Duke Clinical Research Institute, Durham, NC, USA.
Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Ther Innov Regul Sci. 2022 Nov;56(6):934-947. doi: 10.1007/s43441-022-00453-6. Epub 2022 Sep 9.
Recent decades have seen many advances in policy and legislation that support the development of drugs used by neonates, infants, children, and young people. This review summarizes the characteristics and performance of networks capable of conducting studies needed to meet regulatory requirements and make advances in pediatric drug development.
Description of network goals and capabilities by network leaders.
In the United States, Europe, Japan, and Canada, clinical research networks have been organized to meet the needs of biopharmaceutical and academic sponsors for timely access to high-quality sites, as well as to provide advice about drug development with regard to strategic and operational feasibility. Each network addresses the specificities of its context while working toward shared principles including standards and timelines; alignment of goals and processes, while not disturbing arrangements for conducting trials that work well; wide geographic coverage; all age groups and pediatric conditions; sources of funding; sites that compete on performance; performance monitoring for benchmarking, and opportunities to optimize the allocation of resources; and education and training for network members. Facilitation in interactions among these networks is based on a single point-of-contact for each; similar approaches to strategic and operational feasibility assessment, and site selection; and collaborative approaches to education and training.
Within five years, clinical research networks will support the needs of biopharmaceutical and publicly funded pediatric drug development through locally appropriate and globally interoperable approaches.
近几十年来，许多政策和立法方面的进展都支持了新生儿、婴儿、儿童和青少年用药的研发。本文总结了能够开展符合监管要求的研究并推动儿科药物研发进展的网络的特点和性能。
网络负责人对网络目标和能力进行描述。
在美国、欧洲、日本和加拿大，已经组建了临床研究网络，以满足生物制药和学术赞助商对及时获得高质量试验点的需求，并就药物开发的战略和操作可行性提供建议。每个网络都针对其特定环境进行了调整，同时努力实现共享原则，包括标准和时间表、目标和流程的一致性，而不会干扰运行良好的试验安排、广泛的地理覆盖范围、所有年龄组和儿科疾病、资金来源、以绩效竞争的试验点、用于基准测试的绩效监测以及优化资源分配的机会，以及网络成员的教育和培训。这些网络之间的互动是通过每个网络的单点联系人、类似的战略和操作可行性评估和试验点选择方法以及协作的教育和培训方法来实现的。
在五年内，临床研究网络将通过适应当地情况和全球互操作的方法来满足生物制药和公共资助的儿科药物开发的需求。
