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Keywords: RWD, RWE, Causal Inference, medical products, regulatory considerations, FDA
Surging number of available real-world data (RWD) sources (e.g., EHRs, claims data, registries) have attracted many experts from multiple disciplines to actively exploring and developing ways to translate RWD into Real-World Evidence (RWE) to bridge the current gaps in clinical trial enterprise. However, the statistical community can participate more fully in this novel research area. The ASA BIOP Section RWE Scientific Working Group was chartered in 2018 to use statistics generate RWE designed to inform regulatory decisions of medical products. At the end of its Phase I effort, the Working Group has published a paper on causal inference framework for RWE studies (Ho et al. 2021; DOI: 10.1080/19466315.2021.1883475). In this presentation, an hypothetical external controlled single arm oncology study will be used as an example to illustrate each steps of the Framework and details of individual steps will be discussed to highlight the essence of the Framework as a compass guiding RWE statisticians who astray outside the protection of double-blinded randomized clinical trials.