HR1262-119

What This Bill Does

The Mikaela Naylon Give Kids a Chance Act makes a package of FDA, NIH, transplant, and Medicare changes. It lets FDA require molecularly targeted pediatric cancer investigations not only for a sponsor's own adult cancer drug but also for combinations with already approved standard-of-care ingredients or same-sponsor adult cancer drugs aimed at pediatric-relevant molecular targets. It tightens pediatric-study noncompliance procedures, requires FDA reporting on PREA penalties and settlements, extends rare pediatric disease priority review voucher authority to September 30, 2029, directs GAO to study voucher effectiveness, narrows orphan-drug exclusivity to the approved use or indication, funds NIH pediatric drug studies at $25 million per year for 2026-2028, updates Organ Procurement and Transplantation Network data and fee authority, creates an FDA Abraham Accords Office, requires certain generic-drug ingredient sameness disclosures, and increases the Medicare Improvement Fund.

Who Benefits and How

Children with cancer, children with rare diseases, pediatric oncologists, NIH pediatric study networks, generic drug manufacturers, transplant candidates, hospitals, organ procurement organizations, transplant centers, Abraham Accords country regulators, medical product manufacturers in Abraham Accords countries, Medicare policymakers, and FDA reviewers gain clearer authorities, funding, data, or regulatory pathways. Pediatric patients benefit when drug sponsors must generate clinically meaningful pediatric data. Generic applicants benefit when FDA can disclose which inactive ingredients prevent a product from being qualitatively or quantitatively the same as the reference drug. Transplant patients benefit from electronic referral, EHR access, and dashboard transparency.

Who Bears the Burden and How

Brand-name pharmaceutical sponsors, biologics sponsors, orphan-drug holders, FDA review divisions, FDA pediatric and PREA enforcement staff, the Comptroller General, NIH program administrators, OPTN members, hospitals, organ procurement organizations, transplant centers, FDA international-affairs staff, and generic-application reviewers must conduct additional studies, respond to noncompliance letters, report penalties, evaluate voucher effectiveness, operate transplant-network data tools, collect registration fees, stand up the Abraham Accords Office, disclose formulation sameness information, and administer revised Medicare Improvement Fund amounts.

Key Provisions

• Expands FDA pediatric oncology study authority to require molecularly targeted pediatric cancer investigations involving combinations with standard-of-care or same-sponsor adult cancer drugs.
• Requires pediatric studies to yield clinically meaningful pediatric data on dosing, safety, preliminary efficacy, and age-appropriate formulations.
• Requires noncompliance letters, 45-day responses, due-diligence review, and PREA penalty reporting before certain pediatric-study enforcement actions.
• Extends rare pediatric disease priority review voucher authority through September 30, 2029 and requires a GAO study of voucher effectiveness, unmet need, company size, voucher value, and use timing.
• Narrows orphan-drug exclusivity from the same disease or condition to the same approved use or indication.
• Appropriates $25 million per year for 2026 through 2028 for NIH pediatric studies of drugs.
• Requires OPTN support for electronic health record integration, automated hospital referrals, remote OPO access to donor records, transplant dashboards, and member registration fees.
• Establishes an FDA Abraham Accords Office to provide regulatory technical assistance, select a location in an Abraham Accords country, and report to Congress.
• Requires FDA to disclose inactive-ingredient sameness information to certain generic-drug applicants and increases the Medicare Improvement Fund.