Purpose: To explore symptoms and disease impacts of Crohn’s disease and to develop a new patient-reported outcomes (PRO) measure according to industry best practices. Methods: A conceptual model of relevant symptoms experienced by patients with Crohn’s disease was developed following a literature review. Three rounds of combined qualitative semi-structured concept elicitation and cognitive debriefing interviews with 36 patients (≥ 16 years) with Crohn’s disease and 4 clinicians were conducted to further explore the most commonly reported and most bothersome symptoms to patients. Interview results were used to update the conceptual model as well as items and response options included in The Crohn’s Disease Diary, a new PRO measure. Results: All patients (N = 36) reported abdominal pain, loose or liquid bowel movements, and high or increased frequency of bowel movements, with most reporting these symptoms spontaneously (100%, 92%, and 75%, respectively). All patients reported bowel movement urgency, but 61% reported this symptom only when probed. Most also reported that symptoms impacted activities of daily living, work/school, and emotional, social, and physical functioning (overall, 78%–100%; spontaneously, 79% – 92%). Data regarding core symptoms of Crohn’s disease from clinician concept elicitation interviews supported patient data. The 17-item Crohn’s Disease Diary assesses core symptoms and impacts of Crohn’s disease over 24 h, and extraintestinal manifestations over 7 days. The content validity of the diary was confirmed during cognitive debriefing interviews. Conclusion: The Crohn’s Disease Diary is a new PRO measure for the assessment of Crohn’s disease symptoms and impacts, developed according to industry best practices.

Introduction European Alliance of Associations for Rheumatology (EULAR) Sjögren’s Syndrome Disease Activity Index (ESSDAI) is a clinician-reported outcome (ClinRO) instrument, assessing Sjögren’s disease activity from the physician perspective. EULAR Sjögren’s Syndrome Patient Reported Index (ESSPRI) is a patient-reported outcome (PRO) instrument, assessing patient-defined Sjögren’s symptom severity. Both instruments are commonly used as clinical trial endpoints and have been psychometrically validated. However, qualitative evidence supporting content validity and what constitutes a meaningful change is limited. Qualitative evidence supporting Physician/Patient Global Assessment of disease activity and symptom severity (PhGA/PaGA) items used within anchor-based analyses for ESSDAI/ESSPRI is also lacking. Methods Qualitative, semi-structured, telephone/video interviews were conducted with patients with Sjögren’s (n = 12) and physicians who specialise in Sjögren’s (n = 10). Interviews explored: appropriateness of ESSDAI domain weights and meaningful improvements on domain/total scores from the physician perspective, appropriateness of ESSPRI’s 2-week recall period from the patient/physician perspective, patients’ perspectives on meaningful improvements in ESSPRI total scores, and patients’/physicians’ interpretation of PhGA/PaGA items. Results Most ESSDAI domain weights were considered clinically appropriate. Generally, a one-category improvement in domain-level scores and a 3-point improvement in total ESSDAI scores were considered clinically meaningful. Most patients/physicians considered ESSPRI’s 2-week recall period appropriate, and patients considered a 1-to-2-point ESSPRI total score improvement meaningful. PhGA/PaGA items developed for use as ESSDAI/ESSPRI anchors were consistently interpreted. Conclusions The findings support use of ESSDAI and ESSPRI as Sjögren’s clinical trials endpoints, as well as in clinical practice and other research settings. Qualitative data exploring meaningful change supports existing minimal clinically important improvement (MCII) thresholds.

