Unified Program for Therapeutics in Children (UPTiC)

与儿科疾病相比，针对儿科疾病的药物和治疗药物的开发管道是稀疏的。 与成人相比。儿科医生-科学家关于如何跨越这一理解的差距 翻译鸿沟损害了有前途的疗法的发展。这个统一的主要目标 儿童治疗学计划（UPTiC）旨在培养年轻的儿科医生-科学家， 学术潜力，导致新的治疗和儿童药理学的发展，并被认为- 儿科药物开发领域的领导者。该计划将在3个轨道上培训儿科专科研究员 涵盖临床开发的整个管道：1）基于发现的研究（临床前/转化 儿科药物开发），2）临床儿科试验，和3）结果/真实的世界证据。的 该计划将涵盖三个主要培训目标，包括：1）个性化教学和学位导向课程 从事儿科药物开发工作，2）指导临床前、临床试验和成果研究方面的培训， （3）赠款写作培训和模拟审查。这些儿科研究员的培训将通过一个 联合努力汇集了杜克大学医学院，包括儿科系 和杜克临床研究所，以及查佩尔山的北卡罗来纳州大学，包括 儿科学系、药学院、公共卫生学院。UPTIC将由一个多人团队领导， 大学，多学科，资金充足，经验丰富的教师，包括39名全职杜克和 儿科和其他基础，转化和临床科学部门的教师。这 申请要求支持6名博士后儿科学员（每年3名新研究员，支持2年 每个）。受训者将通过杜克领导的已建立的儿科临床试验网络在全国范围内招募 临床研究所和杜克/杜克大学儿科奖学金项目负责人，随后从 杜克和麻省理工的儿科研究员。被任命参加培训计划的个人将被命名为卡茨- 丹尼·费罗斯培训将集中在各个教职员工研究小组正在进行的研究上 在杜克医学中心和北卡罗来纳州大学，以及一个联合教学计划，重点是 儿童的治疗发展管道。在完成这个培训项目后，这些儿科医生- 科学家不仅能够领导以儿科为重点的转化和临床治疗开发， 研究，而且还指导下一代儿科医生的儿科临床基础- 导向研究。实现该计划的目标将直接或间接地解决两个迫切需要：1） 需要更多的全职学术儿科医生研究人员和导师在医学院在整个 2）对创新和新型儿科预防的临床转化的需求未得到满足， 治疗战略，以改善我们社会中最年轻和最脆弱成员的健康。

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.