数字初级疗效终点的第一个监管资格的证据基础
Laurent Servais1,2, Paul Strijbos3, Margaux Poleur4
1Department of Paediatrics, MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK. laurent.servais@paediatrics.ox.ac.uk.
Scientific reports
|November 29, 2024
概括
步伐速度第95个百分点 (SV95C),一个数字终点,现在已获得EMA资格用于杜氏肌肉衰竭 (DMD) 试验. 数据证实了SV95C的存在.
科学领域:
- 数字健康技术数字健康技术
- 可穿戴式传感器应用程序
- 神经肌肉疾病临床试验 神经肌肉疾病临床试验
背景情况:
- 步伐速度第95个百分点 (SV95C) 是一种可穿戴式衍生度量,代表日常生活中最快5%的步伐.
- 欧洲药物管理局 (EMA) 在2023年7月将SV95C认定为杜申肌力发育不良 (DMD) 试验的首要终点,用于4岁及以上的患者.
- 这标志着EMA首次对数字终点进行资格认证,为数字临床结果评估树立了先例.
研究的目的:
- 提交支持SV95C在DMD临床试验中的EMA资格的数据.
- 提供对合格数字临床结果评估所需的证据基础的见解.
- 突出SV95C的潜力,以改变影响运动障碍的临床发展.
主要方法:
- 来自临床试验,自然史研究和患者调查的数据分析,涉及5至14岁的DMD患者.
- 评估SV95C的准确性,有效性,可靠性,敏感性和临床意义.
- 与传统的DMD评估相比,SV95C在检测疾病进展和治疗效果方面的表现的比较.
主要成果:
- 在研究的DMD人群中,SV95C显示了准确性,有效性,可靠性,敏感性和临床意义.
- SV95C与已建立的DMD结果指标有显著的相关性.
- 与传统评估相比,SV95C在稳定治疗方案的患者中呈现迅速增加和稳定下降,对疾病进展的敏感性更早,更大.
结论:
- 欧洲药物管理局对SV95C的资格验证了其在DMD试验中作为数字终点的使用.
- 在DMD中,SV95C提供了一种敏感且具有临床意义的疾病进展和治疗反应的测量方法.
- 这一监管认可为在治疗罕见疾病和其他影响移动性的疾病的药物开发中采用数字终点树立了先例.
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