Early Cost-effectiveness Analysis of Electrochemotherapy as a Prospect Treatment Modality for Skin Melanoma

PurposeElectrochemotherapy is increasingly entering into national and international guidelines, requiring formal evaluation of treatment costs and cost-effectiveness to ensure that its uptake provides value to budget-constrained health care systems. This study analyzed the early cost-effectiveness of electrochemotherapy in patients with Stage IIIc/IV skin melanoma in clinical practice in Slovenia. The costs of electrochemotherapy were compared to those of the standard of care, consisting of palliative treatment and therapy for symptoms.MethodswThe study enrolled 23 patients treated with electrochemotherapy at the Institute of Oncology (Ljubljana, Slovenia). The mean cost of electrochemotherapy was estimated using patient-specific cost data on electrochemotherapy procedures and subsequent follow-up. Quality-adjusted life-years (QALYs) were estimated by collecting EQ-5D-3L questionnaires at baseline, after complete or partial response following the treatment, and after a relapse of skin lesions. A discrete-time Markov model was built to estimate the lifetime costs and consequences of using electrochemotherapy compared to standard of care, from the perspective of the Slovenian health care system. The analysis was conducted separately in the whole patient sample and in the subset of patients with bleeding lesions. Deterministic and probabilistic sensitivity analyses were conducted to test model assumptions and to characterize the uncertainty around model parameters.FindingsIn the whole patient population, electrochemotherapy for skin melanoma Stage IIIc/IV was expected to increase QALYs by 0.29 (95% credible interval [CrI], 0.10–0.50), at the higher cost of 6568 EUR (95% CrI, 4593–8928) in comparison to the standard of care. At the cost-effectiveness threshold of 20,000 EUR/QALY, the estimated probabilities of electrochemotherapy being cost-effective compared to standard of care were 0.30 and 0.91 in the whole patient sample and in patients with bleeding lesions, respectively. In the whole sample population, a 50% reduction in the price of the electrodes was expected to increase the probability of electrochemotherapy being cost-effective from 0.30 to ~0.64.ImplicationsThe findings from this cost-effectiveness analysis of data from clinical practice were based on a small sample size (ie, 23 patents), which made the subgroup of patients with bleeding lesions very small. Therefore, the findings in this patient population should be carefully interpreted.     

Cost-effectiveness of ticagrelor versus clopidogrel for the prevention of atherothrombotic events in adult patients with acute coronary syndrome in Germany

The aim of this health economic analysis was to compare the cost-effectiveness of ticagrelor versus clopidogrel within the German health care system. A two-part decision model was adapted to compare treatment with ticagrelor or clopidogrel in a low-dose acetylsalicylic acid (ASA) cohort (≤150 mg) for all ACS patients and subtypes NSTEMI/IA and STEMI. A decision-tree approach was chosen for the first year after initial hospitalization based on trial observations from a subgroup of the PLATO study. Subsequent years were estimated by a Markov model. Following a macro-costing approach, costs were based on official tariffs and published literature. Extensive sensitivity analyses were performed to test the robustness of the model. One-year treatment with ticagrelor is associated with an estimated 0.1796 life-years gained (LYG) and gained 0.1570 quality-adjusted life-years (QALY), respectively, over the lifetime horizon. Overall average cost with ticagrelor is estimated to be EUR 11,815 vs. EUR 11,387 with generic clopidogrel over a lifetime horizon. The incremental cost-effectiveness ratio (ICER) was EUR 2,385 per LYG (EUR 2,728 per QALY). Comparing ticagrelor with Plavix^® or the lowest priced generic clopidogrel, ICER ranges from dominant to EUR 3,118 per LYG (EUR 3,567 per QALY). These findings are robust under various additional sensitivity analyses. Hence, 12 months of ACS treatment using ticagrelor/ASA instead of clopidogrel/ASA may offer a cost-effective therapeutic option, even when the generic price for clopidogrel is employed.     

The impact of implementing a rapid response system: A comparison of cardiopulmonary arrests and mortality among four teaching hospitals in Australia

Aims

To compare clinical outcomes between a teaching hospital with a mature rapid response system (RRS), with three similar teaching hospitals without a RRS in Sydney, Australia.

Methods

For the period 2002–2009, we compared a teaching hospital with a mature RRS, with three similar teaching hospitals without a RRS. Two non-RRS hospitals began implementing the system in 2009 and a third in January 2010. We compared the rates of in-hospital cardiopulmonary arrest (IHCA), IHCA-related mortality, overall hospital mortality and 1-year post discharge mortality after IHCA between the RRS hospital and the non-RRS hospitals based on three separate analyses: (1) pooled analysis during 2002–2008; (2) before–after difference between 2008 and 2009; (3) after implementation in 2009.

