Validation of biomarkers to predict response to immunotherapy in cancer: Volume II - clinical validation and regulatory considerations.
Validation of biomarkers to predict response to immunotherapy in cancer: Volume II - clinical validation and regulatory considerations.
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生物标志物的验证以预测癌症免疫疗法的反应：第二卷 - 临床验证和调节考虑。
DOI：
10.1186/s40425-016-0179-0
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发表时间：
2016
影响因子：
10.9
通讯作者：
Thurin M
中科院分区：
文献类型：
--
作者：
Dobbin KK;Cesano A;Alvarez J;Hawtin R;Janetzki S;Kirsch I;Masucci GV;Robbins PB;Selvan SR;Streicher HZ;Zhang J;Butterfield LH;Thurin M
越来越多的人认识到，免疫疗法可能会在未来几年显著改善癌症患者的健康状况。目前，虽然一小部分患者因不同的免疫治疗方法而获得实质性的临床好处，但大多数患者没有，但仍然暴露在显著的药物毒性中。因此，在癌症免疫治疗领域，对预测生物标记物的开发和临床应用的需求越来越大。预测性癌症生物标志物可以用来识别哪些患者可能从特定的治疗方法中受益，哪些患者不可能受益。为了在临床环境中应用，预测性生物标记物必须仔细地引导一个循序渐进的、高度调控的发育过程。这份两卷本文件的第一卷重点介绍了生物标记物开发过程的分析前和分析阶段，提供了背景、实例和“良好做法”建议。在目前的第二卷中，重点放在临床验证、临床实用性的验证和生物标记物开发的监管考虑上。这两卷本系列旨在为整个生物标记物开发过程提供指导，特别关注开发基于免疫的生物标记物的独特方面。具体地说，关于预测性生物标记物临床验证的挑战的知识，从其他情况下的许多成功和失败中获得，将与与偏差和过度匹配有关的统计方法学问题一起审查。还将讨论用于生物标记物临床验证的不同试验设计，因为在生物标记物开发的临床验证阶段，临床指标和终点的选择对于建立生物标记物的临床实用性至关重要。最后，将讨论向美国食品和药物管理局提交生物标记物分析的监管方面，以及欧盟的监管考虑。本文的在线版本(doi：10.1186/s40425-0160179-0)包含补充材料，授权用户可以使用。
There is growing recognition that immunotherapy is likely to significantly improve health outcomes for cancer patients in the coming years. Currently, while a subset of patients experience substantial clinical benefit in response to different immunotherapeutic approaches, the majority of patients do not but are still exposed to the significant drug toxicities. Therefore, a growing need for the development and clinical use of predictive biomarkers exists in the field of cancer immunotherapy. Predictive cancer biomarkers can be used to identify the patients who are or who are not likely to derive benefit from specific therapeutic approaches. In order to be applicable in a clinical setting, predictive biomarkers must be carefully shepherded through a step-wise, highly regulated developmental process. Volume I of this two-volume document focused on the pre-analytical and analytical phases of the biomarker development process, by providing background, examples and “good practice” recommendations. In the current Volume II, the focus is on the clinical validation, validation of clinical utility and regulatory considerations for biomarker development. Together, this two volume series is meant to provide guidance on the entire biomarker development process, with a particular focus on the unique aspects of developing immune-based biomarkers. Specifically, knowledge about the challenges to clinical validation of predictive biomarkers, which has been gained from numerous successes and failures in other contexts, will be reviewed together with statistical methodological issues related to bias and overfitting. The different trial designs used for the clinical validation of biomarkers will also be discussed, as the selection of clinical metrics and endpoints becomes critical to establish the clinical utility of the biomarker during the clinical validation phase of the biomarker development. Finally, the regulatory aspects of submission of biomarker assays to the U.S. Food and Drug Administration as well as regulatory considerations in the European Union will be covered. The online version of this article (doi:10.1186/s40425-016-0179-0) contains supplementary material, which is available to authorized users.
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DOI：
10.1056/nejmoa1003466
发表时间：
2010-08-19
期刊：
The New England journal of medicine
影响因子：
--
作者：
Hodi FS;O'Day SJ;McDermott DF;Weber RW;Sosman JA;Haanen JB;Gonzalez R;Robert C;Schadendorf D;Hassel JC;Akerley W;van den Eertwegh AJ;Lutzky J;Lorigan P;Vaubel JM;Linette GP;Hogg D;Ottensmeier CH;Lebbé C;Peschel C;Quirt I;Clark JI;Wolchok JD;Weber JS;Tian J;Yellin MJ;Nichol GM;Hoos A;Urba WJ
通讯作者：
Urba WJ
影响因子：
11.5
作者：
Pignatti, Francesco;Ehmann, Falk;Rasi, Guido
通讯作者：
Rasi, Guido
影响因子：
45.3
作者：
Cobo, Manuel;Isla, Dolores;Rosell, Rafael
通讯作者：
Rosell, Rafael
影响因子：
64.8
作者：
通讯作者：
--
DOI：
10.1200/jco.2014.59.0703
发表时间：
2015-05-01
期刊：
Journal of clinical oncology : official journal of the American Society of Clinical Oncology
影响因子：
--
作者：
Motzer RJ;Rini BI;McDermott DF;Redman BG;Kuzel TM;Harrison MR;Vaishampayan UN;Drabkin HA;George S;Logan TF;Margolin KA;Plimack ER;Lambert AM;Waxman IM;Hammers HJ
通讯作者：
Hammers HJ