In order to better incorporate patient input in clinical trials, the US Food and Drug Administration has included "patient-focused drug development" in the selection and development of clinical outcome assessments, and formulated a series of guidelines. Based on the third guiding principle, "Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments", this article summarizes the clinical outcome assessments from five aspects: concept, development process, scoring mechanism, interference factors and sensitivity, and introduces four different types of clinical outcome assessments, providing new ideas for "patient-focused drug development" efficacy evaluation in clinical trials.
Citation: ZHANG Chenyao, WU Xiaolei, SHI Menglong, MA Yucong, PANG Wentai, DU Liang, LIU Binglin, CAO Lujia, ZHANG Junhua. Selecting and developing clinical outcome assessments in patient-focused drug development. Chinese Journal of Evidence-Based Medicine, 2024, 24(4): 472-477. doi: 10.7507/1672-2531.202307067 Copy