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

Drummond, Michael, Ciani, Oriana, Fornaro, Giulia, Jommi, Claudio, Dietrich, Eva Susanne, Espin, Jaime, Mossman, Jean and de Pouvourville, Gerard (2023) How are health technology assessment bodies responding to the assessment challenges posed by cell and gene therapy? BMC Health Services Research, 23 (1). ISSN 1472-6963

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Identification Number: 10.1186/s12913-023-09494-5

Abstract

Background: The 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. Methods: A 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. Results: The 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. Conclusions: The 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.

Item Type: Article
Official URL: https://bmchealthservres.biomedcentral.com/
Additional Information: © 2023 The Author(s).
Divisions: LSE Health
Subjects: R Medicine > RA Public aspects of medicine > RA0421 Public health. Hygiene. Preventive Medicine
Q Science > QH Natural history > QH301 Biology
Q Science > QH Natural history > QH426 Genetics
Date Deposited: 01 Jun 2023 11:48
Last Modified: 15 Jul 2024 00:54
URI: http://eprints.lse.ac.uk/id/eprint/119298

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