Abstract
The nature of any experiment is that its outcome is uncertain. Due to their great expense and logistical challenges, key aspects of clinical trials must be carefully planned in advance. However, the parameters required to design the trial cannot be known for certain, and there is no guarantee that the design is optimal. Adaptive trial designs incorporate planned flexibility in the conduct of the trial in response to emerging information. The advantage of flexible designs is that they help to optimize the ultimate design of the trial to make the most efficient use of resources. The disadvantage is that valid adaptive designs increase the complexity of the trial conduct and statistical analysis. Sequential testing designs allow stopping the trial early for superiority or futility. Multi-arm designs allow multiple interventions to undergo early testing to choose the most promising for continued study. Adaptive randomization designs help maintain balance among participant characteristics. Enrichment designs change inclusion criteria after an initial phase to target patients who might benefit the most from an intervention. Internal pilot designs re-estimate the planned sample size to increase efficiency and avoid inconclusive findings.
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Richman, J.S., Boughey, J.C. (2017). Adaptive Trial Designs. In: Itani, K., Reda, D. (eds) Clinical Trials Design in Operative and Non Operative Invasive Procedures. Springer, Cham. https://doi.org/10.1007/978-3-319-53877-8_11
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DOI: https://doi.org/10.1007/978-3-319-53877-8_11
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