SHARING CLINICAL
RESEARCH DATA

WORKSHOP SUMMARY

Steve Olson and Autumn S. Downey, Rapporteurs

Forum on Drug Discovery, Development, and Translation
Forum on Neuroscience and Nervous System Disorders
National Cancer Policy Forum
Roundtable on Translating Genomic-Based Research for Health

Board on Health Sciences Policy
Board on Health Care Services

INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS

Washington, D.C.

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Steve Olson and Autumn S. Downey, Rapporteurs Forum on Drug Discovery, Development, and Translation Forum on Neuroscience and Nervous System Disorders National Cancer Policy Forum Roundtable on Translating Genomic-Based Research for Health Board on Health Sciences Policy Board on Health Care Services

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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 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 AbbVie, Alzheimer’s Association, American Academy of Nursing, American Association for Cancer Re- search, American Cancer Society, American College of Medical Genetics and Genomics, American Diabetes Association, American Heart Association, American Medical Association, American So- ciety for Microbiology, American Society of Clinical Oncology, American Society of Human Genet- ics, Amgen Inc., Association of American Cancer Institutes, Association of American Medical Colleges, Blue Cross and Blue Shield Association, Bristol-Myers Squibb, Burroughs Wellcome Fund, C-Change, Celtic Therapeutics, LLLP, CeNeRx Biopharma, Centers for Disease Control and Prevention (Contract Nos. 200-2005-13434 and 200-2011-38807), CEO Roundtable on Cancer, College of American Pathologists, Critical Path Institute, Department of Health and Human Services (Contract Nos. N01-OD-4-2139 and HHSF22301026T), Department of Health and Human Services’ National Institutes of Health (NIH) (Contract No. N01-OD-4-2139) through the National Eye Insti- tute, National Institute of Mental Health, National Institute of Neurological Disorders and Stroke, National Institute on Aging, National Institute on Alcohol Abuse and Alcoholism, National Institute on Drug Abuse, and NIH Blueprint for Neuroscience Research, Department of the Air Force (Con- tract No. FA7014-10-P-0072), Department of Veterans Affairs (Contract No. V101(93) P-2238), Doris Duke Charitable Foundation, Eli Lilly and Company (Contract No. LRL-0028-07), Fast For- ward, LLC, FasterCures, Foundation for the National Institutes of Health, Friends of Cancer Re- search, GE Healthcare, Inc., Genetic Alliance, GlaxoSmithKline, Health Resources and Services Administration (Contract No. HHSH250201100119P), Janssen Research & Development, LLC, Johnson & Johnson Pharmaceutical Research and Development, LLC, The Kaiser Permanente Pro- gram Offices Community Benefit II at the East Bay Community Foundation (Contract No. 20121257), Life Technologies, Lundbeck Research USA, March of Dimes Foundation, Merck & Co., Inc., Merck Research Laboratories, The Michael J. Fox Foundation for Parkinson’s Research, National Cancer Institute (Contract Nos. HHSN261200900003C and N01-OD-4-2139, TO#189), National Coalition for Health Professional Education in Genetics, National Heart, Lung, and Blood Institute (Contract No. N01-OD-4-2139, TO#275), National Human Genome Research Institute (Contract Nos. N01-OD-4-2139, TO#264 and HHSN263201200074I, TO#5), National Institute of Mental Health (Contract No. N01-OD-4-2139, TO#275), National Institute on Aging (Contract No. N01-OD-4-2139, TO#275), National Science Foundation (Contract No. OIA-0753701), National Society of Genetic Counselors, Northrop Grumman Health IT, Novartis Oncology, Novartis Phar- maceuticals Corporation, Office of Rare Diseases Research (Contract No. N01-OD-4-2139, TO#275), Oncology Nursing Society, One Mind for Research, Pfizer Inc. (Contract No. 140-N- 1818071), Sanofi-Aventis, Society for Neuroscience, and Wellcome Trust. The views presented in this publication do not necessarily reflect the views of the organizations or agencies that provided support for the project. International Standard Book Number-13: 978-0-309-26874-5 International Standard Book Number-10: 0-309-26874-5 Additional copies of this workshop summary are available for sale from the National Academies 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 2013 by the National Academy of Sciences. All rights reserved. Printed in the United States of America Suggested citation: IOM (Institute of Medicine). 2013. Sharing clinical research data: Workshop summary. 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 gov- ernment 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 out- standing 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 engi- neering 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. www.national-academies.org

