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玻璃体内雷珠单抗治疗与罕见病相关的脉络膜新生血管所致视力损害:英国的成本效益。

Intravitreal Ranibizumab for the Treatment of Visual Impairment Due to Choroidal Neovascularization Associated with Rare Diseases: Cost-Effectiveness in the UK.

机构信息

BresMed Health Solutions Ltd, Sheffield, UK.

Novartis Pharmaceuticals UK Limited, Camberley, Surrey, UK.

出版信息

Adv Ther. 2019 Mar;36(3):632-644. doi: 10.1007/s12325-019-0894-2. Epub 2019 Feb 6.

DOI:10.1007/s12325-019-0894-2
PMID:30726549
Abstract

INTRODUCTION

This study sought to determine the cost-effectiveness of intravitreal ranibizumab compared with best supportive care (BSC; considered to be no active treatment) for the treatment of visual impairment due to choroidal neovascularization (CNV) associated with causes other than neovascular age-related macular degeneration (nAMD) and pathologic myopia (PM) in a UK setting.

METHODS

An individual patient-level simulation model was developed to estimate the lifetime costs and quality-adjusted life years (QALYs) of ranibizumab vs. BSC. Regression analyses, performed on patient-level data collected within the pivotal phase III MINERVA trial, modelled visual acuity (VA) progression while patients remained on treatment. Patient utilities were modelled as a function of VA in both eyes and resource use estimates were based on trial data or the literature. Costs were evaluated from the perspective of the UK National Health Service and personal social services, with future costs and health outcomes discounted at 3.5% per annum. Sensitivity and scenario analyses were conducted.

RESULTS

The incremental cost-effectiveness ratio for intravitreal ranibizumab was £1363 per QALY compared to BSC and was associated with an incremental benefit of 1.06 QALYs and an incremental cost of £1444 per patient. Drug and administration costs of intravitreal ranibizumab were offset by the prevention of the development of blindness and its associated costs, while the increase in benefits was driven by a reduction in mortality risk and an improved health-related quality of life attributed to an improvement in VA. The findings were robust to a range of sensitivity analyses and ranibizumab consistently remained cost-effective at a willingness-to-pay threshold of £20,000-30,000 per QALY gained for all sensitivity analyses.

CONCLUSION

Intravitreal ranibizumab is a highly cost-effective intervention for the treatment of CNV due to causes other than nAMD and PM as it delivers substantial QALY gains to patients while making cost savings vs. BSC.

FUNDING

Novartis Pharmaceuticals UK Ltd.

摘要

引言

本研究旨在确定玻璃体内雷珠单抗相对于最佳支持治疗(BSC;被认为是无活性治疗)在治疗由非新生血管性年龄相关性黄斑变性(nAMD)和病理性近视(PM)以外的原因引起的脉络膜新生血管(CNV)相关的视力障碍方面的成本效益,该研究在英国进行。

方法

开发了一个个体患者水平的模拟模型,以估计玻璃体内雷珠单抗与 BSC 相比的终生成本和质量调整生命年(QALY)。基于 MINERVA 关键性 III 期试验中收集的患者水平数据进行的回归分析,对治疗期间视力(VA)进展进行了建模。双眼 VA 的患者效用模型和资源使用估算均基于试验数据或文献。成本从英国国家卫生服务和个人社会服务的角度进行评估,未来成本和健康结果按每年 3.5%贴现。进行了敏感性和情景分析。

结果

与 BSC 相比,玻璃体内雷珠单抗的增量成本效益比为每 QALY 1363 英镑,与每例患者 1444 英镑的增量成本相比,增加了 1.06 个 QALY 的增量获益。玻璃体内雷珠单抗的药物和管理成本被预防失明及其相关成本所抵消,而收益的增加是由于死亡率风险的降低和与 VA 改善相关的健康相关生活质量的提高所致。研究结果在一系列敏感性分析中是稳健的,在所有敏感性分析中,雷珠单抗在支付意愿阈值为 20000-30000 英镑/QALY 时均保持成本效益。

结论

玻璃体内雷珠单抗是一种非常有成本效益的治疗 CNV 的方法,用于治疗 nAMD 和 PM 以外的原因,因为它为患者带来了大量的 QALY 获益,同时与 BSC 相比节省了成本。

资金

诺华制药英国有限公司。

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