CONTEMPORARY ISSUES FOR

PROTECTING
PATIENTS

IN CANCER RESEARCH

WORKSHOP SUMMARY

Sharyl J. Nass and Margie Patlak, Rapporteurs

National Cancer Policy Forum

Board on Health Care Services

INSTITUTE OF MEDICINE
              OF THE NATIONAL ACADEMIES

THE NATIONAL ACADEMIES PRESS

Washington, D.C.

www.nap.edu



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CONTEMPORARY ISSUES FOR PROTECTING PATIENTS IN CANCER RESEARCH WORKSHOP SUMMARY Sharyl J. Nass and Margie Patlak, Rapporteurs National Cancer Policy Forum Board on Health Care Services

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THE NATIONAL ACADEMIES PRESS  500 Fifth Street, NW  Washington, DC 20001 NOTICE: The workshop that is the subject of this workshop summary 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 Engineer- ing, and the Institute of Medicine. This project was supported by Contract No. HHSN261200900003C between the National Academy of Sciences and the Centers for Disease Control and Prevention and by the American Association for Cancer Research, the American Cancer Society, the American Society for Radiation Oncology, the American Society of Clinical Oncology, the Association of American Cancer Institutes, C-Change, the CEO Roundtable on Cancer, EMD Serono, GlaxoSmithKline, Helsinn Healthcare SA, the LIVESTRONG Foundation, the National Comprehensive Cancer Network, Novartis Oncology, the Oncology Nursing Society, and Sanofi Oncology. The views presented in this publica- tion do not necessarily reflect the views of the organizations or agencies that provided support for the activity. International Standard Book Number-13:  978-0-309-30666-9 International Standard Book Number-10:  0-309-30666-3 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 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 almost all cultures and religions since the beginning of recorded history. The serpent adopted 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). 2014. Contemporary issues for pro- tecting patients in cancer research: 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 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 man- date 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 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. Victor J. Dzau 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 N ­ ational 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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WORKSHOP PLANNING COMMITTEE1 ANGELA R. BRADBURY (Cochair), Department of Medicine, Hematology/Oncology, and Department of Medical Ethics and Health Policy, Perelman School of Medicine, University of Pennsylvania STEVEN PIANTADOSI (Cochair), Phase One Foundation Chair and Director, Samuel Oschin Comprehensive Cancer Institute EDWARD J. BENZ, JR., Richard & Susan Smith Professor of Medicine, Harvard Medical School, President, Dana-Farber Cancer Institute LAURA CLEVELAND, Patient Advocate, Alliance for Clinical Trials in Oncology, Member, National Cancer Institute Central Institutional Review Board PATRICIA A. GANZ, Director, Cancer Prevention & Control Research, Jonsson Comprehensive Cancer Center, University of California, Los Angeles ROY HERBST, Professor of Medicine and Chief, Section of Medical Oncology, Yale Cancer Center STEVEN JOFFE, Director, Penn Fellowship in Advanced Biomedical Ethics, Department of Medical Ethics and Health Policy, Perelman School of Medicine, University of Pennsylvania THOMAS J. KEAN, Executive Director, C-Change DANIEL R. MASYS, Affiliate Professor, Biomedical and Health Informatics, University of Washington JOHN MENDELSOHN, Director, Khalifa Institute for Personalized Cancer Therapy, The University of Texas MD Anderson Cancer Center HOLLY TAYLOR, Associate Professor, Health Policy and Management, Johns Hopkins Bloomberg School of Public Health and Core Faculty, Johns Hopkins Berman Institute of Bioethics Project Staff SHARYL J. NASS, Director, National Cancer Policy Forum LAURA LEVIT, Program Officer (through December 2013) SARA THARAKAN, Research Assistant (from November 2013) PAMELA LIGHTER, Research Assistant (through September 2013) HANNAH DURING, 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. v

