Estimating the Potential Economic Impact of Tissue Valve Replacement for Heart Valve Disease in China: Patient-Level and Population-Level Cost-Benefit Simulation Analyses.

OBJECTIVES: This study seeks to estimate the potential societal economic impact of treating patients with heart valve disease (HVD) in China with surgical tissue valve replacement versus mechanical valves. METHODS: This societal economic cost-benefit evaluation is based on an individual simulation model for subgroups of patients with HVD that is also aggregated to a macrosocietal model. The individual simulation model was developed to estimate the likely economic impact of surgical aortic valve replacement (SAVR) with tissue versus mechanical valves for different subgroups among all eligible patients with HVD over their remaining lifetimes. Clinical inputs were informed by health claims database analysis, expert clinical opinion, and published literature. Epidemiological inputs and demographic inputs were sourced from the published literature and the China Statistical Yearbook 2020. Health gains were valued at 3 times the average national income. RESULTS: Projected total lifetime economic gains were greater for patients receiving tissue valves. Costs were reported in 2021 US dollars. The average lifetime net economic gain for tissue valve patients was $51 736 (20.0% more than for mechanical valve patients). Increasing the use of tissue valves to 50% among all eligible patients with HVD would provide aggregate long-term economic gains of $167 billion during their remaining lifetimes. The economic gains from greater tissue valve use were due to avoiding anticoagulation monitoring costs, improved quality of life, and greater post-SAVR labor force participation. CONCLUSION: Increased use of tissue valves versus mechanical values in SAVR procedures in China would be likely to generate a substantial societal economic gain.

Patient Characteristics, Treatment Patterns, Healthcare Resource Utilization, and Costs of Targeted Therapy-Eligible Atopic Dermatitis Patients in Taiwan-A Real-World Study.

INTRODUCTION: The objective of this study was to conduct a retrospective analysis to understand the patient profile, treatment patterns, healthcare resource utilization, and cost of atopic dermatitis (AD) of patients eligible for targeted therapy in Taiwan. METHODS: A retrospective, claims-based analysis was undertaken using Taiwan's National Health Insurance Research Database from 01 January 2014 to 31 December 2017. Patients aged ≥ 2 years and with at least one diagnosis code for AD during 2015 were identified. Patients with comorbid autoimmune diseases were excluded. Enrolled AD patients were categorized using claims-based treatment algorithms by disease severity and their eligibility for targeted therapy treatment. A cohort of targeted therapy-eligible patients was formed, and a matched cohort using patients not eligible for targeted therapy was derived using propensity score matching based on age, gender, and the Charlson Comorbidity Index (CCI). Treatment patterns, resource utilization, and costs were measured during a 1-year follow-up period. RESULTS: A total of 377,423 patients with AD were identified for this study. Most patients had mild AD (84.5%; n = 318,830) with 11.9% (n = 45,035) having moderate AD, and 3.6% (n = 13,558) having severe AD. Within the 58,593 moderate-to-severe AD patients, 1.5% (n = 897) were included in the targeted therapy-eligible cohort. The matched cohort consisted of 3558 patients. During the 1-year follow-up period, targeted therapy-eligible patients utilized antihistamines (85.5%), topical treatments (80.8%), and systemic anti-inflammatories (91.6%) including systemic corticosteroids (51.4%) and azathioprine (59.1%). During the first year of follow-up, targeted therapy-eligible patients (70.5%; 7.01 [SD = 8.84] visits) had higher resource utilization rates and frequency of AD-related outpatient visits compared with the matched cohort (40.80%; 1.85 [SD = 4.71] visits). Average all-cause direct costs during 1-year follow-up were $2850 (SD = 3629) and $1841 (SD = 6434) for the eligible targeted therapy and matched cohorts, respectively. AD-related costs were 17.7% ($506) of total costs for the targeted therapy eligible cohort and 2.2% ($41) for the matched cohort. CONCLUSIONS: AD patients eligible for targeted therapy in Taiwan experienced high resource and economic burden compared with their non-targeted-therapy-eligible counterparts.

Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study.

