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.

Harms reported by patients in rheumatology drug trials: a systematic review of randomized trials in the cochrane library from an OMERACT working group.

BACKGROUND: Underreporting of harms in randomized controlled trials (RCTs) may lead to incomplete or erroneous assessments of the perceived benefit-to-harm profile of an intervention. To compare benefit with harm in clinical practice and future clinical studies, adverse event (AE) profiles including severity need to be understood. Even though patients report harm symptoms earlier and more frequently than clinicians, rheumatology RCTs currently do not provide a reporting framework from the patient's perspective regarding harms. Our objective for this meta-research project was to identify AEs in order to determine harm clusters and whether these could be self-reported by patients. Our other objective was to examine reported severity grading of the reported harms. METHODS: We considered primary publications of RCTs eligible if they were published between 2008 and 2018 evaluating pharmacological interventions in patients with a rheumatic or musculoskeletal condition and if they were included in Cochrane reviews. We extracted data on harms such as reported AE terms together with severity (if described), and categorized AE- and severity-terms into overall groups. We deemed all AEs with felt components appropriate for patient self-reporting. RESULTS: The literature search identified 187 possible Cochrane reviews, of which 94 were eligible for evaluation, comprising 1,297 articles on individual RCTs. Of these RCTs, 93 pharmacological trials met our inclusion criteria (including 31,023 patients; representing 20,844 accumulated patient years), which reported a total of 21,498 AEs, corresponding to 693 unique reported terms for AEs. We further sub-categorized these terms into 280 harm clusters (i.e., themes). AEs appropriate for patient self-reporting accounted for 58% of the AEs reported. Among the reported AEs, we identified medical terms for all of the 117 harm clusters appropriate for patient reporting and lay language terms for 86%. We intended to include severity grades of the reported AEs, but there was no evidence for systematic reporting of clinician- or patient-reported severity in the primary articles of the 93 trials. However, we identified 33 terms suggesting severity, but severity grading was discernible in only 9%, precluding a breakdown by severity in this systematic review. CONCLUSIONS: Our results support the need for a standardized framework for patients' reporting of harms in rheumatology trials. Reporting of AEs with severity should be included in future reporting of harms, both from the patients' and investigators' perspectives. REGISTRATION: PROSPERO: CRD42018108393.

Identifying Possible Outcome Domains from Existing Outcome Measures to Inform an OMERACT Core Domain Set for Safety in Rheumatology Trials.

OBJECTIVE: The Outcome Measures in Rheumatology (OMERACT) Safety Working Group objective was to identify harm domains from existing outcome measurements in rheumatology. METHODS: Systematically searching the MEDLINE database on January 24, 2017, we identified full-text articles that could be used for harm outcomes in rheumatology. Domains/items from the identified instruments were described and the content synthesized to provide a preliminary framework for harm outcomes. RESULTS: From 435 possible references, 24 were read in full text and 9 were included: 7 measurement instruments were identified. Investigation of domains/items revealed considerable heterogeneity in the grouping and approach. CONCLUSION: The ideal way to assess harm aspects from the patients' perspective has not yet been ascertained.

Patient Perspectives on DMARD Safety Concerns in Rheumatology Trials: Results from Inflammatory Arthritis Patient Focus Groups and OMERACT Attendees Discussion.

OBJECTIVE: The Outcome Measures in Rheumatology (OMERACT) Safety Working Group is identifying core safety domains that matter most to patients with rheumatic disease. METHODS: International focus groups were held with 39 patients with inflammatory arthritis to identify disease-modifying antirheumatic drug (DMARD) experiences and concerns. Themes were identified by pragmatic thematic coding and discussed in small groups by meeting attendees. RESULTS: Patients view DMARD side effects as a continuum and consider the cumulative effect on day-to-day function. Disease and drug experiences, personal factors, and life circumstances influence tolerance of side effects and treatment persistence. CONCLUSION: Patients weigh overall adverse effects and benefits over time in relation to experiences and life circumstances.

