Cost-Effectiveness Analysis of Toripalimab Plus Axitinib for Patients with Advanced Renal Cell Carcinoma in the United States.

IF 2.7 3区 医学 Q2 PHARMACOLOGY & PHARMACY
Clinical Drug Investigation Pub Date : 2025-08-01 Epub Date: 2025-07-19 DOI:10.1007/s40261-025-01464-5
Ibrahim Alfayoumi, Asmita Priyadarshini Khatiwada, Surachat Ngorsuraches
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引用次数: 0

Abstract

Objective: The RENOTORCH trial found that toripalimab plus axitinib extended progression-free and overall survival in patients with advanced renal cell carcinoma (RCC), though its financial burden may limit widespread use. This study aimed to assess the cost-effectiveness of toripalimab plus axitinib compared with sunitinib monotherapy as a first-line therapy for patients with previously untreated or intermediate- or poor-risk advanced RCC from a US third-party payer perspective.

Methods: A three-state partitioned survival model (progression-free, progression, death) was utilized, with clinical outcomes obtained from the RENOTORCH trial. Progression-free survival (PFS) and overall survival (OS) were modeled using various parametric functions over a 5-year horizon, applying a 3% annual discount rate. Costs of treatments, administration, monitoring, and management of grade 3/4 adverse events (≥ 5% occurrence) were sourced from Micromedex® and Centers for Medicare & Medicaid Services (CMS) databases. Life years (LY), quality-adjusted life years (QALY), and incremental costs per LY and QALY were estimated. One-way and probabilistic sensitivity analyses were conducted. Subgroup analyses for intermediate- and poor-risk patients, as classified by the International Metastatic RCC Database Consortium (IMDC) criteria, were performed using similar methods.

Results: Toripalimab plus axitinib increased total costs by $332,359, gained 0.68 LY and 0.36 QALY compared with sunitinib, resulting in incremental costs of $489,747 per LY and $923,962 per QALY. One-way sensitivity analysis showed that the incremental cost per QALY was most sensitive to changes in toripalimab plus axitinib's cost. At a $150,000 willingness-to-pay threshold, probabilistic sensitivity analysis showed a nearly 0% probability of toripalimab plus axitinib being cost-effective. Similarly, toripalimab plus axitinib was still not cost-effective for intermediate- and poor-risk patients.

Conclusions: Compared with sunitinib monotherapy, our study suggested that toripalimab plus axitinib was not cost-effective for patients with advanced renal cell carcinoma from a US third-party payer perspective. Further analyses are warranted when more data are available. Despite benefits across different risk groups, toripalimab plus axitinib was not cost-effective for intermediate- and poor-risk patients.

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在美国,托利帕单抗联合阿西替尼治疗晚期肾细胞癌患者的成本-效果分析。
目的:RENOTORCH试验发现,托利帕单抗联合阿西替尼延长了晚期肾细胞癌(RCC)患者的无进展生存期和总生存期,尽管其经济负担可能限制了其广泛使用。本研究旨在从美国第三方付款人的角度评估托帕利单抗加阿西替尼与舒尼替尼单药作为一线治疗的成本效益,用于先前未治疗或中低风险晚期RCC患者。方法:采用三状态分区生存模型(无进展、进展、死亡),并从RENOTORCH试验中获得临床结果。无进展生存期(PFS)和总生存期(OS)使用5年的各种参数函数进行建模,采用3%的年贴现率。治疗、管理、监测和管理3/4级不良事件(发生率≥5%)的费用来源于Micromedex®和美国医疗保险和医疗补助服务中心(CMS)数据库。评估生命年(LY)、质量调整生命年(QALY)以及每LY和QALY的增量成本。进行了单向和概率敏感性分析。根据国际转移性RCC数据库联盟(IMDC)标准分类的中危和低危患者的亚组分析使用类似的方法进行。结果:与舒尼替尼相比,托利帕单抗联合阿西替尼使总成本增加332,359美元,LY增加0.68美元,QALY增加0.36美元,每LY增加成本489,747美元,QALY增加成本923,962美元。单向敏感性分析显示,每QALY的增量成本对托帕利单抗加阿西替尼成本的变化最为敏感。在15万美元的支付意愿阈值下,概率敏感性分析显示,托帕利单抗加阿西替尼具有成本效益的概率接近0%。同样,托帕利单抗加阿西替尼对于中、低风险患者仍然没有成本效益。结论:与舒尼替尼单药治疗相比,我们的研究表明,从美国第三方付款人的角度来看,托帕利单抗加阿西替尼治疗晚期肾细胞癌患者不具有成本效益。当获得更多数据时,需要进一步分析。尽管在不同的风险人群中获益,但托帕利单抗加阿西替尼对中危和低危患者没有成本效益。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
5.90
自引率
3.10%
发文量
108
审稿时长
6-12 weeks
期刊介绍: Clinical Drug Investigation provides rapid publication of original research covering all phases of clinical drug development and therapeutic use of drugs. The Journal includes: -Clinical trials, outcomes research, clinical pharmacoeconomic studies and pharmacoepidemiology studies with a strong link to optimum prescribing practice for a drug or group of drugs. -Clinical pharmacodynamic and clinical pharmacokinetic studies with a strong link to clinical practice. -Pharmacodynamic and pharmacokinetic studies in healthy volunteers in which significant implications for clinical prescribing are discussed. -Studies focusing on the application of drug delivery technology in healthcare. -Short communications and case study reports that meet the above criteria will also be considered. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Clinical Drug Investigation may be accompanied by plain language summaries to assist readers who have some knowledge, but non in-depth expertise in, the area to understand important medical advances.
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