BACKGROUND: Acute hepatic porphyria (AHP) is a family of four rare genetic diseases, each involving deficiency in a hepatic heme biosynthetic enzyme. Resultant overproduction of the neurotoxic intermediates δ-aminolevulinic acid (ALA) and porphobilinogen (PBG) leads to disabling acute neurovisceral attacks and progressive neuropathy. We evaluated the AHP disease burden in patients aged ≥ 12 years in a post hoc analysis of the Phase 3, randomized, double-blind, placebo-controlled ENVISION trial of givosiran (NCT03338816), an RNA interference (RNAi) therapeutic that targets the enzyme ALAS1 to decrease ALA and PBG production. We analyzed baseline AHP severity via chronic symptoms between attacks, comorbidities, concomitant medications, hemin-associated complications, and quality of life (QOL) and evaluated givosiran (2.5 mg/kg monthly) in patients with and without prior hemin prophylaxis on number and severity of attacks and pain scores during and between attacks. RESULTS: Participants (placebo, n = 46; givosiran, n = 48) included patients with low and high annualized attack rates (AARs; range 0-46). At baseline, patients reported chronic symptoms (52%), including nausea, fatigue, and pain; comorbidities, including neuropathy (38%) and psychiatric disorders (47%); concomitant medications, including chronic opioids (29%); hemin-associated complications (eg, iron overload); and poor QOL (low SF-12 and EuroQol visual analog scale scores). A linear relationship between time since diagnosis and AAR with placebo suggested worsening of disease over time without effective treatment. Givosiran reduced the number and severity of attacks, days with worst pain scores above baseline, and opioid use versus placebo. CONCLUSIONS: Patients with AHP, regardless of annualized attack rates, have considerable disease burden that may partly be alleviated with givosiran.

A systematic literature review of economic evaluations of pneumococcal conjugate vaccines in east and southeast Asia (2006-2019).

INTRODUCTION: Pneumococcal infections can lead to serious invasive diseases such as meningitis, septicemia and pneumonia, as well as milder but more common illnesses such as sinusitis and otitis media. The World Health Organization (WHO) recommends the inclusion of pneumococcal conjugate vaccines (PCVs) in infant National Immunization Program (NIP) programs worldwide. Decision-makers in Asian countries planning to introduce PCVs in their respective NIP will need a comprehensive evidence of effectiveness of PCVs at the population level and economic evidence including cost-effectiveness. AREAS COVERED: A systematic literature review (from 1/1/2016 to 10/11/2019) of PCVs in East and Southeast Asia to understand (1) the contributing factors to cost-effectiveness results of PCVs and (2) whether gaps in evidence exist suggesting why the region may have yet to implement full NIPs. EXPERT OPINION: In East and Southeast Asia, vaccination with PCVs was found to significantly reduce the mortality and morbidity of pneumococcal diseases and was cost-effective compared to no vaccination. Study assumptions, specifically vaccine local acquisition, the inclusion or exclusion of indirect effects (serotype replacement and herd effect), cross-protection, and protection against nontypeable haemophilus influenzae and serotype 3, were the main drivers of cost-effectiveness.

The Cost-Effectiveness of 13-Valent Pneumococcal Conjugate Vaccine in Seven Chinese Cities.

Objective: This study estimates the cost-effectiveness of vaccination with the 13-valent pneumococcal conjugate vaccine (PCV13) among infants in Beijing, Shanghai, Shenzhen, Chengdu, Karamay, Qingdao, and Suzhou. Methods: A previously published cost-effectiveness model comparing vaccination with PCV13 to no vaccination was localized to the included Chinese cities. A systematic literature review was undertaken to identify age-specific incidence rates for pneumococcal bacteremia, pneumococcal meningitis, pneumonia, and otitis media (AOM). Age-specific direct medical costs of treating the included pneumococcal diseases were taken from the Chinese Health Insurance Association database. The base case analysis evaluated vaccine efficacy using direct effect and indirect effects (DE+ IDE). A subsequent scenario analysis evaluated the model outcomes if only DE was considered. A vaccination rate of 70% was used. The model reported outcomes over a one-year period after it was assumed the vaccine effects had reached a steady state (5-7 years after vaccine introduction) to include the direct and indirect effects of vaccination. Health outcomes were discounted at 5% during the steady-state period. Results: Vaccination with PCV13 was cost-effective in the base case analysis for all included cities with the incremental cost-effectiveness ratio (ICER) ranging from 1145 CNY(Shenzhen) to 15,422 CNY (Qingdao) per quality-adjusted life-year (QALY) gained. PCV13 was the dominant strategy in Shanghai with lower incremental costs and higher incremental QALYs. PCV13 remained cost-effective in the DE-only analysis with all ICERs falling below a cost-effectiveness threshold of three times GDP per capita in each city. Conclusions: Vaccination with PCV13 was a cost-effective strategy in the analyzed cities for both the DE-only and DE + IDE analyses. PCV13 became very cost-effective when a vaccination rate was reached where IDE is observed.

