With the ICH E20 draft guidance now released and entering the public comment stage, Dr. Kert Viele of Berry Consultants begins a new blog series on this topic. Dr. Viele provides explainers for the designs and principles under discussion, and some initial reactions to the draft ICH E20 related to Group Sequential Designs in this blog.

A Goldilocks trial design is an adaptive clinical trial methodology developed to optimize the sample size dynamically during the course of a trial. Its name references the "just right" principle from the Goldilocks fairy tale—neither too large nor too small. Goldilocks designs seek balance between flexibility and efficiency.

A source of widespread confusion is the entrenched belief that introducing interim analyses “costs” alpha; that is, the assumption that interim adaptations erode the available alpha and require the sponsor to “pay a penalty.” This notion also leads to the myth that just the action of “looking at data” at an interim analysis is bad and costs alpha.

Regression-to-the-mean offers critical insights for drug development, like sports statistics, emphasizing the importance of understanding data variability across multiple levels of inference.

Understanding DSMBs is pivotal in ensuring trial integrity, safety, and success.

A discussion on the intricacies of implementing adaptive clinical trials, their operational processes, and how Berry ensures timely execution.