Synthetic and External Controls in Clinical Trials - A Primer for Researchers

Kristian Thorlund; Louis Dron; Jay J H Park; Edward J Mills

doi:10.2147/CLEP.S242097

Synthetic and External Controls in Clinical Trials - A Primer for Researchers

Clin Epidemiol. 2020 May 8:12:457-467. doi: 10.2147/CLEP.S242097. eCollection 2020.

Authors

Kristian Thorlund^{1

2}, Louis Dron², Jay J H Park^{2

3}, Edward J Mills^{1

2}

Affiliations

¹ Department of Health Research Methods, Evidence & Impact (HEI), McMaster University, Hamilton, ON, Canada.
² MTEK Sciences, Vancouver, BC, Canada.
³ Department of Medicine, University of British Columbia, Vancouver, BC, Canada.

Abstract

There has been a rapid expansion in the use of non-randomized evidence in the regulatory approval of treatments globally. An emerging set of methodologies have been utilized to provide greater insight into external control data used for these purposes, collectively known as synthetic control methods. Through this paper, we provide the reader with a set of key questions to help assess the quality of literature publications utilizing synthetic control methodologies. Common challenges and real-life examples of synthetic controls are provided throughout, alongside a critical appraisal framework with which to assess future publications.

Keywords: RCTs; real-world evidence; synthetic control.

Publication types

Review

Grants and funding

This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.