Zhou Zheng-Yi, Mutebi Alex, Han Simeng, Bensimon Arielle G, Louise Ricculli Marie, Xie Jipan, Dalal Anand, Culver Ken
a Analysis Group, Inc. , New York , NY , USA.
b Novartis Pharmaceuticals Corporation , East Hanover , NJ , USA.
J Med Econ. 2018 Jun;21(6):577-586. doi: 10.1080/13696998.2018.1443111. Epub 2018 Mar 12.
To assess the cost-effectiveness of first-line ceritinib vs crizotinib and platinum doublet chemotherapy for anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) from a US third-party payer's perspective.
A partitioned survival model with three health states (stable disease, progressive disease, death) was developed over a 20-year time horizon. Ceritinib's efficacy inputs (progression-free and overall survival) were estimated from ASCEND-4; parametric survival models extrapolated data beyond the trial period. The relative efficacy of ceritinib vs chemotherapy was obtained from ASCEND-4, the relative efficacy of ceritinib vs crizotinib was estimated using a matching-adjusted indirect comparison based on ASCEND-4 and PROFILE 1014. Drug acquisition, treatment administration, adverse event management, and medical costs were obtained from publicly available databases and the literature, and inflated to 2016 US dollars. Treatment-specific stable-state utilities were derived from trials and progressive-state utility from the literature. Incremental costs per quality-adjusted life year (QALY) were estimated for ceritinib vs each comparator. Cost-effectiveness was assessed based on US willingness-to-pay thresholds. Deterministic and probabilistic sensitivity analyses were performed to test model robustness.
In the base case, first-line ceritinib was associated with total direct costs of $299,777 and 3.28 QALYs (from 4.61 life years gained [LYG]) over 20 years. First-line crizotinib and chemotherapy were associated with 2.73 and 2.41 QALYs, 3.92 and 3.53 LYG, and $263,172 and $228,184 total direct costs, respectively. The incremental cost per QALY gained was $66,064 for ceritinib vs crizotinib and $81,645 for ceritinib vs chemotherapy. In the first 2 years following treatment initiation, ceritinib dominated crizotinib by conferring greater health benefits at reduced total costs. Results were robust to deterministic and probabilistic sensitivity analyses.
In the absence of head-to-head trials, an indirect comparison method was used.
Ceritinib is cost-effective compared to crizotinib and chemotherapy in the treatment of previously untreated ALK-positive metastatic NCSLC in the US.
从美国第三方支付方的角度评估一线色瑞替尼与克唑替尼及铂类双联化疗方案用于间变性淋巴瘤激酶（ALK）阳性转移性非小细胞肺癌（NSCLC）的成本效益。
构建了一个具有三种健康状态（疾病稳定、疾病进展、死亡）的分割生存模型，时间跨度为20年。色瑞替尼的疗效数据（无进展生存期和总生存期）来自ASCEND - 4试验；参数生存模型对试验期外的数据进行了外推。色瑞替尼与化疗的相对疗效来自ASCEND - 4试验，色瑞替尼与克唑替尼的相对疗效通过基于ASCEND - 4试验和PROFILE 1014试验的匹配调整间接比较进行估算。药品采购、治疗管理、不良事件处理及医疗成本数据来自公开可用数据库及文献，并换算为2016年美元。特定治疗的稳定状态效用值来自试验，进展状态效用值来自文献。估算了色瑞替尼与各对照方案相比每获得一个质量调整生命年（QALY）的增量成本。基于美国支付意愿阈值评估成本效益。进行了确定性和概率性敏感性分析以检验模型的稳健性。
在基础案例中，一线色瑞替尼在20年内的总直接成本为299,777美元，获得3.28个QALY（从获得的4.61个生命年[LYG]计算得出）。一线克唑替尼和化疗分别与2.73和2.41个QALY、3.92和3.53个LYG以及263,172美元和228,184美元的总直接成本相关。色瑞替尼与克唑替尼相比每获得一个QALY的增量成本为66,064美元，与化疗相比为81,645美元。在开始治疗后的前两年，色瑞替尼以更低的总成本带来更大的健康效益，优于克唑替尼。结果在确定性和概率性敏感性分析中具有稳健性。
由于缺乏头对头试验，采用了间接比较方法。
在美国，对于既往未治疗的ALK阳性转移性NSCLC，色瑞替尼与克唑替尼及化疗相比具有成本效益。
