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Comment & Response |

Study Design and the Drug Development Process

Robert Goldberg, PhD1; Stuart Kauffman, MD, PhD2; Eric J. Topol, MD3
[+] Author Affiliations
1Center for Medicine in the Public Interest, Springfield, New Jersey
2Institute for Systems Biology, Seattle, Washington
3The Scripps Research Institute, La Jolla, California
JAMA. 2014;311(19):2023. doi:10.1001/jama.2014.3826.
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To the Editor A Viewpoint by Dr Djulbegovic and colleagues1 claimed not only that randomized clinical trials (RCTs) are more ethical but that greater use of randomized designs throughout the drug development process would “improve the efficiency, ie, enable faster development of new, successful treatments.”

However, RCTs are outdated for several reasons. First, RCTs are inadequate to evaluate cancer therapies. Genomic analysis is uncovering the tremendous heterogeneity of what previously were considered single diseases. Genomic analysis of cancers of individual patients is disclosing the large number of mutations, and thus targets, within one person. Developing RCTs for targeted therapies would be difficult and time-consuming, prolonging the wait for effective treatments.


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May 21, 2014
John P. A. Ioannidis, MD, DSc; Iztok Hozo, PhD; Benjamin Djulbegovic, MD, PhD
1Stanford Prevention Research Center, Stanford University, Stanford, California
2Department of Mathematics, Indiana University, Gary
3Division of Evidence-based Medicine, University of South Florida, Tampa
JAMA. 2014;311(19):2023-2024. doi:10.1001/jama.2014.3829.
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