Abstract
BACKGROUND: Patient-focused drug development (PFDD) is an important direction in the field of medical research and is of great significance to the development of medicine. In recent years, PFDD and real-world study (RWS) have gained much interest, of which both have their advantages. This study aims to promote research methods innovation and optimize clinical research design and implementation. METHODS: After a brief introduction of PFDD and RWS, this review focused on the comparison of clinical trials of PFDD and RWS in terms of objectives, Population, Intervention, Comparator, Outcome (PICO) elements, research team members, data acquisition, and research key points, and clarified the feasibility and significance of "patient-focused RWS" research model. RESULTS: PFDD emphasized that patients' needs as well as the willingness and satisfaction of patients should be considered throughout the whole research process and the patient experience data should be collected during the study development and drug use. RWS emphasized the facticity of research implementation environment and the breadth of patient sources, which concerned the problem of the extrapolation of study results, the clinical localization, and patient applicability of the target drug. However, there is a connection between both of the above. Both clinical trials of PFDD and RWS bring benefits to patients. CONCLUSIONS: Combining PFDD idea and RWS research method to carry out new research will maximize the benefits for patients. The study model combining the PFDD concept with RWS can facilitate drug development and dissemination, which can be popularized and applied in various research areas. This study can innovate research methods and provide new ideas for future research.