This module provides an overview of the clinical and scientific issues that must be considered by the many disciplines that collaborate on a clinical trial. In this module, we consider the clinical, scientific, and regulatory setting of clinical trials, describing the phased approach to “treatment discovery” in which the safety, efficacy, and effectiveness of candidate treatments is investigated.

In particular, we discuss the issues surrounding identification of the target population, definition of the treatment, choice of clinical outcomes, and choice of comparators. It will include introductory discussion of the choice of randomization strategies, blinding, conduct and monitoring of the study, and plans for reporting of the result. The importance of a well-defined study protocol is emphasized.