May 2015

Novel Methods and Technologies for 21st-Century Clinical TrialsA Review

Author Affiliations
  • 1Department of Neurology, University of Rochester Medical Center, Rochester, New York
  • 2Center for Human Experimental Therapeutics, University of Rochester Medical Center, Rochester, New York
  • 3currently a medical student at Duke University School of Medicine, Durham, North Carolina
  • 4Clinical and Translational Sciences Institute, University of Rochester Medical Center, Rochester, New York

Copyright 2015 American Medical Association. All Rights Reserved. Applicable FARS/DFARS Restrictions Apply to Government Use.

JAMA Neurol. 2015;72(5):582-588. doi:10.1001/jamaneurol.2014.4524

Importance  New technologies are rapidly reshaping health care. However, their effect on drug development to date generally has been limited.

Objectives  To evaluate disease modeling and simulation, alternative study design, novel objective measures, virtual research visits, and enhanced participant engagement and to examine their potential effects as methods and tools on clinical trials.

Evidence Review  We conducted a systematic search of relevant terms on PubMed (disease modeling and clinical trials; adaptive design, clinical trials, and neurology; Internet, clinical trials, and neurology; and telemedicine, clinical trials, and neurology), references of previous publications, and our files. The search encompassed articles published from January 1, 2000, through November 30, 2014, and produced 7976 articles, of which 22 were determined to be relevant and are included in this review.

Findings  Few of these new methods and technologies have been applied to neurology clinical trials. Clinical outcomes, including cognitive and stroke outcomes, increasingly are captured remotely. Other therapeutic areas have successfully implemented many of these tools and technologies, including web-enabled clinical trials.

Conclusions and Relevance  Increased use of new tools and approaches in future clinical trials can enhance the design, improve the assessment, and engage participants in the evaluation of novel therapies for neurologic disorders.