Basic principles of non-clinical development

8. Non-clinical results that can stop the development

A key goal of non-clinical studies is to discover target organ toxicity. The development of the medicinal product candidate can either be stopped based on this information, or the information can be used to monitor possible toxicities in human studies.

Non-clinical results (outcomes) can stop the development of a new medicine if:
  • A development candidate is found toxic in a target organ in animals, e.g. if it is hepatotoxic (toxic for the liver).
  • Further development may be questioned. The development may be stopped or possible toxicities will be monitored in clinical trials (although the predictive value of animal studies may still be questioned).
  • Poor pharmacokinetic (PK) properties have been found. An example is if a product does not get to its target (poor bioavailability). Other examples are if the medicine builds up, or causes toxicity, or if so-called toxic metabolites (substances that are the result of metabolic processes in the body) are produced.

This also explains why early ADME studies are performed. They can help researchers optimise selection of successful product candidates.

Animal studies that are not designed or reported well may produce bias in the research record. Also, there is a risk that researchers may initiate clinical trials when this is not appropriate. Or they may fail to protect humans from toxic drugs.