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Cost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data

BACKGROUND: Patients with severe hemophilia A (SHA) in Italy are routinely treated with standard half-life recombinant factor VIII (rFVIII) products. rFVIII Fc-fusion protein (rFVIIIFc) is an extended half-life rFVIII product that enables less frequent administration than rFVIII, which may support i...

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Autores principales: Bullement, Ash, McMordie, Samuel Thomas, Hatswell, Anthony James, Li, Nanxin, Wilson, Koo
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7018914/
https://www.ncbi.nlm.nih.gov/pubmed/31280415
http://dx.doi.org/10.1007/s41669-019-0158-8
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author Bullement, Ash
McMordie, Samuel Thomas
Hatswell, Anthony James
Li, Nanxin
Wilson, Koo
author_facet Bullement, Ash
McMordie, Samuel Thomas
Hatswell, Anthony James
Li, Nanxin
Wilson, Koo
author_sort Bullement, Ash
collection PubMed
description BACKGROUND: Patients with severe hemophilia A (SHA) in Italy are routinely treated with standard half-life recombinant factor VIII (rFVIII) products. rFVIII Fc-fusion protein (rFVIIIFc) is an extended half-life rFVIII product that enables less frequent administration than rFVIII, which may support improved adherence. Available data indicate low breakthrough bleed rates and potentially improved long-term joint health for patients treated with rFVIIIFc prophylaxis. OBJECTIVE: This study assessed the cost effectiveness of rFVIIIFc versus rFVIII from an Italian healthcare perspective. METHODS: A Semi-Markov model was constructed to assess the lifetime costs and benefits of rFVIII and rFVIIIFc prophylaxis. rFVIII product acquisition costs from a published Italian database were included for both prophylaxis and the resolution of breakthrough bleeding. Clinical outcomes within the model were determined based on published annualized bleeding rates and literature regarding the development of target joints (TJs) as the incidence of bleeds and TJs is associated with impaired health-related quality of life. Cost effectiveness was assessed using cost per quality-adjusted life-year (QALY) gained. RESULTS: Compared with rFVIII, rFVIIIFc was associated with a per-patient cost saving of approximately €1.3 million and QALY gains of 0.39 over a lifetime horizon. Sensitivity analyses considering alternative efficacy, dosing, and structural assumptions each showed that rFVIIIFc dominated rFVIII (i.e., provided more QALYs at a reduced cost). CONCLUSIONS: This cost-effectiveness analysis demonstrated that rFVIIIFc may offer a cost-effective treatment option for patients with SHA in Italy. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s41669-019-0158-8) contains supplementary material, which is available to authorized users.
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spelling pubmed-70189142020-02-28 Cost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data Bullement, Ash McMordie, Samuel Thomas Hatswell, Anthony James Li, Nanxin Wilson, Koo Pharmacoecon Open Original Research Article BACKGROUND: Patients with severe hemophilia A (SHA) in Italy are routinely treated with standard half-life recombinant factor VIII (rFVIII) products. rFVIII Fc-fusion protein (rFVIIIFc) is an extended half-life rFVIII product that enables less frequent administration than rFVIII, which may support improved adherence. Available data indicate low breakthrough bleed rates and potentially improved long-term joint health for patients treated with rFVIIIFc prophylaxis. OBJECTIVE: This study assessed the cost effectiveness of rFVIIIFc versus rFVIII from an Italian healthcare perspective. METHODS: A Semi-Markov model was constructed to assess the lifetime costs and benefits of rFVIII and rFVIIIFc prophylaxis. rFVIII product acquisition costs from a published Italian database were included for both prophylaxis and the resolution of breakthrough bleeding. Clinical outcomes within the model were determined based on published annualized bleeding rates and literature regarding the development of target joints (TJs) as the incidence of bleeds and TJs is associated with impaired health-related quality of life. Cost effectiveness was assessed using cost per quality-adjusted life-year (QALY) gained. RESULTS: Compared with rFVIII, rFVIIIFc was associated with a per-patient cost saving of approximately €1.3 million and QALY gains of 0.39 over a lifetime horizon. Sensitivity analyses considering alternative efficacy, dosing, and structural assumptions each showed that rFVIIIFc dominated rFVIII (i.e., provided more QALYs at a reduced cost). CONCLUSIONS: This cost-effectiveness analysis demonstrated that rFVIIIFc may offer a cost-effective treatment option for patients with SHA in Italy. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s41669-019-0158-8) contains supplementary material, which is available to authorized users. Springer International Publishing 2019-07-06 /pmc/articles/PMC7018914/ /pubmed/31280415 http://dx.doi.org/10.1007/s41669-019-0158-8 Text en © The Author(s) 2019 Open AccessThis article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International License (http://creativecommons.org/licenses/by-nc/4.0/), which permits any noncommercial use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.
spellingShingle Original Research Article
Bullement, Ash
McMordie, Samuel Thomas
Hatswell, Anthony James
Li, Nanxin
Wilson, Koo
Cost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data
title Cost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data
title_full Cost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data
title_fullStr Cost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data
title_full_unstemmed Cost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data
title_short Cost-Effectiveness Analysis of Recombinant Factor VIII Fc-Fusion Protein (rFVIIIFc) for the Treatment of Severe Hemophilia A in Italy Incorporating Real-World Dosing and Joint Health Data
title_sort cost-effectiveness analysis of recombinant factor viii fc-fusion protein (rfviiifc) for the treatment of severe hemophilia a in italy incorporating real-world dosing and joint health data
topic Original Research Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7018914/
https://www.ncbi.nlm.nih.gov/pubmed/31280415
http://dx.doi.org/10.1007/s41669-019-0158-8
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