创新药物Ⅱ期临床研究设计的一般考虑

General considerations for designing phaseⅡclinical trials of new drugs

Ⅱ期临床研究是新药研究过程中的重要阶段,其主要的目的是探索药物剂量-效应关系,初步评估研究新药的药效作用和安全性,为是否可进入后期的确证性临床试验阶段以及为确证性临床试验的给药方案选择提供决策依据。本文介绍了国际上通用的Ⅱ期临床研究的研究类型和设计、研究目标、统计方法等内容,并给出案例加以说明。针对目前国内的创新药物Ⅱ期研究设计常常采用2个受试药物剂量组和1个阳性药组的设计,本文介绍的内容将有助于认识这种设计所存在的不足,对如何进行Ⅱ期临床试验的合理设计和研究推进等方面也有启发作用。

PhaseⅡclinical trial is a very important step during the clinical development of new drugs.The main purpose in phaseⅡis to explore the dose-response information,evaluate the efficacy and toxicity of new drugs as well as provide dosing regimens for the confirmatory clinical trials.In the present paper,the types and designing schemes of phaseⅡclinical trials,the objectives,and statistic methods are discussed and presented with examples.The contents presented in this paper help analyze the deficiencies existing in the dosing finding study when it is designed with paralleling study with two test dosage groups vs one positive control.Researchers can also benefit from the present paper for rational schemes for designing phaseⅡclinical trials as well as strategies for pushing the clinical trial forward.