Comparative Effectiveness Research (cer) Toolkit

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 a a a a a

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 Medicine NPC'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 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 [email protected] 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 given technology or treatment.2 Federal Coordinatins Council Definition of

a

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: rchill-Livingstone, 2000. 2 Buckley, T. The Complexities of Comparative Effectiveness, October 25,2OO7. Chu

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 decisionmakers, 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:

a a

"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 "Addresses need or gap unlikely to be addressed through other organizations "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 priorities, defines CER this way:

CER

"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 imperative to provide transparency in the process and input to delineating research questions.

is

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.

6

lbid., p. 25. 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

to research and innovation in the methods of the development of methodological guidance for CER study design such as the appropriate use of observational data and more informative, practical, and efficient clinical trials. 7. The CER Program should devote sufficient resources CER, including

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

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 NPC, as well as

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

o

other key factors in the selection of the highest priority research:

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

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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

-

for Medicare and Medicaid Services (CMS) issued a guidance document that allowed the agency to integrate evidence-based decision Medicare Coverage Policy

ln July 2006, the Centers

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. 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. 10 lnstitute for Clinical and Economic Review, , accessed June 5, 2009. 6

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

economic value relative to alternative therapies.

1a

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 o

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

April2008.

United BioSource Corporation, Presentation to the National Pharmaceutical Council,

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 developments related to

t'Agency

.t

bill¡on in funding for CER. For a summary of more recent

CER and EBM, please see

"Legislative Proposals Regarding CER and EBM."

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. 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

4.

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 TriCommittee, 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 s

rbid. U.S. Senate Finance

Committee Chairman Max Baucus, comments at the Brookings lnstitution, June g,2OOg,

, accessed June 11, 2009. 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§iontree=23&itemid=141, accessed May 19,2009. 7 Affordable Health Choices Act, Section 937, "Center for Health Outcomes Research and Evaluation." 6

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

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." 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

s

Tri-Committee Summary of America's Affordable Health Choices Act, July !4,2009, pgs. 18-19. 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. Amendment to the Amendment in the Nature of A Substitute to H.R. 3200 Offered by Mr. Mike Rogers of Michigan.

tt

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

PCORA 2009 NPC Comparative Effectiveness Research

#

Recommendation

(Senators BaucusConrad)

Senate HELP

House TriCommittee Health Reform

Act of 2009

CER

(Rep. Schrader)

DoctorPatient Relationship

Act (Reps.

ARRA

Christensen, Herger,

Boustany) 7

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

4

Silent

methods should be rígorous 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 for continuous innovation of healthcare technologies in areas of

Silent

Silent

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: 07.15.09

Silent: Not mentioned within the Bill Red = change from prior version

Precludes: Not allowed within the Bill

Silent

Silent

Silent

Sílent

Silent

Silent

PCORA 2009 NPC Comparative Effectiveness Research

#

Recommendation

(Senators BaucusConrad)

Senate HELP

House TriCommittee Health Reform

Act of 2009

DoctorPatient Relationship Act

(Rep.

(Reps.

Schrader)

Christensen, Herger,

CER

ARRA

Boustany) 6

Encourage fully transparent sta keholder involvement that allows for multiple organizatíons (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

data emerges 9

Precludes

Precludes

Silent

Silent

Silent

Ensure balanced, effective and timely communication of results to consumers, patients, physicians and health care professionals, including any limitations to findingsu

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). findings. " All bills recognize the need for effective and timely communication of results; however each bill differs in the communication of limitations to i

Legend: 07.15.09

Silent: Not mentioned within the BilI Red : change from prior versron

Precludes= Not allowed within the Bill

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

the Federal Coordinating Council for for Healthcare Research and Quality, and the lnstitute of Medicine, among others. definitions, framework and criteria for

CER, such as

CER,

the Agency

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 NPC

9a a 1 ta2e2l6

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

NPC

1095-8655-4e06-98 10-d 8578e2d 8c39

Statement on Economic Stimulus Package, February 3

17

,2009

10b95-9 bf6-493 6-a dcf -5fL6f69 4c62a

NPC-Funded Study: National lnstitute for Health and Clinical Excellence (NICE): How Does

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

it

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

Public

o

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

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