Costs estimation of male patients with Duchenne muscular dystrophy in France: survey on medical costs non reimbursed, non-medical costs, and indirect costs

Introduction Duchenne muscular dystrophy (DMD) is a severe X-linked neuromuscular disorders with 150 to 200 potential new cases per year in France, characterized by progressive muscular atrophy. As there is very little information available in France about DMD costs, a baseline assessment is needed in view of new treatments’ development, as their impact on patient’s life will have an impact on the numerous expenses related to the disease. The objective was to describe the consumption of all resources not eligible for reimbursement related to the care provided to DMD patients in terms of direct medical costs, direct non-medical costs, medical social costs, informal care costs and indirect costs. Methods Patients with DMD diagnosis and their caregivers were recruited through the National Rare Disease Data Bank and questioned about resource consumption related to DMD using “ad-hoc” questionnaires. Results 25 families with 26 patients were recruited. The average total cost of resources not eligible for reimbursement was €104,144 /year/patient. Direct costs were €67,177/year/patient, among which informal care accounted for €43,090/year/patient. Productivity loss was €48,057/year/patient. Families’ out of pocket expense per year was €5,302. Financial assistance received in the context of DMD and disability amounted to an average of €14,980 /year/patient, with monthly allowances up to €14,237/year/patient. Conclusion This study provides a first inventory of the major financial and time burden borne by DMD patients and their caregivers in France, and confirms the high level of non-reimbursed resource consumption by DMD patients as well as the enormous time commitment from their families. Trial registration NCT06861270 (Initial Release: 09/25/2024)