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NATIONAL CANCER POLICY FORUM1 MICHAEL CALIGIURI (Chair), CEO, James Cancer Hospital and Solove Research Institute, and Director, OSU Comprehensive Cancer Center, The Ohio State University PATRICIA A. GANZ (Vice Chair), Professor, University of California, Los Angeles, School of Medicine & Public Health, Division of Cancer Prevention & Control Research, Jonsson Comprehensive Cancer Center AMY P. ABERNETHY, Associate Professor of Medicine, Duke University School of Medicine, and Director, Duke Cancer Care Research Program LUCILE ADAMS-CAMPBELL, Professor of Oncology, Associate Director for Minority Health and Health Disparities Research, Georgetown University Lombardi Cancer Center RAFAEL G. AMADO, Senior Vice President and Head of R&D, GlaxoSmithKline Oncology KENNETH ANDERSON, Kraft Family Professor of Medicine, American Cancer Society Clinical Research Director, Jerome Lipper Multiple Myeloma Center, Harvard Medical School, Dana-Farber Cancer Institute MONICA BERTAGNOLLI, Professor of Surgery, Harvard University Medical School OTIS BRAWLEY, Chief Medical Officer and Executive Vice President, American Cancer Society CARLTON BROWN, Director of Professional Services, Oregon Nurses Association, and Past President, Oncology Nursing Society ROBERT W. CARLSON, Chief Executive Officer, National Comprehensive Cancer Network SERGIO CANTOREGGI, Chief Scientific Officer and Global Head of Research and Development, the Helsinn Group WILLIAM S. DALTON, CEO, M2Gen Personalized Medicine Institute, Moffitt Cancer Center, Tampa, Florida, and Chair, American Association for Cancer Research Science Policy & Legislative Affairs Committee 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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GWEN DARIEN, Executive Vice President, Programs and Services, Cancer Policy Institute, Cancer Support Community WENDY DEMARK-WAHNEFRIED, Associate Director for Cancer Prevention and Control, University of Alabama at Birmingham Comprehensive Cancer Center CAROL A. HAHN, Associate Professor of Radiation Oncology, Duke University Medical Center, and Medical Director of Radiation Oncology, Duke Raleigh Hospital, and Clinical Affairs and Quality Council Chair, American Society for Radiation Oncology LORI HOFFMAN HOGG, Cancer Program Director, Albany Stratton VA Medical Center THOMAS J. KEAN, President and CEO, C-Change SAMIR N. KHLEIF, Director, Georgia Health Sciences University Cancer Center, Georgia Regents University Cancer Center MICHELLE M. LE BEAU, Arthur and Marian Edelstein Professor of Medicine, Director, Comprehensive Cancer Center, University of Chicago, and President, Association of American Cancer Institutes SHARI M. LING, Deputy Chief Medical Officer, Centers for Medicare & Medicaid Services, Medical Officer, Center for Clinical Standards and Quality DOUGLAS R. LOWY, Deputy Director, National Cancer Institute GRETA MASSETTI, Associate Director for Science, Division of Cancer Prevention and Control, Centers for Disease Control and Prevention DANIEL R. MASYS, Affiliate Professor, Biomedical Informatics, University of Washington, Seattle MARTIN J. MURPHY, Chief Executive Officer, CEO Roundtable on Cancer RICHARD PAZDUR, Director, Office of Oncology and Hematology Products, Food and Drug Administration STEVEN PIANTADOSI, Phase One Foundation Endowed Chair and Director, Samuel Oschin Comprehensive Cancer Institute, Cedars- Sinai Medical Center JENNIFER A. PIETENPOL, Director, Vanderbilt-Ingraham Cancer Center, Benjamin F. Byrd, Jr., Professor of Oncology, Professor of Biochemistry, Vanderbilt University RUTH RECHIS, Vice President of Programs, LIVESTRONG Foundation ANDREW SCHIERMEIER, Senior Vice President, Head of Global Oncology, Merck Serono viii

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RICHARD SCHILSKY, Chief Medical Officer, American Society of Clinical Oncology DEBORAH SCHRAG, Associate Professor, Harvard Medical School, Dana-Farber Cancer Institute ADRIAN SENDEROWICZ, Vice President, Global Regulatory Affairs, Sanofi Oncology YA-CHEN TINA SHIH, Director, Program in the Economics of Cancer, University of Chicago ELLEN V. SIGAL, Chairperson and Founder, Friends of Cancer Research STEVEN STEIN, Senior Vice President, U.S. Clinical Development and Medical Affairs, Novartis Oncology RALPH WEICHSELBAUM, Daniel K. Ludwig Professor and Chair, Department of Radiation Oncology, Director, Ludwig Center for Metastasis Research, The University of Chicago Medical Center National Cancer Policy Forum Staff SHARYL J. NASS, Forum Director and Interim Director, Board on Health Care Services (from June 2014) SARA THARAKAN, Research Assistant HANNAH DURING, Senior Program Assistant CELYNNE BALATBAT, Senior Program Assistant (from January 2014) PATRICK BURKE, Financial Associate ROGER HERDMAN, Director, Board on Health Care Services (until June 6, 2014) ix

