Improving Design, Evaluation and Analysis of early drug development Studies

Livrables

Presentation at scientific events

Each research will have presented at least once at a scientific conference or meeting.

e-learning course 3

"The third e-learning course on ""Multiple Testing"" has been completed by all researchers"

Award of PhDs
e-learning course 1

"The first e-learning course on ""Computational skills in statistics"" has been completed by all researchers"

Release of summary videos
Dissemination workshop
Peer-reviewed publications

The first peer-reviewed publication has been submitted by every researcher

Launch of Website
e-learning course 2

"The second e-learning course on ""Genomics: Technologies and data analyses"" has been completed by all researchers"

Final report
Review papers submitted

At least one paper by each researcher discussing state of the art statistical methods relevant in medical statistics and drug development for a non-statistical audience and has been submitted to a substantive journals.

Release of non-technical summary reports for clinical colleagues

Publications

Sample size for multiple hypothesis testing in biosimilar development

Auteurs: Johanna Mielke, Byron Jones, Bernd Jilma, Franz König
Publié dans: Statistics in Biopharmaceutical Research, 2017, Page(s) 0-0, ISSN 1946-6315
Éditeur: Taylor & Francis
DOI: 10.1080/19466315.2017.1371071

Model selection based on combined penalties for biomarker identification

Auteurs: Eleni Vradi, Werner Brannath, Thomas Jaki, Richardus Vonk
Publié dans: Journal of Biopharmaceutical Statistics, 2017, Page(s) 1-15, ISSN 1054-3406
Éditeur: Marcel Dekker Inc.
DOI: 10.1080/10543406.2017.1378662

Longitudinal assessment of the impact of multiple switches between a biosimilar and its reference product on efficacy parameters

Auteurs: Johanna Mielke, Heike Woehling, Byron Jones
Publié dans: Pharmaceutical Statistics, 2018, ISSN 1539-1604
Éditeur: John Wiley & Sons Inc.
DOI: 10.1002/pst.1849

Subgroup identification in dose-finding trials via model-based recursive partitioning

Auteurs: Marius Thomas, Björn Bornkamp, Heidi Seibold
Publié dans: Statistics in Medicine, 2018, ISSN 0277-6715
Éditeur: John Wiley & Sons Inc.
DOI: 10.1002/sim.7594

A review of the deterministic and diffusion approximations for stochastic chemical reaction networks

Auteurs: Pavel Mozgunov, Marco Beccuti, Andras Horvath, Thomas Jaki, Roberta Sirovich, Enrico Bibbona
Publié dans: Reaction Kinetics, Mechanisms and Catalysis, 2018, ISSN 1878-5190
Éditeur: Akademiai Kiado
DOI: 10.1007/s11144-018-1351-y

Incorporating historical information in biosimilar trials: Challenges and a hybrid Bayesian-frequentist approach

Auteurs: Mielke, Johanna, Schmidli, Heinz, Jones, Byron.
Publié dans: Biometrical Journal, 2018, ISSN 1521-4036
Éditeur: Wiley
DOI: 10.1002/bimj.201700152

Clinical trials for authorized biosimilars in the European Union: a systematic review

Auteurs: Johanna Mielke, Bernd Jilma, Franz Koenig, Byron Jones
Publié dans: British Journal of Clinical Pharmacology, Numéro 82(6), 2016, Page(s) 1444-1457, ISSN 0306-5251
Éditeur: Blackwell Publishing Inc.
DOI: 10.1111/bcp.13076

A proposal for a new PhD level curriculum on quantitative methods for drug development

Auteurs: T. Jaki, A. Gordon, P. Forster, L. Bijnens, B. Bornkamp, W. Brannath, R. Fontana, M. Gasparini, L.V. Hampson, T. Jacobs, B. Jones, X. Paoletti, M. Posch, A. Titman, R. Vonk, F. Koenig
Publié dans: Pharmaceutical Statistics, 2018, ISSN 1539-1604
Éditeur: John Wiley & Sons Inc.
DOI: 10.1002/pst.1873

Interval and point estimation in adaptive Phase II trials with binary endpoint

Auteurs: Arsénio Nhacolo, Werner Brannath
Publié dans: Statistical Methods in Medical Research, 2018, Page(s) 096228021878141, ISSN 0962-2802
Éditeur: SAGE Publications
DOI: 10.1177/0962280218781411

Between-Batch Pharmacokinetic Variability Inflates Type I Error Rate in Conventional Bioequivalence Trials: A Randomized Advair Diskus® Clinical Trial

