Cost-Effectiveness of Axicabtagene Ciloleucel for Adult Patients With Relapsed or Refractory Follicular Lymphoma in the United States.

OBJECTIVES: The results of a recent single-arm trial (ZUMA-5) of axicabtagene ciloleucel (axi-cel) for relapsed/refractory (r/r) follicular lymphoma (FL) demonstrated high rates of durable response and tolerable toxicity among treated patients. To quantify the value of axi-cel compared with standard of care (SOC) to manage r/r FL patients who have had at least 2 prior lines of systemic therapy (3L+), a cost-effectiveness model was developed from a US third-party payer perspective. METHODS: A 3-state partitioned-survival cost-effectiveness model was developed with a lifetime horizon. Patient-level analyses of the 36-month ZUMA-5 (axi-cel) and SCHOLAR-5 (SOC) studies were used to extrapolate progression-free and overall survivals. After 5 years of survival, an estimated 40% of the modeled population was assumed to experience long-term remission based on literature. Results include the incremental cost-effectiveness ratio (ICER) measured as incremental cost per quality-adjusted life year (QALY) gained. One-way sensitivity analysis, probabilistic sensitivity analysis, and scenario analyses were performed. All outcomes were discounted 3% per year. RESULTS: Axi-cel led to an increase of 4.28 life-years, 3.64 QALYs, and a total cost increase of $321 192 relative to SOC, resulting in an ICER of $88 300 per QALY. Across all parameters varied in the one-way sensitivity analysis, the ICER varied between $133 030 and $67 277. In the probabilistic sensitivity analysis, axi-cel had a 99% probability of being cost-effective across 5000 iterations using a $150 000 willingness-to-pay threshold. CONCLUSIONS: Given the robustness of the model results and sensitivity analyses, axi-cel is expected to be a cost-effective treatment in 3L+ r/r FL.

Cost-effectiveness of intensification with sodium-glucose co-transporter-2 inhibitors in patients with type 2 diabetes on metformin and sitagliptin vs direct intensification with insulin in the United Kingdom.

AIM: To evaluate the long-term cost-effectiveness of an intensification strategy with sodium-glucose co-transporter-2 (SGLT2) inhibitors (pathway 1) compared with NPH insulin (pathway 2) in patients with type 2 diabetes (T2D) in the United Kingdom who were not at goal on metformin and sitagliptin. METHODS: Cost-effectiveness analysis was performed using the well-established, validated IQVIA CORE Diabetes Model from the payer perspective over a patient's lifetime. Randomized clinical trials informed treatment effect measures, while public or published sources informed economic inputs. Scenario analyses of glycated haemoglobin (HbA1c), hypoglycaemia rate, body mass index effects, SGLT2 inhibitor cardiovascular protective effects, and population characteristics were conducted to assess the robustness of results. RESULTS: Pathway 1 increased life-years and quality-adjusted life-years (QALYs) compared with pathway 2 (13.49 vs. 13.37, and 9.40 vs. 9.22, respectively). Additional drug costs in pathway 1 were offset by diabetes-related complication decreases, leading to slightly lower direct medical costs for pathway 1 (£25747 vs £26095). Pathway 1 was therefore cost-neutral (no interpretable incremental cost-effectiveness ratio), while improving clinical outcomes. Scenario analyses consistently showed cost-neutrality or cost-effectiveness of pathway 1. The highest result remained less than £3000/QALY, reflecting older patients (≥65 years) with lower baseline HbA1c (7%). CONCLUSIONS: For UK patients with T2D not at goal on metformin and sitagliptin therapy, treatment intensification with SGLT2 inhibitors prior to NPH insulin is cost-neutral or cost-effective compared with immediate NPH insulin intensification.

Cost-effectiveness of treating relapsed or refractory 3L+ follicular lymphoma with axicabtagene ciloleucel vs mosunetuzumab in the United States.

