Emery Paul
Department of Rheumatology, Leeds General Infirmary, Leeds, England.
Pharmacoeconomics. 2004;22(2 Suppl 1):55-69. doi: 10.2165/00019053-200422001-00006.
As the cost of drug treatment for rheumatoid arthritis (RA) constitutes only a small proportion of total costs of the disease to individuals and society, therapeutic interventions have the potential for significant economic benefit. To take advantage of this potential, clinicians need to gain a global, long-term perspective on patient care. Economic evaluations of RA therapies are critically important in influencing decisions regarding the role of costly, but highly effective new therapies, particularly in settings where there are financial constraints on healthcare provisions. Such evaluations, therefore, need to be methodologically similar with valid results to enhance their value to clinicians and policy decision-makers. This requires the use of appropriate elements in the numerator (i.e. total number of dollars spent on healthcare as a result of the intervention) and the denominator (net health effectiveness) components of the cost-effectiveness equation. Other important design factors also need to be managed properly to ensure validity of the evaluation. In this regard, the guidelines proposed by the Outcome Measures in Rheumatoid Arthritis Clinical Trials (OMERACT) Task Force represent a useful approach to help create common standards for economic evaluations in RA. Recently, the development of a number of decision analysis models in RA has helped predict the likely cost-effectiveness of new interventions such as the anti-tumour necrosis factor (TNF)-alpha agents, etanercept and infliximab, both of which have been found to be cost-effective relative to other disease-modifying anti-rheumatic drugs (DMARDs) using short-term efficacy endpoints. In comparisons of these two agents in patients with DMARD-resistant RA, etanercept has been shown to be more cost-effective than the combination of methotrexate and infliximab, administered in various dosages, over a period of 1 year using American College of Rheumatology (ACR) response rates as the primary efficacy measure. However, the criteria for determining clinical efficacy is paramount and other studies that use radiographic progression as a measure of clinical effectiveness show no difference between etanercept and infliximab in clinical efficacy. Important issues that need to be considered in developing economic models in RA include consideration of the connection between the prevention of radiographic progression and downstream economic consequences, and the need to employ lifetime models wherever possible because a long time period is necessary to determine the true cost-effectiveness of agents that modify radiographic progression of RA, such as etanercept, infliximab, and adalimumab. In doing so, it is hoped that such studies will provide optimal information to facilitate important decisions on resource allocation.
由于类风湿性关节炎(RA)的药物治疗成本在该疾病给个人和社会造成的总成本中仅占一小部分,治疗干预措施具有产生显著经济效益的潜力。为了利用这一潜力,临床医生需要对患者护理有一个全面、长期的视角。RA治疗的经济评估对于影响有关昂贵但高效的新疗法作用的决策至关重要,特别是在医疗保健供应存在资金限制的情况下。因此,此类评估在方法上需要相似且结果有效,以提高其对临床医生和政策决策者的价值。这就要求在成本效益等式的分子(即因干预而花费在医疗保健上的总金额)和分母(净健康效益)部分使用适当的要素。其他重要的设计因素也需要妥善管理,以确保评估的有效性。在这方面,类风湿性关节炎临床试验结果测量(OMERACT)特别工作组提出的指南是一种有用的方法,有助于为RA的经济评估制定共同标准。最近,RA中一些决策分析模型的开发有助于预测新干预措施(如抗肿瘤坏死因子(TNF)-α药物依那西普和英夫利昔单抗)可能的成本效益,使用短期疗效终点时,相对于其他改善病情抗风湿药物(DMARDs),这两种药物都被发现具有成本效益。在对DMARD抵抗性RA患者中这两种药物的比较中,以美国风湿病学会(ACR)反应率作为主要疗效指标,在1年的时间里,依那西普已被证明比不同剂量的甲氨蝶呤和英夫利昔单抗联合用药更具成本效益。然而,确定临床疗效的标准至关重要,其他以影像学进展作为临床疗效衡量指标的研究表明,依那西普和英夫利昔单抗在临床疗效上没有差异。在开发RA经济模型时需要考虑的重要问题包括考虑预防影像学进展与下游经济后果之间的联系,以及尽可能采用终身模型的必要性,因为需要很长时间来确定改变RA影像学进展的药物(如依那西普、英夫利昔单抗和阿达木单抗)的真正成本效益。这样做的目的是希望此类研究将提供最佳信息,以促进有关资源分配的重要决策。