Tumor treating fields and maintenance temozolomide for newly-diagnosed glioblastoma: a cost-effectiveness study.

Purpose: The EF-14 trial demonstrated that adding tumor treating fields (TTFields) to maintenance temozolomide (TMZ) significantly extends progression-free survival (PFS) and overall survival (OS) for newly-diagnosed glioblastoma (GBM) patients. This study assessed the cost-effectiveness of TTFields and TMZ for newly-diagnosed GBM from the US healthcare system perspective. Methods and materials: Outcomes for newly-diagnosed GBM patients were estimated over a lifetime horizon using an area under the curve model with three states: stable disease, progressive disease, or death. The survival model integrated the 5-year EF-14 trial results with long-term GBM epidemiology data and US background mortality rates. Adverse event rates were derived from the EF-14 trial data. Utility values to determine quality-adjusted life-years, adverse event costs, and supportive care costs were obtained from published literature. A 3% discount rate was applied to future costs and outcomes. One-way and probabilistic sensitivity analyses were performed to assess result uncertainty due to parameter variability. Results: Treatment with TTFields and TMZ was estimated to result in a mean increase in survival of 1.25 life years (95% credible range [CR] = 0.89-1.67) and 0.96 quality-adjusted life years (QALYs) (95% CR = 0.67-1.30) compared to treatment with TMZ alone. The incremental total cost was $188,637 (95% CR = $145,324-$225,330). The incremental cost-effectiveness ratio (ICER) was $150,452 per life year gained and $197,336 per QALY gained. The model was most sensitive to changes in the cost of TTFields treatment. Conclusions: Adding TTFields to maintenance TMZ resulted in a substantial increase in the estimated mean lifetime survival and quality-adjusted survival for newly-diagnosed GBM patients. Treatment with TTFields can be considered cost-effective within the reported range of willingness-to-pay thresholds in the US.

The Cost of Hypoglycemia Associated With Type 2 Diabetes Mellitus in Taiwan.

OBJECTIVES: To quantify the incremental burden of patients with type 2 diabetes mellitus (T2DM) and a hypoglycemic event in Taiwan using the National Health Insurance Research Database. METHODS: Data from 2000 through 2013 with an index period of 2001 through 2012 from the National Health Insurance Research Database's 2-million-patient sample were used. Using a nested case-control study design, patients were indexed if they reported a diagnosis of T2DM during the index period. Patients with T2DM with a hypoglycemic event (defined by International Classification of Diseases, Ninth Revision, Clinical Modification codes) during the index period were identified. Patients with T2DM without a hypoglycemic event were included to form a 4:1 (controls to cases) matched cohort on the basis of age, sex, the Charlson Comorbidity Index, and the T2DM diagnosis date. Both cohorts were followed up for 1 year after the hypoglycemic event and had their treatment utilization, resource utilization, and healthcare costs measured. RESULTS: A total of 144 213 patients with T2DM were identified, with 3 651 (2.5%) recording a hypoglycemic event. Before matching, patients with T2DM with a hypoglycemic event were, on average, older (64.2 vs 56.6) and had higher mean CCI scores (2.4 vs 1.9) than did patients with T2DM without a hypoglycemic event. After matching, patients with T2DM and a hypoglycemic event incurred an additional $1353 in average direct healthcare costs during the 1 year of follow-up compared with the matched cohort. Patients with T2DM with hypoglycemia also spent an additional 5.9 days in the hospital during the follow-up period compared with the matched cohort. CONCLUSIONS: Patients with hypoglycemic events, on average, experienced a substantially higher economic burden than did their counterparts without a hypoglycemic event during the same period.

Estimated lifetime survival benefit of tumor treating fields and temozolomide for newly diagnosed glioblastoma patients.

