Unified Program for Therapeutics in Children (UPTiC)

相比之下，针对儿科疾病的预防和治疗药物的开发流水线是稀少的 对成年人的影响。儿科内科医生和科学家对如何跨越这一点的理解上的差距 转化性分化损害了有前景的治疗方法的发展。此统一解决方案的主要目标 儿童治疗学计划(UpTIC)旨在培养具有强大能力的年轻儿科医生和科学家 引领儿童新疗法和预防方法发展的学术潜力，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 这跨越了整个临床开发流程：1)基于发现的研究(临床前/转化型 儿科药物开发)，2)儿科临床试验，以及3)结果/真实世界证据。这个 计划将涵盖三个主要培训目标，包括：1)个性化、教学性和学位导向型课程 在儿科药物开发方面的工作，2)临床前、临床试验和结果研究方面的指导培训， (3)赠款撰写培训和模拟审查。对这些儿科研究员的培训将通过 联合努力，将杜克大学医学院，包括儿科 和杜克临床研究所，以及北卡罗来纳大学教堂山分校，包括 儿科、药学院和公共卫生学院。UpTIC将由一个由多名 大学，多学科，资金充足，经验丰富的教职员工，包括39名全职杜克大学和 北卡罗来纳大学儿科和其他基础、翻译和临床科学系的教职员工。这 申请申请支持6名儿科博士后学员(每年3名新研究员，2年支持 每个)。受训人员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 临床研究所和杜克大学/北卡罗来纳大学儿科奖学金计划主任，随后从 杜克大学和北卡罗来纳大学的儿科研究员。被任命参加培训计划的个人将被命名为Katz- 丹尼·费洛斯。培训将集中在各个教职员工研究小组中正在进行的研究 在杜克医学中心和北卡罗来纳大学，以及一个联合教学计划，重点是 儿童治疗发展流水线。在完成这个培训计划后，这些儿科医生- 科学家不仅能够领导以儿科为重点的翻译和临床治疗开发 研究，但也指导下一代儿科医生的儿科临床基础- 以研究为导向。计划目标的实现将直接或间接地解决两个当务之急：1) 需要更多全职学术儿科医生-研究人员和医学院的导师 国家和2)对新型儿科预防和临床翻译的创新和临床翻译的需求尚未得到满足 改善我国社会中最年轻和最脆弱成员的健康的治疗战略。

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.