An Evolving Regulatory Environment for Rare and Orphan Diseases
Advarra
AUGUST 16, 2022
In 2016, the FDA granted 333 (57%) of the 582 orphan drug designation requests it received – nearly 10 times the number of designations the agency awarded in 1986. The agency’s 2019 guidance, Rare Diseases: Common Issues in Drug Development , called attention to the need for natural history studies for rare diseases.
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