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Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A

The objective was to undertake an early cost-effectiveness assessment of valoctocogene roxaparvovec (valrox; Roctavian) compared to factor (F)VIII prophylaxis or emicizumab (Hemlibra; Roche HQ, Bazel, Switzerland) in patients with severe Hemophilia A (HA) without FVIII-antibodies. We also aimed to i...

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Autores principales: ten Ham, Renske M.T., Walker, Simon M., Soares, Marta O., Frederix, Geert W.J., Leebeek, Frank W.G., Fischer, Kathelijn, Coppens, Michiel, Palmer, Stephen J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Lippincott Williams & Wilkins 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8820916/
https://www.ncbi.nlm.nih.gov/pubmed/35141470
http://dx.doi.org/10.1097/HS9.0000000000000679
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author ten Ham, Renske M.T.
Walker, Simon M.
Soares, Marta O.
Frederix, Geert W.J.
Leebeek, Frank W.G.
Fischer, Kathelijn
Coppens, Michiel
Palmer, Stephen J.
author_facet ten Ham, Renske M.T.
Walker, Simon M.
Soares, Marta O.
Frederix, Geert W.J.
Leebeek, Frank W.G.
Fischer, Kathelijn
Coppens, Michiel
Palmer, Stephen J.
author_sort ten Ham, Renske M.T.
collection PubMed
description The objective was to undertake an early cost-effectiveness assessment of valoctocogene roxaparvovec (valrox; Roctavian) compared to factor (F)VIII prophylaxis or emicizumab (Hemlibra; Roche HQ, Bazel, Switzerland) in patients with severe Hemophilia A (HA) without FVIII-antibodies. We also aimed to incorporate and quantify novel measures of value such as treatment durability, maximum value-based price (MVBP) and break-even time (ie, time until benefits begin to offset upfront payment). We constructed a Markov model to model bleeds over time which were linked to costs and quality-of-life decrements. In the valrox arm, FVIII over time was estimated combining initial effect and treatment waning and then linked to bleeds. In FVIII and emicizumab arms, bleeds were based on trial evidence. Evidence and assumptions were validated using expert elicitation. Model robustness was tested via sensitivity analyses. A Dutch societal perspective was applied with a 10-year time horizon. Valrox in comparison to FVIII, and emicizumab showed small increases in quality-adjusted life years at lower costs, and were therefore dominant. Valrox’ base case MVBP was estimated at €2.65 million/treatment compared to FVIII and €3.5 million/treatment versus emicizumab. Mean break-even time was 8.03 years compared to FVIII and 5.68 years to emicizumab. Early modeling of patients with HA in The Netherlands treated with valrox resulted in estimated improved health and lower cost compared to prophylactic FVIII and emicizumab. We also demonstrated feasibility of incorporation of treatment durability and novel outcomes such as value-based pricing scenarios and break-even time. Future work should aim to better characterize uncertainties and increase translation of early modeling to direct research efforts.
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spelling pubmed-88209162022-02-08 Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A ten Ham, Renske M.T. Walker, Simon M. Soares, Marta O. Frederix, Geert W.J. Leebeek, Frank W.G. Fischer, Kathelijn Coppens, Michiel Palmer, Stephen J. Hemasphere Article The objective was to undertake an early cost-effectiveness assessment of valoctocogene roxaparvovec (valrox; Roctavian) compared to factor (F)VIII prophylaxis or emicizumab (Hemlibra; Roche HQ, Bazel, Switzerland) in patients with severe Hemophilia A (HA) without FVIII-antibodies. We also aimed to incorporate and quantify novel measures of value such as treatment durability, maximum value-based price (MVBP) and break-even time (ie, time until benefits begin to offset upfront payment). We constructed a Markov model to model bleeds over time which were linked to costs and quality-of-life decrements. In the valrox arm, FVIII over time was estimated combining initial effect and treatment waning and then linked to bleeds. In FVIII and emicizumab arms, bleeds were based on trial evidence. Evidence and assumptions were validated using expert elicitation. Model robustness was tested via sensitivity analyses. A Dutch societal perspective was applied with a 10-year time horizon. Valrox in comparison to FVIII, and emicizumab showed small increases in quality-adjusted life years at lower costs, and were therefore dominant. Valrox’ base case MVBP was estimated at €2.65 million/treatment compared to FVIII and €3.5 million/treatment versus emicizumab. Mean break-even time was 8.03 years compared to FVIII and 5.68 years to emicizumab. Early modeling of patients with HA in The Netherlands treated with valrox resulted in estimated improved health and lower cost compared to prophylactic FVIII and emicizumab. We also demonstrated feasibility of incorporation of treatment durability and novel outcomes such as value-based pricing scenarios and break-even time. Future work should aim to better characterize uncertainties and increase translation of early modeling to direct research efforts. Lippincott Williams & Wilkins 2022-01-28 /pmc/articles/PMC8820916/ /pubmed/35141470 http://dx.doi.org/10.1097/HS9.0000000000000679 Text en Copyright © 2022 the Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the European Hematology Association. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution-Non Commercial-No Derivatives License 4.0 (CCBY-NC-ND) (https://creativecommons.org/licenses/by-nc-nd/4.0/) , where it is permissible to download and share the work provided it is properly cited. The work cannot be changed in any way or used commercially without permission from the journal.
spellingShingle Article
ten Ham, Renske M.T.
Walker, Simon M.
Soares, Marta O.
Frederix, Geert W.J.
Leebeek, Frank W.G.
Fischer, Kathelijn
Coppens, Michiel
Palmer, Stephen J.
Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A
title Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A
title_full Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A
title_fullStr Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A
title_full_unstemmed Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A
title_short Modeling Benefits, Costs, and Affordability of a Novel Gene Therapy in Hemophilia A
title_sort modeling benefits, costs, and affordability of a novel gene therapy in hemophilia a
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8820916/
https://www.ncbi.nlm.nih.gov/pubmed/35141470
http://dx.doi.org/10.1097/HS9.0000000000000679
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