FDA Issues Final Guidance Closing Pediatric Orphan Drug Loophole

The FDA unveiled final guidance closing a loophole under which drug sponsors could avoid their obligation to study drugs in pediatric indications.

The Pediatric Research Equity Act included an exemption from pediatric study requirements for orphan drugs, which in turn allowed sponsors to dodge required studies by obtaining a pediatric subpopulation designation for a non-orphan disease in adult populations.

The agency issued its draft guidance on eliminating the loophole last December. The final guidance is largely unchanged but includes more details of the problems it created.