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, 明揚, 夏如玉, 等. 國際目標值法臨床研究的文獻和方法學特征分析. 中國循證醫學雜志, 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.	呂德良, 李雪迎, 朱賽楠, 等. 目標值法在醫療器械非隨機對照臨床試驗中的應用. 中國衛生統計, 2009, 26(3): 258-260,263.
4.	韓梅, 曹卉娟, 張穎, 等. 單組試驗目標值法及其在中醫非藥物療法評價中的應用. 北京中醫藥, 2020, 39(5): 499-503.
5.	季聰華, 曹毅, 陳健. 單組試驗目標值法在中醫臨床研究中的應用. 中國中西醫結合雜志, 2012, 32(12): 1589-1591.
6.	沈琳博, 宋玉強, 趙冰驄, 等. 單組目標值法在針灸臨床試驗中應用的思考. 中國針灸, 2023, 43(10): 1175-1179.
7.	李倩. 基于第三方復證和目標值法建立中醫個體化診療循證模式的研究(以IBS為例). 廣州: 廣州中醫藥大學, 2020.
8.	張帥, 陳娟, 秦悅農, 等. 基于單組目標值法探討乳癌術后方加減對三陰性乳腺癌患者三年無病生存率的影響. 中醫雜志, 2019, 60(14): 1209-1212.
9.	史隨隨, 羅越毅, 高君, 等. 基于單組目標值法評估調血和脈、理氣消腫法治療頑固性視網膜靜脈阻塞繼發黃斑水腫的臨床療效. 中國中醫藥科技, 2024, 31(2): 246-250.
10.	陳向明. 質的研究方法與社會科學研究. 北京: 教育科學出版社, 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.	費宇彤, 劉建平, 于河, 等. 報告定性研究個體訪談和焦點組訪談統一標準的介紹. 中西醫結合學報, 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.	劉曉玉, 閆世艷, 劉保延. 針刺臨床研究中的假針刺與安慰針刺. 中國針灸, 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.	陳晨, 韓曉紅. 中國罕見病藥物臨床試驗10年現狀分析: 基于《第一批罕見病目錄》. 協和醫學雜志, 2022, 13(6): 1028-1035.
17.	張虹, 高晨燕, 陳曉媛, 等. 關于采用單臂臨床試驗數據用于支持進口藥品注冊的考慮. 中國新藥雜志, 2013, 22(18): 2126-2129.
18.	周永蓮, 黃明安. 患者就醫時對中西醫選擇偏好的影響因素研究. 中醫藥導報, 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.	張晨, 于明坤, 唐金平, 等. 目標值法在中藥上市后再評價中的應用. 中國中藥雜志, 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.	劉建平. 隨機對照試驗的依從性和意向性治療分析. 中國中西醫結合雜志, 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.	費宇彤, 柴倩云, 夏如玉, 等. 真實世界臨床研究證據分級的思考與初步探索. 中國循證醫學雜志, 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.	李衛, 趙耐青. 單組目標值臨床試驗的統計學考慮. 中國衛生統計, 2017, 34(3): 505-508.
29.	代欣玥, 訾明杰, 楊巧寧, 等. 基于文獻的中醫藥臨床研究結局指標現狀分析及思考. 中醫雜志, 2022, 63(12): 1122-1127.
30.	羅慜婧, 柴倩云, 馮玉婷, 等. 中醫藥臨床研究證候標準化診斷的研究思路與方法. 中醫雜志, 2023, 64(24): 2505-2510.

1. 于明坤, 明揚, 夏如玉, 等. 國際目標值法臨床研究的文獻和方法學特征分析. 中國循證醫學雜志, 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. 呂德良, 李雪迎, 朱賽楠, 等. 目標值法在醫療器械非隨機對照臨床試驗中的應用. 中國衛生統計, 2009, 26(3): 258-260,263.
4. 韓梅, 曹卉娟, 張穎, 等. 單組試驗目標值法及其在中醫非藥物療法評價中的應用. 北京中醫藥, 2020, 39(5): 499-503.
5. 季聰華, 曹毅, 陳健. 單組試驗目標值法在中醫臨床研究中的應用. 中國中西醫結合雜志, 2012, 32(12): 1589-1591.
6. 沈琳博, 宋玉強, 趙冰驄, 等. 單組目標值法在針灸臨床試驗中應用的思考. 中國針灸, 2023, 43(10): 1175-1179.
7. 李倩. 基于第三方復證和目標值法建立中醫個體化診療循證模式的研究(以IBS為例). 廣州: 廣州中醫藥大學, 2020.
8. 張帥, 陳娟, 秦悅農, 等. 基于單組目標值法探討乳癌術后方加減對三陰性乳腺癌患者三年無病生存率的影響. 中醫雜志, 2019, 60(14): 1209-1212.
9. 史隨隨, 羅越毅, 高君, 等. 基于單組目標值法評估調血和脈、理氣消腫法治療頑固性視網膜靜脈阻塞繼發黃斑水腫的臨床療效. 中國中醫藥科技, 2024, 31(2): 246-250.
10. 陳向明. 質的研究方法與社會科學研究. 北京: 教育科學出版社, 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. 費宇彤, 劉建平, 于河, 等. 報告定性研究個體訪談和焦點組訪談統一標準的介紹. 中西醫結合學報, 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. 劉曉玉, 閆世艷, 劉保延. 針刺臨床研究中的假針刺與安慰針刺. 中國針灸, 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. 陳晨, 韓曉紅. 中國罕見病藥物臨床試驗10年現狀分析: 基于《第一批罕見病目錄》. 協和醫學雜志, 2022, 13(6): 1028-1035.
17. 張虹, 高晨燕, 陳曉媛, 等. 關于采用單臂臨床試驗數據用于支持進口藥品注冊的考慮. 中國新藥雜志, 2013, 22(18): 2126-2129.
18. 周永蓮, 黃明安. 患者就醫時對中西醫選擇偏好的影響因素研究. 中醫藥導報, 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. 張晨, 于明坤, 唐金平, 等. 目標值法在中藥上市后再評價中的應用. 中國中藥雜志, 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. 劉建平. 隨機對照試驗的依從性和意向性治療分析. 中國中西醫結合雜志, 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. 費宇彤, 柴倩云, 夏如玉, 等. 真實世界臨床研究證據分級的思考與初步探索. 中國循證醫學雜志, 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. 李衛, 趙耐青. 單組目標值臨床試驗的統計學考慮. 中國衛生統計, 2017, 34(3): 505-508.
29. 代欣玥, 訾明杰, 楊巧寧, 等. 基于文獻的中醫藥臨床研究結局指標現狀分析及思考. 中醫雜志, 2022, 63(12): 1122-1127.
30. 羅慜婧, 柴倩云, 馮玉婷, 等. 中醫藥臨床研究證候標準化診斷的研究思路與方法. 中醫雜志, 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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