Congress's Failure to Renew Priority Review Voucher Program Threatens Rare Disease Drug Development

Key Insights

• The FDA's rare pediatric disease Priority Review Voucher program expired in December 2023, despite unanimous congressional support for renewal, leaving biotech companies unable to receive new vouchers worth approximately $150 million.
• Eleven priority review vouchers were awarded in 2023, marking a record year, with beneficiaries including treatments for sickle cell disease, Duchenne muscular dystrophy, and other rare conditions.
• The program's suspension threatens future rare disease drug development, as many companies rely on voucher sales for funding, with existing designations only valid until September 2026 for voucher eligibility.

The biotechnology industry faces significant disruption as Congress failed to renew the FDA's rare pediatric disease Priority Review Voucher (PRV) program, despite widespread bipartisan support. The program's expiration in December 2023 has created immediate uncertainty for companies developing treatments for rare pediatric diseases.

Impact on Rare Disease Drug Development

The PRV program, established in 2012, has been a crucial catalyst for rare disease drug development. These vouchers, which expedite FDA review times from 12 to 6 months, have become valuable assets trading at approximately $150 million each. Zevra Therapeutics recently demonstrated this value by selling their voucher, received for Miplyffa's approval, at this premium price point.

"The impact on the rare disease drug developers and the investment community [is] already being felt because of the destabilizing effect of the failure to reauthorize the program prior to its expiration," explains Stacey Friske, executive director of the Rare Disease Company Coalition.

Current Status and Implications

The FDA began sunsetting the program on December 20, 2023. Companies with existing rare pediatric disease designations must now secure approval by September 30, 2026, to receive a voucher. This deadline has prompted companies to issue warnings to investors through SEC filings about the potential loss of anticipated vouchers.

Matthew Winton, chief operating officer of Inozyme Pharma, emphasizes the program's urgency: "Time is... sort of running out. We need action on this immediately to continue the program but also continue investment in these pediatric rare diseases."

Program Success Stories

The PRV program has demonstrated significant impact in advancing treatments for severe conditions. Duchenne muscular dystrophy has seen seven drugs approved under the program, with Sarepta Therapeutics securing four vouchers. Spinal muscular atrophy (SMA) treatment has been transformed through medications like Spinraza, Zolgensma, and Evrysdi, all benefiting from the program.

"That disease state has completely changed. You were used to planning funerals. And now you're celebrating birthdays and you're celebrating graduation into high school," Winton reflects on SMA treatment advances.

Legislative Efforts and Future Outlook

The Give Kids a Chance Act of 2024 has been proposed to reinstate the program as Congress works to avoid an impending shutdown. The industry is pushing for inclusion in the March 14 budget deadline legislation.

While critics argue the program creates a "quid pro quo" situation benefiting pharmaceutical companies, supporters emphasize its role in driving innovation for rare diseases. The program has facilitated the development of novel therapeutic modalities, including gene therapies and antisense oligonucleotide technologies.

"These [vouchers] are not necessarily going to the 17th, 18th statin—not that there's anything wrong with that—but these are also going to feed the cycle of innovation and bringing drugs to patients in the U.S. who have no other options," Winton argues, highlighting the program's importance for continued rare disease innovation.