PUBLIC ENGAGEMENT
AND CLINICAL TRIALS

NEW MODELS AND DISRUPTIVE TECHNOLOGIES

WORKSHOP SUMMARY

Victoria Weisfeld, Rebecca A. English, and
Anne B. Claiborne, Rapporteurs

Forum on Drug Discovery, Development, and Translation

Board on Health Sciences Policy

INSTITUTE OF MEDICINE
              OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS
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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, 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 2012 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). 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- ety of distinguished scholars engaged in scientific and engineering research, dedi- cated 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 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 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 In- stitute 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 govern- ment. Functioning in accordance with general policies determined by the Acad- emy, the Council has become the principal operating agency of both the National Academy of Sciences and the National Academy of Engineering in providing ser- vices to the government, the public, and the scientific and engineering communi- ties. The Council 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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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