Illustration: Gabriella Turrisi/Axios
The FDA's priority review vouchers are an under-the-radar health program that would need to be reauthorized in December, with research into drugs to treat rare pediatric diseases on the line if it doesn't happen.
Why it matters: Patient advocates and pharmaceutical companies say a missed deadline could jeopardize the benefits of the program, which gives drugmakers incentives to develop drugs for rare pediatric diseases by allowing streamlined FDA product evaluations.
- But others argue that the program is a handout to drug manufacturers.
What they're saying: The voucher program encourages drug companies "to take on the financial and scientific risks associated with developing treatments for diseases that have historically been overlooked," Terry Wilcox, co-founder of Patients Rising, a pharma-affiliated patient advocacy group, told Axios.
- If the program is allowed to expire, Wilcox said, "we jeopardize the progress we've made in giving patients and families hope for lifesaving therapies."
- The biotech trade association BIO said in a statement to Axios that it is "critical that Congress pass reauthorization as soon as possible to continue this vital path forward."
State of play: The program was due to expire Sept. 30 but was extended in the latest government funding continuing resolution. The new deadline is Dec. 20, the same day the CR expires.
- In September, the House passed legislation by voice vote that would reauthorize the program so the FDA could continue issuing vouchers through 2029.
- Sens. Bob Casey and Markwayne Mullin introduced a bipartisan reauthorization bill in the Senate this summer that would extend the program through 2030.
PAHPA, the pandemic preparedness bill, whose own reauthorization has been stalled since last year, had been seen as a potential vehicle for priority review vouchers to ride on.
- Last year, HELP Ranking Member Bill Cassidy was pushing for a five-year voucher extension to be included in PAHPA. Cassidy's office told Axios the lawmaker still supports extending the program.
- Now it seems more likely that the priority review measure will have to ride on an end-of-year package, if one does materialize, or be extended in the short term in another continuing resolution into next year.
- One point in favor of the program is that a previous estimate by the CBO has put it as costing a minimal amount, and most of the cost is just for administration.
Friction point: But one obstacle could be current HELP Chair Bernie Sanders, who has previously opposed the priority review voucher program, viewing it as a handout to pharmaceutical companies.
- Sanders' office didn't respond to a request for comment.
- During the Energy and Commerce committee's markup of the House bill, Ranking Member Frank Pallone questioned whether the program actually motivated pharmaceutical companies to create drugs for rare diseases. But he still voted to approve the legislation.
Zoom in: The program works by awarding a drugmaker a voucher once it's received approval for a rare disease therapeutic.
- That voucher can then be used to get priority review for other therapeutics that wouldn't otherwise qualify, or it can be sold to other companies.
- Vouchers can sell for tens of millions of dollars.
The most common diseases that the vouchers have been awarded for include Duchenne muscular dystrophy, neuroblastoma and sickle cell anemia.
- Since the program started in 2012, 53 priority review vouchers have been issued, per the National Organization for Rare Disorders.
