The Art of Decentralizing Pediatric Rare Disease Studies: Clinical Trials Suited for Daily Life

Pediatric rare disease trials combine rarity of disease with a restricted and geographically-dispersed population of vulnerable subjects for research, creating unique challenges for study design and execution. Recruitment is challenging, as patients, parents, and caregivers need to juggle school schedules, work responsibilities, and family obligations to accommodate study participation.

Incorporating decentralized clinical trial (DCT) strategies can help sponsors attract patients and increase access, enrollment, and retention by limiting site visits and facilitating participation for those who might not otherwise be able—or willing—to travel to distant sites or specialized centers.

In this webinar, you will learn:

  • Challenges of conducting clinical research in pediatric rare diseases
  • Potential applications of DCT strategies in pediatric rare disease studies
  • Risks to applying DCT strategies in these clinical trials
  • Key considerations when operationalizing pediatric rare disease studies with decentralized elements
  • Future trends in decentralization for pediatric rare disease studies
Meet Our Speakers:

Betsy Reid
Senior Vice President, Pediatrics

Adam Bloomfield, MD, FAAP
Executive Director, Medical Affairs, Rare Diseases and Pediatrics