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Durable cell and gene therapy potential patient and financial impact: US projections of product approvals, patients treated, and product revenues
Drug Discovery Today ( IF 7.4 ) Pub Date : 2021-09-17 , DOI: 10.1016/j.drudis.2021.09.001
Colin M Young 1 , Casey Quinn 1 , Mark R Trusheim 1
Affiliation  

Durable cell and gene therapies potentially transform patient lives, but payers fear unsustainable costs arising from the more than 1000 therapies in the development pipeline. A novel multi-module Markov chain Monte Carlo-based model projects product-indication approvals, treated patients, and product revenues. We estimate a mean 63.5 (54–74 5th to 95th percentile range) cumulative US product-indication approvals through 2030, with a mean 93 000 patients treated in 2030 generating a mean US$24.4 billion (US$17.0B–35.0B, US$73.0B extreme) list price product revenues not including ancillary medical costs or cost offsets. Thus, the likely dozens of durable cell and gene therapies developed through 2030 are unlikely to threaten US health system financial sustainability.



中文翻译:

持久细胞和基因治疗对患者和财务的潜在影响:美国对产品批准、治疗患者和产品收入的预测

持久的细胞和基因疗法可能会改变患者的生活,但付款人担心开发管道中的 1000 多种疗法会产生不可持续的成本。一种基于蒙特卡罗的新型多模块马尔可夫链模型可以预测产品适应症批准、治疗患者和产品收入。我们估计,到 2030 年,平均 63.5(54-74 个第 5 到第 95 个百分位数范围)累积美国产品适应症批准,到 2030 年平均有 93,000 名患者接受治疗,平均产生 244 亿美元(17.0B-35.0B 美元,73.0B 美元)极端)标价产品收入,不包括辅助医疗成本或成本补偿。因此,到 2030 年可能开发的数十种持久细胞和基因疗法不太可能威胁美国卫生系统的财务可持续性。

更新日期:2021-09-17
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