The House of Representatives has passed H.R. 1262, known as the Mikaela Naylon Give Kids a Chance Act. The bipartisan legislation reauthorizes the Food and Drug Administration’s (FDA) Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) Program, which aims to encourage the development of treatments for rare pediatric diseases. It also grants the FDA authority to require companies to study combinations of cancer drugs and therapies in pediatric clinical trials.
Chairman Brett Guthrie of the House Committee on Energy and Commerce commented on the passage: “H.R. 1262, the Mikaela Naylon Give Kids a Chance Act, builds on current programs to accelerate research and drug development for rare pediatric diseases, including cancer,” said Chairman Guthrie. “The reauthorization of the Rare Pediatric Disease Priority Review Voucher Program has led to over 50 new treatment approvals for nearly 40 different rare pediatric diseases, many of which had no options prior. The impact of this program is profound for patients, and I am grateful to the sponsors of this legislation and their commitment to promoting research and addressing gaps in pediatric therapeutics.”
The legislation includes several provisions aimed at increasing access to innovative treatments for children. It extends the RPD PRV Program through Fiscal Year 2029 and clarifies that orphan drug exclusivity applies specifically to approved indications rather than broader designations. The act gives the FDA additional power to require pediatric cancer trials for new drug combinations.
Other measures in H.R. 1262 allow the FDA to take enforcement action against companies not meeting pediatric study requirements under the Pediatric Research Equity Act (PREA). The bill also directs the FDA to establish an office in an Abraham Accord country to improve collaboration with that region. Additionally, it requires the FDA to inform certain generic drug applicants if any ingredients make a medication quantitatively or qualitatively different from its listed counterpart, with a goal of speeding up patient access to more affordable medications.
