The Give Kids a Chance Act of 2025 amends section 505B of the Federal Food, Drug, and Cosmetic Act to define and constrain when the FDA can require "molecularly targeted pediatric cancer investigations." It permits the agency to require pediatric investigations of a sponsor’s drug alone or of that drug in combination with another active ingredient only when the combination meets specific criteria tied to prior approvals and clinical relevance, and it sets minimum design expectations for those pediatric studies.
The bill also phases in the changes (applicable to applications submitted three years after enactment), directs HHS to issue guidance on implementation, requires congressional reporting and multi-year GAO studies of outcomes, and extends the rare pediatric disease priority review voucher (PRV) program through September 30, 2029 while shifting the priority‑review user fee to be due at submission. These changes alter developers’ trial planning, FDA’s review duties, and the incentive landscape for rare pediatric drug development.
At a Glance
What It Does
The bill rewrites how section 505B applies to molecularly targeted pediatric cancer work: it defines eligible combination partners, limits when combinations can trigger pediatric investigations, requires clinically meaningful pediatric study design (age‑appropriate formulations, dosing, safety, preliminary efficacy), and permits FDA to request completed preclinical study results with the initial pediatric study plan. It also extends the rare pediatric disease PRV authority to Sept 30, 2029 and requires the PRV user fee to be paid upon submission of the covered application.
Who It Affects
Drug and biologic applicants filing NDAs or BLAs (including sponsors pursuing combination regimens that pair new active ingredients with adult‑approved oncology agents), pediatric oncology trial sites and CROs that run pediatric studies, FDA review divisions responsible for pediatric and oncology assessments, and companies that obtain or use rare‑pediatric PRVs.
Why It Matters
The bill narrows regulatory uncertainty about when FDA may compel pediatric investigations for molecularly targeted agents and combinations, but it also creates new procedural steps and document submissions that affect development timelines and costs. Extending and tweaking PRV mechanics keeps an existing market incentive in place and shifts the payment timing for that expedited pathway.
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What This Bill Actually Does
At its core, the bill reorganizes and tightens the pediatric study obligations in current section 505B. It introduces the phrase "molecularly targeted pediatric cancer investigation" and then specifies two pathways under which FDA can require such an investigation: (1) for the application drug itself, or (2) for the application drug when used in combination with another active ingredient that meets one of two narrowly drawn conditions.
One condition covers combinations where the partner active ingredient is already in a product approved through abbreviated or biosimilar pathways (505(j) or 351(k)) and that partner is considered part of the standard of care for a pediatric cancer. The other permits combination requirements where the partner is in a product approved for an adult cancer (505(b) or 351(a)) and is held by the same sponsor, provided the partner targets a molecular mechanism the FDA finds substantially relevant to the pediatric cancer.
The bill sets concrete expectations for the pediatric investigations it allows FDA to require: studies must produce clinically meaningful pediatric data on dosing, safety, and preliminary efficacy and use formulations appropriate to each pediatric age group the study targets. It limits FDA’s authority to compel investigations of novel multi‑ingredient products: combination investigations may be required only when the application adds a single new active ingredient or when multiple components are combined in an application and each component was previously approved for adult cancer but the combination itself has not been approved.
The bill also explicitly clarifies that inactive ingredients are outside this combination analysis.Procedurally, the bill requires FDA to decide—before the applicant must submit the initial pediatric study plan—which of the statutory paths applies, preventing double‑coverage under both subparagraphs. HHS must publish draft guidance within 12 months and finalize it within 12 months after the comment period closes.
The statutory changes apply to NDAs/BLAs filed three years after enactment, giving sponsors a transition window. The bill also mandates reporting: HHS must report to relevant committees within six years on implementation, and the Government Accountability Office must study the policy’s effects on pediatric cancer drug development and report findings to Congress on a multi‑year timetable.Separately, the bill extends the rare pediatric disease priority review voucher program to September 30, 2029, and amends the PRV user‑fee timing so the priority review fee is due upon submission of the application that uses the voucher.
The bill directs GAO to analyze whether awarding PRVs has effectively spurred development and expedited access for rare pediatric disease therapies and to report its findings to Congress within the statutory timeframes laid out in the bill.
