The House Energy & Commerce Committee unanimously advanced a bill late Wednesday that would reauthorize the rare pediatric priority review voucher (PRV) program through 2029.
But the House bill and companion legislation in the Senate have stalled, and the program expires on Sept. 30. The Senate version has yet to surface in the Committee on Health, Education, Labor and Pensions, and some have blamed Committee Chair Bernie Sanders (I-VT) for holding up reauthorization. Sanders and others have criticized the program as a handout to pharma companies that would have developed these same treatments without the PRVs.
The final version of the bill that passed the House Energy & Commerce Committee included a 2029 expiration date, instead of a 2030 sunset floated earlier this summer. Nancy Goodman, the founder of Kids v Cancer — a nonprofit that promotes pediatric cancer research — noted that the change in the House bill text seeks to help the FDA, which previously had to deal with a two-step sunset schedule and face a surge of applications for rare pediatric designations after four years, and a six-year sunset for issuing vouchers. FDA now faces a five-year schedule for both designations and issuing vouchers, she said.
“While there are expectations that the PRV program will be renewed, the timing is becoming increasingly murky,” William Blair analysts said in a note on Wednesday. That uncertainty and the limited number of PRVs that could still be awarded under the current program could increase the sale price of PRVs, William Blair said.
Ipsen sold the last publicly-announced PRV last month for the highest price since 2016. For fiscal year 2025, the price of using a PRV is also up, to about $2.5 million, the FDA said Wednesday.
Betting on passage
Close watchers of the program are predicting the reauthorization will likely pass, potentially with an incoming continuing resolution to keep the government funded.
Bobby McMillin, managing director at the law firm Arnold & Porter and a former HELP general counsel, told Endpoints News he’s heard that Sanders’ position is if the PRV reauthorization advances, “he wants to move these three or four other things that everyone may not be in agreement with, if that makes sense; he kind of wants to exact a price.”
Sanders’ staff hasn’t responded to multiple questions on the rare pediatric PRV reauthorization.
McMillin also said he’s surprised Sen. Bob Casey (D-PA), co-author of the Senate version of the rare pediatric PRV reauthorization who’s up for reelection this year, hasn’t taken action.
“We kind of thought it might get a markup, you know, just because it’s a nice thing for him to put out a press release on, and say he’s been invested,” McMillin said.
Advocates of the PRV program have stressed that it’s helped provide hundreds of millions of dollars to companies that may not be able to justify developing a new drug in a rare pediatric indication.
Goodman said she hopes the PRV reauthorization will be included in the must-pass continuing resolution to keep the government running.