FDA seeks to clarify Orphan drug regulations

FDA is seeking to clarify language in Orphan drug regulations and propose areas of minor improvement, according to a proposed rule issued Wednesday. The changes are intended to eliminate sponsors' confusion, which is evident in that 124 (38%) of 324 requests for Orphan drug designation in 2010 were denied or stayed so that the sponsor could submit additional material to respond to deficiencies.

The rule clarifies that a compound under development for a subset of a non-rare disease will not be considered for Orphan designation unless the company demonstrates that the compound would not be appropriate for use in the broader population of patients with the non-rare disease. The agency said 24% (79) of requests for Orphan drug designation in 2010 were denied or stayed because they did not identify a medically plausible subset of a non-rare disease. ...