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德国囊性纤维化疾病负担:来自大型囊性纤维化中心的结果。

Cost of illness of cystic fibrosis in Germany: results from a large cystic fibrosis centre.

机构信息

Institute for Health Care Management and Health Economics, Faculty of Economics and Business Administration, University of Hamburg, Germany.

出版信息

Pharmacoeconomics. 2012 Sep 1;30(9):763-77. doi: 10.2165/11588870-000000000-00000.

Abstract

BACKGROUND

Cystic fibrosis (CF) is the most common life-shortening genetic disorder among Whites worldwide. Because many of these patients experience chronic endobronchial colonization and have to take antibiotics and be treated as inpatients, societal costs of CF may be high. As the disease severity varies considerably among patients, costs may differ between patients.

OBJECTIVES

Our objectives were to calculate the average total costs of CF per patient and per year from a societal perspective; to include all direct medical and non-medical costs as well as indirect costs; to identify the main cost drivers; to investigate whether patients with CF can be grouped into homogenous cost groups; and to determine the influence of specific factors on different cost categories.

METHODS

Resource utilization data were collected for 87 patients admitted to an inpatient unit at a CF treatment centre during the first 6 months of 2004 and 125 patients who visited the centre's CF outpatient unit during the entire year. Fifty-four patients were admitted to the hospital and also visited the outpatient unit. Since all patients were exclusively treated at the centre, data could be aggregated. Costs that varied greatly between patients were measured per patient. The remaining costs were summarized as overhead costs and allocated on the basis of days of treatment or contacts per patient. Costs of the outpatient and inpatient units and costs for drugs patients received at the outpatient pharmacy were summarized as direct medical costs. Direct non-medical costs (i.e. travel expenses), as well as indirect costs (i. e. absence from work, productivity losses), were also included in the analysis. Main cost drivers were detected by the analysis of different cost categories. Patients were classified according to a diagnosis-related severity model, and median comparison tests (Wilcoxon-Mann-Whitney tests) were performed to investigate differences between the severity groups. Generalized least squares (GLS) regressions were used to identify variables influencing different cost categories. A sensitivity analysis using Monte Carlo simulation was performed.

RESULTS

The mean total cost per patient per year was &U20AC;41 468 (year 2004 values). Direct medical costs accounted for more than 90% of total costs and averaged &U20AC;38 869 (&U20AC;3876 to &U20AC;88 096), whereas direct non-medical costs were minimal. Indirect costs amounted to &U20AC;2491 (6% of total costs). Costs for drugs patients received at the outpatient pharmacy were the main cost driver. Costs rose with the degree of severity. Patients with moderate and severe disease had significantly higher direct costs than the relatively milder group. Regression analysis revealed that direct costs were mainly affected by the diagnosis-related severity level and the expiratory volume; the coefficient indicating the relationship between costs for mild CF patients and other patients rose with the degree of severity. A similar result was obtained for drug costs per patient as the dependent variable. Monte Carlo simulation suggests that there is a 90% probability that annual costs will be lower than &U20AC;37 300.

CONCLUSIONS

The share of indirect costs as a percentage of total costs for CF was rather low in this study. However, the relevance of indirect costs is likely to increase in the future as the life expectancy of CF patients increases, which is likely to lead to a rising work disability rate and thus increase indirect costs. Moreover we found that infection with Pseudomonas aeruginosa increases costs substantially. Thus, a decrease of the prevalence of P. aeruginosa would lead to substantial savings for society.

摘要

背景

囊性纤维化(CF)是全世界白人中最常见的缩短寿命的遗传疾病。由于许多此类患者经历慢性支气管内定植,并且必须服用抗生素并作为住院患者接受治疗,因此 CF 的社会成本可能很高。由于患者之间的疾病严重程度差异很大,因此患者之间的成本可能会有所不同。

目的

我们的目标是从社会角度计算每位 CF 患者每年的平均总成本;包括所有直接医疗和非医疗成本以及间接成本;确定主要成本驱动因素;调查 CF 患者是否可以分为同质成本组;并确定特定因素对不同成本类别的影响。

方法

收集了 2004 年上半年在 CF 治疗中心住院部住院的 87 名患者和全年在中心 CF 门诊就诊的 125 名患者的资源利用数据。54 名患者住院并同时就诊于门诊。由于所有患者均仅在中心接受治疗,因此可以汇总数据。对患者之间差异较大的成本进行了逐人测量。其余成本被概括为间接成本和管理费用,并根据每位患者的治疗天数或接触次数进行分配。门诊和住院部门的成本以及患者在门诊药房接受的药物成本被汇总为直接医疗成本。还包括了直接非医疗成本(即旅行费用)和间接成本(即旷工、生产力损失)。通过不同成本类别的分析来检测主要成本驱动因素。根据与诊断相关的严重程度模型对患者进行分类,并进行中位数比较检验(Wilcoxon-Mann-Whitney 检验)以调查严重程度组之间的差异。使用广义最小二乘法(GLS)回归来确定影响不同成本类别的变量。使用蒙特卡罗模拟进行敏感性分析。

结果

每位患者每年的平均总成本为 41468 欧元(2004 年的价值)。直接医疗成本占总成本的 90%以上,平均为 38869 欧元(3876 至 88096 欧元),而直接非医疗成本微不足道。间接成本为 2491 欧元(占总成本的 6%)。患者在门诊药房接受的药物成本是主要的成本驱动因素。成本随严重程度的增加而增加。中度和重度疾病患者的直接费用明显高于相对较轻的组。回归分析显示,直接成本主要受与诊断相关的严重程度水平和呼气量的影响;表明轻度 CF 患者与其他患者之间成本关系的系数随严重程度的增加而增加。作为因变量,药物成本的个体患者也得到了类似的结果。蒙特卡罗模拟表明,年度成本低于 37300 欧元的可能性为 90%。

结论

在这项研究中,CF 总间接成本占总成本的比例相对较低。然而,随着 CF 患者预期寿命的延长,间接成本的相关性可能会增加,这可能会导致工作残疾率上升,从而增加间接成本。此外,我们发现铜绿假单胞菌感染会大大增加成本。因此,降低铜绿假单胞菌的流行率将为社会带来实质性的节省。

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