Results

During the 2002–2008 period, the mature RRS hospital had a greater than 50% lower IHCA rate, a 40% lower IHCA-related mortality, and 6% lower overall hospital mortality. Compared to 2008, in their first year of RRS (2009) two hospitals achieved a 22% reduction in IHCA rate, a 22% reduction in IHCA-related mortality and an 11% reduction in overall hospital mortality. During the same time, the mature RRS hospital showed no significant change in those outcomes but, in 2009, it still achieved a crude 20% lower IHCA rate, and a 14% lower overall hospital mortality rate. There was no significant difference in 1-year post-discharge mortality for survivors of IHCA over the study period.

Conclusions

Implementation of a RRS was associated with a significant reduction in IHCA, IHCA-related mortality and overall hospital mortality.     

Cost-effectiveness and Net Monetary Benefit of Durvalumab Consolidation Therapy Versus No Consolidation Therapy After Chemoradiotherapy in Stage III Non–small Cell Lung Cancer in the Italian National Health Service

PurposeThe aim of this study was to evaluate the cost-effectiveness and net monetary benefit of durvalumab consolidation therapy compared with no consolidation therapy after chemoradiotherapy in patients with stage III non–small cell lung cancer with programmed cell death 1 ligand 1 expression ≥1% from the Italian National Health Service perspective.MethodsWe developed a 12-month decision tree combined with a lifetime cohort Markov model in which patients were assigned to receive durvalumab consolidation therapy or active follow-up (Italian standard of care) after chemoradiotherapy to compare cost-effectiveness and net monetary benefit of the two strategies during a 40-year period. Clinical outcomes data were obtained from the respective clinical trials and extrapolated using survival analysis; cost data were derived from Italian official sources and relevant real-world studies. The incremental cost-effectiveness ratio, incremental cost-utility ratio, and incremental net monetary benefit were computed and compared against a 16,372 € per quality-adjusted life-year (QALY) willingness-to-pay threshold. We performed deterministic sensitivity analysis and probabilistic sensitivity analysis to assess how uncertainty affected results; we also performed scenario analyses to compare results under different pricing settings.FindingsIn the base-case scenario, during a 40-year period, the total costs for patients treated with durvalumab consolidation therapy and active follow-up were €59,860 and €49,840 respectively; life-years gained were 3.47 and 3.31, respectively; and QALYs gained were 2.73 and 2.50, respectively, with an incremental cost-effectiveness ratio of €62,131 per life-year, an incremental cost-utility ratio of €42,322 per QALY, and an incremental net monetary benefit of €−6,144. We found that durvalumab was cost-effective (incremental net monetary benefit = 0) when a discount of 13% and 30% on its official price was applied, considering all other drugs priced according to official or maximum selling prices, respectively. Results were most sensitive to the progression-free survival rate for durvalumab and active follow-up, health utility in progression-free state, and price of subsequent treatments.ImplicationsOur analysis indicates that durvalumab consolidation is cost-effective when a discount is applied on its official price. These results suggest that durvalumab may deliver an incremental health benefit with a contained upfront cost during a 40-year period, from the Italian National Health Service perspective, providing added value in a potentially curative care setting.     

Incremental cost-effectiveness analysis of a multidisciplinary renal education program for patients with chronic renal disease

Aim.?The aim of this economic evaluation is to address the economic impact of a multidisciplinary renal patient education on the work maintenance of patients with chronic renal disease.

Method.?A total of 281 patients were recruited. An incremental cost-effectiveness analysis considering direct medical costs and days in the work force was performed. In addition, a Cox proportional hazards survival analysis was performed to investigate the effect of receiving the intervention on early retirement due to work disability.

Results.?The incremental cost-effectiveness ratio [95% CI] per day in the work force saved was 737.02°€ [?3345.64; 3093.03] in the whole group, 73.74°€ [?995.28; 1429.92] in patients with a mild limitation and ?113.25°€ [?3252.06; 1525.58] in patients with a moderate to severe limitation in kidney function. After adjusting for observed time period, serum creatinine and age, having received the intervention had an almost significant medium-term protective effect on early retirement (hazard ratio 0.314, 95% CI 0.10–1.03, p?=?0.0557).

Conclusions.?The cost-effectiveness of the multidisciplinary patient education highly depends on the level of limitation in kidney function and the intervention may be cost-effective in maintaining patients with mild limitation in kidney function in the work force.     

