Toward a Generalized Framework and Flexible Software Environment for Power Analysis of Alcohol Treatment Randomized Controlled Trials

项目总结 酒精使用障碍的药物和行为治疗只有在他们的 效果是稳健和可靠的。统计能力的先验确定是最重要的步骤之一 最大限度地提高科学可靠性；然而，在历史上，它在健康科学中基本上被忽视了。 酒精研究人员越来越多地使用复杂的统计模型(例如，生长曲线、混合模型、 社交网络)；然而，除了最简单的统计检验外，所有确定样本量的方法都是可获得的 还没有被广泛传播。此外，即使对于软件程序容易实现的模型 在可用情况下，科学家一直对预期参数估计表示不确定，禁止 功率分析本身的效用。因此，许多随机对照试验(RCT)可能并不是最优的 旨在进行确凿的统计推断。这项提议的总体目标是缩小这一差距 通过使用已有的和创新的相结合的方法来开发通用的功率分析方法 可用于生成和测试治疗研究中使用的大量模型的方法。这 目标将通过追求两个具体目标来实现：开发灵活、用户友好的软件界面 统计能力和敏感度分析(目标1)；制定确定模式的一般框架 参数估计(目标2)。这项提议是创新的，因为软件界面将植根于 计算技术和人机交互的最新进展。此外， 框架将通过基于概化理论的元分析和指导演练的进步而得到加强 基于机器学习的个体模型不确定性影响的仿真研究 关于观察到假想效应的可能性的参数。该软件将包括自动感应器 分析以确定对功率影响最大的参数，并将建议启动参数。 开发和测试将以纳曲酮和认知行为疗法文献为基础。他们的 方法论方法的广泛使用和多样性使它们成为理想的试验台 推广到其他治疗领域。这项研究与NIAAA内的每一个目标都是协同的 《战略规划(2017-2021年)》。也许最重要的是，将为以下方面制定方法和指导方针 设计具有足够精度的RCT，以区分轨迹、机制、异质性和 治疗效果的特异性。这些努力将为研究人员提供所需的先验信息 执行研究以确保其结果的稳健性。这些随机对照试验设计工具的开发将 对酒精治疗研究的所有领域都有影响，包括瞬间危险行为，个人- 以服务不足和弱势群体为目标的(并存)特点，以及大规模的举措。 因此，这项提案将允许更明确地确定有效的干预措施和治疗方法。 酒精使用障碍。

PROJECT SUMMARY Pharmacological and behavioral treatments of alcohol use disorder are only useful to the extent that their effects are robust and reliable. A priori determination of statistical power is one of the most significant steps to maximizing scientific reliability; however, it has historically been largely overlooked in the health sciences. Alcohol researchers are increasingly utilizing complex statistical models (e.g., growth curves, mixture models, social networks); yet, accessible methods for sample size determination for all but the simplest statistical tests have not been broadly disseminated. Moreover, even for models in which software programs are readily available, scientists consistently express uncertainty regarding expected parameter estimates, prohibiting the utility of power analysis itself. As a result, many randomized controlled trials (RCTs) are likely not optimally designed to allow for conclusive statistical inference. The overall objective of this proposal is to close this gap by developing a generalized method for power analysis using a combination of established and innovative approaches that can be used to generate and test a vast array of models used in treatment research. This objective will be achieved by pursuing two specific aims: develop a flexible, user-friendly software interface for statistical power and sensitivity analysis (Aim 1); and develop a generalized framework for determining model parameter estimates (Aim 2). The proposal is innovative because the software interface will be grounded in state-of-the-art advancements in computational technology and human-computer interaction. In addition, the framework will be fortified by advances in generalizability-theory-based meta-analyses and guided walk and machine-learning-based simulation studies investigating the effects of uncertainty in individual model parameters on the likelihood of observing hypothesized effects. The software will include automated sensitivity analyses to identify parameters with the largest impact on power and will recommend starting parameters. Development and testing will be grounded in the naltrexone and cognitive behavioral therapy literatures. Their widespread use and diversity of methodological approaches make them ideal testbeds from which to generalize to other treatment domains. This research is synergistic with each of the goals within the NIAAA Strategic Plan (2017-2021). Perhaps most significantly, methodology and guidelines will be developed for designing RCTs that have sufficient precision to distinguish trajectories, mechanisms, heterogeneity, and specificity of treatment efficacy. These efforts will provide researchers with the required a priori information to execute studies that ensure the robustness of their results. The development of these tools for RCT design will have implications for all areas of alcohol treatment research, including in-the-moment risk behaviors, person- level (comorbid) characteristics, targeting underserved and vulnerable populations, and large-scale initiatives. As a result, this proposal will allow for more definitive identification of efficacious interventions and treatments of alcohol use disorder.