Plan to tackle US orphan designation backlog unveiled by FDA

Against a backdrop of ever-increasing requests for Orphan Drug designation in the USA, the Food and Drug Administration today unveiled a strategic plan to eliminate the agency’s existing backlog of requests and ensure continued timely response to all new requests for designation with firm deadlines.

In 2016, the FDA’s Office of Orphan Products Development (OOPD) received 568 new requests for designation – more than double the number of requests received in 2012

The agency’s Orphan Drug Modernization Plan comes a week after FDA Commissioner Scott Gottlieb committed to eliminating the backlog within 90 days and responding to all new requests for designation within 90 days of receipt during his testimony before a Senate subcommittee.