211P Following patient mobility in daily life: the EJP-DT4RD project

The DT4RD (Digital Tools 4 Rare Diseases) project is dedicated to the development of a non-invasive toolbox that measures patient mobility in daily life. Although tremendous progress has been made in this regard in the last few years, there is still a strong need to obtain disease-related information that generates fundamental clinical and patient-relevant outcomes accepted by regulators. Such data can be harnessed through digitised means using tools such as wearable devices, sensors, videos and smartphone apps. For example, movement sensors in the patient's home, worn on the body, or mobility aid can assess general movements whilst distinguishing between the patient's voluntary and involuntary actions. The development of novel clinical outcome assessments (COA) has the potential to facilitate the drug development process across rare diseases and support the improvement of the quality of life for patient care, clinical outcomes and ability to collect information in the home environment. The remote evaluation of patient movements can also reduce indirect costs, such as travel to study sites and lost wages, thereby increasing the accessibility of research studies for patients. The aim of this study is to compare hospital-based COAs with home-based COAs to validate their use for remote monitoring of patients. The recruitment target is 40 patients with confirmed diagnosis of a neuromuscular condition. Participants will be followed for one year with three hospital visits and regular recordings at home. We will present preliminary results obtained from 12 patients followed for six months. This project gives the possibility to question the classical ways of obtaining information from patients and creates new opportunities for acquiring patient-centred real data in daily life. Home monitoring provides a scientifically advanced approach to mobility assessment in clinical trials. By offering objective, continuous, and real-world monitoring of mobility parameters, it enhances data quality, ecological validity, and patient engagement. The integration of home monitoring technologies in clinical trial designs can revolutionise the assessment of mobility outcomes, leading to more accurate and meaningful insights into patients' mobility abilities, changes, and responses to interventions. The DT4RD program is funded by a grant from the European Joint Programme on Rare Diseases and co-funded by Chiesi and CSL Behring.