FDA’s new orphan drug modernization plan

As the the number of orphan drug designation requests has steadily increased over the past five years, the FDA has a new plan to complete reviews of all orphan drug designation requests 90 days, that are older than 120 days (the backlog) while maintaining consistent, scientifically rigorous reviews; and after 90 days, 100% of all new orphan drug designation requests will receive a response by the agency within 90 days of receipt. 

FDA said that 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. 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. 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.

These scientific advances and new opportunities also create additional opportunities for FDA, as well as new challenges, the agency says. Furthermore, it needs to make sure it is able to respond to these requests in a timely fashion, and efficiently grant new designations where it is supported by the underlying data. To ensure timely review of orphan drug designation requests, FDA says it is undertaking a broad modernization of its approach to its orphan drug designation program. This is being done to both eliminate a backlog of existing designation requests, and to make sure that the agency can respond in a timely fashion to new applications, even as we anticipate that the demands on the orphan drug program will continue to grow as a consequence of commercial opportunities as well as the promises of scientific progress.

To achieve these goals, among other steps, the agency said it will create a SWAT Team of senior, experienced, and proficient OOPD reviewers to focus on designation requests in the order that the agency receives these 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. 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, in order to review orphan drug designation requests in a timely manner. These are just a few of our programmatic improvements that are further described below in our Orphan Drug Designation Plan.


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