Initial National Priorities for Comparative Effectiveness Research (2009)

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1 Introduction Abstract: This chapter describes the legislative mandate and scope of work for the current study as well as previous Institute of Medicine (IOM) experience in methodologies for priority setting. The IOM Com- mittee on Comparative Effectiveness Research Prioritization was charged with recommending national priorities—with stakeholder input—for the discretionary expenditure of $400 million by the Secretary of Health and Human Services on comparative effectiveness research (CER), and with addressing the data and infrastructure needs to support and sustain this research. The formation of the committee is described as well as the pro- cedures by which it operated. This report provides definitions for CER, mechanisms the committee used for obtaining public input into the pro- cess and the priorities, methodologies for priority setting, and, finally, a portfolio of research topics recommended for funding by the Secretary and recommendations for an infrastructure to facilitate a sustained research enterprise for CER and its translation and dissemination. In the midst of one of the nation’s most serious economic crises, and in anticipation of major national health care reform, the 111th Congress acted to significantly expand public spending, particularly on the nation’s capacity to conduct comparative effectiveness research (CER). The Ameri- can Recovery and Reinvestment Act (ARRA) of 2009 (P.L. 111-5) defines CER (highlighted in the legislative language that follows) and provides $1.1 billion in CER funding for the Agency for Healthcare Research and Quality (AHRQ), the National Institutes of Health (NIH), and the Secre- tary of Health and Human Services (HHS). More than one-third of the funds—$400 million—is for discretionary spending by the Secretary: 21 

22 INITIAL NATIONAL PRIORITIES FOR CER In addition, $400,000,000 shall be available for comparative effectiveness research to be allocated at the discretion of the Secretary of Health and Human Services (“Secretary”) . . . to accelerate the development and dis- semination of research assessing the comparative effectiveness of health care treatments and strategies, through efforts that: (1) conduct, support, or synthesize research that compares the clinical outcomes, effectiveness, and appropriateness of items, services, and procedures that are used to prevent, diagnose, or treat diseases, disorders, and other health conditions; and (2) encourage the development and use of clinical registries, clinical data networks, and other forms of electronic health data that can be used to generate or obtain outcomes data. The legislation also directs the Secretary to enter into a contract with the Institute of Medicine (IOM), under which the IOM should make rec- ommendations to guide the nation’s priorities for CER and specifically to be taken into consideration by the Secretary in decisions on expenditure of the $400 million available for CER: [T]he Secretary shall enter into a contract with the Institute of Medicine . . . to produce and submit a report to the Congress and the Secretary by not later than June 30, 2009, that includes recommendations on the national priorities for comparative effectiveness research to be conducted or supported with the funds provided in this paragraph and that considers input from stakeholders. This report is the IOM’s response to the congressional mandate. In addition to the federal support for the project, the IOM received support both from the National Academies President’s Fund to finance the project until the federal sponsor could contract with the IOM and to undertake the complete cost of the questionnaire process described in Chapter 3, and from the Robert Wood Johnson Foundation for support of the study director. study scope Pursuant to the congressional mandate, the IOM committee established to carry out the study was charged with obtaining extensive stakeholder in- put for the formulation of national priorities for the Secretary’s investment of the ARRA funds for CER. The Governing Board Executive Committee of the National Research Council, an arm of the National Academies, authorized the study emphasizing stakeholder input (Box 1-1). After con- sultation with congressional staff and AHRQ, the administrative sponsor of the study, the committee concluded that its scope of work encompassed three principal tasks:   American Recovery and Reinvestment Act of 2009, P.L. 111-5, 111th Congress, 1st ses- sion (February 17, 2009). 

INTRODUCTION 23 BOX 1-1 Charge to the IOM Committee on Comparative Effectiveness Research Prioritization An ad hoc committee will conduct a study to recommend national priorities for comparative effectiveness research to be conducted or supported with funds from the American Recovery and Reinvestment Act of 2009. The study will be informed by and extend the views of stakeholders and the recent and ongoing IOM work rel- evant to comparative effectiveness research such as that on the national capacity to identify what works in health care, standards for systematic reviews of evidence, and standards for developing trustworthy clinical practice guidelines. 1.  obtain national input from a wide variety of stakeholders, in- To cluding the public, patients, families, and health care providers in order to develop a list of no fewer than 50 recommended priority CER topics. 2.  define how these recommended priorities could be incorporated To in a balanced portfolio of priority research that encompasses all age groups, underrepresented subpopulations in clinical research, the full care continuum from prevention to diagnosis to monitoring to treatment to end-of-life care, the complete range of health care services from the least to the most invasive, and strategies to ensure rapid and effective translation of knowledge into practice. 3.  recommend priority actions for ensuring the infrastructure and To workforce for a long-term, sustainable national CER enterprise. Committee Formation and Procedures The legislation was signed February 17, 2009, and the IOM appointed most of the Committee on Comparative Effectiveness Research Prioritiza- tion on February 28, 2009, with a final few members in mid-March. The 23-member committee included experts in behavioral health, bioethics, biostatistics, child health, clinical trials, consumer and patient perspec- tives, disabilities, drug development, geriatrics, health care delivery, health care policy, health economics, health insurance, internal medicine, preven- tion, public health, racial and ethnic disparities, surgery, systematic review methods, and women’s health. Brief biographies of the committee members appear in Appendix F. The study required an intense, focused effort across just 19 weeks from 

