Presentation (CTI)

OBJECTIVES: The digital transformation of healthcare has accelerated the integration of digital health technologies (DHTs) into clinical research, enabling the development of novel digital endpoints. This study aims to synthesise current knowledge on digital endpoints and assess their implications for clinical development and subsequent regulatory and reimbursement decision-making.
METHODS: A scoping review was conducted to identify and analyse English-language literature on digital endpoints derived from DHTs. Articles were included if they discussed the development, application, and regulatory considerations of digital endpoints, or the appraisal of data derived from digital endpoints for the purpose of product value assessment as part of Health Technology Assessment (HTA) processes. Relevant data were extracted using a standardised charting form and analysed thematically to identify key challenges and trends.
RESULTS: Thirty publications met the inclusion criteria. Since 2019, there has been a steady rise in literature on DHT use, with 57% focusing on the relevance and impact of digital endpoints and 53% addressing regulatory considerations. Regulatory bodies and HTA agencies increasingly acknowledge the value of DHT-derived endpoints, offering guidance to support their development, however there are material regulatory differences between countries stemming from varying approaches to data validation, evidence requirements and terminology variability. A notable milestone is the European Medicines Agency’s (EMA) 2023 qualification of stride velocity 95th centile as a primary endpoint in ambulatory Duchenne Muscular Dystrophy trials—highlighting regulatory acceptance of digital measures. While foundational principles for digital endpoint development align with traditional endpoints, uncertainties remain around validation, qualification processes, and evidentiary standards for clinical relevance.
CONCLUSIONS: Digital endpoints offer promising avenues to capture real-world, patient-centric outcomes, and enhance the sensitivity of clinical trials. However, gaps persist in the regulatory and evidentiary ecosystem, posing challenges to their adoption. Addressing these gaps is critical to unlocking the full potential of digital endpoints in advancing regulatory and HTA decision-making.