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Abstract:
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In areas of unmet need, conducting randomized controlled trials may be infeasible due to enrollment challenges, timelines, cost, or ethical concerns. For drug development targeting small populations in particular, unique challenges such as small patient numbers, heterogeneity in disease presentation, and a lack of understanding of the mechanism, call for more thoughtful and innovative design and analysis of trials. With the advancement of technology, information available from sources of real-world data have grown rapidly. Under the framework of adaptive designs, such information can be utilized to generate evidence for making efficacy claim during interim or final analysis. Within this design, some important perspectives merit careful rumination to ensure robustness of analytical methods in estimating effects of treatments, for instance, how can the heterogeneity from historical data and the current trial data be accounted for during an interim analysis? How to control for type I error? How to ensure study integrity? In this presentation, a case study in confirmatory pediatric study will be discussed, where the focus will be on methodological and practical considerations.
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