The social costs of Duchenne muscular dystrophy in Austria

Duchenne muscular dystrophy (DMD) is a degenerative muscle disease that is inherited via the X-chromosome. The gradual degeneration of the muscles leads to complete paralysis of the extremities and thus to the loss of the ability to walk, to a high level of care dependency and finally to a premature death. There are no licensed therapies in Austria at present that can stop the degenerative progress of the disease. What are the social costs of DMD in Austria? For the first time, we deter-mined the direct, indirect and intangible costs for each of the four stages of the disease. Wherever possible, we referred to Austrian information on prices and quantities and alternatively used information from international literature or expert estimates. The social costs of DMD totalled EUR 30 million in 2023, which corre-sponds to EUR 131,000 per patient. Of this amount, 78% (EUR 23.5 mil-lion) were indirect costs arising from productivity losses among patients and their relatives (informal caregiving) and from premature death. The indirect costs were significantly higher than the direct costs in all four stages of the disease. The majority of the social costs (48%) were incurred during the last stages of the disease in which the patients have already lost their ability to walk. At last, we estimated the lifetime costs of one patient with an average disease progression to be EUR 4.7 million, accompanied by a loss of ap-proximately 60 quality-adjusted life years (QALYs).