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.

Economic Burden of Illness in Adult Patients with Nocturia.

BACKGROUND: Nocturia is considered to be a very bothersome lower urinary tract disorder. Yet, to date, the economic burden attributable to this poor health condition remains less well known. OBJECTIVE: To compare differences in health care resource utilization (HCRU), health care costs, and work productivity in adult patients with differing frequencies of nocturia episodes (i.e., < 2 vs. ≥ 2 nocturia episodes per night). METHODS: Adult patients with nocturia enrolled in an integrated proprietary database were recruited to complete a survey on their demographics, nocturia characteristics, and work productivity. Using patients' survey data and health care claims from the previous 6 months, those with < 2 (n = 197; 21.9%) versus ≥ 2 (n = 702; 78.1%) nocturia episodes per night were compared for differences in HCRU, health care costs, and work productivity after adjusting for potential confounders. HCRU was reported as the mean number per patient per month (PPPM) for outpatient visits (all types), physician office visits, and prescriptions filled and the proportion of patients with ≥1 hospitalization or emergency department visit in the previous 6 months. Health care costs were reported as mean PPPM. Work productivity was assessed via patient survey and reported as a mean percentage for absenteeism, presenteeism, overall work impairment, and activity impairment during the past week. RESULTS: 899 adult patients (mean age = 71.4 years; 57.2% men) were enrolled and analyzed. Compared with patients with <2 nocturia episodes per night, patients with ≥ 2 nocturia episodes had more outpatient health care visits (unadjusted mean visits PPPM: 2.1 vs. 1.6; P < 0.001; adjusted mean visits PPPM: 2.1 vs. 1.6; P = 0.017), office visits (unadjusted and adjusted mean visits PPPM: 0.9 vs. 0.7; P < 0.001), and prescriptions filled (unadjusted mean prescription fills PPPM: 3.1 vs. 2.1; P < 0.001; adjusted mean prescription fills PPPM: 3.2 vs. 2.2; P = 0.027). Patients with ≥ 2 nocturia episodes per night also displayed significantly higher outpatient health care costs (unadjusted mean PPPM costs: $676 vs. $516; P = 0.028; adjusted mean PPPM costs: $678 vs. $506; P = 0.017). In terms of work productivity impairment, patients with ≥ 2 nocturia episodes per night experienced higher rates of unadjusted (20% vs. 10%; P = 0.002) and adjusted presenteeism (20% vs. 10%; P = 0.004) and unadjusted (20% vs. 10%; P = 0.002) and adjusted overall work impairment (20% vs. 10%; P = 0.001). CONCLUSIONS: Study findings demonstrate that nocturia was associated with higher outpatient encounters and related costs in the presence of a greater occurrence of nocturic episodes. DISCLOSURES: This study was funded by Allergan plc, Dublin, Ireland. Neither honoraria nor payments were provided for authorship. Dmochowski is a consultant and speaker for Allergan plc and a consultant for Serenity Pharmaceuticals. Brucker is a consultant and speaker for Allergan plc, a consultant for Watkins-Conti and Avadel, and an investigator for Medtronic and Ipsen. Cole is a consultant for Allergan plc and an employee of Sharp Rees-Stealy Medical Group. Kawahara and Pulicharam are full-time employees of DaVita Medical Group. Burk is a consultant for Allergan plc and a health outcomes consultant. Tung is an employee of Allergan plc. Hale has served as a consultant/advisor to and has received research funding from Allergan plc. The data from this manuscript were previously presented in poster format by Steve Kawahara at the Academy of Managed Care & Specialty Pharmacy Annual Meeting; April 19-22, 2016; San Francisco, CA.

Cost-offset analysis: bimatoprost versus other prostaglandin analogues in open-angle glaucoma.

OBJECTIVES: To develop a cost-offset model from a US payer perspective comparing glaucomatous progression and costs among primary open-angle glaucoma (POAG) patients using bimatoprost, latanoprost, or travoprost. STUDY DESIGN: Cost-offset model. METHODS: A Markov cohort model was used to estimate glaucomatous progression for POAG patients over 7 years. The model assumed bimatoprost-treated patients had lower resulting intraocular pressure (IOP) (by 1 mm Hg) for all presenting IOP categories than latanoprost- or travoprost-treated patients. Patients with lower IOP were assumed to have lower probability of progression. Those that progressed were assumed to do so at a rate of -0.6 dB per year. Direct costs associated with mean deviation score categories were applied to each treatment cohort to calculate the expected 7-year costs of treating patients with each prostaglandin analogue (PGA). Literature was used to support assumptions. A budget impact analysis was conducted where all travoprost patients switched to generic latanoprost and where all bimatoprost patients switched to generic latanoprost. The base case market share was 22% bimatoprost, 23% travoprost, and 55% latanoprost. RESULTS: Model results demonstrate that for a managed care plan with 9500 PGA-treated glaucoma patients, exclusive bimatoprost use would prevent progression in 136 additional individuals compared with exclusive travoprost or latanoprost treatment. Model results demonstrate that greater IOP reduction from bimatoprost is associated with increased cost savings compared with latanoprost or travoprost treatments. CONCLUSIONS: Model results demonstrate that greater IOP reduction from bimatoprost could reduce managed care spending.