BACKGROUND: Clinical trials and real-world evidence (RWE) studies of biologics have demonstrated reduced exacerbations, decreased use of oral corticosteroids (OCS), and improvements in daily symptoms and health-related quality of life in patients with severe eosinophilic asthma (SEA). OBJECTIVE: To compare direct health care costs associated with biologic use for the treatment of SEA from a US third-party payer perspective. METHODS: We developed a cost-minimization model to compare costs and cost offsets associated with 3 biologics-benralizumab, mepolizumab, and dupilumab-for 2- and 4-year periods. The model relied on longitudinal data from clinical trials to inform the primary (base case) analysis cost comparison and RWE study data, in a separate scenario, to compare costs in nonclinical trial settings. Primary model outcomes included exacerbations (including hospitalizations), OCS-dependent years (including associated complications), and total direct health care biologic costs. Results were calculated at the per patient and population level (per 1,000 patients). Sensitivity analyses with key model parameters were performed. RESULTS: Benralizumab had the lowest total biologic costs per patient for both the 2- and 4-year periods. Over 4 years, the marginal cost difference in total biologic costs per patient was $23,061 lower for benralizumab vs mepolizumab and $17,242 lower for benralizumab vs dupilumab. The 4-year population level analysis of benralizumab vs mepolizumab revealed $4.8 million in marginal cost offsets due to 582 fewer exacerbations and 153 fewer OCS-dependent years and a marginal total cost savings of $27.9 million per 1,000 patients for benralizumab. The 4-year population level analysis of benralizumab vs dupilumab revealed $2.3 million in marginal cost offsets due to 291 fewer exacerbations and 64 fewer OCS-dependent years and marginal total cost savings of $19.5 million per 1,000 patients for benralizumab. RWE data were available for a 2-year cost comparison scenario of benralizumab vs mepolizumab, which showed similar results to the base case analysis. Sensitivity analyses varying assumptions on key model parameter estimates confirmed results, with benralizumab having lower total direct health care costs in all scenarios tested, and showed that model results were most sensitive to changes in biologic costs and exacerbation reduction rates. CONCLUSIONS: Patients receiving benralizumab had higher nonbiologic cost offsets because of reductions in exacerbations and OCS-dependent years, leading to greater cost savings for third-party payers compared with patients receiving mepolizumab or dupilumab. Taken together with biologic costs, benralizumab presents greater savings in health care costs for payers than patients with SEA who use mepolizumab or dupilumab. DISCLOSURES: This study was funded by AstraZeneca (Cambridge, UK). Drs Xu, Chung, Genofre, and Katial are or were AstraZeneca employees at the time this research was conducted and may be shareholders of AstraZeneca. Ms Schaefer and Dr Szende are employees of Labcorp Drug Development, which received funding from AstraZeneca to perform this research.

We conducted a systematic literature review of treatment patterns of eosinophilic esophagitis (EoE) to understand patients who may benefit from novel treatment approaches. The search included the Embase, MEDLINE, Cochrane and Web of Science databases, and relevant observational studies (articles published 1 January 2015 to 20 April 2022) were included. Results were screened against predetermined criteria by two independent reviewers and adjudicated by a third per PRISMA guidelines. Of 311 articles screened, 45 met inclusion criteria; among these, study designs varied. Based on included studies, 16%-100% (median: 74%) of all EoE patients initiate pharmacotherapy; most commonly with PPIs (0%-67%), topical steroids (16%-56%) or combination therapy (0%-20%). Between 6% and 27% go on to receive second-line therapy. Between 72% and 94% (median: 80%) of all EoE patients are reported to be on pharmacotherapy (37%-61% PPIs; 14%-40% topical steroids; 16%-48% combination therapy) at any time. A proportion of patients do not achieve a response to treatment and 30%-67% relapse to active eosinophilia (≥15 eos/hpf). Stricture is reported in up to 30% of EoE patients at initial presentation and in 4%-49% during clinical follow-up. Among patients on treatment, up to 23% undergo ≥1 esophageal dilation procedure; up to 37% experience a food impaction, with higher rates among those undergoing ≥1 dilation; and up to 29% experience a food impaction requiring urgent medical intervention. Treatment patterns indicate a proportion of patients experiences manifestations of more severe disease, despite treatment, pointing to an unmet need in this subgroup that may benefit from novel treatment approaches.

