The US House of Representatives on Monday afternoon unanimously passed a wide-ranging bill via voice vote that will reauthorize the rare pediatric priority review voucher program through 2029, among other FDA-related tweaks.
The bill’s passage comes just before the Senate HELP Committee’s vote on its own version of the reauthorization on Thursday, which includes an extension through 2030. While the House and Senate may not meet the Sept. 30 deadline to reauthorize the program, the short-term continuing resolution that will keep the government’s lights on until Dec. 20 also includes an extension of the rare pediatric PRV program through Dec. 20.
Once the program is reauthorized, biopharma companies can continue to receive the rare pediatric disease PRVs from the FDA, which typically sell for about $100 million. Companies like AstraZeneca, BioMarin, Novartis, Vertex and Sarepta Therapeutics have all won more than one rare pediatric PRV so far for developing rare pediatric disease drugs, and have used the vouchers to speed up the FDA reviews of other drugs.
Also included in the House-passed bill is a new Government Accountability Office report on the effectiveness of rare pediatric disease priority voucher awards in incentivizing rare pediatric disease drug development.
That topic has been a sensitive one as academic research and the FDA have questioned how the PRV programs add new priorities for the agency. A GAO report from 2020 on both the rare pediatric and rare tropical disease PRVs found that academic research did not link the PRVs with incentivizing the development of new drugs in these categories, but all seven drug sponsors GAO spoke with stated that PRVs were a factor in their drug development decisions.
The bill also includes language that would codify the FDA’s position on orphan drug exclusivity. The FDA in January 2023 maintained that it would continue to tie the scope of orphan drug exclusivity to the uses or indications for which a drug is approved, which ran counter to what the US Court of Appeals for the Eleventh Circuit said should be the case with Catalyst, where orphan exclusivity could block any competitors beyond the scope of the approved population.
The bill text removes language from the Federal Food, Drug, and Cosmetic Act that says exclusivity applies to the “same disease or condition” and inserts “same approved use or indication within such rare disease or condition.”
Other tweaks in the bill focus on research into pediatric uses of drugs, ensuring pediatric study compliance and the creation of a new FDA office in the Middle East.