Unjamming the Orphan Drug Backlog

Unjamming the Orphan Drug Backlog

July 07, 2017PAO-M07-17-NI-009

The FDA commissioner unveiled the agency's upcoming strategy.

In an effort to ensure timely responses to all new requests for orphan drug designation, FDA commissioner Scott Gottlieb announced on June 29th during testimony before a Senate subcommittee, the agency’s Orphan Drug Modernization Plan, which commits the FDA to eliminating any potential backlog within 90 days — and to respond to all new designation requests within 90 days.

According to FDA statistics, the agency currently has approximately 200 orphan drug designations pending review, due in part to the steady increase of requests over the last five years. The FDA said in 2016 that 568 requests were made — more than double the amount the agency encountered in 2012.

Characterizing the increased interest in the program as positive, the FDA said it remained committed to advancing the program. In his Senate testimony Commissioner Gottlieb explained “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.”

Part of the FDA’s larger Medical Innovation Development Plan, the Orphan Drug backlog program is among the first of a series of initiatives aimed at making sure the agency’s drug approval tools are sharp and effective. As part of the plan, the FDA said it will deploy a “Backlog SWAT team” made up of “senior, experienced reviewers” with “significant expertise” in orphan drug designation. Focused exclusively on backlogged applications, the team, said the FDA, will start processing the oldest applications first, as well as employ a new streamlined Designation Review Template to help boost the efficiency and consistency of reviews.

Collaboration with the agency’s medical products centers was also identified in the plan–in order, said the agency, to be more effective when conducting joint reviews with (for example) the Office of Pediatric Therapeutics to review “rare pediatric disease designation requests.”

Regarding the commitment to respond to all future requests within 90 days, the FDA said it is going to take a multi-faceted approach which includes establishing a new FDA Orphan Products Council to help address scientific and regulatory issues more carefully, and a review of staff and operations to identify expertise and workload efficiencies, among other things. According to the FDA, the agency plans to communicate its success at eliminating the backlog by mid-September.


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