TRAJECTORIES AND COMPOSITION OF COSTS ACROSS DISEASE PROGRESSION STAGES IN DUCHENNE MUSCULAR DYSTROPHY: A SYSTEMATIC REVIEW
Author(s)
Erik Landfeldt, MSc, PhD1, Maria Åberg, MA2, Luca Bello, MD, PhD3, Alfred Peter Born, MD, PhD4, Silene Giusti, Jr., MSc5, Anne-Berit Ekström, MD, PhD6.
1IQVIA, Stockholm, Sweden, 2Equilib Services, Umeå, Sweden, 3CESP - University of Milano-Bicocca, Milan, Italy, 4Department of Pediatric and Adolescent Medicine, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark, 5Italfarmaco S.p.A., Milan, Italy, 6University of Gothenburg, Gothenburg, Sweden.
1IQVIA, Stockholm, Sweden, 2Equilib Services, Umeå, Sweden, 3CESP - University of Milano-Bicocca, Milan, Italy, 4Department of Pediatric and Adolescent Medicine, Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark, 5Italfarmaco S.p.A., Milan, Italy, 6University of Gothenburg, Gothenburg, Sweden.
OBJECTIVES: Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disease associated with a substantial economic burden. This study aimed to synthesize evidence on costs of DMD, focusing on stage-specific cost levels and shifts in cost composition across the disease trajectory.
METHODS: A systematic literature review (PROSPERO: CRD420251153340) was conducted of studies reporting direct medical, direct non-medical (including informal care), and indirect (productivity) costs of DMD. Searches were implemented in PubMed, MEDLINE, Embase, the Health Technology Assessment Database, and the National Health Service Economic Evaluation Database. Costs were inflated to 2025 values and converted to international dollars using country-specific consumer price indices and purchasing power parities. Stage-stratified evidence was synthesized to evaluate cost trajectories across early/late ambulatory/non-ambulatory disease stages.
RESULTS: Among 19 identified studies, stratified per-patient annual cost data were reported for nine cohorts (comprising 2,378 patients from Egypt, Germany, Italy, the UK, and the USA). In line with the natural history of DMD, direct medical costs increased across disease stages (from $16,080 in the early ambulatory stage to $28,810 in the late non-ambulatory stage), reflecting rising healthcare utilization. However, as a proportion of total costs, direct medical costs decreased from 33% to 26%. Direct non-medical costs (including community/transport services, informal care, home and vehicle adaptations, and non-medical aids/devices) also increased substantially along the disease trajectory, from $20,420 to $57,980, but rose as a share of total costs (from 48% to 52%), often representing the largest component in advanced stages. Indirect (productivity) costs increased with disease progression (from $15,550 to $42,020), although their relative contribution varied across stages/studies.
CONCLUSIONS: The economic burden of DMD exhibits stage-dependent shifts in cost composition, with non-medical and productivity-related costs increasingly dominating in advanced stages. These findings underscore the importance of capturing total disease costs and adopting a societal perspective in economic evaluations of emerging therapies for DMD.
METHODS: A systematic literature review (PROSPERO: CRD420251153340) was conducted of studies reporting direct medical, direct non-medical (including informal care), and indirect (productivity) costs of DMD. Searches were implemented in PubMed, MEDLINE, Embase, the Health Technology Assessment Database, and the National Health Service Economic Evaluation Database. Costs were inflated to 2025 values and converted to international dollars using country-specific consumer price indices and purchasing power parities. Stage-stratified evidence was synthesized to evaluate cost trajectories across early/late ambulatory/non-ambulatory disease stages.
RESULTS: Among 19 identified studies, stratified per-patient annual cost data were reported for nine cohorts (comprising 2,378 patients from Egypt, Germany, Italy, the UK, and the USA). In line with the natural history of DMD, direct medical costs increased across disease stages (from $16,080 in the early ambulatory stage to $28,810 in the late non-ambulatory stage), reflecting rising healthcare utilization. However, as a proportion of total costs, direct medical costs decreased from 33% to 26%. Direct non-medical costs (including community/transport services, informal care, home and vehicle adaptations, and non-medical aids/devices) also increased substantially along the disease trajectory, from $20,420 to $57,980, but rose as a share of total costs (from 48% to 52%), often representing the largest component in advanced stages. Indirect (productivity) costs increased with disease progression (from $15,550 to $42,020), although their relative contribution varied across stages/studies.
CONCLUSIONS: The economic burden of DMD exhibits stage-dependent shifts in cost composition, with non-medical and productivity-related costs increasingly dominating in advanced stages. These findings underscore the importance of capturing total disease costs and adopting a societal perspective in economic evaluations of emerging therapies for DMD.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
EE562
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies, Work & Home Productivity - Indirect Costs
Disease
Neurological Disorders, Rare & Orphan Diseases