Phillips Ceri J
School of Health Science, Centre for Health Economics and Policy Studies, University of Wales-Swansea, Swansea, Wales, UK.
CNS Drugs. 2004;18(9):561-74. doi: 10.2165/00023210-200418090-00002.
Multiple sclerosis (MS) is one of the most common causes of neurological disability in young and middle-aged adults. The full economic cost of MS is substantial given that MS patients experience a major perturbation in their daily activities and the disease affects mainly young people who are obliged to restrict their levels of economic activity, either temporarily or permanently. A positive relationship exists between the direct and indirect costs of MS and its severity. Cost variations between countries exist because of differences in the costs of inpatient care, the number of ambulatory visits, drug usage and the extent and type of informal care. The development and availability of new agents has been accompanied by an increased optimism that treatment regimens for MS would be more effective. However, doubts have been expressed about the effectiveness of these treatments, which have compounded the problems associated with estimating the relative cost effectiveness of such interventions. In addition, variations in the utility scores associated with disease categories, the impact of relapses and the resulting utility losses, plus the speed of disease progression have all contributed to the difficulty of estimating the quality-adjusted life year (QALY) losses for a patient experiencing MS. Differences between studies with respect to the costs associated with each disability level, the timescale of the disease and the period over which costs and QALYs are to be measured, and the perspective employed in relation to costing have also resulted in a wide range of estimates being produced for the cost effectiveness of interferons and glatiramer acetate in the management of MS. These range from situations of cost savings, to over $US1.6 million (euro1.85 million) per QALY gained. Recent cost-effectiveness studies have benefited from more relevant and up-to-date data relating to disease progression and have generally produced more favourable cost-effectiveness ratios. However, the lack of homogeneity in the design of the studies partly accounts for the extent of variation in the estimates of cost effectiveness, and the difficulty of arriving at a consensus. The UK Department of Health has introduced a scheme that provides disease-modifying agents in the National Health Service for those patients with clinically active relapsing disease. Patients are monitored annually and payments to manufacturers are dependent on outcomes achieved. This initiative, although not without its detractors, will hopefully enhance the quantity and quality of evidence on the impact of drugs on disease progression and address some of the current difficulties with estimating the relative cost effectiveness of disease-modifying drugs in the treatment of patients with MS.
多发性硬化症(MS)是中青年人群神经功能残疾的最常见病因之一。鉴于MS患者的日常活动受到严重干扰,且该疾病主要影响年轻人,他们不得不暂时或永久限制其经济活动水平,因此MS的全部经济成本相当高昂。MS的直接和间接成本与其严重程度之间存在正相关关系。由于住院护理成本、门诊就诊次数、药物使用情况以及非正式护理的范围和类型存在差异,各国之间的成本也有所不同。新药物的研发和可及性让人们越来越乐观地认为MS的治疗方案会更有效。然而,有人对这些治疗方法的有效性表示怀疑,这使得评估此类干预措施相对成本效益的问题更加复杂。此外,与疾病类别相关的效用评分变化、复发的影响以及由此导致的效用损失,再加上疾病进展速度,都增加了估算MS患者质量调整生命年(QALY)损失的难度。不同研究在与每个残疾水平相关的成本、疾病的时间尺度以及成本和QALY的测量周期,以及成本核算所采用的视角等方面存在差异,这也导致了对干扰素和醋酸格拉替雷治疗MS的成本效益产生了广泛的估计。这些估计范围从成本节约情况到每获得一个QALY超过160万美元(185万欧元)不等。最近的成本效益研究受益于与疾病进展相关的更相关和最新的数据,并且总体上产生了更有利的成本效益比。然而,研究设计缺乏同质性在一定程度上解释了成本效益估计差异的程度,以及达成共识的困难。英国卫生部推出了一项计划,为患有临床活动性复发疾病的患者在国家医疗服务体系中提供疾病修正药物。患者每年接受监测,向制造商的付款取决于取得的结果。这项举措虽然并非没有批评者,但有望提高关于药物对疾病进展影响的证据数量和质量,并解决目前在估计疾病修正药物治疗MS患者的相对成本效益方面的一些困难。