Epidemiological Characteristics, Target Distribution, and Clinical Value of Targeted Anticancer Drugs Approved in...

China has advanced both domestic innovation and the introduction of imported targeted anticancer drugs (TADs). However, characteristics of these TADs remain unclear. In this cross-sectional study, we identified TADs approved in China from 2017 to 2024. Trend in number, target distributions, endpoint evidence, clinical benefit (via European Society for Medical Oncology-Magnitude of Clinical Benefit Scale [ESMO-MCBS]), and the price were analyzed. Of the approved 143 TADs (315 indications), 64 (44.8%) (145 indications [46.0%]) were domestically developed and 79 (55.2%) (170 indications [54.0%]) were imported, with annual growth rates of 31.0% for domestic TADs (incidence rate ratio 1.310, 95% CI; 1.158-1.482; P < 0.001) vs. 9.8% for imported TADs (1.098, 95% CI; 0.992-1.215; P = 0.071). A total of 52 drug targets were identified (23 domestic; 47 imported). Imported TAD indications more often reported overall survival (OS) than domestic drugs (44.1% vs. 29.0%; P = 0.006), but domestic TAD indications showed better OS (hazard ratio 0.69, 95% CI 0.66-0.71 vs. 0.73, 95% CI 0.71-0.76). Domestic TAD indications were predominantly rated as having intermediate clinical benefit (57.9% vs. 35.9% for imported), while high benefit was less frequent (22.1% vs. 35.3%) and low benefit slightly less common (20.0% vs. 28.8%). Median monthly treatment prices were consistently higher for imported than domestic TADs across all drug categories. In conclusion, both domestic and imported TADs continued to increase. Domestic TAD targets were highly concentrated. Fewer than half of domestic and imported TAD indications reported OS, and less than 40% in either group achieved high-grade clinical benefit. Imported TADs had higher monthly treatment prices than domestic TADs.