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.
Louise Klokker, Peter Tugwell, Daniel E Furst, Dan Devoe, Paula Williamson, Caroline B Terwee, Maria E Suarez-Almazor, Vibeke Strand, Thasia Woodworth, Amye L Leong, Niti Goel, Maarten Boers, Peter M Brooks, Lee S Simon, Robin Christensen, Caroline Flurey, Marieke Voshaar and Pam Richards
Disease Category: Rheumatology
Disease Name: Inflammatory arthritis
Age Range: 18 - 100
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Consumers (patients)
- Epidemiologists
- Pharmaceutical industry representatives
- Policy makers
- Regulatory agency representatives
- Researchers
- COS for clinical trials or clinical research
- COS for practice
- Focus group(s)
- Systematic review
- Consensus meeting
- Interview
This is a mixed-methods study incorporating four distinct phases. The first phase includes a scoping review of the scientific literature to identify domains included in outcome measures developed for use in rheumatology clinical trials. The second phase includes a systematic literature review identifying harms reported in clinical trials, that are appropriate for patients’ own reports. The third phase involves focus groups and individual interviews with patients diagnosed with an inflammatory arthritis condition, to determine which outcome domains are important to them, and to allow for patients’ perspective on identified symptomatic side effects. The final phase of the study involves face-to-face meetings with stakeholders including patients, aiming for consensus that the project can move forward to developing or selecting outcome measurement instruments for safety – this phase also includes a developing a definition for patients’ perception of safety.