The Sun Has Started to Set on the Rare Pediatric Disease Priority Review Voucher Program – But It Can Still be Saved

The Winter Solstice, signifying the shortest day of the year, was Saturday, December 21.  Although the earliest sunset of the year was actually in early December, this day also marked another premature sunset – the beginning of the end of the rare pediatric disease priority review voucher program.

As we blogged recently, the program has had scheduled sunset dates from its very beginning, which have been repeatedly extended.  The first sunset date is the authority to grant a priority review voucher to a rare pediatric disease product application for a drug that has not been designated as a drug for a rare pediatric disease.  Although such designations are not typically a prerequisite for a voucher, the law is drafted so that once this first sunset date has passed, only designated programs may receive priority review vouchers for rare pediatric diseases until the program sunsets completely.  As we mentioned in our blog post, this first sunset date was originally in September, but was given a brief reprieve until December 20, 2024, as part of a continuing resolution to keep the government funded until that date.

As December 20 approached, there was a need for another continuing resolution to keep the government funded.  Earlier in the week, it was reported that there was a bipartisan agreement on a bill that would have funded the government and extended the first sunset date for the rare pediatric disease priority review voucher program for nearly 5 years, until September 30, 2029.  However, the bill ran into political challenges, and as the minutes ticked away until a potential government shutdown, the bill that was ultimately passed was stripped of many of the provisions that were originally included, such as the extension to the rare pediatric disease priority review voucher program.

This means that, at the current moment, FDA cannot award any priority review vouchers for rare pediatric disease product applications unless it is for a drug that was designated as a drug for a rare pediatric disease not later than December 20, 2024, and such application is approved not later than September 30, 2026.

Although this is a truly regrettable outcome of what seemed like a promising beginning to the week in this regard, this does not mean that all hope is lost.  Congress has since passed several of the bills that were originally stripped out of the continuing resolution, such as the bill that would transfer the land under RFK Stadium in DC to the DC government for the purposes of building a new football stadium (or other potential enumerated purposes).

Therefore, there remains hope that Congress could still act to extend the program.  As we mentioned in our previous blog post, and as stated by many other stakeholders, this program has been crucially important for rare pediatric disease product development, and it does not cost a single government dollar (although we do not wish to minimize the additional resources a priority review application demands).  If the program were to sunset completely, as currently scheduled, the impact on the development of drugs for rare pediatric diseases would be difficult to overstate.  Although FDA cannot currently award rare pediatric disease priority review vouchers to any applications not currently designated as a drug for a rare pediatric disease, there is no provision that prevents FDA from continuing to grant such designations.  We would strongly urge companies to continue to request, and FDA to continue to grant, such designations in the hopes that Congress can accomplish what it seemed ready to do on a bipartisan basis just a few days ago.  In the meantime, we encourage all stakeholders to keep the pressure on to get this done as soon as possible.