儿科药物开发是否推动了年轻患者的癫痫治疗?是时候制定新的研究目标了。

Earl B Ettienne, Emilio Russo, Pasquale Striano, Jane M Grant-Kels, Klaus Rose
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引用次数: 0

摘要

现代药物改变了影响各个年龄段人群的癫痫。然而,对于青少年癫痫患者来说,药物开发框架却停滞不前。沙利度胺灾难发生后,出现了一种误解,即对于年龄小于18岁的人群,包括抗癫痫药物(ASM)在内的药物需要单独的疗效和安全性证明,这种误解被统称为 "儿科药物开发"。就 ASM 而言,这种情况已发生了一定程度的改变。现在,当局承认 ASM 对 18 岁以下的儿童也有效,但仍要求 "推断疗效",仿佛未成年人是另一个物种。因此,过去几十年的一些儿科癫痫临床研究是不必要的。更重要的是,这阻碍了对有意义的研究目标的研究。我们不需要证实 ASM 在 18 岁生日之前和 18 岁生日之后一样起作用。相反,我们需要了解如何通过预防癫痫发作来防止年轻患者的脑损伤,并优化 ASMs 的使用。在此,我们将讨论如何开展这项工作。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Did pediatric drug development advance epilepsy treatment in young patients? It is time for new research goals.

Modern drugs have changed epilepsy, which affects people of all ages. However, for young people with epilepsy, the framework of drug development has stalled. In the wake of the thalidomide catastrophe, the misconception emerged that for people < 18 years of age drugs, including antiseizure medications (ASMs), need separate proof of efficacy and safety, overall called "pediatric drug development". For ASMs, this has changed to some degree. Authorities now accept that ASMs are effective in < 18 years as well, but they still require "extrapolation of efficacy," as if minors were another species. As a result, some of the pediatric clinical epilepsy research over the past decades was unnecessary. Even more importantly, this has hampered research on meaningful research goals. We do not need to confirm that ASMs work before as they do after the 18th birthday. Instead, we need to learn how to prevent brain damage in young patients by preventing seizures and optimize ASMs' uses. Herein we discuss how to proceed in this endeavor.

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