Back To Schedule
Wednesday, June 27 • 2:00pm - 3:15pm
#365: Engaging the Rare Disease Community to Design Clinical Trials

Sign up or log in to save this to your schedule, view media, leave feedback and see who's attending!

Component Type: Session
Level: Basic
CE: ACPE 1.25 Knowledge UAN: 0286-0000-18-662-L04-P; CME 1.25; IACET 1.25; RN 1.25

In this session, participants will hear several recent examples of how to best engage with different patient communities with respect to clinical trial design.

Learning Objectives

Discuss examples of how to best engage with different patient communities with respect to clinical trial design.


Scott Schliebner, MPH


Nothing About Us, Without Us: Best Practices for Engaging With the Rare Disease Patient Community
Scott Schliebner, MPH

Enrollment: Using Patient Advocacy Groups to Design a Better Trial and Manage Studies in Rare Diseases
Bruce Wynne, PharmD

Early Engagement with the Kabuki Syndrome Patient Community to Understand Clinical Phenotype and Unmet Needs
Deborah Hartman, PhD


Deborah Hartman

Vice President, Global Program Leader, Takeda Pharmaceuticals International, Inc.
Deborah Hartman is VP Global Program Leader in the Neuroscience Area at Takeda Pharmaceuticals in Cambridge, MA. Deborah leads early clinical development programs focused on new treatments for rare disease indications. Prior to Takeda, Deborah held R&D leadership roles at AstraZeneca... Read More →
avatar for Scott Schliebner

Scott Schliebner

Senior Vice President, Center for Rare Diseases, PRA Health Sciences
Scott Schliebner is a clinical strategist with a 20+ year background in clinical development specializing in rare diseases and orphan drug development. His experience encompasses all trial phases; a broad variety of study designs; interventional and observational studies; across a... Read More →
avatar for Bruce Wynne

Bruce Wynne

Director, Clinical Operations Lead, CSL Behring
I have been with CSL Behring for 6 years working & managing clinical trials in rare diseases. I currently lead the Immunology & Acq. Bleeding operations group CSL. I spent 12 years at Sanofi managing international studies, interacting with study sites and monitoring. I graduated with... Read More →

Wednesday June 27, 2018 2:00pm - 3:15pm EDT
Room 151AB Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  05: Patient Engagement, Session