Access Program for Unapproved and Off‐Label Drug Use in Pediatric BRAF V600E‐Mutated Brain Tumors in Japan

Programs allowing access to investigational drugs and off‐label drug use for serious diseases have often been applied to pediatric cancers. A clinical study conducted under the Japanese “Patient‐Proposed Healthcare Services” evaluated the efficacy and safety of dabrafenib plus trametinib in children with BRAF V600 mutant glioma (jRCTs071210071). This study successfully provided unapproved and off‐label medications to four enrolled patients, two with low‐grade glioma and two with high‐grade glioma (median age: 10.5 years), until regulatory approval. The timeframe and data collection from such access programs need to be optimized for pediatric patients in accordance with the healthcare system of each nation.