An article by Captario Head of Science, Stig Johan Wiklund, published in Digital Biomarkers.
Abstract
Digital biomarkers have significant potential to transform drug development, but only a few have contributed meaningfully to bringing new treatments to market. There are uncertainties in how they will generate quantifiable benefits in clinical trial performance and ultimately to the chances of phase three success. Here is a proposed statistical framework and proof-of-concept model with hypothetical digital biomarkers, visualized in a familiar manner to study power calculation.
A Monte Carlo simulation for Parkinson’s disease (PD) was performed using the Captario SUM® platform and illustrative study technology impact calculations were generated. We took inspiration from the EMA-qualified wearable-derived digital endpoint stride velocity 95th centile (SV95C) for Duchenne muscular dystrophy, and we imagined a similar measurement for PD would be available in the future. DaTscan enrichment and “SV95C-like” endpoint biomarkers were assumed on a hypothetical disease-modifying drug pivotal trial aiming for an 80% probability of achieving a study p-value of less than 0.05.
Four scenarios with different combinations of technologies were illustrated. The model illustrated a way to quantify the magnitude of the contributions that enrichment and endpoint technologies could make to drug development studies.
Quantitative models could be valuable not only for the study sponsors but also as an interactive and collaborative engagement tool for technology players and multi-stakeholder consortia. Establishing values of digital biomarkers could also facilitate business cases and financial investments.