Catalan Institute of Oncology, Hospital Duran i Reynals, IDIBELL, L'Hospitalet de Llobregat, Spain.
Hospital Costa del Sol, Marbella, Spain.
BMC Cancer. 2021 Jun 10;21(1):689. doi: 10.1186/s12885-021-08407-1.
Currently biomarkers play an essential role in diagnosis, treatment, and management of cancer. In non-small cell lung cancer (NSCLC) determination of biomarkers such as ALK, EGFR, ROS1 or PD-L1 is mandatory for an adequate treatment decision. The aim of this study is to determine the clinical and economic impact of current anaplastic lymphoma kinase testing scenario in Spain.
A joint model, composed by decision-tree and Markov models, was developed to estimate the long-term health outcomes and costs of NSCLC patients, by comparing the current testing scenario for ALK in Spain vs a hypothetical no-testing. The current distribution of testing strategies for ALK determination and their sensitivity and specificity data were obtained from the literature. Treatment allocation based on the molecular testing result were defined by a panel of Spanish experts. To assess long-term effects of each treatment, 3-states Markov models were developed, where progression-free survival and overall survival curves were extrapolated using exponential models. Medical direct costs (expressed in €, 2019) were included. A lifetime horizon was used and a discount rate of 3% was applied for both costs and health effects. Several sensitivity analyses, both deterministic and probabilistic, were performed in order test the robustness of the analysis.
We estimated a target population of 7628 NSCLC patients, including those with non-squamous histology and those with squamous carcinomas who were never smokers. Over the lifetime horizon, the current ALK testing scenario produced additional 5060 and 3906 life-years and quality-adjusted life-years (QALY), respectively, compared with the no-testing scenario. Total direct costs were increased up to € 51,319,053 for testing scenario. The incremental cost-effectiveness ratio was 10,142 €/QALY. The sensitivity analyses carried out confirmed the robustness of the base-case results, being the treatment allocation and the test accuracy (sensitivity and specificity data) the key drivers of the model.
ALK testing in advanced NSCLC patients, non-squamous and never-smoker squamous, provides more than 3000 QALYs in Spain over a lifetime horizon. Comparing this gain in health outcomes with the incremental costs, the resulting incremental cost-effectiveness ratio reinforces that testing non-squamous and never-smoker squamous NSCLC is a cost-effective strategy in Spain.
目前，生物标志物在癌症的诊断、治疗和管理中发挥着重要作用。在非小细胞肺癌（NSCLC）中，确定ALK、EGFR、ROS1 或 PD-L1 等生物标志物对于做出适当的治疗决策是必要的。本研究旨在确定目前在西班牙间变性淋巴瘤激酶检测方案的临床和经济影响。
采用决策树和马尔可夫模型联合模型，通过比较西班牙目前的 ALK 检测方案与假设的不检测方案，估计 NSCLC 患者的长期健康结果和成本。从文献中获得目前用于确定 ALK 的检测策略的分布及其敏感性和特异性数据。根据西班牙专家小组的定义，根据分子检测结果进行治疗分配。为了评估每种治疗方法的长期效果，开发了 3 状态马尔可夫模型，其中使用指数模型推断无进展生存期和总生存期曲线。包括医疗直接成本（以欧元表示，2019 年）。使用终生时间范围，并对成本和健康效果应用 3%的折扣率。进行了多次确定性和概率敏感性分析，以检验分析的稳健性。
我们估计了 7628 名 NSCLC 患者的目标人群，包括非鳞状组织学和从不吸烟的鳞状癌患者。在终生时间范围内，与不检测方案相比，当前的 ALK 检测方案分别产生了额外的 5060 和 3906 个生命年和质量调整生命年（QALY）。检测方案的总直接成本增加了 5131.9053 万欧元。增量成本效益比为 10142 欧元/QALY。进行的敏感性分析证实了基础病例结果的稳健性，治疗分配和测试准确性（敏感性和特异性数据）是模型的关键驱动因素。
在西班牙，对晚期非小细胞肺癌患者（非鳞状和从不吸烟的鳞状）进行 ALK 检测，在终生时间范围内可提供超过 3000 个 QALY。将这些健康结果的提高与增量成本进行比较，增量成本效益比证实了对非鳞状和从不吸烟的鳞状 NSCLC 进行检测是一种具有成本效益的策略。
