FDA has released a draft guidance document entitled, Rare Diseases: Common Issues in Drug Development
Guidance for Industry, which is intended to help sponsors
conduct more efficient and successful clinical development programs
for rare diseases—disorders or conditions as defined by the
Orphan Drug Act that affect less than 200,000 persons in the United
States. FDA provides insight on the following components of rare
disease drug development: description and understanding of the
disease's natural history; understanding of the pathophysiology
of the disease and the drug's proposed mechanism of action;
nonclinical pharmacotoxicity considerations to support the proposed
clinical investigations; standards of evidence to establish safety
and effectiveness; reliable endpoints and outcome assessment; and
drug manufacturing considerations during drug development. FDA
announced the draft guidance document in the August 17, 2015, Federal Register. FDA is seeking comments on
the draft guidance by October 16, 2015.
The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.
