Two techniques for investigating interactions between treatment and continuous covariates in clinical trials

Patrick Royston () and Willi Sauerbrei ()
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Patrick Royston: MRC Clinical Trials Unit
Willi Sauerbrei: Freiburg University Medical Center

Stata Journal, 2009, vol. 9, issue 2, 230-251

Abstract: There is increasing interest in the medical world in the possibility of tailoring treatment to the individual patient. Statistically, the relevant task is to identify interactions between covariates and treatments, such that the patient’s value of a given covariate influences how strongly (or even whether) they are likely to respond to a treatment. The most valuable data are obtained in randomized controlled clinical trials of novel treatments in comparison with a control treat- ment. We describe two techniques to detect and model such interactions. The first technique, multivariable fractional polynomials interaction, is based on fractional polynomials methodology, and provides a method of testing for continuous-by- binary interactions and by modeling the treatment effect as a function of a continuous covariate. The second technique, subpopulation treatment-effect pattern plot, aims to do something similar but is focused on producing a nonparametric estimate of the treatment effect, expressed graphically. Stata programs for both of these techniques are described. Real data for brain and breast cancer are used as examples. Copyright 2009 by StataCorp LP.

Keywords: mfpi; mfpi plot; stepp tail; stepp window; stepp plot; continuous covariates; treatment-covariate interaction; clinical trials; fractional polynomials; subpopulation treatment-effect pattern plot (search for similar items in EconPapers)
Date: 2009
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Citations: View citations in EconPapers (2)

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