Statistics in Biopharmaceutical Research, Volume 15, Issue 2 (2023)

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Statistics in Biopharmaceutical Research, Volume 15, Issue 2 (2023)

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Announcement

Announcement
Statistics in Biopharmaceutical Research Best Papers Award 2023
Toshimitsu Hamasaki & Freda Cooner
Pages: 235-236
Published online: 22 May 2023
CitationforStatistics in Biopharmaceutical Research Best Papers Award 2023 | Full Text | References | PDF (395 KB) | EPUB
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Free Access

Special Section: Estimands, Design and Analysis of Clinical Trials with Time-to-Event Outcomes

Editorial
Editor’s Note: Special Section on Estimands, Design and Analysis of Clinical Trials with Time-to-Event Outcomes
Toshimitsu Hamasaki
Page: 237
Published online: 22 May 2023
CitationforEditor’s Note: Special Section on Estimands, Design and Analysis of Clinical Trials with Time-to-Event Outcomes | Full Text | References | PDF (321.1 KB) | EPUB
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Free Access
Article
Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event
Heinz Schmidli, James H. Roger & Mouna Akacha
Pages: 238-248
Published online: 19 Apr 2021
AbstractforEstimands for Recurrent Event Endpoints in the Presence of a Terminal Event | Full Text | References | PDF (1 MB) | EPUB
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Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”
H. M. James Hung & Sue-Jane Wang
Pages: 249-250
Published online: 24 Feb 2022
CitationforComment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event” | Full Text | References | PDF (282.6 KB) | EPUB
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We Need Subject Matter Expertise to Choose and Identify Causal Estimands: Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”
Matias Janvin, Jessica G. Young & Mats J. Stensrud
Pages: 251-254
Published online: 22 May 2023
AbstractforWe Need Subject Matter Expertise to Choose and Identify Causal Estimands: Comment on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event” | Full Text | References | PDF (542 KB) | EPUB
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Open Access
Rejoinder to Commentaries on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event”
Heinz Schmidli, James H. Roger, Mouna Akacha & on behalf of the Recurrent Event Qualification Opinion Consortium
Pages: 255-256
Published online: 22 May 2023
CitationforRejoinder to Commentaries on “Estimands for Recurrent Event Endpoints in the Presence of a Terminal Event” | Full Text | References | PDF (509.8 KB) | EPUB
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Article
Properties of Two While-Alive Estimands for Recurrent Events and Their Potential Estimators
Jiawei Wei, Tobias Mütze, Antje Jahn-Eimermacher & James Roger
Pages: 257-267
Published online: 29 Nov 2021
AbstractforProperties of Two While-Alive Estimands for Recurrent Events and Their Potential Estimators | Full Text | References | PDF (1.6 MB) | EPUB | Supplemental
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Article
Efficiency Comparison of Analysis Methods for Recurrent Event and Time-to-First Event Endpoints in the Presence of Terminal Events—Application to Clinical Trials in Chronic Heart Failure
Arno Fritsch, Patrick Schlömer, Franco Mendolia, Tobias Mütze, Antje Jahn-Eimermacher & on behalf of the Recurrent Event Qualification Opinion Consortium
Pages: 268-279
Published online: 04 Aug 2021
AbstractforEfficiency Comparison of Analysis Methods for Recurrent Event and Time-to-First Event Endpoints in the Presence of Terminal Events—Application to Clinical Trials in Chronic Heart Failure | Full Text | References | PDF (1.8 MB) | EPUB | Supplemental
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Article
Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group
Satrajit Roychoudhury, Keaven M Anderson, Jiabu Ye & Pralay Mukhopadhyay
Pages: 280-294
Published online: 04 Mar 2021
AbstractforRobust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group | Full Text | References | PDF (1.9 MB) | EPUB | Supplemental
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The Strong Null Hypothesis and the MaxCombo Test: Comment on “Robust Design and Analysis of Clinical Trials with Nonproportional Hazards: A Straw Man Guidance form a Cross-Pharma Working Group.”
Dominic Magirr & Carl-Fredrik Burman
Pages: 295-296
Published online: 06 Jul 2021
CitationforThe Strong Null Hypothesis and the MaxCombo Test: Comment on “Robust Design and Analysis of Clinical Trials with Nonproportional Hazards: A Straw Man Guidance form a Cross-Pharma Working Group.” | Full Text | References | PDF (427.7 KB) | EPUB
