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

doi:10.1002/pbc.31510

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

研究成果: ジャーナルへの寄稿 › 学術誌 › 査読

抄録

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.

本文言語	英語
論文番号	e31510
ジャーナル	Pediatric Blood and Cancer
巻	72
号	3
DOI	https://doi.org/10.1002/pbc.31510
出版ステータス	印刷中 - 2025

!!!All Science Journal Classification (ASJC) codes

小児科学、周産期医学および子どもの健康
血液学
腫瘍学

UN SDG

この成果は、次の持続可能な開発目標に貢献しています

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10.1002/pbc.31510

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Suzuki, M., Koga, Y., Kawasaki, T., Ueda, T., Yamamoto, S., Goto, H., Kishimoto, J., Ishida, E., Todaka, K., Sonoda, K. H., Oda, Y., Koji, Y., Sakai, Y., & Ohga, S. (Accepted/In press). Access Program for Unapproved and Off-Label Drug Use in Pediatric BRAF V600E-Mutated Brain Tumors in Japan. Pediatric Blood and Cancer, 72(3), 記事 e31510. https://doi.org/10.1002/pbc.31510

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

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.",

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

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year = "2025",

doi = "10.1002/pbc.31510",

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AU - Suzuki, Maya

AU - Koga, Yuhki

AU - Kawasaki, Terumi

AU - Ueda, Tamaki

AU - Yamamoto, Shunsuke

AU - Goto, Hironori

AU - Kishimoto, Junji

AU - Ishida, Eiko

AU - Todaka, Koji

AU - Sonoda, Koh hei

AU - Oda, Yoshinao

AU - Koji, Yoshimoto

AU - Sakai, Yasunari

AU - Ohga, Shouichi

PY - 2025

Y1 - 2025

N2 - 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.

AB - 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.

KW - access program

KW - BRAF V600E-mutated glioma

KW - dabrafenib

KW - patient-proposed healthcare services

KW - trametinib

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DO - 10.1002/pbc.31510

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SN - 1545-5009

VL - 72

JO - Pediatric Blood and Cancer

JF - Pediatric Blood and Cancer

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M1 - e31510

ER -

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

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!!!All Science Journal Classification (ASJC) codes

UN SDG

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