中医药临床疗效评价中目标值法单臂试验适用性与理论对策的定性研究

Qualitative interview on applicability and theoretical countermeasures of objective performance criteria single arm trial in clinical efficacy evaluation of traditional Chinese medicine

目的了解目标值法单臂试验在中医临床疗效评价中的应用可行性,并针对应用的难点提出理论对策,为未来推广目标值法单臂试验在中医药临床疗效评价中的应用提供参考。方法使用半结构化定性访谈的方法,采用目的性抽样结合滚雪球抽样的方法,采用预设的访谈提纲对实际参与目标值法单臂试验相关临床研究的临床专家和方法学家开展深度访谈,探讨在中医药临床疗效评价中目标值法单臂试验的适用情境、影响应用的因素、应用过程中的难点并提出理论对策。结果共对12名受访者进行访谈,其中临床研究专家7人,方法学家5人。对访谈内容进行编码归纳整理,形成216条编码,类属分析后形成5大类,包括适用情境、优势、局限、应用过程中的难点以及理论对策。受访者认为目标值法单臂试验可用作无法开展对照试验时的替代设计方案,也可作为前期探索或证据补充;具有节约样本量、患者依从性好、节省资源投入、设计可以做到相对严谨等优势;但由于认知度不高、证据等级不明确而限制了其应用;应用难点包括目标值设置、样本量计算、质量控制及统计分析;建议形成规范,以推广其在中医药疗效评价中的应用。结论目标值法单臂试验的设计模式与中医药临床科研的特点有一定契合,不过由于其不设同期对照组,使其科学性受到挑战,阻碍了在中医药临床疗效评价领域内的进一步应用。通过规范目标值选取、样本量计算、统计分析方法及研究实施步骤,可以一定程度上提升其论证强度,提高业内对其认可程度,但应注意避免误用、滥用。未来或可以目标值法单臂试验作为基础,进一步探索中医药临床疗效评价科研方法的新方向。

Objective To understand the feasibility of application of objective performance criteria single arm trial in clinical efficacy evaluation of traditional Chinese medicine,and put forward theoretical countermeasures for the difficulties in application,so as to provide references for promoting the application of objective performance criteria single arm trial in clinical efficacy evaluation of traditional Chinese medicine in the future.Methods Semi-structured qualitative interviews were conducted among clinical experts and methodologists who were involved in objective performance criteria single arm trials using purposive sampling combined with snowball sampling and a preset interview outline.To explore the application situations,influencing factors and difficulties in the application process of objective performance criteria single arm trial in the clinical efficacy evaluation of traditional Chinese medicine,and put forward theoretical countermeasures.Results A total of 12 respondents were interviewed,including 7 clinical experts and 5 methodologists.The interview content was summarized and formed into 216 codes.After category analysis,5 categories were formed,including the applicable situations,advantages,limitations,difficulties in the application process and theoretical countermeasures.The respondents believed that objective performance criteria single arm trial could be used as an alternative plan when controlled trials could not be carried out,or as a preliminary exploration or evidence supplement.It had the advantages of reducing sample size,good patient compliance,saving resource input,and relatively rigorous design.However,its application was limited due to low awareness and unclear level of evidence.The application difficulties included the setting of objective performance criteria,sample size calculation,quality control and statistical analysis.It was suggested to form a standard to promote its application in the efficacy evaluation of traditional Chinese medicine.Conclusion The design of objective performance criteria single arm trial is consistent with the characteristics of clinical research of traditional Chinese medicine.However,because there is no control group,the validity of objective performance criteria single arm trial is challenged,which hinders its further application in the clinical efficacy evaluation of traditional Chinese medicine.By standardizing the selection of objective performance criteria,calculation of sample size,statistical analysis methods and research implementation steps,the strength of its demonstration and the recognition can be improved to a certain extent,but attention should be paid to avoid its misuse.In the future,the objective performance criteria single arm trial may be used as a basis to further explore the new direction of scientific research methods for clinical efficacy evaluation of traditional Chinese medicine.