Pharmaceutical Statistics

Cover image for Vol. 16 Issue 5

Edited By: David Morgan, Thomas Permutt and Jorgen Seldrup

Impact Factor: 1.276

ISI Journal Citation Reports © Ranking: 2016: 46/124 (Statistics & Probability); 46/124 (STATISTICS & PROBABILITY); 209/256 (Pharmacology & Pharmacy); 210/257 (PHARMACOLOGY & PHARMACY)

Online ISSN: 1539-1612

PSI Journal Club

Statisticians in the Pharmaceutical Industry's (PSI) Journal Club has been running since 2009, providing a medium for authors of selected papers to present their work to both PSI members and non-members. Sponsored by Wiley, the Journal Club are hosted quarterly by 90-minute teleconferences. Two papers are presented by their authors, followed by questions from the audience.

The following is a list of papers that have been presented at the PSI Journal Club. For access to slides, flyers, video and audio recordings, please visit PSI's website.

September 2017 - Clinical Trial Safety Monitoring

Adaptive and repeated cumulative meta-analyses of safety data during a new drug development process
Authors: Hui Quan, Yingqui Ma, Yan Zheng, Meehyung Cho, Christelle Lorenzato, Carole Hecquet

July 2017

Enhancement of the adaptive signature design for learning and confirming in a single pivotal trial
Author: Gu Mi

March 2017

A novel approach to estimation of the time to biomarker threshold: applications to HIV
Authors: Tarylee Reddy, Geert Molenberghs, Edmund Njeru Njagi, Marc Aerts

A visualization method measuring the performance of biomarkers for guiding treatment decisions
Authors: Hui Yang, Rui Tang, Mike Hale, Jing Huang

November 2016 - Analysing Adverse Event Data

Statistical issues in the analysis of adverse events in time-to-event data
Authors: Arthur Allignol, Jan Beyersmann, Claudia Schmoor

Analysing adverse events by time-to-event models: the CLEOPATRA study
Authors: Tanja Proctor and Martin Schumacher

July 2016 - Estimands

Estimands: discussion points from the PSI estimands and sensitivity expert group
Authors: Alan Phillips, Juan Abellan-Andres, Andersen Soren, Frank Bretz, Chrissie Fletcher, Lesley France, Andrew Garrett, Raymond Harris, Magnus Kjaer, Oliver Keene, David Morgan, Michael O'Kelly and James Roger

March 2016 - Poisson/Negative Binomial Modelling

The analysis of incontinence episodes and other count data in patients with overactive bladder by Poisson and negative binomial regression
Authors: R Martina, R Kay, R van Maanen, A Ridder

Sensitivity analyses for partially observed recurrent event data
Authors: Mouna Akacha and Emmanuel O. Ogundimu

December 2015 - Go/NoGo Decision Rules and Program Success

A Novel Design for Decision Rules Based on Statistical Testing Strategies of Binary Endpoints in a Definitive Go/No-Go Single-Treatment Clinical Study
Author: Ming Zhou and Larry Z. Shen

Evaluation of program success for programs with multiple trials in binary outcomes
Author: Meihua Wang, G. Frank Liu and Jerald Schindler

October 2015 - Missing Data

Missing data in clinical trials: from clinical assumptions to statistical analysis using pattern mixture models
Author: Bohdana Ratitch, Michael O'Kelly, Robert Tosiello

Missing data sensitivity analysis for recurrent event data using controlled imputation
Author: Oliver N. Keene, James H. Roger, Benjamin F. Hartley, Michael G. Kenward

September 2015 - Simulation and Hypothesis Testing

How to test hypothese if you must
Author: Andrew Grieve

Influenza vaccine efficacy trials: a simulation approach to understand failures from the past
Author: Anne Benoit, Catherine Legrand and Walthère Dewé

May 2015 - Adaptive Designs

Two-stage adaptive randomization for delayed response in clinical trials
Authors: Jiajing Xu and Guosheng Yin

Adaptive graph-based multiple testing procedures
Authors: Florian Klinglmueller, Martin Posch and Franz Koenig

