Qualitative interview on applicability and theoretical countermeasures of objective performance criteria single arm trial in clinical efficacy evaluation of traditional Chinese medicine_Chinese Journal of Evidence-Based Medicine

Authors：

WANG Handong ¹ , CHAI Qianyun ¹ , FENG Yuting ¹ , LUO Minjing ¹ , CAO Rui ¹ , TAO Liyuan ² ,  FEI Yutong ¹

1. Center for Evidence-Based Medicine, Beijing University of Traditional Chinese Medicine, Beijing 100029, P. R. China;
2. Research Center of Clinical Epidemiology, Peking University Third Hospital, Beijing 100191, P. R. China;

Corresponding author：

FEI Yutong, Email: feiyt@bucm.edu.cn

Keywords：

Single arm trial; Qualitative interview; Methodology; Objective performance criteria; Traditional Chinese medicine

DOI：

10.7507/1672-2531.202404038

Video：

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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.

Citation： WANG Handong, CHAI Qianyun, FENG Yuting, LUO Minjing, CAO Rui, TAO Liyuan, FEI Yutong. Qualitative interview on applicability and theoretical countermeasures of objective performance criteria single arm trial in clinical efficacy evaluation of traditional Chinese medicine. Chinese Journal of Evidence-Based Medicine, 2024, 24(11): 1317-1324. doi: 10.7507/1672-2531.202404038 Copy

1.	于明坤, 明扬, 夏如玉, 等. 国际目标值法临床研究的文献和方法学特征分析. 中国循证医学杂志, 2019, 19(11): 1308-1316.
2.	U. S. Department of Health And Human Services Food And Drug Administration. Design considerations for pivotal clinical investigations for medical devices-guidance for industry, clinical investigators, institutional review boards and food and drug administration staff. 2018.
3.	吕德良, 李雪迎, 朱赛楠, 等. 目标值法在医疗器械非随机对照临床试验中的应用. 中国卫生统计, 2009, 26(3): 258-260,263.
4.	韩梅, 曹卉娟, 张颖, 等. 单组试验目标值法及其在中医非药物疗法评价中的应用. 北京中医药, 2020, 39(5): 499-503.
5.	季聪华, 曹毅, 陈健. 单组试验目标值法在中医临床研究中的应用. 中国中西医结合杂志, 2012, 32(12): 1589-1591.
6.	沈琳博, 宋玉强, 赵冰骢, 等. 单组目标值法在针灸临床试验中应用的思考. 中国针灸, 2023, 43(10): 1175-1179.
7.	李倩. 基于第三方复证和目标值法建立中医个体化诊疗循证模式的研究(以IBS为例). 广州: 广州中医药大学, 2020.
8.	张帅, 陈娟, 秦悦农, 等. 基于单组目标值法探讨乳癌术后方加减对三阴性乳腺癌患者三年无病生存率的影响. 中医杂志, 2019, 60(14): 1209-1212.
9.	史随随, 罗越毅, 高君, 等. 基于单组目标值法评估调血和脉、理气消肿法治疗顽固性视网膜静脉阻塞继发黄斑水肿的临床疗效. 中国中医药科技, 2024, 31(2): 246-250.
10.	陈向明. 质的研究方法与社会科学研究. 北京: 教育科学出版社, 2000.
