中药新药"三结合"审评证据体系下探索性临床试验策略思考OA

Due to the influence of the inertial thinking in previous clinical research of traditional Chinese medicine(TCM)new drugs and the insufficient in depth understanding of the R&D requirements under the"three combinations"system,in the R&D process of TCM new drugs,there are still phenomena such as carrying out exploratory clinical trials in accordance with the previous traditional research ideas,or attempting to apply for directly conducting confirmatory clinical trials without objectively and scientifically evaluating the"evidence of human use experience".As a result,the policy advantages are not fully utilized,and the characteristics of TCM new drug R&D are not reflected.This has an adverse impact on the formulation of a reasonable R&D plan and also brings inconvenience to the review work of regulatory authorities.Based on the understanding of regulations and practical experience,this article discussed the importance of exploratory clinical trials under the"three combinations"review system.It pointed out that exploratory clinical trials needed to be evaluated based on human use experience data in aspects such as whether the clinical positioning and applicable population are clear,whether the dosing regimen and cycle are reasonable,and whether the clinical advantages and efficacy evaluation indicators are scientific.Necessary exploratory clinical trials should be designed and carried out around research questions lacking reliable evidence of human use experience support.Appropriate research types and reasonable sample sizes should be selected according to the general requirements of methodology to achieve the purpose of exploration.It is hoped that this article could provide inspiration for the formulation of R&D strategies for TCM new drugs,especially for the design and implementation of exploratory clinical trials.