Reed Shelby D, Gouda Pishoy
Duke Clinical Research Institute, Duke University, Durham, NC, USA.
University of Alberta, Edmonton, Alberta, Canada.
J Clin Transl Sci. 2025 Jul 3;9(1):e151. doi: 10.1017/cts.2025.10054. eCollection 2025.
Significant improvements have been achieved to enhance the patient-centricity of clinical research, including the development and utilization of novel clinical trial endpoints. These include endpoints that harness outcomes that are important to patients and reflect the patients' lived experiences. This may take the form of utilizing variables such as days alive and out of hospital (DAOH) and quality-of-life adjusted outcomes. The use of composite outcomes can be used to enrich patient-centricity by weighting or ranking events. These approaches have several nuances that should be considered including selecting appropriate events, defining outcomes, how to elicit or construct weights, and whose opinions to consider. After weights have been determined, a variety of approaches exist to combine weights with outcomes and make comparisons between groups. The approaches, including the win ratio, weighted win ratio, desirability of outcome ranking (DOOR), multicriteria decision analysis (MCDA), and variations of time-to-first composite event analyses, have unique advantages and challenges depending on the clinical scenario. While improving patient-centric outcomes is of high importance to multiple stakeholders, more comparative work is needed to characterize the implications of alternative approaches.
在提高临床研究的以患者为中心程度方面已取得显著进展，包括新型临床试验终点的开发和应用。这些终点包括利用对患者重要且反映患者生活经历的结果。这可能采用诸如存活且出院天数（DAOH）和生活质量调整结果等变量的形式。复合结果的使用可通过对事件进行加权或排序来丰富以患者为中心的程度。这些方法有几个细微差别需要考虑，包括选择合适的事件、定义结果、如何引出或构建权重以及考虑谁的意见。确定权重后，有多种方法可将权重与结果相结合并在组间进行比较。这些方法，包括胜率、加权胜率、结果排名可取性（DOOR）、多标准决策分析（MCDA）以及首次复合事件时间分析的变体，根据临床情况具有独特的优势和挑战。虽然改善以患者为中心的结果对多个利益相关者至关重要，但需要更多的比较工作来描述替代方法的影响。