Improving Benefit-harm Assessment of Therapies from the Patient Perspective: OMERACT Premeeting Toward Consensus on Core Sets for Randomized Controlled Trials.

OBJECTIVE: Outcome Measures in Rheumatology (OMERACT) convened a premeeting in 2018 to bring together patients, regulators, researchers, clinicians, and consumers to build upon previous OMERACT drug safety work, with patients fully engaged throughout all phases. METHODS: Day 1 included a brief introduction to the history of OMERACT and methodology, and an overview of current efforts within and outside OMERACT to identify patient-reported medication safety concerns. On Day 2, two working groups presented results; after each, breakout groups were assembled to discuss findings. RESULTS: Five themes pertaining to drug safety measurement emerged. CONCLUSION: Current approaches have failed to include data from the patient's perspective. A better understanding of how individuals with rheumatic diseases view potential benefits and harms of therapies is essential.

EVOLVE: The Australian Rheumatology Association's 'top five' list of investigations and interventions doctors and patients should question.

BACKGROUND: The EVOLVE (evaluating evidence, enhancing efficiencies) initiative aims to drive safer, higher-quality patient care through identifying and reducing low-value practices. AIMS: To determine the Australian Rheumatology Association's (ARA) 'top five' list of low-value practices. METHODS: A working group comprising 19 rheumatologists and three trainees compiled a preliminary list. Items were retained if there was strong evidence of low value and there was high or increasing clinical use and/or increasing cost. All ARA members (356 rheumatologists and 72 trainees) were invited to indicate their 'top five' list from a list of 12-items through SurveyMonkey in December 2015 (reminder February 2016). RESULTS: A total of 179 rheumatologists (50.3%) and 19 trainees (26.4%) responded. The top five list (percentage of rheumatologists, including item in their top five list) was: Do not perform arthroscopy with lavage and/or debridement for symptomatic osteoarthritis of the knee nor partial meniscectomy for a degenerate meniscal tear (73.2%); Do not order anti-nuclear antibody (ANA) testing without symptoms and/or signs suggestive of a systemic rheumatic disease (56.4%); Do not undertake imaging for low back pain for patients without indications of an underlying serious condition (50.8%); Do not use ultrasound guidance to perform injections into the subacromial space as it provides no additional benefit in comparison to landmark-guided injection (50.3%) and Do not order anti-double-stranded DNA antibodies in ANA negative patients unless the clinical suspicion of systemic lupus erythematosus remains high (45.3%). CONCLUSIONS: This list is intended to increase awareness among rheumatologists, other clinicians and patients about commonly used low-value practices that should be questioned.

Developing an OMERACT Core Outcome Set for Assessing Safety Components in Rheumatology Trials: The OMERACT Safety Working Group.

OBJECTIVE: Failure to report harmful outcomes in clinical research can introduce bias favoring a potentially harmful intervention. While core outcome sets (COS) are available for benefits in randomized controlled trials in many rheumatic conditions, less attention has been paid to safety in such COS. The Outcome Measures in Rheumatology (OMERACT) Filter 2.0 emphasizes the importance of measuring harms. The Safety Working Group was reestablished at the OMERACT 2016 with the objective to develop a COS for assessing safety components in trials across rheumatologic conditions. METHODS: The safety issue has previously been discussed at OMERACT, but without a consistent approach to ensure harms were included in COS. Our methods include (1) identifying harmful outcomes in trials of interventions studied in patients with rheumatic diseases by a systematic literature review, (2) identifying components of safety that should be measured in such trials by use of a patient-driven approach including qualitative data collection and statistical organization of data, and (3) developing a COS through consensus processes including everyone involved. RESULTS: Members of OMERACT including patients, clinicians, researchers, methodologists, and industry representatives reached consensus on the need to continue the efforts on developing a COS for safety in rheumatology trials. There was a general agreement about the need to identify safety-related outcomes that are meaningful to patients, framed in terms that patients consider relevant so that they will be able to make informed decisions. CONCLUSION: The OMERACT Safety Working Group will advance the work previously done within OMERACT using a new patient-driven approach.

