Estimated costs for Duchenne muscular dystrophy care in Brazil

Orphanet Journal of Rare Diseases

Research

Isabela Possebon Bevilacqua¹  Ana Lúcia Portella Staub¹  Jonas Alex Morales Saute²  Nayê Balzan Schneider³  Carisi Anne Polanczyk⁴  Ana Paula Beck da Silva Etges⁵  Tamiê de Camargo Martins⁶  Priscilla Loze⁶  Ana Carolina de Almeida⁶  Natalia Bergamelli Ramos⁶  Erica Caetano Roos⁷ 

[1] Centro de Pesquisa Clínica, Hospital de Clínicas de Porto Alegre, Neurogenética, Porto Alegre, Brazil;Centro de Pesquisa Clínica, Hospital de Clínicas de Porto Alegre, Neurogenética, Porto Alegre, Brazil;Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil;Programa de Pós-Graduação em Medicina: Ciências Médicas, Faculdade de Medicina, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;Departamento de Medicina Interna, Faculdade de Medicina, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;Serviço de Genética Médica, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil;Serviço de Neurologia, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil;National Institute of Science and Technology for Health Technology Assessment (IATS)- CNPq/Brazil (project: 465518/2014-1), Porto Alegre, Brazil;Programa de Pós-Graduação em Epidemiologia, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;National Institute of Science and Technology for Health Technology Assessment (IATS)- CNPq/Brazil (project: 465518/2014-1), Porto Alegre, Brazil;Programa de Pós-Graduação em Epidemiologia, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil;Departamento de Medicina Interna, Faculdade de Medicina, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;Hospital Moinhos de Vento, Porto Alegre, Rio Grande do Sul, Brazil;National Institute of Science and Technology for Health Technology Assessment (IATS)- CNPq/Brazil (project: 465518/2014-1), Porto Alegre, Brazil;Programa de Pós-Graduação em Epidemiologia, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;School of Technology, Pontifícia Universidade Católica do Rio Grande do Sul, Porto Alegre, Brazil;Produtos Roche Químicos e Farmacêuticos S/A, São Paulo, SP, Brazil;Programa de Pós-Graduação em Engenharia de Produção, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil;

关键词: Cost of illness;    Muscular dystrophy, Duchenne;    Neuromuscular diseases;    Rare diseases;   

DOI  :  10.1186/s13023-023-02767-6

 received in 2022-12-13, accepted in 2023-06-04,  发布年份 2023

来源: Springer

【 摘 要 】

BackgroundThe economic burden of rare diseases on health systems is still not widely measured, with the generation of accurate information about the costs with medical care for subjects with rare diseases being crucial when defining health policies. Duchenne Muscular Dystrophy (DMD) is the most common form of muscular dystrophy, with new technologies recently being studied for its management. Information about the costs related to the disease in Latin America is scarce, and the objective of this study is to evaluate the annual hospital, home care and transportation costs per patient with DMD treatment in Brazil.ResultsData from 27 patients were included, the median annual cost per patient was R$ 17,121 (IQR R$ 6,786; 25,621). Home care expenditures accounted for 92% of the total costs, followed by hospital costs (6%) and transportation costs (2%). Medications and loss of family, and patient’s productivity are among the most representative consumption items. When disease worsening due to loss of the ability to walk was incorporated to the analysis, it was shown that wheelchair users account for an incremental cost of 23% compared with non-wheelchair users.ConclusionsThis is an original study in Latin America to measure DMD costs using the micro-costing technique. Generating accurate information about costs is crucial to provide health managers with information that could help establish more sustainable policies when deciding upon rare diseases in emerging countries.

【 授权许可】

CC BY   
© The Author(s) 2023

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