Introduction
Digital biomarkers derived from wearable sensors, mobile devices, and connected health technologies are increasingly investigated as clinical trial endpoints capable of quantifying disease progression and treatment response with unprecedented temporal resolution. These technologies provide continuous measurements of physiological and behavioral signals in real-world settings and therefore hold the potential to transform the evidentiary basis of drug development. Despite this promise, technological advancement and regulatory acceptance of digital biomarkers as primary efficacy endpoints have been limited by challenges related to analytical validation, clinical interpretability, statistical robustness, and operational feasibility.
Objectives
- What are the regulatory hurdles for the acceptance of digital biomarkers as primary and secondary endpoints? How can digital biomarkers meet the standards set by regulatory agencies?
- How do we define validation in the context of rare diseases and small population research, and how do we validate digital biomarkers across different rare disease populations, devices, and settings? What suitable standards need to be in place to ensure comparability across trials and generalizability to different patient groups, recognising the challenges?
- What resources expertise is needed to develop, qualify/validate, and use digital biomarkers as primary and or secondary endpoints in clinical trials?
- How can we ensure that the digital devices used (e.g., wearables, mobile apps, sensors) are compatible with the clinical trial infrastructure and that they adhere to proper technical standards?
Timeline
Working Group assembly Q1-2026
Recommendations Q1-2027
Members
- Rajesh Krishna (Co-Chair) – Certara, USA
- Daniel O’Connor (Co-Chair) – The Association of the British Pharmaceutical Industry, UK
- Jill Weimer – Sanford Research, USA
- Marjon Pasmooj – Dutch Medicines Evaluation Board, Netherlands
- Michelle Farrar – Sydney Children’s Hospital Randwick, Australia
- Lindsey Murray – Critical Path, USA
- Ally Peter Omary – Africa CDC Youth in Digital Health Network, Tanzania
- Anupriya Parthiban – Centogene, India
- Jeremy Slayter – Dalhousie University, Canada
- Jin Lee Cure – NDD and Education in X-linked Neurodevelopmental Disorders, USA
- Julieta Beatriz Bonvin Sallago – Connecticut Children’s Medical Center, USA
- Lucia Cesnakova – Digital Medicine Society, Czech Republic
- Ozan Emre EYUPOĞLU – IstanbulMedipolUniversity, Turkey
- Thomas Morel – UCB, Belgium
- Venkat Chintapalli – AstraZeneca, USA
- Sylvia Cho – FDA, CBER, USA