儿童中药临床试验设计评价与实施难点问题调研

Research on the difficulties of design,evaluation and implementation of clinical trials of pediatric Chinese medicine

目的:本研究就当前儿童中药临床试验设计评价和试验实施等阶段存在的困难和问题进行广泛调研,为《儿童中药临床试验相关问题专家共识》的制定搭建问题清单。方法:通过网络问卷调研儿童中药临床试验参与各方,归纳试验设计与评价及试验实施的难点。结果:共回收问卷49份,受访者认为当前儿童中药临床试验在设计与评价阶段对适应证定位、试验目的、诊断标准、年龄范围、对照药、有效性评价、安全性观察等选择困难,用药方案制定缺乏科学依据;实施阶段要明确试验启动时间,在伦理审查、受试者招募、知情同意、依从性和临床试验数据监察委员会(DMC)设置等方面需进一步提高。结论:当前儿童中药临床试验在设计评价与实施等方面还存在诸多难点,可从政策、意识、技术等多方面加以改进,推动儿童专用中成药的研发。

Objective:To conduct a research on the current difficulties in the design,evaluation,and implementation of clinical trials of pediatric Chinese medicine and build a list of questions for the development of the Expert Consensus on Issues Related to Clinical Trials of Pediatric Chinese Medicine.Methods:The parties involved in clinical trials of pediatric Chinese medicine were surveyed by online questionnaires to learn the difficulties in the design,evaluation,and implementation of clinical trials.Results:A total of 49 questionnaires were collected,and the experts believed that it was dificult to determine the indications,test purpose,diagnostic criteria,age range,control drugs,efficacy and safety evaluation in the design and evaluation stage of clinical trials of traditional Chinese medicine for children,and there was a lack of scientific basis for developing medication regimen.In the implementation phase,the start time of the trial should be specified,and further improvement should be made in the aspects of ethical review,subject recruitment,informed consent,compliance,and DMC setting.Conclusion:There were many difficulties in the design,evaluation,and implementation of current clinical trials of pediatric Chinese medicines,which should be improved from various aspects such as polic,y,awareness,and technology,thus to promote the research and development of pediatric proprietary Chinese medicines.