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SAGE Publications, Statistical Methods in Medical Research, 6(20), p. 657-666

DOI: 10.1177/0962280210379174

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

Journal article published in 2010 by Stephen Senn, Katie Rolfe, Steven A. Julious ORCID
This paper was not found in any repository, but could be made available legally by the author.
This paper was not found in any repository, but could be made available legally by the author.

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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.