Statistical Considerations On The Use Of RWD/RWE For Oncology Drug Approvals: Overview And Lessons Learned

By Sunhee K. Ro, Weidong Zhang, Qi Jiang, Xiaoyun Nicole Li, Rong Liu, Chengxing Cindy Lu, Olga Marchenko, Linda Sun, and Jing Zhao

Despite the growing adoption of real-world data (RWD) and real-world evidence (RWE) in regulatory submissions, their application in the context of oncology drug approvals has encountered notable challenges and limited success. Typically, RWD is employed as a benchmark control for single-arm studies or to complement concurrent controls in randomized clinical trials (RCTs). While considerable research has explored the utilization of RWD and RWE, our objective is to offer a comprehensive examination of their integration into oncology drug approval submissions, with the aim of guiding future RWD/RWE study design.

In this comprehensive overview, the authors explore various examples of RWD/RWE applications and provide an analysis of the strengths and weaknesses of these examples as perceived by regulatory agencies. Additionally, they delve into select case studies in greater detail to illustrate key insights and discuss operational aspects pertaining to RWD/RWE study design and analysis, enriching the discourse on their integration into the regulatory landscape of oncology drug approvals.