FDA guidance closes pediatric Orphan loophole

FDA issued draft guidance Tuesday that is intended to close a “loophole” in its implementation of the Orphan Drug Act that drug companies have used to avoid conducting pediatric studies. The guidance restricts the indications eligible for Orphan Drug designation.

Drugs that have Orphan Drug status are exempt from requirements under the Pediatric Research Equity Act (PREA) that require sponsors to conduct studies in pediatric indications. Companies whose candidate has Orphan Drug designation for a pediatric subpopulation could thereby submit the same candidate for FDA approval in a non-Orphan adult population and be exempt from the pediatric study requirement...