The U.S. Food and Drug Administration (FDA) has published its recommendation for a new approach to drug development for rare pediatric diseases. The new approach supports the FDA’s commitment to increase the efficiency and speed of developing drugs, as well as supporting the sponsors of the research.

The draft guidance, ‘Pediatric Rare Diseases: Collaborative Approach for Drug Development Using Gaucher Disease as Model’, will help companies navigate a new approach for partnering and evaluating multiple drugs in the same clinical trials. This would reduce the number of patients that need to be treated with placebo in these trials. Through the proposed controlled, multi-arm, multi-company clinical trials, several products can be tested in a more time-efficient manner.

Pediatric Rare Diseases — A Collaborative Approach for Drug Development Using Gaucher Disease as a Model Guidance for Industry