Clinical Outcome Assessments and Digital Health Technologies Supporting Clinical Trial Endpoints in Early Parkinson's Disease: Roundtable Proceedings and Roadmap for Research

Abstract

Roundtable Proceedings and Roadmap for Research N umerous novel drug candidates aiming to slow or stop the progression of Parkinson's disease (PD) are under development, offering hope for a disease that currently has no approved treatments to prevent or delay the onset of progressive disabilities. Many promising candidates are being tested for use in early PD. Regulatory approval for such treatments hinges on the achievement of prespecified endpoints in randomized controlled trials, but there are limited outcome measures that are sensitive to statistically and clinically significant changes in early PD and that have demonstrated sufficient evidence to support the meaningfulness of such changes to patients. While many outcome measures for PD exist, they pose challenges for use in early PD, given the clinical heterogeneity, slow rate of disease progression, lack of functional impairment, and combination of motor and nonmotor symptoms present in the population of patients with early PD. There is thus an urgent need to develop clinical outcome assessments (COAs) that can be used in clinical trials of novel treatments in early PD. Digital health technologies (DHTs), such as mobile