AIM: To estimate the mean lifetime survival benefit, an essential component of health economic evaluations in oncology, of adding tumor treating fields (TTFields) to maintenance temozolomide (TMZ) for newly diagnosed glioblastoma patients. METHODS: We integrated EF-14 trial data with glioblastoma epidemiology data. The model provided for an evidence-based approach to estimate lifetime survival for the material number of EF-14 trial patients still alive at 5 years. RESULTS & CONCLUSION: Patients treated with TTFields and TMZ had an incremental mean lifetime survival of 1.8 years (TTFields/TMZ: 4.2 vs TMZ alone: 2.4). Patients alive at year 2 after starting TTFields had a 20.7% probability of surviving to year 10. The results presented here provide the required incremental survival benefit necessary for a future assessment of the incremental cost-effectiveness of TTFields.

Estimating the cost-effectiveness of an infant 13-valent pneumococcal conjugate vaccine national immunization program in China.

BACKGROUND: The burden of pneumococcal disease in China is high, and a 13-valent pneumococcal conjugate vaccine (PCV13) recently received regulatory approval and is available to Chinese infants. PCV13 protects against the most prevalent serotypes causing invasive pneumococcal disease (IPD) in China, but will not provide full societal benefits until made broadly available through a national immunization program (NIP). OBJECTIVE: To estimate clinical and economic benefits of introducing PCV13 into a NIP in China using local cost estimates and accounting for variability in vaccine uptake and indirect (herd protection) effects. METHODS: We developed a population model to estimate the effect of PCV13 introduction in China. Modeled health states included meningitis, bacteremia, pneumonia (PNE), acute otitis media, death and sequelae, and no disease. Direct healthcare costs and disease incidence data for IPD and PNE were derived from the China Health Insurance and Research Association database; all other parameters were derived from published literature. We estimated total disease cases and associated costs, quality-adjusted life years (QALYs), and deaths for three scenarios from a Chinese Payer Perspective: (1) direct effects only, (2) direct+indirect effects for IPD only, and (3) direct+indirect effects for IPD and inpatient PNE. RESULTS: Scenario (1) resulted in 370.3 thousand QALYs gained and 12.8 thousand deaths avoided versus no vaccination. In scenarios (2) and (3), the PCV13 NIP gained 383.2 thousand and 3,580 thousand QALYs, and avoided 13.1 thousand and 147.5 thousand deaths versus no vaccination, respectively. In all three scenarios, the vaccination cost was offset by cost reductions from prevented disease yielding net costs of ¥29,362.32 million, ¥29,334.29 million, and ¥13,524.72 million, respectively. All resulting incremental cost-effectiveness ratios fell below a 2x China GDP cost-effectiveness threshold across a range of potential vaccine prices. DISCUSSION: Initiation of a PCV13 NIP in China incurs large upfront costs but is good value for money, and is likely to prevent substantial cases of disease among children and non-vaccinated individuals.

Estimating the response and economic burden of rheumatoid arthritis patients treated with biologic disease-modifying antirheumatic drugs in Taiwan using the National Health Insurance Research Database (NHIRD).

BACKGROUND: Previous studies in Taiwan utilizing the Taiwan's National Health Insurance Database (NHIRD) have estimated the direct healthcare costs of RA patients, but they have not focused on patients on bDMARDs, or considered patients' response to therapy. OBJECTIVES: The objective of this study was to estimate the rate of inadequate response for patients newly treated with biologic disease-modifying antirheumatic drugs (bDMARDs) as well as their costs and resource use. METHODS: Data were from the catastrophic illness file within the NHIRD from 1/1/2009 to 12/31/2013. Patients with RA, which was categorized by the presence of a catastrophic illness card, that were previously bDMARD-naïve, were included in this study if they initiated their first bDMARD during the index period. The index period included all of 2010, a pre-index period consisting of the index date- 365 days, and a follow-up period including the index date to 365 days post-index, were also included. Previously biologically-naïve patients were indexed into the study on the date of their first claim for a bDMARD. A validated algorithm was used to examine the rate of inadequate response (IR) in the biologically-naïve cohort of patients. Inadequate responders met one or more of the following criteria during their year of follow-up: low adherence (proportion of days covered <0.80); switched to or added a second bDMARD; added a new conventional synthetic DMARD (csDMARD); received ≥1 glucocorticoid injection; or increased oral glucocorticoid dosing. All-cause mean annual direct costs and resource use were measured in the year of follow-up. Costs were converted from NT$ to USD using 1 NT$ = 0.033 USD. RESULTS: A total of 818 patients with RA initiated their first bDMARD (54% etanercept and 46% adalimumab) in 2010. After one year of follow-up, 32% (n = 258) were classified as stable, 66% (n = 540) had an IR, and 2% (n = 20) were lost to follow-up. During the follow-up period mean annual total direct costs were $16,136 for stable patients compared to $14,154 for patients with IR. Mean annual non-medication direct costs were $937 for stable patients and $1,574 for patients with IR. Mean annual hospitalizations were higher for patients with IR (0.46) compared to stable patients (0.10) during the one year follow-up period. CONCLUSIONS: The majority of patients that were previously naïve to bDMARDs had an IR to their first bDMARD during the year of follow-up. Patients with an IR had numerically increased all-cause resource utilization and non-medication costs during the follow-up period compared to patients with stable disease. This level of IR suggests an unmet need in the RA treatment paradigm.

