In late June 2017, the U.S. Food and Drug Administration (“FDA”) unveiled a strategic plan to both eliminate the agency’s existing orphan designation request backlog and ensure timely responses to all new requests for designation with firm deadlines.
This Orphan Drug Modernization Plan comes hot on the heels of FDA Commissioner Scott Gottlieb’s testimony before a Senate subcommittee, where he made a commitment to (1) eliminate the current backlog within ninety days and (2) respond to all new requests for designation within ninety days of receipt.
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. Various incentives, including tax credits for clinical trial costs, relief from the prescription drug user fee, and eligibility for seven years of marketing exclusivity are some of the incentives for manufacturers to develop orphan drugs.
Currently, the FDA has roughly 200 orphan drug designation requests 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.
“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.”
This is the first of several efforts the FDA plans to undertake under its “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.
To ensure all future requests receive a response within 90 days of receipt, the agency will take a multifaceted approach. These efforts include, among other new steps: 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 successful elimination of the backlog by mid-September and will soon provide more information about the Medical Innovation Development Plan.