Using Target Trial Emulation for Estimation of Treatment Effects Using Real-World Data

PURPOSE: Health technology assessment (HTA) bodies are increasingly considering real-world data (RWD) for informing decision-making. The target trial emulation (TTE) framework has emerged as a promising approach to estimate treatment effects whilst minimizing common biases and methodological pitfalls in non-randomized studies. By applying design principles from randomised controlled trials, TTE explicitly ties the design and analysis of the non-randomised study to the target trial, facilitating the interpretation and communication of its underlying assumptions and study findings. In addition, TTE provides a structured approach for identifying potential limitations of RWD and the extent to which these affect the HTA decision.

The purpose of this workshop is to illustrate how the use of TTE can estimate per-protocol treatment effects when evidence comes primarily from a single-arm trial and an external (real-world) control arm. We will use three advanced non-small-cell lung cancer (aNSCLC) trials for illustrating the advantages of the TTE framework as well as major challenges.

DESCRIPTION:

Participants will be introduced to a broad overview of the target trial framework and the adoption of TTE for HTA. The workshop then will review design considerations and an application in a subset of aNSCLC trials.

Grace Hsu will chair the session. Manuel Gomes will provide a general introduction to TTE (10 min.). Sreeram Ramagopalan will illustrate how these methods have been applied to aNSCLC (10 min.). Stephen Duffield will outline the potential value of TTE from a HTA perspective (10 min.).

Audience participation will include engaging in a poll-driven question-and-answer period to identify potential issues and solutions for TTE scenarios (20 min.). This workshop would be valuable to researchers, industry analysts, and HTA representatives who are interested in trial emulation and analysis of RWD.