FDA announces a strategic plan to eliminate orphan designation backlog

The U.S. Food and Drug Administration (FDA) unveiled 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.

As authorized under the Orphan Drug Act, the Orphan Drug Designation Program provides orphan status to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases, which are generally defined as diseases that affect fewer than 200,000 people in the United States. A request for orphan designation is one step that can be taken in the drug development process and is different than the filing of a marketing application with the FDA.

Currently, the FDA has about 200 orphan drug designation requests that are pending review. The number of orphan drug designation requests has steadily increased over the past five years. The increased interest in the program is a positive development for those with rare diseases and under this new plan, the agency remains committed to advancing the program to ensure it can efficiently and adequately review these requests.

"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. "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."

The agency intends to communicate around the successful elimination of the backlog by mid-September and will soon provide more information about the Medical Innovation Development Plan.