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加速药物开发的二期与三期联合设计AI 解读

For late-stage clinical development, we propose combining phase 2 and 3 trials via a two-stage adaptive design. In the first stage, short-term safety and efficacy are examined, after which low doses that lack efficacy and high doses that cause safety concerns are eliminated from further evaluation. The trial continues to the second stage with doses that are not eliminated. For the second stage, the required sample size is adjusted to maintain power. All patients, including…

两阶段自适应设计的统一理论AI 解读

Adaptation of a clinical trial occurs when there is a change from the planned course of action in design, sample size, or method of analysis. There are many proposed adaptations in the literature, but no comprehensive mathematical theory to support them. The standard Neyman–Pearson theory is not suitable for the setting, and its use may result in invalid inference. This article provides a general formulation of adaptation, under which its validity can be broadly and…