Cost-effectiveness of Dabigatran Compared With Rivaroxaban for Prevention of Stroke and Systemic Embolism in Patients With Atrial Fibrillation in China

PurposeIn China, dabigatran and rivaroxaban are the only approved non–vitamin K antagonist oral anticoagulants for the treatment of atrial fibrillation (AF). The goal of this article was to assess the cost-effectiveness of dabigatran versus rivaroxaban for the prevention of stroke and systemic embolism in Chinese patients with AF from the perspective of the Chinese health care system.MethodsA Markov model was constructed to estimate the cost-effectiveness of dabigatran versus rivaroxaban. Clinical events were modeled for a lifetime horizon, based on clinical efficacy data from indirect treatment comparisons. The weighted average of the most recent prices of these 2 drugs was used as the drug acquisition cost. Other costs, including follow-up costs and event costs, were collected by using a survey from a panel of local experts. Utility inputs (health state utilities, clinical event disutilities, and event history utility) were obtained from published literature. Sensitivity analyses that included scenario analyses and a probabilistic sensitivity analysis were conducted to examine the robustness of the economic model.FindingsOver a lifetime, patients treated with dabigatran experienced fewer ischemic strokes (2.14 dabigatran vs 2.61 rivaroxaban) and fewer intracranial hemorrhage (0.48 vs 0.94) per 100 patient-years. In the base case analysis, dabigatran had an incremental cost of ¥28,128 but with higher life years (10.38 vs 10.14) and quality-adjusted life years (QALYs) (7.95 vs 7.70). The resulting incremental cost-effectiveness ratio of ¥112,910 per QALY gained and net monetary benefit of ¥12,214 versus rivaroxaban showed that dabigatran was a cost-effective alternative to rivaroxaban. Extensive sensitivity analyses indicated that the results were robust over a wide range of inputs. The probabilistic sensitivity analysis indicated that dabigatran was cost-effective in 84.2% of the 10,000 Monte Carlo simulations compared with rivaroxaban.ImplicationsDabigatran reduced the occurrence of clinical events and increased QALYs compared with rivaroxaban. The use of dabigatran for the prevention of stroke and systemic embolism is a cost-effective option compared with rivaroxaban among patients with AF in China.     

A Cost-effectiveness Analysis of Albumin in Septic Shock: A Patient-level Data Analysis

PurposeAlbumin-based fluid therapy in septic shock is a matter of debate and criticism. The aim of this study was to assess the cost-effectiveness of albumin therapy in patients with septic shock.MethodsA retrospective cohort study was conducted in Imam Khomeini, Sina, and Shariati hospitals on patients with septic shock admitted to intensive care units from March 31, 2016 to September 22, 2017. Data sources were the health information system database and patient medical records. The patients with potential septic shock were identified based on norepinephrine use. Septic shock was confirmed after medical record review based on systemic inflammatory response syndrome criteria, antibiotic use, and fluid therapy. Patients who received albumin in the fluid therapy were compared with patients treated without albumin. The 28-day mortality, life-year gain, and cost-effectiveness were evaluated.FindingsThe addition of albumin had no significant increase in life-year gain (mean difference = 0.67; 95% CI, −2.25 to 3.58). However, the addition of albumin increased the total cost of treatment by US $3846.07 (95% CI, US $2093.46–US $5598.98). The incremental cost-effectiveness ratio calculated based on the mean life-years gained was US$5740.40 per a life-year gained. The net monetary benefit was negative (−355.4; 95% CI, −15,387.61 to 14,676.81), and the probability that the addition of albumin will be cost-effective at a gross domestic product per capita was 40.0%.ImplicationsAlbumin-based fluid therapy does not improve the 28-day mortality of patients with septic shock. The addition of albumin in the fluid therapy of patients with septic shock was not cost-effective. Both the observational and retrospective nature of the study was expected to introduce bias. We recommend a cost-effectiveness analysis combined with clinical trials to settle the debate once and for all.     

Cost-effectiveness Analysis of Empagliflozin in Japan Based on Results From the Asian subpopulation in the EMPA-REG OUTCOME Trial

PurposeThe goal of this study was to assess the cost-effectiveness of empagliflozin in Japan based on the Asian subpopulation in the EMPA-REG OUTCOME trial.MethodsThe trial has shown a reduction in the risk for cardiovascular (CV) and renal events with empagliflozin in patients with type 2 diabetes mellitus and established CV disease. A cost-effectiveness analysis based on the overall population of the EMPA-REG OUTCOME trial was reported previously by using a lifetime discrete event simulation model. The same modeling frame was adapted to evaluate the cost-effectiveness of treatment with empagliflozin added to standard of care (SoC) compared with SoC alone in Japan. The time to relevant clinical events and the hazard ratios were derived from an Asian subpopulation in the EMPA-REG OUTCOME trial. The costs for each event were estimated from a Japanese medical claims database. Direct medical costs, life expectancy, and quality-adjusted life years (QALYs) were calculated from the public health care perspective.FindingsTreatment with empagliflozin was estimated to increase life expectancy by 6.2 years and 2.7 QALYs, whereas total cost increased by 1,115,475 yen compared with treatment with SoC alone. The incremental cost-effectiveness ratio was 415,849 yen/QALY. In the sensitivity analysis, there was no case that was in excess of the reference value of the incremental cost-effectiveness ratio in the pilot introduction for price revision in Japan (ie, 5 million yen/QALY).ImplicationsBased on the Asian subpopulation in the EMPA-REG OUTCOME trial, our results suggest that empagliflozin added to SoC is highly cost-effective compared with SoC alone in Japan.     

