The US Food and Drug Administration (FDA) has unveiled a strategic plan to eliminate the agency’s existing orphan designation request backlog for drugs 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 to respond 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 US. Orphan designation qualifies the sponsor of the drug for various development incentives, including tax credits for clinical trial costs, relief from prescription-drug user fees if the indication is for a rare disease or condition, and eligibility for seven years of marketing exclusivity upon approval. 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. In 2016, the FDA’s Office of Orphan Products Development received 568 new requests for designation, more than double the number of requests received in 2012.
The program is the first element of several efforts the FDA said it will undertake under its new Medical Innovation Development Plan, which the FDA said is aimed at ensuring that the FDA’s regulatory tools and policies are "modern, risk-based, and efficient."
Among the elements of the plan to eliminate the backlog, the FDA said it will deploy a Backlog SWAT team comprised of senior, experienced reviewers with expertise in orphan-drug designation. The team will focus solely on the backlogged applications, starting with the oldest requests. The agency said it will also employ a new streamlined Designation Review Template to increase consistency and efficiency of its reviews. The FDA said that the program will also look 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.
To ensure all future requests receive a response within 90 days of receipt, the FDA said it will take a multifaceted approach. These efforts include: reorganizing the review staff to maximize expertise and improve workload efficiencies; better leveraging the expertise across the FDA’s medical product centers; and establishing a new FDA Orphan Products Council that 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 agency intends to communicate around the elimination of the backlog by mid-September and said it will soon provide more information about the Medical Innovation Development Plan.