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Comparative Effectiveness Research Toolkit

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					                          1894 Preston White Drive, Reston, VA 20191-5433 Phone: 703.620.6390 Fax: 703.416.0904 Web: www.npcnow.org




         Comparative Effectiveness Research and Evidence-Based Medicine:
         An lnformational Series from the National Pharmaceutical Council

ln early 2009, Congress approved and President Obama signed into law the American Recovery and
Reinvestment Act (ARRA), an economic stimulus package that includes Sf .f billion for comparative
effectiveness research (CER). By approving those funds, lawmakers made it clear that CER will be an
integral part of health care reform.

Although the concept of CER is not new, it is important to establish a clear definition and understanding
of why it is so prominent today and the many related issues and initiatives under consideration in
Washington.

To facilitate this understanding, the National Pharmaceutical Council has developed a series of
informational pieces, which taken together provide an overview of CER. Each of the following items
outlines a specific aspect of   CER in a   short, easy-to-read format.

    a   Defining Evidence-Based Medicine a nd Comparative Effectiveness Resea rch
    a   A Brief History of Comparative Effectiveness Research and Evidence-Based Medicine
    a   NPC's Key Considerations on Comparative Effectiveness Research
    a   Legislative Proposals Regarding Comparative Effectiveness Research
    a   Comparison of Comparative Effectiveness Research Legislative Activities in the Context of NPC's
        CER Recommendations to lOM, FCCCER and AHRQ
        Additional Resources

We encourage you to share these informational pieces with your colleagues and other organizations
interested in the issue, as well as link to the pieces on NPC's website, where we will be providing
updates to the materials on an ongoing basis.

For additional copies of this information, please contact NPC at info@npcnow.ors or 703-620-6390.
                             1894 Preston Wh¡te Dr¡ve, Reston, VA 20191-5433 Phone: 703.620.6390 Fax: 703.476.0904 Web: www.npcnow.org




                            Defining Evidence-Based Medicine
                      and Comparative Effectiveness Research
lntroduction

Evidence-based medicine (EBM) is a systematic approach to clinical problem solving which allows the
integration of the best available research evidence with clinical expertise and patient values.l

Under this definition, EBM requires clinical expertise and use of the best evidence available, but must
also consider patient preferences, optimal patient outcomes, and the relative effects among competing
alternatives. Broadly considered, EBM includes the comparative effectiveness assessments of drugs,
treatments and devices, and the appropriate interpretation of evidence from these assessments to
support health benefit design and medical decision-making.

EBM is the foundation for Comparative Effectiveness Research (CER), which compares available
treatment options utilizing a range of research methods including randomized controlled trials,
observational studies, and systematic reviews, a structured assessment of evidence available from
multiple primary studies.

Another term that is frequently mentioned with CER and EBM is health technology assessment (HTA).
HTA is a rigorous process of appraisal that examines the effects and impact of a health care technology
or treatment. These assessments inform decision-makers as to the direct and indirect consequences of                           a
given technology or treatment.2

Federal Coordinatins Council Definition of          CER


The Federal Coordinating Council for Comparative Effectiveness Research (FCCCER) was established in
2009 under the American Recovery and Reinvestment Act (ARRA) to "coordinate comparative
effectiveness research across the Federal government, The Council was charged with making
recommendations for the framework and prioritization of spending for the S+00 m¡ll¡on allocated to the
Office of the Secretary [of Health and Human Services] for CER."3

Following a series of public listening sessions regarding how to define CER and prioritize this research,
the FCCCER has developed the following definition and criteria, which it outlined in a report submitted
to Congress on June 30, 2009:

l
  sackett DL, Strauss SE, Richardson WS, etol. Evidence-based Medicine: How to Practice and Teach EBM. London:
Chu rchill-Livingstone, 2000.
2
  Buckley, T. The Complexities of Comparative Effectiveness, October 25,2OO7.
                                                              accessed June L4, 2009.
3
    Comparative Effectiveness Research Funding: Federal Coordinating Council for Comparative Effectiveness
Research,   http://www.hh                                                  accessed May 26, 2009.
"Comparative effectiveness research is the conduct and synthesis of research comparing the benefits
and harms of different interventions and strategies to prevent, diagnose, treat and monitor health
conditions in 'realworld' settings. The purpose of this research is to improve health outcomes by
developing and disseminating evidence-based information to patients, clinicians, and other decision-
makers, responding to their expressed needs, about which interventions are most effective for which
patients under specific circumstances.