The cost-effectiveness of a NSCLC patient assistance program for pemetrexed maintenance therapy in People's Republic of China.

BACKGROUND: Eli Lilly and the China Primary Health Care Foundation are currently implementing a patient assistance program (PAP) in China, which allows first-line nonsquamous non-small-cell lung cancer (NSCLC) patients who complete four cycles of pemetrexed induction therapy to receive free, continuous pemetrexed maintenance therapy. OBJECTIVE: To estimate the cost-effectiveness of pemetrexed maintenance therapy vs basic standard care (BSC) and the economic impacts of providing a PAP for pemetrexed maintenance therapy to NSCLC patients who have completed pemetrexed induction therapy in a Chinese health care setting. METHODS: We developed a novel decision-analytic model to evaluate the long-term costs and clinical efficacy of pemetrexed plus BSC vs BSC alone. We utilized a three-state (progression-free survival, progressed disease, and dead) partition survival model for both the clinical and economic aspects of the analysis. Cost and health utility estimates were derived from the literature. We performed a scenario analysis to estimate the real-world impact of introducing the PAP in China by comparing the use of the PAP vs non-PAP. Model uncertainty was evaluated using one-way and multivariate probabilistic sensitivity analysis. RESULTS: Compared to BSC, pemetrexed plus BSC resulted in a gain of 0.22 years of life (95% credible range [CR]: 0.04-0.46) and 0.13 quality-adjusted life years (95% CR: 0.04-0.26) per patient, at an increased cost of $28,105 (95% CR: -$22,720 to $48,646) without a PAP and $3,068 (95% CR: -$1,263 to $9,163) with a PAP. The incremental cost-effectiveness ratio for pemetrexed plus BSC vs BSC alone was cost-prohibitive at $222,700 for non-PAP, but cost-effective at $24,319 with a PAP. CONCLUSION: Our study suggests that maintenance pemetrexed therapy following pemetrexed induction for patients with advanced NSCLC is likely to be highly non-cost-effective in the absence of a PAP, but the pending implementation of the PAP promises to make it cost-effective, with a >90% probability of cost-effectiveness at a Chinese willingness-to-pay threshold per quality-adjusted life year.

Precision Health Economics and Outcomes Research to Support Precision Medicine: Big Data Meets Patient Heterogeneity on the Road to Value.

The "big data" era represents an exciting opportunity to utilize powerful new sources of information to reduce clinical and health economic uncertainty on an individual patient level. In turn, health economic outcomes research (HEOR) practices will need to evolve to accommodate individual patient-level HEOR analyses. We propose the concept of "precision HEOR", which utilizes a combination of costs and outcomes derived from big data to inform healthcare decision-making that is tailored to highly specific patient clusters or individuals. To explore this concept, we discuss the current and future roles of HEOR in health sector decision-making, big data and predictive analytics, and several key HEOR contexts in which big data and predictive analytics might transform traditional HEOR into precision HEOR. The guidance document addresses issues related to the transition from traditional to precision HEOR practices, the evaluation of patient similarity analysis and its appropriateness for precision HEOR analysis, and future challenges to precision HEOR adoption. Precision HEOR should make precision medicine more realizable by aiding and adapting healthcare resource allocation. The combined hopes for precision medicine and precision HEOR are that individual patients receive the best possible medical care while overall healthcare costs remain manageable or become more cost-efficient.

Estimating the Economic Burden of Rheumatoid Arthritis in Taiwan Using the National Health Insurance Database.

