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INITIAL NATIONAL PRIORITIES FOR COMPARATIVE EFFECTIVENESS RESEARCH Committee on Comparative Effectiveness Research Prioritization Board on Health Care Services

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THE NATIONAL ACADEMIES PRESS 500 Fifth Street, N.W. Washington, DC 20001 NOTICE: The project that is the subject of this report was approved by the Govern- ing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineer- ing, and the Institute of Medicine. The members of the committee responsible for the report were chosen for their special competences and with regard for appropri- ate balance. This study was supported by Task Order number HHSP 23337002T and Contract number HHSP 23320042509XI between the National Academy of Sciences and the Agency for Healthcare Research and Quality, by the National Academies President’s Fund, and by the Robert Wood Johnson Foundation’s Health Policy Fellowships. Any opinions, findings, conclusions, or recommendations expressed in this publica- tion are those of the author(s) and do not necessarily reflect the view of the organi- zations or agencies that provided support for this project. International Standard Book Number-13: 978-0-309-13836-9 International Standard Book Number-10: 0-309-13836-1 Library of Congress Control Number: 2009934993 Additional copies of this report are available from the National Academies Press, 500 Fifth Street, N.W., Lockbox 285, Washington, DC 20055; (800) 624-6242 or (202) 334-3313 (in the Washington metropolitan area); Internet, http://www.nap. edu. For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu. Copyright 2009 by the National Academy of Sciences. All rights reserved. Printed in the United States of America The serpent has been a symbol of long life, healing, and knowledge among almost all cultures and religions since the beginning of recorded history. The serpent ad- opted as a logotype by the Institute of Medicine is a relief carving from ancient Greece, now held by the Staatliche Museen in Berlin. Suggested citation: IOM (Institute of Medicine). 2009. Initial National Priorities for Comparative Effectiveness Research. Washington, DC: The National Academies Press.

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“Knowing is not enough; we must apply. Willing is not enough; we must do.” — Goethe Advising the Nation. Improving Health.

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The National Academy of Sciences is a private, nonprofit, self-perpetuating society of distinguished scholars engaged in scientific and engineering research, dedicated to the furtherance of science and technology and to their use for the general welfare. Upon the authority of the charter granted to it by the Congress in 1863, the Acad- emy has a mandate that requires it to advise the federal government on scientific and technical matters. Dr. Ralph J. Cicerone is president of the National Academy of Sciences. The National Academy of Engineering was established in 1964, under the charter of the National Academy of Sciences, as a parallel organization of outstanding en- gineers. It is autonomous in its administration and in the selection of its members, sharing with the National Academy of Sciences the responsibility for advising the federal government. The National Academy of Engineering also sponsors engineer- ing programs aimed at meeting national needs, encourages education and research, and recognizes the superior achievements of engineers. Dr. Charles M. Vest is presi- dent of the National Academy of Engineering. The Institute of Medicine was established in 1970 by the National Academy of Sciences to secure the services of eminent members of appropriate professions in the examination of policy matters pertaining to the health of the public. The Insti- tute acts under the responsibility given to the National Academy of Sciences by its congressional charter to be an adviser to the federal government and, upon its own initiative, to identify issues of medical care, research, and education. Dr. Harvey V. Fineberg is president of the Institute of Medicine. The National Research Council was organized by the National Academy of Sci- ences in 1916 to associate the broad community of science and technology with the Academy’s purposes of furthering knowledge and advising the federal government. Functioning in accordance with general policies determined by the Academy, the Council has become the principal operating agency of both the National Academy of Sciences and the National Academy of Engineering in providing services to the government, the public, and the scientific and engineering communities. The Coun- cil is administered jointly by both Academies and the Institute of Medicine. Dr. Ralph J. Cicerone and Dr. Charles M. Vest are chair and vice chair, respectively, of the National Research Council. www.national-academies.org

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In MeMorIaM This report is dedicated to Maria Carolina Hinestrosa, a hard-working and devoted committee member who, while stricken with cancer, continued to work tirelessly on this report until its completion.

