Leveraging Real-World Treatment Experience from EA Protocols

See Event Highlights and Download the Meeting Report 

Monday, November 19, 2018   |  10:00 AM to 4:30 PM

U.S. Food and Drug Administration
White Oak Campus, Building 31, Great Room
10903 New Hampshire Avenue, Silver Spring, MD 20993 

Building on the Reagan-Udall Foundation for the FDA’s work with FDA and other stakeholders to develop the Expanded Access Navigator, we are convening stakeholders from government, industry, academia and patient groups to discuss process issues that include:

  • Increase understanding of the expanded access program
    • Identify and discuss strengths, challenges and opportunities
  • Discuss what could be the optimum balance between providing access for treatment and obtaining data in support of regulatory applications
    • How could the data be leveraged?
    • How could it be optimized without overburdening companies and providers?
  • Provide input to FDA on future communications regarding utilizing data from Expanded Access (investigational) protocols





Ellen Sigal, Ph.D., Chair, Board of Directors, Reagan-Udall Foundation for the FDA, and, Chairperson and Founder of Friends of Cancer Research



Opening Remarks

  Scott Gottlieb, M.D., Commissioner, U.S. Food and Drug Administration (FDA)


Investigational Therapies and Data Collection

  Janet Woodcock, M.D., Director, Center for Drug Evaluation Research (CDER), FDA


Panel 1: Patient, Physician, and Bioethics Perspectives


Moderator: Richard L. Schilsky, M.D., FACP, FSCT, FASCO, Vice-Chair, Board of Directors, Reagan-Udall Foundation for the FDA, and Senior Vice President and Chief Medical Officer, American Society of Clinical Oncology


Alison Bateman-House, M.P.H., Ph.D., Assistant Professor, Department of Population Health, New York University School of Medicine

Paul Melmeyer, Director of Federal Policy, National Organization for Rare Disorders

Emil Kakkis, M.D., Ph.D., President/CEO, Ultragenyx, and Member, Board of Directors, EveryLife Foundation



Lunch Break



Panel 2: Industry Case Study Presentations


Moderator: Kay Holcombe, Secretary, Board of Directors, Reagan-Udall Foundation for the FDA


David Meeker, M.D., Chief Executive Officer, KSQ Therapeutics

Paul Aliu, Pharm.D., M.B.A., Global Head, Medical Governance, Novartis

Joanne Waldstreicher, M.D., Chief Medical Officer, Johnson & Johnson

Jayne C. Gershkowitz, Chief Patient Advocate, Amicus Therapeutics

2:15pm Break


Panel 3:  Expanding Impact of Expanded Access 


Moderator: Andrew von Eschenbach, M.D., President, Samaritan Health Initiatives Inc., Member, Reagan-Udall Board of Directors, and 20th Commissioner of the FDA


Jacqueline Corrigan-Curay, M.D., J.D., Director, Office of Medical Policy, CDER, FDA

Peter Marks, M.D., Ph.D., Director, Center for Biologics Evaluation and Research, FDA

Amy McKee, M.D., Deputy Director, Oncology Center of Excellence, FDA

Robert Temple, M.D., Deputy Center Director for Clinical Sciences, CDER, FDA

Peter Stein, M.D., Deputy Director, Office of New Drugs, CDER, FDA



Public Comments



Closing Remarks

  June Wasser, M.A., Executive Director, Reagan-Udall Foundation for the FDA