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PLANNING COMMITTEE ON SHARING CLINICAL RESEARCH DATA: AN INSTITUTE OF MEDICINE WORKSHOP1 SHARON TERRY (Chair), Genetic Alliance, Washington, DC JOSEPHINE P. BRIGGS, National Institutes of Health, Bethesda, MD TIMOTHY COETZEE, National Multiple Sclerosis Society, New York, NY STEVEN GOODMAN, Stanford University School of Medicine, CA ROBERT A. HARRINGTON, Stanford University School of Medicine, CA LYNN HUDSON, Critical Path Institute, Tucson, AZ CHARLES HUGH-JONES, Sanofi-Aventis, Inc., Bridgewater, NJ JAN JOHANNESSEN, U.S. Food and Drug Administration, Silver Spring, MD JEFFREY S. NYE, Janssen Research & Development, LLC, Titusville, NJ RICHARD PLATT, Harvard Medical School, Boston, MA WILLIAM Z. POTTER, FNIH Biomarkers Consortium, Philadelphia, PA FRANK W. ROCKHOLD, GlaxoSmithKline Pharmaceuticals Research and Development, King of Prussia, PA MICHAEL ROSENBLATT, Merck & Co., Inc., Whitehouse Station, NJ VICKI SEYFERT-MARGOLIS, U.S. Food and Drug Administration, Silver Spring, MD DEBORAH A. ZARIN, National Institutes of Health, Bethesda, MD IOM Staff AUTUMN DOWNEY, Program Officer CLARE STROUD, Program Officer REBECCA A. ENGLISH, Associate Program Officer CLAIRE GIAMMARIA, Research Associate 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. Statements, recommendations, and opinions expressed are those of individual presenters and par- ticipants, and are not necessarily endorsed or verified by the Institute of Medicine, and they should not be construed as reflecting any group consensus. v

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FRANK VALLIERE, Research Associate TONIA E. DICKERSON, Senior Program Assistant CRYSTI PARK, Senior Program Assistant BRUCE M. ALTEVOGT, Director, Forum on Neuroscience and Nervous System Disorders ADAM C. BERGER, Director, Roundtable on Translating Genomic- Based Research for Health ANNE B. CLAIBORNE, Director, Forum on Drug Discovery, Development, and Translation ROGER HERDMAN, Director, Board on Health Care Services SHARYL J. NASS, Director, National Cancer Policy Forum ANDREW M. POPE, Director, Board on Health Sciences Policy vi

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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 RUSS BIAGIO ALTMAN, Stanford University, CA MARGARET ANDERSON, FasterCures, Washington, DC CHRISTOPHER AUSTIN, National Center for Advancing Translational Sciences, Bethesda, MD ANN BONHAM, Association of American Medical Colleges, Washington, DC LINDA BRADY, National Institute of Mental Health, Bethesda, MD 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 TAMARA DARSOW, American Diabetes Association, Alexandria, VA JAMES H. DOROSHOW, National Cancer Institute, Bethesda, MD GARY L. FILERMAN, Atlas Health Foundation, McLean, VA MARK J. GOLDBERGER, Abbott Pharmaceuticals, Rockville, MD HARRY B. GREENBERG, Stanford University School of Medicine, CA LYNN HUDSON, Critical Path Institute, Tucson, AZ S. CLAIBORNE JOHNSTON, University of California, San Francisco MICHAEL KATZ, March of Dimes Foundation, White Plains, NY 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 CAROL MIMURA, University of California, Berkeley BERNARD H. MUNOS, InnoThink Center for Research in Biomedical Innovation, Indianapolis, IN 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. vii

