After a lengthy period in regulatory limbo, the US Food and Drug Administration’s (FDA) rare paediatric disease priority review voucher (PRV) programme has returned to the pharma landscape, marking a win for biopharma companies developing therapies for rare diseases in children.
On 3 February, President Donald Trump signed a spending bill that will fund several federal agencies for the remainder of 2026. Included in the bill was the rare paediatric disease PRV programme, which has been inactive since it expired in December 2024.
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The programme’s return is being authorised under the Mikaela Naylon Give Kids a Chance Act, a bill that covers a range of regulatory frameworks to increase the speed at which children battling rare diseases and certain cancers can access therapies.
Jeremiah Kelly, partner at law firm Venable, told Pharmaceutical Technology: “The reauthorisation of the pediatric priority review voucher program will incentivise drug and biologics development for diseases that, otherwise due to low prevalence, lack the return-on-investment in the market to compel companies to undertake the costly risks associated with that development. Thankfully, Congress has reauthorised this program, which should strengthen the pipeline of products on the way to help some of our most vulnerable children – this program shifts the balance more in favor of innovation in the pediatric drug development space.”
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 Reauthorization 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 Act has received bipartisan support during its journey from Congress to Trump’s signature.
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By GlobalDataProgramme’s return welcomed
The rare paediatric disease PRV programme expired over a year ago due to political and congressional delays. Patient advocacy groups and pharma industry bodies continually called for its reauthorisation, given the framework’s importance in bringing innovative therapies to market. Many of these groups have now welcomed the programme’s return to the US pharma scene.
The Rare Disease Company Coalition said in a statement: “[We] are pleased to celebrate the passage of the Mikaela Naylon Give Kids a Chance Act, which reauthorises the lifesaving paediatric rare disease PRV programme.”
“This milestone brings renewed hope to families awaiting lifesaving treatments for children with rare diseases and reaffirms a bipartisan commitment to advancing innovation for paediatric patients who urgently need therapeutic breakthroughs. The PRV program has already helped enable therapies across 47 rare paediatric indications,” added the coalition, of which BioMarin and Sarepta Therapeutics are members.
It is currently unclear when the FDA will resume reviewing requests for designation and if there will be any amendments to the scheme’s regulatory process.
According to the National Organization for Rare Disorders (NORD), 63 PRVs have been awarded across 47 rare paediatric diseases since the programme’s inception. Prior to its creation, only four of these 47 rare paediatric diseases, many of which lead to death before the patient reaches adulthood, had a treatment approved by the FDA.
“The reauthorisation of the PRV programme and the accompanying bills will speed up innovation and access, ensuring that more patients have a chance at a life not limited by rare disease,” the NORD said in a statement.
Rare disease incentive back on the agenda
Companies that receive FDA approval for a drug or biological product for a rare paediatric disease can qualify for a voucher that can be redeemed to receive priority review for a different product. The voucher slashes the FDA review time for a new drug application from the standard 10 months to 6 months, allowing pharma companies to recoup R&D expenses in a more lucrative market.
Companies are also able to sell vouchers for cash. Since the PRV framework was installed in 2014, a secondary market of vouchers has established itself. In January 2026, Jazz Pharmaceuticals announced it had sold a voucher for $200m. Other sales in 2025 hovered around the $150m price point.
The incentive means more rare diseases are pursued as part of pharma pipelines.
SynaptixBio, a biotech developing rare disease drugs, currently holds two rare paediatric disease designations. The company’s CEO, Dan Williams, says the bill’s passage is significant for smaller biotechs.
Williams stated: “The PRV programme means smaller biotechs can see a way to financial security, having typically had to find investment during the long years of drug design and development.
“The very high cost of preparing for, and conducting, clinical trials adds to the financial challenge, and all the risk remains until a candidate drug is approved for the market… big pharma doesn’t face the high investment risk of developing a rare disease drug, and the smaller biotech has a natural target for selling its PRV.”
In June 2025, the FDA rolled out a pilot scheme for a new type of priority voucher. This new framework, called the Commissioner’s National Priority Voucher (CNPV) programme, is available to companies developing a drug with US national interests as a primary focus. While rare diseases can fulfil the criteria, they are not the exclusive target of the initiative. Vouchers awarded under the scheme will shorten FDA review time for a new drug application from 10-12 months to up to two months.
