The situation, according to Gottlieb, arises if sponsors received an orphan designation for a pediatric subtype of an otherwise common and non-orphaned adult disease.
For example, with a condition like inflammatory bowel disease (IBD), a drug may be approved to treat the large 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.
“But once a drug receives an orphan designation for a pediatric population of the adult disease, the drug then becomes statutorily exempt from the requirements” of the Pediatric Research Equity Act (PREA), Gottlieb explains.
“It’s a loophole that is in direct opposition to what Congress intended,” he wrote. “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 FDA will soon issue a draft guidance document that’s aimed at closing this inadvertent loophole.”
Nancy Goodman of Kids v Cancer, whose RACE for Children’s Act was included in the FDA user fee reauthorization law (FDARA), explained to Focus that the language on closing the loophole was initially going to be included in FDARA.
A public meeting announced by Gottlieb will also address some of the statute from FDARA and get input on complex scientific and regulatory issues, such as those raised by molecularly targeted drugs and biologics and the appropriate application of orphan incentives.
In addition to closing that loophole and the meeting, Gottlieb said FDA has now reviewed all orphan drug designation requests older than 120 days and will put in place new policies to improve the efficiency of FDA’s review process to ensure that we meet our new 90-day mandate to prevent new backlogs.