Scott Gottlieb, MD, appointed as FDA commissioner in May 2017, has made some fast progress. According to the FDA Voice, The FDA has reviewed the backlog of orphan designation requests that were more than 120 days old. And they accomplished this goal on August 28, 2017 – nearly a month ahead of their September 21 deadline.
Dr. Gottlieb has promised policy improvements to the Orphan Drug Modernization Act (ODA) – most notably -- to ensure that all applications are reviewed within 90-days. According to the FDA, it will reorganize staff to improve workload efficiencies and to leverage expertise across FDA’s various medical product centers. The FDA will also implement business management principles, including process maps designed to assess redundancies, increase efficacies and track metrics.
Removing the Pediatric Loophole and Other Regs are on the Way
FDA is planning public meetings to review complex scientific and regulatory issues in order for FDA to better determine the appropriate application of orphan incentives. Life science companies can read between the lines here – FDA will raise the bar and scrutinize orphan drug applications because of recent public criticism that some sponsors have used their orphan drug status to sidestep key public health goals.
In particular Dr. Gottlieb made clear that a guidance document will close a loophole that currently allows sponsors to avoid pediatric drug studies. “This circumstance arises if sponsors received an orphan designation for a pediatric subtype of an otherwise common and non-orphaned adult disease.” FDA plans to advance certain guidance documents and other policies to address these issues. Stay tuned for these draft guidance documents and imminent public hearings.
JFK Communications will be watching Dr. Gottlieb closely and will keep you apprised of the direction and any imminent changes to the ODA. We are interested in Dr. Gottlieb’s promise of a public hearing regarding the “pediatric loophole” and any other potential changes to the ODA.
Our firm has worked on more than 40 designated orphan drugs. Our life science public relations firm works with a myriad of patient advocacy, clinical development, biotechnology, and precision medicine partners, including organizations such as #BIONJ, where their credo is “Patients Can’t Wait.”
We believe any efforts to improve the ODA will ultimately serve those patients who have limited or no treatment options. And we share the FDA’s goal to facilitate the development of safe, effective innovations that have the potential to meaningfully impact rare diseases.
We welcome your comments regarding the current status of the Orphan Drug Modernization Act and any imminent changes expected from FDA.