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. |