This spring, the BMS-ANed organises an in-person meeting:

Hans van Houwelingen award ceremony and symposium

Date: June 15th 2023
Time: 13:00-18:00 (CEST)
Location: “Vredenburg 19”, Vredenburg 19, 3511 BB, Utrecht

Registration is required and is through the form below

Public transport

From train station Utrecht Central, Vredenbrug 19 is a 3-4 minute walk.

Speakers

Manuela Zucknick – University of Oslo
Emmanuel Lesaffre – KU Leuven
Tim Friede – University Medical Center Göttingen

Titles and Abstracts

Machine learning with small data: examples from pharmacogenomic screens for personalised medicine
Manuela Zucknick

Borrowing information from historical data: A pharmaceutical perspective
Emmanuel Lesaffre, Akalu Banbeta, Hongchao Qi and Joost van Rosmalen

A non-technical review is given on the use of historical data in the design and analysis of randomized controlled trials using a Bayesian approach. The focus is on comparing the philosophy behind different approaches and practical considerations for their use. The two main approaches, i.e. the power prior and the meta-analytic-predictive prior, are illustrated using fictive and real data sets. Such methods, which are known as dynamic borrowing methods, are becoming increasingly popular in pharmaceutical research because they may imply an important reduction in costs. In some cases, e.g. in pediatric studies, they may be indispensable to address the clinical research question. In addition to the two original approaches, this review also covers various extensions and variations of the methods. The usefulness and acceptance of the approaches by regulatory agencies is also critically evaluated. Finally, references to relevant software are provided.

Combining randomized controlled trials and real world data 
Tim Friede

In his talk, Emmanuel Lesaffre will introduce techniques to borrow information from historical data. Building on these results, we will discuss the integration of randomized controlled trials (RCT) and real world data such as clinical registries. As concerns are frequently raised that large-scale registries might overpower smaller RCT, particular attention will be paid to the weights both sources of information receive. In this context, the analysis of clinical registries will be discussed, in particular with regard to data requirements. The work is motivated and illustrated by examples from rare diseases such as Creutzfeldt-Jakob disease and Alport syndrome. The talk draws on joint work with a number of colleagues including Christian Röver, Sarah Friedrich and Tim Mathes.

Programme

Slides

The slides for the presentations are available below:

• Manuela Zucknick
• Emmanuel Lesaffre
• Tim Friede