How Adaptive Trial Designs can Increase Efficiency in Psychiatric Drug Development: A Case Study.

This paper uses a recently completed study to illustrate how adaptive trial designs can increase efficiency of psychiatric drug development. The design employed allowed a continuous reassessment of the estimated dose-response such that patients were randomized in a double-blind fashion to one of seven doses of the investigational drug, placebo, or active comparator. The study design also permitted early detection of futility allowing for early study termination.

A Bayesian dose-finding trial with adaptive dose expansion to flexibly assess efficacy and safety of an investigational drug.

Background: Adaptive dose-ranging trials are more efficient than traditional approaches and may be designed to explicitly address the goals and decisions inherent in learn-phase drug development. We report the design, implementation, and outcome of an innovative Bayesian, response-adaptive, dose-ranging trial of an investigational drug in patients with diabetes, incorporating a dose expansion approach to flexibly address both efficacy and safety.

Purpose: The design was developed to assess whether one or more doses of an investigational drug demonstrated superior efficacy to an active control while maintaining an acceptable safety profile.