Innovating new treatments for rare diseases is never simple. And when the patients are children, the challenges can be even more complex. The Rare Pediatric Disease Priority Review Voucher (RPD-PRV) program is one of the key tools designed to help address these challenges. It encourages researchers and companies to focus on conditions that urgently need better options, including polycystic kidney disease (PKD).
Unfortunately, the program expired at the end of 2024. This removed a critical incentive for developing treatments for children with rare diseases. Thankfully, the House of Representatives recently passed the Give Kids a Chance Act (H.R. 1262), which would reauthorize the RPD-PRV program for at least five more years.
What is the Rare Pediatric Disease Priority Review Voucher (RPD-PRV)?
Administered by the U.S. Food and Drug Administration (FDA), the RPD-PRV program awards a “voucher” to drug developers when they receive FDA approval for a treatment targeting a rare pediatric disease. Developers then have two options for using the voucher. They can receive a priority (faster) review for another therapy, often for one serving a broader patient population. Alternatively, they can sell it to another company.
This creates a powerful incentive to invest in treatments for rare childhood illnesses, including autosomal recessive PKD (ARPKD). Currently, many of these diseases have no available therapies.
The National Organization for Rare Disorders (NORD), of which the PKD Foundation is a proud member, recently released a new report highlighting the program’s success. Over just 13 years, the RPD-PRV program helped spur the development of more than 60 rare pediatric disease treatments, a remarkable impact for a program aimed at some of the rarest conditions.
5 Reasons the RPD-PRV Matters for PKD
1. Current Treatment Options Focus on Adults
Today, the only FDA-approved treatment for slowing kidney function decline in ADPKD is tolvaptan, which is approved only for adults 18 and older.
2. There are No Approved Therapies for Children with PKD
As of now, children and teens with PKD have no disease-modifying treatments available. This includes both early-onset ADPKD and the rarer ARPKD.
3. ARPKD Presents Unique Challenges
ARPKD, which almost always affects children, can lead to serious kidney and liver complications early in life. Families must navigate complex care with limited treatment options beyond supportive management.
4. Improves a Historical Lack of Investment in Rare Diseases
Historically, research and development for rare childhood kidney diseases haven’t attracted major investment. The voucher program helps solve this by creating a meaningful financial incentive.
5. The Potential Payoff is Real
This program could help accelerate pediatric clinical trials and expand treatment research for younger patients. Importantly, it could bring forward new options for families facing ARPKD or early-onset ADPKD.
How Can We Help the RPD-PRV Right Now?
Progress in rare disease research rarely happens overnight. Through programs like the RPD-PRV, we can help speed up the path toward therapies that children with PKD urgently need. The Senate now has the opportunity to pass its version of the Give Kids a Chance Act (S. 932), ensuring continued innovation by extending the RPD-PRV program.
You can join the rare disease community by taking action today. Tell your Senators to support the bill’s passage by sending a pre-drafted email today. It only takes a few minutes of your time. The more attention and support this issue receives, the closer we move toward a future where children with PKD have real treatment options—and real hope.
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