Give Kids a Chance Act of 2025: FDA Gets New Powers, Pediatric Drug Development Revamped

The "Give Kids a Chance Act of 2025" is a multi-pronged bill focused primarily on pediatric healthcare, particularly in the areas of drug development for cancer and rare diseases, and also touches on organ transplantation and international regulatory cooperation. The core purpose is to incentivize and streamline the creation of new treatments for children, while also tightening up some existing regulations and boosting oversight.

Pediatric Drug Push

The bill makes several key changes to how drugs for kids, especially those targeting cancer, are developed and approved. For starters, it requires drug companies to conduct "molecularly targeted pediatric cancer investigations" when developing new drugs or biologics, or when using existing drugs that hit the same targets as adult cancer treatments (SEC. 2). This means companies can't skip out on testing how these drugs work specifically in children. The goal is to get "clinically meaningful data" on the right doses, safety, and effectiveness for different age groups, from toddlers to teens. Think of it like this: a medication that works for a 180-pound adult might be dangerous for a 40-pound child, so these studies are crucial. A farmer wouldn't use the same amount of fertilizer on a sapling as they would a mature tree, right? Same principle applies here.

It also extends the FDA's authority to issue "priority review vouchers" for rare pediatric disease treatments until September 30, 2029 (SEC. 5). These vouchers are basically fast passes for drug approval, and companies can either use them themselves or sell them to others. This is a big deal because it can significantly speed up getting new drugs to market. The bill does require a study on how well these vouchers actually work, though, with a report due to Congress within five years.

Holding Companies Accountable

This bill isn't just about carrots; there's a stick, too. The Act amends the Federal Food, Drug, and Cosmetic Act to ensure companies actually complete required pediatric studies (SEC. 3). If a company falls behind, the FDA must send a "noncompliance letter" before taking any enforcement action. The company then has 45 days to explain themselves. The FDA will then check for "lack of due diligence." It's like getting a warning before getting a speeding ticket – a chance to explain why you were going over the limit. However, this only applies to failures that occur 180 days after the Act is passed. The FDA is also mandated to provide a report on its enforcement of the Pediatric Research Equity Act (PREA), including penalties assessed (SEC. 4).

Orphan Drug Tweaks and Transplantation Updates

The Act also narrows the scope of "orphan drug" exclusivity (SEC. 6). Orphan drugs are those developed for rare diseases. Previously, exclusivity applied to the entire disease; now, it's tied to the specific approved use. Imagine a pizza shop only being allowed to sell one type of pizza – that’s what this change aims to address, promoting more options within the same rare disease category.

For organ transplantation, the bill requires the Organ Procurement and Transplantation Network to better integrate electronic health records and consider a public dashboard of transplant stats (SEC. 8). The Secretary of Health and Human Services can also collect fees from network members to fund operations, but this authority sunsets in three years. Think of it as upgrading the system to be more transparent and efficient, like switching from paper files to a digital database.

The Abraham Accords Angle

Finally, the bill establishes an "Abraham Accords Office" within the FDA (SEC. 9), located in one of the Abraham Accords countries (nations that have normalized relations with Israel). This office will help those countries align their drug regulations with those of the U.S., provide technical assistance, and facilitate interactions between the FDA and interested parties. This is intended to streamline the process for manufacturers in those countries to meet FDA standards. The Secretary has two years to set up the office and three years to report on its progress. The office is not required to take any action that is inconsistent with national security, as determined by the federal government.

Funding

The bill allocates $25,000,000 annually from 2025 through 2027 for pediatric drug studies (SEC.7).