Fit-for-purpose biomarker method validation in anticancer drug development

Drug Discov Today. 2010 Oct;15(19-20):816-25. doi: 10.1016/j.drudis.2010.07.006. Epub 2010 Aug 11.


The introduction of new anticancer drugs into the clinic is often hampered by a lack of qualified biomarkers. Method validation is indispensable to successful biomarker qualification and is also a regulatory requirement. Recently, the fit-for-purpose approach has been developed to promote flexible yet rigorous biomarker method validation, although its full implications are often overlooked. This review aims to clarify many of the scientific and regulatory issues surrounding biomarker method validation and the analysis of samples collected from clinical trial subjects. It also strives to provide clear guidance on validation strategies for each of the five categories that define the majority of biomarker assays, citing specific examples.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Biomarkers, Pharmacological / analysis*
  • Clinical Trials as Topic
  • Drug Design
  • Humans


  • Antineoplastic Agents
  • Biomarkers, Pharmacological