Alternative statistical designs cannot fully mitigate the limitations of traditional clinical trials in rare cancers. Creative study designs that integrate early clinical data and correlative outcomes from concomitant translational and laboratory models to evaluate the efficacy of druggable targets can potentially expedite access to novel therapies for these patients. See related article by Hunter et al., [p. 6095][1] [1]: /lookup/volpage/27/6095?iss=22

中文翻译：

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CMML 中的鲁索替尼：罕见癌症创新试验设计的案例研究

替代统计设计不能完全减轻传统临床试验在罕见癌症中的局限性。创造性的研究设计将早期临床数据和伴随的转化和实验室模型的相关结果整合起来，以评估可药物靶点的疗效，这可能会加快这些患者获得新疗法的速度。参见 Hunter 等人的相关文章，[p. 6095][1][1]：/lookup/volpage/27/6095?iss=22