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Group-sequential
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Allows a trial to be stopped early for efficacy, futility, or safety, when there is enough evidence to justify doing so.
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On average, the sample size that would be required by a trial is reduced; particularly for those with strong treatment effects.
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Response adaptive randomisation
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Allows the treatment allocation ratio(s) to be altered as the trial progresses.
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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.
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Multi-arm multi-stage (MAMS)
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Allows multiple treatments to be evaluated in a single trial. Interim analyses allow less promising treatments to be removed from the trial early.
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Highly efficient for evaluating multiple treatments at once.
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Sample size re-assessment
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Allows the sample size to be modified in response to the outcome variation or treatment effect observed in the interim.
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The trial is more likely to be powered at the desired level, especially when there is limited data to inform a sample size calculation.
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Biomarker adaptive
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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.
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Patient subgroups who will benefit most from particular treatments can be identified and prioritized.
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Platform trial
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Allows treatments to be added in to an ongoing trial. Typically involves several treatments being evaluated under an overarching protocol.
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Efficient for evaluating multiple treatments as new ones become available over time.
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