Department of Pediatrics, University of Colorado School of Medicine and Children's Hospital Colorado, Aurora CO, USA.
Cystic Fibrosis Foundation, Bethesda MD, USA.
J Cyst Fibros. 2024 Jul;23(4):725-733. doi: 10.1016/j.jcf.2024.02.011. Epub 2024 Feb 29.
The COVID-19 pandemic necessitated a rapid shift in clinical research to perform virtual visits and remote endpoint assessments, providing a key opportunity to optimize the use of remote endpoints for clinical trials in cystic fibrosis. The use of remote endpoints could allow more diverse participation in clinical trials while minimizing participant burden but must be robustly evaluated to ensure adequate performance and feasibility. In response, the Cystic Fibrosis Foundation convened the Remote Endpoint Task Force (Supplemental Table 1), a multidisciplinary group of CF researchers with remote endpoint expertise and community members tasked to better understand the current and future use of remote endpoints for clinical research. Here, we describe the current use of remote endpoints in CF clinical research, address key unanswered questions regarding their use and feasibility, and discuss the next steps to determine clinical trial readiness.
COVID-19 大流行需要迅速将临床研究转向进行虚拟访视和远程终点评估，这为优化远程终点在囊性纤维化临床试验中的使用提供了重要机会。远程终点的使用可以允许更多样化的人参与临床试验，同时最大限度地减少参与者的负担，但必须进行稳健的评估，以确保充分的性能和可行性。有鉴于此，囊性纤维化基金会召集了远程终点工作组（补充表 1），这是一个由具有远程终点专业知识的囊性纤维化研究人员和社区成员组成的多学科小组，其任务是更好地了解远程终点在临床研究中的当前和未来用途。在这里，我们描述了远程终点在 CF 临床研究中的当前使用情况，解决了关于其使用和可行性的关键未解决问题，并讨论了确定临床试验准备情况的下一步措施。
