Medical and Regulatory Affairs

The FDA Is Ending Its Rare Pediatric Disease Priority Review Vouchers – and Time Is Running Out to Get One

Between 2010 and 2018, 33.8 percent of the 402 orphan indications approved by the FDA were approved for children only or targeted pediatric diseases.[1] The rare pediatric disease priority review voucher program is one incentive that’s helped to spur research and development in this area with more than 20 vouchers granted to date, but it is set to expire on September 30, 2020.

However, there is still time for sponsors to request the rare pediatric disease designation that makes a drug eligible for the program. A drug must have rare pediatric disease designation granted by this date and then gain approval for the priority review voucher itself no later than September 30, 2022. 

The FDA guarantees a 60-day review timeline of rare pediatric disease designation requests when issued alongside a fast track or orphan designation request. As a result, the FDA has announced that it cannot commit to responding by the program deadline for applications received after July 31, 2020. However, sponsors who believe their product is eligible are still encouraged to apply – the sooner, the better. Here’s what you need to know to understand your product’s eligibility and maximize your chance of a successful submission.

Benefits of the priority review voucher program for rare disease sponsors

Under the Orphan Drug Act of 1983, companies targeting treatment options for rare diseases are eligible for several additional years of exclusivity in their treatment options without competition from generic alternatives. The priority review voucher program for rare pediatric disease drugs provides an additional benefit by shortening the FDA drug submission review process from the standard ten months to six; however, the priority review process does not require approval within this timeframe.

Eligible candidates are given two separate vouchers, each of which has priority review: one voucher is used for the drug treating the rare disease while the other one can be used for another indication. While the priority review voucher program for rare pediatric diseases is expiring on September 30, 2020, the vouchers themselves do not expire once granted.

Who’s eligible for priority review?

The FDA has published a list of rare diseases that qualify for priority review on its website. For a company to be awarded a priority review voucher, the application must be for a disease that is listed on their website OR another disease that qualifies under the Orphan Drug Act. In addition, the request for a priority review voucher cannot contain an active ingredient that has been approved in any other application. This includes esters and salts of active ingredients.

Improving your chances of a successful submission

To improve the chances of a successful submission, companies should ensure the disease they intend to research has been listed on the FDA website, as this will make the approval process easier. It is also important to review other treatments that are being actively researched. In order to be approved, research cannot match another ingredient that is already under development.

If you are looking to submit a request for rare pediatric disease designation, Regulatory Professionals, A Division of Premier Research, can help. We provide a thorough analysis of the issues and a comprehensive assessment of the regulatory environment to help you enter Agency discussions with strategies to minimize risk and maximize successful negotiations. Contact us today to learn more.


[1] Kimmel L, Conti RM, Volerman A, Chua KP. Pediatric Orphan Drug Indications: 2010-2018. Pediatrics. 2020;145(4):e20193128. doi:10.1542/peds.2019-3128