中医药临床试验核心指标（COS）测评方法学研究

Giving full play to the advantages of traditional Chinese medicine(TCM), clinical effect is the key. Clinical efficacy needs to be expressed through measurement and analysis of outcome measures. The popularization and application of evidence-based medicine methods has significantly improved the quality control level of TCM clinical trials. However, at present, there are many problems in the measurement of outcomes in clinical trials, such as a variety of measurement instruments, low quality, diverse measurement points, self-designed standards, unreported methods, etc., leading to large heterogeneity of outcomes, data bias, impractical, etc., which cannot reflect the true efficacy of interventions. Based on the core outcome set(COS) solving "what to measure", the key to solving "how to measure" outcomes is to establish methodology to select instruments for COS-TCM, in order to provide the most scientific and reliable measurement instruments for each core outcome, and improve the accuracy and practicability of research results. In this study, the COS measurement method of chronic heart failure in TCM clinical trial was as a sample, producing methods on a instruments pool of COS, quality assessment methods and recommendation, and the measurement guidance of COS-TCM was formed. The academic norms guide the COS-TCM system to standardize and efficiently select reasonable measurement instruments of outcomes, so that the research data can be standardized, accurate and valuable. In addition, the quality of TCM clinical researches can be promoted.

中医药特色优势的发挥，关键在疗效。而临床疗效需要通过结局指标的测量和分析来表达。循证医学方法的推广应用，使中医临床试验过程质量控制水平显著提升。然而，目前临床试验结局指标测量普遍存在测量工具种类多、质量低，测量时点多样，标准自拟，方法不报告等问题，导致指标异质性大、数据偏倚、不实用等，无法反应干预措施的真实疗效。在开展核心指标（COS）研究解决“测什么”指标基础上，建立中医药COS测评方法学，为每个核心指标提供当前最科学、可靠的测量工具，提高研究数据的准确性和实用性，是解决“怎么测”指标的关键。本研究以慢性心衰中医临床试验COS测量方法为载体，研究COS测量工具库构建方法、测量工具质量评估方法、测量工具和时点一致性推荐方法等，最终形成中医药核心指标测量方法学规范，引导中医核心指标体系能规范、高效地选择合理的指标测评方法，使每个研究数据都能达到规范、准确、有价值，推动中医临床研究质量提升。

临床疗效是判断临床试验证据价值的金标准，需要通过结局指标测量和分析进行评估。但中医临床试验结局指标测量方法不规范，测量工具缺乏科学的质量评估，严重降低了研究价值。本项目以核心指标集研究为基础，引入半结构化访谈、德尔菲调查、共识会议和Grade方法，围绕核心结局指标测量内容、目标人群、适用场景、指标测量工具库、中医药临床疗效评价量表、方法学质量评价、测量学属性评价、可行性评估、证据分级和推荐强度等要点，建立了中医药临床试验核心指标测评方法学，并进行了实例验证和数据库平台搭建，促进了中医药临床研究高效地选用合理的结局指标测量方法/工具。从而，为结局指标从“测什么”到“怎么测”提供闭环式方法学支撑。该项目推动了我国中医临床试验结局指标应用的规范化，有力提高临床研究质量和价值，为循证医疗卫生决策提供高质量证据支撑。