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                 GOVERNMENT OF
        THE UNITED STATES OF AMERICA

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  DEPARTMENT OF HEALTH AND HUMAN SERVICES

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      FEDERAL COORDINATING COUNCIL FOR
          COMPARATIVE EFFECTIVENESS

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             LISTENING SESSION

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          WEDNESDAY, JUNE 10, 2009


           The Listening Session convened in
the Capitol Ballroom of the Embassy Suites
Hotel, 900 10th Street, N.W., Washington,
D.C., at 12:00 p.m., Patrick Conway, Chair,
presiding.

COUNCIL MEMBERS PRESENT:

PATRICK CONWAY
CAROLYN CLANCY
GARTH GRAHAM
CECILIA RIVERA CASALE
DAVID HUNT
MIKE MILLMAN
JOEL KUPERSMITH
THOMAS VALUCK
JAMES SCANLON
LYNN HUDSON
MICHAEL KILPATRICK
NEERA TANDEN
EZEKIEL EMANUEL
PETER DELANY
                       2

COMMENTORS PRESENT:

PANEL 1:

DIANE DORMAN
DR. JAMES FASULES
DR. FREDERICK GROVER
DAVID JUBA
JOE KANTER
DR. BETH KOSIAK
LARRY COHEN

PANEL 2:

DR. TED BUCKLEY
DR. JOHN CUDDEBACK
BILL FOX
POLLY PITTMAN
DR. PATRICIA SALBER
DR. DENEEN VOJTA
MARTYN HOWGILL
DR. MARK ROBERTS

PANEL 3:

DR. BRENDAN CARR
DR. MATTHEW HOFFMAN
DR. JEFFREY LERNER
DR. EDUARDO SIGUEL
DR. MARK PILLEY
GRACE-MARIE TURNER
DR. RONALD STEWART
DR. HARRY SELKER

PANEL 4:

ANTON CHAITKIN
DR. ALEX HANNENBERG
DR. JACK GORMAN
DIANA WILLIAMS
DR. DIANA ZUCKERMAN
                                                       3

                TABLE OF CONTENTS

                                               PAGE

I.     Opening Remarks and Introductions .... 4

II.    Panel Comments:

       --   Panel   1   ......................... 16
       --   Panel   2   ......................... 62
       --   Panel   3   ........................ 118
       --   Panel   4   ........................ 164

III.   Closing Remarks .................... 184
                                                                             4


1                     P-R-O-C-E-E-D-I-N-G-S

2                                                         (12:06 p.m.)

3                     DR. CLANCY:             Good afternoon.                 I

4    would     like       to    welcome       you        to     the       third

5    listening session of the Federal Coordinating

6    Council        for          Comparative              Effectiveness

7    Research.

8                     I     want     to   thank          you        for    your

9    participation,            and   thank     you     in      advance       to

10   panelists for sharing their comments.                               We all

11   know how hard it is to distill your comments.

12                    And,      first,    before          making          other

13   remarks I would like the Council members to

14   introduce      themselves.               I     am        Dr.     Carolyn

15   Clancy, and I am Director of the Agency for

16   Health Care Research and Quality.                         And we will

17   start with you, Cecilia.

18                    DR. CASALE:             Yes.        I am Cecilia

19   Rivera Casale, and I work at ARC.                              I am the

20   Deputy    to     Patrick        Conway,      working           as    staff

21   support for the Council.

22                    DR.      HUNT:      Hello.              My     name    is

23   David Hunt.          I am a surgeon and Chief Medical

24   Officer     at       the        Office       of     the         National
                                                               5


1    Coordinator.

2                  DR. MILLMAN:          I am Mike Millman

3    from    the    Health      Resources      and       Services

4    Administration.       And I am the alternate for

5    Debra Parham Hopson.

6                  DR. VALUCK:      I am Tom Valuck.            I

7    am a Medical Officer and Senior Advisor at the

8    Centers for Medicare and Medicaid Services.

9                  DR. GRAHAM:      Garth Graham.           I am

10   an     internist     and    the      Deputy        Assistant

11   Secretary for Minority Health at HHS.

12                 DR.    CONWAY:          Patrick        Conway,

13   Executive Director of the Council, and Chief

14   Medical Officer in ASPE.

15                 DR. DELANY:         Pete Delany.         I am

16   with   the    Substance    Abuse    and   Mental      Health

17   Services Administration, and I am an Assistant

18   Surgeon General.

19                 DR. HUDSON:         Lynn Hudson.         I am

20   the Director of the Office of Science Policy

21   Analysis at the National Institutes of Health.

22                 DR.    KILPATRICK:          I   am     Michael

23   Kilpatrick,     an   infectious      disease       physician

24   with    the    Department      of     Defense,        Health
                                                                             6


1    Affairs.

2                     DR. KUPERSMITH:                Joel Kupersmith.

3    I   am     the    Chief           Research      and       Development

4    Officer at the Department of Veterans Affairs.

5                     MR. SCANLON:                Good afternoon.             I

6    am Jim Scanlon, Acting Assistant Secretary for

7    Planning and Evaluation at HHS.

8                     DR. CLANCY:            Thank you.

9                     The          American              Recovery           and

10   Reinvestment Act, which we all know as ARRA,

11   authorized        a     total          of    $1.1        billion      for

12   comparative effectiveness research.                            That is

13   $300 million for the Agency for Health Care

14   Research    and        Quality,         $400    million        for     the

15   National     Institutes                of    Health,          and    $400

16   million     that           will        be    allocated         at     the

17   discretion       of     the       Secretary         of    Health      and

18   Human Services, Secretary Sebelius.

19                    The law also created this Federal

20   Coordinating               Council            for         Comparative

21   Effectiveness          Research,            which        is    a    large

22   mouthful,             to          coordinate              comparative

23   effectiveness          research             across       the       Federal

24   Government.            And,       in    addition,        the       Council
                                                                7


1    will specifically make recommendations for the

2    $400 million allocated to the Office of the

3    Secretary.

4                  Comparative                   effectiveness

5    research, which yesterday I heard Peter Orszag

6    refer to as patient-centered health research,

7    and   I   heard      Senator    Baucus      refer    to    as

8    patient-centered outcomes research -- so we

9    might even have a little straw poll here to --

10   you can express your preferences -- compares

11   treatments and strategies to improve health

12   outcomes.

13                 This information is essential for

14   clinicians,        patients,    and     other       decision

15   makers to decide on the best treatment and

16   achieve better outcomes.          Most importantly for

17   today, we want to gather input from a broad

18   array of diverse stakeholders about how this

19   Council     should     approach       its   mission       and

20   important     considerations          for     comparative

21   effectiveness research.

22                 As    opposed     to    previous      sessions

23   when we said, "Here it is.            It is comparative

24   effectiveness       research.        Tell   us   what     you
                                                                               8


1    think,"      this    time       we    have        actually         posted

2    draft documents from the Council online, and

3    we    will   present       those          documents      in        just    a

4    moment.

5                   These       include         a    draft    definition

6    criteria for our making recommendations to the

7    Secretary, and a strategic framework.                               So in

8    addition     to     any    general          comments         panelists

9    were prepared to make, we would ask you to

10   consider commenting on these draft documents.

11                  President                  Obama          and          the

12   administration            are        committed           to         open,

13   transparent processes in government.                               And in

14   that spirit, we would ask anyone who is a

15   registered     lobbyist          to       identify       himself          or

16   herself before making comments.

17                  So    without          further         ado,     I    would

18   like    to    turn        it    over        to     the       Executive

19   Director, Dr. Patrick Conway, who will give

20   you    a     short     background               and     review        the

21   Council's      draft           definition             criteria        and

22   framework.

23                  DR. CONWAY:                Hi.      Welcome to the

24   listening      session.              So    for     today,      as     was
                                                                 9


1    mentioned, we will have a short background on

2    the Council and CER, we will go through two

3    panels with eight panelists each, have a short

4    break, then have a third panel.                The fourth

5    panel is an open public comment session.                   You

6    can sign up at the registration desk, and then

7    we     randomly    select   people     for    the    fourth

8    panel.

9                  In    terms   of   objectives         for    the

10   listening session, this is an opportunity for

11   the Council to hear from a diverse set of

12   stakeholders; to, number two, obtain public

13   input on the draft definition, criteria, and

14   strategic framework that was mentioned; and,

15   third, to listen to ideas on how this research

16   can empower patients and providers and improve

17   care for all Americans.

18                 Each panelist will have a strict

19   three-minute       time     limit    to      give    public

20   comment.      We have a timer that I will be

21   using, as well as Pamela in the corner will

22   signal you when there is one minute, and when

23   it is time to stop.          Please, you can finish

24   your     sentence,    but     please      make      that    a
                                                                 10


1    relatively         short,        non-extended         run-on

2    sentence.

3                  And    try    to    adhere   to     the      time

4    limit.      The idea really being is it leaves

5    plenty of time for us to ask questions and for

6    it to be a discussion.           So thank you for that.

7    You can submit longer comments online via the

8    website as needed.

9                  And     after        the     panel        gives

10   comments, the Council members will have the

11   opportunity to ask questions.

12                 In     terms        of     the      reporting

13   requirements, by June 30th the Council will

14   submit to the President and Congress a report

15   containing information describing the current

16   activities    and,    importantly,       recommendations

17   for      comparative         effectiveness            funding

18   allocated to the Secretary.

19                 The    Secretary,        jointly    with      the

20   directors of ARC and NIH, shall provide to

21   Congress an operating plan for these funds not

22   later than July 30, 2009.

23                 The    draft       definition      --   it    is

24   slightly lengthy, but it was to capture all of
                                                                       11


1    the    important      components.               So    comparative

2    effectiveness        research       is     the       conduct      and

3    synthesis      of    systematic          research          comparing

4    different      interventions         and        strategies         to

5    prevent, diagnose, treat, and monitor health

6    conditions.         The purpose of this research is

7    to inform patients, providers, and decision

8    makers,      responding       to    their        express       needs

9    about which interventions are most effective

10   for       which        patients            under            specific

11   circumstances.         [See        the     Hastings           Center

12   Statement.]

13                  To     provide            this        information,

14   comparative effectiveness research must assess

15   a     comprehensive       array          of      health-related

16   outcomes      for     diverse       patient          populations.

17   Defined      interventions         compared          may    include

18   medications, procedures, medical and assistive

19   devices      and    technologies,         behavioral         change

20   strategies, and delivery system interventions.

21                  This     research          necessitates            the

22   development, expansion, and use of a variety

23   of    data     sources     add       methods          to      assess

24   comparative         effectiveness.               So        that    is
                                                                12


1    currently the draft definition, building on

2    previous definitions by other groups that the

3    Federal Coordinating Council is using.                  But as

4    was said, the feedback today will help inform

5    changes to that definition as needed.

6                   In terms of criteria, the Council

7    broke   the    criteria        into    threshold    criteria

8    that    must   be    met,      and    then   prioritization

9    criteria,      the    threshold        criteria     included

10   within the statutory limits of the Recovery

11   Act and the FCC definition of CER.                      Number

12   two,    responsiveness          to    express     needs    and

13   preferences of patients, clinicians, and other

14   stakeholders,        including       community    engagement

15   in research.         And, number three, feasibility

16   of research topic.

17                  As      was       mentioned,        we      are

18   collecting comments online.                 One of the ones

19   we have received from multiple sources is on

20   the    community     engagement        in    research,    what

21   that    means,      and   is    that    a    prioritization

22   criteria as opposed to threshold criteria.                  So

23   that is the kind of feedback that will be

24   helpful for us today.
                                                                     13


1                  In         terms         of         prioritization

2    criteria,    we        had    five.         One    is    potential

3    impact.       Two,       the     potential         to     evaluate

4    comparative            effectiveness              in       diverse

5    populations        and          patient       subpopulations.

6    Number three, uncertainty within the clinical

7    and     public         health     communities            regarding

8    management decisions.                Number four, addresses

9    a need or gap unlikely to be addressed through

10   other funding mechanisms.                   And, number five,

11   potential for multiplicative effect, such as

12   the Foundation for Future CER or generating

13   additional investment outside of government.

14                 We        have     a     --     this       schematic

15   represents         a         framework       in         terms   of

16   categorizing the activity.                  One can see this

17   as both it helps us categorize.                    We are doing

18   an inventory of our current federal activity

19   now,    so   it        helps     us     categorize         current

20   activity.     Also, once you categorize activity,

21   you can think about, where are the gaps?                        And

22   specifically,          given     the      Council's        charge,

23   where can Office of Secretary funds fill those

24   gaps?
                                                                14


1                  To try to orient you, there are

2    four   major      activities       that      the       Council

3    distilled.     So one being research, two being

4    human and scientific capital for CER, three

5    being CER data infrastructure, and, four, the

6    dissemination and translation and adoption of

7    CER.

8                  We also felt it was important to

9    identify   that    there     could    be     cross-cutting

10   priority themes where you might want to make

11   investments    that    cut    across        the    different

12   activities,    such    as,    to     give    an    example,

13   priority populations.

14                 Racial   and    ethnic       minorities     was

15   identified as a priority population, and you

16   might consider an investment that deals with

17   research, human and scientific capital, data

18   infrastructure, and translation and adoptions

19   for that priority population, as an example of

20   a cross-cutting priority theme.

21                 There is also conditions or types

22   of interventions.       So as was said, specific

23   investments    could   be     made    within       a   single

24   category or a cross-cutting priority theme.
                                                                   15


1                 I am not going to go through this

2    in detail, but it is the more detailed version

3    of the framework.         I think a couple of key

4    points.     One is the foundational block for

5    each of the activities is a current inventory

6    and an ongoing evaluation of where we stand in

7    terms of research, translation and adoption,

8    data infrastructure, etcetera, with the idea

9    being     that   we    have     an        inventory     and     a

10   recommendation        process        as     part   of        this

11   Council.

12                But      going     forward       it      will     be

13   important for this process and the priority-

14   setting to be iterative, and to be based on

15   the accomplishments to date and where the gaps

16   are still present.

17                And that is the summary.                   So we

18   will move to the first panel, and we will

19   generally start from the end and then move

20   down.   So, Ms. Dorman, if you would begin.

21                MS. DORMAN:         Thank you.            I am a

22   registered lobbyist with National Organization

23   for Rare Disorders, and I have been so for the

24   past nine years.       So I will preface my remarks
                                                                16


1    by that.

2                     Good afternoon, and thank you for

3    giving     the    National      Organization       for   Rare

4    Disorders     the      opportunity      to   address     this

5    Council     regarding      comparative       effectiveness

6    research.        NORD represents the estimated 30

7    million men, women, and children in the United

8    States affected by one of the nearly 7,000

9    known rare diseases.

10                    For those who may not know what a

11   rare disease is, it is a disease, syndrome, or

12   condition affecting fewer than 200,000 people

13   in the United States, or approximately one in

14   10 people.        For many, it can take many years

15   to be diagnosed.           Some estimate as many as

16   seven     years;       others     are    never      properly

17   diagnosed at all.

18                    I would like to preface my remarks

19   by   saying        that    NORD      strongly       supports

20   comparative effectiveness research for drugs,

21   biologics,       and   medical    devices,    as    well   as

22   treatment protocols.            If this country is to

23   address the growing disparities in care, we

24   must find a way to ensure that every American
                                                                    17


1    receives      the        care    they     need   and      rightly

2    deserve.

3                    By   way        of    background,   there      are

4    currently 339 orphan drugs and biologics that

5    treat, according to the FDA, about 12 million

6    people     in       the     United       States.          It   is

7    unfortunate the remaining 18 million people

8    have     no     therapy          or     treatment      protocol

9    addressing their specific disease.                     It is a

10   bit of a hit-and-miss proposition.

11                   As a consequence, most are treated

12   off label, because there is nothing specific

13   to their disease.               As a consequence, many of

14   these people have difficulty getting access to

15   the     treatments          they       need,     because       the

16   indication is not on the label or the product.

17                   Comparative effectiveness research

18   could    have        a     profound       impact     on    these

19   patients, should labeling changes be required.

20   Already insurers continue to deny access to

21   care simply because a disease state is not

22   specified on any labeling.

23                   As you deliberate, we do have a

24   number of general suggestions.                     We ask that
                                                                            18


1    you     consider      the      comparative            effectiveness

2    research typically compares average results of

3    one     therapy      or      treatment          protocol         versus

4    another for a studied population.                           However,

5    these    do    not    take     into       account        differences

6    between       patients       due     to     genetics,           and    80

7    percent       of      rare      diseases            are     genetic,

8    comorbidities, and other important factors.

9                    Comparative effectiveness research

10   should    focus      on   questions            that      reflect       the

11   interactions          among        all         of     the       various

12   components of the health care system, and have

13   the     greatest      potential           to    empower         medical

14   specialists        and    patients         to       make    the       most

15   appropriate        decisions         when      faced       with       real

16   world clinical situations.

17                   There          are          specific             issues

18   surrounding rare diseases and orphan products

19   that    we    think      are   addressed            in    the    newly-

20   introduced Patient-Centered Outcomes Research

21   Act of 2009 that was introduced by Senators

22   Baucus and Conrad yesterday.

23                   Specifically, the legislation says

24   that in the case of comparative effectiveness
                                                          19


1    research studies for rare diseases -- for the

2    study of rare diseases that an expert advisory

3    panel assist on the design of such research

4    studies and determine the relative value and

5    feasibility     of      conducting    such     research

6    studies.

7                  The      draft    language      we    have

8    proposed to the U.S. House of Representatives

9    goes a step further and asks that an ombudsman

10   be appointed to serve as the single point of

11   contact    to       patients   with    rare    diseases

12   regarding funding by the Department of Health

13   and Human Services.

14                 NORD      strongly      supports      this

15   language, and we ask that you remain mindful

16   of those who are considered as outliers, and

17   as you continue deliberations you continue to

18   be mindful of the unique needs of rare disease

19   patients and the challenges they face.

20                 DR. CONWAY:      Thank you very much.

21                 Dr. Fasules?

22                 DR. FASULES:     Fasules.     Okay.

23                 DR. CONWAY:      All right.

24                 DR. FASULES:       Good afternoon.      I
                                                                  20


1    am Jim Fasules, a pediatric cardiologist and

2    Senior Vice President for Advocacy with the

3    American College of Cardiology.                 Thank you for

4    the opportunity to share the ACC's views on

5    priorities      for        comparative          effectiveness

6    research and how the college can help with

7    this critical effort.

8                 The       ACC         strongly            supports

9    investment      in     comparative              effectiveness

10   research.       We    believe       that    well-conducted

11   research is useful in helping physicians and

12   other      providers        deliver        high        quality,

13   effective care.        For over 20 years, the ACC

14   has created and published clinical guidelines

15   for the care of patients with cardiovascular

16   disease.     Interestingly, our first guideline

17   was a study on pacemaker effectiveness.

18                Given         the     reality        of      finite

19   financial resources, cost effectiveness has a

20   legitimate     role.             However,       the      college

21   strongly believes that evidence-based medicine

22   must    be   strictly        focused       on     comparative

23   clinical science.           Decisions regarding cost

24   effectiveness        can     be      made        later     when
                                                                     21


1    scientific merit has been established.

2                     Maintaining         a     distinct     firewall

3    between        clinical       and    cost     comparisons       is

4    essential        to     ensure       that    physicians        and

5    patients trust the ethics and integrity of the

6    work.     Comparative effectiveness research on

7    diagnostic imaging is a high priority for the

8    ACC.     Understanding the effectiveness of the

9    various cardiac imaging modalities could help

10   direct better use of these beneficial tools

11   and help inform sound policy decisions related

12   to     their     use.         Clarifying       this     role    is

13   obviously important for accurate diagnosis so

14   important for clinical outcomes.

15                    The    use     of       clinical     data     over

16   administrative data is also key to success.

17   When used to their full capacity, in-patient

18   and outpatient clinical registries can track

19   key     elements       of   comparative        effectiveness,

20   including        laboratory          results,         medication

21   adherence, and diagnostic decisions.

22                    Emphasis should also be placed on

23   longitudinal studies.                One example would be

24   linking the existing data registries, like the
                                                                      22


1    ACC's National Cardiac Data Registry, or NCDR,

2    on percutaneous coronary intervention with the

3    Society for Thoracic Surgery's Coronary Bypass

4    Surgery     Registry.              This         would     provide

5    longitudinal       data     on    the   best      choices       for

6    coronary revascularization.

7                     The ACC applauds the Council for

8    including the evaluation of effectiveness in

9    diverse populations and subpopulations in its

10   draft criteria to prioritize research.                        Robust

11   clinical    registries,          such   as      the     NCDR,   are

12   great tools that support and enable research

13   on diverse populations who are too often not

14   included in random clinical trials.

15                    Conducting       comparative           effective

16   research           will           require             substantial

17   infrastructure            investments.                  The     ACC

18   recommends creating, interlinking, and using

19   robust    national       registries        be    given     a    high

20   priority.

