Stewart Breanne, Lepola Pirkko, Egger Gunter F, Ali Fahimeda, Allen Albert J, Croker Alysha K, Davidson Andrew J, Dicks Pamela, Faust Saul N, Green Dionna, Hovinga Collin, Klein Agnes V, Langham Robyn, Nakamura Hidefumi, Pioppo Laura, Ramroop Shiva, Sakiyama Michiyo, Sanchez Vigil de la Villa Isabel, Sato Junko, Snyder Donna L, Turner Mark A, Zaidi Sarah, Zimmerman Kanecia, Lacaze-Masmonteil Thierry
Maternal Infant Child and Youth Research Network (MICYRN), Vancouver, BC, Canada.
Department of Children and Adolescents, University of Helsinki and Helsinki University Hospital, Helsinki, Finland.
Front Med (Lausanne). 2025 Apr 15;12:1542408. doi: 10.3389/fmed.2025.1542408. eCollection 2025.
Conducting clinical trials (CTs) with children presents several challenges. A major challenge is the need to enrol participants at multiple sites across different jurisdictions. Regardless of whether the trials involve children, adults, or both, CTs need to meet separate Competent Authority (CA) requirements to proceed in each participating country. This work, undertaken by the Working Group (WG) on International Collaborations at the European Network of Pediatric Research at the European Medicines Agency (Enpr-EMA) aims to describe the regulatory requirements including any specific to pediatrics, as well as current or upcoming changes across six jurisdictions-the European Union (EU), United Kingdom (UK), United States of America (USA), Canada, Japan, and Australia.
An open questionnaire developed by the WG and directed at both the CA and the national pediatric clinical trial networks arranged by jurisdictions.
A synopsis of the current legislative and regulatory requirements for CTs applications, application submission processes and application requirements is presented for each of the six jurisdictions. Requirements were found to be mostly consistent across jurisdictions. No difference was found in processes for CTs submission, review, and authorization for pediatric CTs vs. CTs in adults. However, there are additional Ethics Committee/Institutional Review Board requirements for clinical trials including children. Some jurisdictions are considering adopting a risk-based approach, inspired by the Organization of Economic Co-operation and Development (OECD) recommendations on Governance. Changes currently or soon to be implemented in some jurisdictions are also described.
Regulators from the jurisdictions represented in this WG are collaborating to facilitate regulatory harmonization and foster international alignment of pediatric CTs. By interacting with their respective regulatory bodies and developing expertise in their jurisdiction's regulatory requirements, national pediatric networks can support both academic and industry sponsors in navigating the regulatory process for CTs.
开展儿童临床试验面临诸多挑战。一个主要挑战是需要在不同司法管辖区的多个地点招募参与者。无论试验涉及儿童、成人还是两者皆有，临床试验都需要满足每个参与国家独立的主管当局（CA）要求才能进行。欧洲药品管理局欧洲儿科研究网络（Enpr-EMA）国际合作工作组（WG）开展的这项工作旨在描述包括儿科特有要求在内的监管要求，以及六个司法管辖区——欧盟（EU）、英国（UK）、美国（USA）、加拿大、日本和澳大利亚——当前或即将发生的变化。
工作组制定了一份开放式问卷，面向主管当局以及各司法管辖区安排的国家儿科临床试验网络。
针对六个司法管辖区中的每一个，都给出了临床试验申请的现行立法和监管要求、申请提交流程及申请要求的概要。各司法管辖区的要求大多一致。儿科临床试验与成人临床试验在提交、审查和授权流程方面未发现差异。然而，涉及儿童的临床试验对伦理委员会/机构审查委员会有额外要求。一些司法管辖区正在考虑采用基于风险的方法，这一方法受到经济合作与发展组织（OECD）治理建议的启发。还描述了一些司法管辖区目前或即将实施的变化。
该工作组所代表的各司法管辖区的监管机构正在合作，以促进监管协调并推动儿科临床试验的国际接轨。通过与各自的监管机构互动并培养对其司法管辖区监管要求的专业知识，国家儿科网络可以支持学术和行业赞助商应对临床试验的监管流程。
