Background

In recent years, new drugs have been introduced for second-line treatment of advanced renal cell carcinoma (RCC). Nivolumab increases overall survival and is associated with less toxicity compared to everolimus in this setting according to the CheckMate 025 study. However, because of the high cost of nivolumab, there is a need to define its value by considering both efficacy and cost.

Objective

To estimate the cost effectiveness of nivolumab for second-line treatment of advanced RCC from the US payer perspective.

Design, setting, and participants

A Markov model was developed to compare the costs and effectiveness of nivolumab with those of everolimus and placebo in second-line treatment of advanced RCC. Health outcomes were measured in life-years (LYs) and quality-adjusted LYs (QALYs). Drug costs were based on 2016 Medicare reimbursement rates.

Outcome measurements and statistical analysis

Model robustness was assessed in univariable and probabilistic sensitivity analyses. We addressed the issue of the extensive duration of immunotherapy treatment among long-term survivors, which may or may not be approved by payers.

Results and limitations

The total mean cost per patient was $101 070 for nivolumab and $50 935 for everolimus. Nivolumab generated a gain of 0.24 LYs (0.34 QALYs) compared to everolimus. The incremental cost-effectiveness ratio (ICER) for nivolumab was $146 532/QALY versus everolimus and $226 197/QALY versus placebo. Limiting the maximal treatment duration of nivolumab to 2 yr reduced the ICER to $121 788/QALY versus everolimus. The analysis is limited by data availability and our assumptions.

Conclusions

Our analysis established that with a willingness-to-pay threshold of $100 000 to $150 000 per QALY, nivolumab is estimated to be cost-effective versus everolimus, but not cost-effective versus placebo.

Patient summary

We assessed the cost effectiveness of nivolumab in previously treated metastatic kidney cancer. In the USA, it would cost $146 532 to gain one quality-adjusted life-year with nivolumab versus everolimus, or $226 197 versus placebo. Nivolumab is considered cost-effective versus everolimus, but not versus placebo.

中文翻译：

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Nivolumab在晚期肾细胞癌中的成本效益

背景

近年来，已将新药引入晚期肾细胞癌（RCC）的二线治疗。根据CheckMate 025研究，与依维莫司相比，Nivolumab可以提高总体生存率，并且毒性较小。但是，由于nivolumab的价格昂贵，因此有必要通过兼顾功效和成本来确定其价值。

客观的

从美国付款人的角度评估nivolumab用于晚期RCC的二线治疗的成本效益。

设计，设置和参与者

建立了马尔可夫模型，以比较尼古鲁单抗与依维莫司和安慰剂在晚期RCC的二线治疗中的成本和效果。以生命年（LYs）和质量调整后的LYs（QALYs）衡量健康结局。药品费用基于2016年Medicare报销率。

成果测量和统计分析

在单变量和概率敏感性分析中评估了模型的稳健性。我们解决了长期幸存者中长期接受免疫治疗的问题，付款人可能会批准也可能不会批准。

结果与局限性

尼伏鲁单抗的平均每位患者总费用为101 070美元，依维莫司的平均总费用为50 935美元。与依维莫司相比，Nivolumab产生了0.24 LYs（0.34 QALYs）的增益。与依维莫司相比，nivolumab的增量成本效益比（ICER）为146 532美元/ QALY，而与安慰剂相比为226 197美元/ QALY。与依维莫司相比，将nivolumab的最大治疗持续时间限制为2年可将ICER降至121788美元/ QALY。该分析受到数据可用性和我们的假设的限制。

结论

我们的分析确定，每个QALY的支付意愿门槛为10万至15万美元，据估计，尼伏鲁单抗与依维莫司相比具有成本效益，而与安慰剂相比则无成本效益。

病人总结

我们评估了尼古鲁单抗在先前治疗的转移性肾癌中的成本效益。在美国，使用nivolumab与依维莫司相比，获得一个质量调整生命年的费用为146,532美元，而与安慰剂相比为226,197美元。与依维莫司相比，Nivolumab被认为具有成本效益，但与安慰剂相比却不具成本效益。