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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 ELLEN R. STRAHLMAN, GlaxoSmithKline, Research Triangle Park, NC BRIAN L. STROM, University of Pennsylvania Perelman School of Medicine, Philadelphia NANCY SUNG, Burroughs Wellcome Fund, Research Triangle Park, NC JANET TOBIAS, Ikana Media and Mount Sinai School of Medicine, New York, NY JOANNE WALDSTREICHER, Janssen Research & Development, LLC, Raritan, NJ JANET WOODCOCK, U.S. Food and Drug Administration, White Oak, MD IOM Staff ANNE B. CLAIBORNE, Director, Forum on Drug Discovery, Development, and Translation REBECCA A. ENGLISH, Associate Program Officer ELIZABETH F. C. TYSON, Research Associate ROBIN GUYSE, Senior Program Assistant ANDREW M. POPE, Director, Board on Health Sciences Policy viii

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FORUM ON NEUROSCIENCE AND NERVOUS SYSTEM DISORDERS1 STEVEN HYMAN (Chair), The Broad Institute, Cambridge, MA SUSAN AMARA, University of Pittsburgh School of Medicine, PA MARC BARLOW, GE Healthcare, Inc., Chalfont St. Giles, United Kingdom MARK BEAR, Massachusetts Institute of Technology, Cambridge, MA KATJA BROSE, Neuron, San Francisco, CA DANIEL BURCH, CeNeRx Biopharma, Morrisville, NC C. THOMAS CASKEY, Baylor College of Medicine, Houston, TX TIMOTHY COETZEE, National Multiple Sclerosis Society, New York, NY EMMELINE EDWARDS, National Institutes of Health Neuroscience Blueprint, Bethesda, MD MARTHA FARAH, University of Pennsylvania, Philadelphia RICHARD FRANK, GE Healthcare, Inc., Princeton, NJ DANIEL GESCHWIND, University of California, Los Angeles HANK GREELY, Stanford University, CA MYRON GUTMANN, National Science Foundation, Arlington, VA RICHARD HODES, National Institute on Aging, Bethesda, MD THOMAS INSEL, National Institute of Mental Health, Bethesda, MD PHILLIP IREDALE, Pfizer Global Research and Development, Groton, CT DANIEL JAVITT, Nathan S. Kline Institute for Psychiatric Research, Orangeburg, NY FRANCES JENSEN, University of Pennsylvania School of Medicine, Philadelphia STORY LANDIS, National Institute of Neurological Disorders and Stroke, Bethesda, MD ALAN LESHNER, American Association for the Advancement of Science, Washington, DC HUSSEINI MANJI, Johnson & Johnson Pharmaceutical Research and Development, Inc., Titusville, NJ DAVID MICHELSON, Merck Research Laboratories, North Wales, PA _______________________________ 1 Institute of Medicine forums and roundtables do not issue, review, or approve in- dividual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution. ix

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RICHARD MOHS, Lilly Research Laboratories, Indianapolis, IN ALEXANDER OMMAYA, U.S. Department of Veterans Affairs, Washington, DC ATUL PANDE, GlaxoSmithKline, Inc., Research Triangle, NC STEVEN PAUL, Weill Cornell Medical College, New York, NY TODD SHERER, Michael J. Fox Foundation for Parkinson’s Research, New York, NY PAUL SIEVING, National Institutes of Health, Bethesda, MD JUDITH SIUCIAK, Foundation for the National Institutes of Health, Bethesda, MD MARC TESSIER-LAVIGNE, The Rockefeller University, New York, NY WILLIAM THIES, Alzheimer’s Association, Chicago, IL NORA VOLKOW, National Institute on Drug Abuse, Bethesda, MD KENNETH WARREN, National Institute on Alcohol Abuse and Alcoholism, Bethesda, MD JOHN WILLIAMS, Wellcome Trust, London, United Kingdom STEVIN ZORN, Lundbeck USA, Paramus, NJ CHARLES ZORUMSKI, Washington University School of Medicine IOM Staff BRUCE M. ALTEVOGT, Director, Forum on Neuroscience and Nervous System Disorders DIANA E. PANKEVICH, Program Officer ELIZABETH K. THOMAS, Senior Program Assistant ANDREW M. POPE, Director, Board on Health Sciences Policy x

