It’s an exciting time to be involved in research because there is now the opportunity to combine traditional clinical effectiveness research with patient-centered outcomes research (PCOR).
PCOR includes measures important to patients and caregivers such as quality of life, disease symptoms, and safety and engages patients throughout the research process by weighing in on study objectives, design, conduct, implementation, and dissemination. PCOR encourages other stakeholders such as physicians, payers, and policymakers to understand what is important to patients and address responsive research questions. Essentially, PCOR helps bridge the communication gap between health care payers, systems, and providers with patients or residents and caregivers.
PCOR also speeds the dissemination of research findings back to patients and other stakeholders who use the most current information in health care decisions.
And residents of long term care facilities can participate as well. (For more information, see “How Can Residents Get Involved?”)
For rheumatologic conditions, PCOR has the potential to make a tremendous impact. Whereas treatment decision making for many rheumatologic conditions has traditionally depended primarily on x-rays, lab tests, and clinical assessment of relevance to clinicians, PCOR enables researchers to learn how treatments affect patients’ ability to manage symptoms and maintain their quality of life outside of the clinic.
PCOR brings both perspectives together, identifying optimal approaches to manage and study chronic diseases, particularly complex conditions such as rheumatoid arthritis, vasculitis, or pediatric rheumatic disease.
The PCORI Mandate
Ensuring that stakeholders—who are defined as the actual users of research results—are engaged in the Patient-Centered Outcomes Research Institute (PCORI) research is mandatory. PCORnet provides a formal channel for researchers to approach patient communities as partners as well as participants in eventual studies and analysis. Each Patient Powered Research Network (PPRN) functions as a registry or secured repository of patient-reported data and observational electronic health information.
Most of the PPRNs grew out of existing patient communities, and most will have online (Web) and/or smartphone-based portals that allow patients the ability to access research opportunities directly rather than relying on physician solicitation for participation. This is one example of how PCORI aims to engage and empower patients in health research.
The Arthritis Networks
One of the PPRNs, called ArthritisPower, focuses on rheumatoid arthritis, spondyloarthritis, and other rheumatologic and skin conditions (such as psoriasis). It was created and is managed by the nonprofit arthritis patient advocacy group CreakyJoints in partnership with the University of Alabama at Birmingham.
Within ArthritisPower, a Patient Governor Group (essentially a patient steering committee) combs through research requests coming in from patients and researchers wanting to partner with the patient community, and then helps prioritize which questions will be developed into studies. Participating patients choose which studies to participate in and enter their health care data via a mobile- and computer-based application that runs on iPhones, Android devices, or in a browser on desktop PCs.
Data collected relate to sleep, pain, fatigue, and more, although individual studies can be designed to elicit other types of longitudinal data. Study results will be reported directly to participants inside the application (and shared peer-to-peer via presentations and publications), thus closing the loop from inception to completion and dissemination.
Besides ArthritisPower, two other PPRNs focus on rheumatologic conditions. The Vasculitis PPRN focuses on all forms of vasculitis and is a partnership between the Vasculitis Clinical Research Consortium (investigators) and the Vasculitis Foundation (patients).
The Patients, Advocates, and Rheumatology Teams Partner for Research and Service (PARTNERS) PPRN is a collaboration between disease-specific advocacy groups (Arthritis Foundation, Cure JM Foundation, Lupus Foundation of America), a quality improvement network (PR-COIN—Pediatric Rheumatology Care & Outcomes Improvement Network), and a research network (CARRA—Childhood Arthritis and Rheumatology Research Alliance).
Each of these patient networks is eager to partner with clinical researchers to support new projects and initiatives using the infrastructure that has been newly established.
Patient-Centered Priorities In Rheumatology
Traditional health research has brought remarkable advances in treating rheumatic diseases in children and adults, yet there are limitations.
For example, research subjects in many studies, such as Phase III clinical trials, don’t necessarily reflect “real world” patients who often have comorbidities that exclude them from the studies. Yet these unrepresented patient communities raise important clinical management questions that need to be answered in order for clinicians to deliver the best possible care.
Patient-centered research is already underway in the arthritis/rheumatology community. For example, one of the challenges of caring for a patient with arthritis is the large number of treatment options available. Some patients may only be candidates for certain therapies, others are highly sensitive to cost, and still others may prefer pills versus injections.
Liana Fraenkel, MD, of Yale University will soon conduct a large study of patients with the ArthritisPower network to help identify patient characteristics, or “phenotypes,” that can aid physicians in working with patients to choose the best treatment option given patients’ individual preferences.
Questions To Answer
There are many questions that PCOR research in the rheumatology community could answer, such as:
■ How do clinical and patient-reported outcomes compare across different medical treatments for rheumatoid arthritis? And how does the presence of certain comorbidities affect both the choice of treatments and their outcomes?
■ How does the availability of forthcoming biosimilar medications affect patient treatment selection? What are the patient and/or provider motivations for making a medication switch?
■ How do side effects or less serious but nevertheless bothersome symptoms such as nausea impact medication adherence?
■ What is the relative safety and effectiveness of different biologic medications across both directly and indirectly related health domains (such as sleep, fatigue, depression)?
Indeed, now that PCORnet is in its second year, groundbreaking research that crosses multiple conditions is in the pipeline. Glucocorticoids, newer and traditional synthetic DMARDs, and biologic medications are used to treat a range of rheumatologic and other conditions.
Studies that span multiple PPRNs are being proposed to study the comparative safety and effectiveness of these and other medications currently available and those to come, as well as to revisit the safety of older medications that may have deleterious effects in the long term, such as glucocorticoids.
Physicians Build Confidence
Physicians and other health care providers have reason to be excited about PCORnet and, in particular, about PPRNs like ArthritisPower, the V-PPRN, and PARTNERS, and to encourage their patients to actively participate in patient-centered research. The longitudinal data reported by patients can support understanding of their own health while pushing research forward for everyone.
Within the ArthritisPower application designed to capture registry data, patients can track their own symptoms and generate personal reports to share with a physician. Functionality such as this can help patients and doctors work together to choose the best treatment options.
The new patient-centered research paradigm relies on robust patient engagement. Rheumatologists, rheumatology researchers, and patient advocacy organizations—like CreakyJoints, the Vasculitis Foundation, the Arthritis Foundation, Lupus Foundation of America, and Cure JM Foundation—are making this a reality.
W. Benjamin Nowell, PhD, LMSW, is director, patient-centered research, of Global Healthy Living Foundation/CreakyJoints, and co-principal investigator for ArthritisPower. Peter A. Merkel, MD, MPH, is chief, Division of Rheumatology, and professor of medicine and epidemiology at the University of Pennsylvania and co-principal investigator of the Vasculitis Patient-Powered Research Network. Laura E. Schanberg, MD, is professor of pediatrics and co-chief, Division of Pediatric Rheumatology, at Duke University School of Medicine and principal investigator of PARTNERS PPRN. Jeffrey R. Curtis, MD, MS, MPH, is the William J. Koopman Endowed Professor in Rheumatology and Immunology at the University of Alabama at Birmingham, Division of Clinical Immunology and Rheumatology, and co-principal investigator for ArthritisPower.