Direct Medical Costs of Duchenne Muscular Dystrophy Patients: A Retrospective Observational Study Using Swedish Population-Based Register Data

Speaker(s)

Ekström AB1, Kroksmark AK2, Törnblom A3, Toghanian S3, Ortsäter G4, Sejersen T5
1University of Gothenburg, Gothenburg, Gothenburg, Sweden, 2University of Gothenburg, Gothenburg, Sweden, 3Pfizer AB, Stockholm, Sweden, 4Quantify Research, Stockholm, Sweden, 5Karolinska University Hospital, Stockholm, Sweden

Presentation Documents

OBJECTIVES: The objective of this study was to estimate healthcare resource use and direct medical costs of Duchenne muscular dystrophy (DMD) in Sweden.

METHODS: Patients diagnosed with DMD were identified from the Swedish National Registry for Neuromuscular Disorders. Patients were followed from first assessment of health status (index date) until death or end of the study period (2022-12-31), whatever came first. All healthcare visits and prescribed drugs were collected during follow-up. Patients who were included from date of diagnosis were analyzed in a sub-group analysis. The number of healthcare visits and direct medical costs were calculated as per patient-year together with the associated standard deviation (SD). Cost of in- and out-patient visits were estimated from the national patient registry and cost of filled prescriptions were collected from the prescribed drug registry (includes all pharmacy dispensed drugs in Sweden). All costs were inflated to 2022 price-levels and converted to € (from SEK) using an exchange rate of 10.6.

RESULTS: A total of 215 patients were included in this study and the average age at index date was 10.9 years. Patients were followed for 9.1 years on average. On average, DMD patients had 5.8 (2.9 SD) outpatient visits and 0.4 (0.5 SD) hospital admissions per year of follow-up. The average annualized economic burden of DMD patients was €15,411 (38,723 SD). The main driver of the economic burden was the cost of prescribed drugs (€10,465 [38.723 SD]) which represented 68% of total direct medical costs. The average annualized economic burden in patients (n=47) who were included from date of diagnosis was €17,176 (36,661 SD).

CONCLUSIONS: The direct medical costs associated with DMD is high and imposes a significant burden on the healthcare sector in Sweden. The level of healthcare resource use and associated costs is not associated with time since diagnosis.

Code

EE818

Topic

Economic Evaluation

Disease

Pediatrics, Rare & Orphan Diseases