Biomarker-Driven Oncology Clinical Trials: Novel Designs in the Era of Precision Medicine.

Donald C Moore, Andrew S Guinigundo
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Abstract

Oncology drug development historically has followed a path of sequential phase I, II, and III clinical trials using traditional trial designs, with the goal of achieving regulatory approval. These studies are often conducted with inclusion criteria that limit enrollment to a single tumor type or tumor site of origin, excluding other patients who might also respond. Increased use of precision medicine targeting biomarkers or specific oncogenic mutations has led to novel clinical trial designs that can evaluate these therapies in a less limited fashion. Master protocols such as basket trials, umbrella trials, and platform trials can, for example, evaluate histology-specific therapies targeting a common oncogenic mutation across multiple tumor types or screen for the presence of multiple different biomarkers rather than a single one. In other cases, they can lead to more rapid evaluation of a drug and evaluate targeted therapies in tumor types for which they are not yet currently indicated. As the use of complex biomarker-based master protocols increases, advanced practitioners must understand these novel trial designs, their advantages and disadvantages, and how their use may advance drug development and maximize the clinical benefits of molecular precision therapy.

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生物标志物驱动的肿瘤临床试验:精准医学时代的新设计。
从历史上看,肿瘤药物的开发一直遵循使用传统试验设计的顺序I、II和III期临床试验的路径,目标是获得监管部门的批准。这些研究通常采用纳入标准,将入组限制在单一肿瘤类型或肿瘤起源部位,排除了其他可能也有反应的患者。越来越多地使用针对生物标志物或特定致癌突变的精准医学,导致了新的临床试验设计,可以以不那么有限的方式评估这些疗法。例如,篮子试验、保护伞试验和平台试验等主方案可以评估针对多种肿瘤类型的常见致癌突变的组织学特异性治疗,或筛选多种不同生物标志物的存在,而不是单一的。在其他情况下,它们可以更快地评估一种药物,并评估目前尚未指明的肿瘤类型的靶向治疗。随着基于复杂生物标志物的主方案的使用增加,高级从业人员必须了解这些新颖的试验设计,它们的优点和缺点,以及它们的使用如何促进药物开发和最大化分子精确治疗的临床效益。
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