OBJECTIVES:Real-world evidence (RWE) is essential for the development of pharmaceutical and medical technologies and informs treatment-related decisions by regulatory agencies, payers, healthcare providers, and patients. Given that planning RWE studies present diverse challenges, we developed the RWE Framework, a concise, visual, interactive tool designed to align multidisciplinary stakeholders toward common goals and encourage a methodical approach to RWE study planning.METHODS:A search of published literature and internet-based resources was performed to identify guidance on RWE study planning with decision and/or visual aids. A conceptual framework for a study design tool was developed based on best practices for RWE studies, enhanced with an infographic design, and refined by multidisciplinary input from RWE researchers.RESULTS:The searches confirmed an unmet need for a concise tool to support a broad range of RWE study designs: only two sources with decision/visual aids were identified. The novel RWE Framework comprises sequential decision steps with instructions to guide users through consideration of research objectives, product approval status, study setting, outcomes of interest, data availability in routine practice, need for primary data collection and/or randomization, study type and methodology, and applicable regulatory standards. Pilot testing using case studies of pharmaceutical assets demonstrated the utility of RWE Framework and applicability for use in team environments.CONCLUSIONS:The RWE Framework is a novel, concise, visual, and interactive tool to inform RWE study planning. It addresses a broad range of real-world study types and research objectives and was found to enhance RWE decision-making by multidisciplinary teams. Further validation is warranted.

Background: Longitudinal research on outcomes of patients with fibromyalgia is limited. Objective: To assess clinician and patient-reported outcomes over time among fibromyalgia patients. Methods: At enrollment (Baseline) and follow-up (approximately 2 years later), consented patients were screened for chronic widespread pain (CWP), attended a physician site visit to determine fibromyalgia status, and completed an online questionnaire assessing pain, sleep, function, health status, productivity, medications, and healthcare resource use. Results: Seventy-six fibromyalgia patients participated at both time points (at Baseline: 86.8% white, 89.5% female, mean age 50.9 years, and mean duration of fibromyalgia 4.1 years). Mean number of tender points at each physician visit was 14.1 and 13.5, respectively; 11 patients no longer screened positive for CWP at follow-up. A majority reported medication use for pain (59.2% at Baseline, 62.0% at Follow-up). The most common medication classes were opioids (32.4%), SSRIs (16.9%), and tramadol (14.1%) at Follow-up. Significant mean changes over time were observed for fibromyalgia symptoms (modified American College of Rheumatology 2010 criteria: 18.4 to 16.9; P=0.004), pain interference with function (Brief Pain Inventory-Short Form: 5.9 to 5.3; P=0.013), and sleep (Medical Outcomes Study-Sleep Scale: 58.3 to 52.7; P=0.004). Patients achieving ≥2 point improvement in pain (14.5%) experienced greater changes in pain interference with function (6.8 to 3.4; P=0.001) and sleep (62.4 to 51.0; P=0.061). Conclusion: Fibromyalgia patients reported high levels of burden at both time points, with few significant changes observed over time. Outcomes were variable among patients over time and were better among those with greater pain improvement.

Background A previous fibromyalgia (FM) research reports that 20%–47% of diagnosed patients may not meet the study definition of FM 1–2 years after diagnosis. The aim of this study was to gain a better understanding of the progression of FM in a geographically diverse cohort over a 2-year time period. Methods This cohort study followed 226 subjects recruited online to assess FM and chronic widespread pain (CWP) diagnosis stability over time. At enrollment (baseline), subjects provided informed consent, completed an online questionnaire consisting of the London Fibromyalgia Epidemiology Study Screening Questionnaire to screen for CWP (bilateral pain above/below waist lasting ≥1 week in the past 3 months), visited a site for physician evaluation for FM, and completed a questionnaire with validated patient-reported outcome instruments. Subjects were classified into mutually exclusive groups: FM+CWP+ (screened positive for CWP and received physician diagnosis of FM), FM−CWP+ (screened positive for CWP but did not receive physician diagnosis of FM), and FM−CWP− (screened negative for CWP). Approximately 2 years later (follow-up), subjects were reassessed at the same study site and completed a questionnaire with the same patient-reported outcomes. Results Seventy-six FM+CWP+ subjects completed assessments at both time points; 56 (73.7%) met the FM study definition at follow-up. Twenty subjects no longer met the FM study definition (eleven became FM−CWP− and nine became FM−CWP+). Ten subjects (two from FM−CWP− and eight from FM−CWP+) transitioned into the FM+CWP+ group at follow-up; they reported more tender points and pain interference with sleep and worse physical function at baseline compared with subjects who did not transition to FM+CWP+. Most (76.7%) of the subjects who transitioned into/out of FM+CWP+ experienced changes in CWP, number of positive tender points, or both. Conclusion The results suggest that some FM+CWP+ patients experience fluctuation in symptoms over time, which may reflect the waxing and waning nature of FM and affect diagnosis and treatment.

