Griffin Beth Ann, McCaffrey Daniel, Ramchand Rajeev, Hunter Sarah B, Suttorp Marika
RAND Corporation, 1200 South Hayes Street, Arlington, VA 22202, x 5188.
Health Serv Outcomes Res Methodol. 2012 Jun 1;12(2-3):84-103. doi: 10.1007/s10742-012-0089-7.
We develop a new tool for assessing the sensitivity of findings on treatment effectiveness to differential follow-up rates in the two treatment conditions being compared. The method censors the group with the higher response rate to create a synthetic respondent group that is then compared with the observed cases in the other condition to estimate a treatment effect. Censoring is done under various assumptions about the strength of the relationship between follow-up and outcomes to determine how informative differential dropout can alter inferences relative to estimates from models that assume the data are missing at random. The method provides an intuitive measure for understanding the strength of the association between outcomes and dropout that would be required to alter inferences about treatment effects. Our approach is motivated by translational research in which treatments found to be effective under experimental conditions are tested in standard treatment conditions. In such applications, follow-up rates in the experimental setting are likely to be substantially higher than in the standard setting, especially when observational data are used in the evaluation. We test the method on a case study evaluation of the effectiveness of an evidence-supported adolescent substance abuse treatment program (Motivational Enhancement Therapy/Cognitive Behavioral Therapy-5 [MET/CBT-5]) delivered by community-based treatment providers relative to its performance in a controlled research trial. In this case study, follow-up rates in the community based settings were extremely low (54%) compared to the experimental setting (95%) giving raise to concerns about non-ignorable drop-out.
我们开发了一种新工具,用于评估在比较的两种治疗条件下,治疗效果研究结果对不同随访率的敏感性。该方法对反应率较高的组进行删失,以创建一个综合应答组,然后将其与另一种条件下的观察病例进行比较,以估计治疗效果。删失是在关于随访与结局之间关系强度的各种假设下进行的,以确定相对于假设数据随机缺失的模型估计,不同的失访情况能在多大程度上改变推断。该方法提供了一种直观的度量,用于理解改变治疗效果推断所需的结局与失访之间关联的强度。我们的方法是受转化研究的启发,即在实验条件下被发现有效的治疗方法在标准治疗条件下进行测试。在这类应用中,实验环境中的随访率可能远高于标准环境中的随访率,尤其是在评估中使用观察性数据时。我们在一个案例研究评估中测试了该方法,该评估是关于由社区治疗提供者提供的一种有证据支持的青少年药物滥用治疗项目(动机增强疗法/认知行为疗法-5[MET/CBT-5])相对于其在对照研究试验中的表现的有效性。在这个案例研究中,与实验环境(95%)相比,社区环境中的随访率极低(54%),这引发了对不可忽略的失访问题的担忧。