Cost-effectiveness of home non-invasive ventilation in patients with persistent hypercapnia after an acute exacerbation of COPD in the UK.

Home non-invasive mechanical ventilation (HMV) with home oxygen therapy (HOT) in patients with persistent hypercapnia following an acute exacerbation of chronic obstructive pulmonary disease delays hospital readmission. The economic impact of this treatment is unknown. We evaluated the cost-effectiveness of HMV in the UK healthcare system using data from a previously published efficacy trial. Quality-adjusted life-years (QALYs) were computed from EQ-5D-5L. Accounting for all direct patient costs HOT-HMV was £512 (95%CI £36 to £990) more expensive per patient per year than HOT-alone. This small increase in cost was accompanied by increased quality of life leading to an incremental cost-effectiveness ratio of £10 259 per QALY. HOT-HMV was cost-effective in this clinical population. Trial registration number: NCT00990132.

Healthcare resource use and cost burden of urinary incontinence to United States payers.

OBJECTIVE: To assess healthcare resource utilization and costs for female patients diagnosed with stress or mixed urinary incontinence (SUI/MUI) compared to a matched cohort of patients without SUI/MUI. METHODS: We conducted a retrospective matched cohort study of women using the IBM MarketScan research database. Women diagnosed with SUI/MUI between July 1, 2014 and June 30, 2016 were identified using International Classification of Diseases 9 and 10 codes for SUI or MUI with the date of first diagnosis as the index date from which 2-year postindex healthcare resource use and direct cost data were derived from claims, examined, and compared 1:1 with patients without a SUI/MUI diagnosis, matched by age and Charlson's Comorbidity Index. RESULTS: A total of 68 636 women with SUI/MUI were matched 1:1 with controls. In the 2-year postindex date, a significantly higher proportion of SUI/MUI patients had ≥1 inpatient visit and ≥1 outpatient visit compared to the control group (inpatient: 18.89% vs. 12.10%, p < 0.0001; outpatient: 88.44% vs. 73.23%, p < 0.0001). Mean primary care visits were significantly higher in SUI/MUI patients compared to controls (7.33 vs. 5.53; p < 0.0001) as were specialist visits (1.2 vs. 0.08; p < 0.0001). Mean all-cause outpatient costs were higher in SUI/MUI patients compared to controls ($7032.10 vs. $3348.50; p < 0.0001), as were inpatient costs ($3990.70 vs. $2313.70; p < 0.0001). CONCLUSION: Women with SUI/MUI consume significantly higher medical resources and incur higher costs to payers, compared to women without SUI/MUI. While reasons for this are not fully understood, improved and standardized treatment for women with SUI/MUI may positively affect cost and outcomes.

US payer budget impact of a microarray assay with machine learning to evaluate kidney transplant rejection in for-cause biopsies.

AIM: This study evaluates the economic impact to US commercial payers of MMDx-Kidney used in conjunction with histologic evaluation of for-cause kidney transplant biopsies. MATERIALS AND METHODS: An Excel-based model was developed to assess the cost impact of histology plus MMDx-Kidney versus histology alone for the evaluation of potential rejection in kidney transplant patients who receive a for-cause biopsy. Different model time periods were assessed, ranging from 1 to 5 years post-biopsy. A targeted literature review was used to identify parameter estimates, validated by two external clinicians with expertise in managing kidney transplant rejection. A sensitivity analysis was conducted to evaluate the relative impact of key clinical and cost parameters. In particular, the model identified the magnitude of MMDx-Kidney's impact on graft failure from rejection that would be required for MMDx-Kidney to be cost-neutral. RESULTS: By more accurately characterizing rejection, MMDx-Kidney is estimated to increase antirejection treatment costs by $1,126 per test. Nevertheless, a break-even analysis shows that the costs of MMDx-Kidney and anti-rejection medication, as well as the costs associated with an increase in the number of patients with functioning transplants, may be offset by reductions in costs associated with graft failure (i.e. costs of hospitalizations, dialysis, and repeat transplants) over 5 years, assuming MMDx-Kidney reduces annual graft failure from rejection by at least 5%. For the base case, with a 25% relative reduction in annual rate of graft failures from rejection, MMDx-Kidney increases overall costs incurred in the first year of the model but starts generating savings by the second year of the model. CONCLUSIONS: Compared with histologic evaluation of for-cause kidney transplant biopsies alone, the use of MMDx-Kidney in conjunction with histologic evaluation improves the diagnoses of graft dysfunction and may have the potential to generate overall savings from reductions in rejection-related graft failure.

Dasiglucagon demonstrates reduced costs in the treatment of severe hypoglycemia in a budget impact model.

