5 papers
Blinded continuous information monitoring of recurrent event endpoints with time trends in clinical trials
Tobias Mütze, Susanna Salem, Norbert Benda +2
Blinded sample size re-estimation and information monitoring based on blinded data has been suggested to mitigate risks due to planning uncertainties regarding nuisance parameters.…
Recent advances in methodology for clinical trials in small populations: the InSPiRe project
T. Friede, M. Posch, S. Zohar +19
Where there are a limited number of patients, such as in a rare disease, clinical trials in these small populations present several challenges, including statistical issues. This l…
Optimal Design in Hierarchical Models with application in Multi-center Trials
Maryna Prus, Norbert Benda, Rainer Schwabe
Hierarchical random effect models are used for different purposes in clinical research and other areas. In general, the main focus is on population parameters related to the expect…
On estimands and the analysis of adverse events in the presence of varying follow-up times within the benefit assessment of therapies
Steffen Unkel, Marjan Amiri, Norbert Benda +14
The analysis of adverse events (AEs) is a key component in the assessment of a drug's safety profile. Inappropriate analysis methods may result in misleading conclusions about a th…
Systematic reviews in paediatric multiple sclerosis and Creutzfeldt-Jakob disease exemplify shortcomings in methods used to evaluate therapies in rare conditions
Steffen Unkel, Christian Röver, Nigel Stallard +4
BACKGROUND: Randomized controlled trials (RCTs) are the gold standard design of clinical research to assess interventions. However, RCTs cannot always be applied for practical or e…