随着时间的推移，人们越来越关注在癌症医学领域发现和验证生物标志物，这可以指导识别最有可能从治疗中受益的患者、最可能有效的治疗方法以及可能不安全的治疗方法。制定生物标志物指导治疗的必要证据基础是一个与开发新疗法不同的挑战，许多生物标志物已经被纳入常规临床实践中，但在这些实践中，并未进行以评估生物标志物指导方法的直接影响为主要终点的第III期随机研究。这引发了研究和临床界的激烈讨论，探讨生物标志物验证试验的最适当方法以及支持将个体生物标志物驱动方法纳入标准护理的证据水平。这场持续的辩论是优化临床试验设计、最终为患者提供最佳护理的关键，而这个环境越来越注重个性化和以患者为中心的管理。对于生物标志物驱动临床试验设计的最佳方式的持续讨论对于未来临床试验设计的决策至关重要，最终将极大地受益于患者和照顾他们的临床医生。©2023. BioMed Central Ltd., Springer Nature的一部分。

There is an increasing focus over time on the discovery and validation of biomarkers in cancer medicine, which can inform the identification of patients that are most likely to benefit from treatment, which therapy is most likely to be effective, and treatments that may not be safe. BODY: Creating the necessary evidence base for biomarker-informed management is a different challenge to developing a new therapy, and many biomarkers have been adopted into routine clinical practice without phase III randomised studies where the primary endpoint was to evaluate the direct impact of a biomarker-informed approach. This has generated a robust discussion in the research and clinical community regarding the most appropriate trial methodologies for biomarker validation, and the level of evidence required to support the incorporation of individual biomarker-driven approaches as a standard of care. This ongoing debate is key to optimising clinical trial design and ultimately delivering the best possible care to patients in an environment increasingly focused on personalised and patient-focused management.Ongoing deliberation as to the optimal design of biomarker-driven clinical trials is critical to informing future clinical trial design and will ultimately greatly benefit patients and the clinicians that care for them.© 2023. BioMed Central Ltd., part of Springer Nature.