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Efficient tests for one sample correlated binary data with applications

机译：一个样本的高效测试将二进制数据与应用程序相关联

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Four testing procedures are considered for testing the response rate of one sample correlated binary data with a cluster size of one or two, which often occurs in otolaryngologic and ophthalmologic studies. Although an asymptotic approach is often used for statistical inference, it is criticized for unsatisfactory type I error control in small sample settings. An alternative to the asymptotic approach is an unconditional approach. The first unconditional approach is the one based on estimation, also known as parametric bootstrap (Lee and Young in Stat Probab Lett 71 (2): 143-153,2005). The other two unconditional approaches considered in this article are an approach based on maximization (Basu in J Am Stat Assoc 72(358):355-366, 1977), and an approach based on estimation and maximization (Lloyd in Biometrics 64(3):716-723, 2008a). These two unconditional approaches guarantee the test size and are generally more reliable than the asymptotic approach. We compare these four approaches in conjunction with a test proposed by Lee and Dubin (Stat Med 13(12): 1241-1252, 1994) and a likelihood ratio test derived in this article, in regards to type I error rate and power for sample sizes from small to medium. An example from an otolaryngologic study is provided to illustrate the various testing procedures. The unconditional approach based on estimation and maximization using the test in Lee and Dubin (Stat Med 13(12): 1241-1252, 1994) is preferable due to the power advantageous.
机译：考虑使用四种测试程序来测试一个样本相关的二进制数据的响应率，其簇大小为一或两个，这在耳鼻喉科和眼科研究中经常发生。尽管通常使用渐近方法进行统计推断，但由于在小样本设置中I型错误控制不令人满意而受到批评。渐近方法的替代方法是无条件方法。第一种无条件方法是基于估计的方法，也称为参数引导程序（Lee和Young，Stat Probab Lett 71（2）：143-153，2005）。本文考虑的其他两种无条件方法是基于最大化的方法（Basu in J Am Stat Assoc 72（358）：355-366，1977），以及基于估计和最大化的方法（Lloyd in Biometrics 64（3）） ：716-723，2008a）。这两种无条件方法可以保证测试量，并且通常比渐近方法更可靠。我们将这四种方法与Lee和Dubin提出的测试（Stat Med 13（12）：1241-1252，1994）以及本文得出的似然比测试进行了比较，涉及I类错误率和样本功效大小从小到中。提供了一个来自耳鼻喉科研究的例子来说明各种测试程序。基于Lee and Dubin（Stat Med 13（12）：1241-1252，1994）中的测试，基于估计和最大化的无条件方法是首选，因为它具有强大的功能。

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