Hospital Clínico Universitario Lozano Blesa, IIS Aragón, Zaragoza, Spain.
Hospital General Universitario Gregorio Marañón, Madrid, Spain.
J Med Econ. 2024 Jan-Dec;27(1):1379-1387. doi: 10.1080/13696998.2024.2413289. Epub 2024 Oct 23.
Liquid biopsy (LB) is a non-invasive technique to detect genetic alterations by next-generation sequencing (NGS) when tissue biopsy is not available. This study aims to estimate in the Spanish setting, the cost-effectiveness of using FoundationOne Liquid CDx (F1L CDx), a novel blood-derived LB test based on NGS, versus non-molecular diagnosis (non-mDx) in patients with advanced non-small cell lung cancer (NSCLC) in whom tissue sampling is not feasible.
A joint model was developed combining a decision-tree with partitioned survival models to calculate the costs and health outcomes over a lifetime horizon, comparing F1L CDx in LB versus non-mDx. Only direct costs (expressed in € of 2023) were included and a 3% discount rate for future costs and effects was considered. Health outcomes were expressed in Life Years (LYs) and Quality-Adjusted Life Years (QALYs). Utilities and treatment efficacy were obtained from the literature. An expert panel of 11 Spanish oncologists determined the treatment allocation and validated all model inputs and assumptions. Several sensitivity analyses were performed to assess the robustness of the results.
In a hypothetical cohort of 1,000 patients, LB using F1L CDx would detect 386 alterations, so those patients could be treated with targeted therapies or enrolled in clinical trials. Cost-effectiveness results showed that F1L CDx provides greater effectiveness than non-mDx (+383.95 LYs and +305.94 QALYs), with an additional cost of €2,898,308. The incremental cost-utility ratio was €9,473/QALY gained. The probabilistic sensitivity analysis confirmed the robustness of the cost-effectiveness results.
Various limitations inherent to cost-effectiveness analyses were described.
LB with F1L CDx test is a cost-effective strategy in Spain for patients with advanced NSCLC without tissue sample available for molecular diagnosis, improving the personalized treatment of these patients.
液体活检（LB）是一种通过下一代测序（NGS）检测遗传改变的非侵入性技术，当组织活检不可用时可以使用。本研究旨在评估在西班牙环境下，使用基于 NGS 的新型血液衍生 LB 检测方法 FoundationOne Liquid CDx（F1L CDx）与非分子诊断（non-mDx）相比，在组织采样不可行的晚期非小细胞肺癌（NSCLC）患者中的成本效益。
采用决策树与分割生存模型相结合的联合模型，计算终生成本和健康结果，比较 F1L CDx 在 LB 与非-mDx 中的应用。仅包括直接成本（以 2023 年€表示），并考虑未来成本和效果的 3%贴现率。健康结果以生命年（LYs）和质量调整生命年（QALYs）表示。效用和治疗效果从文献中获得。11 名西班牙肿瘤学家的专家小组确定了治疗分配，并验证了所有模型输入和假设。进行了多次敏感性分析，以评估结果的稳健性。
在一个 1000 名患者的假设队列中，使用 F1L CDx 的 LB 可检测到 386 种改变，因此这些患者可以接受靶向治疗或参加临床试验。成本效益结果表明，F1L CDx 比非-mDx 提供更高的效果（增加 383.95 LYs 和 305.94 QALYs），增加的成本为 2898308 欧元。增量成本-效用比为 9473 欧元/QALY。概率敏感性分析证实了成本效益结果的稳健性。
描述了成本效益分析中固有的各种局限性。
对于没有组织样本进行分子诊断的晚期 NSCLC 患者，使用 F1L CDx 进行 LB 是一种在西班牙具有成本效益的策略，可以改善这些患者的个体化治疗。
