DISCUSSION FRAMEWORK FOR
CLINICAL TRIAL DATA SHARING

GUIDING PRINCIPLES, ELEMENTS, AND ACTIVITIES

Committee on Strategies for Responsible
Sharing of Clinical Trial Data

Board on Health Sciences Policy

INSTITUTE OF MEDICINE
       OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS

Washington, D.C.

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Committee on Strategies for Responsible Sharing of Clinical Trial Data Board on Health Sciences Policy

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THE NATIONAL ACADEMIES PRESS • 500 Fifth Street, NW • Washington, DC 20001 NOTICE: The project that is the subject of this report was approved by the Gov- erning 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. The members of the committee re- sponsible for the report were chosen for their special competences and with re- gard for appropriate balance. This study was supported by contracts between the National Academy of Sci- ences and the U.S. National Institutes of Health (HHSN263201200074I), U.S. Food and Drug Administration, AbbVie Inc., Amgen Inc., AstraZeneca Pharma- ceuticals, Bayer, Biogen Idec, Bristol-Myers Squibb, Burroughs Wellcome Fund, Doris Duke Charitable Foundation, Eli Lilly and Company, EMD Serono, Genen- tech, GlaxoSmithKline, Johnson & Johnson, Medical Research Council (UK), Merck & Co., Inc., Novartis Pharmaceuticals Corporation, Novo Nordisk, Pfizer Inc., Sanofi-Aventis, Takeda, and Wellcome Trust. Any opinions, findings, con- clusions, or recommendations expressed in this publication are those of the au- thor(s) and do not necessarily reflect the views of the organizations or agencies that provided support for the project. Additional copies of this report are available for sale from the National Acade- mies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624- 6242 or (202) 334-3313; http://www.nap.edu. For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu. Copyright 2014 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 al- most all cultures and religions since the beginning of recorded history. The ser- pent adopted as a logotype by the Institute of Medicine is a relief carving from ancient Greece, now held by the Staatliche Museum in Berlin. Suggested citation: IOM (Institute of Medicine). 2014. Discussion framework for clinical trial data sharing: Guiding principles, elements, and activities. Washington, DC: The National Academies Press.

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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 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. C. D. Mote, Jr., 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 re- sponsibility 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 Nation- al 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. C. D. Mote, Jr., are chair and vice chair, respectively, of the National Research Council. www.national-academies.org

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COMMITTEE ON STRATEGIES FOR RESPONSIBLE SHARING OF CLINICAL TRIAL DATA BERNARD LO (Chair), President, The Greenwall Foundation, New York City, New York TIMOTHY COETZEE, Chief Advocacy, Services, and Research Officer, National Multiple Sclerosis Society DAVE DEMETS, Professor and Chair, Department of Biostatistics and Medical Informatics, University of Wisconsin, Madison JEFFREY DRAZEN, Editor-in-Chief, New England Journal of Medicine STEVEN GOODMAN, Professor, Medicine & Health Research & Policy, Stanford University School of Medicine PATRICIA KING, Carmack Waterhouse Professor of Law, Medicine, Ethics, and Public Policy, Georgetown University Law Center TRUDIE LANG, Principal Investigator, Global Health Network, Nuffield Department of Medicine, University of Oxford DEVEN MCGRAW, Director, Health Privacy Project, Center for Democracy & Technology ELIZABETH NABEL, President, Brigham and Women’s Hospital ARTI RAI, Elvin R. Latty Professor of Law, Duke University School of Law IDA SIM, Professor of Medicine and Co-Director of Biomedical Informatics of the Clinical and Translational Science Institute, University of California at San Francisco SHARON TERRY, President and CEO, Genetic Alliance JOANNE WALDSTREICHER, Chief Medical Officer, Johnson & Johnson IOM Staff ANNE B. CLAIBORNE, Senior Program Officer LEIGHANNE OLSEN, Interim Study Director (until November 2013) REBECCA N. LENZI, Study Director (from November 2013) MICHELLE MANCHER, Associate Program Officer RACHEL KIRKLAND, Senior Program Assistant (until October 2013) BARRET J. ZIMMERMANN, Senior Program Assistant (from October 2013) ANDREW M. POPE, Director, Board on Health Sciences Policy Consultant THERESA WIZEMANN, Wizemann Scientific Communications, LLC v

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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 in- stitutional standards for objectivity, evidence, and responsiveness to the study charge. The review comments and draft manuscript remain confi- dential to protect the integrity of the deliberative process. We wish to thank the following individuals for their review of this report: Lawrence J. Appel, Johns Hopkins Medical Institutions Marc Boutin, National Health Council Kay Dickersin, Johns Hopkins Bloomberg School of Public Health David Harrington, Harvard School of Public Health Peter S. Kim, Stanford University School of Medicine Bartha Knoppers, McGill University David Korn, Harvard University Harlan M. Krumholz, Yale University School of Medicine Christine Laine, Annals of Internal Medicine Sir Michael Rawlins, Royal Society of Medicine Jerome P. Reiter, Duke University Donald M. Steinwachs, Johns Hopkins University Alan M. Zaslavsky, Harvard Medical School vii

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viii REVIEWERS Although the reviewers listed above have provided many constructive comments and suggestions, they were not asked to endorse the conclusions or recommendations, nor did they see the final draft of the report before its release. The review of this report was overseen by Enriqueta C. Bond, QE Philanthropic Advisors, and Stephen E. Fienberg, Carnegie Mellon University. Appointed by the Institute of Medicine and the National Research Council, they were responsible for making certain that an inde- pendent 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 authoring committee and the institution.

