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The Intersection of Science, Ethics, and Participant Collaboration in Pediatric Rare Disease Product Development
Session Chair(s)
Donna Snyder, MD
Executive Physician
WCG, United States
Experts in pediatric rare disease product development, pediatric ethics and patients/advocates discuss designing studies to best benefit the patient, and how patients/caregivers assess risks and benefits in early phase trials.
Learning Objective : Discuss the ethical and scientific considerations for designing studies for rare diseases that impact children, including the level of evidence needed to initiate early phase trials; Discuss how patients and caregivers weigh the risks of early-phase studies against the benefits of a potential treatment for their rare disease, especially when there are limited to no other options.
Speaker(s)
Ethical Considerations for Including Children in Rare Disease Trials
Donna Snyder, MD
WCG, United States
Executive Physician
Update from ORPURM/DRDMG
Mehul Desai, MD
FDA, United States
Medical Reviewer, ORDPURM/DRDMG, OND, CDER
Patient Perspective
Mark Dant
The Ryan Foundation for Rare Disease Research, United States
Volunteer Executive Director, The Ryan Foundation
Patient Perspective
Ryan Dant
The Ryan Foundation for Rare Disease Research, United States
Patient Advocate
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