Journal of Biopharmaceutical Statistics, Volume 33, Issue 6 (2023)

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Journal of Biopharmaceutical Statistics, Volume 33, Issue 6 (2023)

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Special Issue on Statistics in Pediatric Drug Development

Editorial

Editorial
Pediatric needs should be considered early in the clinical development lifecycle
Margaret Gamalo, Jingjing Ye, YounJeong Choi & Rima Izem
Pages: 677-680
Published online: 08 Aug 2023
First Page PreviewforPediatric needs should be considered early in the clinical development lifecycle | Full Text | References | PDF (247.9 KB) | EPUB
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Free Access

Research Article

Article
Recent Use of Pediatric Extrapolation in Pediatric Drug Development in US
Jingjing Ye, Vickie Zhang, Foteini Strimenopoulou, Yihua Zhao, Haitao Pan, Mayadah Shabbout & Margaret Gamalo
Pages: 681-695
Published online: 07 Feb 2023
AbstractforRecent Use of Pediatric Extrapolation in Pediatric Drug Development in US | Full Text | References | PDF (361.7 KB) | EPUB
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Article
Design of paediatric trials with benefit-risk endpoints using a composite score of adverse events of interest (AEI) and win-statistics
Yodit Seifu, Shahrul Mt-Isa, Kyle Duke, Margaret Gamalo-Siebers, William Wang, Gaohong Dong & John Kolassa
Pages: 696-707
Published online: 22 Dec 2022
AbstractforDesign of paediatric trials with benefit-risk endpoints using a composite score of adverse events of interest (AEI) and win-statistics | Full Text | References | PDF (595.7 KB) | EPUB
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Article
Composite Likelihoods with Bounded Weights in Extrapolation of Data
Margaret Gamalo, Yoonji Kim, Fan Zhang & Junjing Lin
Pages: 708-725
Published online: 20 Jan 2023
AbstractforComposite Likelihoods with Bounded Weights in Extrapolation of Data | Full Text | References | PDF (1.5 MB) | EPUB
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Article
Use of pharmacodynamic modeling for Bayesian information borrowing in pediatric clinical trials
Bernard Sebastien, S. Y. Amy Cheung, Solange Corriol-Rohou, Margaret Gamalo-Siebers, Rana Jreich, Rajesh Krishna & Jing Liu
Pages: 726-736
Published online: 16 Dec 2022
AbstractforUse of pharmacodynamic modeling for Bayesian information borrowing in pediatric clinical trials | Full Text | References | PDF (1.3 MB) | EPUB
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Article
Utility of propensity score-based Bayesian borrowing of external adult data in pediatric trials: A pragmatic evaluation through a case study in acute lymphoblastic leukemia (ALL)
Antara Majumdar, Rebecca Rothwell, Gregory Reaman, Corinne Ahlberg & Pourab Roy
Pages: 737-751
Published online: 04 Jan 2023
AbstractforUtility of propensity score-based Bayesian borrowing of external adult data in pediatric trials: A pragmatic evaluation through a case study in acute lymphoblastic leukemia (ALL) | Full Text | References | PDF (1.4 MB) | EPUB
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Article
Addressing statistical issues when leveraging external control data in pediatric clinical trials using Bayesian dynamic borrowing
Emmanouil Spanakis, Martina Kron, Mareike Bereswill & Saurabh Mukhopadhyay
Pages: 752-769
Published online: 12 Dec 2022
AbstractforAddressing statistical issues when leveraging external control data in pediatric clinical trials using Bayesian dynamic borrowing | Full Text | References | PDF (3.6 MB) | EPUB
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Article
Extent of safety database in pediatric drug development: types of assessment, analytical precision, and pathway for extrapolation through on-target effects
Margaret Gamalo, Yihua Zhao, Aijun Gao, Jingjing Ye, Junjing Lin, Eiji Eshida, YounJeong Choi & Robert Nelson
Pages: 770-785
Published online: 26 Feb 2023
AbstractforExtent of safety database in pediatric drug development: types of assessment, analytical precision, and pathway for extrapolation through on-target effects | Full Text | References | PDF (2.8 MB) | EPUB
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Discussion

Discussion
Analysis and reporting of pediatric growth and development assessment from clinical trials: overview and challenges
YounJeong Choi, Nathalie Barbier, Sabine Fürst-Recktenwald, Jingjing Ye, Dalia Ballas Wajsbrot, Eiji Ishida & Margaret Gamalo
Pages: 786-799
Published online: 21 Dec 2022
AbstractforAnalysis and reporting of pediatric growth and development assessment from clinical trials: overview and challenges | Full Text | References | PDF (893 KB) | EPUB
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Discussion
Advancing drug development in pediatric oncology, a focus on cancer biology and targeted therapies: iMATRIX platform
Marianne Uguen, Magalie Hilton, Mufiza Farid-Kapadia, Asim Datye, Saibah Chohan, Claudia Carlucci, Mark Dixon, Markus Elze, Yingjia Chen, Kit Wun Kathy Cheung, Rucha Sane, Maoxia Zheng & YounJeong Choi
Pages: 800-811
Published online: 13 Jan 2023
AbstractforAdvancing drug development in pediatric oncology, a focus on cancer biology and targeted therapies: iMATRIX platform | Full Text | References | PDF (1.2 MB) | EPUB
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Research Article

Article
Case study using RWD in the context of a pivotal trial for regulatory approval in a rare disease
Paul O’Connell, Antonia Ridolfi & Nathalie Fretault
Pages: 812-819
Published online: 29 Jan 2023
AbstractforCase study using RWD in the context of a pivotal trial for regulatory approval in a rare disease | Full Text | References | PDF (805.7 KB) | EPUB
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Article
RESTART trial design: two-stage seamless transition design with operational considerations
Min Yi, Bin Zhuo & Freda Cooner
Pages: 820-829
Published online: 18 Jan 2023
AbstractforRESTART trial design: two-stage seamless transition design with operational considerations | Full Text | References | PDF (778.6 KB) | EPUB
  • 220 Views
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Article
Perspectives on informative Bayesian methods in pediatrics
James Travis, Mark Rothmann & Andrew Thomson
Pages: 830-843
Published online: 29 Jan 2023
AbstractforPerspectives on informative Bayesian methods in pediatrics | Full Text | References | PDF (821.5 KB) | EPUB
  • 818 Views
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Article
How to select the initial dose for a pediatric study?
Jingjing Ye, Youwei Bi & Naitee Ting
Pages: 844-858
Published online: 08 Dec 2022
AbstractforHow to select the initial dose for a pediatric study? | Full Text | References | PDF (769.6 KB) | EPUB
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Article
Clinical trial considerations for pediatric cancer drug development
Freda Cooner, Jingjing Ye & Gregory Reaman
Pages: 859-874
Published online: 07 Feb 2023
AbstractforClinical trial considerations for pediatric cancer drug development | Full Text | References | PDF (1 MB) | EPUB
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Article
Real world data (RWD) in pediatrics
Tamar Lasky & Aloka Chakravarty
Pages: 875-880
Published online: 15 Dec 2022
AbstractforReal world data (RWD) in pediatrics | Full Text | References | PDF (264 KB) | EPUB
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Back Matter

Referees for Volume 33
Pages: i-v
Published online: 01 Nov 2023
First Page PreviewforReferees for Volume 33 | Full Text | PDF (128 KB) | EPUB
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