Mikaela Naylon Give Kids a Chance Act

Bill Number: H.R. 1262
Origin Chamber: House
Congress: 119th Congress, Session 1
Policy Area: Health
Status: Passed House
Latest Action: 2025-12-02: Received in the Senate.
Last Updated: 2026-04-01T20:36:37Z

AI-Generated Summary

Purpose of the Legislation

The Mikaela Naylon Give Kids a Chance Act (H.R. 1262) aims to advance research, development, and access to treatments for pediatric cancers and rare pediatric diseases by amending the Federal Food, Drug, and Cosmetic Act (FD&C Act). It also addresses related areas, including enforcement of pediatric drug studies, incentives for rare disease treatments, limitations on drug exclusivity, improvements to organ transplantation systems, international regulatory cooperation, transparency in generic drugs, and increased funding for Medicare improvements. The overall goal is to enhance pediatric health outcomes, streamline drug approvals, and promote efficiency in healthcare systems.

Key Provisions

Pediatric Cancer Drug Research (Sec. 2): Expands requirements for molecularly targeted pediatric cancer investigations under the Pediatric Research Equity Act (PREA). Allows studies of a new drug alone or in combination with approved standard-of-care drugs for pediatric cancers or the sponsor's existing adult cancer drugs targeting relevant molecular pathways. Requires studies to provide meaningful data on dosing, safety, and efficacy for pediatric labeling. Mandates FDA guidance within 12 months and applies to new applications 3 years after enactment. Requires reports to Congress from HHS (in 6 years) and GAO (in 10 years) on implementation and effectiveness.

Enforcement of Pediatric Study Requirements (Sec. 3): Strengthens accountability for completing pediatric studies. Introduces a "noncompliance letter" process with a 45-day response period and a "due diligence" review before penalties. Limits enforcement actions (e.g., injunctions under FD&C Act Sec. 303) to cases showing lack of effort, and exempts non-marketed drugs. Applies to failures occurring 180 days after enactment.

FDA Report on Pediatric Study Enforcement (Sec. 4): Updates the annual FDA report on PREA to include evaluations of deadline compliance and a list of penalties, settlements, or payments for non-compliance, detailing the drug, sponsor, and amount.

Priority Review Vouchers for Rare Pediatric Diseases (Sec. 5): Extends the FDA's authority to issue vouchers (which speed up review of another drug) for rare pediatric disease treatments from December 2024 to September 2029. Adjusts user fee timing to the submission of the voucher-using application. Requires a GAO study and report (in 5 years) on the voucher's effectiveness in incentivizing development, including impacts on unmet needs, company sizes, and FDA workload.

Limitations on Orphan Drug Exclusivity (Sec. 6): Narrows the 7-year exclusive approval period for orphan drugs (for rare diseases) to apply only to the specific approved use or indication, not the entire rare disease or condition. Applies retroactively to all designated orphan drugs.

Funding for Pediatric Drug Studies (Sec. 7): Allocates $25 million annually for fiscal years 2026–2028 to the FDA's pediatric drug studies program under the Public Health Service Act.

Organ Procurement and Transplantation Network (OPTN) Improvements (Sec. 8): Updates OPTN requirements to promote electronic health records integration (via APIs or similar tech) for better donor referrals and access, while complying with HIPAA privacy rules. Requires consideration of a public dashboard for transplant statistics (updated more than annually). Authorizes temporary (3-year) registration fees on OPTN members for listing transplant candidates to fund operations, with transparency requirements and a GAO review in 2 years.

Abraham Accords Office in FDA (Sec. 9): Establishes an FDA office in an Abraham Accords country (e.g., Israel, UAE, as defined by the State Department) within 2 years. Duties include providing technical assistance on regulatory standards (e.g., good manufacturing practices) and facilitating FDA interactions with local parties on drug development. Requires a HHS report to Congress in 3 years on progress. Ensures compliance with national security.

