THE NATIONAL ACADEMIES OF SCIENCES, ENGINEERING, AND MEDICINE
Health and Medicine Division
Board on Health Sciences Policy
Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union
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Congressional Briefing
Wednesday, September 4, 2024 – 9:00 a.m.
via Zoom
on
Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities
Requested by Congress in P.L. 117-328, the Consolidated Appropriations Act, 2023, this new National Academies of Sciences, Engineering, and Medicine report looks at the processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the United States and the European Union. The resulting report provides recommendations for enhancing and promoting rare disease drug development by improving engagement with people affected by a rare disease, advancing regulatory science, and fostering collaboration between FDA and the European Medicines Agency.
This briefing was for members of Congress and congressional staff only. The report was publicly released on September 12, 2024 and can be found, in its entirety, on the Web site of the National Academies Press.