FDA issues guidance for rare pediatric disease drug development

FDA issued draft guidance on Wednesday aimed at accelerating clinical development of treatments for pediatric rare diseases. The guidance builds upon a joint proposal between FDA and EMA that encourages companies to join multidrug platform trials (see BioCentury, July 28).

FDA proposed the use of a multi-arm, multi-company clinical trial, which could reduce the total number of children needed for enrollment compared with separate clinical trials...