BACKGROUND: Approximately 7.3 million people with type 1 or type 2 diabetes (T1D/T2D) are treated with insulin, placing them at higher risk of severe hypoglycemia (SH). SH requires assistance of another individual and often necessitates the prompt administration of intravenous glucose, injectable glucagon, or both. Untreated, SH can progress to unconsciousness, seizures, coma, or death. Before 2018, all glucagon rescue treatments required reconstitution. The complexity of reconstitution is often a barrier to successful administration during a severe hypoglycemic event. Studies suggest successful administration of glucagon emergency kits range from 6%-56% of the time. Second-generation glucagon treatments and glucagon analogs do not require reconstitution and have caregiver administration success rates ranging from 94%-100%. Dasiglucagon is a glucagon analog administered via autoinjector or prefilled syringe and has been shown to result in rapid hypoglycemia recovery. Moreover, the autoinjector can be administered successfully 94% of the time by trained caregivers. Previous evaluation of costs in budget impact models (BIMs) demonstrated the potential for second-generation glucagon treatments to reduce the cost of SH events (SHEs). The current model expands on those findings with a treatment pathway and accompanying assumptions reflecting important aspects of real-world SHE treatment. OBJECTIVE: To evaluate the economic impact of dasiglucagon compared with available glucagon treatments for SHE management, considering direct cost of treatment and health care resource utilization. METHODS: A 1-year BIM with a hypothetical US commercial health plan of 1 million lives was developed with a target population of individuals with diabetes at risk of SHE. The treatment pathway model included initial and secondary treatment attempts, treatment administration success and failure, plasma glucose (PG) recovery within 15 minutes, emergency medical services, emergency department (ED) visits, and hospitalizations. A 1-way sensitivity analysis was conducted to assess the sensitivity of the model to changes in parameter values. RESULTS: In a 1 million-covered lives population, it was estimated that 12,006 SHEs would occur annually. The higher rate of initial treatment success and PG recovery within 15 minutes associated with dasiglucagon treatment resulted in lower total health care costs. Total SHE treatment costs with dasiglucagon were estimated at $13.4 million, compared with $16.7 million for injectable native glucagon, $20.7 million for nasal glucagon, $35.3 million for reconstituted glucagon, and $43.8 million for untreated individuals. Compared with untreated people, the number needed to treat (NNT) with dasiglucagon was 6 individuals to avoid 1 hospitalization. NNT for this same comparison was 59 for injectable native glucagon and 27 for nasal glucagon. CONCLUSIONS: Treatment of SH with dasiglucagon decreased total direct medical costs by reducing health care resource utilization (emergency calls, emergency transports, ED visits, and hospitalizations) and accompanying costs associated with the treatment of SH. DISCLOSURES: This research was funded by Zealand Pharma. Bromley, Hinahara, and Goss are employed by Boston Healthcare Associates, Inc., which received funding from Zealand Pharma for development of the health economic model and the manuscript. Kendall and Hammer are employed by Zealand Pharma. Weinzimer has received consulting fees from Zealand Pharma.

Payer budget impact of an artificial intelligence in vitro diagnostic to modify diabetic kidney disease progression.

AIM: To evaluate the U.S. payer budget-impact of KidneyIntelX, an artificial intelligence-enabled in vitro diagnostic to predict kidney function decline in Type 2 Diabetic Kidney Disease (T2DKD) patients, stages 1-3b. MATERIALS AND METHODS: We developed an Excel-based model according to International Society of Pharmacoeconomics and Outcomes Research (ISPOR) good practices to assess U.S. payer budget impact associated with the use of the KidneyIntelX test to optimize therapy T2DKD patients compared to standard of care (SOC) (without KidneyIntelX). A hypothetical cohort of 100,000 stages 1-3b T2DKD patients was followed for 5 years. Peer-reviewed publications were used to identify model parameter estimates. KidneyIntelX costs incremental to SOC (without KidneyIntelX) included test cost, additional prescription medication use, specialist referrals and PCP office visits. Patients managed with KidneyIntelX experienced a 20% slowed progression rate compared to SOC (without KidneyIntelX) attributed to slowed DKD progression, delayed or prevented dialysis and transplants, and reduced dialysis crashes. Associated costs were compared to SOC (without KidneyIntelX). Sensitivity analyses were conducted by varying the definition of progression and the DKD progression rate associated with KidneyIntelX testing and related interventions. RESULTS: Projected undiscounted base case 5-year savings for 100,000 patients tested with KidneyIntelX were $1.052 billion, attributed mostly to slowed progression through DKD stages. The breakeven point for the health plan adopting KidneyIntelX is expected to occur prior to year 2 after adoption. Sensitivity analysis based on the assessment of the most conservative definition of progression and a 5% reduction in progression rate attributed to KidneyIntelX, resulted in a projected 5-year savings of $145 million associated with KidneyIntelX. LIMITATIONS AND CONCLUSIONS: Limitations included reliance on literature-based parameter estimates, including effect size of delayed progression supported by the literature. Incorporating KidneyIntelX in contemporary care of early-stage T2DKD patients is projected to result in substantial savings to payers.

