Description: In this lecture, we will focus on the role of interim analysis to react to new data emerging from inside and outside the trial. How to incorporate an interim analysis in a confirmatory clinical trial without inflating the risk of declaring a treatment as efficient when it is not. Regulatory guidelines document stresses that for confirmatory clinical trials one of the key features is the strict control of the type 1 error risk. The concept of group sequential designs will be explained which allow both early stopping for efficacy and futility. Adaptive designs will be discussed which allow the adaptations such as sample size reassessment, treatment selection or change of allocation ratio. Both regulatory guidance documents and the Consort extension on adaptive design (Dimairo et al. 2020) will be presented. The latter includes several adaptive case studies in its explanatory document. Finally, the concept of master protocols will be presented highlighting some of the key statistical concepts (Koenig et al. 2024, Meyer et al 2020), especially how to increase efficiency by data sharing concepts.
Lecturer: Franz König, Associate Professor at the Center for Medical Data Science (CeDAS) at the Medical University of Vienna.
Franz König has a master and PhD in statistics from the University of Vienna. The CeDAS cooperates in numerous international research consortia involving academia, industry and regulatory authorities. Franz is currently the head of the working group on adaptive designs at the Institute of Medical Statistics. Furthermore, he is the coordinator of the PhD “Programme Medical Informatics, Biostatistics and Complex Systems” since 2022.
He is currently member of ethics committee of the Medical University of Vienna and was also of the ethics committee of the community of Vienna (Ethikkommission der Stadt Wien). From 2008 till 2010 he was seconded to the European Medicines Agency (London, UK) as statistical expert in the Unit Human Medicines Development and Evaluation. His main research interests are multiple testing, adaptive designs, interim analyses, master protocols and data safety monitoring boards (DSMB). Franz König was associate editor of the journal “Statistics in Biopharmaceutical Research”. Before he served as Guest Editor for Special Issues in Biometrical Journal and Statistics in Medicine. Franz König has published >150 papers (see https://orcid.org/0000-0002-6893-3304).
He has been involved in several international research projects both on methodological and applied studies. For example he was the work package leader on « adaptive designs » in the EU FP7- funded research project IDEAL (Grant Agreement No 602552) and deputy coordinator of an EU Horizon 2020 funded Marie Curie ITN network IDEAS on early drug development studies (Grant Agreement No 633567). He was work package co-lead in the IMI project Pearl on platform trials (start November 2019). He was also member of the drafting team which developed a CONSORT extension for randomised trials using an adaptive design (ACE –project) which were publisheprotod in BMJ and trials.
