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Victoria Weisfeld, Rebecca A. English, and
Anne B. Claiborne, Rapporteurs
Forum on Drug Discovery, Development, and Translation
Board on Health Sciences Policy
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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.
This study was supported by contracts between the National Academy of Sciences
and Department of Health and Human Services (Contract Nos. N01-OD-4-2139
TO No. 158 and HHSF223001003T), American Society for Microbiology; Amgen
Inc.; Association of American Medical Colleges; Bristol-Myers Squibb; Burroughs
Wellcome Fund; Celtic Therapeutics, LLLP; Critical Path Institute; Doris Duke
Charitable Foundation; Eli Lilly and Co.; FasterCures; Foundation for the NIH;
Friends of Cancer Research; GlaxoSmithKline; Johnson & Johnson; Merck and Co.,
Inc.; Novartis Pharmaceuticals Corporation; and Pfizer Inc. Any opinions, find-
ings, conclusions, or recommendations expressed in this publication are those of
the authors and do not necessarily reflect the view of the organizations or agencies
that provided support for this project.
International Standard Book Number-13: 978-0-309-21929-7
International Standard Book Number-10: 0-309-21929-9
Additional copies of this report are available from the National Academies Press,
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For more information about the Institute of Medicine, visit the IOM home page at:
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Copyright 2012 by the National Academy of Sciences. All rights reserved.
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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). 2012. Public Engagement and Clini-
cal Trials: New Models and Disruptive Technologies: Workshop Summary. 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 soci-
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the Academy 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 engi-
neering programs aimed at meeting national needs, encourages education and re-
search, and recognizes the superior achievements of engineers. Dr. Charles M. Vest
is president of the National Academy of Engineering.
The Institute of Medicine was established in 1970 by the National Academy of
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stitute acts under the responsibility given to the National Academy of Sciences
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Harvey V. Fineberg is president of the Institute of Medicine.
The National Research Council was organized by the National Academy of Sci-
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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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PLANNING COMMITTEE FOR THE WORKSHOP
SERIES ON IMPROVING THE CLINICAL TRIAL
PROCESS IN THE UNITED STATES1
JEFFREY M. DRAZEN (Chair), New England Journal of Medicine, Boston, MA
BARBARA ALVING,2 National Center for Research Resources,
Bethesda, MD
ANN BONHAM, Association of American Medical Colleges,
Washington, DC
LINDA BRADY, National Institute of Mental Health, Bethesda, MD
ROBERT CALIFF, Duke University Medical Center, Durham, NC
SCOTT CAMPBELL, Foundation for the National Institutes of Health,
Bethesda, MD
GAIL H. CASSELL, Harvard Medical School (visiting), Carmel, IN
JAMES H. DOROSHOW, National Cancer Institute, Bethesda, MD
PAUL R. EISENBERG, Amgen Inc., Thousand Oaks, CA
GARY L. FILERMAN, Atlas Health Foundation, McLean, VA
GARRET A. FITZGERALD, University of Pennsylvania School of
Medicine, Philadelphia
ANNALISA JENKINS,3 Merck Serono, Geneva
PETRA KAUFMANN, National Institute of Neurological Disorders and
Stroke, Bethesda, MD
RONALD L. KRALL, University of Pennsylvania, Center for Bioethics,
Steamboat Springs, CO
FREDA LEWIS-HALL, Pfizer Inc., New York, NY
ELLEN SIGAL, Friends of Cancer Research, Washington, DC
JANET TOBIAS, Ikana Media and Mount Sinai School of Medicine,
New York, NY
JANET WOODCOCK, Food and Drug Administration, White Oak, MD
IOM Staff
ANNE B. CLAIBORNE, Forum Director
REBECCA A. ENGLISH, Associate Program Officer
ELIZABETH F. C. TYSON, Research Associate
ANDREW M. POPE, Director, Board on Health Sciences Policy
ROBIN GUYSE, Senior Program Assistant
1 Institute of Medicine planning committees are solely responsible for organizing the
workshop, identifying topics, and choosing speakers. The responsibility for the published
workshop summary rests with the workshop rapporteurs and the institution.
2 Barbara Alving was with National Center for Research Resources until September 30,
2011.
