FDA to Eliminate Backlog of Orphan Drug Designation Requests

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The agency announced a plan to eliminate its existing orphan designation request backlog.

On June 29, 2017, FDA announced the Orphan Drug Modernization Plan to eliminate the agency’s backlog of existing orphan drug designation requests. FDA plans to complete review of all requests older than 120 days within 90 days and respond to all new requests within 90 days of receipt. As part of the effort, FDA will reorganize review staff and leverage expertise across departments.

A new FDA Orphan Products Council will address scientific and regulatory issues to ensure a consistent approach to the review of orphan drug requests. The agency will create a Backlog SWAT team that will focus solely on backlog applications, starting with the oldest requests. A Designation Review Template will be used to make the reviews more efficient. The program will collaborate with FDA’s medical product centers to conduct joint reviews, particularly with the Office of Pediatric Therapeutics to review rare pediatric disease designation requests. FDA will also revise grant monitoring processes and modernize reporting requirements. A new “Tracking Dashboard” will be created “to monitor and facilitate efforts to meet the new designation goals and FDA will report on overall workload and progress more regularly.”

The Orphan Drug Act created the Orphan Drug Designation Program, which “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.” Sponsors with drugs that are given orphan designation qualify for development incentives.


In 2016, the agency received 568 new orphan designation requests, which FDA says reflects scientific advances in the industry. “These scientific advances and new opportunities also create additional opportunities for FDA, as well as new challenges. The agency needs to make sure it is able to respond to these requests in a timely fashion, and efficiently grant new designations where it is supported by the underlying data,” FDA stated in the new plan.

Source: FDA