Statistics in Biopharmaceutical Research, Volume 15, Issue 1 (2023)

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Statistics in Biopharmaceutical Research, Volume 15, Issue 1 (2023)

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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

Introduction
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
Toshimitsu Hamasaki
Pages: 1-2
Published online: 07 Feb 2023
CitationforEditor’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 | Full Text | References | PDF (389.6 KB) | EPUB
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Free Access
Article
Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment
Mark Levenson, Weili He, Jie Chen, Yixin Fang, Douglas Faries, Benjamin A. Goldstein, Martin Ho, Kwan Lee, Pallavi Mishra-Kalyani, Frank Rockhold, Hongwei Wang & Richard C. Zink
Pages: 3-13
Published online: 10 Mar 2021
AbstractforBiostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment | Full Text | References | PDF (980.1 KB) | EPUB
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Some Biostatistical Considerations About Real-World Data and Evidence in Clinical Studies, Especially for Regulatory Purposes
Gregory Campbell
Pages: 14-17
Published online: 01 Dec 2021
AbstractforSome Biostatistical Considerations About Real-World Data and Evidence in Clinical Studies, Especially for Regulatory Purposes | Full Text | References | PDF (444.5 KB) | EPUB
  • 430 Views
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Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”: Is Real-World Evidence Real?
Chian Chen & Chin-Fu Hsiao
Pages: 18-19
Published online: 27 Sep 2021
CitationforComment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”: Is Real-World Evidence Real? | Full Text | References | PDF (278.3 KB) | EPUB
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Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”
Yukari Uemura, Tomohiro Shinozaki, Shogo Nomura & Taro Shibata
Pages: 20-22
Published online: 04 Oct 2021
First Page PreviewforComment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment” | Full Text | References | PDF (424.4 KB) | EPUB
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Open Access
Comment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment”
Lisa V. Hampson, Evgeny Degtyarev, Rui (Sammi) Tang, Jianchang Lin, Kaspar Rufibach & Cheng Zheng
Pages: 23-26
Published online: 01 Dec 2021
AbstractforComment on “Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment” | Full Text | References | PDF (444.7 KB) | EPUB
  • 550 Views
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Rejoinder to the Commentaries on “The Current Landscape in Biostatistics of Real-World Data and Evidence: Label Expansion”
Mark Levenson & Weili He
Pages: 27-28
Published online: 18 Jul 2022
CitationforRejoinder to the Commentaries on “The Current Landscape in Biostatistics of Real-World Data and Evidence: Label Expansion” | Full Text | References | PDF (341.4 KB) | EPUB
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Article
The Current Landscape in Biostatistics of Real-World Data and Evidence: Clinical Study Design and Analysis
Jie Chen, Martin Ho, Kwan Lee, Yang Song, Yixin Fang, Benjamin A Goldstein, Weili He, Telba Irony, Qi Jiang, Mark van der Laan, Hana Lee, Xiwu Lin, Zhaoling Meng, Pallavi Mishra-Kalyani, Frank Rockhold, Hongwei Wang & Roseann White
Pages: 29-42
Published online: 15 Mar 2021
AbstractforThe Current Landscape in Biostatistics of Real-World Data and Evidence: Clinical Study Design and Analysis | Full Text | References | PDF (1.2 MB) | EPUB
  • 2150 Views
  • 16 CrossRef citations
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Article
The Current Landscape in Biostatistics of Real-World Data and Evidence: Causal Inference Frameworks for Study Design and Analysis
Martin Ho, Mark van der Laan, Hana Lee, Jie Chen, Kwan Lee, Yixin Fang, Weili He, Telba Irony, Qi Jiang, Xiwu Lin, Zhaoling Meng, Pallavi Mishra-Kalyani, Frank Rockhold, Yang Song, Hongwei Wang & Roseann White
Pages: 43-56
Published online: 15 Mar 2021
AbstractforThe Current Landscape in Biostatistics of Real-World Data and Evidence: Causal Inference Frameworks for Study Design and Analysis | Full Text | References | PDF (2 MB) | EPUB
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Article
Applying Quantitative Approaches in the Use of RWE in Clinical Development and Life-Cycle Management
Weili He, Yixin Fang, Hongwei Wang & Ivan Chan
Pages: 57-68
Published online: 06 Jul 2021
AbstractforApplying Quantitative Approaches in the Use of RWE in Clinical Development and Life-Cycle Management | Full Text | References | PDF (1.2 MB) | EPUB
  • 445 Views
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Article
Comparison of Sample Size Requirements of Randomized and Historically Controlled Trials Based on Calibrated Error Rates
Srinand Nandakumar & Steven M. Snapinn
Pages: 69-73
Published online: 26 Feb 2021
AbstractforComparison of Sample Size Requirements of Randomized and Historically Controlled Trials Based on Calibrated Error Rates | Full Text | References | PDF (714.3 KB) | EPUB
  • 167 Views
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Discussion
Good Data Science Practice: Moving Toward a Code of Practice for Drug Development
Mark Baillie, Conor Moloney, Carsten Philipp Mueller, Jonas Dorn, Janice Branson & David Ohlssen
Pages: 74-85
Published online: 16 May 2022
AbstractforGood Data Science Practice: Moving Toward a Code of Practice for Drug Development | Full Text | References | PDF (1.3 MB) | EPUB
  • 973 Views
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Comment on “Good Data Science Practice: Moving towards a Code of Practice for Drug Development”
Yixin Fang & Weili He
Pages: 86-88
Published online: 18 Jul 2022
AbstractforComment on “Good Data Science Practice: Moving towards a Code of Practice for Drug Development” | Full Text | References | PDF (376.1 KB) | EPUB
  • 142 Views
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Good Data Science Practice: Moving toward a Code of Practice for Drug Development (Rejoinder)
Mark Baillie, Conor Moloney, Carsten Philipp Mueller, Jonas Dorn, Janice Branson & David Ohlssen
Pages: 89-91
Published online: 07 Feb 2023
First Page PreviewforGood Data Science Practice: Moving toward a Code of Practice for Drug Development (Rejoinder) | Full Text | References | PDF (582.9 KB) | EPUB
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The NISS Special Series: The NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions

