Drug Evaluation Committee Evaluation of Validation Necessary to Use Digital Biomarkers as Key Endpoints in Clinical Trials
Data Science Subcommittee
July 2025
The Drug Evaluation Committee's Data Science Subcommittee Continuing Task Force 10 in FY2024 reviewed case studies of what evidence is needed to utilize digital biomarkers (dBMs) as key endpoints in clinical development.
As digital health technologies evolve, dBMs are playing an important role in clinical trials. This report discusses the validation considerations necessary to utilize dBM as a primary endpoint and demonstrates its utility, particularly through the evaluation of the SV95C in patients with Duchenne muscular dystrophy (DMD).
This report, taking advantage of the characteristics of the Data Science Subcommittee, in which statisticians and data management personnel are mainly active, focuses on the generation of evidence such as verification and validation, and explains the objectives of the studies that were actually conducted before SV95C was recognized as a primary endpoint and the content of the studies that were conducted. The purpose of this document is to provide useful information for practitioners in planning clinical trials by explaining the purpose of the studies that were conducted before SV95C was recognized as a primary endpoint, and the contents of the studies conducted.
We hope that this document will be of assistance in the development of dBM for those in charge of planning clinical trials, statisticians, wearable device developers, and those in charge of algorithm development for data processing.
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