Genetic Clinic, Department of Pediatrics, Seth GS Medical College and KEM Hospital, Parel, Mumbai, 400012, India.
Department of Clinical Pharmacology, Seth GS Medical College and KEM Hospital, Parel, Mumbai, 400012, India.
Indian J Pediatr. 2024 May;91(5):463-469. doi: 10.1007/s12098-023-04740-4. Epub 2023 Jul 24.
To estimate the economic burden of patients diagnosed with Gaucher disease at a public hospital from a societal perspective.
Data from 30 Gaucher patients visiting the Genetic Clinic of the Department of Pediatrics at the study site in Mumbai was analyzed between January 2019 and January 2021. A cost of illness analysis was undertaken to estimate direct, indirect and intangible costs. Costs in treated and treatment naive groups were compared.
The total cost (direct and indirect) for 30 patients was ₹25,45,74,743/- (3440199.2 USD). Majority of this cost (99.8%) was due to direct costs of which medications [Enzyme replacement therapy (ERT) and Substrate reduction therapy (SRT)] constituted 98.8%. The notional cost was ₹1,43,94,695. Total costs of 14 treated patients were ₹25,29,67,279 and 16 treatment naive patients were ₹16,15,064 with a ratio of 157:1. Direct costs and cost of school absenteeism were significantly higher in the treated subgroup. Overall, direct, total costs and costs of school absenteeism were significantly associated with age and disease duration.
The economic burden of Gaucher disease is a staggering amount. This is an underestimate, as the expenses are highly subsidized in a public health facility. The highest contributor to cost component was direct costs, especially medication costs. Against the backdrop of the National Policy for Rare Diseases, resource allocation towards Gaucher disease should consider short term measures for judicious funding or reimbursement of disease-specific therapy and long-term cost-effective measures for promoting preventive strategies as the most practically feasible solution to reduce this economic burden.
从社会角度估计在公立医院诊断出戈谢病患者的经济负担。
在 2019 年 1 月至 2021 年 1 月期间，分析了在孟买研究现场儿科遗传诊所就诊的 30 名戈谢病患者的数据。进行了疾病成本分析，以估算直接、间接和无形成本。比较了治疗组和未治疗组的成本。
30 名患者的总费用（直接和间接）为 254574743 卢比（3440199.2 美元）。其中大部分（99.8%）是直接成本，其中药物[酶替代疗法（ERT）和底物减少疗法（SRT）]占 98.8%。名义成本为 14394695 卢比。14 名治疗患者的总费用为 252967279 卢比，16 名未治疗患者为 1615064 卢比，比例为 157:1。治疗亚组的直接成本和学校缺勤成本明显更高。总体而言，直接成本、总费用和学校缺勤费用与年龄和疾病持续时间显著相关。
戈谢病的经济负担是惊人的。这是一个低估，因为在公共卫生机构，费用得到了高度补贴。成本构成中最高的是直接成本，尤其是药物成本。在国家罕见病政策的背景下，戈谢病的资源分配应考虑短期措施，以合理资助或报销疾病特异性治疗，并长期采取具有成本效益的措施，以促进预防策略，作为减轻这一经济负担的最实际可行的解决方案。
