Degraeuwe Eva, van der Geest Tessa, Persijn Laura, Nuytinck Lieve, Raes Ann, Turner Mark, Fernandes Ricardo M, Vande Walle Johan, de Wildt Saskia N
Faculty of Medicine and Health Sciences, Department of Internal Medicine and Pediatrics, Ghent University, Ghent, Belgium.
Department of Pediatrics, Ghent University Hospital (UZGent), Ghent, Belgium.
Front Pediatr. 2023 Dec 21;11:1302272. doi: 10.3389/fped.2023.1302272. eCollection 2023.
The high failure rate of industry-driven pediatric clinical trials leads to insufficient timely labeling of drugs in children and a lack of scientific evidence, resulting in the persistently high off-label drug use. National clinical trial networks can facilitate collaboration between sites, investigators, and experts, increasing the likelihood of successful trials. Within the conect4children (c4c) network, an Innovative Medicines Initiative 2-funded project, National Hubs hosted by National Clinical Trials Networks were set up across 21 European countries to facilitate the setup and execution of pediatric clinical trials. In this paper, we aim to present the performance metrics of the trial feasibility process as well as learnings and challenges encountered by the Belgian and Dutch Networks in working within the European c4c project.
The c4c National Hubs streamline pediatric clinical trials by initiating early country outreach, identifying overlapping studies, recommending quality trial sites, and supporting trial budgeting for both industry and academic settings. To show the impact of Pedmed-NL and Belgian Pediatric Clinical Research Network (BPCRN), internal metrics were collected from 2019 to 2022 on four industry-sponsored and three academic trials performed within the c4c network. Timelines and outcomes of the site identification were collected and analyzed for industry trials. A qualitative analysis was conducted through c4c platforms, sponsor interactions, and stakeholder engagement to evaluate the added value of a research network.
In industry-sponsored trials, full feasibility questionnaires were completed within 2 weeks ( = 48), and inclusion rates were up to 80% of clinical sites. Before committing to c4c, 14% of sites were contacted by industry, leading to communication burdens. Utilizing national infrastructure knowledge and therapeutic environment insights helped optimize trial timelines and address feasibility challenges. In addition, national adaptations, such as bilingual staff and site development, played a role in streamlining trial operations in both academic and industry settings. Performance and experiences were similar for both networks.
The early-facilitation examples from the c4c trials demonstrated promising metrics for two National Hubs, including optimized start-up timelines and aiding site selection quality. The learnings and challenges of the Belgian and Dutch Networks provided insights for the development of clinical research networks.
行业驱动的儿科临床试验失败率很高，导致儿童药物标签更新不及时且缺乏科学证据，从而使药品超说明书使用现象持续高发。国家临床试验网络能够促进各试验点、研究者和专家之间的合作，增加试验成功的可能性。在由创新药物倡议2资助的“connect4children（c4c）”网络项目中，21个欧洲国家设立了由国家临床试验网络主办的国家中心，以促进儿科临床试验的开展与实施。在本文中，我们旨在介绍试验可行性流程的绩效指标，以及比利时和荷兰网络在参与欧洲c4c项目过程中的经验教训和遇到的挑战。
c4c国家中心通过尽早开展国别推广、识别重叠研究、推荐优质试验点以及为行业和学术背景的试验提供预算支持，来简化儿科临床试验流程。为了展示荷兰儿科医学网络（Pedmed-NL）和比利时儿科临床研究网络（BPCRN）的影响，收集了2019年至2022年期间在c4c网络内开展的四项行业资助试验和三项学术试验的内部指标。收集并分析了行业试验中试验点识别的时间线和结果。通过c4c平台、申办方互动以及利益相关者参与进行了定性分析，以评估研究网络的附加价值。
在行业资助的试验中，完整的可行性调查问卷在2周内完成（n = 48），入选率高达临床研究点的80%。在加入c4c之前，14%的试验点曾被行业联系过，这导致了沟通负担。利用国家基础设施知识和治疗环境见解有助于优化试验时间线并应对可行性挑战。此外，诸如配备双语工作人员和开展试验点开发等国别调整措施，在简化学术和行业背景下的试验运作方面发挥了作用。两个网络的表现和经验相似。
c4c试验中的早期促进实例为两个国家中心展示了很有前景的指标，包括优化启动时间线和提高试验点选择质量。比利时和荷兰网络的经验教训为临床研究网络的发展提供了借鉴。
