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Health economic evaluation of gene replacement therapies: methodological issues and recommendations

Objective: To provide recommendations for addressing previously identified key challenges in health economic evaluations of Gene Replacement Therapies (GRTs), including: 1) the assessment of clinical effectiveness; 2) the valuation of health outcomes; 3) the time horizon and extrapolation of effects...

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Autores principales: Aballéa, Samuel, Thokagevistk, Katia, Velikanova, Rimma, Simoens, Steven, Annemans, Lieven, Antonanzas, Fernando, Auquier, Pascal, François, Clément, Fricke, Frank-Ulrich, Malone, Daniel, Millier, Aurélie, Persson, Ulf, Petrou, Stavros, Dabbous, Omar, Postma, Maarten, Toumi, Mondher
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Routledge 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7580851/
https://www.ncbi.nlm.nih.gov/pubmed/33144927
http://dx.doi.org/10.1080/20016689.2020.1822666
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author Aballéa, Samuel
Thokagevistk, Katia
Velikanova, Rimma
Simoens, Steven
Annemans, Lieven
Antonanzas, Fernando
Auquier, Pascal
François, Clément
Fricke, Frank-Ulrich
Malone, Daniel
Millier, Aurélie
Persson, Ulf
Petrou, Stavros
Dabbous, Omar
Postma, Maarten
Toumi, Mondher
author_facet Aballéa, Samuel
Thokagevistk, Katia
Velikanova, Rimma
Simoens, Steven
Annemans, Lieven
Antonanzas, Fernando
Auquier, Pascal
François, Clément
Fricke, Frank-Ulrich
Malone, Daniel
Millier, Aurélie
Persson, Ulf
Petrou, Stavros
Dabbous, Omar
Postma, Maarten
Toumi, Mondher
author_sort Aballéa, Samuel
collection PubMed
description Objective: To provide recommendations for addressing previously identified key challenges in health economic evaluations of Gene Replacement Therapies (GRTs), including: 1) the assessment of clinical effectiveness; 2) the valuation of health outcomes; 3) the time horizon and extrapolation of effects beyond trial duration; 4) the estimation of costs; 5) the selection of appropriate discount rates; 6) the incorporation of broader elements of value; and 7) affordability. Methods: A literature review on economic evaluations of GRT was performed. Interviews were conducted with 8 European and US health economic experts with experience in evaluations of GRT. Targeted literature reviews were conducted to investigate further potential solutions to specific challenges. Recommendations: Experts agreed on factors to be considered to ensure the acceptability of historical cohorts by HTA bodies. Existing prospective registries or, if not available, retrospective registries, may be used to analyse different disease trajectories and inform extrapolations. The importance of expert opinion due to limited data was acknowledged. Expert opinion should be obtained using structured elicitation techniques. Broader elements of value, beyond health gains directly related to treatment, can be considered through the application of a factor to inflate the quality-adjusted life years (QALYs) or a higher cost-effectiveness threshold. Additionally, the use of cost-benefit analysis and saved young life equivalents (SAVE) were proposed as alternatives to QALYs for the valuations of outcomes of GRT as they can incorporate broader elements of value and avoid problems of eliciting utilities for paediatric diseases. Conclusions: While some of the limitations of economic evaluations of GRT are inherent to limited clinical data and lack of experience with these treatments, others may be addressed by methodological research to be conducted by health economists.
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spelling pubmed-75808512020-11-02 Health economic evaluation of gene replacement therapies: methodological issues and recommendations Aballéa, Samuel Thokagevistk, Katia Velikanova, Rimma Simoens, Steven Annemans, Lieven Antonanzas, Fernando Auquier, Pascal François, Clément Fricke, Frank-Ulrich Malone, Daniel Millier, Aurélie Persson, Ulf Petrou, Stavros Dabbous, Omar Postma, Maarten Toumi, Mondher J Mark Access Health Policy Guideline Objective: To provide recommendations for addressing previously identified key challenges in health economic evaluations of Gene Replacement Therapies (GRTs), including: 1) the assessment of clinical effectiveness; 2) the valuation of health outcomes; 3) the time horizon and extrapolation of effects beyond trial duration; 4) the estimation of costs; 5) the selection of appropriate discount rates; 6) the incorporation of broader elements of value; and 7) affordability. Methods: A literature review on economic evaluations of GRT was performed. Interviews were conducted with 8 European and US health economic experts with experience in evaluations of GRT. Targeted literature reviews were conducted to investigate further potential solutions to specific challenges. Recommendations: Experts agreed on factors to be considered to ensure the acceptability of historical cohorts by HTA bodies. Existing prospective registries or, if not available, retrospective registries, may be used to analyse different disease trajectories and inform extrapolations. The importance of expert opinion due to limited data was acknowledged. Expert opinion should be obtained using structured elicitation techniques. Broader elements of value, beyond health gains directly related to treatment, can be considered through the application of a factor to inflate the quality-adjusted life years (QALYs) or a higher cost-effectiveness threshold. Additionally, the use of cost-benefit analysis and saved young life equivalents (SAVE) were proposed as alternatives to QALYs for the valuations of outcomes of GRT as they can incorporate broader elements of value and avoid problems of eliciting utilities for paediatric diseases. Conclusions: While some of the limitations of economic evaluations of GRT are inherent to limited clinical data and lack of experience with these treatments, others may be addressed by methodological research to be conducted by health economists. Routledge 2020-10-11 /pmc/articles/PMC7580851/ /pubmed/33144927 http://dx.doi.org/10.1080/20016689.2020.1822666 Text en © 2020 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 Guideline
Aballéa, Samuel
Thokagevistk, Katia
Velikanova, Rimma
Simoens, Steven
Annemans, Lieven
Antonanzas, Fernando
Auquier, Pascal
François, Clément
Fricke, Frank-Ulrich
Malone, Daniel
Millier, Aurélie
Persson, Ulf
Petrou, Stavros
Dabbous, Omar
Postma, Maarten
Toumi, Mondher
Health economic evaluation of gene replacement therapies: methodological issues and recommendations
title Health economic evaluation of gene replacement therapies: methodological issues and recommendations
title_full Health economic evaluation of gene replacement therapies: methodological issues and recommendations
title_fullStr Health economic evaluation of gene replacement therapies: methodological issues and recommendations
title_full_unstemmed Health economic evaluation of gene replacement therapies: methodological issues and recommendations
title_short Health economic evaluation of gene replacement therapies: methodological issues and recommendations
title_sort health economic evaluation of gene replacement therapies: methodological issues and recommendations
topic Guideline
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7580851/
https://www.ncbi.nlm.nih.gov/pubmed/33144927
http://dx.doi.org/10.1080/20016689.2020.1822666
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