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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 comparativeeffectiveness 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 understandingof 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 ofinformational pieces, which taken together provide an overview of CER. Each of the following itemsoutlines a specific aspect of CER in a short, easy-to-read format.

Defining Evidence-Based Medicine a nd Comparative Effectiveness Resea rch

A Brief History of Comparative Effectiveness Research and Evidence-Based MedicineNPC's Key Considerations on Comparative Effectiveness Research

Legislative Proposals Regarding Comparative Effectiveness Research

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 organizationsinterested in the issue, as well as link to the pieces on NPC's website, where we will be providingupdates to the materials on an ongoing basis.

For additional copies of this information, please contact NPC at info@npcnow.ors or 703-620-6390.

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Defining Evidence-Based Medicine

and Comparative Effectiveness Research

lntroduction

Evidence-based medicine (EBM) is a systematic approach to clinical problem solving which allows theintegration 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 mustalso consider patient preferences, optimal patient outcomes, and the relative effects among competingalternatives. Broadly considered, EBM includes the comparative effectiveness assessments of drugs,treatments and devices, and the appropriate interpretation of evidence from these assessments tosupport health benefit design and medical decision-making.

EBM is the foundation for Comparative Effectiveness Research (CER), which compares availabletreatment options utilizing a range of research methods including randomized controlled trials,observational studies, and systematic reviews, a structured assessment of evidence available frommultiple 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 technologyor 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 in2009 under the American Recovery and Reinvestment Act (ARRA) to "coordinate comparativeeffectiveness research across the Federal government, The Council was charged with makingrecommendations for the framework and prioritization of spending for the S+00 m¡ll¡on allocated to theOffice 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 submittedto 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 benefitsand harms of different interventions and strategies to prevent, diagnose, treat and monitor healthconditions 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 whichpatients under specific circumstances.

"To provide this information, comparative effectiveness research must assess a comprehensivearray of health-related outcomes for diverse patient populations and subgroups."Defined interventions compared may include medications, procedures, medical and assistivedevices and technologies, diagnostic testing, behavioral change, and delivery system strategies.

"This research necessitates the development, expansion, and use of a variety of data sourcesand 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 subgroupsand engage communities in research"Uncertainty within the clinical and public health communities regarding management decisionsand variability in practice

"Addresses need or gap unlikely to be addressed through other organizations"Potentialfor multiplicative effect (e.g, lays foundation forfuture CER such as datainfrastructure and methods development and training, or generates additional investmentoutside government)"s

The FCCCER also has developed a strategic framework for CER activity and investments to categorizecurrent activity, identify gaps, and inform decisions on high priority recommendations. According to theFCCCER, "This framework is intended to support immediate decisions for investment in CER prioritiesand to provide a comprehensive foundation for longer-term strategic decisions on CER priorities and therelated infrastructure. At the framework's core is responsiveness to expressed needs for comparativeeffectiveness 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.

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clinical and public health communities."6 The activities and investments are grouped into four maincategories - 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 comparesthe benefits and harms of alternative methods to prevent, diagnose, treat and monitor a clinicalcondition 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 theindividual and population levels."7

ln addition to describing L00 specific and prioritized CER recommendations for the Department ofHealth 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 dynamicstate of disease, interventions, and public concern,

2. Public (including consumers, patients, and caregivers) participation in the priority-setting process isimperative 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 providingbackground 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 toevaluate the portfolio's distribution, its impact for discovery, and its translation into clinical care in orderto provide a process for continuous quality improvement.

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

6 lbid., p. 25.7 lnstitute 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 resources to research and innovation in the methods ofCER, including the development of methodological guidance for CER study design such as theappropriate 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 tofacilitate 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 capacityto carry out the CER mission.

L0. The CER Program should promote rapid adoption of CER findings and conduct research to identifythe most effective strategies for disseminating new and existing CER findings to health careprofessionals, 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 andIOM 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 theirdefinitions 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 clearsupport for the development of new CER methodologies.

ln addition, however, NPC said that it would continue to monitor and seek clarification in areas thatwere unclear or not included in the FCCCER and IOM reports, because it is vital for CER funding decisionsto be made in the best possible manner and result in information that improves clinical decision makingfor health care providers and patients. ln particular, NPC wants to ensure that CER has a positive impacton 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 madetransparent 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 orderto assist clinical decision making.

o These questions include, but are not limited to, defining how best to address the fullrange of health effects of a new technology including quality of life, functionality, andproductivity, as well as how best to appropriately characterize the strengths,weaknesses, and limitations of various underlying health technology assessmentanalytic 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 variedexperimental 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 unansweredquestions" in the context of that condition. Answering these questions may require comparisonsbetween different types of technologies, processes, or procedures that may be considered totreat the condition; for example, the framework should reflect the need for comparisons of drugvs. 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 toeffect improvements in health.

For further information, see "Key Considerations on CER" or the NPC website, www.npcnow.org.

