Jul 16

Past Event

Enhancing the Development and Use of Patient-Reported Outcomes in Drug Development

Summary

Developing valid and reliable instruments for measuring patient-reported outcomes (PROs) is the most direct approach to gaining insights from patients about their symptoms, functional status, treatment preferences, and health-related quality of life. While there is growing interest among drug developers, clinicians, payers, regulators, and patients in the development and widespread use of PRO instruments, progress has been slow. In particular, there are ongoing challenges in using PROs to support product label claims. The U.S. Food and Drug Administration (FDA) released final guidance to industry on the use of PRO instruments to support labeling claims in 2009, and in 2014 released guidance on qualifying instruments that may be used over time in multiple drug development programs. However, the interpretation and implementation of those guidances have presented significant challenges, which stem from a variety of methodological,  logistical, and communication issues.

On July 16, 2014, the Engelberg Center for Health Care Reform, in cooperation with the FDA, held an expert workshop that focused on the major issues related to the development and use of PRO instruments to support labeling claims. This workshop was the first in a series of meetings, and included representatives from across industry, academia, patient advocacy groups, and government agencies. This discussion will help to inform a public workshop that the FDA will hold in spring 2015, which will focus on the development, use, and qualification of drug development tools more broadly and the state of the science around psychometric measurement theory.

Event Agenda

  • 9:00

    Welcome, Overview, and Meeting Objectives

  • 9:15

    Opening Remarks

    • Theresa Mullin

      U.S. Food and Drug Administration

    • Elektra Papadopoulos

      U.S. Food and Drug Administration

    • Ellis Unger

      U.S. Food and Drug Administration

  • 9:30

    Session I: Experiences with the FDA Guidance on PROs: Evidentiary Standards

    • Moderator

      Mark B. McClellan

      Director, Health Care Innovation and Value Initiative

      Senior Fellow, Economic Studies

    • Ethan Basch

      University of North Carolina at Chapel Hill

    • Ari Gnanasakthy

      RTI Health Solutions

    • Tara Symonds

      Pfizer, Inc.

    • Cynthia Bens

      Alliance for Aging Research

    • Amylou Dueck

      Mayo Clinic

  • 11:15

    Session II: Experiences with the FDA Guidance on PROs: Standardizing Communication Processes

    • Moderator

      Mark B. McClellan

      Director, Health Care Innovation and Value Initiative

      Senior Fellow, Economic Studies

    • Nancy Kline Leidy

      Evidera

    • Debra Silberg

      Shire Pharmaceuticals

    • Katarina Halling

      AstraZeneca

    • Donald Patrick

      University of Washington

    • Pat Furlong

      Parent Project Muscular Dystrophy

  • 1:30

    Session III: Identifying Other Issues Related to the FDA Guidance on PRO Development

  • 2:15

    Session IV: Challenges to Capturing the Patient Voice Across the Drug Development Continuum

    • Moderator

      Mark B. McClellan

      Director, Health Care Innovation and Value Initiative

      Senior Fellow, Economic Studies

    • Stephen Joel Coons

      Critical Path Institute

    • James Witter

      National Institutes of Health

    • Debbie Gipson

      University of Michigan

    • Chad Gwaltney

      ERT

    • Marc Boutin

      National Health Council

  • 3:45

    Session V: Next Steps in Promoting the Development and Qualification of PROs

  • 4:15

    Closing Remarks

Details

July 16, 2014

12:00 AM - 12:00 AM EDT

Brookings Falk Auditorium

Falk Auditorium

1775 Massachusetts Ave., NW

Map

For More Information

Brookings Office of Communications

202.797.6105