Evaluation of the Economic Benefit of Earlier Systemic Lupus Erythematosus (SLE) Diagnosis Using a Multivariate Assay Panel (MAP).

OBJECTIVE: Diagnosis of systemic lupus erythematosus (SLE) made by standard diagnostic laboratory tests (SDLTs) has sensitivity and specificity of 83% and 76%, respectively. A multivariate assay panel (MAP) combining complement C4d activation products on erythrocytes and B cells with SDLTs yields a sensitivity and specificity of 80% and 86%, respectively, presumably enabling earlier SLE diagnosis at lower severity, with associated lower health care costs compared with SDLT diagnoses. We compared the payer budget impact of diagnosing SLE using MAP (incremental cost of $108) versus SDLTs. METHODS: We modeled a health plan of 1 million enrollees. SLE diagnosis among suspected patients was 9.2%. The MAP arm assumed 80%/20% of patients were tested with MAP/SDLTs, versus 100% tested with SDLTs in the SDLT arm. Prediagnosis direct costs were estimated from claims data, and postdiagnosis costs were obtained from the literature. Based on improved MAP performance, the assumed hazard ratio for diagnosis rate compared with SDLTs was 1.74 (71%, 87%, 90%, and 91% of patients who develop SLE are diagnosed in years 1 to 4 compared with 53%, 75%, 84%, and 88% of patients diagnosed with SDLTs). RESULTS: Total 4-year pre- and postdiagnosis direct costs for patients with suspected SLE tested with MAP were $59 183 666 compared with $61 174 818 tested by SDLTs, with lower costs in the MAP arm due primarily to prediagnosis savings related to reduced hospital admissions. CONCLUSION: Incorporating MAP into SLE diagnosis results in estimated 4-year direct cost savings of $1 991 152 ($0.04 per member per month). By facilitating earlier diagnosis of SLE, MAP may enhance patient outcomes.

Treatment Patterns and Medication Use in Patients with Postherpetic Neuralgia.

BACKGROUND: Postherpetic neuralgia (PHN) is a chronic, painful condition characterized by persistent pain following resolution of a herpes zoster (HZ) infection. Epidemiologic data demonstrate that the risks for HZ infections and the development of PHN increase with age. OBJECTIVE: To characterize prescribing patterns, health care utilization, and treatment costs for adults with PHN based on real-world data. METHODS: This study analyzed medical and pharmacy claims from 2010 to 2014 in the MarketScan Commercial and Medicare Supplemental databases. PHN patients were identified based on criteria from a published algorithm. PHN treatment patterns were analyzed by age and reported descriptively for patients aged < 65 or ≥ 65 years. Excess incremental health care costs were calculated for PHN patients by comparing expenditures for a cohort of PHN patients to expenditures of a propensity score-matched control group of patients with HZ alone. RESULTS: Approximately 0.4% of patients aged < 65 years were diagnosed with HZ versus 1.3% of patients aged ≥ 65 years; approximately 15.3% of HZ patients aged < 65 years and 26.4% of patients aged ≥ 65 years were diagnosed with PHN. Overall, opioids remained the most frequently prescribed initial treatment. Approximately 21.6% of PHN patients received an opioid as an initial treatment for PHN, 15.1% received gabapentin; 8.9% received a prescription nonsteroidal anti-inflammatory drug (NSAID); 8.3% received a lidocaine patch; 3.3% received pregabalin; 2.5% received a tricyclic antidepressants (TCAs); 0.8% received other topical lidocaine; and < 1% received capsaicin. Observed first-line use of the lidocaine patch and gabapentin was higher in patients aged ≥ 65 years relative to patients aged < 65 years. When separated by age group, only 24.6% of patients aged < 65 years and 38.5% of patients aged ≥ 65 years were prescribed a recommended first-line treatment for initial PHN therapy (gabapentin, lidocaine patch, pregabalin, and TCAs). Comparisons of treatment costs of PHN patients to matched HZ patients without PHN indicated that PHN patients initiated on opioids had the highest mean additional health care expenditure compared with PHN patients initiated on other medications. On average, PHN patients initiated on opioids had $7,601 additional health care expenditure compared with HZ patients with no PHN; additional expenditures were $6,428 for pregabalin, $4,213 for lidocaine patches, $3,478 for gabapentin, $3,304 for NSAIDs, and $2,797 for TCAs, respectively. CONCLUSIONS: Management of PHN is associated with substantial utilization of opioid-based therapies across all ages. Medications supported by evidence either as first-line therapies or as part of a multimodal regimen for the management of PHN are underused relative to opioid-based PHN therapies. Improving adherence to evidence-based PHN treatment regimens offers the potential to reduce opioid prescribing first line and reduce overall treatment costs. Given the emphasis to reduce opioid prescribing to minimize the risk of dependence, abuse, and diversion, multimodal analgesic treatments that can avoid or reduce opioid use should be considered. DISCLOSURES: Research funding was provided by SCILEX Pharmaceuticals. The sponsor reviewed and approved the research plan and provided support for manuscript preparation through Patel's role as a coauthor of this manuscript. The sponsor's product (lidocaine patch) was not used in this study. Patel is a paid employee of SCILEX Pharmaceuticals. Goss is an employee and minority owner of Boston Healthcare Associates, which received a research grant from SCILEX Pharmaceuticals to conduct this study. Gudin reports advisory board fees from AcelRx Pharmaceuticals and BioDelivery Sciences International and consulting fees from Averitas, Daiichi, Hisumitsu, Nektar, Purdue, Quest Diagnostics, SCILEX Pharmaceuticals, and US WorldMeds, unrelated to this study. Fudin reports advisory board fees from AcelRx Pharmaceuticals, Human Half-Cell, Quest Diagnostics, GlaxoSmithKline, SCILEX Pharmaceuticals, BioDelivery Sciences, Daiichi Sankyo, and Salix Pharmaceuticals; speaker fees from Daiichi Sankyo, Salix Pharmaceuticals, Abbott Laboratories, Acutis Diagnostics, and AstraZeneca; and consulting fees from Firstox Laboratories, unrelated to this study. The other authors have nothing to disclose. Parts of this research were presented at the AMCP Managed Care & Specialty Pharmacy Annual Meeting; April 22, 2016; San Francisco, CA, and at the 35th Annual Scientific Meeting of the American Pain Society; May 11-14, 2016; Austin, TX.

