Animal Models for the Study of Human Disease

The use of conventional healthy animals (CHAs) has long been a mainstay of nonclinical safety testing as well as evaluation of efficacy of new chemical entities (NCEs) in the discovery setting. However, the potential value of directed animal models of human disease (AMDs) has increasingly been of value in evaluation of both disciplines. While more frequently employed in the evaluation of efficacy (discovery phase), there has been increasing interest in utilizing AMDs in investigation of the safety (development phase), particularly in the development of targeted NCEs that may not be appropriate for testing in CHAs. While it is accepted that no animal model (neither CHA nor AMD) will accurately predict all risks (or potential benefits) of an NCE, careful selection of the model with an understanding of the pros and cons of each model will optimize the results of investigations. This chapter will outline some of the major organ systems, human diseases, and associated AMDs along with considerations for their use.