In January 2020, the State Food and Drug Administration issued the guiding principles of real world evidence supporting drug research & development (R&D) and evaluation (Trial) to guide the development of real world study (RWS) for drug R&D and review decision-making in the future. This guideline has important reference value for developing RWS of health food. Based on the interpretation of the core content of the guiding principles, combined with the common points of RWS and health food interventions, this paper preliminarily discusses the application of RWS to support health food regulatory decision-making so as to provide references for further promoting the application of RWS in the field of health food.
Assessment of Real World Observational Studies (ArRoWS) is a tool developed by the Leicester Real World Evidence (LRWE) Unit of the Diabetes Research Centre of the University of Leicester in the United Kingdom to assess the quality of real world evidence research, and has been reported to have good practicability. ArRoWS can be used to quickly and specifically assess the quality of real world evidence research that uses electronic health record information. The tool contains 16 items, nine of which are common items, and seven of which are related to specific research designs. The current study introduces the development background, development process, assessment items, assessment criteria, and application methods of ArRoWS and other related aspects, to provide references for real world researchers in China.
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.