Stool DNA Analysis is Cost-Effective for Colorectal Cancer Surveillance in Patients With Ulcerative Colitis.

BACKGROUND & AIMS: Patients with chronic ulcerative colitis are at increased risk for colorectal neoplasia (CRN). Surveillance by white-light endoscopy (WLE) or chromoendoscopy may reduce risk of CRN, but these strategies are underused. Analysis of DNA from stool samples (sDNA) can detect CRN with high levels of sensitivity, but it is not clear if this approach is cost-effective. We simulated these strategies for CRN detection to determine which approach is most cost-effective. METHODS: We adapted a previously published Markov model to simulate the clinical course of chronic ulcerative colitis, the incidence of cancer or dysplasia, and costs and benefits of care with 4 surveillance strategies: (1) analysis of sDNA and diagnostic chromoendoscopy for patients with positive results, (2) analysis of sDNA with diagnostic WLE for patients with positive results, (3) chromoendoscopy with targeted collection of biopsies, or (4) WLE with random collection of biopsies. Costs were based on 2014 Medicare reimbursement. The primary outcome was the incremental cost-effectiveness ratio (incremental cost/incremental difference in quality-adjusted life-years) compared with no surveillance and a willingness-to-pay threshold of $50,000. RESULTS: All strategies fell below the willingness-to-pay threshold at 2-year intervals. Incremental cost-effectiveness ratios were $16,362 per quality-adjusted life-year for sDNA analysis with diagnostic chromoendoscopy; $18,643 per quality-adjusted life-year for sDNA analysis with diagnostic WLE; $23,830 per quality-adjusted life-year for chromoendoscopy alone; and $27,907 per quality-adjusted life-year for WLE alone. In sensitivity analyses, sDNA analysis with diagnostic chromoendoscopy was more cost-effective than chromoendoscopy alone, up to a cost of $1135 per sDNA test. sDNA analysis remained cost-effective at all rates of compliance; when combined with diagnostic chromoendoscopy, this approach was preferred over chromoendoscopy alone, when the specificity of the sDNA test for CRN was >65%. CONCLUSIONS: Based on a Markov model, surveillance for CRN is cost-effective for patients with chronic ulcerative colitis. Analysis of sDNA with chromoendoscopies for patients with positive results was more cost-effective than chromoendoscopy or WLE alone.

Assessment of motivating factors associated with the initiation and completion of treatment for chronic hepatitis C virus (HCV) infection.

