FDA Issues Draft Guidance on Rare Diseases

On Jan. 17, 2019, FDA announced that it issued a revised draft guidance to enable more efficient and successful drug development programs for the treatment or prevention of rare diseases.

The draft guidance, Rare Diseases: Common Issues in Drug Development Guidance for Industry, was originally issued in 2015. The revised guidance covers a range of topics such as natural history studies, use of biomarkers, and methods for demonstrating effectiveness and safety. 

Comments and suggestions should be submitted within 60 days of publication.