Mikaela Naylon Give Kids a Chance Act

The bill would make several changes to federal drug law. It would expand the scope of molecularly targeted pediatric cancer investigations to permit research on new drugs used in combination with already-approved active ingredients, subject to conditions. It would authorize the FDA to take enforcement action — including civil penalties — against drug sponsors that fail pediatric study requirements, but only when the sponsor demonstrated a lack of due diligence, and only for products still on the market. It would extend the FDA's authority to award priority review vouchers for rare pediatric disease treatments through September 30, 2029. It would codify in statute the FDA's interpretation that the seven-year orphan drug exclusivity period blocks approval of the same drug only for the same approved indication — directly overriding a court ruling to the contrary. It would reauthorize $25 million per year through FY2028 for NIH pediatric drug studies, update the Organ Procurement and Transplantation Network statute, and increase the Medicare Improvement Fund balance.

The bill would also establish an Abraham Accords Office within the FDA, to be located in an Abraham Accords country and charged with providing regulatory technical assistance and facilitating interactions with industry partners in those countries. It would require the FDA to disclose to generic drug applicants whether their formulation matches the listed drug's ingredients, improving transparency in the generic drug review process.

Pharmaceutical and biotechnology companies that develop drugs for pediatric cancers and rare pediatric diseases; children with cancer or rare diseases who could benefit from new treatments; sponsors of orphan drugs whose seven-year exclusivity period would be narrowed to specific indications; generic drug manufacturers seeking formulation guidance from the FDA; hospitals, organ procurement organizations, and transplant centers subject to updated OPTN requirements; and regulatory bodies in Abraham Accords countries.

Drug sponsors who have been able to avoid penalties for ignoring pediatric study requirements would face new enforcement risk. Companies holding orphan drug exclusivity would lose the broader protection a federal court granted, potentially opening markets to competitors sooner for different indications of the same disease. Children with rare cancers could gain access to more combination therapies. Generic manufacturers would receive clearer FDA guidance on ingredient matching, potentially speeding approvals.

The FDA would issue draft guidance within 12 months of enactment on expanded pediatric cancer investigation requirements, with final guidance due 12 months after the comment period closes. Enforcement authority over noncompliant drug sponsors would activate 180 days post-enactment, with a required noncompliance letter and 45-day response window before any penalty. The Secretary would establish the Abraham Accords Office within two years. The GAO would conduct multiple studies — on pediatric cancer drug effectiveness, priority review voucher impact, and OPTN registration fees — and report to Congress at five- to ten-year intervals. FDA must issue generic drug transparency guidance within one year.