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IMPLEMENTING A NATIONAL CANCER CLINICAL TRIALS SYSTEM FOR THE 21ST CENTURY Workshop Summary An American Society of Clinical Oncology and Institute of Medicine Workshop Alison Mack and Sharyl J. Nass, Rapporteurs National Cancer Policy Forum Board on Health Care Services
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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 Insti- tute of Medicine. The members of the committee responsible for planning the workshop were chosen for their special competences and with regard for appropriate balance. This project was sponsored by the American Society of Clinical Oncology. The National Cancer Policy Forum is supported by Contract Nos. HHSN261200900003C and 200- 2005-13434 TO #1 between the National Academy of Sciences and the National Cancer Institute and the Centers for Disease Control and Prevention, respectively. This project was also supported by the American Association for Cancer Research, the American Cancer Society, the Association of American Cancer Institutes, Bristol-Myers Squibb, C-Change, the CEO Roundtable on Cancer, Novartis Oncology, and the Oncology Nursing Society. Any opinions, ﬁndings, conclusions, or recommendations expressed in this publication are those of the author(s) and do not necessarily reﬂect the view of the organizations or agencies that provided support for this project. International Standard Book Number-13: 978-0-309-21268-7 International Standard Book Number-10: 0-309-21268-5 Additional copies of this report are available from the National Academies Press, 500 Fifth Street, N.W., Lockbox 285, Washington, DC 20055; (800) 624-6242 or (202) 334-3313 (in the Washington metropolitan area); Internet, http://www.nap.edu. For more information about the Institute of Medicine, visit the IOM home page at: www.iom.edu. Copyright 2011 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). 2011. Implementing a National Cancer Clinical Trials System for the 21st Century: 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. 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. www.national-academies.org
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WORKSHOP PLANNING COMMITTEE1 RICHARD L. SCHILSKY (Chair), Professor of Medicine and Section Chief, Hematology/Oncology, University of Chicago Medical Center, Chicago, Illinois JAN C. BUCKNER, Professor of Oncology, Mayo Clinic College of Medicine, and Group Chair, North Central Cancer Treatment Group, Rochester, Minnesota MICHAEL A. CALIGIURI, Director, Ohio State Comprehensive Cancer Center, Columbus, Ohio WILLIAM DALTON, President, Chief Executive Ofﬁcer, and Center Director, Mofﬁtt Cancer Center, Tampa, Florida JAMES H. DOROSHOW, Director, Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethesda, Maryland ROY S. HERBST, Chief of Medical Oncology, Yale Cancer Center, New Haven, Connecticut JOHN HOHNEKER, Senior Vice President and Head, Global Development, Integrated Hospital Care, Novartis Pharma AG, Basel, Switzerland JOHN MENDELSOHN, President, M.D. Anderson Cancer Center, Houston, Texas SHARON B. MURPHY, Scholar-in-Residence, Institute of Medicine, Washington, DC GEORGE W. SLEDGE, JR., Ballve-Lantero Professor of Medicine, Indiana University School of Medicine, and Co-leader, Breast Cancer Program, Indiana University Simon Cancer Center, Indianapolis, Indiana MARY LOU SMITH, Co-founder, Research Advocacy Network, Naperville, Illinois Institute of Medicine Project Staff SHARYL J. NASS, Director, National Cancer Policy Forum ERIN BALOGH, Associate Program Ofﬁcer MICHAEL PARK, Senior Program Assistant ROGER HERDMAN, Director, Board on Health Care Services American Society of Clinical Oncology Staff SUANNA BRUINOOGE, Director, Research Policy Division, ASCO Cancer Policy and Clinical Affairs Department 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 JOHN MENDELSOHN (Chair), President, M.D. Anderson Cancer Center, Houston, Texas PATRICIA A. GANZ (Vice Chair), Professor, University of California at Los Angeles (UCLA) School of Medicine & Public Health, Division of Cancer Prevention & Control Research, Jonsson Comprehensive Cancer Center, Los Angeles, California AMY ABERNETHY, Associate Professor of Medicine, Duke University School of Medicine, and Director, Duke Cancer Care Research Program, Durham, North Carolina FRED APPELBAUM, Director, Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington PETER B. BACH, Associate Attending Physician, Memorial Sloan-Kettering Cancer Center, New York EDWARD