Abstract
Previous studies have demonstrated that 10%-25% of patients receive gene-matched therapy (GMT) after comprehensive genomic profiling (CGP). However, its real-world clinical effects remain unclear. This study assessed the feasibility of integrating the Center for Cancer Genomics and Advanced Therapeutics (C-CAT) repository that documented genomic and clinical data and the quality indicator (QI) dataset that included cancer-specific data and administered treatment as a model case of real-world data study of cancer genomic medicine in Japan. We successfully integrated these two datasets and included 1162 patients diagnosed with solid tumors at the National Cancer Center Hospital between 2019 and 2021 who underwent CGP testing. Of these, 432 (37.2%) had druggable mutations, 96 (8.3%) received GMT, and 218 (18.8%) received non-GMT. Among 314 patients who initiated either GMT or non-GMT after CGP, the median 2-year overall survival (OS) was 19.0 and 19.7 months for GMT and non-GMT, respectively (hazard ratio: 0.87, 95% confidence interval: 0.56-1.35, p = 0.53). Stratified analysis by prior treatment lines (0-1 vs. ≥ 2) demonstrated no significant differences in survival. Sensitivity analyses yielded consistent results. This study demonstrated that integrating the C-CAT repository and QI datasets enables real-world comparisons of GMT and non-GMT outcomes. Unlike previous clinical trials reporting enhanced survival with GMT, our findings indicated no significant OS difference. Potential explanations include differences in cancer type, CGP timing, study population selection, and immortal time bias. Future multicenter studies would clarify the real-world utility of the CGP and GMT.