A recent trend to incorporate patient-reported outcomes (PROs) in clinical research, and ultimately clinical practice, is a response to the need to better measure and treat what patients truly care about, and adapt to the changing healthcare environment, which increasingly includes patient satisfaction as a key metric for overall quality of care, a metric tied to such issues as reimbursement.
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Explore This IssueDecember 2015
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In rheumatology, including PROs in research and practice is particularly important given the nature of inflammatory diseases, says Yasser El Miedany, MD, consultant rheumatologist at Medway Maritime Hospital in Kent, United Kingdom, and honorary senior clinical lecturer at King’s College in London, who has written numerous articles about patient-centered care in rheumatology.1-3
“In inflammatory arthritic conditions, excluding gouty arthritis, there is not a single ‘gold standard’ measure to assess outcomes,” says Dr. El Miedany, adding that the introduction of biologics and the recently adopted treat-to-target concept has changed the way clinicians look at these conditions and the short- and long-term impacts they have on patients’ lives.
“The success achieved over past years in controlling disease activity draws attention to other outcomes, such as quality of life, ability to work and absence from work because of the arthritic condition, as well as co-morbidities,” he says.
To fully implement patient-centered care, therefore, requires gathering information from patients about their experience with their condition and developing valid and reliable metrics to measure what traditionally have been viewed as soft data or subjective experience. To that end, the European League Against Rheumatism (EULAR) recently recommended involving patients in research.4
An example of efforts underway to involve patients in research comes from Johns Hopkins University in Baltimore where investigators—collaborating with a broad range of stakeholders worldwide—have been working to develop a metric to quantify and qualify patients’ experience with rheumatoid arthritis (RA) flares.
Creating a Patient-Centered Care Model for RA Flares
Since 2006, Clifton O. Bingham, III, MD, associate professor of medicine at Johns Hopkins University and director of Johns Hopkins Arthritis Center, and Susan Bartlett, PhD, associate professor of medicine at McGill University in Montreal and adjunct associate professor at Johns Hopkins, have worked with a worldwide consortium to better understand and develop a standardized outcome measure for RA flares.
Comprising physicians, researchers and patients, the Outcome Measures in Rheumatology (OMERACT) Flares in RA workgroup is addressing gaps in the assessment of RA flares. These gaps include a definition of what constitutes a flare, as well as a way to measure its severity.
“What we are attempting—as an international group—is to come together to first systematically define what is meant by a flare representing an increase in inflammatory RA disease activity that patients and providers agree on, and then find a way to measure it,” says Dr. Bartlett. Until now, she says, there has been no reliable way to identify and quantify flares.
The need to better identify and measure flares has gained importance “in this new era of treatment favoring lowering or withdrawing medications [because] this places patients with RA at greater risk of flare.
“The reason we think it is important to identify flares is because there is now evidence to show that flares are associated with damage,” says Dr. Bartlett. “It is really important for patients to get evaluated if they are in a flare, because it may mean their treatment is not working as well as it should be.”
Amye L. Leong, MBA, a patient research partner and member of the Steering Committee for the OMERACT Flares in RA group, emphasized the importance of a uniform method for identifying flares, particularly now that treatment can make living with the disease more manageable.
Having lived with RA for 35 years, five of which confined her to a wheelchair because of ineffective treatment, Ms. Leong stressed that RA today does not need to be the crippling disease that she first struggled with—if it is diagnosed and treated early.
“Early on as a new patient, [I heard] the doctors … talking about flares, but it was only 28 years later while working with the OMERACT flare group that I learned there is no standard patient-centered definition of a flare,” Ms. Leong says. “I find that startling!”
Findings of the OMERACT Group In 2012, the OMERACT group published the results of its initial studies on patients with RA who reported what they experienced during flares.5 This work was undertaken to understand the symptoms and impact of flares that both patients and physicians thought were important in understanding flares.
