Hoerger T J, Bala M V, Rowland C, Greer M, Chrischilles E A, Holloway R G
Center for Economics Research, Research Triangle Institute, Research Triangle Park, North Carolina, USA.
Pharmacoeconomics. 1998 Nov;14(5):541-57. doi: 10.2165/00019053-199814050-00006.
Pramipexole was recently approved in the US for treatment of the symptoms of idiopathic Parkinson's disease (PD). Although pramipexole has been found to be safe and efficacious when compared with placebo, little data are yet available on its cost effectiveness when compared with baseline treatment. The aim of this study was to estimate the costs and cost effectiveness (cost utility) of pramipexole compared with baseline treatment in patients with early and advanced PD.
We developed a cost-effectiveness (CE) model in the US setting that linked Unified Parkinson's Disease Rating Scale (UPDRS) Part II (activities of daily life) and III (motor) scores to disease progression, costs and patient utility. Data for the model were obtained from clinical trials, a literature review and a survey of 193 patients' health resource use and utility. We used cost and quality-adjusted life-year (QALY) estimates from the model to estimate the incremental cost effectiveness of pramipexole relative to baseline treatment patterns. We performed separate analyses for patients with early and advanced PD. We also performed extensive sensitivity analyses by adding other dopamine agonists to the no-pramipexole treatment regimen and varying disease progression parameters. The study was conducted from the societal perspective, although data presentation allows interpretation of cost effectiveness from either the societal or payer perspective.
For patients with both early and advanced PD, treatment with pramipexole had higher costs but was more effective than baseline treatment. For patients with early onset of PD, the incremental total CE ratio for pramipexole was $US8837/QALY. For patients with advanced PD, the incremental CE ratio was $US12 294/QALY (1997 costs). These ratios were lower than the CE ratios of many widely used medical treatments.
Subject to the inherent limitations of modelling chronic disease progression and subsequent healthcare costs and patient utility, the results suggested that pramipexole was a cost effective treatment for patients with early and advanced PD in the US.
普拉克索最近在美国被批准用于治疗特发性帕金森病（PD）的症状。尽管与安慰剂相比，普拉克索已被证明是安全有效的，但与基线治疗相比，其成本效益的数据仍然很少。本研究的目的是评估普拉克索与早期和晚期PD患者基线治疗相比的成本和成本效益（成本效用）。
我们在美国背景下开发了一个成本效益（CE）模型，该模型将统一帕金森病评定量表（UPDRS）第二部分（日常生活活动）和第三部分（运动）评分与疾病进展、成本和患者效用联系起来。模型数据来自临床试验、文献综述以及对193名患者健康资源使用和效用的调查。我们使用模型中的成本和质量调整生命年（QALY）估计值来评估普拉克索相对于基线治疗模式的增量成本效益。我们对早期和晚期PD患者进行了单独分析。我们还通过在无普拉克索治疗方案中添加其他多巴胺激动剂并改变疾病进展参数进行了广泛的敏感性分析。本研究是从社会角度进行的，不过数据呈现允许从社会或支付方角度解释成本效益。
对于早期和晚期PD患者，普拉克索治疗成本更高，但比基线治疗更有效。对于早期发病的PD患者，普拉克索的增量总CE比值为8837美元/QALY。对于晚期PD患者，增量CE比值为12294美元/QALY（1997年成本）。这些比值低于许多广泛使用的医疗治疗的CE比值。
尽管在模拟慢性病进展以及后续医疗成本和患者效用方面存在固有局限性，但结果表明普拉克索在美国对于早期和晚期PD患者是一种具有成本效益的治疗方法。