11.	Tong A, Sainsbury P, Craig J. Consolidated criteria for reporting qualitative research (COREQ): a 32-item checklist for interviews and focus groups. Int J Qual Health Care, 2007, 19(6): 349-357.
12.	费宇彤, 刘建平, 于河, 等. 报告定性研究个体访谈和焦点组访谈统一标准的介绍. 中西医结合学报, 2008, (2): 115-118.
13.	Al P, Hey S, Weijer C, et al. Changing patient preferences toward better trial recruitment: an ethical analysis. Trials, 2023, 24(1): 233.
14.	刘晓玉, 闫世艳, 刘保延. 针刺临床研究中的假针刺与安慰针刺. 中国针灸, 2023, 43(7): 821-824.
15.	Fei YT, Cao HJ, Xia RY, et al. Methodological challenges in design and conduct of randomised controlled trials in acupuncture. BMJ, 2022, 376: e064345.
16.	陈晨, 韩晓红. 中国罕见病药物临床试验10年现状分析: 基于《第一批罕见病目录》. 协和医学杂志, 2022, 13(6): 1028-1035.
17.	张虹, 高晨燕, 陈晓媛, 等. 关于采用单臂临床试验数据用于支持进口药品注册的考虑. 中国新药杂志, 2013, 22(18): 2126-2129.
18.	周永莲, 黄明安. 患者就医时对中西医选择偏好的影响因素研究. 中医药导报, 2016, 22(15): 38-40,43.
19.	Gray CM, Grimson F, Layton D, et al. A framework for methodological choice and evidence assessment for studies using external comparators from real-world data. Drug Saf, 2020, 43(7): 623-633.
20.	FDA Center For Devices And Radiological Health. The least burdensome provisions: concept and principles: guidance for industry and FDA staff. 2019.
21.	张晨, 于明坤, 唐金平, 等. 目标值法在中药上市后再评价中的应用. 中国中药杂志, 2021, 46(8): 1999-2003.
22.	Selmon MR, Schwindt AG, Cawich IM, et al. Final results of the Chronic Total Occlusion Crossing With the Ocelot System Ⅱ (CONNECT Ⅱ) study. J Endovasc Ther, 2013, 20(6): 770-781.
23.	刘建平. 随机对照试验的依从性和意向性治疗分析. 中国中西医结合杂志, 2003, (12): 884-886.
24.	Chai QY, Fei YT, Gao R, et al. Quality evaluation and reporting specification for real-world studies of traditional Chinese medicine. Chin J Integr Med, 2022, 28(12): 1059-1062.
25.	费宇彤, 柴倩云, 夏如玉, 等. 真实世界临床研究证据分级的思考与初步探索. 中国循证医学杂志, 2022, 22(10): 1206-1211.
26.	Gressler LE, Marinac-Dabic D, dosReis S, et al. Creation of objective performance criteria among medical devices. BMJ Surg Interv Health Technol, 2022, 4(1): e000106.
27.	Nieuwenhuijse MJ, Randsborg PH, Hyde JH, et al. Evidence-based objective performance criteria for the evaluation of hip and knee replacement devices and technologies. Int J Surg, 2023, 109(5): 1125-1135.
28.	李卫, 赵耐青. 单组目标值临床试验的统计学考虑. 中国卫生统计, 2017, 34(3): 505-508.
29.	代欣玥, 訾明杰, 杨巧宁, 等. 基于文献的中医药临床研究结局指标现状分析及思考. 中医杂志, 2022, 63(12): 1122-1127.
30.	罗慜婧, 柴倩云, 冯玉婷, 等. 中医药临床研究证候标准化诊断的研究思路与方法. 中医杂志, 2023, 64(24): 2505-2510.

