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Open Access Commentary

Statistical challenges in the development and evaluation of marker-based clinical tests

Lisa M McShane

Author Affiliations

Biometric Research Branch and Cancer Diagnosis Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, 6130 Executive Boulevard, EPN 8126, Bethesda, MD 20892-7434, USA

BMC Medicine 2012, 10:52  doi:10.1186/1741-7015-10-52

Published: 29 May 2012


Exciting new technologies for assessing markers in human specimens are now available to evaluate unprecedented types and numbers of variations in DNA, RNA, proteins, or biological structures such as chromosomes. These markers, whether viewed individually, or collectively as a 'signature', have the potential to be useful for disease risk assessment, screening, early detection, prognosis, therapy selection, and monitoring for therapy effectiveness or disease recurrence. Successful translation from basic research findings to clinically useful test requires basic, translational, and regulatory sciences and a collaborative effort among individuals with varied types of expertise including laboratory scientists, technology developers, clinicians, statisticians, and bioinformaticians. The focus of this commentary is the many statistical challenges in translational marker research, specifically in the development and validation of marker-based tests that have clinical utility for therapeutic decision-making.

Marker; biomarker; biostatistics; prognostic; predictive; treatment effect modifier; clinical test; translational research