Lee Wei-Ling, Chou Wan-Hsuan, Chang Wei-Pin, Chang Tsung-Wei, Kuo Chun-Nan, Chang Wei-Chiao
Department of Pharmacy, Taipei Chang Gung Memorial Hospital, Taipei, Taiwan.
Department of Clinical Pharmacy, School of Pharmacy, College of Pharmacy, Taipei Medical University, Taipei, Taiwan.
J Food Drug Anal. 2024 Dec 15;32(4):568-576. doi: 10.38212/2224-6614.3536.
This study was aimed to evaluate the cost-effectiveness of pembrolizumab with chemotherapy (pembrolizumab combination therapy) and compare it with standard-of-care platinum-based chemotherapy (chemotherapy alone) as a first-line treatment for metastatic nonsquamous NSCLC from the perspective of Taiwan's third-party-payer public health-care system. We used a partitioned survival model with an estimated time horizon of 10 years. The partitioned survival model uses Kaplan-Meier estimates of progression-free and overall survival from the KEYNOTE-189 clinical trial. The quality-adjusted life-year (QALY) values were based on utility values by progression status calculated from the KEYNOTE-189 trial. This study examined costs related to treatment regimens, disease management, second-line therapy, end-of-life care, and adverse event management. Cost and utility were discounted at 3% per year. Probabilistic and deterministic sensitivity analyses were performed to test the robustness of the results. The willingness-to-pay threshold was set at 3 × Taiwan's gross domestic product (GDP), equivalent to NT$2,788,290. In the base-case scenario, pembrolizumab combination therapy resulted in an expected gain of 0.89 QALYs and an incremental cost of NT$2,201,203 relative to chemotherapy alone. The ICER was NT$2,478,601/QALY. In the analysis of the PD-L1 tumor proportion score (TPS) ≥ 50% subgroup, the patients who received pembrolizumab combination therapy gained 1.12 QALYs more than those who received chemotherapy alone, and the incremental cost was NT$2,522,528. Therefore, the ICER for this subset of patients was NT$2,258,358/QALY. In conclusion, pembrolizumab combination therapy is a cost-effective option for first-line treatment of metastatic nonsquamous NSCLC. The relative cost-effectiveness of pembrolizumab combination therapy is greatest for patients with PD-L1 TPS ≥50%.
本研究旨在从台湾第三方支付公共医疗系统的角度，评估帕博利珠单抗联合化疗（帕博利珠单抗联合疗法）的成本效益，并将其与标准治疗的铂类化疗（单纯化疗）作为转移性非鳞状非小细胞肺癌一线治疗方案进行比较。我们使用了一个分割生存模型，估计时间跨度为10年。该分割生存模型采用了KEYNOTE-189临床试验中无进展生存期和总生存期的Kaplan-Meier估计值。质量调整生命年（QALY）值基于KEYNOTE-189试验计算的按进展状态的效用值。本研究考察了与治疗方案、疾病管理、二线治疗、临终关怀和不良事件管理相关的成本。成本和效用按每年3%进行贴现。进行了概率性和确定性敏感性分析以检验结果的稳健性。支付意愿阈值设定为台湾国内生产总值（GDP）的3倍，相当于新台币2,788,290元。在基础情景下，相对于单纯化疗，帕博利珠单抗联合疗法导致预期获得0.89个QALY，增量成本为新台币2,201,203元。增量成本效果比为新台币2,478,601元/QALY。在对程序性死亡受体配体1（PD-L1）肿瘤比例评分（TPS）≥50%亚组的分析中，接受帕博利珠单抗联合疗法的患者比接受单纯化疗的患者多获得1.12个QALY，增量成本为新台币2,522,528元。因此，该亚组患者的增量成本效果比为新台币2,258,358元/QALY。总之，帕博利珠单抗联合疗法是转移性非鳞状非小细胞肺癌一线治疗的一种具有成本效益的选择。帕博利珠单抗联合疗法对PD-L1 TPS≥50%的患者相对成本效益最高。
