Read twice and referred to the Committee on Health, Education, Labor, and Pensions.
This bill amends the Federal Food, Drug, and Cosmetic Act to clarify and expand FDA requirements for molecularly targeted pediatric cancer investigations, including when pediatric studies apply to single-agent drugs or certain combinations. It requires study designs to produce clinically meaningful pediatric data with age-appropriate formulations, allows FDA to request relevant preclinical study results, and mandates FDA guidance and delayed applicability.
Liberals emphasize pediatric data and age-appropriate formulations.
Relative to its intended legislative type, this bill is a substantive statutory amendment to the FD&C Act that is reasonably well-constructed: it identifies targeted changes, integrates them into existing law, prescribes study-design expectations, sets implementation timelines, and embeds accountability through guidance and GAO/HHS reporting.
This bill amends the Federal Food, Drug, and Cosmetic Act to clarify and expand FDA requirements for molecularly targeted pediatric cancer investigations, including when pediatric studies apply to single-agent drugs or certain combinations.
It requires study designs to produce clinically meaningful pediatric data with age-appropriate formulations, allows FDA to request relevant preclinical study results, and mandates FDA guidance and delayed applicability.
The bill also extends the rare pediatric disease priority review voucher (PRV) authority through September 30, 2029, changes when the priority review user fee is paid, and orders GAO and HHS reports on the effectiveness and impacts of these programs.
Technical, child-focused bill with compromise features improves prospects, but regulatory burdens, industry reactions, and legislative scheduling reduce probability.
Relative to its intended legislative type, this bill is a substantive statutory amendment to the FD&C Act that is reasonably well-constructed: it identifies targeted changes, integrates them into existing law, prescribes study-design expectations, sets implementation timelines, and embeds accountability through guidance and GAO/HHS reporting. It also contains secondary elements that establish reporting/study requirements and administrative actions for the FDA.
Liberals emphasize pediatric data and age-appropriate formulations.
These are examples from the analysis, not a ranked list of the most-affected groups.
Liberals emphasize pediatric data and age-appropriate formulations.
Generally supportive because the bill strengthens requirements for pediatric cancer evidence and emphasizes age-appropriate formulations.
Concerned about whether incentives like PRVs will translate into affordable access for children and equitable distribution of new therapies.
Cautiously favorable: clarifies regulatory expectations, preserves incentives, and requires oversight studies.
Wants assurance the FDA can manage added responsibilities without slowing approvals or imposing undue industry costs.
Mixed to skeptical: supports accelerating pediatric therapies and use of market incentives, but worries about added FDA authorities and mandates increasing regulatory burden.
Prefers incentives over prescriptive requirements and worries about costs and federal overreach.
Reached or meaningfully advanced
Reached or meaningfully advanced
Still ahead
Still ahead
Still ahead
Technical, child-focused bill with compromise features improves prospects, but regulatory burdens, industry reactions, and legislative scheduling reduce probability.
The bill has not accumulated any surfaced votes yet.
Liberals emphasize pediatric data and age-appropriate formulations.
Technical, child-focused bill with compromise features improves prospects, but regulatory burdens, industry reactions, and legislative sche…
Relative to its intended legislative type, this bill is a substantive statutory amendment to the FD&C Act that is reasonably well-constructed: it identifies targeted changes, integrates them into existing law, prescribe…
Go beyond the headline summary with full stakeholder mapping, legislative design analysis, passage barriers, and lens-by-lens tradeoff breakdowns.