Rare Disease Development Support

[membership level=”0,4″][The content continues for paid members only.] Login or Subscribe [/membership][membership level=”1,5,6,7,8,9,10,11,12″][membership] In mid-August, FDA issued a draft guidance on “Rare Diseases: Common Issues in Drug Development.” The draft aims to assist sponsors of drug and biological products for treating rare diseases in conducting more efficient and successful development pro-grams. In addition to an introduction, background and reference sections, the 19-page guidance includes sections on: ● natural history studies ● disease pathophysiology and identification and use of biomarkers ● nonclinical studies ● efficacy endpoints ● evidence of effectiveness and safety, and ● chemistry, manufacturing, and controls (CMC). Comments are due by October 16. FDA also announced the availability of grant funds for the support of development of a Natural History Database. The National Organization for Rare Disorders (NORD) is developing an Internet-based data collection tool with promise to further the accu-mulation of natural history data for many rare diseases. The goal of the grant is to enable NORD to further develop, refine, and disseminate the database tool.
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