Statistics in Biopharmaceutical Research, Volume 15, Issue 3 (2023)

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Statistics in Biopharmaceutical Research, Volume 15, Issue 3 (2023)

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Statistical Innovation in Healthcare: Celebrating the Past 40 Years and Looking Toward the Future - Special issue for the 2021 Regulatory-Industry Statistics Workshop

Introduction
Statistical Innovation in Healthcare: Celebrating the Past 40 Years and Looking Toward the Future–Special Issue for the 2021 Regulatory-Industry Statistics Workshop
Bo Huang & Gene Pennello
Page: 457
Published online: 02 Aug 2023
CitationforStatistical Innovation in Healthcare: Celebrating the Past 40 Years and Looking Toward the Future–Special Issue for the 2021 Regulatory-Industry Statistics Workshop | Full Text | PDF (406.1 KB) | EPUB
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Free Access
Article
The Role of Statistical Thinking in Biopharmaceutical Research
Frank Bretz & Joel B. Greenhouse
Pages: 458-467
Published online: 24 Jul 2023
AbstractforThe Role of Statistical Thinking in Biopharmaceutical Research | Full Text | References | PDF (1.6 MB) | EPUB
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Article
Developing a Targeted Learning-Based Statistical Analysis Plan
Susan Gruber, Hana Lee, Rachael Phillips, Martin Ho & Mark van der Laan
Pages: 468-475
Published online: 03 Oct 2022
AbstractforDeveloping a Targeted Learning-Based Statistical Analysis Plan | Full Text | References | PDF (1.3 MB) | EPUB | Supplemental
  • 831 Views
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Article
Statistical Considerations and Challenges for Pivotal Clinical Studies of Artificial Intelligence Medical Tests for Widespread Use: Opportunities for Inter-Disciplinary Collaboration
Arkendra De
Pages: 476-490
Published online: 14 Feb 2023
AbstractforStatistical Considerations and Challenges for Pivotal Clinical Studies of Artificial Intelligence Medical Tests for Widespread Use: Opportunities for Inter-Disciplinary Collaboration | Full Text | References | PDF (1.4 MB) | EPUB
  • 350 Views
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Article
A Comparison of Estimand and Estimation Strategies for Clinical Trials in Early Parkinson’s Disease
Alessandro Noci, Marcel Wolbers, Markus Abt, Corine Baayen, Hans Ulrich Burger, Man Jin & Weining Zhao Robieson
Pages: 491-501
Published online: 03 Oct 2022
AbstractforA Comparison of Estimand and Estimation Strategies for Clinical Trials in Early Parkinson’s Disease | Full Text | References | PDF (1 MB) | EPUB | Supplemental
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Article
Comparing Dose Levels to Placebo using a Continuous Outcome in a Small n, Sequential, Multiple Assignment, Randomized trial (snSMART)
Fang Fang, RoyN. Tamura, Thomas M. Braun & Kelley M. Kidwell
Pages: 502-509
Published online: 07 Oct 2022
AbstractforComparing Dose Levels to Placebo using a Continuous Outcome in a Small n, Sequential, Multiple Assignment, Randomized trial (snSMART) | Full Text | References | PDF (1.3 MB) | EPUB | Supplemental
  • 208 Views
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Article
Incorporating Intermediate Endpoint in Two-Stage Design Decision Making
Xiaofei Bai & Qiqi Deng
Pages: 510-518
Published online: 21 Sep 2022
