Patient preferences for treatment attributes in moderate-to-severe atopic dermatitis: a discrete choice experiment.

Purpose: Evidence on treatment preferences of patients with moderate-to-severe atopic dermatitis (AD) in the United States (US) is limited and an assessment of treatment preferences in this group is warranted.Materials and methods: An online discrete choice experiment survey was conducted (June 2023) among US adults with self-reported moderate-to-severe AD or experience with systemic therapy who had inadequate response to topical treatments. Preference weights estimated from conditional logistic regression models were used to calculate willingness to trade off and attributes' relative importance (RI).Results: Participants (N = 300; mean age: 45 years; 70% females; 52% systemic therapy experienced) preferred treatments with higher efficacy, lower risk of adverse events (AEs), and less frequent blood tests (p < .05). Treatment attributes, from high to low RI, were itch control (38%), risk of cancer (23%), risk of respiratory infections (18%), risk of heart problems (11%), sustained improvement in skin appearance (5%), blood test frequency (3%), and frequency and mode of administration (2%); together, AE attributes accounted for more than half of the RI.Conclusions: Participants preferred AD treatments that maximize itch control while minimizing AE risks, whereas mode of administration had little impact on preferences. Understanding patients' preferences may help improve shared decision-making, potentially leading to enhanced patient satisfaction with treatment, increased engagement, and better clinical outcomes.

Respiratory syncytial virus hospitalization and mortality: Systematic review and meta-analysis.

BACKGROUND: Respiratory syncytial virus (RSV) is a major public health burden worldwide. We aimed to review the current literature on the incidence and mortality of severe RSV in children globally. METHODS: Systematic literature review and meta-analysis of published data from 2000 onwards, reporting on burden of acute respiratory infection (ARI) due to RSV in children. Main outcomes were hospitalization for severe RSV-ARI and death. RESULTS: Five thousand two hundred and seventy-four references were identified. Fifty-five studies were included from 32 countries. The global RSV-ARI hospitalization estimates, reported per 1,000 children per year (95% Credible Interval (CrI), were 4.37 (2.98, 6.42) among children <5 years, 19.19 (15.04, 24.48) among children <1 year, 20.01 (9.65, 41.31) among children <6 months and 63.85 (37.52, 109.70) among premature children <1 year. The RSV-ARI global case-fatality estimates, reported per 1,000 children, (95% Crl) were 6.21 (2.64, 13.73) among children <5 years, 6.60 (1.85, 16.93) for children <1 year, and 1.04 (0.17, 12.06) among preterm children <1 year. CONCLUSIONS: A substantial proportion of RSV-associated morbidity occurs in the first year of life, especially in children born prematurely. These data affirm the importance of RSV disease in the causation of hospitalization and as a significant contributor to pediatric mortality and further demonstrate gestational age as a critical determinant of disease severity. An important limitation of case-fatality ratios is the absence of individual patient characteristics of non-surviving patients. Moreover, case-fatality ratios cannot be translated to population-based mortality. Pediatr Pulmonol. 2017;52:556-569. © 2016 The Authors. Pediatric Pulmonology. Published by Wiley Periodicals, Inc.

End-stage renal disease in autosomal dominant polycystic kidney disease: a comparison of dialysis-related utilization and costs with other chronic kidney diseases.

BACKGROUND: Autosomal dominant polycystic kidney disease (ADPKD) is the leading inheritable cause of end-stage renal disease (ESRD) and one of the leading causes of ESRD overall. ADPKD patients differ from the overall dialysis population; however, there is little published data regarding health care costs for ADPKD patients on dialysis. METHODS: This retrospective observational cohort study was designed to quantify health care utilization and costs for ADPKD patients with ESRD who received initial services at a single large dialysis organization between January 1, 2007 and December 31, 2009. Parallel results and baseline patient characteristics for control patients with ESRD etiologies other than ADPKD were performed for reference. Dialysis-related utilization and health care costs for patients with ADPKD in ESRD overall and during time horizons that correspond to Medicare-eligibility benchmarks were analyzed. Baseline patient characteristics were described for all patients and included demographics, comorbid illnesses, and clinical characteristics. Dialysis-related utilization, hospitalization rates, and health care costs were considered longitudinally. RESULTS: Total health care costs for ADPKD patients were high at US$51,048 per patient-year based on the overall analysis. Total health care costs were lower for ADPKD patients than for control patients on dialysis. Patients with ADPKD were generally younger, had a lower Charlson Comorbidity Index, and had lower rates of comorbid conditions, which may have contributed to the lower overall costs seen for patients with ADPKD. CONCLUSION: Health care resource utilization and costs for patients with ADPKD in ESRD requiring dialysis were high, and therapeutic interventions that can prevent or delay the progression to ESRD may increase dialysis-free life for patients with ADPKD.

