Department of Pediatrics, Duke University School of Medicine, Duke Clinical Research Institute, Duke University Medical Center, Durham, North Carolina, USA.
Pediatrics. 2012 Nov;130(5):e1269-77. doi: 10.1542/peds.2011-3565. Epub 2012 Oct 1.
Clinical trials are the gold standard for generating evidence-based knowledge in medicine. Recent legislation requiring trials to be registered at ClinicalTrials.gov has enabled evaluation of the clinical trial enterprise as a whole, which was previously not possible. The purpose of this study was to create a snapshot of the pediatric clinical trial portfolio.
All interventional trials registered at ClinicalTrials.gov from July 2005 to September 2010 were included. Pediatric (ie, enrolling patients aged 0-18 years) trial characteristics, therapeutic area, location, and funding were described. Secondary objectives included describing pediatric trials over time and comparison with nonpediatric trials.
During this time, 5035 pediatric trials were registered compared with >10 times as many nonpediatric trials. Neonates/infants were eligible for enrollment in 46.6% of trials versus children (77.9%) and adolescents (45.2%). Nearly one-half of pediatric trials enrolled <100 subjects, and more pediatric trials versus nonpediatric trials evaluated preventive therapies. The proportion of pediatric trials evaluating a drug intervention declined over time, and there were fewer Phase 0 to II versus Phase III to IV trials. Infectious disease/vaccine studies (23%) were the most common, followed by psychiatric/mental health (13%) studies. Many trials enrolled patients outside the United States, and <15% of trials were sponsored by the National Institutes of Health or other US federal agencies.
Analysis of the ClinicalTrials.gov data set allows description of the current scope of pediatric trials. These data may be useful to stakeholders in informing decisions regarding the conduct of trials in children and provide insight into mechanisms to advance pediatric trial infrastructure and methodology toward improving child health.
临床试验是医学领域产生循证知识的金标准。最近的立法要求试验在 ClinicalTrials.gov 上注册，这使得我们能够评估整个临床试验事业，而此前这是不可能的。本研究的目的是描绘儿科临床试验组合的现状。
纳入 2005 年 7 月至 2010 年 9 月期间在 ClinicalTrials.gov 上注册的所有干预性试验。描述儿科（即招募 0-18 岁患者）试验的特征、治疗领域、地点和资金。次要目标包括描述随时间变化的儿科试验并与非儿科试验进行比较。
在此期间，注册了 5035 项儿科试验，而非儿科试验的数量则是其 10 倍以上。有 46.6%的试验有资格招募新生儿/婴儿入组，而儿童（77.9%）和青少年（45.2%）有资格入组。近一半的儿科试验纳入<100 名受试者，且与非儿科试验相比，更多的儿科试验评估了预防疗法。评估药物干预的儿科试验比例随时间下降，而 0 期至 2 期试验与 3 期至 4 期试验相比则较少。传染病/疫苗研究（23%）是最常见的，其次是精神科/心理健康研究（13%）。许多试验招募了美国境外的患者，且<15%的试验由美国国立卫生研究院或其他美国联邦机构资助。
对 ClinicalTrials.gov 数据集的分析可以描述当前儿科试验的范围。这些数据可能对利益相关者有用，有助于在儿童临床试验的开展方面做出决策，并深入了解促进儿科试验基础设施和方法学的机制，以改善儿童健康。
