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20 November 2024

With an increasing number of vaccines recommended in pregnancy it is important to consider how best to evaluate safety. There are particular challenges in assessing safety in pregnancy such as healthy vaccinee effects, immortal time bias and biases introduced in the days close to birth. In this talk I will discuss these challenges and consider different designs with pros and cons, this will include presenting a study I have done using a nested case -control study within a cohort for Covid-19 vaccine safety and plans for a target trial emulation approach for assessing RSV vaccine in pregnancy.

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Professor Nick Andrews

With an increasing number of vaccines recommended in pregnancy it is important to consider how best to evaluate safety. There are particular challenges in assessing safety in pregnancy such as healthy vaccinee effects, immortal time bias and biases introduced in the days close to birth. In this talk I will discuss these challenges and consider different designs with pros and cons, this will include presenting a study I have done using a nested case -control study within a cohort for Covid-19 vaccine safety and plans for a target trial emulation approach for assessing RSV vaccine in pregnancy.

13 November 2024

It is not hard to find a bad visualisation. But how can you effectively improve it? Lorenz Uhlmann is presenting the positive effects of applying gestalt principles. Visualisations are available on the Wonderful Wednesday blog.

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Several design choices must be made while creating a visualisation. Bar chart or line plot? Faceted plots or overplotting? Should there be used multiple colours? And if yes, what colour scheme is best? How to optimize axes, legend, background and title? See the step-by-step improvement. The next challenge will be about intra- and interrater reliability. 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


23 October 2024

This webinar discusses the complexities and opportunities of clinical trials with hierarchical composite endpoints, covering topics such as win odds, win ratio, net benefit as well as regulatory considerations when defining hierarchical composite endpoints.

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Margaret Gamalo, Cordula Zeller, Patrick Schlömer, Dali Zhou

This webinar discusses the complexities and opportunities of clinical trials with hierarchical composite endpoints, covering topics such as win odds, win ratio, net benefit as well as regulatory considerations when defining hierarchical composite endpoints. This webinar starts with a general introduction to hierarchical composite endpoints and an overview of common analysis methods including win ratio, win odds, and net benefit. Then, the practical considerations are illustrated through case studies from clinical trials in heart failure and chronic kidney disease. The webinar concludes with a discussion and a Q&A.

17 October 2024

In this webinar we will review the range of statistical methodologies used to harness the potential of Real-World Data (RWD) in clinical development, particularly in the context of rare diseases and small populations like paediatrics. The session will include theoretical understanding and practical case studies, with a special focus on Bayesian methods and causal inference.

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In this webinar we will review the range of statistical methodologies used to harness the potential of Real-World Data (RWD) in clinical development, particularly in the context of rare diseases and small populations like paediatrics. The session will include theoretical understanding and practical case studies, with a special focus on Bayesian methods and causal inference.    

09 October 2024

It has been shown that certain patients benefit more from specific drugs while others suffer more heavily from side effects. Personalised dosing could address this issue. Steve Mallett is presenting visualisations that support the identification of patient groups with differential dose-response profile. Visualisations are available on the Wonderful Wednesday blog.

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The interactive Shiny application DoRiS was specifically designed to explore the dose-response in subgroups. It combines the display of summary level data, individual data, and statistical probability. Other proposals were to use augmented box plots, combined box and violin plots, or matrix plots across the subgroup factors.

The next challenge is about improving a plot of longitudinal data. 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


08 October 2024

We will illustrate this with a case study example on testing the toxicity of an oncology formulation in a preclinical setting. This (simulated) study examines the impact of various formulation factors and their interactions on the toxicity and efficacy of a new oncology drug cocktail using a rat model. Key factors include the concentrations of Erlotinib, Cisplatin, and Dexamethasone, with responses measured such as tumor inhibition rate, overall survival rate, and toxicity indicators. Additionally, the pH of the formulation and particle size were evaluated. Responses measured include efficacy, represented by tumor inhibition rate, and various toxicity parameters, such as overall survival rate, hepatic toxicity, renal toxicity, cardiac toxicity, and hematological toxicity.

