Priority Review Vouchers Hit Record High in 2024, Program's Future Uncertain

The pharmaceutical industry saw a surge in priority review vouchers (PRVs) in 2024, with a record number issued amid uncertainty about the program's future. A comprehensive analysis by BioSpace reveals that 11 vouchers were handed out last year, the most since the program's inception in 2014. This spike in activity comes as the rare pediatric disease PRV program faces an uncertain future, leaving many biotech companies in limbo.

Record-Breaking Year and Program Expiration

The FDA's priority review voucher programs, designed to incentivize the development of treatments for rare pediatric diseases, tropical diseases, and medical countermeasures, reached a significant milestone in 2024. A total of 80 vouchers have been issued over the past decade, with 11 in 2024 alone, marking the busiest year for the program.

However, the failure of Congress to renew the rare pediatric disease PRV program at the end of 2024 has cast a shadow over its future. This legislative inaction has left many biotech companies reeling, as the program has been a crucial driver for rare disease drug development.

Voucher Valuation and Notable Transactions

The uncertainty surrounding the program's future has had a dramatic effect on voucher pricing. Current estimates place the value of a PRV at approximately $150 million, a significant increase from previous years. This price spike reflects the scarcity and perceived value of these vouchers in expediting FDA review processes.

Notable transactions in the PRV market include:

• AbbVie's record-setting $350 million purchase of United Therapeutics' voucher in 2015
• Novartis' acquisition of a voucher from Pharming Technologies for $21.2 million in 2023, the lowest recorded price
• Gilead's cumulative spending of at least $621 million on PRV purchases

Impact on Rare Disease Drug Development

The PRV program has had a significant impact on rare disease drug development, particularly for conditions like Duchenne muscular dystrophy (DMD). Seven DMD therapeutics have been approved and granted vouchers, making it the most benefited rare disease from the program.

Sarepta Therapeutics has emerged as a leader in earning PRVs, accumulating four vouchers for its various DMD therapies. This success underscores the program's role in incentivizing research and development for rare pediatric diseases.

As the industry grapples with the potential end of the rare pediatric disease PRV program, stakeholders are closely watching for any legislative action that might revive or replace this crucial incentive for rare disease drug development.