Keeping translational research grounded in human biology

Developing new and effective medicines is perhaps one of the most important, yet difficult endeavors that we pursue as a society. As a drug developer, I am part of a team wrestling with a daunting task - picking the most promising drug targets from the myriad biological pathways in the human body, designing drug molecules that can be administered to patients to modify these targets in a manner that impacts disease with an acceptable safety profile, and demonstrating the efficacy and value of these medicines in clinical trials. These hurdles contribute to the extremely low success rate of drug development programs progressing from target identification to approval (1). Increasingly, drug developers recognize that the most important consideration for improving the chance of success is to integrate data on human diversity (genomics, transcriptomics, proteomics, and other forms of molecular and phenotypic data) (2), from target nomination through all subsequent stages of drug development.