How Have Surrogate Outcomes Been Dealt With in Existing HTA Evaluations by Different HTA Bodies and What Lessons Can Be Learnt?

OBJECTIVES: The use of surrogate outcomes poses challenges to health technology assessment (HTA) agencies and payers when utilising such evidence to inform the clinical and cost-effectiveness of technologies, particularly when the surrogate outcome is novel without clear links to the longer-term outcome. Some HTA agencies have published methodological guidance on the use and validation of surrogate outcomes. However, it is not uncommon that decision makers must make recommendations in the absence of well-established evidence on the validation of surrogate outcomes. We aimed to identify how HTA agencies have considered and dealt with the uncertainties around surrogate outcomes in their technology evaluations.

METHODS: A qualitative study was used to explore the professional experiences of HTA staff globally on the use of surrogate outcomes in decision making. Data were collected using three focus groups with 29 participants representing 20 HTA agencies or associated organisations. A framework analysis was used to identify themes from the transcripts.

RESULTS: Early analysis of the focus group data suggests that HTA agencies frequently see submissions that use surrogates, and this is increasing. They face numerous issues when handling this data, including a lack of evidence. Participants suggested they would benefit from guidance to help them ascertain the required information from industry for economic modelling, on how to present the uncertainty arising from the surrogates, and how to appropriately report the findings from an economic model using surrogates. Full results will be presented during the conference.

CONCLUSIONS: Decision making using surrogate outcomes is a frequent and challenging problem faced by HTA agencies. These results provide the first-hand experiences and reflect the needs of HTA agencies globally. This work will inform a wider project aimed at developing recommendations and considerations for the use of surrogates in economic modelling as part of HTAs.