Design and Rationale for a Real-World Observational Cohort of Patients Living with Progressive Neurological Disease and their care partners: TARGET-NEURO Study

Background: Progressive neurological diseases, such as Alzheimer’s disease and related dementias, Parkinson’s disease, and multiple sclerosis, are leading causes of disability and mortality. Chronic neurological diseases typically develop and progress over many years. Safety and effectiveness data, from long-term real-world sources are needed to guide therapeutic decision making and effectively measure pragmatic trials, and what matters most to patients and their care partners. The increasing availability of data from electronic health records is further enhanced by the growing sophistication of epidemiologic and statistical methods, such as causal inference. Methods: A longitudinal, observational study, TARGET-NEURO, will characterize the natural history of chronic neurological diseases in real world populations, observe practice patterns, directly evaluate patient and care partner experiences and understand the effectiveness and long-term safety of treatments. TARGET-NEURO is a cooperative consortium of centers treating patients with chronic neurological diseases, enrolling over one million participants across three cohorts: 1) Engaged, 2) Disease, and 3) Age. Selection for the Disease cohort is based upon diagnosis codes. Those who consent to completing patient-reported outcomes with optional consent for biospecimen collection, will be enrolled into the Engaged cohort. Care partners are also eligible to participate in surveys. The Age cohort includes adults at least 60 years old, regardless of diagnoses. Conclusions: TARGET-NEURO establishes a large, robust database from participants in both academic medical centers and community practice settings to address important clinical questions. The outcomes can support improved understanding of how different patients progress in their disease and how trajectories vary based on sociodemographic factors, co-morbidities and risk factors. The results can support novel prevention and screening strategies, earlier and more precise diagnoses, and appropriate and equitable use of therapies. Trial registration Clinicaltrials.gov identification number: NCT05796037