Caring for the Individual Patient: Understanding Heterogeneous Treatment Effects (2019)
Chapter: Appendix C: Workshop Agenda
Appendix C
WORKSHOP AGENDA
Evidence and the Individual Patient: Understanding Heterogeneous Treatment Effects for Patient-Centered Care
May 31, 2018
National Academy of Sciences Building
Lecture Room
2101 Constitution Avenue, NW
Washington, DC 20001
| 8:30 a.m. | Coffee and light breakfast available |
| 9:00 a.m. | Welcome, Introductions, and Workshop Overview |
Welcome from the National Academy of Medicine
Anne-Marie Mazza, National Academy of Medicine
Opening Remarks and Workshop Overview
Joe Selby, Patient-Centered Outcomes Research Institute (PCORI)
| 9:15 a.m. | Overview of Heterogeneous Treatment Effects: Moving from Evidence-Based Medicine to Personalized/Precision Medicine |
Speakers will present a conceptual overview of heterogeneous treatment effects, as well as examples of clinical trials analyzed to yield more personalized treatment effect estimates. Discussion will focus on how changes in the design of clinical research might enable a better understanding of how treatment effects vary across individuals.
Moderator: Harry Selker, Tufts Medical Center
Speakers:
David Kent, Tufts Medical Center
Sanjay Basu, Stanford University
Derek Angus, University of Pittsburgh
Discussants:
Sheldon Greenfield, University of California, Irvine
Bob Temple, U.S. Food and Drug Administration
Q&A and Open Discussion
| 10:45 a.m. | Break |
| 11:00 a.m. | An Equation-Free Presentation of New Methods for Prediction of Treatment Benefit and Model Evaluation |
This session will focus on statistical methods. Speakers will discuss lessons learned from the genomics revolution, machine learning methods for the analysis of trial data, and new methods for evaluating models that predict treatment benefit.
Moderator: Ewout Steyerberg, Leiden University Medical Center
Speakers:
A. Cecile J. W. Janssens, Emory University Rollins School of Public Health
Fan Li, Duke University
Patrick Heagerty, University of Washington School of Public Health
Discussants:
Frank Harrell, Vanderbilt University
Michael Pencina, Duke Clinical Research Institute
Q&A and Open Discussion
| 12:20 p.m. | Break |
Participants will pick up lunch.
| 12:35 p.m. | Discussion with Stakeholders |
This session will focus on how representatives of several patient communities have applied research to guide their own care, given their own individual circumstances. Additional stakeholders will contribute to the discussion of how to better align evidence with patient-centered care.
Moderator: Bray Patrick-Lake, Duke University
Panelists:
Thomas Concannon, RAND Corporation
Seth Morgan, National Multiple Sclerosis Society, Advocate and Patient Stakeholder
Christine Stake, Ann & Robert H. Lurie Children’s Hospital of Chicago, Patient Stakeholder
Reactors:
Robert Dubois, National Pharmaceutical Council
Karina Davidson, Columbia University
Q&A and Open Discussion—Engagement with other stakeholders
| 1:30 p.m. | Break |
| 1:45 p.m. | From Research into Practice: Implementation and Oversight |
This session will focus on barriers to implementation applying predictions in clinical practice and how to overcome these barriers. Speakers will discuss how to go beyond “all-or-nothing” quality measures to incentivize more personalized care.
Moderator: Nilay Shah, Mayo Clinic
Speakers:
John Spertus, Saint Luke’s Mid America Heart Institute
Josh Peterson, Vanderbilt University
Rodney Hayward, University of Michigan
Discussants:
Naomi Aronson, Blue Cross Blue Shield Association
Katrina Armstrong, Massachusetts General Hospital
Q&A and Open Discussion
| 3:15 p.m. | Opportunities for Collaborative Action |
The aim of this session is to reflect on key themes from the day’s discussion, focusing on innovative methods for understanding heterogeneous treatment effects, challenges related to implementation and oversight to personalize care,
and outstanding policy and research questions that need to be addressed to accelerate progress.
Moderator: Joe Selby, PCORI
Reactors:
Steven Goodman, Stanford University
Evelyn Whitlock, PCORI
Closing Remarks:
David Kent, Tufts Medical Center
| 4:30 p.m. | Adjourn |
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