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左旋多巴-卡比多巴肠凝胶治疗英国晚期帕金森病的成本效果。

The Cost Effectiveness of Levodopa-Carbidopa Intestinal Gel in the Treatment of Advanced Parkinson's Disease in England.

机构信息

Department of Basic and Clinical Neuroscience, The Maurice Wohl Clinical Neuroscience Institute, King's College London, Cutcombe Road, London, SE5 9RT, UK.

University of Illinois at Chicago, Chicago, IL, USA.

出版信息

Pharmacoeconomics. 2022 May;40(5):559-574. doi: 10.1007/s40273-022-01132-y. Epub 2022 Mar 21.

Abstract

BACKGROUND

Parkinson's disease is a progressive neurodegenerative disease, which significantly impacts patients' quality of life and is associated with high treatment and direct healthcare costs. In England, levodopa/carbidopa intestinal gel (LCIG) is indicated for the treatment of levodopa-responsive advanced Parkinson's disease with troublesome motor fluctuations when available combinations of medicinal products are unsatisfactory.

OBJECTIVE

We aimed to determine the cost effectiveness of LCIG compared to the standard of care for patients with advanced Parkinson's disease in England, using real-world data.

METHODS

A Markov model was adapted from previous published studies, using the perspective of the English National Health System and Personal and Social Services to evaluate the cost effectiveness of LCIG compared to standard of care in patients with advanced Parkinson's disease over a 20-year time horizon. The model comprised 25 health states, defined by a combination of the Hoehn and Yahr scale, and waking time spent in OFF-time. The base case considered an initial cohort of patients with an Hoehn and Yahr score of ≥ 3, and > 4 h OFF-time. Standard of care comprised standard oral therapies, and a proportion of patients were assumed to be treated with subcutaneous apomorphine infusion or injection in addition to oral therapies. Efficacy inputs were based on LCIG clinical trials where possible. Resource use and utility values were based on results of a large-scale observational study, and costs were derived from the latest published UK data, valued at 2017 prices. The EuroQol five-dimensions-3-level (EQ-5D-3L) instrument was used to measure utilities. Costs and quality-adjusted life-years were discounted at 3.5%. Both deterministic and probabilistic sensitivity analyses were conducted.

RESULTS

Total costs and quality-adjusted life-years gained for LCIG vs standard of care were £586,832 vs £554,022, and 2.82 vs 1.43, respectively. The incremental cost-effectiveness ratio for LCIG compared to standard of care was £23,649/quality-adjusted life-year. Results were sensitive to the healthcare resource utilisation based on real-world data, and long-term efficacy of LCIG.

CONCLUSIONS

The base-case incremental cost-effectiveness ratio was estimated to be within the acceptable thresholds for cost effectiveness considered for England.

摘要

背景

帕金森病是一种进行性神经退行性疾病,它显著影响患者的生活质量,并与高昂的治疗和直接医疗费用有关。在英国,左旋多巴/卡比多巴肠凝胶(LCIG)适用于治疗左旋多巴反应性晚期帕金森病,当现有药物组合不满意时,出现令人烦恼的运动波动。

目的

我们旨在使用真实世界的数据,确定 LCIG 相对于晚期帕金森病患者的标准治疗的成本效益。

方法

我们从之前发表的研究中改编了一个马尔可夫模型,使用英国国家卫生系统和个人与社会服务的视角,在 20 年的时间范围内评估 LCIG 相对于晚期帕金森病患者的标准治疗的成本效益。该模型由 25 个健康状态组成,由 Hoehn 和 Yahr 量表的组合以及清醒时间在 OFF 时间中的比例定义。基础病例考虑了一组初始患者,他们的 Hoehn 和 Yahr 评分≥3,并且>4 小时的 OFF 时间。标准治疗包括标准口服治疗,假设一部分患者除了口服治疗外还接受皮下阿朴吗啡输注或注射治疗。疗效输入基于 LCIG 临床试验,尽可能使用这些临床试验。资源利用和效用值基于一项大规模观察性研究的结果,成本来自最新发布的英国数据,按 2017 年价格计算。使用 EuroQol 五维健康量表(EQ-5D-3L)工具来衡量效用。成本和质量调整生命年以 3.5%的贴现率进行贴现。进行了确定性和概率敏感性分析。

结果

LCIG 与标准治疗相比,总费用和获得的质量调整生命年分别为 586832 英镑和 554022 英镑,2.82 和 1.43。LCIG 与标准治疗相比的增量成本效益比为 23649 英镑/质量调整生命年。结果对基于真实世界数据的医疗资源利用和 LCIG 的长期疗效敏感。

结论

基础病例的增量成本效益比估计在英国考虑的成本效益可接受阈值内。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/3570/9095547/f2e8cd333535/40273_2022_1132_Fig1_HTML.jpg

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