BACKGROUND: Rheumatoid arthritis (RA) is a chronic autoimmune disease characterized by inflammation and destruction of the joints. OBJECTIVES: This research aims to estimate the economic burden of RA in Taiwan. METHODS: The National Health Insurance Research Database (NHIRD), a claims-based dataset encompassing 99 % of Taiwan's population, was applied. We used a micro-costing approach for direct healthcare costs and indirect social costs by estimating the quantities and prices of cost categories. Direct costs included surgeries, hospitalizations, medical devices and materials, laboratory tests, and drugs. The costs and quantities of the direct economic burden were calculated based on 2011 data of NHIRD. We identified RA patients and a control cohort matched 1:4 on demographic and clinical covariates to calculate the incremental cost related to RA. Indirect costs were evaluated by missed work (absenteeism) and worker productivity (presenteeism). For the indirect burden, we estimated the rate of absenteeism and presenteeism from a patient survey. Costs were presented in US dollars (US$1 = 30 TWD). RESULTS: A total of 41,269 RA patients were included in the database with incremental total direct cost of US$86,413,971 and indirect cost of US$138,492,987. This resulted in an average incremental direct cost of US$2050 per RA patient. Within direct costs, the largest burdens were associated with drugs (US$73,028,944), laboratory tests (US$6,132,395), and hospitalizations (US$3,208,559). For indirect costs, absenteeism costs and presenteeism costs were US$16,059,681 and US$114,291,687, respectively. CONCLUSIONS: The economic burden of RA in Taiwan is driven by indirect healthcare costs, most notably presenteeism.

The Cost-Effectiveness of Pregabalin Versus Gabapentin for Peripheral Neuropathic Pain (pNeP) and Postherpetic Neuralgia (PHN) in China.

INTRODUCTION: Postherpetic neuralgia (PHN), a type of peripheral neuropathic pain (pNeP), is the most common complication of herpes zoster. The objective of this analysis was to determine the cost-effectiveness of pregabalin compared with gabapentin in pNeP and PHN in China. METHODS: We developed a China-localized 12-week simulation model to determine the cost-effectiveness of pregabalin compared to gabapentin in 1000 patients with pNeP and PHN. We utilized a questionnaire of Chinese key opinion leaders to estimate the pre-treatment distribution of pain scores for pNeP and PHN. Treatment outcomes for pregabalin and gabapentin were acquired from the published literature. RESULTS: Treatment with pregabalin lead to 12-week decreases in pain scores of 0.6 (pNeP) and 0.7 (PHN) when compared to patients receiving gabapentin, at an incremental cost per additional day of mild/no pain of $45. The difference in mean days of no or mild pain, moderate pain, and severe pain was 8.8, -5.7, and -3.1, when comparing pregabalin and gabapentin, respectively. Pregabalin had more mean days with a >30% (7.71 days), 40% (8.97 days), and 50% reduction (9.97 days) in pain when compared with gabapentin. In the pNeP scenario, pregabalin was associated with a lower average pain score compared with gabapentin (3.91 vs. 4.55). The difference in mean days of no or mild pain, moderate pain, and severe pain was 9.39, -5.56, and -3.82, when comparing pregabalin and gabapentin, respectively. Pregabalin had more mean days with a >30% (8.77 days), 40% (9.81 days), and 50% reduction (10.55 days) in pain when compared with gabapentin. CONCLUSION: Pregabalin is an effective treatment for PHN and even for pNeP extensively, but at increased cost. It leads to improved outcomes including lower pain scores and more days with no or mild pain. FUNDING: Pfizer, China.

Estimating the cost-effectiveness of linezolid for the treatment of methicillin-resistant Staphylococcus aureus nosocomial pneumonia in Taiwan.

