Skip Navigation

Center of Excellence in Regulatory Science and Innovation

Assessing and Communicating Heterogeneity of Treatment Effects for Patient Subpopulations: Challenges and Opportunities

8:15 A.M. – 4:30 P.M. (Registration begins at 7:30 A.M.)
NOVEMBER 28, 2018
FDA WHITE OAK CAMPUS, BLDG. 31, THE GREAT ROOM (1503 B & C)

Hosted by Johns Hopkins Center of Excellence in Regulatory Science and Innovation (JH-CERSI) and the Food and Drug Administration (FDA)

ABOUT

Heterogeneity of treatment effects (HTE) is the variation in how individuals respond to a treatment. This is a vital and yet underexplored concept of importance to regulators, manufacturers, payors, healthcare providers, academic researchers, and patient and consumer advocacy groups. HTE is of high relevance to the FDA for product approval, labelling, and post-marketing surveillance. We propose a stimulating symposium comprised of talks and panel discussions by leading experts that engages various stakeholders in how to assess and communicate HTE.

The symposium will focus on the following topics:


Who should attend?

This symposium is open to the public. The intended audience includes government, academia, industry, healthcare providers, payors, patients, and patient and consumer advocacy groups.

 

AGENDA & SPEAKERS


Recordings of Symposium Talks:

Morning: https://collaboration.fda.gov/pla3qi6repj8/ 

Afternoon: https://collaboration.fda.gov/pgh5vfvx1upt/ 

Time

Topic/Title

Speaker

7:30 am

Registration

 

8:15 am
 

Welcome and Introductions
 

Ravi Varadhan, PhD, PhD
Johns Hopkins University

8:25 am- 9:05 am



 

 

Keynote Speakers

History of FDA Encouragement to Consider Subgroup Variability

On Treatment Heterogeneity from the Patient’s Perspective

 

Robert Temple, MD
FDA, CDER

Scott Zeger, PhD
Johns Hopkins University

Session 1: Heterogeneity of Treatment Effects in Diverse Populations

9:05-9:25 am

 

Heterogeneity of Treatment Effects in Diverse Populations
 

Marjorie Jenkins, MD, MEdHP, FACP
FDA, OC/OWH

9:25 am-10:25 am




























 

Panel Discussion
















 












 

Moderator:

Marjorie Jenkins, MD, MEdHP, FACP
FDA, OC/OWH

Panelists:

Rajanikanth Madabushi, PhD 
FDA, CDER
Slides

Barbara Buch, MD
FDA, CBER

Vasum Peiris, MD, MPH, FAAP, FACC, FASE
FDA, CDRH
Slides

Victor Crentsil, MD
FDA, CDER
Slides

Virginia Miller, PhD, MBA
Mayo Clinic
Slides

Jonca Bull, MD
PPD
Slides

10:25-10:40 am

Break

Session 2: Heterogeneity of Treatment Effect Considerations in Design and Analysis
Introduction: Mark Rothmann, PhD (FDA, CDER)

10:40-11:00
 

Subgroup Identification: The Hardest Problem There Is 

Stephen Ruberg, PhD
Analytix Thinking

11:00-11:20

 

Decomposition Models as a Framework for Thinking about Heterogeneity of Treatment Effects 

William Crown, PhD
Optum Labs
 

11:20 am -12:30 pm






























 

Panel Discussion































 

Moderator:

Mark Rothmann, PhD
FDA, CDER

Panelists:

Thomas Permutt, PhD
FDA, CDER
Slides

Thomas Louis, PhD
Johns Hopkins University
FDA, CDER
Slides

John Scott, PhD, MA
FDA, CBER
Slides

Jodi Segal, MD, MPH
Johns Hopkins University
Slides

Heng Li, PhD
FDA, CDRH
Slides

Stephen Ruberg, PhD
Analytix Thinking
Slides

William Crown, PhD
Optum Labs

12:30-1:30 pm

Lunch

Session 3: Communicating Heterogeneity of Treatment Effects to Key Stakeholders
Introduction: Martin Mendoza, PhD (FDA, OC/OMH)

1:30-1:50 pm
 

Communicating HTE Across Populations: Challenges and Opportunities

John Whyte, MD, MPH
WebMD

1:50-2:55 pm

































 

Panel Discussion

































 

Moderator:

John Whyte, MD, MPH
WebMD

Panelists:

Iris Masucci, PharmD
FDA, CDER
Slides

Catherine Spong, MD
University of Texas, Southwestern
Slides

Cindy Geoghegan
Patients and Partners
Slides

David Atkins, MD, MPH
Dept. of Veterans Affairs
Slides

Gene Pennello, PhD, MS
FDA, CDRH
Slides

Daniel Canos, PhD, MPH Centers for Medicare and Medicaid Services
Slides

Paula Rausch, PhD, RN
FDA, CDER/OCOMM
Slides

2:55-3:15 pm

Break

Session 4: HTE from the Perspective of the Patient-Centered Outcomes Research Institute (PCORI)

3:15-4:00 pm

 

 












 

 




 

PCORI: How Heterogeneity of Treatment Effect Informs What We Do

Using Group Data to Treat Individuals: Understanding HTE in the Age of Precision Medicine and Patient Centered Care




 

Moderator:

Ravi Varadhan, PhD, PhD
Johns Hopkins University

Presenters:

Evelyn Whitlock, MD, MPH
PCORI

David Kent, MD, MS
Tufts University

Discussant:

Peter P. Stein, MD
FDA, CDER
Slides 

4:00-4:20 pm












 

Final Thoughts by Keynote Speakers and Session Moderators




 






 

Robert Temple, MD
FDA, CDER

Scott Zeger, PhD
Johns Hopkins University

Marjorie Jenkins, MD, MEdHP, FACP
FDA, OC/OWH

Mark Rothmann, PhD
FDA,CDER

John Whyte, MD, MPH
WebMD

4:20-4:30 pm

 

Summary/ Wrap-up

 

Ravi Varadhan, PhD, PhD
Johns Hopkins University
 

 

CONTACT

For questions and concerns, please email Jamie Heyward at jheywar1@jhu.edu or FDA at FDA-JHUHTE_symposium@fda.hhs.gov.

More information can be found on the FDA webpage for the symposium here:  

https://www.fda.gov/ScienceResearch/SpecialTopics/RegulatoryScience/ucm611410.htm.