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Wednesday, June 27 • 8:00am - 9:15am
#303: Regulatory and Ethical Considerations with Placebo Administration Using a Central Venous Access Device in a Pediatric Trial

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Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-18-624-L04-P; CME 1.25; IACET 1.25; RN 1.25

This session presents a case study of the review of a pediatric protocol by a federal panel under 21 CFR 50.54 (Clinical investigations not otherwise approvable that present an opportunity to understand, prevent, or alleviate a serious problem affecting the health or welfare of children.). The panel review addressed the ethics of the administration of placebo through a central venous access device in pediatric patients. Parent and patient perspectives were instrumental in informing the panel’s decision and will be highlighted by a family presentation at the session.

Learning Objectives

Describe the federal regulations and ethical considerations when using a central venous access device to administer a placebo in a pediatric clinical trial; Discuss prospect of direct benefit, component analysis, and research risk analysis; Recognize how patient engagement and burden can impact risk analysis decisions; Discuss a federal ethics panel review of a protocol referred to the FDA.


Robert Nelson


Additional Safeguards for Children in Research and Protocol Review Under 21 CFR 50.54
Donna L. Snyder, MD

Sponsor Perspective
Jonathan Lu, MD, PhD

Principle Investigator Perspective
Perry Shieh, MD, PhD

Parent Perspective
Brett Bullers

Parent Perspective
Erin Bullers

Patient Perspective
Nicholas Bullers

avatar for Erin Bullers

Erin Bullers

Parent, Parent
Mother of two and caregiver to her 15 yr old son Nicholas diagnosed with Duchenne Muscular Dystrophy at the age of four. Over the last 10 yrs, she has spent countless hours researching DMD, and connecting with doctors, researchers, and specialists. Erin and Nicholas have traveled... Read More →
avatar for Brett Bullers

Brett Bullers

Brett Bullers is a husband and father of two including 14 year old Nicholas who suffers with Duchenne muscular dystrophy (DMD). Brett has been active in searching for the best care available to treat DMD as well as helping Nicholas pursue trials and studies that may advance a cure... Read More →
avatar for Nicholas Bullers

Nicholas Bullers

Patient, NA
Nicholas Bullers is a 14 year old living with Duchenne muscular dystrophy (DMD), an incurable progressive muscle disease in which there is no cure. Duchenne affects 1 out of every 3,500 boys worldwide. Nicholas participated in the DMD Imaging Study at the age of seven for three years... Read More →

Jonathan Lu

Medical Director, Sarepta Therapeutics
Jonathan Lu is a Medical Director in Clinical Development at Sarepta Therapeutics. Dr Lu is the lead physician for the ESSENCE trial.
avatar for Robert Nelson

Robert Nelson

Senior Director, Pediatric Drug Development (CHILD), Johnson & Johnson, United States
Robert “Skip” Nelson, MD, MDiv, PhD. is currently Senior Director, Pediatric Product Development and Pediatric Strategy Lead in Immunology at Johnson & Johnson. Previously, he was the Deputy Director and Senior Pediatric Ethicist in the Office of Pediatric Therapeutics, Office... Read More →

Perry Shieh

Neuromuscular Medicine Specialist, UCLA Medical Center
Perry Shieh, MD, PhD, is Associate Professor of Neurology at UCLA. He received his MD and his PhD in Neuroscience from Johns Hopkins, residency training in neurology at Stanford, and fellowship training Neuromuscular Diseases at Brigham and Women's Hospital. His interests are muscular... Read More →
avatar for Donna Snyder

Donna Snyder

Senior Pediatric Ethicist and Team Leader, Office of Pediatric Therapeutics, OC, FDA
Donna L. Snyder, MD. MBE is the Senior Pediatric Ethicist and Team Leader in the Office of Pediatric Therapeutics (OPT) in the Office of the Commissioner (OC) at the US Food and Drug Administration (FDA). She is a standing member of the FDA Pediatric Review Committee (PeRC) and the... Read More →

Wednesday June 27, 2018 8:00am - 9:15am EDT
Room 258C Boston Convention and Exhibition Center 415 Summer Street, Boston, MA 02210 USA
  02: ClinTrials -ClinOps, Session