Presentation (CTI)

Problem Statement: Duchenne muscular dystrophy (DMD) is the most common hereditary neuromuscular disease. With no causal therapy currently available in Germany, DMD causes progressive muscle degeneration from early childhood, leading to severe disability with mobility loss and premature death. This study aimed to determine the societal costs of DMD in Germany in 2022. Further socioeconomic studies that also assess the potential benefit of novel gene therapies are currently ongoing in Austria and Switzerland.
Description: The average annual costs per person with DMD (pwDMD), lifetime costs for a pwDMD with average disease course, and total societal annual costs for all pwDMD were assessed based on a targeted literature review. Recent studies providing Germany-specific data by DMD stage (ambulatory/non-ambulatory) were included. Costs were adjusted to 2022 price levels based on relevant price developments for individual cost components and analysed by DMD stage and cost type (direct: medical/non-medical expenses; indirect: productivity loss/informal care; intangible: loss of quality of life).
Lessons Learned: Three studies providing data from 2012 were included. For 2022, an estimated 2,905 pwDMD incurred average annual costs of EUR 136,500 per pwDMD, with in-/direct costs accounting for EUR 90,200/46,300. Costs increased steeply in later non-ambulatory stages, with ambulatory stages contributing EUR 40,100-45,900 and non-ambulatory stages contributing EUR 67,500-217,200 per pwDMD per year. Costs increased considerably upon entering non-ambulatory stages until death. Cumulative lifetime costs for a pwDMD with average disease course were EUR 4.98M (million). Nearly half (46%) of the indirect costs were incurred due to patients’ productivity losses following their death. Total annual societal costs were EUR 400M (95% CI regarding prevalence estimate: 280-560M) with 66% indirect costs, including 23% due to informal care and 24% due to premature death (productivity losses over 31 lost working years on average).
Stakeholder Perspective: DMD imposes significant costs on society and the German healthcare system that increase steeply with disease progression, generating lifetime costs of EUR 4.98M per pwDMD and EUR 400M in total annual costs for all pwDMD. Therefore, future therapies with the potential to slow disease progression in its early stages and prolong life expectancy could effectively reduce the socioeconomic impact of DMD.