Impact of biomarker development on drug safety assessment

Toxicol Appl Pharmacol. 2010 Mar 1;243(2):167-79. doi: 10.1016/j.taap.2009.12.015. Epub 2009 Dec 28.


Drug safety has always been a key aspect of drug development. Recently, the Vioxx case and several cases of serious adverse events being linked to high-profile products have increased the importance of drug safety, especially in the eyes of drug development companies and global regulatory agencies. Safety biomarkers are increasingly being seen as helping to provide the clarity, predictability, and certainty needed to gain confidence in decision making: early-stage projects can be stopped quicker, late-stage projects become less risky. Public and private organizations are investing heavily in terms of time, money and manpower on safety biomarker development. An illustrative and "door opening" safety biomarker success story is the recent recognition of kidney safety biomarkers for pre-clinical and limited translational contexts by FDA and EMEA. This milestone achieved for kidney biomarkers and the "know how" acquired is being transferred to other organ toxicities, namely liver, heart, vascular system. New technologies and molecular-based approaches, i.e., molecular pathology as a complement to the classical toolbox, allow promising discoveries in the safety biomarker field. This review will focus on the utility and use of safety biomarkers all along drug development, highlighting the present gaps and opportunities identified in organ toxicity monitoring. A last part will be dedicated to safety biomarker development in general, from identification to diagnostic tests, using the kidney safety biomarkers success as an illustrative example.

Publication types

  • Review

MeSH terms

  • Animals
  • Biomarkers*
  • Chemical and Drug Induced Liver Injury / diagnosis
  • Chemical and Drug Induced Liver Injury / metabolism
  • Drug-Related Side Effects and Adverse Reactions*
  • Heart Diseases / chemically induced
  • Heart Diseases / diagnosis
  • Heart Diseases / metabolism
  • Humans
  • Kidney Diseases / chemically induced
  • Kidney Diseases / diagnosis
  • Kidney Diseases / metabolism
  • Safety
  • Vascular Diseases / chemically induced
  • Vascular Diseases / diagnosis
  • Vascular Diseases / metabolism


  • Biomarkers