New guidance intends to limit product sponsors’ exclusions from the requirement to study pharmaceuticals in pediatric patients.

On December 20, 2017, the US Food and Drug Administration (FDA or Agency) issued draft guidance stating that the Agency no longer intends to grant orphan drug designation to drugs for treating pediatric subpopulations of common diseases, which include diseases or conditions affecting more than 200,000 persons in the United States. The draft guidance, Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases, sets forth the Agency’s position with respect to the use of orphan drug designation in pediatric subpopulations. In particular, the draft guidance is intended to limit access to a provision in the Pediatric Research Equity Act (PREA) that may exempt a sponsor holding a pediatric-subpopulation orphan designation from conducting the pediatric studies normally required under PREA when seeking approval of the adult indication of the same common disease.