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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
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Copyright 2009 by the National Academy of Sciences. All rights reserved.
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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
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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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