基于非恒定危害的信号检测方法的最佳显著性水平和样本量。

IF 4 2区 医学 Q1 PHARMACOLOGY & PHARMACY Drug Safety Pub Date : 2024-11-01 Epub Date: 2024-07-09 DOI:10.1007/s40264-024-01460-2
Odile Sauzet, Julia Dyck, Victoria Cornelius
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引用次数: 0

摘要

背景和目标:用于检测电子健康记录(EHR)中药物不良反应(ADR)信号的统计方法需要关于最佳显著性水平和样本大小的信息,以达到足够的功率。Sauzet 和 Cornelius 提出了基于 Weibull 型分布危险函数的信号检测测试(WSP 测试),该测试使用了电子健康记录中的时间到事件信息。得出了应用 WPS 检验的最佳显著性水平和样本大小:方法:对样本量、药物所致(不良反应)和非药物所致事件发生率以及不良反应发生的随机时间进行了一系列模拟研究。根据接收者操作特征曲线图的曲线下面积,我们得出了在无假设信号检测环境下实施不同 WSP 检验的最佳显著性水平,以及达到 80% 或 90% 功率所需的近似样本量:结果:推荐使用显著性水平为 0.004 的 dWSP-pPWSP(双 WSP 和功率 WSP 的组合)检验。结果发现,在 ADR 率等于 0.1 的背景率时,功率达到 80% 所需的样本量从 60 个事件开始。背景率为 0.05,ADR 率相当于背景率增加 20% 时所需的事件数为 900:根据这项研究,建议使用 dWSP-pWSP 测试组合进行信号检测,当同一测试应用于所有不良事件时,显著性水平为 0.004,而不取决于不良反应率。
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Optimal Significance Levels and Sample Sizes for Signal Detection Methods Based on Non-constant Hazards.

Background and objectives: Statistical methods for signal detection of adverse drug reactions (ADRs) in electronic health records (EHRs) need information about optimal significance levels and sample sizes to achieve sufficient power. Sauzet and Cornelius proposed tests for signal detection based on the hazard functions of Weibull type distributions (WSP tests) which use the time-to-event information available in EHRs. Optimal significance levels and sample sizes for the application of the WPS tests are derived.

Method: A simulation study was performed with a range of scenarios for sample size, rate of event due (ADRs), and not due to the drug and random time to ADR occurrence. Based on the area under the curve of the receiver operating characteristic graph, we obtain optimal significance levels of the different WSP tests for the implementation in a hypothesis free signal detection setting and approximate sample sizes required to reach a power of 80% or 90%.

Results: The dWSP-pPWSP (combination of double WSP and power WSP) test with a significance level of 0.004 was recommended. Sample sizes needed for a power of 80% were found to start at 60 events for an ADR rate equal to the background rate of 0.1. The number of events required for a background rate of 0.05 and an ADR rate equal to a 20% increase of the background rate was 900.

Conclusion: Based on this study, it is recommended to use the dWSP-pWSP test combination for signal detection with a significance level of 0.004 when the same test is applied to all adverse events not depending on rates.

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来源期刊
Drug Safety
Drug Safety 医学-毒理学
CiteScore
7.60
自引率
7.10%
发文量
112
审稿时长
6-12 weeks
期刊介绍: Drug Safety is the official journal of the International Society of Pharmacovigilance. The journal includes: Overviews of contentious or emerging issues. Comprehensive narrative reviews that provide an authoritative source of information on epidemiology, clinical features, prevention and management of adverse effects of individual drugs and drug classes. In-depth benefit-risk assessment of adverse effect and efficacy data for a drug in a defined therapeutic area. Systematic reviews (with or without meta-analyses) that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by the PRISMA statement. Original research articles reporting the results of well-designed studies in disciplines such as pharmacoepidemiology, pharmacovigilance, pharmacology and toxicology, and pharmacogenomics. Editorials and commentaries on topical issues. Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Drug Safety Drugs may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.
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