Session (CTI)

PURPOSE: The de-prioritisation of real-world evidence (RWE) in methodological guidelines for the newly European Union (EU) Health Technology Assessment (HTA) Regulation and the challenges in harmonising clinical evidence across diverse EU healthcare systems have been widely shared. Transportability analyses evaluating whether evidence from one context (e.g., population, setting, country) can be applied to another to answer external validity questions is not formally integrated in EU Joint Clinical Assessment (JCA), likely due to unclear RWE evidentiary standards and acceptance thresholds by Member States. Recent EU regulatory developments (e.g., European Healthcare Data Space) provide platforms for data-sharing and offer the opportunity for transportability analyses to be explored in EU JCAs. DESCRIPTION: This workshop will involve interactive, hypothetical EU JCA evidence generation, role play and submission planning. Dr Sarri will present a transportability framework developed by the International Society for Pharmacoepidemiology special interest group and describe its potential uses in EU JCAs (6minutes). Dr Adamson will present a hypothetical EU JCA case study, outlining how transportability analyses may be used alongside trial data to address gaps for a technology with complex evidence generation needs, borrowing learnings from non-EU HTA transportability analyses. Dr Remiro-Azócar will explore opportunities and challenges of using estimands and doubly robust covariate adjustment methodologies in JCAs. Dr Duffield will discuss the role of international data during National Institute for Health and Care Excellence assessments and where adjustments may add value in the context of decision-making. (13minutes each). Through audience participation, various analytical approaches will be explored to address evidence gaps using transportability analyses and their roadblocks. An HTA Coordination Group discussion will be simulated with emphasis on validating transported comparative effects. The workshop will conclude with an interactive discussion including live polling questions (15minutes). Statisticians, payers, industry and patient representatives will benefit.