FDA outlines efforts to reduce orphan drug designation backlog

SILVER SPRING, Md. — The Food and Drug Administration is committing to eliminate its orphan drug designation request backlog within 90 days and respond to all orphan drug requests within 90 days of receipt. The agency on Thursday outlined its Orphan Drug Modernization plan, which it said is part of a larger effort called the Medical Innovation Development plan modernize and make efficient the FDA’s regulatory tools and policies.

“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,” FDA commissioner Dr. Scott Gottlieb said. “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.”

Under this effort, the FDA is deploying a “backlog SWAT team” of experienced reviewers with experience in the designation, and will employ a streamlined designation review template aimed at bringing consistency and efficiency to the process to clear the backlog.

As for ensuring a 90-day response on future requests, the agency said that it would reorganize its review staff to maximize expertise and improve workload efficiencies, establish a new FDA Orphan Products Council to help address regulatory and scientific issues and ensure consistency in regulating orphan drug requests, among other efforts.

The FDA currently has roughly 200 requests for orphan drug designation pending review, and it has seen a steady increase in these requests in the past five years. The number of requests for the designation in 2016 — 568 — was more than double the number it received in 2012.

The FDA said that it would continue to provide information about the Medical Innovation Development Plan in the future, and would provide an update on the backlog in mid-September.