Health Equity Considerations for Developing and Reporting Patient-reported Outcomes in Clinical Trials: A Report from the OMERACT Equity Special Interest Group.

OBJECTIVE: Despite advances integrating patient-centered outcomes into rheumatologic studies, concerns remain regarding their representativeness across diverse patient groups and how this affects equity. The Outcome Measures in Rheumatology (OMERACT) Equity Working Group aims to determine whether and how to address equity issues within the core outcome sets of domains and instruments. METHODS: We surveyed current and previous OMERACT meeting attendees and members of the Campbell and Cochrane Equity Group regarding whether to address equity issues within the OMERACT Filter 2.0 Core Outcome Sets and how to assess the appropriateness of domains, instruments, and measurement properties among diverse patients. At OMERACT 2016, results of the survey and a narrative review of differential psychosocial effects of rheumatoid arthritis (i.e., on men) were presented to stimulate discussion and develop a research agenda. RESULTS: We proposed 6 moments for which an equity lens could be added to the development, selection, or testing of patient-reported outcome measures (PROM): (1) recruitment, (2) domain selection, (3) feasibility in diverse settings, (4) instrument validity, (5) thresholds of meaning, and (6) consideration of statistical power of subgroup analyses for outcome reporting. CONCLUSION: There is a need to (1) conduct a systematic review to assess how equity and population characteristics have been considered in PROM development and whether these differences influence the ranking of importance of outcome domains or a patient's response to questionnaire items, and (2) conduct the same survey described above with patients representing groups experiencing health inequities.

Toward Ensuring Health Equity: Readability and Cultural Equivalence of OMERACT Patient-reported Outcome Measures.

OBJECTIVE: The goal of the Outcome Measures in Rheumatology (OMERACT) 12 (2014) equity working group was to determine whether and how comprehensibility of patient-reported outcome measures (PROM) should be assessed, to ensure suitability for people with low literacy and differing cultures. METHODS: The English, Dutch, French, and Turkish Health Assessment Questionnaires and English and French Osteoarthritis Knee and Hip Quality of Life questionnaires were evaluated by applying 3 readability formulas: Flesch Reading Ease, Flesch-Kincaid grade level, and Simple Measure of Gobbledygook; and a new tool, the Evaluative Linguistic Framework for Questionnaires, developed to assess text quality of questionnaires. We also considered a study assessing cross-cultural adaptation with/without back-translation and/or expert committee. The results of this preconference work were presented to the equity working group participants to gain their perspectives on the importance of comprehensibility and cross-cultural adaptation for PROM. RESULTS: Thirty-one OMERACT delegates attended the equity session. Twenty-six participants agreed that PROM should be assessed for comprehensibility and for use of suitable methods (4 abstained, 1 no). Twenty-two participants agreed that cultural equivalency of PROM should be assessed and suitable methods used (7 abstained, 2 no). Special interest group participants identified challenges with cross-cultural adaptation including resources required, and suggested patient involvement for improving translation and adaptation. CONCLUSION: Future work will include consensus exercises on what methods are required to ensure PROM are appropriate for people with low literacy and different cultures.

The OMERACT First-time Participant ("Newbie") Program: Initial Assessment and Lessons Learned.

OBJECTIVE: To describe the experience of a first-time participant ("newbie") training program at the Outcome Measures in Rheumatology 12 meeting in 2014. METHODS: We conducted newbie sessions at OMERACT 12, including a 2-hour introductory session on Day 1, followed by 1-h evening followup sessions on days 1-4 of OMERACT 12. Pre- and postmeeting surveys assessed participants' level of comfort with the principles of the OMERACT Filters 1.0 (truth, discrimination, feasibility), and Filter 2.0 (the essential tools for OMERACT methodology), the different types of OMERACT sessions, and whether participants felt welcome. RESULTS: In all, 25 new attendees participated in the introductory session and 10-16 attended followup sessions. Fewer participants reported being somewhat or extremely uncomfortable with the meeting, comparing Day 1 (preintroductory session) to days 1-4 (post): (1) with different OMERACT sessions: 56% (pre) versus 6%, 0%, 8%, and 6% (post days 1-4, respectively); and (2) with principles of the OMERACT filter, 64% (pre) versus 7%, 0%, 8%, and 0% (post), respectively. Most reported feeling welcome (100%) and that they were able to contribute substantively to breakout sessions (87%) on Day 1 evening; results were sustained on days 2-4. CONCLUSION: First-time participant training sessions increased the comfort level of the participants with the OMERACT meeting structure and filter, and increased the ability of the new attendees to feel they could contribute to the OMERACT process.

The association of fatigue, comorbidity burden, disease activity, disability and gross domestic product in patients with rheumatoid arthritis. Results from 34 countries participating in the Quest-RA program.

