心脑血管疾病具有高发病率、高致残率及证候复杂、病程迁延等特点，临床上治疗强调“辨证论治”和个体化综合干预。目标值法单臂试验因不需设立同期平行对照，在特定情境下可作为随机对照试验（RCT）的补充，成为评估中医药疗效的一种可行路径。为进一步规范其应用，阐述目标值法单臂试验的设计与实施基本流程及关键环节，并从适用性、可行性及当前面临的局限与挑战等方面，对该设计用于中医药治疗心脑血管病临床疗效评价进行了系统分析。目标值法单臂试验为中医药治疗心脑血管病临床疗效评价提供了契合中医临床实践诊疗特点的研究设计思路，能够进一步完善该领域的循证证据体系。未来在开展相关研究时，应严格把控结局指标选择、目标值制定、样本量计算、质控等关键环节，确保研究结果的可靠性，并积极探索人工智能（AI）等先进技术在目标值构建过程中的应用。

Cardiovascular and cerebrovascular diseases have the characteristics of high incidence, high disability rate, complex syndrome, and a prolonged course. Clinical treatment emphasizes “syndrome differentiation and treatment” and individualized comprehensive intervention. Because there is no need to set up a concurrent parallel control, objective performance criteria-based single-arm trials can be used as a supplement to randomized controlled trials(RCT) in specific situations, and are expected to become a feasible way to evaluate the efficacy of traditional Chinese medicine. To further standardize its application, we expound on the basic process and key links of the design and implementation of objective performance criteria-based single-arm trials, and analyze the application of the design in the clinical efficacy evaluation of traditional Chinese medicine in the treatment of cardiovascular and cerebrovascular diseases from the aspects of applicability, feasibility, current limitations and challenges. The objective performance criteria-based single-arm trials provides a research design idea for the clinical efficacy evaluation of traditional Chinese medicine in the treatment of cardiovascular and cerebrovascular diseases, which is in line with the characteristics of clinical practice of traditional Chinese medicine, and can further improve the evidence-based system in this field. In the future, when carrying out relevant research, we should strictly control the key steps such as the selection of outcome indicators, the formulation of target values, the calculation of sample size, and quality control to ensure the reliability of research results, and actively explore the application of advanced technologies such as artificial intelligence(AI) in the process of target value construction.

R969.4

国家自然科学基金资助项目(82575254); 2025年度山东省青年科技人才托举工程项目(SDAST2025QTB062); 国家中医药高水平中医药重点学科建设项目(zyyzdxk-2023120); 山东中医药大学临床科研专项(LCKY202415)