Robust and flexible statistical methods for clinical trials with innovative and practical designs

Abstract

Randomized clinical trials (RCTs) are widely viewed as the gold standard study design for determining the efficacy of health interventions. Given that RCTs are time consuming and resource intensive, efficiency is of paramount concern. However, it is crucial to ensure that in the design, conduct, and analysis of clinical trials, expediency does not come at the expense of statistical rigor. In this dissertation, we take a careful look at trial designs that aim to be efficient and practical. The main through line for all of our projects is to propose modifications to the design, conduct, and/or analysis of these trials so that they can still reliably answer their intended questions. We are motivated by real problems encountered in real trials with commonly-used designs. We prioritize solutions to these problems that are easy to implement and understand. We justify our proposals with comprehensive asymptotic theory and practical simulation studies.