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在英国,晚期帕金森病患者使用左旋多巴/卡比多巴肠凝胶与标准护理相比的成本效果分析。

A cost-effectiveness analysis of levodopa/carbidopa intestinal gel compared to standard care in late stage Parkinson's disease in the UK.

机构信息

IMS Health, London NW1 6JB, UK.

出版信息

J Med Econ. 2011;14(5):584-93. doi: 10.3111/13696998.2011.598201. Epub 2011 Jul 6.

Abstract

OBJECTIVE

Evaluation of cost-effectiveness of levodopa/carbidopa intestinal gel (LCIG), compared to standard care (SC) in patients with advanced Parkinson's disease (aPD) in the UK.

DESIGN

Markov model to quantify costs and outcomes associated with LCIG versus SC in aPD patients at Hoehn and Yahr (H&Y) stages 3, 4 or 5 experiencing >50% OFF time per day. Time horizon was lifetime, LCIG treatment was assumed to last maximal 5 years after which patients revert to SC. Model comprised 12 aPD health states according to H&Y status and daily time spent in OFF state. Cost analyses are reported from a UK NHS and Personal Social Services perspective. Uncertainties were assessed through one-way sensitivity analyses.

COMPARATORS

LCIG, providing patients with continuous dopaminergic stimulation to maximise functional ON time during the day and SC, defined as medically determined best available oral medication.

MAIN OUTCOME MEASURES

Cost-effectiveness, based on quality adjusted life years gained, presented as an incremental cost-effectiveness ratio.

RESULTS

Lifetime analysis yields an incremental cost per QALY of £36,024 for LCIG compared to SC (incremental cost £39,644, QALY gain 1.1). Results were sensitive to time on treatment, health state on treatment initiation, and estimates of long term benefit (OWSA results from £32,127 to £66,421 per QALY). Findings must be considered in the context of the study limitations which were mainly due to data availability constraints.

CONCLUSIONS

LCIG is an effective treatment, reducing OFF time and improving quality of life in advanced PD. It provides value for money in levodopa-responsive aPD patients with severe motor fluctuations when no other treatment options are effective or suitable. Given LCIG is an orphan drug, it is reasonable to suggest that it may be considered cost-effective in the UK setting. However, further research is needed to complete current data gaps and increase robustness of the model.

摘要

目的

评估左旋多巴/卡比多巴肠凝胶(LCIG)与标准护理(SC)相比,在英国晚期帕金森病(aPD)患者中的成本效益。

设计

使用马尔可夫模型来量化 LCIG 与 SC 在 H&Y 阶段 3、4 或 5 且每天 >50%OFF 时间的 aPD 患者中的成本和结果。时间范围为终生,假设 LCIG 治疗最长可持续 5 年,之后患者转回 SC。模型包括 12 种根据 H&Y 状态和每天 OFF 状态时间划分的 aPD 健康状态。成本分析基于英国国民保健制度和个人社会服务的观点。通过单向敏感性分析评估不确定性。

比较

LCIG,通过提供持续的多巴胺刺激来最大限度地提高白天的功能性 ON 时间;SC,定义为医学上确定的最佳口服药物。

主要结果测量

基于获得的质量调整生命年,以增量成本效益比表示的成本效益。

结果

终生分析表明,与 SC 相比,LCIG 的每 QALY 增量成本为 36024 英镑(增量成本为 39644 英镑,QALY 增益为 1.1)。结果对治疗时间、治疗开始时的健康状态以及长期获益的估计敏感(OWSA 结果从每 QALY 的 32127 英镑到 66421 英镑)。研究结果必须考虑到研究限制,这些限制主要是由于数据可用性的限制。

结论

LCIG 是一种有效的治疗方法,可减少晚期 PD 患者的 OFF 时间并提高生活质量。对于没有其他有效或合适治疗选择的左旋多巴反应性 aPD 患者,严重运动波动患者,LCIG 具有成本效益。鉴于 LCIG 是一种孤儿药,可以合理地认为它在英国的应用可能具有成本效益。然而,需要进一步研究来弥补当前数据差距并提高模型的稳健性。

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