PEDIATRIC PHARMACOLOGY RESEARCH UNIT

儿科药理学研究室

• 批准号: 2760366
• 负责人: BEVERLY J LANGE
• 金额: $ 27.02万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 1999
• 资助国家: 美国
• 起止时间: 1999-01-07 至 2003-12-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/2760366
• 关键词: age difference; child (0-11); clinical research; clinical trials; cooperative study; drug adverse effect; drug delivery systems; drug metabolism; drug screening /evaluation; gene therapy; human subject; meta analysis; molecular genetics; pediatric pharmacology; pediatrics; pharmacokinetics

Most drugs prescribed for children have not undergone age-specific studies of pharmacology, safety or efficacy. To address this problem, the FDA now requires industry to provide data for pediatric age-specific labeling for some marketed drugs and for many new molecular entities. A major barrier to conducting these trials is a lack of institutions and programs capable of performing them. To develop such facilities and conduct high priority trials, NICHD created the PPRU Network. The Children's Hospital of Philadelphia (CHOP) proposes to join this Network and will bring to it a large, diverse population of pediatric patients, a strong record of performance and publishing in clinical trials, the powerful pharmacology and biostatistical resources of the University of Pennsylvania School of Medicine (PENN), and the capabilities of a contract research organization through CHOP's alliance with the Duke Clinical Research Institute. Aim 1 creates a locus for studies of pharmacology, safety and efficacy of new and available drugs. CHOP's PPRU studies will utilize the General Clinical Research Center. The Pharmacology Core Laboratory is located in close proximity to the Center for Outcomes Research and the Division of Biostatistics. CHOP will use standard operating procedures, systems of internal and external auditing and monitoring and formal training of investigators at all levels to provide quality assurance for protocol performance and data. An Advisory Board will oversee PPRU activities. CHOP proposes to use literature review and metananlyses, and, most importantly, to conduct appropriate clinical trials to gather information about and promote age-specific labeling of drugs ( Aim 2). In addition to studies of conventional drugs, the institution is heavily committed to gene therapy and other molecular approaches to disease and to the development of new delivery systems, formulations, and surrogate markers to promote their use (Aim 3). Aim 4 addresses the issue of genetic polymorphisms and modeling. The Pharmacology Core Laboratory has expertise in analyzing CYP polymorphisms and has a strategic plan for determining important racial and genetic polymorphisms in children. Training of clinical investigators and pharmacologists will take place in the context of standard operating procedures for each trial ( Aim 5). Continued medical education will be provided by the Center for Epidemiology and Biostatistics (CCEB) and masters and PhD level degrees in Clinical Pharmacology will be encouraged. The institution is providing stipends for medical students to nurture interest in clinical pharmacology. CHOP and PENN have gathered a formidable array of resources to create an imaginative, productive and successful PPRU program.

大多数儿童处方药物都没有经过针对年龄的药理学、安全性或有效性研究。为了解决这个问题，FDA现在要求行业为一些上市药物和许多新的分子实体提供儿科年龄特定标签的数据。进行这些试验的一个主要障碍是缺乏能够执行这些试验的机构和计划。为了发展这类设施并进行高度优先的试验，NICHD创建了PPRU网络。费城儿童医院(CHOP)提议加入这一网络，并将通过CHOP与杜克临床研究所的联盟，为其带来大量、多样化的儿科患者群体、在临床试验中的出色表现和发表记录、宾夕法尼亚大学医学院(Penn)强大的药理学和生物统计学资源，以及合同研究机构的能力。目标1为研究新的和可获得的药物的药理学、安全性和有效性创造了一个场所。CHOP的PPRU研究将利用综合临床研究中心。药理学核心实验室位于结果研究中心和生物统计司附近。CHOP将使用标准操作程序、内部和外部审计和监测系统以及对各级调查人员的正式培训，为协议性能和数据提供质量保证。咨询委员会将监督PPRU的活动。CHOP建议使用文献综述和荟萃分析，最重要的是，进行适当的临床试验，以收集关于特定年龄的药物标签的信息并促进药物标签(目标2)。除了常规药物的研究外，该机构还致力于基因疗法和其他治疗疾病的分子方法，并致力于开发新的递送系统、配方和替代标记以促进其使用(目标3)。目标4解决了遗传多态和建模问题。药理学核心实验室在分析CYP多态方面拥有专业知识，并为确定儿童重要的种族和遗传多态制定了战略计划。将根据每项试验的标准操作程序对临床调查人员和药理学家进行培训(目标5)。流行病学和生物统计中心(CCEB)将继续提供医学教育，并鼓励临床药理学硕士和博士学位。该机构正在为医科学生提供津贴，以培养他们对临床药理学的兴趣。乔普和宾夕法尼亚大学聚集了一系列强大的资源，创建了一个富有想象力、富有成效和成功的PPRU项目。