            "To provide this information, comparative effectiveness research must assess a comprehensive
            array of health-related outcomes for diverse patient populations and subgroups.
            "Defined interventions compared may include medications, procedures, medical and assistive
            devices and technologies, diagnostic testing, behavioral change, and delivery system strategies.
            "This research necessitates the development, expansion, and use of a variety of data sources
            and methods to assess comparative effectiveness and actively disseminate the results."a

For a research project first   to be considered by the FCCCER, it must meet these criteria:

.           "lncluded within statutory limits of Recovery Act and FCC definition of CER
.           "Potential to inform decision-making by patients, clinicians, or other stakeholders
.           "Responsiveness to expressed needs of patients, clinicians, or other stakeholders
.           "Feasibility of research topic (including time necessary for research)

"The prioritization criteria for scientifically meritorious research and investments are:

            "Potential impact (based on prevalence of condition, burden of disease, variability in outcomes,
            costs, potential for increased patient benefit or decreased harm)
            "Potentialto evaluate comparative effectiveness in diverse populations and patient subgroups
            and engage communities in research
            "Uncertainty within the clinical and public health communities regarding management decisions
            and variability in practice
       a    "Addresses need or gap unlikely to be addressed through other organizations
       a    "Potentialfor multiplicative effect (e.g, lays foundation forfuture CER such as data
            infrastructure and methods development and training, or generates additional investment
            outside government)"s

The FCCCER also has developed a strategic framework for CER activity and investments to categorize
current activity, identify gaps, and inform decisions on high priority recommendations. According to the
FCCCER, "This framework is intended to support immediate decisions for investment in CER priorities
and to provide a comprehensive foundation for longer-term strategic decisions on CER priorities and the
related infrastructure. At the framework's core is responsiveness to expressed needs for comparative
effectiveness research to inform health care decision-making by patients, clinicians, and others in the



a   Federal Coordinating Council on Comparative Effectiveness Research Report to Congress, June 30, 2009, p. 17.

s
    lbid., page 17.
clinical and public health communities."6 The activities and investments are grouped into four main
categories - research, human and scientific capital, CER data infrastructure, and dissemination and
translation of CER.

lnstitute of Medicine Definition of CER

The lnstitute of Medicíne (lOM), which was tasked under the ARRA with developing a list of national            CER
priorities, defines CER this way:

"Comparative effectiveness research (CER) is the generation and synthesis of evidence that compares
the benefits and harms of alternative methods to prevent, diagnose, treat and monitor a clinical
condition or to improve the delivery of care. The purpose of CER is to assist consumers, clinicians,
purchasers, and policy makers to make informed decisions that will improve health care at both the
individual and population levels."7

ln addition to describing L00 specific and prioritized CER recommendations for the Department of
Health and Human Services' (HHS) portion of the ARRA CER funds, the IOM makes the following general
recommendations:

1. Prioritization of CER topics should be a sustained and continuous process, recognizing the dynamic
state of disease, interventions, and public concern,

2. Public (including consumers, patients, and caregivers) participation in the priority-setting process        is
imperative to provide transparency in the process and input to delineating research questions.

3, Consideration of CER topics requires the development of robust, consistent topic briefs providing
background information, current practice, and research status of the condition and its interventions.

4. Regular reporting of the activities and recommendations of the prioritizing body is necessary to
evaluate the portfolio's distribution, its impact for discovery, and its translation into clinical care in order
to provide a process for continuous quality improvement.

5. The HHS Secretary should establish a mechanism-such as a coordinating advisory              body-with the
mandate to strategize, organize, monitor, evaluate and report on the implementation and impact of the
CER Program.


6. The CER Program should fully involve consumers, patients, and caregivers in key aspects of CER,
including strategic planning, príority setting, research proposal development, peer review, and
dissemination.




6lbid., p. 25.
7lnstitute of Medicine, lnitial National Priorities for Comparative Effectiveness Research, June 2009, p.7.
http://iom.edu/Object.File/Master/7!/IO7/CER%20reporT%20brief%2o6%2030%2009.pdf,accessed                Ju|y2,2009.
7. The CER Program should devote sufficient resourcesto research and innovation in the methods of
CER, includingthe development of methodological guidance for CER study design such as the
appropriate use of observational data and more informative, practical, and efficient clinical trials.

8. The CER Program should help to develop large-scale, clinical and administrative data networks to
facilitate better use of data and more efficient ways to collect new data to inform CER.

9. The CER Program should develop and support the workforce for CER to ensure the nation's capacity
to carry out the   CER mission.


L0. The CER Program should promote rapid adoption of CER findings and conduct research to identify
the most effective strategies for disseminating new and existing CER findings to health care
professionals, consumers, patients, and caregivers and for helping them to implement these results in
daily clinical practice.

NPC's Comments on the IOM and FCCCER Reports


NPC, as well as other health care stakeholder organizations, had submitted comments to FCCCER and
IOM on their draft definitions, criteria and frameworks. Following the release of the FCCCER and IOM
reports, NPC said it was pleased that some of its recommendations were incorporated into their
definitions and criteria, such as focusing on conditions with the greatest impact on morbidity and cost,
including all major therapeutic options, taking into account patient subgroups, and expressing clear
support for the development of new     CER   methodologies.

ln addition, however, NPC said that it would continue to monitor and seek clarification in areas that
were unclear or not included in the FCCCER and IOM reports, because it is vital for CER funding decisions
to be made in the best possible manner and result in information that improves clinical decision making
for health care providers and patients. ln particular, NPC wants to ensure that CER has a positive impact
on incentives for future innovation and that the proposed prioritization of research topics and studies,
their associated research time frames, final study outcomes, and related information will be made
transparent to all stakeholders and disseminated in a timely manner.

