Assessing clinical meaningfulness in clinical trials for Alzheimer's disease: A U.S. regulatory perspective

Assessment of clinical benefit will depend on the stage of Alzheimer's disease (AD) being studied, the clinical symptoms or findings that occur at that stage of disease, and the mechanism of the drug and its anticipated effects.It is critical to obtain input from patients and caregivers with lived experience to understand their perspectives on clinical benefit.The Food and Drug Administration (FDA) encourages the use of clinically meaningful within-patient change, which captures the assessment of improvement or decline based on the perspective of the individual patient, to assess meaningful score differences.