European regulators’ views on a wearable-derived performance measurement of ambulation for Duchenne muscular dystrophy regulatory trials

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 [1McDonald CM Mercuri E. Evidence-based care in Duchenne muscular dystrophy.Lancet Neurol. 2018; 17: 389-391Abstract Full Text Full Text PDF PubMed Scopus (12) Google Scholar]. 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.