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贝伐珠单抗联合卡培他滨维持治疗转移性结直肠癌的成本效果分析

Cost-effectiveness of Maintenance Capecitabine and Bevacizumab for Metastatic Colorectal Cancer.

机构信息

Department of Surgery, University of Chicago, Chicago, Illinois.

Department of Surgery, Medical College of Wisconsin, Milwaukee.

出版信息

JAMA Oncol. 2019 Feb 1;5(2):236-242. doi: 10.1001/jamaoncol.2018.5070.

Abstract

IMPORTANCE

Unregulated drug prices increase cancer therapy costs. After induction chemotherapy, patients with metastatic colon cancer can receive maintenance capecitabine and bevacizumab therapy based on improved progression-free survival, but whether this treatment's cost justifies its benefits has not been evaluated in the United States.

OBJECTIVE

This study sought to determine the influence of capecitabine and bevacizumab drug prices on cost-effectiveness from a Medicare payer's perspective.

DESIGN, SETTING, AND PARTICIPANTS: The incremental cost-effectiveness of capecitabine and bevacizumab maintenance therapy was determined with a Markov model using a quality-of-life penalty based on outcomes data from the CAIRO phase 3 randomized clinical trial (RCT), which included 558 adults in the Netherlands with unresectable metastatic colorectal cancer who had stable disease or better following induction chemotherapy. The outcomes were modeled using Markov chains to account for patients who had treatment complications or cancer progression. Transition probabilities between patient states were determined, and each state's costs were determined using US Medicare data on payments for capecitabine and bevacizumab treatment. Deterministic and probabilistic sensitivity analyses identified factors affecting cost-effectiveness.

MAIN OUTCOMES AND MEASURES

Life-years gained were adjusted using CAIRO3 RCT quality-of-life data to determine quality-adjusted life-years (QALYs). The primary end point was the incremental cost-effectiveness ratio, representing incremental costs per QALY gained using a capecitabine and bevacizumab maintenance regimen compared with observation alone.

RESULTS

Markov model estimated survival and complication outcomes closely matched those reported in the CAIRO3 RCT, which included 558 adults (n = 197 women, n = 361 men; median age, 64 and 63 years for patients in the observation and maintenance therapy groups, respectively) in the Netherlands with unresectable metastatic colorectal cancer who had stable disease or better following induction chemotherapy. Incremental costs for a 3-week maintenance chemotherapy cycle were $6601 per patient. After 29 model iterations corresponding to 60 months of follow-up, mean per-patient costs were $105 239 for maintenance therapy and $21.10 for observation. Mean QALYs accrued were 1.34 for maintenance therapy and 1.20 for observation. The incremental cost-effectiveness ratio favored maintenance treatment, at an incremental cost of $725 601 per QALY. The unadjusted ratio was $438 394 per life-year. Sensitivity analyses revealed that cost-effectiveness varied with changes in drug costs. To achieve an incremental cost-effectiveness ratio of less than $59 039 (median US household income) per unadjusted life-year would require capecitabine and bevacizumab drug costs to be reduced from $6173 (current cost) to $452 per 3-week chemotherapy cycle.

CONCLUSIONS AND RELEVANCE

Antineoplastic therapy is expensive for payers and society. The price of capecitabine and bevacizumab maintenance therapy would need to be reduced by 93% to make it cost-effective, a finding useful for policy decision making and payment negotiations.

摘要

重要性

不受管制的药品价格会增加癌症治疗成本。在诱导化疗后,转移性结直肠癌患者可以根据无进展生存期的改善接受卡培他滨和贝伐珠单抗维持治疗,但这种治疗的成本是否能证明其收益合理,这在美国尚未得到评估。

目的

本研究旨在从医疗保险支付者的角度确定卡培他滨和贝伐珠单抗维持治疗的药物价格对成本效益的影响。

设计、地点和参与者:采用马尔可夫模型,使用基于 CAIRO 3 期随机临床试验(RCT)结局数据的生活质量罚分,确定卡培他滨和贝伐珠单抗维持治疗的增量成本效益。使用来自荷兰的 558 例不可切除转移性结直肠癌患者的结果数据进行建模,这些患者在诱导化疗后病情稳定或改善。结局采用马尔可夫链进行建模,以纳入有治疗并发症或癌症进展的患者。确定患者状态之间的转移概率,并使用美国医疗保险数据确定每个状态的治疗费用。确定性和概率敏感性分析确定了影响成本效益的因素。

主要结局和措施

使用 CAIRO3 RCT 生活质量数据调整生命年增益,以确定质量调整生命年(QALY)。主要终点是增量成本效益比,代表与单独观察相比,使用卡培他滨和贝伐珠单抗维持治疗方案每增加一个 QALY 的增量成本。

结果

马尔可夫模型估计的生存和并发症结果与 CAIRO3 RCT 报告的结果非常吻合,该 RCT 纳入了 558 例成年人(n=197 名女性,n=361 名男性;分别为接受观察和维持治疗的患者的中位年龄为 64 岁和 63 岁),这些患者来自荷兰,患有不可切除的转移性结直肠癌,在诱导化疗后病情稳定或改善。每 3 周维持化疗周期的增量成本为每位患者 6601 美元。经过 29 次对应 60 个月随访的模型迭代,维持治疗的每位患者的平均成本为 105239 美元,观察治疗的成本为 21.10 美元。维持治疗的平均 QALY 为 1.34,观察治疗的平均 QALY 为 1.20。增量成本效益比倾向于维持治疗,增量成本为每 QALY 725601 美元。未经调整的比值为每生命年 438394 美元。敏感性分析表明,成本效益随药物成本的变化而变化。要使增量成本效益比低于每未调整生命年 59039 美元(美国中位数家庭收入),则需要将卡培他滨和贝伐珠单抗维持治疗的药物成本从 6173 美元(当前成本)降低到每 3 周化疗周期 452 美元。

结论和相关性

抗肿瘤治疗对支付者和社会来说是昂贵的。卡培他滨和贝伐珠单抗维持治疗的价格需要降低 93%,才能具有成本效益,这一发现有助于决策制定和支付谈判。

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