The economic impact of retinal diseases for which gene therapy is emerging: a systematic literature review

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dc.contributor.authorWillmington, Claire
dc.contributor.authorKirby, Ann
dc.contributor.authorMurphy, Aileen
dc.contributor.funderEuropean Union
dc.date.accessioned2026-06-12T10:50:02Z
dc.date.available2026-06-12T10:50:02Z
dc.date.issued2025-12-07
dc.description.abstractBACKGROUND: Retinal disease is one of the leading causes of blindness and vision impairment worldwide, including Europe. With the advent of gene therapy, the treatment landscape for retinal disease is changing and clinical trials are underway investigating the therapeutic potential of gene therapy in both acquired and inherited retinal diseases. Given the high price of innovative medicines, it is essential to consider the current costs associated with retinal diseases to inform economic evaluations early in the life cycle of these forthcoming treatments. These could inform future reimbursement decisions, health budgets and service delivery plans. AIM: This systematic literature review sought to examine the economic burden of retinal diseases, for which gene therapy is emerging for patients in Europe. METHODS: Following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) 2020 guideline, a systematic search was performed using the Medline, CINAHL, EconLit and Embase databases. The searches were restricted to English language articles published after 1st January 2000. Following article selection, data were extracted in a tabular form and a narrative synthesis was performed. RESULTS: A total of 28 research studies were identified and included in the review that varied in terms of disease of interest, size, country setting, methodology, as well as how costs were reported and valued. While many retinal diseases were considered, almost half of the articles related to the costs of neovascular age-related macular degeneration (nAMD). Significant cost variations were observed across the studies as costs ranged from 45 USD to almost 30,000 USD per patient per annum, across all conditions combined. CONCLUSION: This systematic literature review evidences the heterogeneity among studies analysing the economics of retinal diseases underlying vision impairment. The paucity in the literature, signals the need for further research investigating the costs associated with retinal diseases, for which innovative therapies are expected to enter the market and will be subject to evaluation by decision-makers, whose decisions will have a significant impact on the delivery of these technologies to patients.en
dc.format.extent21
dc.format.mimetypeapplication/pdfen
dc.identifier.authororcidWillmington, Claire
dc.identifier.authororcidKirby, Ann§0000-0002-9043-5483
dc.identifier.authororcidMurphy, Aileen
dc.identifier.citationWillmington, C, Kirby, A & Murphy, A 2025, 'The economic impact of retinal diseases for which gene therapy is emerging : a systematic literature review', Health Economics Review, vol. 16, no. 1. https://doi.org/10.1186/s13561-025-00707-7
dc.identifier.doi10.1186/s13561-025-00707-7
dc.identifier.issn2191-1991
dc.identifier.issued1
dc.identifier.otherORCID: /0000-0002-9043-5483/work/217517961
dc.identifier.otherPubMedCentral: PMC12922378
dc.identifier.urihttps://hdl.handle.net/10468/18958
dc.identifier.volume16
dc.language.isoen
dc.publisherBioMed Central Ltd
dc.rights© 2025, the Author(s) . Open Access This article is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License, which permits any non-commercial use, sharing, 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 you modified the licensed material. You do not have permission under this licence to share adapted material derived from this article or parts of it. 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-nd/4.0
dc.rights.accessrightsopen access
dc.rights.licensenameAttribution-NonCommercial-NoDerivatives 4.0 International
dc.rights.urihttps://creativecommons.org/licenses/by-nc-nd/4.0/
dc.statusPeer reviewed
dc.subjectRetinal disease
dc.subjectAcquired retinal diseases
dc.subjectInherited retinal diseases
dc.subjectEconomic impact
dc.subjectCost-of-illness
dc.subjectGene therapy
dc.subject[Business]
dc.subject[CUBS]
dc.titleThe economic impact of retinal diseases for which gene therapy is emerging: a systematic literature reviewen
dc.typeReview
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