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Comment & Response
May 21, 2014

Study Design and the Drug Development Process

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

Copyright 2014 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA. 2014;311(19):2023. doi:10.1001/jama.2014.3826

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However, RCTs are outdated for several reasons.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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