Disease-modifying drugs (DMDs) for multiple sclerosis (MS) have been evaluated in pediatric patients in observational studies demonstrating a similar, even better clinical effect compared to adults, with a similar safety. Only fingolimod has been tested in a randomized controlled trial (RCT) and is approved for pediatric multiple sclerosis (ped-MS). Numerous methodological, practical, and ethical issues underline that RCTs are difficult to conduct in ped-MS. This also creates a lack of safety information. To facilitate the availability of new agents in ped-MS, we encourage to develop a different approach based on pharmacokinetic/pharmacodynamic studies to yield information on optimal doses and implementation of obligatory registries to obtain information on safety as primary endpoint.

中文翻译：

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小儿多发性硬化症新药的引入：开放性问题和挑战

多发性硬化症 (MS) 的疾病缓解药物 (DMD) 已在儿童患者中进行了观察性研究，结果表明与成人相比，其临床效果相似，甚至更好，并且具有相似的安全性。只有芬戈莫德在随机对照试验 (RCT) 中进行了测试，并被批准用于儿童多发性硬化症 (ped-MS)。许多方法论、实践和伦理问题强调 RCT 很难在 ped-MS 中进行。这也造成缺乏安全信息。为了促进新药在 ped-MS 中的可用性，我们鼓励开发基于药代动力学/药效学研究的不同方法，以产生关于最佳剂量的信息和强制性注册的实施，以获得作为主要终点的安全性信息。