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COMMITTEE ON COMPARATIvE EFFECTIvENESS RESEARCH PRIORITIzATION HAROLD C. SOx (Co-Chair), Editor, Annals of Internal Medicine, American College of Physicians of Internal Medicine, Philadelphia, PA SHELDON GREENFIELD (Co-Chair), Donald Bren Professor of Medicine and Executive Director, Center for Health Policy Research, University of California, Irvine CHRISTINE K. CASSEL, President and CEO, American Board of Internal Medicine, Philadelphia, PA KAy DICKERSIN, Professor of Epidemiology, Director, Center for Clinical Trials and Director, United States Cochrane Center, Johns Hopkins Bloomberg School of Public Health, Baltimore, MD ALAN M. GARbER, Henry J. Kaiser, Jr. Professor and Professor of Medicine, Director, Center for Health Policy and Center for Primary Care and Outcomes Research, Stanford University, Stanford, CA CONSTANTINE GATSONIS, Professor of Medical Science (Biostatistics) and Director, Center for Statistical Sciences, Brown University, Providence, RI GARy L. GOTTLIEb, President, Brigham and Women’s Hospital, Professor of Psychiatry, Harvard Medical School, Boston, MA JAMES A. GuEST, President and CEO, Consumers Union, Yonkers, NY MARK HELFAND, Professor of Medicine and Director, Oregon Evidence-based Practice Center, Oregon Health and Science University, and Staff Physician, Portland VAMC, Portland MARIA CAROLINA HINESTROSA,* Executive Vice President for Programs and Planning, National Breast Cancer Coalition, Co- Founder, Nueva Vida, Washington, DC GEORGE J. ISHAM, Medical Director and Chief Health Officer, HealthPartners, Inc., Bloomington, MN ARTHuR A. LEvIN, Director, Center for Medical Consumers, New York JOANN E. MANSON, Professor of Medicine and the Elizabeth Fay Brigham Professor of Women’s Health, Harvard Medical School, Chief of the Division of Preventive Medicine, Brigham and Women’s Hospital, Boston, MA KATIE MASLOW, Director, Policy Development, Alzheimer’s Association, Washington, DC *Deceased. vii

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MARK b. McCLELLAN, Director, Engelberg Center for Health Care Reform, The Brookings Institution, Washington, DC SALLy C. MORTON, Vice President for Statistics and Epidemiology, RTI International, Research Triangle Park, NC NEIL R. POWE, Chief, Medical Services, San Francisco General Hospital Professor and Vice Chairman, Department of Medicine, University of California, San Francisco JOE v. SELby, Director, Division of Research, Kaiser Permanente, Oakland, CA LISA SIMPSON, Director, Child Policy Research Center, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH SEAN TuNIS, Founder and Director, Center for Medical Technology Policy, Baltimore, MD I. STEvEN uDvARHELyI, Senior Vice President and Chief Medical Officer, Independence Blue Cross, Philadelphia, PA A. EuGENE WASHINGTON, Executive Vice Chancellor and Provost, University of California, San Francisco JAMES N. WEINSTEIN, Dartmouth College Third Century Professor, Director, The Dartmouth Institute for Health Policy and Clinical Practice; Professor and Chair, Department of Orthopedic Surgery, Dartmouth Medical School and Vice Chair, Board of Governors, Dartmouth-Hitchcock Medical Center, Lebanon, NH Study Staff ROGER HERDMAN, Board Director RObERT RATNER, Study Director JILL EDEN, Senior Program Officer DIANNE MILLER WOLMAN, Senior Program Officer SALLy RObINSON, Program Officer LAuRA LEvIT, Associate Program Officer LEA GREENSTEIN, Research Associate MICHELLE MANCHER, Research Associate ALLISON McFALL, Senior Program Assistant REDA uRMANAvICIuTE, Administrative Assistant HARRIET CRAWFORD, IT Project Manager DWAyNE bELL, Programmer Analyst viii

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Consultants JOSHuA bENNER, The Brookings Institution STEvEN PEARSON, Institute for Clinical and Economic Review, Harvard Medical School NEIL WEISFELD, NEW Associates vICTORIA WEISFELD, NEW Associates ix

