An update on the status of the Pediatric Rare Disease Priority Review voucher program….
This program, which was initiated 12 years ago, has always had scheduled sunset dates. Each time those sunset dates approach, the US Government must decide whether to authorize extension of the program.
As it currently stands, the FDA cannot award any Priority Review Vouchers unless a drug received Rare Pediatric Disease designation not later than December 20, 2024 (this was extended from September 30, 2024, to allow the government time to debate and vote on another extension of the program) and the drug and PRV application is approved not later than September 30, 2026.
In late September, the House of Representatives, with bipartisn support, passed the Give Kids a Chance Act of 2024 which was then introduced in the Senate. This bill would extend the designation deadline until September 30, 2029, a five-year extension. Separately, the Creating Hope Reauthorization Act of 2024 has been introduced into the Senate, also with bipartisan sponsorship. This Act proposes that the FDA be authorized to continue awarding PRVs through to September 2030.
However, the issue of authorizing funding of the US Government took first place as the clock ticked down to a potential government shutdown on December 20. While the bill to refund the government was passed at the last minute, it was ultimately stripped of many other provisions that had been rolled into it, such as the extension to the PRV program.
Consequently, as it now stands, any drug not already awarded Rare Pediatric Disease designation is ineligible to receive a Priority Review Voucher on FDA approval. And a drug that has already been awarded Pediatric Rare Disease designation is only eligible to receive a PRV if the drug if the drug and its PRV application are approved prior to September 30, 2026.
This would mean that NNZ-2591 for the treatment of Phelan McDermid syndrome, although already awarded rare Pediatric Disease designation, would miss the current September 30, 2026 cut-off point.
However, I remain hopeful.
Firstly, both the Give Kids a Chance Act of 2024 and the Creating Hope Reauthorization Act of 2024 are still in train and have bipartisan support.
Also, Congress has since passed several bills that were originally stripped from the bill that was hastily passed before Christmas. There are likely to be more.
Further, as stated before, in his post-Inauguration address to Congress in 2017, Donald Trump highlighted the case of a young woman with a rare paediatric disease (Megan) whose Dad fought for funding to develop a drug to save her life.
President Trump stated
… our slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need.
If we slash the restraints, not just at the FDA but across our Government, then we will be blessed with far more miracles like Megan.
The Rare Pediatric Disease Priority Review Voucher program has proven to be of vital importance for rare paediatric disease product development. Recent analysis from NORD shows that among the 39 rare paediatric diseases for which vouchers were awarded until April last year, only three had any previously FDA-approved products on the market.
Finally, not only is the PRV program an incentive scheme that works, it also comes at no cost to the Government – it is big pharma who pays for the vouchers.
So what is there not to love in this program for an entrepreneurial, miracle-loving Trump, a cost-saving zealot Musk and a big pharma-skeptic Kennedy?
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