IMPLEMENTING A
NATIONAL CANCER
CLINICAL TRIALS SYSTEM
FOR THE 21ST CENTURY

Second Workshop Summary

An American Society of Clinical Oncology and

Institute of Medicine Workshop

Sharyl J. Nass and Margie Patkak, 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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An American Society of Clinical Oncology and Institute of Medicine Workshop Sharyl J. Nass and Margie Patkak, 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 co-sponsored by the American Society of Clinical Oncology. The National Cancer Policy Forum is also supported by Contract Nos. HHSN261200900003C and 200-2011-38807 between the National Academy of Sciences and the National Cancer Institute and the Centers for Disease Control and Prevention, respectively, and by the American Association for Cancer Research, the American Cancer Society, the Ameri- can Society for Radiation Oncology, the American Society of Clinical Oncology, the Association of American Cancer Institutes, Bristol-Myers Squibb, C-Change, the CEO Roundtable on Cancer, GlaxoSmithKline, LIVESTRONG, 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:  978-0-309-28724-1 International Standard Book Number-10:  0-309-28724-3 Additional copies of this report 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 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). 2013. Implementing a national cancer clinical trials system for the 21st century: Second 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. 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 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 MONICA BERTAGNOLLI (Chair), Professor of Surgery, Harvard University Medical School, Boston, MA, and Chair, Alliance for Clinical Trials in Oncology AMY P. ABERNETHY, Associate Professor of Medicine, Duke University School of Medicine, and Director, Duke Cancer Care Research Program, Durham, NC MICHAEL A. CALIGIURI, Director, Ohio State Comprehensive Cancer Center, Columbus, and President, Association of American Cancer Institutes RENZO CANETTA, Vice President, Oncology Global Clinical Research, Bristol-Myers Squibb, Wallingford, CT MICHAELE CHAMBLEE CHRISTIAN, Retired, Washington, DC JAMES H. DOROSHOW, Deputy Director for Clinical and Translational Research, National Cancer Institute, Bethesda, MD PATRICK GAVIN, President, Patrick Gavin R.Ph. Consulting LLC, Marne, MI GREGORY H. REAMAN, Associate Director for Oncology Sciences, Office of Hematology and Oncology Products, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD GEORGE W. SLEDGE, JR., Chief of Oncology, Department of Medicine, Stanford University, CA RALPH R. WEICHSELBAUM, Chair, Radiation and Cellular Oncology, and Director, Ludwig Center for Metastasis Research, University of Chicago Medical Center, IL ROBIN ZON, Partner and Vice President, Michiana Hematology- Oncology, and Principal Investigator, Northern Indiana Cancer Research Consortium–Community Clinical Oncology Program, South Bend, IN 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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IOM Project Staff SHARYL J. NASS, Director, National Cancer Policy Forum PAMELA LIGHTER, Research Assistant MICHAEL PARK, Senior Program Assistant CHER HUANG, Summer Intern, MIT in Washington Program (May-August 2013) ROGER HERDMAN, Director, Board on Health Care Services American Society of Clinical Oncology Project Staff SUANNA BRUINOOGE, Director, Research Policy Division, Cancer Policy and Clinical Affairs Department vi

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NATIONAL CANCER POLICY FORUM1 JOHN MENDELSOHN (Chair), Director, Khalifa Institute for Personalized Cancer Therapy, MD Anderson Cancer Center, Houston, TX 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, Durham, NC RAFAEL G. AMADO, Senior Vice President and Head of R&D, GlaxoSmithKline-Oncology, Collegeville, PA FRED APPELBAUM, Director, Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA PETER B. BACH, Attending Physician, Memorial Sloan-Kettering Cancer Center, New York, NY EDWARD BENZ, JR., President, Dana-Farber Cancer Institute and Director, Harvard Cancer Center, Harvard School of Medicine, Boston, MA MONICA BERTAGNOLLI, Professor of Surgery, Harvard University Medical School, Boston, MA OTIS BRAWLEY, Chief Medical Officer and Executive Vice President, American Cancer Society, Atlanta, GA MICHAEL A. CALIGIURI, Director, Ohio State Comprehensive Cancer Center, Columbus RENZO CANETTA, Vice President, Oncology Global Clinical Research, Bristol-Myers Squibb, Wallingford, CT MICHAELE CHAMBLEE CHRISTIAN, Retired, Washington, DC WILLIAM DALTON, CEO, M2Gen Personalized Medicine Institute, Moffitt Cancer Center, Tampa, FL, 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 indi- vidual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution. vii

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WENDY DEMARK-WAHNEFRIED, Associate Director for Cancer Prevention and Control, University of Alabama at Birmingham Comprehensive Cancer Center ROBERT ERWIN, President, Marti Nelson Cancer Foundation, Davis, CA CAROL HAHN, Medical Director of Radiation Oncology, Duke University Medical Center, Raleigh, NC ROY S. HERBST, Chief of Medical Oncology, Yale Cancer Center, New Haven, CT THOMAS J. KEAN, President and CEO, C-Change, Washington, DC MICHELLE M. LE BEAU, Arthur and Marian Edelstein Professor of Medicine and Director, Comprehensive Cancer Center, University of Chicago, IL, and President, Association of American Cancer Institutes DOUGLAS R. LOWY, Deputy Director, National Cancer Institute, Bethesda, MD DANIEL R. MASYS, Affiliate Professor, Biomedical Informatics, University of Washington, Seattle MARTIN J. MURPHY, Chief Executive Officer, CEO Roundtable on Cancer, Durham, NC BRENDA NEVIDJON, Clinical Professor and Specialty Director, Nursing & Healthcare Leadership, Duke University School of Nursing, Durham, NC, and Past President, Oncology Nursing Society STEVEN PIANTADOSI, Director, Samuel Oschin Comprehensive Cancer Institute, Cedars-Sinai Medical Center, Los Angeles, CA RUTH RECHIS, Vice President of Programs, LIVESTRONG Foundation, Austin, TX LISA C. RICHARDSON, Associate Director for Science, Division of Cancer Prevention and Control, Centers for Disease Control and Prevention, Atlanta, GA DEBASISH ROYCHOWDHURY, Senior Vice President, Global Oncology, Sanofi Oncology, Cambridge, MA YA-CHEN TINA SHIH, Director, Program in the Economics of Cancer, University of Chicago, IL ELLEN SIGAL, Chairperson and Founder, Friends of Cancer Research, Washington, DC STEVEN STEIN, Senior Vice President, U.S. Clinical Development and Medical Affairs, Novartis Oncology, East Hanover, NJ viii

