Analysis of real-world data from the C-CAT database: Accessibility of molecular-targeted agents in pediatric patients with solid tumors

Abstract

[Background] Nearly 3 years ago, comprehensive genomic profiling (CGP) of patients with advanced solid tumors was provided by national health insurance. Center for Cancer Genomics and Advanced Therapeutics (C-CAT) has been aggregating cancer genome data from CGP testing to establish a database optimized to serve the Japanese populace. We summarized here the challenges of accessing molecular-targeted agents for pediatric patients with solid tumors. [Methods] Using the “C-CAT Data Utilization Portal”, we extracted data from 0–15-year-old patients who underwent CGP testing between June 1, 2019, and April 4, 2022, and subsequently received targeted therapy. Using this data, we evaluated the access of this cohort to targeted agents. [Results] Data were obtained from 687 patients (288 patients with CNS tumors and 399 patients with non-CNS tumors), including 352 patients who received at least one molecular-targeted agent option in the C-CAT report. Following CGP testing, 40 (5.8%) patients gained access to a targeted agent due to a genetic alteration, four received an approved drug, eight participated in clinical trials, two received agents through patient-proposed healthcare services, and 26 were treated by off-label use of agents. [Discussion] Only a small portion of pediatric patients receive targeted therapy and off-label use was the major means of access. These results emphasize the importance of facilitating access to targeted agents and establishing evidence for pediatric safety and efficacy.