Mikaela Naylon Give Kids a Chance Act of 2025

Bill Number: S. 3302
Origin Chamber: Senate
Congress: 119th Congress, Session 1
Policy Area: Health
Status: Introduced
Latest Action: 2025-12-02: Read twice and referred to the Committee on Health, Education, Labor, and Pensions.
Last Updated: 2026-06-11T18:43:28Z

AI-Generated Summary

Purpose

The Mikaela Naylon Give Kids a Chance Act of 2025 aims to improve the development and approval of drugs and biological products (like vaccines or proteins used as medicines) for treating pediatric cancers. It does this by expanding requirements for studies on targeted cancer therapies in children and extending incentives for rare pediatric diseases, ultimately seeking to provide more safe and effective treatment options for kids with cancer.

Key Provisions

Amendments to Pediatric Research Requirements (Section 2):
Expands the definition of required "molecularly targeted pediatric cancer investigations" (studies focused on drugs that target specific molecules in cancer cells) to include:
Studies of a new drug or biological product alone.
Combinations with existing drugs that are part of standard pediatric cancer care (after FDA consultation).
Combinations with the same sponsor's approved adult cancer drug if it targets a molecule relevant to pediatric cancer growth.
Requires these studies to produce useful data on dosing (how much medicine to give), safety, and early effectiveness, using age-appropriate formulations (forms of the drug suitable for children).
Limits combination studies to drugs with a single new active ingredient (the key component that makes the drug work) or novel mixes of previously approved adult cancer ingredients.
Mandates submission of completed preclinical studies (early lab or animal tests) with the initial pediatric study plan.
Clarifies that FDA must decide early (before the study plan is due) which requirements apply, and prevents overlap between different types of pediatric study mandates.
Directs the FDA to issue draft guidance on these changes within 12 months of enactment and finalize it 12 months later.
Applies to new drug or biological product applications submitted at least 3 years after enactment.
Requires reports to Congress:
HHS report (within 6 years) on implementation efforts with industry.
GAO study (starting within 8 years) and report (within 10 years) evaluating the effectiveness of these pediatric assessments in advancing cancer drug development, including benefits and burdens.

Extension of Priority Review Vouchers (Section 3):
Extends the FDA's authority to award "priority review vouchers" (incentives that speed up FDA review of a future drug application to about 6 months instead of 10) for rare pediatric disease treatments until September 30, 2030 (previously set to expire December 20, 2024).
Changes the timing of the user fee (payment to FDA for reviewing the application using the voucher) to be due when the sped-up application is submitted, while other fees follow standard rules.
Requires a GAO study (within 5 years) and report to Congress on the voucher's effectiveness, examining factors like:
Approved drugs' indications (what conditions they treat).
Whether they address unmet needs in rare pediatric diseases (conditions affecting fewer than 200,000 people under age 18 in the U.S.).
Company sizes, voucher values if sold/transferred, usage timelines, and motivations for development.
Impacts on FDA workload, public health priorities, and comparisons to other federal incentives.

Significant Changes to Existing Law

Broadens Pediatric Cancer Study Scope: Under the Federal Food, Drug, and Cosmetic Act (FD&C Act, Section 505B), previously limited pediatric investigations now explicitly include combinations with standard-care or relevant adult-targeted drugs, encouraging adaptation of adult cancer therapies for children without requiring entirely new combinations.
Adds Design and Submission Rules: Introduces specific study design standards for pediatric data, limits on combination applicability, and early submission of preclinical results—changes not previously detailed.
Clarifies Applicability and Avoids Overlap: New rules ensure FDA makes timely determinations and that no application faces duplicate pediatric requirements, streamlining processes.
Extends and Refines Voucher Program: Under FD&C Act Section 529, pushes back the sunset date for rare pediatric disease vouchers by nearly 6 years and simplifies user fee timing to reduce upfront financial barriers for voucher users.
Enhances Oversight: Introduces new GAO and HHS reporting requirements to assess program impacts, which were not as comprehensive before.