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Comment on “Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group”: The Test Statistic Should Estimate Some Reasonable Measure of Treatment Benefit
Steven Snapinn, Qi Jiang & Chunlei Ke
Pages: 297-299
Published online: 06 Jul 2021
First Page PreviewforComment on “Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group”: The Test Statistic Should Estimate Some Reasonable Measure of Treatment Benefit | Full Text | References | PDF (444.4 KB) | EPUB
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Discussion
Nonproportional Hazards—An Evaluation of the MaxCombo Test in Cancer Clinical Trials
Yuan-Li Shen, Xin Wang, Mushti Sirisha, Flora Mulkey, Jiaxi Zhou, Xin Gao, Lijun Zhang, Thomas Gwise, Shenghui Tang, Marc Theoret, Richard Pazdur & Rajeshwari Sridhara
Pages: 300-309
Published online: 04 Jan 2022
AbstractforNonproportional Hazards—An Evaluation of the MaxCombo Test in Cancer Clinical Trials | Full Text | References | PDF (1.7 MB) | EPUB
  • 971 Views
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Testing and Interpreting the “Right” Hypothesis—Comment on “Non-proportional Hazards — An Evaluation of the MaxCombo Test in Cancer Clinical Trials”
Martin Posch, Robin Ristl & Franz König
Pages: 310-311
Published online: 10 Aug 2022
CitationforTesting and Interpreting the “Right” Hypothesis—Comment on “Non-proportional Hazards — An Evaluation of the MaxCombo Test in Cancer Clinical Trials” | Full Text | References | PDF (300.1 KB) | EPUB
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Comment on “Non-Proportional Hazards – an Evaluation of the MaxCombo Test in Cancer Clinical Trials” by the Cross-Pharma Non-Proportional Hazards Working Group
Ray S. Lin, Pralay Mukhopadhyay, Satrajit Roychoudhury, Keaven M. Anderson, Tianle Hu, Bo Huang, Larry F. Leon, Jason J. Z. Liao, Ji Lin, Rong Liu, Xiaodong Luo, Yabing Mai, Rui Qin, Kay Tatsuoka, Yang Wang, Jiabu Ye, Jian Zhu, Tai-Tsang Chen, Renee Iacona & Cross-Pharma Non-proportional Hazards Working Group
Pages: 312-314
Published online: 22 May 2023
First Page PreviewforComment on “Non-Proportional Hazards – an Evaluation of the MaxCombo Test in Cancer Clinical Trials” by the Cross-Pharma Non-Proportional Hazards Working Group | Full Text | References | PDF (420.5 KB) | EPUB
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Review Article
Rejoinder to Comments on “Non-Proportional Hazards – An Evaluation of the MaxCombo Test in Cancer Clinical Trials”
Yuan-Li Shen, Sirisha Mushti, Flora Mulkey, Thomas Gwise, Xin Wang, Jiaxi Zhou, Xin Gao, Shenghui Tang, Marc R. Theoret, Richard Pazdur & Rajeshwari Sridhara
Pages: 315-317
Published online: 22 May 2023
First Page PreviewforRejoinder to Comments on “Non-Proportional Hazards – An Evaluation of the MaxCombo Test in Cancer Clinical Trials” | Full Text | References | PDF (368.7 KB) | EPUB
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Discussion
Composite Endpoints in Cardio-Renal Clinical Outcome Trials
H. M. James Hung & John Lawrence
Pages: 318-322
Published online: 26 Jul 2021
AbstractforComposite Endpoints in Cardio-Renal Clinical Outcome Trials | Full Text | References | PDF (523 KB) | EPUB
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Article
The Choice of a Randomization Procedure in Survival Studies with Nonproportional Hazards
Marcia Viviane Rückbeil, Martin Manolov & Ralf-Dieter Hilgers
Pages: 323-331
Published online: 17 Aug 2021
AbstractforThe Choice of a Randomization Procedure in Survival Studies with Nonproportional Hazards | Full Text | References | PDF (1.4 MB) | EPUB | Supplemental
  • 895 Views
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Open Access
Article
Combined Tests Based on Restricted Mean Time Lost for Competing Risks Data
Jingjing Lyu, Yawen Hou & Zheng Chen
Pages: 332-339
Published online: 29 Nov 2021
AbstractforCombined Tests Based on Restricted Mean Time Lost for Competing Risks Data | Full Text | References | PDF (1.4 MB) | EPUB | Supplemental
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Letter
Closed-Form Approximation of Correlation Matrix Among Fleming Harrington Test Statistics in MaxCombo Test: Comments on “Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group”
Madan G. Kundu
Pages: 340-342
Published online: 27 Sep 2021
AbstractforClosed-Form Approximation of Correlation Matrix Among Fleming Harrington Test Statistics in MaxCombo Test: Comments on “Robust Design and Analysis of Clinical Trials With Nonproportional Hazards: A Straw Man Guidance From a Cross-Pharma Working Group” | Full Text | References | PDF (378.8 KB) | EPUB
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Research Articles

