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Patient advocates urge PPRV renewal
The Pediatric Priority Review Voucher (PPRV) Program to encourage treatments for rare diseases must be reauthorized, said panelists during BIO’s March Patient Advocacy Coffee Chat.
What it is: Companies gaining approval for rare pediatric disease drugs can receive a voucher granting expedited review of another drug in the future; the voucher can also be sold to another company to fund research. Renewed with bipartisan backing since its initiation in 2012, the program sunset at the end of last year.
Why it matters: As many as 60 drugs have earned a voucher to date—the vast majority for rare pediatric diseases without treatment options before the program’s inception, Bio.News reports. And the program costs taxpayers nothing.
Why delayed reauthorization is bad: “It’s creating uncertainty. And this means that people aren’t getting resources, and then it means that children are going to have to wait longer to get important medicines,” said Duke University's Dr. David Ridley, who was instrumental in developing the program.
“Last year, the House passed unanimously a bill called the Give Kids a Chance Act,” which included PPRV reauthorization and other beneficial legislation, but it was dropped at the last minute from December’s spending package, said Jamie Sullivan of EveryLife Foundation for Rare Diseases.
“One thing that we can all agree on is the need to give our children a fighting chance,” BIO President & CEO John F. Crowley wrote to Congress. “This program will provide greater certainty to small biotech companies, but more importantly, hope to children and families living with rare diseases.”
Read more on Bio.News.
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