Consequences of hemolytic uremic syndrome among hemodialysis patients.

BACKGROUND: Hemolytic uremic syndrome (HUS) is characterized by hemolytic anemia, low platelets, and renal impairment and is mediated by thrombotic microangiopathy (TMA). A common perception is that HUS becomes dormant in dialysis patients with end-stage renal disease (ESRD). We analyzed patients in a large dialysis organization to understand the potential consequences and burden of HUS. METHODS: We identified patients with ESRD ascribed to HUS and those with ESRD ascribed to another cause (control patients) who received hemodialysis or peritoneal dialysis from 01 January 2007 to 31 December 2012. Outcomes were survival, hospitalization, and longitudinal laboratory values associated with TMA, including lactate dehydrogenase, red cell distribution width (RDW), platelets, and hemoglobin. RESULTS: HUS patients (n = 217) were propensity-score matched 1:5 to control patients (n = 1,085) for age, gender, race, dry weight, insurance, access, comorbidities, and Charlson comorbidity index. Compared to control patients, HUS patients had significantly greater risk for hospitalizations overall (RR = 2.3, p = 0.004) and hospitalization for hematologic (RR = 5.6, p = 0.001), cardiovascular (RR = 2.1, p = 0.02), and pancreatic (RR = 7.9, p = 0.04) causes. HUS patients also had evidence of ongoing TMA: higher lactate dehydrogenase and RDW, lower platelets and hemoglobin, and more frequent lactate dehydrogenase spikes. CONCLUSIONS: Dialysis patients with HUS were at significantly higher risk than matched control patients for hospitalizations due to cardiovascular, hematologic, and pancreatic disease, which were associated with ongoing TMA. Additional studies are needed to determine whether targeted therapy for HUS reduces hospitalizations.

The economic impact of improving phosphate binder therapy adherence and attainment of guideline phosphorus goals in hemodialysis patients: a Medicare cost-offset model.

INTRODUCTION: Hyperphosphatemia (serum phosphorus >5.5 mg/dL) in hemodialysis patients is a key factor in mineral and bone disorders and is associated with increased hospitalization and mortality risks. Treatment with oral phosphate binders offers limited benefit in achieving target serum phosphorus concentrations due to high daily pill burden (7-10 pills/day) and associated poor medication adherence. The economic value of improving phosphate binder adherence and increasing percent time in range (PTR) for target phosphorus concentrations has not been previously assessed in dialysis patients. The current retrospective analysis was conducted to summarize health care cost savings to United States (US) payers associated with improved phosphate binder adherence and increased PTR for target phosphorus concentrations in adult end-stage renal disease (ESRD) patients receiving hemodialysis therapy. METHODS: Phosphate binder adherence and PTR were derived from hemodialysis patients who were treated at a large dialysis organization between January 2007 and December 2011. Cost model inputs were derived from US Renal Data System data between July 2007 and December 2009. A cost-offset model was constructed to estimate monthly and annual incremental health care costs (total Medicare; inpatient, outpatient, and Medicare Part B) associated with different levels of phosphate binder adherence and PTR. Model inputs included number of ESRD patients, population adherence to phosphate binders, PTR associated with adherence to phosphate binders, and per-patient per-month cost associated with PTR. A base case model estimated monthly and annual costs of phosphate binder therapy in the population using estimated model inputs. The estimated adherence rate was used to determine number of patients in compliant and noncompliant groups. Monthly costs were calculated as the sum of per-patient per-month cost times the number of patients in adherent and nonadherent groups. Annual costs were monthly costs times 12 and assumed the same level of adherence, PTR, and per-patient per-month costs over time. To study the impact of improving phosphate binder adherence and PTR on cost outcomes, we hypothetically and simultaneously increased both base phosphate binders adherence and PTR for adherent patients (adherence/PTR: 10/20%, 20/40%, 30/60%). Monthly and annual costs were derived for each scenario and compared against the results of the base case model. One-way sensitivity analysis was performed to test model robustness. RESULTS: The base case model estimated total Medicare and inpatient costs of $5,152,342 and $1,435,644, respectively (N = 1,000). When base case model costs were compared to results of each extended model scenario, overall Medicare cost savings (range 0.3-1.9%) and inpatient cost savings (range 1.2-5.7%) were observed. The one-way sensitivity analysis indicated that results were sensitive to PTR for adherent and nonadherent patients and the factor used to increase adherence rate and PTR associated with adherence in the hypothetical scenarios. However, cost savings in overall Medicare costs and inpatient costs were still noted. CONCLUSION: Increasing phosphate binder adherence and improving phosphorus control were associated with increased cost savings in total Medicare costs and inpatient costs.

