House approves bill extending FDA's pediatric priority voucher pathway

The US House of Representatives has approved legislation restoring the US Food and Drug Administrations (FDA) rare paediatric disease priority review voucher programme, a development widely welcomed by patient organisations and medical advocates. The measure will next move to the Senate and, if passed, will then be sent to the US President for final approval.

The bipartisan bill, known as the Mikaela Naylon Give Kids a Chance Act, includes regulatory mechanisms designed to accelerate access to treatments for children diagnosed with rare diseases and several forms of cancer.

First introduced in February 2025 by Congressman Michael McCaul, the act merges the previous Give Kids a Chance initiative with the Creating Hope Reauthorisation measure. Its purpose is to stimulate innovation in paediatric rare disease therapies, expand research, and improve survival and treatment outcomes. The bill is named in honour of Mikaela Naylon, a young advocate who championed its passage before passing away earlier this year from osteosarcoma.

Central to the legislation is the reinstatement of the rare paediatric disease priority review voucher pathway. Originally established under the Creating Hope Act, the programme offers an incentive to pharmaceutical companies developing therapies for rare childhood illnesses. When a treatment for a qualifying disease receives approval, the FDA grants a voucher that allows the holder to shorten the review timeline of a different product by four months or sell the voucher to another company. Such vouchers currently hold an estimated market value of around $150 million.

The programme expired in December 2024 due to legislative delays, prompting repeated calls from patient groups and pharmaceutical stakeholders to revive it. Supporters argue that without the voucher incentive, development of medicines for small paediatric populations risks stagnation.

Following the House vote, advocacy organisations expressed strong approval. The Rare Disease Company Coalition stated that reinstating the review voucher pathway represents a crucial advancement for children facing severe, uncommon illnesses and urged the Senate to act swiftly to avoid further delays in treatment access.

The Biotechnology Innovation Organization also praised the outcome, describing the vote as a significant win for families affected by rare conditions and emphasising the importance of restoring the voucher programme as a driver of life-saving medical innovation.

The EveryLife Foundation for Rare Diseases noted that the legislative progress reflects sustained efforts from patients, families, and advocacy networks who pushed lawmakers to prioritise therapies for children without existing treatment options.

Author: Riley Thompson