Cost-utility analysis of enfortumab vedotin combined with pembrolizumab in the first-line treatment of advanced urothelial carcinoma

LU Qi; HUANG Jinyue; LING Xin; ZHANG Hongxu

doi:10.6039/j.issn.1001-0408.2025.20.11

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Cost-utility analysis of enfortumab vedotin combined with pembrolizumab in the first-line treatment of advanced urothelial carcinoma

更新时间：2025-10-25

- Cost-utility analysis of enfortumab vedotin combined with pembrolizumab in the first-line treatment of advanced urothelial carcinoma
- ZHONGGUO YAOFANG Vol. 36, Issue 20, Pages: 2548-2554(2025)
- 作者机构：
  
  徐州市肿瘤医院药剂科，江苏徐州　221005
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2025.20.11
  CLC： R956;R979.1
- Received：28 May 2025，
  
  Revised：2025-09-23，
  
  Accepted：25 September 2025，
  
  Published：30 October 2025
- 稿件说明：
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鹿琦,黄金岳,凌心,等.维恩妥尤单抗联合帕博利珠单抗一线治疗晚期尿路上皮癌的成本-效用分析 [J].中国药房,2025,36(20):2548-2554.

LU Qi,HUANG Jinyue,LING Xin,et al.Cost-utility analysis of enfortumab vedotin combined with pembrolizumab in the first-line treatment of advanced urothelial carcinoma[J].ZHONGGUO YAOFANG,2025,36(20):2548-2554.
鹿琦,黄金岳,凌心,等.维恩妥尤单抗联合帕博利珠单抗一线治疗晚期尿路上皮癌的成本-效用分析 [J].中国药房,2025,36(20):2548-2554. DOI： 10.6039/j.issn.1001-0408.2025.20.11.

LU Qi,HUANG Jinyue,LING Xin,et al.Cost-utility analysis of enfortumab vedotin combined with pembrolizumab in the first-line treatment of advanced urothelial carcinoma[J].ZHONGGUO YAOFANG,2025,36(20):2548-2554. DOI： 10.6039/j.issn.1001-0408.2025.20.11.

摘要

目的

评价维恩妥尤单抗联合帕博利珠单抗（简称“PemEV”）相较于吉西他滨联合顺铂或卡铂（简称“GP”）一线治疗晚期尿路上皮癌（aUC）的经济性。

方法

从中国卫生体系角度出发，基于EV-302试验泛亚洲亚组数据构建动态Markov模型，设研究时限为20年，循环周期为21 d，贴现率为5%。以直接医疗总成本及质量调整生命年（QALYs）为产出指标，计算PemEV方案相较于GP方案的增量成本-效果比（ICER）；通过敏感性分析和情境分析验证模型的稳健性，并测算PemEV方案相较于GP方案更具经济性时维恩妥尤单抗和帕博利珠单抗的价格阈值。

结果

成本-效用基础分析结果显示，相较于GP方案，PemEV方案可为aUC患者额外带来2.602 QALYs，但治疗成本增加了3 339 703.56元，ICER为1 283 554.39元/QALY，远超意愿支付（WTP）阈值（3倍我国2024年人均国内生产总值，287 247元/QALY）。单因素敏感性分析结果显示，PemEV方案总生存曲线Exponential分布的率参数值对ICER的影响最大。概率敏感性分析结果显示，在当前WTP阈值下，PemEV方案相较于GP方案更具经济性的可能性为0。情境分析结果显示，无论是将研究模型更改为分区生存模型，将研究时限分别设置为5、10、20年，还是将维恩妥尤单抗或（和）帕博利珠单抗的价格下调60%，PemEV方案相较于GP方案始终不具有经济学优势。若要使PemEV方案相较于GP方案更具经济性，维恩妥尤单抗和帕博利珠单抗的价格需同时下调78.65%，即单价分别降至55.71和38.26元/mg。

结论

基于我国卫生体系角度，PemEV方案相较于GP方案一线治疗aUC不具有经济学优势。

Abstract

OBJECTIVE

To evaluate the cost-utility of enfortumab vedotin combined with pembrolizumab （PemEV） versus gemcitabine combined with cisplatin or carboplatin （GP） in the first-line treatment of advanced urothelial carcinoma （aUC）.

