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纳武利尤单抗对比观察用于高复发风险的尿路上皮癌患者辅助治疗的成本效果分析:美国支付方视角。

Cost-effectiveness of nivolumab versus surveillance for the adjuvant treatment of patients with urothelial carcinoma who are at high risk of recurrence: a US payer perspective.

机构信息

RTI Health Solutions, Ljungskile, Sweden.

RTI Health Solutions, Manchester, UK.

出版信息

J Med Econ. 2024 Jan-Dec;27(1):543-553. doi: 10.1080/13696998.2024.2329019. Epub 2024 Apr 4.

Abstract

AIM

To evaluate the cost-effectiveness of adjuvant nivolumab compared with surveillance for the treatment of patients with high-risk muscle-invasive urothelial carcinoma (MIUC) after radical resection from a US healthcare payer perspective and to investigate the impact of alternative modeling approaches on the cost-effectiveness results.

MATERIAL AND METHODS

A four-state, semi-Markov model consisting of disease free, local recurrence, distant recurrence, and death health states was developed to investigate the cost-effectiveness of nivolumab compared with surveillance over a 30-year time horizon. The model used data from the randomized CheckMate 274 trial (NCT02632409) and published literature to inform transitions among health states, and inputs on cost, utility, adverse event, and disease management. Scenario analyses were conducted to investigate the impact of model structure and key assumptions on the results. One-way deterministic and probabilistic sensitivity analysis were conducted to investigate the robustness of the results.

RESULTS

Total expected costs were higher with nivolumab ($162,278) compared with surveillance ($63,027). Nivolumab was associated with improved survival (1.61 life-years gained compared with surveillance) and an incremental gain of 0.98 quality-adjusted life-years (QALYs). Although total treatment costs were higher for nivolumab, cost offsets were observed because of delayed or avoided recurrences and deaths experienced with nivolumab compared with observation. The incremental cost-effectiveness and cost-utility ratios were $61,462/life-year and $100,930/QALY.

LIMITATIONS

At the time of analysis, CheckMate 274 had limited follow-up on disease-free survival and no overall survival data. The limited evidence necessitated assumptions on modeling survival after each type of recurrence.

CONCLUSIONS

Nivolumab is estimated to be a life-extending and cost-effective option for adjuvant treatment of MIUC for patients who are at high risk of recurrence after undergoing radical resection in the United States. Using a threshold of $150,000/QALY, the cost-effectiveness conclusions remained consistent across the scenario and sensitivity analyses conducted.

摘要

目的

从美国医疗保健支付者的角度评估辅助 nivolumab 与监测治疗根治性切除术后高危肌层浸润性膀胱癌(MIUC)患者的成本效益,并研究替代建模方法对成本效益结果的影响。

材料和方法

建立了一个由四个状态组成的半马尔可夫模型,包括无疾病、局部复发、远处复发和死亡状态,以调查 nivolumab 与监测在 30 年时间范围内的成本效益。该模型使用了随机对照试验 CheckMate 274 (NCT02632409)的数据和已发表的文献来提供健康状态之间的转移信息,并提供成本、效用、不良事件和疾病管理的输入。进行了情景分析以研究模型结构和关键假设对结果的影响。进行了单向确定性和概率敏感性分析,以研究结果的稳健性。

结果

与监测相比,nivolumab 的总预期成本更高($162,278 比 $63,027)。与监测相比,nivolumab 与生存改善相关(与监测相比,获得 1.61 个生命年),并获得 0.98 个质量调整生命年(QALY)的增量收益。尽管 nivolumab 的总治疗成本较高,但由于与观察相比,nivolumab 延迟或避免了复发和死亡,因此出现了成本抵消。增量成本效益和成本效用比分别为 $61,462/生命年和 $100,930/QALY。

局限性

在分析时,CheckMate 274 对无疾病生存的随访时间有限,且没有总生存数据。有限的证据需要对每种类型的复发后的生存建模做出假设。

结论

在接受根治性切除术后有高复发风险的美国患者中,与观察相比,nivolumab 估计是 MIUC 辅助治疗的一种延长生命且具有成本效益的选择。使用 150,000 美元/QALY 的阈值,成本效益结论在进行的情景和敏感性分析中保持一致。

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