FDA Plots Elimination of the Orphan Drug Designation Request Backlog

Regulatory NewsRegulatory News | 29 June 2017 | By

With a flood of new orphan drug designation requests, the US Food and Drug Administration (FDA) on Thursday released its new plan to eliminate the backlog of requests in 90 days and to respond to 100% of all new orphan drug designation requests within 90 days.

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

According to FDA’s database of 4,174 orphan designations, 625 of the designations have won approval for that indication, which means about 15% of all orphan designations win approval. But the total number of treatments that have won approval is significantly less than 625 as many treatments have won multiple designations and separate indication approvals for the same treatment.

For instance, Genentech’s cancer treatment Avastin (bevacizumab) has won 11 different orphan designations and has been approved by FDA for eight of those indications. Similarly, Merck’s cancer treatment Keytruda (pembrolizumab) has received 10 orphan designations from FDA and won approval for two of them, so far.

In contrast, of the 4,174 orphan designations granted by FDA since 1983, 572 have been later withdrawn.

Researchers from Johns Hopkins have called for reforms to be made to the designation process, arguing "companies are gaming the system to use the law for mainstream drugs." Sen. Charles Grassley (R-IA) in February also opened an inquiry into possible abuses of FDA's orphan drug designation process. 


The Orphan Drug Act of 1983 looked to help increase the number of treatment available to the nearly 30 million Americans who have a rare disease, which is defined by the Act as a disease that affects fewer than 200,000 people in the US.

A sponsor seeking orphan designation for a drug must submit a request for designation to FDA’s Office of Orphan Products Development (OOPD) with the information required in 21 CFR 316.20 and 316.21.

"Each designation request must stand on its own merit. Sponsors requesting designation of the same drug for the same rare disease or condition as a previously designated product must submit their own data and information in support of their designation request," FDA says.

Orphan designation qualifies the sponsor of the drug for various development incentives, including tax credits for qualified clinical testing. A marketing application for a prescription drug that has received orphan designation also is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.

Elimination Plan

As part of this new FDA plan, first unveiled by commissioner Scott Gottlieb at a Senate hearing, by 21 September 2017, FDA will complete reviews of all orphan drug designations that are older than 120 days. Following that period, the agency says it will respond to all new orphan drug designation requests within 90 days of their receipt.

To achieve these goals, among other steps, the agency will create a team of senior OOPD reviewers to focus on designation requests.

"To reduce administrative burden on the agency’s experts, FDA will create and implement a new streamlined ‘Designation Review Template.’ The goal of this new template is to facilitate consistent and efficient reviews of new designation requests," FDA says.

CDER and CBER will engage in this cross cutting strategy that will also include the participation of the Offices of Pediatric Therapeutics to jointly review Rare Pediatric Disease Designation requests.

FDA will establish an Orphan Products Council to address scientific and regulatory issues related to orphan products to ensure a consistent approach to regulating these products and modify its Orphan Cluster meetings with the European Medicines Agency from monthly to quarterly and shift FDA Rare Disease Council meetings from monthly to quarterly.

In the next two months, FDA also pledges to report on the progress on these activities and OOPD will create a new "Tracking Dashboard" to monitor and facilitate its efforts to meet the new designation goals.



© 2022 Regulatory Affairs Professionals Society.

Discover more of what matters to you

No taxonomy