Estimated costs for Duchenne muscular dystrophy care in Brazil

Abstract

Background The 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. Results Data 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. Conclusions This 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.

Social difficulties and care burden of adult Duchenne muscular dystrophy in Japan: a questionnaire survey based on the Japanese Registry of Muscular Dystrophy (Remudy)

“…Brain comorbidities can have a greater impact on the family than limited mobility for people living with DMD [ 8 ], although there have been no clinical trials or agents which target the brain of DMD patients. The economic burden and care burden of patients and families have also been reported in many countries [ 9 – 11 ]. Infrequent employment among adult DMD patients [ 12 ] contributes at least in part to the economic burden.…”

Higher Prevalence of Nonsense Pathogenic DMD Variants in a Single-Center Cohort from Brazil: A Genetic Profile Study That May Guide the Choice of Disease-Modifying Treatments

“…Since the discovery of DMD in 1986 [2], many different types of pathogenic variants have been described in dystrophinopathies, including large deletions and duplications, single-nucleotide variants and small rearrangements [3]. Data regarding DMD prevalence in Brazil are scarce and not yet estimated [4].…”