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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

The Current Landscape in Biostatistics of Real-World Data and Evidence: Causal Inference Frameworks for Study Design and Analysis

Martin Hoa CBER, FDA, Silver Spring, MD; Correspondence[email protected]
,
Mark van der Laanb Biostatistics, University of California, Berkeley, Berkeley, CA;
,
Hana Leec CDER, FDA, Silver Spring, MD;
,
Jie Chend Overland Pharmaceuticals, Dover, DE;
,
Kwan Leee Janssen Research and Development, Spring House, PA;
,
Yixin Fangf Global Medical Affairs Statistics, Data and Statistical Sciences, AbbVie, North Chicago, IL;
,
Weili Hef Global Medical Affairs Statistics, Data and Statistical Sciences, AbbVie, North Chicago, IL;
,
Telba Ironya CBER, FDA, Silver Spring, MD;
,
Qi Jiangg Seagen, Bothell, WA;
,
Xiwu Line Janssen Research and Development, Spring House, PA;
,
Zhaoling Mengh Sanofi, Cambridge, MA;
,
Pallavi Mishra-Kalyanic CDER, FDA, Silver Spring, MD;
,
Frank Rockholdi Duke Clinical Research Institute and Duke University Medical Center, Duke University, Durham, NC; ORCID Iconhttps://orcid.org/0000-0003-3732-4765
ORCID Icon,
Yang Songj Vertex Pharmaceuticals, Boston, MA;
,
Hongwei Wangf Global Medical Affairs Statistics, Data and Statistical Sciences, AbbVie, North Chicago, IL;
&
Roseann Whitek Your Third Opinion, Raleigh, NC
 show all
Pages 43-56 | Received 19 May 2020, Accepted 26 Jan 2021, Published online: 15 Mar 2021

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Susan Gruber, Hana Lee, Rachael Phillips, Martin Ho & Mark van der Laan. (2023) Developing a Targeted Learning-Based Statistical Analysis Plan. Statistics in Biopharmaceutical Research 15:3, pages 468-475.
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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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Jeen Liu, Jane Zhang, Alan Mitchell, Mindy Fang & Lu Tian. (2023) Causal inference for longitudinal data based on historical controls. Journal of Biopharmaceutical Statistics 33:3, pages 289-306.
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Martin Ho, Susan Gruber, Yixin Fang, Douglas E. Faris, Pallavi Mishra-Kalyani, David Benkeser & Mark van der Laan. (2023) Examples of Applying RWE Causal-Inference Roadmap to Clinical Studies. Statistics in Biopharmaceutical Research 0:0, pages 1-14.
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Susan Gruber, Rachael V. Phillips, Hana Lee, Martin Ho, John Concato & Mark J. van der Laan. (2023) Targeted Learning: Toward a Future Informed by Real-World Evidence. Statistics in Biopharmaceutical Research 0:0, pages 1-15.
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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 Baillie, Conor Moloney, Carsten Philipp Mueller, Jonas Dorn, Janice Branson & David Ohlssen. (2023) Good Data Science Practice: Moving toward a Code of Practice for Drug Development (Rejoinder). Statistics in Biopharmaceutical Research 15:1, pages 89-91.
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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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Lauren E. Dang, Susan Gruber, Hana Lee, Issa J. Dahabreh, Elizabeth A. Stuart, Brian D. Williamson, Richard Wyss, Iván Díaz, Debashis Ghosh, Emre Kıcıman, Demissie Alemayehu, Katherine L. Hoffman, Carla Y. Vossen, Raymond A. Huml, Henrik Ravn, Kajsa Kvist, Richard Pratley, Mei-Chiung Shih, Gene Pennello, David Martin, Salina P. Waddy, Charles E. Barr, Mouna Akacha, John B. Buse, Mark van der Laan & Maya Petersen. (2023) A causal roadmap for generating high-quality real-world evidence. Journal of Clinical and Translational Science 7:1.
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Ying Wu, Hongwei Wang, Jie Chen & Hana Lee. 2023. Real-World Evidence in Medical Product Development. Real-World Evidence in Medical Product Development 145 165 .
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Xingqiao Wang, Xiaowei Xu, Weida Tong, Qi Liu & Zhichao Liu. (2022) DeepCausality: A general AI-powered causal inference framework for free text: A case study of LiverTox. Frontiers in Artificial Intelligence 5.
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Fang Liu & Demosthenes Panagiotakos. (2022) Real-world data: a brief review of the methods, applications, challenges and opportunities. BMC Medical Research Methodology 22:1.
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Hongwei Wang, Yixin Fang, Weili He, Ruizhe Chen & Su Chen. (2022) Clinical Trials with External Control: Beyond Propensity Score Matching. Statistics in Biosciences 14:2, pages 304-317.
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Thomas Laurent, Jason Simeone, Ryohei Kuwatsuru, Takahiro Hirano, Sophie Graham, Ryozo Wakabayashi, Robert Phillips & Tatsuya Isomura. (2022) Context and Considerations for Use of Two Japanese Real-World Databases in Japan: Medical Data Vision and Japanese Medical Data Center. Drugs - Real World Outcomes 9:2, pages 175-187.
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