The Reality of Target Trial Emulation for Medical Decision-Making and HTA Recommendations - Is the Gap Between Academic and HTA Applications Too Wide?

PURPOSE: Target trial emulation (TTE) is a framework for using non-randomized data to emulate randomized controlled trials (RCTs) with a clearly specified target population, intervention strategies, and outcomes. TTE was recently endorsed by the National Institute for Health and Care Excellence (NICE) and has been touted as the potential magic bullet for improving the quality and reporting of real-world evidence in HTA submissions. Because TTE is a complex topic, and because most applications illustrate its use in close to ideal data scenarios, there is considerable barrier for developers and decision-makers in being able to understand, implement and critique TTE analyses in practical scenarios. This is particularly true for those commonly submitted to HTA agencies involving small sample sizes and externally controlled single arm trials.

This workshop will cover TTE in the context of real-world data scenarios for HTA submission with examples of practical applications for TTE in the context of synthetic control arm analyses.

Kristian Thorlund will introduce the audience to the current state of TTE applications (5 min).

Joy Shi will detail the design of TTE framework, cover the nuts and bolts of causal models, and address the need for multidisciplinary teams (15 min).

Alind Gupta will detail the challenges of applying TTE on real world data sets (15 min).

Stephen Duffield will examine submissions with TTE that were deemed high versus low quality and provide guidance for moving forward (15 min).

DESCRIPTION: Participants will be introduced to the stages required for well-conducted TTEs; challenges of designing causal models and the necessity of multidisciplinary teams; how to perform valid TTE when sample sizes are small; as well as what HTA agencies and payers generally accept as good quality TTEs.

This workshop would be valuable to researchers, industry analysts, and sponsor representatives who are interested in incorporating TTE in HTA submissions.