3 Annalisa Jenkins was with Bristol-Myers Squibb as of the dates of the workshop.
v
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FORUM ON DRUG DISCOVERY,
DEVELOPMENT, AND TRANSLATION1
JEFFREY M. DRAZEN (Co-Chair), New England Journal of Medicine,
Boston, MA
STEVEN K. GALSON (Co-Chair), Amgen Inc., Thousand Oaks, CA
MARGARET ANDERSON, FasterCures, Washington, DC
HUGH AUCHINCLOSS, National Institute of Allergy and Infectious
Diseases, Bethesda, MD
LESLIE Z. BENET, University of California-San Francisco
ANN BONHAM, Association of American Medical Colleges,
Washington, DC
LINDA BRADY, National Institute of Mental Health, Bethesda, MD
ROBERT CALIFF, Duke University Medical Center, Durham, NC
SCOTT CAMPBELL, Foundation for the National Institutes of Health,
Bethesda, MD
C. THOMAS CASKEY, Baylor College of Medicine, Houston, TX
GAIL H. CASSELL, Harvard Medical School (visiting), Carmel, IN
PETER B. CORR, Celtic Therapeutics, LLLP, New York, NY
ANDREW M. DAHLEM, Eli Lilly and Company, Indianapolis, IN
JAMES H. DOROSHOW, National Cancer Institute, Bethesda, MD
GARY L. FILERMAN, Atlas Health Foundation, McLean, VA
GARRET A. FITZGERALD, University of Pennsylvania School of
Medicine, Philadelphia
HARRY B. GREENBERG, Stanford University School of Medicine,
Stanford, CA
STEPHEN GROFT, National Institutes of Health, Bethesda, MD
LYNN HUDSON, Critical Path Institute, Tuscon, AZ
THOMAS INSEL, National Center for Advancing Translational
Sciences, Bethesda, MD
PETRA KAUFMANN, National Institute of Neurological Disorders and
Stroke, Bethesda, MD
JACK D. KEENE, Duke University Medical Center, Durham, NC
RONALD L. KRALL, University of Pennsylvania, Center for Bioethics,
Steamboat Springs, CO
FREDA LEWIS-HALL, Pfizer Inc., New York, NY
MARK B. McCLELLAN, The Brookings Institution, Washington, DC
CAROL MIMURA, University of California-Berkeley
ELIZABETH (BETSY) MYERS, Doris Duke Charitable Foundation,
New York, NY
1Institute of Medicine forums and roundtables do not issue, review, or approve individual
documents. The responsibility for the published workshop summary rests with the work -
shop rapporteurs and the institution.
vi
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JOHN ORLOFF, Novartis Pharmaceuticals Corporation, East
Hanover, NJ
AMY PATTERSON, National Institutes of Health, Bethesda, MD
MICHAEL ROSENBLATT, Merck & Co., Inc., Whitehouse Station, NJ
JANET SHOEMAKER, American Society for Microbiology,
Washington, DC
ELLEN SIGAL, Friends of Cancer Research, Washington, DC
ELLIOTT SIGAL, Bristol-Myers Squibb, Princeton, NJ
NANCY SUNG, Burroughs Wellcome Fund, Research Triangle Park, NC
JANET TOBIAS, Ikana Media and Mount Sinai School of Medicine,
New York, NY
JOANNE WALDSTREICHER, Johnson & Johnson, Raritan, NJ
JANET WOODCOCK, Food and Drug Administration, White Oak, MD
IOM Staff
ANNE B. CLAIBORNE, Forum Director
REBECCA A. ENGLISH, Associate Program Officer
ELIZABETH F. C. TYSON, Research Associate
ANDREW M. POPE, Director, Board on Health Sciences Policy
ROBIN GUYSE, Senior Program Assistant
vii
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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 pub-
lished 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 process. We wish to thank the
following individuals for their review of this report:
Joan Finnegan Brooks, Patient Focused Market Research
Elmer Freeman, Center for Community Health Education Research
and Service, Inc.
Susan Love, Dr. Susan Love Research Foundation
Betsy Myers, Doris Duke Charitable Foundation
Although the reviewers listed above have provided many construc-
tive comments and suggestions, they were not asked to endorse the final
draft of the report before its release. The review of this report was overseen
by Carmen R. Green, University of Michigan Medical School. Appointed
by the Institute of Medicine, she was responsible for making certain that
ix
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x REVIEWERS
an independent examination of this report was carried out in accordance
with institutional procedures and that all review comments were carefully
considered. Responsibility for the final content of this report rests entirely
with the authors and the institution.
