Presentation (CTI)

OBJECTIVES: Transportability asks whether findings can be extended to a target population that is partially or entirely distinct from the study population — for example, using US real-world data (RWD) to inform a UK health technology assessment (HTA) decision. Methods used to adjust for factors that differ between the source and target populations and that influence treatment outcomes include weighting, outcome-regression and doubly-robust methods that combine both. This review assessed HTA guidance across multiple jurisdictions to evaluate i) the acceptability of transportability analyses and ii) the extent to which methodological recommendations are provided.
METHODS: Methods guidance from NICE (UK), HAS (France), IQWIG (Germany), CADTH (Canada), and ICER (USA) was reviewed. For each HTA body, we assessed whether guidance explicitly addresses transportability of non-local data, whether such evidence is considered acceptable, and whether recommendations are provided for conducting or reporting transportability analyses. Recommendations of interest included discussion of key transportability assumptions (consistency, positivity and conditional exchangeability), assessment of assumption violations, and guidance on appropriate statistical methods.
RESULTS: Across the five HTA bodies reviewed, only NICE and CADTH explicitly accept use of non-local data and acknowledge the need to assess its external validity. HAS, IQWiG, and ICER discuss the use of non-local trial data or RWD, but generally prioritise evidence generated within their own jurisdictions. While none of the agencies provide detailed methodological guidance for transportability analyses, several provide some guidance that implicitly aligns with transportability principles.
CONCLUSIONS: As use of RWE in HTA submissions continues to expand, methods for transporting non-local RWD will become increasingly important. Developing clear methodological and reporting guidance for transportability analyses may improve their acceptability and support efforts to address evidence gaps in HTA decision-making.