A modest proposal for dropping poor arms in clinical trials

Stat Med. 2014 Aug 30;33(19):3241-52. doi: 10.1002/sim.6169. Epub 2014 Apr 22.

Abstract

This paper presents a simple procedure for clinical trials comparing several arms with control. Demand for streamlining the evaluation of new treatments has led to phase III clinical trials with more arms than would have been used in the past. In such a setting, it is reasonable that some arms may not perform as well as an active control. We introduce a simple procedure that takes advantage of negative results in some comparisons to lessen the required strength of evidence for other comparisons. We evaluate properties analytically and use them to support claims made about multi-arm multi-stage designs.

Keywords: comparisons with control; estimation bias; family-wise error (FWE) rate; multi-arm multi-stage (MAMS) design; multiple comparisons; sequentially rejective methods.

MeSH terms

  • Bias
  • Biostatistics
  • Blood Pressure
  • Clinical Trials, Phase II as Topic / methods
  • Clinical Trials, Phase II as Topic / statistics & numerical data
  • Clinical Trials, Phase III as Topic / methods
  • Clinical Trials, Phase III as Topic / statistics & numerical data
  • Controlled Clinical Trials as Topic / methods*
  • Controlled Clinical Trials as Topic / statistics & numerical data
  • Humans
  • Hypertension / blood
  • Hypertension / diet therapy
  • Hypertension / physiopathology
  • Models, Statistical
  • Triglycerides / blood

Substances

  • Triglycerides