Prospective evaluation of genomics-guided off-label treatment - PubMed
2 hours ago
- #precision oncology
- #off-label drugs
- #genomics-guided treatment
- Study evaluates off-label use of anticancer drugs guided by genomics in patients with advanced solid tumors lacking standard treatments.
- Included 1,610 patients treated with 37 off-label drugs; 1,363 were response-evaluable, with 39.1% having rare cancers.
- Clinical benefit rate was 34.9%, objective response rate was 15.7%, median progression-free survival was 3.4 months, and median overall survival was 8.2 months.
- Grade 3 or higher treatment-related adverse events occurred in 28.4% of patients.
- Results influenced reimbursement decisions in the Netherlands and highlighted benefit in defined molecular subgroups and exceptional responders (7.0%).
- Recommends off-label precision medicine use within frameworks for systematic evaluation, biomarker refinement, and potential label expansion, prioritizing high-confidence targets and international collaboration.