Introduction: Novel therapies for 3L+ relapsed/refractory (r/r) follicular lymphoma (FL) have been approved recently by the US Food and Drug Administration including anti-CD19 CAR-T therapies such as axicabtagene ciloleucel (axi-cel) and CD20 × CD3 T-cell-engaging bispecific monoclonal antibodies such as mosunetuzumab (mosun). The objective of this study was to assess the cost-effectiveness of axi-cel compared to mosun in 3L+ r/r FL patients from a US third-party payer perspective. Methods: A three-state (progression-free, progressed disease, and death) partitioned-survival model was used to compare two treatments over a lifetime horizon in a hypothetical cohort of US adults (age ≥18) receiving 3L+ treatment for r/r FL. ZUMA-5 and GO29781 trial data were used to inform progression-free survival (PFS) and overall survival (OS). Mosun survival was modeled via hazard ratios (HRs) applied to axi-cel survival curves. The PFS HR value was estimated via a matching-adjusted indirect comparison (MAIC) based on mosun pseudo-individual patient data and adjusted axi-cel data to account for trial populations differences. One-way sensitivity analysis (OWSA) and probabilistic sensitivity analyses (PSA) were conducted. Scenario analyses included: 1) the mosun HRs were applied to the weighted (adjusted) ZUMA-5 24-month data to most exactly reflect the MAIC, 2) mosun HR values were applied to axi-cel 48-month follow-up data, and 3) recent axi-cel health state utility values in diffuse large B-cell lymphoma patients. Results: The analysis estimated increases of 1.82 LY and 1.89 QALY for axi-cel compared to mosun. PFS for axi-cel patients was 6.42 LY vs. 1.60 LY for mosun. Increase of $257,113 in the progression-free state was driven by one-time axi-cel treatment costs. Total incremental costs for axi-cel were $204,377, resulting in an ICER of $108,307/QALY gained. The OWSA led to ICERs ranging from $240,255 to $75,624, with all but two parameters falling below $150,000/QALY. In the PSA, axi-cel had an 64% probability of being cost-effective across 5,000 iterations using a $150,000 willingness-to-pay threshold. Scenarios one and two resulted in ICERs of $105,353 and $102,695, respectively. Discussion: This study finds that axi-cel is cost-effective compared to mosun at the commonly cited $150,000/QALY US willingness-to-pay threshold, with robust results across a range of sensitivity analyses accounting for parameter uncertainty.

Hospitalization-related costs associated with oral agents targeting the prostacyclin pathway for pulmonary arterial hypertension.

AIMS: Pulmonary arterial hypertension (PAH) is a rare, progressive, and ultimately fatal form of the broader condition pulmonary hypertension. ESC/ERS guidelines recommend therapy targeting the prostacyclin pathway for patients not achieving low-risk mortality status. Currently, only oral selexipag (OS) and oral treprostinil (OT) have this mechanism of action and are available in the United States (US). A recent database analysis has shown significantly lower hospitalization risk for patients treated with OS versus OT. Nevertheless, differences in hospitalization and treatment costs among PAH patients taking oral prostacyclin pathway agents (PPAs) in the US healthcare system remain unclear. This study aims to estimate the difference in costs for patients who achieve a stable maintenance dose from a US payer perspective. MATERIALS AND METHODS: We developed a cost calculator including direct medical costs from the US third-party payer perspective to estimate PAH-related hospitalizations and costs associated with oral PPA use over 2 years, in a hypothetical US payer plan with 1 million members. The treatment-eligible population was estimated from real-world epidemiological data. Treatment-specific hospitalizations were estimated from a study using the Optum Clinformatics administrative claims database. Influence of each model parameter was tested in one-way sensitivity analyses (OWSA), while scenario analysis tested the impact of key assumptions. RESULTS: For 78 PAH patients included in the model, the base case scenario estimated total costs of $46,736,768 with 98 PAH-related admissions for OS, and total costs of $60,113,620 and 161 PAH-related admissions over 2 years for OT. Using OS was associated with 22.3% cost reduction and 39.1% hospitalizations averted; the number of patients needed treated with selexipag to avoid one hospital admission was 1.23. OWSA indicated medication cost was the most sensitive parameter, followed by population parameters. LIMITATIONS AND CONCLUSIONS: OS use over 2 years would result in lower total, drug, and hospitalization-related costs compared with OT, thus providing financial savings for payers.

Cost-Effectiveness Analysis of Flash Glucose Monitoring System for People with Type 2 Diabetes Receiving Intensive Insulin Treatment.

