Drug Evaluation Committee Evaluation of Validation Necessary to Use Digital Biomarkers as Key Endpoints in Clinical Trials
July 2025
The Data Science Subcommittee of the Drug Evaluation Committee’s FY2024 Continuing Task Force 10 conducted a case study to determine what evidence is required to utilize digital biomarkers (dBMs) as primary endpoints in clinical development.
With the evolution of digital health technologies, dBMs are playing an important role in clinical trials. This report examines the validation required to utilize dBMs as primary endpoints and demonstrates their usefulness, particularly through the evaluation of SV95C in patients with Duchenne muscular dystrophy (DMD).
Leveraging the unique strengths of the Data Science Subcommittee—where statisticians and data managers are primarily active—this report focuses on verification,validation, and with an Explanation of the objectives and details of the trials actually conducted before SV95C was recognized as a primary endpoint, with the aim of providing useful information to practitioners when formulating development plans.
We hope this document will be of assistance to development personnel, statisticians, wearable device developers, and algorithm developers responsible for data processing as they plan and design clinical trials and develop dBM.