NPC also outlined   other key factors in the selection of the highest priority research:

    o    First, it will be important to conduct research to define rigorous, high quality, and validated CER
         methodologies that are focused on providing timely, accurate and balanced information in order
         to assist clinical decision making.
              o These questions include, but are not limited to, defining how best to address the full
                   range of health effects of a new technology including quality of life, functionality, and
                   productivity, as well as how best to appropriately characterize the strengths,
                   weaknesses, and limitations of various underlying health technology assessment
                   analytic techniques.
              o ln order to minimize the likelihood for inaccurate or inappropriate interpretation of CER,
                   a transparent and readily accessible description of the strengths, weaknesses,
                   limitations, and potential for generalizability of the findings of CER utilizing varied
                   experimental and non-experimental research designs should be included.
       Second, the strategic framework should implicitly assume that health care innovations will be
       considered as an important external input to a flexible CER framework. That is, it should be
       encompassed within and considered integral to the framework.
       Third, the agenda for CER should be driven by the clinical condition and the "key unanswered
       questions" in the context of that condition. Answering these questions may require comparisons
       between different types of technologies, processes, or procedures that may be considered to
       treat the condition; for example, the framework should reflect the need for comparisons of drug
       vs. surgery, drug and diagnostic vs. procedure, procedure vs. surgery, or other combinations.
       Fourth, comparisons should also include delivery system architecture options, insurance plan
       designs, methods for primary/secondary prevention, and approaches to provider incentives to
       effect improvements in health.

For further information, see "Key Considerations on CER" or the NPC website, www.npcnow.org.
     .-'. Natiional
    +2                                ical
'i.            Ëlfliliîceutica
                             1894 Preston White Drive, Reston, VA 2OL9I-5433 Phone: 703.620.6390 Fax: 703.476.0904 Web: www.npcnow.org



          A Brief History of Comparative Effectiveness Research
                      And Evidence-Based Medicine
lntroduction

The conceptsof evidence-based medicine (EBM)and comparative effectiveness research (CER)are not
new. Since the 1-970's, health industry leaders and the federal government have turned to Health
Technology Assessment (HTA), EBM, and more recently, CER as a means to improve quality and
consistency and maximize value in the health care delivery system. However, these concepts have taken
on prominence since the 1990's when legislation created the Agency for Health Care Policy and
Research (later renamed the Agency for Healthcare Research and Quality, or AHRQ), to support studies
on the outcomes of health care services and procedures.

These efforts have taken different names over the decades:

1970s: Health Technology Assessment
1980s: Effectiveness Research
1990s: Outcomes Research
2000s: Evidence-based Medicine and Comparative Effectiveness Research

An Overview of Earlv Efforts

Efforts to improve quality and maximize the value of health care seruices have been undertaken by both
governmental and private entities.

Past governmental efforts include:


           The U.S. Congress Office of Technology Assessmentl - An agency created by Congress in 1972ro
           provide analysis of new technologies, including healthcare. The agency was abolished in 1995 as
           part of the l-O4th Congress' "Contract with America."
           The lnstitute of Medicine's Council on Health Care Technology - Established in 1986 "to
           promote the development and application of technology assessment in health care and to
           review health care technologies for their appropriate use."2 The organization lost public funding
           in 1989.
           The Agency for Health Care Policy and Research           -   Early iteration of AHRQ; focused on
           developing clinical guidelines.3


t
 U.S. Congress, Office of Technology Assessment, The OTA Legacy: tg72-Igg5 (Washington, DC: April 1996)
http ://www. princeton.ed u/-ota/, accessed J u ne 15, 2009.
2
 Medical Technology Assessment Directory: A Pilot Reference to Organizations, Assessments, and lnformation
Resources (1988), lnstitute of Medicine, p. 633. http://books.nap.edu/openbook.php?record_id=1090&page=633,
accessed June 12, 2009.
3
    Luce B, Cohen RS, Hunter C, Cragin L, Johnson J. The Current Evidence-Based Medicine Landscape, April 2008, p.
6.
         Rxlntelligence   -   An independent nonprofit corporation founded by BlueCross BlueShield in
        2000, Rxlntelligence conducted cost-benefit, cost-effectiveness analyses of pharmaceutical
        drugs and provided "evaluation of therapeutic interchangeability of drugs." The entity lasted
        only two years.o
         Medicare Coverage Policy      -                    for Medicare and Medicaid Services (CMS)
                                           ln July 2006, the Centers
         issued a guidance document that allowed the agency to integrate evidence-based decision
         making and research into its coverage determination policies.s This policy is still currently in use
         by CMS and is informed by the Medicare Evidence Development and Coverage Advisory
        Committee (MEDCAC)6, which is a working group designed to supplement CMS' internal
        expertise.


