Informing the Tolerability of Cancer Treatments Using Patient-Reported Outcome Measures: Summary of an FDA and Critical Path Institute Workshop
Informing the Tolerability of Cancer Treatments Using Patient-Reported Outcome Measures: Summary of an FDA and Critical Path Institute Workshop
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DOI：
10.1016/j.jval.2017.09.009
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发表时间：
2018-06-01
期刊：
影响因子：
4.5
通讯作者：
Coons, Stephen Joel
中科院分区：
文献类型：
--
作者：
Kluetz, Paul G.;Kanapuru, Bindu;Coons, Stephen Joel
美国食品药品监督管理局和关键路径研究所的患者报告结果（PRO）联盟共同举办了一个关于使用PRO指标评估癌症临床试验安全性和耐受性的研讨会。参与肿瘤药物开发和PRO测量科学的广泛的国际利益相关者提供了关于PRO测量的作用的观点，以提供关于抗癌药物症状性副作用的补充临床数据。发言者和小组成员探讨了来自现有和新出现的PRO措施的信息的效用，重点是国家癌症研究所的不良事件通用术语标准的PRO版本。小组成员和发言者讨论了改善描述症状性不良事件的PRO数据的收集、分析和呈现的潜在方法，以支持药物开发并更好地为监管和治疗决策提供信息。研讨会的参与者在当天结束时讨论了患者报告的研究药物总体副作用负担评估的可能方法，作为潜在的临床试验终点。美国食品和药物管理局重申其承诺，将与国际药物开发利益相关者合作，确定严格的方法，将患者的观点纳入癌症疗法的开发。
The US Food and Drug Administration and the Critical Path Institute's Patient-Reported Outcome (PRO) Consortium convened a cosponsored workshop on the use of PRO measures to inform the assessment of safety and tolerability in cancer clinical trials. A broad array of international stakeholders involved in oncology drug development and PRO measurement science provided perspectives on the role of PRO measures to provide complementary clinical data on the symptomatic side effects of anticancer agents. Speakers and panelists explored the utility of information derived from existing and emerging PRO measures, focusing on the PRO version of the National Cancer Institute's Common Terminology Criteria for Adverse Events. Panelists and speakers discussed potential ways to improve the collection, analysis, and presentation of PRO data describing symptomatic adverse events to support drug development and better inform regulatory and treatment decisions. Workshop participants concluded the day with a discussion of possible approaches to the patient-reported assessment of an investigational drug's overall side effect burden as a potential clinical trial end point. The Food and Drug Administration reiterated its commitment to collaborate with international drug development stakeholders to identify rigorous methods to incorporate the patient perspective into the development of cancer therapeutics.