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Abstract:
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Clinical trials play a critical role in pharmaceutical drug development. New trial designs often depend on historical data, which may not be accurate for the current study due to changes in study populations, patient heterogeneity, or different medical facilities. As a result, the original plan and study design may need to be adjusted or even altered to accommodate new findings and unexpected interim results. The goal of using adaptive methods in clinical trials is to enhance the flexibility of trial conduct and maintain the integrity of trial findings. Through carefully thought-out and planned adaptation, the right dose can be identified faster, patients can be treated more effectively, and treatment effects can be evaluated more efficiently. The net result makes for a more expeditious drug development process. From the perspective of practicality, this one-day short course will introduce various adaptive methods for Phase I to Phase III clinical trials. Accordingly, different types of adaptive designs will be introduced and illustrated with case studies. This includes dose escalation/de-escalation and dose insertion based on observed data, adaptive dose-finding studies using optimal designs to allocate new cohorts of patients based on the accumulated evidence; population enrichment designs; early stopping for toxicity, futility, or efficacy using group-sequential designs; blinded and unblinded sample size re-estimation; and adaptive designs for confirmatory trials with treatment or population selection at interim.
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