The Pulse on Technology

FDA proposes collaborative approach for pediatric rare disease research

December 18, 2017

For more efficient development of treatments for pediatric rare diseases, sponsors can implement controlled, multiarm, multi-company clinical trials, according to a new draft guidance that the FDA says could help eliminate the need for certain studies.

The proposed, double-blind design would minimize the number of patients receiving a placebo, using a single control group for multiple investigational drugs. In addition, a single treatment arm could be stopped early following clinical decline.