FDA Issues Draft Guidance on Rare Diseases

January 17, 2019

The agency revised draft guidance to enable more efficient and successful drug development programs for the treatment or prevention of rare diseases.

On Jan. 17, 2019, FDA announced that it issued a revised draft guidance to enable more efficient and successful drug development programs for the treatment or prevention of rare diseases.

The draft guidance, Rare Diseases: Common Issues in Drug Development Guidance for Industry, was originally issued in 2015.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. 

Source: FDA