prikaz prve stranice dokumenta A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy
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A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy
British Journal of Clinical Pharmacology, 91 (2025), 1; 95-116. https://doi.org/10.1111/bcp.16267
Belančić, Andrej; Faour, Andrea Katrin; Gkrinia, Elvira Meni Maria; Vitezić, Dinko

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Belančić, A., Faour, A. K., Gkrinia, E. M. M. i Vitezić, D. (2025). A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy. British Journal of Clinical Pharmacology, 91. (1), 95-116. doi: 10.1111/bcp.16267

Belančić, Andrej, et al. "A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy." British Journal of Clinical Pharmacology, vol. 91, br. 1, 2025, str. 95-116. https://doi.org/10.1111/bcp.16267

Belančić, Andrej, Andrea Katrin Faour, Elvira Meni Maria Gkrinia i Dinko Vitezić. "A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy." British Journal of Clinical Pharmacology 91, br. 1 (2025): 95-116. https://doi.org/10.1111/bcp.16267

Belančić, A., et al. (2025) 'A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy', British Journal of Clinical Pharmacology, 91(1), str. 95-116. doi: 10.1111/bcp.16267

Belančić A, Faour AK, Gkrinia EMM, Vitezić D. A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy. British Journal of Clinical Pharmacology [Internet]. 01.2025. [pristupljeno 01.04.2025.];91(1):95-116. doi: 10.1111/bcp.16267

A. Belančić, A. K. Faour, E. M. M. Gkrinia i D. Vitezić, "A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy", British Journal of Clinical Pharmacology, vol. 91, br. 1, str. 95-116, Siječanj 2025. [Online]. Dostupno na: https://urn.nsk.hr/urn:nbn:hr:184:796584. [Citirano: 01.04.2025.]

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Podaci o radu
Naslov (engleski)A systematic review of economic evaluations of orphan medicines for the management of spinal muscular atrophy
AutorAndrej Belančić
AutorAndrea Katrin Faour
AutorElvira Meni Maria Gkrinia
AutorDinko Vitezić
Autorova ustanovaSveučilište u Rijeci
Medicinski fakultet
(Katedra za farmakologiju)
Znanstveno / umjetničko
područje, polje i grana		BIOMEDICINA I ZDRAVSTVO
Kliničke medicinske znanosti
Klinička farmakologija s toksikologijom
Sažetak (engleski)
Spinal muscular atrophy (SMA) is a rare inherited autosomal recessive progressive disease of a varying phenotype, with varying clinical symptoms, and as a result the patients suffering from it require multiple types of care. It was deemed useful to conduct a systematic literature review on the pharmacoeconomic evaluations of all currently registered disease‐modifying therapies in order to inform policy and highlight research gaps. Pharmacoeconomic analyses written in English and published after... Više 2016 were considered for inclusion. PubMed/Medline, Global Health and Embase were systematically and separately searched between 16 October and 23 October 2023. Hand‐searching 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‐effectiveness or cost‐utility) also in cases when the cost of treatment is very high and the incremental cost‐effectiveness 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. Sakrij dio sažetka
Ključne riječi (engleski)
Jezikengleski
Vrsta publikacijeZnanstveni rad - Pregledni rad
Status objaveObjavljen
Vrsta recenzijeRecenziran - međunarodna recenzija
Verzija publikacijeObjavljena verzija rada (izdavačev PDF)
Naslov časopisaBritish Journal of Clinical Pharmacology
Brojčani podacivol. 91, br. 1, str. 95-116
p-ISSN0306-5251
e-ISSN1365-2125
DOIhttps://doi.org/10.1111/bcp.16267
URN:NBNurn:nbn:hr:184:796584
Datum objave publikacije2025-01
URL dokumentahttps://bpspubs.onlinelibrary.wiley.com/doi/10.1111/bcp.16267
Vrsta resursaTekst
Prava pristupaOgraničen pristup
Uvjeti korištenjaIkona licence Zaštićeno autorskim pravom.
Datum i vrijeme pohrane2025-01-21 12:08:09
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