Optimizing Pediatric Trials To Generate High-Quality Data For Regulatory Decision-Making 

Conducting clinical trials involving pediatric participants presents distinctive hurdles. These trials frequently exhibit notably elevated placebo response rates. The intricacies intensify due to the existence of varying age-appropriate iterations and observer-based versions when assessing clinical outcomes. Additionally, obtaining both consent from guardians and assent from the children themselves becomes a more intricate endeavor.

Drawing from her extensive expertise in the domain of designing and facilitating clinical trials for pediatric and rare diseases, Dr. Joan Busner, the chair of ISCTM's Orphan Diseases Working Group, offers illuminating insights into these challenges.