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Reviewers This workshop summary has been reviewed in draft form by individu- als chosen for their diverse perspectives and technical expertise, in accor- dance 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 workshop summary as sound as possible and to ensure that the workshop summary 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 workshop summary: JEFFREY R. BOTKIN, Professor of Pediatrics and Medical Ethics, Associate Vice President for Research, University of Utah MILDRED K. CHO, Research Professor of Pediatrics and Associate Director, Center for Biomedical Ethics, Stanford University DEBORAH R. COLLYAR, President, Patient Advocates in Research JOHN D. LANTOS, Director of Pediatric Bioethics, Professor of Pediatrics, University of Missouri-Kansas City School of Medicine MARY S. McCABE, Director, Survivorship Program, Division of Medical Ethics, Weill Medical College, Memorial Sloan- Kettering Cancer Center xi

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xii REVIEWERS Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the workshop summary before its release. The review of this report was overseen by R. ALTA CHARO, Warren P. Knowles Professor of Law and Bioethics at the School of Law, and Department of Medical History and Bioethics, School of Medicine and Public Health at the University of Wisconsin, Madison. 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 rapporteurs and the institution.

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Acknowledgments Support from the many annual sponsors of the Institute of Medicine’s ­ National Cancer Policy Forum is crucial to the work of the Forum. ­ urrent C sponsors include the Centers for Disease Control and Prevention, the National Cancer Institute, the American Association for Cancer Research, the American Cancer Society, the American Society for Radiation Oncology, ­ the American Society of Clinical Oncology, the Association of American Cancer Institutes, C-Change, the Cancer Support Community, the CEO Roundtable on Cancer, EMD Serono, GlaxoSmithKline Oncology, Helsinn Healthcare SA, the LIVESTRONG Foundation, the National Comprehen- sive Cancer Network, Novartis Oncology, the Oncology Nursing Society, and Sanofi Oncology. The Forum wishes to express its gratitude to the expert speakers whose presentations helped define the current challenges and opportunities for protecting patients in cancer research. The Forum also wishes to thank the members of the planning committee for their work in developing an excel- lent workshop agenda. xiii

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Contents INTRODUCTION 1 CURRENT REGULATORY ARENA 3 Common Rule and HIPAA, 3 SHORTCOMINGS OF CURRENT REGULATIONS AND GUIDANCES 7 Context, 8 Data Deidentification, 8 Impediment to Quality Improvement and Learning, 13 Varied Interpretations, 14 Misaligned Incentives, 14 Lack of Harmonization with International Standards, 14 Informed Consent Forms, 15 Informed Consent Process, 18 Consent Tools, 22 Beyond Consent, 24 HITECH 25 ADVANCED NOTICE OF PROPOSED RULEMAKING 27 PATIENT PERSPECTIVES ON RESEARCH PROTECTIONS 30 ETHICAL CHALLENGES OF GENETIC ADVANCES 35 Patient Consent for Use of Archived Biospecimens, 35 Return of Research Results, 38 Clinically Actionable Findings, 40 xv

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xvi CONTENTS Context Matters, 41 Who Pays, 42 Biospecimens from Deceased Participants, 43 THE CHANGING CONTEXT OF RESEARCH AND CARE 43 Oversight in a Learning Health Care System, 46 Oversight of Pragmatic Trials, 48 MULTISITE STUDIES AND IRB REVIEW 50 Central IRBs, 54 New England Reliance Agreement, 56 Value of Local IRBs, 57 EDUCATIONAL NEEDS 60 RESEARCH NEEDS 61 WRAP-UP 62 REFERENCES 63 ACRONYMS 67 APPENDIX 69

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Boxes, Figures, and Table BOXES 1 Suggestions Made by Individual Workshop Participants, 4 2 HIPAA “Safe Harbor” Deidentification Method, 10 3 Teach to Goal, 19 4 Fair Information Practice Principles, 26 5 Platform for Engaging Everyone Responsibly (PEER), 33 FIGURES 1 A risk-based deidentification model, 12 2 A: Typical registry architecture, 34 B:  ore components of the Platform for Engaging Everyone C Responsibly (PEER), 34 3 Landscape of learning activities, 45 TABLE 1 Elements of Informed Consent, 22 xvii

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