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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 will host 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 is to discuss topics relevant to the development of endpoints for rare diseases.

This workshop will discuss:

• 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 includes 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 will allow 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 will not be addressed, topics in general will be discussed and case studies will be presented as learning examples. 

For more information, please visit the Duke-Margolis Center for Health Policy RDEA public workshop webpage.  Workshop materials and the workshop recording are now available on the webpage.

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