title: Statistical issues in studies of individual response.
abstract: We 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.
authors.affiliation: MRC Biostatistics Unit, Cambridge.
authors.forename: D J
authors.initials: DJ
authors.lastname: Spiegelhalter
mesh_terms: D002986:Clinical Trials as Topic; D003627:Data Interpretation, Statistical; D006801:Humans; D008499:Medical Records; D010919:Placebos; D011897:Random Allocation; D012107:Research Design
chemical_list: D010919:Placebos