24 INITIAL NATIONAL PRIORITIES FOR CER the enactment of ARRA to release of a final, peer-reviewed report. The committee began by clarifying the scope of work and developing and imple- menting an approach to obtaining public and stakeholder suggestions for the Secretary’s CER priorities. The committee identified three mechanisms feasible within its time constraints to achieve the requested stakeholder input (see Chapter 3 for details): 1. Direct input via email and letter correspondence through the IOM website 2.  web-based questionnaire, open to all, asking for specific priority A research recommendations and their justification 3.  day-long public session for stakeholder presentations to the A committee By March 6, 2009, a web-based questionnaire had been developed and field tested to obtain public input into priorities. The questionnaire was active on the project’s website starting that same day and a broadcast an- nouncement was emailed on March 9 to approximately 20,000 recipients, including everyone on the IOM listservs and targeted organizations in- volved in health care announcing all three opportunities for public input. In particular, public, consumer, and patient input was solicited by direct con- tact with major consumer and patient advocacy organizations (e.g., AARP, Consumers Union, National Health Council, National Minority Quality Forum). Despite a very short period to notify the public of this process, and the equally short time for the public to submit information to the committee for consideration and voting, the committee received extensive input from more than 1,700 individuals. Although this input meets the requirements of the legislative language, the committee clearly concludes that future efforts to establish research priorities need to provide more extensive opportunities for public input and discussion (see Chapters 3, 4, 5, and 6). Over the course of the study, the committee held two in-person meet- ings (one in conjunction with the public session, and one during an extended weekend retreat) and three phone conferences. Several workgroups of com- mittee members also communicated by telephone conference and other elec- tronic means to address specific tasks such as the committee’s methods for incorporating public input, consideration of selection criteria for developing a list of priority CER questions, and assessment of key issues related to infra- structure and the long-term sustainability of a national CER enterprise. STUDY Context The IOM has been integrally involved in national priority setting in the past. Almost 20 years ago, the IOM addressed the clinical conditions that 

INTRODUCTION 25 the Health Care Financing Agency (now the Centers for Medicare & Med- icaid Services [CMS]) should prioritize as part of their Effectiveness Initia- tive. The reports set priorities for effectiveness research on acute myocardial infarction, hip fractures, and breast cancer (IOM, 1990a,b,e). Subsequently, the IOM recommended methodologies that NIH should utilize to improve consensus development in evaluating biomedical technologies and practices. This study was one part of a three-part examination of group judgment methods for assessing medical technologies (IOM, 1990d). An additional report set forth criteria and methods for deciding which health care tech- nologies to evaluate (IOM, 1992). A series of reports addressed methods for guideline development. These reports focused on the optimal methods for setting priorities for clinical guideline topics (IOM, 1990c, 1995). Priority setting in the allocation of NIH research funding and mechanisms of involv- ing the public were addressed in a report examining how NIH should set its research priorities, looking at four issues: (1) allocation criteria, (2) the decision-making process, (3) mechanisms for public input, and (4) impact of congressional directives (IOM, 1998). More recently, as part of the Quality Chasm series, the IOM dealt with shortfalls in the quality of health care in the United States (IOM, 2003). It recommended criteria for which priorities should be established for quality improvement efforts, as well as specific priority disease entities and condi- tions including care coordination, health literacy, and end-of-life issues. Much of the CER committee’s efforts have been guided by the findings and recommendations of the IOM Committee on Reviewing Evidence to Identify Highly Effective Clinical Services found in Knowing What Works in Health Care (IOM, 2008). In addition, the CER committee took full ad- vantage of the extensive work of the IOM Roundtable on Evidence-Based Medicine’s experience in The Learning Healthcare System (IOM, 2007a) and Learning What Works Best (IOM, 2007b) in identifying the impor- tance of public input for priority setting, potential models of governance for a national program of CER, potential methodologies for conducting CER, and the requisite workforce to accomplish the task at hand. The Committee on Comparative Effectiveness Research Prioritization operated in parallel with the Federal Coordinating Council for CER, which was also authorized in ARRA. This council consists of 15 members, “all of whom are senior federal officers or employees with responsibility for health- related programs, appointed by the President, acting through the Secretary of Health and Human Services.” Its charge, like that of this committee, is “not later than June 30, 2009, the Council shall submit to the President and the Congress a report containing information describing current federal   American Recovery and Reinvestment Act of 2009, P.L. 111-5, 111th Congress, 1st ses- sion (February 17, 2009). 

