Rheumatoid arthritis (RA) is an autoimmune inflammatory disorder, primarily characterized by synovial joint inflammation, affecting ~0.5 to 1% of the overall population (~2900000 patients in the EU) and is more common in women than men (3:1). RA is a huge public health problem as it leads over time to permanent disability. There is no cure for RA but remission of symptoms is more likely when treatment begins early. However, approximately 40% of RA patients fail to achieve even 20% improvement in disease activity, with significant disability remaining in about a third of patients, and major work-related and social costs for patients and society. In addition, 10-20% of patients do not respond to any current medication, pointing to considerable disease heterogeneity and the need for testing and developing new drugs.
A further point related to RA heterogeneity, is that there are no biomarkers of treatment response to individual drugs. Thus, a number of unmet needs still persist particularly related to response/non-response to powerful but expensive drugs. Conventional randomized clinical trials (RCT) may address some of these challenges, but they are time-consuming, expensive and are ethically doubtful, since many patients (currently ~40% regardless of the modality of action) fail to achieve disease benefit, while being exposed to potentially toxic drugs. Thus, the rheumatology community has a need for developing an alternative strategy to deliver innovative trials.
Supported by NIHR Barts Biomedical Research Centre