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

abstract

Musings

John PA Ioannidis^1,2* and Muin J Khoury^3,4

• * Corresponding author: John PA Ioannidis jioannid@stanford.edu

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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."