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Reviewers This report has been reviewed in draft form by individuals chosen for their diverse perspectives and technical expertise, in accordance with procedures approved by the National Research Council’s Report Review Committee. The purpose of this independent review is to provide candid and critical comments that will assist the institution in making its published report as sound as possible and to ensure that the report meets institutional standards for objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confidential to protect the integrity of the deliberative process. We wish to thank the following individuals for their review of this report: RHONDA J. RObINSON bEALE, United Behavioral Health MARC bOuTIN, National Health Council ELLEN WRIGHT CLAyTON, Center for Biomedical Ethics and Society, Vanderbilt University DON E. DETMER, American Medical Informatics Association ERIC b. LARSON, Group Health, Center for Health Studies DAvID O. MELTzER, Pritzker School of Medicine, University of Chicago GARy A. PuCKREIN, National Minority Quality Forum RICHARD SCHILSKy, Biological Sciences Division, University of Chicago Medical Center xi

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xiv FOREWORD This report is the product of the committee’s effort. Drawing on an ex- tensive body of evidence, including input from lay and professional bodies, stakeholders, researchers, and policy makers, the committee has produced a well-grounded report. More than a list of priority topics, this report clarifies the meaning of comparative effectiveness and sets forth criteria for choosing both individual topics and the portfolio of topics for comparative effective- ness research. It is our hope that this document will prove valuable both as an immediate indicator of priorities and as an ongoing guide to the future selection of new subjects for assessment. Harvey V. Fineberg, M.D., Ph.D. President, Institute of Medicine June 2009

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Preface The U.S. Congress mandated this study in the American Recovery and Reinvestment Act of 2009, which the President signed into law on Febru- ary 17, 2009. The legislation required the Institute of Medicine (IOM) to convene a committee to establish a list of research questions that would have the highest priority for study with comparative effectiveness research (CER) funds that the law placed at the discretion of the Secretary of Health and Human Services. Moreover, the law required the committee to seek advice from stakeholders who might benefit from the research: researchers, physicians, professional organizations, and the general public. Basing its approach on methods developed by the Agency for Healthcare Research and Quality, the committee held a public meeting to get advice from pro- fessional and consumer groups and from the general public and solicited nominations for research questions through a web-based questionnaire. The committee developed a process for deciding which conditions to place on its list of the highest priority research questions, and, over a 10-day period, winnowed over a thousand nominations to a list of 100 high prior- ity topics. The principal products of the committee’s work are a definition of CER, a list of 100 priority topics, and 10 recommendations. To guide its work, the committee developed a working definition of CER, using features of definitions offered by other organizations. The priority list contains 100 research questions divided into four quartiles. The committee discussed each question and refined the wording of most of them, while still striving to preserve the spirit of the original nomination. Finally, the committee xv

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xvi PREFACE developed 10 recommendations for an infrastructure to support a national system for conducting CER. We believe that these elements of our report will help to establish the groundwork for a research program that will serve the nation well. Producing a full IOM report in just over 4 months required an intense, sustained effort. On very short notice, nominees to serve on the committee found time in their schedules to attend 5 days of meetings and spend many hours reading the dossiers of hundreds of research questions and deciding which were the most important. The IOM assembled an experienced, out- standing study staff who simply worked miracles day after day. Collectively, we had the pleasure of working together on a task whose importance was self-evident and the honor of serving our country. Harold C. Sox, Co-Chair Sheldon Greenfield, Co-Chair Committee on Comparative Effectiveness Research Prioritization

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Acknowledgments The committee and staff are indebted to a number of individuals and organizations for their contributions to this report. We extend thanks to the following individuals for their assistance in gathering the objective data that the committee used in its voting and pri- oritization process. Christina bethell, Oregon Health & Science University, School of Medicine James A. Schuttinga, Division of Program Coordination, Planning, and Strategic Initiatives, National Institutes of Health Nancy Sonnenfeld, National Center for Health Statistics Deborah A. zarin, ClinicalTrials.gov, National Library of Medicine We extend thanks to the following individuals who piloted and pro- vided feedback on the web-based questionnaire. Raymond J. baxter, Kaiser Permanente Melanie bella, Center for Health Care Strategies Kathleen buto, Johnson & Johnson Denis A. Cortese, Mayo Clinic Nancy Derr, Food and Drug Administration Daniel M. Fox, Milbank Memorial Fund Jean Paul Gagnon, Sanofi Aventis Mark Gibson, Oregon Health & Science University, Center for Evidence-based Policy xvii

