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Table 1 An overview of various types of adaptive design and their benefits

From: Innovative trial approaches in immune-mediated inflammatory diseases: current use and future potential

Adaptive design Description Benefits
Group-sequential Allows a trial to be stopped early for efficacy, futility, or safety, when there is enough evidence to justify doing so. On average, the sample size that would be required by a trial is reduced; particularly for those with strong treatment effects.
Response adaptive randomisation Allows the treatment allocation ratio(s) to be altered as the trial progresses. Allocation can be skewed in favour of the treatment arm that appears to have higher efficacy; meaning more patients are expected to respond in the trial.
Multi-arm multi-stage (MAMS) Allows multiple treatments to be evaluated in a single trial. Interim analyses allow less promising treatments to be removed from the trial early. Highly efficient for evaluating multiple treatments at once.
Sample size re-assessment Allows the sample size to be modified in response to the outcome variation or treatment effect observed in the interim. The trial is more likely to be powered at the desired level, especially when there is limited data to inform a sample size calculation.
Biomarker adaptive Allows the trial’s population to be adjusted to avoid enrolling patients who don’t benefit from a treatment; typically this involves incorporating information from, or adapting on, a biomarker. Patient subgroups who will benefit most from particular treatments can be identified and prioritized.
Platform trial Allows treatments to be added in to an ongoing trial. Typically involves several treatments being evaluated under an overarching protocol. Efficient for evaluating multiple treatments as new ones become available over time.