FDA’s Plan to Cope with Orphan Drugs

The FDA www.fda.gov has unveiled a strategic plan to eliminate the agency’s existing orphan drug designation request backlog to ensure continued timely responses to all new requests for organ drug designations with firm deadlines.

The Orphan Drug Act provides orphan status to drugs and biologics that are defined as drugs that 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 U.S.

Orphan designation qualifies the sponsor of the drug for tax credits to use towards clinical trial costs, relief from prescription drug user fee if the indication is for a rare disease or conditions, and the sponsor of the drug is eligible for seven years of marketing exclusivity upon approval. A request for orphan designation is just one step that can be taken in the drug development process and is different than the filing of a marketing application with FDA.

Currently, 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. The number of orphan new requests for designation has now reached 568 which is more than double the number of requests received in 2012.

FDA Commissioner Scott Gottlieb reports that he is committed to eliminating the backlog within 90 days and to responding to all new requests for designation within 90 days of receipt. The plan to eliminate the backlog is going to require FDA to deploy a “Backlog SWAT team comprised of senior and experienced reviewers with significant expertise in orphan drug designation.

FDA is also going to employ a new streamlined Designation Review Template to increase consistency and efficiency in reviews. The program will also look to collaborate within the FDA’s medical product centers to create greater efficiency.

Also, a new FDA Orphan Products Council will help address scientific and regulatory issues to ensure that the agency is applying a consistent approach to regulating orphan drug products and reviewing designation requests.

Share Button