From the Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethesda, MD.
Cancer J. 2019 Jul/Aug;25(4):254-263. doi: 10.1097/PPO.0000000000000384.
Oncology clinical trials are undergoing transformation to evaluate targeted therapies addressing a wider variety of biologically defined cancer subgroups. Multiarm basket and umbrella trials conducted under master protocols have become more prominent mechanisms for the clinical evaluation of promising new biologically driven anticancer therapies that are integral to precision oncology medicine. These new trial designs permit efficient clinical evaluation of multiple therapies in a variety of histologically and biologically defined cancers. These complex trials require extensive planning and attention to many factors, including choice of biomarker assay platform, mechanism for processing clinicopathologic and biomarker data to assign patients to substudies, and statistical design, monitoring, and analysis of substudies. Trial teams have expanded to include expertise in the interface between biology, clinical oncology, bioinformatics, and statistics. Strategies for the design, conduct, and analysis of these complex trials will continue to evolve to meet new challenges and opportunities in precision oncology medicine.
肿瘤学临床试验正在发生转变，以评估针对更广泛的生物学定义的癌症亚组的靶向治疗方法。在主方案下进行的多臂篮子和伞式试验已成为更突出的机制，用于临床评估有前途的新的生物学驱动的抗癌治疗方法，这些方法是精准肿瘤医学的组成部分。这些新的试验设计允许在多种组织学和生物学定义的癌症中对多种疗法进行有效的临床评估。这些复杂的试验需要进行广泛的规划并注意许多因素，包括生物标志物检测平台的选择、将临床病理和生物标志物数据处理为子研究以分配患者的机制，以及对子研究的统计设计、监测和分析。试验团队已经扩大到包括生物学、临床肿瘤学、生物信息学和统计学之间的接口专业知识。这些复杂试验的设计、实施和分析策略将继续发展，以应对精准肿瘤医学中的新挑战和机遇。
