Model-Based Approaches to Improve Adaptive Designs in Early Clinical Development

• Activity Number: 429
• Type: Invited
• Date/Time: Wednesday, August 6, 2014 : 8:30 AM to 10:20 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Jose C. Pinheiro*+
• Companies: Janssen
• Keywords: Early stage trials ; Modeling ; Trial simulation

Abstract

Studies in early clinical development are generally done with considerably fewer resources, both in terms of money as well as time available to run them. Despite this resource restriction, these trials are often designed using principles better suited to confirmatory studies, such as hypothesis tests based on change from baseline information. Model-based methods in combination with adaptive strategies can greatly enhance the information-efficiency of early phase studies. Examples include making using of all available data via longitudinal models, investigating research questions of interest via Bayesian decision rules, and including futility rules for early stopping. This talk will use a real proof-of-concept trial to motivate and discuss adaptive and model-based strategies to improve decision making in early development.