Learning about the principles of clinical trials

Clinical trials are designed to address many of the problems we have in observational studies and provide medicine with the most trustworthy results possible. For this reason, clinical trials are designed as more or less controlled experiments.

Clinical trials have a specific primary outcome or outcomes of interest to evaluate. These outcomes are related to the main goal of the study. Some examples can be the safety or efficacy of the drug, side effects, or other specific clinical outcomes, such as the survival of patients, which are still in a way related to the efficacy of drugs.

There are four types of clinical trials—Phase I, Phase II, Phase III, and Phase IV. All these trials are part of the drug discovery and research pipeline and are closely connected to each other. While the initial phases are meant to mainly evaluate the drug safety profile, later phases, especially after Phase III, are mostly focused on the efficacy...