AIMS: For people with type 2 diabetes (T2D) on intensive insulin therapy, the use of flash continuous glucose monitoring ("flash monitoring") is associated with improved average glucose control and/or reduced hypoglycemic exposure. This study assessed the cost-effectiveness of flash monitoring versus traditional blood glucose monitoring (BGM) in people with T2D using intensive insulin in the United Kingdom (UK). METHODS: The IQVIA CORE Diabetes Model (IQVIA CDM; v9.0) was used to analyze the impact of flash monitoring versus BGM over a 40-year time horizon from the UK payer perspective. Model inputs included baseline characteristics, intervention effects, resource utilization, costs, and utilities, based on recently published literature and national databases. UK National Health Service reimbursed costs of flash monitoring and BGM were used. An intervention-related health utility was obtained from a time trade-off study. Alternative scenarios were explored to assess the impact of key assumptions on base case results. RESULTS: In base-case analysis, flash monitoring compared with BGM resulted in an incremental cost of £5781 and an additional 0.47 quality-adjusted life years (QALYs). This provides an incremental cost-effectiveness ratio (ICER) of £12,309/QALY. HbA1c and the intervention-related health utility were the key drivers of differentiation. All scenario analyses, including different discount rates, time horizons, effects on HbA1c and on the intervention-related health utility, as well as glycemic emergencies, generated ICERs of less than £20,000 per QALY. CONCLUSIONS: The consistent results across base case and a range of scenario analyses indicate that long-term flash glucose monitoring use is cost-effective compared with BGM in a UK population of T2D on intensive insulin therapy based on updated clinical effects and a cost-effectiveness threshold of £20,000-30,000 per QALY.

The prevalence, correlates, and impact of logically inconsistent preferences in utility assessments for joint health states in prostate cancer.

BACKGROUND: Variations in health state utilities can impact cost-effectiveness analyses. One potential source of error is when joint health state (JS) utilities are rated higher than the embedded single state (SS) utilities. Knowing when and in whom this occurs can improve cost-effectiveness analyses. METHODS: Men (n = 323) were surveyed at the time of prostate biopsy. Time tradeoff SS and JS utilities for prevalent prostate cancer (PCa) health states were collected. JS utilities assessed included those most prevalent for PCa. "Inconsistency" was defined in the following 3 ways: (1) any size rank order violation; (2) total number of violations; and (3) differences greater than 1 standard deviation (SD). Regression analysis assessed independent patient characteristics associated with inconsistent responses. RESULTS: Aggregate JS utilities were consistent. At the individual level, 36% to 41% of responses violated rank order and 12% to 14% were larger than 1 SD. In all, 69% of respondents had at least 1 JS inconsistency, and 24% had >1 SD inconsistencies. Being married and feeling anxious were independently correlated with giving all types of inconsistent ratings, and lower education correlated with making >SD errors. SS utilities, and not JS utilities, were significantly lower for the inconsistent group. "Correcting" JS inconsistencies decreased aggregate utilities 1 to 9 units. CONCLUSIONS: Inconsistent JS utilities for PCa are prevalent in men at biopsy. Being married, more anxious, and having less education are correlated with inconsistencies. It is the SS utilities, rather than the JS utilities, that differ between consistent and inconsistent raters. Better understanding of the source of these inconsistencies is needed.

Cost-effectiveness of intensification with SGLT2 inhibitors for type 2 diabetes.

OBJECTIVES: Using a US payer perspective, this study aimed to compare the lifetime cost-effectiveness of adding sodium-glucose cotransporter 2 (SGLT2) inhibitors vs switching to glucagon-like peptide 1 receptor agonists (GLP-1 RAs) among patients with type 2 diabetes who were not at glycated hemoglobin A1c target after dual therapy with metformin and dipeptidyl peptidase-4 (DPP-4) inhibitors. STUDY DESIGN: The cost-effectiveness analysis was performed with the validated IQVIA Core Diabetes Model. Treatment effects were obtained from randomized clinical trials with economic data based on published literature. METHODS: Risk of treatment-emergent adverse events and complications were simulated using submodels informed by published risk equations adjusted for patient characteristics, physiological parameters, and history of complications. Outcomes included cumulative incidence of micro- and macrovascular complications, life-years (LYs), quality-adjusted life-years (QALYs), and total costs. Scenario analyses were performed to assess robustness of results to variations in clinical and cost inputs and assumptions. RESULTS: Over a lifetime time horizon, adding an SGLT2 inhibitor dominated the strategy of switching to a GLP-1 RA, improving survival by 0.049 LYs and 0.026 QALYs, and was associated with cost savings of $9511. The majority of the scenario analyses confirmed dominance of the DPP-4 inhibitor + SGLT2 inhibitor pathway vs the GLP-1 RA pathway. The probabilistic sensitivity analysis reinforced the base-case finding of cost savings while gaining QALYs. CONCLUSIONS: Intensification with an SGLT2 inhibitor on top of a DPP-4 inhibitor demonstrated slightly better efficacy and cost savings compared with switching to a GLP-1 RA in patients not at glycemic goal with metformin and a DPP-4 inhibitor.

