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U.S. Department of Health and Human Services

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Cancer Drug Approval Endpoints

  • General Background Documents
  • Multiple Myeloma Endpoints
  • Ovarian Cancer Endpoints
  • Primary Brain Tumor Endpoints 
  • Lung Cancer Endpoints
  • Colorectal Cancer Endpoints
  • Prostate Cancer Endpoints
  • Acute Leukemia Endpoints
  • Contact Information

The Food and Drug Administration is undertaking a project to evaluate potential endpoints for cancer drug approval. Endpoints will be examined for the most common cancers, such as lung cancer, colon cancer, etc. For each cancer, FDA will hold public workshops to identify important issues, and these issues will be discussed in meetings of the Oncologic Drugs Advisory Committee (ODAC). Subsequently, guidance documents will be published describing FDA's current thinking on endpoints for cancer drug approval.

Workshop planning will be guided by a steering committee that includes representation from the FDA, the National Cancer Institute, the American Society of Clinical Oncology, and the American Association for Cancer Research. Workshop participants will include oncology experts, radiation oncologists, statisticians, industry representatives, and patient advocates.

Listed below will be agendas, background materials, meeting transcripts, and links providing additional information on workshops and ODAC meetings.

 General Background Documents

 Multiple Myeloma Endpoints

FDA and the American Society of Hematology (ASH), with co-sponsorship by the American Society of Clinical Oncology (ASCO) and the American Association for Cancer Research (AACR), are holding a public workshop on endpoints for multiple myeloma. Issues identified in the workshop will be further discussed before the Oncologic Drugs Advisory Committee (ODAC) and thereafter will be detailed in FDA Guidance Documents. The meeting will be held on October 26, 2006 in Washington, DC.

 Ovarian Cancer Endpoints

FDA and the American Society of Clinical Oncology (ASCO), with co-sponsorship by the American Association for Cancer Research (AACR), public workshop on endpoints for ovarian cancer, April 26, 2006, 7:30 to 5:00 pm, North Bethesda, Maryland.

 Primary Brain Tumor Endpoints

AACR/FDA Public Workshop on Clinical Trial End Points in Primary Brain Tumors, January 20, 2006, 8:00 a.m. to 4:00 p.m. North Bethesda, MD 4:00 to 5:00 pm, Optional Session: NCI Research Priorities.

 Lung Cancer Endpoints

American Society of Clinical Oncology/FDA Lung Cancer Endpoints Workshop (April 15, 2003)

  • Background: Guidance for Industry: FDA Requirements for Approval of Drugs to Treat Non-Small Cell Lung Cancer  (1991)
  • Executive Summary of Workshop Discussion
  • Workshop Participants
  • Final Summary
  • Meeting Presentations
    • Requirements for Drug Approval, Regular Approval Endpoints in Oncology, Grant Williams, MD, FDA.
    • Non-Small Cell Lung Cancer Approved First and Second Line Treatment, Martin Cohen, MD, FDA.
    • Endpoints Used Internationally, Renzo Canetta, MD, Bristol-Myers Squibb.
    • Advanced Non-Small Cell Lung Cancer, David H. Johnson, Vanderbilt University Medical School.
    • Non-Traditional Endpoints in Lung Cancer -  Patient Reported Outcomes, Richard J. Gralla, MD, New York Lung Cancer Alliance.

December 16, 2003 FDA Oncologic Drugs Advisory Committee Meeting on Lung Cancer Endpoints 

  • Meeting Information  NOTE: The meeting will NOT be at the Bethesda Holiday Inn as stated in the Federal Register, rather it will be held at: Room 1066, CDER Advisory Committee Conference Room, 5630 Fishers Lane, Rockville, MD 20857

 Colorectal Cancer Endpoints

FDA Public Workshop on Clinical Trial Endpoints in Colorectal Cancer, November 12, 2003, 8:00 a.m. to 5:00 p.m., Washington DC.

  • FDA Background for Colon Cancer Endpoints Workshop  
  • Colon Cancer Workshop: Questions for Discussion
  • Meeting Presentations
    • Regulatory Background, Grant Williams, MD, FDA.
    • Design Issues in Colorectal Cancer Trials: Surrogates Endpoints and Non-Inferiority Trials, Thomas R. Fleming, Ph.D., University of Washington.
    • Past FDA Approvals in Colorectal Cancer, Amna Ibrahim M.D, FDA
    • Three Year Disease Free Survival (DFS) vs. Five Year Overall survival (OS) as an Endpoint for Adjuvant Colon Cancer Studies: Data from Randomized Trials, D. Sargent, et. al., Mayo Clinic.
    • Potential Use of Biomarkers or Quality of Life (QoL) Parameters in Colorectal Cancer Drug Approval, Charles D. Blanke, M.D., F.A.C.P., Oregon Health & Science University Cancer Institute.
    • Rectal Cancer Endpoints, Margaret Mooney, National Cancer Institute, NIH.
    • A Case for Time to Tumor Progression as a Clinical Benefit Endpoint in the First-line Therapy of Metastatic Colorectal Cancer, Langdon L. Miller, MD, PTC Therapeutics.
  • May 4, 2004 (PM) FDA Oncologic Drugs Advisory Committee Meeting on Colorectal Cancer Endpoints   

 Prostate Cancer Endpoints

FDA Public Workshop on Clinical Trial Endpoints in Prostate Cancer, June 21-22, 2004, 8:00 am -5:00 pm, Bethesda, MD  

 Acute Leukemia Endpoints

FDA Workshop on Clinical Trial Endpoints in Acute Leukemia, June 24, 2005, 8:30 am – 5:00 pm, Washington, DC

  • Meeting Presentations
    • Purpose of Conference and Regulatory Background Nancy Scher, MD; FDA
    • Scope and Background of Panel Frederick R. Appelbaum, MD; Fred Hutchinson Cancer Research Center
    • Scope of Problem/Heterogeneity Component
      Frederick R. Appelbaum, MD; Fred Hutchinson Cancer Research Center
    • Definition of Response Component Susan O’Brien, MD; M.D. Anderson Cancer Center
    • Bridge to Transplant Component Robert J. Arceci, MD, PhD; Johns Hopkins Oncology Center
    • Quality of Life Component  Philip Breitfeld, MD; Duke University Medical Center
    • Chairs’ Questions to Panel  Frederick R. Appelbaum, MD; Fred Hutchinson Cancer Research Center and Daniel Rosenblum, MD; FDA

 For further information, contact:

Dianne Spillman
Office of Oncology Drug Products (HFD-150),
Center for Drug Evaluation and Research, Food and Drug Administration,
10903 New Hampshire Avenue, Silver Spring MD
Phone: (301) 796-1467
Email: dianne.spillman@fda.hhs.gov

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