Transparency in Generic Drug Applications (Sec. 10): Requires FDA, upon request, to disclose if a generic drug's inactive ingredients (non-active components like fillers) match those in the brand-name (listed) drug qualitatively (type) and quantitatively (amount). If not, identifies differences (except trade secrets). Prevents FDA from reversing such determinations without cause (e.g., safety issues). Mandates guidance within 1 year on assessments, including pH adjusters (substances controlling acidity). Applies immediately upon enactment.

Medicare Improvement Fund (Sec. 11): Increases the fund's appropriation from $1.403 billion to $2.622 billion to support Medicare program enhancements.

Significant Changes to Existing Law

Pediatric Research (Secs. 2–4, 7): Broadens PREA to include drug combinations for pediatric cancers, adds due diligence in enforcement, and boosts funding—shifting from general pediatric assessments to targeted, actionable studies.
Rare Disease Incentives (Sec. 5): Prolongs the voucher program by nearly 5 years and refines fee structures, building on the 2012 FDA Safety and Innovation Act.
Orphan Drugs (Sec. 6): Restricts exclusivity from broad disease coverage to specific indications, potentially allowing more competition (applies retroactively, unlike prior limits).
OPTN (Sec. 8): Introduces modern tech integration and fee authority, modernizing a 1980s-era law (National Organ Transplant Act).
Generics (Sec. 10): Adds disclosure requirements for inactive ingredients, addressing past FDA opacity issues without broadly revealing trade secrets.
International and Funding (Secs. 9, 11): Creates a new FDA office (novel addition) and raises Medicare funding, expanding beyond core FD&C Act focus.

Potential Impacts

Government Agencies: Increases FDA workload for guidance, reviews, reports, and a new international office; HHS/GAO face additional study/report obligations. OPTN gains funding/tools for efficiency but must ensure HIPAA compliance. Medicare Administration benefits from higher fund for program improvements.
Citizens: Pediatric patients may access more tailored cancer treatments faster; rare disease families gain from extended incentives. Generic drug transparency could lower costs and speed approvals. Organ transplant candidates benefit from better data sharing and statistics, potentially increasing transplants. Medicare enrollees see indirect gains from enhanced services.
International Relations: Strengthens U.S. regulatory ties with Abraham Accords countries, promoting aligned standards for medical product manufacturing and trade, without compromising security.

Main Stakeholders Affected

Pediatric Patients and Families: Primary beneficiaries through improved cancer/rare disease drug access.
Pharmaceutical and Biotech Companies: Drug developers (especially for pediatrics/orphans) face new study mandates but gain incentives; generics firms benefit from transparency.
FDA and HHS: Must implement changes, issue guidance, and report progress.
Healthcare Providers and Organizations: Hospitals, transplant centers, and organ procurement groups adapt to electronic systems and fees.
Generic Drug Manufacturers: Easier bioequivalence demonstrations via disclosures.
Medicare Beneficiaries and Payers: Impacted by fund increase for program enhancements.
Abraham Accords Countries and Entities: Receive technical assistance, fostering regulatory convergence.

Notable Legal, Constitutional, or Political Implications

Legal: Amendments clarify and limit enforcement (e.g., due diligence protects against arbitrary penalties) while protecting trade secrets in generics/orphan rules. Retroactive orphan changes could face industry challenges but aim to balance incentives with competition. Fee collections for OPTN are time-limited and transparent to avoid overreach.
Constitutional: No direct challenges; provisions respect privacy (HIPAA) and security, aligning with due process via response periods.
Political: Emphasizes child health and international alliances (Abraham Accords), potentially bipartisan appeal. Reports to Congress enable oversight; GAO studies assess cost-benefit, informing future policy without mandating changes.

This summary was generated by AI and may contain inaccuracies. Refer to the official source document for the authoritative text.