Background/PurposeLittle information exists on the comparative patient and economic burden of chronic widespread pain (CWP) and fibromyalgia (FM) in the United States.MethodsThis multistage, observational study included an online screening survey of a large geographically diverse US sample to assess CWP status, a physician/site visit to determine FM diagnosis, and an online subject questionnaire to capture clinical characteristics, pain, health status, functioning, sleep, healthcare resource use (HRU), productivity, and costs. Based on the screener and physician evaluation, mutually exclusive groups of subjects without CWP (CWP-), with CWP but without FM (CWP+), and with confirmed FM were identified.ResultsDisease burden was examined in 472 subjects (125 CWP-, 176 CWP+, 171 FM). Age, race, and ethnicity were similar across groups. Mean body mass index and number of comorbidities increased from CWP- to CWP+ to FM (P=0.0044, P<0.0001, respectively). From CWP- to CWP+ to FM, there were reductions in health status (EQ-5D, SF-12) and sleep outcomes (MOS-SS, SSQ) (all P<0.05). Pain severity, interference with function (BPI-SF), and overall work impairment (WPAI:SHP) increased from CWP- to CWP+ to FM (all P<0.0001). Higher proportions of CWP+ (52.8%) and FM subjects (62.6%) were taking pain-related prescription medications relative to CWP- subjects (32.8%; P<0.0001). Significant differences in total direct and indirect costs across the three groups (both P<0.0001) were observed, with highest costs among FM subjects.ConclusionFibromyalgia subjects were characterized by the greatest disease burden with more comorbidities and pain-related medications, poorer health status, function, sleep, lower productivity, and higher costs.

Objective. The aim of this study was to evaluate patient-reported burden associated with peripheral and central neuropathic pain (NeP) by pain severity and NeP condition. Design. Six hundred twenty-four subjects with one of six NeP conditions were recruited during routine office visits. Subjects consented to retrospective chart review and completed a one-time questionnaire (including EuroQol-5 dimensions, 12-item Short-Form Health Survey, Brief Pain InventoryShort Form, Medical Outcomes Study Sleep Scale, Hospital Anxiety and Depression Scale, and demographic and clinical characteristics). Pain severity scores were used to stratify subjects by mild, moderate, and severe pain. Summary statistics and frequency distributions were calculated. Differences by severity level were compared using Kruskal– Wallis (continuous variables) and chi-square or Fisher’s exact test (categorical variables). Effect size was computed with Cohen’s d (mild vs severe). Results. Subjects’ mean age was 55.5. The majority (80.8%) had moderate or severe pain. Patientreported outcomes (health status, physical and mental health, pain interference with function, sleep, anxiety, and depression) were significantly

