Give Kids a Chance: How Congress Moved to Restart Pediatric Drug R&D Incentives
After the FDA’s Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) authority lapsed at the end of 2024, patient groups and drug developers warned that one of the few “market-shaping” incentives for ultra-small pediatric populations had gone dark—right when pediatric oncology and rare disease pipelines were starting to mature.
That set the stage for H.R. 1262, the “Mikaela Naylon Give Kids a Chance Act.” The bill advanced through the House Energy & Commerce process earlier in the fall, then came to the floor under suspension of the rules - a procedural fast track typically reserved for broadly supported, non-controversial measures. On December 1, 2025, the House passed the bill by voice vote, sending it to the Senate the next day.
In floor messaging and committee framing, supporters described the act as a package designed to do two things at once: (1) restore an investment signal for rare pediatric therapies, and (2) tighten/clarify FDA authorities so pediatric studies - especially for modern combination regimens in cancer - don’t lag behind adult development.
What It Does To Incentivize R&D?
1. Reauthorizes the Rare Pediatric Disease Priority Review Voucher program (through Sept. 30, 2029)
The centerpiece R&D incentive is the bill’s extension of FDA’s ability to award rare pediatric disease PRVs through September 30, 2029.
Why that matters for R&D: a PRV is effectively a high-value asset. When a sponsor wins approval for a qualifying rare pediatric disease product, FDA can award a voucher that can be used (or sold) to obtain priority review for another drug application - pulling review timelines forward and potentially moving revenue earlier. FDA describes the RPD PRV program under FD&C Act Section 529 and how vouchers are awarded under the statutory criteria.
Historically, PRVs have been sold for substantial amounts; GAO documented sales (FY2014–FY2019) ranging from about $67.5M to $350M, illustrating why the incentive can change capital-raising and portfolio decisions.
2. Codifies FDA’s interpretation of orphan drug exclusivity (reducing regulatory uncertainty)
The act also gives statutory footing to FDA’s long-held view that orphan drug exclusivity attaches to the approved indication/use, not necessarily every use within a broader orphan “designation.”
For R&D, this is about predictability: clearer exclusivity boundaries can affect whether companies pursue pediatric label expansions, follow-on indications, or lifecycle strategies.
3. Expands FDA Leverage To Get Pediatric Studies Done
The bill:
- permits/encourages pediatric cancer investigations for targeted drugs in combination with already-approved active ingredients (under specified conditions), and
- authorizes enforcement action when sponsors fail to meet pediatric study obligations under the Pediatric Research Equity Act (PREA) and lack due diligence.
This is less about a “coupon” incentive and more about reducing the structural lag that often leaves pediatric patients waiting years behind adult oncology practice - especially as cancer care shifts toward rational combinations.
4. Reauthorizes certain NIH pediatric research funding (through FY2027)
The Congress.gov summary also states the bill reauthorizes certain NIH funding for priority pediatric research through FY2027 - a direct federal R&D support element alongside the PRV’s market-based incentive.
The next steps are now Senate-driven: it can take up the House-passed bill, amend it, or move a companion. A Senate companion exists (S. 932), which likewise extends the PRV program through FY2029.
If enacted, FDA would again have clear authority to award RPD PRVs through Sept. 30, 2029, and sponsors would likely restart planning around designation requests, filing strategies, and voucher monetization/usage. Separately, the pediatric study/enforcement and combination-trial provisions would move into FDA guidance/operations over time, with oversight mechanisms (including studies/reports) shaping how aggressively the agency uses the tools Congress is giving it.