Cost-effectiveness of urinary dipsticks to screen asymptomatic catheter-associated urinary infections in an intensive care unit

OBJECTIVE: To assess the cost-effectiveness of urinary dipsticks (UDs) to screen asymptomatic catheterized patients for quantitative urine. DESIGN: Prospective comparison of UD with quantitative urine culture (QUC) (reference technique) and cost-effectiveness analysis performed from the hospital's perspective. SETTING: Medical intensive care unit (ICU) of the Besan?on University Hospital (France). PATIENTS AND PARTICIPANTS: All consecutive, asymptomatic, catheterized patients. INTERVENTIONS: Urinary dipsticks (Multistix 8-SG) were analyzed by the reflectance spectrophotometric method (Clinitek 50). Sensitivity (Se), specificity (Sp), positive predictive value (PPV) and negative predictive value (NPV) of four combinations of the leukocyte (L) test pad and the nitrite (N) test pad were calculated: L and N, L or N, L alone and N alone. A micro-costing technique was used to determine the direct medical cost of each strategy. The calculated cost-effectiveness ratio was the incremental cost-effectiveness (ICE) ratio. MEASUREMENTS AND RESULTS: Three hundred thirty-nine urine samples taken from 144 patients were analyzed. The incidence of asymptomatic catheter-associated urinary tract infections (CAUTIs) was 31.3% (> or =10(5) organisms/ml). The L or N combination was the best detector of asymptomatic CAUTI: Se=87.2%, Sp=61.6%, PPV=30.6% and NPV=96.1%. The cost of QUC strategy and UD strategy was EUR 21.5 and EUR 12.6 per test, respectively. The ICE ratio of QUCs was EUR 69.5 per case of detected CAUTI. CONCLUSION: The UD is a cost-effective test for screening asymptomatic catheterized patients for quantitative urine culture in a medical ICU.     

Cost-Effectiveness of Baricitinib Compared With Standard of Care: A Modeling Study in Hospitalized Patients With COVID-19 in the United States

PurposeIn the Phase III COV-BARRIER (Efficacy and Safety of Baricitinib for the Treatment of Hospitalised Adults With COVID-19) trial, treatment with baricitinib, an oral selective Janus kinase 1/2 inhibitor, in addition to standard of care (SOC), was associated with significantly reduced mortality over 28 days in hospitalized patients with coronavirus disease–2019 (COVID-19), with a safety profile similar to that of SOC alone. This study assessed the cost-effectiveness of baricitinib + SOC versus SOC alone (which included systemic corticosteroids and remdesivir) in hospitalized patients with COVID-19 in the United States.MethodsAn economic model was developed to simulate inpatients' stay, discharge to postacute care, and recovery. Costs modeled included payor costs, hospital costs, and indirect costs. Benefits modeled included life-years (LYs) gained, quality-adjusted life-years (QALYs) gained, deaths avoided, and use of mechanical ventilation avoided. The primary analysis was performed from a payor perspective over a lifetime horizon; a secondary analysis was performed from a hospital perspective. The base-case analysis modeled the numeric differences in treatment effectiveness observed in the COV-BARRIER trial. Scenario analyses were also performed in which the clinical benefit of baricitinib was limited to the statistically significant reduction in mortality demonstrated in the trial.FindingsIn the base-case payor perspective model, an incremental total cost of 17,276 US dollars (USD), total QALYs gained of 0.6703, and total LYs gained of 0.837 were found with baricitinib + SOC compared with SOC alone. With the addition of baricitinib, survival was increased by 5.1% and the use of mechanical ventilation was reduced by 1.6%. The base-case incremental cost-effectiveness ratios were 25,774 USD/QALY gained and 20,638 USD/LY gained; a “mortality-only” scenario analysis yielded similar results of 26,862 USD/QALY gained and 21,433 USD/LY gained. From the hospital perspective, combination treatment with baricitinib + SOC was more effective and less costly than was SOC alone in the base case, with an incremental cost of 38,964 USD per death avoided in the mortality-only scenario.ImplicationsIn hospitalized patients with COVID-19 in the United States, the addition of baricitinib to SOC was cost-effective. Cost-effectiveness was demonstrated from both the payor and the hospital perspectives. These findings were robust to sensitivity analysis and to conservative assumptions limiting the clinical benefits of baricitinib to the statistically significant reduction in mortality demonstrated in the COV-BARRIER trial.     

