Department of Statistics, University of Glasgow, Glasgow, UK.
Stat Methods Med Res. 2011 Dec;20(6):657-66. doi: 10.1177/0962280210379174. Epub 2010 Aug 25.
It is a common belief that individual variation in response to treatment is an important explanation for the variation in observed outcomes in clinical trials. If such variation is large, it seems reasonable to suppose that progress in treating disease will be advanced by classifying patients according to their abilities or not to 'respond' to particular treatments. We consider that there is currently a lost opportunity in drug development. There is a great deal of talk about individual response to treatment and tailor-made drugs. However, relatively little work is being done to formally investigate, using suitable designs, where individual response to treatment may be important. Through a case study from a replicate cross-over study we show how, given suitable replication, it is possible to isolate the component of variation corresponding to patient-by-treatment interaction and hence investigate the possibility of individual response to treatment.
人们普遍认为，治疗反应的个体差异是临床试验中观察到的结果变异的一个重要解释。如果这种变异很大，那么根据患者对特定治疗的“反应”能力对其进行分类，似乎可以合理地认为治疗疾病的进展将会得到推进。我们认为，在药物开发方面，目前存在一个错失的机会。人们经常谈论个体对治疗的反应和量身定制的药物。然而，相对而言，很少有工作使用合适的设计，从正式的角度来研究治疗反应的个体差异可能很重要。通过来自重复交叉研究的一个案例研究，我们展示了在适当的重复情况下，如何分离出与患者-治疗相互作用相对应的变异成分，从而研究治疗反应的个体差异的可能性。
