Journal of the National Cancer Institute Advance Access published online on October 7, 2008
JNCI Journal of the National Cancer Institute, doi:10.1093/jnci/djn326
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COMMENTARY |
Pivotal Evaluation of the Accuracy of a Biomarker Used for Classification or Prediction: Standards for Study Design
Affiliations of authors: Division of Public Health Sciences, Fred Hutchinson Cancer Research Center, Seattle, WA (MSP, ZF, HJ, JDP); Department of Clinical Epidemiology, Academic Medical Center, Amsterdam, The Netherlands (PMB)
Correspondence to: Margaret S. Pepe, PhD, Program in Biostatistics and Biomathematics, Fred Hutchinson Cancer Research Center, 1100 Fairview Ave N, M2-B500, Seattle, WA 98109-1024 (e-mail: mspepe{at}u.washington.edu).
Research methods for biomarker evaluation lag behind those for evaluating therapeutic treatments. Although a phased approach to development of biomarkers exists and guidelines are available for reporting study results, a coherent and comprehensive set of guidelines for study design has not been delineated. We describe a nested case–control study design that involves prospective collection of specimens before outcome ascertainment from a study cohort that is relevant to the clinical application. The biomarker is assayed in a blinded fashion on specimens from randomly selected case patients and control subjects in the study cohort. We separately describe aspects of the design that relate to the clinical context, biomarker performance criteria, the biomarker test, and study size. The design can be applied to studies of biomarkers intended for use in disease diagnosis, screening, or prognosis. Common biases that pervade the biomarker research literature would be eliminated if these rigorous standards were followed.
Manuscript received December 19, 2007; revised August 8, 2008; accepted August 11, 2008.
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J Natl Cancer Inst 2008 100: 1419-1420.
J Natl Cancer Inst 2008 100: 1417.
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