US FDA flexible in reviewing therapies for rare diseases, NORD study concludes

The US National Organization for Rare Disorders (NORD) yesterday released a landmark report documenting flexibility in the Food and Drug Administration review of potential treatments for patients with rare diseases.

Released at the US Conference on Rare Diseases and Orphan Products, the report examined the basis for the FDA's approval of 135 non-cancer orphan drugs - those for rare diseases - since the Orphan Drug Act was enacted in 1983 to provide incentives to encourage development of treatments for these diseases.

This is the first study of its kind ever conducted and the first time that there has been a systematic examination of the basis for approval for any category of drug products extending over such a long period of time. The study demonstrates a decades-long pattern of flexibility in FDA review of orphan drugs.