Cincinnati Training Program in Pediatric Clinical and Developmental Pharmacology

描述（由申请人提供）：本申请申请儿科临床和发展药理学机构国家研究服务奖（T32），要求支持一个新的博士后培训计划，该计划将培训下一代儿科临床研究人员，以在开发创新，与临床和发育药理学相关的高影响力方法，将改善儿童药物的正确使用，以加强儿科治疗和儿童的相关健康结果。许多用于儿童的药物在儿科人群中的使用尚未经过科学评估，要么未经许可使用，要么以标签外方式使用。此外，专门用于治疗儿童疾病的药物很少。辛辛那提儿童医院医学中心（CCHMC）的儿科临床和发育药理学研究培训计划（PCDP-RTP）是专门为解决这一关键需求而设计的。该项目基于儿科临床药理学部门，该部门是儿科部门和辛辛那提大学医学院的联合单位。这个T32儿科临床和发育药理学研究培训项目还包括与辛辛那提大学James L. Winkle药学院和药理学与细胞生物物理学和数学科学系的紧密合作。PCDP-RTP的指导教师来自辛辛那提大学儿科系的12个部门和4个部门。教师在一系列与儿科疾病领域的临床、转化和基础科学研究相关的领域拥有专业知识，包括依从性、生物信息学、生物标志物开发、生物统计学和流行病学、比较有效性、临床结果、临床试验、流行病学、遗传学/基因组学和基因表达、临床和发育药理学、质量改进和系统生物学/药理学。T32培训项目具有创新性，并与项目公告中概述的目标保持一致，因为：1)通过与所有主要儿科亚专科的持续研究合作，重点关注多种儿科人群的早期和后期研究；2)涉及创新定量方法的应用和开发，如基于生理学的PK/PD建模和模拟，以及通过我们的多学科药物计量学核心进行疾病进展分析；3)通过个性化医学和遗传药理学项目，嵌入到机构和学术健康中心的药物遗传/基因组学研究工作中；4)与生物统计学和流行病学、生物信息学、卫生服务和成果研究紧密结合；5)作为NICHD儿科药理学研究单位网络13个站点之一，可以依靠超过十年的经验。对受训者的支助期一般为两年，但也可以提供额外的支助期，特别是如果这将使他们在随后建立独立的研究事业方面更具竞争力的话。PCDP-RTP得到了辛辛那提大学学术健康中心的核心设施和各种学术项目的良好支持，其中包括辛辛那提医学中心、UCCOM、詹姆斯·l·温克尔药学院和其他几个联合学院。我们相信，PCDP-RTP提供了一个独特的资源，将打开一个重要的新途径，扩大有才华的年轻临床研究人员的库与儿科治疗的职业兴趣。拟议的培训计划将使他们获得一个精细化的视野，将临床和发展药理学的最新原则应用于健全的临床研究和基于证据的临床实践，以改善儿科健康结果。

DESCRIPTION (provided by applicant): This application for an institutional National Research Service Award (T32) in Pediatric Clinical and Developmental Pharmacology requests support for a new postdoctoral training program that will train the next generation of pediatric clinical investigators to assume leadership roles in developing innovative, high impact clinical and developmental pharmacology related approaches that will improve proper use of medicines in children to enhance pediatric therapeutics and related health outcomes of children. Many medicines used in children have not been scientifically evaluated for use in the pediatric population and are either used unlicensed or in an off-label manner. In addition, few medicines are developed specifically to treat childhood diseases. The Pediatric Clinical and Developmental Pharmacology Research Training Program (PCDP-RTP) at Cincinnati Children's Hospital Medical Center (CCHMC) is designed specifically to address this critical need. The program is based in the Division of Pediatric Clinical Pharmacology, which is jointly a unit within the Department of Pediatrics and in the University of Cincinnati, College of Medicine. This T32 Pediatric Clinical and Developmental Pharmacology Research Training Program also involves strong collaborations with the James L. Winkle College of Pharmacy and the Departments of Pharmacology & Cell Biophysics and Mathematical Sciences at the University of Cincinnati. Faculty advisors for the PCDP-RTP come from 12 divisions within the Department of Pediatrics and four Departments at the University of Cincinnati. Faculty have expertise in an array of fields relevant to clinical, translational and basic science research of pediatric disease areas that include adherence, bioinformatics, biomarker development, biostatistics and epidemiology, comparative effectiveness, clinical outcomes, clinical trials, epidemiology, genetics/genomics and gene expression, clinical and developmental pharmacology, quality improvement, and systems biology/pharmacology. The T32 training program is innovative and well aligned with the objectives outlined in the program announcement because: 1) it has a focus in early and later phase studies in multiple and diverse pediatric populations through ongoing research collaborations with all major pediatric subspecialties; 2) involves the application and development of innovative quantitative methodologies such as physiologically based PK/PD modeling and simulation and disease progression analysis through our multidisciplinary Pharmacometrics Core; 3) is embedded in the institutional and Academic Health Center's pharmacogenetic/genomics research endeavors through the Personalized Medicine and Genetic Pharmacology Programs; 4) is closely integrated with biostatistics and epidemiology, bio-informatics, and health services and outcomes research; 5) can rely on over ten years of experience as one of 13 sites of the NICHD Pediatric Pharmacology Research Unit Network. The typical period of support for trainees will be two years, though an additional period of support may be provided, especially if it will make them more competitive for subsequently establishing an independent research career. The PCDP-RTP is well supported by core facilities and a variety of academic programs at the University of Cincinnati's Academic Health Center, which includes CCHMC, UCCOM, the James L. Winkle College of Pharmacy and several other allied colleges. We believe that the PCDP-RTP provides a unique resource that will open an important new avenue to enlarge the pool of talented young clinical investigators with a career interest in pediatric therapeutics. The proposed training program will allow them to acquire a refined vision for applying state of the art principles of clinical and developmental pharmacology to sound clinical research and evidence-based clinical practice to improve pediatric health outcomes.