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January 22/29, 2014

Improving the Drug Development ProcessMore Not Less Randomized Trials

Author Affiliations
  • 1Division of Evidence-Based Medicine, Department of Internal Medicine; and Departments of Hematology and Health Outcome Behavior, H. Lee Mofitt Cancer Center and Research Institute, University of South Florida, Tampa
  • 2Department of Mathematics, Indiana University, Gary
  • 3Stanford Prevention Research Center, Department of Medicine, and Department of Health Research and Policy, Stanford University School of Medicine; Department of Statistics, Stanford University School of Humanities and Sciences; and Meta-Research Innovation Center at Stanford, Stanford, California
JAMA. 2014;311(4):355-356. doi:10.1001/jama.2013.283742

Drug development is often a lengthy and expensive process. Extensive preclinical testing via in vitro and animal experimentation aims to select drugs most likely to work in humans. Under the current system, only about half of the drugs succeed in moving from phase 1 (dose-finding) to phase 2 (safety and efficacy).1 For drugs that enter phase 2, less than 1 in 3 succeed; for those entering phase 3 (pivotal efficacy), that number decreases to less than 1 in 2.1,2 Less than 20% of drugs entering phase 1 testing successfully reach the end of the 3-phase evaluation. The percentage can vary from one specialty area to another, and it can be less than 5% to 10% for oncologic and neurologic diseases.3

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