Measurement in clinical trials: A neglected issue for statisticians?

Stephen Senn*, Steven Julious

*Corresponding author for this work

Research output: Contribution to journalArticleResearchpeer-review

108 Citations (Scopus)

Abstract

Biostatisticians have frequently uncritically accepted the measurements provided by their medical colleagues engaged in clinical research. Such measures often involve considerable loss of information. Particularly, unfortunate is the widespread use of the so-called 'responder analysis', which may involve not only a loss of information through dichotomization, but also extravagant and unjustified causal inference regarding individual treatment effects at the patient level, and, increasingly, the use of the so-called number needed to treat scale of measurement. Other problems involve inefficient use of baseline measurements, the use of covariates measured after the start of treatment, the interpretation of titrations and composite response measures. Many of these bad practices are becoming enshrined in the regulatory guidance to the pharmaceutical industry. We consider the losses involved in inappropriate measures and suggest that statisticians should pay more attention to this aspect of their work.

Original languageEnglish
Pages (from-to)3189-3209
Number of pages21
JournalStatistics in Medicine
Volume28
Issue number26
DOIs
Publication statusPublished - 20 Nov 2009
Externally publishedYes

Keywords

  • Baselines
  • Dichotomies
  • Number needed to treat
  • Ordered categorical data
  • Responder analysis
  • Titration

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