Research Brief

AI-Driven Diagnosis Accelerates Undiagnosed Rare Syndromes

A collaboration between Toronto's SickKids, São Paulo's Genomic Center, and Tokyo Medical AI Lab demonstrates how multimodal machine learning fast-tracks diagnoses for children with unresolved rare phenotypes.

Model Architecture

The ORIGIN platform merges facial phenotyping, clinical notes embeddings, and trio exome sequencing data. A transformer-based fusion layer prioritizes candidate diagnoses.

Performance

Top-5 diagnosis accuracy reached 84% across 1,100 cases, cutting time-to-diagnosis from 24 months to 7 months.

Bias Mitigation

Model retraining with diverse phenotypic datasets reduced accuracy gaps across ancestry groups from 18% to 5%.

Clinical Integration

Clinicians upload patient data through a secure portal. AI-generated differential diagnosis lists align with HPO terms and suggest confirmatory tests.

  • • Integration with telehealth consults for remote triage.
  • • Automated consent workflows for data sharing across borders.
  • • API endpoints enabling linkage to national newborn screening registries.

Pilot Hospitals

  • SickKids, Toronto, Canada
  • Hospital das Clínicas, São Paulo, Brazil
  • Tokyo Medical AI Lab, Japan
  • Red Cross Children's Hospital, Cape Town, South Africa

Global Ethics Council ensures privacy safeguards and algorithmic transparency.

Future Directions

Next-phase improvements include federated learning to remove central data pooling, expanded cohort enrollment from South Asia, and patient-facing explainability dashboards.

  • • Collaboration with WHO Rare Disease Working Group for standardization.
  • • Open benchmark challenges inviting external validation.
  • • Funding pipeline via Global Innovation Fund to support scale-up.