Assessing safety and efficacy in subpopulations in Alzheimer's disease clinical trials: contextualizing representativeness.

ABSTRACT: Efficacy outcomes in clinical trials are based on well-powered analyes of the entire participating population. Trial populations will comprise many types of demographic and biological subgroups, including individuals of different sexes, groups of older and younger individuals, participants with or without the apolipoprotein E ε4 (APOE) genotype, ethnoracial groups, participants from urban versus rural communities, participants with lower and higher educational levels, or individuals who have or have not undergone previous therapies such as anti-amyloid monoclonal antibodies (MABs). Each subgroups is underpowered to draw definitive outcomes, and analyses can lead to inaccurate conclusions. Disciplined subgroup analysis can be hypothesis generating and can help guide drug development

SUBMITTER: Shan G 

PROVIDER: S-EPMC12664824 | biostudies-literature | 2025 Oct-Dec

REPOSITORIES: biostudies-literature