Patient‐reported outcomes for arthritis: Time to focus on personal life impact measures?

Only patients themselves can report their perspective on the outcomes of illness and its treatment. For some treatment effects, the patient may be the only source of information. Therefore, patient-reported outcomes (PROs) are standard measures of pain and have become standards for functional disability and global health/disease activity due to their inclusion in American College of Rheumatology (ACR) criteria and other disease activity measures (1,2). They have been shown to be as effective as the traditional physicianor laboratory-reported outcomes in reflecting changes in disease activity over time and predicting longterm morbidity and mortality (3). In addition, there are a range of health-related quality of life instruments to measure the broader impact of conditions (4). However, these currently used PROs are not necessarily synonymous with the patient perspective. The US Food and Drug Administration, recognizing the need to incorporate this wider patient perspective in drug evaluation programs, has called for (and issued draft guidance on) standardization of methods for developing PROs that incorporate the patient perspective (5). We believe that the development of measures across rheumatic conditions and a wide range of interventions will be beneficial. Examining the patient’s experience, as reported in new data from the BeST study in this issue of Arthritis Care & Research (including patient-reported functional difficulties) (6), may be particularly crucial in the context of chronic conditions that have a major impact on patients but are only partially treatable, which is a common situation in rheumatology. The multidisciplinary team intervenes not only to change the disease process itself, but also to ameliorate the shortand long-term consequences of the condition. This can be achieved through other therapeutic measures (such as compensating for loss of function by increasing general fitness) or through changes in personal or social circumstances (such as education about appropriate domestic arrangements or altering the living environment). PROs have increasingly been used in rheumatology to test treatment strategies in clinical trials where interventions may be close to the disease pathology, but PROs have a much wider use in health-related domains such as informing individual patient care in a routine practice setting, in health policy and resource allocation, in the measurement of health care quality (7), and capturing participation in society (8). The patient’s perspective of the impact of rheumatic conditions is broad (9), whereas the clinician tends to focus on pathology and functional disability. The growing amount of literature on the patient perspective has revealed 2 important issues: clinicians and patients have different perspectives on outcomes (10), and patients prioritize treatment outcomes that are not routinely measured, such as well-being, normality, and sleep (11–14). Differences between what clinicians and patients believe is important are sometimes due to prioritizing treatment of disease over its consequences, and/or a lack of research about particular aspects of the disease. For example, the significance or multidimensionality of fatigue in rheumatoid arthritis (RA) was not recognized by health professionals (15), nor was dyscognition in fibromyalgia (“fibro-fog”) (16). Mapping the experience of symptoms in detail, the impact of conditions on different domains of life, and patients’ important treatment outcomes may enhance rheumatology research at many levels, from the underlying mechanisms of the disease to the broad social impact. First, understanding the experience of symptoms in detail may provide new insights into the mechanisms of the disease and/or provide evidence for the cohesion (or otherwise) of symptoms caused by different pathologic processes. For example, morning stiffness was improved by time-released glucocorticoids when other symptoms remained unchanged (17). Second, discrepancies between the patient’s experience and apparent disease control may be identified. Treatment may result in a clinically significant reduction in the Disease Activity Score (DAS), but not change in a patient’s important outcomes, such as being able to climb stairs. Third, patient-clinician communication may be improved by establishing the different ways in which common terms, such as flare, are used (18), or the Tessa Sanderson, MSc: University of West England, Bristol, UK; John Kirwan, MD: University of Bristol, Bristol, UK. Address correspondence to John Kirwan, MD, Division of Medicine, Bristol Royal Infirmary, Marlborough Street, Bristol, Avon, UK BS2 8HW. E-mail: john.kirwan@bristol. ac.uk. Submitted for publication October 6, 2008; accepted in revised form October 20, 2008. Arthritis & Rheumatism (Arthritis Care & Research) Vol. 61, No. 1, January 15, 2009, pp 1–3 DOI 10.1002/art.24270 © 2009, American College of Rheumatology