21                    Comparative effectiveness research

22   should,     by     its     very     nature,       improve        the

23   quality    of     care    and     ensure     greater      patient

24   value.      We     urge     the    Council        to    take    the
                                                                 23


1    crucial   next     step     to    make    sure   comparative

2    effectiveness research fulfills its potential

3    to improve care.            This involves integrating

4    research findings into guidelines and decision

5    support tools for clinical use.

6                  The ACC would be pleased to engage

7    with the Council and offer the lessons we have

8    learned through our experience in guidelines

9    development        to     support        dissemination      of

10   national comparative effectiveness results.

11                 Thank you for the opportunity to

12   speak with you this afternoon.                   The college

13   looks forward to continuing to work with you

14   on this endeavor.

15                 DR. CONWAY:          Thank you very much.

16                 Dr. Grover?

17                 DR. GROVER:          Thank you.      I am Fred

18   Grover, the past President and current Chair

19   of the Council on Quality Research and Patient

20   Safety for the Society of Thoracic Surgeons.

21                 On        behalf     of    the     Society    of

22   Thoracic Surgeons, I would like to thank you

23   for the opportunity to submit testimony today.

24   We   agree    with       the     criteria   that   you     have
                                                              24


1    established         for     comparative      effectiveness

2    research      and     believe     that      the   following

3    proposals strongly comply with these criteria.

4                  We would like to propose specific

5    examples of comparative effectiveness research

6    in our field.         The STS national database is

7    the    gold    standard       clinical       registry    for

8    cardiothoracic surgery in this country.                  We

9    have     participation        from   greater      than    90

10   percent of all cardiac surgery centers in the

11   country, and there are more than 3.8 million

12   records in the database, which began 20 years

13   ago.

14                 The American College of Cardiology

15   also has mature clinical databases.               Combined,

16   the STS and ACC databases cover virtually the

17   entire spectrum of cardiovascular care in this

18   country.      Both the STS and the ACC recognize

19   the importance of longitudinal followup.

20                 Linking our two clinical databases

21   with CMS administrative data can provide long-

22   term   followup       out    to   several    years.      The

23   linked     data      set      will   contain       clinical

24   characteristics, as well as long-term outcomes
                                                                  25


1    and cost data.          This data set gives us an

2    unprecedented         opportunity       to     exhaustively

3    examine       the    comparative        effectiveness        of

4    surgical versus medical treatment strategies

5    to    treat   coronary    artery    disease,         i.e.   PCI

6    with stents versus coronary bypass.

7                   This will allow us for the first

8    time to examine very large numbers of real

9    world patients rather than the small numbers

10   and    very    select    populations         used    in     most

11   randomized clinical trials.

12                  This      type      of        study        merits

13   consideration for several reasons.                 It will be

14   the largest and most comprehensive study ever

15   performed in this field.           It has the potential

16   to optimize treatment, thereby minimizing both

17   over-use and under-use, and establishing the

18   most appropriate therapy for various subsets

19   of patients.

20                  It,    therefore,    has      the     potential

21   to improve care and reduce the costs of one of

22   the most commonly encountered conditions in

23   medicine today.         Since both the STS and the

24   ACC     databases        are    mature,            the      data
                                                             26


1    infrastructure       is     already      in     place   and

2    operational and has been for years.               It will,

3    therefore,   be     possible    to    obtain     important

4    long-term results very quickly.

5                 The     approach      can     be   completely

6    generalized, so this study should serve as a

7    roadmap for investigators in many other fields

8    of medicine.       In addition, we ask the Council

9    to strongly consider the following research

10   studies:     followup to assess the impact of

11   long-term    compliance       with       NQF    performance

12   measures, a comparative longitudinal followup

13   of patients undergoing various forms of lung

14   cancer surgery; and an STS/ACC/CMS data set

15   study   to     compare       the      effectiveness      of

16   percutaneous       versus    surgical      treatment     of

17   atrial fibrillation.

18                Once again, we applaud the Council

19   for convening this meeting, and we sincerely

20   thank you for this opportunity to provide this

21   testimony.

22                Thank you.

23                DR. CONWAY:       Thank you very much.

24                MR. Juba?
                                                                      27


1                   MR.        JUBA:            Thanks.             Good

2    afternoon.      My name is David Juba.                       I am a

3    Senior       Research       Analyst         at        Fundamental

4    Clinical Consulting.              However, I am actually

5    here today representing the American Health

6    Care     Association,           AHCA,     and       its    National

7    Center for Assisted Living.

8                   And as the other panelists did, I

9    want to begin by thanking each of you for

10   making available this opportunity for comment.

11                  The AHCA is the nation's largest

12   association of post-acute and long-term care

13   providers.          It   represents        more      than    10,000

14   nonprofit and for-profit providers nationwide

15   who care for more than a million and a half

16   frail, elderly, and disabled individuals every

17   day.     And I am personally pleased to support

18   AHCA as the current member and past chairman

19   of     its      Health           Information              Technology

20   Committee.

21                  In        fact,          health        information

22   technology is proliferating rapidly throughout

23   the    post-acute        care    sector,        a   sector    whose

24   2007   revenues      exceeded       $131        billion.       This
                                                                            28


1    places us third behind acute care hospitals

2    and    physicians        in      terms       of     total     national

3    health     expenditures.                As     you      might      know,

4    Medicare and Medicaid fund a vast majority of

5    the care we provide.

6                     Now,        I    make       these       points         to

7    impress upon you the importance of post-acute

8    and long-term-care sector, a sector that is

9    really      fertile              ground        for       comparative

10   effectiveness       research.                It      allows      me    to

11   impress upon you the fact that the clinical

12   profile     of    the        residents         we    care        for   in

13   skilled nursing facilities today is greatly

14   different from only a few years ago.

15                    Increasing              numbers            of         our

16   residents        require         short-term          rehabilitation

17   care.     Many of them are high acuity patients

18   with     multiple        diagnoses           and      comorbidities

19   requiring multiple medications.

20                    Given       the        push      for     integrated

21   care, coordination across sites, and payment

22   bundling,    assessing            the     appropriateness              and

23   effectiveness           of       care     across        and       within

24   settings     will        be       an     important          area       for
                                                                 29


1    comparative effectiveness research.                    In fact,

2    the post-acute and long-term care sector is

3    making its own initial efforts in this regard.

4                      Now, many of you hearing me and

5    listening might know that the long-term care

6    sector already has a form of electronic health

7    record.       It is called the minimum data set, or

8    MDS.       Now, if you could combine MDS data with

9    data       from   pharmacy      and   lab   providers,      you

10   would actually have a powerful foundation for

11   comparative effectiveness studies.

12                     And,    in     fact,   an     AHCA     member

13   company, Sava Senior Care based in Atlanta,

14   Georgia,      is    doing      just   that,     with    results

15   leading to relatively important improvements

16   in their clinical outcomes.

17                     Long-term care and post-acute care

18   has    a    history      of    sharing   data    and    working

19   cooperatively with the research community on

20   issues      ranging      from    quality    assessment      and

21   improvement to appropriate staffing.                   We would

22   be pleased to cooperate in a similar way with

23   comparative effectiveness researchers.

24                     Two trends loom on the horizon as
                                                                  30


1    we see it.         One is the aging of the baby boom

2    generation.          The other is the evolution of

3    alternative care settings.             Patients today are

4    touched by multiple providers as they move it

5    along    an    evolving       and   dynamic    continuum      of

6    care.

7                   Consequently, the challenge facing

8    us is to develop a better understanding of

9    what works for individuals 85 and over.                    These

10   are     the    fastest-growing             segment    of    our

11   population, and we must be ready for them.

12                  Thank you.

13                  DR. CONWAY:          Thank you very much.

14                  Next, Mr. Kanter?

15                  MR. KANTER:           Thank you very much

16   for your invitation, ladies and gentlemen, the

17   opportunity to share my story and my vision

18   for better health and health care.

19                  My name is Joe Kanter.                 I am a

20   World    War    II    veteran,       an     entrepreneur,      a

21   cancer    survivor,       a     philanthropist,       and     an

22   advocate for better health information.

23                  I     founded         the     Kanter    Family

24   Foundation in 1988 after my personal battle
                                                                 31


1    with prostate cancer.            As a patient, I faced

2    many choices for my treatment, from surgery to

3    doing   nothing.      I     had    access    to     the    best

4    medical resources available, but I was still

5    unable to accurately determine what would work

6    best for me.

7                 It was then I decided to dedicate

8    my time and money to improve health care for

9    all Americans.       I have worked over the past

10   decade to make my vision of a national health

11   outcomes database a reality.

12                The    idea      is    simple:           a    man

13   diagnosed with prostate cancer, like me, or a

14   woman with a chronic condition like diabetes,

15   or a physician unsure about what call, the

16   right call, could plug into the user-friendly

17   tool and pull up information on how different

18   treatments worked in similar patients, like

19   me.

20                In    short,    the    two     would    provide

21   health care professionals and patients useful,

22   scientific   evidence       on    what    works     best   for

23   whom.

24                I am pleased to see that after 10
                                                                        32


1    years       of     the           Kanter     Family       Foundation

2    promoting these ideas, the administration and

3    Congress are now using the same language and

4    investing in HIT and comparative effectiveness

5    and outcomes research.

6                     These initiatives are necessary as

7    a first step in our nation's long-range goal

8    to    harness      a    real-time          data       from    personal

9    electronic health records and provide health

10   care     providers          and     average       Americans       with

11   easily           accessible               and         understandable

12   scientific data to make evidence-based health

13   care decisions.

14                    It must be understood that experts

15   believe that we are 10 to 12 years behind the

16   use    of   personal             electronic      medical       records

17   with the rest of the world.                       As the Council

18   considers        how        to     invest       the    appropriated

19   funds, I hope that you will support a complete

20   inventory              of           existing            comparative

21   effectiveness research studies, both in the

22   United States and globally.

23                    Why reinvent the wheel?                     I believe

24   we have a major obligation to make certain
                                                                         33


1    that the expenditure of funds is supported by

2    usable and meaningful results.                     So we need to

3    evaluate the methodologies and to use to our

4    advantage        success        and        failures           in    any

5    comparative effectiveness research in the U.S.

6    and globally.

7                    Patients         want        and        deserve      a

8    greater voice in their health care.                            But in

9    order    to     exercise     judgment,           they    must      have

10   access     to    reliable,        scientific            information

11   about how treatments are performed compared to

12   one     another.       We       have       evidence       that      the

13   national health outcomes database, utilizing

14   electronic medical records, will reduce the

15   costs and embrace the quality of comparative

16   effectiveness research.

17                   For    several         months,          the    Kanter

18   Family Foundation has been conducting research

19   and      organizing         a     global          health-sharing

20   network,        in     order          to     determine             what

21   comparative effectiveness exists in the United

22   States and globally.

23                   We    also      have       the    first       printed

24   document in a medical journal that has just
                                                                   34


1    come   out    that     lists    all     of     the    personal

2    electronic medical record uses, and they have

3    126 evaluation studies that they consider.

4                  DR. CONWAY:        Thank you, sir.             Sir?

5    Mr.    Kanter?         Thank    you     very       much,     sir.

6    Appreciate it.

7                  Dr. Kosiak?

8                  DR.    KOSIAK:           Thank       you.      The

9    American      Urological             Association,          which

10   represents     16,000     urologists         worldwide       and

11   almost 10,000 in the U.S., greatly appreciates

12   the opportunity to testify here today.

13                 The      public        health        burden     of

14   urologic     disease    in     the    U.S.    is     large   and

15   growing, with an estimated annual impact of

16   over $11 billion.            The AUA nominates three

17   areas that warrant future investigation in a

18   CER framework.

19                 First, prostate cancer treatment.

20   In 2008, an estimated 200,000 men were newly

21   diagnosed with prostate cancer in the U.S.,

22   and about 29,000 men died from the disease.

23   Significant prostate cancer disparities exist

24   between rural and urban populations and across
                                                                       35


1    racial and ethnic groups.

2                     The range of treatment options can

3    vary    significantly,           and       patient     preference

4    regarding treatment side effects and quality

5    of life plays a particularly prominent role in

6    prostate cancer treatment choice.

7                     In addition, prostate cancer is a

8    focus of the Medicare program.                       It has been

9    identified        as      one         of     eight        high-cost

10   conditions selected for the physician resource

11   utilization report pilot program.                    Yet despite

12   its prevalence, cost, and complexity, there is

13   a    distinct     lack     of    evidence          comparing      the

14   treatment        options        for        localized       prostate

15   cancer.

16                    The   second         area    we     nominate     is

17   comparison       of    imaging        modalities       for   major

18   urologic    conditions.           A        number    of   analyses

19   conducted from MedPAC have established that at

20   least     some    portion       of     the     rapid      rate    of

21   increase in physician-ordered imaging services

22   is      attributable             to          duplicative          or

23   inappropriate imaging.

24                    The     AUA     is        embarking       upon    a
                                                                   36


1    comparative       effectiveness           study    of    imaging

2    modalities       for    ureteral       stones,         with   the

3    intent of producing evidence-based guidance on

4    the most effective and efficient imaging for

5    this condition to disseminate to urologists,

6    primary care practitioners, and others who may

7    treat this condition.

8                    Such guidance across a wide range

9    of conditions could go a long way to help the

10   medical profession order only those imaging

11   studies        that     are        most      effective        and

12   appropriate       for   the    condition          in    question.

13   And,     lastly,        we    ask      for        support     for

14   development and maintenance over time for the

15   quality infrastructure through creation of a

16   public/private partnership under the auspices

17   of AHRQ or NIH, both of which are positioned

18   to accept private funds.

19                   This venue could be used to pool

20   the    resources       of    those    public       and    private

21   stakeholders, including government, business,

22   private        insurers,      research       entities,        and

23   medical specialty societies, all of whom have

24   a     vested    interest      in     quality      measurement,
                                                                37


1    improvement, and evidence-based medicine.

2                 Thank you.

3                 DR. CONWAY:          Thank you.

4                 So we are now going to move to the

5    phone.    I believe we have Mr. Larry Cohen on

6    the phone.

7                 MR. COHEN:       Good morning.          Can you

8    hear me okay?      Hello?

9                 DR.   CONWAY:          Yes.      We   can    hear

10   you, sir.

11                MR. COHEN:           Oh, great.         Can you

12   hear me now?       Good morning.            I am actually

13   calling from L.A., so I am the first one to

14   say good morning.        I am Executive Director of

15   Prevention     Institute.          We   are    a     national

16   nonprofit    based     in    Oakland,       moving    beyond

17   approaches that target individuals one person

18   at a time to quality prevention, which means

19   creating systematic, comprehensive strategies

20   that     change    the      conditions        that     impact

21   community    health.         So    we   are    focused     on

22   improving    places      where     people     play,      work,

23   study, live.

24                Prevention Institute is dedicated
                                                                       38


1    to     translating          research        into       effective

2    community      practice        and     to        ensuring     that

3    effective      community           practice        shapes     our

4    research agenda.            And it is important to note

5    that     effective     prevention         can      often     solve

6    multiple medical problems at one time, which

7    is why we are asking for an investment in

8    primary prevention solutions.

9                   Let me, in my time, give you six

10   reasons.      Most importantly, primary prevention

11   works.     It saves lives, it reduces illness and

12   injury, and it reduces misery.                    We have seen

13   multiple examples from minimum drinking age

14   laws     to    lead     poisoning           to     anti-smoking

15   legislation.        I worked on the nation's first

16   multi-city     no     smoking      laws.         We   have    seen

17   thousands     fewer     deaths       in     California       as    a

18   result of primary prevention.

19                  Secondly,        primary          prevention       is

20   cost effective, and there are potential for

21   major savings.         A recent study with conducted

22   with Trust for America's Health showed that an

23   investment     of     $10    per    person       in   prevention

24   would save the first year, and then the second
                                                                          39


1    year, $2.8 billion annually, and by the fifth

2    year     that    same        investment             would    save    $16

3    billion.

4                     So     at    a        time     when    we    have    a

5    financial crisis, here is an opportunity to

6    actually reduce costs.

7                     Also, the third reason is because

8    our health care system is crumbling.                          Clearly,

9    we need to do better for the people who need

10   care,    and     we     can       do    so     by     simultaneously

11   reducing the number of people who become ill

12   or injured in the first place, and reducing

13   the impact of potential illness or injury.                           So

14   primary prevention then lessens the burden on

15   the health care system.

16                    Currently,            we     are    seeing    primary

17   prevention       finally       as       an     important      part   of

18   what     is     being    considered             for     health      care

19   reform, an important part of the stimulus, and

20   recent    polls       just    this          past     week    confirmed

21   that an investment in primary prevention is

22   strongly supported by most Americans as the

23   most important part of the solution.

24                    While       our       country's        expenditures
                                                                              40


1    are     focused        on        medical             treatments          and

2    services, health is not primarily derived from

3    health care.             Many studies have shown that

4    there       are       four         determinants                  --      the

5    environment,         behavior,          heredity,           of    course,

6    and health care.               And environment and behavior

7    together       are        60     to        70     percent         of    the

8    determinants         of     health,         and       yet   we        rarely

9    focus on them.

10                  Very        important            in    terms      of     your

11   emphasis      on     equity,          primary         prevention        can

12   play    a     vital       role        in        eliminating           health

13   disparities          by        changing              the    underlying

14   conditions        that     led    to       these       inequities        in

15   communities in the first place.

16                  And, finally, I should note that

17   prevention         complements              medical         care        and

18   treatment.           The       same        things      that       prevent

19   illness and injury will hasten recovery.                                For

20   example, if we need to buy and eat healthy

21   food    and    need       places       to       exercise         to    avoid

22   diabetes,      this        availability               is    even       more

23   critical for those with diabetes to maintain

24   their health.             Health is too important for
                                                                  41


1    every one of us to only think about it in the

2    clinical setting.

3                 I have given you, I hope, the why

4    of   focusing     on    primary    prevention.           I   am

5    happy, of course, to discuss in more detail

6    the what and the how.            As Einstein said, "No

7    problem can be solved from the same level of

8    consciousness that created it."                 We need to

9    look at other methodology.

10                Thank you very, very much.

11                DR. CONWAY:         Thank you very much.

12                Is    Dr.     Diana       Zuckerman    on       the

13   phone?

14                (No response.)

15                Okay.       So we are going to open it

16   up to questions from the Council.

17                DR.       GRAHAM:     I    think   I   will     go

18   ahead start.       Dr. Grover and Dr. Fasules, you

19   started off by talking about the importance of

20   clinical     databases           over      administrative

21   databases.      I would like to hear a little bit

22   about the diversity represented in some of the

23   databases that you talked about.

24                I think you noted that that was
                                                      42


1    one of the very important criteria that we

2    alluded to in terms of being able to look and

3    see across the diversity of populations.       That

4    would be question number one.

5                 My second question is, you talked

6    a little bit about some priority studies that

7    you thought needed to be done, alluding to

8    such things like looking at surgical versus

9    medical management around atrial fibrillation.

10   And I think some of the recent data around

11   the WATCHMAN studies and some of those kinds

12   of things I think are important, especially

13   given the prevalence of atrial fibrillation.

14   So I think that those are some good examples.

15                But could you give me kind of your

16   thought pattern in terms of how some of those

17   other studies kind of rose to the top of your

18   list?

19                DR. FASULES:     Okay.   Let me do --

20   start     with   diversity   and   clinical   versus

21   claims.    For small amounts, the claims data is

22   all over the place.     When you have a huge data

23   set like CMS has, and you can compare that and

24   link that with the clinical data, you have a
                                                                         43


1    very powerful tool.

2                    The        thing        about       registries       as

3    opposed        to     random        trials        is     a   registry

4    captures       everyone         who     is    having     that    done.

5    And when you have a huge registry -- take the

6    ICD   registry,           for     instance        --    it   captures

7    everyone who has received an ICD.                        So you have

8    all     populations,              and      then        you   can     do

9    comparisons               amongst            populations,          does

10   something need to be done differently with a

11   different group.

12                   So the strength is the breadth of

13   the registry, and then when you have enough

14   and putting it with the claims data in a huge

15   claims database, you get some -- you get a

16   double impact.              But small claims data is --

17   doesn't work.

18                   DR. GROVER:                Yes, let me address

19   that, too.          Ninety percent of patients in the

20   United States that have a cardiac operation,

21   excluding           the     VA,       which       has    their     own

22   database, are entered into this database.                          And

23   that includes all races, gender, age, those

24   types     of        things.           So      the      diversity     is
                                                                 44


1    absolutely there.            These are the cases that

2    are done across the board in the U.S., and I

3    think the same is true with the ACC database.

4                   DR.      CLANCY:     But,    Fred,    do    you

5    actually collect data on race and ethnicity

6    when you are entering it--

7                   DR. GROVER:        Yes.

8                   DR. CLANCY:        Okay.    Thanks.

9                   DR. GROVER:         Yes, yes.         And the

10   clinical aspect of it is, what we hope to do

11   with this study is, it is not whether stents

12   are   better       than     coronary     bypass,    or     vice

13   versa.     It's if you get down to the patient

14   level, the individual patient level, which --

15   the individual patient treatment, which is the

16   best treatment?