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Phil Kay and Chandramouli Ramnarayanan, JMP

Advances in AI, lab automation and closed-loop optimization promise big productivity gains for pharma R&D. But experimenting with maximum efficiency - that is, the least number of runs - will always be important. This is especially true where there is an ethical imperative, such as in pre-clinical animal experiments.  Recent advances in statistical Design Of Experiments (DOE) including Definitive Screening Designs (DSDs) and the broader class of Orthogonal Minimally Aliasing Response Surface (OMARS) designs have given us new options for understanding complex systems with small experiments. Self-Validated Ensemble Modelling (SVEM) is an innovative analysis approach that applies ideas from Machine Learning to small data from designed experiments. In this presentation we will show how SVEM works and how it can overcome common challenges when you are building useful models of complex systems from small experiments.

12 September 2024

PSI Journal Club presents “Subgroup and Covariate Analysis”. Chaired by Chris Harbron with presentations from: Thomas Jemielita: - Investigating Stability in Subgroup Identification for Stratified Medicine and Björn Holzhauer: - “Super-covariates”: Using predicted control group outcome as a covariate in randomized clinical trials.

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PSI Journal Club presents “Subgroup and Covariate Analysis”. Chaired by Chris Harbron with presentations from: Thomas Jemielita: - Investigating Stability in Subgroup Identification for Stratified Medicine and Björn Holzhauer: - “Super-covariates”: Using predicted control group outcome as a covariate in randomized clinical trials.

11 September 2024

Many cancer trials use the progression free survival as primary outcome measure, i.e. the length of time that a patient lives on without worsening of the disease. Various reasons can lead to censoring of the data. And differential censoring could influence the interpretation of the results. Paolo Eusebi presents visualisations that help to explore the pattern of events and reasons for censoring. Visualisations are available on the Wonderful Wednesday blog.

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Many cancer trials use the progression free survival as primary outcome measure, i.e. the length of time that a patient lives on without worsening of the disease. Various reasons can lead to censoring of the data. And differential censoring could influence the interpretation of the results. Paolo Eusebi presents visualisations that help to explore the pattern of events and reasons for censoring. Visualisations are available on the Wonderful Wednesday blog.

The basic plot for progression free survival is the survival plot using Kaplan-Meier estimates. Augmented with information on censoring this gives a great overview. An innovative way of displaying events and censoring is by stacked lollipop plot. See the panel discussing pros and cons. The next challenge is about visualising dose response in subgroups for personalized dosing. 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

14 August 2024

The development of a new patient reported outcome (PRO) needs to include the interpretation of the results. It should be possible to define a minimal clinically important difference and a meaningful between-group difference. Rhys Warham is presenting ways how visualisations can support that process. They are available on the Wonderful Wednesday blog.

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The development of a new patient reported outcome (PRO) needs to include the interpretation of the results. It should be possible to define a minimal clinically important difference and a meaningful between-group difference. Rhys Warham is presenting ways how visualisations can support that process. They are available on the Wonderful Wednesday blog.

For comparing the new PRO with the gold standard eCDF plots and PDF plots can be shown overlapping or stacked. There are also many different ways to show the distribution of within the standard ratings to easily spot differences or possible inconsistencies. The next challenge is on the event pattern in a study aiming for improvement on progression free survival. 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


07 August 2024

This webinar will help listeners gain a broader understanding of a recent and rapidly advancing core area of pharmaceutical drug development. The content will be broad, but will focus on clinical design and analysis strategies for advanced therapeutics such as cell/gene therapies, and strategic options to advance candidate drugs in this space.