Cost-effectiveness Analysis of Ado-trastuzumab Emtansine (T-DM1) for the Adjuvant Treatment of Patients With Residual Invasive HER2+ Early Breast Cancer in the United States.

OBJECTIVE: Ado-trastuzumab emtansine (T-DM1) was recently approved for patients with human epidermal growth factor receptor 2 positive (HER2+) early breast cancer (eBC) with residual invasive disease after neoadjuvant taxane and trastuzumab-based treatment. Cost-effectiveness analysis was conducted to compare T-DM1 versus trastuzumab in the United States. MATERIALS AND METHODS: A Markov cohort-based model tracked clinical and economic outcomes over a lifetime horizon from a US payer perspective. The model included 6 health states: invasive disease-free, nonmetastatic (locoregional) recurrence, remission, first-line and second-line metastatic BC and death. Model state transitions were based on statistical extrapolation of the head-to-head KATHERINE study and published sources. Dosing and treatment duration reflected prescribing information and trials. Costs (2019 US dollars) associated with pharmaceutical treatment (wholesale acquisition costs), health state specific care, adverse events, and end-of-life care were included. Health state utilities were obtained from KATHERINE and published literature. RESULTS: T-DM1 dominated trastuzumab, yielding lower lifetime costs (-$40,271), and higher life-years (2.980) and quality-adjusted life-years (2.336). Results were driven by patients receiving T-DM1 spending less time in more costly downstream health states, as these patients are less likely to experience a recurrence overall, despite having a higher likelihood of metastatic disease (distant recurrence) in the subset of patients who experience recurrence. Probabilistic sensitivity analysis indicated robust results, with 96.7% of 5000 stochastic simulations producing dominance for T-DM1. The most influential variables were related to treatment costs, off treatment utilities, and health state costs. Additional scenario analyses tested a range of model inputs and assumptions, and produced consistent results. CONCLUSION: Relative to trastuzumab, T-DM1 treatment for patients with HER2+ eBC who have residual invasive disease after neoadjuvant taxane and trastuzumab-based treatment is likely to reduce the overall financial burden of cancer, while simultaneously improving patient outcomes.

US cost-effectiveness and budget impact of point-of-care NAAT for streptococcus.

OBJECTIVES: In the United States, approximately 12 million individuals seek medical care for pharyngitis each year, accounting for about 2% of ambulatory care visits. Although the gold standard for diagnosing group A streptococcus (GAS) is culture, it is time intensive. Rapid antigen detection tests (RADT) with or without culture confirmation are commonly used instead. Although RADT provide results quickly, they generally have lower test sensitivity. Recently, point-of-care nucleic acid amplification tests (POC NAAT) have emerged. This study evaluates the cost-effectiveness and budget impact to the US payer of adopting POC NAAT. STUDY DESIGN: This study was a cost-effectiveness analysis, with costs and outcomes calculated via a decision tree. METHODS: A decision-tree model quantified costs and outcomes associated with a GAS diagnostic strategy using POC NAAT compared with RADT + culture confirmation. Model inputs were derived from the published literature. Model outputs included costs and clinical effects: quality-adjusted life-days lost, GAS and antibiotic complications, number of patients appropriately treated, and antibiotic utilization. Sensitivity and scenario analyses were performed. RESULTS: Base-case analysis projected that a POC NAAT strategy would cost $44 per patient compared with $78 for RADT + culture. Compared with RADT + culture, POC NAAT would increase the number of appropriately treated patients and avert unnecessary use of antibiotics. The budget impact of POC NAAT was -0.4% relative to current budget over 5 years. Findings were robust in sensitivity analyses. CONCLUSIONS: Our results suggest that POC NAAT would be less costly and more effective than RADT + culture; POC NAAT adoption may yield cost savings to US third-party payers. Access to POC NAAT is important to optimize GAS diagnosis and treatment decisions in the United States.

Relationship Between Patient Characteristics and Number of Procedures as well as Length of Stay for Patients Surviving Severe Burn Injuries: Analysis of the American Burn Association National Burn Repository.

