FDA Closes Loophole Companies Used to Skirt Pediatric Study Requirements

Regulatory NewsRegulatory News | 19 December 2017 |  By 

The US Food and Drug Administration (FDA) on Tuesday released draft guidance indicating that it no longer intends to grant orphan drug designation to drugs for pediatric subpopulations of common diseases except for under certain conditions.

Those conditions are if the use of the drug in the pediatric subpopulation are for a valid orphan subset, meaning "that use of the drug in a subset of persons with a non-rare disease or condition may be appropriate but use of the drug outside of that subset (in the remaining persons with the non-rare disease or condition) would be inappropriate owing to some property(ies) of the drug," or if a sponsor "can adequately demonstrate that the disease in the pediatric subpopulation is a different disease from the disease in the adult population."


The draft guidance is meant to close a loophole, first discussed by FDA Commissioner Scott Gottlieb in September, that has allowed companies to avoid their obligation to study pharmaceuticals in pediatric populations.

According to the draft, section 505B(k) of the Food Drug & Cosmetics Act (FD&C Act) contains a statutory exemption from the requirement to conduct pediatric studies under the Pediatric Research Equity Act (PREA) for certain drugs with orphan designation.

"The interplay of [the] pediatric-subpopulation designation and the PREA orphan exemption has created an unintended loophole where a sponsor holding pediatric-subpopulation designation can submit a marketing application for use of its drug in the non-orphan adult population of that disease, get a pediatric-subpopulation designation for the pediatric subset of the disease, and, due to this designation, be exempt from conducting the pediatric studies normally required under PREA when seeking approval of the adult indication," the draft says.

It offers the example of FDA granting a pediatric-subpopulation designation for a sponsor’s drug for pediatric ulcerative colitis. If the sponsor submits a new drug application (NDA) or biologics license application (BLA) for its drug to treat ulcerative colitis in adults, the sponsor would then be exempt from having to conduct pediatric studies.

Gottlieb said in a statement: "Unfortunately, some of the efforts intended to encourage pediatric drug development have been used by some drug developers to avoid their obligations to study drugs in pediatric populations. One such area is the longstanding practice of designating pediatric subpopulations of common diseases as orphan conditions. By making this designation, drug developers took advantage of an unintended loophole in a law that was, to the contrary, specifically designed to ensure that drugs are studied for pediatric indications. Today, we’re closing that loophole and announcing that we no longer intend to grant pediatric-subpopulation designations through the orphan drug program."

FDA said it expects to implement this policy upon publication of the final version of this guidance dependent upon comments received. "In the interim, FDA will refrain from issuing final decisions on requests for pediatric-subpopulation designation until the guidance is finalized," a Federal Register notice said. 

The agency said Tuesday that it welcomes public comment on the draft, which is part of FDA’s Orphan Drug Modernization Plan announced in June.

Clarification of Orphan Designation of Drugs and Biologics for Pediatric Subpopulations of Common Diseases: FDA Draft Guidance for Industry


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