BACKGROUND: Real world evidence (RWE) data can be leveraged for counterfactual prediction when appropriate design and analytic methods are applied. Target trial emulation is the state-of-the-art framework to this effect and has been increasingly applied in studies as part of post approval safety and effectiveness requirements, commitments, and evaluations. This symposium showcases the experience and challenges encountered while applying the target trial emulation framework in regulatory grade pharmacoepidemiology/RWE studies by research groups and multi-country consortia (SIGMA Consortium) and others. Studies using target trial emulation are increasingly applied in the pre-approval phases. To integrate the point of view of the regulator in the adoption of novel methodology for real-world studies
OBJECTIVES:
To present the challenge of evaluating the safety of healthcare interventions that are sustained over time by: Elaborating on the implications of considering a healthcare intervention as a single-time exposure vs. an exposure that is sustained over time. Explaining how the approach of target trial emulation can be applied for this purpose
To present an actual regulatory application of the target trial emulation in the evaluation of the safety of a cancer drug (EUPAS33448), with a focus on the following: The importance of specifying treatment strategies that comply with the “consistency” identifying assumption. The challenge of defining time zero when studying a cancer drug that is indicated for several lines of treatment.
To propose solutions to computational and statistical challenges that can arise when emulating a target trial. This session will share and discuss open-source solutions, including simulations and practical examples. This symposium is aimed to researchers who work with RWD, particularly in studies using databases from diverse countries to estimate the safety of healthcare interventions for regulatory purposes.
