Research and innovation are critical to improving the health and well-being of the world's children; clinical trials yield important information on a medical product's safety, dosing, and effectiveness. While the prescribing information available to pediatric providers has substantially improved, approximately 50% of medicines still do not have data on their labels to guide their appropriate use in children. Regulatory bodies have recently taken measures to ensure, if drugs have a potential pediatric indication, that the safety and efficacy clinical trials include the pediatric population. However, there are significant challenges with pediatric trials, including study design and feasibility, suitable formulations, and patient recruitment and retention. The authors propose that these challenges can be addressed by actively involving young people and families in study design so their insights can inform successful trial implementation. As the volume of pediatric research increases, there needs to be a concomitant effort for researchers and providers to seek input from patients and families in the development of their work, which can lead to a transformation of the cultural and regulatory environment of pediatric medicine. The benefits of patient and public involvement in research have been well documented. In 2006, the National Institute for Health Research Clinical Research Network: Children started its first Young Persons' Advisory Group in an effort to include young people in the design and delivery of pediatric research. Since their initial efforts, the youth advisory concept has grown into a global effort known as the International Children's Advisory Network (iCAN). This article describes the foundational building blocks of iCAN and provides tools to investigators and practitioners in an effort to increase the instances of children and families being invited to share their unique point of view in medicine.