Cost-Effectiveness of the Wellness Incentives and Navigation (WIN) Program

ObjectivesPromoting patient involvement in managing co-occurring physical and mental health conditions is increasingly recognized as critical to improving outcomes and controlling costs in this growing chronically ill population. The main objective of this study was to conduct an economic evaluation of the Wellness Incentives and Navigation (WIN) intervention as part of a longitudinal randomized pragmatic clinical trial for chronically ill Texas Medicaid enrollees with co-occurring physical and mental health conditions.MethodsThe WIN intervention used a personal navigator, motivational interviewing, and a flexible wellness expense account to increase patient activation, that is, the patient’s knowledge, skills, and confidence in managing their self-care and co-occurring physical and mental health conditions. Regression models were fit to both participant-level quality-adjusted life years (QALYs) and total costs of care (including the intervention) controlling for demographics, health status, poverty, Medicaid managed care plan, intervention group, and baseline health utility and costs. Incremental costs and QALYs were calculated based on the difference in predicted costs and QALYs under intervention versus usual care and were used to calculate the incremental cost-effectiveness ratios (ICERs). Confidence intervals were calculated using Fieller’s method, and sensitivity analyses were performed.ResultsThe mean ICER for the intervention compared with usual care was $12 511 (95% CI $8971-$16 842), with a sizable majority of participants (70%) having ICERs below $40 000. The WIN intervention also produced higher QALY increases for participants who were sicker at baseline compared to those who were healthier at baseline.ConclusionThe WIN intervention shows considerable promise as a cost-effective intervention in this challenging chronically ill population.     

Cost-Effectiveness of Care Coordination for Children With Chronic Noncomplex Medical Conditions: Results From a Multicenter Randomized Clinical Trial

ObjectivesTo assess the cost-effectiveness of care coordination, compared with standard care, for children with chronic noncomplex medical conditions.MethodsA total of 81 children aged between 2 and 15 years newly diagnosed with a noncomplex chronic condition were randomized to either care coordination or standard care as part of a multicenter randomized controlled trial. Families receiving care coordination were provided access to an Allied Health Liaison Officer, who facilitated family-centered healthcare access across hospital, education, primary care, and community sectors. Costs were estimated over a 12-month period from the perspective of the Australian health system. Health outcomes were valued as quality-adjusted life-years (QALYs). Caregiver productivity costs were included in an alternative base-case analysis, and key assumptions were tested in a series of one-way sensitivity analyses. A probabilistic sensitivity analysis was conducted to investigate the overall impact of uncertainty in the data.ResultsChildren in the intervention arm incurred an average of $17 in additional health system costs (95% confidence interval ?3861 to 1558) and gained an additional 0.031 QALYs (95% confidence interval ?0.29 to 0.092) over 12 months, producing an incremental cost-effectiveness ratio of $548 per QALY. When uncertainty was considered, there was a 73% likelihood that care coordination was cost-effective from a health system perspective, assuming a willingness to pay of $50 000 per QALY. This increased to 78% when caregiver productivity costs were included.ConclusionsCare coordination is likely to be a cost-effective intervention for children with chronic noncomplex medical conditions in the Australian healthcare setting.     

Cost-Effectiveness of a Computerized Provider Order Entry System in Improving Medication Safety Ambulatory Care

BackgroundComputerized provider order entry (CPOE) is the process of entering physician orders directly into an electronic health record. Although CPOE has been shown to improve medication safety and reduce health care costs, these improvements have been demonstrated largely in the inpatient setting; the cost-effectiveness in the ambulatory setting remains uncertain.ObjectiveThe objective was to estimate the cost-effectiveness of CPOE in reducing medication errors and adverse drug events (ADEs) in the ambulatory setting.MethodsWe created a decision-analytic model to estimate the cost-effectiveness of CPOE in a midsized (400 providers) multidisciplinary medical group over a 5-year time horizon— 2010 to 2014—the time frame during which health systems are implementing CPOE to meet Meaningful Use criteria. We adopted the medical group’s perspective and utilized their costs, changes in efficiency, and actual number of medication errors and ADEs. One-way and probabilistic sensitivity analyses were conducted. Scenario analyses were explored.ResultsIn the base case, CPOE dominated paper prescribing, that is, CPOE cost $18 million less than paper prescribing, and was associated with 1.5 million and 14,500 fewer medication errors and ADEs, respectively, over 5 years. In the scenario that reflected a practice group of five providers, CPOE cost $265,000 less than paper prescribing, was associated with 3875 and 39 fewer medication errors and ADEs, respectively, over 5 years, and was dominant in 80% of the simulations.ConclusionsOur model suggests that the adoption of CPOE in the ambulatory setting provides excellent value for the investment, and is a cost-effective strategy to improve medication safety over a wide range of practice sizes.     

