Degraeuwe Eva, Hovinga Collin, De Maré Annelies, Fernandes Ricardo M, Heaton Callie, Nuytinck Lieve, Persijn Laura, Raes Ann, Vande Walle Johan, Turner Mark A
Department of Internal Medicine and Pediatrics, Ghent University, Ghent, Belgium.
Critical Path Institute, Tucson, AZ, United States.
Front Pediatr. 2024 Nov 7;12:1388170. doi: 10.3389/fped.2024.1388170. eCollection 2024.
BACKGROUND/AIMS: Due to a lack of standard pediatric prescribing information, medicines are often used in a dosage form or for an indication that has not been investigated in children. Pediatric clinical trial research networks aim to facilitate the timely availability of innovative drugs for children by developing standardized trial facilitation and conduct processes. This paper aims to assess the (pre)feasibility duration and characteristics of a US-sponsored clinical trial, in collaboration with I-ACT for Children, for distribution across European sites via European clinical research facilitation networks.
A transatlantic partnership between the Belgian Pediatric Clinical Research Network (BPCRN,) and I-ACT for Children conducted feasibilities in Europe for industry-sponsored early-stage pharmacological clinical trials between 2019 and 2022. The collaboration recorded time to event for key elements of feasibility, influences on successful feasibility, and benefits of collaboration.
Trials were conducted across 17 European countries with 202 participating hospital sites. The initial phase, the pre-feasibility questionnaire had a 70% response rate from 142 sites, and sites took a median 38 days (IQR 20 days) to complete the questionnaire for five trials. All responses underwent a quality control, addressing inaccuracies in site capabilities and recruitment. The first trial's CDA and feasibility questionnaire were completed in roughly 2 months for 7 countries. Time to completion was affected by precontracted sites, limited scope of studies, changes in timelines, COVID-related disruptions, and a learning curve for collaboration.
Collaboration between European collaborative national networks and US-network I-ACT for Children has supported site identification of global pediatric clinical trials. This illustrates one method for the importance of early engagement with sponsors and implementation of effective communication systems.
背景/目的：由于缺乏标准的儿科处方信息，药物常常以未在儿童中研究过的剂型或适应证使用。儿科临床试验研究网络旨在通过制定标准化的试验促进和实施流程，促进创新药物及时供应给儿童。本文旨在评估一项由美国发起、与儿童I-ACT合作、通过欧洲临床研究促进网络在欧洲各站点开展的临床试验的（预）可行性持续时间及特点。
比利时儿科临床研究网络（BPCRN）与儿童I-ACT建立了跨大西洋伙伴关系，于2019年至2022年在欧洲开展了由行业赞助的早期药理学临床试验的可行性研究。该合作记录了可行性关键要素的事件发生时间、对成功可行性的影响以及合作的益处。
试验在17个欧洲国家的202个参与医院站点进行。初始阶段，预可行性调查问卷在142个站点的回复率为70%，各站点完成五项试验调查问卷的中位时间为38天（四分位间距20天）。所有回复都经过了质量控制，解决了站点能力和招募方面的不准确问题。第一个试验的CDA和可行性调查问卷在大约2个月内完成了7个国家的工作。完成时间受到预先签约的站点、研究范围有限、时间表变化、与新冠疫情相关的干扰以及合作的学习曲线的影响。
欧洲国家合作网络与美国儿童网络I-ACT之间的合作支持了全球儿科临床试验的站点识别。这说明了早期与赞助商接触以及实施有效沟通系统的重要性的一种方法。
