Presentation (CTI)

OBJECTIVES: To systematically identify, compare, and synthesise key recommendations from major global guidelines on the generation and use of real-world evidence (RWE) in regulatory and health technology assessment (HTA) decision-making.
METHODS: A targeted literature review was conducted using PubMed and Google Scholar to identify guidance documents on RWE. Search terms included “real-world evidence,” “real-world data,” “RWE,” “RWD,” and “guidelines.” Searches were supplemented by manual review of HTA and regulatory agency websites. Eligible documents included formal RWE guidance issued by regulatory bodies or HTA agencies. Extracted data were qualitatively synthesised to compare recommendations across domains, including data sources, study design, methodological rigour, and decision-making applications.
RESULTS: Five key guidelines were included: NICE (UK), HAS (France), CADTH (Canada), NMPA (China), and the REALISE framework. Across guidelines, RWD was consistently defined as routinely collected data from healthcare delivery, and RWE as evidence derived using observational or pragmatic study designs. All agencies recognise the growing role of RWE, particularly in:

• Characterising disease epidemiology and treatment pathways
• Assessing comparative effectiveness and safety
• Supporting economic evaluations and reimbursement decisions

However, differences were observed in evidentiary thresholds, methodological expectations, and acceptable study designs. NICE and CADTH emphasise transparency, protocol pre-specification, and causal inference methods, while NMPA places a stronger focus on supporting regulatory approvals. The REALISE framework provides cross-cutting methodological recommendations aimed at improving study validity and reproducibility.
CONCLUSIONS: Global RWE guidelines show broad alignment in recognising the importance of RWE but vary in methodological expectations and decision contexts. Harmonisation of standards and increased emphasis on data quality, transparency, and analytical rigour will be critical to maximise the acceptance and impact of RWE in HTA and regulatory decision-making.