November 2014 - Safety and HTA Data

Guidance on the implementation and reporting of a drug safety Bayesian network meta-analysis
Authors: Karen L. Price, H. Amy Xia, Hwanhee Hong, Jouni Kerman, Haoda Fu, George Quartey, Cory R. Heilmann, Haijun Ma and Bradley P. Carlin

Journal Club Slides

Subgroup analyses in cost-effectiveness analyses to support health technology assessments
Authors: Christy Chuang-Stein, Marie-Ange Paget, Carol Reid and Neil Hawkins
Journal Club Slides

July 2014 - Hot Topics in Drug Development

Use of historical control data for assessing treatment effects in clinical trials
Authors: Kert Viele, Scott Berry, Beat Neuenschwander, Billy Amzal, Fang Chen, Nathan Enas, Brian Hobbs, Joseph G. Ibrahim, Nelson Kinnersley, Stacy Lindborg, Sandrine Micallef, Satrajit Roychoudhury and Laura Thompson
Journal Club Slides

Preparing individual patient data from clinical trials for sharing: the GlaxoSmithKline approach
Authors: Sara Hughes, Karen Wells, Paul McSorley and Andrew Freeman
Journal Club Slides

April 2014 - Combination Therapies - Joint PSI/RSS Journal Club

A Bayesian dose finding design for oncology clinical trials of combinational biological agents
Authors: Chunyan Cai, Ying Yuan and Yuan Ji
(published in Journal of the Royal Statistical Society: Series C: Applied Statistics)
Journal Club slides

Escalation strategies for combination therapy Phase I trials (OnlineOpen article)
Authors: Michael J. Sweeting and Adrian P. Mander
Journal Club slides

December 2013 - Extreme value modelling

Extreme value modelling of laboratory safety data from clinical studies
Authors: Harry Southworth and Janet E. Heffernan
Journal Club slides

Multivariate extreme value modelling of laboratory safety data from clinical studies
Authors: Harry Southworth and Janet E. Heffernan

October 2013 - Sample size re-estimation techniques - Joint PSI/DIA Journal Club

Adaptive blinded sample size adjustment for comparing two normal means - a mostly Bayesian approach
Authors: Andrew M. Hartley
Journal Club slides

August 2013 - Papers chosen by you!

A structured framework for assessing sensitivity to missing data assumptions in longitudinal clinical trials
Authors: Craig H. Mallinckrodt, Q. Lin and M. Molenberghs
Journal Club slides

On the practical application of mixed effects models for repeated measures to clinical trial data
Authors: Scott W. Andersen and Brian A. Millen
Journal Club slides

April 2013 - Survival analysis - PSI/RSS Journal Club

Evaluating joint effects of induction–salvage treatment regimes on overall survival in acute leukaemia
Authors: Abdus S. Wahed, Peter F. Thalli
Journal Club slides

Attenuation of treatment effect due to measurement variability in assessment of progression-free survival
Authors: Nicola Schmitt, S. Hong, A. Stone, J. Denne
Journal Club slides

December 2012 - Modelling in drug development

Application of optimal design methodologies in clinical pharmacology experiments
Authors: Kayode Ogungbenro, Aristides Dokoumetzidis and Leon Aarons
Journal Club slides

Discounting phase 2 results when planning phase 3 clinical trials
Authors: Simon Kirby, J. Burke, C. Chuang-Stein and C. Sin
Journal Club slides

September 2012 - Special Edition on PSI's Special Interest Groups (SIGs)

Toxicology SIG
An assessment of the statistical methods used to analyse toxicology studies

Authors: Helena Geys, Philip Jarvis, Jim Saul, Mike Aylott, Simon Bate and John Sherington
Journal Club slides

Biomarker SIG
A statistician's perspective on biomarkers in drug development

Authors: Martin Jenkins, Chris Harbron, Aiden Flynn, Trevor Smart, Tony Sabin, Jayantha Ratnayake, Paul Delmar, Athula Herath, Philip Jarvis and James Matcham
Journal Club slides

June 2012 - Decision making at intermins and DMCs - Joint PSI/DIA Journal Club

Informing the selection of futility stopping thresholds: case study from a late-phase clinical trial
Authors: Sara Hughes, Robert L. Cuffe, Alfons Lieftucht and W. Garrett Nichols
Journal Club slides