“One of our first findings was that flares are, indeed, a universal experience and far more common than any of us realized,” says Dr. Bartlett. “We thought maybe 15% of patients in a given year would flare, but now our data suggest flare is much more common.” Emerging data suggest that up to one-third or more of patients who come in for their regular office visit are in a flare.5
The need for a consensus-based definition of a flare is evident from another study result that found a lack of agreement among patients and physicians about when a patient is in a flare. According to Dr. Bartlett, data from a longitudinal early RA cohort in Canada suggest that doctors agree with patients about 70% of the time when a patient is experiencing a severe flare. However, in the 30% of cases where there is lack of agreement, in 15%, patients think they are experiencing a flare when they are not, and in 15% the doctor says a patient is experiencing a flare, when they are not.5
Another surprise from the study, according to Dr. Bartlett, was how little clinicians know about the nature of flares and how they impact patients.5 Although the study found a lot of overlap in what physicians and patients thought were important symptoms of flares, patients described a lot more things that were important to them than physicians recognized.
Along with trouble with mobility and moving joints, patients also emphasized fatigue and said that this was often one of the first signs of a flare coming. Many felt fatigue interfered more with quality of life than pain. “One of the first things that patients told us was that when they start to go into a flare … they … stop everything other than work to conserve energy,” says Dr. Bartlett.
“Depending on the severity of the flare, I may disengage from social activity and will do anything to keep working,” says Ms. Leong.
Dr. Bingham points out that symptoms, including fatigue, that clinicians routinely address in assessing disease activity are important to patients because they affect such everyday activities as brushing their hair, opening doors and being able to fulfill important roles as a parent. “These are the things patients care about and change when they are flaring [that] they want us to measure,” he says, adding that this shows that the current metrics used for RA (e.g., disease activity score [DAS]) in clinical practice and research really do not reflect many of the things patients care about.
Lessons learned from that study, says Dr. Bartlett, are that patients are pretty good at managing their flares (e.g., by eliminating unnecessary activity, sleeping more, increasing medications as agreed with their doctors) and that they seek out their physician when self-management strategies no longer work.
‘The point of measuring fatigue is not to treat a symptom of RA, but to treat the disease itself better.’ —Clifton O. Bingham, III, MD
“Many times, patients are able to successfully manage flares through a series of creative behavioral strategies, but there is, of course, a point at which self-management no longer works,” she says Overall, Dr. Bartlett says the research offers a clearer indication of the prevalence and burden of flares and that it can have an impact on patients’ quality of life.
From this and ongoing work through OMERACT, the investigators arrived at a consensus of five common core domains for the measurement of flares: pain, physical function, fatigue, participation in activities and stiffness (in press).5-8 Using these measurements, the group is now collecting data on patients who experience flares to understand the range of each domain (e.g., how much pain, how great the fatigue).
Results of the final version of the flare measure will be presented at the May 2016 OMERACT meeting and open to ratification by OMERACT attendees.
Patient-Centered Care Approach to Management
Building on the work done through the OMERACT collaboration, Drs. Bingham and Bartlett are collaborating on several projects funded through the national Patient-Centered Outcomes Research Institute (PCORI), a major funding agency in the U.S.
In their first project, initiated in 2011, the investigators evaluated how patient-reported outcome information could be integrated within the clinical setting.9
A major focus was to understand whether the generic Patient-Reported Outcome Measurement Information System (PROMIS) measures could reflect the symptoms and impact that patients with RA described. Developed by the National Institutes of Health, PROMIS offers precise, reliable ways to evaluate patient-reported symptoms across disease (see PROMIS Instruments, above left).
The overall project goals, Dr. Bingham says, were to see if more patient-centered information could be gathered in the context of a clinical visit, to see what the information could tell physicians about people with RA, and to determine how that information would be interpreted by patients and their physicians and whether it could be used in making treatment decisions.
Results of the project, published in September 2015, provide preliminary evidence of the validity of PROMIS measures to assess RA symptoms and their impact on patients.9
“The measurement system of PROMIS is highly precise in the ways it can measure symptoms and impacts across the spectrum of RA disease activity,” says Dr. Bingham.
“Results are provided in the context of where someone falls in relation to the general population.”
PROMIS can measure symptoms and functional status in people with RA, such as fatigue, sleep, emotional distress and participation in everyday activities. Results are reported using T-scores in which all scales have a mean of 50 and standard deviation of 10. Using a common metric across all scales makes it easier to interpret and compare results across visits, between RA patients and those with other medical conditions.
The ability to use such measures as PROMIS to quantify patient reports that traditionally have been seen as subjective, and therefore harder to quantify, comes from the use of an advanced statistical methodology, called item response theory. Dr. Bartlett likens the methodology to the way standardized tests are constructed. “The answer you give on one question will determine the next question you get, and the computer will give you questions that help pinpoint what is being evaluated,” she says.
