Economic Burden of Gaucher Disease at a Tertiary Care Public Hospital in Mumbai.

Autor: Mhatre SP; Genetic Clinic, Department of Pediatrics, Seth GS Medical College and KEM Hospital, Parel, Mumbai, 400012, India., Muranjan M; Genetic Clinic, Department of Pediatrics, Seth GS Medical College and KEM Hospital, Parel, Mumbai, 400012, India. muranjanmamta@rediffmail.com., Gogtay NJ; Department of Clinical Pharmacology, Seth GS Medical College and KEM Hospital, Parel, Mumbai, 400012, India.
Jazyk: angličtina
Zdroj: Indian journal of pediatrics [Indian J Pediatr] 2024 May; Vol. 91 (5), pp. 463-469. Date of Electronic Publication: 2023 Jul 24.
DOI: 10.1007/s12098-023-04740-4
Abstrakt: Objectives: To estimate the economic burden of patients diagnosed with Gaucher disease at a public hospital from a societal perspective.
Methods: 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.
Results: 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.
Conclusions: 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.
(© 2023. The Author(s), under exclusive licence to Dr. K C Chaudhuri Foundation.)
Databáze: MEDLINE
Externí odkaz: