Formation of the list of methodological quality evaluation and explanation for the selection of objective performance criteria in single arm trial_Chinese Journal of Evidence-Based Medicine

Authors：

CAO Rui ¹ , CHAI Qianyun ^1,2 , WANG Handong ¹ , LUO Minjing ^1,3,4 , LI He ⁵ , LI Shuying ⁵ , LIU Zhihan ¹ ,  TAO Liyuan ⁶ ,  LIU Jianping ¹ ,  FEI Yutong ¹

1. Center for Evidence-based Medicine, Beijing University of Traditional Chinese Medicine, Beijing 100029, P. R. China;
2. Journal of Traditional Chinese Medicine, Beijing 100700, P. R. China;
3. Guangdong Provincial Hospital of Traditional Chinese Medicine, Guangzhou 511400, P. R. China;
4. Guangdong Academy of Traditional Chinese Medicine, Guangzhou 511400, P. R. China;
5. The First Clinical College, Beijing University of Traditional Chinese Medicine, Beijing 100027, P. R. China;
6. Clinical Epidemiology Research Center of Peking University Third Hospital, Beijing 100191, P. R. China;

Corresponding?author：

TAO Liyuan, Email: tendytly@163.com; LIU Jianping, Email: liujp@bucm.edu.cn; FEI Yutong, Email: feiyt@bucm.edu.cn

Keywords：

Single-arm trial; Objective performance criteria; Nominal group method; Expert consensus; Methodology

DOI：

10.7507/1672-2531.202410031

Video：

Export PDF Favorites Scan Get Citation

Abstract Full text Figures/Tables Video References Cited by

Objective To form the list of methodological quality evaluation and explanation for the selection of objective performance criteria in single arm trial, and initially construct a methodological guiding tool for the selection and evaluation of target value, so as far to provide a research basis for improving the system of single-arm trial quality evaluation by objective performance criteria method. Methods Combining the bias risk assessment method of observational study, interventional trails, domestic and foreign policy documents, and systematically collecting the common bias and evaluation key points, preliminarily proposing the list of methodological quality evaluation and explanation for the selection of objective performance criteria in single-arm trial, via two rounds of expert consensus voting using nominal group method, finally formulating the list based on the voting results and expert opinions. Results Through two rounds of discussion, sorting, comprehensive expert opinions, we improved the corresponding items in the list and finally formed the list of methodological quality evaluation and explanation for the selection of objective performance criteria in single-arm trial, which included four areas: authority, traceability, comparability, and reliability, including 10 items. The entries in each field considered both design and implementation throughout the clinical trial. Conclusion The methodology list developed in this study provides methodological guidance for the selection of objective performance criteria and quality evaluation, and provides a solid theoretical basis for the establishment of a complete methodological system of quality evaluation for objective performance criteria single arm trails.

Citation： CAO Rui, CHAI Qianyun, WANG Handong, LUO Minjing, LI He, LI Shuying, LIU Zhihan, TAO Liyuan, LIU Jianping, FEI Yutong. Formation of the list of methodological quality evaluation and explanation for the selection of objective performance criteria in single arm trial. Chinese Journal of Evidence-Based Medicine, 2025, 25(4): 447-453. doi: 10.7507/1672-2531.202410031 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.	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.
3.	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.
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.	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.
6.	FDA Center for Devices and Radiological Health. The least burdensome provisions: concept and principles: guidance for industry and FDA staff. 2019.
7.	國家藥監局. 關于發布真實世界數據用于醫療器械臨床評價技術指導原則(試行)的通告(2020年第77號). 2020.
8.	中國國家藥監局藥品審評中心. 藥物真實世界研究設計與方案框架指導原則(試行). 2023.
9.	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.
10.	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.
11.	EQUATOR Network. Enhancing the quality and transparency of health research.
12.	Delbecq AL, van de Ven AH, Gustafson DH. Group techniques for program planning, a guide to nominal group and Delphi processes. Glenview, IL: Scott, Foresman and Company, 1975.
13.	Tully MP, Cantrill JA. The use of the nominal group technique in pharmacy practice research: processes and practicalities. J Soc Admin Pharm, 1997, 14: 93-104.
14.	Jones J, Hunter D. Consensus methods for medical and health services research. BMJ, 1995, 311: 376-380.
15.	Wei W, Liu Y, Zhou N, et al. Constructing an emergency preparedness evaluation index system for public use during major emerging infectious disease outbreaks: a Delphi study. BMC Public Health, 2023, 23(1): 1109.
16.	Zhang D, Yan Y, Liao MX, et al. How to evaluate surgical tourism service organizations in China: indicators system development and a pilot application. Glob Health Res Policy, 2022, 7(1): 26.
17.	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.
18.	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.
19.	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.
20.	Dong Z, Dai H, Gao Y, et al. Effect of Mahuang Fuzi and Shenzhuo decoction on idiopathic membranous nephropathy: a multicenter, nonrandomized, single-arm clinical trial. Front Pharmacol, 2021, 12: 724744.
21.	霍雨晴. 注射用丹參多酚酸治療缺血性腦卒中恢復期療效評價及精準定位研究. 天津: 天津中醫藥大學, 2021.
22.	Mehran R, Cao D, Angiolillo DJ, et al. 3- or 1-month DAPT in patients at high bleeding risk undergoing everolimus-eluting stent implantation. JACC Cardiovasc Interv, 2021, 14(17): 1870-1883.
23.	Kandzari DE, Kirtane AJ, Windecker S, et al. One-month dual antiplatelet therapy following percutaneous coronary intervention with Zotarolimus-eluting stents in high-bleeding-risk patients. Circ Cardiovasc Interv, 2020, 13(11): e009565.
24.	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.
25.	牛毅. 運用桂枝茯苓丸加減治療子宮肌瘤療效的臨床研究. 成都: 成都中醫藥大學, 2020.
26.	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.

