Building a Precision Therapeutics program at a tertiary care Children's Hospital

Background: Pediatric medicine is shifting from population-based approaches to precision medicine, fueled by advances in genomics, pharmacogenomics, and multi-omic data integration. Children, who are disproportionately affected by genetic and rare diseases, stand to benefit significantly from individualized diagnostics and targeted therapies. Cook Children's Health Care System (CCHCS) launched a Precision Therapeutics Program to unify and advance efforts in oncogenomics, pharmacogenomics, genetics, research, and education, establishing a scalable model for pediatric precision care. Objective: To develop and evaluate a comprehensive, cross-disciplinary precision medicine infrastructure integrating molecular diagnostics, clinical care, research, and education, with measurable outcomes in pediatric oncology and rare disease management. Methods: CCHCS implemented a unified precision medicine model built on four pillars: oncology-targeted therapeutics, pharmacogenomics, clinical genetics/genetic oncology, and research-education infrastructure. Multidisciplinary teams-led by physician-scientists, pharmacists, geneticists, and informaticians-coordinated efforts across hospital departments. The model included molecular tumor board discussions, precision medicine clinics, pharmacogenomic consultations embedded in the electronic medical record, and genetic oncology surveillance. Utilization, integration, and educational reach were prospectively tracked. Results: Since 2019, the Molecular Tumor Board has reviewed over 150 pediatric oncology cases, delivering actionable therapies and achieving cost savings through coordinated pharmaceutical oversight. The Precision Medicine Clinic extended targeted and investigational therapies to non-oncologic conditions, improving patient access. Pharmacogenomics services were fully integrated into Epic within 15 months, completing over 100 consultations with high satisfaction and automated decision support. The Genetic Oncology Clinic identified 54 patients with heritable cancer syndromes, enabling early detection and familial cascade testing. Institution-wide education-including monthly accredited conferences, student involvement, and public engagement-further promoted adoption. Conclusion: The CCHCS Precision Therapeutics Program demonstrates that structured, multidisciplinary integration of genomics, pharmacogenomics, and informatics is feasible and transformative in pediatric care. The model highlights collaboration, education, and patient partnership, offering a replicable framework for other institutions seeking to implement sustainable precision medicine ecosystems. Ongoing innovation, research alignment, and family engagement will be crucial for expanding precision care across pediatric specialties.