BACKGROUND/PURPOSE: Methicillin-resistant Staphylococcus aureus (MRSA) nosocomial pneumonia (NP) is associated with higher resource utilization, increased hospital stays, and mortality. We present a health economics model to understand the impact of using linezolid as the first-line treatment of MRSA NP in Taiwan. METHODS: We developed a cost-effectiveness model to estimate the costs and clinical outcomes of using linezolid 600 mg b.i.d. versus vancomycin 15 mg/kg b.i.d. as the first-line treatment of MRSA NP in Taiwan. The model is a decision-analytic analysis in which a MRSA-confirmed patient is simulated to utilize one of the treatments, using data from a clinical trial. Within each treatment arm, the patient can or cannot achieve clinical cure. Regardless of whether the clinical cure was achieved or not, the patient may or may not have experienced an adverse event. The per-protocol results for clinical cure were 57.6% and 46.6% for linezolid and vancomycin, respectively. RESULTS: The total cost of linezolid was $376 more per patient than that of vancomycin. Drug costs were higher for linezolid than for vancomycin ($1108 vs. $233), and hospitalization costs were lower ($4998 vs. $5496). With higher cost and higher cure rates for linezolid, the incremental cost per cure was $3421. CONCLUSION: This study projects linezolid to have higher drug costs, lower hospital costs, and higher overall costs compared with vancomycin. This is balanced against the higher clinical cure rate for linezolid. Depending on the willingness to pay for clinical cure, linezolid could be cost effective as the first-line treatment of NP in Taiwan.

The cost reduction in hospitalization associated with paliperidone palmitate in the People's Republic of China, Korea, and Malaysia.

BACKGROUND: Schizophrenia results in substantial health care utilization costs. Much of these costs can be attributed to health care use resulting from nonadherence to treatment, relapse, and hospitalization. AIMS OF THE STUDY: The objective of this research is to further estimate the health care resource utilization costs of patients with schizophrenia in the People's Republic of China, Korea, and Malaysia with a specific focus on the reduction in hospitalization costs associated with the use of long-acting, injectable paliperidone palmitate (PP) relative to alternative treatment medications. METHODS: The study focuses exclusively on the estimated reduction in hospitalization days following treatment with PP and the potential associated cost savings. Cost analysis was done using a payer's perspective and only includes direct health care costs associated with hospitalization. Localized cost data were taken from published sources, and health care utilization was estimated based on a clinical study conducted in countries in the Asia-Pacific region. People's Republic of China, Korea, and Malaysia had the highest number of patients enrolled in the clinical study, and thus were chosen for this research. Analysis looked at 12-month and 18-month periods following initial treatment with PP relative to a retrospective 12-month period utilizing alternative treatment medications. RESULTS: Results suggest that reductions in hospital utilization cost over 12 months may occur through the use of PP relative to alternatives-ranging from $1,991 for the People's Republic of China to $6,698 for Korea and $6,716 for Malaysia. CONCLUSION: Given the substantial costs associated with the treatment of schizophrenia both worldwide and in Asia, it is important to fully understand the costs and outcomes associated with various treatment options. In this research, we have specifically analyzed the direct health care cost savings associated with hospital utilization for patients taking PP relative to alternative treatment methods. The results suggest that reductions in hospital utilization cost were associated with PP treatment, likely largely due to increased adherence to treatment.

The health and economic impact of vaccination with 7-valent pneumococcal vaccine (PCV7) during an annual influenza epidemic and influenza pandemic in China.

BACKGROUND: China has experienced several severe outbreaks of influenza over the past century: 1918, 1957, 1968, and 2009. Influenza itself can be deadly; however, the increase in mortality during an influenza outbreak is also attributable to secondary bacterial infections, specifically pneumococcal disease. Given the history of pandemic outbreaks and the associated morbidity and mortality, we investigated the cost-effectiveness of a PCV7 vaccination program in China from the context of typical and pandemic influenza seasons. METHODS: A decision-analytic model was employed to evaluate the impact of a 7-valent pneumococcal vaccine (PCV7) infant vaccination program on the incidence, mortality, and cost associated with pneumococcal disease during a typical influenza season (15% flu incidence) and influenza pandemic (30% flu incidence) in China. The model incorporated Chinese data where available and included both direct and indirect (herd) effects on the unvaccinated population, assuming a point in time following the initial introduction of the vaccine where the impact of the indirect effects has reached a steady state, approximately seven years following the implementation of the vaccine program. Pneumococcal disease incidence, mortality, and costs were evaluated over a one year time horizon. Healthcare costs were calculated using a payer perspective and included vaccination program costs and direct medical expenditures from pneumococcal disease. RESULTS: The model predicted that routine PCV7 vaccination of infants in China would prevent 5,053,453 cases of pneumococcal disease and 76,714 deaths in a single year during a normal influenza season.The estimated incremental-cost-effectiveness ratios were ¥12,281 (US$1,900) per life-year saved and ¥13,737 (US$2,125) per quality-adjusted-life-year gained. During an influenza pandemic, the model estimated that routine vaccination with PCV7 would prevent 8,469,506 cases of pneumococcal disease and 707,526 deaths, and would be cost-saving. CONCLUSIONS: Routine vaccination with PCV7 in China would be a cost-effective strategy at limiting the negative impact of influenza during a typical influenza season. During an influenza pandemic, the benefit of PCV7 in preventing excess pneumococcal morbidity and mortality renders a PCV7 vaccination program cost-saving.

