Mikaela Naylon Give Kids a Chance Act

What Would Change

Today: Drug companies have certain requirements for pediatric cancer drug studies, but this bill would expand them to include molecularly targeted investigations and combination therapies. Enforcement for pediatric study requirements can lead to penalties, but the process for determining "due diligence" is not as clearly defined. Orphan drugs receive market exclusivity for a broad "same disease or condition." The FDA's authority to issue rare pediatric disease priority review vouchers is set to expire in 2024. The Organ Procurement and Transplantation Network (OPTN) has existing requirements but lacks specific mandates for electronic health record integration, a public dashboard for transplant statistics, and the ability to collect registration fees from members. There is no dedicated Abraham Accords Office within the FDA. Generic drug applicants may not easily get information about inactive ingredients in brand-name drugs. The Medicare Improvement Fund is set at $1,403,000,000. After this bill: Drug companies would face expanded requirements for molecularly targeted pediatric cancer investigations, including for combination drugs, with new FDA guidance and reports to Congress. The FDA's enforcement actions for pediatric study noncompliance would require a due diligence process and would not apply to drugs no longer marketed. Orphan drug exclusivity would be limited to the "same approved use or indication within such rare disease or condition." The FDA's authority to issue rare pediatric disease priority review vouchers would be extended until September 30, 2029, and the user fee payment timing would change. The OPTN would be encouraged to integrate electronic health records, establish a public dashboard for transplant data, and be authorized to collect registration fees from members (with a 3-year sunset). An Abraham Accords Office would be established within the FDA to provide technical assistance and facilitate international regulatory interactions. The FDA would be required to disclose inactive ingredient information to generic drug applicants upon request for bioequivalence determinations. The Medicare Improvement Fund would increase to $2,622,000,000.