17                  And to do that you really have to

18   have reliable clinical data.                That includes

19   pre-operative        risk    factors,     details    of    the

20   operation,        and     risk-adjusted     post-operative

21   outcomes.

22                  The key here is that currently we

23   -- to the present we have done one month's

24   outcomes     or     index    hospitalization,        and    by
                                                                       45


1    combining these two robust clinical databases,

2    or     linking     them      with      CMS's     administrative

3    database, we are able to follow patients long

4    term     for     reintervention,          death,       myocardial

5    infarction, that type of thing, out a number

6    of years, as well as look at the cost data.

7                     And         I         think          comparative

8    effectiveness           is   a        balance       between      high

9    quality, the best quality measure you can get,

10   and    assuming        you   are      keeping       that   quality,

11   what is the most cost effective measure you

12   can get.       So I think this combines and offers

13   a    very   rich    group        of    patients      to    evaluate

14   this.

15                    And    I    know      one     of    your     cross-

16   cutting things, too, is, how do you use this

17   to educate the population?                      How do you use

18   this     to      educate         the     surgeons          and    the

19   internists?        And I know I have just been with

20   the ACC group the last couple of days here,

21   and their database group, talking about real-

22   time decision-making elements being built into

23   the databases.

24                    So that this can be put in -- not
                                                                    46


1    only published and educating physicians in the

2    public by publications through the American

3    Heart lay publications, but can be built into

4    real decision time database work, where you

5    have    a    patient     with    certain     criteria,    age,

6    level       of   angina,       what     is   their     coronary

7    anatomy,         what    is     their    left    ventricular

8    function,        all    of    these   things,    and    make    a

9    specific recommendation based on data.

10                    DR. GRAHAM:          One quick follow-on

11   question.        I am sorry, Tom, for monopolizing.

12   I think that AHA has done a great job with

13   the -- kind of the dissemination of a lot of

14   the -- with the guidelines, tools.                      And we

15   have done some projects in terms of trying to

16   extrapolate        those      into    minority   communities

17   and face some challenges in terms of being

18   able        to     adequately         capture     the     data

19   appropriately.

20                    Going back to my first question, I

21   know you answered it in an interesting way.

22   So you would say within your -- within some of

23   the priority databases that you are talking

24   about that you are able to adequately capture
                                                                   47


1    differences    in    terms     of     urban    versus       rural

2    populations,    Hispanics          versus     non-Hispanics,

3    Africans    versus    --      African-Americans          versus

4    other populations.            Are you able to kind of

5    adequately     capture     some       of     the     impact   of

6    various interventions in those populations?

7                  DR. FASULES:            I think the number

8    is   around     --     well         over     three      million

9    percutaneous    interventions          in     the     database,

10   and that database -- everyone who has had an

11   ICD on Medicare had to be -- has to be in the

12   database.     What Dr. Grover is talking about is

13   one of -- is trying to look longitudinally.

14   And I think what we are trying to do is move

15   those databases longitudinally.                And with the

16   CMS claims data, we have a better -- linking

17   it to that we have the longitudinal.

18                 We also have a new database that

19   is   now    piloted      at     600        sites,     improving

20   continuous cardiac care, so IC3.                     And it is

21   actually going to be -- it is a longitudinal

22   database     that     will         capture     all     of     the

23   different      things         of      --      distinct        NQF

24   measurements and use those measurements as you
                                                                       48


1    are putting them in the database to make sure

2    that you have measured the hemoglobin A1C, for

3    instance, you have measured the risk factors.

4    And then, if you didn't, you go back and take

5    care of it.

6                  DR. VALUCK:         So, thank you, Garth.

7    Those were questions that I was going to ask

8    to highlight the opportunity that we have with

9    clinical registries, and particularly Medicare

10   and   Medicaid     data.         But    now    I    can     ask    a

11   different    question      beyond       the       ongoing    work

12   that we have with ACC and STS around those

13   databases.

14                 My   question        to       Dr.    Fasules        is

15   about what could be characterized as your two-

16   step approach to incorporating cost into the

17   effectiveness      discussion          or     considerations.

18   Would you articulate that again, please, and

19   possibly expand a bit on that position?

20                 DR. FASULES:         I think in the long

21   run cost is important.             It is whether -- it

22   is, do you do the science on the disease and

23   the treatment, the two treatments, and compare

24   the   treatments?          And    then,       you    --     as     a
                                                                49


1    society, you put a cost value on that.                    So I

2    don't think the science should be -- the cost

3    value     shouldn't      be    done    while       you     are

4    comparing the science or the treatment.

5                  It    is   easy    when    there       is     no

6    difference and the cost is big.              There is --

7    it is when there is a little difference and

8    the cost is bigger, but not huge, that -- that

9    is going to be a societal judgment.                       That

10   shouldn't necessarily be -- and then, some of

11   that has to be choice of the patient as well.

12                 So if you -- but you have to know,

13   does the one give you anything, improvement

14   over    the   other,     and    how     great      is     that

15   improvement, and then put the cost evaluation

16   in there.     At least that is how we feel, as

17   opposed to -- otherwise, I don't think you are

18   going to be evaluating the effectiveness of

19   the difference in treatment.

20                 DR. VALUCK:       Thank you.          You get

21   at issues of societal values and also patient

22   preferences, which play in.

23                 DR.   FASULES:          Definitely,         Tom,

24   patient    preference     has    to   play     a   role     in
                                                                  50


1    there.      That is important.

2                   DR. GROVER:        Can I follow up with

3    that question just for a second, too?                 I mean,

4    I think if you find a -- number one, I said,

5    you have to keep quality or outcomes the same.

6    But then, if you find, for example, in a

7    given patient, say with very complex coronary

8    anatomy, the -- that particular patient would

9    have a high incidence of reintervention or two

10   or three reinterventions.

11                  That   patient        might    be     one    that

12   ought to go to surgery first, for a coronary

13   bypass first, because that would be -- save

14   the patient procedures and at the same time

15   reduce costs.         And I think that is how you

16   kind   of    weigh    all    of   these      things    in   and

17   balance them.

18                  DR. HUNT:       Hi.     Mr. Kanter, thank

19   you very much for relating your story.                 I just

20   have one question.          In your personal struggle,

21   how long did it take you to make the decision,

22   once   you    had    the    information       that    you   had

23   cancer, how long did it take you to make the

24   decision of what definitive treatment to use?
                                                                         51


1    And what tools did you use to help make that

2    decision?      As imperfect as those tools were,

3    what did you find useful?

4                   MR.    KANTER:              That    is    prostate

5    cancer.       I took close to two years.                      I was

6    fortunate on belonging on the board of a large

7    nonprofit      prostate         cancer      group       that      Dr.

8    Grover was also on.             And I found that they --

9    the board -- mostly had metastasized cancer,

10   and I had a beginning cancer.

11                  And        I     consulted         --    I      first

12   attempted Web MD, and I then talked to my

13   urologist.      I then met the Medical Director of

14   this    nonprofit         cancer       institute,           and     he

15   explained to me that cancers -- and this was

16   15 years ago -- he was on the right track, and

17   he didn't have any evidence.                   So sometimes a

18   doctor's      intuition          is    maybe       better         than

19   anything else.

20                  And he says, "Look, I can't tell

21   you    why.     I    am       going   to    direct      you    to    a

22   hospital that is not my hospital.                           I also

23   can't   tell    you why.              But I know that the

24   patients that I recommended to them generally
                                                                 52


1    had a better recovery."

2                  And it was a new system that was

3    coming out of Sloan Kettering regarding the

4    methodology of radiation.               But he basically

5    felt then, watchful waiting.              And the studies

6    that AHRQ has made, and the studies since then

7    that    the   physicians      have      made,    et     cetera,

8    seems to indicate there are a small number of

9    very rapidly growing prostate cancer.                    Those,

10   if it happens, you need to have major surgery,

11   major chemo, major everything, because they

12   are the ones that create the deaths.

13                 But        generally      speaking,        people

14   with cancer, prostate cancer, die with it, not

15   from it.      And that is a major, major change

16   for the medical system of the United States.

17   And I think it is -- more and more of the

18   physicians         are    recognizing      that        watchful

19   waiting was the answer.

20                 Now, I discussed with doctors, and

21   it     came   to    my    attention      that     it    wasn't

22   invasive, and there is a new type of radiation

23   coming    from     New    York,   the    Sloan    Kettering,

24   that moves the radiation so it doesn't hit all
                                                                          53


1    of the vital organs in one place, because it

2    turns around you.

3                  And I decided to take that, and

4    against my doctor's recommendations.                         He says,

5    "I    will   do    it,       but   my    feeling        is     is    do

6    nothing."         And, of course, at that time we

7    didn't have anybody doing nothing as much as

8    we are having today.

9                  But the radiation was successful,

10   and it took me two years.

11                 MS. TANDEN:            Can I just ask a --

12   and   I   think    we    need      to    do   --     we      need    to

13   shorten this up, because we have time.                            So if

14   you want to --

15                 DR.    KUPERSMITH:              Okay.           I    just

16   have a quick question --

17                 MS. TANDEN:            -- ask a very quick

18   question, and then I will --

19                 DR.        KUPERSMITH:               --        to     the

20   representative from the American College of

21   Cardiology.         Full      disclosure,        I      am    also    a

22   cardiologist.           To    what      extent     have       studies

23   from -- and you have been working with these

24   databases for a long time, I know that.                              To
                                                                        54


1    what extent have they thus far informed your

2    guidelines as compared to randomized control

3    trials?      To what extent have studies, based on

4    these guidelines, on these databases, informed

5    your guidelines?

6                    DR. FASULES:        I can't give you a

7    percent, because I think the first thing we do

8    is, of course, use the randomized trials as

9    the    first    line.     We     are    starting         to   mine

10   several of the databases to look at how they

11   can affect care, and changing the guidelines

12   as that happens.

13                   One thing that I can give you an

14   example of -- it was just presented, though,

15   in abstract form -- is the use of the data

16   online as you are entering it right after the

17   cath to see whether the patient is at high

18   risk    of   bleeding    post      PCI,      and   direct       you

19   which     one    you    should     be     putting        in     the

20   hospital.

21                   Now,    but    I    can't         give    you       a

22   specific number.         We do look at the data, but

23   it is early on.

24                   MS.    TANDEN:         And    I    just       --    I
                                                                     55


1    realize that we don't have very much time, so

2    I would like as concise an answer as possible,

3    because I know we are switching over.                          But,

4    Mr.     Kanter,       you      raised        the       issue     of

5    international         --    what     other      countries       are

6    doing.

7                    And   leaving        aside      what    they    use

8    comparative       effectiveness        research         for,    how

9    would you think -- how do you recommend that

10   we       access            international           comparative

11   effectiveness research information?                    Or how do

12   we better do that?           And I am directing that at

13   Mr. Kanter, since he raised the issue.

14                   MR. KANTER:          You would have to --

15   I couldn't quite get the --

16                   MS. TANDEN:           I'm sorry.          So you

17   raised in your discussion, in your testimony,

18   the idea of accessing or us using as a country

19   data     done      by       comparative          effectiveness

20   research done by other countries.

21                   MR. KANTER:        Yes.

22                   MS. TANDEN:          And leaving aside how

23   those      countries           use        the      comparative

24   effectiveness           research,         obviously         other
                                                                     56


1    countries --

2                  MR. KANTER:         Yes.

3                  MS.     TANDEN:            --   conduct          that

4    research --

5                  MR. KANTER:         Yes.

6                  MS.     TANDEN:         --      do        you    have

7    particular views -- and I appreciate we need

8    to do this in a concise way --

9                  MR. KANTER:         Yes.     We have some --

10                 MS. TANDEN:          -- how we would do

11   that?

12                 MR.     KANTER:        Well,    we        are    also

13   creating a global health-sharing network to

14   share that information.             And we are planning

15   an international conference on this.                      We have

16   a   tremendous      amount   of    data.           We    had    two

17   people doing research for almost three months.

18                 And     at   first     blush    they        end   up

19   saying     they     have     got     electronic           medical

20   records,       they        have       got          comparative

21   effectiveness,       et    cetera,    but     when       you    get

22   down to start to analyze an individual one, I

23   don't think it would pass a peer review group

24   in the United States.
                                                                  57


1                 However,      we    also   found,       despite

2    the fact that we are supposed to be 12 years,

3    10 to 12 years behind, we found in the United

4    States, with some of the major groups like

5    Kaiser   that     have   really    come      up   and      have

6    gotten a good deal of data that they indicated

7    to me they were willing to share, and they

8    want to be part of this program.

9                 But it is very complex, and the

10   data that is put up for review in the journals

11   has to be investigated as to the manner and

12   the methodology they utilize in coming up with

13   their conclusions.

14                MS. TANDEN:        Thank you.        Thank you

15   very much.

16                DR. CONWAY:        Thank you very much.

17                So    we    are    going   to    move      on   to

18   panel number two, please.          So panelists -- the

19   second panel can come on up.              It is Dr. Ted

20   Buckley, Dr. John Cuddeback, Mr. Bill Fox, Mr.

21   Martyn Howgill, Ms. Polly Pittman, Dr. Mark

22   Roberts, Dr. Patricia Salber, and Dr. Deneen

23   Vojta.          Apologize        if     that         was      a

24   mispronunciation.
                                                                  58


1                 All right.           So I think people are

2    settling in.       We will start at the end with

3    Dr. Buckley, please.

4                 DR. BUCKLEY:           Sure.        I would like

5    to state, first of all, that I am a registered

6    lobbyist.          BIO       is    the      largest        trade

7    organization       to    serve      and     represent        the

8    biotechnology industry in the United States

9    and around the globe.             BIO is pleased to have

10   the    opportunity      to    submit      comments      to   the

11   Federal    Coordinating       Council       on    comparative

12   effectiveness research.

13                In order to honor the three-minute

14   time limit, I will read an abbreviated version

15   of the comments that BIO has submitted to the

16   FCC.

17                As a representative of an industry

18   committed    to      discovering         new      cures,     and

19   ensuring patient access to them, BIO strongly

20   supports efforts to increase the availability

21   of    accurate,    scientific       evidence       to    inform

22   clinical decision-making.

23                BIO        believes         that      individual

24   patients    and    their     doctors      should    be     armed
                                                                     59


1    with the best-available information to help

2    assess     the      relative     clinical       benefits        and

3    risks    of      various        treatment       alternatives.

4    However,      BIO    is   concerned          that    comparative

5    effectiveness information may be used strictly

6    as a means to contain cost rather than deliver

7    health     care       value      by     improving        patient

8    outcomes.

9                     BIO believes it is important that

10   this    panel     evaluate      the    lack     of    consistent

11   methodologies        that     are     used    in     comparative

12   effectiveness research.               Doing so will enable

13   CER to provide maximum benefits to patients.

14   Careful consideration should be given as to

15   what methods should be selected.                    In addition,

16   rigorous      standards       must     be     applied    to    the

17   research      method      selected.          These     standards

18   should     consider         both       the      benefits        and

19   challenges        associated          with     the     different

20   methodologies.

21                    Comparative        effectiveness        studies

22   should     capture        all       relevant         aspects     of

23   diseases      and     their      treatments          using     high

24   standards of evidence.                 Government policies
                                                                          60


1    addressing comparative effectiveness need to

2    acknowledge          the        limitations           of      current

3    methodologies and ensure that they do not lead

4    to conclusions and decisions that discourage

5    or       impede           medical         advancements              and

6    breakthroughs that can address unmet medical

7    needs.

8                    It         is         critical         that         all

9    stakeholders be involved and represented in

10   these     efforts.              BIO    believes        that        broad

11   stakeholder       involvement           is    the    best     way    to

12   create     a      neutral         advisory          body,      ensure

13   thoughtful discussion, and generate rigorous

14   and also feasible recommendation.

15                   BIO urges the Federal Coordinating

16   Council    to     advise         the    IOM    to     improve       the

17   quality of its advisory committee by including

18   additional      representatives               from    the     patient

19   minority and innovator groups.                       Including all

20   stakeholders         at    the    table       will    enhance       the

21   committee's discussions and deliberations.

22                   Finally,         given       the     funding       that

23   has     been    made       available         elsewhere        in    the

24   Department        of      Health       and     Human       Services'
                                                                   61


1    budget for health IT, BIO urges the FCC to

2    focus on ways to link the various databases

3    within its operating agencies to each other,

4    as   well   as       to   those    systems     that    will    be

5    utilized       by    providers     under      the    health    IT

6    initiatives.          By electronically coordinating

7    these data, the value of CER will be realized

8    by informing practitioners' clinical decision-

9    making.

10                   Thank you for your time.

11                   DR. CONWAY:        Thank you very much.

12                   Dr. Cuddeback?

13                   DR.       CUDDEBACK:       Thank      you,    and

14   good afternoon.            I am John Cuddeback, CMI of

15   Anceta, the collaborative data warehouse of

16   the American Medical Group Association.                      AMGA

17   provides a forum for multi-specialty medical

18   groups and other organized systems of care to

19   learn from each other.

20                   One-third         of   AMGA     members       are

21   integrated          delivery      systems.          Eighty-five

22   percent have adopted EHRs, and the majority of

23   those are using e-prescribing.                 So there is a

24   wealth    of    detailed       clinical      and    process    of
                                                                   62


1    care data and patient outcome data across the

2    continuum.

3                  Building       on     a     series      of    best

4    practice collaboratives organized by AMGA over

5    the   past    decade,       Anceta        is   creating       an

6    ongoing,      data-driven            collaboration           for

7    improvement, beginning with diabetes and its

8    common comorbidities.              Seven medical groups

9    are currently participating, and we project

10   rapid growth over the coming year.

11                 We     have        promised      the     group's

12   anonymity for the moment, but they represent

13   nearly 3,000 physicians caring for about three

14   million patients.

15                 The        graphic        in     your        packet

16   illustrates        our     two      recommendations          for

17   comparative effectiveness, plus a third factor

18   to be considered regarding cost.                As you know,

19   complex      patients       with        multiple       chronic

20   conditions often pose challenges in treatment,

21   and they account for a very high percentage of

22   health care costs.

23                 Some       practice       guidelines     address

24   multiple comorbidities, but most do not.                     You
                                                                     63


1    may have seen Cynthia Boyd's paper in JAMA in

2    which a hypothetical elderly woman with five

3    common        conditions,         not     including        heart

4    failure, would get 12 different medications a

5    day in 19 or more doses.                In the real world,

6    physicians are making thoughtful compromises

7    to optimize treatment for such patients.                       But

8    we     aren't        systematically            tracking        what

9    compromises         are   made    and    how    they   work     in

10   various subgroups of patients.

11                   Most of the data we need are in

12   our EHRs, but we have to extract and normalize

13   them     to    allow      meaningful      comparisons,         and

14   bring in external data to fill gaps.                   We also

15   need     to    engage      clinicians      in     constructive

16   collaboration         around      the   data.       Those      are

17   Anceta's roles.

18                   It is a very hands-on version of

19   comparative effectiveness.                The analytics are

20   only one step.            Our medical groups closed the

21   loop     with       rapid-cycle         improvement,       which

22   allows        the    findings      to     be     refined       and

23   ultimately to be validated.

24                   Our       first    recommendation         is    to
                                                                    64


1    make two deliverables a priority for funding

2    -- a set of optimized protocols for various

3    groups    of    complex        patients,    and    a     set    of

4    learnings about how to replicate data-driven

5    improvement across multiple organizations.

6                    Our           second        recommendation

7    addresses delivery system design.                   Organized

8    systems of care have been cited as models for

9    delivering high-quality, coordinated, patient-

10   centered care at low relative cost.                 President

11   Obama     and   others        have   publicly      recognized

12   these models and have said we should ask why.

13                   Based    on     AMGA's     experience         with

14   best      practice           collaboratives,        we        have

15   identified aspects of structure and process

16   that appear to support effective collaboration

17   and favorable outcomes.              We recommend funding

18   an     objective,       independent        study       of    such

19   factors     aimed       at     understanding       which       are

20   replicable and which are the strongest drivers

21   of high-quality care at low cost.

22                   We endorse a view of comparative

23   effectiveness           that     goes      beyond           simply

24   comparing medications, devices, and existing
                                                                      65


1    guidelines.         We    recommend          using    real-world

2    data in the context of collaborative, rapid-

3    cycle improvement to expand the evidence base

4    for costly and vulnerable patient populations,

5    plus   an   objective       study      of     delivery       system

6    design for effective care coordination.

7                  Thank you.

8                  DR. CONWAY:         Thank you.

9                  Mr. Fox?

10                 MR. FOX:          Thank you.          The National

11   Center      for     Patient       Interactive             Research

12   appreciates this opportunity to speak at this

13   important event.