This study establishes important, national benchmarks for burn centers to assess length of stay (LOS) and number of procedures across patient profiles. We examined the relationship between patient characteristics such as age and total body surface area (TBSA) burned and number of procedures and LOS in the United States, using the American Burn Association National Burn Repository (NBR) database version 8.0 (2002-2011). Among 21,175 surviving burn patients (TBSA > 10-60%), mean age was 33 years, and mean injury size was 19.9% TBSA. Outcomes included the number of debridement, excision, autograft procedures, and LOS. Independent variables considered were: age (linear, squared, and cubed to account for nonlinearity), TBSA, TBSAs of partial-thickness and mixed/full-thickness burns, sex, hospital-acquired infection, other infection, inhalation injury, and diabetes status. Regression methods included a mixed-effects model for LOS and ordinary least squares for number of procedures. A backward stepwise procedure (P <0.2) was used to select variables. Number of excision and autografting procedures increased with TBSA; however, this relationship did not hold for debridement. After adjusting for sex, age, and comorbidities, predicted LOS for adults (18+) was 12.1, 21.7, 32.2, 43.7, and 56.1 days for 10, 20, 30, 40, and 50% TBSA, respectively. Similarly, predicted LOS for pediatrics (age < 18) was 8.1, 18.8, 33.2, 47.6, and 56.1 days for the same TBSA groups, respectively. While average estimates for adults (1.12 days) and pediatrics (1.01) are close to the one day/TBSA rule-of-thumb, consideration of other important patient and burn features in the NBR can better refine predictions for LOS.

Cost-effectiveness of omalizumab for the treatment of moderate-to-severe uncontrolled allergic asthma in the United States.

Objective: Uncontrolled asthma is associated with considerable clinical burden and costs to payers and patients. US economic models evaluating biologics using data from clinical trials demonstrate high incremental cost-effectiveness ratios (ICERs), but the cost-effectiveness based on real-world treatment patterns is unknown. This analysis used real-world evidence to assess the cost-effectiveness of adding omalizumab to standard of care (SOC).Methods: A Markov model was applied to track patients' health states in 2-week cycles, comparing costs and treatment effects of SOC alone versus SOC + omalizumab over a lifetime (US payer perspective). Outcomes included exacerbation events, life years, quality-adjusted life years (QALYs), total costs, and an ICER. Patient characteristics, exacerbations, patient-reported outcomes, and work productivity were derived from the real-world PROSPERO (Prospective Study to Evaluate Predictors of Clinical Effectiveness in Response to Omalizumab) study. Published literature informed mortality, exacerbation-related disutility, and unit costs. Sensitivity analyses assessed model robustness.Results: Over a lifetime horizon, omalizumab was associated with an increase of 2.0 QALYs at a cost of $US 148,319 in patients with uncontrolled asthma (ICER of $75,319/QALY gained) and a reduction in exacerbations of 6.0 events/patient. Accounting for responder status improved the ICER ($70,505/QALY); incorporating indirect costs further reduced the ICER. One-way and multivariate sensitivity analyses confirmed that the base case outcome was robust to variation in inputs.Conclusions: Based on real-world outcomes, omalizumab may be cost-effective for uncontrolled asthma from the US payer perspective. Including broader evidence on treatment discontinuation, caregiver burden, and oral corticosteroid reduction from real-world studies may better reflect the effects and value of omalizumab for all healthcare stakeholders.

Cost-effectiveness of DPP-4 inhibitor and SGLT2 inhibitor combination therapy for type 2 diabetes.

OBJECTIVES: Maintaining glycemic control limits costly health risks in patients with type 2 diabetes (T2D), but accomplishing this may require individualized strategies. Generic medications (eg, sulfonylureas [SU], insulin) are common in T2D management due to their efficacy and costs; however, relatively new drug classes (eg, dipeptidyl peptidase 4 [DPP-4] inhibitors, sodium-glucose cotransporter 2 [SGLT2] inhibitors) have demonstrated clinical benefits in combination therapy. The objective of this study was to evaluate the long-term cost-effectiveness of a strategy involving branded combination therapy with DPP-4 inhibitors and SGLT2 inhibitors (pathway 1) compared with a generic alternative with SU and insulin (pathway 2) on a background of metformin. STUDY DESIGN: Cost-effectiveness analysis using the validated IQVIA CORE Diabetes Model from the US payer perspective. METHODS: Cost-effectiveness analysis. Lifetime clinical and economic outcomes (discounted 3%/year) were modeled for a T2D cohort failing to achieve glycemic goal on metformin monotherapy. Patient baseline data and treatment effects reflect results of clinical trials. Direct medical cost inputs are from multiple published sources. Scenario analyses on key intervention effects and assumptions tested robustness of results. RESULTS: Pathway 1 had higher direct medical costs compared with pathway 2, yet also increased total quality-adjusted life-years (QALYs) by 0.24. Increased costs were partially offset by a reduction in diabetes-related complications and delayed insulin initiation. The incremental cost-effectiveness ratio (ICER) for pathway 1 is favorable at $64,784/QALY. Scenario analyses showed limited impact; nearly all ICERs were less than $100,000/QALY. CONCLUSIONS: In the United States, sequential addition of SGLT2 inhibitors to DPP-4 inhibitors may be considered cost-effective compared with traditional treatment with generic medications for patients who fail to achieve glycemic goal on metformin.

