title: [The N-of-1 trial: the ideal study design that is underused].
abstract: The randomised experiment in a single patient, the N-of-1 trial, is the best study design for demonstrating causality, for example between agent and effect. Despite this, this type of study is only encountered sporadically in medical journals. One reason for this is that even this type of design cannot definitively demonstrate causality, because different points in time are compared with one another. Moreover, the design is rather inefficient, since the results correspond with those from observation without randomisation, placebo control or blinding. Even so, the N-of-1 trial is the ultimate form of verification in, for example, the individualisation of treatment. For this reason, this form of study might be used more often.
authors.affiliation: Leids Universitair Medisch Centrum, afd. Klinische Epidemiologie, Postbus 9600, 2300 RC Leiden. j.p.vandenbroucke@lumc.nl
authors.forename: J P
authors.initials: JP
authors.lastname: Vandenbroucke
mesh_terms: D018592:Cross-Over Studies; D019317:Evidence-Based Medicine; D006801:Humans; D016032:Randomized Controlled Trials as Topic / Q000379:methods; D012107:Research Design / Q000592:standards