14                 DR. CLANCY:           Could you get closer

15   to the microphone?

16                 MR.        FOX:          We     appreciate         the

17   opportunity       to     speak    at        this    event.       We

18   commend     the    Council       for    structuring          a   new

19   approach to evaluating the outcomes of patient

20   care in order to identify the best clinical

21   practices         and     to      apply            them    to     a

22   rationalization of the cost structure of U.S.

23   health care.

24                 I am Bill Fox, Executive Director
                                                                  66


1    of CPR, and I have been a patient for many

2    years, since my open heart surgery at age 17.

3    Because of the excellence of that care, I was

4    able to go on to be a law firm partner, state

5    and     federal       attorney,      and     health         care

6    executive.

7                   All of us who have been patients

8    know that we are the experts on our disease,

9    we     know   how    it    is    affecting      us,   and    we

10   experience the symptoms.             And we desperately

11   want     to   share       that   information      with      our

12   physicians, so that we can develop a treatment

13   plan together.            In today's world of health

14   care, doctors are pushed to tell, not ask.                   So

15   they miss something important which affects

16   the outcomes and treatment plans.

17                  Our     organization        is     developing

18   innovative      approaches         to   the       successful

19   engagement of the patient in the care process,

20   and creating a true therapeutic partnership

21   between the patient, the physician, and other

22   caregivers.         This therapeutic partnership is

23   crucial to the success of CER.

24                  We see two points of true patient
                                                                        67


1    interactivity -- one in the physician's office

2    where      the     physician        is    coached      to    ask   and

3    listen and to support the patient in setting

4    personal         goals        for    self-care.              Medical

5    research has shown conclusively that a patient

6    who sets his own goals is far more likely to

7    achieve       them       and    to       enjoy    better      health

8    outcomes.

9                       The second point of interactivity

10   is    as     the    patient         leaves       the   physician's

11   office,          where        simple,      easily       used       and

12   understood technologies allow the patient to

13   report what services he was provided, what his

14   doctor communicated to him, and what he feels

15   about his role in managing his disease.

16                      This independently collected data

17   can     be   used        by    health      systems,         insurance

18   companies, and researchers to compare patient

19   perceptions         across      offices,         physicians,       and

20   regions.           We would like to offer a set of

21   principles from our work which you might find

22   useful in funding CER research.

23                      The patient should, and can, have

24   a     central       role       in    developing         their      own
                                                                      68


1    treatment plan.             This therapeutic partnership

2    is      key      to        reducing       disparities       among

3    vulnerable          populations,       particularly         those

4    with    chronic       conditions.           Patient-generated

5    data can help to transform the current health

6    care model.

7                     At the moment, this is the only

8    voice       which      is     not     being      heard      on    a

9    systematic,           standardized,          and     measurable

10   basis.        We all know that culture eats strategy

11   for lunch.          So in order to achieve this true

12   interactivity, several cultural shifts will be

13   necessary.          Physicians will have to learn to

14   ask more than tell, and patients will have to

15   learn both how to tell and ask about their

16   systems, and how to set their own goals.

17                    Health systems will have to learn

18   how    to     analyze       comparative     patient       data   to

19   discern trends and design positive change.                        A

20   neutral       third        party,   not    the     provider      or

21   payer, must collect and analyze this data to

22   understand what is and is not working.                       This

23   will        bring       confidence,         integrity,           and

24   transparency          to    the   process.         More   patient
                                                                 69


1    engagement leads to healthier populations and

2    reduced health care expenditures, which is the

3    goal of both CER and this Council.

4                    As     a    patient          researcher     and

5    advocate,       I    appreciate    the        opportunity   to

6    share these insights with the panel.

7                    DR. CONWAY:       Thank you very much.

8                    Mr. Howgill?

9                    MR. HOWGILL:       Thank you very much

10   for       the   opportunity       to        comment   on    the

11   Council's            definition         of         comparative

12   effectiveness research. I am the -- I am not a

13   lobbyist.       I am the Executive Director of the

14   Institute for Health Technology Studies, which

15   is    a     501(c)(3)      research         and    educational

16   foundation focused on measuring the value of

17   medical technology innovation.

18                   Since our establishment five years

19   ago, In-Health has funded more than a dozen

20   studies         by     university-level            researchers

21   resulting in numerous articles in peer review

22   journals,        contributing,         we     hope,   to    the

23   understanding         of   the   value       and   appropriate

24   applications in medical technologies.
                                                                70


1                  On behalf of In-Health, we offer

2    the following two comments.               We applaud the

3    proposed assessment of a comprehensive array

4    of health-related outcomes for diverse patient

5    populations and want to affirm that this must

6    include    comparisons      of    the    broader     longer-

7    range     socioeconomic       effects       of     different

8    interventions.

9                  We    suggest       that     studies     which

10   concentrate on clinical and disability affects

11   alone     may      ignore        important,        currently

12   unmeasured,     longer-term       values    produced       for

13   patients, families, and their employers.

14                 The Council's first criterion for

15   scientifically       meritorious           research        and

16   investment calls for measurements of impacts,

17   "Based on prevalence of condition, burden of

18   disease, variability in outcomes, and costs of

19   care."     We wish to underscore that if these

20   four    definitional    areas      of     impact    were    to

21   exclude     either     broader       or     longer     term

22   socioeconomic      consequences,         then    comparisons

23   and contrasts among diagnostic and therapeutic

24   alternatives would be impaired.
                                                                      71


1                    So thank you for the opportunity

2    to contribute those comments.

3                    DR. CONWAY:       Thank you very much.

4                    Next, Ms. Pittman?

5                    MS.    PITTMAN:         Members             of   the

6    Coordinating           Council         for         Comparative

7    Effectiveness         Research,    thank         you    for      the

8    opportunity to address you today.                  My name is

9    Polly Pittman.          I am Executive Vice President

10   of Academy Health, a professional society for

11   health services research and policy.

12                   Ours is a multi-disciplinary field

13   that includes comparative effectiveness, among

14   other types of research.               As such, we know

15   first-hand the challenges that can result from

16   a lack of a common definition.                   In our case,

17   the preeminent example of an -- is our annual

18   attempt    to    report      funding   levels          of    health

19   services     research        across    federal         agencies,

20   which are complicated by the wide variety of

21   operational definitions that are used.

22                   On     the     issue        of     comparative

23   effectiveness         research    specifically,             Academy

24   Health     recently      published      a    study          on   the
                                                             72


1    volume and cost of ongoing research.                A copy

2    of the report, which found over 600 current

3    studies and a wide variety of costs, were sent

4    to you this week.

5                    In order to conduct the count, we

6    needed an operational definition of what and

7    what      is      not     comparative      effectiveness

8    research.        We began by convening a group of

9    experts         with      different       methodological

10   backgrounds.             The     group     agreed      that

11   comparative effectiveness research includes a

12   range of study types that must be considered

13   along two dimensions.

14                   First is the degree of comparison,

15   ranging        from,    for    example,    comparing    an

16   intervention to placebo or usual care, on the

17   one hand going to head-to-head trials of two

18   different active interventions on the other.

19   The second dimension relates to the degree of

20   experimental       control,     ranging    from   clinical

21   trials that measure efficacy to observational

22   studies, with broader inclusion criteria and

23   less tightly controlled procedures.

24                   For     operational       purposes,     the
                                                                          73


1    group agreed that three primary study designs

2    should     be     counted       --     head-to-head            trials,

3    observational         studies,         and       syntheses.           A

4    number     of     issues,    including             how    and       when

5    conservative management should be used as a

6    comparator, were not resolved.

7                     All,       however,              agreed            that

8    organizational,          behavioral,         and        system-level

9    factors        that     provide       the        context       of    an

10   intervention are critical components of good

11   comparative effectiveness research.                       Patients,

12   doctors, and administrators want to understand

13   why,     for     whom,    and        under       what     conditions

14   intervention A works better than intervention

15   B.

16                    And that means translating complex

17   organizational           contexts           into         independent

18   variables to be employed either as confounders

19   or stratifying variables.

20                    It is in this context that Academy

21   Health         strongly     supports             the       Council's

22   proposed           definition               of           comparative

23   effectiveness research.                 We appreciate that

24   the    definition        supports      the       need     to    inform
                                                                          74


1    stakeholders about the effectiveness of a wide

2    range    of    medical        interventions           for     diverse

3    patient    populations         while       at     the    same       time

4    acknowledging         differences            in       health        care

5    settings.

6                    Academy Health is pleased that the

7    definition       also     acknowledges             the       need    to

8    develop    better       data       sources      and     methods      to

9    assess        comparative           effectiveness.                   The

10   emphasis on the multi-disciplinary nature of

11   comparative      effectiveness             research          is     also

12   welcomed       because        it     sets       the      stage      for

13   collaboration            among              clinicians              and

14   researchers.

15                   Academy       Health        will      continue       to

16   track comparative effectiveness research and

17   would     welcome       the     opportunity             to    provide

18   assistance      and     work        with     the      Coordinating

19   Council on this important effort.

20                   Thank you.

21                   DR. CONWAY:          Thank you very much.

22                   Dr. Roberts?

23                   DR. ROBERTS:          Thank you.             My name

24   is Mark Roberts, and I am an internist and
                                                                 75


1    professor      of   medicine      at    the    University   of

2    Pittsburgh, and I am speaking here today in my

3    role    as    the     President    of    the    Society     for

4    Medical Decision Making, an academic society

5    concerned      with     making     better       health    care

6    decisions.

7                   I      appreciate        the     Coordinating

8    Council's invitation to speak on behalf of our

9    society's members, many of whom have developed

10   the methodologies that are used in comparative

11   effectiveness research, both here and abroad.

12   Today I will emphasize three points regarding

13   the          prioritization             of          comparative

14   effectiveness research.

15                  First,      we      support          continuing

16   investment in the development and advancement

17   of      comparative          effectiveness             methods

18   themselves, and the rigorous training in their

19   use.         Comparative        effectiveness         research

20   requires        the      application           of     multiple

21   methodologies,         including        advanced      clinical

22   trial        design,     data      synthesis          methods,

23   observational methods, mathematical modeling,

24   and many others.
                                                                        76


1                      We     cannot      rely      solely      on      the

2    randomized control trials to answer complex

3    clinical      questions,          such       as,    what    is    the

4    optimal time to initiate HIV therapy, or what

5    set of complex patient characteristics make

6    one therapy superior to another?

7                      We     support         research          in      the

8    development and evaluation of the methods used

9    to conduct comparative effectiveness, so that

10   the most appropriate methods for the specific

11   task can be used with surety.

12                     Secondly,       we     believe      that       costs

13   are     an   important          outcome,       in    addition       to

14   effectiveness.            We believe that comparative

15   effectiveness           analyses       are    enhanced       by   the

16   inclusion of costs, so that patients, doctors,

17   and society has a measure of the value of the

18   decisions they face.                 However, costs should

19   never        be        used     in      isolation,          without

20   consideration of health effects.

21                     As Alan Garber, a member of our

22   society has explained, conducting comparative

23   effectiveness without including the cost is

24   like    choosing         from    a     menu    without      prices.
                                                                            77


1    Patients    and         their       doctors         can      and       do

2    understand these tradeoffs.

3                  Third,       comparative              effectiveness

4    research    must        account           for    the      individual

5    nature of patient characteristics, including

6    their     specific        preferences              and       personal

7    values.    The best treatment for an individual

8    patient with a specific disease simply cannot

9    be   determined         from        the     knowledge         of       the

10   average    affect         of        that        treatment         in    a

11   narrowly-defined randomized control trial.

12                 While this is obvious for biologic

13   variability,        such            as      the         choice         of

14   chemotherapy      in     breast          cancer     patients           who

15   carry   specific        genetic          markers,       it   is    also

16   true for the values that patients have for the

17   possible    outcomes           of    their        care       and       its

18   treatments.         I    have       --     that     a     particular

19   therapy has a higher five-year survival may be

20   irrelevant to an ailing grandmother who wants

21   the therapy that maximizes her ability to be

22   alive at her granddaughter's wedding in two

23   months.

24                 CER       must    develop          the    ability         to
                                                                   78


1    account       for         the      important         individual

2    differences in physiology and risk faced by

3    patients making decisions about their care,

4    and   it   must      also        account     for     individual

5    patient preferences.

6                  Tools and methods to help patients

7    and   their    doctors           include    these     important

8    characteristics            are     necessary,         so    that

9    patients      can        become     partners       with     their

10   caregivers     in        their     search     for    the    best

11   therapy, and that the practice of evidence-

12   based medicine can become more personalized

13   and patient-centered.

14                 If     conducted        with    well-validated

15   methods        that             incorporate          individual

16   characteristics of patients and their values,

17   comparative         effectiveness           has      tremendous

18   opportunity         to     improve     the        quality    and

19   efficiency     of        health     care     in     the    United

20   States.

21                 Thank you.

22                 DR. CONWAY:           Thank you very much.

23                 Dr. Vojta?

24                 DR.         VOJTA:           Good      afternoon.
                                                                     79


1    United Health Group is very excited about your

2    efforts to advance comparative effectiveness,

3    which we feel is vital to raising the quality

4    of health care in this country.

5                    In   many      ways,    the     work    of    this

6    Council,      similar    to     the    work     we    at   United

7    Health Group do every day, we use data to help

8    stakeholders make good decisions.                    Simply put,

9    we      believe         that        patients,          families,

10   physicians,       and    purchasers        deserve      to    know

11   what treatments work best for which patients,

12   and what the value is, so that they can make

13   more informed choices.

14                   That is the value of CER.                    It is

15   an investment in better health for all of us.

16                   Among our recommendations for the

17   new     CER   funding,      first,        we   ask     that    you

18   consider focusing on broader dissemination of

19   existing solid evidence.               For many years, the

20   United        Health     Foundation            provided       U.S.

21   physicians with a copy of the British Medical

22   Journal's clinical evidence.

23                   More    recently,       we     have    supported

24   broad    dissemination         of   the      USPSTF    Guide    to
                                                                   80


1    Clinical   Preventive          Services.        And    in    our

2    experience,     physicians         greatly         appreciate

3    receiving this rigorous credible information.

4    There are, however, many more ways to connect

5    physicians     across      the     country      with        vital

6    existing CER and drive this momentum further.

7                  Next,       we     suggest       disseminating

8    existing CER to consumers.                  Not all CER is

9    appropriate for consumer use, but there are

10   multiple     conditions,        such    as    diabetes       and

11   prostate   cancer,        where    such      research       could

12   benefit    patients       today,       enabling       them     to

13   engage in more knowledgeable discussions with

14   their physicians and work with them to arrive

15   at more informed choices.

16                 Some    portion          of    the      new     CER

17   funding    might     be    used    to       develop    simple,

18   secure ways to share this information with the

19   public.    And there are vital areas of medicine

20   where new CER studies should be done to help

21   address       the         needs        of       underserved,

22   unresearched, and other priority populations

23   who experience a disproportionate burden of

24   health risk, disease, and cost.
                                                                        81


1                     Quality       care    is    the       number      one

2    focus for comparative effectiveness research,

3    but     this     research       can     also       help     improve

4    efficiency.           We know from our experience in

5    two    decades     of    experience         --    we   documented

6    this recently in the first report issued by

7    the     United     Health      Center       for     Health      Care

8    Reform       and      Modernization         --     that      better

9    quality not only produces better health but

10   reduces unnecessary spending.

11                    Let's     look       at    diabetes.              The

12   numbers      are   staggering.             Nearly      24   million

13   people with diabetes, another 57 million with

14   pre-diabetes, cost in 2007 $174 billion.                           And

15   much    of     this     cost    comes       from    the      use    of

16   expensive new drugs and procedures, yet in a

17   recently released NIH funded study involving

18   diabetics with heart disease, those receiving

19   these    newer     modalities         did    no     better      than

20   patients receiving the older, less expensive

21   treatments.

22                    United        Health         Care          recently

23   launched the diabetes health plan, responding

24   to consumers' interest in a health plan option
                                                                  82


1    that reduced their out-of-pocket expenses if

2    they     comply    with     evidence-based        standards.

3    This plan represents an extremely promising

4    approach that companies like GE and Hewlett-

5    Packard are embracing, but patients and their

6    physicians would benefit further from CER that

7    provided additional answers.

8                  What       combinations     of     drugs      work

9    best for me?          What are the advantages and

10   disadvantages       of     new   therapies     for    Type    2

11   versus more established therapies?                 What care

12   management        strategies      work     best       in    the

13   outpatient arena?

14                 We    are     highly    encouraged      by     our

15   early findings on consumer acceptance of this

16   health plan.        As investments in CER are made,

17   the research base strengthens, approaches like

18   these can be extended to other conditions and

19   other    patient     groups,     and,    again,      move   the

20   health     system    towards      a     higher     level      of

21   quality and value performance.

22                 Thank you.

23                 DR. CONWAY:        Thank you very much.

24                 Is     Dr.    Patricia      Salber      on    the
                                                                       83


1    phone?

2                     DR. SALBER:           Yes, I am.          Can you

3    hear me okay?

4                     DR. CONWAY:         Yes, we can.

5                     DR.       SALBER:               Okay.           Good

6    afternoon.             I   am     Patricia        Salber,      Chief

7    Medical Officer and Executive Vice President

8    of Universal American Corporation, a senior-

9    focused health care company that sponsors a

10   number      of        Medicare         programs,         including

11   Medicare     Advantage             plans     that        focus    on

12   coordinating patient care.

13                    We        believe         that      comparative

14   effectiveness research can inform and help to

15   rationalize       all      aspects     of    the    health       care

16   enterprise, ranging from head-to-head trials

17   of   therapeutics          to     comparisons      of    the     most

18   effective ways to organize and deliver health

19   care services.

20                    With       respect         to     the      latter,

21   Universal    American           uses    operational         CER   to

22   determine        not       only     whether       our     clinical

23   programs are effective, but also to gain an

24   understanding of which aspects of the program
                                                              84


1    add value and which add only cost.             To achieve

2    our   goals,     we     have    engaged       an    outside

3    independent researcher, Dr. Thomas Wilson, who

4    testified on behalf of the Population Health

5    Impact     Institute      in    the    last        listening

6    session.

7                   Dr.    Wilson   has    been    helping    us

8    make better resource allocation decisions for

9    our   health     care    dollars.          Based    on   our

10   collective work, we have a learning culture at

11   Universal American, and here are a few of the

12   lessons that we have learned.

13                  Number   one,    it    is    important    to

14   involve an evaluation expert at the outset of

15   health improvement program design, so that the

16   program is launched in a way that facilitates

17   evaluation of its effectiveness.

18                  Two,   there    must   be    understanding

19   and support for this type of evaluation from

20   the C suite, as well as from departments like

21   IT, finance, and marketing.

22                  And,   number    three,      well-designed

23   randomized control trials are often called the

24   gold standard.        However, they are not always
                                                                           85


1    possible        or     even        desirable        in        business

2    settings.        In fact, we have found that many

3    valuable insights can be gleaned just from the

4    process of doing the evaluation.

5                    Our        learning       culture       now     shapes

6    many      of         our         programs.              We      employ

7    observational, quasi-experimental designs with

8    a comparable reference to evaluate them.                             And

9    a couple of examples: we are analyzing impact

10   of the addition of a psychosocial enhancement

11   towards COPD disease management programs to

12   assess its impact on rehospitalization.

13                   Another           example     is    a        study    to

14   determine the effectiveness of a program that

15   engages     community            pharmacists       to        encourage

16   patients to take their medications and follow

17   their physician's guidance.

18                   Based on our experiences, we would

19   like       to         make          the      following               four

20   recommendations             to     the     Council.             First,

21   projects       that        are     dedicated       to    operations

22   delivery system assessment should be given a

23   funding    priority.               Second,     operational           CER

24   projects       that    are        funded     should      advance       a
                                                                        86


1    culture    of      learning        in     the     health          care

2    enterprise.

3                  Third, operational CER projects do

4    not necessarily have to be designed to achieve

5    definitive results.             We can learn and advance

6    operational efficiency and effectiveness with

7    studies of less rigorous design.                       And then,

8    finally, please consider developing different

9    criteria     for    the        assessment       of    operations

10   delivery   system        research       projects           that    use

11   observational       and        quasi-experimental                study

12   design from those that are used to evaluate

13   strongly-controlled experimental studies.

14                 Thank       you      very      much          for     the

15   opportunity to comment.

16                 DR. CONWAY:          Thank you.

17                 We        will     now     open        it      up     to

18   questions from the Council.

19                 MR. MILAN:          Dr. Cuddeback, what is

20   the   nature,      do    you     think,      of      the    federal

21   investment      that       could        be   made          to     help

22   organizations like yours use data warehouses?

23   Is    there     some      sort     of    central          activity,

24   investment, that would help a broad array of
                                                                  87


1    organizations like you are trying to do, and

2    our last speaker?

