Principles of precision medicine and its application in toxicology

J Toxicol Sci. 2018;43(10):565-577. doi: 10.2131/jts.43.565.

Abstract

Precision medicine is an approach to developing drugs that focuses on employing biomarkers to stratify patients in clinical trials with the goal of improving efficacy and/or safety outcomes, ultimately increasing the odds of clinical success and drug approval. Precision medicine is an important tool for toxicologists to utilize, because its principles can be used to decide whether to pursue a drug target, to understand interindividual differences in response to drugs in both nonclinical and clinical settings, to aid in selecting doses that optimize efficacy or reduce adverse events, and to facilitate understanding of a drug's mode-of-action. Nonclinical models such as the mouse and non-human primate can be used to understand genetic variation and its potential translation to humans, and are available for toxicologists to employ in advance of drugs moving into clinical development. Understanding interindividual differences in response to drugs and how these differences can influence the drug's risk-benefit profile and lead to the identification of biomarkers that enhance patient efficacy and safety is of critical importance for toxicologists today, and in the future, as the fields of pharmacogenomics and genetics continue to advance.

Keywords: Interindividual variability; Pharmacogenomics; Precision medicine; Review; Safety assessment.

Publication types

  • Review

MeSH terms

  • Animals
  • Drug Delivery Systems
  • Drug Discovery
  • Genetic Variation
  • Humans
  • Mice
  • Pharmacogenetics / trends
  • Precision Medicine* / trends
  • Risk Assessment
  • Toxicology*