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

Dorthe B Berthelsen; Lee S Simon; John P A Ioannidis; Marieke Voshaar; Pam Richards; Niti Goel; Vibeke Strand; Sabrina M Nielsen; Beverly J Shea; Peter Tugwell; Susan J Bartlett; Glen S Hazlewood; Lyn March; Jasvinder A Singh; Maria E Suarez-Almazor; Maarten Boers; Randall M Stevens; Daniel E Furst; Thasia Woodworth; Amye Leong; Peter M Brooks; Caroline Flurey; Robin Christensen; OMERACT Safety Working Group

doi:10.1016/j.semarthrit.2023.152288

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

Semin Arthritis Rheum. 2023 Dec:63:152288. doi: 10.1016/j.semarthrit.2023.152288. Epub 2023 Oct 26.

Authors

Dorthe B Berthelsen¹, Lee S Simon², John P A Ioannidis³, Marieke Voshaar⁴, Pam Richards⁵, Niti Goel⁶, Vibeke Strand⁷, Sabrina M Nielsen⁸, Beverly J Shea⁹, Peter Tugwell¹⁰, Susan J Bartlett¹¹, Glen S Hazlewood¹², Lyn March¹³, Jasvinder A Singh¹⁴, Maria E Suarez-Almazor¹⁵, Maarten Boers¹⁶, Randall M Stevens¹⁷, Daniel E Furst¹⁸, Thasia Woodworth¹⁸, Amye Leong¹⁹, Peter M Brooks²⁰, Caroline Flurey²¹, Robin Christensen²²; OMERACT Safety Working Group

Affiliations

¹ Section for Biostatistics and Evidence-Based Research, the Parker Institute, Bispebjerg and Frederiksberg Hospital, Copenhagen, Denmark; Research Unit of Rheumatology, Department of Clinical Research, University of Southern Denmark, Odense University Hospital, Denmark; Department of Rehabilitation, Municipality of Guldborgsund, Nykoebing F, Denmark.
² SDG LLC Cambridge, MA 02138, USA.
³ Departments of Medicine, Epidemiology and Population Health, Biomedical Data Science, and Statistics, and Meta-Research Innovation Center at Stanford (METRICS), Stanford University, California, USA.
⁴ Department of Pharmacy, Sint Maartenskliniek, Department of Pharmacy, RadboudUMC, Nijmegen, the Netherlands.
⁵ Department of Rheumatology, University of Bristol, Bristol, United Kingdom.
⁶ Division of Rheumatology, Duke University School of Medicine, Durham, NC, USA.
⁷ Division of Immunology/Rheumatology, Stanford University, Palo Alto CA, USA.
⁸ Section for Biostatistics and Evidence-Based Research, the Parker Institute, Bispebjerg and Frederiksberg Hospital, Copenhagen, Denmark; Research Unit of Rheumatology, Department of Clinical Research, University of Southern Denmark, Odense University Hospital, Denmark.
⁹ Ottawa Hospital Research Institute, Clinical Epidemiology Program and School of Epidemiology and Public Health, University of Ottawa, Canada.
¹⁰ Department of Medicine, School of Epidemiology, Public Health and Community Medicine, University of Ottawa, Canada.
¹¹ Department of Medicine, McGill University, Montreal, Canada.
¹² Department of Medicine, Cumming School of Medicine, University of Calgary; Department of Community Health Sciences, Cumming School of Medicine, University of Calgary; Arthritis Research Canada.
¹³ Department of Rheumatology, Royal North Shore Hospital, Kolling Institue and The University of Sydney, Sydney, Australia.
¹⁴ Medicine Service, VA Medical Center, Birmingham, AL, USA; Department of Medicine and the School of Medicine, University of Alabama at Birmingham (UAB), Birmingham, AL, USA; Department of Epidemiology and the UAB School of Public Health, Birmingham, AL, USA.
¹⁵ Department of Health Services Research and Section of Rheumatology and Clinical Immunology, University of Texas MD Anderson Cancer Centre, Houston, TX, USA.
¹⁶ Department of Epidemiology and Data Science, Amsterdam UMC, Vrije Universiteit, Amsterdam, the Netherlands.
¹⁷ Centrexion Therapeutic Corp, Boston, MA, USA.
¹⁸ David Geffen School of Med. Department of Medicine, Division of Rheumatology, UCLA, Los Angeles, California, USA.
¹⁹ Healthy Motivation, Santa Barbara, California USA.
²⁰ Centre for Health Policy Melbourne School of Population and Global Health University of Melbourne, Australia.
²¹ Faculty of Health and Applied Sciences, University of the West of England, Bristol, United Kingdom.
²² Section for Biostatistics and Evidence-Based Research, the Parker Institute, Bispebjerg and Frederiksberg Hospital, Copenhagen, Denmark; Research Unit of Rheumatology, Department of Clinical Research, University of Southern Denmark, Odense University Hospital, Denmark. Electronic address: Robin.Christensen@regionh.dk.

PMID: 37918049
DOI: 10.1016/j.semarthrit.2023.152288

Abstract

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.

Keywords: Adverse events; Core outcome set; Harms; OMERACT; Safety; rheumatology.

MeSH terms

Clinical Trials as Topic
Humans
Musculoskeletal Diseases* / therapy
Outcome Assessment, Health Care
Patient Reported Outcome Measures
Rheumatology*

Supplementary concepts

Rippling muscle disease, 1