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Please use this identifier to cite or link to this item: http://hdl.handle.net/1942/24101

Title: Precision medicine needs randomized clinical trials
Authors: Saad, Everardo D.
Paoletti, Xavier
Burzykowski, Tomasz
Buyse, Marc
Issue Date: 2017
Citation: NATURE REVIEWS CLINICAL ONCOLOGY, 14(5), p. 317-323
Abstract: The advent of precision medicine has prompted profound changes in clinical cancer research, and the rising numbers of new therapeutic agents pose challenges in terms of the most appropriate trial designs and effects on the drug-approval process. In the past 5 years, some remarkably efficacious drugs have been approved based on evidence from uncontrolled phase I trials. We challenge the view that the expected benefits from new drugs are generally sufficient to forgo a randomized trial with patients assigned to a control arm (a regimen other than the experimental treatment). Relying on efficacy results from uncontrolled clinical trials can result in expedited drug approval, but the disadvantages of this practice must be taken into account. For example, the apparent improvements in outcomes observed in an early single-arm trial of a new therapy might reflect the prognostic nature of the target, rather than a true treatment effect. Moreover, the predictive role of biomarkers cannot be definitively ascertained without randomly assigning patients to a control arm. We discuss the need for such randomization to a true control in all phases of drug development and the role of companion biomarker testing. We propose that an increased use of randomization will facilitate a seamless transition between phases of drug and/or biomarker development.
Notes: [Saad, Everardo D.; Burzykowski, Tomasz] IDDI, Ave Provinciale 30, B-1340 Ottignies, Belgium. [Paoletti, Xavier] Inst Gustave Roussy, 114 Rue Edouard Vaillant, F-94800 Villejuif, France. [Burzykowski, Tomasz] Hasselt Univ, Interuniv Inst Biostat & Stat Bioinformat I Biost, Martelarenlaan 42, B-3500 Hasselt, Belgium. [Buyse, Marc] Hasselt Univ, I BioStat, Martelarenlaan 42, B-3500 Hasselt, Belgium. [Buyse, Marc] IDDI, 185 Alewife Brook Pkwy,Suite 410, Cambridge, MA 02138 USA.
URI: http://hdl.handle.net/1942/24101
DOI: 10.1038/nrclinonc.2017.8
ISI #: 000399452400009
ISSN: 1759-4774
Category: A1
Type: Journal Contribution
Appears in Collections: Research publications

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