Wong Ian, Sweis Dimah, Cope Judith, Florence Alexander
The School of Pharmacy, University of London, London, United Kingdom.
Drug Saf. 2003;26(8):529-37. doi: 10.2165/00002018-200326080-00001.
There have been numerous studies to show that many of the medicines used in children are used off-label or are unlicensed for use in children. When children are prescribed unlicensed and off-label medications, some people may see them as unknowing participants in informal and uncontrolled experiments. However, the licensing status of a drug can be seen as a by-product of the real issues: the safety, efficacy and quality of these medicines in the current licensing system. It is important to conduct research in order to provide high quality data regarding safety and efficacy to support evidence-based paediatric prescribing. Clinical trials will always be an invaluable means of acquiring vital information about a drug; but when it comes to children, we may find that these trials are not always practical for technical, ethical and financial reasons; therefore, it is important to explore other methodologies in paediatric medicines research. Pharmacoepidemiological and prospective cohort studies could provide vital safety and efficacy data on paediatric medicines; however, resources need to be invested in the methodological research. Paediatric drug formulation research is under-resourced and under-valued, and, unfortunately, fatal and serious adverse reactions due to inappropriate formulations have been reported in many instances. Paediatric medication is a complex problem; we need to use all available tools for research on safety, efficacy and formulation. The reason for lack of progress in paediatric drug research is most likely due to lack of resources and research capacity. The industry and government should work together and invest more money in paediatric drug research. Finally, regulatory authorities, healthcare professionals and academics need to rethink the research strategy in order to provide better medicines for children.
已有大量研究表明，许多用于儿童的药物属于超说明书用药或未获儿童用药许可。当给儿童开具未获许可和超说明书的药物时，有些人可能会将他们视为参与非正式且不受控制实验的不知情参与者。然而，药物的许可状态可被视为实际问题的一个副产品：即这些药物在当前许可体系中的安全性、有效性和质量。开展研究以提供有关安全性和有效性的高质量数据，以支持循证儿科处方，这一点很重要。临床试验始终是获取有关药物重要信息的宝贵手段；但对于儿童而言，我们可能会发现，由于技术、伦理和财务方面的原因，这些试验并不总是可行的；因此，探索儿科药物研究的其他方法很重要。药物流行病学和前瞻性队列研究可为儿科药物提供重要的安全性和有效性数据；然而，需要对方法学研究投入资源。儿科药物制剂研究资源不足且未得到重视，不幸的是，许多案例中都报告了因制剂不当导致的致命和严重不良反应。儿科用药是一个复杂的问题；我们需要利用所有可用工具来研究安全性、有效性和制剂。儿科药物研究缺乏进展的原因很可能是资源和研究能力不足。制药行业和政府应共同努力，加大对儿科药物研究的投入。最后，监管机构、医疗保健专业人员和学者需要重新思考研究策略，以便为儿童提供更好的药物。
