Implementing a National Cancer Clinical Trials System for the 21st Century: Second Workshop Summary (2013)
Chapter: Front Matter
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
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 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 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 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: 978-0-309-28724-1
International Standard Book Number-10: 0-309-28724-3
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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.
“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
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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.
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
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 individual documents. The responsibility for the published workshop summary rests with the workshop rapporteurs and the institution.
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
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
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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:
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 workshop summary before its release. The review of this report was overseen by
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.
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 implementing 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.
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Lessons from the National Clinical Trials System in the United Kingdom
Studies More Appropriate for NCTN Than Industry
INCENTIVIZING PATIENT AND PHYSICIAN PARTICIPATION
Increasing Physician Participation
Regulatory Oversight for Trials of Small Subsets of Cancer Patients
Progression-Free Survival Endpoints and Bias
New Models for Regulation of Drug Development
A Workshop Statement of Task and Agenda
B Executive Summary of A National Cancer Clinical Trials System for the 21st Century
Boxes, Figures, and Table
BOXES
1 Overview of Key Achievements Since 2010
2 Overview of Suggestions Made by Individual Participants
3 Reconfigured Groups of the NCTN
4 Common IT Data Management System (CDMS)
5 Criteria for Evaluating NCTN Trials
FIGURES
3 Clinical development times vary by period and across therapeutic classes, 2000-2011
4 2013 site map for the NCI Community Oncology Research Program (NCORP)
5 Adaptive licensing captures more of the patient experience
TABLE
