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25 June 2025

The iRISE (improving Reproducibility In SciencE) project aims to deepen our understanding of the drivers contributing to poor reproducibility and to conduct a detailed evaluation – including primary research – of the effectiveness of interventions to increase reproducibility.

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The iRISE (improving Reproducibility In SciencE) project aims to deepen our understanding of the drivers contributing to poor reproducibility and to conduct a detailed evaluation – including primary research – of the effectiveness of interventions to increase reproducibility. The project’s work package (WP) on theory is dedicated to clarifying terms and underlying concepts related to reproducibility. To ensure efficient communication within the iRISE consortium, we produced a glossary with working definitions of terms including reproducibility, replicability, and replication.  A concept important in pre-clinical drug discovery, translatability, was for example defined as "the ability to apply research discoveries from experimental models to applications that directly benefit humans".  The definitions are complemented with measures to quantify different types of reproducibility, and theories on the possible factors leading to irreproducibility.

To view the presented slides, please click here.

11 June 2025

This month it’s all about improving an existing plot from a recent publication on a hyperkalemia trial. Bodo Kirsch is leading the panel discussion on dos and don’ts of effective visualisations. All visualisations are available on the Wonderful Wednesday blog.

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The discussion touched various issues in scientific data visualisation, like messaging, axis limits, background grids and shading, interpolation, choice of legend, reference lines and explanatory text. This was followed by a demonstration of instant patient level data simulation using AI. The next challenge is to include these patient level data into the graphical display of the hyperkalemia trial results. See the Wonderful Wednesday homepage for more detail.

Wonderful Wednesdays are brought to you by the Visualisation SIG. The Wonderful Wednesday team includes Bodo Kirsch, Zachary Skrivanek, Lorenz Uhlmann, Steve Mallett, Rhys Warham, Mark Baillie, Paolo Eusebi, Martin Brown, Benjamin Lang, Lovemore Gakava and Aditeya Pandey

11 June 2025

EU HTA - Communicating Statistics & Uncertainty - The Case of Health Technology Assessment - Lara Wolfson Help your non-statistician audience make sense of risk - Maricarmen Climente Communicating statistical concepts to an HTA Audience - Nick Latimer

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EU HTA - Communicating Statistics & Uncertainty - The Case of Health Technology Assessment - Lara Wolfson
Help your non-statistician audience make sense of risk - Maricarmen Climente
Communicating statistical concepts to an HTA Audience - Nick Latimer

11 June 2025

Chair: Kate Taylor How to be wrong (T006) - Simon Cleall Stepping into leadership: How will I manage? (T002) - Catherine Dixon Enhancing Cross-functional Partnership in Early Oncology Clinical Development: A Practical Guide for Biostatisticians (T008) - Laura Barker Trust actually: Building teams that love to work together (T007) - Zainab Walsh Building High-Performing Teams: Leadership Strategies for Navigating Change and Driving Growth(T010) - Aga Rasinska Trust: The Backbone of Leadership (T005) - Alun Bedding

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Chair: Kate Taylor
How to be wrong (T006) - Simon Cleall
Stepping into leadership: How will I manage? (T002) - Catherine Dixon
Enhancing Cross-functional Partnership in Early Oncology Clinical Development: A Practical Guide for Biostatisticians (T008) - Laura Barker
Trust actually: Building teams that love to work together (T007) - Zainab Walsh

Building High-Performing Teams: Leadership Strategies for Navigating Change and Driving Growth(T010) - Aga Rasinska
Trust: The Backbone of Leadership (T005) - Alun Bedding

11 June 2025

Chair: Jyoti Soni Steps in using healthcare systems data as outcome data in clinical trials (O024) - Sharon Love Why Accurate Time to response prediction matters? (O026) - Donia Skanji Survival of the Fittest: Digitising Survival Data for Enhanced Decision-Making in Clinical Trials (O044) - James Sykes and Nelson Kinnersley

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Chair: Jyoti Soni
Steps in using healthcare systems data as outcome data in clinical trials (O024) - Sharon Love
Why Accurate Time to response prediction matters? (O026) - Donia Skanji
Survival of the Fittest: Digitising Survival Data for Enhanced Decision-Making in Clinical Trials (O044) - James Sykes and Nelson Kinnersley