ADPKD imposes substantial burdens on HRQoL at all stages of the disease. Few studies have examined disease impacts and symptomatology on HRQoL. This analysis aimed to assess whether disease-specific questionnaires allow for improved assessment of HRQoL in ADPKD. In a large global observational study of 3,409 subjects, patient-reported outcomes (PRO) instruments (including SF-12v2, EQ-5D, ADPKD-IS) were completed in 6-month intervals. Scores were calculated at Baseline and longitudinally, overall and by chronic kidney disease (CKD) stage. The overall population scores were normal/near normal regardless of instrument with little change overtime (mean score at Baseline and 18 months): SF-12 Mental Component Summary score (MCS) 50.15 to 49.77, SF-12 Physical Component Summary score (PCS) 49.94 to 49.12; EQ-5D US Index 0.91 to 0.90; ADPKD-IS Physical 1.59 to 1.66, Emotional 1.78 to 1.79, Fatigue 1.90 to 1.95. Only the SF-12 PCS and ADPKD-IS instruments were able to differentiate between early and late stage disease at Baseline (CKD1 vs CKD4 at baseline (SD): PCS 52.22 (8.41) vs 46.79 (9.89), MCS 49.66 (9.90) vs 51.09 (9.63), EQ-5D US Index 1.06 (0.25) vs 1.13 (0.34), ADPKD-IS Physical 1.47 (0.82) vs 1.83 (0.88), Emotional 1.72 (0.88) vs 2.07 (0.91), Fatigue 1.77 (1.03) vs 2.25 (1.23)). Longitudinal changes (Baseline and 18 months) could only be measured in later disease stages. Changes in EQ-5D and SF-12 were driven by impacts on overall health state. The disease-specific ADPKD-IS questionnaire was more sensitive to HRQoL in a cross-sectional analysis by disease severity vs all generic HRQoL instruments except SF-12 PCS. In a slowly progressing disease like ADPKD, observations of longitudinal change in HRQoL will require longer follow-up. These results support that ADPKD disease impacts are incapable of being measured by blunt generic tools.

Fibromyalgia (FM) is characterized by chronic widespread pain (CWP), fatigue, and functional limitations. Previous research suggests remission from FM and its symptoms is rare, though some studies report 20%-47% of diagnosed patients may no longer meet the study definition of FM at 1-2 years after diagnosis.1 This study followed a cohort of 226 subjects recruited online to assess transitions into and out of FM over 2 years. At enrollment (Year 1) subjects completed a brief online questionnaire to screen for CWP (bilateral pain, above/below waist lasting ≥1 week) based on the 4 pain questions of the London Fibromyalgia Epidemiology Study Screening Questionnaire (LFESSQ), site visit for physician evaluation of FM, and another online questionnaire with several validated patient-reported outcome instruments. Subjects were classified into mutually-exclusive groups: FM (study definition required CWP-positive screen and physician diagnosis of FM), CWP+ (CWP-positive screen without physician diagnosis of FM), and CWP- (CWP-negative screen). At the follow-up visit (approximately 2 years later), subjects completed the same questionnaires and were reassessed by the physician. Among FM subjects who completed assessments at both time points (n=76), 56 (73.7%) met the study definition of FM at follow-up. Twenty subjects no longer met the FM study definition (11 switched to CWP-, 9 to CWP+). Ten subjects (2 from CWP-, 8 from CWP+) transitioned into the FM group; they reported greater number of tender points, lower physical function, and more sleep disturbance in Year 1 compared with subjects who did not transition to FM. Most subjects (23/30, 76.7%) who transitioned into/out of FM experienced corresponding changes in CWP, number of positive tender points (≥11), or both. Results suggest some patients may experience fluctuation in symptoms over time, which may reflect the waxing and waning nature of FM and further complicate diagnosis. (1. White, Curr Pain Headache Rep, 2001) Research supported by Pfizer Inc.