When measuring fatigue, for example, the computer algorithm draws on 90 questions in the PROMIS item bank to find the right set of questions (often four to six) that can precisely identify fatigue level.
“This is a new way of measuring things,” says Dr. Bingham, adding that results are able to quantify for physicians not only how much fatigue patients are experiencing, but also how it is affecting their overall quality of life.
Because the measures are very sensitive, even when measuring very low or high levels of a symptom, this approach offers new insight into RA. For example, even when patients are in remission or experiencing low disease activity, PROMIS measures indicate that a subset was experiencing debilitating levels of fatigue that affected their ability to work and participate in life roles. Many patients also told the investigators that severe fatigue was one of the earliest signs of a major RA flare.
“The question becomes, ‘Are we managing people as well as we can, or do we need to think about also measuring other key symptoms that really affect how people feel and function?’” Dr. Bingham says.
Treat the Whole Patient
An answer to that question comes from those who live with RA daily. “Patients with rheumatoid arthritis are whole people,” says Ms. Leong. “Just to address the flare is not to address the whole person and the family, work and social context in which we live.”
As a patient advocate, along with her many advisory and research roles, including president and CEO of Healthy Motivation, Ms. Leong says that the work with OMERACT and Drs. Bingham and Bartlett is aimed at activating patients with RA to get engaged with their own care. “We know that activated, engaged patients have better outcomes and [fewer] flares and manage their health better,” she adds.
Anne Lyddiatt, a patient living with RA who participates in the OMERACT group and research projects, says having a better way to measure RA flares would lead to better self-management by patients. “Rheumatologists can do blood work for indications, but often, by the time we get [to their office], things have changed so they are reliant on patient reporting,” Ms. Lyddiatt says.
Having a better way to measure flares would help patients know how to better manage their flares, for example, by reducing activities, following their rheumatologist’s orders for medications (often an increase or additional medications) or deciding whether they need to see their rheumatologist.
In the Hands of Rheumatologists
Dr. Bingham says the ultimate goal of OMERACT’s work in developing patient-centered measures in RA is to get information to clinicians so they can better understand how RA symptoms and flares affect their patients and then incorporate the findings into treatment decisions.
However, he acknowledges that rheumatologists can get frustrated when they hear about the importance of measuring a symptom, such as fatigue, because of the perceived lack of treatment for it. But ongoing data suggest that fatigue tracks with other measures of RA disease activity and is often an early warning sign of impending flares.
Patients are pretty good at managing their flares (e.g., by eliminating unnecessary activity, sleeping more, increasing medications as agreed with their doctors), & they seek out their physician when self-management strategies no longer work.
“The point of measuring fatigue is not necessarily to target this particular symptom, but to ensure the RA is being optimally controlled,” Dr. Bingham says. When RA is well controlled, fatigue returns to levels seen in the general population, he notes.
“The point of measuring fatigue is not to treat a symptom of RA, but to treat the disease itself better,” he says, adding that the hope is to improve fatigue with better management of RA.
Dr. Bingham says that if rheumatologists and other healthcare providers can better understand the patient experience by measuring it more precisely, “then we have great opportunities to combine this with other clinical and laboratory information and really come up with a way of treating both the disease and the person living with the disease.”
One of the most important things about the information gained from the studies, says Dr. Bingham, is that it opens up the conversation between physicians and patients. “Patients recognized they hadn’t always related changes in how they were feeling to their RA, and physicians found the information helped them understand how well patients were doing overall and even pick up [on] early flares,” says Dr. Bingham.
For Dr. El Miedany, one of the challenges of implementing this new metric and patient-centered approach in the clinical setting will be incorporating the patient-reported outcome measure questionnaires into standard clinical practice.
However, he stresses that having patients complete a questionnaire as part of the infrastructure of usual care, similar to routine monitoring of predictors of premature mortality, such as blood pressure or cholesterol in cardiovascular disease, will help patients prepare for their visit and improve communication between the doctor and patient.
Dr. Bingham noted that PROMIS measures are being embedded within many large electronic health records (EHR) systems. Providers can potentially have the system generate e-mails prompting patients to complete selected PROMIS measures between visits via a patient portal. The results are automatically entered into the EHR. “I think this ability to track how patients are feeling between visits will be incorporated into how we provide care in the future,” he says.