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. 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.
3. 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.
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. 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.
6. FDA Center for Devices and Radiological Health. The least burdensome provisions: concept and principles: guidance for industry and FDA staff. 2019.
7. 國家藥監局. 關于發布真實世界數據用于醫療器械臨床評價技術指導原則(試行)的通告(2020年第77號). 2020.
8. 中國國家藥監局藥品審評中心. 藥物真實世界研究設計與方案框架指導原則(試行). 2023.
9. 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.
10. 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.
11. EQUATOR Network. Enhancing the quality and transparency of health research.
12. Delbecq AL, van de Ven AH, Gustafson DH. Group techniques for program planning, a guide to nominal group and Delphi processes. Glenview, IL: Scott, Foresman and Company, 1975.
13. Tully MP, Cantrill JA. The use of the nominal group technique in pharmacy practice research: processes and practicalities. J Soc Admin Pharm, 1997, 14: 93-104.
14. Jones J, Hunter D. Consensus methods for medical and health services research. BMJ, 1995, 311: 376-380.
15. Wei W, Liu Y, Zhou N, et al. Constructing an emergency preparedness evaluation index system for public use during major emerging infectious disease outbreaks: a Delphi study. BMC Public Health, 2023, 23(1): 1109.
16. Zhang D, Yan Y, Liao MX, et al. How to evaluate surgical tourism service organizations in China: indicators system development and a pilot application. Glob Health Res Policy, 2022, 7(1): 26.
17. 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.
18. 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.
19. 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.
20. Dong Z, Dai H, Gao Y, et al. Effect of Mahuang Fuzi and Shenzhuo decoction on idiopathic membranous nephropathy: a multicenter, nonrandomized, single-arm clinical trial. Front Pharmacol, 2021, 12: 724744.
21. 霍雨晴. 注射用丹參多酚酸治療缺血性腦卒中恢復期療效評價及精準定位研究. 天津: 天津中醫藥大學, 2021.
22. Mehran R, Cao D, Angiolillo DJ, et al. 3- or 1-month DAPT in patients at high bleeding risk undergoing everolimus-eluting stent implantation. JACC Cardiovasc Interv, 2021, 14(17): 1870-1883.
23. Kandzari DE, Kirtane AJ, Windecker S, et al. One-month dual antiplatelet therapy following percutaneous coronary intervention with Zotarolimus-eluting stents in high-bleeding-risk patients. Circ Cardiovasc Interv, 2020, 13(11): e009565.
24. 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.
25. 牛毅. 運用桂枝茯苓丸加減治療子宮肌瘤療效的臨床研究. 成都: 成都中醫藥大學, 2020.
26. 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.

Chinese Journal of Evidence-Based Medicine

Formation of the list of methodological quality evaluation and explanation for the selection of objective performance criteria in single arm trial

Abstract Full text Figures/Tables Video References Cited by

Previous Article

Next Article

Format

Content