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BIO Comments on FDA Draft Guidance on Rare Diseases: Natural History Studies for Drug Development

May 24, 2019

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Rare Diseases: Natural History Studies for Drug Development.

BIO says the guidance is as an important communication tool for the FDA and Sponsors. It ensures Sponsors have the appropriate information for developing new therapies for patients with rare diseases ­– especially since many rare diseases do not have FDA-approved treatment.

BIO provides recommendations on how to improve natural history studies so the FDA can have robust data for making regulatory decisions, as well as how to improve the guidance. BIO recommends the draft guidance align with other draft guidance covering natural history studies, like Rare Diseases: Common Issues in Drug Development


Download Full Comments Below
BIO Comment Letter Natural History Studies Rare Disease Drug Development FDA–2017-D-0481
Read the full comment letter below
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