Closing the Gap: Improving Drug Access for Pediatric and AYA Cancer Care

May 15, 2026

In Japanese

Since January 2024, the National Cancer Center Japan (NCCJ) has been conducting the PARTNER trial (NCCH2220) under Japan’s Patient-Proposed Healthcare Services framework, in collaboration with Hokkaido University, Kyushu University, Okayama University and Nagoya University. Designed to expand access to off-label drugs or unapproved drugs in Japan for pediatric and adolescent and young adult (AYA) cancer patients, this investigator-initiated clinical research evaluates the safety and efficacy of the drugs.

In March 2026, after confirming safety in pediatric and AYA patients, the PARTNER trial moved forward with the new cohort investigating eflornithine hydrochloride for patients newly diagnosed with high-risk neuroblastoma (HRNB). Although eflornithine hydrochloride was approved by the U.S. Food and Drug Administration (FDA) for maintenance therapy for HRNB in December 2023, it remains unapproved in Japan. As a result, no approved treatment option is currently available, leaving patients and their families eagerly awaiting access to this therapy as soon as possible.

Dr. Ayumu Arakawa, Chief of the Department of Pediatric Oncology and the Principal Investigator of the trial, commented enthusiastically: ‘With the addition of the new eflornithine cohort, we hope that patients seeking additional treatment options will have the opportunity to receive this therapy under appropriate physician supervision. Furthermore, the data on treatment efficacy and safety obtained through this trial are expected to contribute to the future provision of beneficial medical care for patients, as well as to serve as supporting evidence for regulatory approval and reimbursement considerations. Moving forward, we will continue to expand the participating institutions and target drugs in order to provide treatment opportunities to a greater number of patients.’

Through initiatives such as the PARTNER trial, NCCJ will continue working to responsibly expand cancer patients’ access to promising new treatment options that were previously unavailable in Japan, while generating evidence to inform future regulatory review and clinical practice.