THE UNITED STATES OF AMERICA
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
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FEDERAL COORDINATING COUNCIL FOR
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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,
COUNCIL MEMBERS PRESENT:
CECILIA RIVERA CASALE
DR. JAMES FASULES
DR. FREDERICK GROVER
DR. BETH KOSIAK
DR. TED BUCKLEY
DR. JOHN CUDDEBACK
DR. PATRICIA SALBER
DR. DENEEN VOJTA
DR. MARK ROBERTS
DR. BRENDAN CARR
DR. MATTHEW HOFFMAN
DR. JEFFREY LERNER
DR. EDUARDO SIGUEL
DR. MARK PILLEY
DR. RONALD STEWART
DR. HARRY SELKER
DR. ALEX HANNENBERG
DR. JACK GORMAN
DR. DIANA ZUCKERMAN
TABLE OF CONTENTS
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
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
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
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
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
1 will specifically make recommendations for the
2 $400 million allocated to the Office of the
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
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
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
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
23 DR. CONWAY: Hi. Welcome to the
24 listening session. So for today, as was
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
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
1 relatively short, non-extended run-on
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
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
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
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.
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
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
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.
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
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
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
1 receives the care they need and rightly
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
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
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
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
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
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
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
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
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
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
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
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
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
22 Thank you.
23 DR. CONWAY: Thank you very much.
24 MR. Juba?
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
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
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
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
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
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
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
20 In short, the two would provide
21 health care professionals and patients useful,
22 scientific evidence on what works best for
24 I am pleased to see that after 10
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
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
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
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
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
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,
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
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
1 year, $2.8 billion annually, and by the fifth
2 year that same investment would save $16
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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
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?
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
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
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
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
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
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
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
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
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.
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
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
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
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
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
21 Comparative effectiveness studies
22 should capture all relevant aspects of
23 diseases and their treatments using high
24 standards of evidence. Government policies
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
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'
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-
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
1 care data and patient outcome data across the
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
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
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
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
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
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
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
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
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
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.
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
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.
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
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
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
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
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
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
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
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
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.
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.
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
24 CER must develop the ability to
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
21 Thank you.
22 DR. CONWAY: Thank you very much.
23 Dr. Vojta?
24 DR. VOJTA: Good afternoon.
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
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.
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
22 United Health Care recently
23 launched the diabetes health plan, responding
24 to consumers' interest in a health plan option
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
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
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
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
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
1 culture of learning in the health care
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
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
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
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
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
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
1 research protocols. It isn't just simply
2 let's grab a bunch of people and do some
4 So I think your comment on that
5 from the patient perspective would be very
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 --
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
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
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
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,
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
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
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
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
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
23 I would be interested in your
24 thoughts how, you know, we can think as a
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,
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
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
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
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.
1 DR. EMANUEL: Dr. Buckley, I have
2 -- sorry. Usually, I am pretty loud, or loud
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
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
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.
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
1 a surgery versus drug evaluation in the study
3 DR. EMANUEL: What do you mean the
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.
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
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
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
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
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
2 DR. CONWAY: Thank --
3 DR. KUPERSMITH: Could I ask --
4 too late?
5 DR. CONWAY: Yes, go ahead.
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
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
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
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
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
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,
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
1 not been rigorously evaluated, and this
2 represents a major missed opportunity to
3 improve patient outcomes and to conserve
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
1 represent a major missed opportunity to
2 improve patient outcomes and to conserve
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
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.
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.
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
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
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
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
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
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
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
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
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 --
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.
23 Everything else has a 110 percent
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
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
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
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
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
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
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
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
24 However, for those who sell
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
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
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
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
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
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
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
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
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
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
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.
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
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
24 Trauma, as I think most of you are
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
22 The economic burden of trauma
23 reaches about $400 billion a year. This
24 includes both health care costs and lost
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
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
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
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
24 So it's a question of making
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,
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
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
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
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
1 incorporate that information into our
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
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
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
21 And you know, the unintended
22 consequences of these unplanned medical visits
23 are substantial, and I think worth
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
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
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
23 DR. CONWAY: I would like to thank
24 panel three. We're now going to move to the
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
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
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
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
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
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
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
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
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.
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
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
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
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.
23 A quick break. I'm going to ask
24 for questions and comments.
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
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
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
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
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
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
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
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
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
1 care delivery that often fails to meet the
2 needs of people with disabilities, culturally
3 diverse populations, and those with chronic
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
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,
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
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
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.
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,
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.
1 (Whereupon, at 3:02 p.m., the proceedings in
2 the foregoing matter were
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.