FDA Funds Digital Biomarker Validation Projects Targeting Wearable Endpoints

Wearable-derived endpoints are moving closer to regulatory acceptability for IND and NDA submissions, as FDA-funded demonstration projects at Massachusetts General Hospital, Biosensics LLC, and the University of Rochester advance clinical validation of digital health technologies (DHTs) across three rare neurological indications.

The three projects target distinct measurement gaps. Massachusetts General Hospital is completing usability and clinical validation of stride velocity 95th centile (SV95C) as a secondary or exploratory endpoint in Phase II and III ALS trials, establishing construct validity, longitudinal responsiveness, and clinically meaningful within-patient change thresholds using Global Rating of Change methodology. For regulatory affairs teams preparing ALS programs, the construct validity and endpoint performance data generated here will be directly relevant to endpoint justification sections in trial protocols and submissions.

Biosensics LLC is pursuing wearable sensor validation in CMT1A, covering foot drop metrics, ambulation metrics acquired passively during activities of daily living, and upper limb use metrics. The study design emphasises objective, scalable, at-home assessment alongside standardised in-clinic measurement, addressing a core 21 CFR Part 312 concern around endpoint reliability across decentralised collection environments. Patient interview components examining perceived ease of use and attitude toward the technology will also inform usability documentation requirements under FDA's 2023 DHT guidance framework.

The University of Rochester's MEND-HD project is evaluating daily living mobility, chorea, physical activity, heart rate variability, and sleep as continuously captured remote endpoints in early-stage Huntington's disease. The project responds directly to the limitations of episodic, subjective traditional measures in detecting subtle early motor features, a gap that becomes operationally significant as disease-modifying interventions enter late-stage development and require sensitive, validated outcome measures. Findings are expected to inform larger-scale validation efforts with potential applicability to other neurological indications.

All three projects are funded through FDA mechanisms including the Broad Agency Announcement and the CERSI Program, positioning the outputs as shared learning resources for external stakeholders rather than proprietary sponsor data. R&D and regulatory affairs teams developing DHT-enabled endpoints should monitor published outputs from these projects as reference datasets for endpoint qualification submissions to FDA's Drug Development Tool qualification program.

Validation data from the SV95C and CMT1A wearable studies will set a measurable precedent for the evidence standard FDA expects when sponsors propose digital mobility endpoints in rare neurological disease programs.

Source: FDA What's New: Drugs RSS Feed via FDA.gov, 30 October 2026.