Uses and limitations of randomization-based efficacy estimators
- 1 August 2005
- journal article
- research article
- Published by SAGE Publications in Statistical Methods in Medical Research
- Vol. 14 (4), 327-347
- https://doi.org/10.1191/0962280205sm406oa
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.Keywords
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