Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development

Frontiers in Oncology

Laurent Azoulay²  Timothy A. Yap³  Lauren J. Lee⁴  Elodie Baumfeld Andre⁴  Ira Jacobs⁴  Darrin Beaupre⁴ 

[1] Centre for Clinical Epidemiology Lady Davis Institute, Jewish General Hospital, Montreal, QC, Canada;Department of Epidemiology, Biostatistics, and Occupational Health and Gerald Bronfman Department of Oncology, McGill University, Montreal, QC, Canada;Department of Investigational Cancer Therapeutics (Phase I Program), Division of Cancer Medicine, the University of Texas MD Anderson Cancer Center, Houston, TX, United States;Pfizer Inc., New York, NY, United States;

关键词: cancer;    clinical trial;    design;    drug development;    external control group;    oncology;   

DOI  :  10.3389/fonc.2021.695936

来源: DOAJ

【 摘 要 】

Randomized controlled trials (RCTs) that assess overall survival are considered the “gold standard” when evaluating the efficacy and safety of a new oncology intervention. However, single-arm trials that use surrogate endpoints (e.g., objective response rate or duration of response) to evaluate clinical benefit have become the basis for accelerated or breakthrough regulatory approval of precision oncology drugs for cases where the target and research populations are relatively small. Interpretation of efficacy in single-arm trials can be challenging because such studies lack a standard-of-care comparator arm. Although an external control group can be based on data from other clinical trials, using an external control group based on data collected outside of a trial may not only offer an alternative to both RCTs and uncontrolled single-arm trials, but it may also help improve decision-making by study sponsors or regulatory authorities. Hence, leveraging real-world data (RWD) to construct external control arms in clinical trials that investigate the efficacy and safety of drug interventions in oncology has become a topic of interest. Herein, we review the benefits and challenges associated with the use of RWD to construct external control groups, and the relevance of RWD to early oncology drug development.

【 授权许可】

Unknown