Proof of Concept: Functional Models for Drug Development in Humans

A drug developed for human use classically goes through a number of steps, including discovery, extensive preclinical studies for safety in experimental animals, and then human safety and efficacy studies. The Food and Drug Administration (FDA) requires successful completion of all the above tasks before approval for therapeutic use in human beings. The exciting advances in human genomics and combinatorial chemistry promise substantial applications to new drugs for human diseases; however, the time and expense associated with the processes necessary to bring a new drug to market are rising exponentially. Thus, there is a great need for functional models of disease progression, including animal models of human disease and biomarkers in human clinical trials.