By Clinical Informatics News Staff 

June 29, 2017 Today, the U.S. Food and Drug Administration 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.

Currently, the FDA has about 200 orphan drug designation requests that are pending review. Last year, the FDA’s Office of Orphan Products Development (OOPD) received 568 new requests for designation—more than double the number of requests received in 2012. FDA Commissioner Scott Gottlieb has committed to eliminating the backlog (all requests older than 120 days) in 90 days. In the future, all new requests for designation will be addressed within 90 days of receipt.

The agency’s Orphan Drug Modernization Plan lays out steps to do that.          

First, a “Backlog SWAT team” comprised of senior, experienced OOPD reviewers will focus solely on the backlogged applications, starting with the oldest requests. FDA plans to minimize discretionary work during this time so that review teams can focus on core activities, and create and employ a new streamlined Designation Review Template to increase consistency and efficiency of its reviews. Collaboration is essential for speedy review, so OOPD plans to conduct joint reviews with the Office of Pediatric Therapeutics to review rare pediatric disease designation requests, and CDER and CBER for some other drug designation requests.

By September 21, 2017, FDA plans to have reviewed and responded to all designation requests older than 120 days.

To ensure all future requests receive a response within 90 days of receipt, the agency outlined a multifaceted approach focused on streamlining review efficiency. The new plan calls for reorganizing review staff to maximize expertise and improve workload efficiencies; decreasing meeting frequency; improving all technology infrastructure; creating a new tracking dashboard to monitor progress; 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. For sponsors, OOTD plans to roll out web-based training to improve the quality of submissions.

FDA plans to report on the progress of these activities within the next two months.

“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. in a press release. “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.”

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. Orphan designation qualifies the sponsor of the drug for various development incentives, including tax credits for clinical trial costs, relief from prescription drug user fee 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.

The Orphan Drug Modernization Plan is the first element of several efforts the FDA will undertake under its new “Medical Innovation Development Plan,” which is aimed at ensuring that the FDA’s regulatory tools and policies are modern, risk based, and efficient. The goal of the plan is to seek ways the FDA can help facilitate the development of safe, effective and transformative medical innovations that have the potential to significantly impact disease and reduce overall health care costs.