Overview of Drug Development

The pathologist is a vital partner in a team of pharmaceutical scientists in assuring that only safe and potentially efficacious new drug candidates are administered to human subjects. Pathologists play a key role in the selection and testing of new drug candidates, in decisions of whether to pursue or terminate research programs, in defining the potential hazards associated with new chemical entities (NCE), in defining how those hazards can be monitored in human subjects, and in translating those hazards into human risk assessments and the benefit : risk analyses critical to drug approval. While classically trained in disease diagnosis, pharmaceutical pathologists draw on their knowledge of the normal biology and morphology of body tissues and organs and their understanding of how these tissues can become perturbed during insult from disease, vascular disturbances, or toxins. Interpreting when tissue changes occur with respect to dose and systemic exposure and the reversibility of these changes is vital in defining margins between efficacious and toxic outcomes. While the pathologist is most critical in the description and interpretation of tissue damage during toxicology and carcinogenicity studies, the pathologist can also contribute to the entire preclinical development program for pharmaceutical candidates. These roles are briefly summarized in this chapter.