Safety, dosing, and pharmaceutical quality for studies that evaluate medicinal products (including biological products) in neonates
Safety, dosing, and pharmaceutical quality for studies that evaluate medicinal products (including biological products) in neonates
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DOI：
10.1038/pr.2016.221
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发表时间：
2017-05-01
影响因子：
3.6
通讯作者：
Turner, Mark A.
中科院分区：
文献类型：
--
作者：
Ward, Robert M.;Benjamin, Daniel;Turner, Mark A.
自 1997 年以来，为了响应新的立法和法规，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，世界各地监管机构制定的现有指南并未完全解决新生儿药物开发的特殊性，特别是目前存活的极早产新生儿。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来解决指南的内容。该小组包括新生儿科医生、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、临床试验专家和制药行业的儿科医生。该小组制定了一份全面的参考白皮书来指导新生儿药物临床试验，特别是早期研究。要点包括：产品开发需要以新生儿生理学和药理学为基础，同时充分利用在其他环境中获得的知识；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、实施和解释中的价值。本白皮书应通过向监管机构、申办者和新生儿社区通报现有的良好实践，促进新生儿药物临床试验的成功。
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.