Background Published findings on natural history of fibromyalgia (FM) are limited. Objectives The multisite US prospective observational cohort study followed FM subjects to assess disease burden and natural history. Methods At enrollment, consented subjects completed an online questionnaire to screen for chronic widespread pain (CWP, pain on both sides and above and below waist for ≥1 week); a physician site visit to determine FM diagnosis; and another online questionnaire to capture demographics, clinical characteristics, medications, healthcare resource use (HRU), pain (Brief Pain Inventory-Short Form [BPI-SF]), FM (modified [self-report] American College of Rheumatology [ACR] 2010 criteria, Revised Fibromyalgia Impact Questionnaire [FIQ-R]), sleep (Medical Outcomes Study-Sleep Scale [MOS-SS]), and health status (Short Form 12-item Health Survey [SF-12]). At follow-up, approximately 2 years later, subjects and physicians completed the same assessments. Statistical significance of changes was tested (p<0.05) using analysis of covariance and generalized McNemar9s test. Results A total of 76 FM subjects participated at both time points (at enrollment: 89.5% female, mean age 50.9 years, mean duration of FM 4.1 years, 86.8% white). Mean number of tender points at each physician visit was 14.1 and 13.5, respectively. Distribution of FM severity based on physician evaluation at enrollment was 19.7% mild/very mild, 48.7% moderate, 31.6% severe/very severe, and at follow-up was 15.6% mild/very mild, 43.8% moderate, 40.6% severe/very severe. At follow-up, 20.3% of subjects reported their overall health status had improved since enrollment, while 47.5% reported a decline. Comorbidities reported by ≥20% of subjects at follow-up: arthritis, lower back pain, depression, high cholesterol, hypertension, headache/migraine, anxiety, and sleep apnea. Medication use for pain was reported by 59.2% and 62.0% of subjects at each time point, respectively. Most common medication classes were opioids (32.4%), SSRIs (16.9%), and tramadol (14.1%) at follow-up. Mean number of healthcare provider visits for pain over the past 3 months (among those with ≥1) was 5.4 and 4.9 at each time point, respectively. There were no statistically significant changes in pain-related HRU. Significant mean changes (p-value <0.05) over time were observed for mean modified ACR 2010 criteria score (18.4 to 16.9, p=0.004), BPI-SF pain interference index (5.9 to 5.3, p=0.013), and MOS-SS overall sleep problems index (58.3 to 52.7, p=0.004). No statistically significant differences were observed for BPI-SF pain severity index (5.2 to 5.1), FIQ-R overall score (53.2 to 53.2), SF-12 physical (32.8 to 34.1) and mental (41.9 to 42.4) component summary scores. Conclusions FM patients continued to report high levels of disease burden at follow-up. There was variability among patients in outcomes, with few significant differences over time. Data suggest improvement in symptoms, sleep and pain interference with function due potentially to active management (pharmacologic, nonpharmacologic) of the condition. Disclosure of Interest C. Schaefer Consultant for: Pfizer Inc (paid consulting services provided by Covance Market Access Services), E. Adams Consultant for: Pfizer Inc (paid consulting services provided by Covance Market Access Services), M. Udall Shareholder of: Pfizer Inc., Employee of: Pfizer Inc., E. Masters Shareholder of: Pfizer Inc., Employee of: Pfizer Inc., R. Mann Consultant for: Pfizer Inc (paid consulting services provided by Covance Market Access Services), S. Daniel Consultant for: Pfizer Inc (paid consulting services provided by Covance Market Access Services), H. McElroy Consultant for: Pfizer Inc (paid consulting services provided by Covance Market Access Services), J. Cappelleri Shareholder of: Pfizer Inc., Employee of: Pfizer Inc., A. Clair Shareholder of: Pfizer Inc., Employee of: Pfizer Inc., M. Hopps Employee of: Pfizer Inc., R. Staud Grant/research support from: Investigator initiated grant from Pfizer Inc., P. Mease Grant/research support from: Pfizer Inc., Consultant for: Pfizer Inc., Speakers bureau: Pfizer Inc., S. Silverman Grant/research support from: Pfizer Inc., Consultant for: Pfizer Inc. and Lilly, Speakers bureau: Pfizer Inc. and Lilly

Background: Comprehensive studies on costs of moderate to severe plaque psoriasis (MSPP) have not been conducted in the United States.Objective: We sought to evaluate current health care resource use, productivity, and costs among patients with MSPP in routine practice.Methods: A total of 200 adults seeking MSPP treatment enrolled in 9 US sites. Consented patients reported symptoms, treatment, lost productivity, and costs; 6-month retrospective chart review captured health care resource use and clinical characteristics. Costs were assigned to health care resource use and lost productivity using standard algorithms. Differences by Psoriasis Area and Severity Index (PASI) group, based on PASI score (<= 10, >10-<= 20, >20) at enrollment, were evaluated. Analyses included descriptive statistics and analysis of variance or Kruskal-Wallis tests.Results: Most patients (79.5%) were prescribed 1 or more MSPP medications (mean: 1.5); 36.0% and 9.0% received self-administered biologics and systemic therapies, respectively. Mean number of nonprescription treatments was 12.3. Differences by PASI group were observed for overall work and activity impairment (P<.02). Six-month total MSPP direct costs per patient were $11,291; indirect costs were $2101 and differed across PASI groups (P =.0008).Limitations: This study enrolled patients with MSPP actively seeking care.Conclusion: Despite treatment, a number of patients with MSPP continue to experience moderate to severe PASI scores, impaired functioning, and high costs suggesting a need for new treatment options.

