'Am I doing this right?' Physician perceptions of the global assessment in clinical trials of systemic sclerosis

Hana Sabanovic¹, John D Pauling^{2

3}, Murray Baron⁴, Laurence Clemens⁵, Francesco Del Galdo^{6

7}, Christopher P Denton^{8

9}, Oliver Distler¹⁰, Tracy Frech¹¹, Anna-Maria Hoffmann-Vold^{10

12}, Marie Hudson⁴, Dinesh Khanna¹³, Nancy Maltez¹⁴, Thomas A Medsger¹⁵, Peter A Merkel^{16

17}, Mandana Nikpour^{18

19

20}, Janet Pope²¹, Virginia D Steen²², Wendy Stevens^{5

23}, Elizabeth R Volkmann²⁴, Laura Ross^{5

23}

Affiliations

¹ Centre for Health Policy, School of Population and Global Health, University of Melbourne, Parkville, Australia.
² Department of Rheumatology, North Bristol NHS Trust, Bristol, UK.
³ Musculoskeletal Research Unit, School of Translational Health Sciences, University of Bristol, Bristol, UK.
⁴ Division of Rheumatology, Jewish General Hospital, McGill University, Montreal, Canada.
⁵ Department of Rheumatology, St Vincent's Hospital Melbourne, Fitzroy, Australia.
⁶ Leeds Institute of Rheumatic and Musculoskeletal Medicine, LIRMM, Leeds, UK.
⁷ NIHR Leeds Biomedical Research Centre, Leeds Teaching Hospitals Trust, Leeds, UK.
⁸ Centre for Rheumatology and Connective Tissue Diseases, Royal Free Hospital, London, UK.
⁹ Division of Medicine, University College London, London, UK.
¹⁰ Department of Rheumatology, University Hospital Zurich, University of Zurich, Zurich, Switzerland.
¹¹ Department of Medicine, Division of Rheumatology and Immunology, Vanderbilt University Medical Center, Tennessee Valley Healthcare System for Veterans Affairs, Nashville, USA.
¹² Department of Rheumatology, Oslo University Hospital, Oslo, Norway.
¹³ University of Michigan Scleroderma Program, Division of Rheumatology, Department of Internal Medicine, University of Michigan, Ann Arbor, USA.
¹⁴ Department of Medicine, Division of Rheumatology, The Ottawa Hospital, University of Ottawa, Ottawa, ON, Canada.
¹⁵ University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.
¹⁶ Division of Rheumatology, Department of Medicine, University of Pennsylvania, Philadelphia, USA.
¹⁷ Division of Epidemiology, Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania, Philadelphia, USA.
¹⁸ Sydney Musculoskeletal Research Flagship Centre, Faculty of Medicine and Health, University of Sydney, Sydney, Australia.
¹⁹ University of Sydney School of Public Health, Sydney, Australia.
²⁰ Department of Rheumatology, Royal Prince Alfred Hospital, Sydney, Australia.
²¹ Schulich School of Medicine and Dentistry, University of Western Ontario, London, ON, Canada.
²² Division of Rheumatology, Department of Medicine, Georgetown University Medical Center, Washington, DC, USA.
²³ Department of Medicine, The University of Melbourne, Parkville, Australia.
²⁴ Division of Rheumatology, Department of Medicine, University of California, Los Angeles, Los Angeles, USA.

PMID: 40644620
PMCID: PMC12596065
DOI: 10.1093/rheumatology/keaf377

'Am I doing this right?' Physician perceptions of the global assessment in clinical trials of systemic sclerosis

Hana Sabanovic et al. Rheumatology (Oxford). 2025.

. 2025 Nov 1;64(11):5844-5852.

doi: 10.1093/rheumatology/keaf377.

