抗肿瘤药物早期临床研究的新颖试验设计方法

Novel designs for early clinical trials of anticancer drugs

在抗肿瘤药物的早期研究中,新颖的临床试验设计方法得到广泛关注并在国内外实际药物研发中得以应用。本文分别在药物单剂量探索和多剂量探索中,介绍了基于算法、模型和模型辅助的3种设计方法,并分别介绍了这些方法的基本思想和统计方法,之后阐述其优劣性。新颖临床试验设计不仅可以减少研发成本和研究时间,也可以较早分辨药物是否有效,从而提高临床研究效率。

Novel designs for early clinical trials of anticancer drugs have received widespread attention and have been applied in drug development.This paper introduced three methods,i.e.,algorithm-based designs,model-based designs and model-assisted designs,in single-dose exploration and multiple-dose exploration respectively.Their basic ideas and statistical methods were introduced and then their strengths and weaknesses were explained.These novel designs can not only reduce R&D costs and research time but also determine whether the medicine is effective in clinic earlier,thereby increasing the development efficiency.