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

Maya Suzuki, Yuhki Koga, Terumi Kawasaki, Tamaki Ueda, Shunsuke Yamamoto, Hironori Goto, Junji Kishimoto, Eiko Ishida, Koji Todaka, Koh hei Sonoda, Yoshinao Oda, Yoshimoto Koji, Yasunari Sakai, Shouichi Ohga

Research output: Contribution to journalArticlepeer-review

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 languageEnglish
Article numbere31510
JournalPediatric Blood and Cancer
Volume72
Issue number3
DOIs
Publication statusAccepted/In press - 2025

All Science Journal Classification (ASJC) codes

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

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