Biomarker definitions and their applications
Biomarker definitions and their applications
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DOI：
10.1177/1535370217750088
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发表时间：
2018-02-01
影响因子：
3.2
通讯作者：
Califf, Robert M.
中科院分区：
文献类型：
--
作者：
Califf, Robert M.
生物标志物对于合理发展医学治疗学至关重要，但在研究和临床实践中，特别是在慢性疾病和营养领域，其使用所涉及的基本定义和概念仍然存在重大混乱。澄清不同生物标志物的定义并更好地理解其适当应用可能会带来重大益处。本文综述了美国食品和药物管理局和国立卫生研究院最近建立的生物标志物定义，作为其联合生物标志物，终点和其他工具（BEST）资源的一部分。这些定义是根据其在患者护理、临床研究或治疗开发中的各自用途来定义的。我们探讨了生物标志物和临床结局评估之间的区别，并讨论了诊断，监测，药效学/反应，预测，预后，安全性和敏感性/风险生物标志物的具体定义和应用。我们还探讨了当前生物标志物发展趋势的影响，包括复杂的复合生物标志物和来自传感器和移动的技术的数字生物标志物。最后，我们讨论了生物标志物驱动的预测毒理学和系统药理学的挑战和潜在好处，确保生物标志物开发科学的质量和可重复性的必要性，以及促进整个医疗产品开发生态系统合作的重要性。
Biomarkers are critical to the rational development of medical therapeutics, but significant confusion persists regarding fundamental definitions and concepts involved in their use in research and clinical practice, particularly in the fields of chronic disease and nutrition. Clarification of the definitions of different biomarkers and a better understanding of their appropriate application could result in substantial benefits. This review examines biomarker definitions recently established by the U.S. Food and Drug Administration and the National Institutes of Health as part of their joint Biomarkers, EndpointS, and other Tools (BEST) resource. These definitions are placed in context of their respective uses in patient care, clinical research, or therapeutic development. We explore the distinctions between biomarkers and clinical outcome assessments and discuss the specific definitions and applications of diagnostic, monitoring, pharmacodynamic/response, predictive, prognostic, safety, and susceptibility/risk biomarkers. We also explore the implications of current biomarker development trends, including complex composite biomarkers and digital biomarkers derived from sensors and mobile technologies. Finally, we discuss the challenges and potential benefits of biomarker-driven predictive toxicology and systems pharmacology, the need to ensure quality and reproducibility of the science underlying biomarker development, and the importance of fostering collaboration across the entire ecosystem of medical product development.