Optimizing Pediatric Trials to Generate High Quality Data

Pediatric clinical trials present formidable challenges that distinguish them from adult research: obtaining meaningful consent and assent from children who didn't choose to participate, implementing age-appropriate clinical outcome assessments across developmental stages, managing the highest placebo response rates observed in any therapeutic area, and navigating heightened ethical scrutiny for this vulnerable population. These complexities directly impact data quality and regulatory acceptance, yet therapeutic products licensed for adults are routinely used in pediatric populations without sufficient safety, dosing, or efficacy data.

Dr. Joan Busner, Clinical Vice President for Pediatrics and Orphan Diseases at Signant Health, provides strategic guidance for sponsors navigating pediatric drug development. With over 35 years as an academic psychiatric researcher, service as Principal Investigator for 49 clinical trials, and chair of the ISCTM Orphan Diseases Working Group, Dr. Busner brings unparalleled expertise in addressing pediatric trial complexities.

Critical success strategies covered:

• Designing age-appropriate consent/assent processes that engage children as empowered research partners
• Implementing clinical outcome assessments validated for pediatric populations with appropriate observer-reported versions
• Applying evidence-based training approaches that establish consensus and standardization among raters in rare disease trials
• Balancing protocol requirements with children's cognitive/physical capabilities and visit length tolerances
• Managing caregiver dynamics while ensuring child understanding and authentic participation

Watch below to discover effective methodologies for addressing unique challenges in pediatric clinical trials.