{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T18:41:59Z","timestamp":1790707319623,"version":"4.1.0"},"reference-count":63,"publisher":"Wiley","license":[{"start":{"date-parts":[[2025,12,14]],"date-time":"2025-12-14T00:00:00Z","timestamp":1765670400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Br J Clin Pharmacol"],"abstract":"<jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients. There is reason to believe that there are economic benefits of switching patients from nusinersen to risdiplam, especially because real\u2010world analysis has shown non\u2010inferiority in clinical outcomes along with a favourable safety profile of switching.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>To conduct the cost\u2010utility analysis of switching from nusinersen to risdiplam.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>A discrete event simulation model was created for three different groups of patients with SMA (types 1\u20133), comparing the cost\u2010utility of nusinersen to the risdiplam switch and continuing nusinersen therapy. Analysis was conducted from the perspective of the Croatian Health Insurance Fund for the time horizon of 80\u2009years. A deterministic, one\u2010way, one\u2010factor sensitivity analysis was carried out using the Monte Carlo simulation.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>The incremental cost\u2010effectiveness ratio of nusinersen to risdiplam switch versus staying on nusinersen was EUR\u2010136930\u2009\u00b1\u200982\u2009336 for type 1 SMA (i.e. the switch dominated), for type 2 SMA it was EUR 2681860\u2009\u00b1\u2009321\u2009859, and for type 3 SMA it was EUR 2576667\u2009\u00b1\u2009676\u2009078. For all three types of SMA, switching to risdiplam showed positive net monetary benefit (type 1: EUR 35449\u2009\u00b1\u200917\u2009113; type 2: EUR 799264\u2009\u00b1\u200930\u2009765; type 3: EUR 767498\u2009\u00b1\u20097283). Market forces may substantially impact pricing dynamics; therefore, these results should be interpreted with consideration of inherent uncertainties.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>The cost\u2010effectiveness of switching from nusinersen to risdiplam is evident across all SMA types. Further research on the long\u2010term impact of switching treatments in SMA is essential to address uncertainties and optimize cost\u2010effective therapeutic strategies.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/bcp.70379","type":"journal-article","created":{"date-parts":[[2025,12,15]],"date-time":"2025-12-15T02:58:09Z","timestamp":1765767489000},"source":"Crossref","is-referenced-by-count":2,"title":["Cost\u2010utility analysis of nusinersen\u2013risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model"],"prefix":"10.1002","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7848-6600","authenticated-orcid":false,"given":"Andrej","family":"Belan\u010di\u0107","sequence":"first","affiliation":[{"name":"Department of Basic and Clinical Pharmacology and Toxicology University of Rijeka, Faculty of Medicine  Rijeka 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