Clinical characteristics and outcomes of end-stage renal disease patients with self-reported pruritus symptoms.

One of the most common conditions affecting end-stage renal disease (ESRD) patients undergoing hemodialysis (HD) is pruritus. Studies report that itchy and dry skin, symptoms of pruritus, affect 40%-90% of ESRD patients. Yet, in clinical practice the condition is often underdiagnosed resulting in inadequate management and an underappreciated impact on patient outcomes. Two retrospective analyses were conducted: a preliminary analysis of ESRD patients with pruritus symptoms (n=73,124) undergoing HD or peritoneal dialysis at a large dialysis provider and a subsequent detailed analysis of a homogenous subset of patients undergoing in-center HD (n=38,315). The goal was to better understand the clinical burden of pruritus as it relates to patient characteristics, quality of life, medication use, and HD compliance. This population is commonly burdened by multiple comorbidities and related polypharmaceutical management; identifying the relationship of pruritus to these ailments can help guide future research and resource allocation. The detailed analysis confirmed trends observed in the preliminary analysis: 30% reported being "moderately" to "extremely bothered" by itchiness. The HD patient population with the highest severity of self-reported pruritus also had a consistent trend in overall increased resource utilization - higher monthly doses of erythropoietin-stimulating agents (53,397.1 to 63,405.4 units) and intravenous (IV) iron (237.2 to 247.6 units) and higher use of IV antibiotics (14.1% to 20.7%), as well as poorer quality-of-life measures (25-point reductions in Burden of Disease Score and Effects on Daily Life subscales of the Kidney Disease Quality of Life-36 survey). These results highlight the need to better identify and manage ESRD patients impacted by pruritus, as this symptom is associated with negative clinical outcomes and increased resource utilization. Further studies are needed to evaluate the current economic burden of pruritus in ESRD patients and create possible options for an improved pharmacoeconomic profile in this patient population.

Economic implications of treatment-resistant depression among employees.

BACKGROUND: Conservative estimates indicate between 10% and 20% of all individuals with major depressive disorders (MDDs) fail to respond to conventional antidepressant therapies. Amongst those with MDD, individuals with treatment-resistant depression (TRD) have been found to be frequent users of healthcare services and to incur significantly greater costs than those without TRD. Given the prevalence of the disorder, it is understandable that MDDs are responsible for a significant amount of both direct and indirect healthcare costs. OBJECTIVE: To provide empirical findings for employees likely to have TRD based on analysis of employer claims data, in the context of previous research. METHODS: We conducted a claims data analysis of employees of a large national (US) employer. The data source consisted of medical, pharmaceutical and disability claims from a Fortune 100 manufacturer for the years 1996-1998 (total beneficiaries >100000). The employee sample included individuals with medical or disability claims for MDDs (n = 1692). A treatment pattern algorithm was applied to classify MDD patients into TRD-likely (n = 180) and TRD-unlikely groups. Treated prevalence of select comorbid conditions and the patient costs (direct and indirect) from the employer perspective by condition were compared among TRD-likely and TRD-unlikely employees, and with a 10% random sample of the overall employee population for 1998. RESULTS: The average annual cost of employees considered TRD-likely was dollars US 14490 per employee, while the cost for depressed but TRD-unlikely employees was dollars US 6665 per employee, and dollars US 4043 for the employee from the random sample. TRD beneficiaries used more than twice as many medical services compared with TRD-unlikely patients, and incurred significantly greater work loss costs. CONCLUSION: TRD has gained increasing recognition due to both the clinical challenges and economic burdens associated with the condition. TRD imposes a significant economic burden on an employer. TRD-likely employees are more likely to be treated for selected comorbid conditions and have higher medical and work loss costs across all conditions.

