FDA to Close PREA/Orphan Loophole

In July of this year, we reported that FDA Announces Strategic Plan to Eliminate Orphan Designation Request Backlog. On September 12, 2017, Commissioner Gottlieb stated that “Reviews of all orphan drug designation requests older than 120 days were completed on August 28th.” In the same report, entitled “FDA is Advancing the Goals of the Orphan Drug Act,” the Commissioner also noted the following: “For all the success of the ODA, there’s been criticism that some sponsors are using designations as a way to sidestep other important public health goals set out by Congress. We need to make sure our policies take notice of all of these new challenges and opportunities. FDA plans to advance certain guidance documents and other policies to address these issues. One guidance document will close a loophole that allows sponsors to avoid an obligation to study drugs in pediatric indications. This circumstance arises if sponsors received an orphan designation for a pediatric subtype of an otherwise common and non-orphaned adult disease.” Click here for the full story and here for a report from RAPS Regulatory Focus entitled “FDA to Close Loophole Allowing Companies to Skirt Pediatric Study Requirements”. Feel free to contact us for help with your iPSP or Orphan drug development program.