The Patient Voice: Is Anyone Listening?
Course Director: Jeanine R. Jarnes, PharmD, MSc, BCOP, BCPS
Sunday, February 4, 2024
4:00 – 5:45 PM
WORLDSymposium presented an exciting new program on Sunday, February 4, 2024 from 4:00 – 5:45 PM just prior to the 20th Annual WORLDSymposium opening event, “Be the Catalyst“. This new session: The Patient Voice: Is Anyone Listening? explored measures that industry and other researchers have taken to better understand what is most important to patients in terms of clinical trial endpoints, unmet needs, and goals of therapies.
The goal of this new 1.75-hour non-CE session was to enhance awareness of the importance of listening to what therapy outcomes and unmet needs are most important to patients and their families, and incorporating those outcomes into clinical trial design and post-trial marketing follow-up. This session was recorded and offered as part of the On Demand program through March 14, 2024.
Preliminary Agenda
| 4:00 PM | Welcome (Jeanine R. Jarnes) |
| 4:01 PM | Introductory Remarks from Family Perspective (Alan Finglas) |
| 4:05 PM | Earlier is better: Updates on a Phase I clinical trial of in utero enzyme replacement therapy (Tippi MacKenzie) |
| 4:15 PM | From Commitment to Co-Creation: Over 30 Years of Partnership with the Patient Community (Danielle Dong) |
| 4:25 PM | As Unique as You: A Personalized Approach to Supporting the Patient’s Journey (Heather Park) |
| 4:35 PM | The voice of caregiver study in MPS II and other notable caregiver observations made in JCR’s clinical MPS programs (Sairei So) |
| 4:45 PM | Incorporating the patient/caregiver voice in rare disease drug development using the biopsychosocial model (Heather Lau) |
| 4:55 PM | Best practices in patient education: How to create tools that meet community needs (Nita Patel) |
| 5:05 PM | Embracing the silence: Taking listening to a new level. Mindfully rare mental health in the Fabry community. (Andres Trevino) |
| 5:15 PM | Improving Therapeutic Options in Cystinosis Through Partnerships with Patients, Caregivers and Healthcare Providers (Andrea Atherton) |
| 5:25 PM | Closing Remarks from Family Perspective (Cara O’Neill) |
| 5:35 PM | Q&A and Panel Discussion: Moderated by Matthew Ellinwood |
| 5:45 PM | Adjourn |
Program Chair:
Jeanine R. Jarnes, PharmD, BCOP, BCPS, MSc
Assistant Professor, Department of Pediatrics
Pharmacotherapy for Inherited Metabolic Diseases
Advanced Therapies Department
College of Pharmacy, Experimental and Clinical Pharmacology
University of Minnesota
Minneapolis, MN, United States
Faculty
Alan Finglas
MSD Action Foundation
Tippi MacKenzie
Professor of Surgery
Division of Pediatric Surgery
UCSF School of Medicine
University of California, San Francisco
Danielle Dong
Rare Diseases Scientific Advocacy Lead, Global Medical Affairs
Sanofi
Heather Park
Head, US Rare Diseases Patient Support Services
Sanofi
Sairei So
Director, International Project Unit
JCR Pharmaceuticals
Heather Lau
Executive Director, Global Clinical Development
Ultragenyx
Nita Patel
Executive Director, Patient & Professional Advocacy
Amicus Therapeutics
Andres Trevino
Patient Advocacy Director
Chiesi USA, Inc.
Andrea Atherton
Regional Medical Director
Amgen
Cara O’Neill
Chief Science Officer & Co-Founder
Cure Sanfilippo Foundation
Matthew Ellinwood
Chief Scientific Officer
National MPS Society
Thank you to the sponsors for this session
