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. 2021 Apr;16(4):537-545.
doi: 10.1016/j.jtho.2021.01.1616. Epub 2021 Feb 2.

Biomarker Discovery and Validation: Statistical Considerations

Fang-Shu Ou  1 , Stefan Michiels  2 , Yu Shyr  3 , Alex A Adjei  4 , Ann L Oberg  5
Affiliations

Biomarker Discovery and Validation: Statistical Considerations

Fang-Shu Ou et al. J Thorac Oncol. 2021 Apr.

Abstract

Biomarkers have various applications including disease detection, diagnosis, prognosis, prediction of response to intervention, and disease monitoring. In this era of precision medicine, having validated biomarkers to inform clinical decision making is more important than ever. In this article, we discuss best the practices and potential issues in biomarker discovery and validation. We encourage team science partnerships to bring cutting-edge discovery from bench to bedside, leading to improved patient care and outcomes.

Keywords: Biomarker; Clinical trial; Confirmation analysis; Exploratory analysis.

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Conflict of interest statement

CONFLICTS OF INTEREST:

FSO / SM / ALO/ AAA: None

Dr. Shyr reports personal fees from AstraZeneca, Eisai, Janssen, Novartis, Pfizer, Roche, outside the submitted work.

Figures

Figure 1.
Figure 1.
Use of biomarkers in relation to the course of disease
Figure 2.
Figure 2.. Simplified schematic of biomarker development
PRoBE: prospective-specimen-collection, retrospective-blinded-evaluation.
Figure 3.
Figure 3.
Trial design schema
See this image and copyright information in PMC

References

    1. Group F-NBW: BEST (Biomarkers, EndpointS, and other Tools) Resource, 2020 - PubMed
    1. National Comprehensive Cancer Network. Lung cancer screening. 2020. version. https://www.nccn.org/patients/guidelines/content/PDF/lung-early-stage-pa.... Accessed 04JAN2021.
    1. Nicholson AG, Sauter JL, Nowak AK, et al.: EURACAN/IASLC Proposals for Updating the Histologic Classification of Pleural Mesothelioma: Towards a More Multidisciplinary Approach. J Thorac Oncol 15:29–49, 2020 - PubMed
    1. Remon J, Ahn MJ, Girard N, et al.: Advanced-Stage Non-Small Cell Lung Cancer: Advances in Thoracic Oncology 2018. J Thorac Oncol 14:1134–1155, 2019 - PubMed
    1. Pepe MS, Etzioni R, Feng Z, et al.: Phases of biomarker development for early detection of cancer. J Natl Cancer Inst 93:1054–61, 2001 - PubMed

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