University of Miami Sylvester Comprehensive Cancer Center/Jackson Memorial Hospital, Miami, Florida.
University of Miami Miller School of Medicine, Miami, Florida.
JAMA Netw Open. 2024 May 1;7(5):e2413938. doi: 10.1001/jamanetworkopen.2024.13938.
Standard of care for unresectable locally advanced non-small cell lung cancer (NSCLC) involves definitive chemoradiotherapy followed by maintenance therapy with durvalumab. However, the cost of durvalumab has been cited as a barrier to its use in various health systems.
To evaluate the cost-effectiveness of durvalumab vs placebo as maintenance therapy in patients with unresectable stage III NSCLC from 4 international payer perspectives (US, Brazil, Singapore, and Spain).
DESIGN, SETTING, AND PARTICIPANTS: In this economic evaluation, a Markov model was designed to compare the lifetime cost-effectiveness of maintenance durvalumab for unresectable stage III NSCLC with that of placebo, using 5-year outcomes data from the PACIFIC randomized placebo-controlled trial. Individual patient data were extracted from the PACIFIC, KEYNOTE-189, ADAURA, ALEX, and REVEL randomized clinical trials to develop a decision-analytic model to determine the cost-effectiveness of durvalumab compared with placebo maintenance therapy over a 10-year time horizon. Direct costs, adverse events, and patient characteristics were based on country-specific payer perspectives and demographic characteristics. The study was conducted from June 1, 2022, through December 27, 2023.
Life-years, quality-adjusted life years (QALYs), lifetime costs, and incremental cost-effectiveness ratios (ICERs) were estimated at country-specific willingness-to-pay thresholds ([data reported in US$] US: $150 000 per QALY; Brazil: $22 251 per QALY; Singapore: $55 288 per QALY, and Spain: $107 069 per QALY). One-way and probabilistic sensitivity analyses were performed to account for parameters of uncertainty. A cost-threshold analysis was also performed.
The US base-case model found that treatment with durvalumab was associated with an increased cost of $114 394 and improved effectiveness of 0.50 QALYs compared with placebo, leading to an ICER of $228 788 per QALY. Incremental cost-effectiveness ratios, according to base-case models, were $141 146 for Brazil, $153 461 for Singapore, and $125 193 for Spain. Durvalumab price adjustments to the PACIFIC data improved cost-effectiveness in Singapore, with an ICER of $45 164. The model was most sensitive to the utility of durvalumab.
In this cost-effectiveness analysis of durvalumab as maintenance therapy for unresectable stage III NSCLC, the therapy was found to be cost-prohibitive from the perspective of various international payers according to country-specific willingness-to-pay thresholds per QALY. The findings of the study suggest that discounted durvalumab acquisition costs, as possible in Singapore, might improve cost-effectiveness globally.
不可切除的局部晚期非小细胞肺癌（NSCLC）的标准治疗包括明确的放化疗，随后用度伐鲁单抗进行维持治疗。然而，度伐鲁单抗的成本已被认为是在各种医疗体系中使用它的障碍。
从 4 个国际支付者的角度（美国、巴西、新加坡和西班牙）评估不可切除的 III 期 NSCLC 患者中，度伐鲁单抗与安慰剂作为维持治疗的成本效益。
设计、地点和参与者：在这项经济评估中，设计了一个马尔可夫模型，以比较不可切除的 III 期 NSCLC 患者使用度伐鲁单抗与安慰剂作为维持治疗的终生成本效益，使用 PACIFIC 随机安慰剂对照试验的 5 年结果数据。从 PACIFIC、KEYNOTE-189、ADAURA、ALEX 和 REVEL 随机临床试验中提取患者个体数据，以开发一个决策分析模型，以确定在 10 年时间范围内，与安慰剂维持治疗相比，度伐鲁单抗的成本效益。直接成本、不良事件和患者特征基于国家特定的支付者视角和人口统计学特征。该研究于 2022 年 6 月 1 日至 2023 年 12 月 27 日进行。
在国家特定的支付意愿阈值下（美国：每 QALY 150000 美元；巴西：每 QALY 22251 美元；新加坡：每 QALY 55288 美元，西班牙：每 QALY 107069 美元），估计了寿命年、质量调整生命年（QALYs）、终生成本和增量成本效益比（ICERs）。进行了单向和概率敏感性分析，以考虑不确定性参数。还进行了成本阈值分析。
美国基本案例模型发现，与安慰剂相比，使用度伐鲁单抗治疗的成本增加了 114394 美元，效果提高了 0.50 QALY，导致每 QALY 的 ICER 为 228788 美元。根据基本案例模型，巴西的增量成本效益比为 141146 美元，新加坡为 153461 美元，西班牙为 125193 美元。对 PACIFIC 数据进行度伐鲁单抗价格调整可提高新加坡的成本效益，ICER 为 45164 美元。该模型对度伐鲁单抗的效用最为敏感。
在这项关于不可切除的 III 期 NSCLC 患者中，使用度伐鲁单抗作为维持治疗的成本效益分析中，根据每个 QALY 的国家特定支付意愿阈值，该疗法被认为在各种国际支付者中是成本不可承受的。研究结果表明，在新加坡，可能会降低度伐鲁单抗的折扣获得成本，从而在全球范围内提高成本效益。