May 2012 - PSI Annual Conference - Plenary session on proposed best practice for statisticians in the reporting and publication of pharmaceutical industry- supported clinical trials

Proposed best practice for statisticians in the reporting and publication of pharmaceutical industry-sponsored clinical trials
Authors: James Matcham, Steven Julious, Stephen Pyke, Michael O'Kelly, Susan Todd, Jorgen Seldrup and Simon Day
Journal Club slides

April 2012 - Meta-analysis in clinical trials

Investigating the assumption of homogeneity of treatment effects in clinical studies with application to meta-analysis
Authors: Steven Julious and Anne Whitehead
Journal Club slides

Reporting cumulative proportion of subjects with an adverse event based on data from multiple studies
Authors: Christy Chuang-Stein and Mohan Beltangady
Journal Club slides

December 2011 - Discussion on publications from the pharmaceutical industry

Making available information from studies sponsored by the pharmaceutical industry: some current practices
Authors: Michael O'Kelly, Steven A. Julious, Stephen Pyke, Simon Day, Sue Todd, Jorgen Seldrup and James Matcham
Journal Club slides

Proposed best practice for statisticians in the reporting and publication of pharmaceutical industry-sponsored clinical trials
Authors: James Matcham, Steven Julious, Stephen Pyke, Michael O'Kelly, Susan Todd, Jorgen Seldrup and Simon Day
Journal Club slides

The potential for bias in reporting of industry-sponsored clinical trials
Authors: Stephen Pyke, Steven A. Julious, Simon Day, Michael O'Kelly, Susan Todd, James Matcham and Jorgen Seldrup

July 2011 - Early phase clinical trials

Sequential design approaches for bioequivalence studies with crossover designs
Authors: Diane Potvin, Charles E. DiLiberti, Walter W. Hauck, Alan F. Parr, Donald J. Schuirmann and Robert A. Smith
Journal Club slides

Planning a Bayesian early-phase phase I/II study for human vaccines in HER2 carcinomas
Authors: Sarah Zohar, Ileana Baldi, Guido Forni, Franco Merletti, Giuseppe Masucci and Dario Gregori
Journal Club slides

April 2011 - Multiregional trials - Joint PSI/DIA Journals Club

Consideration of regional differences in design and analysis of multi-regional trials
Authors: James Hung, Sue-Jane Wang, Robert O'Neill
Journal Club slides

October 2010 - Survival Analysis

On assessing the presence of evaluation-time bias in progression-free survival in randomized trials
Authors: Richard Kay, Jane Wu and Janet Wittes
Journal Club slides

Weibull prediction of event times in clinical trials
Author: Gui-shuang Ying and Daniel F. Heitjan
Journal Club slides

June 2010 - Missing data (discussion points from PSI expert group)/Modelling dropouts in count data

Missing Data: Discussion points from the PSI Missing Data Expert Group
Authors: Alan Phillip, Tomasz Burzykowski, James Carpenter, Corneel Coens, Daniel Evans, Lesley France, Mike Kenward, Peter Lane, James Matcham, David Morgan, James Roger, Brian Sullivan, Ian White, and Ly-Mee Yu, of the PSI Missing Data Expert Group
Journal Club slides

Modeling longitudinal count data with dropouts
Author: Mohamed Alosh
Journal Club slides

March 2010 - Bayesian Analysis

The rheumatoid arthritis drug development model: a case study in Bayesian clinical trial simulation
Authors: Richard M. Nixon, Anthony O'Hagan, Jeremy Oakley, Jason Madan, John W. Stevens, Nick Bansback, and Alan Brennan
Journal Club slides

Bayesian fitting of a logistic dose-response curve with numerically derived priors
Authors: Les W. Huson and N. Kinnersley
Journal Club slides

December 2009 - The first PSI Journal Club

Action following the discovery of a global association between the whole genome and adverse event risk in a clinical drug-development programme
Authors: John Whitehead, Patrick Kelly, Yinghui Zhou, Nigel Stallard, Helene Thygesen and Clive Bowman
Journal Club slides

Back to basics: explaining sample size in outcome trials, are statisticians doing a thorough job?
Author: Kevin J. Carroll
Journal Club slides