We discuss the challenges and opportunities we face at The Hospital for Sick Children in Toronto, ON,Canada in conducting clinical investigation in children. Three areas are discussed: (i) the program structure of the Research Institute; (ii) the Toronto Centre for Phenogenomics as a common platform for preclinical studies across the university campus; and (iii) evolving clinical research infrastructure. Because preclinical and clinical investigation are becoming increasingly resource-intensive, we are witnessing a trend toward functional amalgamation of research teams, centralization of major experimental facilities, and the expansion of research infrastructure at local and national levels. Although these trends are common to clinical research in all age groups, pediatric investigation is further challenged by its relative paucity of research-related human resources, and patient numbers at local and even national levels. To promote drug research in children, these organizational changes in the research framework need to be seen on a much broader international level.