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Contents
ACRONYMS xv
1 INTRODUCTION 1
2 FRAMING THE PROBLEM 5
The Current Landscape of Clinical Trials, 5
Panel Presentations and Discussion, 11
3 RECRUITMENT CHALLENGES IN CLINICAL TRIALS FOR
DIFFERENT DISEASES AND CONDITIONS 17
Cardiovascular Surgery, 17
Working with Underserved Communities, 21
Mental Health, 27
4 MODELS FOR PUBLIC ENGAGEMENT 31
Love/Avon Army of Women, 31
23andMe, 33
ResearchMatch, 36
5 MESSAGES AND METHODS FOR PUBLIC ENGAGEMENT 39
Messages, 39
Messengers, 43
xi
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xii CONTENTS
6 THE MEDIA 49
Balanced Coverage, 49
Relationships with Researchers, 50
Relationships with the Audience, 51
Conflict of Interest, 52
7 NOVEL CLINICAL TRIAL DESIGNS 55
Adaptive Clinical Trial Designs, 55
Using Point-of-Care Clinical Trials to Create a Learning
Health Care System, 58
The Food and Drug Administration Perspective, 62
8 THE HEALTH SYSTEM’S STRUCTURE AND CULTURE 65
Building a Supportive Clinical Trials Environment in
Academia, 65
Public-Private Partnerships in Cancer Trials, 69
Engaging Community Physicians in Rheumatoid Arthritis
Research, 71
9 TOWARD A PATIENT-CENTERED STRATEGY FOR
CLINICAL TRIALS 75
The Changing Politics of Clinical Trial Engagement, 75
Closing Panel, 81
REFERENCES 85
APPENDIXES
A WORKSHOP AGENDA 89
B THE CLINICAL TRIALS PROCESS 99
C PARTICIPANT BIOGRAPHIES 101
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Table, Figure, and Boxes
TABLE
3-1 Comparison of Researcher and Participant Perspectives on Clinical
Trials, 23
FIGURE
4-1 A timeline comparing a conventional NIH clinical trial versus the
23andMe research model, 35
BOXES
2-1 Elements for Successful Clinical Trials, 6
2-2 Key Principles for Recruiting and Retaining People in Clinical
Trials, 13
3-1 Targeted Strategies to Manage Patient Recruitment, 22
5-1 An Urgent Case for Clear Messaging, 46
7-1 Point-of-Care Clinical Trial Pilot Study, 60
xiii
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xiv TABLE, FIGURE, AND BOXES
8-1 Building Connections Between Community Physicians and
Clinical Trials: Challenges and Potential Solutions, 66
9-1 Principles of Public Engagement Discussed During the
Workshop, 83
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Acronyms
ACC American College of Cardiology
ACTION Analgesic Clinical Trials Innovation, Opportunities,
and Networks
AHA American Heart Association
CABG coronary artery bypass graft
CBPR community-based participatory research
CDER FDA Center for Drug Evaluation and Research
CDRH FDA Center for Devices and Radiological Health
CF cystic fibrosis
CRO contract research organization
CTSA Clinical and Translational Science Awards
CTSN Cardiothoracic Surgical Trials Network
CTTI Clinical Trials Transformation Initiative
EHR electronic health record
FDA U.S. Food and Drug Administration
FURRThER Families Undergoing Risk Reduction Through Educational
Reinforcement
GIST gastrointestinal stromal tumor
HRA Health Research Alliance
xv
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xvi ACRONYMS
IND investigational new drug
IOM Institute of Medicine
IRB Institutional Review Board
LVAD left ventricular assist device
NCATS National Center for Advancing Translational Sciences
NHLBI National Heart, Lung, and Blood Institute
NIH National Institutes of Health
NIMHD National Institute on Minority Health and Health
Disparities
PDUFA Prescription Drug User Fee Act
PMA premarket approval
RAIN Rheumatoid Arthritis Investigational Network
REMATCH Randomized Evaluation of Mechanical Assistance for the
Treatment of Congestive Heart Failure
STICH Surgical Treatment for Ischemic Heart Failure
TEAR Treatment of Early Aggressive Rheumatoid Arthritis
VA U.S. Department of Veterans Affairs
WICER Washington Heights/Inwood Informatics Infrastructure
for Community-Centered Comparative Effectiveness
Research
