Office of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, USA.
Pediatrics. 2012 Dec;130(6):e1583-91. doi: 10.1542/peds.2011-3687. Epub 2012 Nov 5.
To examine the characteristics of pediatric trials conducted under US legislation and to compare results with data from 2002 to 2007.
We reviewed all pediatric trials provided to the US Food and Drug Administration in submissions that were approved between September 28, 2007 and December 21, 2010. We extracted data for each trial including age range, therapeutic indication, design, duration, and patient and center enrollment by location.
Overall 346 studies on 113 drugs and biologicals enrolled 55 819 pediatric patients. The United States participated in 86% of the studies, providing 71% of the centers and 74% of the patients. Corresponding percentages for non-US countries were 43%, 29%, and 26% respectively. Developing or transition countries participated in 22% of the studies, providing 12% of the centers and 10% of the patients; our earlier analysis found corresponding percentages of 38%, 12%, and 23%. The most common therapeutic areas studied in the latter countries were infectious, neurologic, and pulmonary diseases. Seventy-eight vaccine studies enrolled 147 692 patients. The United States participated in 40% of the studies, providing 39% of the centers and 22% of the patients. Corresponding percentages for non-US countries were 74%, 61%, and 78% respectively. Developing or transition countries participated in 27% of the studies, providing 15% of the centers and 52% of the patients.
The United States remains an important location for pediatric trials. Developing country involvement in pediatric drug development is not increasing, although these countries participate significantly in vaccine trials.
研究在美国法规下进行的儿科试验的特点，并将结果与 2002 年至 2007 年的数据进行比较。
我们回顾了所有在 2007 年 9 月 28 日至 2010 年 12 月 21 日期间批准的提交给美国食品和药物管理局的儿科试验。我们为每个试验提取了数据，包括年龄范围、治疗指征、设计、持续时间以及按地点登记的患者和中心。
共有 346 项关于 113 种药物和生物制剂的研究纳入了 55819 名儿科患者。美国参与了 86%的研究，提供了 71%的中心和 74%的患者。非美国国家的相应百分比分别为 43%、29%和 26%。发展中国家或转型国家参与了 22%的研究，提供了 12%的中心和 10%的患者；我们之前的分析发现相应的百分比为 38%、12%和 23%。在后一类国家中，最常见的研究治疗领域是传染病、神经病学和肺部疾病。78 项疫苗研究纳入了 147692 名患者。美国参与了 40%的研究，提供了 39%的中心和 22%的患者。非美国国家的相应百分比分别为 74%、61%和 78%。发展中国家或转型国家参与了 27%的研究，提供了 15%的中心和 52%的患者。
美国仍然是儿科试验的重要地点。发展中国家参与儿科药物开发的情况并未增加，尽管这些国家在疫苗试验中发挥了重要作用。
