Methods and applications of sample size estimation in real world studies_Chinese Journal of Evidence-Based Medicine

Authors：

CAO Xue ¹ , HE Zhenfan ² ,  LIU Jia ¹ ,  CHEN Xinlin ²

1. Institute of Basic Research in Clinical Medicine, China Academy of Chinese Medical Sciences, Beijing 100700, P. R. China;
2. School of Basic Medical Sciences, Guangzhou University of Chinese Medicine, Guangzhou 510006, P. R. China;

Corresponding?author：

LIU Jia, Email: marie_liujia@163.com; CHEN Xinlin, Email: chenxlsums@126.com

Keywords：

Real world study; Sample size estimation; Current situation; Reporting guidance

DOI：

10.7507/1672-2531.202510109

Video：

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Objective To review the current application of sample size estimation in real-world studies (RWS), analyse parameter settings and commonly used methods, and provide methodological guidance for researchers conducting RWS. Methods First, ClinicalTrials.gov was searched to identify RWS with documented sample size calculations. Key information was extracted for descriptive analysis. Secondly, critical parameters and common estimation methods for RWS sample size calculations were systematically reviewed, and strategies were proposed for addressing common challenges. Finally, relevant international reporting standards were interpreted. Results The literature review included 44 clinical trials with a wide range of sample sizes (30 to 30 400 cases). While most studies detailed the sample size estimation process, the parameter settings were often incomplete and many failed to adequately consider the characteristics of real-world data. Therefore, we proposed key parameters for RWS sample size estimation, including effect size, significance level and statistical power. Researchers should also consider issues such as heterogeneity, confounding factors and data quality. This study clarified the essential elements of reporting sample size estimation. Conclusion Methodological guidance for real-world evidence sample size estimation is lacking. We advise researchers to standardise reporting procedures for sample size estimation in future studies and to set parameters reasonably based on research objectives, study design types and data characteristics. This will enhance the transparency and scientific rigour of real-world evidence.

Citation： CAO Xue, HE Zhenfan, LIU Jia, CHEN Xinlin. Methods and applications of sample size estimation in real world studies. Chinese Journal of Evidence-Based Medicine, 2026, 26(2): 202-209. doi: 10.7507/1672-2531.202510109 Copy

