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News|Articles|January 17, 2019
FDA Issues Draft Guidance on Rare Diseases
Author(s)Pharmaceutical Technology Editors
The agency revised draft guidance to enable more efficient and successful drug development programs for the treatment or prevention of rare diseases.
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On Jan. 17, 2019, FDA announced that it issued a revised draft
The draft guidance, Rare Diseases: Common Issues in Drug Development Guidance for Industry, was originally issued in 2015.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.
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