Next Article in Journal
Impact of Non-Medical Switching of Prescription Medications on Health Outcomes: An E-Survey of High-Volume Medicare and Medicaid Physician Providers
Previous Article in Journal
Chinese Guidelines Related to Novel Coronavirus Pneumonia
 
 
Search for Articles:
Title / Keyword
Author / Affiliation / Email
Journal
Article Type
 
 
Advanced Search
 
Section
Special Issue
Volume
Issue
Number
Page
 
Journals
JMAHP
Volume 8
Issue 1
10.1080/20016689.2020.1822666
jmahp-logo
Submit to this Journal Review for this Journal Propose a Special Issue
► Article Menu

Article Menu

share Share announcement Help format_quote Cite question_answer Discuss in SciProfiles thumb_up
...
Endorse textsms
...
Comment
Journal of Market Access & Health Policy (JMAHP) is published by MDPI from Volume 12 Issue 1 (2024). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by MDPI on mdpi.com as a courtesy and upon agreement with Taylor & Francis.
first_page
settings
Order Article Reprints
Font Type:
Arial Georgia Verdana
Font Size:
Aa Aa Aa
Line Spacing:
Column Width:
Background:
Guidelines

Health Economic Evaluation of Gene Replacement Therapies: Methodological Issues and Recommendations

by
Samuel Aballéa
1,*,
Katia Thokagevistk
2,
Rimma Velikanova
3,
Steven Simoens
4,
Lieven Annemans
5,
Fernando Antonanzas
6,
Pascal Auquier
7,
Clément François
2,8,
Frank-Ulrich Fricke
9,
Daniel Malone
10,
Aurélie Millier
2,
Ulf Persson
11,
Stavros Petrou
12,
Omar Dabbous
13,
Maarten Postma
3 and
Mondher Toumi
2,8,†
1
Creativ-Ceutical, HEOR, Rotterdam, The Netherlands
2
Creativ-Ceutical, HEOR, Paris, France
3
Groningen Research Institute of Pharmacy, PharmacoTherapy, Epidemiology & Economics, University of Groningen, Groningen, The Netherlands
4
Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium
5
Department of Public Health, Ghent University, Ghent, Belgium
6
Department of Economics, University of La Rioja, Logroño, Spain
7
CEReSS - Health Service Research and Quality of Life Center, Aix-Marseille University, Marseille, France
8
Public Health Department - Research Unit, Aix-Marseille University, Marseille, France
9
Technische Hochschule Nürnberg, Nürnberg, Germany
10
Pharmacotherapy Faculty, College of Pharmacy, University of Utah, Salt Lake City, USA
11
The Swedish Institute for Health Economics (IHE), Lund, Sweden
12
Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, UK
13
Global Health Economics and Outcomes Research and Real World EvidenceAveXis Inc., Novartis Gene Therapies, Bannockburn, IL, USA
 
add Show full affiliation list
 
remove Hide full affiliation list
*
Author to whom correspondence should be addressed.
†
Mondher Toumi is the Editor-in-Chief of the Journal of Market Access and Health Policy but did not participate in the editorial review process of this article.
J. Mark. Access Health Policy 2020, 8(1), 1822666; https://doi.org/10.1080/20016689.2020.1822666
Submission received: 20 March 2020 / Revised: 31 August 2020 / Accepted: 4 September 2020 / Published: 11 October 2020

Abstract

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.
Keywords: gene replacement therapy; guidelines; health economic evaluation; methods; cost-effectiveness analysis; QALY gene replacement therapy; guidelines; health economic evaluation; methods; cost-effectiveness analysis; QALY

Share and Cite

MDPI and ACS Style

Aballéa, S.; Thokagevistk, K.; Velikanova, R.; Simoens, S.; Annemans, L.; Antonanzas, F.; Auquier, P.; François, C.; Fricke, F.-U.; Malone, D.; et al. Health Economic Evaluation of Gene Replacement Therapies: Methodological Issues and Recommendations. J. Mark. Access Health Policy 2020, 8, 1822666. https://doi.org/10.1080/20016689.2020.1822666

AMA Style

Aballéa S, Thokagevistk K, Velikanova R, Simoens S, Annemans L, Antonanzas F, Auquier P, François C, Fricke F-U, Malone D, et al. Health Economic Evaluation of Gene Replacement Therapies: Methodological Issues and Recommendations. Journal of Market Access & Health Policy. 2020; 8(1):1822666. https://doi.org/10.1080/20016689.2020.1822666

Chicago/Turabian Style

Aballéa, Samuel, Katia Thokagevistk, Rimma Velikanova, Steven Simoens, Lieven Annemans, Fernando Antonanzas, Pascal Auquier, Clément François, Frank-Ulrich Fricke, Daniel Malone, and et al. 2020. "Health Economic Evaluation of Gene Replacement Therapies: Methodological Issues and Recommendations" Journal of Market Access & Health Policy 8, no. 1: 1822666. https://doi.org/10.1080/20016689.2020.1822666

APA Style

Aballéa, S., Thokagevistk, K., Velikanova, R., Simoens, S., Annemans, L., Antonanzas, F., Auquier, P., François, C., Fricke, F. -U., Malone, D., Millier, A., Persson, U., Petrou, S., Dabbous, O., Postma, M., & Toumi, M. (2020). Health Economic Evaluation of Gene Replacement Therapies: Methodological Issues and Recommendations. Journal of Market Access & Health Policy, 8(1), 1822666. https://doi.org/10.1080/20016689.2020.1822666

Article Metrics

Back to TopTop