BPCA PHARMACOEPIDEMIOLOGY (REAL WORLD DATA) STUDIES

自20世纪90年代初以来，科学界，包括学术界、NIH、FDA和 制药行业一直在努力提高人们对药物使用的认识 孩子们。国会通过立法，为#年的药物开发计划提供激励 儿科。提高药品质量的实施和监督责任 儿科的开发主要委托给FDA(用于专利药物)和 给NIH(用于非专利药物)。尽管立法已经提高了 在儿童中进行的临床研究，在发育方面仍然存在差距 药理学，药物代谢的个体发生学，结果衡量标准的验证和 儿科研究中的终端，以及最近利用数据库和电子健康 记录(真实世界的健康数据)以利用临床研究。在经历了30多年的 立法，面对临床护理和研究领域蓬勃发展的技术进步， 显然，要继续成功地弥合在以下领域的知识差距 儿科药物开发，以适当地治疗各种疾病的儿科患者， 需要对儿科药物开发研究采取综合和全面的方法。 此外，NICHD和科学界的其他人得出结论， 统一临床研究数据(即PK、药效学、药物基因组学、 效率和安全数据)与使用现有的临床相关数据库、登记和 其他整理真实世界患者健康数据的方法对于有效地 以个性化的方式治疗儿科患者。

Since the early 1990s, the scientific community, including academia, NIH, FDA and the pharmaceutical industry have worked to improve the knowledge of medications used in children. Congress has passed legislations to provide incentives for drug development plans in pediatrics. The responsibility for the implementation and oversight for improving drug development in pediatrics has been delegated primarily to the FDA (for on-patent drugs) and to the NIH (for off-patent drugs). Even though the legislations have improved the number of clinical studies conducted in children, gaps remain in areas such as developmental pharmacology, ontogeny of drug metabolism, the validation of outcome measures and endpoints in pediatric research, and most recently utilizing databases and electronic health records (real world health data) to leverage clinical research. After more than 30 years of legislation, and in the face of burgeoning technological advances in clinical care and research, it has become clear that to be successful in continuing to close knowledge gaps in the area of pediatric drug development to appropriately treat pediatric patients for a variety of diseases, an integrated and comprehensive approach to pediatric drug development research is required. Furthermore, NICHD and others in the scientific community are concluding that the harmonization of clinical research data (i.e., PK, pharmacodynamic, pharmacogenomic, efficiency, and safety data) with the use of existing clinically relevant databases, registries and other means of collating real world patient health data are vital to the success of effectively treating pediatric patients in a personalized way.