Center for Care Delivery and Outcomes Research, Minneapolis VA HCS, Minneapolis, Minnesota, USA.
Department of Medicine, University of Minnesota, Minneapolis, Minnesota, USA.
Stat Med. 2023 Oct 30;42(24):4377-4391. doi: 10.1002/sim.9866. Epub 2023 Aug 13.
Missing outcomes are commonly encountered in randomized controlled trials (RCT) involving human subjects and present a risk for substantial bias in the results of a complete case analysis. While response rates for RCTs are typically high there is no agreed upon universal threshold under which the amount of missing data is deemed to not be a threat to inference. We focus here on binary outcomes that are possibly missing not at random, that is, the value of the outcome influences its possibility of being observed. Salient information that can assist in addressing these missing outcomes in such situations is the anticipated response rate in each study arm; these can often be anticipated based on prior research in similar populations using similar designs and outcomes. Further, in some areas of human subjects research, we are often confident or we suspect that response rates among RCT participants with successful treatment outcomes will be at least as great as those among participants without successful treatment outcomes. In other settings we may suspect the opposite relationship. This direction of the differential response between those with successful and unsuccessful outcomes can further aid in addressing the missing outcomes. We present simple Bayesian pattern-mixture models that incorporate this information on response rates to analyze the relationship between a binary outcome and an intervention while addressing the missing outcomes. We assess the performance of this method in simulation studies and apply this method to the results of an RCT of a smoking abstinence intervention.
在涉及人体受试者的随机对照试验 (RCT) 中,常见的情况是缺失结果,并且在完整病例分析的结果中存在严重偏差的风险。虽然 RCT 的响应率通常很高,但对于缺失数据量被认为不会对推断造成威胁的通用阈值,目前还没有达成一致意见。我们这里关注的是可能不是随机缺失的二分类结局,也就是说,结局的值会影响其被观察的可能性。在这种情况下,有助于解决这些缺失结局的重要信息是每个研究臂中预期的响应率;这些通常可以根据类似人群中使用类似设计和结局的先前研究来预测。此外,在人体受试者研究的某些领域,我们通常有信心或怀疑在有成功治疗结果的 RCT 参与者中,与没有成功治疗结果的参与者相比,反应率至少会更高。在其他情况下,我们可能会怀疑相反的关系。这种成功和失败结局之间的差异反应方向可以进一步帮助解决缺失结局的问题。我们提出了简单的贝叶斯混合模型,这些模型将响应率的信息纳入其中,以分析二分类结局与干预之间的关系,同时解决缺失结局的问题。我们在模拟研究中评估了该方法的性能,并将该方法应用于一项吸烟戒断干预的 RCT 结果。