Cost savings associated with timely treatment of botulism with botulism antitoxin heptavalent product.

BACKGROUND: Botulism is a rare, serious, and sometimes fatal paralytic illness caused by exposure to neurotoxins produced by Clostridium botulinum bacteria. Patients with documented or suspected exposure to botulinum toxin serotypes A-G can be treated with BAT® [Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G)-(Equine)] product, which was approved in 2013 in the United States (US). Patients with botulism have demonstrated greater clinical benefit with early BAT product treatment (≤2 days from symptom onset) versus late treatment (>2 days). OBJECTIVE: Economic outcomes associated with improved clinical outcome benefits of BAT product treatment have not yet been reported. This ad hoc analysis aimed to estimate and compare costs associated with hospitalization, intensive care unit stay, and mechanical ventilation for patients with botulism administered BAT product treatment early or late. METHODS: Clinical outcomes data for early and late BAT product treatment were obtained from a patient registry conducted between October 2014 and July 2017. Total per patient mean daily costs were estimated based on information from published literature. Total population costs per group were calculated by multiplying estimated mean cost per patient by the average annual number of non-infant botulism cases in the US. RESULTS: Mean per patient costs were 2.5 times lower for patients treated with BAT product early versus late. On average in the US, early BAT product treatment could save greater than $3.9 million per year versus late treatment. CONCLUSION: Substantial economic savings can be achieved with early BAT product treatment. The findings support the recommendation for public health authorities to ensure antitoxin treatment is readily available in sufficient quantities to manage botulism cases, including sporadic outbreaks and potential mass exposure biological attacks.

Cost-effectiveness of screening for HIV in the era of highly active antiretroviral therapy.

BACKGROUND: The costs, benefits, and cost-effectiveness of screening for human immunodeficiency virus (HIV) in health care settings during the era of highly active antiretroviral therapy (HAART) have not been determined. METHODS: We developed a Markov model of costs, quality of life, and survival associated with an HIV-screening program as compared with current practice. In both strategies, symptomatic patients were identified through symptom-based case finding. Identified patients started treatment when their CD4 count dropped to 350 cells per cubic millimeter. Disease progression was defined on the basis of CD4 levels and viral load. The likelihood of sexual transmission was based on viral load, knowledge of HIV status, and efficacy of counseling. RESULTS: Given a 1 percent prevalence of unidentified HIV infection, screening increased life expectancy by 5.48 days, or 4.70 quality-adjusted days, at an estimated cost of 194 dollars per screened patient, for a cost-effectiveness ratio of 15,078 dollars per quality-adjusted life-year. Screening cost less than 50,000 dollars per quality-adjusted life-year if the prevalence of unidentified HIV infection exceeded 0.05 percent. Excluding HIV transmission, the cost-effectiveness of screening was 41,736 dollars per quality-adjusted life-year. Screening every five years, as compared with a one-time screening program, cost 57,138 dollars per quality-adjusted life-year, but was more attractive in settings with a high incidence of infection. Our results were sensitive to the efficacy of behavior modification, the benefit of early identification and therapy, and the prevalence and incidence of HIV infection. CONCLUSIONS: The cost-effectiveness of routine HIV screening in health care settings, even in relatively low-prevalence populations, is similar to that of commonly accepted interventions, and such programs should be expanded.

Cost-effectiveness Analysis of a Flash Glucose Monitoring System for Patients with Type 1 Diabetes Receiving Intensive Insulin Treatment in Sweden.

Flash glucose monitoring - an alternative to traditional self-monitoring of blood glucose (SMBG) - prevents hypoglycaemic events without impacting glycated haemoglobin (HbA1c).21 Given the potential benefits, this study assessed the cost-effectiveness of using flash monitoring versus SMBG alone in patients with type 1 diabetes (T1D) receiving intensive insulin treatment in Sweden. Methods: This study used the IQVIA CORE Diabetes Model (IQVIA CDM, v9.0) to simulate the impact of flash monitoring versus SMBG over 50 years from the Swedish societal perspective. Trial data informed cohort data, intervention effects, and resource utilisation; literature and Tåndvards-Läkemedelförmånsverket (TLV) sources informed utilities and costs. Scenario analyses explored the effect of key base case assumptions. Results: In base case analysis, direct medical costs for flash monitor use were SEK1,222,333 versus SEK989,051 for SMBG use. Flash monitoring led to 0.80 additional quality-adjusted life years (QALYs; 13.26 versus 12.46 SMBG) for an incremental cost effectiveness ratio (ICER) of SEK291,130/QALY. ICERs for all scenarios remained under SEK400,000/QALY. Conclusion: Hypoglycaemia and health utility benefits due to flash glucose monitoring may translate into economic value compared to SMBG. With robust results across scenario analyses, flash monitoring may be considered cost-effective in a Swedish population of T1D intensive insulin users.

