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阿达木单抗治疗幼年特发性关节炎相关葡萄膜炎的成本-效果分析。

Cost-Effectiveness Analysis of Adalimumab for the Treatment of Uveitis Associated with Juvenile Idiopathic Arthritis.

机构信息

Centre for Health Economics and Medicines Evaluation, Bangor University, Bangor, United Kingdom.

Centre for Health Economics and Medicines Evaluation, Bangor University, Bangor, United Kingdom.

出版信息

Ophthalmology. 2019 Mar;126(3):415-424. doi: 10.1016/j.ophtha.2018.09.043. Epub 2018 Oct 16.

Abstract

PURPOSE

To investigate the cost effectiveness of adalimumab in combination with methotrexate, compared with methotrexate alone, for the management of uveitis associated with juvenile idiopathic arthritis (JIA).

DESIGN

A cost-utility analysis based on a clinical trial and decision analytic model.

PARTICIPANTS

Children and adolescents 2 to 18 years of age with persistently active uveitis associated with JIA, despite optimized methotrexate treatment for at least 12 weeks.

METHODS

The SYCAMORE (Randomised controlled trial of the clinical effectiveness, SafetY and Cost effectiveness of Adalimumab in combination with MethOtRExate for the treatment of juvenile idiopathic arthritis associated uveitis) trial (identifier, ISRCTN10065623) of methotrexate (up to 25 mg weekly) with or without fortnightly administered adalimumab (20 or 40 mg, according to body weight) provided data on resource use (based on patient self-report and electronic records) and health utilities (from the Health Utilities Index questionnaire). Surgical event rates and long-term outcomes were based on data from a 10-year longitudinal cohort. A Markov model was used to extrapolate the effects of treatment based on visual impairment.

MAIN OUTCOME MEASURES

Medical costs to the National Health Service in the United Kingdom, utility of defined health states, quality-adjusted life-years (QALYs), and incremental cost per QALY.

RESULTS

Adalimumab in combination with methotrexate resulted in additional costs of £39 316, with a 0.30 QALY gain compared with methotrexate alone, resulting in an incremental cost-effectiveness ratio of £129 025 per QALY gained. The probability of cost effectiveness at a threshold of £30 000 per QALY was less than 1%. Based on a threshold analysis, a price reduction of 84% would be necessary for adalimumab to be cost effective.

CONCLUSIONS

Adalimumab is clinically effective in uveitis associated with JIA; however, its cost effectiveness is not demonstrated compared with methotrexate alone in the United Kingdom setting.

摘要

目的

评估阿达木单抗联合甲氨蝶呤与单独使用甲氨蝶呤治疗幼年特发性关节炎(JIA)相关葡萄膜炎的成本效果。

设计

基于临床试验和决策分析模型的成本效益分析。

参与者

2 至 18 岁的持续活跃性 JIA 相关葡萄膜炎患者,尽管接受了至少 12 周的优化甲氨蝶呤治疗。

方法

SYCAMORE(阿达木单抗联合甲氨蝶呤治疗幼年特发性关节炎相关葡萄膜炎的临床疗效、安全性和成本效果的随机对照试验)试验(标识符:ISRCTN81451366)提供了资源利用(基于患者自我报告和电子记录)和健康效用(来自健康效用指数问卷)的数据。手术事件发生率和长期结果基于 10 年纵向队列的数据。Markov 模型用于根据视力损害情况推断治疗效果。

主要观察指标

英国国家医疗服务体系的医疗成本、定义健康状态的效用、质量调整生命年(QALY)和每 QALY 的增量成本。

结果

阿达木单抗联合甲氨蝶呤治疗的额外费用为 39316 英镑,与单独使用甲氨蝶呤相比,获得了 0.30 个 QALY 的收益,导致每获得一个 QALY 的增量成本效益比为 129025 英镑。在 30000 英镑/QALY 的阈值下,成本效益的可能性小于 1%。基于阈值分析,阿达木单抗的价格需要降低 84%才能具有成本效益。

结论

阿达木单抗在 JIA 相关葡萄膜炎中具有临床疗效;然而,在英国背景下,与单独使用甲氨蝶呤相比,其成本效果并不显著。

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