Moving Beyond Quality-Adjusted Life-Years in Elderly Care: How Can Multicriteria Decision Analysis Complement Cost-Effectiveness Analysis in Local-Level Decision Making

ObjectivesThis study aimed to investigate how multicriteria decision analysis (MCDA) could complement cost-effectiveness analysis (CEA) to support investment decisions in elderly care at local level.MethodsWe used an integrated elderly care program in The Netherlands as a case study to demonstrate the application of both methods. In a 12-month quasi-experimental study (n = 384), data on the following outcome measures were collected: quality-adjusted life-years (CEA) and physical functioning, psychological well-being, social relationships and participation, enjoyment of life, resilience, person centeredness, continuity of care, and costs (MCDA). We performed regression analysis on inversed probability weighted data and controlled for potential confounders to obtain a double robust estimate of the outcomes. Probabilistic sensitivity analyses determined uncertainty for both methods.ResultsThe integrated elderly care program was not likely (ie, 36%) to be cost-effective according to the CEA (incremental cost-effectiveness ratios: €88 249 from a societal perspective) using the conventional Dutch willingness-to-pay threshold (ie, €50 000). The MCDA demonstrated that informal caregivers and professionals slightly preferred the intervention over usual care, driven by enjoyment of life and person centeredness. Patients did not prefer either the intervention or usual care, whereas payers and policy makers slightly preferred usual care, mainly due to higher costs of the intervention.ConclusionsMCDA could provide local-level decision makers with a broader measurement of effectiveness by including outcomes beyond health and longevity and the preferences of multiple stakeholders. This additional information could foster the acceptability and implementability of cost-effective innovations in elderly care.     

Is Dietetic Treatment for Undernutrition in Older Individuals in Primary Care Cost-Effective?

ObjectivesUndernutrition in older age is associated with adverse clinical outcomes and high health care costs. This study aimed to evaluate the cost-effectiveness of a dietetic treatment in primary care compared with usual care in older, undernourished, community-dwelling individuals.DesignA parallel randomized controlled trial.SettingPrimary care.ParticipantsA total of 146 undernourished, independently living older (≥65 years) individuals.InterventionDietetic treatment.MeasurementsMain outcomes were change in kilogram body weight compared with baseline and quality-adjusted life years (QALYs) after 6 months. Costs were measured from a societal perspective. The main analysis was performed according to the intention-to-treat principle. Multiple imputation was used to impute missing data and bootstrapping was used to estimate uncertainty surrounding cost differences and incremental cost-effectiveness ratios. Cost-effectiveness planes and cost-effectiveness acceptability curves were estimated.ResultsThe participants were randomized to receive either dietetic treatment (n = 72) or usual care (n = 74). After 6 months, no statistically significant differences were found between the dietetic treatment and usual care group in body weight change (mean difference 0.78 kg, 95% CI −0.26–1.82), QALYs (mean difference 0.001, 95% CI −0.04–0.04) and total costs (mean difference €1645, 95% CI −525–3547). The incremental cost-utility ratio (ICUR) for QALYs was not interpretable. The incremental cost-effectiveness ratio (ICER) for body weight gain was 2111. The probability that dietetic treatment is cost-effective compared with usual care was 0.78 for a ceiling ratio of €5000 for body weight and 0.06 for a ceiling ratio of €20.000 for QALY.ConclusionIn this study, dietetic treatment in older, undernourished, community-dwelling individuals was not cost-effective compared with usual care.     

