慢性疲劳综合征/肌痛性脑脊髓炎的治疗药物研究指南简介

美国食品和药品监督管理局（FDA）于2014年3月发布了“慢性疲劳综合症/肌痛性脑脊髓炎（CFS/ME）指南草案”。指南草案介绍CFS/ME的临床症状,严重的CFS/ME影响患者的工作、学习和日常生活。目前CFS/ME的诊断比较困难,也没有任何有效药物获批用于该病的治疗,CFS/ME已成为远未得到满足的公众健康问题。在草案中,FDA从药物的临床试验设计等多方面,如目标人群定位、疗效终点、安全性等来指导相关新药的研发。介绍了指南的主要内容,期望为我国这方面的临床试验及药物的开发提供参考。

Brief introduction of guidance for developing drug products used in treatment ofindustry chronic fatigue syndrome/myalgic encephalomyelitis

The US Food and Drug Administration (FDA) issues "Guidance for Industry Chronic Fatigue Syndrome (CFE)/Myalgic Encephalomyelitis (ME): Developing Drug Products for Treatment" in March 2014. Clinical syndromes were contained in the draft guidance. CFS/ME affects the function in daily activities of work, school, household management, and personal care of patients. Currently CFS/ME is a serious disease and there are no approved therapies to treat CFS/ME, which is unmet medical need in the treatment. The guidance focused on specific drug development and clinical trials design issues is drafted, which contain drug development population, efficacy points, and safety etc. This article describes the main contents of the guidelines, expected to provide a reference for the development of this aspect of clinical trials and drug.