Heinrich Ria, Claus Franziska, Schoenfelder Tonio
Scientific Institute of Health Economics and Health System Research (WIG2 GmbH), Leipzig, Germany.
Chair Health Sciences-Public Health, Technische Universität Dresden, Dresden, Germany.
Orphanet J Rare Dis. 2024 Dec 16;19(1):462. doi: 10.1186/s13023-024-03492-4.
Lysosomal storage diseases (LSDs) can be treated with intravenous enzyme replacement therapy (ERT). ERT is being administered either in specialized clinics or in the home care setting. Studies indicate that home-based ERT can be considered safe and positively effects patient reported outcomes. Due to the higher level of individual care required by nurses when administering ERT at home, questions arise whether this will result in additional costs for health care systems. Since cost data to the German health care system are currently unavailable, this study aims to assess home care ERT costs of LSD patients.
The longitudinal study assessed the direct costs of home-based ERT for patients with LSD diseases Fabry, Pompe, Gaucher, and MPS I from 2019 to 2021 on a patient-by-patient basis by evaluating the healthcare documentation. Costs assessed included ERT drug and further administration equipment, time needed to prepare, administer, and post-process the infusion, and travel expenses of nursing staff.
Data from 62 patients was analyzed: of these, 29 (46.8%) with Fabry disease, 19 (30.6%) with Pompe disease, 10 (16.1%) with Gaucher disease, and 4 (6.5%) with MPS I. Patients ranged in age from 3 to 79 (mean 40 years); 42% of patients reported comorbidities (19.4% with hypertension, and 10% with heart disease). Mean total ERT-related costs were €369,047 per year across all patients. Approximately 98.5% of these costs were attributable to the infusion therapy and administration itself (€363,880), and approximately 1.5% were attributable to personnel and travel costs. Patients with Pompe disease incurred the highest average annual costs (€483,907) and patients with Fabry disease the lowest (€264,896). Cost differences among underlying LSDs were due primarily to ERT drug costs; the drug infused for Fabry disease costs about half as much as that for Gaucher or for Pompe disease. Despite MPS I patients requiring about twice as many infusions, significantly lower drug costs for this disease resulted in the second lowest total mean costs of all LSD subtypes analyzed.
As total costs are almost entirely driven by infusion-related material, especially the ERT drug, moving ERT into the home environment is not expected to increase costs.
溶酶体贮积症（LSDs）可用静脉内酶替代疗法（ERT）进行治疗。ERT可在专科诊所或居家护理环境中给药。研究表明，居家ERT可被认为是安全的，并且对患者报告的结局有积极影响。由于护士在家中进行ERT给药时需要更高水平的个体化护理，因此出现了这是否会给医疗保健系统带来额外成本的问题。由于目前尚无德国医疗保健系统的成本数据，本研究旨在评估LSD患者居家护理ERT的成本。
这项纵向研究通过评估医疗保健文档，逐个患者地评估了2019年至2021年期间法布里病、庞贝病、戈谢病和I型粘多糖贮积症（MPS I）患者居家ERT的直接成本。评估的成本包括ERT药物和其他给药设备、准备、给药和输液后处理所需的时间，以及护理人员的差旅费。
分析了62例患者的数据：其中，29例（46.8%）患有法布里病，19例（30.6%）患有庞贝病，10例（16.1%）患有戈谢病，4例（6.5%）患有MPS I。患者年龄在3岁至79岁之间（平均40岁）；42%的患者报告有合并症（19.4%患有高血压，10%患有心脏病）。所有患者的平均每年ERT相关总成本为369,047欧元。这些成本中约98.5%归因于输液治疗和给药本身（363,880欧元），约1.5%归因于人员和差旅费。庞贝病患者的平均年度成本最高（483,907欧元），法布里病患者的成本最低（264,896欧元）。基础LSDs之间的成本差异主要归因于ERT药物成本；用于法布里病的输注药物成本约为戈谢病或庞贝病的一半。尽管MPS I患者需要的输液次数约为其他患者的两倍，但该疾病显著较低的药物成本导致其在所有分析的LSD亚型中总平均成本第二低。
由于总成本几乎完全由输液相关材料，尤其是ERT药物驱动，因此将ERT转移到家庭环境中预计不会增加成本。
