The development pipeline for prophylactics and therapeutics that target pediatric diseases is sparse compared to that of adults. A gap in the understanding of pediatric physician-scientists regarding how to span this translational divide compromises the development of promising therapies. The primary goal of this Unified Program for Therapeutics in Children (UPTiC) is to train young pediatric physician-scientists with strong academic potential to lead the development of novel therapeutics and prophylactics for children and be thought- leaders in the field of pediatric drug development. The program will train subspecialty pediatric fellows in 3 tracks that span the full pipeline of clinical development: 1) discovery-based research (preclinical/translational pediatric drug development), 2) clinical pediatric trials, and 3) outcomes/real world evidence. The program will cover three major training aims, including: 1) individualized didactic and degree-oriented course work in pediatric drug development, 2) mentored training in preclinical, clinical trials, and outcomes research, and 3) grant-writing training and mock review. The training of these pediatric research fellows will be through a combined effort bringing together the Duke University School of Medicine, including the Department of Pediatrics and the Duke Clinical Research Institute, and the University of North Carolina at Chapel Hill, including the Department of Pediatrics, School of Pharmacy, and School of Public Health. UPTiC will be led by a team of multi- university, multi-disciplinary, well-funded, and experienced faculty members, including 39 full-time Duke and UNC faculty members in Pediatrics and other basic, translational, and clinical science departments. This application requests support for six postdoctoral pediatric trainees (three new fellows per year, 2 years of support each). Trainees will be nationally recruited through established pediatric clinical trials networks led by the Duke Clinical Research Institute and Duke/UNC pediatric fellowship program directors and subsequently drawn from the pool of pediatric fellows at Duke and UNC. Individuals appointed to the training program will be named Katz- Denny Fellows. The training will center on research ongoing in the various faculty members' research groups across the Duke Medical Center and the University of North Carolina and a joint didactic program focusing on the therapeutic development pipeline for children. Upon completion of this training program, these pediatrician- scientists will be capable of not only leading translational and clinical pediatric-focused therapeutic development research, but also mentoring the next generation of pediatricians in the fundamentals of pediatric clinically- oriented research. Fulfillment of the program's objective would directly or indirectly address two imperatives: 1) the need for more full-time academic pediatric physician-researchers and mentors in medical schools throughout the country and 2) the unmet need for innovations and clinical translation of novel pediatric prevention and treatment strategies to improve the health of the youngest and most vulnerable members of our society.
The pipeline of novel therapeutics for children is extremely limited compared to that of adults due to several unique features of a pediatric population, including safety, regulatory and ethical concerns, low frequency of pediatric diseases, and age-dependent pharmacokinetic factors. There is a paramount need, therefore, for pediatric physician-scientists who are knowledgeable in pediatric drug discovery and clinical trial design. The proposed Unified Program for Therapeutics in Children (UPTiC) will train pediatric physician-scientists to lead the development, implementation, and evaluation of novel prophylactics and therapeutics for pediatric populations to improve the health and well-being of children.