What Are Pediatric Priority Review Vouchers — and Why Do They Matter for the Rare Disease Community?
If you’re part of the rare disease community, you may have heard the term Pediatric Priority Review Voucher (PRV). Although the name includes “pediatric,” these vouchers can ultimately impact treatments for both children and adults.
What Is a Pediatric Priority Review Voucher?
A Pediatric Priority Review Voucher is an incentive created by the U.S. Food and Drug Administration (FDA). It is awarded to a company that successfully develops and receives approval for a drug that treats a rare pediatric disease.
Developing treatments for rare conditions — especially rare childhood diseases — is scientifically complex and financially risky. Patient populations are small, research costs are high, and the return on investment is uncertain. To encourage companies to take on this work, the FDA offers a reward: a voucher that allows any future drug application to receive priority review.
Under standard review, the FDA typically takes about 10 months to review a new drug application. With priority review, that timeline is shortened to about 6 months. That four-month difference can be very meaningful for patients waiting for new therapies.
Why Are Vouchers Sold?
The company that earns the voucher does not have to use it for its own product. Instead, it can sell the voucher to another company.
These vouchers have sold for substantial amounts — sometimes hundreds of millions of dollars. Why? Because accelerating FDA review for a high-impact or high-revenue drug can be extremely valuable to the purchasing company.
For smaller biotechnology companies working in rare diseases, selling a PRV can provide critical funding. The proceeds may be used to:
- Advance additional rare disease research
- Launch or complete clinical trials
- Expand manufacturing capacity
- Strengthen long-term financial sustainability
In many cases, this funding helps companies continue developing treatments for other rare conditions — including those affecting both children and adults.
How Does the Buying Company Benefit?
The company that purchases the voucher can use it to speed up FDA review of almost any eligible medication — whether it treats adults, children, or both.
A faster review may:
- Bring a treatment to market sooner
- Allow patients to access therapy earlier
- Provide earlier revenue that supports future research
In this way, the voucher program creates a connection between rare pediatric drug development and broader innovation across the healthcare system.
How Does the Rare Disease Community Benefit?
Even though the voucher is awarded for a pediatric rare disease treatment, its impact extends beyond children.
- It encourages investment in rare conditions that might otherwise be overlooked.
- It provides financial support to rare disease companies, helping them sustain and expand research pipelines.
- It accelerates access to other therapies, potentially benefiting adult and pediatric patients alike.
For patients and care partners navigating a rare diagnosis, understanding programs like Pediatric Priority Review Vouchers can shed light on how policy tools are used to stimulate research. While no single program solves every challenge in rare disease drug development, PRVs are one mechanism designed to promote innovation — and ultimately expand treatment options for the entire rare disease community.
