Division of Emergency Medicine, Boston Children's Hospital, 300 Longwood Ave, Boston, MA 02115.
Pediatrics. 2014 Jan;133(1):78-87. doi: 10.1542/peds.2013-2567. Epub 2013 Dec 16.
The allocation of research resources should favor conditions responsible for the greatest disease burden. This is particularly important in pediatric populations, which have been underrepresented in clinical research. Our aim was to measure the association between the focus of pediatric clinical trials and burden of disease and to identify neglected clinical domains.
We performed a cross-sectional study of clinical trials by using trial records in ClinicalTrials.gov. All trials started in 2006 or after and studying patient-level interventions in pediatric populations were included. Age-specific measures of disease burden were obtained for 21 separate conditions for high-, middle-, and low-income countries. We measured the correlation between number of pediatric clinical trials and disease burden for each condition.
Neuropsychiatric conditions and infectious diseases were the most studied conditions globally in terms of number of trials (874 and 847 trials, respectively), while intentional injuries (5 trials) and maternal conditions (4 trials) were the least studied. Clinical trials were only moderately correlated with global disease burden (r = 0.58, P = .006). Correlations were also moderate within each of the country income levels, but lowest in low-income countries (r = .47, P = .03). Globally, the conditions most understudied relative to disease burden were injuries (-260 trials for unintentional injuries and -160 trials for intentional injuries), nutritional deficiencies (-175 trials), and respiratory infections (-171 trials).
Pediatric clinical trial activity is only moderately associated with pediatric burden of disease, and least associated in low-income countries. The mismatch between clinical trials and disease burden identifies key clinical areas for focus and investment.
研究资源的分配应优先考虑导致疾病负担最大的条件。这在儿科人群中尤为重要，因为他们在临床研究中代表性不足。我们的目的是衡量儿科临床试验的重点与疾病负担之间的关联，并确定被忽视的临床领域。
我们通过使用 ClinicalTrials.gov 中的试验记录，对临床试验进行了横断面研究。所有于 2006 年或之后开始、针对儿科人群中患者干预措施的试验均被纳入研究。我们获得了 21 种不同疾病在高、中、低收入国家的特定年龄疾病负担的衡量指标。我们测量了每个疾病条件下儿科临床试验数量与疾病负担之间的相关性。
从试验数量来看，神经精神疾病和传染病是全球研究最多的疾病（分别为 874 项和 847 项试验），而意外伤害（5 项试验）和孕产妇疾病（4 项试验）研究最少。临床试验与全球疾病负担的相关性仅为中度（r = 0.58，P =.006）。在每个国家收入水平内相关性也是中度的，但在低收入国家最低（r =.47，P =.03）。全球范围内，相对于疾病负担而言，研究最少的疾病条件是伤害（意外伤害未报告的试验为-260 项，故意伤害为-160 项）、营养缺乏（-175 项）和呼吸道感染（-171 项）。
儿科临床试验活动与儿科疾病负担仅呈中度相关，在低收入国家相关性最低。临床试验与疾病负担之间的不匹配确定了重点关注和投资的关键临床领域。