1. 于明坤, 明扬, 夏如玉, 等. 国际目标值法临床研究的文献和方法学特征分析. 中国循证医学杂志, 2019, 19(11): 1308-1316.
2. U. S. Department of Health And Human Services Food And Drug Administration. Design considerations for pivotal clinical investigations for medical devices-guidance for industry, clinical investigators, institutional review boards and food and drug administration staff. 2018.
3. 吕德良, 李雪迎, 朱赛楠, 等. 目标值法在医疗器械非随机对照临床试验中的应用. 中国卫生统计, 2009, 26(3): 258-260,263.
4. 韩梅, 曹卉娟, 张颖, 等. 单组试验目标值法及其在中医非药物疗法评价中的应用. 北京中医药, 2020, 39(5): 499-503.
5. 季聪华, 曹毅, 陈健. 单组试验目标值法在中医临床研究中的应用. 中国中西医结合杂志, 2012, 32(12): 1589-1591.
6. 沈琳博, 宋玉强, 赵冰骢, 等. 单组目标值法在针灸临床试验中应用的思考. 中国针灸, 2023, 43(10): 1175-1179.
7. 李倩. 基于第三方复证和目标值法建立中医个体化诊疗循证模式的研究(以IBS为例). 广州: 广州中医药大学, 2020.
8. 张帅, 陈娟, 秦悦农, 等. 基于单组目标值法探讨乳癌术后方加减对三阴性乳腺癌患者三年无病生存率的影响. 中医杂志, 2019, 60(14): 1209-1212.
9. 史随随, 罗越毅, 高君, 等. 基于单组目标值法评估调血和脉、理气消肿法治疗顽固性视网膜静脉阻塞继发黄斑水肿的临床疗效. 中国中医药科技, 2024, 31(2): 246-250.
10. 陈向明. 质的研究方法与社会科学研究. 北京: 教育科学出版社, 2000.
11. Tong A, Sainsbury P, Craig J. Consolidated criteria for reporting qualitative research (COREQ): a 32-item checklist for interviews and focus groups. Int J Qual Health Care, 2007, 19(6): 349-357.
12. 费宇彤, 刘建平, 于河, 等. 报告定性研究个体访谈和焦点组访谈统一标准的介绍. 中西医结合学报, 2008, (2): 115-118.
13. Al P, Hey S, Weijer C, et al. Changing patient preferences toward better trial recruitment: an ethical analysis. Trials, 2023, 24(1): 233.
14. 刘晓玉, 闫世艳, 刘保延. 针刺临床研究中的假针刺与安慰针刺. 中国针灸, 2023, 43(7): 821-824.
15. Fei YT, Cao HJ, Xia RY, et al. Methodological challenges in design and conduct of randomised controlled trials in acupuncture. BMJ, 2022, 376: e064345.
16. 陈晨, 韩晓红. 中国罕见病药物临床试验10年现状分析: 基于《第一批罕见病目录》. 协和医学杂志, 2022, 13(6): 1028-1035.
17. 张虹, 高晨燕, 陈晓媛, 等. 关于采用单臂临床试验数据用于支持进口药品注册的考虑. 中国新药杂志, 2013, 22(18): 2126-2129.
18. 周永莲, 黄明安. 患者就医时对中西医选择偏好的影响因素研究. 中医药导报, 2016, 22(15): 38-40,43.
19. Gray CM, Grimson F, Layton D, et al. A framework for methodological choice and evidence assessment for studies using external comparators from real-world data. Drug Saf, 2020, 43(7): 623-633.
20. FDA Center For Devices And Radiological Health. The least burdensome provisions: concept and principles: guidance for industry and FDA staff. 2019.
21. 张晨, 于明坤, 唐金平, 等. 目标值法在中药上市后再评价中的应用. 中国中药杂志, 2021, 46(8): 1999-2003.
22. Selmon MR, Schwindt AG, Cawich IM, et al. Final results of the Chronic Total Occlusion Crossing With the Ocelot System Ⅱ (CONNECT Ⅱ) study. J Endovasc Ther, 2013, 20(6): 770-781.
23. 刘建平. 随机对照试验的依从性和意向性治疗分析. 中国中西医结合杂志, 2003, (12): 884-886.
24. Chai QY, Fei YT, Gao R, et al. Quality evaluation and reporting specification for real-world studies of traditional Chinese medicine. Chin J Integr Med, 2022, 28(12): 1059-1062.
25. 费宇彤, 柴倩云, 夏如玉, 等. 真实世界临床研究证据分级的思考与初步探索. 中国循证医学杂志, 2022, 22(10): 1206-1211.
26. Gressler LE, Marinac-Dabic D, dosReis S, et al. Creation of objective performance criteria among medical devices. BMJ Surg Interv Health Technol, 2022, 4(1): e000106.
27. Nieuwenhuijse MJ, Randsborg PH, Hyde JH, et al. Evidence-based objective performance criteria for the evaluation of hip and knee replacement devices and technologies. Int J Surg, 2023, 109(5): 1125-1135.
28. 李卫, 赵耐青. 单组目标值临床试验的统计学考虑. 中国卫生统计, 2017, 34(3): 505-508.
29. 代欣玥, 訾明杰, 杨巧宁, 等. 基于文献的中医药临床研究结局指标现状分析及思考. 中医杂志, 2022, 63(12): 1122-1127.
30. 罗慜婧, 柴倩云, 冯玉婷, 等. 中医药临床研究证候标准化诊断的研究思路与方法. 中医杂志, 2023, 64(24): 2505-2510.

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

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