11 June 2025

Chair: Julia Saperia Unexpected results and challenges when using mixture priors for Bayesian borrowing (O031) - Darren Scott Non-monotonic power in Bayesian dynamic borrowing: insights and practical remedies (O035) - Gianmarco Caruso Biased borrowing or borrowing bias? Leveraging Bayesian borrowing and quantitative bias analysis for robust comparative effectiveness insights (O049) - Grace Hsu

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Chair: Julia Saperia
Unexpected results and challenges when using mixture priors for Bayesian borrowing (O031) - Darren Scott
Non-monotonic power in Bayesian dynamic borrowing: insights and practical remedies (O035) - Gianmarco Caruso
Biased borrowing or borrowing bias? Leveraging Bayesian borrowing and quantitative bias analysis for robust comparative effectiveness insights (O049) - Grace Hsu

11 June 2025

Chair: Sarwar Mozumder Session Introduction - David Wright and Sarwar Mozumder Marginal hazard ratios and covariate adjustment – A causal inference perspective - Rhian Daniel Efficiency of nonparametric superiority tests based on restricted mean survival time versus the log-rank test under proportional hazards - Dominic Magirr Covariate adjustment in time-toevent data: single and doubly-robust methods - Sanne Roels Ensuring covariate adjustment methods for TTE outcomes are fit for use - Tim Morris Discussion Panel: Thoughts from A Regulator’s Perspective – What are the Expectations? Armin Koch, David Wright, Rhian Daniel, Dominic Magirr, Sanne Roels and Tim Morris

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Chair: Sarwar Mozumder
Session Introduction - David Wright and Sarwar Mozumder
Marginal hazard ratios and covariate adjustment – A causal inference perspective - Rhian Daniel
Efficiency of nonparametric superiority tests based on restricted mean survival time versus the log-rank test under proportional hazards - Dominic Magirr
Covariate adjustment in time-toevent data: single and doubly-robust methods - Sanne Roels
Ensuring covariate adjustment methods for TTE outcomes are fit for use - Tim Morris Discussion Panel: Thoughts from A Regulator’s Perspective – What are the Expectations?
Armin Koch, David Wright, Rhian Daniel, Dominic Magirr, Sanne Roels and Tim Morris

11 June 2025

Chair: Conny Berlin Published patient preference studies can influence the choice of endpoints in clinical trials: An example from Atopic Dermatitis (O018) – Byron Jones Assessing the Readiness of the Patient Preference Study Landscape for Meta-Analyses and Benefit Transfers: Do We Always Need a New Preference Study (O021) - Michael Bui Enhancing Generalizability in Patient Preference Studies: Addressing Sample Skewness in the associated Covariate Distribution (O020) - Divya Mohan Patient Preferences in Clinical Trials, Challenges and Opportunities (O013) - Cecilia Jimenez Moreno

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Chair: Conny Berlin
Published patient preference studies can influence the choice of endpoints in clinical trials: An example from Atopic Dermatitis (O018) – Byron Jones
Assessing the Readiness of the Patient Preference Study Landscape for Meta-Analyses and Benefit Transfers: Do We Always Need a New Preference Study (O021) - Michael Bui
Enhancing Generalizability in Patient Preference Studies: Addressing Sample Skewness in the associated Covariate Distribution (O020) - Divya Mohan
Patient Preferences in Clinical Trials, Challenges and Opportunities (O013) - Cecilia Jimenez Moreno

11 June 2025

Chair: Marco Boeri This session is a joint effort of the EFSPI/PSI Benefit-Risk ESIG. The speakers will emphasize recent developments in BRA methodologies for medicinal products. What does the CIOMS WG XII Benefit-Risk Assessment Report say? - Shahrul Mt-Isa Innovative trial designs and effect size estimation - bias, de-biasing, and when is it considered to be important - Ursula Garczarek Implementing innovative safety evaluation methods: Overcoming challenges and sharing successes - Naomi Givens Methodological aspects and practical application of a drug quantitative benefit-risk assessment: a case study - Zhaoyang Teng, Hua Liu, Zhaowei [Zoe] Hua, Rui [Sammi] Tang and Gaëlle Saint-Hilary Shahrul Mt-Isa, Ursula Garczarek Naomi Givens and Pavel Mozgunov

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Chair: Marco Boeri
This session is a joint effort of the EFSPI/PSI Benefit-Risk ESIG. The speakers will emphasize recent developments in BRA methodologies for medicinal products.