The Cost-effectiveness of a Flash Glucose Monitoring System for Management of Patients with Type 2 Diabetes Receiving Intensive Insulin Treatment in Sweden.

Flash glucose monitoring, an alternative to traditional self-monitoring of blood glucose (SMBG), prevents hypoglycaemic events without impacting glycated haemoglobin (REPLACE trial). Given the potential benefits, this study assessed the cost-effectiveness of using flash monitoring versus SMBG alone in patients with type 2 diabetes (T2D) receiving intensive insulin treatment in Sweden.Methods: This study used the IQVIA CORE Diabetes Model (IQVIA CDM, v8.5) to simulate the impact of flash monitoring versus SMBG over 40 years from the Swedish societal perspective. Baseline characteristics, intervention effects, and resource utilisation were derived from REPLACE; literature and Tandvårds-Läkemedelförmånsverket (TLV) sources informed utilities and costs. Scenario analyses explored the effect of key base case assumptions. Results: In base case analysis, direct medical costs for flash monitoring use were SEK1,630,586 (€158,523) versus SEK1,459,394 (€141,902) for SMBG use. Flash monitoring led to 0.56 additional quality-adjusted life years (QALYs; 6.21 versus 5.65 SMBG) for an incremental cost-effectiveness ratio (ICER) of SEK306,082/QALY (€29,762/QALY). ICERs for all scenarios remained under SEK400,000/QALY (€38,894/QALY). Conclusions: Hypoglycaemia and health utility benefits due to flash glucose monitoring may translate into economic value compared to SMBG. With robust results across scenario analyses, flash monitoring may be considered cost-effective in a Swedish population of T2D intensive insulin users.

Economic Burden of Toxicities Associated with Treating Metastatic Melanoma in the United States.

BACKGROUND: Little has been reported on the costs of managing the adverse events (AEs) associated with current therapies for patients with regional or distant metastatic melanoma. OBJECTIVES: To identify treatment-related AEs in patients with metastatic melanoma and to estimate the associated costs of treating these AEs in the United States. METHODS: A cost-estimation study for AEs associated with treatment of metastatic melanoma was conducted from 2012 to 2013 by identifying grades 3 and 4 AEs through the use of a comprehensive search of drug labels and English-language, published phase 2/3 studies in PubMed, conference abstracts, and the National Comprehensive Cancer Network guidelines. Resource utilization for the management of each type of AE in the outpatient setting was obtained via interviews with 5 melanoma specialists in the United States. Unit costs for an AE associated with melanoma treatment in the outpatient setting were assigned using Medicare reimbursement rates to obtain these costs. Hospitalization and length-of-stay costs were estimated for each associated AE using the large national claims database Optum Clinformatics Data Mart for the period of July 1, 2004, to November 30, 2012. RESULTS: The most common AEs associated with chemotherapies used for melanoma were neutropenia, vomiting, and anemia. The most common AEs associated with vemurafenib were cutaneous squamous-cell carcinoma or keratoacanthoma, rash, and elevated liver enzymes; the most common AEs associated with dabrafenib were cutaneous squamous-cell carcinoma and pyrexia. Trametinib was most often associated with hypertension and rash. The most common AEs with ipilimumab were immune-related diarrhea or colitis, dyspnea, anemia, vomiting, and, less frequently, hypophysitis. The most common grade 3/4 AE with talimogene laherparepvec was cellulitis. The highest treatment costs for an AE in the outpatient setting were for neutropenia ($2092), headache ($609), and peripheral neuropathy ($539). The highest mean inpatient costs for an AE were for acute myocardial infarction, sepsis, and coma, which ranged from $31,682 to $47,069. Colitis or diarrhea, cutaneous squamous-cell carcinoma, thrombocytopenia, hyponatremia, oliguria or anuria, hypertension, anemia, and elevated liver enzymes were associated with mean costs for hospitalization ranging from $19,122 to $26,861. CONCLUSION: The costs of managing treatment-related AEs in patients with metastatic melanoma are substantial. Effective treatments with improved safety profiles may help to reduce these costs. Until real-world evidence for the costs associated with treatment toxicity is available in the outpatient and inpatient settings, the costs estimated in this study can help inform decision makers about the cost-effectiveness of managing patients with metastatic melanoma.

