Talk titles and abstracts:

Mr Brennan Kahan (UCL)

Estimands in randomised trials: ensuring trials get the right answer to the right question

The exact treatment effect being estimated in a randomised trial is often unclear, or in some cases, not relevant to clinical decision making. Estimands are a way of clarifying the treatment effect of interest, and can be used to (i) ensure the question being addressed is clear and clinically relevant; and (ii) ensure the trial is appropriately designed and analysed to answer the question of interest. In this talk I will describe the estimands framework, including: (a) what estimands are; (b) how investigators can choose appropriate estimands for their own trials; and (c) how estimands can be used to guide choices on trial design and analysis strategies.

Dr Kaspar Rufibach (Roche)

ICH E9 estimands addendum: Old wine in new bottles or a genuine step forward?

In this talk I will introduce the ICH E9(R1) estimand addendum and how it helps clarifying scientific objectives in clinical trials (https://www.ich.org/). Using real hematology trial examples I will discuss how the estimand framework helps to design trials with complex treatment options such as potential curative procedures or treatment sequence across different phases. If time permits I will also illustrate how the framework helped clarify whether and how clinical trials might need to be adapted in reaction to the Covid pandemic.

Prof Stijn Vansteelandt (LSHTM/Ghent University)
His talk will be based around recent paper with PhD student Hege Michiels on a novel estimand to adjust for rescue treatment within clinical trials.

Mr Brennan Kahan (UCL)
Brennan Kahan is a Senior Research Fellow in the Methodology Theme at the MRC Clinical Trials Unit at UCL. His research interests include estimands, complex trial designs, and efficient statistical estimators.

Dr Kaspar Rufibach (Roche)
Kaspar Rufibach is an expert Statistical Scientist in Roche's Methods, Collaboration, and Outreach group located in Basel. He is active in methodological research, provides consulting to Roche statisticians and broader project teams, gives biostatistics training to statisticians and non-statisticians in- and externally, mentors students, and interacts with external partners in industry, regulatory agencies, and the academic community in various working groups and collaborations. He co-founded and co-leads the European special interest group “Estimands in oncology” (sponsored by PSI and EFSPI, which also has the status as an ASA scientific working group, a subsection of the ASA biopharmaceutical section). The group currently has 77 members representing 37 companies and several Health Authorities, and works on various topics around estimands in oncology. Kaspar’s research interests are methods to optimize study designs, advanced survival analysis, probability of success, estimands and causal inference, estimation of treatment effects in subgroups, and general nonparametric statistics. Before joining Roche, Kaspar received training and worked as a statistician at the Universities of Bern, Stanford, and Zurich.

More on the oncology estimand WG: http://www.oncoestimand.org
More on Kaspar: http://www.kasparrufibach.ch

Prof Stijn Vansteelandt (Ghent University & LSHTM)
Since 2017, Stijn has split his time as Professor of Statistics at Ghent University and Professor of Statistical Methodology at the LSHTM. He obtained a PhD in Mathematics (Statistics) in 2002 from Ghent University, and subsequently completed postdoctoral research in the Department of Biostatistics at the Harvard School of Public Health. Stijn research mainly focuses on developing statistical methods for inferring the causal effect of an exposure on an outcome from experimental and observational data under minimal and well-understood assumptions. His work focuses on a variety of topics in biostatistics, epidemiology and medicine, including research focused on data-adaptive inference to obtain honest confidence intervals when variable selection or machine learning algorithms are employed to obtain estimators of effects or associations. Stijn has recently joined the editorial board of the Journal of the Royal Statistical Society - Series B, after previously serving as Co-Editor for the journal Biometrics, and Associate Editor for the journals of Biometrics, Biostatistics, Epidemiology, Epidemiologic Methods and the Journal of Causal Inference.

Joint event between RSS Manchester Local Group and the Centre for Biostatistics at the University of Manchester