Opinion | A program targeting rare pediatric diseases lapsed. Kids need Congress to fix it.

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When a clinical trial slips from “this month” to “next quarter,” nothing dramatic happens in the Capitol — but in a living room somewhere, a parent reassesses hope.

Since the Rare Pediatric Disease Priority Review Voucher (PRV) Program lapsed on Dec. 20, 2024, those slips stopped being hypothetical. The PRV Program is one of the few levers that make investment in research and development for devastating pediatric diseases viable. Without it, investments hesitate, timelines drift and children pay in loss of function they cannot regain. As residents and providers to the Minnesota rare disease community, we know that kids cannot wait — Congress must restore the PRV Program now.

The PRV Program is simple and effective. When the FDA approves a therapy for a qualified rare pediatric disease, the sponsor also earns a voucher for priority FDA review of another medicine, shortening review time to roughly six months instead of 10. That predictability helps pull private capital into complex diseases with limited populations, especially for small and midsize biotechs whose future is contingent on securing the next round of funding. In fact, advocacy-founded biotechs often rely on this program to create effective business models. Restoring the PRV Program doesn’t create a new spending program; it aligns incentives so promising and innovative science isn’t stranded for lack of financing. And there is no cost to Minnesota taxpayers.

The need is enormous. One in 10 Americans lives with a rare disease, with nearly two-thirds of rare diseases starting in childhood. Of the roughly 10,000 rare diseases, 95% still lack an FDA-approved treatment. For these families, PRV isn’t an abstraction. It’s the difference between trials that launch and trials that stall; between a study coordinator being hired or a site being canceled; between a therapy reaching the finish line and one that never leaves the notebook.

The program’s impact is real. Since its inception, the FDA has awarded 60-plus pediatric PRVs across more than 40 devastating conditions, accelerating a wave of development barely imaginable a decade ago. Developers, especially the emerging companies that carry so much of the early financial risk, consistently cite PRV as a factor in whether to start, sustain or expand pediatric programs. That’s why the policy has long enjoyed bipartisan support: It channels private dollars toward children’s hardest problems without adding new federal spending or taxpayer resources.

With the promise of a PRV opportunity on the horizon, multiple investigational therapies are being pursued for Dravet syndrome, some of which have already demonstrated substantial seizure reduction and, for the first time, improvements in other unmet needs.

Despite the incredible potential of new therapies for Dravet syndrome and other rare diseases affecting children, investors face the question of whether earlier-stage therapies will make it to the market if Congress doesn’t restore the PRV Program. If Congress takes no further action, any prospective therapy that received Rare Pediatric Designation before Dec. 20, 2024, would have to submit its final application to the FDA within weeks to have a chance of gaining approval before the Sept. 30, 2026, PRV Program expiration, a date that is rapidly approaching. For the Dravet syndrome patient community and so many other rare disease communities with therapies in the pipeline — with hope in the pipeline — this timeline is not feasible.

In communities across the nation, families are navigating diagnoses without approved options. Trials are being run that depend on stable policy to plan staffing, sites and enrollment. Rare pediatric disease advocates have recently met with members of Congress all over the country, including in Minnesota, to show what delay looks like in real life: a child losing function while a start date slips on a calendar.

Congress has a clear path. The Give Kids a Chance Act was introduced to restore the FDA’s authority to award rare pediatric PRVs and extend it for multiple years. Passing it would switch the signal back to “go” for sponsors planning pivotal studies, for sites hiring coordinators, and for families deciding whether to uproot for a trial.

For children with rare diseases, time is life. Congress should renew the PRV Program immediately and keep hope on schedule for Minnesota children and families who cannot afford another lost week.