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NATIONAL CANCER POLICY FORUM1 JOHN MENDELSOHN (Chair), MD Anderson Cancer Center, Houston, TX PATRICIA A. GANZ (Vice Chair), University of California, Los Angeles AMY P. ABERNETHY, Duke University School of Medicine, Durham, NC RAFAEL G. AMADO, GlaxoSmithKline-Oncology, Collegeville, PA FRED APPELBAUM, Fred Hutchinson Cancer Research Center, Seattle, WA PETER B. BACH, Memorial Sloan-Kettering Cancer Center, New York, NY EDWARD BENZ, JR., Harvard School of Medicine, Boston, MA MONICA BERTAGNOLLI, Harvard University Medical School, Boston, MA OTIS BRAWLEY, American Cancer Society, Atlanta, GA MICHAEL A. CALIGIURI, Ohio State University, Columbus RENZO CANETTA, Bristol-Myers Squibb, Wallingford, CT MICHAELE CHAMBLEE CHRISTIAN, Retired, Washington, DC WILLIAM DALTON, Moffitt Cancer Center, Tampa, FL WENDY DEMARK-WAHNEFRIED, University of Alabama at Birmingham ROBERT ERWIN, Marti Nelson Cancer Foundation, Davis, CA ROY S. HERBST, Yale Cancer Center, New Haven, CT THOMAS J. KEAN, C-Change, Washington, DC MICHELLE LE BEAU, University of Chicago, IL DOUGLAS R. LOWY, National Cancer Institute, Bethesda, MD DANIEL R. MASYS, University of Washington, Seattle, WA MARTIN J. MURPHY, Roundtable on Cancer, Durham, NC BRENDA NEVIDJON, Duke University School of Nursing, Durham, NC STEVEN PIANTADOSI, Cedars-Sinai Medical Center, Los Angeles, CA _______________________________ 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. xi

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LISA C. RICHARDSON, Centers for Disease Control and Prevention, Atlanta, GA DEBASISH ROYCHOWDHURY, Sanofi Oncology, Cambridge, MA YA-CHEN TINA SHIH, University of Chicago, IL ELLEN SIGAL, Friends of Cancer Research, Washington, DC STEVEN STEIN, Novartis Oncology, East Hanover, NJ JOHN A. WAGNER, Merck and Company, Inc., Rahway, NJ RALPH R. WEICHSELBAUM, University of Chicago, IL JANET WOODCOCK, U.S. Food and Drug Administration, Rockville, MD IOM Staff SHARYL J. NASS, Director, National Cancer Policy Forum LAURA LEVIT, Program Officer ERIN BALOGH, Associate Program Officer PAMELA LIGHTER, Research Assistant MICHAEL PARK, Senior Program Assistant PATRICK BURKE, Financial Associate SHARON B. MURPHY, Scholar in Residence ROGER HERDMAN, Director, Board on Health Care Services xii

