FDA Orphan Drug Modernization Plan Released

  In late June 2017, the U.S. Food and Drug Administration (“FDA”) unveiled a strategic plan to both eliminate the agency’s existing orphan designation request backlog and ensure timely responses to all new requests for designation with firm deadlines.   This Orphan Drug Modernization Plan comes hot on the heels of FDA Commissioner Scott Gottlieb’s testimony before a Senate subcommittee, where he made a commitment to (1) eliminate the current backlog within ninety days and (2) respond to all new requests for designation within ninety days of receipt.   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. Various incentives, including tax credits for clinical trial costs, relief from the prescription drug user fee, and eligibility for seven years of marketing exclusivity are some of the incentives for manufacturers to develop orphan drugs.   Currently, the FDA has roughly 200 orphan drug designation requests 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 doubl...
Source: Policy and Medicine - Category: American Health Authors: Source Type: blogs