Pediatric Pharmacology Research Unit Network

描述（由申请人提供）： UCSD/CHHC儿科药理学研究单位（PPRU）的总体目标是从事具有已证实或潜在活性的药物的研究，这些药物将导致婴儿和儿童的安全有效的治疗用途。这将通过仔细的合作计划和遵守FDA指南来实现，以便在PPRU研究的开发和实施过程中收集的药物文件可以提供足够的数据，用于以安全有效的剂量许可用于儿科适应症的新（或旧）药物。这项使命的很大一部分将与制药行业合作完成，因此所进行的研究的直接、切实的结果将是增加标记为儿科使用的药物数量，并将儿科标签扩展到所有儿科人群，包括新生儿，以便所有儿科患者都可以接受智能和合理的基于科学的治疗。PPRU赠款的范围是在UCSD和圣地亚哥儿童医院提供组织和运营基础设施，以实现以下具体目标：1。为新药和已上市药物的生物利用度、制剂、药物代谢、药代动力学、药效学、安全性和有效性研究提供场所。2.收集或促进儿童特定年龄药物标签所需的临床数据的积累。对广泛使用的非专利药物和新生儿使用的药物的研究将是高度优先事项。3.开展儿科药理学新方法和创新研究。感兴趣的具体领域包括但不限于：从成人研究中鉴定、开发、验证和/或外推诊断、预后、疗效、毒性和疾病活动的生物标志物; B）开发和验证非侵入性药效学测量; c）开发和/或调整儿科新的药代动力学-药效学建模技术（例如，基于机械和生理的药代动力学）; d）新分子方法的使用; e）新药物递送系统在儿科治疗中的应用。4.实施药物代谢酶（DME）、转运蛋白和受体的发育特征和遗传多态性研究，并描述表型-基因型相关性。5.在临床研究中应用药物基因组学和蛋白质组学工具。6.提供临床和发展药理学方面的培训和教育计划。

DESCRIPTION (provided by applicant): The overall objective of the UCSD/CHHC Pediatric Pharmacology Research Unit (PPRU) is to engage in studies of drugs with proven or potential activity which will lead to safe and effective therapeutic use in infants and children. This will be accomplished through careful collaborative planning and adherence to FDA guidelines so that drug files assembled during development and conduct of PPRU studies can provide sufficient data for licensing of new (or old) drugs for pediatric indications at safe, effective doses. A significant portion of this mission will be accomplished in collaboration with pharmaceutical industry so that a direct, tangible consequence of the studies performed will be to increase the number of drugs which are labeled for pediatric use and expand pediatric labeling to all pediatric populations, including newborns, so that all pediatric patients may receive intelligent and rational science-based therapy. The scope of the PPRU grant is to provide an organizational and operational infrastructure at UCSD and Children's Hospital of San Diego to implement the following specific aims: 1. To provide a locus for the conduct of studies in bioavailability, formulations, drug metabolism, pharmacokinetics, pharmacodynamics, safety and effectiveness of new drugs and drugs already in the market. 2. To gather or promote the accrual of necessary clinical data for pediatric age-specific labeling of drugs. Studies of widely used off-patent drugs and drugs used in the newborn population will be of high priority. 3. To carry out research on novel approaches and innovations in pediatric pharmacology. Specific areas of interest include, but are not limited to: a. Identification, development, validation and or extrapolation from adult studies of biomarkers of diagnosis, prognosis, efficacy, toxicity and of disease activity; b) Development and validation of non-invasive pharmacodynamic measurements; c) Development and/or adaptation of novel pharmacokinetic-pharmacodynamic modeling technology in pediatrics (e.g. mechanistic and physiologic based pharmacokinetics); d) Use of new molecular approaches; e)Applications of new drug delivery systems to pediatric therapy. 4. To implement studies of the developmental characteristics and genetic polymorphisms of drug metabolizing enzymes (DMEs), transporters, and receptors and describe phenotypic-genotypic correlations. 5. To apply pharmacogenomic and proteonomic tools in clinical studies. 6. To provide a training and educational program in clinical and developmental pharmacology.