Investigating variability in patient response to treatment - A case study from a replicate cross-over study

Stephen Senn*, Katie Rolfe, Steven A. Julious

*Corresponding author for this work

Research output: Contribution to journalArticleResearchpeer-review

54 Citations (Scopus)

Abstract

It is a common belief that individual variation in response to treatment is an important explanation for the variation in observed outcomes in clinical trials. If such variation is large, it seems reasonable to suppose that progress in treating disease will be advanced by classifying patients according to their abilities or not to 'respond' to particular treatments. We consider that there is currently a lost opportunity in drug development. There is a great deal of talk about individual response to treatment and tailor-made drugs. However, relatively little work is being done to formally investigate, using suitable designs, where individual response to treatment may be important. Through a case study from a replicate cross-over study we show how, given suitable replication, it is possible to isolate the component of variation corresponding to patient-by-treatment interaction and hence investigate the possibility of individual response to treatment.

Original languageEnglish
Pages (from-to)657-666
Number of pages10
JournalStatistical Methods in Medical Research
Volume20
Issue number6
DOIs
Publication statusPublished - Dec 2011
Externally publishedYes

Keywords

  • components of variation
  • personalised medicine
  • replicate cross-over
  • treatment response
  • within subject variability

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