Cost-effectiveness of dronedarone in atrial fibrillation: results for Canada, Italy, sweden, and Switzerland

BackgroundDronedarone is a therapy for the treatment of patients with paroxysmal and persistent atrial fibrillation or atrial flutter. According to results in the ATHENA trial, dronedarone on top of standard of care (SOC) decreases the risk of cardiovascular hospitalizations or death by 24% compared with SOC alone.ObjectivesA patient-level health economic model was developed to evaluate the cost-effectiveness of dronedarone on top of SOC versus SOC alone.MethodsThe risk of experiencing stroke, congestive heart failure, acute coronary syndromes, treatment discontinuation, and death was modeled by separate health states, whereas adverse events were included as 1-time cost and utility decrements. State transition probabilities were primarily deduced from the patient-level data from ATHENA using survival analysis. Four sets of analyses were performed to reflect costs and treatment effects in Canada, Italy, Sweden, and Switzerland. Cost-effectiveness analysis was also conducted in a newly defined patient population identified by the European Medicines Agency (EMA) to avoid the use of dronedarone in permanent AF patients resembling those in the PALLAS study.ResultsThe predicted survival time was, for the Canadian cohort, extended from 10.11 to 10.24 years when dronedarone was added to SOC. Similar results were found for the other countries, resulting in incremental cost-effectiveness ratios (ICERs) of €5828, €5873, €14,970, and €8554 per QALYs for Canada, Italy, Sweden, and, Switzerland, respectively. These results are all well below current established cost-effectiveness thresholds. In the EMA-restricted population, all patients were predicted to live longer, and the ICER increased but remained within established thresholds, with an average cost per QALY gained of €15,900.ConclusionsDronedarone on top of SOC appears to be a cost-effective treatment for atrial fibrillation compared with SOC alone. Despite the differences in the local settings considered, the results were consistent among all the countries included in the study. ClinicalTrials.gov identifier: NCT00174785.     

Cost-effectiveness Analysis of Prophylaxis Versus On-demand Treatment for Children With Hemophilia B Without Inhibitors in China

PurposeHemophilia B (HB) is a hereditary bleeding disorder caused by a deficiency of coagulation factor IX (FIX), which represents 15% to 20% of all patients with hemophilia. Clinical studies have found significant benefits of prophylaxis treatment with FIX versus on-demand (OD) treatment. However, these benefits are associated with an increase in FIX consumption and a considerable increase in cost. Most Chinese children with HB receive OD treatment. Only a small proportion of patients with HB receive prophylaxis treatment in China. The patients with inhibitors could result in more complicated bleeding events, joint status, or treatment patterns. The objective of this study is to assess the cost-effectiveness of prophylaxis compared with OD treatment in children with HB without inhibitors from the Chinese health care perspective.MethodsA Markov model was used to analyze cost-effectiveness by comparing prophylaxis with OD treatment. The model uses a 17-year time horizon with a yearly cycle. Transition probabilities and utility weights were estimated using published studies. The cost data for patients with HB were collected from Beijing Children's Hospital and Capital Medical University. One-way and probabilistic sensitivity analyses were performed to assess the robustness of the results.FindingsThe model projects that prophylaxis has an incremental 1.23 quality-adjusted life-years (QALYs). The incremental cost per QALY gained for individuals with HB receiving prophylaxis was ¥155,709.80 ($23,530.36) compared with the OD group, which is under the willingness-to-pay threshold (3 times the gross domestic product per capital according to the World Health Organization recommendations) in China of ¥193,932 ($29,306.37). Moreover, 1-way sensitivity analysis found that the results were sensitive to the utility of nonarticular bleeding. The lower this parameter is, the higher the probability is of the incremental cost-effectiveness ratio for prophylaxis not being cost-effective. This finding infers that when the patients have a better QALY (higher utility) at the beginning, the cost for benefit from prophylaxis treatment is lower. The results of the probabilistic sensitivity analyses indicate that the probability of prophylaxis being cost-effective is 89.3%.ImplicationsAlthough prophylaxis is a costly treatment, the results of this study indicate that it is cost-effective compared with OD treatment for children with HB in China.     

A preliminary economic evaluation of percutaneous neuromuscular electrical stimulation in the treatment of hemiplegic shoulder pain

Objective. The objective of this study was to compare the cost-effectiveness of various treatment modalities for hemiplegic shoulder pain.

Design. A stage II economic evaluation.

Main outcome measures. Incremental cost effectiveness ratio of P-NMES, compared to slings and anti-inflammatory injections.

Results. The incremental cost effectiveness ratio (ICER) of p-NMES, compared to anti-inflammatory injections is €6,061 (±3,285). The incremental cost of the first quality-adjusted life year after implantation of the P-NMES device compared to anti-inflammatory injections is €33,007 (±5,434). This decreases to ≈ €7,000 after 5 years, and to ≈ €5,000 after 10 survival years.

