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Costs of Illness of Spinal Muscular Atrophy: A Systematic Review

OBJECTIVES: The objective of our study was to conduct a systematic literature review of estimates of costs of illness of spinal muscular atrophy (SMA). METHODS: We searched MEDLINE (through PubMed), CINAHL, Embase, Web of Science, National Health Service Economic Evaluation Database, and the Nationa...

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Autores principales: Landfeldt, Erik, Pechmann, Astrid, McMillan, Hugh J., Lochmüller, Hanns, Sejersen, Thomas
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8270802/
https://www.ncbi.nlm.nih.gov/pubmed/33576939
http://dx.doi.org/10.1007/s40258-020-00624-2
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author Landfeldt, Erik
Pechmann, Astrid
McMillan, Hugh J.
Lochmüller, Hanns
Sejersen, Thomas
author_facet Landfeldt, Erik
Pechmann, Astrid
McMillan, Hugh J.
Lochmüller, Hanns
Sejersen, Thomas
author_sort Landfeldt, Erik
collection PubMed
description OBJECTIVES: The objective of our study was to conduct a systematic literature review of estimates of costs of illness of spinal muscular atrophy (SMA). METHODS: We searched MEDLINE (through PubMed), CINAHL, Embase, Web of Science, National Health Service Economic Evaluation Database, and the National Health Service Health Technology Assessment Database for studies published from inception up until 31 August, 2020, reporting direct medical, direct non-medical, and/or indirect costs of any phenotype of SMA. Two reviewers independently screened records for eligibility, extracted the data, and assessed studies for risk of bias using the Newcastle–Ottawa Scale. Costs were adjusted and converted to 2018 US dollars. RESULTS: The search identified 14 studies from eight countries (Australia, France, Germany, Italy, Spain, Sweden, the UK, and the USA). The mean per-patient annual direct medical cost of illness was estimated at between $3320 (SMA type III, Italy) and $324,410 (SMA type I, USA), mean per-patient annual direct non-medical cost between $25,880 (SMA types I–III, Spain) and $136,800 (SMA type I, Sweden), and mean per-patient annual indirect cost between $9440 (SMA type I, Germany) and $74,910 (SMA type II, Australia). Most studies exhibited a risk of bias. CONCLUSIONS: The current body of evidence of costs of illness of SMA is relatively scarce and characterized by considerable variability across geographical settings and disease phenotypes. Our review provides data pertaining to the economic impact of SMA, which is of particular relevance in light of emerging treatments and ongoing research in this field, and underscores the substantial unmet medical need in this patient population. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s40258-020-00624-2) contains supplementary material, which is available to authorized users.
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spelling pubmed-82708022021-07-20 Costs of Illness of Spinal Muscular Atrophy: A Systematic Review Landfeldt, Erik Pechmann, Astrid McMillan, Hugh J. Lochmüller, Hanns Sejersen, Thomas Appl Health Econ Health Policy Systematic Review OBJECTIVES: The objective of our study was to conduct a systematic literature review of estimates of costs of illness of spinal muscular atrophy (SMA). METHODS: We searched MEDLINE (through PubMed), CINAHL, Embase, Web of Science, National Health Service Economic Evaluation Database, and the National Health Service Health Technology Assessment Database for studies published from inception up until 31 August, 2020, reporting direct medical, direct non-medical, and/or indirect costs of any phenotype of SMA. Two reviewers independently screened records for eligibility, extracted the data, and assessed studies for risk of bias using the Newcastle–Ottawa Scale. Costs were adjusted and converted to 2018 US dollars. RESULTS: The search identified 14 studies from eight countries (Australia, France, Germany, Italy, Spain, Sweden, the UK, and the USA). The mean per-patient annual direct medical cost of illness was estimated at between $3320 (SMA type III, Italy) and $324,410 (SMA type I, USA), mean per-patient annual direct non-medical cost between $25,880 (SMA types I–III, Spain) and $136,800 (SMA type I, Sweden), and mean per-patient annual indirect cost between $9440 (SMA type I, Germany) and $74,910 (SMA type II, Australia). Most studies exhibited a risk of bias. CONCLUSIONS: The current body of evidence of costs of illness of SMA is relatively scarce and characterized by considerable variability across geographical settings and disease phenotypes. Our review provides data pertaining to the economic impact of SMA, which is of particular relevance in light of emerging treatments and ongoing research in this field, and underscores the substantial unmet medical need in this patient population. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s40258-020-00624-2) contains supplementary material, which is available to authorized users. Springer International Publishing 2021-02-12 2021 /pmc/articles/PMC8270802/ /pubmed/33576939 http://dx.doi.org/10.1007/s40258-020-00624-2 Text en © The Author(s) 2021 https://creativecommons.org/licenses/by-nc/4.0/Open AccessThis article is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License, which permits any non-commercial use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) .
spellingShingle Systematic Review
Landfeldt, Erik
Pechmann, Astrid
McMillan, Hugh J.
Lochmüller, Hanns
Sejersen, Thomas
Costs of Illness of Spinal Muscular Atrophy: A Systematic Review
title Costs of Illness of Spinal Muscular Atrophy: A Systematic Review
title_full Costs of Illness of Spinal Muscular Atrophy: A Systematic Review
title_fullStr Costs of Illness of Spinal Muscular Atrophy: A Systematic Review
title_full_unstemmed Costs of Illness of Spinal Muscular Atrophy: A Systematic Review
title_short Costs of Illness of Spinal Muscular Atrophy: A Systematic Review
title_sort costs of illness of spinal muscular atrophy: a systematic review
topic Systematic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8270802/
https://www.ncbi.nlm.nih.gov/pubmed/33576939
http://dx.doi.org/10.1007/s40258-020-00624-2
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