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

Wednesday, November 28, 2018, 8:15 a.m. - 4:30 p.m. ET

Past Event

8:15 A.M. – 4:30 P.M. (REGISTRATION BEGINS AT 7:30 A.M.)
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:

Major sources of HTE including subpopulations (e.g., sex, race, pediatric and geriatric populations)
HTE considerations in study design and statistical analysis
How HTE may be communicated to stakeholders via various avenues such as drug trial snapshots and labeling.

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 and 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 Barbara Buch, MD FDA, CBER Vasum Peiris, MD, MPH, FAAP, FACC, FASE FDA, CDRH Victor Crentsil, MD FDA, CDER Virginia Miller, PhD, MBA Mayo Clinic Jonca Bull, MD PPD
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 Thomas Louis, PhD Johns Hopkins University FDA, CDER John Scott, PhD, MA FDA, CBER Jodi Segal, MD, MPH Johns Hopkins University Heng Li, PhD FDA, CDRH Stephen Ruberg, PhD Analytix Thinking 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 Catherine Spong, MD University of Texas, Southwestern Cindy Geoghegan Patients and Partners David Atkins, MD, MPH Dept. of Veterans Affairs Gene Pennello, PhD, MS FDA, CDRH Daniel Canos, PhD, MPH Centers for Medicare and Medicaid Services Paula Rausch, PhD, RN FDA, CDER/OCOMM
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
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.

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