The randomised clinical trial (RCT) is the cornerstone for evaluation of medical interventions, whether these interventions concern (new) medicines, medical technology or non-medical interventions to improve quality of life for patients. Our R&E group has a strong track record improving methodology for clinical trials, ranging from cluster randomised trials for complex health interventions to subgroup analyses in trials, hybrid Bayesian – frequentist approaches and multi-arm trials such as basket and umbrella designs. Based on both international academic partnerships and our long standing collaboration with the Dutch Medicines Evaluation Board and the European Medicines Agency, there is particular focus on drug development and regulatory decision making. A more recent development in that context is the use of real world data to generate evidence for such decision making, as augmentation to randomised trials. Methodology development to establish added value for use of real world data and innovative approaches to augment trials are at the core of this research theme: hybrid designs, use of real world data to establish drug effects in target populations and use of real world data for extrapolation of trial results to broader populations.
More-EUROPA
(HORIZON-HLTH-2022-TOOL-11-02; 2023-2027)
The aim of the project is to establish the value of registry-based RWD in augmenting RCT data and to enable the more effective and ethical use of registry data to support patient-centred regulatory and HTA decision-making. Biostatistics leads the Methodology development Work Package, which (a.o.) includes the following key areas: statistical methods to incorporate registry data in early drug development decision making; methods to extrapolate clinical trial data to target populations of different composition; methods for identification of subgroup for which treatment effects differ. As founding element, innovative methods for Bayesian federated inference are implemented and extended, allowing advanced modelling in federated databases with only one pass of the data.
Collaboration CBG-MEB
Biostatistics has a long standing collaboration with the Dutch Medicines Evaluation Board (CBG-MEB), with three biostatisticians seconded to the CBG-MEB for advanced Scientific Advice and assessment. In addition, the third CBG-MEB funded Biostatistics PhD project is undertaken, focusing on improving Scientific Advice for early dose finding in oncology, given the significantly different mechanisms of action of modern anticancer drugs (compared to more traditional chemotherapy).
Collaboration Oslo University
Dr. Marleen van Gelder is seconded at Oslo University, with research focusing on using observational data to gain more insight into the safety of medication use, in particular for populations in which trials are often not feasible (pregnant people, children). Through this collaboration, it is at the forefront of this research domain, where (a.o.) Oslo University is preparing to join DARWIN-EU, one of the key initiatives of EMA to leverage Real World Data for regulatory decision making.
Kit Roes, Steven Teerenstra, Aysun Cetinyurek Yavuz, Laura Rodwell, Njeri Kamau, Bergas Fayyad