Predictive Efficacy and Toxicology

Predicting the biological effects of drugs, chemicals and interventions is fraught with hazard. Many candidate drugs fail in human clinical trials because they are found to be unsafe or ineffective, despite promising preclinical studies in animal and cell models.

NCATS is developing model systems for drug and toxicity testing that more closely resemble human physiology and more accurately reflect how our bodies will react to an experimental compound. Such advances could save enormous amounts of time and expense by preventing people in clinical studies from being exposed to potentially harmful or ineffective candidate drugs. Scientists also can use these models to study the basic biology of disease, predict toxicity or other physiological processes, and evaluate environmental chemicals. The model systems support NIH’s position that non-animal model approaches can reduce the need for animals in research but will require further improvement to completely replace them.

Several NCATS programs and initiatives — including Assay Development and Screening TechnologiesEarly Translation BranchTissue Chip for Drug Screening and Toxicology in the 21st Century — are designed to address the translational issue of predictive efficacy and toxicology.

Learn more about other translational issues NCATS aims to address: