将最初试验的疗效数据纳入后续评价:呼吸道合胞病毒疫苗的应用

IF 4.7 2区 医学 Q1 PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH
Epidemiology Pub Date : 2024-03-01 Epub Date: 2023-11-14 DOI:10.1097/EDE.0000000000001690
Joshua L Warren, Maria Sundaram, Virginia E Pitzer, Saad B Omer, Daniel M Weinberger
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引用次数: 0

摘要

背景:当一项随机对照试验未能证明对主要终点有统计学意义的疗效时,需要进行一项潜在昂贵的新试验以获得许可。纳入以前试验的数据可能允许更有效的后续试验来证明有效性,从而加快有效疫苗的供应。方法:基于一项失败的抗呼吸道合胞病毒(RSV)母亲疫苗试验的结果,我们模拟了一项新的贝叶斯组序贯试验的数据。我们要么忽略之前试验的数据(即,弱信息先验分布),要么使用将历史数据纳入分析的先验分布来分析数据。我们评估了新试验的疗效与原试验相同、大于或小于原试验的情况。对于每种情况,我们评估了中期分析后估计疫苗效果的统计效力和I型错误率。结果:当我们使用严格的阈值来控制第一类错误率时,纳入历史数据的分析比没有纳入历史数据的分析有一点优势。如果对第一类错误的控制不太重要(例如,在许可后评估中),则合并历史数据可以大大提高效率。结论:由于需要控制用于疫苗许可的试验中的I型错误率,纳入历史数据在早期停止试验方面几乎没有额外的好处。然而,这些统计方法在许可后使用真实世界证据的评估中可能很有希望。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Incorporating Efficacy Data from Initial Trials Into Subsequent Evaluations: Application to Vaccines Against Respiratory Syncytial Virus.

Background: When a randomized controlled trial fails to demonstrate statistically significant efficacy against the primary endpoint, a potentially costly new trial would need to be conducted to receive licensure. Incorporating data from previous trials might allow for more efficient follow-up trials to demonstrate efficacy, speeding the availability of effective vaccines.

Methods: Based on the outcomes from a failed trial of a maternal vaccine against respiratory syncytial virus (RSV), we simulated data for a new Bayesian group-sequential trial. We analyzed the data either ignoring data from the previous trial (i.e., weakly informative prior distributions) or using prior distributions incorporating the historical data into the analysis. We evaluated scenarios where efficacy in the new trial was the same, greater than, or less than that in the original trial. For each scenario, we evaluated the statistical power and type I error rate for estimating the vaccine effect following interim analyses.

Results: When we used a stringent threshold to control the type I error rate, analyses incorporating historical data had a small advantage over trials that did not. If control of type I error is less important (e.g., in a postlicensure evaluation), the incorporation of historical data can provide a substantial boost in efficiency.

Conclusions: Due to the need to control the type I error rate in trials used to license a vaccine, incorporating historical data provides little additional benefit in terms of stopping the trial early. However, these statistical approaches could be promising in evaluations that use real-world evidence following licensure.

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来源期刊
Epidemiology
Epidemiology 医学-公共卫生、环境卫生与职业卫生
CiteScore
6.70
自引率
3.70%
发文量
177
审稿时长
6-12 weeks
期刊介绍: Epidemiology publishes original research from all fields of epidemiology. The journal also welcomes review articles and meta-analyses, novel hypotheses, descriptions and applications of new methods, and discussions of research theory or public health policy. We give special consideration to papers from developing countries.
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