Auteurs: E. Burmeister Getz, K.J. Carroll, J. Mielke, L.Z. Benet, B. Jones
Publié dans: Clinical Pharmacology & Therapeutics, 2016, ISSN 0009-9236
Éditeur: Nature Publishing Group
DOI: 10.1002/cpt.535

A Bayesian model to estimate the cutoff and the clinical utility of a biomarker assay

Auteurs: Eleni Vradi, Thomas Jaki, Richardus Vonk, Werner Brannath
Publié dans: Statistical Methods in Medical Research, 2018, Page(s) 096228021878477, ISSN 0962-2802
Éditeur: SAGE Publications
DOI: 10.1177/0962280218784778

An information theoretic phase I-II design for molecularly targeted agents that does not require an assumption of monotonicity

Auteurs: Pavel Mozgunov, Thomas Jaki
Publié dans: Journal of the Royal Statistical Society: Series C (Applied Statistics), 2018, Page(s) 68(2):347-367, ISSN 0035-9254
Éditeur: Blackwell Publishing Inc.
DOI: 10.1111/rssc.12293

Generalization of Cramér-Rao and Bhattacharyya inequalities for the weighted covariance matrix

Auteurs: Kelbert, Mark; Mozgunov, Pavel
Publié dans: Mathematical Communications, Numéro 8, 2017, ISSN 1848-8013
Éditeur: University of Osijek

An update on the clinical evidence that supports biosimilar approvals in Europe

Auteurs: Johanna Mielke, Bernd Jilma, Byron Jones, Franz Koenig
Publié dans: British Journal of Clinical Pharmacology, Numéro 84/7, 2018, Page(s) 1415-1431, ISSN 0306-5251
Éditeur: Blackwell Publishing Inc.
DOI: 10.1111/bcp.13586

A benchmark for dose finding studies with continuous outcomes

Auteurs: Pavel Mozgunov, Thomas Jaki, Xavier Paoletti
Publié dans: Biostatistics, 2018, ISSN 1465-4644
Éditeur: Oxford University Press
DOI: 10.1093/biostatistics/kxy045

Bayesian knowledge integration for an in vitro-in vivo correlation model

Auteurs: Elvira M. Erhardt, Moreno Ursino, Jeike Biewenga, Tom Jacobs, Mauro Gasparini
Publié dans: Biometrical Journal, 2018, ISSN 0323-3847
Éditeur: John Wiley & Sons Ltd.
DOI: 10.1002/bimj.201700263

Comparing Approaches to Treatment Effect Estimation for Subgroups in Clinical Trials

Auteurs: Marius Thomas, Björn Bornkamp
Publié dans: Statistics in Biopharmaceutical Research, 2016, Page(s) 0-0, ISSN 1946-6315
Éditeur: Taylor & Francis
DOI: 10.1080/19466315.2016.1251490

Quantifying the association between progression-free survival and overall survival in oncology trials using Kendall's τ

Auteurs: Enya M. Weber, Andrew C. Titman
Publié dans: Statistics in Medicine, 2018, ISSN 0277-6715
Éditeur: John Wiley & Sons Inc.
DOI: 10.1002/sim.8001

Cancer phase I trial design using drug combinations when a fraction of dose limiting toxicities is attributable to one or more agents

Auteurs: Jose L. Jimenez, Mourad Tighiouart, Mauro Gasparini
Publié dans: Biometrical Journal, 2018, ISSN 0323-3847
Éditeur: John Wiley & Sons Ltd.
DOI: 10.1002/bimj.201700166

Subgroup identification in clinical trials via the predicted individual treatment effect

Auteurs: Nicolás M. Ballarini, Gerd K. Rosenkranz, Thomas Jaki, Franz König, Martin Posch
Publié dans: PLOS ONE, Numéro 13/10, 2018, Page(s) e0205971, ISSN 1932-6203
Éditeur: Public Library of Science
DOI: 10.1371/journal.pone.0205971

Randomized dose-escalation designs for drug combination cancer trials with immunotherapy

Auteurs: Pavel Mozgunov, Thomas Jaki, Xavier Paoletti
Publié dans: Journal of Biopharmaceutical Statistics, 2018, Page(s) 1-19, ISSN 1054-3406
Éditeur: Marcel Dekker Inc.
DOI: 10.1080/10543406.2018.1535503

Properties of the weighted log-rank test in the design of confirmatory studies with delayed effects

