Enhancing the Pediatric Drug Development Framework to Deliver Better Pediatric Therapies Tomorrow
Enhancing the Pediatric Drug Development Framework to Deliver Better Pediatric Therapies Tomorrow
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DOI：
10.1016/j.clinthera.2017.07.043
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发表时间：
2017-10-01
影响因子：
3.2
通讯作者：
Bucci-Rechtweg, Christina
中科院分区：
文献类型：
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作者：
Bucci-Rechtweg, Christina
参与儿童临床管理的卫生保健专业人员长期以来一直认识到，经过适当评估以使其在儿科人群中最佳使用的疗法数量有限。在过去的一个世纪中，监管政策的进步极大地发展了成人药物评价。可用患者群体的稀缺、药物开发研究的实际复杂性以及最低的财务回报阻碍了对儿童疗法研究的制药投资。最近，美国和欧洲的儿科政策和立法制定了义务和激励制度，以刺激对儿科药物开发的投资。这些举措与更复杂的药物发现和开发过程相结合，使儿科药物标签取得了重大进展。在新靶点、精准医疗和监管科学创新的推动下，现在药物开发研究的重点正在微妙地转向针对儿童的药物开发研究，而不仅仅是针对儿童。尽管对研究药物的儿科研究和儿科信息使用标签有所增加，但现有政策却产生了意想不到的后果。因此，在某些治疗领域和专利到期疗法方面取得的进展有限。未来旨在提高儿科药品的可及性和可及性的政策改革不仅应反映对现有政策和立法举措成功之处的理解，而且还应建设性地解决失败和意外后果。总而言之，政策改革、全球合作和监管科学创新将更有能力在明天提供更好的儿科治疗。 (C) 2017 Elsevier HS Journals, Inc. 保留所有权利。
Health care professionals involved in the clinical management of children have long appreciated the limited number of therapies suitably evaluated for their optimal use in the pediatric population. In the past century, advances in regulatory policy significantly evolved adult drug evaluation. The scarcity of available patient populations, practical complexities of drug development research, and minimal financial returns have hampered pharmaceutical investment in the study of therapies for children. More recently, pediatric policy and legislation in the United States and Europe have instituted a system of obligations and incentives to stimulate investment in pediatric drug development. These initiatives, in conjunction with a more sophisticated process of drug discovery and development, have led to significant advancements in the labeling of drugs for pediatric use. Facilitated by the emergence of new targets, precision medicine, and innovations in regulatory science, there is now a subtle shift in focus toward drug development research for children rather than simply in children. Although there has been an increase in pediatric studies of investigational agents and labeling of pediatric information for use, there have been unintended consequences of existing policies. As a result, limited progress has been made in certain therapeutic areas and for off-patent therapies. Future policy reform to enhance the availability and accessibility of pediatric medicines should not only reflect an understanding not only of the successes of existing policy and legislative initiatives but also constructively address failures and unintended consequences. Taken together, policy reform, global cooperation, and innovation in regulatory science will more ably deliver better pediatric therapies tomorrow. (C) 2017 Elsevier HS Journals, Inc. All rights reserved.