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Abstract:
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Real-world data (RWD) and real-world evidence (RWE) have traditionally been used in medical product development. Since the passage of the 21st Century Cures Act in 2016, the use of RWD and RWE in product development and regulatory decision has received increased attention. Stein (2019) provides a wide spectrum on the use of RWD and RWE at different stages of clinical trials with different study designs. Recently, the US Food and Drug Administration issued several guidance documents on the use of RWD and RWE to support regulatory decision-making for drugs and biologics. This IOL will provide an overview on the interface between randomized controlled trials (RCTs) and real-world studies (RWSs), offering a systematic, integrated viewpoint on the use of RWD and RWE in precision medicine development. Specifically, this lecture will provide (1) an overview of traditional use of RWD and RWE in clinical trials, (2) points-to-consider in defining estimands for studies that use RWD and RWE, (3) assessment of fit-purpose RWD for regulatory purpose, (4) causal inference frameworks including counterfactual framework, target trials, and integration of data from various sources, (5) a roadmap of using RWD and RWE for product development, and (6) challenges and opportunities. Illustrative examples are given throughout the lecture.
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