由于频繁的干预和昂贵的随访，转移性尿路上皮癌 (mUCa) 被列为每位患者治疗成本最高的癌症。在研究一线疗法时，与标准治疗（SoC；吉西他滨/顺铂）相比，enfortumab vedotin pembrolizumab (EV P) 和吉西他滨/顺铂 nivolumab 等组合表现出显着的总体生存获益。在这里，我们对 mUCa 进行了成本效益分析。我们从付款人的角度开发了马尔可夫模型，过滤了来自 3 期 Checkmate-901 和 EV302/Keynote-A39 试验的临床数据。德国和美国使用蒙特卡罗模拟从社会经济角度确定最佳治疗方案。最后，我们比较了不同支付意愿 (WTP) 阈值下每种方式的增量成本效益比 (ICER)。 在生命周期中，SoC、吉西他滨/顺铂 nivolumab 和 EV P 与平均成本 € 163 424（美国：458 006 美元）、206 853 欧元（美国：597 802 美元）和 401 170 欧元（美国：1 228 455 美元），质量调整生命年 (QALY) 分别为 1.21、1.71 和 2.31 。新策略的 ICER 为 87 340 欧元（美国：281 142 美元；吉西他滨/顺铂 nivolumab）和 216 140 欧元（美国：700 448 美元；EV P）。在常用的 WTP 门槛为 100 000 欧元/美元时，吉西他滨/顺铂 nivolumab 将是德国的最佳策略，而 EV P 需要降价 46%（美国：82%）才能实现成本效益。QALY 几乎翻倍EV P与当前SoC对比；然而，从严格的社会经济角度来看，目前的成本可能并不合理。尽管吉西他滨/顺铂纳武单抗的肿瘤学益处较低，但由于其良好的成本效益，尤其是在欧洲，应考虑将其用于一线治疗。确定个体危险因素对于优化未来的治疗反应和治疗成本至关重要。本报告对转移性尿路上皮癌的新兴治疗方案进行了成本效益分析。 enfortumab vedotin pembrolizumab 组合成为最有效的治疗方法；然而，它也被证明是最昂贵的。从纯粹的社会经济角度来看，吉西他滨/顺铂和纳武单抗的组合至少在德国是一种具有成本效益的替代方案。版权所有 © 2024 作者。由 Elsevier B.V. 出版。保留所有权利。

Metastatic urothelial carcinoma (mUCa) ranks as the costliest cancer to treat per patient due to frequent interventions and expensive follow-ups. Investigating first-line therapies, combinations such as enfortumab vedotin + pembrolizumab (EV + P) and gemcitabine/cisplatin + nivolumab exhibit significant overall survival benefits compared with the standard treatment (SoC; gemcitabine/cisplatin). Here, we conducted a cost-effectiveness analysis for mUCa.We developed a Markov model from a payer perspective, filtering clinical data from the phase 3 Checkmate-901 and EV302/Keynote-A39 trials. Monte Carlo simulation was used to identify the optimal treatment from a socioeconomic perspective in Germany and the USA. Finally, we compared the incremental cost-effectiveness ratio (ICER) of each modality at different willingness-to-pay (WTP) thresholds.At a lifetime horizon, SoC, gemcitabine/cisplatin + nivolumab, and EV + P were associated with average costs of €163 424 (USA: $458 006), €206 853 (USA: $597 802), and €401 170 (USA: $1 228 455), and gained quality-adjusted life years (QALYs) of 1.21, 1.71, and 2.31, respectively. The ICERs of the newer strategies were €87 340 (USA: $281 142; gemcitabine/cisplatin + nivolumab) and €216 140 (USA: $700 448; EV + P). At a commonly used WTP threshold of €/$100 000, gemcitabine/cisplatin + nivolumab would be the optimal strategy in Germany, while EV + P would require a price reduction of 46% (USA: 82%) to be cost effective.QALYs nearly double with EV + P compared with the current SoC; yet, current costs may not be justified from a strict socioeconomic perspective. Despite its lower oncological benefit, gemcitabine/cisplatin + nivolumab should be considered for first-line therapy due to favorable cost effectiveness, especially in Europe. Establishing individual risk factors is essential for optimizing therapeutic response and treatment costs in the future.This report presents a cost-effectiveness analysis of emerging treatment options for metastatic urothelial carcinoma. The combination of enfortumab vedotin + pembrolizumab emerged as the most effective treatment; however, it also proved to be the costliest. From a purely socioeconomic standpoint, the combination of gemcitabine/cisplatin and nivolumab represents a cost-effective alternative at least in Germany.Copyright © 2024 The Authors. Published by Elsevier B.V. All rights reserved.