Description: Modelling longitudinal natural history data is challenging and need some special knowledge when reaching efficient results. In the presentation I will discuss some common approaches and their assumptions, invalidating these analysis strategies. I will recommend to use progression modelling via Linear of nonlinear mixed effects modelling. Nonlinear mixed effects modelling will be important, when observations of the primary endpoint variable are affected by ceiled or floored effects, as with SARA score in the ataxia field, best corrected visus acuity in ophthalmology etc. I will further show and comment on how these methods can be used to inform sample size justification of a comparative clinical trial. As I will derive my arguments mainly along ataxia examples, I will avoid formal description.
Lecturer: Dr. Ralf-Dieter Hilgers, Professor at SFU and RealiseD coordinator
Prof. Dr. Ralf-Dieter Hilgers studied mathematics at RWTH Aachen University. He finished his doctoral thesis at the statistical faculty of the University of Dortmund in 1991. In 2000 he received the Venia Legendi for Medical Statistics at the University of Cologne. Since 2001 he is head of the Institute of Medical Statistics (IMSA) at the Medical Faculty, RWTH Aachen University. His research interest is in optimal design of experiments, randomizations procedure and clinical trials. Since 1987 he gives biostatistical advice to clinical and experimental trials in all clinical and preclinical areas. Professor Hilgers teaches 300 students in different bio-scientific areas per year and is responsible for the education of investigators in clinical trials. He also acts as reviewer for methodological and clinical journals. He receives funding form German funders like BMBF, DFG and also private partners. According to his main research activity, he coordinates the IDeAl project funded by the European Community within the 7th framework program (GA N° 602552) (www.ideal.rwth-aachen.de) form 2013-2017, which established new methodologies for small population group trials. Currently, he is co‐lead of WP20 “Accelerating the validation, use and development of innovative methodologies tailored for clinical trials in RDs“ within the H2020 funded European Joint Program on rare diseases (GA N° 825575). He is co‐lead of WP4 of the European Rare Disease Research Coordination and Support Action consortium (ERICA) project serving for the ERN’s.
