零期临床助力中国原创药研发新范式迭代

Phase 0 clinical trials facilitate new paradigm iteration of original drug research and development in China

新药研发是一项耗时长且风险高的系统工程,需要投入大量资源。由于药物的安全性很难预料,药物开发失败花费的75%左右都在早期临床阶段。零期临床研究主要通过“微剂量”研究快速获得人体药代动力学及药效学等重要信息,为后期的药物临床试验节约资源,提高成功率。但在我国零期临床研究尚处于起步阶段,没有相应的法规和指导原则,缺乏合理的研究设计和专业的研究人员。本文就创新药物零期临床研究的内涵、目前存在的问题及解决策略与如何在中国有效开展零期临床等作一概述,以期为我国的原创药研发提供新范式参考。

Drug development is a lengthy and high-risk process that demands significant resource investment.With drug safety being unpredictable,approximately 75%of the expenses linked to drug development failures arise during the early stages of clinical trials.Phase 0 clinical trials involve administering"microdoses"to swiftly gather essential data on human pharmacokinetics and pharmacodynamics,thereby conserving resources and enhancing the success rates of subsequent trials.However,in China,phase 0 clinical research is still in its nascent stages,lacking corresponding regulations,guidelines,well-designed studies,and adequately trained researchers.This paper presents a comprehensive overview of the importance,current obstacles,suggested solutions,and effective strategies concerning phase 0 clinical research in innovative drug development,aiming to introduce a new approach to original drug research and development in the country.