6th CEN Conference

Session

Clinical trials 2

20 May 2026, 13:45

Room 13 B

Conveners

Clinical trials 2

• Sonja Zehetmayer (Medical University of Vienna)

61. A systematic empirical comparison of different statistical approaches for a multi- aspect analysis of clinical trial data in rare diseases

Martin Geroldinger (Research Program Biomedical Data Science, Paracelsus Medical University Salzburg)

20/05/2026, 13:45

oral presentation

The servEB project (WISS 2025, federal state of Salzburg, 20102/F2300645-FPR) combines
clinical expertise, advanced statistical analyses, and AI-driven imagine classification technology to improve the assessment of trial outcomes in rare diseases, especially Epidermolysis Bullosa research as an example. When defining meaningful endpoints, multiple aspects of the disease have to be considered,...

164. Assessing covariate-adjusted risk differences in small-sample trials: A comparative evaluation of statistical methods

Martin Schnuerch (Global Biostatistics & Data Sciences, Boehringer Ingelheim Pharma GmbH & Co. K)

20/05/2026, 14:03

oral presentation

Binary endpoints are commonly used to measure clinical outcomes in randomized controlled trials. In this context, conditional odds ratios (ORs) based on logistic regression have been routinely used as population-level summary to quantify treatment effects. However, ORs have been criticized for a lack of interpretability, non-collapsibility, and sensitivity to model specification. In response,...

206. When randomization is not random: Allocation bias in small sample, group sequential randomized clinical trials

Daniel Bodden (RWTH Aachen University)

20/05/2026, 14:39

oral presentation

Even in rare diseases, where the sample size is limited and blinding is less frequently implemented, randomized controlled trials are considered the gold standard to proof efficacy. Randomization is used to mitigate bias and regulatory guidance recommend the investigation of the impact of bias on the test decision. We quantified how allocation bias affects the test decision in small sample...

193. Multiple Treatment Arms, Multiple Biases? Allocation and chronological biases in platform trials

Nico Bruder (Institute of Medical Statistics, Medical University of Vienna)

20/05/2026, 14:57

oral presentation

In rare diseases, the need for innovative clinical trial designs is increasing. Platform trials are becoming particularly popular, as they allow for flexible adding and dropping of arms and reduce sample size requirements by using a shared control. In a platform trial setting with two experimental arms and one control, we use clinical trial simulations to quantify the impact on operating...