Attention-deficit/hyperactivity disorder: increased costs for patients and their families.

OBJECTIVE: To estimate the direct (medical and prescription drug) and indirect (work loss) costs of children treated for attention-deficit/hyperactivity disorder (ADHD) and their family members. METHOD: The data source was an administrative database from a national, Fortune 100 manufacturer that included all medical, pharmaceutical, and disability claims for beneficiaries (n > 100,000). The analysis involved four samples. The ADHD patient sample included individuals age 18 or younger with at least one ADHD claim during the study period (1996-1998). Resource utilization of ADHD patients was contrasted with a matched control sample of patients who did not have claims for ADHD. The ADHD and non-ADHD family samples included non-ADHD family members of ADHD patients and their matched controls. RESULTS: The annual average expenditure (direct cost) per ADHD patient was $1,574, compared to $541 among matched controls. The annual average payment (direct plus indirect cost) per family member was $2,728 for non-ADHD family members of ADHD patients versus $1,440 for family members of matched controls. Both patient and family cost differences were significant at the 95% confidence level. CONCLUSIONS: ADHD imposes a significant financial burden regarding the cost of medical care and work loss for patients and family members.

Economic cost and epidemiological characteristics of patients with fibromyalgia claims.

OBJECTIVE: Fibromyalgia (FM) is characterized by widespread pain that can lead to significant patient disability, complex management decisions for physicians, and economic burden on society. We investigated the total costs of FM in an employer population. METHODS: Administrative claims data of a Fortune 100 manufacturer were used to quantify direct (i.e., medical and pharmaceutical claims) and indirect (i.e., disability claims and imputed absenteeism) costs associated with FM. A total of 4699 patients with at least one FM claim between 1996 and 1998 were contrasted with a 10% random sample of the overall beneficiary population. Employee-only subsets of both samples also were drawn. RESULTS: Medical utilization, receipt of prescription drugs, and annual total costs were proportionately similar yet significantly greater among FM claimants than the overall sample (all p < 0.0001). Total annual costs for FM claimants were $5945 versus $2486 for the typical beneficiary (p < 0.0001). Six percent of these costs were attributable to FM-specific claims. The prevalence of disability was twice as high among FM employees than overall employees (p < 0.0001). For every dollar spent on FM-specific claims, the employer spent another $57 to $143 on additional direct and indirect costs. CONCLUSION: Hidden costs of disability and comorbidities greatly increase the true burden of FM. Regardless of the clinical understanding of FM, when a claim for FM is present, considerable costs are involved. Findings suggest that within the management of FM there may be large cost-offset opportunities for reductions in patient, physician, and employer burdens.

Identification of a claims data "signature" and economic consequences for treatment-resistant depression.

BACKGROUND: Major depressive disorder (MDD) is a debilitating condition with significant economic consequences. Conservative estimates indicate that between 10% and 20% of all individuals with MDD are treatment resistant. The objectives for this study were (1) to use current treatment strategies identified in the literature to evaluate the validity of studying treatment-resistant depression (TRD) using claims data and (2) to estimate cost differences between TRD-likely and TRD-unlikely patients identified by use of treatment patterns. METHOD: The data source consisted of medical, pharmaceutical, and disability claims from a Fortune 100 manufacturer for 1996 through 1998 (N = 125,242 continuously enrolled beneficiaries between the ages of 18 and 64 years). The sample included individuals with medical or disability claims for MDD (NMDD = 4186). A treatment pattern algorithm was applied to classify adult MDD patients into TRD-likely (NTRD = 487) and TRD-unlikely groups. Resource utilization and costs were compared among TRD-likely and TRD-unlikely patients and a random sample of average beneficiaries (i.e., 10% of all beneficiaries) for 1998. RESULTS: Consistent with the epidemiologic literature, the algorithm classified 12% of the MDD sample as TRD-likely. Mean annual costs were $10,954 for TRD-likely patients, $5025 for TRD-unlikely patients, and $3006 for average beneficiaries. TRD-likely patients used almost twice as many medical services as did TRD-unlikely patients and incurred significantly greater indirect costs (p < .0001). CONCLUSION: It is feasible to use an administrative dataset to develop a claim-based treatment algorithm to identify TRD-likely patients. Resource utilization by TRD-likely patients was substantial, not only for direct treatment of depression but also for treatment of comorbid medical conditions. Additionally, TRD imposed on employers substantial indirect costs resulting from high rates of depression-associated disability.