BACKGROUND: Infection with hepatitis C virus (HCV) is associated with high morbidity and increased mortality but many patients avoid initiation of treatment or report challenges with treatment completion. The study objective was to identify motivators and barriers for treatment initiation and completion in a community sample of HCV-infected patients in the United States. METHODS: Survey methods were employed to identify factors reported by patients as important in their decision to start or complete HCV treatment. Study participants included 120 HCV-infected individuals: 30 had previously completed treatment with pegylated interferon/ribavirin (PR), 30 had discontinued PR, 30 were treated with PR at the time of the survey, and 30 were treatment‒naïve. Telephone interviews occurred between May and August of 2011 and employed a standardized guide. Participants assigned factors a rating from 1 (not at all important) to 5 (extremely important). Trained researchers coded and analyzed interview transcripts. RESULTS: Of 33 factors, expected health problems from not treating HCV infection was reported as most encouraging for treatment initiation and completion, while treatment side effects was most discouraging. Sixty-nine percent of participants reported that the ability to obtain information during treatment on the likelihood of treatment success (i.e., results of viral load testing) would motivate them to initiate therapy. Median preferred timing for learning about test results was 5 weeks (range: 1-23 weeks). CONCLUSION: Understanding challenges and expectations from patients is important in identifying opportunities for education to optimize patient adherence to their HCV treatment regimen.

The economic burden of treatment-resistant depression.

BACKGROUND: Major depressive disorder (MDD) is a leading cause of disability, morbidity, and mortality worldwide. The lifetime prevalence in the United States is estimated at 17%. Treatment-resistant depression (TRD) is generally defined as failure to achieve remissions despite adequate treatment. About 30% of patients do not achieve remission after 4 different antidepressant treatment trials. A few studies have examined the economic burden of TRD, but none has investigated the cost associated with more chronic and extensive forms of TRD characterized by nonresponse to ≥4 treatment trials. OBJECTIVE: The objective of this study was to compare the health care utilization (HCU) and direct medical expenditures of TRD patients with those of chronic MDD patients. METHODS: Patients with chronic MDD (defined as ≥2 years of continuous treatment) and patients with TRD (defined as undergoing at least 4 different qualifying antidepressant therapy trials) were identified in the PharMetrics Patient-centric Database. The association between TRD and medical expenditures was measured by using multivariate regression analysis. RESULTS: The classification of TRD had a clinically meaningful and statistically significant association with increased medical expenditures. Holding all else equal, the classification of TRD was associated with a 29.3% higher costs (P < 0.001) in medical expenditures compared with patients not meeting the study definition of TRD. CONCLUSIONS: These results demonstrate that TRD is associated with significantly higher per-patient medical costs due to higher HCU. The findings suggest that the development of treatment alternatives for TRD is warranted. Limitations related to the use of secondary administrative data are noted.

Cost-effectiveness model of use of genetic testing as an aid in assessing the likely benefit of aspirin therapy for primary prevention of cardiovascular disease.

BACKGROUND: Aspirin use for the primary prevention of cardiovascular disease (CVD) is controversial because of the need to balance the risk of major bleeding events caused by aspirin with the benefit of CVD events prevented by aspirin. The United States Preventive Services Task Force (USPSTF) proposed guidelines that use CVD risk thresholds, based on the Framingham Risk Score, to identify patients likely to benefit from aspirin use. Genetic information could be used to modify this CVD risk assessment; for example, 2 variants of the LPA gene, which encodes apolipoprotein(a), are associated with increased risk of CVD. OBJECTIVES: To estimate the incremental cost-effectiveness of using genetic test results for 2 LPA variants to derive modified Framingham Risk Score estimates and to use these estimates to identify patients likely to benefit from aspirin use according to USPSTF guidelines for aspirin use in the primary prevention of CVD. METHODS: A cost-effectiveness model of 1 million patients representative of the US population was developed based on the association of 2 LPA variants (rs3798220 and rs10455872) with CVD. The cost of testing was estimated for patients whose 10-year CVD risk would exceed the USPSTF treatment threshold if they were to test positive for the LPA variants. Patient utility estimates for myocardial infarction and stroke, and cost estimates (using a 3.5% annual discount rate) for myocardial infarction, stroke, and gastrointestinal bleeding events were based on published estimates. RESULTS: Recommending aspirin to patients whose CVD risk surpassed the risk threshold when LPA information was included in their risk assessment would prevent an estimated 65 CVD events over 10 years. At a genetic test cost of $150, the incremental cost-utility of testing for LPA variants is estimated at $24,942 per quality-adjusted life-year. CONCLUSIONS: LPA genotyping in the context of the aspirin use guidelines for primary prevention of CVD could be cost-effective.