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xviii ACKNOWLEDGMENTS Carmen Hooker Odom, Milbank Memorial Fund Richard A. Justman, United HealthCare Michael S. Lauer, National Institutes of Health Sandy Leonard, AstraZeneca Samuel R. Nussbaum, WellPoint, Inc. Steven D. Pearson, Institute for Clinical and Economic Review, Harvard Medical School John W. Rowe, Columbia University Edward M. Rozynski, Stryker John Santa, Consumers Union Chad Shearer, Center for Health Care Strategies Jeffrey Shuren, Food and Drug Administration Jean Slutsky, Agency for Healthcare Research and Quality Karen Smith, AstraZeneca Stephen A. Somers, Center for Health Care Strategies Frank Torres, Microsoft Corporation The individuals who testified before the committee during the public meeting are all listed in Appendix A and their written testimony is avail- able at www.iom.edu/cerpriorities. The committee appreciates all 1,758 individuals who responded to its questionnaire, either for themselves or their organization. We especially thank Joshua Benner and Steven Pearson for their valuable contribution to Chapter 2 of the report. Funding for this study was provided by the National Academies Presi- dent’s Fund, the Agency for Healthcare Research and Quality, and the Rob- ert Wood Johnson Foundation. The committee appreciates the opportunity and support for the development of this report. Finally, many within the Institute of Medicine were helpful to the study staff. The staff would especially like to thank Clyde Behney, Patrick Burke, William McLeod, Abbey Meltzer, LeighAnne Olsen, Lauren Tobias, Jackie Turner, and Jordan Wyndelts.

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Contents SUMMARY 1 1 INTRODUCTION 21 Study Scope, 22 Committee Formation and Procedures, 23 Study Context, 24 Organization of the Report, 26 References, 27 2 WHAT IS COMPARATIVE EFFECTIVENESS RESEARCH? 29 The Need for More and Better Evidence of What Works in Health Care, 30 Defining Comparative Effectiveness Research, 33 Characteristics of CER, 37 Examples of CER Studies: Coronary Artery Disease, 42 Existing CER Activity in the United States, 46 Conclusion, 54 References, 56 3 OBTAINING INPUT TO IDENTIFY NATIONAL PRIORITIES FOR COMPARATIVE EFFECTIVENESS RESEARCH 61 Introduction, 61 Invitations to Provide Input, 62 Communications Directly to the Committee, 62 Presentations at an Open Meeting of Stakeholders, 65 xix

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xx CONTENTS Input from a Web-Based Questionnaire, 68 Reference, 75 4 THE CRITERIA AND PROCESS FOR SETTING PRIORITIES 77 Introduction, 77 Portfolio Considerations, 78 Criteria Chosen for Priority Setting, 80 Data Collection to Aid Topic Selection, 83 Lessons from Previous Priority-Setting Processes, 84 Voting Procedures, 90 Lessons Learned from the Current Prioritization Process and Committee Recommendations, 92 References, 94 5 PRIORITIES FOR STUDY 97 Assembling a Diverse Portfolio, 98 Diversity of Research Areas, 99 Diversity of Populations, 102 Diversity of Interventions, 103 Diversity of Study Methodologies, 104 Introduction to Final List of Priority Topics, 105 Discussion of the Priority Topics by Research Area, 116 Timeliness and Limitations of the Committee’s Priority List, 136 References, 137 6 ESSENTIAL PRIORITIES FOR A ROBUST CER ENTERPRISE 139 The Imperative for Effective Coordination of the CER Enterprise, 140 Meaningful Consumer, Patient, and Caregiver Engagement, 142 Robust Data and Information Systems, 146 Develop, Deploy, and Support a CER Workforce, 155 Bringing Knowledge into Practice, 159 Conclusion, 159 References, 160 APPENDIXES A Public Meeting Agenda—March 20, 2009 167 B Stakeholder Questionnaire 171 C Data Tables: Burden of Disease and Variation of Care 189 D Cardiovascular and Peripheral Vascular Cover Sheet 199 E Definitions of Medical Terminology in CER Priority List 203 F Committee Biographies 213