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ROUNDTABLE ON TRANSLATING GENOMIC-BASED RESEARCH FOR HEALTH1 WYLIE BURKE (Co-Chair), University of Washington, Seattle SHARON TERRY (Co-Chair), Genetic Alliance, Washington, DC NAOMI ARONSON, Blue Cross and Blue Shield Association, Chicago, IL EUAN ANGUS ASHLEY, Stanford University School of Medicine, Palo Alto, CA PAUL R. BILLINGS, Life Technologies, Carlsbad, CA BRUCE BLUMBERG, The Permanente Medical Group, Oakland, CA DENISE E. BONDS, National Heart, Lung, and Blood Institute, Bethesda, MD PHILIP J. BROOKS, National Center for Advancing Translational Research, Rockville, MD C. THOMAS CASKEY, Baylor College of Medicine, Houston, TX SARA COPELAND, Health Resources and Services Administration, Rockville, MD MICHAEL J. DOUGHERTY, American Society of Human Genetics, Bethesda, MD VICTOR DZAU, Duke University, Durham, NC W. GREGORY FEERO, Journal of the American Medical Association, Chicago, IL ANDREW N. FREEDMAN, National Cancer Institute, Rockville, MD GEOFFREY GINSBURG, Duke University, Durham, NC RICHARD J. HODES, National Institute on Aging, Bethesda, MD SHARON KARDIA, University of Michigan School of Public Health, Ann Arbor MOHAMED KHAN, BC Cancer Agency, Vancouver, British Columbia, Canada MUIN KHOURY, Centers for Disease Control and Prevention, Atlanta, GA THOMAS LEHNER, National Institute of Mental Health, Bethesda, MD _________________________ 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. xiii

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DEBRA LEONARD, Weill Cornell Medical Center of Cornell University, New York, NY ELIZABETH MANSFIELD, U.S. Food and Drug Administration, Silver Spring, MD KELLY MCVEARRY, Northrop Grumman Information Systems, Rockville, MD GARRY NEIL, Johnson & Johnson, New Brunswick, NJ ROBERT L. NUSSBAUM, University of California, San Francisco, School of Medicine MICHELLE A. PENNY, Eli Lilly and Company, Indianapolis, IN AIDAN POWER, Pfizer Inc., Groton, CT VICTORIA M. PRATT, Quest Diagnostics Nichols Institute, Chantilly, VA RONALD PRZYGODZKI, Department of Veterans Affairs, Washington, DC ALLEN D. ROSES, Cabernet, Shiraz and Zinfandel Pharmaceuticals; Duke University, Durham, NC KEVIN A. SCHULMAN, Duke University School of Medicine, Durham, NC JOAN A. SCOTT, National Coalition for Health Professional Education in Genetics, Lutherville, MD DAVID VEENSTRA, University of Washington, Seattle MICHAEL S. WATSON, American College of Medical Genetics and Genomics, Bethesda, MD DANIEL WATTENDORF, DARPA/Defense Sciences Office, Arlington, VA CATHERINE A. WICKLUND, Northwestern University, Chicago, IL IOM Staff ADAM C. BERGER, Director, Roundtable on Translating Genomic- Based Research for Health SEAN P. DAVID, James C. Puffer, M.D./American Board of Family Medicine Fellow TONIA E. DICKERSON, Senior Program Assistant ANDREW M. POPE, Director, Board on Health Sciences Policy xiv

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Reviewers This workshop summary has been reviewed in draft form by individ- uals chosen for their diverse perspectives and technical expertise, in ac- cordance 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 summary as sound as possible and to ensure that the summary meets institutional standards for objectivity, evidence, and re- sponsiveness to the study charge. The review comments and draft manu- script remain confidential to protect the integrity of the process. We wish to thank the following individuals for their review of this workshop summary: William W. Chin, Harvard Medical School Carolyn Compton, Critical Path Institute Barry Gertz, Affairs, Merck Research Laboratories Jeffrey Nye, Janssen Research & Development, LLC Although the reviewers listed above have provided many construc- tive comments and suggestions, they did not see the final draft of the workshop summary before its release. The review of this summary was overseen by Melvin Worth, Jr. Appointed by the Institute of Medicine, he 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 summary rests entirely with the authors and the institution. xv

