临床试验证据支持FDA批准新型治疗剂,2005-2012年
Nicholas S Downing1, Jenerius A Aminawung2, Nilay D Shah3
1Yale University School of Medicine, New Haven, Connecticut.
JAMA
|January 23, 2014
概括
支持美国食品和药物管理局 (FDA) 对新型治疗剂的批准的临床试验证据的强度有很大的不同. 这种变化会影响患者和医生对新疗法的决策.
科学领域:
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
- 监管科学 监管科学
背景情况:
- 医生和患者经常认为新批准的药物的安全性和有效性已经被充分理解.
- 支持美国食品和药物管理局 (FDA) 批准决定的临床试验证据的实际强度尚未被系统评估.
研究的目的:
- 描述用于FDA批准新型治疗剂的关键疗效试验的设计和特征.
- 评估支持近期药物批准的证据质量.
主要方法:
- 对2005年至2012年间批准的新型治疗剂的FDA公开可用的文件进行横截面分析.
- 关键试验根据设计特征进行分类:随机化,盲目化,比较器和试验终点 (代用品,临床结果或临床规模).
- 还分析了患者数量,试验持续时间和完成率.
主要成果:
- 448个关键试验支持了188种新药的206种适应症.
- 大多数试验是随机的,双盲的,并使用活性或安慰剂对比剂.
- 很大一部分 (36.8%) 的适应症是基于单一试验获得批准的.
- 几乎一半的迹象 (45.3%) 完全依赖替代终点,而33.3%使用临床结果.
- 试验特征因治疗领域,孤儿状态和加速批准指定而有所不同.
结论:
- 支持FDA批准新型治疗剂的关键临床试验证据的质量和设计显示出相当大的差异.
- 证据强度的这种异质性对患者和医生的临床决策有重大影响.
- 进一步评估证据质量对于明智使用新药至关重要.
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