Evolving and evaluating the OMERACT fellows program: insights and implications from OMERACT 2023 fellows

Objective: To describe the evolution of the OMERACT Fellows Program (OM FP) and to evaluate the innovative changes implemented in the 2023 program. Methods: The OM FP, the first of its kind in global rheumatology, was developed in 2000 to mentor early career researchers in methods and processes for reaching evidence-driven consensus for outcome measures in clinical studies. The OM FP has evolved through continuing iterations of face to face and online feedback. Key new features delivered in 2023 included e-learning modules, virtual introductory pre-meetings, increased networking with Patient Research Partners (PRPs), learning opportunities to give and receive personal feedback, ongoing performance feedback during the meeting from Fellow peers, PRPs, senior OMERACTers (members of the OMERACT community) and Emerging Leader mentors, involvement in pitching promotions, two-minute Lightning Talks in a plenary session and an embedded poster tour. An online survey was distributed after the meeting to evaluate the program. Results: OM FP has included 208 fellows from 16 countries across 4 continents covering 47 different aspects of rheumatology outcomes since its inception. Over 50 % have remained engaged with OMERACT work. In 2023, 18 Fellows attended and 15 (83 %) completed the post-meeting survey. A dedicated OM FP was deemed important by all respondents, and 93 % would attend the meeting in future. The PRP/Fellow Connection Carousel and Lightning Talks were rated exceptional by 93 %. Key components to improve included clarification of expectations, overall workload, the Emerging Leaders Mentoring Program, and the content and duration of daily summary sessions. Conclusion: The innovations in the 2023 OM FP were well received by the majority of participants and supports early career rheumatology researchers to develop collaborations, skills and expertise in outcome measurement. Implementation of feedback from Fellows will enhance the program for future meetings, continuing to facilitate learning and succession planning within OMERACT

Treatment effect sizes vary in randomized trials depending on the type of outcome measure

Objective: To compare estimated treatment effects of physical therapy (PT) between patient-reported outcome measures (PROMs) and outcomes measured in other ways. Study Design and Setting: We selected randomized trials of PT with both a PROM and a non-PROM included in Cochrane systematic reviews (CSRs). Two reviewers independently extracted data and risk-of-bias assessments. Our primary outcome was the ratio of odds ratios (RORs), used to quantify how effect varies between PROMs and non-PROMs; an ROR > 1 indicates larger effect when assessed by using PROMs. We used REML-methods to estimate associations of trial characteristics with effects and between-trial heterogeneity. Results: From 90 relevant CSRs, 205 PT trials were included. The summary ROR across all the comparisons was not statistically significant (ROR, 0.88 [95% CI: 0.70–1.12]; P = 0.30); however, the heterogeneity was substantial (I2 = 88.1%). When stratifying non-PROMs further into clearly objective non-PROMs (e.g., biomarkers) and other non-PROMs (e.g., aerobic capacity), the PROMs appeared more favorable than did clearly objective non-PROMs (ROR, 1.92 [95% CI: 0.99–3.72]; P = 0.05). Conclusion: Estimated treatment effects based on PROMs are generally comparable with treatment effects measured in other ways. However, in our study, PROMs indicate a more favorable treatment effect compared with treatment effects based on clearly objective outcomes

‘I couldn’t carry on taking a drug like that’::a qualitative study of patient perspectives on side effects from rheumatology drugs

Objectives: There is growing interest in collecting outcome information directly from patients in clinical trials. This study evaluates what patients with rheumatic and musculoskeletal diseases (RMDs) consider important to know about symptomatic side effects they may experience from a new prescription drug.Methods: Patients with inflammatory arthritis, who had one or more prescribed drugs for their disease for at least 12 months, participated in focus groups and individual interviews. Discussions were analysed using reflexive thematic analysis.Results: We conducted seven focus groups with 34 participants across three continents. We found four overarching and two underpinning themes. The ‘impact on life’ was connected to participants’ ‘daily life’, ‘family life’, ‘work life’ and ‘social life’. In ‘psychological and physical aspects’ participants described ‘limitation to physical function’, ‘emotional dysregulation’ and ‘an overall mental state’. Extra tests, hospital visits and payment for medication were considered a ‘time, energy and financial burden’ of side effects. Participants explained important measurement issues to be ‘severity’, ‘frequency’ and ‘duration’. Underpinning these issues, participants evaluated the ‘benefit–harm balance’ which includes ‘the cumulative burden’ of having several side effects and the persistence of side effects over time.Conclusions: In treatment for RMDs, there seems to be an urgent need for feasible measures of patient-reported bother (impact on life and cumulative burden) from side effects and the benefit–harm balance. These findings contribute new evidence in support of a target domain—an outcome that represents the patient voice evaluating the symptomatic treatment-related side effects for people with RMDs enrolled in clinical trials

