European regulators' views on a wearable-derived performance measurement of ambulation for Duchenne muscular dystrophy regulatory trials
European regulators' views on a wearable-derived performance measurement of ambulation for Duchenne muscular dystrophy regulatory trials
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DOI：
10.1016/j.nmd.2019.06.003
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发表时间：
2019-07-01
影响因子：
2.8
通讯作者：
Magrelli, Armando
中科院分区：
文献类型：
--
作者：
Haberkamp, Marion;Moseley, Jane;Magrelli, Armando
杜氏肌营养不良症（DMD）的新疗法的开发推动了对检测DMD疾病进展变化的更有效方法的需求[1]。然而，药品的批准必须基于从监管角度来看可以接受的结果措施。在这篇文章中，欧洲监管机构提供了一个新的终点（步幅速度95百分位数（SV95C）），可用于治疗DMD试验的最新监管考虑的更新。这一新的端点旨在通过可穿戴设备在家庭环境中直接、可靠和连续地量化患者的安培数。(C)2019年，任作家。由爱思唯尔公司出版
Development of novel therapies for Duchenne muscular dystrophy (DMD) are driving the need for more efficient ways of detecting changes in disease- progression in DMD [1]. However, medicines' approval must be based on outcome measures that are acceptable from a regulatory perspective. In this article, European regulators provide an update on the recent regulatory consideration of a new endpoint (Stride Velocity 95th Centile (SV95C)) that could be used in therapeutic DMD trials. This new endpoint aims to quantify a patient's ambulation directly, reliably and continuously in a home environment with a wearable device. (C) 2019 The Authors. Published by Elsevier B.V.