Model Based vs. Rule Based Designs in Phase I Dose Finding Clinical Trials

Abstract

A phase I clinical trial is needed when a new drug or treatment is first used in human subjects. For ethnic reason, in such a trial, we have to carefully balance the possible benefit and harm so that the benefit is maximized and harm is minimized, which deems that the design is adaptive with small sample size, and is primarily focusing on safety end-points with efficacy end-points as secondary ones.