40 years of the Orphan Drug Act, the Rare Disease Endpoint Advancement (RDEA) Pilot Program, and Transforming Rare Disease Development

2023 marks the 40th anniversary of the Orphan Drug Act (ODA). Many advances have been made in the research, development, and approval of products for people with rare diseases. While much progress has been made, there is still a great opportunity for additional advancements. Recent years have seen additional activity and opportunities to further this work. The seventh iteration of the Prescription Drug User Fee Act (PDUFA VII) established the rare disease endpoint advancement (RDEA) pilot program to support the development of efficacy endpoints for rare diseases and drive the general advancement of rare disease drug development, which officially opens for applications in July. FDA has also recently embarked on efforts such as the CDER ARC Program to provide resources, educational opportunities, and increased stakeholder engagement in the rare disease space. With all these initiatives, we have the opportunity to transform rare disease development for the future.

In this session, panelists will reflect on the progress that has been made since the ODA was passed, identify areas of opportunity for the future, discuss stakeholder positions regarding the upcoming RDEA pilot, and envision what the ideal future state for rare disease development is, including implementing lessons learned from the RDEA pilot to ensure advancement of endpoint development.