REAL-WORLD HEALTHCARE RESOURCE UTILIZATION AND RELATED COSTS OF DUCHENNE MUSCULAR DYSTROPHY IN FRANCE: THE EPIDUCH STUDY
Author(s)
JULIA BONASTRE, PhD1, Laure Le Goff, PhD2, Catherine Kerting, PhD3, David Pau, MSc3, Alexandre Civet, MSc3, Fatiha Messaoudi, MSc4, Solenne Tauty, MSc5, Mélanie Goguillot, MSc5, Francoise BUGNARD, MSc5, Cecile Halbert, PhD6.
1Gustave roussy, VILLEJUIF, France, 2Hospices Civils de Lyon, Villeurbanne, France, Villeurbanne, France, 3Roche, Boulogne-Billancourt, France, 4IVIDATA, Paris, France, 5Cytel, Oullins, France, 6AP-HM, Marseille, France.
1Gustave roussy, VILLEJUIF, France, 2Hospices Civils de Lyon, Villeurbanne, France, Villeurbanne, France, 3Roche, Boulogne-Billancourt, France, 4IVIDATA, Paris, France, 5Cytel, Oullins, France, 6AP-HM, Marseille, France.
OBJECTIVES: To describe annual healthcare resource utilization (HCRU) and related costs in France for the management of Duchenne Muscular Dystrophy (DMD), a chronic life-threatening condition.
METHODS: EpiDuch was a national retrospective cohort study. A deterministic indirect linkage method was used to match a cohort of DMD male patients selected in the French National Registry for Rare Diseases with claims data from the French National Health Data System (SNDS) over 2014-2023. Index date was defined as the DMD diagnosis date. Healthcare costs were assessed from a collective perspective on an annual basis distinguishing prevalent (diagnosed before 2017) and incident patients (diagnosed in 2017-2023).
RESULTS: The study population consisted in 669 DMD male patients on average 11.3 (±standard deviation SD:8.6) years old (range: from birth to 48 years). For prevalent patients (N=466), mean annual HCRU per patient included 46.1 (±SD:67.4) days with paramedical care and 6.5 (±SD: 8.9) medical procedures. For incident patients (N=203), HCRU increased significantly as the disease progressed: mean annual number of days with paramedical care per patient increased from 23.1 (±SD: 32.4) during the 1st year to a peak at 28.8 days (± SD: 38.6) during the 3rd year. Similarly, the mean annual number of procedures rose from 0.2 (± SD: 1.4) in the 1st year to 1.2 (± SD: 6.3) in the 3rd year. Per patient annual healthcare cost was €30,188 (median: €7,872, interquartile range [IQR: € 3,357-€22,293]). It was higher in prevalent patients with a mean annual healthcare cost per patient of €36,768 (median: €11,719, [IQR: €5,307-€29,258]) and €15,201 (median: €4,040, [IQR: €2,295-€7,403]) in incident patients.
CONCLUSIONS: The EpiDuch study shows that DMD is associated with a substantial healthcare burden increasing as the disease progresses, characterized by a highly intensive paramedical care follow-up and repeated medical procedures. The overall economic burden of DMD is however largely underestimated in this study.
METHODS: EpiDuch was a national retrospective cohort study. A deterministic indirect linkage method was used to match a cohort of DMD male patients selected in the French National Registry for Rare Diseases with claims data from the French National Health Data System (SNDS) over 2014-2023. Index date was defined as the DMD diagnosis date. Healthcare costs were assessed from a collective perspective on an annual basis distinguishing prevalent (diagnosed before 2017) and incident patients (diagnosed in 2017-2023).
RESULTS: The study population consisted in 669 DMD male patients on average 11.3 (±standard deviation SD:8.6) years old (range: from birth to 48 years). For prevalent patients (N=466), mean annual HCRU per patient included 46.1 (±SD:67.4) days with paramedical care and 6.5 (±SD: 8.9) medical procedures. For incident patients (N=203), HCRU increased significantly as the disease progressed: mean annual number of days with paramedical care per patient increased from 23.1 (±SD: 32.4) during the 1st year to a peak at 28.8 days (± SD: 38.6) during the 3rd year. Similarly, the mean annual number of procedures rose from 0.2 (± SD: 1.4) in the 1st year to 1.2 (± SD: 6.3) in the 3rd year. Per patient annual healthcare cost was €30,188 (median: €7,872, interquartile range [IQR: € 3,357-€22,293]). It was higher in prevalent patients with a mean annual healthcare cost per patient of €36,768 (median: €11,719, [IQR: €5,307-€29,258]) and €15,201 (median: €4,040, [IQR: €2,295-€7,403]) in incident patients.
CONCLUSIONS: The EpiDuch study shows that DMD is associated with a substantial healthcare burden increasing as the disease progresses, characterized by a highly intensive paramedical care follow-up and repeated medical procedures. The overall economic burden of DMD is however largely underestimated in this study.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EPH127
Topic
Economic Evaluation, Epidemiology & Public Health, Real World Data & Information Systems
Disease
Musculoskeletal Disorders (Arthritis, Bone Disorders, Osteoporosis, Other Musculoskeletal), Pediatrics, Rare & Orphan Diseases