Patient-Reported Outcomes in Cancer Clinical Trials: Measuring Symptomatic Adverse Events With the National Cancer Institute's Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE).
Patient-Reported Outcomes in Cancer Clinical Trials: Measuring Symptomatic Adverse Events With the National Cancer Institute's Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE).
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DOI：
10.14694/edbk_159514
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发表时间：
2016-01-01
期刊：
影响因子：
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通讯作者：
Mitchell, Sandra A
中科院分区：
文献类型：
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作者：
Kluetz, Paul G;Chingos, Diana T;Mitchell, Sandra A
系统地捕捉患者的观点可以为开发新的癌症疗法提供信息。患者报告结局（PRO）通常包括在癌症临床试验中;然而，所使用的结构、测量和分析方法存在异质性，使得这些终点难以解释。有新的努力，以确定严格的方法，以获得高质量和翔实的PRO数据，从癌症临床试验。在这种情况下，PRO用于解决特定的研究目标，并且跨越产品开发生命周期的重要目标是评估安全性和耐受性。美国食品药品监督管理局（FDA）血液学和肿瘤产品办公室（OHOP）已将症状性不良事件（AE）确定为核心PRO概念，对患者报告的症状性AE进行系统评估可提供数据以补充临床医生报告。美国国家癌症研究所的不良事件通用术语标准（PRO-CTCAE）的患者报告结局版本正在由包括FDA在内的多个利益相关者进行评估，并被认为是一种有前途的工具，可提供标准而灵活的方法，从患者的角度评估症状性AE。在本文中，我们简要回顾了FDA OHOP对提交给FDA的癌症试验中PRO的观点，并重点关注使用PRO-CTCAE评估症状性AE。最后，我们讨论了必须做的进一步工作，以扩大PRO-CTCAE的使用，作为一种提供以患者为中心的数据的方法，可以补充癌症临床试验中现有的安全性和耐受性评估。
Systematic capture of the patient perspective can inform the development of new cancer therapies. Patient-reported outcomes (PROs) are commonly included in cancer clinical trials; however, there is heterogeneity in the constructs, measures, and analytic approaches that have been used making these endpoints challenging to interpret. There is renewed effort to identify rigorous methods to obtain high-quality and informative PRO data from cancer clinical trials. In this setting, PROs are used to address specific research objectives, and an important objective that spans the product development life cycle is the assessment of safety and tolerability. The U.S. Food and Drug Administration's (FDA) Office of Hematology and Oncology Products (OHOP) has identified symptomatic adverse events (AEs) as a central PRO concept, and a systematic assessment of patient-reported symptomatic AEs can provide data to complement clinician reporting. The National Cancer Institute's Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) is being evaluated by multiple stakeholders, including the FDA, and is considered a promising tool to provide a standard yet flexible method to assess symptomatic AEs from the patient perspective. In this article, we briefly review the FDA OHOP's perspective on PROs in cancer trials submitted to the FDA and focus on the assessment of symptomatic AEs using PRO-CTCAE. We conclude by discussing further work that must be done to broaden the use of PRO-CTCAE as a method to provide patient-centered data that can complement existing safety and tolerability assessments across cancer clinical trials.