Patient experience of medication administration and development of a Patient Experience and Preference Questionnaire (PEPQ) for patients with advanced or metastatic cancer.

Introduction: A better understanding of patient experience of intravenous (IV) or subcutaneous (SC) routes of administration is fundamental to providing optimal administration of medical therapies to oncology patients. The objective of this study was to examine patient experiences of IV and SC treatment with nivolumab and confirm the relevance of item concepts in the Patient Experience and Preference Questionnaire (PEPQ). The PEPQ is a clinical outcomes' assessment instrument developed to obtain patient-centric data and understand the experience with IV and SC treatment administration. Methods: Embedded qualitative interviews were conducted with a subset of participants from three treatment cohorts with metastatic non-small-cell lung cancer (NSCLC), renal cell carcinoma (RCC), unresectable or advanced metastatic melanoma, hepatocellular carcinoma (HCC), or colorectal cancer (CRC) from the CA209-8KX clinical trial. Concept elicitation interviews were conducted within 14 days of the initial treatment cycle and patient experiences with IV and SC treatment administration were assessed. Concepts from interviews were mapped to the PEPQ version 1.0 questions to assess relevance and convergence of concepts. Results: Interviews were conducted with 43 trial participants from clinical sites opting to participate from six countries (Argentina, France, the Netherlands, Poland, Spain, and New Zealand). The mean age of sub-study participants was 66 ± 11.3 years (range 24-80 years), and 67.4% (N = 29) were male. Sub-study participants with experience of SC most frequently reported symptoms or signs of injection-related redness (27.9%), itching (14.0%), and pain (of needle), and described the pain as pricking, stinging, or tingling (11.0% each). The amount of pain and time burden were widely endorsed as important factors for satisfaction and related to the route of medication administration. For 11 sub-study participants with experience with both IV and SC treatments, 10 (90.9%) preferred SC over IV treatment administration. Conclusion: This study summarizes the experience and satisfaction of receiving IV or SC treatment and confirms the relevance of the PEPQ in a subgroup of CA209-8KX clinical trial participants with metastatic NSCLC, RCC, melanoma, HCC, and CRC. Participant treatment experience and satisfaction with the route of medication mapped to the PEPQ question content support the relevance of PEPQ v2.0 in clinical trials as a self-report measure.

Patient experiences in ulcerative colitis: conceptual model and review of patient-reported outcome measures.

PURPOSE: To identify symptoms and their impacts on daily functioning and health-related quality of life (HRQoL) experienced by adult patients with ulcerative colitis (UC) and evaluate patient-reported outcome (PRO) measures for UC clinical studies. METHODS: A conceptual model of symptoms and impacts of UC were developed from a literature review. PRO measures were identified from the literature, clinical trials databases, health technology assessment submissions, and regulatory label claims, and were selected for conceptual analysis based on disease specificity and use across information sources. PRO measures covering the most concepts when mapped against the conceptual model were assessed for gaps in psychometric properties using Food and Drug Administration (FDA) guidance and consensus-based standards for the selection of health measurement instruments (COSMIN) criteria. RESULTS: The conceptual model grouped the 52 symptom concepts and 72 proximal and distal impacts into eight, two, and five dimensions, respectively. Of 65 PRO measures identified, eight underwent conceptual analysis. Measures covering the most concepts and assessed for psychometric properties were the Inflammatory Bowel Disease Questionnaire, Symptoms and Impacts Questionnaire for UC, UC-PRO symptoms modules, UC-PRO impact modules, and Crohn's and UC Questionnaire; all had good or excellent support for content validity. The UC-PRO Signs and Symptoms fully met FDA guidance and COSMIN criteria for content validity and most psychometric properties. CONCLUSION: Existing PRO measures assess concepts relevant to patients with UC, but all PRO measures reviewed require further psychometric evaluation to demonstrate they are fit for purpose.

Patient Experiences, Satisfaction, and Expectations with Current Systemic Lupus Erythematosus Treatment: Results of the SLE-UPDATE Survey.

OBJECTIVES: To provide information on systemic lupus erythematosus (SLE) patients' experiences, satisfaction, and expectations with treatments and examine the association between treatment satisfaction and patient-reported outcomes (PRO). METHODS: A cross-sectional, non-interventional, online survey of US adult patients with SLE was conducted in 2019. The survey consisted of 104 questions about SLE and the following PRO instruments: LupusPRO™, Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue, Work Productivity and Activity Impairment (WPAI), an 11-point Worst Pain Numerical Rating scale (NRS), and an 11-point Worst Joint Pain NRS. RESULTS: Five hundred participants (75% female, 76% White/Caucasian, mean age 42.6 ± 12.7 years, 63% with an associate degree or higher) completed the survey. Most participants were "completely" or "somewhat satisfied" with their treatments, although satisfaction rates were lower for corticosteroids (65%), immunosuppressants (71%), and anti-malarials (55%) than for belimumab (intravenous or subcutaneous) (86%) and rituximab (94%). Treatments were more often considered "burdensome" or "very burdensome" for belimumab (67%) and rituximab (63%) than for corticosteroids (48%), immunosuppressants (49%), and anti-malarials (30%). Pain and productivity assessments supported substantial impairment for the majority of participants, even those who indicated that they were completely satisfied with treatments. The treatment goals most commonly reported as "very important" were reducing fatigue, pain, and the frequency or severity of flares. Three-quarters of participants (76.6%) indicated that their physician's goals for their therapy matched their own goals "very" or "somewhat closely." Despite high levels of satisfaction, most participants (63.0%) indicated that their physicians had not asked about their treatment goals during the past 3 months. CONCLUSION: SLE patients reported high rates of satisfaction with current therapies despite identifying substantial treatment burdens, residual pain, and fatigue. Reduced fatigue, pain, and flares were the most important treatment goals for these patients.