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Updated: Jul 30, 2025

Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System
Published on: December 11, 2016
Statistical Considerations on the Use of RWD/RWE for Oncology Drug Approvals: Overview and Lessons Learned
Sunhee K Ro1, Weidong Zhang2, Qi Jiang3
1Sierra Oncology Inc: GlaxoSmithKline Inc, San Mateo, USA. skwonro@gmail.com.
Abstract:
Despite increasing utilization of real-world data (RWD)/real-world evidence (RWE) in regulatory submissions, their application to oncology drug approvals has seen limited success. Real-world data is most commonly summarized as a benchmark control for a single arm study or used to augment the concurrent control in a randomized clinical trial (RCT). While there has been substantial research on usage of RWD/RWE, our goal is to provide a comprehensive overview of their use in oncology drug approval submissions to inform future RWD/RWE study design. We will review examples of applications and summarize the strengths and weaknesses of each example identified by regulatory agencies. A few noteworthy case studies will be reviewed in detail. Operational aspects of RWD/RWE study design/analysis will be also discussed.
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