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A bill to reauthorize a program that encourages the development of pediatric rare disease treatments was introduced in the Senate on June 20.
Bill S.4583 would reauthorize the Pediatric Priority Review Voucher (PPRV) program, which expires Sept. 30. The bill mirrors a bill introduced in the House in February.
It was sponsored in the Senate by Sens. Bob Casey (D-PA) and Markwayne Mullin (R-OK), and co-sponsored by Sens. Sherrod Brown (D-OH) and Susan Collins (R-ME).
“As a father of two children with a rare disease, I am grateful that lawmakers are working together, on a bipartisan basis, to continue this vital program and hope it’s signed into law as soon as possible,” said John F. Crowley, President & CEO of the Biotechnology Innovation Organization (BIO).
What is the PPRV program?
The PPRV program encourages the development of treatments for rare diseases impacting children.
Rare diseases are those that affect fewer than 200,000 people in the U.S. Taken together, more than 7,000 rare diseases impact 30 million Americans, and 95% have no treatment, according to the National Organization for Rare Disorders (NORD).
Under the PPRV program, companies gaining approval for drugs targeting rare pediatric diseases can receive a voucher granting expedited review of another drug in the future. Drug makers can also fund current and future research by selling the voucher to another company.
“Since its creation in 2012, this program has awarded 53 vouchers for 39 rare pediatric diseases that have led to innovations benefitting over 200,000 patients. 36 of those rare diseases had no previously approved therapies on the market at the time of approval,” according to Sen. Mullin.
What’s next for the PPRV program?
The bill that was just introduced into the Senate would extend the program for six years to “provide greater stability to innovators, encourage investment, and spur innovation in rare and neglected diseases that disproportionately impact children,” said Sen. Casey. “Our bipartisan bill will keep this critical voucher program going so drug companies don’t stop innovating new treatments to help sick kids.”
The PPRV program has already done a lot of good, BIO’s Crowley noted.
“For more than a decade, the rare pediatric disease priority review voucher program has offered immeasurable hope to countless families,” he said.
“At no cost to taxpayers, the voucher program has helped incentivize the development of nearly four dozen new medicines for children with rare diseases,” Crowley continued. “The program demonstrates how smart government policy, combined with the scientific skill and dedication of America’s biotechnology industry, can lead to innovative new medicines for patients.”