METHODS

From the perspective of China’s health system， a dynamic Markov model was established based on the pan-Asian subgroup data from the EV-302 trial. The study timeframe was set at 20 years， with a cycle length of 21 days and a discount rate of 5%. Using total direct medical costs and quality-adjusted life years （QALYs） as outcome measures， the incremental cost-effectiveness ratio （ICER） of the PemEV regimen compared to the GP regimen was calculated. The robustness of the model was validated through sensitivity analysis and scenario analysis， and the price thresholds for enfortumab vedotin and pembrolizumab were estimated under conditions where the PemEV regimen was more cost-effective compared to the GP regimen.

RESULTS

Cost-utility analysis indicated that compared to the GP regimen， PemEV regimen could generate an additional 2.602 QALYs in aUC patients， but the treatment cost increased by 3 339 703.56 yuan， with an ICER of 1 283 554.39 yuan/QALY. This figure significantly exceeded the willingness-to-pay （WTP） threshold （3 times China’s gross domestic product per capita in 2024， 287 247 yuan/QALY）. The rate parameter of the exponential distribution fitted to the overall survival curve in the PemEV regimen had the greatest impact on ICER， according to the one-way sensitivity analysis. Probabilistic sensitivity analysis suggested that the PemEV regimen had no chance of being more cost-effective than the GP regimen at the current WTP threshold. Scenario analysis revealed that the PemEV regimen consistently lacked cost-utility advantage over the GP regimen， regardless of whether the study model was changed to a partitioned survival model， the study timeframe was set to 5， 10 or 20 years， or the prices of enfortumab vedotin and/or pembrolizumab were reduced by 60%. The prices of enfortumab vedotin and pembrolizumab should be simultaneously reduced by 78.65% （55.71 yuan/mg and 38.26 yuan/mg， respectively） when the PemEV regimen had a cost-utility advantage over the GP regimen.

CONCLUSIONS

From the perspective of China’s healthcare system， PemEV regimen does not demonstrate a cost-utility advantage over GP regimen in the first-line treatment of aUC.

关键词

Keywords

references

中国临床肿瘤学会指南工作委员会 . 中国临床肿瘤学会（CSCO）尿路上皮癌诊疗指南2024 [M ] . 北京：人民卫生出版社， 2024 ： 12-23，79-94，122-140，155-171 .

HEPP Z ， SHAH S N ， SMOYER K ， et al . Epidemiology and treatment patterns for locally advanced or metastatic urothelial carcinoma：a systematic literature review and gap analysis [J ] . J Manag Care Spec Pharm ， 2021 ， 27 （ 2 ）： 240 - 255 .

NADAL R ， VALDERRAMA B P ， BELLMUNT J . Pro-gress in systemic therapy for advanced-stage urothelial carcinoma [J ] . Nat Rev Clin Oncol ， 2024 ， 21 （ 1 ）： 8 - 27 .

HOFFMAN-CENSITS J ， MALDONADO L . Targeted treatment of locally advanced and metastatic urothelial cancer：enfortumab vedotin in context [J ] . Onco Targets Ther ， 2022 ， 15 ： 1519 - 1529 .

KATIMS A B ， REISZ P A ， NOGUEIRA L ， et al . Targeted therapies in advanced and metastatic urothelial carcinoma [J ] . Cancers（Basel）， 2022 ， 14 （ 21 ）： 5431 .

CHALLITA-EID P M ， SATPAYEV D ， YANG P ， et al . Enfortumab vedotin antibody-drug conjugate targeting nectin-4 is a highly potent therapeutic agent in multiple preclinical cancer models [J ] . Cancer Res ， 2016 ， 76 （ 10 ）： 3003 - 3013 .

RUCK J M ， BRODERICK S R . Neoadjuvant and adjuvant pembrolizumab for the treatment of early-stage resectable non-small cell lung cancer： an editorial regarding the interim data analysis of the KEYNOTE-671 phase Ⅲ trial of neoadjuvant and adjuvant pembrolizumab [J ] . Ann Surg Oncol ， 2024 ， 31 （ 1 ）： 4 - 5 .

