Improving benefit-harm assessment of glucocorticoid therapy incorporating the patient perspective: The OMERACT glucocorticoid core domain set.

OBJECTIVE: Our primary objective was to develop an Outcome Measures in Rheumatology (OMERACT) core domain set to capture the impact of glucocorticoids (GC), both positive and negative, on patients with Rheumatic conditions. METHODS: The OMERACT Filter 2.1 was used to guide core domain selection. Systematic literature reviews, qualitative studies and quantitative surveys were conducted by the OMERACT GC Impact working group to identify candidate domains for a core domain set. A summary of prior work and Delphi exercise were presented at the OMERACT 2020 virtual GC workshop. A proposed GC Impact core domain set derived from this work was presented for discussion in facilitated breakout groups. Participants voted on the proposed GC Impact core domain set. RESULTS: 113 people, including 23 patient research partners, participated in two virtual workshops conducted at different times on the same day. The proposed mandatory domains to be evaluated in clinical trials involving GCs were: infection, bone fragility, hypertension, diabetes, weight, fatigue, mood disturbance and death. In addition, collection of disease specific outcomes was included in the core domain set as "mandatory in specific circumstances". The proposed core domain set was endorsed by 100% (23/23) of the patient research partners and 92% (83/90) of the remaining participants, including clinicians, researchers and industry stakeholders. CONCLUSION: A GC Impact core domain set was endorsed at the OMERACT 2020 virtual workshop. The OMERACT GC Impact working group will now progress to identify, develop and validate measurement tools to best address these domains in clinical trials.

Patient preferences to value health outcomes in rheumatology clinical trials: Report from the OMERACT special interest group✰.

OBJECTIVE: To inform a research plan for future studies by obtaining stakeholder input on the application of preference-based methods to clinical trial design. METHODS: We conducted a virtual OMERACT session to encourage stakeholder engagement. We developed materials for the session to facilitate discussion based on identified case examples and feedback sessions. RESULTS: Participants prioritized incorporating patient preferences in all aspects of trial design with an emphasis on outcome selection. Participants highlighted the need for careful consideration around preference heterogeneity and equity factors. CONCLUSION: Including patient preferences in trial design was considered a priority requiring further exploration to develop comprehensive guidance.

Economic aspects of treatment options in rheumatoid arthritis: a systematic literature review informing the EULAR recommendations for the management of rheumatoid arthritis.

OBJECTIVE: To review the cost effectiveness of rheumatoid arthritis (RA) treatments and inform the clinical recommendations by the European League Against Rheumatism. METHODS: A systematic literature search and review of the health economic evidence on RA treatment options was performed. RESULTS: Despite diverse methodological approaches, health economic analyses are concordant: at onset of disease, traditional disease-modifying antirheumatic drugs (DMARDs) are cost effective-that is, treatment merits outweigh treatment costs. If DMARDs fail, therapeutic escalation with tumour necrosis factor alpha inhibitors (TNFi) is cost effective when standard dosing schemes are employed. If TNFi fail, rituximab or abatacept is cost effective. Economic evidence for switching TNFi remains sparse. CONCLUSIONS: The costly sequelae of insufficiently controlled RA justify intensive escalations of treatment in this disease. By maintaining function, patients are kept in the work process, reducing indirect costs. Quality of life is improved at an expense commonly accepted for chronic diseases. Effective control of disease activity seems to be a prudent use of societal resources.

Progress on incorporating the patient perspective in outcome assessment in rheumatology and the emergence of life impact measures at OMERACT 9.

The Patient Perspective Workshop included over 100 researchers and 18 patient participants from 8 countries. Following preconference reading and short plenary presentations, breakout groups considered work undertaken on measurement of sleep, assessing interventions to develop the effective consumer, and assessing psychological and educational interventions. The workshop explored the best way to identify other outcome domains (and instruments) that should be measured in observational or interventional studies with broader intentions than simply altering outcomes captured in the traditional "core set" plus fatigue. Four sleep questionnaires showed promise and will be the subject of further study. The Effective Consumer scale (EC-17) was reviewed and the concept Effective Consumer was well received. Participants thought it worthwhile to measure the skills and attributes of an effective consumer and develop an intervention that would include education in all of the scale's categories. Assessment of educational and psychological interventions requires a wider set of instruments than is currently used; these should relate to the purpose of the intervention. This principle was extended to include wider measures of the impact of disease on life, as indicated in the International Classification of Functioning, Disability and Health. Life impact measure sets covering domains appropriate to different rheumatic conditions and focused on different interventions might be defined by future OMERACT consensus. Measurement instruments within these domains that are valid for use in rheumatic conditions can then be identified and, in the case of psychological and educational interventions, chosen to fit with the purpose of the intervention.