Data Driven Modelling for Clinical Trials on Alzheimer’s Disease

Most, if not all, papers on data-driven computational modelling of neurodegenerative disease progression only claim to improve clinical trials, e.g., through trial enrichment, and/or reduced sample sizes. This is because individual patient-level data from clinical trials is rarely shared. The A4 Study has bucked this trend — all data became available in July 2024 from this secondary prevention clinical trial of solanezumab in preclinical Alzheimer’s disease. We performed a post hoc subgroup analysis on this data by stratifying trial participant data into brain atrophy map subtypes discovered by Subtype and Stage Inference (SuStaIn). Treatment response differed between subgroups, with one subtype showing a trend towards treatment efficacy. I’ll discuss the implications of this and explore how data-driven brain mapping can be incorporated into clinical trial design.