Introduction Sjögren’s Syndrome Symptom Diary (SSSD) and Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) are patient-reported outcome (PRO) instruments assessing Sjögren’s symptoms. Original SSSD items have demonstrated content validity, however qualitative evidence supporting the updated ‘tiredness’ item and two new supplementary items is lacking. Although well established and validated in other rheumatic diseases, there is no qualitative evidence supporting content validity of FACIT-F in Sjögren’s. This study addressed these evidence gaps to support use of SSSD and FACIT-F as clinical trial endpoints, in clinical practice and in other research settings. Methods Qualitative, semi-structured telephone interviews were conducted with patients with Sjögren’s (n = 12) and expert Sjögren’s physicians (n = 10). Patient interviews explored content validity (e.g., understanding and relevance) of the new and updated SSSD items, perceptions of item and total score meaningful change on SSSD, and understanding and relevance of FACIT-F items. Physician interviews explored opinions on various SSSD scoring approaches. Results The new and updated SSSD items and FACIT-F demonstrated good content validity. Most patients considered a two-point improvement on most SSSD items meaningful, as well as a one- or two-point total score improvement. Most physicians reported tracking changes in patient responses to individual items as the most appropriate SSSD scoring approach. Conclusions SSSD and FACIT-F are content valid in a Sjögren’s population, meeting an important criterion to support their use as clinical trial endpoints, but also their use in clinical practice and other research settings. Qualitative data exploring meaningful change will be valuable in supporting psychometrically derived responder definitions.

Purpose: Endometriosis is a chronic disorder of the female reproductive system characterized by debilitating symptoms, particularly endometriosis-related pain (ERP). Patient-reported outcome (PRO) measures of symptoms and impacts are required to assess disease severity in ERP clinical studies and clinical practice. A content-valid instrument was developed by modifying the Dysmenorrhea Daily Diary (DysDD) to form the Endometriosis Daily Diary (EDD), an electronic PRO administered via handheld device.Methods: Qualitative research with US females with ERP was conducted in three stages: (1) Development of an endometriosis conceptual model based on qualitative literature and conduct of concept elicitation (CE) interviews (N = 30). (2) Cognitive debriefing (CD) interviews (N = 30) conducted across two rounds to assess relevance and understanding of the EDD, with modifications between interview rounds. (3) Pilot testing to assess usability/feasibility of administrating the EDD daily on an electronic handheld device (N = 15). Clinical experts provided guidance throughout the study.Results: The conceptual model provided a comprehensive summary of endometriosis to inform modifications to the DysDD, forming the EDD. CD results demonstrated that EDD items were relevant for most participants. Instructions, items, response scales, and recall period were well-understood. The resulting daily diary assesses severity of cyclic and non-cyclic pelvic pain, dyspareunia, impact of ERP on functioning and daily life, symptoms associated with ERP, and bowel symptoms. Participants were able to complete the diary daily and found the device easy to use.Conclusion: The EDD demonstrated good content validity in females experiencing ERP. The next step is to perform psychometric validation in an ERP sample.

Background/Objectives: Up to 95% of cancer patients report sleep disturbances or disorders. These can potentially influence cancer development and outcomes. This study aimed to review the existing literature to understand the patient experience of cancer-related sleep impairment across indications and treatments, to improve awareness and inform disease treatment/management. Methods: A targeted literature review of published qualitative studies exploring sleep impairment in oncology was conducted in MEDLINE, Embase, and PsycINFO. Concepts were extracted from the reviewed articles, and patient quotes or author descriptions/interpretations were subject to secondary analysis using semantic, qualitative, directed content analysis techniques via ATLAS.ti. Results informed the development of a patient-centric conceptual model. Results: From 482 identified publications, 22 publications were selected for in-depth review and concept extraction, collectively including 531 patients across 25 different oncology indications. Cancer-related sleep impairment frequently affected patients living with cancer, who reported difficulty obtaining good quality sleep, including difficulty falling asleep and staying asleep. Sleep problems were triggered by the emotional anxiety of life with cancer, cancer-related symptoms, treatments and side effects, and external factors. Poor sleep greatly impacted daily life for patients, with difficulties in daily life causing further sleep impairment, in a cyclical relationship. Conclusions: This study determined that there is an unmet need for treatment of sleep impairment in oncology to improve patient outcomes. The holistic conceptual model developed in this study can provide an important basis for selecting, developing, or modifying outcome measures to assess relevant sleep outcomes, facilitating discussion between patients and clinicians and improving disease treatment/management.