In March 2024, the European Commission ratified methodological guidelines specifying the statistical analyses for the new Joint Clinical Assessment (JCA). The guidelines address the inclusion of SATs and external controls through discussion on methods for analysing non-randomised data and disconnected networks.

However, the pre-conditions for the acceptance of non-randomised comparisons, which include pre-specification of the analysis methods, are not without their challenges, especially given the time available for the JCA. Moreover, such pre-conditions are likely to differ between EU member states.

With the new regulation taking effect in January 2025, there is an imperative to clarify the conditions under which the use of non-randomised evidence from SATs in benefit dossiers will be accepted. This session will serve as a pivotal platform for fostering dialogue between HTA bodies and the industry regarding expectations and achievable outcomes.

Mr. Scott will outline the current guidelines regarding the use of SATs and external controls, as well as offering insights into the ongoing discourse surrounding their acceptance. Dr. Jen, Dr. McConnell, and Dr. Ren will put forward their position regarding the conditions under which treatment comparisons from SATs using external controls can be relied upon for the purpose of providing evidence of additional benefit. The remainder of the session will be left for debate with the audience on the expectations and efforts required for HTA submissions, considering the challenges of clinical trial design, development costs, ethical considerations, and the imperative to expedite patient access to effective medications.