Aug 1

Past Event

Special Medical Use: Limited Use for Drugs Developed in an Expedited Manner to Meet an Unmet Medical Need

Event Materials

Summary

The President's Council of Advisors on Science and Technology’s (PCAST) 2012 report, Propelling Innovation in Drug Discovery, Development, and Evaluation recommended that U.S. Food and Drug Administration (FDA) develop a new regulatory pathway to accelerate the development and approval of drugs by allowing new treatments to be studied in a narrow subpopulation of high-risk patients with unmet therapeutic needs.

On August 1, 2013, the Engelberg Center for Health Care Reform (ECHCR) convened an expert workshop to discuss a proposed limited-use approval pathway for drugs developed in an expedited manner to meet unmet medical needs based upon the PCAST recommendation. This workshop is part of an ongoing cooperative agreement with the FDA, and brought together a diverse group of stakeholders, including clinicians, pharmacists, provider systems, health plans, industry representatives, and patients to discuss the potential benefits and implementation challenges of such a pathway. The conversation covered a variety of topics including:

  • possible characteristics of a new pathway,
  • how stakeholders can cooperate to promote the appropriate use of limited-use products once they enter the market, and
  • additional evidence development and safety surveillance in the postmarket setting.

Event Agenda

  • 9:00

    Welcome, Introduction and Meeting Objectives

  • 9:15

    Facilitating Drug Development for Unmet Medical Needs

    • Janet Woodcock

      U.S. Food and Drug Administration

  • 9:30

    Session I The Special Medical Use Pathway Proposal

    • Moderator

      mmclellanimage

      Mark B. McClellan

      Director, Health Care Innovation and Value Initiative

      Senior Fellow, Economic Studies

    • Jim Greenwood

      Biotechnology Industry Organization

    • John Castellani

      Pharmaceutical Research and Manufacturers of America

    • Jack W. Lasersohn

      The Vertical Group

    • Margaret Anderson

      FasterCures

    • Jeff Allen

      Friends of Cancer Research

  • 10:30

    Session II Implementation and Impact of Special Medical Use Products in Clinical Practice

    • Moderator

      mmclellanimage

      Mark B. McClellan

      Director, Health Care Innovation and Value Initiative

      Senior Fellow, Economic Studies

    • Samuel Nussbaum

      WellPoint, Inc.

    • Brian Solow

      OptumRx

    • Edward J. Septimus

      HCA Healthcare

    • Cheryl Bettigole

      Complete Care Health Network and National Physicians Alliance

    • Thomas Davis

      CVS Caremark

  • 12:45

    Session III: Postmarket Considerations to Promote Safe Use and Continued Evidence Development of Special Medical Use Products

    • mmclellanimage

      Mark B. McClellan

      Director, Health Care Innovation and Value Initiative

      Senior Fellow, Economic Studies

    • Marc Boutin

      National Health Council

    • Robert Califf

      Duke Translational Medicine Institute and Duke University Medical Center

    • Elizabeth Chrischilles

      University of Iowa College of Public Health

    • Preeti Pinto

      Preeti Pinto and Associates, LLC

  • 1:45

    Recap and Closing Remarks

Details

August 1, 2013

9:00 AM - 2:00 PM EDT

The Brookings Institution

1775 Massachusetts Ave., NW

Map

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