Autoimmune and inflammatory diseases, including rheumatoid arthritis, inflammatory bowel disease, juvenile arthritis, psoriasis, and vasculitis, collectively are relatively common conditions and are a significant source of disability and decreased quality of life. Fortunately, recent advances in therapy, including the development of biologic agents, have generally improved health outcomes for many people with these conditions. Nevertheless, patients and physicians face many uncertainties when making treatment decisions. The safety profile of newer treatments is not well established, and there are few studies of comparative effectiveness between newer treatments. Most published studies include only patients who meet strict clinical trial inclusion criteria and focus on physician-derived disease-specific clinical outcomes that typically fail to encompass important patient-facing health concepts, such as fatigue, that are important to people living with these conditions.
To address these uncertainties and improve decision making about treatment, we bring together the data and resources of five patient-powered research networks (PPRNs: AR-PoWER, PARTNERS, CCFA Partners, ImproveCare Now, and Vasculitis) and two clinical data research networks (CDRNs: Mid-South, PEDSnet). Each PPRN represents disease conditions commonly treated with biologic and immunosuppressive medications; each PPRN’s members have independently identified comparative effectiveness and safety of medications as a top research priority.
Using electronic health record data from CDRNs and linked hospital discharge and outpatient pharmacy data when available, we will evaluate the relationship between newer biologic agents and the important safety events of hospitalized infection, heart attack, stroke, and cancer. Using disease-specific and cross-cutting patient-reported outcomes collected directly from PPRN participants who are changing biologic and other treatments, we will evaluate their effectiveness to improve patient-reported outcomes. In addition, to support PPRN recruitment, we will utilize CDRN resources to identify persons with the autoimmune and inflammatory conditions of interest and invite them to join the respective PPRNs, thus enhancing the PPRNs with new, previously unidentified members.
Throughout this process, we will engage patients and other stakeholders from both PPRNs and CDRN constituents in the design of these studies, including the creation of a steering committee with patient representation. We will disseminate our study findings broadly throughout the PPRNs and their associated professional societies and national patient advocacy groups.