Ask the Senate to Extend the Priority Review Voucher

The U.S. House of Representatives has passed the Give Kids a Chance Act, which includes an extension of the Priority Review Voucher -- a critical tool to encourage rare disease drug development. Now is the time for the Senate to take action. Reach out today to let your Senator know to prioritize rare disease drug development by passing the Give Kids a Chance Act. 

To send the below customized letter to your senator, simply:

1. Enter Your Name, Address, and email in the Take Action Now Box to the right and click "Next." 

2. Review the body of the email -- feel free to add any language you feel fits the ask relating to your loved one.

3. Click Send Email. 

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On behalf of the mitochondrial disease community and the more than one in ten Americans affected by a rare disease, we urge you to support swift reauthorization of the Rare Pediatric Disease Priority Review Voucher (PRV) program by prioritizing passage of S. 932 / H.R. 1262, the Give Kids a Chance Act, before the end of the year. The authorization for the PRV program expired last December. Without urgent Congressional action, hope for thousands of children living with mitochondrial disease—and millions more across the rare-disease spectrum—may fade irreversibly.

The program's lapse is already having measurable consequences. Companies developing therapies for mitochondrial disease are reconsidering their early-stage pipelines, delaying or shelving programs, and increasingly struggling to attract investment. Drug development for rare pediatric conditions takes many years; without the predictability the PRV program provides, companies cannot responsibly plan long-term R&D or make the financial commitments needed to advance therapies toward approval.

Reauthorizing the program would help ensure continued progress for children living with mitochondrial disease and related rare pediatric conditions. The PRV program has enjoyed broad, bipartisan, and bicameral support since its creation in 2012. The Give Kids a Chance Act has already advanced through the House of Representatives and now only needs your Senate support.

The need could not be more pressing. Mitochondrial diseases—like many rare conditions—are devastating, progressive, and often fatal. For mitochondrial disease specifically, there are only two FDA-approved disease-modifying treatments, despite more than 300 known genetic subtypes and a highly complex clinical course. The challenges of rare pediatric drug development—including small patient populations, heterogeneous progression, limited natural history data, and the absence of validated endpoints—make targeted incentives such as the PRV program essential to sustaining industry investment.

At a moment when mitochondrial disease therapies are finally within reach, Congress has the opportunity to ensure that progress does not stall. Reauthorizing the Rare Pediatric Disease PRV program is a critical step in giving children with mitochondrial disease a real chance at life-changing treatments.  We appreciate your support and appreciation.