What does the CIOMS WG XII Benefit-Risk Assessment Report say? - Shahrul Mt-Isa
Innovative trial designs and effect size estimation - bias, de-biasing, and when is it considered to be important - Ursula Garczarek
Implementing innovative safety evaluation methods: Overcoming challenges and sharing successes - Naomi Givens
Methodological aspects and practical application of a drug quantitative benefit-risk assessment: a case study - Zhaoyang Teng, Hua Liu, Zhaowei [Zoe] Hua, Rui [Sammi] Tang and Gaëlle Saint-Hilary

Shahrul Mt-Isa, Ursula Garczarek Naomi Givens and Pavel Mozgunov

11 June 2025

Chair: Nicola Scott The GSK Biostatistics team has successfully used Bayesian Dynamic Borrowing (BDB) in a commercial setting, which allows for the re-use of external data, synthesising new and existing data to increase efficiency whilst maintaining rigorous standards for regulatory decision making. The judging panel was impressed by the culmination of years of work invested in this project – starting with the development and publication of innovative methodology, followed by diligent efforts to communicate this methodology to regulators and stakeholders. The acceptance of Bayesian approaches by regulators is a big step forward, widely acknowledged within the industry and beyond. The award presentation took place at the PSI annual conference in Amsterdam, where Nicky Best and Andrea Callegaro collected the award on behalf of the Biostatistics team. Nicky Best, Andrea Callegaro, Dawn Edwards and Jodie Crawford

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Chair: Nicola Scott
The GSK Biostatistics team has successfully used Bayesian Dynamic Borrowing (BDB) in a commercial setting, which allows for the re-use of external data, synthesising new and existing data to increase efficiency whilst maintaining rigorous standards for regulatory decision making. The judging panel was impressed by the culmination of years of work invested in this project – starting with the development and publication of innovative methodology, followed by diligent efforts to communicate this methodology to regulators and stakeholders. The acceptance of Bayesian approaches by regulators is a big step forward, widely acknowledged within the industry and beyond. The award presentation took place at the PSI annual conference in Amsterdam, where Nicky Best and Andrea Callegaro collected the award on behalf of the Biostatistics team.
Nicky Best, Andrea Callegaro, Dawn Edwards and Jodie Crawford 

11 June 2025

Chair: Julia Saperia Predicting the probability of clinical trials success from AI-based approaches using multimodal data (O032) - Nils Ternes Enhancing Treatment Effect Estimation in Clinical Trials using Machine Learning: A Within-Study Prognostic Score Approach (O038) - Antigoni Elefsinioti Application of causal inference to identify determinants of seizure reduction and quality of life in patients with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC) treated with cannabidiol (CBD) (O048) - Teresa Greco

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Chair: Julia Saperia
Predicting the probability of clinical trials success from AI-based approaches using multimodal data (O032) - Nils Ternes
Enhancing Treatment Effect Estimation in Clinical Trials using Machine Learning: A Within-Study Prognostic Score Approach (O038) - Antigoni Elefsinioti
Application of causal inference to identify determinants of seizure reduction and quality of life in patients with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), and tuberous sclerosis complex (TSC) treated with cannabidiol (CBD) (O048) - Teresa Greco 

11 June 2025

Chair: Sue Todd INVENTS: Going Beyond Conventional RCTs for Rare and Paediatric Diseases – Insights from Year 1 of the European Collaboration (O022) - Marcus Elze Statistical Challenges in Health Technology Assessment (HTA) for Rare Diseases (O042) - Samadhan Ghubade Randomization-based Inference for MCP-Mod (O037) - Lukas Pin

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Chair: Sue Todd
INVENTS: Going Beyond Conventional RCTs for Rare and Paediatric Diseases – Insights from Year 1 of the European Collaboration (O022) - Marcus Elze
Statistical Challenges in Health Technology Assessment (HTA) for Rare Diseases (O042) - Samadhan Ghubade
Randomization-based Inference for MCP-Mod (O037) - Lukas Pin
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