BACKGROUND:Autosomal dominant polycystic kidney disease (ADPKD) results in kidney cyst development and enlargement, resulting in chronic kidney disease (CKD) leading to renal failure. This study sought to determine if ADPKD patients in the early stages of CKD contribute to a sizable economic burden for the US health care system.METHODS:This was a retrospective, matched cohort study, reviewing medical resource utilization (MRU) and costs for adults in a US private-payer claims database with a diagnosis code of ADPKD (ICD-9-CM 753.13). ADPKD patients were matched by age grouping (0-17, 18-34, 35-44, 45-54, 55-64, and 65+ years) and sex to controls to understand the burden of ADPKD. Descriptive statistics on 6-month MRU and costs were assessed by CKD stages, dialysis use, or previous renal transplant.RESULTS:The analysis included ADPKD patients in CKD stages 1-5 (n=316 to n=860), dialysis (n=586), and post-transplant (n=615). Mean ages did not differ across CKD stages (range 43-56 years). Men were the majority in the later stages but the minority in the early stages. The proportion of patients with at least one hospitalization increased with CKD stage, (12% to >40% CKD stage 2 to stage 5, dialysis or post-transplant). The majority had at least one hospital outpatient visit and at least one pharmacy claim. Total 6-month per-patient costs were greater among ADPKD patients than in age-matched and sex-matched healthy non-ADPKD controls (P<0.001 for all comparisons).CONCLUSION:ADPKD patients with normal kidney function are associated with a significant economic burden to the health care system relative to the general population. Any treatments that delay progression to later stages of CKD may provide potential health care cost offsets.

BACKGROUND:HIV-related neuropathic pain (HIV-NeP) is common; however, the burden of HIV-NeP is not well-understood.METHODS:The cross-sectional study aimed to characterize the HIV-NeP burden. A total of 103 patients with HIV-NeP recruited during routine office visits completed a questionnaire to assess patient-reported outcomes, including pain severity, health status, sleep, mood, and lost productivity. Physicians completed a 6-month retrospective chart review.RESULTS:The sample was predominantly male and not employed for pay. A majority (75.7%) of patients experienced moderate or severe pain. Pain interference, general health, physical health, and depression were worse among patients with more severe pain (all Ps < .006). Most (87.4%) patients were prescribed at least 1 medication for NeP. HIV-related neuropathic pain was associated with 36.1% work impairment. Adjusted annualized costs increased with increasing pain severity (P < .0001).CONCLUSION:The impact of HIV-NeP on health status, physical function, and depression increases with severity, resulting in substantial clinical and economic burden.

To evaluate disease burden, clinical and patient-reported outcomes and healthcare costs of patients admitted to hospital for management of plaque or erythrodermic psoriasis. This observational study enrolled 61 eligible patients from 107 hospital stays across 9 UK hospitals. Sites recorded Psoriasis Area Severity Index (PASI) at admission and discharge, psoriasis treatments, and length of stay (LOS). Patients reported psoriasis-related symptoms, health status (SF-12v2, EQ-5D-3L), mood (HADS), productivity (WPAI), and dermatology-related quality of life (DLQI) at admission, and also reported psoriasis-related symptoms, EQ-5D-3L, and DLQI at discharge. An algorithm assigned cost/hospital stay. Descriptive statistics are based on those responding to each item. Statistical significance evaluated at the 0.05 level. Mean age was 45.5 years; 50.8% were male. Mean time since psoriasis diagnosis was 20.0 years. Most (78.7%) had ≥1 previous psoriasis-related hospitalization. Mean number of physician-diagnosed co-morbid conditions was 2.5. At admission, mean SF-12v2 Physical and Mental component summary scores were 35.4 and 32.1, respectively; mean HADS scores were 9.7 (anxiety) and 9.6 (depression) indicating substantial impairment. Forty-five percent reported changing job, role, or position at work due to psoriasis. Mean WPAI activity impairment at admission was 68.7%; among the 35.1% employed for pay, mean WPAI work impairment was 79.2%. Mean PASI improved from admission to discharge (25.2→12.1; p<0.0001). Also, improvement was seen at discharge for EQ-5D-3L (0.34 →0.60), DLQI total score (20.1→12.0), and psoriasis symptom scores (all p<0.05). Mean (range) LOS was 17.0 (2,71) days; for 8 patients achieving a 75% reduction in PASI (PASI75), mean LOS was 18.1 vs. 13.1 days for 27 patients not achieving PASI75 (p=0. 11). Mean (SD) cost/hospital stay was £4,875 (±£3,096). Disease burden, LOS and cost are substantial among patients hospitalized for psoriasis. On average, patients improved during their hospital stay. Nonetheless, they reported suboptimal clinical and patient-reported outcomes at discharge.

