As many as half of all people living with a rare disease are children, and rare pediatric disease priority review vouchers (PRVs) offer a crucial incentive for companies to develop therapies for these particularly challenging patient populations. Reauthorizing this vital program before the September 30, 2024 deadline would maintain an important tool in ongoing efforts to address the significant unmet treatment needs of the pediatric rare disease population.
If you would like to get involved or share your story of how the pediatric PRV program helped you or someone you love, please email policy@rarediseases.org.
Background on PRVs
The Rare Pediatric Disease PRV has helped spur rare disease drug development in pediatric populations and brought almost four dozen therapies to market with critical safety and dosing data specific to children.
The PRV program began in 2006 to address the unmet need of developing drugs for tropical diseases and was ultimately expanded in 2012 to include rare pediatric diseases. Under this program, companies that develop novel therapies for rare pediatric diseases can be awarded a PRV. The PRV allows a sponsor to obtain priority review for a new drug application (NDA) or biologic license application (BLA) that would otherwise not qualify for priority review; it can also be sold to another manufacturer to obtain a priority review for their product.
This program’s authorization ends on September 30, 2024, and without a timely reauthorization, FDA will no longer be allowed to initiate the process necessary to issue new rare pediatric disease PRVs.
Timely reauthorization is needed to support rare disease drug development
To date, 46 PRVs have been awarded for 35 different rare pediatric diseases. More than half of these PRVs were awarded after 2019, the year prior to when the most recent Government Accountability Office (GAO) analysis ended.[1]
It is important to note that at the time of the prior analysis, the program had only been in place for 7 years; on average, it takes 10+ years to bring a new rare disease therapy to market.[2] The significant uptake in pediatric rare disease approvals in recent years demonstrates the value of the program to the rare disease patient community, where more than 95% of rare diseases lack an FDA approved treatment.
The National Organization for Rare Disorders (NORD) urges Congress to reauthorize the Rare Pediatric Disease PRV program before the September 30, 2024, deadline.
NORD is thankful to Reps. McCaul, Eshoo, Bilirakis, Barragan, Burgess, and Trahan for introducing the Creating Hope Reauthorization Act of 2024 (H.R.7384), and for their long leadership in this space.
Footnotes
[1] U.S. Government Accountability Office. (2020, January 31). Drug development: FDA’s Priority Review Voucher Programs. Drug Development: FDA’s Priority Review Voucher Programs | U.S. GAO. https://www.gao.gov/products/gao-20-251
[2] Orphanet: About orphan drugs. (n.d.). https://www.orpha.net/consor/cgi-bin/Education_AboutOrphanDrugs.php?lng=EN
