Healthcare resource utilisation and direct medical cost for individuals with 5q spinal muscular atrophy in Sweden

Thomas Sejersen (), Sophie Graham (), Anne-Berit Ekström (), Anna-Karin Kroksmark (), Marta Kwiatkowska (), Michael L. Ganz (), Nahila Justo (), Karl Gertow () and Alex Simpson ()
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Thomas Sejersen: Karolinska University Hospital
Sophie Graham: Evidera Ltd
Anne-Berit Ekström: Sahlgrenska University Hospital
Anna-Karin Kroksmark: Sahlgrenska University Hospital
Marta Kwiatkowska: Evidera Ltd
Michael L. Ganz: Evidera Inc
Nahila Justo: Evidera-PPD
Karl Gertow: Roche AB
Alex Simpson: F. Hoffmann-La Roche Ltd

The European Journal of Health Economics, 2025, vol. 26, issue 1, No 4, 35-48

Abstract: Abstract Background Spinal muscular atrophy (SMA) is a rare, progressive, neuromuscular disorder. Recent advances in treatment require an updated assessment of burden to inform reimbursement decisions. Objectives To quantify healthcare resource utilisation (HCRU) and cost of care for patients with SMA. Methods Cohort study of patients with SMA identified in the Swedish National Patient Registry (2007–2018), matched to a reference cohort grouped into four SMA types (1, 2, 3, unspecified adult onset [UAO]). HCRU included inpatient admissions, outpatient visits, procedures, and dispensed medications. Direct medical costs were estimated by multiplying HCRU by respective unit costs. Average annual HCRU and medical costs were modelled for SMA versus reference cohorts to estimate differences attributable to the disease (i.e., average treatment effect estimand). The trajectory of direct costs over time were assessed using synthetic cohorts. Results We identified 290 SMA patients. Annualised HCRU was higher in SMA patients compared with reference cohorts. Highest risk ratios were observed for inpatient overnight stays for type 1 (risk ratio [RR]: 29.2; 95% confidence interval [CI]: 16.0, 53.5) and type 2 (RR: 23.3; 95% CI: 16.4,33.1). Mean annual direct medical costs per patient for each year since first diagnosis were greatest for type 1 (€114,185 and SMA-attributable: €113,380), type 2 (€61,876 and SMA-attributable: €61,237), type 3 (€45,518 and SMA-attributable: €44,556), and UAO (€4046 and SMA-attributable: €2098). Costs were greatest in the 2–3 years after the first diagnosis for all types. Discussion and conclusion The economic burden attributable to SMA is significant. Further research is needed to understand the burden in other European countries and the impact of new treatments.

Keywords: Cohort study; Direct medical costs; Healthcare resource use; Spinal muscular atrophy; Sweden; Registries (search for similar items in EconPapers)
JEL-codes: I11 (search for similar items in EconPapers)
Date: 2025
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DOI: 10.1007/s10198-024-01678-y

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