FDA Must Now Study Adult Cancer Drugs on Kids, Plus New Fees Hit Organ Transplant Centers

This bill, named the Mikaela Naylon Give Kids a Chance Act, is a massive legislative package focused primarily on improving drug development for children and rare diseases, but it also includes major updates to the national organ transplant system and tweaks to generic drug transparency.

The core purpose of the bill is to force drug companies to test promising new cancer treatments on children when the drug’s molecular target is relevant to a pediatric cancer. Specifically, Section 2 amends the Federal Food, Drug, and Cosmetic Act to require a "molecularly targeted pediatric cancer investigation" for new adult cancer drugs if the Secretary of Health and Human Services determines the drug’s target is “substantially relevant” to a pediatric cancer. This means companies can no longer easily skip pediatric testing just because the drug was initially developed for adults. The goal is to ensure that kids get access to the latest, most targeted therapies with appropriate dosing and safety data, rather than relying on educated guesses.

Pediatric Drug Development: Less Skipping, More Studying

If a drug company fails to complete these required pediatric studies, Section 3 tightens the screws on enforcement. The FDA must now issue a formal noncompliance letter and, before taking action, give the company 45 days to show “due diligence” in trying to meet the requirement. This closes a loophole where companies might face little penalty for simply failing to submit the required data. Section 4 backs this up by requiring the FDA to publicly report all penalties and settlements collected for these failures, naming the drug, the sponsor, and the amount—adding a layer of public accountability.

To keep the pipeline moving for truly rare diseases, Section 5 extends the rare pediatric disease priority review voucher program until September 30, 2029. This is the incentive where a company developing a drug for a very small patient population gets a valuable voucher that speeds up the review of a different, more profitable drug. A key part of this extension is a required study by the Comptroller General to figure out if these vouchers are actually doing their job—are they inspiring new treatments, or just being flipped for cash?

Orphan Drug Exclusivity Gets a Haircut

Section 6 makes a significant change to market exclusivity for orphan drugs—drugs developed for rare diseases. Currently, a company gets 7 years of market exclusivity for treating a “rare disease or condition.” This bill narrows that protection. Instead of covering the whole disease, the exclusivity will only apply to the specific “approved use or indication” within that disease. For example, if a drug is approved to treat Symptom A of a rare disease, a competitor could potentially get approval to treat Symptom B of the same disease, even during the 7-year window. This is a big deal for pharmaceutical companies, potentially reducing the financial protection that incentivizes rare disease research, but it could also open the door for competitors to bring forward new treatments faster for different aspects of the same condition.

Organ Transplant System Gets a Tech Upgrade (and a Bill)

Shifting gears entirely, Section 8 tackles the Organ Procurement and Transplantation Network (OPTN). The bill modernizes the network by requiring functions like integrating electronic health records among hospitals and transplant centers and promoting automated donor referrals, all while adhering to HIPAA privacy rules. This move aims to fix the current patchwork system by allowing better, faster data sharing—critical when every minute counts for an organ recipient.

However, this upgrade comes with a cost. Section 8 also grants the Secretary of Health and Human Services authority to collect registration fees from any OPTN member (like a hospital or transplant center) for each transplant candidate placed on the national waiting list. These fees are intended to support OPTN operations. While this fee collection authority has a 3-year sunset clause, it introduces a new operational cost for transplant centers, which could ultimately affect how they manage their waiting lists and budgets.

Transparency and International Relations

Rounding out the bill, Section 10 increases transparency in generic drug applications. If a generic manufacturer asks, the FDA must now confirm whether their generic drug is “qualitatively and quantitatively the same” as the brand-name reference drug. If it’s not, the FDA has to identify the specific ingredient and the amount of the deviation. This should help generic manufacturers avoid costly delays and speed up the process of getting cheaper alternatives to market.

Finally, Section 9 establishes an Abraham Accords Office within the FDA. This office will be located in an Abraham Accords country and will focus on providing technical assistance to regulatory partners there to strengthen oversight and align regulatory requirements for FDA-regulated products. This aims to streamline international commerce and regulatory alignment in the region. And in a final, unrelated note, Section 11 increases the amount in the Medicare Improvement Fund by over $1.2 billion, raising it from $1.4 billion to $2.6 billion.