Improving Design, Evaluation and Analysis of early drug development Studies

Deliverables

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

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

Model selection based on combined penalties for biomarker identification

Author(s): Eleni Vradi, Werner Brannath, Thomas Jaki, Richardus Vonk
Published in: Journal of Biopharmaceutical Statistics, 2017, Page(s) 1-15, ISSN 1054-3406
Publisher: 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

Author(s): Johanna Mielke, Heike Woehling, Byron Jones
Published in: Pharmaceutical Statistics, 2018, ISSN 1539-1604
Publisher: John Wiley & Sons Inc.
DOI: 10.1002/pst.1849

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

Author(s): Marius Thomas, Björn Bornkamp, Heidi Seibold
Published in: Statistics in Medicine, 2018, ISSN 0277-6715
Publisher: John Wiley & Sons Inc.
DOI: 10.1002/sim.7594

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

Author(s): Pavel Mozgunov, Marco Beccuti, Andras Horvath, Thomas Jaki, Roberta Sirovich, Enrico Bibbona
Published in: Reaction Kinetics, Mechanisms and Catalysis, 2018, ISSN 1878-5190
Publisher: Akademiai Kiado
DOI: 10.1007/s11144-018-1351-y

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

Author(s): Mielke, Johanna, Schmidli, Heinz, Jones, Byron.
Published in: Biometrical Journal, 2018, ISSN 1521-4036
Publisher: Wiley
DOI: 10.1002/bimj.201700152

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

Author(s): Johanna Mielke, Bernd Jilma, Franz Koenig, Byron Jones
Published in: British Journal of Clinical Pharmacology, Issue 82(6), 2016, Page(s) 1444-1457, ISSN 0306-5251
Publisher: Blackwell Publishing Inc.
DOI: 10.1111/bcp.13076

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

Author(s): 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
Published in: Pharmaceutical Statistics, 2018, ISSN 1539-1604
Publisher: John Wiley & Sons Inc.
DOI: 10.1002/pst.1873

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

Author(s): Arsénio Nhacolo, Werner Brannath
Published in: Statistical Methods in Medical Research, 2018, Page(s) 096228021878141, ISSN 0962-2802
Publisher: 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

Author(s): E. Burmeister Getz, K.J. Carroll, J. Mielke, L.Z. Benet, B. Jones
Published in: Clinical Pharmacology & Therapeutics, 2016, ISSN 0009-9236
Publisher: Nature Publishing Group
DOI: 10.1002/cpt.535

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

Author(s): Eleni Vradi, Thomas Jaki, Richardus Vonk, Werner Brannath
Published in: Statistical Methods in Medical Research, 2018, Page(s) 096228021878477, ISSN 0962-2802
Publisher: 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

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

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

Author(s): Kelbert, Mark; Mozgunov, Pavel
Published in: Mathematical Communications, Issue 8, 2017, ISSN 1848-8013
Publisher: University of Osijek

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

Author(s): Johanna Mielke, Bernd Jilma, Byron Jones, Franz Koenig
Published in: British Journal of Clinical Pharmacology, Issue 84/7, 2018, Page(s) 1415-1431, ISSN 0306-5251
Publisher: Blackwell Publishing Inc.
DOI: 10.1111/bcp.13586

A benchmark for dose finding studies with continuous outcomes

Author(s): Pavel Mozgunov, Thomas Jaki, Xavier Paoletti
Published in: Biostatistics, 2018, ISSN 1465-4644
Publisher: Oxford University Press
DOI: 10.1093/biostatistics/kxy045

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

Author(s): Elvira M. Erhardt, Moreno Ursino, Jeike Biewenga, Tom Jacobs, Mauro Gasparini
Published in: Biometrical Journal, 2018, ISSN 0323-3847
Publisher: John Wiley & Sons Ltd.
DOI: 10.1002/bimj.201700263

Comparing Approaches to Treatment Effect Estimation for Subgroups in Clinical Trials

