Patient perspective of measuring treatment efficacy: The Rheumatoid Arthritis Patient Priorities for Pharmacological Interventions (RAPP-PI) outcomes

Abstract/Description

Background: Collaboration with patients with rheumatoid arthritis (RA) highlights that outcomes important to them include fatigue, coping and life enjoyment. However, these are not commonly measured in clinical trials. There is little evidence about which outcomes patients would prioritise, nor what factors influence patients’ prioritisation. Objective: To develop a complementary core set with patients to promote inclusion of their priority outcomes in pharmacological interventions. Methods: Nominal groups were conducted with RA patients to rank 63 outcomes generated from previous in-depth interviews. A multi-centre postal survey provided the final selection of core outcomes for the RAPP-PI, in which RA patients rated the importance of the priority outcomes from the nominal groups and ranked the top 6. Results: 26 patients participated in 5 nominal group discussions, and reduced the 63 initial outcomes to 32 most important. 254 participants in the survey ranked priority treatment outcomes to form the RAPP-PI: pain, activities of daily living, joint damage, mobility, life enjoyment, independence, fatigue, and valued activities. The 8 priorities represent three domains of treatment outcomes: direct impact of RA, psychosocial well-being, and function/participation. Chi-squared tests showed that disease severity, disease duration, gender and patients’ perceptions of managing, self-efficacy and normality influenced the selection of priority treatment outcomes. Conclusion: Collaboration with patients has captured their perspective of priority outcomes from pharmacological interventions. Whilst there is some overlap with professional core outcomes, the additional use of this complementary set will give a broader evaluation of effectiveness of interventions from the key stakeholders: patients.