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JOHN A. WAGNER, Vice President, Clinical Pharmacology, Merck and Company, Inc., Rahway, NJ RALPH R. WEICHSELBAUM, Chair, Radiation and Cellular Oncology, and Director, Ludwig Center for Metastasis Research, University of Chicago Medical Center, IL JANET WOODCOCK, Director, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Rockville, MD National Cancer Policy Forum Staff SHARYL J. NASS, Director LAURA LEVIT, Program Officer ERIN BALOGH, Associate Program Officer PAMELA LIGHTER, Research Assistant MICHAEL PARK, Senior Program Assistant CHER HUANG, Summer Intern, MIT in Washington Program (May-August 2013) PATRICK BURKE, Financial Associate SHARON B. MURPHY, Scholar in Residence ROGER HERDMAN, Director, Board on Health Care Services 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: JAN BUCKNER, Chair and Professor, Department of Oncology, Mayo Clinic BARBARA CONLEY, Associate Director, Cancer Diagnosis Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute MARY LOU SMITH, Co-founder, Research Advocacy Network Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the work- shop summary before its release. The review of this report was overseen by xi

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xii REVIEWERS JOHANNA T. DWYER, Professor of Medicine and Community Health, Tufts University School of Medicine, and Senior Nutrition Scientist, National Institutes of Health. 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 We thank the American Society of Clinical Oncology for generously co-sponsoring this workshop. The support of the many sponsors of the Institute of Medicine’s National Cancer Policy Forum was crucial to the planning and conduct of this workshop. Federal sponsors are the National Cancer Institute and the Centers for Disease Control and Prevention. Non- federal sponsors include 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, Bristol-Myers Squibb, C-Change, the CEO Roundtable on Cancer, GlaxoSmithKline, LIVESTRONG, 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 challenges and opportunities in implement- ing a national cancer clinical trials system. The Forum also wishes to thank the members of the planning committee for their work in developing an excellent workshop agenda. xiii

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Contents INTRODUCTION, 1 IMPROVING SPEED AND EFFICIENCY OF TRIALS, 9 Reorganization of the NCTN, 9 Aggressive Timelines, 11 Information Technology Improvements, 13 More Collaborations, 13 Partnering with Industry, 15 Global Collaborations, 18 Partnering with Cancer Centers, 21 Partnering with Foundations, 23 An Example of Collaboration in Cardiovascular Research, 24 The Timeline for Cancer Drug Development, 27 FOSTERING INNOVATION, 29 Collecting Biospecimens, 30 Testing Biospecimens, 32 Interpreting Molecular Tests, 32 Innovative Trial Designs, 35 NCI Initiatives, 40 IMPROVING PRIORITIZATION, SELECTION, SUPPORT, AND COMPLETION OF TRIALS, 41 Trial Reimbursements, 42 Identifying Strengths and Gaps in Trial Portfolios, 42 xv

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xvi CONTENTS Lessons from the National Clinical Trials System in the United Kingdom, 45 Studies More Appropriate for NCTN Than Industry, 49 INCENTIVIZING PATIENT AND PHYSICIAN PARTICIPATION, 51 Boosting Patient Accruals, 51 Increasing Physician Participation, 53 Tapping Community Practices, 54 REGULATORY ISSUES, 58 Regulation of Biomarker Tests, 59 Regulatory Oversight for Trials of Small Subsets of Cancer Patients, 61 Less Burdensome Reporting, 62 Progression-Free Survival Endpoints and Bias, 64 Global Regulation, 67 New Models for Regulation of Drug Development, 69 WRAP-UP, 72 REFERENCES, 75 ACRONYMS, 79 APPENDIXES A Workshop Statement of Task and Agenda, 83 B Executive Summary of A National Cancer Clinical Trials System for the 21st Century, 89

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Boxes, Figures, and Table BOXES 1 Overview of Key Achievements Since 2010, 3 2 Overview of Suggestions Made by Individual Participants, 3 3 Reconfigured Groups of the NCTN, 9 4 Common IT Data Management System (CDMS), 14 5 Criteria for Evaluating NCTN Trials, 43 FIGURES 1 Timeline comparison of trial activation, historical versus post- implementation of the recommendations from the OEWG, April 2010 to August 2012, 12 2 TIMI trial organization, 26 3 Clinical development times vary by period and across therapeutic classes, 2000-2011, 28 4  site map for the NCI Community Oncology Research 2013 Program (NCORP), 56 5 Adaptive licensing captures more of the patient experience, 71 TABLE 1  Progress Toward Recommendations of the IOM Report A NCI National Cancer Clinical Trials System for the 21st Century (2010), 4 xvii

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