How are health technology assessment bodies responding to the assessment challenges posed by cell and gene therapy?

BMC Health Services Research

Research

Jaime Espin¹  Giulia Fornaro²  Oriana Ciani²  Claudio Jommi²  Michael Drummond³  Gerard de Pouvourville⁴  Jean Mossman⁵  Eva Susanne Dietrich⁶ 

[1]Andalusian School of Public Health, Andalusia, Spain

[2]CERGAS, SDA Bocconi School of Management, Milan, Italy

[3]Centre for Health Economics, University of York, York, UK

[4]CERGAS, SDA Bocconi School of Management, Milan, Italy

[5]ESSEC, Paris, France

[6]Patient Representative and Visiting Senior Research Associate in the Medical Technology Research Group, LSE Health, London School of Economics, London, UK

[7]University of Bonn Pharmaceutical Institute, Bonn, Germany

关键词: Cost-effectiveness analysis;    Advanced therapy medicinal products;    Health technology assessment;    Managed entry agreements;    Reimbursement;   

DOI  :  10.1186/s12913-023-09494-5

 received in 2022-11-04, accepted in 2023-05-03,  发布年份 2023

来源: Springer

【 摘 要 】

BackgroundThe aims of this research were to provide a better understanding of the specific evidence needs for assessment of clinical and cost-effectiveness of cell and gene therapies, and to explore the extent that the relevant categories of evidence are considered in health technology assessment (HTA) processes.MethodsA targeted literature review was conducted to identify the specific categories of evidence relevant to the assessment of these therapies. Forty-six HTA reports for 9 products in 10 cell and gene therapy indications across 8 jurisdictions were analysed to determine the extent to which various items of evidence were considered.ResultsThe items to which the HTA bodies reacted positively were: treatment was for a rare disease or serious condition, lack of alternative therapies, evidence indicating substantial health gains, and when alternative payment models could be agreed. The items to which they reacted negatively were: use of unvalidated surrogate endpoints, single arm trials without an adequately matched alternative therapy, inadequate reporting of adverse consequences and risks, short length of follow-up in clinical trials, extrapolating to long-term outcomes, and uncertainty around the economic estimates.ConclusionsThe consideration by HTA bodies of evidence relating to the particular features of cell and gene therapies is variable. Several suggestions are made for addressing the assessment challenges posed by these therapies. Jurisdictions conducting HTAs of these therapies can consider whether these suggestions could be incorporated within their existing approach through strengthening deliberative decision-making or performing additional analyses.

【 授权许可】

CC BY   
© The Author(s) 2023

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