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Boxes, Figures, and Tables Summary Table S-1 Final List of 100 Priority Topics, by Quartile Ratings, 3 Chapter 1 Box 1-1 Charge to the IOM Committee on Comparative Effectiveness Research Prioritization, 23 Chapter 2 Box 2-1 Methods Commonly Used in CER, 40 Tables 2-1 Definitions of CER, 35 2-2 Selected CER Studies of Management of Acute Coronary Syndrome, 44 Chapter 3 Box 3-1 Organizations Represented at the Stakeholder Meeting, 66 xxi

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xxii BOXES, FIGURES, AND TABLES Figure 3-1 Stakeholder response to web-based questionnaire, 69 Tables 3-1 Solicited Stakeholder Groups, 63 3-2 Respondents to the IOM Questionnaire by Stakeholder Category, 70 3-3 Comparative Effectiveness Research Priorities Submitted by Primary Area of Study, 71 3-4 Comparative Effectiveness Research Priorities by Proposed Population to Be Studied, 72 3-5 Comparative Effectiveness Research Priorities by Proposed Intervention, 72 3-6 Comparative Effectiveness Research Priorities by Proposed Study Methodology, 73 Chapter 4 Figure 4-1 Voting process and selection of priority topics, 91 Tables 4-1 Portfolio and Priorities Criteria, 79 4-2 Criteria and Priorities for Quality Improvement, 86 4-3 A Variety of Priority-Setting Initiatives and Their Selected Criteria, 87 Chapter 5 Box 5-1 Round 3 Voting Procedure, 106 Figure 5-1 Distribution of the recommended research priorities by primary and secondary research areas, 101 Tables 5-1 Recommended Research Priorities by Research Area, 100 5-2 Committee’s Recommended Research Priorities by Study Populations, 103 5-3 Committee’s Recommended Research Priorities by Types of Intervention, 104

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xxiii BOXES, FIGURES, AND TABLES 5-4 Committee’s Recommended Research Priorities by Study Methodology, 105 5-5 Results of the IOM Committee’s Final Vote for Priority Topics, by Quartile, 106 5-6 Final List of Priority Topics, by Quartile Ratings, 107 5-7 Health Care Delivery Systems Priority Topics, 118 5-8 Cardiovascular and Peripheral Vascular Diseases Priority Topics, 121 5-9 Psychiatric Disorders Priority Topics, 122 5-10 Neurologic Disorders Priority Topics, 123 5-11 Oncology and Hematology Priority Topics, 124 5-12 Women’s Health Priority Topics, 124 5-13 Musculoskeletal Disorders Priority Topics, 125 5-14 Infectious Disease and Liver and Biliary Tract Disorder Priority Topics, 126 5-15 Endocrinology and Metabolism Disorders and Geriatric Priority Topics, 127 5-16 Birth and Developmental Disorders Priority Topics, 128 5-17 Complementary and Alternative Medicine Priority Topics, 129 5-18 Nutrition Priority Topics, 130 5-19 Race and Ethnic Disparities Priority Topics, 130 5-20 Skin Disorders Priority Topics, 131 5-21 Alcoholism, Drug Dependency, and Overdose Priority Topics, 131 5-22 Functional Limitations and Disability Priority Topics, 132 5-23 Ears, Eyes, Nose, and Throat Disorders Priority Topics, 132 5-24 Kidney and Urinary Tract Disorders Priority Topics, 133 5-25 Oral Health Priority Topics, 133 5-26 Palliative and End-of-Life Care Priority Topics, 134 5-27 Gastrointestinal System Disorders Priority Topics, 134 5-28 Immune System, Connective Tissue, and Joint Disorders Priority Topics, 135 5-29 Pediatric Disorders Priority Topics, 135 5-30 Respiratory Disorders Priority Topics, 136 5-31 Trauma, Emergency Medicine, and Critical Care Medicine Priority Topics, 136 Chapter 6 Box 6-1 IOM Recommendations for Changes to the HIPAA Privacy Rule and Associated Guidance Beyond the HIPAA Privacy Rule: Enhancing Privacy, Improving Health Through Research, 156

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