Auteurs: José L. Jiménez, Viktoriya Stalbovskaya, Byron Jones
Publié dans: Pharmaceutical Statistics, 2019, ISSN 1539-1604
Éditeur: John Wiley & Sons Inc.
DOI: 10.1002/pst.1923

Pharmacological Treatment of Arterial Hypertension in Children and Adolescents

Auteurs: Jacopo Burrello, Elvira M. Erhardt, Gaelle Saint-Hilary, Franco Veglio, Franco Rabbia, Paolo Mulatero, Silvia Monticone, Fabrizio D’Ascenzo
Publié dans: Hypertension, Numéro 72/2, 2018, Page(s) 306-313, ISSN 0194-911X
Éditeur: Lippincott Williams & Wilkins Ltd.
DOI: 10.1161/hypertensionaha.118.10862

A Bayesian K-PD model for synergy: A case study

Auteurs: Fabiola La Gamba, Tom Jacobs, Helena Geys, Luc Ver Donck, Christel Faes
Publié dans: Pharmaceutical Statistics, Numéro 17/6, 2018, Page(s) 674-684, ISSN 1539-1604
Éditeur: John Wiley & Sons Inc.
DOI: 10.1002/pst.1887

The Assessment of Quality Attributes for Biosimilars: a Statistical Perspective on Current Practice and a Proposal

Auteurs: Johanna Mielke, Franz Innerbichler, Martin Schiestl, Nicolas M. Ballarini, Byron Jones
Publié dans: The AAPS Journal, Numéro 21/1, 2019, ISSN 1550-7416
Éditeur: Springer New York
DOI: 10.1208/s12248-018-0275-9

A novel measure of drug benefit–risk assessment based on Scale Loss Score

Auteurs: Gaelle Saint-Hilary, Veronique Robert, Mauro Gasparini, Thomas Jaki, Pavel Mozgunov
Publié dans: Statistical Methods in Medical Research, 2018, Page(s) 096228021878652, ISSN 0962-2802
Éditeur: SAGE Publications
DOI: 10.1177/0962280218786526

Dose-Finding Methods: Moving Away from the 3 + 3 to Include Richer Outcomes

Auteurs: Xavier Paoletti, Damien Drubay, Laurence Collette
Publié dans: Clinical Cancer Research, Numéro 23/15, 2017, Page(s) 3977-3979, ISSN 1078-0432
Éditeur: American Association for Cancer Research
DOI: 10.1158/1078-0432.ccr-17-1306

Comparison of different approaches for dose response analysis

Auteurs: Saswati Saha, Werner Brannath
Publié dans: Biometrical Journal, Numéro 61/1, 2019, Page(s) 83-100, ISSN 0323-3847
Éditeur: John Wiley & Sons Ltd.
DOI: 10.1002/bimj.201700276

A robust Bayesian meta-analytic approach to incorporate animal data into phase I oncology trials

Auteurs: Haiyan Zheng, Lisa V Hampson, Simon Wandel
Publié dans: Statistical Methods in Medical Research, 2019, Page(s) 096228021882004, ISSN 0962-2802
Éditeur: SAGE Publications
DOI: 10.1177/0962280218820040

Repeated measures dose-finding design with time-trend detection in the presence of correlated toxicity data

Auteurs: Jun Yin, Xavier Paoletti, Daniel J Sargent, Sumithra J Mandrekar
Publié dans: Clinical Trials, Numéro 14/6, 2017, Page(s) 611-620, ISSN 1740-7745
Éditeur: SAGE Publications
DOI: 10.1177/1740774517723829

Interim analysis incorporating short- and long-term binary endpoints

Auteurs: Julia Niewczas, Cornelia U. Kunz, Franz König
Publié dans: Biometrical Journal, 2019, ISSN 0323-3847
Éditeur: John Wiley & Sons Ltd.
DOI: 10.1002/bimj.201700281

Loss Functions in Restricted Parameter Spaces and Their Bayesian Applications

Auteurs: Mozgunov P, Jaki T, Gasparini M
Publié dans: Journal of Applied Statistics, 2019, ISSN 0266-4763
Éditeur: Routledge
DOI: 10.1080/02664763.2019.1586848

Bayesian sequential integration within a preclinical PK-PD modeling framework: Lessons learned

Auteurs: Fabiola La Gamba, Tom Jacobs, Helena Geys, Thomas Jaki, Jan Serroyen, Moreno Ursino, Alberto Russu, Christel Faes
Publié dans: Pharmaceutical Statistics, 2019, ISSN 1539-1604
Éditeur: John Wiley & Sons Inc.
DOI: 10.1002/pst.1941

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