A structured literature review of the direct costs of adult systemic lupus erythematosus in the US.

OBJECTIVE: A structured review of the literature was undertaken to examine the direct costs of adult systemic lupus erythematosus (SLE) in a US population. METHODS: English-language studies published from January 2000 to April 2010 were systematically reviewed from both Medline's PubMed and the Cochrane databases. Studies were included if they reported direct medical costs of SLE among adults in the US. RESULTS: Seven studies published since January 2000 that reported direct medical costs associated with SLE in the US were identified. Studies examined main cost categories of inpatient, outpatient, and pharmacy services; each contributed substantially to total costs. Wide SDs were reported, consistent with variability in disease manifestation. Mean annual direct costs of SLE patients ranged from $13,735-$20,926; the costs of those with and without nephritis ranged from $29,034-$62,651 and $12,273-$16,575, respectively. Across studies of a general SLE population, pharmaceutical costs composed 19-30% of total expenditures, with inpatient costs accounting for 16-50% and outpatient costs accounting for 24-56% of overall costs. Methodologies varied across studies, with patient self-reported resource utilization generating the lowest estimates versus claims-based analyses; Medicaid claims analyses generated lower incremental cost estimates for SLE patients versus control patients compared to estimates based on commercial claims analysis. CONCLUSION: SLE is associated with substantial annual direct cost burden in the US; however, little research has been done examining costs associated with specific treatments or cost variation by disease severity and disease manifestations. Future research elucidating the causes in variation of costs will help in the appraisal of emerging therapies and in developing clinical management strategies.

Frequency and outcomes of blood products transfusion across procedures and clinical conditions warranting inpatient care: an analysis of the 2004 healthcare cost and utilization project nationwide inpatient sample database.

The objective of this retrospective cohort study was to assess frequency and outcomes associated with blood products transfusion. Data from the 2004 Nationwide Inpatient Sample database were used. Length of stay (LOS), postoperative infections, noninfectious transfusion-related complications, in-hospital mortality, and total charges were evaluated for transfused and nontransfused cohorts. Of the estimated 38.66 million discharges in the United States in 2004, 5.8% (2.33 million) were associated with blood products transfusion. Average LOS was 2.5 days longer, and charges were $17 194 higher for the transfused cohort (P < .0001). Odds of death were 1.7 times higher (P < .0001) and odds of infection 1.9 times higher (P < .0001) for the transfused cohort. Increased provider awareness and recognition of the frequency and potential negative outcomes of blood products transfusion may encourage the adoption of novel approaches to minimize intraoperative and early postoperative bleeding, reduce transfusion requirements, and most important, improve patient-level postoperative outcomes and health-related quality of life.

Valuation of transfusion-free living in MDS: results of health utility interviews with patients.

BACKGROUND: This study measured how myelodysplastic syndrome (MDS) patients value transfusion independence (TI), reduced transfusions (RT) and transfusion-dependence (TD) using health utility assessment methodology. METHODS: 47 MDS patients were interviewed, US (n = 8), France (n = 9), Germany (n = 9) and the UK (n = 21), to elicit the utility value of TI, RT and TD. Health states were developed based on literature; patient forum discussions; and were validated by a hematologist. Face-to-face interviews used the feeling thermometer Visual Analogue Scale (VAS) and the Time Trade-Off (TTO) method to value the health states on a 0 (dead) to 1 (perfect health) scale. Socio-demographic, clinical, and quality-of-life (EQ-5D) characteristics were surveyed to describe the patient sample. RESULTS AND DISCUSSION: The mean age was 67 years (range: 29-83); 45% male, 70% retired; 40% had secondary/high school education, or higher (32%), and 79% lived with family, a partner or spouse, or friends. The mean time from MDS diagnosis was 5 years (range:1-23). Most patients (87%) received previous transfusions and 49% had received a transfusion in the last 3 months. Mean EQ-5D index score was 0.78; patients reported at least some problem with mobility (45%), usual activities (40%), pain/discomfort (47%), and anxiety/depression (34%). Few patients had difficulty understanding the VAS (n = 3) and TTO (n = 4) exercises. Utility scores for TI were higher than for RT (0.84 vs. 0.77; p < 0.001) or TD (0.84 vs. 0.60; p < 0.001). Three patients rated TD worse than dead. Corresponding VAS scale scores were 78 vs. 56; (p < 0.001), and 78 vs. 31 (p < 0.001), respectively. CONCLUSION: Patients value TI, suggesting an important role for new treatments aiming to achieve greater TI in MDS. These results can be used in preference-based health economic evaluation of new MDS treatments, such as in future cost-utility studies.

An assessment of medical resource utilization and hospitalization cost associated with a diagnosis of anemia in women with obstetrical bleeding in the United States.

