精准医学的新研究设计——篮子试验、伞式试验和平台试验。

Renju Ravi, Harshad V Kesari
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引用次数: 4

摘要

“一刀切”的概念——对患有某种疾病的病人采用一种治疗方法——似乎已经过时了。精准医学的出现促使临床研究发生了深刻的变化,它使研究人员能够更准确地预测特定疾病人群的预防和治疗策略。因此,新颖的研究设计对于建立安全有效的个性化医疗至关重要。篮子、伞式和平台试验设计(统称为主方案)是生物标志物富集设计,允许在一个方案中测试多个假设,从而加速药物开发。这些试验设计是根据患者的风险因素量身定制干预策略,可以帮助预测他们是否对特定治疗有反应。篮子试验评估具有共同分子改变的各种疾病的治疗方法,而伞形试验评估针对单一疾病的多种靶向治疗方法,该方法根据不同的分子改变/风险因素分层为亚组。这些设计是复杂的,它们的主要局限性源于这样一个事实,即仅用分子谱完全取代组织学分型是不合适的。然而,在未来的几十年里,这些试验设计可能会得到普及,并提高临床研究的效率。本文简要概述了主方案设计的特点,并举例说明了使用这些研究设计的已完成和正在进行的临床试验。
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Novel Study Designs in Precision Medicine - Basket, Umbrella and Platform Trials.

The concept of 'one size fits all' - one treatment for patients with a particular disease, seems to be outdated. The advent of precision medicine has prompted profound changes in clinical research and it allows researchers to predict more accurately, the prevention and treatment strategies for a specific disease population. Novel study designs are, therefore, essential to establish safe and effective personalized medicine. Basket, umbrella and platform trial designs (collectively referred to as master protocols) are biomarker enrichment designs that allow for testing more than one hypotheses within a protocol, thus accelerating drug development. These trial designs tailor intervention strategies based on patient's risk factor(s) that can help predict whether they will respond to a specific treatment. Basket trials evaluate therapy for various diseases that share a common molecular alteration, while umbrella trials evaluate multiple targeted therapies for a single disease that is stratified into subgroups based on different molecular alterations/ risk factors. These designs are complex and their major limitations stem from the fact that it would be inappropriate to completely replace histological typing with molecular profiling alone. However, in the upcoming decades, these trial designs are likely to gain popularity and improve the efficiency of clinical research. This article briefly overviews the characteristics of master protocol designs with examples of completed and ongoing clinical trials utilizing these study designs.

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来源期刊
CiteScore
4.80
自引率
9.10%
发文量
55
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