Dzhambazova Elizabet, Kostadinov Kostadin, Tsenkova-Toncheva Lilia, Galabova Fani, Ezeldin Fares, Iskrov Georgi, Stefanov Rumen
Department of Social Medicine and Public Health, Faculty of Public Health, Medical University of Plovdiv, 4002 Plovdiv, Bulgaria.
Department of Pediatrics, Medical Faculty, Medical University of Plovdiv, 4002 Plovdiv, Bulgaria.
Healthcare (Basel). 2025 Feb 13;13(4):401. doi: 10.3390/healthcare13040401.
: The objective of our study was to quantify the annual costs, from a societal perspective, encompassing direct health care costs, direct non-health care costs, and labor productivity losses associated with spinal muscular atrophy (SMA) patients in Bulgaria and their caregivers. : We applied a prevalence-based, bottom-up costing methodology to assess the socio-economic burden of SMA from a societal perspective. We evaluated and summed up all costs for health services (diagnosis, treatment, follow-up, and rehabilitation), educational and social services, and formal and informal care in the community, as well as indirect costs due to the loss of productivity and work capacity of the SMA patients' caregivers. : Nine parents of SMA patients provided consent and completed the study's questionnaire. Two children had SMA type III, and seven had SMA type II. The median annual socio-economic burden per SMA patient was EUR 254,968.80. The high direct costs, primarily driven by drug expenses, and the substantial indirect costs resulting from the loss of productivity among informal caregivers were the primary causes. We found no utilization of social care and educational services. : We emphasize the need for careful consideration of long-term outcomes, real-world data collection, and performance-based reimbursement. An ideal scenario could achieve these objectives in synergy. A second layer of policy actions and measures must address the unmet needs of SMA patients and their families using a holistic approach. The indirect costs associated with SMA, particularly the productivity loss of informal caregivers, underscore the need for comprehensive support programs.
我们研究的目的是从社会角度量化与保加利亚脊髓性肌萎缩症（SMA）患者及其照料者相关的年度成本，包括直接医疗保健成本、直接非医疗保健成本以及劳动生产率损失。
我们采用基于患病率的自下而上成本核算方法，从社会角度评估SMA的社会经济负担。我们评估并汇总了医疗服务（诊断、治疗、随访和康复）、教育和社会服务、社区正式和非正式照料的所有成本，以及由于SMA患者照料者生产力和工作能力丧失而产生的间接成本。
9名SMA患者的家长同意并完成了研究问卷。2名儿童患有III型SMA，7名患有II型SMA。每名SMA患者的年度社会经济负担中位数为254,968.80欧元。主要由药品费用驱动的高额直接成本以及非正式照料者生产力丧失导致的大量间接成本是主要原因。我们发现社会照料和教育服务未得到利用。
我们强调需要认真考虑长期结果、收集实际数据以及基于绩效的报销。理想的情况可以协同实现这些目标。第二层政策行动和措施必须采用整体方法解决SMA患者及其家庭未满足的需求。与SMA相关的间接成本，特别是非正式照料者的生产力损失，凸显了全面支持计划的必要性。