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A Brief History of Comparative Effectiveness Research

And Evidence-Based Medicine

lntroduction

The conceptsof evidence-based medicine (EBM)and comparative effectiveness research (CER)are notnew. Since the 1-970's, health industry leaders and the federal government have turned to HealthTechnology 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 takenon 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 studieson the outcomes of health care services and procedures.

These efforts have taken different names over the decades:

1970s: Health Technology Assessment1980s: 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 bothgovernmental and private entities.

Past governmental efforts include:

The U.S. Congress Office of Technology Assessmentl - An agency created by Congress in 1972roprovide 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 "topromote the development and application of technology assessment in health care and toreview health care technologies for their appropriate use."2 The organization lost public fundingin 1989.

The Agency for Health Care Policy and Research - Early iteration of AHRQ; focused ondeveloping 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 lnformationResources (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 - ln July 2006, the Centers for Medicare and Medicaid Services (CMS)

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 threatto innovation, medical autonomy, and market access.t

Private efforts include:

Cochrane Collaboration - Founded in 1993, this global nonprofit network is dedicated toevaluating health care interventions through systematic reviews. The major product of theCollaboration 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 reviewsinterventions and evidence to determine effectiveness and guide clinical decision-making.

Center for Medical Technology Policy (CMTP) - CMTP was created in 2006 to generate reliableand credible information about the real world risks, benefits and costs of promising new medicaltechnologies. lnitial funding was provided by the California Healthcare Foundation and Blue

Shield of California Foundation, with ongoing funding from organizations including the NationalPharmaceutical Council.s

lnstitute for Clinical and Economic Review (ICER): This organization was created by a grant fromthe Blue Shield of California Foundation in 2006, and roduces appraisals of clinical effectivenessand cost effectiveness of medical innovations, with the goal of providing new information todecision-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 nonprofitagency 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 AdvisoryCommittee. , 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,

2009.10 lnstitute for Clinical and Economic Review, , accessed

June 5, 2009.

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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 technicalassistance services.llHayes, lnc. - This is an independent organization that specializes in health technologyassessment reports for health care organizations, including health plans, managed carecompanies, hospitals, and health networks. Hayes' medical research analysts assess such

technologies as medical and surgical procedures, drugs, biologics, diagnostic and screeningtests, medical devices and equipment, and complementary and alternative therapies.l2Oregon Drug Effectiveness Review Project - Established in 2003, this project "producessystematic, evidence-based reviews of the comparative effectiveness and safety of drugs inmany widely used drug classes, and applies the findings to ¡nform public policy and relatedactivities in local settings."13

AMCP Format for Formulary Submissions -Established by the Academy of Managed CarePharmacy, this is a set of guidelines for submitting new and existing pharmaceuticals for a

health system's Pharmacy and Therapeutics Committee. The form requires detailedinformation, not only on the drug's safety and efficacy, but also on its overall clinical andeconomic value relative to alternative therapies. 1a

It is believed that these private sector activities have succeeded largely because they have beenperceived as useful by the market in clinical decision making, purchasing, coverage and formularyplacement, and cost containment. For the most part, these initiatives have been insulated from politicalinfluence, 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 thecreation 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 researchcenters and private-public partnerships. ln 2005, AHRQ launched its Effective Health Care Program,which has three core mandates:

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To review and synthesize existing knowledge through Evidence-based Practice Centers (EPCs)

To promote and generate new knowledge through the DEcIDE (Developing Evidence to lnformDecisions about Effectiveness) Research NetworkTo compile the findings from the EPCs and DEcIDE Network and then translate that knowledgefor 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 ofalternative interventions; be a transparent and open process; determine usability and real-worldapplicability; and drive research forward.l6 Since 2005, AHRQ has published more than 150 reports onvarious 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 toachieve its stated goal for 90 percent of all healthcare decisions to be patient-specific and based on thebest 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 primarycare, and conducts assessment of various health services.te

Department of Veterans Affairs and Department of Defense - Both of these entities use datafrom their patient populations to assess the effectiveness of various interventions and makecoverage decisions based on findings. The Department of Veterans Affairs' program is called theTechnology Assessment Program (VATAP)20 and the Department of Defense manages theseefforts through TRICARE Management Activity,2l the Department of Defense agency responsiblefor 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 AnnuatPerformance

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 HealthcareResearch and Quality - ECRI. , accessed June 15, 2009.

utical

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NPC's Key Considerat¡onsOn Comparative Effectiveness Research

The goal of comparative effectiveness research (CER) should be to support the dialogue between healthcare 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.

Study and monitor how best to employ CER in a manner that preserves incentives forcontinuous innovation of healthcare technologies in areas of unmet need.

Consider the needs of patient subgroups who may respond differently to medicines and

treatments based on age, genetic variation and co-morbidities.

Encourage fully transparent stakeholder involvement that allows for multiple organizations(including industry) to participate in both governance and evidence generation/assessment.

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.

Be current and allow for amendment when new data emerges.

