Congress has until the end of September to reauthorize a voucher program that’s meant to incentivize new drug development for rare pediatric diseases. But the program faces a tough congressional schedule ahead of the presidential election and questions over whether it’s working as intended.
The vouchers, known as priority review vouchers, seek to incentivize rare disease research and the majority of recent ones have sold for about $100 million. Unlike the voucher programs for rare tropical diseases and medical countermeasures, the PRV program for rare pediatric diseases is set to run out at the end of September, and its fate is unclear at the moment.
The House Energy & Commerce Committee is currently working on a six-year reauthorization, but Karin Hoelzer, director of policy and regulatory affairs at the National Organization for Rare Disorders (NORD), told Endpoints News that the organization is “deeply concerned” that there “might be a lapse” in the program’s authorization.
“Any additional uncertainty about whether these political incentives will continue to be available has a chilling effect on innovation,” Hoelzer said.
The E&C committee is working on a package of bills that includes the rare pediatric PRV reauthorization, which has bipartisan support. Two weeks ago, the E&C’s health subcommittee advanced the reauthorization, but it has yet to be taken up by the full committee or in the Senate.
“While the rare pediatric PRV program was started in 2012, more than half of the designations, awards and voucher redemptions came in the last four years,” E&C Chair Cathy McMorris Rodgers (R-WA) said at a hearing in May. She noted again at a hearing on Thursday that the reauthorization “is a top priority.”
With the election in November, the timeline for Congress to act is short.
Hoelzer explained how even if the rare pediatric PRV program is not reauthorized, there is a provision in the current program that states any company that already received a rare pediatric designation from the FDA would still win a voucher over the next two years.
“This legislation, which is proposed, will hopefully move forward, and we hope that there will be a companion bill in the next couple of days,” Hoelzer said.
A GAO report from 2020 raised questions about whether the PRV program had incentivized new R&D — or if companies were advancing older drugs just to win the $100 million voucher. NORD recently updated a report that found 53 PRVs have been awarded for 39 pediatric rare diseases since 2012. Before this PRV program, three of those same 39 rare diseases had an FDA-approved drug, NORD said.