Right now, Congress has a chance to save the lives of countless sick children—without spending a dime of taxpayer money.
It could seize that opportunity by reauthorizing the Pediatric Priority Review Voucher (PPRV) program—which incentivizes companies to invest in lifesaving but financially risky treatments for rare pediatric diseases.
The PPRV program is one of the most successful conservative health policies in recent decades. It has delivered dozens of new treatments for rare and terminal conditions without placing any burden on taxpayers. Instead, it harnesses market forces to drive innovation. Yet last year, its authorization expired.
Here’s how it works. When a company successfully develops a treatment for a rare pediatric disease and receives FDA approval, it gets awarded a voucher that grants expedited FDA review of another drug.
The company can use the voucher to speed up the approval of one of its own treatments in the development pipeline—or sell the voucher to another firm, generating revenue that can be reinvested into rare disease R&D.
The PPRV program is an elegant solution to the problem that has long hindered medical progress on rare diseases—the fact that these diseases’ small patient populations limit companies’ potential returns, even if they successfully develop a new treatment.
While rare diseases collectively afflict roughly 30 million Americans—most of them children—each individual disease affects fewer than 200,000 people.
So historically, drugmakers haven’t been able to sell enough of each new treatment to make the development process financially viable. Approximately 95% of known rare diseases have no FDA-approved treatment.
An expedited FDA review process means that a drug will have a longer exclusivity period—and thus generate more revenue. By creating incentives for rare disease research, the PPRV program has paid substantial dividends for patients. In just over a decade, it has driven the approval of 50 life-saving treatments for 40 different ailments—including conditions like spinal muscular atrophy and Duchenne muscular dystrophy.
In fact, every drug developer interviewed by the Government Accountability Office in a 2020 report confirmed that the PPRV program influenced their decision to invest in pediatric rare disease research.
Without the PPRV program, many existing rare disease treatments simply would not exist—and other key treatments would be slower to come to market. It’s exactly the kind of free-market policy conservatives should embrace—one that’s proven to spur medical breakthroughs and save lives at zero cost to taxpayers.
And reauthorizing the PPRV program wouldn’t only be good policy—it’d be smart politics. Lawmakers have already reintroduced a bill to reauthorize PPRV: the Give Kids a Chance Act.
Passing the bill as soon as possible would help deliver a healthcare victory for President Trump.
PPRV is a common-sense, conservative solution that accelerates FDA approvals, promotes free-market innovation, and gives hope to children suffering from rare diseases.
President Trump, Speaker Johnson, and Congressional Republicans have a golden opportunity to save sick children, spur medical innovation, and advance free-market reforms by reviving the program.