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 ofMedicine's CER Priority Setting Committee, the Agency for Healthcare Research and Quality and theFederal Coordinating Council for Comparative Effectiveness Research. lt is critical for these entities toestablish appropriate guiding principles that will be used by decision makers in determining CER

priorities.

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Legislative Proposals RegardingComparative 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 andReinvestment 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 theNational 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 debateas 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 ComparativeEffectiveness Research (FCCCER) that is composed of 15 senior federal employees who are in leadershiproles in government organizations that impact health care. At least half are physicians or other expertswith clinical expertise. The task of the committee is to coordinate CER efforts across governmentagencies, and to make recommendations on CER spending priorities to Congress. The Council submittedits 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 initiatedwithin 120 days. Every six months, HHS, AHRQ and NIH must submit a report to Congress detailing howfunds 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 shoulduse the information generated. lt is unclear whether ARRA's funding for CER will be followed by theenactment 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. lnJune 2009, U.S. Senate Finance Committee Chairman Max Baucus (D-MT) and U.S. Senate BudgetCommittee 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 ComparativeEffectiven ess Research.

Research Act of 2OO9," to establish a "private, nonprofit corporation, called the Patient-CenteredOutcomes 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 andprimary 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 TrustFu nd."a

Chairman Baucus said that he intends for this legislation to be included as part of a comprehensivehealth 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 similarlegislation to create a Health Care Comparative Effectiveness Research lnstitute "responsible formanaging and guiding health research programs." This institute would be governed by a 21-memberboard that would include representatives from government agencies, state health agencies, and theinsurance and pharmaceutical industries, along with physicians, patients, researchers and otherexperts. 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-reviewprocess. 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) Committeeapproved 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 spectrumof 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 additionalmembers" 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 LaborCommittees. Among its sweeping changes to the health care system, the "America's Affordable HealthChoices Act" would create a "Center for Comparative Effectiveness Research within the AHRQtoconduct, support and synthesize research relevant to the comparative effectiveness of the full spectrumof 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 thatthe 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 fieldof specialty. Any recommendations made or best practices developed by the Commission or theCenter shall be based upon evidence-based medicine and shall not violate standards and

protocols of clinical excellence of the specialty colleges and academies." s

o Prevent CMS from using federally funded clinical CER data to make coverage determinations formedical 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 ofPennsylvania (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 ofMichigan.

Comparison of Various Comparative Effectiveness Research Legislative Activitiesin the Context of NPC's CER Recommendations to lOM, FCCCER, and AHRQ

#NPC Comparative Effectiveness Research

Recommendation

PCORA 2009(Senators

Baucus-Conrad)

Senate HELP

House Tri-Committee

HealthReform

CER Actof 2009(Rep.

Schrader)

Doctor-Patient

RelationshipAct

(Reps.

Christensen,Herger,

Boustany)

ARRA

7 Focus on conditions with high burden of illness andcost such as chronic diseases and provide evidencethat will facilitate good decision-making by healthcare professionals and patients

Silent Silent Silent

2 Encompass all healthcare services, including devices,diagnostics, healthcare delivery methods,pharmaceuticals and medical and surgicalprocedures, 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 ofmethods guidelines

Silent Sílent

4 Study and monitor how best to employ CER in amanner that preserves incentives for continuousinnovation of healthcare technologies in areas ofunmet need

Silent Silent Silent Silent

5 Consider the needs of patient subgroups who may

respond differently to medicines and treatmentsbased on age, genetic variation and co-morbidities"

Legend:07.15.09

Silent: Not mentioned within the BillRed = change from prior version

Precludes: Not allowed within the Bill

#NPC Comparative Effectiveness Research

Recommendation

PCORA 2009(Senators

Baucus-Conrad)

Senate HELP

House Tri-Committee

HealthReform

CER Actof 2009

(Rep.

Schrader)

Doctor-Patient

RelationshipAct

(Reps.

Christensen,Herger,

Boustany)

ARRA

6 Encourage fully transparent sta keholderinvolvement that allows for multiple organizatíons(including industry)to partic¡pate in both governance

and evidence generation/assessment"'

Precludes Precludes

7 Utilize a full range of types and sources of evidencethat consider both direct and indirect benefits tosociety, such as quality of life, patient functionalityand economic prod uctivity'u

8 Be current and allow for amendment when new

data emergesSilent Silent Silent

9 Ensure balanced, effective and timelycommunication of results to consumers, patients,physicians and health care professionals, includingany 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:07.15.09

Silent: Not mentioned within the BilI Precludes= Not allowed within the BillRed : 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 CER, such as the Federal Coordinating Council for 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 ,20093 10b95-9 bf6-493 6-a dcf -5fL6f69 4c62a

NPC-Funded Study: National lnstitute for Health and Clinical Excellence (NICE): How Does itWork and What Are the lmplications for the U.S.?,

NPC-Funded Study: The Current Evidence-Based Medicine Landscape,

Agency for Healthcare Research and Qualityo 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 lawo Comparative Effectiveness Research Sections of the American Recovery and Reinvestment Act