Lowin Julia, Sail Kavita, Baj Rakhi, Jalundhwala Yash J, Marshall Thomas S, Konwea Henrietta, Chaudhuri K R
a QuintilesIMS , London , UK.
b AbbVie , North Chicago , IL , USA.
J Med Econ. 2017 Nov;20(11):1207-1215. doi: 10.1080/13696998.2017.1379411. Epub 2017 Sep 21.
Parkinson's disease (PD) is an incurable, progressive neurological condition, with symptoms impacting movement, walking, and posture that eventually become severely disabling. Advanced PD (aPD) has a significant impact on quality-of-life (QoL) for patients and their caregivers/families. Levodopa/carbidopa intestinal gel (LCIG) is indicated for the treatment of advanced levodopa-responsive PD with severe motor fluctuations and hyper-/dyskinesia when available combinations of therapy have not given satisfactory results.
To determine the cost-effectiveness of LCIG vs standard of care (SoC) for the treatment of aPD patients.
A Markov model was used to evaluate LCIG vs SoC in a hypothetical cohort of 100 aPD patients with severe motor fluctuations from an Irish healthcare perspective. Model health states were defined by Hoehn & Yahr (H&Y) scale-combined with amount of time in OFF-time-and death. SoC comprised of standard oral therapy ± subcutaneous apomorphine infusion and standard follow-up visits. Clinical efficacy, utilities, and transition probabilities were derived from published studies. Resource use was estimated from individual patient-level data from Adelphi 2012 UK dataset, using Irish costs, where possible. Time horizon was 20 years. Costs and outcomes were discounted at 4%. Both one-way and probabilistic sensitivity analyses were conducted.
The incremental cost-effectiveness ratio for LCIG vs SOC was €26,944/quality adjusted life year (QALY) (total costs and QALYs for LCIG vs SoC: €537,687 vs €514,037 and 4.37 vs 3.49, respectively). LCIG is cost-effective at a payer threshold of €45,000. The model was most sensitive to health state costs.
LCIG is a cost-effective treatment option compared with SoC in patients with aPD.
帕金森病（PD）是一种无法治愈的进行性神经疾病，其症状会影响运动、行走和姿势，最终导致严重残疾。晚期帕金森病（aPD）对患者及其护理人员/家庭的生活质量（QoL）有重大影响。左旋多巴/卡比多巴肠凝胶（LCIG）适用于治疗晚期左旋多巴反应性帕金森病，当现有治疗组合未取得满意效果时，可用于治疗伴有严重运动波动和异动症/运动障碍的患者。
确定LCIG与标准治疗（SoC）相比治疗aPD患者的成本效益。
从爱尔兰医疗保健角度出发，使用马尔可夫模型评估100名伴有严重运动波动的aPD患者的假设队列中LCIG与SoC的情况。模型健康状态由Hoehn & Yahr（H&Y）量表结合“关”期时间和死亡情况来定义。SoC包括标准口服治疗±皮下注射阿扑吗啡以及标准随访。临床疗效、效用和转移概率来自已发表的研究。资源使用尽可能根据2012年英国阿德尔菲数据集的个体患者水平数据，采用爱尔兰成本进行估算。时间范围为20年。成本和结果按4%进行贴现。进行了单向和概率敏感性分析。
LCIG与SoC相比的增量成本效益比为26,944欧元/质量调整生命年（QALY）（LCIG与SoC的总成本和QALY分别为537,687欧元对514,037欧元以及4.37对3.49）。在支付者阈值为45,000欧元时，LCIG具有成本效益。该模型对健康状态成本最为敏感。
与SoC相比，LCIG对于aPD患者是一种具有成本效益的治疗选择。