The question becomes, ‘Are we managing people as well as we can, or do we need to think about also measuring other key symptoms that really affect how people feel & function?’ —Clifton O. Bingham, III, MD
Another way patients can track their symptoms over time and report this information to their rheumatologists is through a site called Arthritis Power, the first patient-led, patient-generated and patient-centered research registry for arthritis.10 All the information patients enter about their health, including sleep, pain, flares or emotional health is tracked and stored in the registry.
Ben Nowell, PhD, a co-investigator of Arthritis Power, says it’s designed to allow patients to track their personal health while also making it easy for them to participate in research projects. Dr. Nowell is also director of patient-centered research at CreakyJoints, an online nonprofit, patient-supported community.11
“When it comes time for a patient’s next appointment, he or she can e-mail their doctor this information directly from the application in advance,” says Dr. Nowell, an advisory board member for some of the projects led by Drs. Bingham and Bartlett. “Therefore, the conversation the patient and doctor have in the office may be driven by data and not reliant on a patient’s memory or a doctor eliciting for symptoms.”
“This interaction is a cornerstone of patient-centered care, [because] patients are speaking about their illness in their own terms and are prioritizing their biggest concerns,” he says.
Mary Beth Nierengarten is a writer, editor and journalist from Minneapolis with more than 20 years of experience writing about clinical medicine and health-related issues.
Developed by the National Institutes of Health, PROMIS provides a standardized approach for assessing patients’ physical and mental health status and social well-being. PROMIS instruments measure patient-reported symptoms of physical function, depression, anxiety, social function, pain and fatigue. The instruments are available at no cost as paper-based short forms (four to 10 items) or by using item banks with computerized adaptive testing where three to seven items can offer more precise results. Several EHR systems now offer access to PROMIS measures through patient portals.
Patient-Reported Outcome Tools
A toolbox of patient-reported outcomes (PROs) for use is available on the European League Against Rheumatism (EULAR) website. The goal of the toolbox is to provide a comprehensive database of validated patient-reported instruments, such as questionnaires, indices and scales, which emphasize PROs used in rheumatology. Interested users can search for an outcome measure by language, by disease or by name.
Online Patient Resources
- El Miedany Y. Adopting patient-centered care in standard practice: PROMs moving toward disease-specific era. Clin Exp Rheumatol. 2014 Sep–Oct;32(5 Suppl 85):S40–S46.
- El Miedany Y. PROMs in inflammatory arthritis: Moving from static to dynamic. Clin Rheumatol. 2013 Jun;32(6):735–742.
- El Miedany Y, El Gaafary M, Palmer D. Assessment of the utility of visual feedback in the treatment of early rheumatoid arthritis patients: A pilot study. Rheumatol Int. 2012 Oct;32(10):3061–3068.
- Smolen JS, Landewé R, Breedveld FC, et al. EULAR recommendations for the management of rheumatoid arthritis with synthetic and biological disease-modifying antirheumatic drugs: 2013 update. Ann Rheum Dis. 2014 Mar;73(3):492–509.
- Bartlett SJ, Hewlett S, Bingham CO 3rd, et al. Identifying core domains to assess flare in rheumatoid arthritis: An OMERACT international patient and provider combined Delphi consensus. Ann Rheum Dis. 2012 Nov;71(11):1856–1860.
- Hewlett S, Sanderson T, May J, et al. ‘I’m hurting, I want to kill myself’: Rheumatoid arthritis flare is more than a high joint count—An international patient perspective on flare where medical help is sought. Rheumatology (Oxford). 2012 Jan;51(1):69–76.
- Bartlett SJ, Bykerk VP, Cooksey R, et al. Feasibility and domain validation of rheumatoid arthritis (RA) flare core domain set: Report of the OMERACT 2014 RA Flare Group Plenary. J Rheumatol. 2015 Nov;42(11):2185–2189.
- Bykerk VP, Lie E, Bartlett SJ, et al. Establishing a core domain set to measure rheumatoid arthritis flares: Report of the OMERACT 11 RA Flare Workshop. J Rheumatol. 2014 Apr;41(4):799–809.
- Bartlett SJ, Orbai AM, Duncan T, et al. Reliability and validity of selected PROMIS measures in people with rheumatoid arthritis. PLoS One. 2015 Sep 17;10(9):e0138543.
- Arthritis Power.