A renewal of the FDA’s pediatric rare disease voucher program is couched within a massive $1.2 trillion spending bill the House is set to vote on later this week.
There is hope yet for the FDA’s rare pediatric disease priority vouchers as key legislation is set to come before the U.S. House of Representatives later this week and the Senate next week. If passed, it would renew the program and make these expedited review tickets available to drugmakers once again.
The House Appropriations Committee on Tuesday released the text of a funding package to keep the government open after a Jan. 30 deadline. That package includes $1.2 trillion in spending across the federal government, including Defense, Transportation, Housing and Urban Development and Health and Human Services.
The rare pediatric priority review voucher (PRV) program—now up for renewal in the HHS section of the legislation—first came into play in 2012 to encourage drugmakers to develop therapies for rare pediatric diseases. As a reward for developing and securing an approval for such conditions, companies are given a voucher that they can claim to speed up the review of a different drug application.
Companies can also choose to sell their vouchers for some liquidity. Last week, for instance, Jazz Pharmaceuticals offloaded a ticket for $200 million, the highest sale price in nearly 10 years.
In September 2024, Congress failed to authorize the renewal of the program, forcing the FDA in December of that year to wind down the scheme. Review vouchers have become more expensive as the program expired.
Then, in September last year, the House Committee on Energy and Commerce reignited hopes for the program after it advanced a bill that would reauthorize the awards. The proposed legislation was dubbed the Mikaela Naylon Give Kids a Chance Act of 2025 and, if passed, would keep the vouchers going through Sept. 30, 2029.
A vote on the spending proposal is expected to take place later this week, according to reporting from The Washington Post.
Also of interest to the pharma sector is the inclusion in the spending bill of a series of reforms to pharmacy benefits managers (PBMs), including a new HHS review of fees paid to PBMs and a requirement that PBMs submit reports to HHS covering drug pricing and rebate data, as well as written justifications for coverage choices.
The overall funding package is designed to prevent another government shutdown and would keep federal offices running past the Jan. 31 deadline, as per the Post.
The proposal also puts the National Institutes of Health’s budget at $48.7 billion—a $415 million increase from 2025 and in direct contrast with the Trump administration’s thrust to cut the agency’s appropriation by $18 billion.
If the spending bill passes the House vote, it will go on to the Senate next, though no schedule for this deliberation has yet been announced.
