有效应对流行病的新型临床试验设计

Innovative clinical trial design for effective epidemic response

对于迅速演变的流行病来说,仅开展传统临床试验还是不能及时得到有意义的研究结果。在未来更好地应对流行病暴发,可能需要接受一种灵活的试验设计,这种设计可以绕过、加速或重新安排传统的3个临床研究阶段,同时又遵循候选药物安全性和有效性相关的科学规律。介绍了治疗新型冠状病毒肺炎(COVID-19)的8个适应性临床试验,及1个平台试验,同时还列举了2个疫苗研发的由真实世界证据(RWE)和随机对照试验(RCT)组成的混合设计方案。为了有效应对流行病,鼓励新型的临床试验设计,鼓励疫苗开发的新型研究策略。建议为未来可能发生的流行病制定药物研发计划,以进行适应性的、持续的、多地区的临床研究。

For rapidly evolving epidemics,only conducting traditional clinical trials can not get meaningful research results in time.In the future,to better respond to epidemic outbreaks,it may be necessary to accept a flexible trial design that can bypass,accelerate,or rearrange the traditional three clinical research stages,while at the same time complying with scientific laws related to the safety and effectiveness of drug candidates.This article introduces 8 adaptive clinical trials for the treatment of new coronavirus diseases,and 1 platform trial.Two hybrid designs of real world evidence(RWE)and randomized controlled trials(RCT)for vaccine development are presented.In order to effectively respond to epidemics,innovative clinical trial design and novel research strategies for vaccine development are encouraged.It is recommended to formulate drug development plans for future epidemic diseases in order to conduct adaptive,continuous,and multi-regional clinical research.