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

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.

Automated summary

The research aimed to understand the evidence needs for assessing the clinical and cost-effectiveness of cell and gene therapies, and to explore how HTA processes consider the relevant evidence. The study identified specific categories of evidence and analysed HTA reports from different jurisdictions. It found that HTA bodies consider some evidence positively, such as rare diseases and substantial health gains, but react negatively to unvalidated surrogate endpoints and inadequate reporting of risks.