3                      DR.    CUDDEBACK:           Well,   I    think

4    actually the strategic framework that you have

5    laid out is -- hits a number of areas where we

6    have    needs.           Dr.    Roberts        had    mentioned

7    investment        in     methods       development     and   an

8    example of newer methods that are evolving to

9    allow      inferences       from       observational       data,

10   inferences about causality as opposed to just

11   observing association.             You know, that sort of

12   method would be very helpful in the kinds of

13   studies     we     are    doing     using     data    that   are

14   byproducts of the actual process of care.

15                     We    obviously       are    interested     as

16   well in methods of enhancing adoption.                    We are

17   doing      that    as    part     of    our    collaborative,

18   closed-loop feedback process, but I think that

19   is one of the areas that clearly is -- there

20   are opportunities for new contributions there.

21                     DR. DELANY:       Mr. Fox, I wanted to

22   follow up with you on what -- I am going to

23   use the term "expert patient model" or expert

24   --   you    know, and I have seen a number of
                                                                   88


1    studies out of the UK and Canada, as well as

2    in Australia, about this.             And they talk about

3    a   number   of   precursors         that    had   to   happen

4    before they got physicians to the point where

5    they would let the patient be an expert.                     What

6    has to change here?

7                 MR.    FOX:        I    think    what      has   to

8    change here is the idea that when we look at

9    current patient satisfaction surveys, or the

10   kind of information that we are drawing out of

11   patients currently and measuring, it pretty

12   much stops at the satisfaction-dignity kind of

13   questions.

14                We    need    to    develop      methodologies

15   and technologies when we are developing these

16   HIT solutions that allow the patient to enter

17   clinically significant information into this

18   system that becomes part of these EMRs and

19   PHRs as they go forward.

20                So what we are talking about is

21   using health literacy concepts and cultural

22   competency concepts to be able to interpret

23   the   information    that       is   coming    out      of    the

24   primary research that is going to be happening
                                                                           89


1    in   CER,      so       we     can    place         that     into     the

2    technology in such a way so that the patient

3    can be reporting on the effectiveness of the

4    doctors     that         are     implementing              these     best

5    practices in their clinical practice.

6                     So that if you are talking about,

7    say, the eight big chronic diseases, you can

8    formulate      a     question         type     of    basis     through

9    technology          that       can        be   utilized       by      the

10   patient, so that they can get used to, in that

11   atmosphere, being a part of that team.

12                    We see a lot of talk about Health

13   2.0 and information therapy and the way we

14   would like this to work going forward down the

15   road,    and     information              being     pushed    to     cell

16   phones, et cetera.               But when you look at the

17   studies, you see that very few people actually

18   utilize this, and most of the people that do

19   are highly educated, high income, et cetera.

20                    I went to a conference where Neil

21   Calman     spoke,        who    is        on   President       Obama's

22   committee, and he said, you know, "You people"

23   --   and    he looked around at us, and I was

24   sitting     at      a   table        of    Kaiser     people        where
                                                                           90


1    everybody, you know, two degrees was like the

2    minimum entry requirement, he said, you know,

3    "You are not the people we need to design this

4    for.          As      you        all     are     designing         these

5    solutions, they need to be inclusive."

6                        So I think we have to start in the

7    clinical suite with allowing the doctor and

8    the patient to work together at a very sort of

9    primary       level       with         the    findings      that      are

10   coming out of CER to build a foundation to do

11   all of the rest that we want to do.

12                       DR. KILPATRICK:              Mr. Fox, I just

13   want     to     kind        of    follow        up    in    the    same

14   direction           you     talked       about        the   physician

15   allowing the patient to interact.                              We have

16   heard a lot on the Council about involving the

17   patients       in     the    sort       of     design,      selection,

18   processing of developing CER.

19                       And     could       you     perhaps        talk     a

20   little        bit     about       that?          We     have      heard,

21   obviously, there is training requirements, and

22   what would you see as how to get the cadre of

23   patients up to that level to be able to really

24   understand the complexities of some of these
                                                                        91


1    research      protocols.          It     isn't       just    simply

2    let's    grab      a    bunch    of    people       and     do    some

3    studies.

4                    So I think your comment on that

5    from    the   patient      perspective         would        be    very

6    helpful.

7                    MR.      FOX:         Well,     I    think        what

8    patients are looking for is an ability to get

9    into the game.           I have talked to people that

10   are    developing        these    technologies            who     were

11   told, you know, Medicaid patients won't play

12   in    this    game,      they    don't    understand,             they

13   don't care, whatever it is.                   You are going to

14   get zero results.

15                   And     that     didn't    pan       out    in     the

16   studies.           And     if     they        were     given       an

17   opportunity to get into the game, they were

18   willing       to       participate,       and        they        could

19   understand because they do understand their

20   symptoms.       But I think where we really have to

21   -- it is the health literacy problem that has

22   to be overcome.

23                   And to be able to translate, you

24   know, 10,000 CPT codes in clinical studies --
                                                                           92


1    you    know,    I    read    something            that    said    when

2    Marcus       Welby   was     a    doctor      there       were    200

3    clinical studies a year, now there is 30,000.

4    So we can't expect patients to keep pace with

5    that,    particularly            when    they      are    concerned

6    with their particular problem on a day-to-day

7    basis.

8                    But I think that we could start in

9    designing and utilizing HIT technology to be

10   able    to    get    them    into       at    least       the    basic

11   understanding        of     what        should      be    happening

12   between      their    clinician,         whether         that    is    a

13   physician or somebody else as we go forward,

14   and then at each and every visit, and what

15   they    can    do    to     implement        that        into    their

16   lives, and then contribute information back to

17   the clinician or the study that is valuable to

18   them in sort of a CQI process of getting the

19   CER over into practice with the patients.

20                   MS. TANDEN:         Dr. Vojta, I had a --

21   you talked a little bit about how you are

22   designing      insurance         plans       to    interact      with

23   comparative effectiveness research, and what

24   the receptivity of that is.                   And I wanted to
                                                                   93


1    get   a   sense,      is   that    something        unique    to

2    United Health, or is that a new arena?                       And

3    could you just spend like another -- just a

4    minute    or   two     describing     exactly        how   that

5    works?

6                   DR. VOJTA:          Sure.      This year we

7    launched the diabetes health plan.                     And we

8    took the approach that most people think about

9    health care in two spaces.                 One is coverage,

10   and one is care, the care they receive in the

11   office.     And we wondered, could we -- today

12   most of our health benefits are the same.

13                  If we go around the room, we use

14   -- all have employer -- generally, employer-

15   sponsored health care, and the benefit package

16   looks the same, yet we know people who live

17   with chronic disease, as an example, have a

18   disproportionate higher out-of-pocket expense.

19   And so we recognize that that is a reality,

20   and that has been well documented.

21                  And    we   said,     could    we     create    a

22   personalized         benefit      design     that    actually

23   worked for people living with diabetes?                      And

24   we picked diabetes because of the prevalence,
                                                                     94


1    the cost, and because it is an ambulatory-

2    sensitive condition.

3                   And we said to consumers, "If you

4    -- we recognize that the benefit -- that you

5    would    like       to   reduce      your       out-of-pocket

6    expense.       Would you be willing, in exchange

7    for compliance with ADA standards, so that you

8    --   that     you   go   get    your      --    you   get      your

9    hemoglobin A1C checked, you follow your LDL,

10   you get your eyes checked once a year.                          In

11   exchange      for    that,     if    we    offered       you    an

12   enhanced benefit, so we significantly reduce

13   your    out-of-pocket        expense,      would      you      find

14   that acceptable?"

15                  And before we rolled this health

16   plan out, we did a large amount of research,

17   both    quantitative         and    qualitative.               And,

18   fascinating,        73   percent     of    diabetics        said,

19   "Sign    me    up."       And       we    had    three      major

20   employers sign on with us to run this pilot.

21   And the really exciting part was it starts,

22   first and foremost, with a screening, so that

23   we can identify the 57 million pre-diabetics.

24   And even in one of the screens with a very
                                                             95


1    commercial,        highly     paid      population,      I

2    wondered, would the numbers pan out?              If I am

3    a pediatrician, would it pan out?

4                  And, in fact, 26 percent of people

5    screened who were previously unaware that they

6    had pre-diabetes screened positive.              And four

7    percent of those screened screened positive

8    for diabetes.

9                  So   we    start   with   the     screening.

10   We   offer   the   enhanced      benefit,     which   feels

11   like it is for me, the diabetic.            And then, we

12   offer technology.        It is hard to take care of

13   yourself every day.         Health care happens every

14   day for diabetics.

15                 So    we      provide     them     with    an

16   automatic reminder system, tracking tool, just

17   like the dentist with a postcard, but we do it

18   constantly, because there is lots to do.                And

19   we wrap it up in a package that real Americans

20   are used to getting, and that is your health

21   plan.   It has really gone quite well, but we

22   are in the evaluation phase.

23                 MS. TANDEN:         Right.       And if you

24   have any sense of, you know, its impact on
                                                                      96


1    care and quality of coverage and how providers

2    feel,    but   also,    you    know,     impact       and    cost

3    effectiveness, that would be interesting to

4    share.

5                   DR. VOJTA:      So it started in -- it

6    was   first    rolled   out        in   2009,   number       one.

7    Number two, we do have an evaluation.                        But,

8    number    three,    phase      2    includes     --    we        are

9    talking to providers about new payment options

10   to -- for diabetic care, and they are very

11   excited.

12                  DR. CONWAY:          I have a question --

13   I will be brief with my question -- for Dr.

14   Cuddeback, and I think Dr. Roberts, either/or.

15   So we -- and I have some personal experience

16   with this and hear this a lot from the outside

17   -- the struggle of if you want to look at

18   effectiveness,      collecting           the    data        in    a

19   registry, and translating those into results,

20   and then you try to fit that question into a

21   typical    RO1     funded      principal        investigator

22   model.

23                  I   would      be    interested        in     your

24   thoughts how, you know, we can think as a
                                                                     97


1    Federal Government how to both, you know, fund

2    important       RO1   work,     but       also   think      about

3    funding       these    types    of        studies    that      are

4    broader than a singular question, and actually

5    do                 have,                  you               know,

6    registries/translation/adoption

7    infrastructure.

8                    DR.    CUDDEBACK:                Well,      there

9    certainly are infrastructure requirements, and

10   just one example of even varying levels of

11   data        that      are      available,           and        some

12   opportunities to use funding like this, maybe

13   to     push    the    level    up     a     little      bit,    we

14   obviously are thinking a lot about disparities

15   in    our     population,     but    two    of    the     medical

16   groups that are part of Anceta today as a

17   matter of policy don't collect data on race

18   and ethnicity in their operational systems.

19                   And so as part of the work we are

20   doing with them -- now, you heard earlier that

21   the    registries      are     collecting        those      data,

22   because they define their data set and their

23   definitions        prospectively.           As   part     of   the

24   work we are doing with them, a couple of them
                                                                    98


1    now are beginning to make that change.                        They

2    understand the policy issues, but they also

3    understand the value of having the data.

4                   So     we     are       taking    observational

5    data    that    are        the     byproduct      of    actually

6    providing      care    and       essentially      raising      the

7    standards for those data.                And we find this in

8    a number of other aspects.                 Entering discrete

9    data into EHRs rather than narrative data --

10   that    migration          is    already        occurring,      of

11   course, but one of the best ways to encourage

12   it is for people to be able to see the data

13   and    see   the    results        of    those   data    in    the

14   analyses.

15                  So I think there is -- by funding

16   the process of using existing data, real-world

17   data,   and    with    the       understanding         that   that

18   process itself may raise the quality of the

19   data and improve the usability of the data,

20   improve the inferences you can draw from it, I

21   think that is a valuable contribution.

22                  DR. ROBERTS:             Let me just say that

23   I   think     there    are       two    different      kinds    of

24   research that needs to be and is funded in
                                                                99


1    those     arenas.         There       is,    first,       the

2    application of methods to particular problems

3    that are not the traditional basic science,

4    RO1 activity.       And those are in fact being

5    funded, and they are being funded across the

6    NIH, and they are being funded through AHRQ

7    and many other places.

8                  What has been a little bit more

9    complicated is what I would call the envelope-

10   pushing   development     of    the    methods      of    CER,

11   much like the NIH funded the development of

12   methods in many scientific and basic science

13   disciplines     over    the   years.        And    AHRQ   has

14   currently, and to some extent the NSF, have

15   been the only bodies that have been sort of

16   willing    to    fund     the     basic      science       of

17   comparative effectiveness research to advance

18   the methods themselves, which need advancing.

19                 And so I would strongly recommend

20   that some component of what is proposed is be

21   that we move into the realm of true envelope-

22   pushing RO1 funded research, development of

23   strong,    rigorous       methodologies           that    are

24   advancing that field.
                                                                 100


1                  DR. EMANUEL:       Dr. Buckley, I have

2    -- sorry.     Usually, I am pretty loud, or loud

3    enough.

4                  I was just wondering, if BIO was

5    to create a comparative effectiveness program

6    that would be acceptable to it, what would the

7    structure be like, and how would it compare to

8    what we have heard from some of the other

9    individuals here?

10                 DR.    BUCKLEY:      Well,       I    think    it

11   would incorporate quite a lot of the aspects

12   that they have talked about, pushing forward

13   the methodological aspects.             I mean, RCTs are

14   great,    but,      unfortunately,          they    look     at

15   efficacy,     not     effectiveness,         in    the     real

16   world.    They are based on a homogenous group

17   of   people        that   unfortunately            does     not

18   represent      many       of     the        patients        and

19   subpopulations.

20                 I think we would want to see --

21   so, one, really up front a large investment in

22   methodological        issues.          We    are     at     the

23   forefront     of    the   21st   century      of    medicine

24   where it is going to be molecular-based, not
                                                                        101


1    just    race      and       ethnicity,       but    down      to   the

2    molecular level.               And so we need to think

3    through, how do we incorporate those sorts of

4    biomarkers        into        comparative          effectiveness

5    research?        Is it NF1 trials, adaptive clinical

6    design, et cetera?

7                     So     I    would     say     that     the    first

8    thing   that      you want to do is lay a great

9    foundation,           because     we     are       at   also       the

10   beginning         of         comparative           effectiveness

11   research in the U.S.                   And so when you are

12   building     a    large       building       you    always     begin

13   with the foundation.

14                    The second thing I would say is

15   that    we       really       want     to     incorporate          all

16   stakeholders.           One of the -- when thinking

17   through NICE, one of the criticisms of NICE is

18   that    they          haven't        really        involved        the

19   patients.        People would -- NICE would say that

20   they have.        But when you talk to the patient

21   groups, they bring up the point that one of

22   these CE studies often costs 80,000 pounds.

23   And they have researchers that they employ,

24   and that is their full-time job.
                                                               102


1                   The patients, on the other hand,

2    are supposed to gather the data themselves,

3    train themselves as to how to put that data

4    forward   in     a   way     that   is     scientifically

5    acceptable.      Now, this is on top of holding

6    down a job, dealing with their family, and

7    dealing with their disability or illness.

8                   So how do you get that stakeholder

9    group involved?            Also, how do you get the

10   other stakeholders involved -- the insurance

11   companies, the innovator groups, et cetera,

12   the   doctors        and     clinicians,      and        other

13   clinicians,     nurse      practitioners,     et       cetera,

14   who are on the forefront, so -- in order to

15   set the proper research agenda and also get

16   the right questions there.          So that is how BIO

17   would start it.

18                  DR.   EMANUEL:        So,    if     I     could

19   summarize, we need stakeholder advice, and we

20   need methodological research.

21                  DR. BUCKLEY:         That would be the

22   foundation of it.          Further, we would focus on

23   the totality of the health care system rather

24   than just a simple drug-to-drug evaluation or
                                                          103


1    a surgery versus drug evaluation in the study

2    design.

3                  DR. EMANUEL:     What do you mean the

4    "totality"?

5                  DR.   BUCKLEY:     The   totality,     the

6    way in which health care is delivered should

7    also   be   taken   into   account.      Insurance    --

8    value-based insurance design, how does that --

9    and how does --

10                 DR. EMANUEL:      We should evaluate

11   whether that works or not.

12                 DR. BUCKLEY:      We should evaluate

13   how that impacts patient care and outcomes,

14   yes, absolutely.

15                 DR. EMANUEL:     Okay.

16                 DR.   GRAHAM:     Mr.    Fox,   actually,

17   taking from the last question and applying it

18   to some of the things you talked about, what

19   would you describe as the key components of a

20   programmatic set of activities, or some kind

21   of structure that encourages those therapeutic

22   partnerships that you alluded to?              Which I

23   thought was a great way to articulate true

24   kind of patient-provider partnership.
                                                               104


1                  MR.    FOX:      I    mean,   19    years    of

2    trial, I am not used to -- I think what we

3    need to be able to do is implement some kind

4    of systematic measurement of the effectiveness

5    of the communication between the clinician and

6    the patient as this is rolled out.

7                  We     were    using    the    example       in

8    Pennsylvania there is 67 counties, and at some

9    point they are going to be told to roll out

10   some form of universal health care, and they

11   are going to be able to -- and as CER comes

12   forward, how are you going to measure best

13   practices across those 67 public health areas?

14                 So if you can measure the way in

15   which the effectiveness by which clinicians

16   are communicating the -- whatever the current

17   state of the art is in evidence-based medicine

18   for   these    chronic       care    conditions      across

19   populations,        across     different         vulnerable

20   populations, that is how you are going to cut

21   down on the disparities.

22                 Say,     why     are     we    getting        a

23   measurement over here of -- you know, we are

24   having an 83 percent understanding of these
                                                              105


1    certain     criteria,     and    the       consistency    in

2    application       of   evidence-based       medicine,    and

3    over here it is only 56 percent.              What are the

4    factors that are contributing to that?                   And

5    how can we implement these best practices over

6    here?

7                  So I think in terms of getting the

8    patients involved in having their input into

9    the system actually affect the effectiveness

10   of   the   care    and   the    use   of    evidence-based

11   medicine.      That is sort of like the first

12   level in involving the patient in this sort of

13   HIT transformation that we are undergoing now.

14                 DR. CLANCY:        So, Dr. Roberts, we

15   very much appreciate your bringing to bear the

16   considerable       intellectual        capital    of     the

17   Society for Medical Decision-making.                   There

18   are some who believe that once you get beyond

19   the world of randomized trials that it is kind

20   of the Wild West in terms of methods -- I am

21   quite serious -- and that we need policies or

22   agreed-upon criteria to certify studies that

23   use other methods.

24                 And I wondered if the society had
                                                                        106


1    thought about this, because there is obviously

2    a       tension        between            that            level      of

3    standardization, if you will, and continued

4    innovation that you spoke about.

5                    DR. ROBERTS:              Sure.           Although I

6    would remind people -- I mean, there is no

7    randomized control trial that proves the earth

8    went around the sun, and not vice versa, yet I

9    believe it almost as well as I believe that

10   beta     blockers        improve          outcomes         in     heart

11   attacks.     So much of science has learned a lot

12   about    what     it     learns         not    from        randomized

13   control trial but from thoughtful observation

14   with rigorous underlying theory.

15                   And    so     we     in       the    society       have

16   struggled    with       this,      because          in    fact    these

17   methods,     at       least     at      the     level       that    we

18   understand        them      currently,              are     in     fact

19   complicated and difficult.                     But that doesn't

20   mean they are impossible, and it doesn't mean

21   they are not valuable.

22                   There is a little bit of a worry

23   about     certification,           in     that       certification

24   itself assumes that we really understand those
                                                               107


1    methods     well      enough        to    make     what    an

2    appropriate certification would be.

3                  And so I think we are a long ways

4    away from there, but I do think that when we

5    even -- even looking at the incredibly strong

6    methodology     that      is    a    randomized     control

7    trial, and when you have 10 percent of the

8    people who died on therapy A and 20 percent of

9    the people who died on therapy B, and you say,

10   "A is better than B," that is true.

11                 But true wisdom about that disease

12   is:    why did those 10 percent of the people

13   die?      And why did those 20 percent of the

14   people die?        Did some people who got A who

15   died, would they have done better on B?                   And

16   vice versa.

17                 So we would -- as a society would

18   believe that there is substantial benefit to

19   be made by enhancing and understanding these

20   methods,       and,       again,          by     repetitive

21   demonstration that they get the right answer

22   in experimental circumstances and in natural

23   experiments,       that    you      can    begin    to    get

24   confidence      about          their       validity       and
                                                           108


1    robustness.

2                  DR. CONWAY:     Thank --

3                  DR. KUPERSMITH:      Could I ask --

4    too late?

5                  DR.   CONWAY:       Yes,       go     ahead.

6    Sorry.

7                  DR.   KUPERSMITH:        It    is   only    a

8    comment, so -- no, I would like to commend

9    everybody's interest in methodology, because I

10   do think one of the things that is going to

11   happen because of the interest in comparative

12   effectiveness research is we are going to look

13   at these methodologies differently than we did

14   before.     There is no proof for the randomized

15   control trial either, and so nobody certified

16   it.