BENZ, JR., President, Dana-Farber Cancer Institute, and Director, Harvard Cancer Center, Harvard University Medical School, Boston, Massachusetts MONICA BERTAGNOLLI, Professor of Surgery, Harvard University Medical School, Boston, Massachusetts THOMAS G. BURISH, Provost, Notre Dame University, South Bend, Indiana, and Past Chair, American Cancer Society Board of Directors MICHAEL A. CALIGIURI, Director, Ohio State Comprehensive Cancer Center, Columbus, Ohio, and President, Association of American Cancer Institutes RENZO CANETTA, Vice President, Oncology Global Clinical Research, Bristol-Myers Squibb, Wallingford, Connecticut MICHAELE CHAMBLEE CHRISTIAN, Retired, Washington, DC WILLIAM DALTON, President, Chief Executive Ofﬁcer, and Center Director, Mofﬁtt Cancer Center, Tampa, Florida, and Chair, AACR Committee on Science Policy and Legislative Affairs WENDY DEMARK-WAHNEFRIED, Associate Director for Cancer Prevention and Control, University of Alabama at Birmingham Comprehensive Cancer Center, Birmingham, Alabama ROBERT ERWIN, President, Marti Nelson Cancer Foundation, Davis, California 1 Institute of Medicine forums and roundtables do not issue, review, or approve indi- vidual documents. The responsibility for the published meeting summary rests with the rapporteurs and the institution. vi
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ROY S. HERBST, Chief of Medical Oncology, Yale Cancer Center, New Haven, Connecticut JOHN HOHNEKER, Senior Vice President and Head, Global Development, Integrated Hospital Care, Novartis Pharma AG, Basel, Switzerland THOMAS J. KEAN, Executive Director, C-Change, Washington, DC DOUGLAS R. LOWY, Deputy Director, National Cancer Institute, Bethesda, Maryland DANIEL R. MASYS, Afﬁliate Professor, Department of Medical Education and Biomedical Informatics, University of Washington, Seattle, Washington MARTIN J. MURPHY, Chief Executive Ofﬁcer, CEO Roundtable on Cancer, Durham, North Carolina BRENDA NEVIDJON, Clinical Professor and Specialty Director, Nursing & Healthcare Leadership, Duke University School of Nursing, Durham, North Carolina, and Past President, Oncology Nursing Society STEVEN PIANTADOSI, Director, Samuel Oschin Comprehensive Cancer Institute, Cedars-Sinai Medical Center, Los Angeles, California LISA C. RICHARDSON, Associate Director for Science, Division of Cancer Prevention and Control, Centers for Disease Control and Prevention, Atlanta, Georgia YA-CHEN TINA SHIH, Director, Program in the Economics of Cancer, University of Chicago, Chicago, Illinois ELLEN SIGAL, Chairperson and Founder, Friends of Cancer Research, Washington, DC JOHN A. WAGNER, Vice President, Clinical Pharmacology, Merck and Company, Inc., Rahway, New Jersey RALPH R. WEICHSELBAUM, Chair, Radiation and Cellular Oncology, and Director, Ludwig Center for Metastasis Research, The University of Chicago Medical Center, Chicago, Illinois JANET WOODCOCK, Director, Center for Drug Evaluation and Research, Food and Drug Administration, Rockville, Maryland National Cancer Policy Forum Staff SHARYL J. NASS, Director, National Cancer Policy Forum LAURA LEVIT, Program Ofﬁcer CHRISTINE MICHEEL, Program Ofﬁcer ERIN BALOGH, Associate Program Ofﬁcer MICHAEL PARK, Senior Program Assistant PATRICK BURKE, Financial Associate SHARON B. MURPHY, Scholar-in-Residence ROGER HERDMAN, Director, Board on Health Care Service vii
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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 deliberative process. We wish to thank the following individuals for their review of this report: Bettye Green, Chair, ACOSOG Patient Advocates, South Bend, Indiana Michael Katz, Chair, Patient Advisory Board, Coalition of Cancer Cooperative Groups, Bayside, New York Heidi Nelson, Fred C. Andersen Professor of Surgery, Division of Colon and Rectal Surgery, Mayo Clinic, Rochester, Minnesota, and Group Co-Chair, ACOSOG Edith A. Perez, Deputy Director, Mayo Clinic Cancer Center, Serene M. and Frances C. Durling Professor of Medicine, Mayo Clinic Florida, Jacksonville, Florida, and Breast Committee Chair, NCCTG ix
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x REVIEWERS Although the reviewers listed have provided many constructive com- ments and suggestions, they were not asked to endorse the conclusions or recommendations nor did they see the ﬁnal draft of this report before its release. The review of this report was overseen by Melvin Worth. 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 report rests entirely with the rapporteurs and the institution.
