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It was deemed useful to conduct a systematic literature review on the pharmacoeconomic evaluations of all currently registered disease\u2010modifying therapies in order to inform policy and highlight research gaps. Pharmacoeconomic analyses written in English and published after 2016 were considered for inclusion. PubMed\/Medline, Global Health and Embase were systematically and separately searched between 16 October and 23 October 2023. Hand\u2010searching was also conducted on PubMed based on reference lists of published literature. After the exclusion criteria were applied, 14 studies were included. BMJ checklist was used for quality assessment and the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist was used to assess the quality of reporting of all included studies. Data extraction was performed manually. Regarding evidence synthesis, data were heterogeneous and are thus presented based on comparison. This study confirms the need for pharmacoeconomic analyses (cost\u2010effectiveness or cost\u2010utility) also in cases when the cost of treatment is very high and the incremental cost\u2010effectiveness ratio values exceed the usual, acceptable values for standard therapy. Specific willingness to pay thresholds for orphan medicines are of the utmost importance, to allow patients with SMA to have access to safe and effective treatments. With such economic evaluations, it is possible to compare the value of medications with the same indication, but it should be emphasized that in the interpretation of data and in making decisions about the use of medicines, the impact of new knowledge should be considered.<\/jats:p>","DOI":"10.1111\/bcp.16267","type":"journal-article","created":{"date-parts":[[2024,10,8]],"date-time":"2024-10-08T23:00:28Z","timestamp":1728428428000},"page":"95-116","source":"Crossref","is-referenced-by-count":20,"title":["A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy"],"prefix":"10.1111","volume":"91","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7848-6600","authenticated-orcid":false,"given":"Andrej","family":"Belan\u010di\u0107","sequence":"first","affiliation":[{"name":"Department of Clinical Pharmacology Clinical Hospital Centre Rijeka  Rijeka Croatia"},{"name":"Department of Basic and Clinical Pharmacology with Toxicology University of Rijeka, Faculty of Medicine  Rijeka Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0006-9039-0197","authenticated-orcid":false,"given":"Andrea Katrin","family":"Faour","sequence":"additional","affiliation":[{"name":"Vancouver Coastal Health  Vancouver BC Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elvira Meni Maria","family":"Gkrinia","sequence":"additional","affiliation":[{"name":"Athens Greece"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dinko","family":"Vitezi\u0107","sequence":"additional","affiliation":[{"name":"Department of Clinical Pharmacology Clinical Hospital Centre Rijeka  Rijeka Croatia"},{"name":"Department of Basic and Clinical Pharmacology with Toxicology University of Rijeka, Faculty of Medicine  Rijeka Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,10,8]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pcl.2015.03.010"},{"key":"e_1_2_9_3_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010017\u20100671\u20108"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.3390\/ijms21093297"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2017.52"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.05.011"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.005"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.11.004"},{"key":"e_1_2_9_9_1","doi-asserted-by":"publisher","DOI":"10.1080\/14656566.2019.1704732"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40271\u2010020\u201000415\u2010w"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41582\u2010020\u201000410\u20107"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.3390\/jcm12082839"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1186\/s13023\u2010021\u201001695\u20107"},{"key":"e_1_2_9_14_1","volume-title":"PRISMA transparent reporting of systematic reviews and meta\u2010analyses"},{"key":"e_1_2_9_15_1","volume-title":"Parliamentary Office of Science & Technology. 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