Novel and rediscovered trial designs: should we embrace or avoid them?
Randomized controlled trial (RCT) has long been the gold standard for clinical research, representing the best way to determine effectiveness of different interventions. However, this commonly used design may not be the best practical option for answering various research questions in many clinical situations. Recent years have seen a strong increase in the number of studies using pragmatic alternatives to the standard randomized controlled trial design.
In this tutorial by Dr. Peter van den Ven, a (bio)statistician at the Department of Epidemiology and Data Science at the University of Amsterdam, you learn about alternative trial designs, their strengths and limitations. He started the tutorial with introducing some of these new alternative designs. The stepped-wedge trial design is often used as an alternative to the cluster-randomized trial because the willingness among the clusters to participate is considered higher. The trial within cohorts (TwiCs) design avoids attrition in the control arm by not explicitly informing subjects randomized to the control arm about them being part of the trial population. Series of N-of-1 trials reduce the total number of patients needed by alternating experimental and control treatment within patients. In this tutorial I will discuss pros and cons of these designs focusing both on the practical aspects and methodological consequences.