17                 I would like also to correct you,

18   Dr. Roberts, on one thing.          Besides NSF and

19   AHRQ that funds those research, VA does as

20   well.

21                 DR. ROBERTS:      I apologize.          You

22   are   absolutely    right.     Quite     a   bit,    as   a

23   matter of fact.

24                 DR. CONWAY:      Thank you to panel
                                                                    109


1    number two.

2                  We are going to take a very short

3    five-minute     break,     and    then       reconvene         for

4    panel number three.

5         (Whereupon,         the    above-entitled           matter

6    went off the record at 1:50 p.m. and resumed

7    at 2:02 p.m.)

8                  DR. CONWAY:         All right.             We are

9    going to go ahead and get started with panel

10   number three.       Apologize for the delay.

11                 So    we   will    --   same    format.           We

12   will start with Dr. Brendan Carr.

13                 DR.    CARR:       Hello.        My    name       is

14   Brendan Carr.       I am an Assistant Professor of

15   Emergency    Medicine     and     Epidemiology           at    the

16   University    of    Pennsylvania.         And       it    is    my

17   honor today to speak to you on behalf of the

18   American College of Emergency Physicians and

19   the Society for Academic Emergency Medicine.

20   Collectively, ACEP and SAEM represent about

21   30,000 members.

22                 I would like to highlight a few

23   things about our specialty that I think are

24   relevant, and I would like to briefly offer
                                                                    110


1    some    perspective       on     the        opportunities       to

2    improve the health of the American people and

3    to     preserve       resources           using     comparative

4    effectiveness research in the emergency care

5    setting.

6                   Specifically, the organizations I

7    represent      urge     you     to        define    comparative

8    effectiveness         broadly        in     order     to    allow

9    careful study of the processes of emergency

10   care    that   have     profound          impact     on    patient

11   outcomes and health care costs.

12                  Emergency       medicine        represents       an

13   increasingly important part of the health care

14   system, both with respect to the magnitude and

15   the breadth of care that we provide.                         There

16   are    about   120     million       emergency       department

17   visits annually, an average of one visit for

18   every three       Americans.

19                  Emergency       providers           treat    nearly

20   all    patients    with       time-sensitive          and    life-

21   threatening illness and injury, and emergency

22   departments serve the entire spectrum of the

23   U.S.   population      and     the     entire       spectrum    of

24   disease     states,     in      the       words     of     EMTALA,
                                                                     111


1    "Anyone       needing         emergency              treatment,

2    regardless of citizenship, legal status, or

3    the ability to pay."

4                  EDs    also     disproportionately                care

5    for the underserved, minorities, the elderly,

6    children, those with both acute conditions and

7    exacerbations of chronic illness.

8                  I    would    like    to    focus          on   three

9    areas of interest -- diagnosis, disposition,

10   and     design.      With     respect          to    diagnosis,

11   emergency providers use lab test technology-

12   based     diagnostic       aids    to    facilitate           rapid

13   identification and treatment of serious injury

14   and   illness.       Whether       we    are    evaluating        a

15   patient who may be bleeding from trauma, blood

16   clotting causing stroke or heart attack, or an

17   overwhelming infection, the goal of emergency

18   care is rapid, accurate diagnosis and life-

19   saving treatment.

20                 Emergency       providers         rely      heavily

21   on lab and technology-based testing, including

22   cross-sectional      imaging       such    as       CT    and   MR.

23   However,     the    comparative          effectiveness           of

24   different approaches to rapid diagnosis has
                                                                          112


1    not   been     rigorously            evaluated,              and     this

2    represents        a       major    missed         opportunity         to

3    improve      patient        outcomes        and         to     conserve

4    resources.

5                   Disposition.            Emergency providers

6    either make or strongly influence the decision

7    to admit patients to the hospital, and this

8    decision     is       a    key     determinant           of     patient

9    outcomes and health care costs.                     Fully half of

10   all   U.S.    hospital            missions        come        from   the

11   emergency      department,            and        many        treatments

12   initiated in the ED are continued during the

13   hospital course.

14                  The admission of a patient to the

15   hospital should be considered as an important

16   treatment decision, and yet the comparative

17   effectiveness of inpatient versus outpatient

18   treatment in many conditions, including low-

19   risk chest pain and neonatal fever, have not

20   been rigorously evaluated.

21                  The          failure         to      include          the

22   question      of          inpatient     versus               outpatient

23   evaluation and treatment in the definition of

24   comparative           effectiveness              research          would
                                                                      113


1    represent      a     major     missed        opportunity          to

2    improve      patient     outcomes        and     to     conserve

3    resources.

4                   Finally, design.              There are many

5    unanswered         questions         related           to        the

6    fundamental        organization        and       delivery         of

7    emergency     care,     and    we    caution       against       too

8    narrowly      defining       comparative        effectiveness

9    research.

10                  The Institute of Medicine released

11   a   series    of     reports    titled       "The     Future       of

12   Emergency      Care"     three       years       ago.            They

13   described      the    emergency      care       system      as     --

14   their language -- at the breaking point, and

15   suggested      the     development         of    coordinated,

16   regionalized, and accountable systems of care.

17                  Substantial efforts are ongoing to

18   build regional emergency care systems for the

19   life-saving           treatment          of           myocardial

20   infarction,        stroke,     cardiac     arrest,       sepsis,

21   trauma.       These     efforts      and     the      associated

22   resources      will    only     be    well      spent       if    we

23   compare      the   effectiveness        of      the    different

24   systems and the approaches.
                                                                          114


1                   We         hope       that         comparison          of

2    different       organizational                   structures          for

3    emergency      care       systems          will    be        explicitly

4    included      in      the          meaning        of     comparative

5    effectiveness research.

6                   And        I    thank       you     very       much    on

7    behalf   of    the    emergency            care        community     for

8    allowing us to contribute to this discussion.

9                   DR. CONWAY:               Thank you.

10                  Dr. Hoffman?

11                  DR.        HOFFMAN:              Members        of    the

12   Council, I likewise appreciate the opportunity

13   to   give    comment          today.        I     am    Dr.     Matthew

14   Hoffman, and I will be speaking on behalf of

15   members of the Maternal Fetal Medicine Units

16   Network.      I am not a lobbyist.

17                  For        those      of     you        who    are    not

18   familiar      with     the         work     of     the       MFMU,    we

19   represent 14 perinatal centers that deliver

20   obstetrical         care       to        approximately          140,000

21   women on an annual basis.                         Our fundamental

22   core   mission       is       to    do    comparative          research

23   trials      amongst       this       group        to    improve      the

24   outcomes of pregnant women and their neonates.
                                                                 115


1                     My purpose in being here today is

2    to underscore the importance obstetrics has in

3    this country.          In 2004, there were 4.7 million

4    hospitalizations, which were the direct result

5    of pregnancies and its complications.                       When

6    coupled together with the normal outcome of

7    these, the newborn, there were an additional

8    4.4 million admissions that were the direct

9    result    of     pregnancy.         This    represents       the

10   single    largest       cause   of    hospitalization         in

11   this country.

12                    Moreover, government, through its

13   involvement in the state Medicaid, remains the

14   single largest guarantor of maternity care in

15   this country.          The importance of this care is

16   monumental to both women and their neonates,

17   and progressively research has also emphasized

18   the issue that many adult diseases have in

19   fact     their    beginnings        and    origins    in     the

20   perinatal period.

21                    Despite the broad use of prenatal

22   care, there is tremendous differences and wide

23   variation        in    the    way    that     the    care    is

24   delivered.            And,   moreover,      there    has    been
                                                                       116


1    progressive          introduction          of     technologies

2    without meaningful assessment as to the way

3    they impact both women and their neonates.

4                    We believe that this is largely a

5    deficit due to the fact that there is not

6    currently       a     large        maternal       child        data

7    repository      to    address       such    an    issue.            We

8    believe specifically that the development of a

9    maternal    child      data       repository      should       be    a

10   recommendation by this group, and a goal that

11   can be achieved within the confines of the

12   ARRA,      or        the        American        Recovery          and

13   Reinvestment Act.

14                   We believe that such an effort is

15   achievable           using          currently           available

16   electronic      health      records.          However,       to     be

17   successful,         such    an     effort       will    be     best

18   undertaken       by        an     established          group        of

19   researchers with a cooperative infrastructure

20   who care for an ethnically and geographically

21   diverse population.                Specifically, the MFMU

22   would be interested in potentially cooperating

23   or developing and help guiding this effort.

24                   To    be        successful,      this     project
                                                                      117


1    must entail three separate functions.                          First

2    of all, there needs to be development of a

3    common     language,         so       that    data         can    be

4    meaningfully           and          comparably           combined.

5    Secondly, there needs to be an ongoing method

6    and data repository that is developed.                           And,

7    most importantly, that there be research done

8    to   validate      and       ensure     both       quality       and

9    accuracy of the data that is obtained.

10                And, finally, there needs to be a

11   mechanism    that      is      made    available         to    allow

12   researchers to have access to this data both

13   internal and external to this organization.

14                In        summary,         we        believe        that

15   perinatal    care       is     --     can    be    meaningfully

16   impacted    by    the     development         of     a    maternal

17   child data repository.                 We believe that by

18   using    existing      research        infrastructure            that

19   exists within this country that this is an

20   achievable goal.

21                We ask you to support an effort to

22   develop    such    a     data       repository       to    improve

23   outcomes,     as       Vermont         Oxford        and       other

24   registries    have       had    for    the    care        of   their
                                                                 118


1    patients.

2                    Thank you.

3                    DR. CONWAY:         Thank you very much.

4                    Dr. Lerner?

5                    DR. LERNER:         I am Jeff Lerner.         I

6    am the President and CEO of ECRI Institute,

7    also known a E-C-R-I.                    My proposal to you

8    today    is    to     consider      using     your    financial

9    resources to fund a national patient library

10   of evidence-based information.

11                   I     have      submitted      a     1,500-word

12   document,       and,      if    I   am    successful     today,

13   perhaps I can intrigue you enough to read that

14   document and look at some of the work products

15   I will describe briefly.

16                   My organization is known to many

17   of you, since we have been doing evidence-

18   based medicine, since the late 1960s.                    We are

19   nonprofit.          We are an evidence-based practice

20   center.        We have conflict of interest rules

21   for      our        300    employees,         that       include

22   examination of IRS returns to ensure there are

23   no investments in pharmaceutical or medical

24   device    companies,           since     we   evaluate    their
                                                             119


1    products.

2                  My proposal is to establish this

3    entity   as   a   trusted     central        resource    for

4    patients and their families, so that they can

5    work with their clinicians to figure out what

6    works    best,       especially       when     they     have

7    particularly troubling illnesses.

8                  I believe this would also make the

9    advantages of comparative effectiveness more

10   understandable       and   usable     for    the   public.

11   There is obviously a lot of feathers that are

12   ruffled by comparative effectiveness, and part

13   of the task I think is to convince the public

14   that science really is their friend, it is

15   working in their interest.            It is not just a

16   subterfuge for cost control, as important as

17   cost control is.

18                 Now,    a    national    patient     library

19   would    gather       existing      publicly-available

20   public and private patient information that

21   meets a standard of being evidence-based.                It

22   would also look at the new studies that you

23   are funding through AHRQ and NIH, as well as

24   other elements of the ARRA.           And this would be
                                                                   120


1    translated        information.             It    is    actually

2    professional information that gets translated

3    for patients.

4                  But     I     also        want     to    emphasize

5    creating    de     novo   information           that   from   the

6    get-go   is   designed        for       patients,      evidence-

7    based information.

8                  Now,     this      type      of    entity     would

9    also     bring        together           researchers          from

10   behavioral         economics,           cognitive       science

11   disciplines, communications, so that we would

12   understand how to use the information.                        This

13   would be helpful, particularly as we move into

14   things     like    electronic           health    records      and

15   PHRs.    We have to figure out how to use it.

16                 The examples, which you can look

17   at later, that lead me to this conclusion were

18   our experiences with bone marrow transplant in

19   the 1990s when no one trusted our findings

20   from professional studies, because they said

21   it didn't work and it was killing more people

22   than standard chemotherapy.

23                 No      one        had      an     interest      in

24   promoting     that.         So     we    went     directly     to
                                                                           121


1    patients through, for example, the National

2    Breast    Cancer        Coalition     and         others,       who    we

3    brought     in    to     create      a       patient       reference

4    guide, which we published, and which is free

5    on the web.       That was translated information.

6                     More    recently,           we    have       produced

7    guides on bulimia, but that was written by

8    coming up with the key research questions from

9    consulting patients and their families as well

10   as   researchers           and       then          building           the

11   information.            And    the       idea       is     to    bring

12   patients    together          with   their         physicians          to

13   make decisions.           It is not prescriptive.                      We

14   know that people have different risk profiles.

15                    I see I am out of time, so I am

16   going to have to wait for the --

17                    DR.     CONWAY:             Thank        you.          I

18   appreciate it.

19                    Dr. Siguel?

20                    DR.    SIGUEL:          I    agree       that    CMOs

21   used to evaluate outcomes and costs in the

22   amount     of    HMOs.         Beware        that        CE     can    be

23   counterproductive.            Powerful incentives create

24   clean-cut        data     that       appears          to        include
                                                            122


1    selected biomarkers.         Undesirable biomarkers

2    are   not   measured.         Some       best      evidence

3    encourages profitable treatments since there

4    are simpler ones.

5                Paid for performance doctors would

6    provide patients with best evidence drugs.              No

7    doctor gets extra performance pay for getting

8    people to lose weight instead of taking drugs.

9                You should distinguish between the

10   optimum medicine versus optimum health care

11   delivery.   For example, for chronic conditions

12   that represent the largest chunk of cost in

13   the U.S., we already know the best treatments.

14   For diabetes type 2, for example, the best

15   treatment   is   weight      loss,       healthy    meals,

16   exercise.   Evidence that drugs prevent short-

17   term complications and improve biomarkers is

18   mostly irrelevant.

19               Three    years    ago    I    proposed    that

20   policymakers misunderstood biochemistry.              Low-

21   fat diets were a mistake.           A diet low in fat

22   implemented as one high in carbohydrates was

23   biochemically equivalent to a high saturated

24   fat diet.   I found that the major culprits in
                                                                    123


1    cardiovascular disease were high calories, not

2    enough essential fats, and too many trans.

3                     So    you     can    tell    people    to    lose

4    weight,        eat    healthy        foods,    avoid    smoking,

5    exercise, and more.             Use what I call my secret

6    rope.      You put it around your waist, and you

7    measure it.           If you see that after a time it

8    goes up, then you move to my new treatment.

9    It    is   a    secret      treatment        that   consists    of

10   using duct tape and you put it around your

11   mouth like this --

12                    (Laughter.)

13                    -- for the duration of the meals.

14   Extensive            research        has   shown,      with    100

15   percent certainty, that people who cut caloric

16   intake, they lose weight, no matter what their

17   metabolism is like.

18                    I     have     my    insurance      plan      that

19   covers 100 percent.             It covers 100 percent of

20   the    payment        for     the    rope,    the   duct      tape,

21   penicillin, bandaids, all for $5 per month.

22                    (Laughter.)

23                    Everything else has a 110 percent

24   co-payment.
                                                              124


1                 (Laughter.)

2                 To    level      the   playing     field    and

3    improve education, because research shows that

4    better education improves health outcomes and

5    lowers    costs.        Properly    implemented,        on    a

6    statistical basis, research has shown that my

7    plan increases life expectancy more than 10

8    years,    beating       any   other     treatment.            I

9    challenge you to prove otherwise, and I urge

10   you to use it as the gold standard to compare

11   all other CE studies.

12                I     also       suggest    you      allocate

13   $300 million       to    create     better      models       of

14   disease          diagnosis,         treatment,           and

15   consequences.             Without       them,      CE        is

16   meaningless,      because     clinical    trials    cannot

17   evaluate what they cannot measure.                And they

18   cannot measure what is going to happen in the

19   future, particularly when currently mostly we

20   measure biomarkers that prove what a special

21   interest needs to profit.

22                I would use this $300 million to

23   create predictive models with high R-squared,

24   a task dutifully avoided by researchers who
                                                          125


1    rarely publish R-squareds now, perhaps because

2    they are too low.      My models should integrate

3    medical     and   behavioral    data    to     accurately

4    predict changes in morbidity and mortality.

5                  CE does not exist in a vacuum of

6    cause,    behavior,    environment,      and     accurate

7    models.     Drastic assumptions are implicit in

8    clinical trials.      Beware of the physicist who

9    seeks to predict horse races by assuming the

10   horses are both running on a field, a uniform

11   surface with constant friction.          This is what

12   happens when your models are incorrect.

13                 Thank you for your time.

14                 DR. CONWAY:      Thank you, sir.

15                 Ms. Turner?

16                 MS. TURNER:      Thank you very much.

17   I name is Grace-Marie Turner.           I am President

18   of   the      Galen     Institute,       a       research

19   organization       devoted      to     health     policy

20   research.     And I am also a former member of

21   the National Advisory Council of the Agency

22   for Healthcare Research and Quality, where I

23   have the chance and the opportunity to work

24   with Dr. Clancy.      And I appreciate her and all
                                                                    126


1    of your important work on this topic, this

2    crucially important issue.

3                    It is widely agreed that doctors

4    and patients need improved information about

5    medicines       and     treatments      to   make       the    best

6    decisions about health care and to eliminate

7    much of the waste that we hear about in our

8    health     care       system.        However,       many        are

9    concerned about how this new federal program

10   or     these     new     federal        programs        will     be

11   structured       and     the     consequences       of        those

12   programs.

13                   As we have heard today, one of the

14   large concerns is the -- addressing the unique

15   medical needs of individual patients, so that

16   they     are    fully     considered.            Experts       from

17   Europe         and      Canada,         where      comparative

18   effectiveness         systems     are    established,          warn

19   that    those     who    fall    outside     the    mainstream

20   have a much more difficult time receiving the

21   care that they need.

22                   As      Dr.     Cuddeback        said     in     an

23   earlier        panel,         comparative        effectiveness

24   research       must     support     both        treatment       and
                                                                   127


1    collaboration of those with rare diseases and

2    multiple          comorbidities.                  Comparative

3    effectiveness also has the risk of stifling

4    innovation.          If    allowed       to     dictate       the

5    preferences        of     medicine,         physicians        are

6    concerned         that     centralized            comparative

7    effectiveness       decision         making   could     replace

8    their experience, wisdom, and knowledge with

9    bureaucracies that reduce their decisions to

10   formulas.

11                 It is also important that we not

12   interfere with the progress of innovation in

13   our health sector.             Faced with another major

14   bureaucratic        hurdle      to      introducing       their

15   products to markets, medical companies would

16   be less likely to pursue research on new and

17   potentially        life-saving         drugs,         biologics,

18   medical devices, and surgeries.

19                 A     new    study       from     the    Institut

20   Économique     Molinari         in     France    says     that,

21   despite the best of intentions, the inevitable

22   consequence        of     comparative           effectiveness

23   research     can    be    to    push     up     the    cost    of

24   innovation    substantially           and     undervalue      its
                                                                    128


1    benefits, reducing the number of new products

2    and making certain projects unprofitable.

3                      Many    also      hope    that     comparative

4    effectiveness research could be used to lower

5    health spending.          Professor Michael Schlander,

6    who is a well-respected physician, researcher,

7    and economist in Europe, found that decisions

8    by     the   National         Institute       for    Health     and

9    Clinical Excellence in the UK have actually

10   led to higher spending for the NIH, not the

11   savings that have been expected.

12                     He      also         says         that      those

13   experienced with CER abroad say it is almost

14   impossible to integrate clinical findings and

15   cost    estimates,        because       they    use    different

16   methods of evaluation.                 As a result, he said

17   many subjective decisions are made, and what

18   many believe to be scientific processes.

19                     I see my time is up.                My primary

20   recommendation           is     that    the     infrastructure

21   created      by    this       panel,    and    throughout      the

22   Federal Government, is going to establish how

23   this     information           is    used      in    perpetuity.

24   Because we now are in an information age, we
                                                              129


1    have an opportunity to rely on development of

2    information that provides an opportunity for

3    collaboration --

4                  DR. CONWAY:         Thank you very much.

5                  Dr. Selker?

6                  DR. SELKER:           I'm Harry Selker, a

7    general internist and researcher and Dean of

8    Clinical and Translational Science at Tufts

9    University,        representing       the     Society     for

10   General Internal Medicine, an organization of

11   academic      general        internists       focused     on

12   research, education, and primary care, with a

13   long     history       in   comparative     effectiveness

14   research.     We are delighted to get a chance to

15   provide this statement to the Council.

16                 Unless compromised by poor quality

17   or conflicts of interest by stakeholders, CER

18   should     have    a    direct,     positive    impact     on

19   health.     Also, for our nation, even if total

20   costs of health care do not fall, CER should

21   have a positive impact on cost effectiveness.

