NINDS
Advancing Digital Endpoints and Digital Health Technologies for Neurological and Neuromuscular Disorders
About
Background
The NINDS and NICHD/NCMRR, in collaboration with NIA, NIMH, and NIAMS, are co-sponsoring a three-day virtual workshop to share lessons from current digital endpoint validation efforts, highlight existing and emerging digital health technologies, and discuss shared gaps and opportunities across neurological and neuromuscular disorders. This multi-stakeholder workshop will bring together clinical and translational researchers from a diverse spectrum of neurological and neuromuscular conditions along with device developers, regulators, industry professionals, and patient advocacy organizations/non-profits and individuals with lived experience. Goals of the workshop are to discuss best practices and lessons learned from existing validation efforts, highlight available and emerging digital health technologies, and discuss shared needs, gaps, and opportunities for collaboration among different neurological and neuromuscular communities. Each day of the workshop is centered on a set of functional domains, and closes with a special session or topic area such as regulatory pathways and breakout sessions.
Digital endpoints are expected to revolutionize clinical trials by enabling real-world data collection with more granular longitudinal data, increasing participation of under-represented study participants, and improving participant engagement and retention. However, regulatory acceptance of endpoints used in clinical trials requires extensive fit for purpose validation and must be able to demonstrate that quantifiable changes in the endpoints reflect clinically meaningful changes in how participants feel, function, or survive. Digital Health Technologies (DHTs), used to measure digital endpoints, include wearable sensors, mobile/app based cognitive or functional assessments, and in-home video analysis. Advancing DHT development and digital endpoint validation is expected to advance therapeutic development and improve patient care. Currently there are only a few neurological and neuromuscular conditions with active digital endpoint validation efforts underway, however there are many more conditions that could benefit from emerging technologies and collaborating on shared digital endpoint validation needs.