Abstract
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.