International patient and physician consensus on a psoriatic arthritis core outcome set for clinical trials

Abstract

Objective: To identify a core set of domains (outcomes) to be measured in psoriatic arthritis (PsA) clinical trials that represent both patients' and physicians' priorities.

Methods: We conducted (1) a systematic literature review (SLR) of domains assessed in PsA; (2) international focus groups to identify domains important to people with PsA; (3) two international surveys with patients and physicians to prioritise domains; (4) an international face-to-face meeting with patients and physicians using the nominal group technique method to agree on the most important domains; and (5) presentation and votes at the Outcome Measures in Rheumatology (OMERACT) conference in May 2016. All phases were performed in collaboration with patient research partners.

Results: We identified 39 unique domains through the SLR (24 domains) and international focus groups (34 domains). 50 patients and 75 physicians rated domain importance. During the March 2016 consensus meeting, 12 patients and 12 physicians agreed on 10 candidate domains. Then, 49 patients and 71 physicians rated these domains' importance. Five were important to >70% of both groups: musculoskeletal disease activity, skin disease activity, structural damage, pain and physical function. Fatigue and participation were important to >70% of patients. Patient global and systemic inflammation were important to >70% of physicians. The updated PsA core domain set endorsed by 90% of OMERACT 2016 participants includes musculoskeletal disease activity, skin disease activity, pain, patient global, physical function, health-related quality of life, fatigue and systemic inflammation.

Conclusions: The updated PsA core domain set incorporates patients' and physicians' priorities and evolving PsA research. Next steps include identifying outcome measures that adequately assess these domains.

Keywords: Outcomes research; Psoriatic Arthritis; Qualitative research; Spondyloarthritis.

Conflict of interest statement

Competing interests: A-MO reports grants from Celgene and Janssen (to Johns Hopkins University), during the conduct of the study; consulting fees from Janssen, outside the submitted work. MdW reports grants from Celgene and Janssen (to Johns Hopkins University), during the conduct of the study; personal fees from Novartis, personal fees from BMS, outside the submitted work. PM reports grants and other from AbbVie, grants and other from Amgen, grants and other from Bristol Myers Squibb, grants and other from Celgene, other from Crescendo, other from Corrona, other from Demira, other from Genentech, grants and other from Janssen, grants and other from Lilly, other from Merck, grants and other from Novartis, grants and other from Pfizer, grants and other from Sun, grants and other from UCB, other from Zynerba, during the conduct of the study. WT reports grants, consulting and speaker fees from Abbvie, Pfizer, UCB, Novartis and Celgene. OFG reports grants and/or consulting fees from Abbvie, Pfizer, BMS, Celgene, Janssen, Novartis, UCB, Lilly. NG reports she is a full-time employee of Quintiles, Inc. PH reports personal fees from Celgene, outside the submitted work. NMcH reports grants from UK Government to Host Institution, during the conduct of the study. MdW and VS are members of the executive of OMERACT, an organisation that develops outcome measures in rheumatology and receives arms-length funding from 36 companies.

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Figures

Figure 1

Study diagram: research work streams that led to the updated psoriatic arthritis (PsA) core domain set are represented: the process started with a systematic literature review (SLR) and international focus groups with patients with PsA to generate a large pool of candidate domains. These were then reduced through an iterative consensus process consisting of surveys with patients and physicians, a consensus nominal group technique (NGT) meeting with patients and physicians, and breakout group discussions and voting at the Outcome Measures in Rheumatology (OMERACT) 2016.

Figure 2

Domains important to patients and physicians for inclusion in psoriatic arthritis (PsA) clinical trials: The first panel shows domains selected important* by >70% of either patients or physicians in the first international survey and patient and physician percentages. The second panel shows patient and physician percentages rating each domain as important* in the second international survey (*important was designated as receiving a rating of ≥8 on a numerical rating scale from 0, not important at all, to 10, very important). The brackets identify the 70% level of consensus within a group. MSK, musculoskeletal.

Figure 3

Updated 2016 psoriatic arthritis (PsA) core domain set. Musculoskeletal (MSK) disease activity includes peripheral joints, enthesitis, dactylitis and spine symptoms; skin activity includes skin and nails; patient global is defined as patient-reported disease-related health status. The inner circle (core) includes domains that should be measured in all PsA randomised controlled trials (RCTs) and longitudinal observational studies (LOS). The middle circle includes domains that are important but may not be feasible to assess in all RCTs and LOS. The outer circle or research agenda includes domains that may be important but need further study.