Author(s): Marius Thomas, Björn Bornkamp
Published in: Statistics in Biopharmaceutical Research, 2016, Page(s) 0-0, ISSN 1946-6315
Publisher: 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

Author(s): Enya M. Weber, Andrew C. Titman
Published in: Statistics in Medicine, 2018, ISSN 0277-6715
Publisher: 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

Author(s): Jose L. Jimenez, Mourad Tighiouart, Mauro Gasparini
Published in: Biometrical Journal, 2018, ISSN 0323-3847
Publisher: John Wiley & Sons Ltd.
DOI: 10.1002/bimj.201700166

Subgroup identification in clinical trials via the predicted individual treatment effect

Author(s): Nicolás M. Ballarini, Gerd K. Rosenkranz, Thomas Jaki, Franz König, Martin Posch
Published in: PLOS ONE, Issue 13/10, 2018, Page(s) e0205971, ISSN 1932-6203
Publisher: Public Library of Science
DOI: 10.1371/journal.pone.0205971

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

Author(s): Pavel Mozgunov, Thomas Jaki, Xavier Paoletti
Published in: Journal of Biopharmaceutical Statistics, 2018, Page(s) 1-19, ISSN 1054-3406
Publisher: 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

Author(s): José L. Jiménez, Viktoriya Stalbovskaya, Byron Jones
Published in: Pharmaceutical Statistics, 2019, ISSN 1539-1604
Publisher: John Wiley & Sons Inc.
DOI: 10.1002/pst.1923

Pharmacological Treatment of Arterial Hypertension in Children and Adolescents

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

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

Author(s): Fabiola La Gamba, Tom Jacobs, Helena Geys, Luc Ver Donck, Christel Faes
Published in: Pharmaceutical Statistics, Issue 17/6, 2018, Page(s) 674-684, ISSN 1539-1604
Publisher: 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

Author(s): Johanna Mielke, Franz Innerbichler, Martin Schiestl, Nicolas M. Ballarini, Byron Jones
Published in: The AAPS Journal, Issue 21/1, 2019, ISSN 1550-7416
Publisher: Springer New York
DOI: 10.1208/s12248-018-0275-9

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

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

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

Author(s): Xavier Paoletti, Damien Drubay, Laurence Collette
Published in: Clinical Cancer Research, Issue 23/15, 2017, Page(s) 3977-3979, ISSN 1078-0432
Publisher: American Association for Cancer Research
DOI: 10.1158/1078-0432.ccr-17-1306

Comparison of different approaches for dose response analysis

Author(s): Saswati Saha, Werner Brannath
Published in: Biometrical Journal, Issue 61/1, 2019, Page(s) 83-100, ISSN 0323-3847
Publisher: John Wiley & Sons Ltd.
DOI: 10.1002/bimj.201700276

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

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

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

Author(s): Jun Yin, Xavier Paoletti, Daniel J Sargent, Sumithra J Mandrekar
Published in: Clinical Trials, Issue 14/6, 2017, Page(s) 611-620, ISSN 1740-7745
Publisher: SAGE Publications
DOI: 10.1177/1740774517723829

Interim analysis incorporating short- and long-term binary endpoints

Author(s): Julia Niewczas, Cornelia U. Kunz, Franz König
Published in: Biometrical Journal, 2019, ISSN 0323-3847
Publisher: John Wiley & Sons Ltd.
DOI: 10.1002/bimj.201700281

Loss Functions in Restricted Parameter Spaces and Their Bayesian Applications

Author(s): Mozgunov P, Jaki T, Gasparini M
Published in: Journal of Applied Statistics, 2019, ISSN 0266-4763
Publisher: Routledge
DOI: 10.1080/02664763.2019.1586848

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

Author(s): Fabiola La Gamba, Tom Jacobs, Helena Geys, Thomas Jaki, Jan Serroyen, Moreno Ursino, Alberto Russu, Christel Faes
Published in: Pharmaceutical Statistics, 2019, ISSN 1539-1604
Publisher: John Wiley & Sons Inc.
DOI: 10.1002/pst.1941

Deliverables

Publications

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