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
Washington, D.C.
www.nap.edu
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 Governing Board of the National Research Council, whose members are drawn from the councils of the National Academy of Sciences, the National Academy of Engineering, 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, findings, 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
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Suggested citation: IOM (Institute of Medicine). 2012. Public Engagement and Clinical Trials: New Models and Disruptive Technologies: Workshop Summary. Washington, DC: The National Academies Press.
“Knowing is not enough; we must apply.
Willing is not enough; we must do.”
—Goethe
THE NATIONAL ACADEMIES
Advisers to the Nation on Science, Engineering, and Medicine
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 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 engineers. 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 engineering programs aimed at meeting national needs, encourages education and research, 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 Institute 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 Sciences 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 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.
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.
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
1 Institute of Medicine forums and roundtables do not issue, review, or approve individual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.
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
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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 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 constructive 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
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.
Relationships with Researchers
Relationships with the Audience
7 NOVEL CLINICAL TRIAL DESIGNS
Adaptive Clinical Trial Designs
Using Point-of-Care Clinical Trials to Create a Learning Health Care System
The Food and Drug Administration Perspective
8 THE HEALTH SYSTEM’S STRUCTURE AND CULTURE
Building a Supportive Clinical Trials Environment in Academia
Public-Private Partnerships in Cancer Trials
Engaging Community Physicians in Rheumatoid Arthritis Research
9 TOWARD A PATIENT-CENTERED STRATEGY FOR CLINICAL TRIALS
Table, Figure, and Boxes
TABLE
3-1 Comparison of Researcher and Participant Perspectives on Clinical Trials
FIGURE
4-1 A timeline comparing a conventional NIH clinical trial versus the 23 and Me research model
BOXES
2-1 Elements for Successful Clinical Trials
2-2 Key Principles for Recruiting and Retaining People in Clinical Trials
3-1 Targeted Strategies to Manage Patient Recruitment
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 |
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 |