Real-world evidence in rheumatic diseases: relevance and lessons learnt

Abstract

An emerging trend in the medical literature, including the Rheumatology literature, is that of accumulating large, multicentric, multi-national data based on registries of patients seen in real life situations. Such real-world evidence (RWE) may help provide valuable insights into the long-term outcomes of disease in unselected patients seen in daily practice, including patients belonging to vulnerable populations such as extremes of age, during pregnancy and lactation. Evidences gathered from real life practice settings can help understand drug prescription patterns, including adherence to treatment guidelines, cost-effectiveness of therapy, and real-life long-term outcomes, and adverse effects of treatment with particular medications. Registry-based data also helps analyze comorbidities in patients with rheumatic diseases, and their impact on quality of life, morbidity and mortality. Traditionally, a randomized controlled trial (RCT), or systematic reviews of multiple, homogenous RCTs, have been considered the cornerstone of evidence-based medicine, and RWE does, at times, provide differing viewpoints from the results of particular drugs in clinical trial settings. Therefore, in the present day, it is prudent to consider the complementary nature of information derived from RWE to that obtained from rigorous, clinical trial settings. Future guidelines for disease management may consider it relevant to include information from RWE in addition to that available from clinical trials, to help devise management guidelines that are harmonious with routine practice settings.

Keywords: Big data; Randomized controlled trial; Real-world data; Registries; Rheumatology.