Evaluating (cost-)effectiveness/safety of ‘off-label’ rituximab in people with multiple sclerosis in comparison with approved treatments.
To extend registry-based RCT evidence supporting the use of rituximab by using RWD from different European multiple sclerosis registries for effectiveness and cost-benefit.
Registries are not delivering upon their promise, due to inadequate enrolment, insufficient data elements and lack of shared data access. Established patient registries, however, do capture large patient numbers and capture these patient data longitudinally. Another point of critique is that real-world data (RWD) are dependent of the health care setting, i.e., country where data originate from. In contrast with multi-centre clinical trials, outcomes may differ between countries. Therefore, capturing representative RWD from multiple countries may facilitate generalisation of results for the European decision maker. While a central data warehouse containing individual patient data would allow efficient and powerful analyses, this may often be unfeasible to data privacy or registry management constraints that hinder data sharing. This problem arises in this case study on multiple sclerosis, where we will evaluate the effectiveness and cost-benefit of rituximab using RWD from different European multiple sclerosis registries. The traditional ‘multi-step’ federated analyses or a non-federated dataset of merged data in a central data warehouse will be compared to a novel ‘one pass’ federated analysis. These federated analysis methods will be applied to the substantive research question on the effectiveness of ‘off-label’ prescribed rituximab in patients with multiple sclerosis compared to approved disease-modifying multiple sclerosis treatment.
Patient registry: Multiple Sclerosis registry (SMSreg) (www.neuroreg.se/en/multipelskleros/) and MS patient registries from three other European countries.
