pediatric clinical trials stockFDA commissioner Scott Gottlieb plans to close a loophole currently allowing companies to avoid their obligation to study pharmaceuticals in pediatric populations. Gottlieb plans to close the loophole currently impacting pediatric clinical trials through a new guidance, he announced on Tuesday, September 12.

When and how companies take advantage of the loophole

Gottlieb explains that the opportunity occurs when sponsors receive an orphan designation for a pediatric subtype of an otherwise common and non-orphaned adult disease.1 RAPS provides an example to highlight the situation: with a condition like inflammatory bowel diseases, a drug may be approved to treat the population of adults with the condition but then the same drug may be granted an orphan designation to treat a subset of children suffering from IBD. Gottlieb describes that “once a drug receives an orphan designation for a pediatric population of the adult disease, the drug then becomes statutorily exempts from the requirements [of the Pediatric Research Equity Act (PREA)].”

In a post on FDA’s blog, Gottlieb wrote that the loophole is in “direct opposition to what Congress intended… nobody envisioned this unintended conflict between the original ODA [Orphan Drug Act] and the provisions outlined in PREA. In effect, by letting sponsors designate pediatric subpopulations of drugs intended to treat adult diseases, the drug makers receive an unintended ‘free pass’ from having to study drugs in these or other pediatric uses. Thus, rather than ensuring more pediatric research, as Congress envisioned, we can end up with fewer pediatric studies. FDA will soon issue a draft guidance document that’s aimed at closing this inadvertent loophole.”2

Closing the loophole and implementing new policies to improve the efficiency of FDA’s orphan drug designation review process are priorities for the agency in the coming months. Stay tuned to the Pearl Pathways blog and follow us on Twitter for updates on the potential impacts to pediatric clinical trials.