Fung Albert, Yue Xiaomeng, Wigle Patricia R, Guo Jeff J
Division of Pharmacy Practice & Administrative Sciences, James L. Winkle College of Pharmacy, University of Cincinnati Academic Health Center, Cincinnati, Ohio, USA.
Joulé, Edison, New Jersey, USA.
Intractable Rare Dis Res. 2021 Nov;10(4):238-245. doi: 10.5582/irdr.2021.01104.
Many pediatric patients with rare diseases use drugs off-label due to limited data in pediatric patients. Off-label treatment remains an important public health issue for neonates, infants, children, and adolescents, especially for pediatric patients with rare diseases. For patients with rare diseases, the majority of medications have no or limited information in labelling for pediatric use. Children present unique considerations in clinical trials due to ethical and clinical concerns, which have limited and even discouraged testing of drugs in the pediatric population. Numerous legislative measures have been enacted to address barriers in pediatric drug testing. This research reviewed off-label medication use in rare pediatric diseases, evaluated recent medication uses in pediatric clinical practice, discussed key regulations for rare pediatric diseases, and summarized recent drug approvals for rare pediatric diseases. This study demonstrates the ongoing medical need for newly approved medications to treat pediatric rare diseases and revealed the positive impact of regulations from the Orphan Drug Act of 1983 to the Research to Accelerate Cures and Equity (RACE) for Children Act on drug development and off-label medication practice in rare pediatric disease management. This article provides informative historical background and current considerations of off-label use of medications in neonates, infants, children, and adolescents with rare diseases.
许多患有罕见病的儿科患者因儿科患者数据有限而使用未标明适应证的药物。未标明适应证的治疗对于新生儿、婴儿、儿童和青少年来说仍然是一个重要的公共卫生问题，尤其是对于患有罕见病的儿科患者。对于患有罕见病的患者，大多数药物在儿科用药标签上没有或只有有限的信息。由于伦理和临床方面的考虑，儿童在临床试验中存在独特的考量因素，这限制甚至阻碍了在儿科人群中进行药物测试。已经颁布了许多立法措施来解决儿科药物测试中的障碍。本研究回顾了儿科罕见病中未标明适应证的药物使用情况，评估了儿科临床实践中近期的药物使用情况，讨论了儿科罕见病的关键法规，并总结了近期儿科罕见病的药物批准情况。这项研究表明，持续存在对新批准药物治疗儿科罕见病的医疗需求，并揭示了从1983年《孤儿药法案》到《加速儿童治疗与公平研究（RACE）法案》的法规对罕见儿科疾病管理中药物开发和未标明适应证用药实践的积极影响。本文提供了关于患有罕见病的新生儿、婴儿、儿童和青少年未标明适应证用药的丰富历史背景和当前考量因素。
