By Fineline Cube The US Congress has introduced the Give Kids a Chance Act, legislation aimed at broadening the Food and Drug Administration’s (FDA) authority over rare pediatric disease research. The bill modifies requirements for molecularly targeted pediatric cancer investigations, extends the Priority Review Voucher (PRV) program until September 30, 2029, and reauthorizes National Institutes of Health (NIH) funding through Fiscal Year 2027.
Key Legislative Provisions
| Provision | Detail |
|---|---|
| Bill Name | Give Kids a Chance Act |
| Target Agency | FDA |
| Pediatric Cancer Research | Permits research on new drugs in combination with approved active ingredients under specified conditions |
| Enforcement Authority | FDA may take action against sponsors failing to comply with pediatric study requirements due to lack of due diligence |
| PRV Program Extension | Extended until September 30, 2029 (originally sunset December 20, 2024) |
| PRV Mechanism | Approval for rare pediatric disease drug qualifies for voucher redeemable for priority review of different product; transferable/sellable |
| NIH Funding | Reauthorized through Fiscal Year 2027 for priority pediatric research |
Market Impact & Strategic Implications
- Pediatric Oncology Pipeline: Combination therapy provision accelerates development of novel regimens for rare pediatric cancers, addressing ~15% of childhood cancers with molecular targets
- PRV Market Value: Extended program maintains $100–150 million voucher market value; incentivizes investment in rare pediatric disease R&D
- Regulatory Compliance: Enhanced enforcement authority ensures sponsors demonstrate due diligence, reducing delays in pediatric study completion
- NIH Funding Stability: FY 2027 reauthorization provides $2–3 billion annual funding certainty for pediatric research infrastructure
- Industry Response: Biotech and pharma trade associations support extension; rare disease advocacy groups praise combination therapy flexibility
Forward‑Looking Statements
This brief contains forward‑looking statements regarding legislative implementation timelines, regulatory enforcement patterns, and market impacts of the Give Kids a Chance Act. Actual outcomes may differ due to congressional approval processes, FDA rulemaking, and industry adaptation.-Fineline Info & Tech