U.S. flag An official website of the United States government

On Oct. 1, 2024, the FDA began implementing a reorganization impacting many parts of the agency. We are in the process of updating FDA.gov content to reflect these changes.

U.S. Food and Drug Administration
  1. Home
  2. Drugs
  3. News & Events for Human Drugs
  4. FDA CDER and CBER & Duke-Margolis Center for Health Policy | Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development - 06/07/2023
  1. News & Events for Human Drugs

Public | Virtual

Event Title
FDA CDER and CBER & Duke-Margolis Center for Health Policy | Rare Disease Endpoint Advancement Pilot Program Workshop: Novel Endpoints for Rare Disease Drug Development
June 7 - 8, 2023

Date:
June 7 - 8, 2023
Day1:
Wed, Jun 07 1:00 p.m. - 05:00 p.m. ET
Day2:
Thu, Jun 08 1:00 p.m. - 05:00 p.m. ET

Meeting Summary and Objectives

On June 7 and 8, 2023, the FDA’s Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and the Duke-Margolis Center for Health Policy hosted a jointly sponsored virtual public workshop on the Rare Disease Endpoint Advancement (RDEA) Pilot Program and novel endpoint development for rare disease drug development. The purpose of this public workshop was to discuss topics relevant to the development of endpoints for rare diseases.

This workshop discussed:

  • RDEA Pilot Program, including application process and required elements for the RDEA proposal
  • Scientific and technical issues associated with developing study endpoints for rare diseases
  • Lessons learned from previous PDUFA meeting programs that can be applied to the RDEA Pilot Program

The lack of regulatory precedent, small trial populations, and/or limited understanding of disease natural history associated with rare diseases creates unique challenges when determining the appropriate efficacy endpoint(s) for clinical trials intended to evaluate the effectiveness of rare disease therapies.  Well-developed efficacy endpoints, especially those that could apply to other rare diseases with similar manifestations, can help drive the general advancement of rare disease drug development.

The primary audience for the workshop included academic investigators, pharmaceutical and biotechnology companies, patient advocacy organizations, and anyone looking to learn how to develop rigorous endpoints for rare disease drug development.

Participation in this workshop allowed attendees to improve their familiarity and understanding of the RDEA Pilot Program and the issues associated with development of efficacy endpoints for clinical trials of therapies for rare diseases. While specific questions about applications were not addressed, topics in general were discussed and case studies were presented as learning examples.

Workshop materials and recordings are available on the Duke-Margolis Center for Health Policy RDEA public workshop webpage and this webpage.

Workshop Agenda

Speaker Bios

Workshop Resource Guide

Workshop Recordings

Presentation Slides

Transcripts

For questions, please email: RDEA.Meetings@fda.hhs.gov.

Back to Top