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Avery McIntosh (Pfizer) and Oleksandr Sverdlov (Novartis)

This webinar will help listeners gain a broader understanding of a recent and rapidly advancing core area of pharmaceutical drug development. The content will be broad, but will focus on clinical design and analysis strategies for advanced therapeutics such as cell/gene therapies, and strategic options to advance candidate drugs in this space.

One of the recent advances in 21st century medicine is the emergence of gene therapies, drugs that affect the basic biology of genetic disease. The field has seen some notable setbacks in the past, but in recent years has exploded as decades of basic science have been successfully translated into the most complex biologics ever constructed, leading to regulatory approval of several gene therapy products in oncology, hematology, neurology, and ophthalmology indications. These drugs are at the apex of biological manufacturing complexity, and have the potential to be disease modifying or even curative. Evidence-based and innovative quantitative clinical development and lifecycle management strategies will be required as fixtures in the development for these unique drugs in order to reach patients in need. In this webinar we provide an overview of the history and future of gene therapies, and discuss the crucial role of the statistician in the drug development process of these drugs, with a focus on innovative trial design and analysis techniques.

A list of useful references from the presentation can be found below:
- McIntosh, Avery, and Oleksandr Sverdlov, eds. Development of Gene Therapies: Strategic, Scientific, Regulatory, and Access Considerations. CRC Press, 2024.
- Rohde, Maximilian, et al. "Practical and Statistical Considerations for the Long Term Follow‐Up of Gene Therapy Trial Participants." Clinical Pharmacology & Therapeutics 115.1 (2024): 139-146.
- McIntosh, Avery, et al. "Clinical design and analysis strategies for the development of gene therapies: considerations for quantitative drug development in the age of genetic medicine." Clinical Pharmacology & Therapeutics 110.5 (2021): 1207-1215.
- Mueller, Arne, et al. "Digital endpoints for self‐administered home‐based functional assessment in pediatric Friedreich’s ataxia." Annals of Clinical and Translational Neurology 8.9 (2021): 1845-1856.
- Hudry, Eloise, and Luk H. Vandenberghe. "Therapeutic AAV gene transfer to the nervous system: a clinical reality." Neuron 101.5 (2019): 839-862.
- Cox, Gerald F. "The art and science of choosing efficacy endpoints for rare disease clinical trials." American Journal of Medical Genetics Part A 176.4 (2018): 759-772

10 July 2024

This webinar follows up on last month's challenge on platform trial design. This time the impact of specific design changes is highlighted with effective visualisations. Bodo Kirsch is presenting examples for exploratory and explanatory visualisations. They are available on the Wonderful Wednesday blog.

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This webinar follows up on last month's challenge on platform trial design. This time the impact of specific design changes is highlighted with effective visualisations. Bodo Kirsch is presenting examples for exploratory and explanatory visualisations. They are available on the Wonderful Wednesday blog.

Exploring a huge variety of possible scenarios can be tedious. Exploratory visualisations can improve that process dramatically by using interactive parameter setting. That's what the two shiny apps of today's webinar do. Once an effect has been explored an explanatory visualisation is a powerful tool to communicate this. The next challenge is about responder definition. 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.


02 July 2024

We will review how the Graduate study team interpreted the estimand framework and used its elements to cover the primary clinical question of interest. We will review the challenges we met and the solutions we implemented.

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Rachid Abbas, Angeliki Thanaspoulou, Marcel Wolbers

GRADUATE I(NCT03444870) and GRADUATE II (NCT03443973) were two global, Phase III, randomized, placebo-controlled trials assessing the efficacy and safety of gantenerumab in early Alzheimer’s disease. The GRADUATE studies were among the first pivotal trials in Alzheimer’s Disease to adopt the estimand framework and to conduct a primary analysis which is fully aligned with the targeted estimand. In this webinar we will review the key elements of the estimand framework through the experience of the Graduate study team. We will review how the Graduate study team interpreted the estimand framework and used its elements to cover the primary clinical question of interest. We will review the challenges we met and the solutions we implemented.
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