Training a system-literate care coordination workforce.

People with chronic complex conditions continue to experience increasing health system fragmentation and poor coordination. To reverse these trends, one solution has been an investment in effective models of care coordination that use a care coordinator workforce. Care coordinators are not a homogenous workforce - but an applied professional role, providing direct and indirect care, and is often undertaken by nurses, allied health professionals, social workers or general practitioners. In Australia, there is no training curriculum nor courses, nor nationally recognised professional quality standards for the care coordinator workforce. With the growing complexity and fragmentation of the health care system, health system literacy - shared understanding of the roles and contributions of the different workforce professions, organisations and systems, among patients and indeed the health workforce is required. Efforts to improve health system literacy among the health workforce are increasing at a policy, practice and research level. However, insufficient evidence exists about what are the health system literacy needs of care coordinators, and what is required for them to be most effective. Key areas to build a health system literate care coordination workforce are presented. Care coordination is more than an optional extra, but one of the only ways we are going to be able to provide equitable health services for people with chronic complex conditions. People with low health literacy require more support with the coordination of their care, therefore we need to build a high performing care coordinator workforce that upholds professional quality standards, and is health literacy responsive.

How to choose core outcome measurement sets for clinical trials: OMERACT 11 approves filter 2.0.

OBJECTIVE: The Outcome Measures in Rheumatology (OMERACT) initiative works to develop core sets of outcome measures for trials and observational studies in rheumatology. At the OMERACT 11 meeting, substantial time was devoted to discussing a conceptual framework and a proposal for a more explicit working process to develop what we now propose to term core outcome measurement sets, collectively termed "OMERACT Filter 2.0." METHODS: Preconference work included a literature review, and discussion of preliminary proposals through face-to-face discussions and Internet-based surveys with people within and outside rheumatology. At the conference, 5 interactive sessions comprising plenary and small-group discussions reflected on the proposals from the viewpoint of previous and ongoing OMERACT work. These considerations were brought together in a final OMERACT presentation seeking consensus agreement for the Filter 2.0 framework. RESULTS: After debate, clarification, and agreed alterations, the final proposal suggested all core sets should contain at least 1 measurement instrument from 3 Core Areas: Death, Life Impact, and Pathophysiological Manifestations, and preferably 1 from the area Resource Use. The process of core set development for a health condition starts by selecting core domains within the areas ("core domain set"). This requires literature searches, involvement (especially of patients), and at least 1 consensus process. Next, developers select at least 1 applicable measurement instrument for each core domain. Applicability refers to the original OMERACT Filter and means that the instrument must be truthful (face, content, and construct validity), discriminative (between situations of interest) and feasible (understandable and with acceptable time and monetary costs). Depending on the quality of the instruments, participants formulate either a preliminary or a final "core outcome measurement set." At final vote, 96% of participants agreed "The proposed overall framework for Filter 2.0 is a suitable basis on which to elaborate a Filter 2.0 Handbook." CONCLUSION: Within OMERACT, Filter 2.0 has made established working processes more explicit and includes a broadly endorsed conceptual framework for core outcome measurement set development.

Updating the OMERACT filter: implications for imaging and soluble biomarkers.