The Lifetime Economic Burden of Inaccurate HER2 Testing: Estimating the Costs of False-Positive and False-Negative HER2 Test Results in US Patients with Early-Stage Breast Cancer.

BACKGROUND: Patients with breast cancer whose tumors test positive for human epidermal growth factor receptor 2 (HER2) are treated with HER2-targeted therapies such as trastuzumab, but limitations with HER2 testing may lead to false-positive (FP) or false-negative (FN) results. OBJECTIVES: To develop a US-level model to estimate the effect of tumor misclassification on health care costs and patient quality-adjusted life-years (QALYs). METHODS: Decision analysis was used to estimate the number of patients with early-stage breast cancer (EBC) whose HER2 status was misclassified in 2012. FP results were assumed to generate unnecessary trastuzumab costs and unnecessary cases of trastuzumab-related cardiotoxicity. FN results were assumed to save money on trastuzumab, but with a loss of QALYs and greater risk of disease recurrence and its associated costs. QALYs were valued at $100,000 under a net monetary benefit approach. RESULTS: Among 226,870 women diagnosed with EBC in 2012, 3.12% (n = 7,070) and 2.18% (n = 4,955) were estimated to have had FP and FN test results, respectively. Approximately 8400 QALYs (discounted, lifetime) were lost among women not receiving trastuzumab because of FN results. The estimated incremental per-patient lifetime burden of FP or FN results was $58,900 and $116,000, respectively. The implied incremental losses to society were $417 million and $575 million, respectively. CONCLUSIONS: HER2 tests result in misclassification and nonoptimal treatment of approximately 12,025 US patients with EBC annually. The total economic societal loss of nearly $1 billion suggests that improvements in HER2 testing accuracy are needed and that further clinical and economic studies are warranted.

The short-term cost-effectiveness of once-daily liraglutide versus once-weekly exenatide for the treatment of type 2 diabetes mellitus in the United States.

BACKGROUND: Type 2 diabetes mellitus (T2DM) is a chronic metabolic disease with substantial morbidity, mortality, and economic impacts. Glucagon-like peptide-1 (GLP-1) receptor agonists, such as once-daily (QD) liraglutide and once-weekly (QW) exenatide, are FDA-approved treatment for T2DM. Head-to-head trials and meta-analyses comparing these agents have reported clinically meaningful improvements but small differences in glycemic control between both agents. In this study, we calculate and compare the cost-effectiveness implications of these alternative effectiveness outcomes. METHODS: We developed a decision model to evaluate the short-term cost-effectiveness of exenatide QW 2 mg versus liraglutide QD 1.8 mg in T2DM patients, with effectiveness measured as reduction in glycated hemoglobin (HbA1c). In the base case, the model tracks change in HbA1c and direct medical expenditure over a 6-month time horizon. We calculated and compared the cost per 1% reduction in HbA1c of models populated with clinical data from a head-to-head randomized, controlled trial (DURATION-6) and a network meta-analysis. Expenditure inputs were derived from wholesale acquisition costs and published sources. RESULTS: In the base case, 6-month expenditure for the liraglutide and exenatide strategies were $3,509 and $2,618, respectively. Using clinical data from DURATION-6 and the network meta-analysis, the liraglutide strategy had an incremental cost per 1% reduction in HbA1c of $4,773 and $27,179, respectively. The most influential model parameters were drug costs, magnitude of HbA1c reduction in patients on treatment for >1 month, and liraglutide gastrointestinal adverse event rate. In probabilistic sensitivity analyses (PSA) using DURATION-6 data, the exenatide strategy was optimal at willingness-to-pay levels below $4,800 per 1% reduction in HbA1c. In a PSA using meta-analysis data, the exenatide strategy was dominant. CONCLUSIONS: Our modeled results demonstrate that the effectiveness and cost-effectiveness of liraglutide QD 1.8 mg relative to exenatide QW 2 mg depend largely on the chosen source of the clinical data.