From Data to Decisions: The Potential of RWE Transportability in Healthcare Decision Making

PURPOSE: Health Technology Assessment (HTA) bodies increasingly consider supplementary evidence from real-world data (RWD) alongside traditional data sources like RCTs. However there remain concerns about bias and the applicability of RWD from other countries to the local settings. Further complicated by the uneven availability of high-quality RWD across different geographical regions. This workshop will discuss how transportability analysis, like methods used to adjust for confounding factors, can help bridge this gap. Recently NICE updated their real-world evidence framework to include transportability methods as a potential approach to adjust for external validity concerns.

DESCRIPTION: Workshop attendees will hear from different stakeholders across the industry including decision makers on how transportability methods can address the lack of quality local data within an HTA submission. The workshop will a) present a transportability framework and methods, and current stakeholder guidance and b) address how transportability methods can be applied in practice. Philani Mpofu will open the workshop with a brief introduction on the concepts (5 min). Alexandra Starry will draw learnings from previous NICE and G-BA appraisals and introduce a transportability analysis framework (15 min). Marian Eberl will discuss challenges from the industry perspective to address requirements for local data and present a pilot transportability analysis in lung cancer using US and German data as an example explaining how the study was adapted to the previously outlined framework (15 min). Stephen J. Duffield will discuss key considerations for the acceptability of international data and transportability analysis approaches from a decision maker perspective (10 min). Then Philani Mpofu will lead the audience participation, by walking them through a hypothetical case study, asking them to identify differences that should be considered across a multi-country transportability project (15 min). This interactive and informative workshop will inform researchers and industry analysts on how to design their own transportability study.