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

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...

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Hauptverfasser: Suzuki, Maya, Koga, Yuhki, Kawasaki, Terumi, Ueda, Tamaki, Yamamoto, Shunsuke, Goto, Hironori, Kishimoto, Junji, Ishida, Eiko, Todaka, Koji, Sonoda, Koh‐hei, Oda, Yoshinao, Koji, Yoshimoto, Sakai, Yasunari, Ohga, Shouichi
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container_title Pediatric blood & cancer
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creator Suzuki, Maya
Koga, Yuhki
Kawasaki, Terumi
Ueda, Tamaki
Yamamoto, Shunsuke
Goto, Hironori
Kishimoto, Junji
Ishida, Eiko
Todaka, Koji
Sonoda, Koh‐hei
Oda, Yoshinao
Koji, Yoshimoto
Sakai, Yasunari
Ohga, Shouichi
description 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.
doi_str_mv 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
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