Mikaela Naylon Give Kids a Chance Act of 2025
Analysis under review: This bill has generated analysis that may be too generic or incomplete. Clause-level evidence remains available below.
Summary
What This Bill Does
The Mikaela Naylon Give Kids a Chance Act of 2025 aims to accelerate the development of cancer treatments for children. It does this by requiring pharmaceutical companies to study whether their molecularly targeted cancer drugs could also work for pediatric cancers, and by extending a valuable incentive program that rewards companies for developing treatments for rare childhood diseases.
Who Benefits and How
- Children with cancer and rare pediatric diseases gain access to potentially life-saving treatments that might not otherwise be developed for their small patient populations
- Pharmaceutical and biotech companies benefit significantly from the 6-year extension of the priority review voucher program, which awards vouchers worth $100+ million that can be sold or used to fast-track FDA approval of other drugs
- Clinical trial organizations and researchers in pediatric oncology see increased business opportunities as more pediatric cancer studies are required
Who Bears the Burden and How
- Pharmaceutical companies developing new molecularly targeted cancer drugs must conduct additional pediatric cancer studies, adding time and cost (potentially millions per study) to their drug development process
- The FDA faces increased workload for reviewing pediatric study plans and processing priority review vouchers
- The Government Accountability Office must conduct comprehensive studies evaluating the effectiveness of both programs within 5-10 years
Key Provisions
- Expands the definition of "molecularly targeted pediatric cancer investigation" to include combination therapies with approved drugs that target molecular pathways relevant to pediatric cancers
- Extends the rare pediatric disease priority review voucher program from December 2024 to September 2030
- Requires the FDA to issue implementation guidance within 12 months of enactment
- Mandates GAO studies to evaluate whether these programs effectively improve pediatric cancer drug development
- Clarifies that drug applications must be subject to either the pediatric cancer study requirement OR the general pediatric study requirement, but not both
Evidence Chain:
This summary is generated from the full bill text using AI analysis. Expand "Detailed Analysis" below for identified beneficiaries/burden bearers.
At a Glance
What This Bill Does
Amends the Federal Food, Drug, and Cosmetic Act to improve pediatric cancer drug development by expanding requirements for molecularly targeted pediatric cancer investigations and extending the rare pediatric disease priority review voucher program through September 2030.
Who Benefits
- Children with cancer who may gain access to new treatments
- Pharmaceutical companies developing pediatric cancer drugs (receive priority review vouchers worth hundreds of millions of dollars)
- Drug manufacturers with adult cancer drugs that target molecular pathways relevant to pediatric cancers
Who Bears Costs
- Pharmaceutical companies submitting new drug applications (must conduct additional pediatric studies)
- FDA (increased workload for pediatric study reviews and priority review voucher processing)
- Taxpayers (indirect costs through FDA operations)
Key Policy Areas
Healthcare, Pharmaceuticals, Pediatric Medicine, Drug Regulation
Primary Purpose
Amends the Federal Food, Drug, and Cosmetic Act to improve pediatric cancer drug development by expanding requirements for molecularly targeted pediatric cancer investigations and extending the rare pediatric disease priority review voucher program through September 2030.
Policy Domains
Legislative Strategy
"Incentivize pharmaceutical companies to develop pediatric cancer treatments by requiring pediatric studies for molecularly targeted cancer drugs and extending the valuable priority review voucher program that rewards rare pediatric disease drug development."
Identified Gains
- Children with cancer who may gain access to new treatments
- Pharmaceutical companies developing pediatric cancer drugs (receive priority review vouchers worth hundreds of millions of dollars)
- Drug manufacturers with adult cancer drugs that target molecular pathways relevant to pediatric cancers
Identified Costs
- Pharmaceutical companies submitting new drug applications (must conduct additional pediatric studies)
- FDA (increased workload for pediatric study reviews and priority review voucher processing)
- Taxpayers (indirect costs through FDA operations)
Sponsors
Legislative Progress
In CommitteeMr. Mullin (for himself, Mr. Bennet, Ms. Klobuchar, Mrs. Shaheen, …
Read twice and referred to the Committee on Health, Education, …
Introduced in Senate
Stakeholder Effects
cui bono?How this legislation distributes effects. Mention counts reflect frequency, not effect magnitude.
Large pharmaceutical companies seeking priority FDA review, Pharmaceutical companies developing drugs for rare pediatric diseases, Pharmaceutical companies developing molecularly targeted cancer drugs
Positive-direction: Large pharmaceutical companies seeking priority FDA review, Pharmaceutical companies developing drugs for rare pediatric diseases
Negative-direction: Pharmaceutical companies developing molecularly targeted cancer drugs, Pharmaceutical companies with approved adult cancer drugs targeting molecular pathways relevant to pediatric cancer
Biotech firms developing combination cancer therapies, Small and mid-sized biotech companies focused on rare diseases
Positive-direction: Small and mid-sized biotech companies focused on rare diseases
Negative-direction: Biotech firms developing combination cancer therapies
Children with rare pediatric diseases, Pediatric cancer patients and families
FDA Center for Drug Evaluation and Research, FDA priority review processing
Pediatric oncology clinical trial organizations
Bill Structure & Actor Mappings
Who is "The Secretary" in each section?
- "the_secretary"
- → Secretary of Health and Human Services (acting through the Commissioner of Food and Drugs)
- "the_comptroller_general"
- → Comptroller General of the United States
- "the_secretary"
- → Secretary of Health and Human Services
- "the_comptroller_general"
- → Comptroller General of the United States
Key Definitions
Terms defined in this bill
A voucher awarded under section 529 of the Federal Food, Drug, and Cosmetic Act for drugs treating rare pediatric diseases, which can be used to obtain priority FDA review for any subsequent drug application.
An investigation of a drug or biological product that is directed at a molecular target the Secretary determines to be substantially relevant to the growth or progression of a pediatric cancer, or a drug used in combination with an active ingredient that is part of the standard of care for treating a pediatric cancer.
We use a combination of our own taxonomy and classification in addition to large language models to assess meaning and potential beneficiaries. High confidence means strong textual evidence. Always verify with the original bill text.
Learn more about our methodology