Potential Impacts

On Government Agencies: Increases FDA responsibilities for guidance, determinations, study reviews, and implementation coordination with industry; may raise workload for priority reviews but includes reporting to evaluate efficiency. HHS and GAO will need resources for mandated studies and reports.
On Citizens: Primarily benefits children with cancer (especially rare or molecularly targeted types) by potentially leading to more pediatric-specific approvals, faster access to treatments, and better labeling for safe use—addressing gaps where adult drugs are used off-label (not officially approved for that purpose) in kids.
On International Relations: Minimal direct impact, though it could indirectly influence global pharmaceutical development if U.S. incentives attract more international investment in pediatric oncology (cancer treatment).
Broader Effects: May accelerate innovation in pediatric cancer drugs but could impose development costs on companies; overall, aims to reduce the 15-20% lower survival rates for some childhood cancers compared to adults by filling research gaps.

Main Stakeholders Affected

Pediatric Cancer Patients and Families: Direct beneficiaries through expanded research and incentives for new treatments.
Pharmaceutical and Biotechnology Companies: Required to conduct more targeted pediatric studies; gain from extended vouchers that can be redeemed or sold for financial incentives (vouchers have historically sold for millions).
Food and Drug Administration (FDA): Tasked with implementation, guidance, consultations, and reviews—central to enforcing changes.
Federal Government and Congress: HHS oversees coordination; congressional committees (Energy and Commerce, Health, Education, Labor, and Pensions) receive reports for oversight; bipartisan sponsors indicate broad political support.
Healthcare Providers and Researchers: Involved in standard-of-care determinations and study designs, potentially seeing more clinical trial opportunities.

Notable Legal, Constitutional, or Political Implications

Legal Implications: Strengthens the FD&C Act's pediatric provisions (e.g., Pediatric Research Equity Act) by promoting evidence-based pediatric use of targeted therapies, potentially reducing reliance on untested adult extrapolations. Ensures compliance through clear limitations and FDA discretion, minimizing legal challenges over study requirements. The 3-year delayed applicability provides transition time, avoiding retroactive burdens.
Constitutional Implications: None significant; aligns with Congress's authority under the Commerce Clause to regulate drugs affecting interstate commerce and public health, without infringing on individual rights.
Political Implications: Bipartisan introduction (over 20 co-sponsors from both parties) highlights rare consensus on child health issues, potentially setting precedent for extending similar incentives (e.g., tropical disease vouchers). Reporting requirements enable future adjustments based on effectiveness, influencing drug policy debates on incentives versus mandates.

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

Sponsor

Sen. Mullin, Markwayne [R-OK]

Cosponsors (37)

Sen. Bennet, Michael F. [D-CO], Sen. Klobuchar, Amy [D-MN], Sen. Shaheen, Jeanne [D-NH], Sen. Marshall, Roger [R-KS], Sen. Scott, Rick [R-FL], Sen. Collins, Susan M. [R-ME], Sen. Coons, Christopher A. [D-DE], Sen. Capito, Shelley Moore [R-WV], Sen. Kelly, Mark [D-AZ], Sen. Husted, Jon [R-OH], Sen. Kim, Andy [D-NJ], Sen. Sheehy, Tim [R-MT], Sen. Moody, Ashley [R-FL], Sen. Hickenlooper, John W. [D-CO], Sen. Duckworth, Tammy [D-IL], Sen. Boozman, John [R-AR], Sen. Schiff, Adam B. [D-CA], Sen. Kennedy, John [R-LA], Sen. Reed, Jack [D-RI], Sen. Schmitt, Eric [R-MO], Sen. Markey, Edward J. [D-MA], Sen. Booker, Cory A. [D-NJ], Sen. Justice, James C. [R-WV], Sen. Budd, Ted [R-NC], Sen. Gillibrand, Kirsten E. [D-NY], Sen. Slotkin, Elissa [D-MI], Sen. Daines, Steve [R-MT], Sen. Warner, Mark R. [D-VA], Sen. Blumenthal, Richard [D-CT], Sen. Curtis, John R. [R-UT], Sen. Fischer, Deb [R-NE], Sen. Warnock, Raphael G. [D-GA], Sen. Heinrich, Martin [D-NM], Sen. Banks, Jim [R-IN], Sen. McCormick, David [R-PA], Sen. Van Hollen, Chris [D-MD], Sen. Gallego, Ruben [D-AZ]

Recent Actions

2025-12-02: Read twice and referred to the Committee on Health, Education, Labor, and Pensions.
2025-12-02: Introduced in Senate

Bill Versions

Mikaela Naylon Give Kids a Chance Act of 2025 — issued 2025-12-02 — PDF (13 pages)