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在无法获取总生存数据的情况下,我们能否准确预测成本效益?纳武单抗联合伊匹单抗治疗英国晚期黑色素瘤患者的案例研究。

Can We Accurately Predict Cost Effectiveness Without Access to Overall Survival Data? The Case Study of Nivolumab in Combination with Ipilimumab for the Treatment of Patients with Advanced Melanoma in England.

作者信息

Lee D, Amadi A, Sabater J, Ellis J, Johnson H, Kotapati S, McNamara S, Walker A, Cooper M, Patterson K, Roskell N, Meng Y

机构信息

BresMed Health Solutions, Sheffield, UK.

Bristol-Myers Squibb Pharmaceuticals, Uxbridge, UK.

出版信息

Pharmacoecon Open. 2019 Mar;3(1):43-54. doi: 10.1007/s41669-018-0080-5.

Abstract

BACKGROUND

Nivolumab with ipilimumab (the Regimen) is the first immuno-oncology combination treatment to demonstrate long-term clinical benefit for advanced melanoma patients. We evaluated the cost effectiveness of the Regimen in this population, with and without the availability of overall survival (OS) data.

METHODS

A partitioned survival model and a Markov state-transition model were developed to estimate the lifetime costs and benefits of the Regimen versus ipilimumab. These models were built with and without the availability of OS data, as only progression-free survival data were available from the head-to-head, phase III trial against ipilimumab at the time of the National Institute for Health and Care Excellence (NICE) submission. Patient utilities and resource use data were sourced from trial data or the literature.

RESULTS

Incremental cost-effectiveness ratios (ICERs) and absolute costs were similar between the models with and without OS data, but the model with OS data generated more than 1 additional quality-adjusted life-year (QALY) across both treatment arms. In both models, based on list prices, the Regimen was the most cost-effective treatment.

CONCLUSIONS

The analyses show that the Regimen is a cost-effective treatment for advanced melanoma patients in England, and methods to overcome the lack of OS can give reasonable estimates of QALYs gained and ICERs.

摘要

背景

纳武利尤单抗联合伊匹木单抗(该治疗方案)是首个对晚期黑色素瘤患者显示出长期临床获益的免疫肿瘤联合治疗方案。我们评估了该治疗方案在这一人群中的成本效益,有无总生存期(OS)数据。

方法

构建了一个分割生存模型和一个马尔可夫状态转换模型,以估计该治疗方案与伊匹木单抗相比的终身成本和效益。这些模型在有和没有OS数据的情况下构建,因为在向英国国家卫生与临床优化研究所(NICE)提交资料时,与伊匹木单抗进行的头对头III期试验仅提供了无进展生存期数据。患者效用和资源使用数据来自试验数据或文献。

结果

有和没有OS数据的模型之间的增量成本效益比(ICER)和绝对成本相似,但有OS数据的模型在两个治疗组中均产生了超过1个额外的质量调整生命年(QALY)。在两个模型中,根据标价,该治疗方案是最具成本效益的治疗方法。

结论

分析表明,该治疗方案对英国的晚期黑色素瘤患者是一种具有成本效益的治疗方法,克服OS数据缺乏的方法可以合理估计获得的QALY和ICER。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/df1a/6393277/1f75739fd33f/41669_2018_80_Fig1_HTML.jpg

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