A Cost-Utility Analysis of Add-On Cannabidiol Versus Usual Care Alone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in England and Wales.

IF 2 Q2 ECONOMICS

PharmacoEconomics Open Pub Date : 2024-07-01 Epub Date: 2024-03-05 DOI:10.1007/s41669-024-00474-x

Colin Burke, Catriona Crossan, Emma Tyas, Matthew Hemstock, Dawn Lee, Sally Bowditch

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引用次数: 0

Abstract

Objectives: The aim of this study was to evaluate the cost effectiveness of plant-derived highly purified cannabidiol (Epidyolex^® in the UK; 100 mg/mL oral solution) as an add-on treatment to usual care for the management of treatment-refractory seizures associated with tuberous sclerosis complex (TSC) in patients aged ≥ 2 years.

Methods: A cohort-based model was developed using a National Health Service perspective and lifetime horizon. Health states were based on weekly seizure frequency and seizure-free days, utilizing patient-level data from the GWPCARE6 trial (ClinicalTrials.gov identifier: NCT02544763). Two independent regression models were applied to individual patient-level data to predict seizure-free days and seizure frequency. Healthcare resource utilization data were sourced from a Delphi panel, and patient and caregiver health-related quality of life values were elicited using vignettes valued by the general public. Outcomes relating to TSC-associated neuropsychiatric disorders were modeled with costs and quality-adjusted life-years sourced from published literature.

Results: In the base case, compared with usual care alone, 12 mg/kg/day cannabidiol was associated with an incremental cost-effectiveness ratio (ICER) of £23,797. The National Institute for Health and Care Excellence disease severity modifier reduced the ICER to £19,831. Probabilities of cost effectiveness at willingness-to-pay thresholds of £20,000 and £30,000 were 30% and 52%, respectively, for the base case and 39% and 66%, respectively, for the disease severity modifier. Results were robust to sensitivity and scenario analyses.

Conclusions: At 12 mg/kg/day and an ICER threshold of £20,000-£30,000, we provide evidence for the cost effectiveness of add-on cannabidiol treatment for patients with TSC-associated seizures aged ≥ 2 years who are refractory to current treatment.

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英格兰和威尔士治疗结节性硬化综合症相关癫痫发作的附加大麻二酚与常规护理的成本效用分析》（A Cost-Utility Analysis of Add-On Cannabidiol Vers Usual Care Alone for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in England and Wales）。

研究目的本研究旨在评估植物提取的高纯度大麻二酚（英国为 Epidyolex®；100 毫克/毫升口服溶液）作为常规治疗的附加治疗方法，在治疗年龄≥ 2 岁的结节性硬化综合征（TSC）相关难治性癫痫发作时的成本效益：方法：采用国民健康服务视角和终生视角开发了一个基于队列的模型。健康状况以每周发作频率和无发作天数为基础，利用来自 GWPCARE6 试验（ClinicalTrials.gov 标识符：NCT02544763）的患者级别数据。两个独立回归模型应用于单个患者层面的数据，以预测无发作天数和发作频率。医疗资源利用率数据来自德尔菲小组，患者和护理人员与健康相关的生活质量值则是通过公众评价的小故事得出的。与 TSC 相关的神经精神疾病的治疗结果与成本和质量调整生命年的模型均来自已发表的文献：在基本病例中，与单纯常规护理相比，12 毫克/千克/天大麻二酚的增量成本效益比 (ICER) 为 23,797 英镑。国家健康与护理卓越研究所的疾病严重程度修正系数将 ICER 降至 19,831 英镑。在20,000英镑和30,000英镑的支付意愿阈值下，基本病例的成本效益概率分别为30%和52%，疾病严重程度修正因子的成本效益概率分别为39%和66%。结果对敏感性分析和情景分析都很可靠：在 12 毫克/千克/天和 ICER 临界值为 20,000 英镑至 30,000 英镑的条件下，我们为年龄≥ 2 岁且对当前治疗难治的 TSC 相关癫痫发作患者接受加用大麻二酚治疗的成本效益提供了证据。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

PharmacoEconomics Open Multiple-

CiteScore

3.50

自引率

0.00%

发文量

审稿时长

8 weeks

期刊介绍： PharmacoEconomics - Open focuses on applied research on the economic implications and health outcomes associated with drugs, devices and other healthcare interventions. The journal includes, but is not limited to, the following research areas:Economic analysis of healthcare interventionsHealth outcomes researchCost-of-illness studiesQuality-of-life studiesAdditional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in PharmacoEconomics -Open may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.