Abstract
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.
Original language | English |
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Article number | e31510 |
Journal | Pediatric Blood and Cancer |
Volume | 72 |
Issue number | 3 |
DOIs | |
Publication status | Accepted/In press - 2025 |
All Science Journal Classification (ASJC) codes
- Pediatrics, Perinatology, and Child Health
- Hematology
- Oncology