What outcomes from pharmacologic treatments are important to people with rheumatoid arthritis? Creating the basis of a patient core set

Abstract

Objective. Function, patient global assessment, and pain are routinely measured in rheumatoid arthritis (RA) clinical trials. However, other patient-reported outcomes identified as important to patients in qualitative studies, such as fatigue and quality of life, are commonly not included, and modern treatment regimens may have changed patients' expectations of treatment outcomes. Our objective was to elicit patient priority treatment outcomes for pharmacologic interventions since the common use of anti-tumor necrosis factor (anti-TNF) therapy, which will form the basis of a core set of patient priorities to complement existing professional core sets. Methods. In-depth interviews were conducted with 23 RA patients, purposively sampled for age, sex, medication (anti-TNF or other disease-modifying antirheumatic drugs), disease severity, and work status. Grounded theory guided iterative data collection and analysis. Coding of the data was peer reviewed. A patient research partner collaborated in the research design and analysis. Results. Sixty-three different outcomes important to patients were generated from the interviews. Four major categories of patient outcomes from pharmacologic treatments were developed: "RA under control," "Doing things," "Emotional health," and "Coping with illness." The core category (or overall theme) was "Minimizing the personal impact of RA." Conclusion. Although the routine outcomes of pain, function, and overall well-being were raised by the patients, they also generated a further 60 important outcomes that they look for from treatment. This difference in perspective may potentially influence treatment decisions. The next step is therefore to use these data to develop a patient core set.

Polish Rheumatologists and Rheumatoid Arthritis Patients Differ in Their Opinions About Treatment Inefficacy

“…For patients, more understandable treatment goals are relief of physical symptoms and achieving optimal physical and social functioning. 11,12 Our study shows that joint swelling without pain or progression of erosions sometimes is not linked to disease activity by patients. Particularly, older patients may not perceive abnormal joints or be unaware of the importance of the presence of swollen joints in the assessment of RA activity.…”

Item Development and Face Validity of the Rheumatoid Arthritis Patient Priorities in Pharmacological Interventions Outcome Measures

“…The priority of Visible joint damage provided the greatest challenge because no existing instrument could be found that measured patients' selfreporting of joint damage. The three NRS were developed from previous interview data [17], and discussed during the consultation meetings and focus groups. A minority of Phase 1 participants were concerned about the effect of the term 'joint damage' on newly diagnosed patients, but the overall consensus was that RA patients should be realistic about the potential consequences of progression of their disease and the implications for measurement were more important.…”

“…A strength is that these new outcome measures are based on the development of a conceptual framework grounded in patient data from a previous study [17], in collaboration with patients as recommended by the FDA [10], and benefiting from a higher level of user involvement [47]. Further research aims to construct a final tool, which will assess priorities important to patients in one instrument, constructing an algorithm from observational data to produce a single score.…”

“…For example, different understandings of the word 'mobility' were apparent: "Moving around", "Get from room to room", "Doing your chores", "Movement in your arm" and "Dexterity". There was a consensus from participants that the phrase 'getting around' should be used to convey the original meaning of the priority from the previous interview data [17].…”

“…First, differences have been shown between physician and patient assessments of outcomes such as pain, overall health, and physical and mental function [14]. Second, RA patients identify treatment outcomes that are not routinely measured, such as sleep, well-being, and normality [15][16][17], which may relate to disease activity.…”