The NISS Ingram Olkin Forum on Unplanned Clinical Trial Disruptions
Nancy Flournoy
Pages: 92-93
Published online: 07 Feb 2023
CitationforThe NISS Ingram Olkin Forum on Unplanned Clinical Trial Disruptions | Full Text | References | PDF (525 KB) | EPUB
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Estimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions
Kelly Van Lancker, Sergey Tarima, Jonathan Bartlett, Madeline Bauer, Bharani Bharani-Dharan, Frank Bretz, Nancy Flournoy, Hege Michiels, Camila Olarte Parra, James L. Rosenberger & Suzie Cro
Pages: 94-111
Published online: 14 Sep 2022
AbstractforEstimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions | Full Text | References | PDF (2.3 MB) | EPUB | Supplemental
  • 836 Views
  • 6 CrossRef citations
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Comment on” Estimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions”
Stijn Vansteelandt
Pages: 112-115
Published online: 07 Feb 2023
First Page PreviewforComment on” Estimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions” | Full Text | References | PDF (539.6 KB) | EPUB
  • 332 Views
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Comment on “Estimands and Their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions”
Mouna Akacha & Tianmeng Lyu
Pages: 116-118
Published online: 07 Feb 2023
First Page PreviewforComment on “Estimands and Their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions” | Full Text | References | PDF (351.4 KB) | EPUB
  • 306 Views
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Rejoinder: Estimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions
Kelly Van Lancker, Sergey Tarima, Jonathan Bartlett, Madeline Bauer, Bharani Bharani-Dharan, Frank Bretz, Nancy Flournoy, Hege Michiels, Camila Olarte Parra, James L Rosenberger & Suzie Cro
Pages: 119-124
Published online: 07 Feb 2023
First Page PreviewforRejoinder: Estimands and their Estimators for Clinical Trials Impacted by the COVID-19 Pandemic: A Report from the NISS Ingram Olkin Forum Series on Unplanned Clinical Trial Disruptions | Full Text | References | PDF (1.9 MB) | EPUB | Supplemental
  • 401 Views
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Research Articles

