Tassinari Melissa S, Benson Kimberly, Elayan Ikram, Espandiari Parvaneh, Davis-Bruno Karen
Pediatric and Maternal Health Staff, Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration, 10903 New Hampshire Ave.,Silver Spring, MD 20993, USA.
Birth Defects Res B Dev Reprod Toxicol. 2011 Aug;92(4):261-5. doi: 10.1002/bdrb.20304. Epub 2011 May 18.
Juvenile animal toxicity studies are conducted to support applications for drugs intended for use in children. They are designed to address specific questions of potential toxicity in the growing animal or provide data about long-term safety effects of drugs that cannot be obtained from clinical trials. Decisions to conduct a juvenile animal study are based on existing data, such as a safety signal already identified in adult studies, or previous knowledge of the drug or chemical class for its potential to impair growth or developmental milestones. In 2006, the FDA issued an industry guidance in which considerations for determining when a juvenile animal study is warranted were outlined. A retrospective study was conducted covering years both before and after the issued guideline to examine the contribution of juvenile animal toxicity studies to the risk/benefit assessment of pediatric drugs at the FDA. The initial findings were presented as part of the May 2010 HESI workshop on the value of juvenile animal studies. The objective of the review was to better understand the value that the juvenile animal study contributes to regulatory decision making for pediatric drug development by looking at when the studies have been included in the product assessment; what, if any, impact the studies had on the regulatory decisions made; and whether the data were incorporated into the label. The data described below represent a first look at impact of the juvenile animal study since the pediatric legislation and the juvenile animal guidance were issued in the US.
开展幼年动物毒性研究是为了支持用于儿童的药物的申请。这些研究旨在解决生长中的动物潜在毒性的特定问题，或提供从临床试验中无法获得的药物长期安全性影响的数据。决定开展幼年动物研究是基于现有数据，例如在成年动物研究中已经确定的安全信号，或者对药物或化学类别可能损害生长或发育里程碑的先前了解。2006年，美国食品药品监督管理局（FDA）发布了一份行业指南，其中概述了确定何时有必要开展幼年动物研究的考量因素。开展了一项回顾性研究，涵盖指南发布前后的年份，以审查幼年动物毒性研究对FDA儿科药物风险/效益评估的贡献。初步研究结果作为2010年5月HESI关于幼年动物研究价值研讨会的一部分进行了展示。该综述的目的是通过研究这些研究何时被纳入产品评估；这些研究对做出的监管决策有何影响（如果有的话）；以及数据是否被纳入标签，来更好地理解幼年动物研究对儿科药物开发监管决策的贡献。以下数据代表了自美国颁布儿科立法和幼年动物指南以来，对幼年动物研究影响的初步观察。