The Five Things You Need to Know
The bill amends FD&C Act section 505B to create a defined category, 'molecularly targeted pediatric cancer investigation,' and to specify when FDA can require such investigations for an applicant’s drug alone or when combined with another active ingredient.
FDA may require pediatric combination investigations only if the added active ingredient is either (A) in a product approved under 505(j) or 351(k) and judged part of pediatric standard of care, or (B) in an adult‑cancer product approved under 505(b) or 351(a) held by the same sponsor and directed at a molecular target the agency finds substantially relevant to the pediatric cancer.
Required pediatric investigations must be designed to produce clinically meaningful pediatric data—age‑appropriate formulations and information on dosing, safety, and preliminary efficacy sufficient to inform potential pediatric labeling—and FDA may request completed preclinical study results with the initial pediatric study plan.
The statutory changes do not take effect immediately: they apply only to NDAs/BLAs submitted three years after enactment; HHS must issue draft guidance within 12 months and finalize it within 12 months after the comment period closes, and HHS and GAO must report to Congress on implementation and outcomes on multi‑year schedules.
Section 3 extends the rare pediatric disease PRV program through September 30, 2029, changes the PRV priority‑review user fee to be due at application submission, and directs GAO to study whether PRVs meaningfully incentivize rare pediatric drug development.
Section-by-Section Breakdown
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Defines molecularly targeted pediatric cancer investigations and allowed combination partners
This provision redesignates existing subparagraphs and inserts a new structure that spells out when FDA may require molecularly targeted pediatric cancer studies. It enumerates two alternative bases for requiring combination investigations: (i) the partner active ingredient is in a product approved under 505(j) or 351(k) and is part of pediatric standard of care; or (ii) the partner is in an adult‑cancer product approved under 505(b) or 351(a) and owned by the same sponsor and targets a molecular mechanism the Secretary deems substantially relevant to the pediatric cancer. The change narrows agency discretion by tying combination obligations to prior approval status and to either standard‑of‑care use in pediatrics or sponsor ownership plus target relevance.
Study design and limits on when combination investigations may be required
The bill requires pediatric investigations to be designed to yield clinically meaningful pediatric study data: appropriate formulations for each age group and data on dosing, safety, and preliminary efficacy that could inform labeling. It also constrains requirements for novel combinations: FDA can only compel an investigation of a combination if the application contains a single new active ingredient or if multiple active ingredients are combined where the combination has not been previously approved but each active ingredient was previously approved to treat an adult cancer. This reduces the risk FDA will impose broad combination study obligations for complex, multi‑ingredient filings.
Pre‑submission determinations and preclinical data timing
The bill requires the agency to determine whether the applicant falls under subparagraph (A) or (B) before the date the applicant must submit the initial pediatric study plan, removing later surprises about which statutory path applies. It also permits FDA to request that the results of already‑completed preclinical studies relevant to the initial pediatric study plan be submitted at the time that plan is filed, front‑loading documentation sponsors may need to assemble.
Mutually exclusive application paths and inactive ingredient carve‑out
A clean rule of construction states that an application covered by the requirements of one subparagraph is not simultaneously subject to the other, preventing overlapping obligations. The amendment also clarifies that the combination analysis applies to active ingredients and does not extend to inactive ingredients, which narrows the set of combinations that can trigger additional pediatric obligations.
Guidance deadline, three‑year phase‑in, HHS and GAO reporting
HHS must publish draft guidance on implementing these amendments within 12 months of enactment and finalize it within 12 months after the comment period closes. The statutory changes apply to applications submitted three years after enactment, providing a transition window. HHS must report to congressional health committees within six years on implementation, and GAO is directed to study effectiveness of the new §505B requirements and report findings to Congress on the multi‑year schedule set by the bill.
Extends rare pediatric disease PRV program and studies PRV effectiveness
Section 3 amends the PRV statute to extend authority to award rare pediatric disease priority review vouchers through September 30, 2029. It changes the priority‑review user fee to be payable when a sponsor submits the application that uses a voucher (rather than another timing mechanism). The section also directs GAO to conduct a study of the PRV program’s effectiveness—including who receives vouchers, company sizes, transfer values, timelines between award and use, and impacts on FDA workload—and to report findings to Congress within the statutory timeframes.