OLSON D ， YOUNAN P ， LIU B ， et al . 1187Enfortumab vedotin induces immunogenic cell death，elicits antitumor immune memory，and shows enhanced preclinical activity in combination with immune checkpoint inhibitors [J ] . J Immuno Ther Cancer ， 2022 ， 10 （ Suppl. 2 ）： A1231 - A1231 .

POWLES T ， VALDERRAMA B P ， GUPTA S ， et al . Enfortumab vedotin and pembrolizumab in untreated advanced urothelial cancer [J ] . N Engl J Med ， 2024 ， 390 （ 10 ）： 875 - 888 .

KIKUCHI E ， VAN DER HEIJDEN M S ， VALDERRAMA B P ， et al . 269O enfortumab vedotin plus pembrolizumab（EV+P）vs chemotherapy（Chemo）in previously untreated locally advanced or metastatic urothelial carcinoma（la/mUC）：pan-Asian subgroup analysis from EV-302/KEYNOTE-A39 [J ] . Ann Oncol ， 2024 ， 35 ： S1507 - S1508 .

European Society for Medical Oncology . Enfortumab vedotin plus pembrolizumab（EV+P）vs chemotherapy（Chemo）in previously untreated locally advanced or metastatic urothelial carcinoma（la/mUC）：pan-Asian subgroup analysis from EV-302/KEYNOTE-A39 [EB/OL ] .（ 2024-12-07 ）[ 2025-03-10 ] . https：//oncologypro.esmo.org/congress-resources/esmo-asia-congress-2024session=proffered_paper_session_genitourinary_tumours_59dd090 https://oncologypro.esmo.org/congress-resources/esmo-asia-congress-2024session=proffered_paper_session_genitourinary_tumours_59dd090 .

ZHU Y W ， LIU K ， ZHU H ， et al . Enfortumab vedotin plus pembrolizumab for previously untreated locally advanced or metastatic urothelial carcinoma：a cost-effectiveness analysis [J ] . Ther Adv Med Oncol ， 2025 ， 17 ： 1758835 - 9241295544 .

YOU M J ， ZHENG Q Y ， HE Y . Enfortumab vedotin plus pembrolizumab as a first-line treatment for advanced urothelial carcinoma：a cost-effectiveness analysis from China based on the EV-302 trial [J ] . Front Pharmacol ， 2024 ， 15 ： 1412292 .

药智网 . 药品采购中标价格 [EB/OL ] .（ 2025-02-28 ）[ 2025-03-10 ] . https：//www.yaozh.com/yaopinzhongbiao https://www.yaozh.com/yaopinzhongbiao .

GRANDE E ， ARRANZ J Á ， DE SANTIS M ， et al . Atezolizumab plus chemotherapy versus placebo plus chemotherapy in untreated locally advanced or metastatic urothelial carcinoma（IMvigor130）：final overall survival analysis results from a randomised，controlled，phase 3 study [J ] . Lancet Oncol ， 2024 ， 25 （ 1 ）： 29 - 45 .

National Comprehensive Cancer Network . NCCN clinical practice guidelines in oncology：bladder cancer [EB/OL ] .（ 2024-05-09 ）[ 2025-03-10 ] . https：//www.nccn.org/professionals/physician _gls/pdf/bladder.pdf https://www.nccn.org/professionals/physician_gls/pdf/bladder.pdf .

BELLMUNT J ， DE WIT R ， VAUGHN D J ， et al . Pembrolizumab as second-line therapy for advanced urothelial carcinoma [J ] . N Engl J Med ， 2017 ， 376 （ 11 ）： 1015 - 1026 .

刘国恩 . 中国药物经济学评价指南2020 [M ] . 北京：中国市场出版社， 2020 ： 45-47，71-76 .

国家统计局 . 国家数据 [EB/OL ] .（ 2024-12-31 ）[ 2025-04-21 ] . https：//data.stats.gov.cn/sj/ https://data.stats.gov.cn/sj/ .

SMITH D H ， GRAVELLE H . The practice of discounting in economic evaluations of healthcare interventions [J ] . Int J Technol Assess Health Care ， 2001 ， 17 （ 2 ）： 236 - 243 .

