Division of Child Neurology, Centre de Références des Maladies Neuromusculaires, Department of Pediatrics, University Hospital Liège and University of Liège, Liège, Belgium.
Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK.
J Neuromuscul Dis. 2022;9(2):335-346. doi: 10.3233/JND-210743.
In 2019, stride velocity 95th centile (SV95C) became the first wearable-derived digital clinical outcome assessment (COA) qualified by the European Medicines Agency (EMA) for use as a secondary endpoint in trials for Duchenne muscular dystrophy. SV95C was approved via the EMA's qualification pathway for novel methodologies for medicine development, which is a voluntary procedure for assessing the regulatory acceptability of innovative methods used in pharmaceutical research and development. SV95C is an objective, real-world digital ambulation measure of peak performance, representing the speed of the fastest strides taken by the wearer over a recording period of 180 hours. SV95C is correlated with traditional clinic-based assessments of motor function and has greater sensitivity to clinical change over 6 months than other wearable-derived stride variables, for example, median stride length or velocity. SV95C overcomes many limitations of episodic, clinic-based motor function testing, allowing the assessment of ambulation ability between clinic visits and under free-living conditions. Here we highlight considerations and challenges in developing SV95C using evidence generated by a high-performance wearable sensor. We also provide a commentary of the device's technical capabilities, which were a determining factor in the regulatory approval of SV95C. This article aims to provide insights into the methods employed, and the challenges faced, during the regulatory approval process for researchers developing new digital tools for patients with diseases that affect motor function.
在 2019 年，步速 95 百分位（SV95C）成为第一个由可穿戴设备衍生的数字临床结局评估（COA），通过欧洲药品管理局（EMA）的资格认证，可作为杜氏肌营养不良症临床试验的次要终点。SV95C 通过 EMA 的新药开发方法资格认证途径获得批准，这是一个自愿程序，用于评估在药物研发中使用的创新方法的监管可接受性。SV95C 是一种客观的、真实世界的、峰值性能的数字步行测量指标，代表了佩戴者在 180 小时记录期内迈出的最快步伐的速度。SV95C 与传统的基于诊所的运动功能评估相关，并且比其他可穿戴衍生的步速变量（例如，中位数步长或速度）在 6 个月内对临床变化更敏感。SV95C 克服了基于偶发事件的诊所运动功能测试的许多局限性，允许在诊所就诊之间和在自由生活条件下评估步行能力。在这里，我们使用高性能可穿戴传感器生成的证据来强调开发 SV95C 时需要考虑的因素和面临的挑战。我们还对该设备的技术能力进行了评论，这是 SV95C 获得监管批准的决定因素。本文旨在为开发影响运动功能疾病患者的新数字工具的研究人员提供有关监管批准过程中所采用的方法和面临的挑战的见解。
