The Development and Content Validation of the Sjögren's Related Quality of Life Instrument (SRQoL)

IntroductionSeveral clinical outcome assessment (COA) instruments assess Sj & ouml;gren's disease (Sj & ouml;gren's) symptoms, but do not provide comprehensive assessment of the health-related quality of life (HRQoL) impact of Sj & ouml;gren's. This study aimed to develop a patient-reported outcome (PRO) instrument for the assessment of HRQoL, intended for use in clinical trials and clinical practice in the assessment of treatment benefit.MethodsReview of study sponsor proprietary data and qualitative interviews informed the development of a conceptual model, the Sj & ouml;gren's Related Quality of Life (SRQoL) and patient global impression of severity (PGI-S) and change (PGI-C) items. Combined concept elicitation and cognitive debriefing interviews with patients with Sj & ouml;gren's explored their HRQoL impact experience and content validity of the SRQoL and PGI items.ResultsTwenty participants were interviewed about their Sj & ouml;gren's experience. Following inductive analysis of interviews, concepts were categorized into eight domains: emotional well-being (e.g., worry and stress; n = 20/20; 100%), sleep (e.g., daytime sleepiness and waking up during the night; n = 20/20; 100%), activities of daily living (e.g., difficulty looking at screens and difficulty driving; n = 20/20; 100%), cognition (e.g., concentration difficulties and word finding difficulties; n = 19/20; 95.0%), physical functioning (e.g., difficulty walking and difficulty exercising; n = 19/20; 95.0%), social and family functioning (e.g., dependent on others and relationship difficulties; n = 17/20; 85.0%), work (n = 15/20; 75.0%), and sexual functioning (n = 12/20; 60.0%). SRQoL and PGI items, instructions, response options, and recall period were well understood and relevant to participants.ConclusionsThe SRQoL is a new PRO instrument to assess Sj & ouml;gren's impact on HRQoL, developed in accordance with regulatory guidance. This study provides considerable insight into the patient experience of Sj & ouml;gren's and evidence to support the content validity of the SRQoL. Future research should evaluate the psychometric properties of the SRQoL to support its use in clinical trials and clinical practice and further validate its use as an assessment of treatment benefit. Patient and physician completed questionnaires used in clinical trials and clinical practice have previously focused on symptoms. These questionnaires do not cover the full experience of living with Sj & ouml;gren's, such as differences in symptoms between individual patients, and their use has also been known to result in inaccurate reports. Another way of thinking about patients' experiences is to use a questionnaire to ask about the impact Sj & ouml;gren's has on patients' health-related quality of life, meaning how Sj & ouml;gren's might affect their life in different ways including physically, emotionally, and socially. The goal of this study was to develop a new questionnaire (named the Sj & ouml;gren's-Related Quality of Life [SRQoL] questionnaire) to measure the impact that Sj & ouml;gren's has on patients' lives. Interviews were conducted with people with Sj & ouml;gren's to explore their understanding of the new questionnaire and how relevant the content is to their experience of the condition. The results of this study suggest that the SRQoL includes relevant and easy-to-understand questions, suitable for use in future clinical trials and clinical practice, to measure if patients' HRQoL improves with treatment. Future research is needed to confirm that the questionnaire accurately measures the impact of Sj & ouml;gren's on HRQoL for use with patients with Sj & ouml;gren's.