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Contents ACRONYMS xxi 1 INTRODUCTION AND THEMES OF THE WORKSHOP 1 Organization of the Report, 2 Themes of the Workshop, 4 2 THE BENEFITS OF DATA SHARING 9 The Uses of Shared Participant-Level Data, 11 The Rationale for Data Sharing, 12 Cautions on Data Sharing, 17 3 BARRIERS TO DATA SHARING 19 Legal Issues Relating to Patient Privacy, 20 Cultural Barriers, 22 Technical Challenges, 25 4 MODELS OF DATA SHARING 27 ClinicalTrials.gov, 28 The DataSphere Project, 31 The Yale-Medtronic Experience, 32 The Biomarkers Consortium, 36 The NEWMEDS Consortium, 37 PatientsLikeMe, 39 Distributed Systems for Clinical Research Information Sharing, 40 xvii

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xviii CONTENTS 5 STANDARDIZATION TO ENHANCE DATA SHARING 43 How Standards Benefit Sharing, 44 Developing Standards to Enable Data Sharing, 47 Retrospective Versus Prospective Approaches to Data Standardization, 50 Data-Sharing Approaches That Have Benefited from the Use of Standards, 51 Governance Issues, 54 6 CHANGING THE CULTURE OF RESEARCH 57 The Role of Regulators, 58 The Role of Journals, 60 The Role of Funders, 61 Incentivizing Change by Ensuring Credit, 63 Protecting Against Misuse of Shared Data, 65 Patient-Driven Sharing of Clinical Research Data, 66 7 FINAL REFLECTIONS ON SHARING CLINICAL RESEARCH DATA 73 Low-Hanging Fruit, 74 The Boundaries of Precompetitive Collaboration, 75 Partnering with Patients, 76 REFERENCES 81 APPENDIXES A Workshop Agenda 85 B List of Data-Sharing Initiatives 101 C Participant Biographies 105

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Boxes and Figures BOXES 1-1 Statement of Task for the Workshop, 3 2-1 What Is Participant-Level Data?, 10 2-2 Treatment Advances for Cardiovascular Disease: A Success Story for Data Sharing, 14 FIGURES 2-1 A “cycle of quality” from discovery science to the measurement of outcomes can generate evidence to inform policy, 14 4-1 Information loss as clinical trials data progresses from raw uncoded data to summary data, 29 4-2 Distributed networks can facilitate working remotely with research datasets derived from routinely collected electronic health information, often eliminating the need to transfer sensitive data, 41 xix

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Acronyms BRIDG Biomedical Research Integrated Domain Group CAMD Coalition Against Major Diseases CDA clinical documentation architecture CDASH Clinical Data Acquisition Standards Harmonization CDE common data element CDER Center for Drug Evaluation and Research CDISC Clinical Data Interchange Standards Consortium CEO chief executive officer CFAST Coalition for Accelerating Standards and Therapies CLIA Clinical Laboratory Improvement Amendments C-Path Critical Path Institute CSR clinical study report eDISH Electronic Drug-Induced Serious Hepatoxicity EHR electronic health record EMA European Medicines Agency FDA Food and Drug Administration FDASIA FDA Safety and Innovation Act FNIH Foundation for the National Institutes of Health FTC Federal Trade Commission HHMI Howard Hughes Medical Institute HIPAA Health Insurance Portability and Accountability Act HL7 Health Level 7 International xxi

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xxii ACRONYMS iDASH Integrated Data for Analysis, Anonymization, and Sharing IHE Integrating the Healthcare Enterprise IOM Institute of Medicine IRB institutional review board NDA new drug application NEWMEDS Novel Methods for Development of Drugs in Depression and Schizophrenia NHLBI National Heart, Lung, and Blood Institute NIH National Institutes of Health NINDS National Institute of Neurological Disorders and Stroke NSF National Science Foundation PDUFA Prescription Drug User Fee Act PHI protected health information SDO standards development organization SDTM Study Data Tabulation Model YODA Yale University Open Data Access