本研究旨在评估下一代测序 (NGS) 与序贯单基因检测 (SGT) 的成本效益，包括晚期 EGFR/ALK 阴性非小细胞相关治疗的长期成本和生存结果肺癌（NSCLC）。我们开发了一个与分区生存模型相关的决策树，以估计五年分析期内的临床结果和成本。决策树由基于分子生物标志物（ROS1、BRAF、NTRK、MET、RET 和 KRAS 改变）测试结果的治疗类型组成。接受每种靶向治疗的概率是根据 1) 检测率、2) 检测到的改变的比例以及 3) 接受与检测结果一致的治疗的患者比例来估计的。我们通过重建临床试验中的患者水平数据，使用参数估计来估计每种治疗的长期总体生存率和无进展生存率。测试、药物、管理、就诊、监测、不良事件、进展后和临终关怀的费用都包括在内。效用值是从之前的研究中获得的。增量成本效益比 (ICER) 用于在每个质量调整生命年 (QALY) 38,701 美元（50,000,000 KRW）的阈值内评估 NGS 的成本效益。 NGS 组与 SGT 组分别为 0.028 和 0.023。 NGS 组的总医疗费用比 SGT 组高出 8,375 美元。药品费用的差异占医疗总费用差异的大部分。与序贯 SGT 相比，NGS 不具有成本效益，ICER 分别为 300,233 美元/LY 和 359,405 美元/QALY。NGS 对于晚期 EGFR/ALK 阴性 NSCLC 不具有成本效益，但比序贯 SGT 具有生存获益。我们的研究结果为有关 EGFR 突变流行地区的 NGS 覆盖范围和临床应用的决策提供了基础。版权所有 © 2024。由 Elsevier B.V. 出版。

This study aimed to evaluate the cost-effectiveness of next-generation sequencing (NGS) versus sequential single-gene testing (SGT), including the long-term costs and survival outcomes of relevant treatments for advanced EGFR/ALK-negative non-small cell lung cancer (NSCLC).We developed a decision tree linked to a partitioned survival model to estimate the clinical outcomes and costs over the five-year analysis period. The decision tree consisted of treatment types based on molecular biomarker (ROS1, BRAF, NTRK, MET, RET, and KRAS alterations) test results. The probability of receiving each targeted therapy was estimated based on 1) the testing rate, 2) the proportion of alterations detected, and 3) the proportion of patients receiving treatment consistent with the testing results. We estimated the long-term overall survival and progression-free survival for each treatment using parametric estimation by reconstructing patient-level data from clinical trials. The costs of testing, drugs, administration, physician visits, monitoring, adverse events, post-progression, and end-of-life care were included. The utility values were obtained from a previous study. The incremental cost-effectiveness ratio (ICER) was used to evaluate the cost-effectiveness of NGS within a threshold of $38,701 (50,000,000 KRW) per quality-adjusted life year (QALY).The incremental life-years (LYs) and QALYs for the NGS group versus the SGT group were 0.028 and 0.023, respectively. The total medical cost for the NGS group was $8,375 higher than that for the SGT group. The difference in drug costs accounted for most of the differences in total medical costs. NGS was not cost-effective compared to sequential SGT, with an ICER of $300,233/LY and $359,405/QALY, respectively.NGS is not cost-effective for advanced EGFR/ALK-negative NSCLC, but has a survival benefit over sequential SGT. Our findings provide a basis for decision-making regarding the coverage and clinical utilization of NGS in regions where EGFR alterations are prevalent.Copyright © 2024. Published by Elsevier B.V.