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Preface Sharing data generated through the conduct of clinical trials offers the promise of placing evidence about the safety and efficacy of thera- pies and clinical interventions on a firmer basis and enhancing the bene- fits of clinical trials. Ultimately, such data sharing—if carried out appropriately—could lead to improved clinical care and greater public trust in clinical research and health care. However, recommending strat- egies for how clinical trial data could be shared responsibly is a difficult process—many stakeholders have valid interests that sometimes are not aligned; different models and approaches have been proposed or tried; and proposing an approach that works for the wide range of clinical trials may be daunting. Undergirding any discussion of possibilities and needs for clinical trial data sharing is a fundamental philosophical question: To whom ought the benefits of the data belong, and, accordingly, where lies the presumption as to whether data ought to be shared? The Institute of Medicine (IOM) has assembled a committee with in- terdisciplinary expertise and a wide range of backgrounds to study how data from clinical trials might best be shared. The fulfillment of the committee’s task requires careful gathering of evidence and listening to and understanding different viewpoints. We plan to learn from what has already been proposed and tried. As the committee is charged in its statement of task, this document is designed as a framework for discussion. This framework is being re- leased to stimulate reactions and comments from stakeholders and the public. In addition to seeking comments generally, we also have posed several specific questions for which feedback and suggestions would be particularly useful as we move our deliberations forward. Although we welcome comments at any time throughout the study process, comments ix

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x PREFACE on the issues raised in this framework document would be most helpful if received by March 24, 2014. At this early stage in our work, we have reached no conclusions. As all IOM committees are called to do, we ap- proach our charge with minds open to sound evidence and convincing arguments, and we look forward to hearing them. This document would not have been possible without the dedicated and skilled work of IOM staff, including Anne B. Claiborne, LeighAnne Olsen, Rebecca N. Lenzi, Michelle Mancher, Rachel Kirkland, Barret J. Zimmermann, and Andrew M. Pope, whom the committee gratefully acknowledges and thanks. Bernard Lo, Chair Committee on Strategies for Responsible Sharing of Clinical Trial Data

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Contents CONTEXT OF THE STUDY 1 Charge to the Committee and Scope of the Study, 3 A FRAMEWORK FOR DISCUSSION AND CALL FOR COMMENTS 5 Invitation for Public Comments, 7 GUIDING PRINCIPLES FOR RESPONSIBLE SHARING OF CLINICAL TRIAL DATA 8 Respect the Individual Participants Whose Data Are Shared, 9 Respect Through Research Participant Protections, 9 Respect Through Engagement, 10 Maximize Benefits to Participants in Clinical Trials and to Society, While Minimizing Harm, 10 Potential Benefits, 11 Potential Harms, 13 Increase Public Trust in Clinical Trials, 14 Trust in Clinical Trial Data, 14 Trust in the Data Sharing Process, 15 Carry Out Sharing of Clinical Trial Data in a Manner That Enhances Fairness, 15 Fairness in Exposure to Potential Risks of Data Sharing, 16 Fairness in the Distribution of the Potential Benefits of Research, 16 xi

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xii CONTENTS ADDITIONAL OBSERVATIONS RELATING TO PRINCIPLES FOR DATA SHARING 17 Operational Strategies Following from Guiding Principles, 17 Framing Rights and Responsibilities: To Whom Do the Benefits of Clinical Trial Data Belong?, 18 DATA SHARING ELEMENTS AND ACTIVITIES 19 What Types of Data Could Be Shared?, 20 Current Practices in Data Disclosure, 20 Data, 21 Reports Generated from Data, 23 Metadata and Additional Documentation, 25 Who Are the Providers of Shared Data?, 27 Who Are the Recipients of Shared Data?, 28 When Might Clinical Trial Data Be Shared?, 29 How Might Data Be Shared?, 29 Open Access, 33 Controlled Access, 33 Selected Set of Clinical Trial Data Sharing Activities, 34 REFERENCES 37 APPENDIXES A Statement of Task 43 B Committee Biographies 45 C Figure 1: Data Flow from Participant to Analyzed Data and Reporting 57

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Boxes and Figure BOXES 1 Specific Topics for Public Feedback, 6 2 Provisional Guiding Principles for Responsible Sharing of Clinical Trial Data, 8 3 Types of Summary Reports, 23 4 Metadata and Additional Documentation to Support the Use of Shared Clinical Trial Data, 26 5 Set of Clinical Trial Data Sharing Activities, 32 FIGURE 1 Data flow from raw data to analyzable data, 21 xiii

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