Economic burden of Duchenne muscular dystrophy from a societal perspective in Mumbai, India

INTRODUCTION: Healthcare demands of Duchenne muscular dystrophy (DMD) impose a significant burden with the possibility of country-specific differences. MATERIAL AND METHODS: Analysis to determine cost-of-illness from a societal perspective was performed by prevalence-based, bottom-up microcosting in 60 boys with DMD between July 2022 and January 2024. Direct (medical and nonmedical), indirect (productivity losses of caregivers) and intangible (cost of suffering monetized by willingness to pay method) costs were estimated. National burden was calculated using India-specific prevalence. RESULTS: The cumulative annual cost for 60 patients was ₹ 23,987,323 (USD 290,157). The total median annual cost per patient was ₹ 268,325 (USD 3245). The national economic burden of DMD was ₹ 20,549,902,776 (248,577,510 USD). The highest cost driver of direct costs was expenditure on complementary and alternative medicines and unapproved therapies (₹ 8,549,790 or USD 103,420). Nature of therapy was a significant predictor of total, direct, and intangible costs ( P < 0.001, 0.003, and < 0.001, respectively). Residence in tier X city was a predictor for direct ( P = 0.042) and intangible costs ( P = 0.032). Residence in urban areas ( P = 0.022) and Mumbai ( P = 0.002) were significant predictors of intangible costs. CONCLUSIONS: Despite some limitations, the study provides directions for planning healthcare services for DMD in India by developing standard treatment guidelines, allocation of resources through national health schemes for carrier screening and prenatal diagnosis, promoting evidence-based therapeutic practices, and regulation to curb unscientific therapies. Baseline data are available to decide cost-effectiveness of public expenditure for disease-modifying drugs for DMD.