An Overview of Innovative Designs and Statistical Considerations: Lessons Learned

• Activity Number: 262 - The FDA Complex Innovative Trial Design Pilot Program: Learning from Case Examples and Simulations for the Public Good
• Type: Topic Contributed
• Date/Time: Tuesday, August 4, 2020 : 1:00 PM to 2:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Laura Lee Johnson*
• Companies: FDA
• Keywords: complex innovative design (CID); adaptive designs; master protocols

Abstract

We will discuss a few of the authorized parts of the complex innovative design proposals FDA has accepted into its pilot program to date and recent CDER and CBER guidance on related topics. Discussion will include the importance of incorporating all the adaptations in simulation scenarios, issues that may arise when determining the range of plausible values for an endpoint when borrowing data from other datasets, exchangeability of persons across studies, and the nuisance of nuisance parameters. We will discuss how plans evolved and were developed to handle these issues and highlight the importance of simulations to bring to light unanticipated issues and promote pre-trial decision making.