Lovelace Clinic Foundation, Albuquerque, MN 87106-4264, USA.
Pharmacoeconomics. 2012 Oct 1;30(10):869-85. doi: 10.2165/11633330-000000000-00000.
Chronic obstructive pulmonary disease (COPD) is one of the leading causes of death and disability worldwide, but before the development of several new pharmacological treatments little could be done for COPD patients. Recognition that these new treatments could significantly improve the prognosis for COPD patients has radically changed clinical management guidelines from a palliative philosophy to an aggressive approach intended to reduce chronic symptoms, improve quality of life and prolong survival. These new treatments have also sparked interest in COPD cost-effectiveness research. Most COPD cost-effectiveness studies have been based on clinical trial populations, limited to direct medical costs, and used standard analysis methods such as Markov modelling, and they have usually found that newer therapies have favourable cost effectiveness. However, new insights into the clinical progression of COPD bring into question some of the assumptions underlying older analyses. In this review, we examine clinical factors unique to COPD and recent changes in clinical perspectives that have important implications for pharmacoeconomic analyses. The main parameters explored include (i) the high indirect medical costs for COPD and their relevance in assessing the societal benefits of new therapy; (ii) the importance of acute deteriorations in COPD, known as exacerbations, and approaches to modelling the cost benefit of exacerbation reduction; (iii) quality/utility instruments for COPD; (iv) the prevalence of co-morbid conditions and confounding between COPD and co-morbid disease utilization; (v) the limitations of Markov modelling; and (vi) the problem of outliers.
慢性阻塞性肺疾病（COPD）是全球主要的死亡和致残原因之一，但在几种新的药理学治疗方法发展之前，对 COPD 患者几乎无能为力。人们认识到，这些新的治疗方法可以显著改善 COPD 患者的预后，这从根本上改变了临床管理指南，从姑息治疗理念转变为旨在减轻慢性症状、提高生活质量和延长生存时间的积极治疗方法。这些新的治疗方法也激发了对 COPD 成本效益研究的兴趣。大多数 COPD 成本效益研究都是基于临床试验人群，仅限于直接医疗成本，并使用标准分析方法，如马尔可夫模型，并且通常发现新的治疗方法具有良好的成本效益。然而，对 COPD 临床进展的新认识使一些基于旧分析的假设受到质疑。在这篇综述中，我们考察了 COPD 特有的临床因素和最近在临床观点上的变化，这些变化对药物经济学分析具有重要意义。主要探讨的参数包括：（i）COPD 的高额间接医疗成本及其在评估新疗法对社会的益处方面的相关性；（ii）COPD 急性恶化（称为恶化）的重要性，以及建模减轻恶化成本效益的方法；（iii）COPD 的质量/效用工具；（iv）合并症的流行率以及 COPD 与合并疾病利用之间的混杂；（v）马尔可夫模型的局限性；以及（vi）离群值的问题。
