Innovative approaches for designing and analyzing adaptive dose-ranging trials

J Biopharm Stat. 2007;17(6):965-95. doi: 10.1080/10543400701643848.

Abstract

Inadequate selection of the dose to bring forward in confirmatory trials has been identified as one of the key drivers of the decreasing success rates observed in drug development programs across the pharmaceutical industry. In recognition of this problem, the Pharmaceutical Research and Manufacturers of America (PhRMA), formed a working group to evaluate and develop alternative approaches to dose finding, including adaptive dose-ranging designs. This paper summarizes the work of the group, including the results and conclusions of a comprehensive simulation study, and puts forward recommendations on how to improve dose ranging in clinical development, including, but not limited to, the use of adaptive dose-ranging methods.

MeSH terms

  • Clinical Trials as Topic / methods*
  • Clinical Trials as Topic / statistics & numerical data
  • Data Interpretation, Statistical*
  • Dose-Response Relationship, Drug
  • Drug Industry*
  • Humans
  • Research Design*
  • Sample Size