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：

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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.

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26.	牛毅. 运用桂枝茯苓丸加减治疗子宫肌瘤疗效的临床研究. 成都: 成都中医药大学, 2020.
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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. 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.
3. 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.
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. 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.
7. 国家药监局. 关于发布真实世界数据用于医疗器械临床评价技术指导原则(试行)的通告(2020年第77号). 2020.
8. 中国国家药监局药品审评中心. 药物真实世界研究设计与方案框架指导原则(试行). 2023.
9. 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.
10. 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.
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. FDA Center For Devices And Radiological Health. The least burdensome provisions: concept and principles: guidance for industry and FDA staff. 2019.
18. 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.
19. 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.
20. 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.
21. 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.
22. 霍雨晴. 注射用丹参多酚酸治疗缺血性脑卒中恢复期疗效评价及精准定位研究. 天津: 天津中医药大学, 2021.
23. 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.
24. 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.
25. 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.
26. 牛毅. 运用桂枝茯苓丸加减治疗子宫肌瘤疗效的临床研究. 成都: 成都中医药大学, 2020.
27. 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.

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