Few of these efforts took hold, mostly because they lost political support due to their perceived threat
to innovation, medical autonomy, and market access.t

Private efforts include:

        Cochrane Collaboration - Founded in 1993, this global nonprofit network is dedicated to
        evaluating health care interventions through systematic reviews. The major product of the
        Collaboration is the Cochrane Database of Systematic Reviews, which is published quarterly as
        part of The Cochrane Library.8
        Blue Cross/Blue Shield Technology Evaluation Center- Established in 1995, this entity reviews
        interventions and evidence to determine effectiveness and guide clinical decision-making.
        Center for Medical Technology Policy (CMTP) - CMTP was created in 2006 to generate reliable
        and credible information about the real world risks, benefits and costs of promising new medical
        technologies. lnitial funding was provided by the California Healthcare Foundation and Blue
        Shield of California Foundation, with ongoing funding from organizations including the National
        Pharmaceutical Council.s
        lnstitute for Clinical and Economic Review (ICER): This organization was created by a grant from
        the Blue Shield of California Foundation in 2006, and roduces appraisals of clinical effectiveness
        and cost effectiveness of medical innovations, with the goal of providing new information to
        decision-makers intent on improving the value of health care services. Ongoing funding is
        provided by a group of organizations, including the National Pharmaceutical Council. 10
        ECRI lnstitute (formerly the Emergency Care Research lnstitute) - ECRI lnstitute is a nonprofit
        agency and is a Collaborating Center of the World Health Organization (WHO) and an Evidence-


a
 Luce B, Cohen RS, Hunter C, A Critical Analysis of the 2008 National Landscape of Evidence-Based Medicine and
Comparative Effectiveness Policies, April 2008, p.4.
t  rbid, p.24.
6
  Centers for Medicare and Medicaid Services, Medicare Evidence Development and Coverage Advisory
Committee.                                                    , accessed June 15, 2009.
t
  Bryan R. Luce, PhD, MBA, United BioSource Corporation, Presentation to the National Pharmaceutical Council,
April 2008.
8
  The Cochrane Collaboration - About the Cochrane Collaboration,
accessed June 12, 2009.
s
  The Center for Medical Technology Policy - About Us, http://www.cmtpnet.orglabout-cmtp, accessed June 5,
2009.
    lnstitute for Clinical and Economic Review,
10
                                                                                                      , accessed
June 5, 2009.
                                                                                                                   2
            based Practice Center (EPC) for AHRQ. ECRI evaluates safety, quality, and cost-effectiveness in
            health care. lt offers more than 10 databases, publications, information services, and technical
            assistance services.ll
            Hayes, lnc. - This is an independent organization that specializes in health technology
            assessment reports for health care organizations, including health plans, managed care
            companies, hospitals, and health networks. Hayes' medical research analysts assess such
            technologies as medical and surgical procedures, drugs, biologics, diagnostic and screening
            tests, medical devices and equipment, and complementary and alternative therapies.l2
            Oregon Drug Effectiveness Review Project - Established in 2003, this project "produces
            systematic, evidence-based reviews of the comparative effectiveness and safety of drugs in
            many widely used drug classes, and applies the findings to ¡nform public policy and related
            activities in local settings."13
            AMCP Format for Formulary Submissions -Established by the Academy of Managed Care
            Pharmacy, this is a set of guidelines for submitting new and existing pharmaceuticals for a
            health system's Pharmacy and Therapeutics Committee. The form requires detailed
            information, not only on the drug's safety and efficacy, but also on its overall clinical and
                                                                  1a
            economic value relative to alternative therapies.

It is believed that these private sector activities have succeeded largely because they have been
perceived as useful by the market in clinical decision making, purchasing, coverage and formulary
placement, and cost containment. For the most part, these initiatives have been insulated from political
influence, thus improving their longer term viability.ls

Growins lnterest in      CER    in Recent Years

The federal government's interest in CER has been accelerating over the past few years, with the
creation of new and expansion of several existing initiatives.

ln 2003, the Medicare Modernization Act (MMA) ensured funding for CER through AHRQ. Today AHRC[s
authority has expanded to generate new knowledge, which it does through a network of research
centers and private-public partnerships. ln 2005, AHRQ launched its Effective Health Care Program,
which has three core mandates:

       a   To review and synthesize existing knowledge through Evidence-based Practice Centers (EPCs)
       o   To promote and generate new knowledge through the DEcIDE (Developing Evidence to lnform
           Decisions about Effectiveness) Research Network
           To compile the findings from the EPCs and DEcIDE Network and then translate that knowledge
           for consumers, physicians, payers and policy makers.



