Regulatory Processes for Rare Disease Drugs in the United States and European Union
Contributors
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; Editors: Carolyn K. Shore, Tequam L. Worku, Carson W. Smith, and Jeffrey P. Kahn.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 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.
Suggested citation:
National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory processes for rare disease drugs in the United States and European Union: Flexibilities and collaborative opportunities. Washington, DC: The National Academies Press. https://doi.org/10.17226/27968.
Digital Object Identifier: https://doi.org/10.17226/27968
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Created: September 12, 2024; Last Update: October 30, 2024.