GUYOT P ， ADES A E ， OUWENS M J N M ， et al . Enhanced secondary analysis of survival data：reconstructing the data from published Kaplan-Meier survival curves [J ] . BMC Med Res Methodol ， 2012 ， 12 ： 9 .

周挺，马爱霞 . 生存分析在药物经济学评价Markov模型转移概率计算中的应用 [J ] . 中国循证医学杂志， 2018 ， 18 （ 10 ）： 1129 - 1134 .

WU B ， LI T ， CAI J ， et al . Cost-effectiveness analysis of adjuvant chemotherapies in patients presenting with gastric cancer after D2 gastrectomy [J ] . BMC Cancer ， 2014 ， 14 ： 984 .

赵连莹 . 晚期非小细胞肺癌一线治疗方案的药物经济学评价 [D ] . 南京：南京医科大学， 2012 .

BALAR A V ， CASTELLANO D ， O’DONNELL P H ， et al . First-line pembrolizumab in cisplatin-ineligible patients with locally advanced and unresectable or metastatic urothelial cancer（KEYNOTE-052）：a multicentre，single-arm，phase 2 study [J ] . Lancet Oncol ， 2017 ， 18 （ 11 ）： 1483 - 1492 .

PATTERSON K ， PRABHU V ， XU R F ， et al . Cost-effectiveness of pembrolizumab for patients with advanced，unresectable，or metastatic urothelial cancer ineligible for cisplatin-based therapy [J ] . Eur Urol Oncol ， 2019 ， 2 （ 5 ）： 565 - 571 .

LIU S X ， DOU L ， WANG K X ， et al . Cost-effectiveness analysis of nivolumab combination therapy in the first-line treatment for advanced esophageal squamous-cell carcinoma [J ] . Front Oncol ， 2022 ， 12 ： 899966 .

LIU Q ， TAN C Q ， YI L D ， et al . Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line therapy for extensive-stage small-cell lung cancer [J ] . PLoS One ， 2021 ， 16 （ 11 ）： e0258605 .

孙利华，吴晶 . 药物经济学 [M ] . 北京：人民卫生出版社， 2022 ： 141 - 145 .

LATIMER N R . Survival analysis for economic evaluations alongside clinical trials：extrapolation with patient-level data：inconsistencies，limitations，and a practical guide [J ] . Med Decis Making ， 2013 ， 33 （ 6 ）： 743 - 754 .

EDLIN R ， MCCABE C ， HULME C ， et al . Cost effectiveness modelling for health technology assessment：a practical course [M ] . Cham ： Springer International Publishing ， 2015 ：98， 102 ， 120 - 129 .

CRANMER H ， SHIELDS G E ， BULLEMENT A . A comparison of partitioned survival analysis and state transition multi-state modelling approaches using a case study in oncology [J ] . J Med Econ ， 2020 ， 23 （ 10 ）： 1176 - 1185 .

胡善联 . 中国医保药品价格谈判回顾和展望 [J ] . 卫生经济研究， 2024 ， 41 （ 1 ）： 9 - 13 .

孟蕊，芮明军，王欣恬，等 . 不同决策分析模型在抗肿瘤药物经济学评价中的应用：以加拿大CADTH为例 [J ] . 中国药房， 2021 ， 32 （ 14 ）： 1752 - 1757 .

CHIDDARWAR T V ， JALAL H ， ALARID-ESCUDERO F ， et al . Cost-effectiveness of enfortumab vedotin and pembrolizumab for first-line metastatic urothelial cancer in the United States [J ] . Value Health ， 2025 ， 28 （ 7 ）： 1009 - 1017 .

LI A D ， WU M Y ， OUYANG X ， et al . Cost-effectiveness of first-line enfortumab vedotin in addition to pembrolizumab for metastatic urothelial carcinoma in the United States [J ] . Front Immunol ， 2024 ， 15 ： 1464092 .

RIEGER C ， SCHLÜCHTERMANN J ， LEHMANN M ， et al . Cost-effectiveness analysis in the new era of treatment strategies in metastatic urothelial carcinoma based on Checkmate-901 and EV302/Keynote-A39 [J ] . Eur Urol Oncol ， 2025 ， 8 （ 3 ）： 681 - 688 .

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