Personalized medicine is a growing area of research which aims to tailor the treatment given to a patient according to one or more personal characteristics. These characteristics can be demographic such as age or gender, or biological such as a genetic or other biomarker.
Prior to utilizing a patient’s biomarker information in clinical practice, robust testing in terms of analytical validity, clinical validity and clinical utility is necessary. A number of clinical trial designs have been proposed for testing a biomarker’s clinical utility, including Phase II and Phase III clinical trials which aim to test the effectiveness of a biomarker-guided approach to treatment; these designs can be broadly classified into adaptive and non-adaptive. While adaptive designs allow planned modifications based on accumulating information during a trial, non-adaptive designs are typically simpler but less flexible.
Antoniou et al, as members of the MRC Hubs for Trials Methodology Research’s Stratified Medicine Working Group, have undertaken a comprehensive review of biomarker-guided trial designs based on an in-depth search strategy which identified 211 relevant papers, and the results of the review have been published in two separate papers, one focusing on adaptive trial designs and the other on non-adaptive trial designs. On this website, each of the trial designs identified in the review is represented graphically together with an overview of its key characteristics, methodology, and its pros and cons.