OBJECTIVE:To characterize the burden of idiopathic painful peripheral neuropathy with small fiber involvement (idiopathic SFN) by pain severity in the US. METHODS:One hundred previously diagnosed idiopathic SFN subjects were enrolled during routine office visits. Subjects completed a one-time questionnaire, and investigators reported clinical characteristics and healthcare resource use, based on 6 month retrospective chart review. Annualized direct and indirect costs were estimated. Results were stratified across pain severity groups. RESULTS:Mean age was 63.5 years; 53.0% were female; 76.0% had moderate or severe pain. Most common comorbidities were sleep disturbance/insomnia (37.0%), anxiety (34.0%), and depressive symptoms (33.0%). Overall mean health status (0.59; -0.11-1.00 scale), physical and mental health (31.7 and 45.6, respectively, 0-100 scale), sleep index (45.1; 0-100 scale), and pain interference with function (5.0; 0-10 scale) differed by pain severity, with worse outcomes among those with greater pain (all p < 0.002). 84.0% were prescribed ≥1 SFN medication. 16.0% were employed; mean overall work impairment was 36.9%. Annualized average adjusted direct and indirect costs per subject ($8055 and $13,733, respectively) differed by pain severity. CONCLUSIONS:Idiopathic SFN subjects with pain experience moderate or severe pain, which negatively impacts health status, function, and productivity, and leads to substantial direct and indirect costs.

Autosomal dominant polycystic kidney disease (ADPKD) is the most common type of polycystic kidney disease (PKD) and the fourth leading cause of end-stage renal disease. Currently, there is no published information on medical resource utilisation (MRU) in patients with ADPKD in Europe. This study aimed to better understand MRU associated with ADPKD by disease stage in six European countries (Germany, France, the United Kingdom, Italy, Spain and Sweden). This study was a retrospective review of medical charts collected via an online physician survey. Participating physicians abstracted data for the last three eligible ADPKD patients treated in their practice, each within a different Chronic Kidney Disease (CKD) stage. Data collected over the past 2 years included socio-demographics, clinical characteristics and MRU, such as diagnostic tests, specialist visits, dialysis, emergency department visits, inpatient admissions and medications. A total of 1,055 ADPKD patients were enrolled. The mean (SD) age of the sample was 50.4 (14.4) years, 53.6% were male, with 39.4% employed full time. Only 40% of patients had a known PKD genotype, of which 83% had PKD-1 and 17% had PKD-2. Almost all patients (99.1%) experienced at least 1 ADPKD-related complication, most commonly hypertension (75.4%). On average (SD), patients had 4.4 (9.3) annual specialist visits. Approximately 19.7% of patients experienced at least 1 hospitalisation over the 2 year study period with a mean (SD) length of stay of 7.0 (8.5) days. Patients in more advanced disease stages reported higher mean (SD) annual specialist visits and more disease-related hospitalisations, 1.5 (1.1) and 9.2% in CKD Stage 1 compared to 10.0 (17.2) and 32.4% in Dialysis, respectively. This is the first study to provide evidence on MRU among patients with ADPKD in Europe. Results demonstrate that ADPKD patients require substantial MRU, which accumulate with disease progression.