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Review
. 2023 Jul;57(4):899-910.
doi: 10.1007/s43441-023-00528-y. Epub 2023 May 13.

Statistical Considerations on the Use of RWD/RWE for Oncology Drug Approvals: Overview and Lessons Learned

Sunhee K Ro #  1 , Weidong Zhang #  2 , Qi Jiang  3 , Xiaoyun Nicole Li  4 , Rong Liu  5 , Chengxing Cindy Lu  6 , Olga Marchenko  7 , Linda Sun  8 , Jing Zhao  8
Affiliations
Review

Statistical Considerations on the Use of RWD/RWE for Oncology Drug Approvals: Overview and Lessons Learned

Sunhee K Ro et al. Ther Innov Regul Sci. 2023 Jul.

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.

Keywords: Clinical study design; Control arm augmentation; Oncology drug approval; Real world data; Real world evidence.

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Conflict of interest statement

Sunhee K. Ro is an employee of Sierra/GSK and owns stocks of BeiGene and Amgen. Weidong Zhang is an employee of Sana Biotechnology and owns stocks of Sana Biotechnology and Pfizer, Inc. Qi Jiang is an employee of Seagen and owns stocks. Xiaoyun Nicole Li is an employee of BeiGene and owns stock of BeiGene and Merck. Rong Liu is an employee of BMS and owns stocks. Cindy Chengxing Lu is an employee of AstraZeneca and owns stocks. Olga Marchenko is an employee of Bayer. Linda Sun is an employee of Merck and owns stocks. Jing Zhao is an employee of Merck.

References

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