1. |
Wieseler B, Neyt M, Kaiser T, et al. Replacing RCTs with real world data for regulatory decision making: a self-fulfilling prophecy. BMJ, 2023, 380: e073100.
|
2. |
国家卫生健康委. 药品临床综合评价管理指南(2021年版试行)(国卫办药政发〔2021〕16号). 2021.
|
3. |
海南省医疗保障局. 海南省医疗保障局关于推进真实世界数据医保创新应用的若干意见(琼医保〔2023〕24号). 2023.
|
4. |
Wang W, Tan J, Wu J, et al. Use of real world data to improve drug coverage decisions in China. BMJ, 2023, 381: e068911.
|
5. |
刘炳林, 薛斐然. 药物临床价值评估的主要考虑因素及问题. 中国新药杂志, 2017, 26(5): 504-508.
|
6. |
孙鑫, 谭婧, 唐立, 等. 基于真实世界证据的上市后药品评价技术框架体系: 思考与建议. 中国循证医学杂志, 2018, 18(4): 277-283.
|
7. |
Agrawal S, Arora S, Amiri-Kordestani L, et al. Use of single-arm trials for US food and drug administration drug approval in oncology, 2002-2021. JAMA Oncol, 2023, 9(2): 266-272.
|
8. |
王雯, 谭婧, 任燕, 等. 重新认识真实世界数据研究: 更新与展望. 中国循证医学杂志, 2020, 20(11): 1241-1246.
|
9. |
孙鑫, 谭婧, 唐立, 等. 重新认识真实世界研究. 中国循证医学杂志, 2017, 17(2): 126-130.
|
10. |
谭婧, 彭晓霞, 舒啸尘, 等. 患者登记数据库构建技术规范. 中国循证医学杂志, 2019, 19(7): 771-778.
|
11. |
Ford I, Norrie J. Pragmatic trials. N Engl J Med, 2016, 375(5): 454-463.
|
12. |
王雨宁, 姚明宏, 任燕, 等. 真实世界数据作为外部对照用于药械临床评价的特殊考虑. 中华流行病学杂志, 2023, 44(2): 335-340.
|
13. |
Cox E, Martin BC, Van Staa T, et al. Good research practices for comparative effectiveness research: approaches to mitigate bias and confounding in the design of nonrandomized studies of treatment effects using secondary data sources: the International Society For Pharmacoeconomics and Outcomes Research Good Research Practices For Retrospective Database Analysis Task Force report--Part II. Value Health, 2009, 12(8): 1053-1061.
|
14. |
国家药品监督管理局药品审评中心. 药物真实世界研究设计与方案框架指导原则(试行). 2023.
|
15. |
温泽淮, 李玲, 刘艳梅, 等. 实效性随机对照试验的技术规范. 中国循证医学杂志, 2019, 19(7): 794-802.
|
16. |
Loudon K, Treweek S, Sullivan F, et al. The PRECIS-2 tool: designing trials that are fit for purpose. BMJ, 2015, 350: h2147.
|
17. |
Hernán MA, Robins JM. Using big data to emulate a target trial when a randomized trial is not available. Am J Epidemiol, 2016, 183(8): 758-764.
|
18. |
FDA. Best practices for conducting and reporting pharmacoepidemiologic safety studies using electronic healthcare data. 2013.
|
19. |
彭晓霞, 舒啸尘, 谭婧, 等. 基于真实世界数据评价治疗结局的观察性研究设计技术规范. 中国循证医学杂志, 2019, 19(7): 779-786.
|
20. |
Yoshida K, Solomon DH, Kim SC. Active-comparator design and new-user design in observational studies. Nat Rev Rheumatol, 2015, 11(7): 437-441.
|
21. |
Kyriacou DN, Lewis RJ. Confounding by indication in clinical research. JAMA, 2016, 316(17): 1818-1819.
|
22. |
Yadav K, Lewis RJ. Immortal time bias in observational studies. JAMA, 2021, 325(7): 686-687.
|
23. |
Seeger JD, Davis KJ, Iannacone MR, et al. Methods for external control groups for single arm trials or long-term uncontrolled extensions to randomized clinical trials. Pharmacoepidemiol Drug Saf, 2020, 29(11): 1382-1392.
|
24. |
Schmidli H, Häring DA, Thomas M, et al. Beyond randomized clinical trials: use of external controls. Clin Pharmacol Ther, 2020, 107(4): 806-816.
|
25. |
Carpenter JR, Smuk M. Missing data: a statistical framework for practice. Biom J, 2021, 63(5): 915-947.
|
26. |
严洁. 缺失数据的多重插补. 重庆: 重庆大学出版社, 2017.
|