用家用视频进行专家审查,为单臂试验与外部控制生成盲证的策略
Xinruo Zhang1,2, Katherine Brind'Amour1,3, Kelly E King4
1CERobs Consulting, LLC, Wrightsville Beach, NC, USA.
Therapeutic innovation & regulatory science
|August 17, 2023
概括
一种新方法使用家庭视频的盲目专家审查来评估神经退行性疾病儿童的发育里程碑,为临床试验提供了可扩展和家长友好的方法.
科学领域:
- 儿科神经学 儿科神经学
- 临床试验方法论 临床试验方法论
- 结果测量 结果测量
背景情况:
- 儿童的神经退行性疾病导致发育延迟和里程碑的丧失.
- 现有的结果指标缺乏可扩展性和父母/照顾者 (P/CG) 对临床试验的意义.
- 需要客观的,监管所接受的措施,以提高儿科神经退行性疾病的治疗疗效.
研究的目的:
- 开发和试验一种新的,盲目的策略,用于评估儿科神经退行性疾病临床试验中的发育里程碑.
- 创建一个可扩展的结果测量,包括P/CG输入,并利用家庭视频录像.
- 为弥补这一群体监管所接受的,有意义的结果措施的差距.
主要方法:
- 通过P/CG和专家采访确定了关键的发展里程碑.
- 家庭录像记录儿童执行特定活动的标准化程序.
- 实施视频评估的盲目专家小组审查过程,随机插入录制以进行质量控制.
主要成果:
- P/CGs发现指令清晰,活动相关,在家进行记录比在诊所进行评估更可取.
- 专家评级人员发现视频易于评估,里程碑具有临床意义.
- 试点研究表明了标准化战略的可行性.
结论:
- 使用家庭视频对发展里程碑进行盲目专家评分的标准化策略是可行的和有效的.
- 这种方法提供了一个严格的,客观的评分系统,适用于各种疾病环境,有或没有外部控制.
- 该方法允许灵活的数据收集和未来的视频存档以进行进一步分析.
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