Abstract：
Cell and gene therapy (CGT) innovations have provided several significant breakthroughs in recent years. However, CGTs often come with a high upfront cost, raising questions about patient access, affordability, and long-term value. This study reviewed cost-effectiveness analysis (CEA) studies that have attempted to assess the long-term value of Food and Drug Administration (FDA)-approved CGTs. Two reviewers independently searched the Tufts Medical Center CEA Registry to identify all studies for FDA-approved CGTs, per January 2023. A data extraction template was used to summarize the evidence in terms of the incremental cost-effectiveness ratio expressed as the cost per quality-adjusted life year (QALY) and essential modeling assumptions, combined with a template to extract the adherence to the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist. The review identified 26 CEA studies for seven CGTs. Around half of the base-case cost-effectiveness results indicated that the cost per QALY was below $100,000-$150,000, often used as a threshold for reasonable cost-effectiveness in the United States. However, the results varied substantially across studies for the same treatment, ranging from being considered very cost-effective to far from cost-effective. Most models were based on data from single-arm trials with relatively short follow-ups, and different long-term extrapolations between studies caused large differences in the modeled cost-effectiveness results. In sum, this review showed that, despite the high upfront costs, many CGTs have cost-effectiveness evidence that can support long-term value. Nonetheless, substantial uncertainty regarding long-term value exists because so much of the modeling results are driven by uncertain extrapolations beyond the clinical trial data.
摘要：
近年来，细胞和基因治疗创新提供了一些重大突破。然而,细胞和基因疗法往往有很高的前期成本，提出关于病人接触的问题，负担能力，和长期价值。这项研究回顾了成本效益分析研究，这些研究试图评估FDA批准的细胞和基因疗法的长期价值。两名审稿人独立搜索了塔夫茨医学中心成本效益分析登记处，以确定2023年1月FDA批准的细胞和基因治疗的所有研究。使用数据提取模板来总结增量成本效益比的证据，该增量成本效益比表示为每质量调整寿命年的成本（QALY）和基本建模假设。结合模板，以提取对综合卫生经济评估报告标准（CHEERS）清单的遵守情况。该综述确定了26项针对7种细胞和基因治疗的CEA研究。大约一半的基本情况成本效益结果表明，每个QALY的成本低于$100,000-$150,000，通常用作美国合理成本效益的阈值。然而,对于相同治疗的研究，结果差异很大，从被认为非常具有成本效益到远远没有成本效益。大多数模型是基于单臂试验的数据，随访时间相对较短，和研究之间不同的长期外推导致建模的成本效益结果存在很大差异。总之,这项审查表明，尽管前期成本很高，许多细胞和基因疗法的成本效益证据可以支持长期价值.尽管如此，长期价值存在很大的不确定性,因为很多建模结果都是由临床试验数据之外的不确定外推驱动的.
