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.
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 Engineering, 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 publication 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
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Suggested citation: IOM (Institute of Medicine). 2014. Contemporary issues for protecting patients in cancer research: Workshop summary. Washington, DC: The National Academies Press.
“Knowing is not enough; we must apply.
Willing is not enough; we must do.”
—Goethe
INSTITUTE OF MEDICINE
OF THE NATIONAL ACADEMIES
Advising the Nation. Improving Health.
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. 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 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. C. D. Mote, Jr., are chair and vice chair, respectively, of the National Research Council.
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.
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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.
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
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)
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Reviewers
This workshop summary 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 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
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.
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. Current 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 Comprehensive 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 excellent workshop agenda.
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Contents
SHORTCOMINGS OF CURRENT REGULATIONS AND GUIDANCES
Impediment to Quality Improvement and Learning
Lack of Harmonization with International Standards
ADVANCED NOTICE OF PROPOSED RULEMAKING
PATIENT PERSPECTIVES ON RESEARCH PROTECTIONS
ETHICAL CHALLENGES OF GENETIC ADVANCES
Boxes, Figures, and Table
BOXES
1 Suggestions Made by Individual Workshop Participants
2 HIPAA “Safe Harbor” Deidentification Method
4 Fair Information Practice Principles
5 Platform for Engaging Everyone Responsibly (PEER)
FIGURES
1 A risk-based deidentification model
2 A: Typical registry architecture
B: Core components of the Platform for Engaging Everyone Responsibly (PEER)
3 Landscape of learning activities
TABLE