Department of Economics, Universitat de Barcelona, Diagonal 696, 08034, Barcelona, Spain.
Clin Drug Investig. 2020 Apr;40(4):335-341. doi: 10.1007/s40261-020-00897-4.
Spinal muscular atrophy (SMA) is a rare disorder, estimated to affect 1 per 10,000 live births. Patients affected with SMA often require intensive, chronic healthcare, which represents great social and economic costs.
This study aimed to evaluate the direct medical costs of SMA, from the National Health System perspective in Catalonia, and provide regional data for the development of optimal disease management protocols and resource allocation decisions at the regional level.
A retrospective, population-based study was designed based on admission records from primary care centres, hospitals and specialised care settings (inpatient and outpatient care), emergency services and extended care facilities obtained from a regional governmental claims database.
A total of 396 patients met the inclusion criteria. Annual direct medical costs summed €58,606 per patient, taking into account the use of healthcare resources at all levels of care and excluding the cost of prescription medication. Specialised care represented 81% of the expenses that were mostly associated with respiratory manifestations of SMA. In the year 2016, 71.26% of patients with SMA had four or more systems affected by a chronic condition, versus 23.50% in the general population, which had an impact on healthcare use.
Inpatient extended care and the increased presence of multimorbid chronic conditions in patients with SMA must be taken into account in order to develop multidisciplinary treatment protocols that reflect the complexity of SMA. Forthcoming resource allocation decisions should reflect the intensive use of specialised care registered in patients with SMA.
脊髓性肌萎缩症（SMA）是一种罕见疾病，估计每 10000 例活产儿中就有 1 例受其影响。患有 SMA 的患者通常需要密集、慢性的医疗保健，这代表着巨大的社会和经济成本。
本研究旨在从加泰罗尼亚国家卫生系统的角度评估 SMA 的直接医疗成本，并提供区域数据，为制定最佳疾病管理方案和在区域层面分配资源做出决策。
设计了一项基于初级保健中心、医院和专门护理机构（住院和门诊护理）、急诊服务和延长护理设施的入院记录的回顾性、基于人群的研究，这些记录均来自一个地区政府的报销数据库。
共有 396 名患者符合纳入标准。考虑到在所有护理水平上使用医疗资源，不包括处方药物的成本，每名患者的年直接医疗费用总计为 58606 欧元。专门护理占费用的 81%，这些费用主要与 SMA 的呼吸表现有关。在 2016 年，71.26%的 SMA 患者有四个或更多系统受到慢性疾病的影响，而普通人群中这一比例为 23.50%，这对医疗保健的使用产生了影响。
必须考虑 SMA 患者的住院延长护理和多种慢性合并症的增加，以制定反映 SMA 复杂性的多学科治疗方案。即将做出的资源分配决策应反映 SMA 患者登记的专门护理的密集使用。