AbstractforIncorporating Intermediate Endpoint in Two-Stage Design Decision Making | Full Text | References | PDF (2 MB) | EPUB
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Article
The Use of Machine Learning in Regulatory Drug Safety Evaluation
Di Zhang, Jaejoon Song, Sai Dharmarajan, Tae Hyun Jung, Hana Lee, Yong Ma, Rongmei Zhang & Mark Levenson
Pages: 519-523
Published online: 23 Sep 2022
AbstractforThe Use of Machine Learning in Regulatory Drug Safety Evaluation | Full Text | References | PDF (630.8 KB) | EPUB
  • 440 Views
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Article
Beyond the Cox Hazard Ratio: A Targeted Learning Approach to Survival Analysis in a Cardiovascular Outcome Trial Application
David Chen, Maya L. Petersen, Helene Charlotte Rytgaard, Randi Grøn, Theis Lange, Søren Rasmussen, Richard E. Pratley, Steven P. Marso, Kajsa Kvist, John Buse & Mark J. van der Laan
Pages: 524-539
Published online: 03 Apr 2023
AbstractforBeyond the Cox Hazard Ratio: A Targeted Learning Approach to Survival Analysis in a Cardiovascular Outcome Trial Application | Full Text | References | PDF (2.5 MB) | EPUB
  • 2701 Views
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Open Access
Article
Power and Sample Size Calculations for the Restricted Mean Time Analysis of Prioritized Composite Endpoints
Lu Mao
Pages: 540-548
Published online: 03 Oct 2022
AbstractforPower and Sample Size Calculations for the Restricted Mean Time Analysis of Prioritized Composite Endpoints | Full Text | References | PDF (1.2 MB) | EPUB | Supplemental
  • 1815 Views
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Open Access
Article
What Can Be Achieved with the Estimand Framework?
Susan Mayo & Yongman Kim
Pages: 549-553
Published online: 02 Mar 2023
AbstractforWhat Can Be Achieved with the Estimand Framework? | Full Text | References | PDF (848.2 KB) | EPUB
  • 605 Views
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Article
Clinical and Statistical Perspectives on the ICH E9(R1) Estimand Framework Implementation
Alexei C. Ionan, Miya Paterniti, Devan V. Mehrotra, John Scott, Bohdana Ratitch, Sylva Collins, Shanti Gomatam, Lei Nie, Kaspar Rufibach & Frank Bretz
Pages: 554-559
Published online: 06 Jul 2022
AbstractforClinical and Statistical Perspectives on the ICH E9(R1) Estimand Framework Implementation | Full Text | References | PDF (900.6 KB) | EPUB
  • 958 Views
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Article
From Logic-Respecting Efficacy Estimands to Logic-Ensuring Analysis Principle for Time-to-Event Endpoint in Randomized Clinical Trials with Subgroups
Yi Liu, Miao Yang, Siyoen Kil, Jiang Li, Shoubhik Mondal, Yue Shentu, Hong Tian, Liwei Wang & Godwin Yung
Pages: 560-573
Published online: 18 Apr 2023
AbstractforFrom Logic-Respecting Efficacy Estimands to Logic-Ensuring Analysis Principle for Time-to-Event Endpoint in Randomized Clinical Trials with Subgroups | Full Text | References | PDF (1.9 MB) | EPUB | Supplemental
  • 228 Views
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Article
Experience with Advisory Committee Meeting Preparation and Execution
Christine Gause & Jing Zhao
Pages: 574-581
Published online: 26 Jul 2023
AbstractforExperience with Advisory Committee Meeting Preparation and Execution | Full Text | References | PDF (1.4 MB) | EPUB
  • 72 Views
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Research Articles

