A new report from the NASEM Health and Medicine Division offers recommendations for enhancing rare disease drug development. CIRS is delighted to have been able to collaborate with NASEM and conduct the data analyses that helped inform this important report, which is expected to shape future legislative changes and collaborative regulatory efforts for rare diseases.
Rare diseases, such as sickle cell disease and thalassemia, affect up to 30 million people in the United States and at least 300 million across the globe. Congress called on the U.S. Food and Drug Administration (FDA) to sponsor a National Academies study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the U.S. and the European Union. The resulting report, Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities, 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 (EMA).
Access the report and additional resources, including policy briefs and an infographic, on the project webpage.
NASEM is organising a public webinar during which members of the report authoring committee will discuss the report’s key messages and recommendations and answer questions from the audience on 27th September 2024, 1:00pm-2:00pm ET.
Register for the webinar here.
