Session (CTI)

Issue: Although randomised controlled trials (RCTs) are the gold standard for assessing treatment efficacy and safety, certain conditions make it difficult to conduct adequately powered trials. In these cases, single-arm trials (SATs) and non-randomised studies are often pursued. However, their lack of a control group limits causal inference. External control (EC) groups are increasingly used to generate comparative evidence using real-world data (RWD) when RCTs are impractical or unethical. However, it is unclear to what extent these EC studies are fit for purpose. Overview: The GetReal Institute developed an External Comparator Best Practices Framework to help decision-makers determine when to consider an EC study, what requirements need to be met and how to design and execute such a study. This panel will discuss whether the framework can support regulatory decision-making and health technology assessment (HTA) by providing structured guidance on data selection, study design, statistical analysis, and regulatory engagement. Each panelist will offer a critical perspective on the framework over the course of 10 minutes each, considering what is required to ensure broad adoption across stakeholder groups. The session will close with a Q&A for 15-20 mins. People from Industry, Regulatory, HTA and RWD research backgrounds would benefit from attending this session.