A roadmap for affordable genetic medicines

• 成果类型: Review
• 署名作者: Kliegman, Melinda; Zaghlula, Manar; Abrahamson, Susan; Esensten, Jonathan H.; Wilson, Ross C.; Urnov, Fyodor D.; Doudna, Jennifer A.
• 署名单位: University of California System; University of California Berkeley; Tel Aviv University; Chaim Sheba Medical Center; University of California System; University of California Berkeley; Howard Hughes Medical Institute; University of California System; University of California Berkeley; United States Department of Energy (DOE); Lawrence Berkeley National Laboratory; University of California System; University of California Berkeley; University of California System; University of California Berkeley
• 刊物名称: Nature
• ISSN/ISSBN: 0028-5448
• DOI: 10.1038/s41586-024-07800-7
• 发表日期: 2024-10-10
• 关键词: cell therapies; drug development; voucher program; INNOVATION; management; cost

摘要

Twenty genetic therapies have been approved by the US Food and Drug Administration to date, a number that now includes the first CRISPR genome-editing therapy for sickle cell disease-CASGEVY (exagamglogene autotemcel, Vertex Pharmaceuticals). This extraordinary milestone is widely celebrated owing to the promise for future genome-editing treatments of previously intractable genetic disorders and cancers. At the same time, such genetic therapies are the most expensive drugs on the market, with list prices exceeding US$4 million per patient. Although all approved cell and gene therapies trace their origins to academic or government research institutions, reliance on for-profit pharmaceutical companies for subsequent development and commercialization results in prices that prioritize recouping investments, paying for candidate product failures and meeting investor and shareholder expectations. To increase affordability and access, sustainable discovery-to-market alternatives are needed that address system-wide deficiencies. Here we present recommendations of a multidisciplinary task force assembled to chart such a path. We describe a pricing structure that, once implemented, could reduce per-patient cost tenfold and propose a business model that distributes responsibilities while leveraging diverse funding sources. We also outline how academic licensing provisions, manufacturing innovation and supportive regulations can reduce cost and enable broader patient treatment. Implementation of new pricing and business structures and improved licensing and manufacturing processes could reduce the per-patient cost of gene therapy tenfold.