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In response to a Congressional request, an ad hoc committee of the National Academies of Sciences, Engineering, and Medicine will conduct a study on processes for evaluating the safety and efficacy of drugs for rare diseases or conditions in the United States and the European Union.
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Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities
Consensus
·2024
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 e...
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