tt
   Luce B, Cohen RS, Hunter C, Cragin L, Johnson J. The Current Evidence-Based Medicine Landscape, April 2008, p.
47.
tt
   rbid, p. 48.
tt
     Oregon Health & Science University-Centerfor Evidence-based Policy Drug Effectiveness Review Project,
     tp://www.ohsu.edu/ohsuedu/research/policvcenter/DERP/index.cfm,       accessed June 11, 2009.
     Luce B, Cohen RS, Hunter C, Cragin L, Johnson J. The Current Evidence-Based Medicine Landscape, April 2008, p.
48.
ttBryan
           R. Luce, PhD, MBA,   United BioSource Corporation, Presentation to the National Pharmaceutical Council,
April2008.
The program is meant to focus on effectiveness, as in the evidence of the relative benefits and risks of
alternative interventions; be a transparent and open process; determine usability and real-world
applicability; and drive research forward.l6 Since 2005, AHRQ has published more than 150 reports on
various interventions and treatments.lT

ln addition to MMA, the lnstitute of Medicine's Roundtable on Evidence-Based Medicine has engaged
major stakeholders in an effort to "help transform the way evidence on clinical effectiveness is
generated and used to improve health and health care." Through workshops and publications, the IOM
hopes to engage health care stakeholders and "identify key issues that are not being adequately
addressed, the nature of the barriers and possible solutions, and the priorities for action" in order to
achieve its stated goal for 90 percent of all healthcare decisions to be patient-specific and based on the
best available evidence by 2020.18


Other ongoing government-supported progra ms include:

           U.S. Preventive Services Task Force (USPSTF)     -
                                                         Established in 1984, and sponsored by AHRQ
        since 1998, USPSTF is an independent panel of private-sector experts in prevention and primary
        care, and conducts assessment of various health services.te
        Department of Veterans Affairs and Department of Defense - Both of these entities use data
        from their patient populations to assess the effectiveness of various interventions and make
        coverage decisions based on findings. The Department of Veterans Affairs' program is called the
        Technology Assessment Program (VATAP)20 and the Department of Defense manages these
        efforts through TRICARE Management Activity,2l the Department of Defense agency responsible
        for administering the health benefits of military beneficiaries.

Under the Obama administration, momentum for the advancement of CER continues to grow. ln
January 2009, as part of the economic stimulus law known as the American Recovery and Reinvestment
Act (ARRA), the new Congress set aside Sf      .t   bill¡on in funding for CER. For a summary of more recent
developments related to     CER and EBM, please see      "Legislative Proposals Regarding CER and EBM."




t'Agency
           for Healthcare Research and Quality, Effective Health Care   -   The Program,


"  u.s. Department of Heatth and Human services, Agency for Hearthcar:it""rt:.ii'iåi;iÎir1 rt 2008 Annuat
Performance
Report,
accessed June 5, 2009.
18
   lnstitute of Medicine Roundtable on Evidence-Based Medicine - Charter and Vision Statement,
                                                               , accessed June 11, 2009.
   Luce B, Cohen RS, Hunter C, Cragin L, Johnson J. The Current Evidence-Based Medicine Landscape, April 2008, p.
9.
'o U.S. Department of Veterans Affairs, VA Technology Assessment Program.
http://www.va.gov/VATAP/i ndex. htm, accessed Ju ne 15, 2009.
"Jacobsen G, CRS Reportfor Congress: Comparative Clinical Effectiveness and Cost-Effectiveness Research:
Background, History, and Overview, October L5, 2007, p. 28.
                                                       , accessed June 15, 2009; and Agency for Healthcare
Research and Quality - ECRI.                                             , accessed June 15, 2009.
                                utical

                            1894 Preston White Drive, Reston, VA 20191-5433 Phone: 703.620.6390 Fax: 703.476.0904 Web: www.npcnow.org




                           NPC's Key Considerat¡ons
                      On Comparative Effectiveness Research

The goal of comparative effectiveness research (CER) should be to support the dialogue between health
care providers and patients, thus enhancing the quality of patient care.

To ensure the successful implementation of CER, policy makers should consider the following issues:

     L    Focus on conditions with high burden of illness and cost such as chronic diseases and provide
          evidence that will facilitate good decision-making by health care professionals and patients.
     2.   Encompass all healthcare services, including devices, diagnostics, healthcare delivery methods,
          pharmaceuticals and medical and surgical procedures, and establish priorities for research in an
          explicit and transparent manner.
     3.   CER   methods should be rigorous and transparent, and conducted in accordance with a clear set
        of methods guidelines.
    4.  Study and monitor how best to employ CER in a manner that preserves incentives for
        continuous innovation of healthcare technologies in areas of unmet need.
     5. Consider the needs of patient subgroups who may respond differently to medicines and
       treatments based on age, genetic variation and co-morbidities.
    6. Encourage fully transparent stakeholder involvement that allows for multiple organizations
       (including industry) to participate in both governance and evidence generation/assessment.
    7. Utilize a full range of types and sources of evidence that consider both direct and indirect
       benefits to society, such as quality of life, patient functionality and economic productivity.
    8. Be current and allow for amendment when new data emerges.
    9. Ensure balanced, effective and timely communication of results to consumers, patients,
          physicians and health care professionals, including any limitations to findings.