Article
Generalizing Clinical Trial Results to a Target Population
Shuang Li & Daniel F. Heitjan
Pages: 125-132
Published online: 26 Jul 2021
AbstractforGeneralizing Clinical Trial Results to a Target Population | Full Text | References | PDF (841.8 KB) | EPUB
  • 283 Views
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Article
Trigger Strategy in Repeated Tests on Multiple Hypotheses
Jiangtao Gou
Pages: 133-140
Published online: 26 Jul 2021
AbstractforTrigger Strategy in Repeated Tests on Multiple Hypotheses | Full Text | References | PDF (765.6 KB) | EPUB
  • 139 Views
  • 2 CrossRef citations
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Article
Statistical Inference of the Probability of Passing the USP Dissolution Test
Tee-Chin Chang
Pages: 141-153
Published online: 18 Aug 2021
AbstractforStatistical Inference of the Probability of Passing the USP Dissolution Test | Full Text | References | PDF (2.9 MB) | EPUB
  • 215 Views
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Article
Comparative Effectiveness Research using Bayesian Adaptive Designs for Rare Diseases: Response Adaptive Randomization Reusing Participants
Fengming Tang & Byron J. Gajewski
Pages: 154-163
Published online: 31 Aug 2021
AbstractforComparative Effectiveness Research using Bayesian Adaptive Designs for Rare Diseases: Response Adaptive Randomization Reusing Participants | Full Text | References | PDF (1.8 MB) | EPUB
  • 144 Views
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Article
Multiarmed Bandit Designs for Phase I Dose-Finding Clinical Trials With Multiple Toxicity Types
Lan Jin, Guodong Pang & Demissie Alemayehu
Pages: 164-177
Published online: 31 Aug 2021
AbstractforMultiarmed Bandit Designs for Phase I Dose-Finding Clinical Trials With Multiple Toxicity Types | Full Text | References | PDF (1.7 MB) | EPUB
  • 241 Views
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Article
Reference-Based Multiple Imputation—What is the Right Variance and How to Estimate It
Jonathan W. Bartlett
Pages: 178-186
Published online: 12 Nov 2021
AbstractforReference-Based Multiple Imputation—What is the Right Variance and How to Estimate It | Full Text | References | PDF (977.8 KB) | EPUB
  • 5056 Views
  • 5 CrossRef citations
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Open Access
Article
Weighted Approach for Estimating Effects in Principal Strata With Missing Data for a Categorical Post-Baseline Variable in Randomized Controlled Trials
Shengchun Kong, Dominik Heinzmann, Sabine Lauer & Lu Tian
Pages: 187-197
Published online: 28 Jan 2022
AbstractforWeighted Approach for Estimating Effects in Principal Strata With Missing Data for a Categorical Post-Baseline Variable in Randomized Controlled Trials | Full Text | References | PDF (1.5 MB) | EPUB | Supplemental
  • 234 Views
  • 3 CrossRef citations
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Article
Optimal Spiking Experiment for Noninferiority of Qualitative Microbiological Methods on Accuracy With Multiple Microorganisms
Mona Emampour, Pieta C. IJzerman-Boon, Md Abu Manju & Edwin R. van den Heuvel
Pages: 198-213
Published online: 02 Feb 2022
AbstractforOptimal Spiking Experiment for Noninferiority of Qualitative Microbiological Methods on Accuracy With Multiple Microorganisms | Full Text | References | PDF (3.1 MB) | EPUB | Supplemental
  • 974 Views
  • 1 CrossRef citations
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Open Access
Article
Efficient Estimation of the Efficacy and Safety Endpoints for Clinical Trials with Preplanned Dose Titrations
Yongming Qu, Xuewei Cui, Ross Bray & Nicholas Sun
Pages: 214-224
Published online: 24 Feb 2022
AbstractforEfficient Estimation of the Efficacy and Safety Endpoints for Clinical Trials with Preplanned Dose Titrations | Full Text | References | PDF (1.6 MB) | EPUB | Supplemental
  • 211 Views
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Article
Continuous Method Validation: Beyond One-Time Studies to Characterize Analytical Methods
Paul Faya, Steven Novick, John W. Seaman Jr., John J. Peterson, Tony Pourmohamad, Dwaine Banton & Yanbing Zheng
Pages: 225-233
Published online: 01 Apr 2022
AbstractforContinuous Method Validation: Beyond One-Time Studies to Characterize Analytical Methods | Full Text | References | PDF (1 MB) | EPUB
  • 312 Views
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