Identifying Digital Endpoints to Assess FAtigue, Sleep and acTivities in daily living in Neurodegenerative disorders and Immune-mediated inflammatory diseases

Fatigue and sleep disturbances are two common and disabling symptoms that affect patients with neurodegenerative disorders (NDD) and immune-mediated inflammatory diseases (IMID). These symptoms are major predictors of poor quality of life and increased healthcare cost. Current questionnaire-based approaches to measure these symptoms have key limitations preventing them from being used as reliable endpoints in clinical trials to evaluate the effect of therapies. IDEA-FAST aims to address these issues by identifying novel digital endpoints for fatigue and sleep disturbances that will provide more objective, sensitive and reliable measures of the severity and impact of these symptoms in ecological settings. Such digital endpoints will not only help to gain insight into the underpinning mechanisms of fatigue and sleep disturbances, but will also vastly improve the efficiency of clinical trials, ultimately reducing the time and cost to bring new therapies to patients.To identify these digital endpoints, we will follow the recommendations of the Clinical Trials Transformation Initiative (CTTI). We will identify the characteristics that fatigue and sleep disturbances will have impact, then select the digital measures (endpoints) to quantify them, followed by choosing the appropriate digital device/technology accordingly. We will then perform a pilot study to prioritise a few of these candidate digital endpoints for validation. We will test the performance of these digital endpoints in two NDD and four IMID in a large longitudinal study during which extensive relevant clinical data will be collected. If these digital endpoints were validated, we will seek support from EMA/FDA for their qualification. Patient users’ perspective, ethical, data privacy, legal and other regulatory issues will be taken into consideration in all aspects of our proposal. The resultant digital biobank from the longitudinal study will become an invaluable resource for future exploitation.