22   We would be spending health care dollars more

23   wisely.

24                 However,        for     those     who      sell
                                                                   130


1    treatments, the consequences are more mixed,

2    and   this    has    led       to    a    blurring     of     the

3    processes of the conduct of science and the

4    conduct of health insurance coverage.                       There

5    are important issues that need to be addressed

6    to ensure the best possible impact of both

7    health care delivery and health.

8                  I   would     like         to    highlight     two.

9    First, comparative effectiveness research is

10   research     intended      to       affect     treatments      of

11   patients, of people.                Thus, like biomedical

12   research,    it     deserves        to    be    done   at     the

13   highest    standards      of    science        and   free    from

14   conflicts of interest by stakeholders.                      Thus,

15   it should be done at a science agency, not at

16   a new hybrid entity that would require a new

17   science infrastructure, that would involve in

18   its governance those who have a direct stake

19   in its results.

20                 Public input to research agenda is

21   a social good and should be sought.                         It is

22   very reasonable that agencies doing CER and

23   health care research should have high-level

24   public-private advisory boards.                   However, it
                                                                    131


1    must not be a governing board, which would

2    constitute an avenue for conflict of interest

3    that scientists, clinicians, policymakers, and

4    the      public       would         and         should        find

5    objectionable.

6                   The     AHRQ       has     the       most     broad

7    experience     and    expertise          in   CER    and     could

8    continue as a lead agency for CER.                         The NIH

9    also has a very important role to play, and

10   both are likely to benefit from collaboration

11   with the FDA, CDC, and other federal agencies.

12                  For     example,           AHRQ       could      be

13   responsible          for         research         looking       at

14   effectiveness,        harm,       and     safety,     that     use

15   analyses      of    current       evidence,        health     care

16   databases,     and    health       care       delivery.        NIH

17   could    be   responsible         for     large,     randomized

18   comparative        effectiveness          trials     needed     to

19   accurately assess the benefits of treatment.

20                  Links among the involved agencies

21   will be important as reflected by the role of

22   this Council.        This link might be facilitated

23   by      involving          the      NIH        clinical        and

24   translational         science           awards,      which      is
                                                              132


1    included in many of these institutions already

2    AHRQ CER centers, and would be an excellent

3    link from AHRQ to NIH, and a portal to its

4    institutes and centers, as well as to other

5    agencies.

6                  Second, coverage decisions should

7    not be the purview of the research agencies

8    doing CER.     Those decisions should be made by

9    CMS and other payers, as they are now and

10   presumably with changes that will come with

11   health care reform.          Consideration of payment

12   decisions      regarding         cost,     equity,       and

13   compassionate care, among many issues, should

14   be overseen by agencies under long-established

15   procedures.

16                 We believe it's an excellent sign

17   that the ARRA recognize the importance of CER,

18   and that its natural home is in the science

19   agencies, namely AHRQ in conjunction with NIH,

20   where   peer       review   processes      and    research

21   infrastructure       are    in   place    to    ensure   the

22   highest quality science.

23                 We     encourage      the        Coordinating

24   Council to allocate ARRA funds for CER in a
                                                                   133


1    way that preserves the conduct of CER at AHRQ,

2    NIH,     and      other     extant          federal     science

3    agencies, and it serves as a model that will

4    serve      future         CER       activities,         thereby

5    maximizing     the       important      impact    of    CER    on

6    health care and on the nation's health.

7                   DR. CONWAY:         Thank you very much.

8                   We will now move to the phone.                  Is

9    Dr. Mark Pilley on the phone?

10                  DR. PILLEY:           Good morning.            Yes.

11   I'm     Mark   Pilley,      Medical         Director    for    RS

12   Medical, a pain and function solutions company

13   and medical equipment manufacturer located in

14   Vancouver,        Washington.           I    appreciate,       on

15   behalf    of   the       company,      the    opportunity      to

16   address the Federal Coordinating Council for

17   Comparative Effectiveness Research.

18                  We are committed and interested in

19   demonstrating the effectiveness of our pain

20   management         and          functional        improvement

21   solutions         with     comparative          effectiveness

22   criteria, seeking reliable evidence and trends

23   that support our interest in the reduction of

24   opiates     and    the     use    of    NSAIDs    and    their
                                                                    134


1    sequelae in diverse populations.

2                   We are embarking on a multi-center

3    research involving the sickest sick of home

4    care and hospice patients in concert with the

5    American      Society      for    Home     Care    Physicians,

6    Janus, and AAFP, a national research network,

7    consistent         with     the      medical        home       and

8    independence at home models.

9                   We     believe         that        applying        a

10   systematic approach that blends subjective and

11   objective       probabilities             with     comparative

12   research      criteria      and     intermittent      outcomes

13   will be most attractive in demonstrating the

14   effectiveness of our products, which require

15   evidentiary support to ensure wider adoption

16   and payment.

17                  Good       science    blended       with    real-

18   world   observation         and     experience      is     a    key

19   note.         RS     Medical        has     been     providing

20   electrotherapeutic          products       that     have       been

21   beneficial for thousands of patients over the

22   last    two    decades       with     limited       supportive

23   evidence.      Electrotherapy continues to be used

24   at all levels of health care services, because
                                                                             135


1    it's safe, it is effective for many patients,

2    and     does       reduce          use     of       the     need        for

3    pharmacotherapy and reduced costs.

4                       We are now committed to validate

5    what     we        know       empirically.                Comparative

6    effectiveness           can    set       the    tone      for      us    to

7    participate             as         vanguards          rather        than

8    naysayers.          This will provide an opportunity

9    to     collect      observation           outcomes          data    real

10   time, providing needed scientific research and

11   information                   regarding                   therapeutic

12   effectiveness and cost effectiveness.

13                      We   also        embrace         the   expectation

14   that this helps to level the playing field and

15   potentially               accelerate                adoption            and

16   implementation of more useful products being

17   accessible to patients.                  The twentieth century

18   double-blinded            evidentiary           pedigree      we    have

19   savored       is    ill-suited           for    a    wide    range       of

20   clinical applications and to the complexity of

21   the clinical forum.

22                      Time      and    financial         barriers      have

23   further exacerbated and created difficulties

24   in this area.             The ability to scientifically
                                                                    136


1    validate     and        demonstrate          the     value     and

2    effectiveness we have seen in our patients and

3    those whom we serve needs to be captured.

4                  We        believe        in      the        faithful

5    expression    through         the     data    and       experience

6    means fear nothing from the finest arguments.

7    We       invite        this      research          in      medical

8    epistemological          breakthrough.               Comparative

9    effectiveness can help to set the tone for

10   synergy,     appreciation,             and     expansion        of

11   knowledge, creating informed vanguards on the

12   forefront of health care decision-making and

13   research.

14                 Essential to the delivery of safe

15   and effective medical care is the foundation

16   of   evidence           rooted       both     in        scientific

17   methodology             and         established           through

18   observation            and       real-life           experience.

19   Applying     a         methodology       that        is    openly

20   receptive, applying skills and due diligence

21   of   observational            effectiveness,            beneficial

22   modalities        of     intervention         and        treatment

23   become     available          and     accessible          to   all

24   patients despite payment systems.
                                                               137


1                   We    are    committed    to     comparative

2    effectiveness research to establish what we

3    know to be effective.

4                   Thank you.

5                   DR. CONWAY:      Thank you very much.

6                   Is    Dr.    Ronald      Stewart      on   the

7    phone?

8                   DR. STEWART:      Yes.     My name is Dr.

9    Ronnie Stewart.       I'm a professor of surgery at

10   the University of Texas Health Science Center

11   at San Antonio, and I'm a trauma surgeon in

12   San Antonio, Texas.

13                  I currently serve as the Chairman

14   of the Board of the National Trauma Institute.

15   We    respectfully         request    that     the   Council

16   recommend             substantial               comparative

17   effectiveness       research     funding       for   trauma.

18   Trauma is one of the most lethal and costly

19   health care conditions affecting Americans of

20   all   ages,    yet    today     any    federal       research

21   funding     has     been    sporadic     and     inadequate

22   relative      to    the    magnitude     of    the    health

23   problem.

24                  Trauma, as I think most of you are
                                                                      138


1    aware, is any serious injury resulting from

2    intentional       or    unintentional      violence.              As

3    such, it includes fractures, head injuries,

4    burns, hemorrhage, or secondary complications,

5    such as shock, respiratory failure, infection,

6    and    post-traumatic         stress    disorder,          and    of

7    course,       ultimately      death.       Trauma         is     the

8    leading cause of death for young people in the

9    United States from the ages of 1 to 44, and

10   it's    the     fifth    leading    cause       of    death       in

11   Americans of all ages.

12                   Over     160,000    Americans         die      from

13   injury each year, and trauma accounts for 37

14   million emergency department visits and 2.6

15   million hospital admissions across the nation.

16   It      affects        both   civilians        and        military

17   personnel, and according to the CDC, injury

18   accounts for 30 percent of life-years lost in

19   the United States, equal to the combined life-

20   years    lost    from     cancer,      heart    disease,         and

21   HIV.

22                   The     economic       burden        of     trauma

23   reaches    about       $400   billion     a     year.          This

24   includes      both      health   care     costs       and      lost
                                                                           139


1    productivity.          The past 50 years has seen the

2    development       of     sophisticated          trauma          centers

3    and    regional        trauma        systems.             Trauma       is

4    probably the most regionalized of any major

5    health problem.          However, there is very little

6    high      quality        translational               or        clinical

7    research addressing comparative efficacy.

8                     Those    care        providers           --     that's

9    trauma       surgeons,                emergency                medicine

10   physicians,                orthopedic                      surgeons,

11   neurosurgeons, and public health practitioners

12   -- realize that many treatments provided are

13   not    evidence-based,           and       we   need           them    to

14   determine        the     best    possible        treatment             in

15   patients    where        there's       often     an       incredibly

16   short amount of time to make the decision.

17                    The purpose of the National Trauma

18   Institute is to create awareness of the burden

19   of trauma on society, and to raise funds for a

20   variety     --    from     a     variety        of     public         and

21   private sources for trauma-related research.

22   The National Trauma Institute represents the

23   national     community          of    trauma         providers         in

24   calling     for     funding          and   infrastructure              to
                                                               140


1    address the -- what we view as the obvious and

2    urgent medical need.

3                  The     problem       is       significantly

4    underfunded relative to the magnitude of the

5    public   health     problem,    and     we   believe      that

6    trauma     fits    extraordinarily        well     into    the

7    draft definition of comparative effectiveness

8    research     and    meets    all    the      criteria      for

9    inclusion.

10                 This concludes my remarks, and I

11   thank you for the opportunity to present.

12                 DR. CONWAY:      Thank you.

13                 We'll now open it up to questions

14   from the Council.

15                 DR. CLANCY:       I'll ask a question.

16   So Jeff, I'd have to say the notion of a

17   national      patient       library       sounds        pretty

18   intriguing.        Does the UK NICE have something

19   like this, or am I making that up?

20                 DR. LERNER:       No, you're not making

21   it up.     They do have a fair amount of patient

22   information.        I think, Carolyn, that the big

23   distinction,       though,     would     be,     you    know,

24   really     centralizing      this      and     making     this
                                                                        141


1    somebody's first job, you know, so that it's

2    not something that we -- you know, we do in

3    addition to our professional studies.

4                  And I don't think anyone has that,

5    so    you   know,      it's       an    issue       of     mass    and

6    bringing    together         both       public       and       private

7    information,       you      know,      to     create       something

8    that really is trustable as large scale.

9                  DR.          CLANCY:           How         would     you

10   distinguish      it,       say,       from    healthfinder,         or

11   MedlinePlus, or other resources like that?

12                 DR. LERNER:               Sure.        It's a good

13   question.        A     lot    of       that     information         is

14   essentially        either         repurposed         professional

15   information,         and     sometimes         it's        not    even

16   repurposed.          It's got essentially guides to

17   how to use the professional literature.                            But

18   that's a far cry from actually being usable

19   for    people.             After       all,        not     just    the

20   individuals,       but      you       know,    there       are    many

21   professionals who really, you know, don't have

22   a good grasp of statistics and science and so

23   on.

24                 So      it's        a    question           of     making
                                                               142


1    things, not just usable, but also embedding

2    this in the technologies we're now using to

3    reach the public.           And it's very different.

4    You know, you can take a very sophisticated

5    person and say, okay.          Well, they're not going

6    to be fooled by a certain set of statistics.

7    But then you look at the results of behavioral

8    economics, and you know, they sure can be.

9    And I think we need a kind of disinterested

10   body that would sort this through for people.

11                  Now I do want to make one point

12   about    it     that       I   --     that    could        be

13   misinterpreted.        You know, you look at a lot

14   of   data,    and   it's    designed,    let's     say,    by

15   manufacturers to sell a product.              That's not

16   wrong.       That's what they're supposed to do.

17   But where's the counterbalance for the public?

18   You know, who undoes that and gives somebody

19   something else to look at it so that they can

20   make a decision?         In other words, it has to be

21   purpose-built for the use by consumers with

22   their physicians.

23                  DR. DELANY:         Well I mean, I want

24   to   follow    up   on     that,    because   in   itself,
                                                                 143


1    you're actually talking about a great deal of

2    comparative effectiveness research needs to be

3    done just to design that system, because we

4    need    to    be   looking    at     --    I    mean,    you're

5    talking about bringing in cognitive behavior.

6                   You're      talking         about       bringing

7    behavioral economics, which I'm still trying

8    to figure out what that actually means when I

9    read the literature.               But I think it means

10   people just don't behave rationally, period,

11   according to economics rules.

12                  This   is     the    --    if,    in   all   good

13   world, people would follow a certain set of

14   rules,   then      you'd   come      out       with   the   same

15   thing.        And I think that's fine.                  I think

16   actually looking at the fact that people --

17   and    this    gets   into    some       of     the   questions

18   about, you know, patient preferences, often

19   patients will choose something over another

20   that has nothing to do with the best outcome.

21                  So I think that's good, but I --

22   how would you -- I mean, I'm kind of intrigued

23   by it myself, but I'm wondering, how do we get

24   to this?       Is this -- it seems to me it's got
                                                                            144


1    to be an iterative system, but there has to be

2    a foundational set of research studies before

3    we go into something like this, or we're going

4    to set up a structure that, in the endpoint,

5    is going to cost more to continue to figure

6    out what the structure should look like.

7                        DR. LERNER:             Okay.        That's a good

8    point.     You know, I don't think you, you know,

9    build a library and then, you know, see what

10   happens.        And by library, I meant a modern

11   library.            But        what    --    we     have     a   lot    of

12   information              out     now        that     --      but     it's

13   disparate.           No one pulls it together.                         You

14   could     start          the     way     you       would    start      any

15   project        --        with,    you       know,     a     significant

16   planning       effort          that    looked       at,     well,    what

17   kind of -- what is out there?                            Where are the

18   gaps?    Would we be able to fill them?

19                       Now    to     some      extent,        there's     not

20   going     to        be    an     answer        to    that     question,

21   because we don't know what we don't know.                              But

22   we   don't          even       have,        you     know,    a     really

23   significant effort in this regard.                               We have

24   pockets    of        it.         We    have       very     well-meaning
                                                                 145


1    people.     It's not brought together.

2                  And you know, behavioral economics

3    is I think a very interesting part of that.

4    You know, it's interesting because -- yes, it

5    may show that people are irrational, but it

6    may not.      You know, you can look at nuclear

7    physics     and     say,   well,        because    we     don't

8    understand quantum mechanics, it's irrational.

9    No, it's really that we just don't understand

10   it yet.

11                 You know, so, you know, it's still

12   also a relatively new field.                   But the point

13   is, how are you ever going to get from where

14   we are now to something with some force?                    And

15   I   think    that     we   owe     it     to    the     public,

16   especially if we're going to charge them more

17   money.    We might as well give them something.

18                 DR. KILPATRICK:           Like Dr. Carr and

19   Dr. Stewart, both of you were talking about

20   areas where people are getting what I would

21   perhaps call unplanned medical care.                    And I'm

22   wondering, as we take a look at comparative

23   effectiveness research, what you would offer

24   as to how we do that medical literacy and
                                                                 146


1    involve the patients in the process to look at

2    the outcomes, as you so clearly stated, that

3    we don't have a lot of evidence-based data at

4    this point.

5                  DR. STEWART:         This is Ron Stewart.

6                  DR. CARR:        Dr. Stewart, we can't

7    hear   you   if   you're      speaking.        I    hear    him

8    speaking in the microphone.

9                  Unplanned        medical      care       --     I

10   appreciate the euphemism very much.                  I think

11   that there are -- I guess I have two answers,

12   and the first is that the way that we think

13   about planning for emergency care, it needs to

14   be from the population perspective, not from

15   the    hospital-based      individual          perspective.

16   And I think there are good examples of this.

17   I'll     celebrate      Dr.        Stewart's        sort     of

18   recognition of the trauma system.                  They think

19   in terms of how you get someone to the right

20   care promptly.

21                 There have been gaps in knowledge.

22   We don't know much about EMS systems.                        We

23   don't have great electronic medical records

24   for    EMS   systems.         We    don't   know      how    to
                                                               147


1    incorporate        that     information          into     our

2    learning.

3                  DR. CARR:          So there are gaps in

4    the EMS systems and in the EMS knowledge base,

5    IT   solutions,      involving        emergency     medical

6    services,    and    helping      us   to     understand   how

7    what they do, or how long they take, or where

8    they choose to go, and if they choose to take

9    a helicopter versus a ground ambulance, what

10   they choose to do on the way, how that impacts

11   care.

12                 Those are giant questions that we

13   don't have the answers to, but the perspective

14   is   from     the    population,           not    from    the

15   individual.    Comparing outcomes for a cervical

16   spine injury at one facility versus another

17   facility    falls    very     short     of    understanding

18   what happened from the moment of injury.

19                 It's very much akin to the door-

20   to-balloon initiative.           It's very much akin to

21   -- in cardiology.         It's very much akin to time

22   is brain, time is muscle.             All of these public

23   health     campaigns      that    have       happened     have

24   happened because we understand that time is
                                                                   148


1    medicine.

2                  So I guess I would say that that

3    is the one piece of it is planning from the

4    population perspective.              And the other piece

5    is understanding that what we're talking about

6    are     undifferentiated         complaints.                 We're

7    talking    about     people    who     don't     know    what's

8    wrong with them, and that is not the emphasis

9    of most of the medical literature, at least

10   that I read.

11                 The undifferentiated complaint is

12   someone with -- fever and abdominal pain is a

13   perfect example.        Where do you go from there?

14   Because the answer is you go to CAT scan from

15   there.     And then where do you go from there

16   three     weeks     later     when    they       are    in     the

17   emergency department, or 24 hours later when

18   they are in a different hospital's emergency

19   department?         You go to CAT scan again and

20   again.

21                 And     you      know,       the     unintended

22   consequences of these unplanned medical visits

23   are      substantial,         and      I     think           worth

24   investigating.
                                                                     149


1                    DR.    MILLMAN:        Dr.       Carr    and     Dr.

2    Stewart,      there     are     many       topics       that     are

3    possible in those sort of areas you talked

4    about.       Do you have any thoughts about how

5    priorities for CER study should be developed

6    in these areas?         Once you say some area like

7    this    is   important,        and    there      are     lots     of

8    possibilities, how do you narrow down what --

9    is it a process?         Is it an infrastructure that

10   we ought to be funding?

11                   DR. STEWART:         I think it should --

12   it's awkward here, so -- over the phone.                           I

13   think    that,    really,       a    good    starting          point

14   would be to look at the relative magnitude of

15   the public health problem to determine, you

16   know,    what    gets     the       main    priority.            The

17   National Trauma Institute, with really all of

18   its stakeholders, has taken a look at that.

19                   And if you look for -- from the

20   trauma point of view, dealing with hemorrhage,

21   particularly non-compressible trunk or torso

22   hemorrhage,       is    very        high    on      that       list,

23   resuscitation,          then         infection,          disaster

24   preparedness, burns.                So in many ways, the
                                                                 150


1    trauma community has made a priority list of

2    the problems.

3                     Traumatic     brain    injury      would    be

4    very    high     on   that   list,     as   well,       although

5    traumatic brain injury, as I think most of you

6    are aware, has gotten a fair amount of funding

7    over the past two years.

8                     DR. CARR:     I would add that, from

9    the     systems       standpoint,       I    think        shared

10   infrastructure is one of the things that you

11   said and that -- and I guess in my opinion,

12   not speaking for ACEP or SAM, is what I think

13   the     answer    is.        The   system    is     a    shared

14   infrastructure.

15                    Whether the person in need of care

16   needs    an    interventional        cardiologist,         needs

17   Dr. -- I'm sorry -- Stewart, a trauma surgeon,

18   needs     a    vascular       enterologist        or     stroke

19   doctor, what they really need is they need to

20   be in the right place at the right time, and

21   we need to figure out how to get them there

22   promptly.

