Institute for Technology Assessment, Massachusetts General Hospital, Boston, MA, United States of America.
Division of General Internal Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, United States of America.
PLoS One. 2020 Jan 29;15(1):e0228288. doi: 10.1371/journal.pone.0228288. eCollection 2020.
OBJECTIVES: While previous cost-effectiveness studies on pembrolizumab in stage IV non-small cell lung cancer (NSCLC) have found these regimens to be cost-effective, their reliance on randomized controlled trial (RCT) data with strict inclusion criteria limits generalizability to patients with comorbidities. We estimated the cost-effectiveness of first-line pembrolizumab for patients with various comorbidities. MATERIALS AND METHODS: In our base case analysis, we studied pembrolizumab plus chemotherapy (pembrolizumab combination therapy) versus chemotherapy alone. In a secondary analysis, we considered only patients with PD-L1 expression of at least 50% (PD-L1-high) and evaluated pembrolizumab monotherapy, pembrolizumab combination therapy, and chemotherapy alone. Microsimulation models were developed for the base case and the PD-L1-high analyses. To estimate outcomes of patients with differing comorbidities, we combined survival data from patients with few or no comorbidities from the RCTs with estimates from the general population obtained from the Surveillance, Epidemiology, and End Results (SEER)-Medicare database. Comorbidity burden level was divided into three groups based on the Charlson score (equal to 0, 1, or 2+); patients with various other specific comorbidities were also analyzed. Incremental cost-effectiveness ratios (ICER) were compared to a willingness-to-pay (WTP) threshold of $100,000/quality-adjusted life-year (QALY). RESULTS: In the Charlson 0, Charlson 1, and Charlson 2+ patient populations, estimated ICERs for pembrolizumab combination therapy in the base case model were $173,919/QALY, $175,165/QALY, and $181,777/QALY, respectively, compared to chemotherapy. In the PD-L1-high model, the Charlson 0, Charlson 1, and Charlson 2+ patients had ICERs of $147,406/QALY, $149,026/QALY, and $154,521/QALY with pembrolizumab combination therapy versus chemotherapy. Pembrolizumab monotherapy was weakly dominated for each comorbidity group in the PD-L1-high model. CONCLUSION: For patients with stage IV NSCLC and varying comorbidity burden, first-line treatment with pembrolizumab does not represent a cost-effective strategy compared to chemotherapy. Resources should be focused on collecting immunotherapy survival data for more representative NSCLC patient populations.
目的：先前关于 pembrolizumab 在 IV 期非小细胞肺癌（NSCLC）中的成本效益研究发现，这些方案具有成本效益，但它们依赖于具有严格纳入标准的随机对照试验（RCT）数据，这限制了它们在伴有合并症的患者中的推广。我们评估了一线 pembrolizumab 治疗各种合并症患者的成本效益。
材料和方法：在我们的基本案例分析中，我们研究了 pembrolizumab 联合化疗（pembrolizumab 联合治疗）与单纯化疗的比较。在二次分析中，我们仅考虑 PD-L1 表达至少为 50%（PD-L1 高）的患者，并评估了 pembrolizumab 单药治疗、pembrolizumab 联合治疗和单纯化疗。为基本案例和 PD-L1 高分析建立了微观模拟模型。为了估计具有不同合并症的患者的结局，我们将来自 RCT 中合并症少或无的患者的生存数据与来自监测、流行病学和最终结果（SEER）-医疗保险数据库的一般人群中的估计相结合。根据 Charlson 评分（等于 0、1 或 2+）将合并症负担水平分为三组；还分析了具有各种其他特定合并症的患者。增量成本效益比（ICER）与 100,000 美元/QALY 的支付意愿（WTP）阈值进行比较。
结果：在 Charlson 0、Charlson 1 和 Charlson 2+患者人群中，基本案例模型中 pembrolizumab 联合治疗的估计 ICER 分别为 173,919/QALY、175,165/QALY 和 181,777/QALY，与化疗相比。在 PD-L1 高模型中，Charlson 0、Charlson 1 和 Charlson 2+患者的 pembrolizumab 联合治疗与化疗相比的 ICER 分别为 147,406/QALY、149,026/QALY 和 154,521/QALY。在 PD-L1 高模型中，对于每个合并症组，pembrolizumab 单药治疗均为弱占优。
结论：对于伴有不同合并症负担的 IV 期 NSCLC 患者，与化疗相比，一线治疗使用 pembrolizumab 并非具有成本效益的策略。应集中精力为更具代表性的 NSCLC 患者群体收集免疫治疗生存数据。
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