Key result
Digital outcome measures for motor function outside a clinical setting are feasible and employed across a broad range of rare neurological diseases, though few are robustly validated as clinical trial endpoints.
Why the study?
Developing drugs for rare diseases is challenging and requires precise, objective outcome measures, prompting a summary of progress in digital outcome measures for real-life motor function assessment.
Systematic Review (n=6,590)
Digital outcome measures for motor function in rare neurological diseases are feasible but require further validation for regulatory qualification and widespread adoption as clinical trial endpoints.
Answers are grounded in this paper and cite their sources.
Supports remote motor monitoring in rare neurological diseases; extends feasibility evidence but leaves robust endpoint validation open.
Developing drugs for rare diseases is challenging, and the precision and objectivity of outcome measures is critical to this process. In recent years, a number of technologies have increasingly been used for remote monitoring of patient health. We report a systematic literature review that aims to summarize the current state of progress with regard to the use of digital outcome measures for real-life motor function assessment of patients with rare neurological diseases. Our search of published literature identified 3826 records, of which 139 were included across 27 different diseases. This review shows that use of digital outcome measures for motor function outside a clinical setting is feasible and employed in a broad range of diseases, although we found few outcome measures that have been robustly validated and adopted as endpoints in clinical trials. Future research should focus on validation of devices, variables, and algorithms to allow for regulatory qualification and widespread adoption.
No takes yet. Share an insight, caveat, or question.
Poleur et al. (2023) conducted a systematic review in Rare neurological diseases (n=6,590). Digital outcome measures (wearable sensors, apps, software) vs. Standard clinical outcomes or healthy controls was evaluated on Feasibility and validation of digital outcome measures for real-life motor function assessment. Digital outcome measures for motor function outside a clinical setting are feasible and employed across a broad range of rare neurological diseases, though few are robustly validated as clinical trial endpoints.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: