3 papers
stat.AP2018
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…
stat.OT2018
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…
q-bio.QM2016
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…