OBJECTIVE: Anemia during pregnancy has been associated with adverse maternal and fetal outcomes. Although women with obstetrical bleeding are at increased risk for developing anemia, little is known about the prevalence and burden associated with anemia in hospitalized women with this condition. This study was conducted to estimate the prevalence, demographic characteristics, medical resource utilization, and hospitalization cost associated with a diagnosis of anemia in hospitalized women with obstetrical bleeding in the United States. METHODS: The Healthcare Cost and Utilization Project Nationwide Inpatient Sample (2003) was queried using ICD-9-CM codes to identify all pregnancy-related discharges as well as discharges with diagnosis codes for conditions associated with obstetrical bleeding. Descriptive statistics were used to evaluate demographic characteristics, medical resource utilization components and hospitalization cost for two groups: patients with a diagnosis of anemia and patients without a diagnosis of anemia. RESULTS: Of the estimated 4,525,714 pregnancy-related discharges in the United States in 2003, more than 250,000 recorded diagnosis codes associated with obstetrical bleeding. Nearly 1 in 5 of these women had an anemia diagnosis. A diagnosis of anemia in hospitalized women with obstetrical bleeding was associated with a 9-fold increase in blood transfusion (p < 0.0001), 33% longer average length of stay (p < 0.0001), and 50% higher average total cost per hospitalization (p < 0.0001). CONCLUSIONS: Anemia and blood transfusion are frequently observed in hospitalized women with obstetrical bleeding. To improve outcomes in these patients and alleviate the adverse impact of anemia on postpartum health status, greater provider awareness of the prevalence and burden of illness associated with a diagnosis of anemia in hospitalized women with obstetrical bleeding is warranted.

Assessment of the prevalence and impact of anemia on women hospitalized for gynecologic conditions associated with heavy uterine bleeding.

OBJECTIVE: Women with heavy uterine bleeding often are untreated or inadequately treated for anemia. This study was conducted to estimate the prevalence and impact of anemia in women hospitalized for gynecologic conditions associated with heavy uterine bleeding. STUDY DESIGN: The largest all-payer inpatient care database, the Healthcare Cost and Utilization Project's 2003 Nationwide Inpatient Sample, was queried using International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM) codes to identify and group women with gynecologic diagnoses associated with heavy uterine bleeding into 2 categories: those with or without anemia. Groups were evaluated for demographic characteristics, medical resource utilization and hospitalization costs using descriptive statistics. RESULTS: More than 25% of the estimated 300,589 women in the study had a diagnosis of anemia. Compared to patients without a diagnosis of anemia, those with an anemia diagnosis were more likely to have a blood transfusion (24% vs. 0.7%, p<0.0001), an emergency department admission (26.8% vs. 3.2%, p<0.0001) and higher hospitalization costs ($5,631 vs. $5,101, p <0.0001). CONCLUSIONS: Anemia and blood transfusions are common in women hospitalized for gynecologic conditions associated with heavy uterine bleeding. Greater patient and provider awareness of the prevalence and burden associated with anemia may increase opportunities to reduce blood transfusions and improve general health status and quality of life in this patient population.

Economic burden of bilateral neovascular age-related macular degeneration: multi-country observational study.

BACKGROUND: There is limited previous research examining the healthcare costs of neovascular age-related macular degeneration (NV-AMD), which constrains our understanding of the economic impact of this condition. With aging populations, this leading cause of rapid vision loss in Western countries is expected to become a pressing health predicament, requiring decision makers to evaluate alternative treatment strategies for AMD. OBJECTIVE: To document the economic burden of bilateral NV-AMD, the late stage of AMD, in elderly patients, from a societal perspective. STUDY DESIGN, SETTING AND PARTICIPANTS: A cross-sectional, observational study surveyed 401 patients with bilateral NV-AMD and 471 non-AMD subjects in Canada, France, Germany, Spain and the UK. Physicians' records and subjects' standardized telephone interviews were used to record medical resource utilization, assistance with daily living and social benefits. Annual bilateral NV-AMD-related socioeconomic costs were calculated in euro, year 2005 values. MAIN OUTCOME MEASURES: Societal costs including direct vision-related medical costs (e.g. treatment of AMD and vision-related equipment), direct non-vision-related medical costs (e.g. medications) and direct non-medical-related costs (e.g. home healthcare and social services) were the main outcome measures. RESULTS: The demographic profile of NV-AMD patients was similar across countries; however, co-morbid condition profiles varied. NV-AMD patients reported substantial health-related problems and associated health resource utilization (HRU). In the previous 12 months, 12-22% of patients fell, and half of these patients required medical treatments. More than 20% (range 21-59%) of patients were prescribed vision-enhancing equipment. More than half of the patients (54-81%) were living with a spouse or family member and 19-41% reported receiving assistance for activities of daily living. The average annual societal cost per bilateral NV-AMD patient treated was estimated to be euro 7879 in Canada, euro 7349 in France, euro 12 445 in Germany, euro 5732 in Spain and euro 5300 in the UK, and direct vision-related medical costs accounted for 23-63% of the total cost. Half of the patients were diagnosed with bilateral NV-AMD for <1 year, with an average length of 5 months; there were no statistically significant differences in total annual costs per patient between these patients and those who were diagnosed with bilateral disease for > or =1 year. Estimated annual societal costs of bilateral NV-AMD patients in these countries ranged from euro 268 to euro 1311 million. Estimated annual societal costs of all NV-AMD patients in these countries ranged from euro 671 to euro 3278 million. CONCLUSIONS: Bilateral NV-AMD imposes significant functional impairment on patients, leading to increased HRU and a high societal cost burden. Differences in national healthcare systems and NV-AMD treatment patterns were reflected in the wide variation of NV-AMD costs across the five surveyed countries. Even though the prevalence rates and per-patient costs varied by country, the societal costs of NV-AMD patients were substantial in each country. Earlier intervention with effective therapies is expected to reduce disease burden and disability associated with NV-AMD and, thus, decrease the overall societal cost.