Over the past several months, NPC has presented these considerations before the lnstitute of
Medicine's CER Priority Setting Committee, the Agency for Healthcare Research and Quality and the
Federal Coordinating Council for Comparative Effectiveness Research. lt is critical for these entities to
establish appropriate guiding principles that will be used by decision makers in determining CER
priorities.
                            1894 Preston White Drive, Reston, VA 20191-5433 Phone: 703.620.6390 Fax: 703.476.0904 Web:www.npcnow.org



                             Legislative Proposals Regarding
                           Comparative Effectiveness Research
lntroduction

Congress made a major investment in ensuring high-quality, patient-centered health care by allocating
Sf .f bilt¡on for comparative effectiveness research (CER) as part of the American Recovery and
Reinvestment Act (ARRA). Of those funds, the Agency for Healthcare Research and Quality (AHRQ)
received SZOO mill¡on; of that Sz00 mill¡on, 5400 million was transferred to the Office of Director of the
National lnstitutes of Health (NlH)to support research projects. The remaining 5+OO million will be
allocated at the discretion of the Secretary of Health and Human Services (HHS).l While there is debate
as to whether this is enough money to meaningfully integrate CER into the health care delivery system,
it is a clear recognition by government of the importance of CER as a public good.

Who Will Decide What to Do With the Fundine?

Within ARRA, Congress created a committee known as the Federal Coordinating Council for Comparative
Effectiveness Research (FCCCER) that is composed of 15 senior federal employees who are in leadership
roles in government organizations that impact health care. At least half are physicians or other experts
with clinical expertise. The task of the committee is to coordinate CER efforts across government
agencies, and to make recommendations on CER spending priorities to Congress. The Council submitted
its initial report to Congress on June 30, 2009. Funds for CER within ARRA must be obligated by
September 30,20t0, and the law states a preference for "quick start" projects that can be initiated
within 120 days. Every six months, HHS, AHRQ and NIH must submit a report to Congress detailing how
funds have been spent.2

ls the United States Heading Toward an EBM/CER lnstitute?

Many unanswered questions remain about how a government-sponsored CER effort would be
structured, the type of research it should conduct, and how health care decision makers would or should
use the information generated. lt is unclear whether ARRA's funding for CER will be followed by the
enactment of legislation that creates a new government CER body, or whether existing agencies will be
used to facilitate CER and to help enable its application by various stakeholders in the health care
delivery system.

Several lawmakers have proposed legislation that includes the creation of a permanent CER entity. ln
June 2009, U.S. Senate Finance Committee Chairman Max Baucus (D-MT) and U.S. Senate Budget
Committee Chairman Kent Conrad (D-ND) introduced legislation, "The Patient-Centered Outcomes


1
  American Recovery and Reinvestment Act of 2OO9, Title Vlll - Departments of Labor, Health and Human Services,
and Education, and Related Agencies.
2
  American Recovery and Reinvestment Act of 2009, Sec. 804 - Federal Coordinating Council for Comparative
Effectiven ess Research.
Research Act of 2OO9," to establish a "private, nonprofit corporation, called the Patient-Centered
Outcomes Research lnstitute, to generate scientific evidence and new information on how diseases,
disorders and other health conditions can be treated to achieve the best clinicaloutcome for patients,"3

The lnstitute would "be governed by a multi-stakeholder Board of Governors" and would "establish a
national agenda of research priorities." lt could "contract with federal agencies, such as AHRQ, and
appropriate private entities to conduct the research, which lwould] include both systematic reviews and
primary research. The lnstitute and its activities lwould] be funded by contributions from both public
and private payers, made available to the lnstitute through a Patient-Centered Outcomes Research Trust
Fu   nd."a

Chairman Baucus said that he intends for this legislation to be included as part of a comprehensive
health reform bill that will be considered by his committee.s

ln May 2009, U.S. Representatives Kurt Schrader (D-OR) and Allyson Schwartz (D-PA) introduced similar
legislation to create a Health Care Comparative Effectiveness Research lnstitute "responsible for
managing and guiding health research programs." This institute would be governed by a 21-member
board that would include representatives from government agencies, state health agencies, and the
insurance and pharmaceutical industries, along with physicians, patients, researchers and other
experts. Public comment would be solicited prior to establishing research guidelines, and all research
would have to account for patients' gender, race, age and ethnicity and be subject to a peer-review
process. Their legislation also would establish a research fund paid for with unspent stimulus money and
fees on Medicare and private health plans.6

Separately, in mid-July 2009, the U.S. Senate Health, Education, Labor and Pensions (HELP) Committee
approved comprehensive health reform legislation to establish a "Center for Health Outcomes Research
and Evaluation" within AHRQ. Under the legislation, the Center will "coordinate, conduct, support and
synthesize research relevant to the comparative health outcomes and effectiveness of the full spectrum
of health care treatments." lt will be governed by an advising council composed of the AHRQ director,
the chief medical officer of the Centers for Medicare and Medicaid Services (CMS), and "19 additional
members" who "collectively have experience" in epidemiology, health services research, bioethics,
communication and decision sciences, health economies, and safe use of medical products.i

