Azam F., Phillips R., Newport R., Johnson R., Midgley R., Kerr DJ.
Early clinical trials are pivotal steps in the humanization of a drug development project. Their design has to accomplish both regulatory aspects and scientific gain regarding the drug target, human mechanism of action, and viable biomarkers; they should ultimately support proof-of-concept (PoC) as early as possible. This chapter details the basic characteristics of such trials and shows the opportunities elicited by smart designs of exploratory trials, exploratory investigational new drug filing, microdosing studies, early decision making, adaptive trial design, and the combination of regulatory and exploratory trials. All this aims at accelerating PoC as a major value inflection point of drug development.