Conclusion. In this early evaluation, P-NMES seems to be cost-effective according to known guidelines. Treatment with P-NMES is recommended for patients with chronic HSP.     

Cost-effectiveness Comparison of Ceftazidime/Avibactam Versus Meropenem in the Empirical Treatment of Hospital-acquired Pneumonia,Including Ventilator-associated Pneumonia,in Italy

PurposeCeftazidime/avibactam (CAZ-AVI) is a fixed-dose combination antibiotic approved in Europe and the United States for patients with hospital-acquired pneumonia, including ventilator-associated pneumonia (HAP/VAP). The economic benefits of a new drug such as CAZ-AVI are required to be assessed against those of available comparators, from the perspective of health care providers and payers, through cost-effectiveness and cost-utility analyses. The objective of this analysis was to compare the cost-effectiveness of CAZ-AVI versus meropenem in the empirical treatment of appropriate hospitalized patients with HAP/VAP caused by gram-negative pathogens, from the perspective of publicly funded health care in Italy (third-party perspective, based on the data from the REPROVE (Ceftazidime-Avibactam Versus Meropenem In Nosocomial Pneumonia, Including Ventilator-Associated Pneumonia) clinical study; ClinicalTrials.gov NCT01808092).MethodsA patient-level, sequential simulation model of the HAP/VAP clinical course was developed using spreadsheet software. The analysis focused on direct medical costs. The time horizon of the model selected was 5 years, with an annual discount rate of 3% on costs and quality-adjusted life-years (QALYs). Clinical inputs for treatment comparisons were mainly obtained from the REPROVE clinical study data. In addition to clinical outcomes observed in the trial, the model incorporated impact of resistance pathogens, based on data from published studies and expert opinion. Certain assumptions were made for some model parameters due to a lack of data.FindingsThe analysis demonstrated that the intervention sequence (CAZ-AVI followed by colistin + high-dose meropenem) versus the comparator sequence (meropenem followed by colistin + high-dose meropenem) provided a better clinical cure rate (+13.52%), which led to a shorter hospital stay (−0.40 days per patient), and gains in the number of life-years (+0.195) and QALYs (+0.350) per patient. The intervention sequence had an estimated net incremental total cost of €1254 ($1401) per patient, and the estimated incremental cost-effectiveness ratio was €3581 ($4000) per QALY gained, well below the willingness-to-pay threshold of €30,000 ($33,507) per QALY in Italy.ImplicationsThe model results showed that CAZ-AVI is expected to provide clinical benefits in hospitalized patients with HAP/VAP in Italy at an acceptable cost compared to meropenem.     

Cost-Effectiveness and Net Monetary Benefit of Olaparib Maintenance Therapy Versus No Maintenance Therapy After First-line Platinum-based Chemotherapy in Newly Diagnosed Advanced BRCA1/2-mutated Ovarian Cancer in the Italian National Health Service

PurposeThe aim of this study was to evaluate the cost-effectiveness and net monetary benefit of olaparib maintenance therapy compared with no maintenance therapy after first-line platinum-based chemotherapy in newly diagnosed advanced BRCA1/2-mutated ovarian cancer from the Italian National Health Service (NHS) perspective.MethodsWe developed a lifetime Markov model in which a cohort of patients with newly diagnosed advanced BRCA1/2-mutated ovarian cancer was assigned to receive either olaparib maintenance therapy or active surveillance (Italian standard of care) after first-line platinum-based chemotherapy to compare cost-effectiveness and net monetary benefit of the 2 strategies. Data on clinical outcomes were obtained from related clinical trial literature and extrapolated using parametric survival analyses. Data on costs were derived from Italian official sources and relevant real-world studies. The incremental cost-effectiveness ratio (ICER), incremental cost-utility ratio (ICUR), and incremental net monetary benefit (INMB) were computed and compared against an incremental cost per quality-adjusted life-year (QALY) gained of €16,372 willingness-to-pay (WTP) threshold. We used deterministic sensitivity analysis (DSA) and probabilistic sensitivity analysis (PSA) to assess how uncertainty affects results; we also performed scenario analyses to compare results under different pricing settings.FindingsIn the base-case scenario, during a 50-year time horizon, the total costs for patients treated with olaparib therapy and active surveillance were €124,359 and €97,043, respectively, and QALYs gained were 7.29 and 4.88, respectively, with an ICER of €9,515 per life-year gained, an ICUR of €11,345 per QALY gained, and an INMB of €12,104. In scenario analyses, considering maximum selling prices for all other drugs, ICUR decreased to €11,311 per QALY and €7,498 per QALY when a 10% and 20% discount, respectively, was applied to the olaparib official price, and the INMB increased to €12,186 and €21,366, respectively. DSA found that the model results were most sensitive to the proportion of patients with relapsing disease in response to platinum-based chemotherapy, time receiving olaparib first-line maintenance treatment, and subsequent treatments price. According to PSAresults, olaparib was associated with a probability of being cost-effective at a €16,372 per QALY WTP threshold ranging from 70% to 100% in the scenarios examined.ImplicationsOur analysis indicates that olaparib maintenance therapy may deliver a significant health benefit with a contained upfront cost during a 50-year time horizon, from the Italian NHS perspective, providing value in a setting with curative intent.     