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Contents INTRODUCTION, 1 PANEL I: NCI AND THE COOPERATIVE GROUPS, 4 NCI Perspective and Current Activities, 4 Overview of the NCI Response, 4 Response of the NCI Community Clinical Oncology Program (CCOP), 19 Cooperative Group Leadership Perspective and Current Activities, 22 Cooperative Group Chairs’ Perspective, 22 Group Statisticians’ Perspective, 26 Experience from the Consolidation of the Children’s Oncology Group (COG), 27 Panel Discussion, 30 Consolidation of Cooperative Groups, 30 Encouraging Collaboration, 32 Reducing NCI Oversight of the Cooperative Groups, 34 Going Forward, 36 PANEL II: PAYORS, 36 Payor Policies, 37 An Insurer’s Perspective, 37 Kaiser Permanente’s Dual Role, 39 Medicare and Medicaid Support of Clinical Trials, 40 Demonstrating Utility to Payors, 40 xi
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xii CONTENTS Analysis of Cancer Clinical Trials Coverage, 42 Coverage of Phase I Trials, 44 Determining the Standard of Care, 48 Establishing New Common Procedural Terminology (CPT) Codes for Care of Patients Enrolled in Clinical Trials, 48 PANEL III: INTERACTIONS BETWEEN INDUSTRY, THE FDA, AND THE PUBLICLY FUNDED CANCER CLINICAL TRIALS SYSTEM, 50 Pharmaceutical Industry Perspective, 51 Diagnostic Industry Perspective, 52 FDA Perspective, 54 Central IRB and Informed Consent, 57 Partnerships Between Industry and the Cooperative Groups, 59 Informatics, 64 Vendor Perspective, 64 Completeness Versus Flexibility, 66 PANEL IV: CLINICAL TRIALS INVESTIGATORS AND PATIENT ADVOCATES, 69 Clinical Trials Investigators, 69 CCOP Physician Perspective, 69 Academic Clinical Investigator Perspective, 74 Community Cancer Center Perspective, 76 Patient Advocates, 78 Cooperative Group Patient Advocate Perspective, 78 Research Foundation Perspective, 80 Panel Discussion, 81 Maintaining Engagement of Cooperative Group Investigators, 81 Measuring Success, 82 Open Trial Enrollment, 84 Selection of Trial Sites, 85 Funding Clinical Trials, 86 CLOSING REMARKS, 87 REFERENCES, 91 ACRONYMS, 95 APPENDIXES A Workshop Agenda, 99 B Executive Summary of A National Cancer Clinical Trials System for the 21st Century, 105
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xiii CONTENTS BOXES, TABLES, AND FIGURES BOX 1: Summary of the IOM Consensus Recommendations, 3 BOX 2: Timeline for Implementing a New Funding Opportunity Announcement for the Cooperative Group Program, 14 BOX 3: NCCN Categories of Evidence and Consensus, 38 BOX 4: CCOP Strategic Goals, 2010, 71 TABLE 1: Progress by NCI Toward Goals and Recommendations of the IOM Report A National Cancer Clinical Trials System for the 21st Century (2010), 6 TABLE 2: Overall Cooperative Group Funding Structure, Fiscal Year 2007, 24 TABLE 3: Rates of Approval for Coverage of Care in a Cancer Clinical Trial, by Insurance Provider, at Johns Hopkins University Hospital, from July 2003 to July 2008, 45 FIGURE 1: NCI’s proposed new organizational structure for the Cooperative Group Program, 17 FIGURE 2: States with laws mandating clinical trials coverage, 44 FIGURE 3: Distribution of the 63 CCOP and Minority CCOP sites across the United States and in Puerto Rico, as of June 2010, 71
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