Discussion
Survey and Recommendations on the Use of P-Values Driving Decisions in Nonclinical Pharmaceutical Applications
Stan Altan, Dhammika Amaratunga, Javier Cabrera, Jeonifer Garren, Helena Geys, John Kolassa, David LeBlond, Dingzhou Li, Jason Liao, Jia Liu, Mariusz Lubomirski, Guillermo Miro-Quesada, Steven Novick, Martin Otava, John Peterson, Katharina Reckermann, Tim Schofield, Charles Tan, Kanaka Tatikola, Fetene Tekle, Jennifer Thomas & Kim Vukovinsky
Pages: 343-358
Published online: 21 Mar 2022
First Page PreviewforSurvey and Recommendations on the Use of P-Values Driving Decisions in Nonclinical Pharmaceutical Applications | Full Text | References | PDF (1.7 MB) | EPUB
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Article
Estimands for Continuous Longitudinal Outcomes in the Presence of Treatment Discontinuation—A Simulation Study in Hyperkalemia Treatments
Masaru Tsuchikawa & Kentaro Sakamaki
Pages: 359-371
Published online: 14 Apr 2022
AbstractforEstimands for Continuous Longitudinal Outcomes in the Presence of Treatment Discontinuation—A Simulation Study in Hyperkalemia Treatments | Full Text | References | PDF (1.6 MB) | EPUB | Supplemental
  • 302 Views
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Article
Quantifying Replicability and Consistency in Systematic Reviews
Iman Jaljuli, Yoav Benjamini, Liat Shenhav, Orestis A. Panagiotou & Ruth Heller
Pages: 372-385
Published online: 22 Apr 2022
AbstractforQuantifying Replicability and Consistency in Systematic Reviews | Full Text | References | PDF (4 MB) | EPUB | Supplemental
  • 333 Views
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Article
A Simulation Study on the Estimation of the Effect in the Hypothetical Scenario of No Use of Symptomatic Treatment in Trials for Disease-Modifying Agents for Alzheimer’s Disease
Florian Lasch, Lorenzo Guizzaro, Frank Pétavy & Ciro Gallo
Pages: 386-399
Published online: 29 Apr 2022
AbstractforA Simulation Study on the Estimation of the Effect in the Hypothetical Scenario of No Use of Symptomatic Treatment in Trials for Disease-Modifying Agents for Alzheimer’s Disease | Full Text | References | PDF (2 MB) | EPUB | Supplemental
  • 4008 Views
  • 2 CrossRef citations
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Open Access
Article
Assessing the Impacts of Cluster Effects and Covariate Imbalance in Cluster Randomized Equivalence Trials
Joseph Ficek, Henian Chen, Yuanyuan Lu, Yangxin Huang & John M. Mayer
Pages: 400-407
Published online: 06 Jun 2022
AbstractforAssessing the Impacts of Cluster Effects and Covariate Imbalance in Cluster Randomized Equivalence Trials | Full Text | References | PDF (1.3 MB) | EPUB | Supplemental
  • 155 Views
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Article
Combining Real-World and Randomized Control Trial Data Using Data-Adaptive Weighting via the On-Trial Score
Joanna Harton, Brian Segal, Ronac Mamtani, Nandita Mitra & Rebecca A. Hubbard
Pages: 408-420
Published online: 24 May 2022
AbstractforCombining Real-World and Randomized Control Trial Data Using Data-Adaptive Weighting via the On-Trial Score | Full Text | References | PDF (2.5 MB) | EPUB | Supplemental
  • 326 Views
  • 1 CrossRef citations
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Article
Hypothetical Estimands in Clinical Trials: A Unification of Causal Inference and Missing Data Methods
Camila Olarte Parra, Rhian M. Daniel & Jonathan W. Bartlett
Pages: 421-432
Published online: 06 Jul 2022
AbstractforHypothetical Estimands in Clinical Trials: A Unification of Causal Inference and Missing Data Methods | Full Text | References | PDF (1.4 MB) | EPUB | Supplemental
  • 6300 Views
  • 3 CrossRef citations
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Open Access
Article
On Variance Estimation for the One-Sample Log-Rank Test
Moritz Fabian Danzer, Andreas Faldum & Rene Schmidt
Pages: 433-443
Published online: 18 Jul 2022
AbstractforOn Variance Estimation for the One-Sample Log-Rank Test | Full Text | References | PDF (1.2 MB) | EPUB | Supplemental
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Letters to the Editor

