Cost-utility analysis of pembrolizumab combined with chemotherapy in the first-line treatment of advanced or unresectable biliary tract carcinoma

DING Ying; WANG Xuanhui; GONG Chunbo

doi:10.6039/j.issn.1001-0408.2023.23.13

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Cost-utility analysis of pembrolizumab combined with chemotherapy in the first-line treatment of advanced or unresectable biliary tract carcinoma

更新时间：2023-12-08

- Cost-utility analysis of pembrolizumab combined with chemotherapy in the first-line treatment of advanced or unresectable biliary tract carcinoma
- ZHONGGUO YAOFANG Vol. 34, Issue 23, Pages: 2891-2895(2023)
- 作者机构：
  
  山东中医药大学药学院，济南　250300
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2023.23.13
  CLC： R956;R735.8
- Published：15 December 2023，
  
  Received：29 April 2023，
  
  Revised：10 November 2023，
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丁樱,王旋辉,宫春博.帕博利珠单抗联合化疗方案一线治疗晚期或不可切除胆道恶性肿瘤的成本-效用分析 ^Δ[J].中国药房,2023,34(23):2891-2895.

DING Ying,WANG Xuanhui,GONG Chunbo.Cost-utility analysis of pembrolizumab combined with chemotherapy in the first-line treatment of advanced or unresectable biliary tract carcinoma[J].ZHONGGUO YAOFANG,2023,34(23):2891-2895.
丁樱,王旋辉,宫春博.帕博利珠单抗联合化疗方案一线治疗晚期或不可切除胆道恶性肿瘤的成本-效用分析 ^Δ[J].中国药房,2023,34(23):2891-2895. DOI： 10.6039/j.issn.1001-0408.2023.23.13.

DING Ying,WANG Xuanhui,GONG Chunbo.Cost-utility analysis of pembrolizumab combined with chemotherapy in the first-line treatment of advanced or unresectable biliary tract carcinoma[J].ZHONGGUO YAOFANG,2023,34(23):2891-2895. DOI： 10.6039/j.issn.1001-0408.2023.23.13.

摘要

目的

从我国卫生体系角度出发，评价帕博利珠单抗联合化疗方案对比安慰剂联合化疗方案一线治疗晚期或不可切除胆道恶性肿瘤（BTC）的经济性。

方法

根据KEYNOTE-966试验数据构建分区生存模型，模拟周期为21 d，模拟时限为患者终生。以质量调整生命年（QALY）为产出指标，采用成本-效用分析法评价上述两种方案的经济性。通过单因素敏感性分析和概率敏感性分析验证基础分析结果，并探讨有赠药方案情境下的经济性。

结果

基础分析结果显示，帕博利珠单抗组的成本与效果均高于安慰剂组，增量成本-效果比（ICER）为3 909 359.78元/QALY，高于以3倍我国2022年人均国内生产总值（GDP）即257 094元作为的意愿支付（WTP）阈值，证明没有经济性。单因素敏感性分析结果显示，效用贴现率、无进展生存状态效用值、成本贴现率、帕博利珠单抗成本等对ICER的影响较大。概率敏感性分析验证了基础分析结果的稳健性，并得出当WTP阈值大于1 500 000元/QALY时，帕博利珠单抗组方案开始具有经济性。情境分析结果表明，当考虑针对低收入人群的帕博利珠单抗慈善赠药方案时，虽然治疗成本大幅下降，但仍不具有经济性。

结论

在以3倍我国2022年人均GDP作为WTP阈值时，相比于安慰剂联合化疗方案，帕博利珠单抗联合化疗方案治疗晚期或不可切除BTC不具有经济性。

Abstract

OBJECTIVE

To evaluate the cost-effectiveness of pembrolizumab combined with chemotherapy versus placebo combined with chemotherapy in the first-line treatment of advanced or unresectable biliary tract carcinoma （BTC） from the perspective of China’s health system.

METHODS

A partitioned survival model was constructed based on the KEYNOTE-966 study data. The simulation period was 21 days， and the simulation time was the patient’s whole life. Using quality-adjusted life year （QALY） as the output indicator， the cost-utility analysis method was used to evaluate the cost-effectiveness of the two schemes mentioned above. Univariate and probabilistic sensitivity analyses were performed to verify the results of the basic analysis， and to explore the cost-effectiveness under the scenario of drug donation scheme.

RESULTS

The basic analysis showed that both the cost and effectiveness of the pembrolizumab group were higher than those of the placebo group， and the incremental cost-effectiveness ratio （ICER） was 3 909 359.78 yuan/QALY， which was higher than the willingness-to-pay （WTP） threshold of 3 times 2022 gross domestic product （GDP） per capita （257 094 yuan）， indicating no cost-effectiveness. The results of univariate sensitivity analysis showed that the utility discount rate， the utility value of progression-free survival （PFS） status， the cost discount rate， and the cost of pembrolizumab had a great influence on ICER. Probabilistic sensitivity analysis verified the robustness of the results of basic analysis， and concluded that when the WTP threshold was greater than 1 500 000 yuan/QALY， the pembrolizumab group became cost-effective. The results of the scenario analysis showed that considering the drug donation scheme of pembrolizumab for low-income people， although its treatment cost was significantly reduced， it was still not cost-effective.

CONCLUSIONS

At the WTP threshold of 3 times China’s GDP per capita in 2022， pembrolizumab combined with chemotherapy is not cost-effective compared with placebo combined with chemotherapy for advanced or unresectable BTC.

关键词

帕博利珠单抗分区生存模型胆道恶性肿瘤成本-效用分析药物经济学评价

Keywords

partitioned survival modelbiliary tract carcinomacost-utility analysispharmacoeconomic evaluation

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