26 INITIAL NATIONAL PRIORITIES FOR CER activities on comparative effectiveness research and recommendations for such research conducted or supported from funds made available for allot- ment by the Secretary for comparative effectiveness research in this Act.” Additionally, “the Council shall submit to the President and Congress an annual report regarding its activities and recommendations concerning the infrastructure needs, organizational expenditures and opportunities for bet- ter coordination of comparative effectiveness research by relevant federal departments and agencies.” The IOM committee intends its report to serve as a complementary document to that of the Council, which may serve as the blueprint for federal efforts to develop a structure and process for the implementation of CER efforts. Organization of the Report This introductory chapter has described the context for this report, including related past IOM studies, the committee’s charge, and the objec- tives, scope, and study methods for this report. Subsequent chapters address the following topics: • Chapter 2—What Is Comparative Effectiveness Research? This chapter has two primary objectives: first, to establish a conceptual framework for CER by defining key terms and research methods, and second, to describe several current private and public CER programs. • Chapter 3—Obtaining Input to Identify National Priorities for Com- parative Effectiveness Research documents the committee’s methods for soliciting stakeholder input and nominations for priority CER topics. Direct communications by letter and email are described, pre- sentations at the open meeting are reviewed, and the questionnaire soliciting nominations for priority topics is presented in detail. The distribution of the public nominations is presented with their clinical characteristics pertinent to the portfolio distribution. • Chapter 4—The Criteria and Process for Setting Priorities describes priority selection criteria used in past IOM committee initiatives and presents the committee’s recommendations. It further lays out the concept of the “portfolio,” by which the committee proposes to establish balance and scope of the priorities. Finally, it describes the process by which more than 2,600 nominated CER topics were narrowed to the final list of 100 priority CER topics. Recom- mendations are presented for a sustained priority setting process moving forward. 

INTRODUCTION 27 • Chapter 5—Priorities for Study presents the committee’s portfolio and list of recommended national priority topics for CER to be conducted or supported with the Secretary’s portion of funds from ARRA 2009. • Chapter 6—Essential Priorities for a Robust CER Enterprise ex- plains the imperative for effective coordination of the HHS Secre- tary’s sustained CER strategy and outlines four essential program priorities: (1) ensuring meaningful consumer, patient, and caregiver participation; (2) building robust information systems including research and innovation in the methods of CER; (3) development and support of a highly skilled CER workforce; and (4) vigorous support of research and efforts to translate CER knowledge into everyday clinical practice. references IOM (Institute of Medicine). 1990a. Acute myocardial infarction: Setting priorities for effec- tiveness research. Edited by P. H. Mattingly and K. N. Lohr. Washington, DC: National Academy Press. ———. 1990b. Breast cancer: Setting priorities for effectiveness research. Washington, DC: National Academy Press. ———. 1990c. Clinical practice guidelines: Directions for a new program. Edited by M. J. Field and K. N. Lohr. Washington, DC: National Academy Press. ———. 1990d. Consensus development at the NIH: Improving the program Washington, DC: National Academy Press. ———. 1990e. Hip fracture: Setting priorities for effectiveness research. Edited by K. A. Heithoff and L. K. N. Washington, DC: National Academy Press. ———. 1992. Setting Priorities for Health Technology Assessment: A Model Process. Edited by M. S. Donaldson and H. C. Sox. Washington, DC: National Academy Press. ———. 1995. Setting priorities for clinical practice guidelines. Edited by M. J. Field. Wash- ington, DC: National Academy Press. ———. 1998. Scientific opportunities and public needs: Improving priority setting and public input at the National Institutes of Health. Washington, DC: National Academy Press. ———. 2003. Priority areas for national action: Transforming health care quality. Edited by K. Adams and J. Corrigan, Quality chasm series; Variation: Quality chasm series. Washington, DC: The National Academies Press. ———. 2007a. The Learning healthcare system: Workshop summary. The IOM Roundtable on Evidence-Based Medicine. Edited by L. Olsen, D. Aisner, and J. M. McGinnis. Wash- ington, DC: The National Academies Press. ———. 2007b. Learning what works best: The nation’s need for evidence on comparative effectiveness in health care. http://www.iom.edu/ebm-effectiveness (accessed April 15, 2009). ———. 2008. Knowing what works in health care: A roadmap for the nation. Edited by J. Eden, B. Wheatley, B. J. McNeil, and H. Sox. Washington, DC: The National Academies Press.