Statistical issues in multisite effectiveness trials: the case of brief strategic family therapy for adolescent drug abuse treatment.
Statistical issues in multisite effectiveness trials: the case of brief strategic family therapy for adolescent drug abuse treatment.
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DOI：
10.1191/1740774504cn041oa
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发表时间：
2004-01-01
期刊：
影响因子：
--
通讯作者：
Szapocznik, Jose
中科院分区：
文献类型：
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作者：
Feaster, Daniel J;Robbins, Michael S;Szapocznik, Jose
美国国家药物滥用研究所临床试验网络的多地点简短战略家庭治疗（BSFT）试验的统计发展提供了一个有用的，真实的例子，说明有效性试验如何不同于疗效试验。特别是，两个设计要素将该有效性试验与功效试验区分开来。首先，由于试验的目的是证明BSFT的使用将是对当前实践的改进，因此决定在每个地点将BSFT与常规治疗进行比较。这一决定确保了试验对参与的社区治疗提供者具有最大的生态有效性。其次，希望将结果推广到一般临床实践，这就要求使用随机效应模型来估计社区治疗提供者之间的差异（实际上）。这两个决定共同影响样本量计算。与固定的研究中心效应估计值相比，允许治疗变化将增加研究中心间效应量的变异性，并且将这种变异性估计为随机效应需要更大的样本量（社区治疗提供者和每个社区治疗提供者的参与者数量）。详细介绍了这些影响及其对统计设计的影响。
The statistical development of the multisite Brief Strategic Family Therapy (BSFT) Trial of the National Institute on Drug Abuse's Clinical Trials Network provides a useful, real example of how an effectiveness trial can differ from an efficacy trial. In particular, two design elements distinguish this effectiveness trial from an efficacy trial. First, because the goal of the trial is to show that the use of BSFT would be an improvement on current practice, it was decided to compare BSFT to treatment as usual at each location. This decision ensures that the trial has the most ecological validity to the participating community treatment providers. Second, the desire to generalize the results to general clinical practice dictates that variability (in effect) across community treatment providers be estimated using a random effects model. These two decisions jointly influence the sample size calculations. Allowing variation in treatment as usual, will increase the variability in effect sizes across sites and estimation of this variability as a random effect necessitates a larger sample size (both number of community treatment providers and participants per community treatment provider), than is the case for a fixed site effect estimate. Details of these effects and their implications for the statistical design are presented.