Pediatric Pharmacology Research Unit Network

儿科药理学研究单位网络

• 批准号: 7393850
• 负责人: EDMUND Vincent CAPPARELLI
• 金额: $ 39.04万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 依托单位国家: 美国
• 财政年份: 1994
• 资助国家: 美国
• 起止时间: 1994-01-01 至 2009-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7393850
• 关键词: Adherence; Adult; Age; Amendment; Area; Biological Availability; Biological Markers; Canada; Cessation of life; Characteristics; Child; Childhood; Chloramphenicol; Class; Clinical; Clinical Data; Clinical Pharmacology; Clinical Research; Collaborations; Cosmetics; Data; Development; Diagnosis; Disease; Dose; Drug Approval; Drug Approval Processes; Drug Delivery Systems; Drug Evaluation; Drug Formulations; Drug Industry; Drug Kinetics; Drug Labeling; Drug Legislation; Drug Prescriptions; Drug usage; Effectiveness; Enzymes; Europe; Evolution; Exposure to; Failure; Food, Drug and Cosmetic Act; Funding; Genetic Polymorphism; Grant; Gray unit of radiation dose; Guidelines; Human; Infant; Invasive; Knowledge; Label; Laws; Lead; Legal patent; Licensing; Marketing; Measurement; Medical; Mission; Molecular; Newborn Infant; Numbers; Orphan; Patients; Pediatric Hospitals; Pediatrics; Perinatal Exposure; Pharmaceutical Preparations; Pharmacodynamics; Pharmacogenomics; Pharmacologic Substance; Pharmacology; Pharmacotherapy; Physiological; Population; Process; Regulation; Research; Research Contracts; Research Infrastructure; Research Personnel; Safety; Science; Screening procedure; Statutes and Laws; Sulfanilamide; Sulfanilamides; Supervision; Syndrome; Technology; Thalidomide; Therapeutic; Therapeutic Uses; Toxic effect; Training; United States Food and Drug Administration; Validation; Work; base; diethylene glycol; drug development; drug metabolism; innovation; interest; malformation; novel; novel strategies; outcome forecast; pediatric pharmacology; pharmacodynamic model; programs; receptor; tool

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.

描述(由申请人提供)： UCSD/CHHC儿科药理研究组(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，这些药物将导致在婴儿和儿童中安全和有效的治疗使用。这将通过仔细的合作计划和遵守FDA的指南来实现，以便在PPRU研究的开发和实施期间收集的药物文件可以为以安全、有效的剂量许可用于儿科适应症的新药(或旧药)提供足够的数据。这项任务的很大一部分将与制药业合作完成，以便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的数量，并将儿科标记扩大到包括新生儿在内的所有儿科人群，以便所有儿科患者都可以接受基于科学的智能和合理的治疗。PPRU赠款的范围是为加州大学圣迭戈分校和圣地亚哥儿童医院提供一个组织和业务基础设施，以实现以下具体目标：1.为开展关于生物利用度、配方、药物代谢、药代动力学、药效学、安全性和有效性的研究提供场所。2.收集或促进必要的临床数据的积累，以用于儿科特定年龄的药物标签。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的。3.开展儿科药理学的新方法、新创新研究。感兴趣的具体领域包括但不限于：a)诊断、预后、疗效、毒性和/或疾病活动性的生物标记物的确定、开发、验证和/或推断；b)非侵入性药效学测量的开发和验证；c)儿科新型药代动力学-药效学建模技术的开发和/或应用(例如基于机械学和生理学的药代学)；d)新的分子方法的使用；e)新型给药系统在儿科治疗中的应用。4.开展药物代谢酶(DMES)、转运体和受体的发育特征和遗传多态性研究，描述表型-基因相关性。5.将药物基因组学和蛋白质组学工具应用于临床研究。6.提供临床和发育药理学方面的培训和教育计划。