癌症患者的最佳管理依赖于晚期肿瘤学随机对照试验。对晚期试验设计和解释的关键考虑因素进行全面了解，对改善随后的试验设计、执行和临床决策至关重要。本综述探讨了晚期肿瘤学试验设计的重要方面。首先，我们讨论了主要终点的选择，包括使用替代终点的优缺点。我们解释了评估肿瘤进展所涉及的挑战，并讨论了减轻偏倚的策略。我们定义了信息性截尾偏倚及其对试验结果的影响，并给出了可能导致信息性截尾的情景的示例。我们强调了对于生存分析的对数秩检验和风险比的传统作用，以及在非比例风险存在时的局限性，介绍了诸如限制性平均生存时间或最大组合等替代生存估计指标。我们强调了优越性和非劣效性试验设计和解释之间的区别，并比较了贝叶斯和频率主义统计方法。最后，我们讨论了在塑造临床管理建议中适当利用二期和三期试验结果，并评估依赖二期数据进行治疗决策的固有风险和益处。版权所有© 2023 Elsevier Inc. 保留所有权利。

Optimal management of cancer patients relies heavily on late-phase oncology randomized controlled trials. A comprehensive understanding of the key considerations in designing and interpreting late-phase trials is crucial for improving subsequent trial design, execution, and clinical decision-making. In this review, we explore important aspects of late-phase oncology trial design. We begin by examining the selection of primary endpoints, including the advantages and disadvantages of using surrogate endpoints. We address the challenges involved in assessing tumor progression and discuss strategies to mitigate bias. We define informative censoring bias and its impact on trial results, including illustrative examples of scenarios that may lead to informative censoring. We highlight the traditional roles of the log-rank test and hazard ratio in survival analyses, along with their limitations in the presence of nonproportional hazards as well as an introduction to alternative survival estimands, such as restricted mean survival time or MaxCombo. We emphasize the distinctions between the design and interpretation of superiority and noninferiority trials, and compare Bayesian and frequentist statistical approaches. Finally, we discuss appropriate utilization of phase II and phase III trial results in shaping clinical management recommendations and evaluate the inherent risks and benefits associated with relying on phase II data for treatment decisions.Copyright © 2023 Elsevier Inc. All rights reserved.