FDA announces plan to eliminate orphan drug designation backlog

The FDA has released the details of its Orphan Drug Modernization Plan, developed to eliminate the existing orphan drug request backlog and create a system for timely response deadlines for all future requests. This plan will be one of several efforts as part of the agency’s encompassing Medical Innovation Development Plan.

“People who suffer with rare diseases are too often faced with no, or limited, treatment options, and what treatment options they have may be quite expensive due in part to significant costs of developing therapies for smaller populations,” Scott Gottlieb, MD, FDA commissioner, said in a press release from the agency. “Congress gave us tools to incentivize the development of novel therapies for rare diseases and we intend to use these resources to their fullest extent in order to ensure Americans get the safe and effective medicines they need, and that the process for developing these innovations is as modern and efficient as possible.”

There are currently 200 orphan drug designation requests pending review. According to the FDA, designation requests have been steadily increasing over the last 5 years, with 568 new requests in 2016 — more than double the requests received in 2012.

“The uptick in designation requests reflects, among other factors, advances in science that allow researchers to target rare diseases that were previously not readily amenable to therapy,” the agency wrote in the modernization plan. “This is good news. It is a reflection of substantial medical progress that’s allowing us to effectively target many vexing diseases. It is also a reflection of our better understanding of the genetic basis of diseases, which unlocks our ability to define and target rare disorders.”

During a testimony before a Senate subcommittee, Gottlieb committed to eliminate the backlog within 90 days and respond to future designation requests within 90 days of receipt.

To eliminate the backlog, the FDA will employ a team of senior, experienced reviewers and begin with the oldest requests. Additionally, the agency will make use of a new, streamlined Designation Review Template to increase consistency and efficacy and will collaborate with medical product centers such as the Office of Pediatric Therapeutics to review rare pediatric disease designation requests.

To ensure the review of future requests within 90 days, the FDA plans to reorganizing their review staff to maximize expertise and improve workload designation, leverage work across the medical product centers and establish a new FDA Orphan Products Council to ensure the agency is applying a consistent approach in the review process.

This plan follows the recent release of the FDA’s Drug Competition Action Plan, designed to prioritize the review process for generic drugs and increase competition in the prescription drug market.

The FDA will provide an update on its progress to eliminate the backlog in mid-September of this year. – by Talitha Bennett

Reference:

FDA. Orphan drug modernization plan. https://www.fda.gov/downloads/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/UCM565068.pdf. Published June 29, 2017. Accessed June 29, 2017.