The US House of Representatives has passed a new piece of legislation that reauthorises the US Food and Drug Administration’s (FDA) rare paediatric disease (RPD) priority review voucher (PRV) programme in a move welcomed by industry bodies and charities.
The Mikaela Naylon Give Kids a Chance Act, which received bipartisan support, includes a range of regulatory frameworks to increase the speed at which children battling rare diseases and certain cancers can access therapies.
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The bill will now move to the Senate, and if passed there, it will be handed off to US President Donald Trump for approval.
First put forward by Republican congressman Michael McCaul in February 2025, the Mikaela Naylon Give Kids a Chance Act combines two initiatives – the original Give Kids a Chance Act and the Creating Hope Reauthorisation Act. The aim of the legislation is to spur drug development for paediatric rare diseases, improve outcomes for patients, and close gaps in paediatric drug research.
The bill is named after Mikaela Naylon, a young woman who advocated for its passage. Naylon died this year after complications from osteosarcoma, a rare type of bone cancer.
McCaul said: “I’ve met with countless cancer patients and advocates who have asked me for one thing: to give kids their best chance of beating cancer. Today, the House of Representatives shined a beacon of hope for those children and their families by unanimously passing the Mikaela Naylon Give Kids A Chance Act.”
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By GlobalDataImpacts of the proposed Act
Chief among the proposed Act’s effects is the reauthorisation of the RPD PRV programme. This pathway, originally created under McCaul’s Creating Hope Act, incentivises pharma companies to develop drugs for rare paediatric diseases.
Given that the indication might not be profitable for a company, the FDA rewards approval in a disease with a priority voucher. This voucher can either be used to slash four months off review time for another product or be sold for cash. Currently, these vouchers go for around $150m. For example, Bavarian Nordic secured $160m from selling one in June this year.
However, the programme expired in December 2024 due to political and congressional delays. Patient advocacy groups and pharma industry bodies have been calling for its reuthorisation, given the framework’s importance in brining innovative therapies to market. The Mikaela Naylon Give Kids a Chance Act’s passage through the House has been welcomed by the aforementioned groups.
The Rare Disease Company Coalition’s executive director Stacey Frisk “Today’s House passage of the Mikaela Naylon Give Kids a Chance Act is a massive step forward for the rare disease community.
“We applaud every member who voted for this bill and thank the sponsors for their dedication to advancing solutions for children who cannot wait. With House approval secured, we are urging the Senate to prioritise this legislation and ensure the PRV programme is reinstated as quickly as possible. Children living with rare, devastating diseases need action, not uncertainty.”
In a statement, the Biotechnology Innovation Organization (BIO) commented: “The unanimous House passage of the Mikaela Naylon Give Kids a Chance Act is a victory for children and families living with rare diseases. We applaud the House for their commitment to this life-saving measure, and to a key part of the bill – the reauthorisation of the Pediatric Priority Review Voucher programme.”
EveryLife Foundation for Rare Diseases, a US nonprofit, said: “Today’s milestone comes as a result of community-driven advocacy and dedication from rare disease patients, families, organisations, and others who shared their stories and used their voices to ensure Congress acted.”
