New FDA Rules Force Drug Companies to Test Cancer Drugs on Kids, Extends Rare Disease Voucher Program to 2029

This bill, the Give Kids a Chance Act of 2025, is focused squarely on fixing a major gap in drug development: making sure new treatments for cancer and rare diseases are specifically tested and tailored for children. It’s essentially updating the playbook the FDA uses to regulate pharmaceutical companies, especially when it comes to pediatric medicine.

The core of the bill is twofold: first, expanding the FDA’s power to demand pediatric testing for cancer drugs, and second, extending a successful incentive program designed to spur development for rare childhood diseases. If you’re a parent, or if you know someone dealing with pediatric illness, this bill is about getting those kids access to cutting-edge, age-appropriate treatments faster.

Pediatric Cancer: No More Hand-Me-Down Dosing

Section 2 tackles the issue of pediatric cancer drugs. Historically, many drugs were approved for adults, and doctors had to guess at the right dose for kids—a practice known as “off-label use.” This bill aims to stop that, particularly for molecularly targeted cancer drugs. When a drug company submits an application for a new cancer drug, the FDA can now require them to study that drug specifically for children’s cancer.

Crucially, this new requirement expands to cover combination therapies. If a company is developing a new drug that targets a specific molecular pathway (like a genetic mutation common in certain cancers), the FDA can now mandate that the company study that drug in combination with existing standard pediatric treatments. This is a big deal because cancer treatment often involves multiple drugs working together. For the pharmaceutical industry, this means an increased upfront cost and workload, as they can no longer just focus on the adult market and hope the pediatric data follows later. They must now design specific, clinically meaningful studies for kids, looking closely at dosing and safety from the start.

Extending the Rare Disease Fast Pass

Section 3 extends a program that has been critical for getting treatments for rare pediatric diseases (those affecting fewer than 200,000 people). This program uses Priority Review Vouchers (PRVs), which are basically golden tickets that let a drug company skip the long line and get their drug application reviewed by the FDA faster—sometimes saving months or even a year. Companies earn these vouchers by developing a drug for a rare pediatric disease, and they can either use it themselves or sell it to another company for potentially hundreds of millions of dollars.

The authority for the FDA to issue these vouchers was set to expire in December 2024. This bill pushes that deadline out to September 30, 2029. For patients with rare diseases, this extension means the incentive for drug companies to tackle these difficult, small-market conditions remains strong for another five years. The bill also clarifies that if a company uses one of these vouchers, they must pay the associated priority review user fee at that time, though all other standard user fees remain due as scheduled.

What’s the Catch? More Oversight, More Work

While this bill is a clear win for pediatric health, it introduces new administrative complexity. The FDA will have to issue new guidance within the next two years to define exactly how these new combination study requirements will work. Furthermore, the bill mandates extensive studies by the Government Accountability Office (GAO) and the Department of Health and Human Services (HHS).

Essentially, the GAO has to become a policy detective, digging into how effective the voucher program has been over the past decade—looking at who got the vouchers (big pharma vs. small biotech), how much they sold for, and whether the drugs actually met an unmet medical need. This level of required oversight ensures that these regulatory tools and financial incentives are actually doing what they’re supposed to: generating real treatments, not just paperwork.