Translation of new therapies: From bench to bedside

The process of drug development is often long and complex, often beginning at the bedside. This is related to the fact that the strongest evidence supporting the existence of a drug target in the human is the ability to demonstrate alteration of the target in the setting of disease relative to the healthy condition. The vast majority of compounds developed and tested die in the preclinical phase of drug development. A molecule targeting a pathway entering the clinic often has well over a decade of study behind it including work in academic and industry laboratories and even those which survive to enter Phase 1 human trials have a failure rate of over 90%. This chapter discusses the current process of drug development in rheumatology and immunology, including target and pathway identification.