DEVELOPING BIOMARKER-BASED TOOLS FOR Cancer Screening, Diagnosis, and Treatment

The State of the Science, Evaluation, Implementation, and Economics

WORKSHOP SUMMARY

National Cancer Policy Forum

Margie Patlak and Sharyl Nass, Rapporteurs

INSTITUTE OF MEDICINE OF THE NATIONAL ACADMIES

THE NATIONAL ACADEMIES PRESS

Washington, D.C.
www.nap.edu



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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics DEVELOPING BIOMARKER-BASED TOOLS FOR Cancer Screening, Diagnosis, and Treatment The State of the Science, Evaluation, Implementation, and Economics WORKSHOP SUMMARY National Cancer Policy Forum Margie Patlak and Sharyl Nass, Rapporteurs INSTITUTE OF MEDICINE OF THE NATIONAL ACADMIES THE NATIONAL ACADEMIES PRESS Washington, D.C. www.nap.edu

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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics 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. The members of the committee responsible for the report were chosen for their special competences and with regard for appropriate balance. This study was supported by Contract Nos. HHSH25056133, HHSN261200611002C, 200-2005-13434, HHSM-500-2005-00179P, HHSP23320042509XI, and 223-01-2460 between the National Academy of Sciences and the Department of Health and Human Services. 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-10 0-309-10134-4 International Standard Book Number-13 978-0-309-10134-9 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 2006 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.

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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics “Knowing is not enough; we we must apply. Willing is not enough; we must do.” —Geothe INSTITUTE OF MEDICINE OF THE NATIONAL ACADEMIES Advising the Nation. Improving Health.

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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics 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. Wm. A. Wulf 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. Wm. A. Wulf are chair and vice chair, respectively, of the National Research Council. www.national-academies.org

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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics NATIONAL CANCER POLICY FORUM HAROLD L. MOSES, MD (Chair), Professor of Cancer Biology, Medicine, and Pathology, Director Emeritus, Vanderbilt-Ingram Comprehensive Cancer Center, Vanderbilt University Medical Center PETER BACH, MD, MAPP, Senior Advisor, Office of the Administrator, Centers for Medicare & Medicaid Services EDWARD BENZ, JR., MD, President, Dana-Farber Cancer Center THOMAS BURISH, PhD, Chair, ACS Board and Provost, Notre Dame University MARK CLANTON, MD, MPH, Deputy Director, Cancer Delivery Systems, National Cancer Institute BETTY FERRELL, PhD, RN, FAAN, Research Scientist, City of Hope National Medical Center JOSEPH FRAUMENI, JR., MD, Director, Division of Cancer Epidemiology and Genetics, National Cancer Institute STEPHEN FRIEND, MD, PhD, Executive Vice President, Oncology, Merck PATRICIA GANZ, MD, ASCO Board Member and Professor of Medicine, University of California, Los Angeles THOMAS KEAN, MPH, Executive Director, C-Change WILLIAM LAWRENCE, MD, MS, Director, Center for Outcomes and Evidence, Agency for Healthcare Research and Quality LYNN MATRISIAN, PhD, Chair, Division of Cancer Biology, Vanderbilt University DAVID R. PARKINSON, MD, Senior Vice President, Oncology Research and Development, Biogen Idec EDITH PEREZ, MD, Director, Cancer Clinical Study Unit, Mayo Clinic SCOTT RAMSEY, MD, PhD, Full Member, Fred Hutchinson Cancer Research Center EDDIE REED, MD, Director, Division of Cancer Prevention and Control, Centers for Disease Control and Prevention WILLIAM ROBINSON, MD, MPH, Director, Office of Minority Health and Health Disparities, Health Resources and Services Administration CHARLES SAWYERS, MD, Chairman, Human Oncology and Pathogenesis Program, Memorial Sloan-Kettering Cancer Center

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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics MARGARET SPITZ, MD, Chair of Epidemiology, M.D. Anderson Cancer Center ELLEN STOVALL, President and CEO, National Coalition for Cancer Survivorship JANET WOODCOCK, MD, Deputy Commissioner for Operations, Food and Drug Administration Staff SHARYL NASS, PhD, Study Director ROGER HERDMAN, MD, Director, ALIZA NORWOOD, Research Assistant MARY ANN PRYOR, Senior Program Assistant

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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics 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 (NRC’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: Margaret Pepe, PhD, University of Washington School of Public Health G. Gregory Raab, PhD, Consultant, Raab & Associates, Inc. David Ransohoff, MD, University of North Carolina, Chapel Hill Howard Schulman, PhD, Pharmaceutical Product Development, Inc. (PPD), Biomarker Discovery Sciences Janet Warrington, PhD, Affymetrix, Inc. Although the reviewers listed above have provided many constructive comments and suggestions, they did not see the final draft of the report before its release. The review of this report was overseen by Melvin Worth, MD. Appointed by the NRC, he was responsible for making certain that an

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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics 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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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics Contents     Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics          Introduction   1      Overview of Technologies Used to Discover Cancer Biomarkers   3      Genomics, Proteomics, and Metabolomics   3      Molecular Imaging   9      Meeting the Technical Challenges of Biomarker Validation and Qualification   11      Coordinating the Development of Biomarkers and Targeted Therapies   20      Therapeutics Industry Perspective   21      Diagnostics Industry Perspective   24      NCI Perspective   26      Clinical Investigator Perspective   28      Biomarker Development and Regulatory Oversight   29      FDA Critical Path Initiative   30      Oversight of Diagnostic Tests   32      Designing Clinical Studies of Biomarkers   36      Assessment and Adoption of Biomarker-Based Technologies   39      Federal Programs for Technology Assessment   40      Insurance Coverage Decisions and Practice Guidelines   43      CMS Coverage of Biomarkers   46

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Developing Biomarker-Based Tools for Cancer Screening, Diagnosis, and Treatment: The State of the Science, Evaluation, Implementation, and Economics      Economic Impact of Biomarkers   48      Cost-Effectiveness Analysis   48      The Value of Information and Research   53      Technology Assessment in the Private Sector   53      Clinical Development Strategies for Biomarker Utilization Discussion   57      Strategies for Implementing Standardized Biorepositories Discussion   61      Strategies for Determining Analytic Validity and Clinical Utility of Biomarkers Discussion   66      Strategies to Develop Biomarkers for Early Detection Discussion   69      Mechanisms for Developing an Evidence Base Discussion   73      Evaluation of Evidence in Decision Making Discussion   76      Incorporating Biomarker Evidence into Clinical Practice Discussion   80      Acronyms   85      Glossary   86      References   92     APPENDIXES     A   Workshop Agenda   93 B   Workshop Speakers, Moderators, Invited Discussants, and Participants   99