Mikaela Naylon Give Kids a Chance Act

The Mikaela Naylon Give Kids a Chance Act, which passed the House and is now under Senate review, would strengthen the FDA's oversight of medications developed for children with rare diseases. The bill would allow researchers to test new drugs in combination with already-approved medications for pediatric cancers, making it easier to develop treatments for conditions affecting young patients. It would also give the FDA new enforcement tools to penalize pharmaceutical companies that fail to conduct required pediatric studies, ensuring drug sponsors take their research obligations seriously.

The legislation would extend several key research support programs. It would continue the FDA's priority review voucher program, which provides incentives for developing treatments for rare pediatric diseases, through September 2029. Additionally, it would reauthorize funding through fiscal year 2027 for the National Institutes of Health to support priority pediatric research initiatives. These provisions aim to maintain momentum in developing new treatments for conditions that affect relatively small numbers of children.

The bill also addresses a legal dispute about orphan drug exclusivity, which grants seven-year market protection for medications treating rare diseases. It would clarify that this exclusivity applies specifically to the approved use or indication rather than all uses of a drug for a disease, codifying the FDA's longstanding interpretation. This clarification would help ensure that pharmaceutical companies can develop different formulations or applications of existing drugs for rare pediatric conditions without losing market protection.