OBJECTIVE: The Outcome Measures in Rheumatology (OMERACT) Filter provides a framework for the validation of outcome measures for use in rheumatology clinical research. However, imaging and biochemical measures may face additional validation challenges because of their technical nature. The Imaging and Soluble Biomarker Session at OMERACT 11 aimed to provide a guide for the iterative development of an imaging or biochemical measurement instrument so it can be used in therapeutic assessment. METHODS: A hierarchical structure was proposed, reflecting 3 dimensions needed for validating an imaging or biochemical measurement instrument: outcome domain(s), study setting, and performance of the instrument. Movement along the axes in any dimension reflects increasing validation. For a given test instrument, the 3-axis structure assesses the extent to which the instrument is a validated measure for the chosen domain, whether it assesses a patient-centered or disease-centered variable, and whether its technical performance is adequate in the context of its application. Some currently used imaging and soluble biomarkers for rheumatoid arthritis, spondyloarthritis, and knee osteoarthritis were then evaluated using the original OMERACT Filter and the newly proposed structure. Breakout groups critically reviewed the extent to which the candidate biomarkers complied with the proposed stepwise approach, as a way of examining the utility of the proposed 3-dimensional structure. RESULTS: Although there was a broad acceptance of the value of the proposed structure in general, some areas for improvement were suggested including clarification of criteria for achieving a certain level of validation and how to deal with extension of the structure to areas beyond clinical trials. CONCLUSION: General support was obtained for a proposed tri-axis structure to assess validation of imaging and soluble biomarkers; nevertheless, additional work is required to better evaluate its place within the OMERACT Filter 2.0.

Updating the OMERACT filter: implications for patient-reported outcomes.

OBJECTIVE: At a previous Outcome Measures in Rheumatology (OMERACT) meeting, participants reflected on the underlying methods of patient-reported outcome (PRO) instrument development. The participants requested proposals for more explicit instrument development protocols that would contribute to an enhanced version of the "Truth" statement in the OMERACT Filter, a widely used guide for outcome validation. In the present OMERACT session, we explored to what extent these new Filter 2.0 proposals were practicable, feasible, and already being applied. METHODS: Following overview presentations, discussion groups critically reviewed the extent to which case studies of current OMERACT Working Groups complied with or negated the proposed PRO development framework, whether these observations had a more general application, and what issues remained to be resolved. RESULTS: Several aspects of PRO development were recognized as particularly important, and the need to directly involve patients at every stage of an iterative PRO development program was endorsed. This included recognition that patients contribute as partners in the research and not merely as subjects. Correct communication of concepts with the words used in questionnaires was central to their performance as measuring instruments, and ensuring this understanding crossed cultural and linguistic boundaries was important in international studies or comparisons. CONCLUSION: Participants recognized, endorsed, and were generally already putting into practice the principles of PRO development presented in the plenary session. Further work is needed on some existing instruments and on establishing widespread good practice for working in close collaboration with patients.

A framework to support team-based models of primary care within the Australian health care system.

Health systems with strong primary care orientations are known to be associated with improved equity, better access for patients to appropriate services at lower costs, and improved population health. Team-based models of primary care have emerged in response to health system challenges due to complex patient profiles, patient expectations and health system demands. Successful team-based models of primary care require a combination of interprofessional education and learning; organisational and management policies and systems; and practice support systems. To ensure evidence is put into practice, we propose a framework comprising five domains (theory, implementation, infrastructure, sustainability and evaluation) to assist policymakers, educators, researchers, managers and health professionals in supporting team-based models of primary care within the Australian health care system.

Improving musculoskeletal health: global issues.

Musculoskeletal (MSK) disorders are among the leading reasons why patients consult a family or primary health practitioner, take time off work and become disabled. Many of the MSK disorders are more common in the elderly. Thus, as the proportion of the elderly increases all over the world, MSK disorders will make a greater contribution to the global burden of disease. Epidemiological studies have shown that the spectrum of MSK disorders in developing countries is similar to that seen in industrialised countries, but the burden of disease tends to be higher due to a delay in diagnosis or lack of access to adequate health-care facilities for effective treatment. Musculoskeletal pain is very common in the community while fibromyalgia is being recognised as part of a continuum of chronic widespread pain rather than a narrowly defined entity. This will allow research to improve our understanding of pain in a variety of diffuse pain syndromes. The availability of newer more effective therapies has resulted in efforts to initiate therapy at an earlier stage of diseases. The new criteria for rheumatoid arthritis, and the diagnosis of axial and peripheral involvement in spondyloarthritis, permit an earlier diagnosis without having to wait for radiological changes. One of the major health challenges is the global shortage of health workers, and based on current training of health workers and traditional models of care for service delivery, the global situation is unlikely to change in the near future. Thus, new models of care and strategies to train community health-care workers and primary health-care practitioners to detect and initiate the management of patients with MSK disorders at an earlier stage are required. There is also a need for prevention strategies with campaigns to educate and raise awareness among the entire population. Lifestyle interventions such as maintaining an ideal body weight to prevent obesity, regular exercises, avoidance of smoking and alcohol abuse, intake of a balanced diet and nutrients to include adequate calcium and vitamin D, modification of the work environment and avoidance of certain repetitive activities will prevent or ameliorate disorders such as osteoarthritis, osteoporosis, rheumatoid arthritis, gout and MSK pain syndromes including low back pain and work-related pain syndromes. These prevention strategies also contribute to reducing the prevalence and outcome of diseases such as hypertension, cardiovascular diseases, diabetes and respiratory diseases. Thus, prevention strategies require urgent attention globally.

