FDA creates team to tackle backlog of orphan drug designation requests

The FDA said Thursday that it will deploy a team of "senior, experienced reviewers" as it looks to eliminate the agency's existing orphan designation request backlog of around 200 applications. The regulator noted that the team, which comprises staff with "significant expertise in orphan drug designation," will focus solely on the outstanding requests, starting with the oldest first. 

The moves comes shortly 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. Gottlieb remarked "Congress gave us tools to incentivise 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 FDA also unveiled plans to employ a new streamlined designation review template to boost the consistency and efficiency of its reviews. The programme will additionally seek to cooperate within the agency's medical product centres to increase efficiency, including conducting joint reviews with the Office of Pediatric Therapeutics for reviewing rare paediatric disease designation requests. 

Meanwhile, to ensure that a response is provided for all future requests within 90 days of receipt, the FDA said it will take multiple steps, including reorganising review staff to maximise expertise and increase workload efficiencies, leveraging expertise across the agency's medical product centres and creating a new Orphan Products Council that will focus on scientific and regulatory issues to ensure that the FDA applies a consistent approach to regulating orphan drug products and reviewing designation requests. 

Since being confirmed by the US Senate last month, Gottlieb has called for the agency to take a more active role in controlling drug pricing and increasing competition from the generic sector.  The agency later unveiled initiatives to facilitate such competition for prescription drugs and ease the entry of generic competitors.