帕博利珠单抗联合化疗与单纯化疗作为日本不可切除晚期食管癌患者一线化疗的成本效益分析

Cost-effectiveness analysis of pembrolizumab plus chemotherapy versus chemotherapy as first line chemotherapy for patients with unresectable advanced esophageal cancer in Japan.

作者信息

Kakinuma Hirohito, Takada Daisuke, Itoshima Hisashi, Kunisawa Susumu, Moriwaki Kensuke, Honda Michitaka, Fushimi Kiyohide, Imanaka Yuichi

机构信息

Department of Healthcare Economics and Quality Management, School of Public Health, Kyoto University Graduate School of Medicine, Kyoto, Japan.

Department of Minimally Invasive Surgical and Medical Oncology, Fukushima Medical University, Fukushima, Japan.

出版信息

Esophagus. 2025 Jul 12. doi: 10.1007/s10388-025-01144-5.

DOI:10.1007/s10388-025-01144-5

PMID:40652068

Abstract

BACKGROUND

Although pembrolizumab plus chemotherapy (Pembro + Chemo) is the recommended first-line therapy for advanced esophageal cancer, it remains unclear whether it is more cost-effective than chemotherapy (Chemo). We evaluated the cost-effectiveness of Pembro + Chemo versus Chemo from a Japanese healthcare payer perspective.

METHODS

A partitioned survival analysis model was developed to predict costs and quality-adjusted life years (QALY) for Pembro + Chemo and Chemo. The model parameters were based on a previous randomized controlled trial and a nationwide administrative database in Japan. The incremental cost-effectiveness ratio (ICER) of Pembro + Chemo compared with Chemo was estimated. A subgroup analysis on the level of PD-L1 Combined Positive Score (CPS) ≥ 10 was also conducted. Additionally, one-way deterministic sensitivity analysis and probabilistic sensitivity analysis with Monte Carlo simulations (5,000 simulations) were conducted.

RESULTS

The ICER of Pembro + Chemo over Chemo was estimated at USD 176,479 per QALY. The subgroup analysis for patients with PD-L1 CPS ≥ 10 showed an ICER of USD 126,862 per QALY. One-way deterministic sensitivity analysis demonstrated that the ICER remained above the willingness-to-pay threshold of USD 50,000-100,000 per QALY in all scenarios, with the utility of progression-free survival in the Pembro + Chemo group having the greatest influence on the ICER. Probabilistic sensitivity analysis showed that the probability of Pembro + Chemo being cost-effective was 0% at the willingness-to-pay threshold.

CONCLUSION

The addition of pembrolizumab to chemotherapy was not cost-effective in treating advanced esophageal cancer in Japan.

摘要

背景

尽管帕博利珠单抗联合化疗（帕博利珠单抗 + 化疗）是晚期食管癌推荐的一线治疗方案，但它是否比单纯化疗更具成本效益仍不明确。我们从日本医疗支付方的角度评估了帕博利珠单抗 + 化疗与单纯化疗的成本效益。

方法

建立了一个分区生存分析模型，以预测帕博利珠单抗 + 化疗和单纯化疗的成本及质量调整生命年（QALY）。模型参数基于先前的一项随机对照试验和日本的一个全国性行政数据库。估计了帕博利珠单抗 + 化疗与单纯化疗相比的增量成本效益比（ICER）。还对程序性死亡受体配体 1（PD-L1）联合阳性评分（CPS）≥10 的水平进行了亚组分析。此外，进行了单向确定性敏感性分析和蒙特卡洛模拟（5000 次模拟）的概率敏感性分析。

结果

帕博利珠单抗 + 化疗相对于单纯化疗的 ICER 估计为每 QALY 176,479 美元。对 PD-L1 CPS≥10 的患者进行的亚组分析显示，ICER 为每 QALY 126,862 美元。单向确定性敏感性分析表明，在所有情况下，ICER 均高于每 QALY 50,000 - 100,000 美元的支付意愿阈值，帕博利珠单抗 + 化疗组无进展生存期的效用对 ICER 影响最大。概率敏感性分析表明，在支付意愿阈值下，帕博利珠单抗 + 化疗具有成本效益的概率为 0%。