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Explore Healthcare in Codify Search →Who Benefits and Who Bears the Cost
Every bill creates winners and losers. Here's who stands to gain and who bears the cost.
Who Benefits
- Children with certain cancers: by tying pediatric investigations to molecular relevance and requiring age‑appropriate formulations and dosing data, the bill aims to generate more actionable pediatric safety and dosing information that can support labeling and appropriate pediatric use.
- Pediatric oncology investigators and trial sites: clearer statutory criteria and a requirement that FDA declare which path applies before the initial pediatric study plan reduce regulatory uncertainty when negotiating study scope and enrollment strategies.
- Sponsors holding adult‑approved oncology products: companies that already hold 505(b)/351(a) approvals for adult cancers and that pursue pediatric‑relevant targets may find it easier to be required (or to justify) combination studies when they control the partner product, aligning development programs across indications.
- Recipients and buyers of PRVs: companies that earn rare pediatric disease PRVs retain a time‑limited marketable asset (now available through 2029) and can plan transfers or use with a clearer fee timing rule.
- Policymakers and Congress: the mandated HHS and GAO reports create an evidence base to evaluate whether these regulatory changes improve pediatric oncology development and to inform future legislative fixes.
Who Bears the Cost
- Drug and biologic sponsors (especially smaller firms): new or clarified pediatric investigation obligations, front‑loaded requests for preclinical data, and potential combination trials increase development costs and planning complexity.
- FDA (Center for Drug Evaluation and Research): issuing guidance, making determinations about which statutory path applies, reviewing potentially more pediatric data, and handling PRV timing and transfers increase administrative and review workload.
- Clinical trial networks and CROs: conducting studies that meet the bill’s 'clinically meaningful' standard—age‑appropriate formulations and pediatric dosing/safety—will require specialized protocols and perhaps additional resources to recruit rare pediatric populations.
- Health systems and payers: if the bill prompts more pediatric labeling changes or new combination regimens, hospitals and insurers may face decisions about coverage and formulary inclusion for niche, potentially high‑cost pediatric treatments.
- Sponsors relying on complex multi‑ingredient products: where combinations include new active ingredients or non‑sponsor owned partners, the bill’s limits and evidentiary expectations could delay or complicate development strategies.
Key Issues
The Core Tension
The central dilemma is balancing two legitimate goals: require and incentivize the right pediatric evidence for molecularly targeted cancers so children get safer, labeled treatments, while avoiding rules and costs that make developers less willing to invest in pediatric oncology or that force burdensome, low‑value studies in fragile patient populations. The statute narrows agency discretion but leaves key definitional and procedural choices to FDA, so implementation will determine whether the balance favors actionable pediatric evidence or greater regulatory friction for drug developers.
The bill attempts to strike a balance between encouraging targeted pediatric oncology research and limiting overbroad agency demands, but it leaves several important implementation questions unresolved. The phrase 'substantially relevant to the growth or progression of a pediatric cancer' vests substantial discretion in the Secretary; how FDA operationalizes that judgment—what evidence counts, how molecular relevance is measured across histologies and ages—will determine how often combination obligations attach.
Requiring FDA to decide which statutory path applies before the initial pediatric study plan helps predictability, but the statute does not specify a strict timeline or a dispute resolution process if the sponsor disagrees with the agency’s determination.
The three‑year phase‑in gives sponsors runway to adjust development plans, yet it also introduces strategic timing considerations: companies may rush filings to fall before the effective date or delay to avoid new obligations. The bill also overlaps with existing pediatric statutes and incentives (PREA, BPCA, existing pediatric regulations, and orphan/PRV incentives); it does not harmonize conflicts such as when PREA pediatric requirements diverge from the molecularly targeted approach or how exclusivity and voucher economics interact.
Finally, extending PRVs through 2029 preserves a market incentive, but the bill’s GAO study mandate acknowledges concerns that vouchers may not always produce meaningful unmet‑need gains and that they impose review prioritization costs on FDA.
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