儿童眼部用药：与临床研究相关的监管情况。
Ocular medicines in children: the regulatory situation related to clinical research.
机构信息
Laboratory for Mother and Child Health, Department of Public Health, Mario Negri Institute for Pharmacological Research, Milan, Italy.
出版信息
BMC Pediatr. 2012 Jan 20;12:8. doi: 10.1186/1471-2431-12-8.
BACKGROUND
Many ocular medications are prescribed for paediatric patients, but the evidence for their rational use is very scant. This study was planned to compare the availability and the licensing status of ocular medications marketed in Italy, the United Kingdom (UK), and the United States of America (USA) related to the amount of published and un-published RCTs testing these drugs in the paediatric population.
METHODS
A quantitative analysis was performed to evaluate the number of ocular medications with a paediatric license in Italy, the UK, and the USA. A literature search was also performed in MEDLINE, EMBASE, and The Cochrane Central Register of Controlled Trials for randomized controlled trials (RCTs) on ophthalmic pharmacological therapy in children aged < 18 years, published up to December 2010. A search in the international clinical trial registries, the list of paediatric investigation plans (PIPs) approved by European Medicines Agency (EMA), and the table of medicines with new paediatric information approved by Food and Drug Administration (FDA) was also performed.
RESULTS
In all, of 197 drugs identified, 68 (35%) single drugs are licensed for paediatric use at least in one considered country, while 23 (12%) were marketed in all three countries. More specifically, in Italy 43 single drugs (48% of those marketed) had a paediatric license, while 39 (64%) did in the UK and 22 (54%) did in the USA. Only 13 drugs were marketed with a paediatric license in all countries. The percentage of drugs licensed for paediatric use and for which at least one RCT had been performed ranged between 51% in Italy and 55% in the USA. No published RCTs were found for 11 (48%) drugs licensed for paediatric use in all three countries. In all, 74 (35%) of the retrieved RCTs involved mydriatic/cycloplegic medications.A total of 62 RCTs (56% completed) on 46 drugs were found in the international clinical trial registries. Cyclosporin and bevacizumab were being studied in many ongoing trials. Twenty-six drugs had new paediatric information approved by FDA based on new paediatric clinical trials, while only 4 PIPs were approved by EMA.
CONCLUSIONS
There is a pressing need for further research and clinical development in the pediatric ophthalmic area, where effective up-to-date treatments, and additional research and education on use in children, remain priorities.
背景
许多眼部药物都被开给儿科患者使用，但关于这些药物合理使用的证据却很少。本研究旨在比较意大利、英国和美国上市的眼部药物的可获得性和许可状态，以及这些药物在儿科人群中进行的已发表和未发表的随机对照试验（RCT）的数量。
方法
我们进行了一项定量分析，以评估在意大利、英国和美国具有儿科许可的眼部药物数量。我们还在 MEDLINE、EMBASE 和 The Cochrane Central Register of Controlled Trials 中检索了截至 2010 年 12 月发表的关于儿童年龄<18 岁的眼科药理学治疗的 RCT 文献。我们还在国际临床试验注册处、欧洲药品管理局（EMA）批准的儿科研究计划（PIP）清单以及美国食品和药物管理局（FDA）批准的具有新儿科信息的药物列表中进行了搜索。
结果
在总共确定的 197 种药物中，有 68 种（35%）单一药物至少在一个被考虑的国家被批准用于儿科用途，而有 23 种（12%）在所有三个国家都有销售。具体而言，在意大利，有 43 种单一药物（市场上的 48%）具有儿科许可，而英国有 39 种（64%），美国有 22 种（54%）。只有 13 种药物在所有国家都有儿科许可和销售。有儿科许可且至少进行了一项 RCT 的药物比例在意大利为 51%，在美国为 55%。在所有三个国家都有儿科许可的 11 种（48%）药物均未发现已发表的 RCT。在检索到的 RCT 中，有 74 项（35%）涉及散瞳/睫状肌麻痹药物。在国际临床试验注册处共发现了 62 项（56%已完成）关于 46 种药物的 RCT。环孢素和贝伐单抗正在进行许多正在进行的试验。根据新的儿科临床试验，FDA 批准了 26 种药物具有新的儿科信息，而 EMA 仅批准了 4 个 PIP。
结论
在儿科眼科领域，迫切需要进一步研究和临床开发，以提供有效的最新治疗方法，并开展更多关于儿童用药的研究和教育。
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