‘I couldn’t carry on taking a drug like that’::a qualitative study of patient perspectives on side effects from rheumatology drugs

Objectives: There is growing interest in collecting outcome information directly from patients in clinical trials. This study evaluates what patients with rheumatic and musculoskeletal diseases (RMDs) consider important to know about symptomatic side effects they may experience from a new prescription drug.Methods: Patients with inflammatory arthritis, who had one or more prescribed drugs for their disease for at least 12 months, participated in focus groups and individual interviews. Discussions were analysed using reflexive thematic analysis.Results: We conducted seven focus groups with 34 participants across three continents. We found four overarching and two underpinning themes. The ‘impact on life’ was connected to participants’ ‘daily life’, ‘family life’, ‘work life’ and ‘social life’. In ‘psychological and physical aspects’ participants described ‘limitation to physical function’, ‘emotional dysregulation’ and ‘an overall mental state’. Extra tests, hospital visits and payment for medication were considered a ‘time, energy and financial burden’ of side effects. Participants explained important measurement issues to be ‘severity’, ‘frequency’ and ‘duration’. Underpinning these issues, participants evaluated the ‘benefit–harm balance’ which includes ‘the cumulative burden’ of having several side effects and the persistence of side effects over time.Conclusions: In treatment for RMDs, there seems to be an urgent need for feasible measures of patient-reported bother (impact on life and cumulative burden) from side effects and the benefit–harm balance. These findings contribute new evidence in support of a target domain—an outcome that represents the patient voice evaluating the symptomatic treatment-related side effects for people with RMDs enrolled in clinical trials

Stakeholder endorsement advancing the implementation of a patient-reported domain for harms in rheumatology clinical trials: Outcome of the OMERACT Safety Working Group

Objectives To develop an understanding of the concept of safety/harms experienced by patients involved in clinical trials for their rheumatic and musculoskeletal diseases (RMDs) and to seek input from the OMERACT community before moving forward to developing or selecting an outcome measurement instrument. Methods OMERACT 2023 presented and discussed interview results from 34 patients indicating that up to 171 items might be important for patients’ harm-reporting. Results Domain was defined in detail and supported by qualitative work. Participants in the Special-Interest-Group endorsed (96 %) that enough qualitative data are available to start Delphi survey(s). Conclusion We present a definition of safety/harms that represents the patient voice (i.e., patients’ perception of safety) evaluating the symptomatic treatment-related adverse events for people with RMDs enrolled in clinical trials

Core outcome measurement set for shared decision making in rheumatic and musculoskeletal conditions: A scoping review to identify candidate instruments

OBJECTIVES Shared decision making (SDM) is a central tenet in rheumatic and musculoskeletal care. The lack of standardization regarding SDM instruments and outcomes in clinical trials threatens the comparative effectiveness of interventions. The Outcome Measures in Rheumatology (OMERACT) SDM Working Group is developing a Core Outcome Set for trials of SDM interventions in rheumatology and musculoskeletal health. The working group reached consensus on a Core Outcome Domain Set in 2020. The next step is to develop a Core Outcome Measurement Set through the OMERACT Filter 2.2. METHODS We conducted a scoping review (PRISMA-ScR) to identify candidate instruments for Powered by Editorial Manager® and ProduXion Manager® from Aries Systems Corporation the OMERACT Filter 2.2 We systematically reviewed five databases (Ovid MEDLINE®, Embase, Cochrane Library, CINAHL and Web of Science). An information specialist designed search strategies to identify all measurement instruments used in SDM studies in adults or children living with rheumatic or musculoskeletal diseases or their important others. Paired reviewers independently screened titles, abstracts, and full text articles. We extracted characteristics of all candidate instruments (e.g., measured construct, measurement properties). We classified candidate instruments and summarized evidence gaps with an adapted version of the Summary of Measurement Properties (SOMP) table. RESULTS We found 14,464 citations, read 239 full text articles, and included 99 eligible studies. We identified 220 potential candidate instruments. The five most used measurement instruments were the Decisional Conflict Scale (traditional and low literacy versions) (n=38), the Hip/Knee-Decision Quality Instrument (n=20), the Decision Regret Scale (n=9), the Preparation for Decision Making Scale (n=8), and the CollaboRATE (n=8). Only 44 candidate instruments (20%) had any measurement properties reported by the included studies. Of these instruments, only 57% matched with at least one of the 7-criteria adapted SOMP table. CONCLUSION We identified 220 candidate instruments used in the SDM literature among people with rheumatic and musculoskeletal diseases. Our classification of instruments showed evidence gaps and inconsistent reporting of measurement properties. The next steps for the OMERACT SDM Working Group are to match candidate instruments with Core Domains, assess feasibility and review validation studies of measurement instruments in rheumatic diseases or other conditions. Development and validation of new instruments may be required for some Core Domains