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 conducted a case study on what kind of evidence is needed to utilize digital biomarkers (dBM) 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 purpose of the actual studies conducted before SV95C was recognized as the primary endpoint, the contents of the conducted studies, and the results of the studies. Explanation of the purpose of the studies that were conducted before SV95C was recognized as a primary endpoint, as well as the contents of the studies conducted, is intended to provide useful information for those in charge of clinical development planning.
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.