Ohio State University, Columbus, Ohio, USA.
Clin Cancer Res. 2010 Mar 15;16(6):1745-55. doi: 10.1158/1078-0432.CCR-09-2167. Epub 2010 Mar 9.
The National Cancer Institute (NCI) Investigational Drug Steering Committee (IDSC) charged the Biomarker Task Force to develop recommendations to improve the decisions about incorporation of biomarker studies in early investigational drug trials. The Task Force members reviewed biomarker trials, the peer-reviewed literature, NCI and U.S. Food and Drug Administration (FDA) guidance documents, and conducted a survey of investigators to determine practices and challenges to executing biomarker studies in clinical trials of new drugs in early development. This document provides standard definitions and categories of biomarkers, and lists recommendations to sponsors and investigators for biomarker incorporation into such trials. Our recommendations for sponsors focus on the identification and prioritization of biomarkers and assays, the coordination of activities for the development and use of assays, and for operational activities. We also provide recommendations for investigators developing clinical trials with biomarker studies for scientific rationale, assay criteria, trial design, and analysis. The incorporation of biomarker studies into early drug trials is complex. Thus the decision to proceed with studies of biomarkers should be based on balancing the strength of science, assay robustness, feasibility, and resources with the burden of proper sample collection on the patient and potential impact of the results on drug development. The Task Force provides these guidelines in the hopes that improvements in biomarker studies will enhance the efficiency of investigational drug development.
美国国家癌症研究所（NCI）的试验药物指导委员会（IDSC）责成生物标志物工作组制定建议，以改进早期试验药物试验中纳入生物标志物研究的决策。工作组审查了生物标志物试验、同行评议的文献、NCI 和美国食品和药物管理局（FDA）的指导文件，并对研究人员进行了调查，以确定在新药早期开发临床试验中执行生物标志物研究的实践和挑战。本文件提供了生物标志物的标准定义和分类，并列出了向赞助商和研究人员提出的关于将生物标志物纳入此类试验的建议。我们对赞助商的建议侧重于确定和优先考虑生物标志物和检测，协调开发和使用检测的活动，以及开展运营活动。我们还为开发具有生物标志物研究的临床试验的研究人员提供了关于科学原理、检测标准、试验设计和分析的建议。将生物标志物研究纳入早期药物试验非常复杂。因此，进行生物标志物研究的决定应基于平衡科学的强度、检测的稳健性、可行性和资源，以及对患者适当样本采集的负担和结果对药物开发的潜在影响。工作组提供这些准则，希望改善生物标志物研究将提高试验药物开发的效率。
