Santhera Pharmaceuticals Ltd Liestal, Switzerland.
Front Pharmacol. 2013 May 22;4:66. doi: 10.3389/fphar.2013.00066. eCollection 2013.
The study intended to substantiate healthcare resource utilization, costs, and funding patterns of US and Canadian Friedreich's Ataxia (FRDA) populations, to assess compliance with treatment guidance and to identify areas where novel healthcare measures or improved access to existing care may improve patients' functional and social capabilities and reduce the financial impact on the healthcare systems.
Healthcare resource utilization and costs were collected in a cross-sectional study in the US (N = 197) and Canada (N = 43) and analyzed across severity of disease categories. Descriptive statistics, correlation analysis, and hypothesis testing were applied.
In the US, healthcare costs of FRDA patients were higher than those of "adults with two and more chronic conditions." Significantly higher costs were incurred in advanced stages of the disease, with paid homecare being the main driver. This pattern was also observed in Canada. Compliance with the recommended annual neurological and cardiological follow-up was high, but was low for the recommended regular speech therapy. In the US public and private funding ratios were similar for the FRDA and the general populations. In Canada the private funding ratio for FRDA was higher than average.
The variety of healthcare measures addressing the broad range of symptoms of FRDA, and the increasing use of paid home care as disease progresses made total US healthcare costs of FRDA exceed the costs of US adults with two and more chronic conditions. Therefore, measures delaying disease progression will allow patients to maintain their independence longer and may reduce costs to the healthcare system. Novel measures to address dysarthria and to ensure access to them should be further investigated. The higher than average private funding ratio in Canada was due to the relatively high cost of the pharmacological treatment of FRDA.
本研究旨在证实美国和加拿大弗里德里希共济失调（FRDA）人群的医疗资源利用、成本和资金模式，评估治疗指南的遵守情况，并确定可能改善患者功能和社交能力并减轻医疗系统财务负担的新医疗措施或改善现有护理的获取途径。
在美国（N=197）和加拿大（N=43）进行了一项横断面研究，收集了医疗资源利用和成本数据，并按疾病严重程度类别进行了分析。应用了描述性统计、相关分析和假设检验。
在美国，FRDA 患者的医疗保健费用高于“有两种或多种慢性疾病的成年人”。在疾病的晚期，成本显著增加，而付费家庭护理是主要驱动因素。这一模式在加拿大也观察到。建议的年度神经学和心脏病学随访的依从性很高，但推荐的常规言语治疗的依从性较低。在美国，公共和私人资金的比例对于 FRDA 和一般人群相似。在加拿大，FRDA 的私人资金比例高于平均水平。
针对 FRDA 广泛症状的各种医疗措施的使用，以及随着疾病的进展越来越多地使用付费家庭护理，使得 FRDA 的总美国医疗保健成本超过了美国有两种或多种慢性疾病的成年人的成本。因此，延迟疾病进展的措施将使患者能够更长时间地保持独立，并可能降低医疗系统的成本。应进一步研究解决构音障碍的新措施，并确保获得这些措施。加拿大高于平均水平的私人资金比例归因于 FRDA 药物治疗的相对较高成本。