Taking stock of interprofessional learning in Australia.

Changes in health service delivery and issues of quality of care and safety are driving interprofessional practice, and interprofessional learning (IPL) is now a requirement for medical school accreditation. There is international agreement that learning outcomes frameworks are required for the objectives of IPL to be fully realised, but there is debate about the most appropriate terminology. Interprofessional skills can be gained in several ways - from formal educational frameworks, at pre- and post-registration levels to work-based training. Research activity suggests that many consider that IPL delivers much-needed skills to health professionals, but some systematic reviews show that evidence of a link to patient outcomes is lacking. Australian efforts to develop an evidence base to support IPL have progressed, with new research drawing on recommendations of experts in the area. The focus has now shifted to curriculum development. The extent to which IPL is rolled out in Australian universities will depend on engagement and endorsement from curriculum managers and the broader faculty.

Simulation in clinical teaching and learning.

Simulation-based education (SBE) is a rapidly developing method of supplementing and enhancing the clinical education of medical students. Clinical situations are simulated for teaching and learning purposes, creating opportunities for deliberate practice of new skills without involving real patients. Simulation takes many forms, from simple skills training models to computerised full-body mannequins, so that the needs of learners at each stage of their education can be targeted. Emerging evidence supports the value of simulation as an educational technique; to be effective it needs to be integrated into the curriculum in a way that promotes transfer of the skills learnt to clinical practice. Currently, SBE initiatives in Australia are fragmented and depend on local enthusiasts; Health Workforce Australia is driving initiatives to develop a more coordinated national approach to optimise the benefits of simulation.

Identifying preliminary domains to detect and measure rheumatoid arthritis flares: report of the OMERACT 10 RA Flare Workshop.

BACKGROUND: While disease flares in rheumatoid arthritis (RA) are a recognized aspect of the disease process, there is limited formative research to describe them. METHODS: The Outcome Measures in Rheumatology Clinical Trials (OMERACT) RA Flare Definition Working Group is conducting an international research project to understand the specific characteristics and impact of episodic disease worsening, or "flare," so that outcome measures can be developed or modified to reflect this uncommonly measured, but very real and sometimes disabling RA disease feature. Patient research partners provided critical insights into the multidimensional nature of flare. The perspectives of patients and healthcare and research professionals are being integrated to ensure that any outcome measurement to detect flares fulfills the first OMERACT criteria of Truth. Through an iterative data-driven Delphi process, a preliminary list of key domains has been identified to evaluate flare. RESULTS: At OMERACT 10, consensus was achieved identifying features of flare in addition to the existing core set for RA, including fatigue, stiffness, symptom persistence, systemic features, and participation. Patient self-report of flare was identified as a component of the research agenda needed to establish criterion validity for a flare definition; this can be used in prospective studies to further evaluate the Discrimination and Feasibility components of the OMERACT filter for a flare outcome measure. CONCLUSION: Our work to date has provided better understanding of key aspects of the RA disease process as episodic, potentially disabling disease worsening even when a patient is in low disease activity. It also highlights the importance of developing ways to enhance communication between patients and clinicians and improve the ability to achieve "tight control" of disease.