Reproducibility and replicability of high-frequency, in-home digital biomarkers in reducing sample sizes for clinical trials

Alzheimers Dement (N Y). 2021 Dec 31;7(1):e12220. doi: 10.1002/trc2.12220. eCollection 2021.

Abstract

Introduction: Reproducibility and replicability of results are rarely achieved for digital biomarkers analyses. We reproduced and replicated previously reported sample size estimates based on digital biomarker and neuropsychological test outcomes in a hypothetical 4-year early-phase Alzheimer's disease trial.

Methods: Original data and newly collected data (using a different motion sensor) came from the Oregon Center for Aging & Technology (ORCATECH). Given trajectories of those with incident mild cognitive impairment and normal cognition would represent trajectories of the control and experimental groups in a hypothetical trial, sample sizes to provide 80% power to detect effect sizes ranging from 20% to 50% were calculated.

Results: For the reproducibility, identical P-values and slope estimates were found with both digital biomarkers and neuropsychological test measures between the previous and current studies. As for the replicability, a greater correlation was found between original and replicated sample size estimates for digital biomarkers (r = 0.87, P < .001) than neuropsychological test outcomes (r = 0.75, P < .001).

Discussion: Reproducibility and replicability of digital biomarker analyses are feasible and encouraged to establish the reliability of findings.

Keywords: early prevention; linear mixed‐effect models; mild cognitive impairment; randomized controlled trials; technology assessment.