Risk of Bias and Clinical Benefit of Phase III Trials Supporting FDA-Approved Anticancer Medicines, 2018–2025: A Cross-Sectional Analysis

Among 43 pivotal phase III randomized trials supporting FDA approvals of anticancer drugs, only 30% of trials were at low risk of bias and 33% demonstrated substantial clinical benefit, while just 9% achieved both. FDA regulatory review documents frequently provided methodological information that reduced uncertainty compared with published reports. These findings highlight the importance of integrating trial quality and clinical benefit assessments to strengthen regulatory decision-making, inform clinical practice, and support value-based oncology care.