Biomarker method validation in anticancer drug development

Br J Pharmacol. 2008 Feb;153(4):646-56. doi: 10.1038/sj.bjp.0707441. Epub 2007 Sep 17.

Abstract

Over recent years the role of biomarkers in anticancer drug development has expanded across a spectrum of applications ranging from research tool during early discovery to surrogate endpoint in the clinic. However, in Europe when biomarker measurements are performed on samples collected from subjects entered into clinical trials of new investigational agents, laboratories conducting these analyses become subject to the Clinical Trials Regulations. While these regulations are not specific in their requirements of research laboratories, quality assurance and in particular assay validation are essential. This review, therefore, focuses on a discussion of current thinking in biomarker assay validation. Five categories define the majority of biomarker assays from 'absolute quantitation' to 'categorical'. Validation must therefore take account of both the position of the biomarker in the spectrum towards clinical end point and the level of quantitation inherent in the methodology. Biomarker assay validation should be performed ideally in stages on 'a fit for purpose' basis avoiding unnecessarily dogmatic adherence to rigid guidelines but with careful monitoring of progress at the end of each stage. These principles are illustrated with two specific examples: (a) absolute quantitation of protein biomarkers by mass spectrometry and (b) the M30 and M65 ELISA assays as surrogate end points of cell death.

Publication types

  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology*
  • Antineoplastic Agents / therapeutic use
  • Biomarkers, Pharmacological / analysis*
  • Cell Death / drug effects
  • Clinical Trials as Topic / legislation & jurisprudence
  • Clinical Trials as Topic / methods*
  • Clinical Trials as Topic / standards
  • Drug Evaluation, Preclinical / methods*
  • Drug Evaluation, Preclinical / standards
  • Enzyme-Linked Immunosorbent Assay* / standards
  • Guideline Adherence
  • Guidelines as Topic
  • Humans
  • Keratin-18 / analysis
  • Laboratories* / legislation & jurisprudence
  • Laboratories* / standards
  • Mass Spectrometry* / standards
  • Proteins / analysis
  • Quality Control
  • Reproducibility of Results
  • Terminology as Topic

Substances

  • Antineoplastic Agents
  • Biomarkers, Pharmacological
  • Keratin-18
  • Proteins