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April 28, 2015

The Platform Trial: An Efficient Strategy for Evaluating Multiple Treatments

Author Affiliations
  • 1Berry Consultants LLC, Austin, Texas
  • 2Department of Biostatistics, University of Kansas Medical Center, Kansas City
  • 3University of Central Florida College of Medicine, Orlando
  • 4Department of Emergency Medicine, Harbor-UCLA Medical Center, Torrance, California
JAMA. 2015;313(16):1619-1620. doi:10.1001/jama.2015.2316

The drug development enterprise is struggling. The development of new therapies is limited by high costs, slow progress, and a high failure rate, even in the late stages of development. Clinical trials are most commonly based on a “one population, one drug, one disease” strategy, in which the clinical trial infrastructure is created to test a single treatment in a homogeneous population.

This approach has been largely unsuccessful for multiple diseases, including sepsis, dementia, and stroke. Despite promising preclinical and early human trials, there have been numerous negative phase 3 trials of treatments for Alzheimer disease1 and more than 40 negative phase 3 trials of neuroprotectants for stroke.2 Effective treatments for such diseases will likely require combining treatments to affect multiple targets in complex cellular pathways and, perhaps, tailoring treatments to subgroups defined by genetic, proteomic, metabolomic, or other markers.3