DESIGN DIFFERENCES EXPLAIN VARIATION IN RESULTS BETWEEN RANDOMIZED TRIALS AND THEIR NON-RANDOMIZED EMULATIONS.
DESIGN DIFFERENCES EXPLAIN VARIATION IN RESULTS BETWEEN RANDOMIZED TRIALS AND THEIR NON-RANDOMIZED EMULATIONS.
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设计差异解释了随机试验与其非随机模拟之间结果的差异。
DOI：
10.1101/2023.07.13.23292601
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发表时间：
2023
期刊：
影响因子：
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通讯作者：
Wang,ShirleyV
中科院分区：
文献类型：
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作者：
Heyard,Rachel;Held,Leonhard;Schneeweiss,Sebastian;Wang,ShirleyV
虽然随机对照试验（RCT）被认为是医学治疗有效性证据的标准，但使用健康保险索赔或电子健康记录数据的非随机真实世界证据（RWE）研究可以提供重要的补充证据。由于对非随机化研究中的混杂因素和使用次要数据的担忧，使用RWE为决策提供信息受到质疑。RCT-DUPLICATE是一个示范项目，模拟了32项RCTs的设计，采用非随机RWE研究。我们试图探讨仿真差异如何与RCT-RWE study pairs.MethodsWe包括所有RCT-RWE研究对RCT-DUPLICATE的影响的措施是一个风险比，并使用探索性荟萃回归方法来解释差异和变化的影响大小之间的结果从RCT和RWE研究的变化。所考虑的解释变量与设计和人群差异有关。结果在该样本中RCT-RWE研究对之间观察到的效果估计值的大部分变化可以通过元回归模型中的三个仿真差异来解释：（i）在医院开始治疗（在索赔数据中未观察到），（ii）随机化时停止某些基线治疗（不是临床实践的一部分），（iii）延迟起效的药物作用（错过了短期药物治疗的持久性在临床实践中）。ConclusionsThis分析表明，相当大的比例的随机对照试验和RWE研究的结果之间观察到的变化可以归因于设计仿真差异。(238字）
ObjectivesWhile randomized controlled trials (RCTs) are considered a standard for evidence on the efficacy of medical treatments, non-randomized real-world evidence (RWE) studies using data from health insurance claims or electronic health records can provide important complementary evidence. The use of RWE to inform decision-making has been questioned because of concerns regarding confounding in non-randomized studies and the use of secondary data. RCT-DUPLICATE was a demonstration project that emulated the design of 32 RCTs with non-randomized RWE studies. We sought to explore how emulation differences relate to variation in results between the RCT-RWE study pairs.MethodsWe include all RCT-RWE study pairs from RCT-DUPLICATE where the measure of effect was a hazard ratio and use exploratory meta-regression methods to explain differences and variation in the effect sizes between the results from the RCT and the RWE study. The considered explanatory variables are related to design and population differences.ResultsMost of the observed variation in effect estimates between RCT-RWE study pairs in this sample could be explained by three emulation differences in the meta-regression model: (i) in-hospital start of treatment (not observed in claims data), (ii) discontinuation of certain baseline therapies at randomization (not part of clinical practice), (iii) delayed onset of drug effects (missed by short medication persistence in clinical practice).ConclusionsThis analysis suggests that a substantial proportion of the observed variation between results from RCTs and RWE studies can be attributed to design emulation differences. (238 words)