A similar bill that places a CER entity under AHRQ s purview was introduced by the U.S. House Tri-
Committee, which comprises the Energy and Commerce, Ways and Means, and Education and Labor
Committees. Among its sweeping changes to the health care system, the "America's Affordable Health
Choices Act" would create a "Center for Comparative Effectiveness Research within the AHRQto
conduct, support and synthesize research relevant to the comparative effectiveness of the full spectrum
of health care items, services, and systems." A l7-member commission would be appointed by the HHS

t   Baucus, Conrad lntroduce Bill   to lnvest in Research on Best Practices in Health Care, press release, June 9, 2009.
                                                                         accessed June 11, 2009.
o
    rbid.
s
    U.S. Senate Finance   Committee Chairman Max Baucus, comments at the Brookings lnstitution, June g,2OOg,
                                                                     , accessed June 11, 2009.
6
  Schrader, New Dems lntroduce Bill Empowering Doctors, Nurses to Make the Best Patient Care Decisions, press
release, May 19, 2009. hllp://www.schrader.house.gov/?sectionid=24&parentid=23&sectiontree=23&itemid=141,
accessed May 19,2009.
7
  Affordable Health Choices Act, Section 937, "Center for Health Outcomes Research and Evaluation."
Secretary, and members would include "the Director of AHRQ, the Chief Medical Officer of CMS, and
stakeholders including clinicians, patients, researchers, third-party payers and consumers. Clinical
perspective advisory panels [would] provide advice on specific research questions, methods and gaps in
evidence in terms of clinical outcomes for priorities identified by the commission in order to ensure that
the research is clinically relevant."s

During House Energy and Commerce Committee consideration, three CER-related amendments were
added to the Tri-Committee legislation, The amendments would:

       o   Ensure that work performed by the CER entity is "based upon consultation with, and review by,
           the specialty colleges and academies of medicine to determine best practices within their field
           of specialty. Any recommendations made or best practices developed by the Commission or the
           Center shall be based upon evidence-based medicine and shall not violate standards and
                                                                                      s
           protocols of clinical excellence of the specialty colleges and academies."
       o   Prevent CMS from using federally funded clinical CER data to make coverage determinations for
           medical treatments, services, or items on the basis of cost.1o
       .   Specify that CER may not be used by the federal government to deny or ration care.tt

The Tri-Committee legislation was approved by the House Energy and Commerce Committee on July 31,
2009.




8
    Tri-Committee Summary of America's Affordable Health Choices Act, July !4,2009, pgs. 18-19.
s
    Amendment to the Amendment in the Nature of A Substitute to H.R. 3200 Offered by Mr. Tim Murphy of
Pennsylvania (Al NS-EC_001).
to
 Amendment to the Amendment in the Nature of A Substitute to H.R. 3200 Offered by Mr. Phil Rangel of Georgia.
tt
 Amendment to the Amendment in the Nature of A Substitute to H.R. 3200 Offered by Mr. Mike Rogers of
Michigan.
                                              Comparison of Various Comparative Effectiveness Research Legislative Activities
                                                in the Context of NPC's CER Recommendations to lOM, FCCCER, and AHRQ



                                                                                                                     Doctor-
                                                                                                                     Patient
                                                            PCORA 2009                 House Tri-       CER Act    Relationship
           NPC Comparative Effectiveness Research            (Senators                 Committee        of 2009        Act
#                                                                        Senate HELP                                              ARRA
                     Recommendation                           Baucus-                    Health          (Rep.        (Reps.
                                                              Conrad)                   Reform         Schrader)   Christensen,
                                                                                                                     Herger,
                                                                                                                    Boustany)
7   Focus on conditions with high burden of illness and
    cost such as chronic diseases and provide evidence
                                                                                          Silent                      Silent      Silent
    that will facilitate good decision-making by health
    care professionals and patients

2   Encompass all healthcare services, including devices,
    diagnostics, healthcare delivery methods,
    pharmaceuticals and medical and surgical
    procedures, and establish priorities for research in
    an explicit and transparent manner'

3   CER methods should be rígorous and transparent,
    and conducted in accordance with a clear set of                                                                   Silent      Sílent
    methods guidelines
4   Study and monitor how best to employ CER in a
    manner that preserves incentives for continuous
                                                                            Silent        Silent                      Silent      Silent
    innovation of healthcare technologies in areas of
    unmet need
5   Consider the needs of patient subgroups who may
    respond differently to medicines and treatments
    based on age, genetic variation and co-morbidities"


