Health economic evaluation of gene replacement therapies: methodological issues and recommendations
Autor: | Samuel Aballéa, Pascal Auquier, Frank-Ulrich Fricke, Stavros Petrou, Aurélie Millier, Mondher Toumi, Steven Simoens, Daniel C. Malone, K. Thokagevistk, Ulf Persson, Maarten J. Postma, Lieven Annemans, Fernando Antoñanzas, Clément François, Rimma Velikanova, O. Dabbous |
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Přispěvatelé: | PharmacoTherapy, -Epidemiology and -Economics, Value, Affordability and Sustainability (VALUE), Real World Studies in PharmacoEpidemiology, -Genetics, -Economics and -Therapy (PEGET), Microbes in Health and Disease (MHD) |
Rok vydání: | 2020 |
Předmět: |
qaly
Actuarial science lcsh:Public aspects of medicine cost-effectiveness analysis lcsh:RA1-1270 Time horizon Disease Cost-effectiveness analysis Guideline lcsh:Business Health outcomes Gene replacement therapy methods QALY Gene replacement Economic evaluation guidelines lcsh:HF5001-6182 Psychology Methodological research health economic evaluation Research Article gene replacement therapy Valuation (finance) |
Zdroj: | Journal of Market Access & Health Policy, Vol 8, Iss 1 (2020) Journal of market access & health policy, 8(1):1822666 Journal of Market Access & Health Policy article-version (VoR) Version of Record |
ISSN: | 2001-6689 |
Popis: | 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. ispartof: J Mark Access Health Policy vol:8 issue:1 pages:1822666- ispartof: location:United States status: Published online |
Databáze: | OpenAIRE |
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