Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings.
Clinical outcome measures for trials in Duchenne muscular dystrophy: report from International Working Group meetings.
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DOI：
10.4155/cli.11.113
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发表时间：
2011-09
期刊：
影响因子：
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通讯作者：
Connor E
中科院分区：
文献类型：
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作者：
Bushby K;Connor E
2010年6月，来自欧洲和美国的25名代表在美国华盛顿举行会议，讨论在临床试验设计和分析背景下杜氏肌营养不良症（DMD）的临床结局指标。该研讨会是为了响应2009年9月欧洲药品管理局会议而组织的，该会议明确指示需要制定国际共识，为DMD新兴疗法临床试验中使用的年龄适当的临床结局措施提供基础。数据来自8个多中心纵向数据集，代表近1900例患者在20年的时间段。这一经验证实了在多个地点进行重复评估的可行性，并解决了DMD药物开发中的几个核心问题，例如类固醇的“新”自然史，特定结局指标的可靠性和敏感性，以及疾病分期和患者选择。这些数据为参与DMD治疗的学术研究人员、制药赞助商和监管机构提供了宝贵的资产。该小组仍然致力于在许多合作目标上共同努力，以支持这种孤儿疾病的治疗开发工作，并将这些数据提供给在该领域工作的利益相关者。
In June 2010, 25 representatives from Europe and the US met in Washington, DC, USA, to discuss clinical outcome measures in Duchenne muscular dystrophy (DMD) in the context of clinical trial design and analysis. The workshop was organized in response to a September 2009 European Medicines Agency meeting where a clear directive was given that an international consensus needs to be developed that provides a foundation for age-appropriate clinical outcome measures for use in clinical trials of emerging therapeutics for DMD. Data were presented from eight multicenter longitudinal datasets, representing nearly 1900 patients over a 20-year time period. This experience confirmed the feasibility of repeated evaluations performed at multiple sites and addressed several core issues in drug development for DMD, such as the ‘new’ natural history in the steroidera, reliability and sensitivity of specific outcome measures, as well as disease staging and patient selection. These data form a valuable asset for academic investigators, pharmaceutical sponsors and regulatory agencies involved in DMD therapeutics. The group remains committed working together on a number of collaborative goals to support the therapeutics development effort in this orphan disease and to make these data available to stakeholders working in the field.