Pre-Clinical Testing → Limitation of Pre-Clinical Studies

An example of in vivo study for efficacy – animal behavioural studies

Remember, the success of a drug in pre-clinical studies does not warrant its performance in clinical trials, which could be attributed to inappropriate models used (e.g., an animal model does not fully represent a disease pathology) and inter-species differences (i.e., the efficacy of a candidate drug in mouse may not be translated to that in human). Approximately 95% of drugs that look promising during pre-clinical testing stage fails Phase 1 clinical trial (fail to demonstrate safety), from which 90% fails Phase 2 clinical trial (fail to demonstrate efficacy). Translation of data from pre-clinical studies to justify any clinical trial must be carefully pondered in order to minimize the risk of clinical trial withdrawal or failure.


Figure source: https://www.nature.com/articles/nrd3630/figures/2)