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

🎥   Watch the webinar recording: 


Day 1: Monday, November 30, 2020

Opening remarks (MRCT Center)
Richardae Araojo
Sylva Collins
Barbara Bierer
8:45-9:10 Keynote speaker: A clinical trialist’s perspective
“System Redesign needed to make sense of findings that suggest heterogeneity of treatment effect”
Robert Califf
9:10-10:15Challenges 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
10:30-11:00Goals of subgroup analyses, assessing standards for analysis of differences, and limits of interpretationSteven Snapinn
11:00-11:30Subgroup identification: Utility, impact, and methods Scott Evans
11:30-12:45Statistical Methods I
Moderator: Li Chen
Stuart Pocock
Stephen Ruberg
Lisa Lavange
Mark Rothmann
Kathleen Fritsch
Ravi Varadhan
12:45-12:50Closing remarksRichardae Araojo and Barbara Bierer

Day 2: Tuesday, December 1, 2020

TimeProposed AgendaProposed Speakers
 8:30-9:30Statistical Methods II
Moderator : Steve Snapinn
Donald Berry
Thomas Louis
Gene Pennello
Frank Bretz
9:30- 10:00Summary Discussion of Statistical Methods I and II 
10:15-11:15Data Visualization and the role of graphical representation
Moderator: Marcia Levenstein
Frank Harrell
Ilya Lipkovich
Michael Griswold
11:15-12:15Panel: Statistical methodologies, pros and cons
Moderator: Thomas Gwise
Frank Rockhold
Qi Jiang
Thomas Gwise
John Scott
12:15-12:45Open discussion and ConclusionsFDA and MRCT Center
12:45- 1:00Core team to discuss Day 1 & Day 2 wrap upPlanning committee