PEDIATRIC PHARMACOLOGY RESEARCH UNIT

大多数儿童用药都没有经过针对儿童年龄的药理学、安全性或有效性研究。为了解决这个问题，FDA现在要求行业提供一些已上市药物和许多新分子实体的儿科年龄特异性标签数据。进行这些试验的一个主要障碍是缺乏能够进行这些试验的机构和方案。为了发展这类设施并进行高度优先的试验，人口与发展中心建立了PPRU网络。费城儿童医院（CHOP）提议加入该网络，并将为其带来大量不同的儿科患者，在临床试验中的良好表现和发表记录，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，以及通过CHOP与杜克临床研究所联盟的合同研究组织的能力。目标1为新药和现有药物的药理学、安全性和有效性研究创造了一个场所。CHOP的PPRU研究将利用普通临床研究中心。药理学核心实验室位于结果研究中心和生物统计部门附近。CHOP将使用标准操作程序、内部和外部审计和监测系统，并对各级调查人员进行正式培训，以提供协议执行和数据的质量保证。一个咨询委员会将监督PPRU的活动。CHOP建议使用文献综述和meta分析，最重要的是，开展适当的临床试验，以收集有关药物年龄特异性标签的信息并促进其发展（目标2）。除了传统药物的研究外，该机构还致力于基因治疗和其他疾病分子方法的研究，并致力于开发新的给药系统、配方和替代标记物，以促进其使用（目标3）。目的4解决遗传多态性和建模的问题。药理学核心实验室在分析CYP多态性方面具有专业知识，并制定了确定儿童重要种族和遗传多态性的战略计划。临床研究者和药理学家的培训将在每个试验的标准操作程序的背景下进行（目标5）。流行病学与生物统计中心（CCEB）将提供继续医学教育，鼓励临床药理学硕士和博士学位。该机构为医学生提供津贴，以培养他们对临床药理学的兴趣。CHOP和PENN已经收集了大量资源来创建一个富有想象力，富有成效和成功的PPRU计划。

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.