Mortality Cost of Duchenne Muscular Dystrophy

Abstract

Background Despite advances in management and care, Duchenne muscular dystrophy (DMD) remains universally fatal. The objective of this study was to estimate the mortality cost of DMD. Methods We estimated the mean total national annual mortality cost associated with DMD by simulating the mean number of patients who would have been alive in 2012 in the absence of DMD using data on the mean DMD incidence, mean number of live male births, mean life expectancy at birth in DMD and the male general population, and a societal willingness-to-pay (WTP) for a life-year. We attributed each patient who would have been alive in 2012 a lost life-year. Finally, to estimate the mean mortality cost of DMD, we multiplied the estimated mean number of life-years lost with a societal WTP for a life-year of €75,000. Results The mean total number of patients who would have been alive in 2012 in the absence of DMD was estimated at 4470 (95% bootstrapped CI: 4449-4492) in Germany, 3313 (3297-3329) in Italy, 3564 (3547-3581) in the UK, and 16,105 (16,029-16,186) in the USA. The corresponding mean mortality cost (in millions) was estimated at €335 (€334-€337) for Germany, €248 (€247-€250) for Italy, €267 (€266-€269) for the UK, and €1,208 (€1,202-€1,214) for the USA. Conclusions We show that DMD is associated with a considerable mortality cost and a substantial total economic burden to society. Our findings serve as important intelligence input to health economic policy decisions, allocation of funds for research, and cost-effective care delivery systems.