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Are randomized trials obsolete or more important than ever in the genomic era?

John PA Ioannidis12* and Muin J Khoury34

Author Affiliations

1 Stanford Prevention Research Center, Department of Medicine and Department of Health Research and Policy, Stanford University School of Medicine, 1265 Welch Road, MSOB X306, Stanford, CA 94305, USA

2 Department of Statistics, Stanford University School of Humanities and Sciences, Stanford, CA 94305, USA

3 Office of Public Health Genomics, Centers for Disease Control and Prevention, Atlanta, GA 30333, USA

4 Epidemiology and Genomics Research Program, Division of Cancer Control and Population Sciences, National Cancer Institute, National Institutes of Health, Rockville, MD 20892, USA

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Genome Medicine 2013, 5:32  doi:10.1186/gm436

Published: 18 April 2013

First paragraph (this article has no abstract)

The genomic era has raised the possibility of major changes in the design, conduct, and even the existence of randomized trials as we know them [1-3]. Randomized trials are often seen as a slow, laborious, expensive, and difficult step in the translational process and are associated with a high attrition rate for drugs. Indeed, most tests that are in use for the screening, diagnosis, prognosis, monitoring or management of patients have never been scrutinized by a randomized trial. This has largely been due to a failure to realize that tests can do as much harm and as much good as drugs or devices; thus, a rigorous appraisal of their clinical utility, including both the possible benefits and the possible harms, is necessary. Moreover, numerous new omics-based tests are continually being proposed, especially in the context of targeted preventive or therapeutic interventions. Given rapid development of these new biomarkers, can we make randomized trials more adaptable to a changing landscape? Furthermore, do we still need randomized trials at all? Our answers to these two questions are: yes, to some extent; and yes, definitely. We will explain our reasoning in this article.