Successful Reauthorization of the Rare Pediatric Disease Priority Review Voucher Program
Author:
Dr Susan McCune
Executive Principal Medical Consultant
Key Details
FDA’s Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program was established in 2012 as a financial incentive to encourage the development of therapeutics for rare pediatric diseases. The program officially ended as of September 30, 2024 due to a sunset clause. Since that time, there have been a number of efforts to reauthorize the program, but it wasn’t until the legislation finally passed on February 3, 2026 to reauthorize the program through to September 30, 2029. The Bill called the Mikaela Naylon Give Kids A Chance Act was named for Mikeala Naylon, a 15 year old who courageously advocated for the bill throughout her battle with osteosarcoma, which she lost earlier this year. The Bill strengthens incentives for pediatric drug development for children battling cancer and rare diseases.
What this means for sponsors / how they can benefit:
The RPD PRV program is aimed to incentivize drug development for rare pediatric diseases. A sponsor who receives approval for a drug or biological product for a rare pediatric disease may qualify for a voucher that can be redeemed so that a different product can receive priority review.
Eligibility or key requirements:
A rare pediatric disease is defined as one where the serious or life-threatening manifestations of the disease primarily affect individuals who are aged birth to 18 years of age, and the disease is a rare disease or condition. The voucher is requested at the time of the original NDA/BLA submission. FDA strongly recommends that sponsors planning to request the voucher request a Rare Pediatric Disease Designation. This can be submitted at the same time as request for orphan-drug designation or fast track designation. It can be a stand-alone submission as long as it is submitted before the filing of the NDA/BLA.
How Scendea can support:
Scendea has extensive experience with the RPD RPV program and can help with preparation and submission of requests for both the Rare Pediatric Disease Designation and the RPD PRV.