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Statistics in Biopharmaceutical Research Volume 15, 2023 - Issue 1
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Special Section: A Collection of Articles on Opportunities and Challenges in Utilizing Real-World Data for Clinical Trials and Medical Product Development

Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment

Mark Levensona CDER, FDA, Silver Spring, MD;; Correspondence[email protected]
,
Weili Heb Global Medical Affairs Statistics, Data and Statistical Sciences, AbbVie, North Chicago, IL;;
,
Jie Chenc Overland Pharmaceuticals, Dover, DE;;
,
Yixin Fangb Global Medical Affairs Statistics, Data and Statistical Sciences, AbbVie, North Chicago, IL;;
,
Douglas Fariesd Global Statistical Sciences, Eli Lilly & Company, Indianapolis, IN;;
,
Benjamin A. Goldsteine Department of Biostatistics & Bioinformatics, Duke University, Durham, NC;; ;f Duke Clinical Research Institute, Duke University, Durham, NC;;
,
Martin Hog CBER, FDA, Silver Spring, MD;;
,
Kwan Leeh Statistics and Decision Sciences, Janssen Research and Development (retired), Spring House, PA;;
,
Pallavi Mishra-Kalyania CDER, FDA, Silver Spring, MD;;
,
Frank Rockholde Department of Biostatistics & Bioinformatics, Duke University, Durham, NC;; ;f Duke Clinical Research Institute, Duke University, Durham, NC;; ORCID Iconhttps://orcid.org/0000-0003-3732-4765
ORCID Icon,
Hongwei Wangb Global Medical Affairs Statistics, Data and Statistical Sciences, AbbVie, North Chicago, IL;;
&
Richard C. Zinki Lexitas Pharma Services, Inc., Durham, NC
 show all
Pages 3-13 | Received 01 May 2020, Accepted 26 Jan 2021, Published online: 10 Mar 2021

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Read on this site (12)

Olivia McDermott & Breda Kearney. (2023) The value of using real-world evidence as a source of clinical evidence in the European medical device regulations: a mixed methods study. Expert Review of Medical Devices 0:0, pages 1-15.
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Mark Levenson, Weili He, Li Chen, Sai Dharmarajan, Rima Izem, Zhaoling Meng, Herbert Pang & Frank Rockhold. (2023) Statistical Consideration for Fit-for-Use Real-World Data to Support Regulatory Decision Making in Drug Development. Statistics in Biopharmaceutical Research 15:3, pages 689-696.
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Freda Cooner, Ran Liao, Junjing Lin, Sophie Barthel, Yodit Seifu & Shiling Ruan. (2023) Leveraging Real-World Data in COVID-19 Response. Statistics in Biopharmaceutical Research 15:3, pages 582-595.
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Toshimitsu Hamasaki. (2023) Editor’s Note: Special Section on a Collection of Articles on Opportunities and Challenges in Utilizing Real-World Data for Clinical Trials and Medical Product Development. Statistics in Biopharmaceutical Research 15:1, pages 1-2.
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Mark Levenson & Weili He. (2023) Rejoinder to the Commentaries on “The Current Landscape in Biostatistics of Real-World Data and Evidence: Label Expansion”. Statistics in Biopharmaceutical Research 15:1, pages 27-28.
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Gregory Campbell. (2023) Some Biostatistical Considerations About Real-World Data and Evidence in Clinical Studies, Especially for Regulatory Purposes. Statistics in Biopharmaceutical Research 15:1, pages 14-17.
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Lisa V. Hampson, Evgeny Degtyarev, Rui (Sammi) Tang, Jianchang Lin, Kaspar Rufibach & Cheng Zheng. (2023) Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”. Statistics in Biopharmaceutical Research 15:1, pages 23-26.
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Yukari Uemura, Tomohiro Shinozaki, Shogo Nomura & Taro Shibata. (2023) Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”. Statistics in Biopharmaceutical Research 15:1, pages 20-22.
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Chian Chen & Chin-Fu Hsiao. (2023) Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”: Is Real-World Evidence Real?. Statistics in Biopharmaceutical Research 15:1, pages 18-19.
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Weili He, Yixin Fang, Hongwei Wang & Ivan Chan. (2023) Applying Quantitative Approaches in the Use of RWE in Clinical Development and Life-Cycle Management. Statistics in Biopharmaceutical Research 15:1, pages 57-68.
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Xiang Yin, Pallavi S. Mishra-Kalyan, Rajeshwari Sridhara, Mark D. Stewart, Elizabeth A. Stuart & Ruthanna C. Davi. (2022) Exploring the Potential of External Control Arms created from Patient Level Data: A case study in non-small cell lung cancer. Journal of Biopharmaceutical Statistics 32:1, pages 204-218.
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Nelson Lu, Chenguang Wang, Wei-Chen Chen, Heng Li, Changhong Song, Ram Tiwari, Yunling Xu & Lilly Q. Yue. (2022) Propensity score-integrated power prior approach for augmenting the control arm of a randomized controlled trial by incorporating multiple external data sources. Journal of Biopharmaceutical Statistics 32:1, pages 158-169.
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Jonathan Cimino & Claude Braun. (2023) Design a Clinical Research Protocol: Influence of Real-World Setting. Healthcare 11:16, pages 2254.
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Susan Gruber, Rachael V. Phillips, Hana Lee, John Concato & Mark van der Laan. (2023) Evaluating and improving real-world evidence with Targeted Learning. BMC Medical Research Methodology 23:1.
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Stella Erdmann, Dominic Edelmann & Meinhard Kieser. (2023) Using real‐world data to predict health outcomes—The prediction design: Application and sample size planning. Biometrical Journal 65:6.
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Liang Li & Thomas Jemielita. (2023) Confounding adjustment in the analysis of augmented randomized controlled trial with hybrid control arm. Statistics in Medicine 42:16, pages 2855-2872.
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Youngsoo Lee, Ji-Hyang Lee, So Young Park, Ji-Ho Lee, Joo-Hee Kim, Hyun Jung Kim, Sang-Heon Kim, Kian Fan Chung & Woo-Jung Song. (2023) Roles of real-world evidence in severe asthma treatment: challenges and opportunities. ERJ Open Research 9:2, pages 00248-2022.
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Jixian Wang, Hongtao Zhang & Ram Tiwari. 2023. Real-World Evidence in Medical Product Development. Real-World Evidence in Medical Product Development 233 254 .
Weili He, Yixin Fang, Hongwei Wang & Charles Lee. 2023. Real-World Evidence in Medical Product Development. Real-World Evidence in Medical Product Development 3 16 .
Jing Liu, Jeffrey S. Barrett, Efthimia T. Leonardi, Lucy Lee, Satrajit Roychoudhury, Yong Chen & Panayiota Trifillis. (2022) Natural History and Real‐World Data in Rare Diseases: Applications, Limitations, and Future Perspectives. The Journal of Clinical Pharmacology 62:S2.
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Olivier Collignon, Anja Schiel, Carl‐Fredrik Burman, Kaspar Rufibach, Martin Posch & Frank Bretz. (2022) Estimands and Complex Innovative Designs. Clinical Pharmacology & Therapeutics 112:6, pages 1183-1190.
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Julian C. Hong. (2021) Strategies to Turn Real-world Data Into Real-world Knowledge. JAMA Network Open 4:10, pages e2128045.
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