Current Status of Single-arm Trials with External Controls and Case Analysis of Bias Risk Assessment

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LI Ge, SUN Feng. Current Status of Single-arm Trials with External Controls and Case Analysis of Bias Risk Assessment[J]. CHINESE JOURNAL OF DRUG EVALUATION, 2022, 39(5): 363-369.

[1]Zhang Y， Naci H， Wagner AK， et al. Overall Survival Benefits of Cancer Drugs Approved in China From 2005 to 2020[J]. JAMA Netw Open， 2022，5(8)： e2225973.
     [2]唐凌，周明，夏琳，等. 对单臂试验支持抗肿瘤药物上市策略的再思考[J]. 中华肿瘤杂志，2022，44(6)： 587-92.
     [3]Jahanshahi M， Gregg K， Davis G， et al. The Use of External Controls in FDA Regulatory Decision Making[J]. Ther Innov Regul Sci，2021，
     [4]Demystifying Synthetic Control Arms[EB/OL]. https：//www.cytel.com/hubfs/ebook-SCA-digi_v4_SM%20(002).pdf (accessed October 09 2022).
     [5]Securing market access with single-arm trial data[EB/OL]. https：//www.iqvia.com/blogs/2021/06/securing-market-access-with-single-arm-trial-data#：~：text=Based%20on%20our%20data%20extraction%20parameters%20we%E2%80%99ve%20identified，2019%2C%20mostly%20in%20oncology%20indications%20%28see%20Figure%201%29. (accessed October 09 2022).
     [6]Song Y， Wu J， Chen X， et al. A Single-Arm， Multicenter， Phase II Study of Camrelizumab in Relapsed or Refractory Classical Hodgkin Lymphoma[J].Clin Cancer Res，2019，25(24)： 7363-9.
     [7]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[J]. Pharmacoepidemiol Drug Saf，2020，29(11)： 1382-1392.
     [8]Burcu M， Dreyer NA， Franklin JM， et al. Real-world evidence to support regulatory decision-making for medicines： Considerations for external control arms[J]. Pharmacoepidemiol DrugSaf，2020，29(10)： 1228-35.
     [9]Sterne JA， Hernan MA， Reeves BC， et al. ROBINS-I： a tool for assessing risk of bias in non-randomised studies of interventions[J]. BMJ，2016，355： i4919.
     [10]Doebele RC， Perez L， Trinh H， et al. Comparative effectiveness analysis between entrectinib clinical trial and crizotinib real-world data in ROS1+ NSCLC[J].Journal of Comparative Effectiveness Research，2021，10(17)： 1271-82.
     [11]孙凤，高乐，杨智荣，等. 偏倚风险评估系列：(五)非随机干预性研究[J]. 中华流行病学杂志， 2018，39(3)： 374-81.
     [12]Risk Of Bias In Non-randomized Studies of Interventions (ROBINS-I)： detailed guidance[EB/OL]. https：//www.bristol.ac.uk/media-library/sites/social-community-medicine/images/centres/cresyda/ROBINS-I_detailed_guidance.pdf (accessed October 22 2022).
     [13]Franklin JM， Schneeweiss S. When and How Can Real World Data Analyses Substitute for Randomized Controlled Trials?； (1532-6535 (Electronic)).
     [14]Simon R， Blumenthal Gm Fau-Rothenberg ML， Rothenberg Ml Fau-Sommer J， et al. The role of nonrandomized trials in the evaluation of oncology drugs. (1532-6535 (Electronic)).
     [15]Suissa S. Single-arm Trials with Historical Controls： Study Designs to Avoid Time-related Biases. Epidemiology 2021； 32(1)： 94-100.
     [16]石舒原，赵厚宇，周庆欣，等. 真实世界证据与随机对照试验：RCT DUPLICATE项目方法学介绍[J]. 药物流行病学杂志， 2020，29(3)： 8.
     [17]Ghadessi M， Tang R， Zhou J， et al. A roadmap to using historical controls in clinical trials-by Drug Information Association Adaptive Design Scientific Working Group (DIA-ADSWG)[J]. Orphanet J Rare Dis，2020，15(1)： 69.
     [18]Wang CY， Berlin JA， Gertz B， et al. Uncontrolled Extensions of Clinical Trials and the Use of External Controls-Scoping Opportunities and Methods[J]. Clin Pharmacol Ther，2022，111(1)： 187-199.

Current Status of Single-arm Trials with External Controls and Case Analysis of Bias Risk Assessment

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