Heterogeneity of Treatment Effects in Clinical Trials: Methods and Innovations


  1. Gain a common understanding on the desired goals of subgroup analyses
  2. Establish standards for assessing effects within subgroups and for defining meaningful differences in effect between subgroups
  3. Review the characteristics, assumptions, advantages and limitations of traditional and innovative models for subgroup analyses
  4. Review contribution of data visualization techniques to complement and enhance statistical modeling approaches and interpretation
  5. Develop and communicate recommendations
8:30-8:45 Welcome
Opening remarks (MRCT Center)
Richardae Araojo
Sylva Collins
Barbara Bierer


Keynote speaker: A clinical trialist’s perspective
“System Redesign needed to make sense of findings that suggest heterogeneity of treatment effect”
Robert Califf
9:10-10:15 Challenges for the Regulator, Clinician, and Patient: Population safety and efficacy but individual treatment
Moderator: David Strauss
Christopher Granger
Ellis Unger
Janet Wittes
Donna Cryer
Danielle Campbell
Aloka Chakravarty
10:15-10:30 Break  


10:30-11:00 Goals of subgroup analyses, assessing standards for analysis of differences, and limits of interpretation Steven Snapinn
11:00-11:30 Subgroup identification: Utility, impact, and methods


Scott Evans
11:30-12:45 Statistical Methods I
Moderator: Li Chen
Stuart Pocock
Stephen Ruberg
Lisa Lavange
Mark Rothmann
Kathleen Fritsch
Ravi Varadhan
12:45-12:50 Closing remarks Richardae Araojo and Barbara Bierer
Time Proposed Agenda Proposed Speakers
 8:30-9:30 Statistical Methods II
Moderator : Steve Snapinn
Donald Berry
Thomas Louis
Gene Pennello
Frank Bretz
9:30- 10:00 Summary Discussion of Statistical Methods I and II
10:00-10:15 Break  
10:15-11:15 Data Visualization and the role of graphical representation
Moderator: Marcia Levenstein
Frank Harrell
Ilya Lipkovich
Michael Griswold
11:15-12:15 Panel: Statistical methodologies, pros and cons
Moderator: Thomas Gwise
Frank Rockhold
Qi Jiang
Thomas Gwise
John Scott
12:15-12:45 Open discussion and Conclusions FDA and MRCT Center
12:45- 1:00 Core team to discuss Day 1 & Day 2 wrap up Planning committee