Pre-Clinical Testing

What Is Pre-Clinical Assessment of Drug Efficacy and Safety?

Pre-clinical studies are intended to provide a key resource for risk versus benefit assessment prior to phase 1 clinical trials. An efficacy study is designed to illustrate the benefit (i.e., its clinical promise) of a pre-clinical drug candidate, while the safety study is designed to evaluate the risk associated with the pre-clinical drug candidate. Usually, both in vitro (cell-based) and in vivo (animal-based) studies are performed to comprehensively assess the efficacy and safety of a pre-clinical drug candidate. However, passing the pre-clinical assessment does not warrant the efficacy and safety in clinical study, partly due to the use of inappropriate pre-clinical models and inter-species differences. Therefore, the careful selection and use of multiple cell and animal models is critical to reduce the chances of a pre-clinical drug candidate failing when it reaches clinical trials.

Learning Objectives

• Appreciate the use of in vitro and in vivo models in pre-clinical studies
• Describe some basic techniques used in pre-clinical assessment
• Understand the limitation of pre-clinical data in clinical translation