Kobelt Gisela, Berg Jenny, Atherly Deborah, Hadjimichael Olympia
Karolinska Institute, Stockholm, Sweden.
Neurology. 2006 Jun 13;66(11):1696-702. doi: 10.1212/01.wnl.0000218309.01322.5c.
To estimate current costs and quality of life (utility) of patients treated with disease modifying drugs (DMDs) in the United States, and to investigate the effect of disease severity on costs and utility.
Data on demographics, disease information, resource utilization (including work capacity), and utility were collected directly from patients in a cross-sectional postal survey. Patients were selected randomly among participants in the North American Committee on Multiple Sclerosis Patient Registry (NARCOMS) receiving DMDs. Mean annual costs per patient (2004 USD) and mean utilities for the sample and for different levels of functional disability are estimated from the societal perspective.
The target answer rate of 50% was reached and 1,909 patients were included in the analysis. The mean age was 49 years and time since diagnosis was 13 years. A total of 10.5% of patients had primary progressive, 47.6% relapsing-remitting, and 33.3% secondary progressive disease. A total of 28.8% of patients indicated to have experienced a relapse during the past 3 months. Total average costs are estimated at 47,215 dollars per patient and year. Of these, 53% were for direct medical and non-medical costs, 37% for production losses, and 10% for informal care. Mean utility in the sample was 0.698.
Consistent with other studies, costs and utilities were significantly correlated with functional capacity. The proportion of costs represented by disease modifying drugs is considerably lower than estimated in other studies, as all costs related to the disease are included.
评估美国使用疾病修饰药物(DMDs)治疗的患者的当前成本及生活质量(效用),并研究疾病严重程度对成本和效用的影响。
通过横断面邮政调查直接从患者处收集人口统计学、疾病信息、资源利用(包括工作能力)和效用数据。在北美多发性硬化症患者登记委员会(NARCOMS)接受DMDs治疗的参与者中随机选择患者。从社会角度估计每位患者的年均成本(2004年美元)以及样本和不同功能残疾水平的平均效用。
达到了50%的目标应答率,1909名患者纳入分析。平均年龄为49岁,确诊时间为13年。10.5%的患者患有原发性进行性疾病,47.6%为复发缓解型,33.3%为继发性进行性疾病。共有28.8%的患者表示在过去3个月内经历过复发。估计每位患者每年的总平均成本为47215美元。其中,53%用于直接医疗和非医疗成本,37%用于生产损失,10%用于非正式护理。样本中的平均效用为0.698。
与其他研究一致,成本和效用与功能能力显著相关。疾病修饰药物所占成本比例远低于其他研究中的估计,因为纳入了与该疾病相关的所有成本。