Abstract
BACKGROUND: The incidence of renal cell carcinoma (RCC) and its associated economic burden have risen significantly in Taiwan. While targeted therapy has become a standard treatment, its real-world effectiveness and cost-effectiveness remain to be fully evaluated. METHODS: This population-based cohort study utilized the Taiwan National Health Insurance Research Database and Cancer Registry to analyze 14,131 RCC cases diagnosed between 1998 and 2016. Key outcomes included life expectancy (LE), loss of LE, and lifetime medical costs. RESULTS: The cumulative incidence rate of RCC increased from 0.37 to 0.73% in men and from 0.23 to 0.36% in women. Significant LE loss was observed, particularly in patients under 50 years of age (14.38 years in men; 12.89 years in women). In advanced cases, targeted therapy yielded a slightly higher LE (4.43 years) compared to non-targeted therapy (3.63 years); however, the loss of LE was similar between groups. CONCLUSION: The real-world relationship between survival outcomes and lifetime medical costs of targeted therapy in Taiwan suggests suboptimal efficiency under current clinical practice. These findings suggest a need to re-evaluate reimbursement strategies by considering pharmacogenomic heterogeneity, implementing genomic profiling for precision medicine, and transitioning toward more effective combination therapy paradigms.