Developers Voice Concern Over Draft Guidance Closing Orphan Drug Loophole

FDA guidance that proposes to close what the agency sees as a “loophole” allowing drug developers to bypass pediatric study requirements would negatively impact child drug development if it becomes final, according to some drug developers and patient advocacy groups.

In a draft guidance issued in December, the FDA said that the agency no longer intends to grant orphan drug designation to drugs for pediatric subpopulations of common diseases due to the way pediatric subpopulation designations and the Pediatric Research Equity Act (PREA) orphan exemption work together. The agency said their interplay creates an “unintended loophole” in which the drug’s sponsor can exempt itself from conducting pediatric studies required by PREA for adult indication approval.

Aevi Genomic Medicine commented that “the proposed remedy is likely to impede and delay development of important new medicines for children” because without orphan drug designation, developing novel drugs and biologics for children is more difficult and “in many cases, practically impossible.”