Current guidelines for the management of patients with rheumatoid arthritis (RA) focus on early intervention and a treat-to-target goal of remission or low disease activity [
1,
2]. Over the past decade, this approach has been extremely successful in reducing disease activity and joint damage in patients with RA [
3,
4], and patients are now being diagnosed after a shorter symptom duration and with less severe inflammation. Paradoxically, however, these changes have not always been paralleled by an improvement in patient well-being with respect to various patient-reported outcomes (PROs), such as pain, fatigue, morning stiffness and disease activity, possibly due to greater societal and patient health expectations [
4].
The challenge now in RA is to encourage rheumatologists and other healthcare professionals (HCPs) to focus on individual patient perceptions of disease impact as well as disease activity measures, with the aims of providing patient-centered care through shared decision making and improving outcomes considered important by patients themselves. This should be achievable using PROs to complement disease activity as a treatment target, and with a view to informing management decisions about appropriate interventions. This is important for all patients, but particularly for individuals who fail to achieve and sustain the target of remission or low disease activity with pharmacotherapy, and those meeting low disease activity or remission states who continue to experience symptoms, such as pain and fatigue, that impact on sense of well-being.
The importance of PROs in patients with RA was highlighted during recent discussions regarding the latest (2016) update to European League Against Rheumatism (EULAR) recommendations for the management of patients with RA [
2]. In these discussions, patients suggested that the list of treatment recommendations should end with an item about PROs to convey the importance of PROs in disease management. Fortunately, today, the clinical care of patients with RA increasingly involves patient-centered management by a multidisciplinary team that includes rheumatologists as well as other HCPs, such as general physicians, physiotherapists, occupational therapists and nurses, many of whom are experienced in guiding and steering patient care based on the use of PROs.
The aim of this expert opinion article is to discuss what information can usefully be provided by patient input in the form of PROs to assist in shared decision-making between patients, rheumatologists and other HCPs in daily clinical practice, encouraging a more complete and patient-centered approach to the treatment of patients with RA. This in turn should improve patient care and help achieve optimal health outcomes. This manuscript was developed with the aid of references identified through non-systematic searches of the internet, including PubMed and Google Scholar, using the search terms: ‘patient-reported outcomes’; ‘patient-reported outcome measures’; ‘clinical practice’; ‘rheumatoid arthritis’.
What are PROs and how are they currently used in the assessment of RA?
Patient-reported outcomes (PROs) are outcomes that focus on the patient’s perspective: they are any reports of a patient’s health status that come directly from the patient [
5], and include assessments of symptoms (nature and severity), and patient functioning, health-related quality of life (HRQOL) and satisfaction with current health state. PROs are included in the core set of RA outcome measures recommended for clinical trials by the American College of Rheumatology (ACR), EULAR and Outcome Measures in Rheumatology Clinical Trials (OMERACT) [
6‐
9], and are now mandatory for submissions to drug agencies [
5,
10]. PROs provide useful information about the impact of RA on patients’ lives, as well as the effect of an intervention on clinical symptoms in clinical trials and research studies. In this setting, data from PROs are aggregated for group comparisons, and an individual patient’s results are not provided. In addition, the PROs used tend to reflect the requirements of the stakeholders [
11]. In real-world clinical practice, however, the need for PROs is different: PROs should provide key information about the individual patient perspective that complements information provided by physical assessment and composite scores, and can also be used to guide patient care [
12]. As part of a value-based case, PROs can also be used to shape guidelines [
13]. However, the uptake of PROs in daily clinical practice has generally been limited. Although PROs could help to explore the patient perspective and motivations, which is an important consideration in the shared decision-making process, most HCPs focus on objective signs of inflammation, such as tender joint count (TJC) and swollen joint count (SJC) or acute-phase reactants [
11,
14‐
16].
Both ACR and EULAR guidelines for the treatment of patients with RA recommend that treatment is based on a shared decision between the patient and rheumatologist [
1,
2]. The benefits of shared decision making include alignment of patient and rheumatologist considerations and aims, improved patient adherence to medication and improved patient satisfaction with management decisions [
2,
17]. PROs can aid in the shared decision-making process by presenting patient perspective on the disease impact.
