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Scand. J. Gastroenterol. Suppl. · Jan 1988
Comparative StudyStatistical issues in studies of individual response.
- D J Spiegelhalter.
- MRC Biostatistics Unit, Cambridge.
- Scand. J. Gastroenterol. Suppl. 1988 Jan 1; 147: 40-5.
AbstractWe consider intensive studies of individual response to therapy in a controlled experiment. It is helpful to distinguish between strict 'N = 1' studies, which are pragmatic trials intended to draw conclusions concerning only the patient under consideration, and 'N much greater than 1' studies, which are explanatory trials intended to make more general statements about a treatment with highly variable response, for which aggregate measures of effect on groups are inappropriate. Issues of design, measurement, and statistical significance are discussed, and the rule of permutation tests emphasised. Three published examples are used as illustrations.
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