University of Birmingham Birmingham, United Kingdom
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Benjamin A Fisher1, Linda Stone2, Jessica Marvel3, Pushpendra Goswami4, Monia Steenackers4, Gayle Kenney4, Chiara Perella5, Wolfgang Hueber4, Chloe Howse6, Elizabeth Gargon6, Aishwarya Chohan6, Megan Mayhew6 and Nicola Williamson6, 1University of Birmingham, Birmingham, United Kingdom, 2The British Sjogren’s Syndrome Association, Birmingham, United Kingdom, 3Novartis Pharmaceuticals Corporation, East Hanover, NJ, 4Novartis Pharma AG, Basel, Switzerland, 5Novartis Pharma AG, Riehen, Switzerland, 6Adelphi Values Patient-Centered Outcomes, Bollington, United Kingdom
Background/Purpose: Sjögren's is a heterogenous chronic auto-immune disease, characterized by excessive dryness of the eyes and mouth, as well as systemic complications which can significantly impact patients' health-related quality of life (HRQoL). Existing patient-reported outcome (PRO) measures focus on the frequency/severity of symptoms but do not capture the impacts that Sjögren's has on patients' HRQoL. To address this gap, the Sjögren's-Related Quality of Life (SRQoL), was developed in line with Food and Drug Administration's Clinical Outcome Assessment (COA) guidance documents.1-4 Qualitative interviews were conducted to evaluate the content validity and appropriateness of the SRQoL.
Methods: Patient advisory boards, social media listening, previous patient interviews, and findings from a prior literature review were reviewed to gain insights into the patient experience and HRQoL impact of Sjögren's to develop the SRQoL. Global impression of severity (PGI-S) and change (PGI-C) items were developed to inform further validation of the SRQoL. SRQoL and PGI-S/C development involved a patient advocate and physician as research partners. Combined qualitative concept elicitation (CE) and cognitive debriefing (CD) interviews are being conducted with adult (≥18 years) patients with primary Sjögren's from the US and UK, over two rounds. Round-one has been completed (n=10; 70% female; mean age = 52 years).
Results: From the review, an initial 36-item draft PRO measure was developed to assess HRQoL across seven hypothesized domains including physical functioning, activities of daily living, cognition, sleep, emotional wellbeing, social/family functioning, and work. Round-one CE results were consistent with Sjögren's related symptoms and HRQoL impacts elicited from the review (difficulty walking, driving difficulties, low mood/sadness, and daytime sleepiness). Concepts from the participant interviews (round one) were organized into a preliminary conceptual model in Figure 1. SRQoL and PGI-S/C items were well understood and relevant to most participants during round-one CD. Participants endorsed the 7-day recall period and frequency and severity response scales.
Conclusion: Initial findings support the content validity and suitability of the draft SRQoL, with round-two interviews currently underway. Following further testing and psychometric evaluation, the SRQoL is intended to provide greater understanding of patient burden and treatment benefit of Sjögren's in randomized clinical trials and may be used in clinical practice to monitor patients' HRQoL and improve patient-physician communication.
References:
FDA PFDD: Collecting Comprehensive and Representative Input, 2018
FDA PFDD: Methods to Identify What Is Important to Patients, 2022
FDA PFDD: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments, 2022
FDA PFDD: Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision-Making, 2023
B. A Fisher: Bristol-Myers Squibb(BMS), 2, Celgene, 5, Galapagos, 2, 5, Janssen, 2, 5, Novartis, 2, Roche, 2, Sanofi, 2, Servier, 2, 5, UCB, 2; L. Stone: Novartis, 12, Patient advocate, Servier Saclay, 12, Member Patient Board; J. Marvel: Novartis, 3; P. Goswami: Novartis, 3; M. Steenackers: Novartis, 3; G. Kenney: Novartis Pharma AG, 3; C. Perella: Novartis, 3; W. Hueber: Novartis, 3, 11; C. Howse: Novartis, 2, 7; E. Gargon: Novartis, 2, 7; A. Chohan: Novartis, 2, 7; M. Mayhew: Novartis, 2, 7; N. Williamson: Novartis, 2, 7.
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