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

Timothy A. Yap, Ira Jacobs, Elodie Baumfeld Andre, Lauren J. Lee, Darrin Beaupre, Laurent Azoulay

Research output: Contribution to journalReview articlepeer-review

18 Scopus citations

Abstract

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.

Original languageEnglish (US)
Article number695936
JournalFrontiers in Oncology
Volume11
DOIs
StatePublished - Jan 6 2022

Keywords

  • RCT
  • cancer
  • clinical trial
  • design
  • drug development
  • external control group
  • oncology
  • real-world data

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

Fingerprint

Dive into the research topics of 'Application of Real-World Data to External Control Groups in Oncology Clinical Trial Drug Development'. Together they form a unique fingerprint.

Cite this