The US Food and Drug Administration (FDA) is seeking to permanently authorise the rare pediatric disease priority review voucher (PRV) programme, putting an end to cycles of expirations and renewals by Congress.
The proposal was outlined by FDA commissioner Marty Makary as part of the agency’s $7.2bn budget proposal to Congress for fiscal year 2027. The document contained justifications for the 3.3% budget rise compared to fiscal year 2026, as well as several legislative shifts.
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Makary said permanently reauthorising the rare pediatric disease PRV programme would create “more predictability for sponsors”, as well as ensuring the framework fulfils its goal of accelerating therapies to children with debilitating or life-threatening diseases.
The programme has historically been reauthorised every four years, although political roadblocks can occur. Until recently, it had been in regulatory limbo, mainly due to congressional and funding delays, after expiring in December 2024.
On 3 February, President Donald Trump signed a spending bill that included a reauthorisation of the programme, now active until 2029. The programme’s return was brought back under the Mikaela Naylon Give Kids a Chance Act, a range of regulatory frameworks to increase the speed at which children battling rare diseases and certain cancers can access therapies.
A former FDA reviewer told Pharmaceutical Technology: “By making the programme permanent, it avoids the uncertainty that is created with the run-up to every potential expiration deadline date. There can be negative impacts on product development resulting from the “rush to submit” versus a programme that is established with reliable continuity.”
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.
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 six 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.
Accelerating US pharma interests
The pharma legislative proposals also include a further clampdown on misleading advertising, more flexibility for companies manufacturing generics, and more transparency on complete response letters (CRLs).
The FDA will also double down on the Commissioner’s National Priority Voucher (CNPV) pilot programme, a new voucher framework that prioritises US-centric pharma development. The programme has had a mixed reception since being launched in mid-2025, leading the FDA to convene a public hearing later this year to gather feedback. The CNPV programme and the FDA PreCheck, the latter an initiative to help build pharma facilities in the US, will benefit from an extra $9m in funding.
In a letter preceding the fiscal outlay, Makary said: “The FDA is in the process of transitioning from a reactionary system to a proactive system and creating new pathways to usher new products to market with transformational potential to Americans’ health.”