Sponsor

Rep. McCaul, Michael T. [R-TX-10]

Cosponsors (313)

Rep. Bilirakis, Gus M. [R-FL-12], Rep. Dingell, Debbie [D-MI-6], Rep. Schrier, Kim [D-WA-8], Rep. Harshbarger, Diana [R-TN-1], Rep. Matsui, Doris O. [D-CA-7], Rep. Crenshaw, Dan [R-TX-2], Rep. Castor, Kathy [D-FL-14], Rep. Kelly, Mike [R-PA-16], Rep. Trahan, Lori [D-MA-3], Rep. Weber, Randy K. Sr. [R-TX-14], Rep. Wittman, Robert J. [R-VA-1], Rep. Allen, Rick W. [R-GA-12], Rep. Zinke, Ryan K. [R-MT-1], Rep. Evans, Gabe [R-CO-8], Rep. Higgins, Clay [R-LA-3], Rep. Gimenez, Carlos A. [R-FL-28], Rep. Miller, Carol D. [R-WV-1], Rep. Cline, Ben [R-VA-6], Rep. Sessions, Pete [R-TX-17], Rep. Rulli, Michael A. [R-OH-6], Rep. Mullin, Kevin [D-CA-15], Rep. Soto, Darren [D-FL-9], Rep. Keating, William R. [D-MA-9], Rep. McIver, LaMonica [D-NJ-10], Rep. Case, Ed [D-HI-1], Rep. Castro, Joaquin [D-TX-20], Rep. Sewell, Terri A. [D-AL-7], Rep. Wasserman Schultz, Debbie [D-FL-25], Del. Norton, Eleanor Holmes [D-DC-At Large], Rep. McGarvey, Morgan [D-KY-3], Rep. Diaz-Balart, Mario [R-FL-26], Rep. Wilson, Joe [R-SC-2], Rep. LaHood, Darin [R-IL-16], Rep. Van Drew, Jefferson [R-NJ-2], Rep. Malliotakis, Nicole [R-NY-11], Rep. Larson, John B. [D-CT-1], Rep. Bishop, Sanford D. [D-GA-2], Rep. DelBene, Suzan K. [D-WA-1], Rep. Harder, Josh [D-CA-9], Rep. Grijalva, Raúl M. [D-AZ-7], Rep. Schneider, Bradley Scott [D-IL-10], Rep. Scanlon, Mary Gay [D-PA-5], Rep. Finstad, Brad [R-MN-1], Rep. Huizenga, Bill [R-MI-4], Rep. Miller, Mary E. [R-IL-15], Rep. Yakym, Rudy [R-IN-2], Rep. Costa, Jim [D-CA-21], Rep. McCollum, Betty [D-MN-4], Rep. Hayes, Jahana [D-CT-5], Rep. Garcia, Robert [D-CA-42] and 263 more

Recent Actions

2025-12-02: Received in the Senate.
2025-12-01: Motion to reconsider laid on the table Agreed to without objection.
2025-12-01: On motion to suspend the rules and pass the bill, as amended Agreed to by voice vote. (text: CR H4929-4932)
2025-12-01: Passed/agreed to in House: On motion to suspend the rules and pass the bill, as amended Agreed to by voice vote. (text: CR H4929-4932)
2025-12-01: DEBATE - The House proceeded with forty minutes of debate on H.R. 1262.
2025-12-01: Considered under suspension of the rules. (consideration: CR H4929-4935)
2025-12-01: Mr. Carter (GA) moved to suspend the rules and pass the bill, as amended.
2025-10-31: Placed on the Union Calendar, Calendar No. 304.
2025-10-31: Reported (Amended) by the Committee on Energy and Commerce. H. Rept. 119-352.
2025-10-31: Reported (Amended) by the Committee on Energy and Commerce. H. Rept. 119-352.
2025-09-17: Ordered to be Reported in the Nature of a Substitute (Amended) by the Yeas and Nays: 47 - 0.
2025-09-17: Committee Consideration and Mark-up Session Held
2025-02-12: Referred to the House Committee on Energy and Commerce.
2025-02-12: Introduced in House
2025-02-12: Introduced in House

Bill Versions

Mikaela Naylon Give Kids a Chance Act — issued 2025-12-01 — PDF (32 pages)
Give Kids a Chance Act of 2025 — issued 2025-02-12 — PDF (26 pages)
Give Kids a Chance Act of 2025 — issued 2025-10-31 — PDF (34 pages)