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An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models
BACKGROUND: Gene therapies can treat, prevent, or cure a disease by changing the expression of a person’s genes. They are an innovative strategy for treating genetic disorders; however, they are still emerging on the market access and in the healthcare system. Health technology assessment (HTA) agen...
Autores principales: | , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Routledge
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8592603/ https://www.ncbi.nlm.nih.gov/pubmed/34790341 http://dx.doi.org/10.1080/20016689.2021.2002006 |
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author | Pochopień, Michał Paterak, Ewelina Clay, Emilie Janik, Justyna Aballea, Samuel Biernikiewicz, Małgorzata Toumi, Mondher |
author_facet | Pochopień, Michał Paterak, Ewelina Clay, Emilie Janik, Justyna Aballea, Samuel Biernikiewicz, Małgorzata Toumi, Mondher |
author_sort | Pochopień, Michał |
collection | PubMed |
description | BACKGROUND: Gene therapies can treat, prevent, or cure a disease by changing the expression of a person’s genes. They are an innovative strategy for treating genetic disorders; however, they are still emerging on the market access and in the healthcare system. Health technology assessment (HTA) agencies have not yet elaborated any standardised approach for assessing gene therapies; therefore, significant differences can be seen during HTAs carried out in various countries. In this review, we focused on submitted economic models of gene therapies approved for use by the US FDA and EMA with the aim to provide a comprehensive summary of how selected HTA bodies assessed the cost-effectiveness of gene therapies. An additional objective was to examine and discuss differences in the methods used in economic models across countries and drugs. METHODS: We identified economic models of gene therapies from six countries (NICE, IQWiG, SMC, HAS, CADTH, ICER) and focused on nine agents (Glybera, Imlygic, Strimvelis, Yescarta, Kymriah, Luxturna, Zynteglo, Zolgensma, Tecartus). Details of cost-utility evaluations and budget impact models were reviewed and extracted. RESULTS: Overall, 983 publications were identified, and 17 studies were included for the analysis. Reviewed evaluations of gene therapies differed in terms of the study perspective, discounting, extrapolation of outcomes based on limited and immature data, time horizon, and adequate estimation of benefits in terms of quality-adjusted life-years. Methods of economic evaluations were in line with the current recommendations; however, long-term follow-up studies are still missing. CONCLUSIONS: Discrepancies in an economic evaluation of gene therapies between different HTA bodies are rooted in a lack of general assessment frameworks specific to gene therapies. Although challenges were resolved by adjustments to the currently used value assessment framework, new methodological approaches would be useful. In addition, to improve the methods and quality of an evaluation, further research would be valuable. |
format | Online Article Text |
id | pubmed-8592603 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Routledge |
record_format | MEDLINE/PubMed |
spelling | pubmed-85926032021-11-16 An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models Pochopień, Michał Paterak, Ewelina Clay, Emilie Janik, Justyna Aballea, Samuel Biernikiewicz, Małgorzata Toumi, Mondher J Mark Access Health Policy Review Article BACKGROUND: Gene therapies can treat, prevent, or cure a disease by changing the expression of a person’s genes. They are an innovative strategy for treating genetic disorders; however, they are still emerging on the market access and in the healthcare system. Health technology assessment (HTA) agencies have not yet elaborated any standardised approach for assessing gene therapies; therefore, significant differences can be seen during HTAs carried out in various countries. In this review, we focused on submitted economic models of gene therapies approved for use by the US FDA and EMA with the aim to provide a comprehensive summary of how selected HTA bodies assessed the cost-effectiveness of gene therapies. An additional objective was to examine and discuss differences in the methods used in economic models across countries and drugs. METHODS: We identified economic models of gene therapies from six countries (NICE, IQWiG, SMC, HAS, CADTH, ICER) and focused on nine agents (Glybera, Imlygic, Strimvelis, Yescarta, Kymriah, Luxturna, Zynteglo, Zolgensma, Tecartus). Details of cost-utility evaluations and budget impact models were reviewed and extracted. RESULTS: Overall, 983 publications were identified, and 17 studies were included for the analysis. Reviewed evaluations of gene therapies differed in terms of the study perspective, discounting, extrapolation of outcomes based on limited and immature data, time horizon, and adequate estimation of benefits in terms of quality-adjusted life-years. Methods of economic evaluations were in line with the current recommendations; however, long-term follow-up studies are still missing. CONCLUSIONS: Discrepancies in an economic evaluation of gene therapies between different HTA bodies are rooted in a lack of general assessment frameworks specific to gene therapies. Although challenges were resolved by adjustments to the currently used value assessment framework, new methodological approaches would be useful. In addition, to improve the methods and quality of an evaluation, further research would be valuable. Routledge 2021-11-13 /pmc/articles/PMC8592603/ /pubmed/34790341 http://dx.doi.org/10.1080/20016689.2021.2002006 Text en © 2021 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group. https://creativecommons.org/licenses/by-nc/4.0/This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License (http://creativecommons.org/licenses/by-nc/4.0/ (https://creativecommons.org/licenses/by-nc/4.0/) ), which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Review Article Pochopień, Michał Paterak, Ewelina Clay, Emilie Janik, Justyna Aballea, Samuel Biernikiewicz, Małgorzata Toumi, Mondher An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models |
title | An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models |
title_full | An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models |
title_fullStr | An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models |
title_full_unstemmed | An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models |
title_short | An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models |
title_sort | overview of health technology assessments of gene therapies with the focus on cost-effectiveness models |
topic | Review Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8592603/ https://www.ncbi.nlm.nih.gov/pubmed/34790341 http://dx.doi.org/10.1080/20016689.2021.2002006 |
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