Pediatric Clinical Trials: Past, Present and Future

DESCRIPTION OF SESSION

“Children are not just small adults” is a common refrain, but less commonly known is what that means, when and why it arose, and how it has impacted clinical research and drug development. The journey towards inclusion of pediatric participants in clinical trials is littered with regulatory carrots and sticks, such that we have shifted from protecting children from research to protecting them through research, and the global regulatory landscape that contribute to this continues to shift and grow. There is still a long way to go. In Canada, 50-80% of drugs are used off-label in children. To encourage pediatric drug development, Canada is currently conducting a 2 year pilot (2024-2026) on a pediatric study submission policy that is modeled after those that have been in effect in the USA and Europe for the past couple of decades. These continue to develop, with notable advances such as the RACE for Children Act that came into effect in the USA in 2020, and setbacks such as the recent surprising cancellation in the USA of provisions that support pediatric research, particularly for oncology and rare diseases. Here, I will explain what “children are not small adults” means from an historical, regulatory, physiological, and ethical perspective. Highlighting key regulatory milestones, current developments, and future needs along with discussion of practical aspects of pediatric trial planning will be accomplished with reference to the ethical considerations that underpin pediatric clinical research. Common assumptions will be touched on, and topics such as consent/assent, inclusion of adolescents, pediatric medical trauma, and pediatric plans will be considered from an ethical and practical perspective.