Burden and health care resource utilization in neovascular age-related macular degeneration: findings of a multicountry study.

OBJECTIVE: To describe the burden of bilateral neovascular age-related macular degeneration (NV-AMD) on patient-reported functioning and health resource utilization. METHODS: A cross-sectional study of 401 patients with bilateral NV-AMD and 471 elderly control subjects without AMD was conducted in 5 countries. Subjects completed a telephone survey, including the National Eye Institute 25-Item Visual Function Questionnaire, the EuroQol instrument, the Hospital Anxiety and Depression Scale, and history of falls, fractures, and health care resource utilization. RESULTS: The mean age for patients with NV-AMD was 78.1 years, and 65% were women. The patients reported 45% worse vision-related functioning, 13% worse overall well-being, and 30% more anxiety and 42% more depression symptoms than controls after adjusting for covariates (all, P < .001). The effect of NV-AMD was also observed as a doubled fall rate (16% vs 8% [P < .001]) and a quadrupled need for assistance with daily activities (29% vs 7% [P < .001]) in the patients compared with controls. CONCLUSIONS: The evidence of extensive decline in quality of life and increased need of daily living assistance for patients with NV-AMD compared with a control population substantiates the need for new treatments that prevent vision loss and progression to blindness.

Assessment of the impact of weekly versus monthly erythropoiesis stimulating protein therapy on patients with CKD and their families.

This cross-sectional study measured patient and family member reported treatment and economic burden associated with monthly versus weekly erythropoiesis stimulating protein therapies for anemia in patients with CKD who were not yet on dialysis. The results indicated that compared to a weekly regimen, a monthly regimen saved patients 7 hours and family members 19 hours per month for routine anemia management. The monthly regimen substantially reduced the reported economic and treatment burdens of patient and their family members.

Cost effectiveness of lenalidomide in the treatment of transfusion-dependent myelodysplastic syndromes in the United States.

Lenalidomide has been approved for the treatment of transfusion-dependent low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a chromosome 5q deletion with or without additional cytogenetic abnormalities. We evaluated the cost effectiveness of lenalidomide versus best supportive care (BSC) in these patients. We developed a decision analytic model to compare costs and outcomes of lenalidomide with BSC without recombinant erythropoietin (EPO) versus BSC with EPO over 1 year. Outcome measures were transfusion independence and quality-adjusted life years (QALYs) gained. The model incorporated costs of medications, transfusions, chelation, laboratory tests, office visits, and other resources associated with each therapy. Lenalidomide therapy was associated with an estimated incremental 0.53 transfusion-free and 0.25 QALY gain compared to BSC at 1 year. The costs of lenalidomide therapy were substantially offset by reduced blood transfusion and EPO costs. One-year total treatment costs were estimated at $63,385 for lenalidomide and $54,940 for BSC. The incremental cost-effectiveness ratio for lenalidomide vs BSC was estimated at $16,066 per transfusion-free year and $35,050 per QALY gained, values within the acceptable cost-effectiveness ranges for a new therapy. Results suggest that oral lenalidomide is cost effective in the United States in the treatment of transfusion-dependent, low- or intermediate-1-risk MDS associated with a deletion 5q cytogenetic abnormality. Confirmation of these findings awaits results of an ongoing randomized phase III trial (MDS-004 study).