Assessing the Cost-effectiveness of a Hypothetical Disease-modifying Therapy With Limited Duration for the Treatment of Early Symptomatic Alzheimer Disease

PurposeClinical trials have produced promising results for disease-modifying therapies (DMTs) for Alzheimer’s disease (AD); however, the evidence on their potential cost-effectiveness is limited. This study assesses the cost-effectiveness of a hypothetical DMT with a limited treatment duration in AD.MethodsWe developed a Markov state–transition model to estimate the cost-effectiveness of a hypothetical DMT plus best supportive care (BSC) versus BSC alone among Americans living with mild cognitive impairment (MCI) due to AD or mild AD. AD states included MCI due to AD, mild AD, moderate AD, severe AD, and death. A hypothetical DMT was assumed to confer a 30% reduction in progression from MCI and mild AD. The base case annual drug acquisition cost was assumed to be $56,000. Other medical and indirect costs were obtained from published literature or list prices. Utilities for patients and caregivers were obtained from the published literature and varied by AD state and care setting (community care or long-term care). We considered 3 DMT treatment strategies: (1) treatment administered until patients reached severe AD (continuous strategy), (2) treatment administered for a maximum duration of 18 months or when patients reached severe AD (fixed-duration strategy), and (3) 40% of patients discontinuing treatment at 6 months because of amyloid plaque clearance and the remaining patients continuing treatment until 18 months or until they reached severe AD (test-and-discontinue strategy). Incremental cost-effectiveness ratios (ICERs) were calculated as the incremental cost per quality-adjusted life-year (QALY) gained.FindingsFrom the health care sector perspective, continuous treatment with a hypothetical DMT versus BSC resulted in an ICER of $612,354 per QALY gained. The ICER decreased to $157,288 per QALY gained in the fixed-duration strategy, driven by large reductions in treatment costs. With 40% of patients discontinuing treatment at 6 months (test-and-discontinue strategy), the ICER was $125,631 per QALY gained. In sensitivity and scenario analyses, the ICER was the most sensitive to changes in treatment efficacy, treatment cost, and the initial population AD state distribution. From the modified societal perspective, ICERs were 6.3%, 20.4%, and 25.1% lower than those from the health care sector perspective for the continuous, fixed-duration, and test-and-discontinue strategies, respectively.ImplicationsUnder a set of assumptions for annual treatment costs and the magnitude and duration of treatment efficacy, DMTs used for a limited duration may deliver value consistent with accepted US cost-effectiveness thresholds.     

Economic Evaluation of Adding Daratumumab to a Regimen of Bortezomib + Dexamethasone in Relapsed or Refractory Multiple Myeloma: Based on the Latest Updated Analysis of CASTOR

PurposeAdding daratumumab to a regimen of bortezomib + dexamethasone (Vd) has been reported to provide a benefit of longer progression-free survival in patients with relapsed or refractory multiple myeloma (RRMM). However, it is still unclear whether the addition of daratumumab is cost-effective in RRMM. Based on the latest updated analysis of data from the CASTOR trial, this study performed an economic evaluation of the addition of daratumumab to Vd in patients with RRMM.MethodsA Markov decision model was used for estimating the long-term costs and efficacy of Vd with or without daratumumab in patients with RRMM. Data on efficacy were taken from the CASTOR trial to compare Vd + daratumumab with Vd. Costs were taken from the US Centers for Medicare & Medicaid Services and from the literature. A series of sensitivity analyses were performed to address the robustness of the model. Variations in the price of daratumumab and subgroup analyses were conducted.FindingsThe base-case analysis showed that adding daratumumab to Vd provided an additional 1.256 quality-adjusted life-years (QALYs) or 1.645 life-years (LYs), with incremental 213,164 USD (163,184 USD) per QALY (LY) gained. Univariate sensitivity analyses suggested that the subsequent treatment cost of DVd and the price of daratumumab had the greatest effect on the incremental cost-effectiveness ratio. According to the variations analysis of the price of daratumumab, the addition of daratumumab would be cost-effective when daratumumab was priced at 70% (30%) of the current price at a willingness-to-pay threshold of 200,000 USD/QALY (150,000 USD/QALY). Subgroup analysis indicated that adding daratumumab to Vd was most cost-effective in patients with 1 prior line of therapy.ImplicationsFrom a US-payer perspective, daratumumab added to Vd in RRMM is likely to exceed the common accepted values of cost-effectiveness     

Assessing the impact of a home-based stroke rehabilitation programme: a cost-effectiveness study

Background: Stroke is often a severe and debilitating event that requires ongoing rehabilitation. The Community Stroke Rehabilitation Teams (CSRTs) offer home-based stroke rehabilitation to individuals for whom further therapy is unavailable or inaccessible. The objective of this study was to evaluate the cost-effectiveness of the CSRT programme compared with a “Usual Care” cohort.

