New Act Extends Rare Pediatric Drug Vouchers and Changes Rules for Cancer Drug Testing and Organ Donations

The Give Kids a Chance Act of 2025 is a comprehensive piece of legislation that looks to tighten up drug testing requirements for children, especially those with cancer, while also making some big changes to how the FDA handles generic drugs and even how the organ transplant system is funded. This bill is less about a single silver bullet and more about adjusting the gears across several critical areas of public health.

The Fine Print on Pediatric Cancer Drugs

If you’ve ever wondered why drug development seems slow for kids, especially those with rare cancers, part of it is regulatory complexity. This bill tries to fix that by setting new, highly specific rules for drug companies developing molecularly targeted cancer drugs (SEC. 2). Now, when a company submits a drug application, the required pediatric investigation must cover the drug alone or in combination with other ingredients already considered the standard of care for a pediatric cancer. This is a big deal because it forces companies to test their new drugs in the real-world context of combination therapies, which is often how cancer is actually treated. The goal is better data sooner, but it also adds complexity, potentially slowing down development if the combination requirements are too onerous.

Accountability and the Orphan Drug Trade-Off

One major incentive for developing treatments for rare diseases is the Orphan Drug designation, which grants market exclusivity. This bill narrows that protection significantly (SEC. 6). Currently, exclusivity is granted based on the “same disease or condition.” This bill changes that to the “same approved use or indication.” Think of it this way: if a drug is approved for a rare disease that causes both bone loss and vision problems, a competitor could potentially develop a new drug for the vision problem aspect of that disease without violating the first drug's exclusivity. This change is designed to encourage more competition and treatment options within the same rare disease, but it will definitely make drug companies that rely on broad exclusivity nervous.

On the accountability front, the bill addresses the Pediatric Research Equity Act (PREA), which requires studies on how drugs work in children. If a company fails to complete a required study, the FDA can issue a noncompliance letter. This bill adds a due diligence requirement, giving the company 45 days to respond before the FDA can officially determine a failure (SEC. 3). However, there’s a loophole: the FDA is prohibited from taking enforcement action if the drug is no longer being marketed. This means a company could simply pull a product off the shelf to avoid penalties for failing to conduct required pediatric studies, raising concerns about accountability.

Extending Incentives and Funding the Organ Network

One clear win for patients is the extension of the Rare Pediatric Disease Priority Review Voucher (PRV) program until September 30, 2029 (SEC. 5). These vouchers are like golden tickets: they incentivize companies to develop drugs for rare childhood diseases by allowing them to cut the review time for a different, more profitable drug. Extending this program is a direct boost to getting treatments for conditions that affect only a handful of children. The bill also requires a new study from the Government Accountability Office (GAO) to measure just how effective this voucher program actually is.

Separately, the bill makes a temporary, but significant, change to the Organ Procurement and Transplantation Network (OPTN) (SEC. 8). The Secretary of HHS now has the authority to collect registration fees from OPTN members—like transplant centers and organ procurement organizations—for every candidate placed on the waiting list. This fee is meant to support the network's operations, and the authority to collect it sunsets after three years. It also pushes the OPTN to integrate modern technology, like electronic health records and 24-hour IT service, which should streamline the complex process of matching organs to recipients.

Making Generic Drugs Less of a Mystery

For generic drug manufacturers, the process of proving their product is identical to the brand-name version can be frustratingly opaque. This bill mandates increased transparency (SEC. 10). If a generic company asks the FDA if their proposed drug is “qualitatively and quantitatively the same” as the brand-name version, the FDA must now disclose two key pieces of information if they determine the drugs are not the same: 1) the specific ingredient causing the difference, and 2) the exact amount of that difference. This cuts through months of back-and-forth for manufacturers, speeding up the pathway to market for cheaper generic options. The FDA must issue guidance on how they make these “sameness” determinations within a year.

Finally, the bill creates an Abraham Accords Office within the FDA (SEC. 9). This new office will be physically located in an Abraham Accords country and is tasked with helping those countries align their regulatory oversight with the FDA’s standards. The goal is to strengthen the global supply chain and regulatory compliance, particularly for medical products manufactured in those regions.