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...

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Autores principales:	Michał Pochopień, Ewelina Paterak, Emilie Clay, Justyna Janik, Samuel Aballea, Małgorzata Biernikiewicz, Mondher Toumi
Formato:	article
Lenguaje:	EN
Publicado:	Taylor & Francis Group 2021
Materias:	gene therapy economic evaluation cost-effectiveness model health technology assessment Public aspects of medicine RA1-1270 Business HF5001-6182
Acceso en línea:	https://doaj.org/article/754b4c674be641ef872d82a64d8b4e17
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spelling	oai:doaj.org-article:754b4c674be641ef872d82a64d8b4e172021-11-17T14:21:57ZAn overview of health technology assessments of gene therapies with the focus on cost-effectiveness models2001-668910.1080/20016689.2021.2002006https://doaj.org/article/754b4c674be641ef872d82a64d8b4e172021-01-01T00:00:00Zhttp://dx.doi.org/10.1080/20016689.2021.2002006https://doaj.org/toc/2001-6689Background: 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.Michał PochopieńEwelina PaterakEmilie ClayJustyna JanikSamuel AballeaMałgorzata BiernikiewiczMondher ToumiTaylor & Francis Grouparticlegene therapyeconomic evaluationcost-effectiveness modelhealth technology assessmentPublic aspects of medicineRA1-1270BusinessHF5001-6182ENJournal of Market Access & Health Policy, Vol 9, Iss 1 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	gene therapy economic evaluation cost-effectiveness model health technology assessment Public aspects of medicine RA1-1270 Business HF5001-6182
spellingShingle	gene therapy economic evaluation cost-effectiveness model health technology assessment Public aspects of medicine RA1-1270 Business HF5001-6182 Michał Pochopień Ewelina Paterak Emilie Clay Justyna Janik Samuel Aballea Małgorzata Biernikiewicz Mondher Toumi An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models
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	article
author	Michał Pochopień Ewelina Paterak Emilie Clay Justyna Janik Samuel Aballea Małgorzata Biernikiewicz Mondher Toumi
author_facet	Michał Pochopień Ewelina Paterak Emilie Clay Justyna Janik Samuel Aballea Małgorzata Biernikiewicz Mondher Toumi
author_sort	Michał Pochopień
title	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_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_sort	overview of health technology assessments of gene therapies with the focus on cost-effectiveness models
publisher	Taylor & Francis Group
publishDate	2021
url	https://doaj.org/article/754b4c674be641ef872d82a64d8b4e17
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An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models

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