Pragmatic issues in biomarker evaluation for targeted therapies in cancer

Nat Rev Clin Oncol. 2015 Apr;12(4):197-212. doi: 10.1038/nrclinonc.2014.202. Epub 2014 Nov 25.


Predictive biomarkers are becoming increasingly important tools in drug development and clinical research. The importance of using both guidelines for specimen acquisition and analytical methods for biomarker measurements that are standardized has become recognized widely as an important issue, which must be addressed in order to provide high-quality, validated assays. Herein, we review the major challenges in biomarker validation processes, including pre-analytical (sample-related), analytical, and post-analytical (data-related) aspects of assay development. Recommendations for improving biomarker assay development and method validation are proposed to facilitate the use of predictive biomarkers in clinical trials and the practice of oncology.

Publication types

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

MeSH terms

  • Antineoplastic Agents / therapeutic use*
  • Biomarkers, Tumor / metabolism*
  • Drug Monitoring / methods
  • Humans
  • Molecular Targeted Therapy
  • Neoplasms / drug therapy*
  • Prognosis
  • Reproducibility of Results


  • Antineoplastic Agents
  • Biomarkers, Tumor