Real-World Evidence

RWE shapes regulatory and reimbursement decisions with growing FDA acceptance. The session covers tokenised RWE platforms (Datavant, Truveta), federated learning across health systems (TriNetX, Aetion, Owkin), the FDA RWE framework and 21st Century Cures implementation, Project Optimus dose-optimisation, synthetic control arms, and payer evidence requirements. Discussion addresses RWE for label expansion, the comparison of EHR vs claims data, NLP in RWE extraction, equity considerations in RWE source data, international acceptance (EMA, MHRA, PMDA), and emerging RWE for cell and gene therapy long-term follow-up.