Background: The American College of Medical Genetics and Genomics/Association for Molecular Pathology (ACMG/AMP) guidelines represent the gold standard for clinical variant interpretation. Despite widespread adoption, a comprehensive comparison of software tools designed to implement these guidelines has been lacking, creating a significant gap in evidence-based guidance for clinicians.
Methods: We benchmarked four ACMG/AMP-based tools (Franklin, InterVar, TAPES, Genebe) selected from 22 available tools, and compared their performance with LIRICAL, a top-performing phenotype-driven tool. Selection criteria included free availability, VCF compatibility, operational reliability, and absence of disease-specificity. We used 151 expert-curated datasets from Mendelian disorders. Our evaluation framework assessed top-N accuracy (N=1,5,10,20,50), retention rates, precision, recall, F1 scores, and Area Under the Curve (AUC). Statistical validation employed bootstrap confidence intervals (n=1000) and Friedman tests.
Results: LIRICAL (68.21%) and Franklin (61.59%) demonstrated superior top-10 variant prioritization accuracy in Mendelian disorders, significantly outperforming other tools (p=0.0000).
Conclusions: Tools with advanced phenotypic integration significantly outperform those relying primarily on genomic features for variant prioritization in Mendelian disorders.