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  • Review Article
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Pragmatic issues in biomarker evaluation for targeted therapies in cancer

Nature Reviews Clinical Oncology volume 12, pages 197–212 (2015)Cite this article

Subjects

Key Points

  • Predictive biomarkers are essential tools with regard to personalized medicine and health economics, and are crucial to improve the success rate of new therapies

  • Implementation of biomarkers into clinical practice presents biological, clinical and logistical challenges, in particular, relating to standardization across multiple countries and clinical practices

  • During biomarker development, robust laboratory methodology is necessary at all analytical phases, from pre-analytical (sample definition, handling and processing) to analytical (data and quality-control recording) and post-analytical (data reporting and interpretation)

  • A series of recommendations can be made to increase biomarker reliability and facilitate development of predictive biomarkers that can ultimately be used to provide benefit for patients with cancer

Abstract

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.

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Acknowledgements

The work of the authors has been supported by the Aide et Recherche en Cancérologie Digestive (ARCAD) foundation, a not-for-profit organization, and editorial assistance was provided by M. Benetkiewicz.

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Authors and Affiliations

  1. Department of Oncology, New Drug Evaluation Laboratory, Centre of Experimental Therapeutics, Centre Hospitalier Universitaire Vaudois (CHUV), Lausanne, 1011, Switzerland

    Armand de Gramont

  2. INSERM U830, Genetics and Biology of Paediatric Tumours Group, Institut Curie, France

    Sarah Watson

  3. Departments of Surgical Oncology, and Molecular and Cellular Oncology, University of Texas MD Anderson Cancer Center, USA

    Lee M. Ellis

  4. Division of Pathology and Laboratory Medicine, University of Texas MD Anderson Cancer Center, USA

    Stanley R. Hamilton

  5. Medical Oncology, Vall d'Hebron University Hospital, Vall d'Hebron Institute of Oncology (VHIO) and Universitat Autonoma de Barcelona (UAB), Spain

    Jordi Rodón & Josep Tabernero

  6. Medical Oncology Department, Institut Hospitalier Franco-Britannique, France

    Aimery de Gramont

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Contributions

Armand de Gramont, S.W., Aimery de Gramont and S.R.H. researched the data from the article; Armand de Gramont, L.M.E., J.R., J.T. Aimery de Gramont and S.R.H. contributed substantially to writing the article; and all authors contributed to discussion of content and review/editing of the manuscript before submission.

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de Gramont, A., Watson, S., Ellis, L. et al. Pragmatic issues in biomarker evaluation for targeted therapies in cancer. Nat Rev Clin Oncol 12, 197–212 (2015). https://doi.org/10.1038/nrclinonc.2014.202

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