Hiroyo Kuwabara

Background: The development of later-line drugs for metastatic colorectal cancer (mCRC) has expanded treatment options. However, real-world evidence of treatment sequences and transition rates from early- to later-line treatments are limited. Patients and methods: This was a retrospective study using hospital administrative data from patients in Japan who underwent colorectal cancer surgery or first-line treatment after January 2017. Transition rates were calculated and treatment sequences were summarized using a Sankey diagram. Logistic regression was performed to identify factors associated with the transition from second- to third-line treatment. Results: In total, 27,100 patients (median age: 69 years) were included in the study population. Transition rates to subsequent treatment lines from first to fifth ranged from 66.6% to 71.3% (first to second: 69.4%; second to third: 71.3%; third to fourth: 71.1%; fourth to fifth: 66.6%). Among 9061 patients who received second-line treatment, 6456 continued to third-line treatment, and 2605 received the best supportive care. Longer first- (≥180 days; OR: 1.24; 95% CI: 1.13–1.37) and second-line (≥120 days; OR: 1.70; 95% CI: 1.55–1.86) treatment durations were significant factors for continuing to third-line treatment. Prior therapy with oxaliplatin and irinotecan plus molecular targeted drugs was also associated with a higher likelihood of proceeding to third-line treatment (OR: 1.41; 95% CI: 1.27–1.56). Conclusions: This study describes the current mCRC treatment landscape in Japan. Considering the findings, appropriate early treatments are critical for transition to later-line treatment. Additionally, many later-line options are necessary to provide treatment continuation opportunities for better outcomes.

Motohashi T, et al. J Gynecol Oncol. 2024 Jul;36:e19. https://doi.org/10.3802/jgo.2025.36.e19

Aims: The treatment landscape of renal cell carcinoma has changed with the introduction of targeted therapies. While the clinical benefit of cabozantinib is well established for Japanese patients who have received prior treatment, the economic benefit remains unclear. The objective of this study was to assess the cost-effectiveness of cabozantinib compared with everolimus, axitinib, and nivolumab in patients with advanced renal cell carcinoma who have failed at least one prior therapy in Japan.Methods: A cost-effectiveness model was developed using a partitioned survival approach and a public healthcare payer's perspective. Over a lifetime horizon, clinical and economic implications were estimated according to a three-health-state structure: progression-free, post-progression, and death. Key clinical inputs and utilities were derived from the METEOR trial, and a de novo network meta-analysis and cost data were obtained from publicly available Japanese data sources. Costs, quality-adjusted life-years, and incremental cost-effectiveness ratios were estimated. Costs and health benefits were discounted annually at 2%.Results: Cabozantinib was more costly and effective compared with everolimus and axitinib, with deterministic incremental cost-effectiveness ratios of ¥5,375,559 and ¥2,223,138, respectively. Compared to nivolumab, cabozantinib was predicted to be less costly and more effective. Sensitivity and scenario analyses demonstrated that the key drivers of cost-effectiveness results were the estimation of overall survival and treatment duration, relative efficacy, drug costs, and subsequent treatment costs.Limitations: METEOR was an international trial but did not enroll any patients from Japan. Efficacy and safety data from METEOR were used as a proxy for the Japanese population following validation by clinical experts, and alternative assumptions specific to clinical practice in Japan were evaluated in scenario analyses.Conclusions: In Japan, cabozantinib is a cost-effective alternative to everolimus, axitinib, and nivolumab for the treatment of patients with advanced renal cell carcinoma who have received at least one prior line of therapy.

Background Vedolizumab (VDZ) was approved by the Japanese Ministry of Health, Labor and Welfare in 2018 for the treatment of patients with moderate-to-severe active ulcerative colitis (UC). The comparative cost-effectiveness of VDZ compared with other biologics is unknown in Japan. This information could be useful for decision makers at the time of repricing biologics for the treatment of patients with moderate-to-severe UC. Objective The aim was to assess the cost-effectiveness of VDZ versus other branded biologics for the treatment of patients with moderate-to-severe UC who were anti-tumor necrosis factor (TNF)-naïve, from the Japanese public healthcare payer perspective. Methods A hybrid decision tree/Markov model was developed to predict the number of patients who achieved response and remission at the end of the induction phase and sustained it during the maintenance phase, translating this into quality-adjusted life-years (QALYs) and costs. Treatment-related adverse events, discontinuation and surgery, and their impact on QALYs and costs were also modeled. A systematic literature review and network meta-analysis were conducted to estimate the comparative efficacy of each treatment versus placebo. Rates of adverse events, surgery, surgery complications, and utilities were from the literature. Costs (2018 Japanese yen) were obtained from the Japanese National Health Insurance drug price list and medical fee table and local claims databases. Clinical and economic outcomes were projected over a lifetime and discounted at 2% annually. Results Over a lifetime, VDZ yielded greater QALYs and cost savings compared with golimumab and was cost-effective compared with adalimumab and infliximab (incremental cost-effectiveness ratios ¥4,821,940 and ¥4,687,692, respectively). Deterministic and probabilistic analyses supported the robustness of the findings in the base-case analysis, indicating that VDZ was either dominant or cost-effective in most scenarios and replications. The main limitations of this analysis include excluding tofacitinib and infliximab biosimilar as comparators, health-state utility estimates were obtained from population studies in the United Kingdom, and the impact of subsequent (i.e., second-line) biologic treatment was not evaluated. Conclusion Our analysis suggests that VDZ is dominant or cost-effective compared with other branded biologics for the treatment of anti-TNF-naïve patients with moderate-to-severe UC in Japan.