In clinical practice, disease activity is measured using composite indices, such as Disease Activity Score with 28-joint count (DAS28), Clinical Disease Activity Index (CDAI) or Simplified Disease Activity Index (SDAI) [
18]. PROs are incorporated into these indices in the form of the Patient’s Global Assessment of Disease Activity (PGA) [
19,
20]. The PGA covers two patient-reported concepts: global health and overall disease activity [
21]. In its guidelines, EULAR also recommends improvement of disability, HRQOL, and social and work capacity as a treatment target without specifying measurement instruments [
2]. OMERACT has also highlighted the concept of patient well-being, measured using the Patient Acceptable Symptom State (PASS). This is a PRO and is defined as a symptom state that the patient considers acceptable. It recognizes that patients consider feeling good to be more important than feeling better [
22].
Although PROs are included in composite measures currently used to evaluate patients with RA, they do not fully address the needs of patients for several reasons. In clinical trials, many PROs are used to provide a comprehensive assessment of symptoms, functional status and HRQOL. In clinical practice, however, the number of PROs included in composite measures is limited and they do not reflect aspects of the disease that are important from the patient perspective, such as fatigue, sleep disturbance and psychological aspects [
11,
15]. Studies have also shown that many patients in DAS28 remission are not pain-free, suggesting that, in such cases, perceived pain may be non-inflammatory in origin or reflect central sensitization [
23,
24]. In addition, composite scores used to assess disease activity do not provide information about disability [
11]. The PGA, which is the main PRO in ACR- and EULAR-endorsed instruments for measuring disease activity and patient functioning, is also not without limitations. These include lack of gold-standards for PGA wording/phrasing, assessment period and scoring system; broad concepts that may cause interpretation difficulties; and being affected by patient factors unrelated to RA itself, such as level of education, psychological distress and comorbidities [
21]. Furthermore, although the PGA is included in the DAS28, its weighting is low, so its presence does not affect the final DAS28 score to any great extent.
Clearly, it should not be overlooked that treat-to-target strategies provide the best means of addressing inflammation and its consequences in patients with RA; nonetheless, treating-to-target per se may miss opportunities to enhance overall patient well-being through PRO-directed adaptations in disease management. With a treat-to-target approach, many patients will fail to attain low disease activity or remission and in these situations PROs may be particularly useful for guiding treatment decisions, with the aim of improving health outcomes through patient empowerment.
What are the unmet needs of patients with RA and how could use of PROs address them?
The treat-to-target approach in patients with RA focuses on reducing inflammation to prevent joint damage, physical disability and mortality [
1,
2]. However, patients with the disease consider control of pain and fatigue, and maintenance of physical function and HRQOL to be the most important aspects of care [
25‐
29]. A recent literature review of the unmet needs of patients with RA showed that, despite ongoing treatment, many patients still experience significant pain and fatigue, which have a substantial negative impact on HRQOL. Many patients also have mental health symptoms, such as anxiety and depression, that are not being addressed, and may also experience reduced sexual functioning, and social and work participation, which again have a negative impact on HRQOL [
28]. Together, these findings suggest that there may be discordance between the needs of patients with RA and current treatment goals. Indeed, a study assessing discordance between patients’ and physicians’ global assessment of disease activity found that nearly 36% of patients experienced discordance from their rheumatologist [
26]. However, it should be stressed that optimal management of the inflammatory component of RA, particularly when sustained remission is achievable, may be the best means of addressing the long-term needs of individual patients.
The timely and effective use of PROs could bridge the gap between HCP and patient perspectives in RA by encouraging HCPs to focus more on individual patient perceptions of disease impact. This in turn would facilitate shared decision making between patients and HCPs, encouraging customized and more comprehensive patient care. In addition, PROs may be used to identify flares, and to indicate when a clinic visit is necessary when patients can no longer self-manage their condition. However, PROs will only add value if they inform a management decision that helps to improve patient overall well-being and provide information that rheumatologists and other HCPs would otherwise be unable to capture (e.g. disease activity status between clinic visits), and if obtaining the PRO data and acting on the information obtained do not add to rheumatologist workload. The involvement of a multidisciplinary team in patient care can aid in this. Furthermore, some symptoms, such as fatigue, are not easy to manage, and attempts to do so could prove problematic because this symptom is affected by many factors (e.g. systemic inflammation, comorbidities, sleep quality, physical functioning, mood, pain and sense of control), and interplay between these factors is poorly understood [
30]. Currently available PRO instruments could be used to achieve the goals mentioned above. However, additional PROs may be useful to help us understand the extent to which pain is driven by inflammation, and the role of pain and fatigue in causing disability and vice versa.