The Rare Disease Pediatric Review Voucher (PRV) is set to expire in late September. This would not be good for the rare disease community.
The Foundation’s thoughts on the PRV are:
- Pediatric Rare Disease Priority Review Voucher (PRV) program is a critical incentive to stimulate companies to develop drugs for rare diseases in children.
- The opportunity to receive a PRV is a key factor when businesses are deciding which disease to work on.
- Companies can sell their PRV and that revenue is part of the incentive for them to invest in developing treatments for rare pediatric conditions.
Some of the important impacts of the PRV, from the Every Life Foundation’s website are:
- Developing drugs for rare pediatric diseases is challenging due to the small populations affected, difficulties associated with conducting clinical trials for children, delays in diagnosis and more.
- About 70% of rare diseases are exclusively pediatric onset and overall, 95% of rare diseases have no approved treatments.
- The impact of the PRV program has continued to increase since the last reauthorization. A new incentive takes time to fully impact decision making since it takes an average of 15 years for a drug to be developed and approved by the FDA.
- 49 Rare Pediatric PRVs have been issued since 2012 for innovative treatments in 40 diseases like spinal muscular atrophy, Duchenne muscular dystrophy, and progeria syndrome.
- 7 out of 7 drug developers interviewed by the GAO reported that PRVs were a factor in drug development decisions.
The EveryLife is asking supporters to take action and have made writing a letter to your state representatives very easy. You’ll see on the right side of this page, you can enter your story information, and then your name, address, phone, and it will automatically populate those representatives in your state and will send for you.
Please consider taking action.
TAKE ACTION: https://everylifefoundation.org/rare-advocates/take-action/
MORE INFORMATION ON THE PRV: https://everylifefoundation.org/prv/