Unified Program for Therapeutics in Children

ABSTRACT The pipeline for prophylactics and therapeutics that target pediatric diseases is sparse compared to that of adults. A limited pool of pediatric physician-scientists knowledgeable in the myriad of factors that create this translational divide compromises the development of promising therapies. The primary goal of the Unified Program for Therapeutics in Children (UPTiC) has been to train early-career pediatric physician-scientists with strong academic potential to lead the discovery and development of novel therapeutics and prophylactics for children and be thought-leaders in the field of pediatric drug development. The program trains subspecialty pediatric fellows in 3 tracks that span the full pipeline of drug development: 1) discovery-based research (preclinical/translational pediatric drug development), 2) pediatric clinical trials and prospective cohort studies, and 3) outcomes/real world evidence. The program covers 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. The training of these pediatric research fellows features a combined effort of 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 is led by a team of multi-institutional, multi-disciplinary, well-funded, and experienced faculty members, including 44 full- time Duke and UNC faculty members in Pediatrics and other basic, translational, and clinical science departments. This renewal application requests support for four postdoctoral pediatric trainees (two new fellows per year, 2 years of support each). Trainees are drawn from the national pool of pediatric fellow applicants at Duke and UNC through early engagement with fellow applicants and program directors and direct outreach to pediatric research and clinical organizations. In its initial cycle, the program trained 17 pediatric subspecialty “Katz-Denny” fellows who studied new therapeutic approaches including small molecules, biologics and devices, sought new biomarkers, and discovered new biological underpinnings of diseases in children. Katz-Denny fellows have been well-published, and the majority have obtained grant funding during and/or beyond the period of training. As the program evolves, two primary goals for the next cycle include expanding cross-institutional programming and research engagement across Duke and UNC, and enhancing programmatic diversity. Fulfillment of the program's objectives will 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 need for innovations, basic discoveries, and clinical translation of novel pediatric prevention and treatment strategies to improve the health of the youngest and most vulnerable members of society.