BPCA-PTN COVID-19 STUDIES

在过去的25年里，科学界，包括学术界，美国国家科学院， 美国国家卫生研究院（NIH）、美国食品药品监督管理局（FDA）和制药行业一直致力于 提高对儿童用药的认识。负责执行 改善药物开发的监督主要委托给FDA（对于专利 药物）和NIH（非专利药物）。儿科试验网络（PTN） 根据《儿童最佳药物法案》（BPCA）临床计划建立并资助 Eunice Kennedy Shriver国家儿童健康和人类发展研究所 （NICHD）。 许多PTN研究历史上利用标准护理（SOC）程序，例如数据和 从已经接受目标药物（DOI）的婴儿和儿童中采集生物样本， 并产生了有意义的药代动力学（PK）数据，从而改善了给药建议 在婴儿和儿童身上。这些研究并不给儿童开处方或给药， 从已经按照SOC接受药物治疗的儿童中采集样本， 治疗提供者此外，通过机会主义研究获得的初步数据也有助于 设计第一阶段到第三阶段的儿童试验，并支持校外申请 research. 2019年冠状病毒病（COVID-19）大流行是由新型严重急性呼吸道感染引起的。 冠状病毒2型综合征（SARS-CoV-2）。感染的范围可能从病人没有症状， 引起广泛的症状，如轻微的感冒样症状到危及生命的肺炎， 败血症和多器官系统衰竭COVID-19于2019年12月首次出现，当时一群 中国武汉的患者出现了不明原因的严重肺炎。[Wiersinga等人，JAMA doi：10.1001/jama2020.12839]。截至2020年8月11日，SARS-CoV-2已影响200多个 20,124,437亿例确诊病例，超过73.7万例确诊死亡 [https：//www.cdc.gov/coronavirus/2019-ncov]. 2020年4月，在COVID-19疫情爆发的高峰期， 在患有活动性关节炎的儿童中， 疾病或先前暴露于COVID-19的儿童，无论是否有症状。这 疾病预防控制中心于2020年5月将其命名为儿童多系统炎症综合征（MIS-C）。作为 截至2020年7月29日，美国有570名患者符合MIS-C标准 [https：//www.cdc.gov/mmwr/volumes/69/wr/mm6932e2.htm].随着大流行病继续蔓延 在美国和世界各地，科学界预计， 受感染和影响的人数将继续上升。必须有一个有组织的协调合作 公共卫生、病人护理和研究之间的努力，以进行适当的预防， 识别和治疗受COVID-19影响的儿童。 由于COVID-19以极高的感染率和死亡率肆虐世界， 对于预防，研究和临床数据已经成为NIH作为一个 研究和资助机构。此外，NIH，学术界，临床医生和其他人在 科学界得出结论，所有流行病学和临床相关的协调 数据以及对患者识别和治疗至关重要的临床相关结局 有患上COVID相关疾病的风险，如儿童多系统炎症综合征 （MIS-C）是成功控制大流行病的关键。 这些COVID-19相关研究将利用PTN内的专业知识和现有数据 BPCA计划的基础设施（BPCA PTN和数据协调中心），以扩大 SARS-CoV-2（COVID-19）儿童研究。

For the last 25 years the scientific community, including academia, the National Institutes of Health (NIH), the Food and Drug Administration (FDA) and the pharmaceutical industry have worked to improve the knowledge of medications used in children. The responsibility for the implementation and oversight for improving drug development has been delegated primarily to the FDA (for on-patent drugs) and to the NIH (for off-patent drugs). The Pediatric Trials Network (PTN) has been established and funded under the Best Pharmaceuticals for Children Act (BPCA) Clinical Program implemented by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). Many PTN studies historically capitalize on standard of care (SOC) procedures, such as data and biological-sample collection from infants and children already receiving drugs of interest (DOIs), and have produced meaningful pharmacokinetic (PK) data resulting in improved dosing recommendations in infants and children. These studies do not prescribe or administer drugs to children, but rather collected samples from children who were already receiving drugs per SOC as prescribed by their treating provider. In addition, preliminary data obtained through opportunistic studies have served to design phase 1 through 3 trials in children as well as support applications for extramural research. The coronavirus disease 2019 (COVID-19) pandemic is caused by the novel severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). The infection may range from the patient having no symptoms to causing a wide range of symptoms, such as mild cold like symptom to life-threatening pneumonias, sepsis, and multi-organ system failure. COVID-19 first emerged in December 2019 when a cluster of patients in Wuhan, China developed severe pneumonias of unknown cause. [Wiersinga et al, JAMA doi:10.1001/jama2020.12839]. As of August 11, 2020, SARS-CoV-2 has affected more than 200 countries, resulting in 20,124,437 million identified cases with over 737K confirmed deaths [https://www.cdc.gov/coronavirus/2019-ncov]. In April 2020 at the peak of the COVID-19 outbreak, there was recognized a new onset of hyperinflammatory symptoms in children who either had active disease or in children who had been previously exposed to COVID-19 with or without symptoms. This syndrome was named multisystem inflammatory syndrome in children (MIS-C) by the CDC in May 2020. As of July 29, 2020, 570 patients have met the criteria for MIS-C in the US [https://www.cdc.gov/mmwr/volumes/69/wr/mm6932e2.htm]. As the pandemic continues to spread throughout the US and the world, the scientific community anticipates that the number of children infected and affected will continue to rise. There must be an organized concerted collaborative effort between public health, patient care, and research in order for the appropriate prevention, identification and treatment of children impacted by COVID-19. Since COVID-19 has ravaged the world with inordinate high infection rates and death, the need for preventive, research and clinical data have become paramount and top priority for NIH as a research and funding institution. Furthermore, NIH, academia, clinicians, and others in the scientific community are concluding that the harmonization of all epidemiology and clinical related data as well as clinically relevant outcomes vital to identification and treatment of patients at-risk for COVID-related illnesses, such as the multi-system inflammatory syndrome in children (MIS-C), are critical to successfully managing the pandemic. These COVID-19 related studies will leverage the expertise and existing data within the PTN infrastructure of the BPCA program (BPCA PTN and Data Coordinating Center) to expand the research on SARS-CoV-2 (COVID-19) in children.