FDA Unveils Plan to Eliminate Backlog of Orphan Drug Designation Applications

The U.S. Food and Drug Administration (FDA) has announced a plan to eliminate the agency’s backlog of orphan drug designation requests within 90 days, as well as respond to any new requests for the designation within 90 days of receipt. The FDA has approximately 200 pending orphan drug designation requests, and the number of requests has been increasing each year.

As part of its Medical Innovation Development Plan (MIDP), the agency will create a team of senior reviewers with significant expertise in orphan drug designation to focus solely on the backlogged applications, starting with the oldest requests. The agency will also use a streamlined Designation Review Template to increase consistency and efficiency of drug reviews.

To ensure that all future requests receive a response within 90 days of receipt, the agency said it will reorganize its staffing to maximize expertise, improve workload efficiencies, and better leverage expertise across the FDA’s medical product centers. The FDA also plans to establish a new Orphan Products Council to address scientific and regulatory issues to guarantee that the agency is applying a consistent approach to regulating orphan drug products and reviewing designation requests.

The MIDP aims to ensure that the FDA’s regulatory tools and policies are modern, risk-based, and efficient under the new direction of Commissioner Scott Gottlieb, MD.

Source: U.S. Food and Drug Administration news release, June 29, 2017.