WANG Mingqi 1,2,3 , JIA Yulong 1,2,3 , WANG Yuning 1,2,3 , LI Ling 1,2,3 , WANG Wen 1,2,3 , REN Yan 1,2,3 , YAO Minghong 1,2,3 , SUN Xin 1,2,3
  • 1. Chinese Evidence-based Medicine Center, West China Hospital, Sichuan University, Chengdu 610041, P. R. China;
  • 2. NMPA Key Laboratory for Real World Data Research and Evaluation in Hainan, Chengdu 610041, P. R. China;
  • 3. Sichuan Center of Technology Innovation for Real World Data, Chengdu 610041, P. R. China;
YAO Minghong, Email: ymhldjxa@sina.com; SUN Xin, Email: sunxin@wchscu.cn
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High-quality randomized controlled trials (RCTs) are regarded as the gold standard for assessing the efficiency and safety of drugs. However, conducting RCTs is expensive and time consumed, and providing timely evidence by RCTs for regulatory agencies and medical decision-makers can be challenging, particularly for new or emerging serious diseases. Additionally, the strict design of RCTs often results in a weakly external validity, making it difficult to provide the evidence of the clinical efficacy and safety of drugs in a broader population. In contrast, large simple clinical trials (LSTs) can expedite the research process and provide better extrapolation and reliable evidence at a lower cost. This article presents the development, features, and distinctions between LSTs and RCTs, as well as special considerations when conducting LSTs, in accordance with literature and guidance principles from regulatory agencies both from China and other countries. Furthermore, this paper assesses the potential of real-world data to bolster the development of LSTs, offering relevant researchers’ insight and guidance on how to conduct LSTs.

Citation: WANG Mingqi, JIA Yulong, WANG Yuning, LI Ling, WANG Wen, REN Yan, YAO Minghong, SUN Xin. Large scale simple clinical trial designs supported by real world data. Chinese Journal of Evidence-Based Medicine, 2024, 24(5): 605-611. doi: 10.7507/1672-2531.202311044 Copy

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