FDA Proposes Collaborative Approach for Pediatric Rare Disease Research

December 14, 2017

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rare diseases, sponsors can implement controlled, multi-arm, multi-company clinical trials, according to a new draft guidance that the FDA says could help eliminate the need for certain studies.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

While the guidance itself focuses on treatments for Gaucher disease, a rare, inherited metabolic disorder, the FDA hopes the plan can be extended as a model for other areas of pediatric drug development, according to CDER Director Janet Woodcock.

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