PURPOSE:

Producing plausible lifetime extrapolations is fundamental to appropriate decision-making within health technology assessment (HTA). However, continuing development of novel therapies and reliance on surrogate outcomes lead to increasingly immature data, complicating this essential task. Real-world evidence (RWE) and expert elicitation have been proposed as potential solutions to incorporate external evidence, with both ICER in the U.S. and NICE in the UK seeking to increase their use. But how exactly should external evidence be used?

Whilst guidance provided so far by HTA bodies and in the literature is welcome; practical guidance to inform selection of methods is sorely lacking. Existing guidance includes frameworks which provide rationale for the intention behind using external evidence more widely, how to source such data (such as the NICE RWE framework), and the use of individual patient-level data from clinical trials for extrapolation (such as NICE TSD21). However, no guidance considers how external evidence should be incorporated into survival extrapolations for HTA.

More research is needed to determine the best solutions; however, there is an urgent need for guidance on how to manage the seemingly endless options available to analysts in the interim.

DESCRIPTION:

This workshop will take the first steps towards development of practical guidance.

Bart Heeg will demonstrate how the DIVE framework can be used to transparently document developer considerations and how this fits with existing HTA guidance.

Ash Bullement will provide an overview of methodological approaches in use and pros/cons based upon the results of a systematic review of methods literature.

Brett McQueen will provide his insights on the use of RWE in a recent multiple myeloma appraisal in collaboration with ICER.

Dawn Lee will seek audience input on a draft framework for decision-making based on her insights from attempted application of NICE guidance to a multiple technology appraisal in RCC.