Adaptive Designs Workshop

Opportunities, Challenges and Scope in Drug Development

November 13-14, 2006

Adaptive clinical trial designs have recently attracted attention beyond the statistical community.  These designs provide a potential pathway for drug development that, under certain circumstances, can improve quality, speed and efficiency of decision making. By introducing flexibility within trial design, adaptive approaches should facilitate early identification of negative and positive therapeutic responses and conserve precious patient resources for treatments that have a higher probability of success. Thus, the appropriate use of adaptive clinical trial designs should minimize patient exposure to ineffective treatments, while increasing the efficiency of clinical trials.  However, as with any proposed new technology or new paradigm, it is appropriate for these to be questioned and challenged.  Given the context of human clinical research, this rigorous critical debate could not be more important.

The aim of this workshop was to establish a constructive dialogue among statisticians, clinicians, regulators and other functions within the pharmaceutical industry, health authority agencies, and academia to contribute to the development of consensus positions in the emerging field of adaptive designs.

Due to the growing scientific interest in adaptive designs and their potential impact on a broad variety of clinical research, clinical operations, data management, and regulatory issues, this PhRMA conference was opened to all of the FDA regulated industry and other interested parties.  The workshop agenda and presentations (where permitted) are being made publicly available.

 Click here to download the agenda.

GENERAL SESSION:

Walt Offen, Ph.D., Eli Lilly and Company
 “Welcome and Introduction: Moving From Emotional Debate to a Fact Driven Dialogue”
 
Michael Krams, M.D., Wyeth
 “Welcome and Introduction: Moving From Emotional Debate to a Fact Driven Dialogue”

 

Robert Ruffolo, Ph.D., Wyeth
 “Discovery and Development of New Medicines”

 

Declan Doogan, M.D., Pfizer Inc
 “Transformational Change in R&D”
 
Marc Walton, M.D., Ph.D., FDA
 “Adaptive Design Trial Issues: Goals and Needs”

 

Michael Krams, M.D., Wyeth
 “Improving the Efficiency of Drug Development: The Bigger Picture”

 

GENERAL SESSION:  Adaptive Designs - Taxonomy and Scope

Vlad Dragalin, Ph.D., Wyeth
  “Classification and Taxonomy”

 

Robert Temple, M.D., FDA
  "Myth Busting - Clinical"

 

Robert O'Neill, Ph.D., FDA
  "Myth Busting - Statistical"

 

(Kuang-Kuo) Gordon Lan, Ph.D., Johnson & Johnson
  “Technical and Practical Problems In Adaptive Designs”

GENERAL SESSION:   Adaptive Approaches

Brenda Gaydos, Ph.D., Eli Lilly and Company
 “Overview of Adaptive Dose Finding Methods”

 

Keaven Anderson, Ph.D., Merck & Co., Inc.
 “Sample Size Re-estimation”

 

Jeff Maca, Ph.D., Novartis Pharmaceuticals Corporation
 “Seamless Designs”

 

Don Stanski, M.D., Ph.D., Novartis Pharmaceuticals Corporation
 “PK/PD Modeling to Choose the Dose for Phase III”

 

Jose Pinheiro, Ph.D., Novartis Pharmaceuticals Corporation
 “Scenario Planning: Comparing Methods, Quantifying Potential Gains from Adaptive Dose Finding”

 

Cyrus Mehta, Ph.D., Cytel Inc.
 “Example Seamless Phase II/III Design”

 

Paul Gallo, Ph.D., Novartis Pharmaceuticals Corporation
Bram Zuckerman, M.D., FDA
 ”Issues Relating to Decision Making / Implementation and Data Monitoring Committees in Adaptive Design Trials”

 

Paul Gallo, Ph.D., Novartis Pharmaceuticals Corporation
 “Monitoring and confidentiality issues for adaptive design trials”

 

Ning Li, Ph.D., FDA
 “Statistical Issues of Data Monitoring Committee in Adaptive Designs”

 

Richard Kuntz , M.D., Medtronic Inc.
 “Data Monitoring Committees and Adaptive Clinical Trial Design”

 

Bram Zuckerman, M.D., FDA
 “Practical Issues with Adaptive Trial Design: An FDA Division of Cardiovascular Devices (DCD) Perspective”

 

Susan Ellenberg, Ph.D., University of Pennsylvania
 “The Role of Data Monitoring Committees In Implementing Adaptive Designs”

 

Sue-Jane Wang, Ph.D., FDA
 “Regulatory Experience of Adaptive Designs in Well-Controlled Clinical Trials”

GENERAL SESSION:  Regulatory Interactions and Planning

Robert Powell, PharmD, FDA
 “Current FDA Activities, Future Concepts and Barriers”

 

Greg Enas, Ph.D., Eli Lilly and Company
 “A New Process for Interactions Between Sponsors and FDA To Facilitate Planning/Implementation of Adaptive Designs”
 

BREAKOUT SESSIONS:

Joel Schiffenbauer, M.D.
Bo Zhen, Ph.D.
Howard Fingert, M.D., Pfizer Inc
Steve Snapinn, Ph.D., Amgen Inc.
 “Session A:  Procedural & DMC Issues in Adaptive Paradigm”

 

Robert Meyer, M.D.
Jim Hung, Ph.D.
Ron Marcus, M.D., Bristol-Myers Squibb Company
Willi Maurer, Ph.D., Novartis Pharmaceuticals Corporation
 “Session B:  Consideration for Adaptation in Confirmatory Trials”

 

Dave Ross, M.D.
Telba Irony, Ph.D.
Stephen J. Victor, M.D., Johnson & Johnson
Qing Liu, Ph.D., Johnson & Johnson
 “Session C:  Seamless Phase II/III Design”
 “Session C:  Seamless Phase II/III Design Summary”

 

Joga Gobburu, M.D.
Stella Machado, Ph.D.
Frederic Sax, M.D., AstraZeneca
Alex Dmitrienko, Ph.D., Eli Lilly and Company
 “Session D:  Dose Finding”

GENERAL SESSION:  The Future of Adaptive Design

Donald A. Berry, Ph.D., MD Anderson
 ”The Promise and the Perils of Adaptive Designs”

 

Andy Grieve, Ph.D., Pfizer Inc
 “Adaptive Designs: A Fad or the Future of Clinical Research?”

FEEDBACK FROM BREAKOUT SESSION

David DeBrota, M.D., Eli Lilly and Company
 “Feedback from Breakout Session”

 

Brenda Gaydos, Ph.D., Eli Lilly and Company
 “Summary of Recommendations and Next Steps”