Regulatory Processes for Rare Disease Drugs in the United States and European Union
Flexibilities and Collaborative Opportunities
Description
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.
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‘Regulatory Processes for Rare Disease Drugs in the United States and European Union - National Academies of Sciences, Engineering, 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’.
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