因果推断和现实世界的证据:对比阿比拉酸与恩扎胺胺的比较有效性评估
Per Johansson1,2,3,4, Paulina Jonéus1, Sophie Langenskiöld2,5
1Department of Statistics, Uppsala University, Uppsala, Sweden.
PloS one
|October 26, 2023
概括
预发布研究协议可以提高现实世界的证据 (RWE) 质量,以便在监管中使用. 使用已发表的协议进行的观察性研究发现,阿比拉乙酸在晚期前列腺癌中与恩扎胺相比,增加了死亡风险.
科学领域:
- 药监和药物安全 药监和药物安全
- 监管科学中的现实世界证据 (RWE)
- 瘤学和癌症治疗学 癌症治疗学
背景情况:
- 监管机构越来越重视现实世界的证据 (RWE),并随机对照试验的药物批准.
- 确保RWE适用于监管决策是至关重要的.
- 预先公布的研究方案对RWE质量的影响仍在讨论中.
研究的目的:
- 为了证明使用预先发布的协议的观察性研究可以达到随机试验协议的严格性.
- 通过评估在临床实践中阿比拉酸和恩扎胺的比较有效性,以示范这一策略.
- 评估与这些治疗晚期前列腺癌相关的死亡风险.
主要方法:
- 设计了两个互补的观察性研究协议,并制定了预分析计划.
- 在数据采集之前,在clinicaltrials.gov等存储库上发布了协议.
- 在晚期前列腺癌患者中对阿比拉酸盐与恩扎胺进行了比较有效性分析.
主要成果:
- 预先公布的协议使详细的研究设计能够与随机试验相提并论.
- 分析显示,与阿比拉酸处方相关的死亡风险增加.
- 在这种患者群体中,与阿比拉酸相比,恩扎胺与较低的死亡风险有关.
结论:
- 对观察性研究的协议的预发布提高了现实世界的证据的质量和监管适用性.
- 在患有晚期前列腺癌的患者中,阿比拉酸的死亡风险高于恩扎胺.
- 这种方法支持使用真实世界的数据进行强有力的比较有效性研究.
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