Policy Issues in the Development of Personalized Medicine in Oncology

WORKSHOP SUMMARY

Margie Patlak and Laura Levit, 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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Margie Patlak and Laura Levit, 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 Institute of Medicine. This study was supported by Contract Nos. HHSN261200611002C, 200-2005- 13434 TO #1, and 223-01-2460 to #27, between the National Academy of Sciences and the National Cancer Institute, the Centers for Disease Control and Prevention, and the Food and Drug Administration, respectively. This study was also supported by the American Cancer Society, the American Society of Clinical Oncology, the Association of American Cancer Institutes, and C-Change. 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: 978-0-309-14575-6 International Standard Book Number-10: 0-309-14575-9 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 2010 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. Cover art created by Tim Cook and used with permission from the National Institutes of Health, 2004. Suggested citation: IOM (Institute of Medicine). 2010. Policy issues in the development of personalized medicine in oncology: Workshop summary. Washington, DC: The National Academies Press.

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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 ROy HERbST (Cochair), Professor and Chief, Section on Thoracic Medical Oncology, Department of Thoracic/Head and Neck Medical Oncology, M.D. Anderson Cancer Center, Houston, TX DAvID PARKINSON (Cochair), President and Chief Executive Officer, Nodality, Inc., San Francisco, CA FRED APPELbAuM, Director, Clinical Research Division and Head, Division of Medical Oncology, Fred Hutchinson Cancer Research Center, Seattle, WA PETER bACH, Associate Attending Physician, Memorial Sloan-Kettering Cancer Center, New York, NY RObERT ERWIN, President, Marti Nelson Cancer Foundation, Davis, CA STEPHEN FRIEND, President, Chief Executive Officer, and Cofounder, Sage Bionetworks, Seattle, WA STEvEN GuTMAN, Professor of Pathology, University of Central Florida, Orlando, FL GAIL JAvITT, Law and Policy Director, Genetics and Public Policy Center, Johns Hopkins University, Washington, DC SAMIR KHLEIF, Senior Investigator and Chief of Cancer Vaccine Section, National Cancer Institute, Bethesda, MD Study Staff LAuRA LEvIT, Study Director CASSANDRA L. CACACE, Research Assistant MICHAEL PARK, Senior Program Assistant ASHLEy McWILLIAMS, Senior Program Assistant PATRICK buRKE, Financial Associate SHARyL J. NASS, Director, National Cancer Policy Forum ROGER HERDMAN, Director, Board on Health Care Services SHARON b. MuRPHy, Scholar in Residence 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 HAROLD MOSES (Chair), Director Emeritus, Vanderbilt-Ingram Cancer Center, Nashville, TN FRED APPELbAuM, Director, Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA PETER b. bACH, Associate 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 THOMAS G. buRISH, Past Chair, American Cancer Society Board of Directors and Provost, Notre Dame University, South Bend, IN MICHAELE CHAMbLEE CHRISTIAN, Retired, Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethsda, MD RObERT ERWIN, President, Marti Nelson Cancer Foundation, Davis, CA bETTy R. FERRELL, Research Scientist, City of Hope National Medical Center, Duarte, CA JOSEPH F. FRAuMENI, JR., Director, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, MD PATRICIA A. GANZ, Professor, University of California, Los Angeles, Schools of Medicine & Public Health, Division of Cancer Prevention & Control Research, Jonsson Comprehensive Cancer Center, Los Angeles, CA RObERT R. GERMAN, Associate Director for Science (Acting), Division of Cancer Prevention and Control, Centers for Disease Control and Prevention, Atlanta, GA ROy S. HERbST, Chief, Thoracic/Head & Neck, Medical Oncology, M.D. Anderson Cancer Center, Houston, TX THOMAS J. KEAN, Executive Director, C-Change, Washington, DC JOHN MENDELSOHN, President, M.D. Anderson Cancer Center, Houston, TX 1 IOM 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. vi

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JOHN E. NIEDERHubER, Director, National Cancer Institute, Bethesda, MD DAvID R. PARKINSON, President and Chief Executive Officer, Nodality, Inc., San Francisco, CA SCOTT RAMSEy, Full Member, Cancer Prevention Program, Fred Hutchinson Cancer Research Center, Seattle, WA JOHN WAGNER, Executive Director, Clinical Pharmacology, Merck and Company, Inc., Whitehouse Station, NJ JANET WOODCOCK, Deputy Commissioner and Chief Medical Officer, Food and Drug Administration, Rockville, MD National Cancer Policy Forum Staff SHARyL NASS, Director, National Cancer Policy Forum LAuRA LEvIT, Program Officer CHRISTINE MICHEEL, Program Officer ERIN bALOGH, Research Associate ASHLEy McWILLIAMS, Senior Program Assistant MICHAEL PARK, Senior Program Assistant PATRICK buRKE, Financial Associate SHARON b. MuRPHy, Scholar in Residence ROGER HERDMAN, Director, Board on Health Care Services 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 process. We wish to thank the following individuals for their review of this report: ELI ADASHI, Professor of Medical Sciences, Brown University, Providence, RI STEvEN GuTMAN, Professor of Pathology, University of Central Florida, Orlando, FL GAIL JAvITT, Law and Policy Director, Genetics and Public Policy Center, Johns Hopkins University, Washington, DC MuIN KHOuRy, Director, Office of Public Health Genomics, Centers for Disease Control and Prevention, Atlanta, GA Although the reviewers listed above have provided many constructive comments and suggestions, they were not asked to endorse the final draft ix

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x REVIEWERS of the 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 authors and the institution.

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Contents INTRODUCTION 1 PERSONALIZED CANCER MEDICINE TECHNOLOGY 3 Deciphering the Clinical Implications, 6 Increasing Complexity of Predictive Tests, 9 Test Validation, 15 Test Reliability, 19 Translation Challenges, 21 Codevelopment Challenges, 23 REGULATION OF PREDICTIVE TESTS 25 Overview of the FDA’s Regulation of Predictive Tests, 25 Overview of CMS’s Regulation of Laboratories Performing Predictive Tests, 28 Should the FDA Do More?, 30 Is the Status Quo Appropriate?, 33 Policy Suggestions, 36 Improve Laboratory Proficiency, 37 Increase Transparency, 38 Restructure and Coordinate Oversight, 39 Improve Enforcement, 40 Assess Clinical Utility, 41 Ways to Capture Clinical Utility Data, 43 xi

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xii CONTENTS REIMBURSEMENT 49 Medicare Coverage of Predictive Tests, 49 Reimbursement Rates, 52 Bundling of Payments, 54 Value of Biomarkers, 56 SUMMARY 63 REFERENCES 65 ACRONYMS 71 GLOSSARY 73 APPENDIXES A Workshop Agenda 77 B Workshop Speakers and Moderators 81