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The European Journal of Health Economics

Open Access 20-04-2024 | Spinal Muscular Atrophy | Original Paper

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

Authors: Thomas Sejersen, Sophie Graham, Anne-Berit Ekström, Anna-Karin Kroksmark, Marta Kwiatkowska, Michael L. Ganz, Nahila Justo, Karl Gertow, Alex Simpson

Published in: The European Journal of Health Economics

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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.
Appendix
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Literature
1.
2.
3.
Wirth, B., Karakaya, M., Kye, M.J., Mendoza-Ferreira, N.: Twenty-five years of spinal muscular atrophy research: from phenotype to genotype to therapy, and what comes next. Annu. Rev. Genomics Hum. Genet. 21, 231–261 (2020)CrossRefPubMed
4.
5.
Ogino, S., Wilson, R.B., Gold, B.: New insights on the evolution of the SMN1 and SMN2 region: simulation and meta-analysis for allele and haplotype frequency calculations. Eur. J. Hum. Genet. 12, 1015–1023 (2004)CrossRefPubMed
6.
Jones, C., Oskoui, M., Zielinski, D., Vinikoor, L., Farwell, W.: PP09.1 – 2352: Systematic review of incidence and prevalence of spinal muscular atrophy (SMA). J. Paediatr. Neurol. 19, S64–S65 (2015)CrossRef
7.
Burr, P., & Reddivari, A.K.R.: Spinal muscle atrophy. StatPearls, Treasure Island, FL (2022)
8.
Wang, C.H., Finkel, R.S., Bertini, E.S., et al.: Consensus statement for standard of care in spinal muscular atrophy. J. Child Neurol. 22, 1027–1049 (2007)CrossRefPubMed
9.
Mercuri, E., Finkel, R.S., Muntoni, F., et al.: Diagnosis and management of spinal muscular atrophy: part 1: recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromuscul. Disord. 28, 103–115 (2018)CrossRefPubMed
10.
11.
12.
13.
Hjartarson, H.T., Nathorst-Boos, K., Sejersen, T.: Disease modifying therapies for the management of children with spinal muscular atrophy (5q SMA): an update on the emerging evidence. Drug Des Devel Ther 16, 1865–1883 (2022)CrossRefPubMedPubMedCentral
14.
15.
Paracha, N., Hudson, P., Mitchell, S., Sutherland, C.S.: Systematic literature review to assess economic evaluations in spinal muscular atrophy (SMA). Pharmacoeconomics 40, 69–89 (2022)CrossRefPubMed
16.
Dangouloff, T., Botty, C., Beaudart, C., Servais, L., Hiligsmann, M.: Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments. Orphanet J. Rare Dis. 16, 47 (2021)CrossRefPubMedPubMedCentral
17.
Paracha, N., Hudson, P., Mitchell, S., Sutherland, C.S.: Systematic literature review to assess the cost and resource use associated with spinal muscular atrophy management. Pharmacoeconomics 40, 11–38 (2022)CrossRefPubMed
18.
Landfeldt, E., Pechmann, A., McMillan, H.J., Lochmuller, H., Sejersen, T.: Costs of illness of spinal muscular atrophy: a systematic review. Appl. Health Econ. Health Policy 19, 501–520 (2021)CrossRefPubMedPubMedCentral
19.
20.
Kostera-Pruszczyk, A., Napiorkowski, L., Szymanska, K., et al.: Spinal muscular atrophy: epidemiology and health burden in children—a Polish national healthcare database perspective before introduction of SMA-specific treatment. Neurol. Neurochir. Pol. 55, 479–484 (2021)CrossRefPubMed
21.
Chen, G., Sharif, B., Gerber, B., et al.: Epidemiology, healthcare resource utilization and healthcare costs for spinal muscular atrophy in Alberta Canada. J. Med. Econ. 24, 51–59 (2021)CrossRefPubMed
22.
Dangouloff, T., Hiligsmann, M., Deconinck, N., et al.: Financial cost and quality of life of patients with spinal muscular atrophy identified by symptoms or newborn screening. Dev. Med. Child Neurol. 65, 67–77 (2023)CrossRefPubMed
23.
24.
25.
Darba, J.: Management and current status of spinal muscular atrophy: a retrospective multicentre claims database analysis. Orphanet J. Rare Dis. 15, 8 (2020)CrossRefPubMedPubMedCentral
26.
Lally, C., Jones, C., Farwell, W., Reyna, S.P., Cook, S.F., Flanders, W.D.: Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States. Orphanet J. Rare Dis. 12, 175 (2017)CrossRefPubMedPubMedCentral
27.
28.
29.
30.
Campbell, R.T., Hudson, C.M.: Synthetic cohorts from panel surveys. An approach to studying rare events. Res. Aging 7, 81–93 (1985)CrossRefPubMed
31.
32.
Walter, M.C., Hiebeler, M.: Adult spinal muscular atrophy. Fortschr. Neurol. Psychiatr. 90, 421–430 (2022)PubMed
33.
Chambers, G.M., Settumba, S.N., Carey, K.A., et al.: Prenusinersen economic and health-related quality of life burden of spinal muscular atrophy. Neurology 95, e1–e10 (2020)CrossRefPubMed
34.
Metadata
Title
Healthcare resource utilisation and direct medical cost for individuals with 5q spinal muscular atrophy in Sweden
Authors
Thomas Sejersen
Sophie Graham
Anne-Berit Ekström
Anna-Karin Kroksmark
Marta Kwiatkowska
Michael L. Ganz
Nahila Justo
Karl Gertow
Alex Simpson
Publication date
20-04-2024
Publisher
Springer Berlin Heidelberg
Published in
The European Journal of Health Economics
Print ISSN: 1618-7598
Electronic ISSN: 1618-7601
DOI
https://doi.org/10.1007/s10198-024-01678-y