Department of Paediatrics, University of Toronto, Canada.
Division of Paediatric Clinical Pharmacology, Department of Paediatrics, University of Western Ontario, Canada.
Br J Clin Pharmacol. 2022 Oct;88(10):4250-4257. doi: 10.1111/bcp.14769. Epub 2021 May 2.
The development of specific drug therapy for children was a paradigm-changing event that transformed paediatric medical practice. However, a series of tragedies involving drug treatment for children resulted in a gap developing between drug regulation and practice, with the majority of drugs used in child healthcare being used off-label, rendering children therapeutic orphans. Over the past two decades changes in drug regulation led by the US Food and Drug Administration and followed by the European Union's European Medicines Agency have led to substantial changes in how new drugs with potential use in children are studied and labelled. While these changes have substantially improved labelling for new drugs, there has been much less progress with older drugs. Although the unique challenges of conducting clinical research in children have been addressed by novel clinical trial designs, many of these innovations have not been translated into approaches accepted for the drug approval process. The regulations applying to the need for paediatric studies currently are only applicable in the United States and the European Union, and there is less impetus for paediatric labelling in other jurisdictions. This impacts on a number of issues beyond labelling, including the availability of child-friendly formulations. Finally, the impact of Brexit on paediatric drug studies in the UK remains unclear and is subject to ongoing negotiations between the UK government and the European Union.
儿童专用药物疗法的发展是一个具有划时代意义的事件，它改变了儿科医疗实践。然而，一系列涉及儿童药物治疗的悲剧导致了药物监管与实践之间出现了差距，大多数用于儿童保健的药物都是超适应证使用的，这使得儿童成为治疗孤儿。在过去的二十年中，美国食品和药物管理局（FDA）和欧盟的欧洲药品管理局（EMA）主导的药物监管变革，导致了对具有儿童潜在用途的新药的研究和标签的实质性变化。尽管这些变化大大改善了新药的标签，但对于旧药的进展却少得多。尽管新型临床试验设计已经解决了在儿童中进行临床研究的独特挑战，但许多创新并没有转化为药物批准过程中可接受的方法。目前适用于儿科研究需求的法规仅适用于美国和欧盟，其他司法管辖区对儿科标签的推动力较小。这不仅影响标签，还影响到包括儿童友好型制剂的可获得性等多个问题。最后，英国脱欧对英国儿科药物研究的影响仍不清楚，英国政府和欧盟之间正在进行持续谈判。
