Li SiNi, Li JianHe, Peng LiuBao, Li YaMin, Wan XiaoMin
Clinical Nursing Teaching and Research Section, The Second Xiangya Hospital, Central South University, Changsha, China.
The Xiangya Nursing School, Central South University, Changsha, China.
Front Oncol. 2021 May 28;11:684073. doi: 10.3389/fonc.2021.684073. eCollection 2021.
Recently, a phase III CROWN trial compared the efficacy of two anaplastic lymphoma kinase (ALK) inhibitors and demonstrated that lorlatinib displayed clinical improvement over crizotinib for advanced non-small cell lung cancer (NSCLC) patients. Therefore, the aim of this study was to estimate the cost-effectiveness of lorlatinib as a first-line therapy for patients with advanced ALK-positive (+) NSCLC.
A cost-effectiveness analysis was performed using a microsimulation model from the US payer perspective and a lifetime horizon (30 years) in patients with previous untreated advanced ALK+ NSCLC. Based on the CROWN trial, patient characteristics were obtained, and the transition probabilities were estimated. All direct costs were derived from official sources and published literature. The main outcomes of the model were total costs, incremental cost-effectiveness ratio (ICER), quality-adjusted life years (QALYs), and life years (LYs). One-way and probabilistic sensitivity analyses and multiple scenario analyses were conducted to test the robustness of the model outcomes.
In the base case analysis, in which 1 million patients were simulated, treatment with lorlatinib or crizotinib as the first-line treatment was related to a mean cost of $909,758 and $616,230 (incremental cost: $293,528) and a mean survival of 4.81 QALYs and 4.09 QALYs (incremental QALY: 0.72) per patient, respectively. The main drivers of cost effectiveness were drug price and subsequent cost. PAS indicated that lorlatinib has 90% cost-effectiveness when compared to crizotinib when the willingness-to-pay (WTP) threshold in increased to $448,000/QALY. Scenario analysis demonstrated that lorlatinib has 100% cost-effectiveness at a WTP threshold of 200,000/QALY compared to crizotinib treatment when the price of lorlatinib is decreased to 75% ($424.5) of its original price.
In this study, lorlatinib was unlikely to be cost effective compared with crizotinib for patients with previously untreated advanced ALK+ NSCLC at a WTP threshold of 200,000/QALY.
最近，一项III期CROWN试验比较了两种间变性淋巴瘤激酶（ALK）抑制剂的疗效，结果表明，对于晚期非小细胞肺癌（NSCLC）患者，洛拉替尼相较于克唑替尼在临床疗效上有改善。因此，本研究的目的是评估洛拉替尼作为晚期ALK阳性（+）NSCLC患者一线治疗的成本效益。
从美国医保支付方的角度，采用微观模拟模型对既往未接受治疗的晚期ALK+ NSCLC患者进行了为期30年的全生命周期成本效益分析。基于CROWN试验，获取患者特征并估计转移概率。所有直接成本均来自官方来源和已发表文献。该模型的主要结果包括总成本、增量成本效益比（ICER）、质量调整生命年（QALY）和生命年（LYs）。进行了单向和概率敏感性分析以及多情景分析，以检验模型结果的稳健性。
在基础案例分析中，模拟了100万名患者，使用洛拉替尼或克唑替尼作为一线治疗的平均成本分别为909,758美元和616,230美元（增量成本：293,528美元），每位患者的平均生存期分别为4.81个QALY和4.09个QALY（增量QALY：0.72）。成本效益的主要驱动因素是药品价格和后续成本。概率敏感性分析（PAS）表明，当支付意愿（WTP）阈值提高到448,000美元/QALY时，与克唑替尼相比，洛拉替尼具有90%的成本效益。情景分析表明，当洛拉替尼价格降至原价的75%（424.5美元）时，与克唑替尼治疗相比，在WTP阈值为200,000美元/QALY时，洛拉替尼具有100%的成本效益。
在本研究中，对于既往未接受治疗的晚期ALK+ NSCLC患者，在支付意愿阈值为200,000美元/QALY的情况下，与克唑替尼相比，洛拉替尼不太可能具有成本效益。
