Jeffrey Curtis, M.D., professor at the University of Alabama at BirminghamSchool of MedicineDivision of Clinical Immunology and Rheumatology, has been approved for a $2.5 million research award by the Patient-Centered Outcomes Research Institute to study the comparative effectiveness and safety of biologic and newer therapies for a number of autoimmune and inflammatory diseases.
Conditions such as rheumatoid arthritis, inflammatory bowel disease, Crohn’s and ulcerative colitis, juvenile idiopathic arthritis, psoriasis, and vasculitis are relatively common conditions causing a significant source of disability, decreased quality of life and premature mortality. Recent advances in therapy, including biologic agents or treatments made from living organisms, have dramatically improved health outcomes for many people with these conditions, although questions remain about how best to employ these agents.
“Patients and physicians face many uncertainties when making treatment decisions,” said Curtis, co-director of the UAB Center for Education and Research on Therapeutics of Musculoskeletal Disorders. “The safety profile of newer treatments is not well-established, and there are few studies of comparative effectiveness between newer treatments. Our research addresses these uncertainties and will bring improved decision-making about treatment.”
Curtis notes that most published studies of these newer agents are from clinical trials and therefore include only patients who meet strict clinical trial inclusion criteria. Moreover, these studies focus largely on physician-derived disease-specific clinical outcomes that typically fail to encompass important health issues that patients face, such as fatigue, that can have a significant impact on people living with these conditions.
To address these uncertainties and improve treatment, Curtis will bring together the data and resources of five patient-powered research networks and two clinical data research networks. Each PPRN represents disease conditions commonly treated with biologic and immunosuppressive medications, and its members have independently identified comparative effectiveness and safety of medications as a top research priority.
These PPRNs are part of PCORnet, a national network created by PCORI to enable the study of comparative effectiveness research questions that matter to patients. PCORnet consists of both patient-generated data from PPRNs and electronic health record data from large health systems.
For example, one of these five PPRNs is the ArthritisPower registry that studies adult arthritis and skin conditions, including rheumatoid arthritis, psoriatic arthritis, psoriasis and ankylosing spondylitis. Like the other PPRNs, the ArthritisPower registry is a joint venture funded by PCORI and a collaboration between UAB and the CreakyJoints patient community.
Electronic health record data from Clinical Data Research Networks, and linked hospital discharge and outpatient pharmacy data when available, will be used to evaluate the relationship between newer immune-related treatments and important safety events such as hospitalized infection, heart attack, stroke and cancer.
|Investigators will evaluate the effectiveness of disease-specific and crosscutting patient-reported outcomes collected directly from patients who are changing biologic and other treatments and will work with the five patient communities represented to track patient-reported outcomes.|
Investigators will evaluate the effectiveness of disease-specific and crosscutting patient-reported outcomes collected directly from patients who are changing biologic and other treatments and will work with the five patient communities represented to track patient-reported outcomes.
The CDRN resources will identify persons with the autoimmune and inflammatory conditions of interest and invite them to join the respective PPRNs, growing the PPRNs with new members.
Throughout this process, patients and other stakeholders from both PPRNs and CDRN constituents will be included in the creation of a steering committee with patient representation. Study findings will be published broadly throughout the PPRNs and their associated professional societies and national patient advocacy groups.
“This project was selected for PCORI funding, not only for its scientific merit and commitment to engaging patients and other stakeholders, but also for its potential to fill an important gap in our health knowledge and give people information to help them weigh the effectiveness of their care options,” said PCORI Executive Director Joe Selby, M.D., MPH. “We look forward to following the study’s progress and working with Dr. Curtis to share the results.”
Curtis’ study was selected for PCORI funding through a highly competitive review process in which patients, clinicians and other stakeholders joined clinical scientists to evaluate the proposals. Applications were assessed for scientific merit, how well they will engage patients and other stakeholders, and their methodological rigor, among other criteria.
Curtis’ award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
PCORI is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers and clinicians with the evidence-based information needed to make better-informed health care decisions. PCORnet is an innovative initiative of PCORI that aims to improve the nation’s capacity to conduct comparative clinical effectiveness research efficiently by creating a large, highly representative network for conducting clinical outcomes research that directly involves patients in the development and execution of the research. For more information about PCORI’s funding, visit www.pcori.org.