FDA New Pilot Program for Orphan Designation Requests

For Rare Disease Day, the FDA lunched a new pilot program to streamline orphan designation requests to make it easier for applicants and reviewers. The program also includes a tutorial and standardizes FDA communication processes. Further, the FDA entered into a new Memorandum of Understanding with the National Organization for Rare Disorders to conduct outreach for strategies that incorporate patient while also providing a more in-depth understanding of urgent patient needs and informing drug development.

This new actions demonstrate FDA commitment to developing rare disease drugs and devices and face the increasing application for orphan drug designation.

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 increased interest in the program is a positive development for those with rare diseases and under this new plan, the agency remains committed to advancing the program to ensure it can efficiently and adequately review these requests.

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.

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