Legend:                     Silent: Not mentioned within the Bill        Precludes: Not allowed within the Bill
07.15.09                       Red = change from prior version
                                                                                                                                   Doctor-
                                                                                                                                   Patient
                                                              PCORA 2009                       House Tri-         CER Act        Relationship
           NPC Comparative Effectiveness Research              (Senators                       Committee          of 2009            Act
#                                                                             Senate HELP                                                           ARRA
                      Recommendation                            Baucus-                          Health             (Rep.           (Reps.
                                                                Conrad)                         Reform           Schrader)       Christensen,
                                                                                                                                   Herger,
                                                                                                                                  Boustany)
6   Encourage fully transparent sta keholder
    involvement that allows for multiple organizatíons
                                                                               Precludes                                                          Precludes
    (including industry)to partic¡pate in both governance
    and evidence generation/assessment"'

7   Utilize a full range of types and sources of evidence
    that consider both direct and indirect benefits to
    society, such as quality of life, patient functionality
    and economic prod uctivity'u

8   Be current and allow   for amendment when new
                                                                                 Silent                                             Silent          Silent
    data emerges
9   Ensure balanced, effective and timely
    communication of results to consumers, patients,
    physicians and health care professionals, including
    any limitations to findingsu



i All bills recognized and encompass a variety of healthcare service including devices, pharmaceuticals and medical and surgical procedures. Differences
in bills occur based upon the inclusion of healthcare delivery methods.
ü All bilts recognize the need to consider patient subgroups; however the degree and types of patient subgroups considered differ.
ü Stakeholder involvement from multiple organizations (including industry) varies, and may include either inclusion in governance and/or evidence
generation/ assessment.
i" All bitls recognize a broad range of types and sources of evidence; however only PCORA 2009 and CER Act of 2A09 address types of indirect benefits to
society such as (i.e. quality of life, patient functionality and economic productivity).
" All bills recognize the need for effective and timely communication of results; however each bill differs in the communication of limitations to
                                                                                                                                                   findings.



Legend:                       Silent: Not mentioned within the BilI           Precludes= Not allowed within the Bill
07.15.09                         Red : change from prior versron
                          1894 Preston White Drive, Reston, VA 20I9t-5433 Phone: 703 620.6390 Fax: 703.476.0904 Web: www.npcnow.org


                                        Additional Resources
Below are additional resource materials from the National Pharmaceutical Council related to the
ongoing conversations among U.S. health care stakeholders about evidence-based medicine and
comparative effectiveness research (CER). All       NPC   documents are available at www.npcnow.ors.

This document also includes links to key documents from organizations charged with developing the
definitions, framework and criteria for             the Federal Coordinating Council for
                                           CER, such as                                                  CER,   the Agency
for Healthcare Research and Quality, and the lnstitute of Medicine, among others.

National Pharmaceutical Council

        NPC Testimony    submitted to the Federal Coordinating Council on Comparative Effectiveness
        Research on the Draft Definition and Strategic Framework for Comparative Effectiveness
        Research, June 10, 2009
                                                                  -95d3-49de-89 b8-d       9a a 1 ta2e2l6

        NPC   Testimony before the Federal Coordinating Council on Comparative Effectiveness Research,
        April 14, 2009


        NPC   Testimony before the Agency for Healthcare Research and Quality, April 3, 2009


        NPC   Testimony before the lnstitute of Medicine Comparative Effectiveness Research
        Committee, March 20, 20Og
        http://www. npcnow.orslNews.aspx?newsid=5d8d0e9f-1100-4b39-866e-fcbf453bdf5c
        NPC Podcast; Briefing on Clinical Comparative Effectiveness, February 10, 2009
                                                                 cf   1095-8655-4e06-98 10-d 8578e2d 8c39
        NPC   Statement on Economic Stimulus Package, February                17   ,2009
                                                                 3    10b95-9 bf6-493 6-a dcf -5fL6f69 4c62a
        NPC-Funded Study: National lnstitute for Health and Clinical Excellence (NICE): How Does                     it
        Work and What Are the lmplications for the U.S.?,
        NPC-Funded Study: The Current Evidence-Based Medicine Landscape,




Agency for Healthcare Research and Quality
    o   National Advisory Council for Healthcare Research and Quality
Congressional Budget Office
    o     Research on the Comparative Effectiveness of Medical Treatments, December 2OO7


    o    CBO Budget Options, Volume l: Health Care, 2008




Federal Coordinating Cou ncil for Comparative Effectiveness Research
    o     Report to the President and Congress on Comparative Effectiveness Research, June 30, 2009


    ¡     Federal Coordinating Council for Comparative Effectiveness Research, main page



lnstitute of Medicine
    o     lnitial National Priorities for Comparative Effectiveness Research, June 30, 2009

    o     lnstitute of Medicine Comparative Effectiveness Research Prioritization
                                                      X

    o     lnstitute of Medicine Comparative Effectiveness Research Committee Public Meetings, March
          20,2009



Public   law
    o    Comparative Effectiveness Research Sections of the American Recovery and Reinvestment Act

				
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