通过幂似然结合实验和观测数据。

IF 1.4 4区 数学 Q3 BIOLOGY
Biometrics Pub Date : 2025-01-07 DOI:10.1093/biomtc/ujaf008
Xi Lin, Jens Magelund Tarp, Robin J Evans
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引用次数: 0

摘要

随机对照试验是因果推理的黄金标准,在现代循证医学中发挥着关键作用。然而,他们使用的样本量往往太有限,无法提供足够的力量来得出因果结论。相比之下,观测数据越来越容易获得,但由于隐藏的混杂因素,可能存在偏差。鉴于这些互补特征,我们提出了一种幂似然方法来增加随机对照试验与观察数据,以提高治疗效果估计的效率。我们提供了一个数据自适应过程来最大化期望对数预测密度(ELPD),以选择最能调节观测数据信息的学习率。我们通过模拟研究验证了我们的方法,该方法显示在保持近似标称覆盖率的同时增加了功率。最后,我们将我们的方法应用于一项真实世界的数据融合研究,该研究扩大了美国健康声明数据集的PIONEER 6临床试验,证明了我们方法的有效性,并就如何解决其应用中的实际问题提供了详细的指导。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Combining experimental and observational data through a power likelihood.

Randomized controlled trials are the gold standard for causal inference and play a pivotal role in modern evidence-based medicine. However, the sample sizes they use are often too limited to provide adequate power for drawing causal conclusions. In contrast, observational data are becoming increasingly accessible in large volumes but can be subject to bias as a result of hidden confounding. Given these complementary features, we propose a power likelihood approach to augmenting randomized controlled trials with observational data to improve the efficiency of treatment effect estimation. We provide a data-adaptive procedure for maximizing the expected log predictive density (ELPD) to select the learning rate that best regulates the information from the observational data. We validate our method through a simulation study that shows increased power while maintaining an approximate nominal coverage rate. Finally, we apply our method in a real-world data fusion study augmenting the PIONEER 6 clinical trial with a US health claims dataset, demonstrating the effectiveness of our method and providing detailed guidance on how to address practical considerations in its application.

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来源期刊
Biometrics
Biometrics 生物-生物学
CiteScore
2.70
自引率
5.30%
发文量
178
审稿时长
4-8 weeks
期刊介绍: The International Biometric Society is an international society promoting the development and application of statistical and mathematical theory and methods in the biosciences, including agriculture, biomedical science and public health, ecology, environmental sciences, forestry, and allied disciplines. The Society welcomes as members statisticians, mathematicians, biological scientists, and others devoted to interdisciplinary efforts in advancing the collection and interpretation of information in the biosciences. The Society sponsors the biennial International Biometric Conference, held in sites throughout the world; through its National Groups and Regions, it also Society sponsors regional and local meetings.
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