Design Characteristics and Survival Outcomes of Local Trials Supporting the Approval of Targeted Anticancer Drugs in China.

BACKGROUND: China has rapidly advanced in precision oncology, driven by numerous targeted anticancer drugs approved locally. However, characteristics of design and outcomes of pivotal trials that enrolled Chinese patients and were conducted locally to support these approvals remain unknown. METHOD: We identified all targeted anticancer drugs approved in China before 31 December 2022, and collected the therapeutic outcomes of the trials involving Chinese patients, based on publicly available data and regulatory comments. RESULT: Among 300 oncology indications for 112 targeted anticancer drugs approved in China, 201 (67.0%) were supported by randomized controlled trials (RCTs). Of 201 indications, 36 (17.9%) indications were supported by 36 local RCTs showing outcomes from the Chinese population. Across these RCTs, 22 (61.1%) had a double-blind design and 19 (52.8%) used a placebo as control. Drugs' HRs were 0.68 (95% CI 0.63-0.73, I2 = 37.2%) for OS and 0.47 (95% CI 0.42-0.52, I2 = 81.8%) for PFS, whereas tumor response was 1.63 (95% CI 1.44-1.85, I2 = 82.0%). Targeted anticancer drugs extended survival time by a median of 2.90 months (interquartile range, IQR 2.10-7.40 months) for OS and 2.95 months (IQR 1.85-5.40 months) for PFS. First-in-class drugs had significantly improved OS gain (8.20 months) when compared with non-first-in-class drugs (2.45 months) (p = 0.006). CONCLUSION: Targeted anticancer drugs significantly reduced the risk of death and cancer progression in Chinese populations, although OS gain was modest. However, the local trials involving Chinese patients still need to be increased. This study revealed characteristics of local trial evidence, facilitating treatment decision-making for Chinese physicians and patients and supporting sponsors in planning and designing local trials.