Functional outcomes in head and neck cancer.

For advanced head and neck cancer (HNC) patients, the effects of disease and the side effects of aggressive treatments have the potential to severely affect function and quality of life. More recent treatment strategies offer patients many options and have increased rates of locoregional control. However, they have not eliminated either acute treatment side effects or the spectrum of negative late sequelae, such as eating and speech dysfunction, residual pain, and troublesome dryness of the mouth. Understanding this broad spectrum of side effects and how patients experience them as well as the functional and quality of life implications is important to treatment evaluation and patient decision making. The heterogeneity of HNC patients (in terms of tumor site), the diversity of surgical techniques and chemoradiotherapy regimens, together with individual patient differences in response to these variables, make it particularly difficult to describe precise outcomes attached to various treatment options. However, in the context of this caveat, there are increasing data documenting the impact of various treatment modalities on physical, functional, and QOL outcomes. This article presents some of these data with a focus on the performance and functional results of radiation therapy, surgery, or concomitant chemoradiation therapy.

Evaluations of quality of life and organ function.

For advanced head and neck cancer (HNC) patients, aggressive radiation and chemoradiation treatments offer new therapy options. The aims of these regimens are increased survival and organ preservation, with the goals of preserving organ function, minimizing late effects, and improving quality of life (QOL). At the same time, the toxicities of these regimens are acknowledged as is the potential for long-term dysfunction. Thus, particularly now, with the increasing use of aggressive chemoradiation therapy (CRT) regimens, documentation of the QOL and functional outcomes of these treatments is critical. The implications for speech and swallowing are widely recognized and the broader effects of these impairments on overall QOL have received some attention. This article presents data on the performance, functional, and QOL results of radiation therapy (RT) and various CRT regimens in HNC.

The association between noncancer pain, cognitive impairment, and functional disability: an analysis of the Canadian study of health and aging.

BACKGROUND: Noncancer pain and cognitive impairment affect many older adults and each is associated with functional disability, but their combined impact has yet to be rigorously studied. METHODS: This is a cross-sectional analysis of the Canadian Study of Health and Aging. Pain was collapsed from a 5-point to a dichotomous scale (no and very mild vs moderate and greater). Cognitive status was dichotomized from the Modified Mini-Mental State Examination (0-100) to no (>77) or mild-moderate (77-50) impairment. Five Instrumental Activities of Daily Living (IADL) and seven Activities of Daily Living (ADL) were self-rated as "accomplished without any help" (0), "with some help" (1), or "completely unable to do oneself" (2) and then summed to create a composite score of 0-10 and 0-14, respectively. Multivariate linear regression analysis was conducted to determine the associations between self-reported functional status with moderate or greater pain, cognitive impairment, and the interaction of the two. RESULTS: A total of 5,143 (90.2%) participants were eligible, 1,813 (35.6%) reported pain at a moderate intensity or greater and 727 (14.3%) were cognitively impaired. The median IADL and ADL summary scores increased among the pain and cognition categories in the following order: no pain and cognitively intact (0.63 SD 1.24, 0.23 SD 0.80), pain and cognitively intact (1.18 SD 1.69, 0.57 SD 1.27), no pain and cognitively impaired (1.64 SD 2.22, 0.75 SD 1.57), and pain and cognitively impaired (2.27 SD 2.47, 1.35 SD 2.09), respectively. Multivariate linear regression found IADL summary scores were associated with pain, coefficient .17 (95% confidence interval [CI] 0.07-0.26), p < .01; cognitive impairment, coefficient .67 (95% CI 0.51-0.83), p < .01; and an interaction effect of pain with cognitive impairment, coefficient .24 (95% CI 0.01-0.49), p = .05. ADL summary scores were associated with pain coefficient .10 (95% CI 0.04-0.17), p < .01 and cognitive impairment, coefficient .29 (95% CI 0.19-0.39), p < .01, but had a nonsignificant interaction term, coefficient .12 (95% CI -0.03 to 0.29), p = .12. CONCLUSIONS: Noncancer pain and cognitive impairment are independently associated with IADL and ADL impairment and IADL impairment is even greater when both conditions are present.