Bullement, A. orcid.org/0000-0001-7091-0972, Knowles, E.S., DasMahapatra, P. et al. (2 more authors) (2021) Cost-effectiveness analysis of rFVIIIFc versus contemporary rFVIII treatments for patients with severe hemophilia A without inhibitors in the United States. PharmacoEconomics - Open, 5 (4). pp. 625-633. ISSN 2509-4262
Abstract
Background
A range of treatments for patients with severe hemophilia A (HA) have been developed over the last decade, allowing for reduced frequency of administration and improved outcomes (joint health and breakthrough bleeding rates). While clinically effective, the cost effectiveness of these treatments has not been established.
Objective
This study presents a cost-effectiveness analysis of contemporary rFVIII treatments for severe HA patients without inhibitors.
Methods
A published semi-Markov model was used to compare three different prophylaxis regimens: (1) extended half-life (EHL) recombinant Factor VIII (rFVIII) Fc-fusion protein (rFVIIIFc, Eloctate®, Sanofi), (2) EHL PEGylated rFVIII (PEG-rFVIII, Adynovate®, Takeda), and (3) standard half-life (SHL) rFVIII (antihemophilic factor [recombinant], Advate®, Takeda), used as a proxy for all SHL rFVIII treatments. Acquisition costs were included based on published dosing and weight data. Benefits were incorporated through published annualized bleeding rates, rates of target joint development/resolution, and improvements in the modified hemophilia joint health score. Results were presented as total, discounted costs, and quality-adjusted life-years (QALYs).
Results
rFVIIIFc was shown to provide the most QALYs (27.922) compared with both PEG-rFVIII (27.454) and SHL rFVIII (27.071), at lower costs. Discounted lifetime costs were estimated at US$18.235m (rFVIIIFc), US$20.198m (PEG-rFVIII), and US$18.285m (SHL rFVIII), and were predominantly affected by model settings related to acquisition costs, patient weight, and dosing.
Conclusions
rFVIIIFc may offer a cost-effective option for severe HA patients. Uncertainties owing to the limited evidence base is the main limitation of the study.
Metadata
Item Type: | Article |
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Authors/Creators: |
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Copyright, Publisher and Additional Information: | © 2021 The Author(s). This 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/. |
Dates: |
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Institution: | The University of Sheffield |
Academic Units: | The University of Sheffield > Faculty of Medicine, Dentistry and Health (Sheffield) > School of Health and Related Research (Sheffield) |
Depositing User: | Symplectic Sheffield |
Date Deposited: | 30 Jul 2021 10:03 |
Last Modified: | 09 Mar 2022 10:00 |
Status: | Published |
Publisher: | Springer Nature |
Refereed: | Yes |
Identification Number: | 10.1007/s41669-021-00283-6 |
Related URLs: | |
Open Archives Initiative ID (OAI ID): | oai:eprints.whiterose.ac.uk:176678 |