OBJECTIVES: The aim is to assess the prevalence of comorbidities and to further analyse to which degree fatigue can be explained by comorbidity burden, disease activity, disability and gross domestic product (GDP) in patients with rheumatoid arthritis (RA). METHODS: Nine thousands eight hundred seventy-four patients from 34 countries, 16 with high GDP (>24.000 US dollars [USD] per capita) and 18 low-GDP countries (<24.000 USD) participated in the Quantitative Standard monitoring of Patients with RA (QUEST-RA) study. The prevalence of 31 comorbid conditions, fatigue (0-10 cm visual analogue scale [VAS] [10=worst]), disease activity in 28 joints (DAS28), and physical disability (Health Assessment Questionnaire score [HAQ]) were assessed. Univariate and multivariate linear regression analyses were performed to assess the association between fatigue and comorbidities, disease activity, disability and GDP. RESULTS: Overall, patients reported a median of 2 comorbid conditions of which hypertension (31.5%), osteoporosis (17.6%), osteoarthritis (15.5%) and hyperlipidaemia (14.2%) were the most prevalent. The majority of comorbidities were more common in high-GDP countries. The median fatigue score was 4.4 (4.8 in low-GDP countries and 3.8 in high-GDP countries, p<0.001). In low-GDP countries 25.4% of the patients had a high level of fatigue (>6.6) compared with 23.0% in high-GDP countries (p<0.001). In univariate analysis, fatigue increased with increasing number of comorbidities, disease activity and disability in both high- and low-GDP countries. In multivariate analysis of all countries, these 3 variables explained 29.4% of the variability, whereas GDP was not significant. CONCLUSIONS: Fatigue is a widespread problem associated with high comorbidity burden, disease activity and disability regardless of GDP.

Cost-effectiveness analysis of interferon beta-1b for the treatment of patients with a first clinical event suggestive of multiple sclerosis.

OBJECTIVES: To assess, from a Swedish societal perspective, the cost effectiveness of interferon ß-1b (IFNB-1b) after an initial clinical event suggestive of multiple sclerosis (MS) (ie, early treatment) compared with treatment after onset of clinically definite MS (CDMS) (ie, delayed treatment). METHODS: A Markov model was developed, using patient level data from the BENEFIT trial and published literature, to estimate health outcomes and costs associated with IFNB-1b for hypothetical cohorts of patients after an initial clinical event suggestive of MS. Health states were defined by Kurtzke Expanded Disability Status Scale (EDSS) scores. Model outcomes included quality-adjusted life years (QALYs), total costs (including both direct and indirect costs), and incremental cost-effectiveness ratios. Sensitivity analyses were performed on key model parameters to assess the robustness of model results. RESULTS: In the base case scenario, early IFNB-1b treatment was economically dominant (ie, less costly and more effective) versus delayed IFNB-1b treatment when QALYs were used as the effectiveness metric. Sensitivity analyses showed that the cost-effectiveness results were sensitive to model time horizon. Compared with the delayed treatment strategy, early treatment of MS was also associated with delayed EDSS progressions, prolonged time to CDMS diagnosis, and a reduction in frequency of relapse. CONCLUSION: Early treatment with IFNB-1b for a first clinical event suggestive of MS was found to improve patient outcomes while controlling costs.

Real-Time Contrast Echo Assessment of Myocardial Perfusion at Low Emission Power: First Experimental and Clinical Results Using Power Pulse Inversion Imaging.

Power pulse inversion (PPI) has been developed for echocontrast specific imaging in order to reduce destruction of microbubbles. The purpose of this study was to evaluate PPI for real-time contrast echocardiography. Therefore, in vitro studies in a physiological flow-phantom and clinical examinations in patients with coronary artery disease were performed. The in vitro rersults of this study indicate that PPI allows real-time imaging at low emission power and is almost nondestructive to contrast microbubbles of Definity. At this low emission power a strong linear relationship between the dosage of the contrast agent and the resulting PPI signal intensity was found (R = 0.998, p < 0.001). In the clinical examinations real-time imaging using low mechanical index PPI resulted in strong myocardial signals and a complete filling of the cavities indicating absence of bubble destruction. Most striking was the ability of PPI to display myocardial thickening and wall motion simultaneously with the assessment of myocardial contrast replenishment following ultrasound induced bubble destruction by high power frames. We conclude that PPI allows nondestructive contrast imaging both in experimental and clinical settings. Therefore, real-time imaging of myocardial perfusion and real-time assessment of contrast replenishment following ultrasound induced destruction of microbubbles is feasible. Moreover, PPI allows simultaneous assessment of perfusion and myocardial function.