title: Uses and limitations of randomization-based efficacy estimators.
abstract: In randomized trials with departures from allocated treatment, intention-to-treat analysis is important but not always sufficient. The most common supplement to intention-to-treat analysis is per-protocol analysis, whose assumption of comparability between different nonrandomized groups is often implausible. Randomization-based methods avoid making this assumption and are preferable. Situations where intention-to-treat analysis is insufficient and a randomization-based method is useful include provision of patient information, exploration of treatment-covariate and treatment-time interactions, meta-analysis, and equivalence trials.
authors.affiliation: MRC Biostatistics Unit, Institute of Public Health, Robinson Way, Cambridge CB2 2SR, UK. ian.white@mrc-bsu.cam.ac.uk
authors.forename: Ian R
authors.initials: IR
authors.lastname: White
mesh_terms: D000368:Aged; D002648:Child; D002985:Clinical Protocols; D019983:Guideline Adherence; D006801:Humans; D008297:Male; D015233:Models, Statistical; D017711:Nonlinear Dynamics; D016032:Randomized Controlled Trials as Topic; D012107:Research Design