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1. 石舒原, 趙厚宇, 劉佐相, 等. 增強真實世界證據可信度與應用價值的國際研究經驗與啟示. 醫藥導報, 2023, 42(1): 24-31.
2. 秦雪妮, 陳維生, 邵華, 等. 真實世界研究在醫藥領域的應用及研究方法. 藥學進展, 2021, 45(7): 512-523.
3. 張慧娥, 肖夢麗, 譚琴, 等. 真實世界研究與藥品臨床綜合評價: 思考與應用. 臨床合理用藥, 2025, 18(10): 164-166, 175.
4. 徐嘉悅, 何俏, 劉艷梅, 等. 真實世界數據用于藥品臨床價值評價的關鍵技術考量. 中國循證醫學雜志, 2024, 24(5): 523-528.
5. 國家藥監局藥審中心. 關于發布《藥物臨床試驗樣本量估計指導原則(試行)》的通告(2024年第54號). 2024.
6. 王詩淳, 李紅艷, 馬文昊, 等. 基于中國臨床試驗注冊中心的真實世界研究現況分析. 中國循證醫學雜志, 2023, 23(1): 75-79.
7. Kim HS, Kim JH. Proceed with caution when using real world data and real world evidence. J Korean Med Sci, 2019, 34(4): e28.
8. Yan YY, Yang YH, Wang WW, et al. Post-marketing safety surveillance of the salvia miltiorrhiza depside salt for infusion: a real world study. PLoS One, 2017, 12(1): e0170182.
9. Jeong IK, Kim SR. Efficacy and safety of Pitavastatin in a real-world setting: observational study evaluating safety in patient treated with Pitavastatin in Korea (PROOF study). Endocrinol Metab (Seoul), 2020, 35(4): 882-891.
10. Sun W, Ou Q, Zhang Z, et al. Chinese acute ischemic stroke treatment outcome registry (CASTOR): protocol for a prospective registry study on patterns of real-world treatment of acute ischemic stroke in China. BMC Complement Altern Med, 2017, 17(1): 357.
11. Goulart Rosa R, Spinardi J, Allen KE, et al. BNT162b2 against COVID-19 in Brazil using a test-negative design: study protocol and statistical analysis plan. PLoS One, 2022, 17(10): e0276384.
12. Guo J, Hu X, Wang J, et al. Safety and efficacy of compound methyl salicylate liniment for topical pain: a multicenter real-world study in China. Front Pharmacol, 2022, 13: 1015941.
13. Thomas FP, Saporta M, Attarian S, et al. Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study. Neurodegener Dis Manag, 2021, 11(1): 21-33.
14. Katakami N, Mita T, Takahara M, et al. Rationale and design for the J-DISCOVER study: DISCOVERing the treatment reality of type 2 diabetes in a real-world setting in Japan-a protocol. Diabetes Ther, 2018, 9(1): 165-175.
15. Zhao X, Tong Z, Sun L, et al. Clinical characteristics, treatment patterns, and effectiveness in Chinese patients with angina pectoris using electronic patient-reported outcomes: protocol for a multicenter, prospective, cohort Study (GREAT). Adv Ther, 2023, 40(4): 1899-1912.
16. Zhuang W, Tang Y, Xu W, et al. Should psychological distress be listed as a surgical indication for indeterminate pulmonary nodules: protocol for a prospective cohort study in real-world settings. J Thorac Dis, 2022, 14(3): 769-778.
17. Liu Y, Wang Q, Wu Z, et al. A prospective, multicenter, real-world observational study evaluating the impact of tibial runoff on clinical outcomes after endovascular therapy for femoropopliteal lesions: research protocol. Front Cardiovasc Med, 2022, 9: 1035659.
18. He F, Sun Y, Zhang W, et al. Safety and efficacy of generic nab-paclitaxel-based therapy in Chinese patients with malignant tumors in a real-world setting: a multicenter prospective observational study. Discov Oncol, 2024, 15(1): 712.
19. Pan Q, Zhai X, Wang H, et al. Real-world pharmacological treatment pattern of neuropathic pain in China: a retrospective, database, multicenter study (ReTARdant) protocol. Pain Ther, 2025, 14(5): 1611-1627.
20. Shen N, Meng Q, Zhang L, et al. Evaluation of safety, effectiveness and treatment patterns of sodium zirconium cyclosilicate in management of hyperkalaemia in China: a real-world study protocol. BMJ Open, 2023, 13(3): e070530.
21. Sun Y, Guo N, Zhang M, et al. Association between preoperative frailty and myocardial injury after noncardiac surgery in geriatric patients: study protocol for a prospective, multicentre, real-world observational, cohort trial. BMC Geriatr, 2024, 24(1): 271.
22. An Y, Liu T, He D, et al. The usage of biological DMARDs and clinical remission of rheumatoid arthritis in China: a real-world large scale study. Clin Rheumatol, 2017, 36(1): 35-43.
23. Rodrigues CO, Spinardi J, Rosa RG, et al. Real-world effectiveness of original BNT162b2 mRNA COVID-19 against symptomatic Omicron infection among children 5-11 years of age in Brazil: a prospective test-negative design study. Immunol Lett, 2024, 269: 106903.
24. Procopio G, Bamias A, Schmidinger M, et al. Real-world effectiveness and safety of Pazopanib in patients with intermediate prognostic risk advanced renal cell carcinoma. Clin Genitourin Cancer, 2019, 17(3): e526-e533.
25. Krag A, Schuchmann M, Sodatonou H, et al. Design of the prospective real-world outcomes study of hepatic encephalopathy patients' experience on Rifaximin-α (PROSPER): an observational study among 550 patients. Hepatol Med Policy, 2018, 3: 4.
26. Soriano A, Montravers P, Bassetti M, et al. The use and effectiveness of Ceftazidime-avibactam in real-world clinical practice: EZTEAM study. Infect Dis Ther, 2023, 12(3): 891-917.
27. Augustin M, Sommer R, Daudén E, et al. Patient-reported well-being in value-based care using tildrakizumab in a real-world setting: protocol of a multinational, phase IV, 1-cohort prospective observational study (the POSITIVE study). BMJ Open, 2023, 13(2): e060536.
28. Li Y, Chen W, Xie F, et al. Novel electromagnetic navigation bronchoscopy system for the diagnosis of peripheral pulmonary nodules: a prospective, multicentre study. Thorax, 2023, 78(12): 1197-1205.
29. Pelle J, Briant AR, Branger P, et al. Real-world effectiveness of Natalizumab extended interval dosing in a French cohort. Neurol Ther, 2023, 12(2): 529-542.
30. Lee JY, Park JY, Park SY, et al. Real-world effectiveness of bevacizumab based on AURELIA in platinum-resistant recurrent ovarian cancer (REBECA): a Korean Gynecologic Oncology Group study (KGOG 3041). Gynecol Oncol, 2019, 152(1): 61-67.
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Chinese Journal of Evidence-Based Medicine

Methods and applications of sample size estimation in real world studies

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