Pediatric Versus Adult Drug Trials for Conditions With High Pediatric Disease Burden
Pediatric Versus Adult Drug Trials for Conditions With High Pediatric Disease Burden
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DOI：
10.1542/peds.2012-0139
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发表时间：
2012-08-01
期刊：
影响因子：
8
通讯作者：
Mandl, Kenneth D.
中科院分区：
文献类型：
--
作者：
Bourgeois, Florence T.;Murthy, Srinivas;Mandl, Kenneth D.
背景与目的：儿童的最佳治疗决策需要充分的证据证明药物对儿童患者的安全性和有效性。然而，令人担忧的是，在儿童中进行的试验不够，儿科试验与成人试验不同。我们的目的是衡量临床药物试验中儿科研究的患病率，并比较儿科和成人药物试验的试验特征和质量指标。方法：对于代表儿科疾病高负担的条件，我们确定了在ClinicalTrials.gov上注册的所有药物试验，开始日期在2006年至2011年之间，并跟踪结果出版物。我们测量了每种情况下儿科试验和受试者的比例，并比较了儿科和成人试验的特征和质量指标。结果：在所选择的条件中，59.9%的疾病负担归因于儿童，但只有12.0%（292/2440）的试验是儿童（P < 0.001）。在儿童试验中，58.6%的试验是在没有行业资助的情况下进行的，而成人试验中这一比例为35.0% （P < 0.001）。与成人随机试验相比，儿科试验较少检查安全性结果（10.1% vs 16.9%, P = 0.008）。儿科随机试验在研究开始前进行适当登记的可能性略高（46.9% vs 39.3%, P = 0.04），在研究时间框架内发表的可能性略高（32.8% vs 23.2%, P = 0.04）。结论：儿童疾病负担与针对儿童人群的临床试验研究数量之间存在实质性差异。这可能部分与试验资金有关，儿科试验主要依赖于政府和非营利组织。儿科2012;130:285 - 292
BACKGROUND AND OBJECTIVE: Optimal treatment decisions in children require sufficient evidence on the safety and efficacy of pharmaceuticals in pediatric patients. However, there is concern that not enough trials are conducted in children and that pediatric trials differ from those performed in adults. Our objective was to measure the prevalence of pediatric studies among clinical drug trials and compare trial characteristics and quality indicators between pediatric and adult drug trials.METHODS: For conditions representing a high burden of pediatric disease, we identified all drug trials registered in ClinicalTrials.gov with start dates between 2006 and 2011 and tracked the resulting publications. We measured the proportion of pediatric trials and subjects for each condition and compared pediatric and adult trial characteristics and quality indicators.RESULTS: For the conditions selected, 59.9% of the disease burden was attributable to children, but only 12.0% (292/2440) of trials were pediatric (P < .001). Among pediatric trials, 58.6% were conducted without industry funding compared with 35.0% of adult trials (P < .001).Fewer pediatric compared with adult randomized trials examined safety outcomes (10.1% vs 16.9%, P = .008). Pediatric randomized trials were slightly more likely to be appropriately registered before study start (46.9% vs 39.3%, P = .04) and had a modestly higher probability of publication in the examined time frame (32.8% vs 23.2%, P = .04).CONCLUSIONS: There is substantial discrepancy between pediatric burden of disease and the amount of clinical trial research devoted to pediatric populations. This may be related in part to trial funding, with pediatric trials relying primarily on government and nonprofit organizations. Pediatrics 2012;130:285-292