23                    DR. CONWAY:       I would like to thank

24   panel three.          We're now going to move to the
                                                                         151


1    open public comment.                  I believe there's four

2    selected panelists.              Is that correct?

3                      So Mr. Anton Chaitkin -- apologize

4    if I said that wrong -- Dr. Alex Hannenberg,

5    Dr.     Jack      Gorman,       and       Ms.    Diana       Williams.

6    We've even got name tags.                  That's great.

7                      So we'll do the same format.                       It

8    will    be       three    minutes,         and       then    time   for

9    questions.         We'll start with Mr. Chaitkin.

10                     MR.     CHAITKIN:              Anton       Chaitkin.

11   I'm    a    historian          and    a    history       editor     for

12   Executive         Intelligence            Review.            President

13   Obama      has    put     in    place      a    reform       apparatus

14   reviving the euthanasia of Hitler Germany in

15   1939 that began the genocide there.

16                     The     apparatus         here       is     to    deny

17   medical care to elderly, chronically ill, and

18   poor people, and thus save, as the President

19   says,      $2-    to     $3    trillion         by    taking       lives

20   considered not worthy to be lived, as the Nazi

21   doctors said.

22                     Dr.     Ezekiel          Emanuel          and    other

23   avowed cost-cutters on this panel also lead a

24   propaganda               movement              for          euthanasia
                                                             152


1    headquartered at the Hastings Center, of which

2    Dr. Emanuel is a fellow.           They shape public

3    opinion and the medical profession to accept a

4    death culture, such as the Washington State

5    law passed in November to let physicians help

6    kill   patients    whose       medical    care     is    now

7    rapidly    being   withdrawn       in     the     universal

8    health disaster.

9                 Dr.       Emanuel's         movement        for

10   bioethics and euthanasia, and this Council's

11   purpose,    directly       continue       the      eugenics

12   movement that organized Hitler's killing of

13   patients, and then other costly and supposedly

14   unworthy    people.      Dr.     Emanuel        wrote   last

15   October 12th that a crisis, war, and financial

16   collapse would get the frightened public to

17   accept the program.

18                Hitler told Dr. Brandt, his -- in

19   1935 that the euthanasia program would have to

20   wait until the war began to get the public to

21   go along.     Dr. Emanuel wrote last year that

22   the    Hippocratic      oath     should     be      junked.

23   Doctors should no longer just serve the needs

24   of the patient.       Hoche and Binding, the German
                                                                            153


1    eugenicists, exactly said the same thing to

2    start the killing.

3                       You on the Council are drawing up

4    the procedures to -- list to be used to deny

5    care,    which         will   kill      millions           if    it    goes

6    ahead in the present world crash.                               You think

7    perhaps       the         backing         of        powerful           men,

8    financiers,               will          shield             you         from

9    accountability,            but     you     are        now        in    the

10   spotlight.

11                      Disband       this    Council       and        reverse

12   the whole course of this Nazi revival now.

13                      DR. CONWAY:          Thank you.

14                      We'll move to Dr. Hannenberg.

15                      DR.    HANNENBERG:               Good    afternoon.

16   Thank you very much for the chance to speak

17   with you.              My name is Dr. Alex Hannenberg.

18   I'm     the    President-elect                 of     the        American

19   Society       of       Anesthesiologists,             whose        43,000

20   members are responsible for about 25 million

21   anesthetics annually.

22                      I     applaud     the       Council           for    its

23   recognition, particularly of the importance of

24   infrastructure             investments          in         support      of
                                                                     154


1    comparative         effectiveness         research,       and    in

2    particular,         patient        registries.           They    do

3    indeed have the opportunity to be reused and

4    continually provide the basis for important

5    information.

6                       I'm also here to tell you that the

7    members       of    our     society       have    embraced      and

8    demonstrated with their own investments their

9    readiness and eagerness to participate in this

10   kind of work.              A consortium of 15 academic

11   anesthesia departments has somehow found a way

12   to     pool        data     from      disparate        electronic

13   anesthesia records into a registry, a clinical

14   database.

15                      Twelve       large      private       practice

16   anesthesia         groups       across     the    country       have

17   invested      their       own    dollars        and   efforts    in

18   similar       exercises,         as      have    most     of     our

19   subspecialty              organizations.                    These,

20   unfortunately, are relatively narrow in scope,

21   and by the very nature of being fragmented,

22   beg    for    a    national      integrative          effort,    not

23   only    among       the     anesthesia      data      aggregation

24   efforts,          but     across      specialties        and    the
                                                                      155


1    perioperative team.

2                    Such        an    interoperable           database

3    would    serve       multiple      purposes.            The   reason

4    these people have done it is because of their

5    desire         for         benchmarking           and         quality

6    improvement.           This in turn begets important

7    performance measures and forms the basis for,

8    if you'll forgive me, public reporting we can

9    believe in.

10                   We have set in place a mechanism

11   to      take         the     information           from         these

12   benchmarking reports and tie them to focused

13   educational      efforts         to     address    shortcomings

14   that    are    identified         there.        The     connection

15   with comparative effectiveness research goes

16   beyond that.

17                   We     hope      that,     as     we     give    our

18   members the tools for benchmarking and data

19   aggregation for these purposes, and get them

20   in the habit of doing it, that we can add to a

21   core data set for this purpose variable and

22   rotating       data        elements      tied      to     key    and

23   priority clinical research needs, especially

24   those     that        give       rise     to    evidence-based
                                                                             156


1    practice guidelines.

2                      Some     examples         of     the       kinds       of

3    things people are working on, the influence of

4    drug    choices         and     anesthetic         techniques            on

5    unplanned         admissions          to     the       hospital          or

6    unplanned admissions to the ICU, or length of

7    stay.        There       is    some    provocative               research

8    relating      anesthetic            technique           and       cancer

9    survival.          You        can   easily        imagine         how    an

10   anesthesia registry linked to a tumor registry

11   could    very       effectively            answer        those         very

12   important questions.

13                     The     Council           and        the        medical

14   community               recognizes               the             enormous

15   contributions and potential of projects like

16   STS and American College of Cardiology, and I

17   think the challenge for you today is to find

18   ways    to   disseminate            this     across          a    broader

19   range        of         medical        practices.                       And

20   anesthesiology           is    here    to    say       that       we    are

21   eager and ready to work with you toward that

22   goal.    These registries clearly are the gift

23   that keeps on giving.

24                     Thank you.
                                                                     157


1                     DR. CONWAY:       Thank you.

2                     Dr. Gorman?

3                     DR. GORMAN:         Thank you very much.

4    My     name      is    Jack     Gorman.         I'm    the     Chief

5    Scientific            Officer     for      Care        Management

6    Technologies,          a   subsidiary      of     Comprehensive

7    Neuroscience.           Care Management Technologies is

8    in the business of improving care for people

9    affected      by      mental    illness     and       the   medical

10   comorbidities           that     these     people           commonly

11   suffer with.            Our clients include Medicaid,

12   Medicare, and private insurance providers.

13                    Prior to coming to Care Management

14   Technologies, I spent my professional career

15   as an academic psychiatrist specializing in

16   research into the neurobiology and treatment

17   of a diverse group of psychiatric disorders.

18   As part of that work, I was involved in the

19   design and conduct of many of what we consider

20   to    be   the     gold    standard       for    assessing       the

21   efficacy      of       therapeutic       interventions,         the

22   randomized control trial.

23                    As you know, somewhat uniquely in

24   the    psychiatric          field,      randomized           control
                                                                       158


1    trials,      RCTs,        that    are     done   to    establish

2    treatment efficacy, especially for the purpose

3    of     FDA     approval,           are     usually         placebo-

4    controlled because of the lack of biomarkers

5    in our field to tell us whether a treatment

6    works        and     our         subsequent       reliance         on

7    subjective ratings.

8                       RCTs        established          the          first

9    generation          of     anti-depressant            and        anti-

10   psychotic medications in the 1950s and 1960s,

11   the second generation of these medications in

12   the '80s and 1990s, and have also given us

13   clear    evidence         that     many     forms     of    focused

14   psychotherapy             and       other        non-medication

15   treatments         are     highly        efficacious        in    the

16   treatment of psychiatric illness.

17                      But    along    the    way    we    have      also

18   learned that RCTs do not tell us very much

19   about how psychiatric treatments work in the

20   real world.              The majority of patients with

21   psychiatric          illness       have     multiple        medical

22   comorbidities            and     psychiatric      comorbidities

23   that      complicate             treatment       response          and

24   generally      exclude          them     from    enrolling        and
                                                                       159


1    participating in RCTs.

2                   The fact that the placebo response

3    rate is very high in many RCTs -- for example,

4    in     about     50        percent       of    trials      involving

5    currently marketed anti-depressant drugs, the

6    drug has failed to beat placebo.                       But this is

7    almost never taken into account when we design

8    behavioral health care strategies.

9                   Furthermore,              RCTs      have    told    us

10   very little about which treatments within any

11   given class are most effective.                         To fill in

12   these gaps, some attempts have been made to

13   launch    large        effectiveness             trials    that   are

14   more inclusive and directly compare treatments

15   within classes.

16                  Studies           such     as     the   large   NIMH-

17   sponsored      CATIE,           STAR*D     and    STEP-BD      trials

18   have     given        us     very     valuable         information.

19   These studies are expensive, however, and took

20   many      years            to       plan         and      implement.

21   Furthermore, there is evidence that they have

22   had very little influence on actual treatment

23   provision        to         patients           with     psychiatric

24   illness.
                                                                              160


1                    Currently, as we and others have

2    shown,    the       biggest    influence             on    what      drugs

3    psychiatrists              prescribe            is         from          the

4    pharmaceutical industry.                   Psychiatrists, for

5    unclear         reasons,          are           biased           against

6    prescribing           generic         drugs,               and           have

7    decreasingly                accepted                 evidence-based

8    psychotherapies.              Perhaps          because         of    this,

9    psychiatric         care    has     become       among         the       most

10   costly    and       controversial              forms      of     medical

11   intervention that we have today.

12                   A    complementary              and       increasingly

13   frequently          used      way         of      assessing              the

14   comparative           effectiveness                  of        existing

15   treatments is, as you know, the use of large

16   administrative databases.                  To be sure, these

17   have their limitations, because patients are

18   not   randomly        assigned       to    treatments               in    the

19   naturalistic           setting,                and         therefore,

20   assortment biases can occur.

21                   DR.        CONWAY:             Thank        you,          Dr.

22   Gorman.

23                   A quick break.                 I'm going to ask

24   for questions and comments.
                                                            161


1                   Ms. Zuckerman, if you can come up,

2    and I'm opening it up to the panel.               Yes, we

3    are still going to -- Ms. Williams, you are

4    still going to get to talk.            I'm going to ask

5    Ms. Zuckerman to come up and join you.

6                   We're going to take a quick stop

7    in the panel to open it up for comments or

8    questions, because we have a few folks who

9    have to potentially go.         The whole transcript

10   will be available, though, and all comments

11   will be shared with all Council members.

12                  DR. EMANUEL:      I apologize that I

13   have to go back to an important meeting.               I do

14   want to just clarify one thing about my own --

15   since my reputation has been besmirched here,

16   is I think I do have a very long record of

17   writing        against    the         legalization      of

18   euthanasia.

19                  So the association of me and that

20   seemed a little strange given -- I don't know

21   -- at least 30 years or 25 years of writing on

22   the topic, against the legalization.              So just

23   to   clarify    the   record    for    everyone   in    the

24   room.
                                                                     162


1                     Thank you.

2                     MR. CHAITKIN:        You stated that you

3    were opposed to assisted suicide, but that you

4    are   in   favor        of    the   withdrawal         of   medical

5    care, which accomplishes the same thing.                          So

6    you had this article?

7                     DR. CONWAY:          Sir, your statement

8    was read into the record.                It's not the time

9    for debate, but we appreciate your comments.

10   And we apologize for the break in the panel,

11   but we just wanted to have that break.

12                    So we're going to move on.                    We'll

13   go    to   Ms.         Williams     next,        and    then    Ms.

14   Zuckerman, and then we'll open it up to the

15   entire panel for questions and comments.                          I

16   apologize        for    catching     you      while     you    were

17   eating.

18                    (Laughter.)

19                    MS.     WILLIAMS:          Good       afternoon,

20   panel.       I    am     encouraged      by      the    panelists

21   before me who -- it appears that we are all on

22   the   same   page        in    wanting      to    maintain      and

23   deliver optimal health care for our patients.

24                    My name is Diana Williams, and I
                                                                     163


1    serve as the Secretary for the Foundation for

2    Environmental         --     Environmentally-Triggered

3    Illnesses, also known as FETI.

4                  The                 Foundation                     for

5    Environmentally-Triggered                   Illnesses            was

6    established      to    identify          and      fund     health

7    research    that      will       add     to     the      body    of

8    knowledge of environmental causes of diseases

9    and   to   promote         and    fund      the    evaluation,

10   testing,    and       diagnosis        of      patients         with

11   environmentally-triggered illnesses in a real-

12   world setting.

13                 FETI     is        requesting       funding         to

14   conduct    an     initial        three-year        comparative

15   effectiveness research project to thoroughly

16   evaluate patients in an environmental control

17   unit, ECU, under the direction of a medical

18   director experienced in this advanced method

19   of evaluation and treatment.

20                 The ECU will serve as a research

21   tool and assist in conducting, supporting, and

22   synthesizing        research        that       compares         the

23   clinical        outcomes,          effectiveness,               and

24   appropriateness        of        items,        services,        and
                                                                   164


1    procedures that are used to prevent, diagnose,

2    or treat illnesses.               Research using the ECU

3    can be used to encourage the use of clinical

4    registries, clinical data networks, and other

5    forms of electronic health data that can be

6    used    to     generate      or    obtain    outcomes         data

7    related           to          environmentally-triggered

8    illnesses.

9                    Such research can specifically be

10   used      to    address       the      needs         and      life

11   circumstances           of        individual          patients,

12   including      women,     children,     minorities,          thus

13   delivering more personal care by keeping down

14   the cost of the delivering health care, the

15   gold    standard       research      model     in     medicine,

16   which   can     clarify      many     issues    of     accurate

17   diagnosis and effective treatment in the ECU.

18                   Consistent with President Obama's

19   insistence on transparency and integrity in

20   government, there is no area more important

21   than    health     care       where     transparency           and

22   integrity are imperative.              We need objective,

23   accurate,       non-lobbied          data      and         outcome

24   information if we are to reverse the downward
                                                                      165


1    spiral    of    chronic        disease   and    its       enormous

2    financial burden on U.S. citizens personally

3    and financially.

4                    The        most       prevalent            chronic

5    diseases in America are lung disease, heart

6    disease, hypertension, mental illness, cancer,

7    diabetes, and stroke is on the rise.                             Yet

8    billions of dollars are spent, often wasted,

9    by     trying        to    suppress      symptoms          without

10   identifying the cause of the illness.                     We must

11   stop    this    trend      by    identifying        the   causes,

12   eliminating them, and provide treatments that

13   restore patients' health rather than merely

14   medicate the symptoms.

15                   An    environmental       control         unit    is

16   the research tool that can accomplish our best

17   medical goals with complex medical conditions.

18   In its simplest form, the ECU is a set of

19   patient care rooms, either on a wing of a

20   medical facility or located in a freestanding

21   building.

22                   And       in    an   effort    to    keep    this

23   short, I would like to say that we have an

24   economically         unsupportable       system      of    health
                                                                166


1    care delivery that often fails to meet the

2    needs of people with disabilities, culturally

3    diverse populations, and those with chronic

4    illnesses.

5                   Funding the ECU is needed by the

6    CRE, and will identify the cause of chronic

7    illnesses and enable treatment in real-world

8    settings.

9                   Thank you very much.

10                  DR. CONWAY:        Thank you.

11                  Ms. Zuckerman?

12                  DR.       ZUCKERMAN:       Thanks    for     the

13   opportunity         to     be   here.     I'm    Dr.      Diana

14   Zuckerman.           I'm    President    of   the   National

15   Research Center for Women and Families, which

16   is a nonprofit research center that translates

17   complicated scientific and medical information

18   into        usable       information      that      can      be

19   effectively used by patients, consumers, and

20   health professionals, as well as policymakers.

21                  My post-doctoral training at Yale

22   Medical School was in epidemiology and public

23   health, and prior to my current position, I

24   was    on     the    faculty      at    Vassar   and      Yale,
                                                                     167


1    directed research at Harvard, and I worked in

2    the   U.S.      Congress       and    the        Public      Health

3    Service for over a dozen years.                     I'm also a

4    fellow     at    the       University       of     Pennsylvania

5    Center for Bioethics.

6                    I've spent much of the last 15 or

7    20 years working on FDA issues, and that's

8    relevant to your task today, because I've seen

9    time and time again how new medical products

10   are   approved         by     the     FDA        despite      very

11   questionable       data      in     terms     of    safety      and

12   effectiveness.

13                   And    so    often,    as     you      all   know,

14   these new products cost a lot more than the

15   previously approved products, even though they

16   may be less effective and less safe.                      And yet

17   they drive up costs of our health care system,

18   because      there's         so      much        pressure       and

19   advertising and other way -- and for other

20   reasons, these products are so -- these new

21   products     are      so    often    taking      the    place    of

22   older products that may be, as I said, more

23   effective and safer.

24                   In fact, I just testified before
                                                          168


1    the FDA yesterday on three anti-psychotic --

2    atypical anti-psychotic medications which are

3    a perfect case in point.           There are 20 million

4    prescriptions a year for three anti-psychotic

5    medications that are supposed to be -- that

6    are     approved    only     for    schizophrenia    and

7    psychosis    related    to    bipolar    disorder,   but

8    that are being used for anxiety, depression,

9    ADHD, Alzheimer's patients who are a little

10   bit too feisty, and in other ways are driving

11   up health care costs but harming patients.

12                 So when I saw one of the questions

13   was, how can we deal with the differences in

14   consumers' views and pharmaceutical companies'

15   views       about      comparative        effectiveness

16   training, comparative effectiveness research,

17   my feeling is sometimes you can't, but that it

18   is your job to figure out how best we can make

19   sure that the research really is moving us in

20   a direction where patients and consumers are

21   using the most effective, safest medication,

22   and as well as cost effective medications.

23                 Thank you.

24                 DR.   CONWAY:          Thank   you,    Dr.
                                                               169


1    Zuckerman, and thank you, panel number four.

2                   Unfortunately, we are out of time,

3    so I'll turn it over to Neera Tanden.

4                   MS.   TANDEN:         I    want    to   thank

5    everyone for participating.              This is our last

6    listening session, and so I just wanted to say

7    both Secretary Sebelius and President Obama

8    are    committed     to   an    open     and     transparent

9    process.      And that is one of the reasons why

10   we     have    these      listening       sessions        with

11   stakeholders, which has very much informed the

12   thinking of the Council as we go forward.

13                  And I would say I appreciate that

14   there were some loaded charges, in this panel

15   in particular, and I think one thing that we

16   would respond -- and I know this is a panel

17   made    up    of   experts     and   thinkers,      and   the

18   Council is made up of real professionals who

19   are dedicated to ensuring that we are doing

20   this right and providing good guidance to the

21   Secretary on how these funds should be spent,

22   but that the proof will be in the pudding.

23                  You will see the report that the

24   -- that we are working on, and you will see,
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1    you know, the spend plan that the Secretary

2    puts forward is available to the public.

3                    And again, this is meant to be a

4    completely transparent process, and so those

5    people with concerns that rise to the level of

6    allegations, will see at the end of the day

7    what     this       Council     does,        and     what     the

8    government does with the funding allocated for

9    comparative effectiveness research.

10                   And we are committed to ensuring

11   the basic goal of this funding, which is to

12   ensure that we are using this money in a way

13   that    has   the     most    effect    on    improving       the

14   quality of care for our people, and ensuring

15   that we are empowering providers and patients

16   with the best information.

17                   And     so     thank         you     all      for

18   participating.           I     know     we've       learned     a

19   tremendous       amount       over      the        last     three

20   sessions, and that it has been critical that

21   we     get    the     stakeholder        involvement          and

22   learning in order for us to do a good job for

23   the Secretary and for Congress.

24                   Thank you.
                                                   171


1    (Whereupon, at 3:02 p.m., the proceedings in

2                the    foregoing      matter     were

3                concluded.)

 4
 5   Appended Note:
 6   The Hastings Center thanks the Department of
 7   Health and Human Services and the Federal
 8   Coordinating Council for Comparative
 9   Effectiveness for providing it with an
10   opportunity to clarify its mission in response to
11   remarks made at this listening session. The
12   Center is a nonpartisan, nonprofit research
13   organization dedicated to bioethics and public
14   policy since 1969. The Center does not advocate
15   for legislation, and contrary to remarks made at
16   this hearing, has not been, and is not now an
17   advocate for euthanasia.

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