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Recent advances in treatment require an updated assessment of burden to inform reimbursement decisions.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Objectives<\/jats:title>\n            <jats:p>To quantify healthcare resource utilisation (HCRU) and cost of care for patients with SMA.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Methods<\/jats:title>\n            <jats:p>Cohort study of patients with SMA identified in the Swedish National Patient Registry (2007\u20132018), 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.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Results<\/jats:title>\n            <jats:p>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 (\u20ac114,185 and SMA-attributable: \u20ac113,380), type 2 (\u20ac61,876 and SMA-attributable: \u20ac61,237), type 3 (\u20ac45,518 and SMA-attributable: \u20ac44,556), and UAO (\u20ac4046 and SMA-attributable: \u20ac2098). Costs were greatest in the 2\u20133\u00a0years after the first diagnosis for all types.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Discussion and conclusion<\/jats:title>\n            <jats:p>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.<\/jats:p>\n          <\/jats:sec>","DOI":"10.1007\/s10198-024-01678-y","type":"journal-article","created":{"date-parts":[[2024,4,21]],"date-time":"2024-04-21T02:56:11Z","timestamp":1713668171000},"page":"35-48","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Healthcare resource utilisation and direct medical cost for individuals with 5q spinal muscular atrophy in Sweden"],"prefix":"10.1007","volume":"26","author":[{"given":"Thomas","family":"Sejersen","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sophie","family":"Graham","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne-Berit","family":"Ekstr\u00f6m","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna-Karin","family":"Kroksmark","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marta","family":"Kwiatkowska","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael L.","family":"Ganz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nahila","family":"Justo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karl","family":"Gertow","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alex","family":"Simpson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,4,20]]},"reference":[{"key":"1678_CR1","doi-asserted-by":"publisher","first-page":"743","DOI":"10.1016\/j.pcl.2015.03.010","volume":"62","author":"BT Darras","year":"2015","unstructured":"Darras, B.T.: Spinal muscular atrophies. 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Karl Gertow is employed by Roche AB, Sweden. Nahila Justo, Michael Ganz, Sophie Graham and Marta Kwiatkowska are employed by Evidera, which provides consulting and other research services to pharmaceutical, medical device, and related organizations. In their salaried positions, they work with a variety of companies and organizations, and are precluded from receiving payment or honoraria directly from these organizations for services rendered. Thomas Sejersen is a recipient of honoraria received for lectures or consultancy from Biogen, Novartis, PTC Therapeutics, Sarepta Therapeutics, Roche, Hansa Biopharma and Sanofi Genzyme. Anne-Berit Ekstr\u00f6m is a recipient of honoraria for consultancy from Novartis. Anna-Karin Kroksmark has acted as a clinical evaluator and consultant to Biogen.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"The study was approved by the Swedish Ethical Review Authority DNR:2020\u201304656.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}}]}}