Cancer Clinical Trials beyond Pandemic: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion
Rajeshwari Sridhara, Elizabeth Barksdale, Olga Marchenko, Qi Jiang, Yuki Ando, Erick Bloomquist, Michael Coory, Melissa Crouse, Evgeny Degtyarev, Theodor Framke, Boris Freidlin, David E. Gerber, Thomas Gwise, Filip Josephson, Lorenzo Hess, Paul Kluetz, Daniel Li, Sumithra Mandrekar, Martin Posch, Khadija Rantell, Bohdana Ratitch, Andrew Raven, Kit Roes, Kaspar Rufibach, Sinan B. Sarac, Richard Simon, Harpreet Singh, Marc Theoret, Andrew Thomson, Emmanuel Zuber, Yuan Li Shen & Richard Pazdur
Pages: 444-449
Published online: 22 Sep 2022
AbstractforCancer Clinical Trials beyond Pandemic: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion | Full Text | References | PDF (593.1 KB) | EPUB
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Evaluation of Treatment Effect in Underrepresented Population in Cancer Trials: Discussion with International Regulators
Rajeshwari Sridhara, Olga Marchenko, Qi Jiang, Elizabeth Barksdale, Jie Chen, Nancy Dreyer, Lola Fashoyin-Aje, Elizabeth Garrett-Mayer, Nicole Gormley, Thomas Gwise, Lorenzo Hess, Sumithra Mandrekar, Francesco Pignatti, Khadija Rantell, Andrew Raven, Yuan-Li Shen, Harpreet Singh, Craig L. Tendler, Marc Theoret & Richard Pazdur
Pages: 450-456
Published online: 31 Oct 2022
AbstractforEvaluation of Treatment Effect in Underrepresented Population in Cancer Trials: Discussion with International Regulators | Full Text | References | PDF (675.8 KB) | EPUB
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