Clinical Epidemiology (May 2020)
Synthetic and External Controls in Clinical Trials – A Primer for Researchers
Abstract
Kristian Thorlund,1,2 Louis Dron,2 Jay JH Park,2,3 Edward J Mills1,2 1Department of Health Research Methods, Evidence & Impact (HEI), McMaster University, Hamilton, ON, Canada; 2MTEK Sciences, Vancouver, BC, Canada; 3Department of Medicine, University of British Columbia, Vancouver, BC, CanadaCorrespondence: Kristian ThorlundMTEK Sciences, 802-777 West Broadway, Vancouver, BC V5Z 1J5, CanadaEmail [email protected]: 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: synthetic control, RCTs, real-world evidence
