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从美国医保支付方角度进行的度伐利尤单抗联合曲美木单抗与索拉非尼用于不可切除肝细胞癌一线治疗的成本效益分析

Tremelimumab plus durvalumab versus sorafenib in first-line treatment of unresectable hepatocellular carcinoma: a cost-effectiveness analysis from the US payer perspective.

作者信息

Cheng Shuqiao, Li Bin, Tang Lanhua, Liu Shao, Xiao Jian

机构信息

Department of Pharmacy, Xiangya Hospital Central South University, Changsha, Hunan, China.

Department of Oncology, Xiangya Hospital Central South University, Changsha, Hunan, China.

出版信息

BMJ Open. 2025 Apr 29;15(4):e090992. doi: 10.1136/bmjopen-2024-090992.

Abstract

OBJECTIVE

In a recently published 4-year overall survival (OS) update from the phase III clinical trial named HIMALAYA (NCT03298451), single tremelimumab plus regular interval durvalumab (a regimen termed STRIDE) demonstrated significantly improved OS compared with sorafenib in the first-line setting of unresectable hepatocellular carcinoma (uHCC). Although dual immunotherapy represents a novel treatment option for uHCC, the economic implications of these high-priced drugs require further exploration. This study aimed to evaluate the cost-effectiveness of STRIDE in uHCC to inform first-line treatment decisions and help allocate medical resources most effectively.

DESIGN

Using a partitioned survival model, we conducted a cost-effectiveness analysis comparing STRIDE to sorafenib in the first-line treatment of uHCC. Clinical information was gathered from the phase III HIMALAYA trial. Costs and health state utilities data were derived from previous literature. Uncertainty of the model was assessed through one-way sensitivity analysis and probabilistic sensitivity analysis.

OUTCOME MEASURES

Total costs, life years, quality-adjusted life years (QALYs), incremental QALYs and incremental cost-effectiveness ratio (ICER).

SETTING

US payer perspective.

PARTICIPANTS

393 participants in the STRIDE group and 389 participants in the sorafenib group who were diagnosed with uHCC and had no previous systemic treatment.

INTERVENTIONS

Single-dose tremelimumab plus monthly durvalumab (STRIDE) versus sorafenib.

RESULTS

Treatment with STRIDE provided an additional 0.51 QALYs at an incremental total cost of United States dollar ($)9812. The ICER of STRIDE was $19 239 per QALY compared with sorafenib, which falls below the willingness-to-pay threshold of $150 000 per QALY. Sensitivity analyses indicated that our results were robust to the variation ranges of key inputs.

CONCLUSION

In this economic evaluation comparing two first-line systemic therapies for uHCC patients, STRIDE was cost-effective compared with sorafenib from a US payer perspective. Our study is the first to demonstrate that immunotherapy can provide both survival benefits and economic viability in uHCC.

摘要

目的

在一项名为HIMALAYA(NCT03298451)的III期临床试验最近公布的4年总生存期(OS)更新中,单药曲美木单抗加定期度伐利尤单抗(一种称为STRIDE的方案)在不可切除肝细胞癌(uHCC)一线治疗中与索拉非尼相比,OS有显著改善。尽管双重免疫疗法是uHCC的一种新型治疗选择,但这些高价药物的经济影响需要进一步探索。本研究旨在评估STRIDE方案治疗uHCC的成本效益,为一线治疗决策提供参考,并帮助最有效地分配医疗资源。

设计

使用分区生存模型,我们进行了一项成本效益分析,比较STRIDE方案与索拉非尼在uHCC一线治疗中的效果。临床信息来自III期HIMALAYA试验。成本和健康状态效用数据来自先前的文献。通过单向敏感性分析和概率敏感性分析评估模型的不确定性。

观察指标

总成本、生命年、质量调整生命年(QALY)、增量QALY和增量成本效益比(ICER)。

研究背景

美国支付方视角。

研究对象

STRIDE组393名和索拉非尼组389名被诊断为uHCC且既往未接受过全身治疗的参与者。

干预措施

单剂量曲美木单抗加每月一次度伐利尤单抗(STRIDE)与索拉非尼。

结果

STRIDE方案治疗可额外获得0.51个QALY,增量总成本为9812美元。与索拉非尼相比,STRIDE方案的ICER为每QALY 19239美元,低于每QALY 150000美元的支付意愿阈值。敏感性分析表明,我们的结果对关键输入的变化范围具有稳健性。

结论

在这项比较uHCC患者两种一线全身治疗方案的经济学评估中,从美国支付方的角度来看,与索拉非尼相比,STRIDE方案具有成本效益。我们的研究首次证明免疫疗法在uHCC中既能带来生存益处又具有经济可行性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9933/12049948/22ffa68dbc60/bmjopen-15-4-g001.jpg

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