Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.

Autor: El-Sayed AA; Public Health Institute, Faculty of Health, Liverpool John Moores University, Liverpool, UK.; Medical Affairs Department, Novo Nordisk Egypt, Cairo, Egypt., Bolous NS; Department of Global Pediatric Medicine, St. Jude Children's Research Hospital, Memphis, TN, USA. nancy.bolous@stjude.org.
Jazyk: angličtina
Zdroj: PharmacoEconomics - open [Pharmacoecon Open] 2024 Nov 15. Date of Electronic Publication: 2024 Nov 15.
DOI: 10.1007/s41669-024-00540-4
Abstrakt: Introduction: Haemophilia is a rare genetic bleeding disorder that leads to musculoskeletal complications. The high cost of haemophilia treatment necessitates a thorough evaluation of its economic burden. However, due to the difficulty of estimating direct non-medical, indirect, and intangible costs, studies often underestimate the actual economic burden of haemophilia. This scoping review aims to summarise economic studies in haemophilia conducted from a societal perspective.
Methods: A systematic search across eight scholarly databases, grey literature, and reference lists until the 5 th of July 2023 was conducted to identify relevant studies. The inclusion criteria encompassed full-text, English-language publications of economic analyses in congenital haemophilia from a societal perspective. Model-based studies and those adopting a payer perspective were excluded. Costs were adjusted to international dollars (I$) and US dollars (US$) in 2022 for comparability.
Results: Out of 2993 potential sources identified, 25 studies met the inclusion criteria, covering 7226 persons with haemophilia across 22 countries. All studies reported direct medical costs, with four excluding the cost of haemostatic therapy. Fifteen studies reported direct formal non-medical costs, while eight reported direct informal non-medical costs. All but one study reported the indirect costs. The average annual costs of haemophilia varied widely based on treatment modality, disease severity, geographical location, and included cost categories. When including the cost of clotting factor replacement therapy (CFRT), the total cost for severe haemophilia without inhibitors ranged from 1566 I$ to 700,070 I$ per person per year (lowest value reported in India and highest in the United States). CFRT represented up to 99.9% of the total cost for those receiving prophylaxis and up to 95.1% for episodic treatment. Haemostatic therapies accounted for 82% of the total cost in patients with inhibitors.
Conclusion: There is a significant heterogeneity in defining cost categories required for a comprehensive economic analysis from a societal perspective. While haemostatic therapies constitute a substantial portion of the overall cost, direct non-medical and indirect costs are crucial as they are often paid out-of-pocket and may impede access to treatment. It is essential for haematologists and economists to establish a standardised costing framework for future studies, particularly in the era of novel therapies.
Competing Interests: Declarations Author contributions AAE set the review design; conducted the systematic literature search; retrieved, screened, and reviewed potential sources of evidence; extracted and analysed data; synthesised results from the included studies; and wrote the manuscript. NSB supervised; contributed to data extraction, data analysis, and synthesising results; and reviewed and wrote the manuscript. Funding NSB is partly funded by ALSAC. The funding source had no role in the design and conduct of the study; management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; or the decision to submit the manuscript for publication. Availability of data and materials Charts and figures in this manuscript will be available under the journal policy. Code availability All the information included in this manuscript will be available on the journal website. Ethics approval and consent to participate The approval of the institutional review board (IRB) was not required for conducting this scoping review. Competing interests AAE is an employee of Novo Nordisk Egypt. NSB does not have conflicts of interest for conducting this research project.
(© 2024. The Author(s).)
Databáze: MEDLINE
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