Viewpoints on the FDA draft adaptive designs guidance from the PhRMA working group

J Biopharm Stat. 2010 Nov;20(6):1115-24. doi: 10.1080/10543406.2010.514452.

Abstract

The US Food and Drug Administration has recently released a draft guidance document on adaptive clinical trials. We comment on the document from the particular perspective of the authors as members of a PhRMA working group on this topic, which has interacted with FDA personnel on adaptive trial issue during recent years. We describe the activities and prior work of our working group, and use this as a basis to discuss the content of the guidance document as it relates to several issues of current relevance, such as data monitoring processes, adaptive dose finding, so-called seamless trial designs, and sample size reestimation.

MeSH terms

  • Clinical Trials as Topic / methods*
  • Clinical Trials as Topic / statistics & numerical data
  • Data Interpretation, Statistical
  • Dose-Response Relationship, Drug
  • Drug Approval / methods*
  • Drug Dosage Calculations
  • Guidelines as Topic
  • Humans
  • Models, Statistical
  • Reproducibility of Results
  • Research Design*
  • Sample Size
  • Treatment Outcome
  • United States