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Home » FDA Proposes Collaborative Approach for Pediatric Rare Disease Research
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rare diseases, sponsors can implement controlled, multi-arm, multi-company clinical trials, according to a new draft guidance that the FDA says could help eliminate the need for certain studies.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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