Target Trial Emulation in Practice: An Introduction and Exploration of Application Through Case Studies

PURPOSE: Target Trial Emulation (TTE) represents an advancement in HEOR by offering a rigorous approach to establish treatment effectiveness from observational data, thus overcoming the limitations of traditional real-world evidence (RWE) studies. This workshop will delve into how TTE addresses the critical challenge of bridging the gap between randomized controlled trials and RWE, enhancing the accuracy and applicability of healthcare interventions in diverse patient populations, including case studies involving target trial emulation applications in practice. Topics include (1) how to benchmark studies, (2) per-protocol estimation and what adherence means, (3) external comparators from RWE, and (4) data limitations, including unmeasured confounders and missing data problems

DESCRIPTION: Participants will gain insights into crucial TTE aspects, including benchmarking studies, per-protocol estimation and understanding adherence, leveraging real-world external comparators, and addressing missing data. Winson Cheung will introduce TTE, emphasizing its significance in bridging the gap between observational studies and randomized controlled trials. The discussion leaders will guide participants through real-world case studies illuminating the practical application of TTE. Manuel Gomes will describe the nuances of benchmarking in TTE and its role in ensuring robust study design and interpretation. He will also speak about the intricacies of per-protocol estimation and challenges in determining treatment adherence, with practical insights from relevant case studies. Alind Gupta will describe the use of external comparators in the context of target trial emulation, focusing on their calibration, application and interpretation. Dr Gupta will also address challenges related to data limitations, including unmeasured confounders and missing data. Audience participation will be encouraged through interactive discussions, where attendees can share their perspectives and experiences. We will tailor this workshop for researchers, statisticians, and practitioners interested in gaining practical insights into applying TTE in real-world scenarios.