To amend the Federal Food, Drug, and Cosmetic Act with respect to molecularly targeted pediatric cancer investigations, and for other purposes.
Sponsors
Legislative Progress
Passed HouseAdditional sponsors: Mr. Wittman, Mr. Allen, Mr. Zinke, Mr. Evans …
Reported with an amendment, committed to the Committee of the …
Passed House (inferred from eh version)
Mr. McCaul (for himself, Mr. Bilirakis, Mrs. Dingell, Ms. Schrier, …
Summary
What This Bill Does
The Give Kids a Chance Act of 2025 strengthens requirements for pharmaceutical companies to study how cancer drugs work in children, extends a program that rewards companies for developing treatments for rare childhood diseases, and narrows a loophole that allows drug companies to block competition for certain rare disease medicines. It also modernizes the organ transplant network with better technology and creates an FDA office to work with Middle Eastern countries that signed the Abraham Accords.
Who Benefits and How
Children with cancer and rare diseases benefit most, as drug companies will be required to study whether adult cancer treatments also work for pediatric patients, and incentives for rare disease drug development are extended through 2029. Generic drug manufacturers gain because the bill narrows orphan drug exclusivity to specific uses rather than entire diseases, making it easier to develop competing treatments. Companies in Israel, UAE, Bahrain, Morocco, and Sudan benefit from easier access to FDA regulatory guidance through the new Abraham Accords Office.
Who Bears the Burden and How
Brand-name pharmaceutical companies face new compliance costs: they must now conduct pediatric cancer studies for molecularly targeted drugs, and can face FDA penalties for failing to complete required pediatric studies. Companies with orphan drug exclusivity lose some market protection as competitors can now develop treatments for different uses within the same rare disease. Organ transplant hospitals and procurement organizations must invest in electronic health record integration. Taxpayers fund an additional 1.2 billion dollars for the Medicare Improvement Fund and 75 million for NIH pediatric drug research.
Key Provisions
- Expands pediatric cancer drug testing requirements to include combination therapies with standard-of-care drugs
- Extends the rare pediatric disease priority review voucher program from December 2024 to September 2029
- Narrows orphan drug 7-year market exclusivity from entire diseases to specific approved uses, allowing more competition
- Authorizes FDA penalties for drug sponsors who fail to complete required pediatric studies with due diligence
- Requires organ transplant networks to integrate electronic health records and publish transparency dashboards
- Creates an Abraham Accords Office within FDA to facilitate regulatory cooperation with signatory countries
- Increases Medicare Improvement Fund by 1.24 billion dollars and authorizes 25 million per year for NIH pediatric studies
Evidence Chain:
This summary is derived from the structured analysis below. See "Detailed Analysis" for per-title beneficiaries/burden bearers with clause-level evidence links.
Primary Purpose
To enhance pediatric drug development requirements, extend incentives for rare pediatric disease treatments, modify orphan drug exclusivity, improve organ transplant networks, and establish an FDA Abraham Accords Office for international regulatory cooperation.
Policy Domains
Legislative Strategy
"Expand pediatric drug development requirements, provide regulatory incentives for rare disease treatments, facilitate generic drug competition in orphan drug markets, modernize organ transplant infrastructure, and establish international regulatory partnerships."
Likely Beneficiaries
- Children with cancer and rare diseases (more treatment options)
- Pharmaceutical companies developing pediatric cancer drugs (clearer pathway, priority vouchers)
- Generic drug manufacturers (clearer bioequivalence guidance, narrower orphan exclusivity)
- Organ transplant patients (improved system efficiency)
- Abraham Accords countries' pharmaceutical industries (FDA cooperation)
- Medicare beneficiaries (increased improvement fund)
Likely Burden Bearers
- Brand-name pharmaceutical companies (narrower orphan drug exclusivity)
- Drug sponsors with pediatric study requirements (enforcement now possible under Section 303)
- FDA (new reporting requirements, new Abraham Accords Office to staff)
- Taxpayers (appropriations for pediatric studies program, Medicare fund increase)
Bill Structure & Actor Mappings
Who is "The Secretary" in each section?
- "the_secretary"
- → Secretary of Health and Human Services (acting through FDA Commissioner)
- "the_secretary"
- → Secretary of Health and Human Services
- "the_secretary"
- → Secretary of Health and Human Services
- "comptroller_general"
- → Comptroller General of the United States
- "the_secretary"
- → Secretary of Health and Human Services
- "the_director"
- → Director of the Abraham Accords Office
- "the_secretary"
- → Secretary of Health and Human Services
- "the_commissioner"
- → Commissioner of Food and Drugs
- "the_secretary"
- → Secretary of Health and Human Services (acting through FDA)
Note: 'The Secretary' consistently refers to the Secretary of Health and Human Services throughout this bill, often acting through the Commissioner of Food and Drugs (FDA Commissioner).
Key Definitions
Terms defined in this bill
As defined in section 529 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ff).
A country identified by the Department of State as having signed the Abraham Accords Declaration.
An investigation of a drug or biological product (alone or in combination) that is directed at a molecular target substantially relevant to the growth or progression of a pediatric cancer, designed to yield clinically meaningful pediatric study data regarding dosing, safety, and preliminary efficacy.
The use or indication approved under section 505 of the FFDCA or licensed under section 351 of the Public Health Service Act for a drug designated under section 526 for a rare disease or condition.
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