CHARACTERIZING THE USE OF DIGITAL HEALTH TECHNOLOGY-DERIVED ENDPOINTS TO CAPTURE PATIENT EXPERIENCE DATA IN CLINICAL TRIALS
Author(s)
Khang Nguyen, PharmD1, Jaymin Patel2, Danny Yeh, PhD3;
1University of North Carolina at Chapel Hill, chapel hill, NC, USA, 2AESARA, Associate Director - HEOR, Wake Forest, NC, USA, 3Aesara, Burlingame, CA, USA
1University of North Carolina at Chapel Hill, chapel hill, NC, USA, 2AESARA, Associate Director - HEOR, Wake Forest, NC, USA, 3Aesara, Burlingame, CA, USA
OBJECTIVES: Patient experience data (PED) has expanded with digital health technologies (DHTs). DHT derived measures can complement traditional endpoints by providing continuous, real-world insights into treatment experience, yet the literature offers limited detail on how they are used within clinical trials. This review characterizes current approaches to using DHTs as trial endpoints and identifies themes for integration.
METHODS: Due to inconsistent reporting of DHT use in public registries, we conducted a targeted search of Embase and PubMed (2015 to 2025) for clinical trials reporting DHT-derived endpoints used to support pharmaceutical product benefits. We extracted device type (eg, accelerometers, smartphone apps, smartwatches), DHT based outcome measures, clinical concepts of interest (eg, activity, mobility, sleep), and digital outcome results.
RESULTS: Thirty one studies were identified across neurology, rheumatology, pulmonary and respiratory disease, genetic disorders, and dermatology. Most evaluated DHT endpoints as exploratory or secondary outcomes (n=28); three used them as primary endpoints. Digital endpoints most often measured physical activity, mobility or gait, and motor function (n=19), with fewer assessing sleep (n=12), balance (n=3), or posture (n=2). Data collection relied mainly on wearable accelerometers and actigraphy, with limited use of smartphone or smartwatch inertial sensors. Endpoint definitions and analytic approaches varied widely, and few studies reported systematic psychometric evaluation of DHT endpoints, such as reliability, validity, or sensitivity to change. Common challenges included high variability in sensor measures, weak alignment with clinical and patient reported outcomes, and declining adherence over time.
CONCLUSIONS: Interest in DHT derived endpoints in clinical trials is increasing, but variability in devices, validation, and implementation limits interpretability and comparability. Clearer guidance on endpoint development and validation, standardization of analytic approaches, and practical implementation of playbooks may improve regulatory readiness and accelerate adoption of DHT derived endpoints in trials.
METHODS: Due to inconsistent reporting of DHT use in public registries, we conducted a targeted search of Embase and PubMed (2015 to 2025) for clinical trials reporting DHT-derived endpoints used to support pharmaceutical product benefits. We extracted device type (eg, accelerometers, smartphone apps, smartwatches), DHT based outcome measures, clinical concepts of interest (eg, activity, mobility, sleep), and digital outcome results.
RESULTS: Thirty one studies were identified across neurology, rheumatology, pulmonary and respiratory disease, genetic disorders, and dermatology. Most evaluated DHT endpoints as exploratory or secondary outcomes (n=28); three used them as primary endpoints. Digital endpoints most often measured physical activity, mobility or gait, and motor function (n=19), with fewer assessing sleep (n=12), balance (n=3), or posture (n=2). Data collection relied mainly on wearable accelerometers and actigraphy, with limited use of smartphone or smartwatch inertial sensors. Endpoint definitions and analytic approaches varied widely, and few studies reported systematic psychometric evaluation of DHT endpoints, such as reliability, validity, or sensitivity to change. Common challenges included high variability in sensor measures, weak alignment with clinical and patient reported outcomes, and declining adherence over time.
CONCLUSIONS: Interest in DHT derived endpoints in clinical trials is increasing, but variability in devices, validation, and implementation limits interpretability and comparability. Clearer guidance on endpoint development and validation, standardization of analytic approaches, and practical implementation of playbooks may improve regulatory readiness and accelerate adoption of DHT derived endpoints in trials.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
PCR37
Topic
Patient-Centered Research
Disease
No Additional Disease & Conditions/Specialized Treatment Areas