Practical Considerations for Bayesian and Frequentist Adaptive Clinical Trials

Clinical trials play a critical role in drug development. New trial designs often depend on historical data, which, however, may not be accurate for the current study due to changes in study populations, patient heterogeneity, or different medical standards. Consequently, the original study design may need to be adjusted to accommodate new findings and unexpected interim results. Through carefully thought-out and planned adaptations, the right dose can be identified faster, patients can be treated more effectively, and treatment effects evaluated more efficiently. Reflecting the increasing importance and use of adaptive clinical trials, the International Council for Harmonisation (ICH) has tasked a working group to develop harmonized regulatory guidance that is expected to be released for public consultation by the time of this course. This one-day short course introduces various adaptive methods for Phase I to Phase III clinical trials using both, frequentist and Bayesian methods. Accordingly, we introduce different types of adaptive designs and illustrate practical considerations with case studies. Types of adaptive designs covered in this course include dose escalation / de-escalation, adaptive dose finding studies, master protocol designs, trials with sample size adaptations as well as adaptive designs for confirmatory trials with treatment or population selection at interim.