Authors

Affiliations

¹ Centre for Health Policy, School of Population and Global Health, University of Melbourne, Parkville, Australia.
² Department of Rheumatology, North Bristol NHS Trust, Bristol, UK.
³ Musculoskeletal Research Unit, School of Translational Health Sciences, University of Bristol, Bristol, UK.
⁴ Division of Rheumatology, Jewish General Hospital, McGill University, Montreal, Canada.
⁵ Department of Rheumatology, St Vincent's Hospital Melbourne, Fitzroy, Australia.
⁶ Leeds Institute of Rheumatic and Musculoskeletal Medicine, LIRMM, Leeds, UK.
⁷ NIHR Leeds Biomedical Research Centre, Leeds Teaching Hospitals Trust, Leeds, UK.
⁸ Centre for Rheumatology and Connective Tissue Diseases, Royal Free Hospital, London, UK.
⁹ Division of Medicine, University College London, London, UK.
¹⁰ Department of Rheumatology, University Hospital Zurich, University of Zurich, Zurich, Switzerland.
¹¹ Department of Medicine, Division of Rheumatology and Immunology, Vanderbilt University Medical Center, Tennessee Valley Healthcare System for Veterans Affairs, Nashville, USA.
¹² Department of Rheumatology, Oslo University Hospital, Oslo, Norway.
¹³ University of Michigan Scleroderma Program, Division of Rheumatology, Department of Internal Medicine, University of Michigan, Ann Arbor, USA.
¹⁴ Department of Medicine, Division of Rheumatology, The Ottawa Hospital, University of Ottawa, Ottawa, ON, Canada.
¹⁵ University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.
¹⁶ Division of Rheumatology, Department of Medicine, University of Pennsylvania, Philadelphia, USA.
¹⁷ Division of Epidemiology, Department of Biostatistics, Epidemiology, and Informatics, University of Pennsylvania, Philadelphia, USA.
¹⁸ Sydney Musculoskeletal Research Flagship Centre, Faculty of Medicine and Health, University of Sydney, Sydney, Australia.
¹⁹ University of Sydney School of Public Health, Sydney, Australia.
²⁰ Department of Rheumatology, Royal Prince Alfred Hospital, Sydney, Australia.
²¹ Schulich School of Medicine and Dentistry, University of Western Ontario, London, ON, Canada.
²² Division of Rheumatology, Department of Medicine, Georgetown University Medical Center, Washington, DC, USA.
²³ Department of Medicine, The University of Melbourne, Parkville, Australia.
²⁴ Division of Rheumatology, Department of Medicine, University of California, Los Angeles, Los Angeles, USA.

PMID: 40644620
PMCID: PMC12596065
DOI: 10.1093/rheumatology/keaf377

Abstract

Objectives: Physician global assessments (PhyGAs) are commonly performed in randomized controlled trials (RCTs) in SSc. However, there is no single PhyGA applied across RCTs. We performed an exploratory qualitative study to explore perceptions of the PhyGA, its role in RCTs and how physicians perform their own assessment.

Methods: Participants with expertise in the clinical assessment and, or actively involved in research on SSc were invited to participate. Participants were asked to define disease constructs of activity, damage, severity, and overall health, and to describe how they perform a PhyGA and their perception of what a PhyGA should assess. Interview transcripts were analysed using deductive and inductive thematic analysis.

Results: Eighteen rheumatologists and one patient research partner were interviewed. Four major themes were identified: (i) physician uncertainty; (ii) variation in the conduct of a PhyGA; (iii) physician efforts to improve PhyGA consistency; (iv) utility of a PhyGA. Most participants felt a PhyGA should assess changeable aspects of SSc, commonly conceived of as disease activity. There was considerable uncertainty about the optimal method for assessing disease activity. Participants were uncertain about their own methods of performing a PhyGA, and variability in the application of the instrument was identified. Despite these limitations, physicians generally agreed that the PhyGA is useful and can assess unquantifiable aspects of SSc.

Conclusion: We identified significant heterogeneity in the approach to PhyGAs in SSc. This variation was considered a limitation of the PhyGA. Overall, a PhyGA was viewed as a useful instrument that can aid the assessment of treatment response in RCTs.

Keywords: clinical trials; global assessment; outcome measures; physician assessment; systemic sclerosis.

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Figures

**Figure 1.**
Proposed methods for standardization of a SSc physician global assessment instrument

See this image and copyright information in PMC

References

1. Hao Y, Hudson M, Baron M et al. ; Australian Scleroderma Interest Group. Early mortality in a multinational systemic sclerosis inception cohort. Arthritis Rheumatol 2017;69:1067–77. - PubMed
1. Pope JE. The future of treatment in systemic sclerosis: can we design better trials? Lancet Rheumatol 2020;2:e185–94. - PubMed
1. Lafyatis R, Valenzi E. Assessment of disease outcome measures in systemic sclerosis. Nat Rev Rheumatol 2022;18:527–41. - PubMed
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'Am I doing this right?' Physician perceptions of the global assessment in clinical trials of systemic sclerosis

Affiliations

'Am I doing this right?' Physician perceptions of the global assessment in clinical trials of systemic sclerosis

Authors

Affiliations

Abstract

Figures

References

MeSH terms

Grants and funding

LinkOut - more resources

Full Text Sources

Medical