Leveraging Real-World Data in COVID-19 Response
Freda Cooner, Ran Liao, Junjing Lin, Sophie Barthel, Yodit Seifu & Shiling Ruan
Pages: 582-595
Published online: 14 Sep 2022
AbstractforLeveraging Real-World Data in COVID-19 Response | Full Text | References | PDF (1.4 MB) | EPUB
  • 175 Views
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Article
An Alternative to Traditional Sample Size Determination for Small Patient Populations
Holly Jackson & Thomas Jaki
Pages: 596-607
Published online: 21 Sep 2022
AbstractforAn Alternative to Traditional Sample Size Determination for Small Patient Populations | Full Text | References | PDF (2 MB) | EPUB | Supplemental
  • 1842 Views
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Open Access
Article
Testing the Equality of Proportions for Combined Unilateral and Bilateral Data Under Equal Intraclass Correlation Model
Chang-Xing Ma & Huipei Wang
Pages: 608-617
Published online: 23 Sep 2022
AbstractforTesting the Equality of Proportions for Combined Unilateral and Bilateral Data Under Equal Intraclass Correlation Model | Full Text | References | PDF (1.2 MB) | EPUB
  • 94 Views
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Article
Performance of LTMLE in the Presence of Missing Data in Control-Matched Longitudinal Studies
Sue-Jane Wang, Zhipeng Huang & Hai Zhu
Pages: 618-637
Published online: 03 Oct 2022
AbstractforPerformance of LTMLE in the Presence of Missing Data in Control-Matched Longitudinal Studies | Full Text | References | PDF (2.5 MB) | EPUB
  • 158 Views
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Article
Bayesian Simultaneous Credible Intervals for Effect Measures from Multiple Markers
Ashwini Joshi, Terhi Ollila, Ilkka Immonen & Sangita Kulathinal
Pages: 638-653
Published online: 03 Oct 2022
AbstractforBayesian Simultaneous Credible Intervals for Effect Measures from Multiple Markers | Full Text | References | PDF (2.9 MB) | EPUB | Supplemental
  • 1196 Views
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Open Access
Article
Sample Size Calculation and Timing of Dose Selection in a Multiple-Dose Clinical Trial
Liji Shen, Jin Zhang & Paul DeLucca
Pages: 654-660
Published online: 11 Oct 2022
AbstractforSample Size Calculation and Timing of Dose Selection in a Multiple-Dose Clinical Trial | Full Text | References | PDF (813 KB) | EPUB | Supplemental
  • 139 Views
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Article
Assessing Contribution of Treatment Phases through Tipping Point Analyses via Counterfactual Elicitation Using Rank Preserving Structural Failure Time Models
Sudipta Bhattacharya & Jyotirmoy Dey
Pages: 661-674
Published online: 07 Oct 2022
AbstractforAssessing Contribution of Treatment Phases through Tipping Point Analyses via Counterfactual Elicitation Using Rank Preserving Structural Failure Time Models | Full Text | References | PDF (2.3 MB) | EPUB
  • 1270 Views
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Open Access
Article
Probability of Study Success (PrSS) Evaluation Based on Multiple Endpoints in Late Phase Oncology Drug Development
Zhen Zhang, Yong Lin & Jingyi Liu
Pages: 675-688
Published online: 28 Oct 2022
AbstractforProbability of Study Success (PrSS) Evaluation Based on Multiple Endpoints in Late Phase Oncology Drug Development | Full Text | References | PDF (2 MB) | EPUB | Supplemental
  • 226 Views
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Article
Statistical Consideration for Fit-for-Use Real-World Data to Support Regulatory Decision Making in Drug Development
Mark Levenson, Weili He, Li Chen, Sai Dharmarajan, Rima Izem, Zhaoling Meng, Herbert Pang & Frank Rockhold
Pages: 689-696
Published online: 07 Oct 2022
AbstractforStatistical Consideration for Fit-for-Use Real-World Data to Support Regulatory Decision Making in Drug Development | Full Text | References | PDF (916.7 KB) | EPUB
  • 613 Views
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Letter to the Editor

Designing Dose-Optimization Studies in Cancer Drug Development: Discussions with Regulators
Olga Marchenko, Rajeshwari Sridhara, Qi Jiang, Elizabeth Barksdale, Yuki Ando, Dinesh De Alwis, Katie Brown, Laura Fernandes, Mark T. J. van Bussel, Qiuyi Choo, Michael Coory, Elizabeth Garrett-Mayer, Thomas Gwise, Lorenzo Hess, Rong Liu, Sumithra Mandrekar, Daniele Ouellet, José Pinheiro, Martin Posch, Nam Atiqur Rahman, Khadija Rerhou Rantell, Andrew Raven, Sarem Sarem, Suman Sen, Mirat Shah, Yuan Li Shen, Richard Simon, Marc Theoret, Ying Yuan & Richard Pazdur
Pages: 697-703
Published online: 14 Feb 2023
AbstractforDesigning Dose-Optimization Studies in Cancer Drug Development: Discussions with Regulators | Full Text | References | PDF (769.3 KB) | EPUB
  • 558 Views
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