Methods: We collected data on CSRT clients from January 2012 to February 2013. Comparator data were derived from a study of stroke survivors with limited access to specialised stroke rehabilitation. Literature-derived values were used to inform a long-term projection. Using Markov modelling, we projected the model for 35?years in six-month cycles. One-way, two-way, and probabilistic sensitivity analyses were performed. Results were discounted at 3% per year.

Results: Results demonstrated that the CSRT programme has a net monetary benefit (NMB) of $43,655 over Usual Care, and is both less costly and more effective (incremental cost?=??$17,255; incremental effect?=?1.65 Quality Adjusted Life Years [QALYs]). Results of the probabilistic sensitivity analysis revealed that incremental cost-effectiveness of the CSRT programme is superior in 100% of iterations when compared to Usual Care.

Conclusions: The study shows that CSRT model of care is cost-effective, and should be considered when evaluating potential stroke rehabilitation delivery methods.

• Implications for Rehabilitation

• Ongoing rehabilitation following stroke is imperative for optimal recovery.

• Home-based specialised stroke rehabilitation may be an option for individuals for whom ongoing rehabilitation is unavailable or inaccessible.

• The results of this study demonstrated that home-based rehabilitation is a cost-effective means of providing ongoing rehabilitation to individuals who have experienced a stroke.

     

Cost-effectiveness of a one-year coaching program for healthy physical activity in early rheumatoid arthritis

Abstract

Purpose: To describe cost-effectiveness of the Physical Activity in Rheumatoid Arthritis (PARA) study intervention. Method: Costs were collected and estimated retrospectively. Cost-effectiveness was calculated based on the intervention cost per patient with respect to change in health status (EuroQol global visual analog scale – EQ-VAS and EuroQol – EQ-5D) and activity limitation (Health assessment questionnaire – HAQ) using cost-effectiveness- and cost-minimization analyses. Results: Total cost of the one-year intervention program was estimated to be €67?317 or €716 per participant. Estimated difference in total societal cost between the intervention (IG) and control (CG) was €580 per participant. Incremental cost-effectiveness ratio (ICER) for one point (1/100) of improvement in EQ-VAS was estimated to be €116. By offering the intervention to more affected participants in the IG compared to less affected participants, 15.5 extra points of improvement in EQ-VAS and 0.13 points of improvement on HAQ were gained at the same cost. “Ordinary physiotherapy” was most cost-effective with regard to EQ-5D. Conclusions: The intervention resulted in improved effect in health status for the IG with a cost of €116 per extra point in VAS. The intervention was cost-effective if targeted towards a subgroup of more affected patients when evaluating the effect using VAS and HAQ.

• Implications for Rehabilitation

• The physical activity coaching intervention resulted in an improved effect on VAS for the intervention group, to a higher cost.

• In order to maximize cost-effectiveness, this type of physical activity coaching intervention should be targeted towards patients largely affected by their RA.

• The intervention is cost-effective from the patients’ point of view, but not from that of the general population.

     

Cost-effectiveness analysis of early point-of-care lactate testing in the emergency department

PurposeTo determine the cost-effectiveness of implementing a point-of-care (POC) Lactate Program in the emergency department (ED) for patients with suspected sepsis to identify patients who can benefit from early resuscitation.Materials and methodsWe constructed a cost-effectiveness model to examine an ED with 30 000 patients annually. We evaluated a POC lactate program screening patients with suspected sepsis for an elevated lactate ≥4 mmol/L. Those with elevated lactate levels are resuscitated and their lactate clearance is evaluated by serial POC lactate measurements. The POC Lactate Program was compared with a Usual Care Strategy in which all patients with sepsis and an elevated lactate are admitted to the intensive care unit. Costs were estimated from the 2014 Medicare Inpatient and National Physician Fee schedules, and hospital and industry estimates.ResultsIn the base-case, the POC Lactate Program cost $39.53/patient whereas the Usual Care Strategy cost $33.20/patient. The screened patients in the POC arm resulted in 1.07 quality-adjusted life years for an incremental cost-effectiveness ratio of $31 590 per quality-adjusted life year gained, well below accepted willingness-to-pay-thresholds.ConclusionsImplementing a POC Lactate Program for screening ED patients with suspected sepsis is a cost-effective intervention to identify patients responsive to early resuscitation.