van der Schans Simon, Goossens Lucas M A, Boland Melinde R S, Kocks Janwillem W H, Postma Maarten J, van Boven Job F M, Rutten-van Mölken Maureen P M H
Unit of Pharmacoepidemiology and Pharmacoeconomics, Department of Pharmacy, University of Groningen, Groningen, The Netherlands.
Institute of Health Policy and Management, Erasmus University Rotterdam, Rotterdam, The Netherlands.
Pharmacoeconomics. 2017 Jan;35(1):43-63. doi: 10.1007/s40273-016-0448-2.
Worldwide, chronic obstructive pulmonary disease (COPD) is a highly prevalent chronic lung disease with considerable clinical and socioeconomic impact. Pharmacologic maintenance drugs (such as bronchodilators and inhaled corticosteroids) play an important role in the treatment of COPD. The cost effectiveness of these treatments has been frequently assessed, but studies to date have largely neglected the impact of treatment sequence and the exact stage of disease in which the drugs are used in real life.
We aimed to systematically review recently published articles that reported the cost effectiveness of COPD maintenance treatments, with a focus on key findings, quality and methodological issues.
We performed a systematic literature search in Embase, PubMed, the UK NHS Economic Evaluation Database (NHS-EED) and EURONHEED (European Network of Health Economics Evaluation Databases) and included all relevant articles published between 2011 and 2015 in either Dutch, English or German. Main study characteristics, methods and outcomes were extracted and critically assessed. The Quality of Health Economic Studies (QHES) instrument was used as basis for quality assessment, but additional items were also addressed.
The search identified 18 recent pharmacoeconomic analyses of COPD maintenance treatments. Papers reported the cost effectiveness of long-acting muscarinic antagonist (LAMA) monotherapy (n = 6), phosphodiesterase (PDE)-4 inhibitors (n = 4), long-acting beta agonist/inhaled corticosteroid (LABA/ICS) combinations (n = 4), LABA monotherapy (n = 2) and LABA/LAMA combinations (n = 2). All but two studies were funded by the manufacturer, and all studies indicated favourable cost effectiveness; however, the number of quality-adjusted life-years (QALYs) gained was small. Less than half of the studies reported a COPD-specific outcome in addition to a generic outcome (mostly QALYs). Exacerbation and mortality rates were found to be the main drivers of cost effectiveness. According to the QHES, the quality of the studies was generally sufficient, but additional assessment revealed that most studies poorly represented the cost effectiveness of real-life medication use.
The majority of studies showed that pharmacologic COPD maintenance treatment is cost effective, but most studies poorly reflected real-life drug use. Consistent and COPD-specific methodology is recommended.
在全球范围内,慢性阻塞性肺疾病(COPD)是一种高度流行的慢性肺部疾病,具有相当大的临床和社会经济影响。药物维持治疗(如支气管扩张剂和吸入性糖皮质激素)在COPD治疗中发挥着重要作用。这些治疗的成本效益已被频繁评估,但迄今为止的研究在很大程度上忽略了治疗顺序的影响以及在现实生活中使用药物时疾病的确切阶段。
我们旨在系统回顾最近发表的报道COPD维持治疗成本效益的文章,重点关注关键发现、质量和方法学问题。
我们在Embase、PubMed、英国国家医疗服务体系经济评估数据库(NHS-EED)和欧洲卫生经济评估数据库网络(EURONHEED)中进行了系统的文献检索,并纳入了2011年至2015年间以荷兰语、英语或德语发表的所有相关文章。提取并严格评估主要研究特征、方法和结果。以卫生经济研究质量(QHES)工具为质量评估基础,但也涉及其他项目。
检索确定了18项最近关于COPD维持治疗的药物经济学分析。论文报道了长效毒蕈碱拮抗剂(LAMA)单药治疗(n = 6)、磷酸二酯酶(PDE)-4抑制剂(n = 4)、长效β激动剂/吸入性糖皮质激素(LABA/ICS)联合治疗(n = 4)、LABA单药治疗(n = 2)和LABA/LAMA联合治疗(n = 2)的成本效益。除两项研究外,所有研究均由制造商资助,且所有研究均表明成本效益良好;然而,获得的质量调整生命年(QALY)数量较少。不到一半的研究除了报告一般结果(大多为QALY)外,还报告了COPD特异性结果。发现急性加重率和死亡率是成本效益的主要驱动因素。根据QHES,研究质量总体上是足够的,但进一步评估显示,大多数研究未能很好地反映现实生活中药物使用的成本效益。
大多数研究表明,COPD药物维持治疗具有成本效益,但大多数研究未能很好地反映现实生活中的药物使用情况。建议采用一致且针对COPD的方法学。