FEDERAL AGENCY ROLES IN CANCER DRUG DEVELOPMENT FROM PRECLINICAL RESEARCH TO NEW DRUG APPROVAL:

THE NATIONAL CANCER INSTITUTE AND THE FOOD AND DRUG ADMINISTRATION

Background paper prepared for the National Cancer Policy Board

Joseph P.Newhouse, Harvard University

John Mendelsohn, M.D. Anderson Cancer Center

Hellen Gelband and Roger Herdman, Institute of Medicine

MARCH 2005




INSTITUTE OF MEDICINE AND
NATIONAL RESEARCH COUNCIL
OF THE NATIONAL ACADEMIES



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Federal Agency Roles in Cancer Drug Development from Preclinical Research to New Drug Approval: The National Cancer Institute and the Food and Drug Administration FEDERAL AGENCY ROLES IN CANCER DRUG DEVELOPMENT FROM PRECLINICAL RESEARCH TO NEW DRUG APPROVAL: THE NATIONAL CANCER INSTITUTE AND THE FOOD AND DRUG ADMINISTRATION Background paper prepared for the National Cancer Policy Board Joseph P.Newhouse, Harvard University John Mendelsohn, M.D. Anderson Cancer Center Hellen Gelband and Roger Herdman, Institute of Medicine MARCH 2005 INSTITUTE OF MEDICINE AND NATIONAL RESEARCH COUNCIL OF THE NATIONAL ACADEMIES

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Federal Agency Roles in Cancer Drug Development from Preclinical Research to New Drug Approval: The National Cancer Institute and the Food and Drug Administration 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 No. NO2-CO-01029 between the National Academy of Sciences and the National Cancer Institute. 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. 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 2005 by the National Academy of Sciences. All rights reserved. Printed in the United States of America.

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Federal Agency Roles in Cancer Drug Development from Preclinical Research to New Drug Approval: The National Cancer Institute and the Food and Drug Administration “Knowing is not enough; we must apply. Willing is not enough; we must do.” —Goethe INSTITUTE OF MEDICINE OF THE NATIONAL ACADEMIES Adviser to the Nation to Improve Health

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Federal Agency Roles in Cancer Drug Development from Preclinical Research to New Drug Approval: The National Cancer Institute and the Food and Drug Administration 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. Bruce M.Alberts 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. Bruce M.Alberts and Dr. Wm. A.Wulf are chair and vice chair, respectively, of the National Research Council. www.national-academies.org

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Federal Agency Roles in Cancer Drug Development from Preclinical Research to New Drug Approval: The National Cancer Institute and the Food and Drug Administration PREFACE The interplay between the public and private sectors in health care is nowhere more intricate than in the case of cancer. From basic research to the panoply of control measures, to the subject of this background paper—the development of new drugs and biological agents to treat the disease most feared by Americans—the leading federal government agencies have played essential roles. The Food and Drug Administration (FDA) is an obvious player, responsible for expediting the flow of new agents for the full range of health conditions to the public, while protecting from undue risk. Challenges for FDA abound throughout the medical spectrum, but cancer occupies a special place, with rules and procedures peculiar to the disease, its treatment, and the public’s clamor for faster progress. It should surprise no one that the National Cancer Institute (NCI) is at the forefront of basic research on cancer, but the historic and current depth of the Institute’s involvement in drug development is an anomaly among the government’s disease-oriented institutes. Its role in relation to the traditional pharmaceutical industry, small and large biotechnology companies, and the academic community has been central since the 1940s, but has changed over time as the capacity and priorities of the other parties have evolved. Continued success will depend on the flexibility to fully complement the other sectors with cutting-edge technical capabilities as well as having in place the administrative means to join easily with them throughout the discovery and development process. These issues were part of the very ambitious scope of work of a committee created under the aegis of the National Cancer Policy Board to analyze every aspect of the way new agents to fight cancer are developed and to search for ways to streamline the process. This scope was overwhelming, and it became clear that shorter reports, a typical committee report and three separate authored papers on various aspects of the process, would be more manageable and provide more accessible products. This authored background paper, describing the federal role in cancer drug development, is one of those. The other products from this study are a committee report on the development of agents to treat childhood cancers and two authored background papers covering the use of human tissues in research to develop new anticancer agents and issues in intellectual property among government, industry, and academia. We believe this paper provides useful background detail for those interested in exploring issues that will be informed by how the FDA and NCI act and interact to develop cancer drugs from preclinical research to new drug approval. Joseph P.Newhouse, Ph.D.

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Federal Agency Roles in Cancer Drug Development from Preclinical Research to New Drug Approval: The National Cancer Institute and the Food and Drug Administration ACKNOWLEDGMENTS The authors gratefully acknowledge the contributions of the following individuals who provided information and insight for this paper, either through formal presentations or through informal contacts: Scott Armstrong, M.D., Ph.D. William Cotreau Timothy Hoey, Ph.D. Peter Houghton, Ph.D. Tyler Jacks, Ph.D. Elizabeth Jaffee, M.D. Nina Mani, Ph.D. Cheryl Marks, Ph.D. Sharyl J.Nass, Ph.D. Andrew Neighbour, Ph.D. Drew Pardoll, M.D. Richard Pazdur, M.D. Emanuel Petricoin, Ph.D. Edward A.Sausville, M.D., Ph.D. David Sidransky, M.D. Malcolm Smith, M.D., Ph.D. Robert Spiegel, M.D. Sudhir Srivastava, Ph.D. Beverly A.Teicher, Ph.D. Robert Tepper, M.D. Joseph E.Tomaszewski, Ph.D. J.Gregory Townsend Jeffrey M.Trent, Ph.D.

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Federal Agency Roles in Cancer Drug Development from Preclinical Research to New Drug Approval: The National Cancer Institute and the Food and Drug Administration TABLE OF CONTENTS     PREFACE   i     ACKNOWLEDGEMENTS   ii 1—   SCIENTIFIC RATIONALE   1 2—   THE NATIONAL CANCER INSTITUTE   7 3—   THE FOOD AND DRUG ADMINISTRATION   28 4—   SUMMARY: THE ROLES OF THE NCI AND FDA IN DEVELOPING ANTICANCER AGENTS   54     REFERENCES   56     ACRONYMS AND ABBREVIATIONS   61

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