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Regulatory Processes for Rare Disease Drugs in the United States and European Union
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Regulatory Processes for Rare Disease Drugs in the United States and European Union

Book Details

Format Paperback / Softback
ISBN-10 0309726557
ISBN-13 9780309726559
Publisher National Academies Press
Imprint National Academies Press
Country of Manufacture GB
Country of Publication GB
Publication Date Nov 30th, 2024
Print length 386 Pages
Product Classification: Medicine
Ksh 7,550.00
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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.


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