PharmacoEconomics - open · 2026
Incremental Disease Burden (Healthcare Costs and Resources) of Duchenne Muscular Dystrophy in the US: A Matched Cohort Analysis.
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Scavina Mena, Posner Nate, Dorling Patricia, Talaga Anna, Stachel Anna, Alvir Jose
Аннотация
BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare genetic condition that causes progressive muscle weakness and reduced life expectancy. Detailed economic studies on the impact of DMD disease burden, costs, and healthcare resource utilization (HCRU) are limited.
OBJECTIVE: To compare healthcare costs, HCRU, and ambulatory status between a cohort of DMD patients and a matched cohort of patients without muscular dystrophies (non-MD).
- METHODS: A retrospective study was conducted using Merative MarketScan® Commercial and Medicaid databases. Patients with DMD were identified using a validated algorithm. The index date for the DMD cohort was the first date with an ICD-10 code: G71.01. The study included males without cancer, aged ≤ 40 years, and consistently enrolled for 3 months pre-index and 12 months post-index. Patients with DMD were matched 1:1 with non-MD patientsby age, region, race, insurance, and index date year. Patients with DMD were compared for healthcare costs and HCRU on the basis of ambulatory function versus non-ambulatory status (wheelchair purchase).
RESULTS: The DMD and non-MD cohorts included 782 matched pairs. Patients with DMD had significantly higher mean unadjusted total healthcare costs (US$103,826.05 ± 309,335.71) per patient per year compared with patients with non-MD (US$3225.30 ± 13,865.14; p < 0.0001) per patient per year. The total healthcare cost was primarily driven by pharmacy and outpatient costs. The DMD cohort showed significantly higher HCRU than the non-MD cohort across physician offices, outpatient, emergency room, speech therapy, physical therapy, endocrinology, cardiology, orthopedic, pulmonology, and physical medicine visits. DMD patients with non-ambulatory status were older, medically progressed, and incurred higher costs than patients with ambulatory function.
CONCLUSIONS: DMD imposes a substantial cost and healthcare resource burden on patients and the healthcare system. The results suggest a high unmet need that requires the development of novel therapies to treat DMD.
OBJECTIVE: To compare healthcare costs, HCRU, and ambulatory status between a cohort of DMD patients and a matched cohort of patients without muscular dystrophies (non-MD).
- METHODS: A retrospective study was conducted using Merative MarketScan® Commercial and Medicaid databases. Patients with DMD were identified using a validated algorithm. The index date for the DMD cohort was the first date with an ICD-10 code: G71.01. The study included males without cancer, aged ≤ 40 years, and consistently enrolled for 3 months pre-index and 12 months post-index. Patients with DMD were matched 1:1 with non-MD patientsby age, region, race, insurance, and index date year. Patients with DMD were compared for healthcare costs and HCRU on the basis of ambulatory function versus non-ambulatory status (wheelchair purchase).
RESULTS: The DMD and non-MD cohorts included 782 matched pairs. Patients with DMD had significantly higher mean unadjusted total healthcare costs (US$103,826.05 ± 309,335.71) per patient per year compared with patients with non-MD (US$3225.30 ± 13,865.14; p < 0.0001) per patient per year. The total healthcare cost was primarily driven by pharmacy and outpatient costs. The DMD cohort showed significantly higher HCRU than the non-MD cohort across physician offices, outpatient, emergency room, speech therapy, physical therapy, endocrinology, cardiology, orthopedic, pulmonology, and physical medicine visits. DMD patients with non-ambulatory status were older, medically progressed, and incurred higher costs than patients with ambulatory function.
CONCLUSIONS: DMD imposes a substantial cost and healthcare resource burden on patients and the healthcare system. The results suggest a high unmet need that requires the development of novel therapies to treat DMD.