FDA Delivering 90-Day Guarantee on Orphan Drug Designation Requests

The number of orphan drug designation requests has steadily increased over the past five years. In fact, it more than doubled from 267 in 2012 to 568 in 2016.

While that’s good news for scientific advances and drug discovery, it presents challenges in the approval of safe and effective treatments for rare diseases—among them granting approval in a timely fashion. There are currently about 200 orphan drug designation requests pending review.

To address these issue, the U.S. Food and Drug Administration (FDA) announced a strategic plan to eliminate the agency’s existing orphan designation request backlog and ensure continued timely response to all new requests for designation with firm deadlines.

The agency’s Orphan Drug Modernization Plan comes a week after FDA Commissioner Scott Gottlieb committed to eliminating the backlog within 90 days and responding to all new requests for designation within 90 days of receipt during his testimony before a Senate subcommittee.

“People who suffer with rare diseases are too often faced with no, or limited, treatment options, and what treatment options they have may be quite expensive due in part to significant costs of developing therapies for smaller populations,” said FDA Commissioner Scott Gottlieb, M.D. in an agency statement. “Congress gave us tools to incentivize the development of novel therapies for rare diseases and we intend to use these resources to their fullest extent in order to ensure Americans get the safe and effective medicines they need, and that the process for developing these innovations is as modern and efficient as possible.”

Those tools include a new “Designation Review Template” to facilitate consistent and efficient reviews of new review requests.

The agency will also create a SWAT Team of senior, experienced reviewers to focus on designation requests that are older than 120 days. The team will review these requests in the order that the agency received them—working from the oldest submission up to the most recent. The deadline to clear the backlog is September 21, 2017.

Other efforts to reach this deadline include plans to collaborate within the agency’s medical product centers to create greater efficiency, including conducting joint reviews with the Office of Pediatric Therapeutics to review rare pediatric disease designation requests.

For future requests, a reorganization of review staff will maximize expertise and improve workload efficiencies and a new FDA Orphan Products Council will help address scientific and regulatory issues to ensure the agency is applying a consistent approach to regulating orphan drug products and reviewing designation requests.

The FDA plans to report on their progress in mid-September.