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Separating Perceptions from Reality in the Acceptance of Adaptive Trials by Regulatory Agencies

Statistical Science and Quantitative Thinking

Day & Time:
June 18, 10:30AM - 12:00PM (Pacific Standard Time)

Session Number:

Room Number:



Separating Perceptions from Reality in the Acceptance of Adaptive Trials by Regulatory Agencies

David Ohlssen, PhD
Senior Expert Methodologist
Novartis Pharmaceuticals Corporation, United States

A high level of uncertainty still persists within industry over the acceptance of innovative trials by regulatory agencies. With the FDA guidance document on adaptive trials near final, and the recent EMA qualification for statistical methodology combining proof of concept (PoC) and dose finding, this session aims to separate perceptions from reality, and demonstrate that agencies are more accepting of new methods than perhaps industry realizes. Although the draft FDA guidance document on adaptive clinical trials was issued in 2010 and the European Reflection Paper in 2007, a high level of uncertainty persists and, in some cases, disagreement relating to what is perceived as actually acceptable and what is not, from a regulatory point of view. As a consequence, often a more conservative position is taken within companies. This session looks at the barriers within industry and presents an opportunity to remove uncertainty and provide education and clarity regarding the acceptance of adaptive trials by regulatory agencies. One particular example further demonstrating the acceptance of innovative methods in drug development is given by EMA’s CHMP favorable opinion on MCP-Mod as an efficient statistical methodology for model-based design and analysis of dose finding studies. The session will provide a case study of the MCP-Mod approach, which is an adaptive analysis method for combining PoC and model-based dose-finding.

Learning Objective(s):
Interpret the intent of the FDA guidance on adaptive designs; Identify key factors required by regulators in the review of adaptive trials; Apply adaptive trials within their own clinical development programs.

Presentation(s) & Speaker(s):
FDA Viewpoint
Lisa M. LaVange, PhD
Director, Office of Biostatistics, Office of Translational Science, CDER
FDA, United States

Innovative Dose-Finding Using Novel Adaptive Design and Analysis Approaches
Silke Jörgens
Senior Statistical Consultant
ICON Plc, Germany

Adaptive Designs: An Exercise in Change Management
Vladimir Dragalin, PhD
Vice President, Scientific Fellow, Quantitative Sciences
Janssen Research & Development, LLC, United States