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Monday, June 17 • 11:00am - 12:00pm
#105: The Intersection of Science, Ethics, and Participant Collaboration in Pediatric Rare Disease Product Development

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Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-24-519-L04-P; CME 1.00; RN 1.00

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 Objectives

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.

Chair

Donna Snyder, MD

Speaker

Ethical Considerations for Including Children in Rare Disease Trials
Donna Snyder, MD

Update from ORPURM/DRDMG
Mehul Desai, MD

Patient Perspective
Mark Dant

Patient Perspective
Ryan Dant


Speakers
avatar for Donna Snyder

Donna Snyder

Executive Physician, WCG, United States
Donna Snyder, MD, MBE is the Executive Physician of WCG where she contributes subject matter expertise and operational guidance across WCG to ensure that research will be conducted to the highest ethical standards. Prior to WCG, Dr. Snyder served as the leader and Senior Pediatric... Read More →
avatar for Mark Dant

Mark Dant

Volunteer Executive Director, The Ryan Foundation, The Ryan Foundation for Rare Disease Research, United States
Mark is the Founder and Volunteer Executive Director of the Ryan Foundation and is the former Board Chair of the of the EveryLife Foundation for Rare Diseases. Mark and his family founded the Ryan Foundation in 1992 shortly after their only child Ryan was diagnosed with MPS I. Since... Read More →
avatar for Ryan Dant

Ryan Dant

Patient Advocate, The Ryan Foundation for Rare Disease Research, United States
Ryan Dant is a long-time patient advocate diagnosed at the age of three with Mucopolysaccharidosis type 1 (MPS 1), a rare lysosomal storage disorder. When doctor’s predicted Ryan’s life to be cut short due to the disease, Ryan’s parents, Mark and Jeanne, spearheaded the funding... Read More →
avatar for Mehul Desai

Mehul Desai

Medical Reviewer, ORDPURM/DRDMG, OND, CDER, FDA, United States
Mehul Desai M.D. is a medical officer in the Division of Rare Diseases and Medical Genetics (DRDMG), Office of New Drugs (OND), Center for Drug Evaluation Research (CDER), with the US Food and Drug Administration (FDA). Dr. Desai has clinical development experience in both the pharmaceutical... Read More →

Monday June 17, 2024 11:00am - 12:00pm PDT
Room 17AB San Diego Convention Center 111 W Harbor Drive, San Diego, CA 92101 USA
  05: Patient-Focused Drug Dev, Session |   09: Regulatory, Session |   02: ClinTrials -ClinOps, Session