Basics of Designing General Toxicology Studies

Drug development is a complex process and is tailored specifically to a given therapeutic indication, the target patient population, the therapeutic platform/s of choice (small molecules/biotherapeutics/gene therapy) and the route of administration. Non-clinical toxicology studies are critical to establishing the safety of new pharmaceutical candidates to enable first in human (FIH) clinical studies, to support the continued progression through phases of clinical development and finally for the registration with regulatory authorities to support a marketing application. Well designed and executed toxicology studies thus enable a smooth transition of new molecular entities through milestones of clinical development. This chapter aims to explain the general scientific principles and key considerations in the basic design of repeat-dose toxicology studies with a focus on small molecules and the exceptions applicable to study designs for biotherapeutics and gene therapies.