Ward Robert M, Benjamin Daniel, Barrett Jeffrey S, Allegaert Karel, Portman Ronald, Davis Jonathan M, Turner Mark A
Department of Pediatrics, Divisions of Neonatology and Clinical Pharmacology, University of Utah, Salt Lake City, Utah.
Department of Pediatrics and Duke Clinical Research Institute, Duke University, Durham, North Carolina.
Pediatr Res. 2017 May;81(5):692-711. doi: 10.1038/pr.2016.221. Epub 2016 Nov 3.
The study of medications among pediatric patients has increased worldwide since 1997 in response to new legislation and regulations, but these studies have not yet adequately addressed the therapeutic needs of neonates. Additionally, extant guidance developed by regulatory agencies worldwide does not fully address the specificities of neonatal drug development, especially among extremely premature newborns who currently survive. Consequently, an international consortium from Canada, Europe, Japan, and the United States was organized by the Critical Path Institute to address the content of guidance. This group included neonatologists, neonatal nurses, parents, regulators, ethicists, clinical pharmacologists, specialists in pharmacokinetics, specialists in clinical trials and pediatricians working in the pharmaceutical industry. This group has developed a comprehensive, referenced White Paper to guide neonatal clinical trials of medicines - particularly early phase studies. Key points include: the need to base product development on neonatal physiology and pharmacology while making the most of knowledge acquired in other settings; the central role of families in research; and the value of the whole neonatal team in the design, implementation and interpretation of studies. This White Paper should facilitate successful clinical trials of medicines in neonates by informing regulators, sponsors, and the neonatal community of existing good practice.
自1997年以来，为响应新的法律法规，全球范围内针对儿科患者的药物研究有所增加，但这些研究尚未充分满足新生儿的治疗需求。此外，全球监管机构制定的现有指南并未完全涵盖新生儿药物研发的特殊性，尤其是对于目前存活的极早产儿。因此，关键路径研究所组织了一个来自加拿大、欧洲、日本和美国的国际联盟，以探讨指南的内容。该团队包括新生儿科医生、新生儿护士、家长、监管人员、伦理学家、临床药理学家、药代动力学专家、临床试验专家以及制药行业的儿科医生。该团队编写了一份全面且有参考文献的白皮书，以指导药物的新生儿临床试验——尤其是早期研究。要点包括：在充分利用其他环境中获得的知识的同时，基于新生儿生理学和药理学进行产品研发的必要性；家庭在研究中的核心作用；以及整个新生儿团队在研究设计、实施和解读中的价值。这份白皮书应通过向监管机构、申办者和新生儿群体通报现有良好做法，促进药物在新生儿中的成功临床试验。