Cost-effectiveness analysis of a surveillance program to prevent hip dislocation in children with cerebral palsy

ObjectiveIn this study we conducted an economic evaluation of a surveillance programme to prevent hip dislocation in children with cerebral palsy.MethodWe developed a model that compared costs and health outcomes of children with cerebral palsy with and without a surveillance programme. Information from a number of sources was combined into a decision analytical model, primarily based on data from a comparative study with a 20-year follow-up. Effectiveness was measured using Quality-Adjusted Life Years (QALYs). The analysis took the perspective of the Spanish National Health Service. We undertook extensive sensitivity analyses including a probabilistic sensitivity analysis.ResultsThe surveillance programme led to higher QALYs and higher health care costs, with an estimated incremental cost per QALY gained of 12,282€. The results were robust to model assumptions. The probability that the programme was cost-effective was estimated to be over 80% at the threshold of 25.000€/QALY recommended in Spain.ConclusionThis study indicates that surveillance programmes to prevent hip dislocation in children with cerebral palsy are likely to be cost-effective.     

Revisión de la medicación en ancianos polimedicados en riesgo vascular: ensayo aleatorizado y controlado

ObjectiveTo analyse the effectiveness of a medication review based on intervention directed at improving the appropriateness of drug treatments according to the established guidelines, as well as blood pressure, serum lipid and blood glucose control in elderly patients on multiple medication, and cardiovascular disease or high risk of cardiovascular disease.DesignA randomised controlled trial with blind evaluation.SettingFourteen Primary Health Care centres in AndalusiaParticipantsA total of 323 patients older than 65 on polypharmacy and cardiovascular disease or high risk of cardiovascular disease.InterventionA pharmacist interviewed the patient, reviewed the appropriateness of the drug treatment, taking in account health record data, proposed modifications and communicated them to the general practitioner or nurse. The control group received usual health care.Main measurementsPercentage of patients with appropriate use of low doses of acetylsalicylic acid, blood pressure, LDL-cholesterol, HbA_1c, and quality of life scores.ResultsA total of 41% of patients (average age 74, 61% women) had cardiovascular disease. Ten months after the intervention (18.3% withdrawals), more patients in the intervention group used low dose acetylsalicylic acid than in the control group (52.3% vs 38.6%; P=.024). There were no differences between groups in intermediate clinic outcomes. Quality of life scores improve in intervention group by 6.1 points (100 points scale), but was not statistically significant (P=.051).ConclusionClinical medication review improves the appropriateness of antiplatelet treatment in the elderly on polypharmacy and with high risk of cardiovascular disease. No improvement in biochemistry measurements was found.     

Cost-Effectiveness of Magnetic Resonance Imaging-Guided Thrombolysis for Patients With Stroke With Unknown Time of Onset

ObjectivesPatients waking up with stroke symptoms are often excluded from intravenous thrombolysis with alteplase (IV-tpa). The WAKE-UP trial, a European multicenter randomized controlled trial, proved the clinical effectiveness of magnetic resonance imaging-guided IV-tpa for these patients. This analysis aimed to assess the cost-effectiveness of the intervention compared to placebo.MethodsA Markov model was designed to analyze the cost-effectiveness over a 25-year time horizon. The model consisted of an inpatient acute care phase and a rest-of-life phase. Health states were defined by the modified Rankin Scale (mRS). Initial transition probabilities to mRS scores were based on WAKE-UP data and health state utilities on literature search. Costs were based on data from the University Medical Center Hamburg-Eppendorf, literature, and expert opinion. Incremental costs and effects over the patients’ lifetime were estimated. The analysis was conducted from a formal German healthcare perspective. Univariate and probabilistic sensitivity analyses were performed.ResultsTreatment with IV-tpa resulted in cost savings of €51 009 and 1.30 incremental gains in quality-adjusted life-years at a 5% discount rate. Univariate sensitivity analysis revealed incremental cost-effectiveness ratio being sensitive to the relative risk of favorable outcome on mRS for placebo patients after stroke, the costs of long-term care for patients with mRS 4, and patient age at initial stroke event. In all cases, IV-tpa remained cost-effective. Probabilistic sensitivity analysis proved IV-tpa cost-effective in >95% of the simulations results.ConclusionsMagnetic resonance imaging-guided IV-tpa compared to placebo is cost-effective in patients with ischemic stroke with unknown time of onset.     

Economic Evaluations of Remote Patient Monitoring for Chronic Disease: A Systematic Review

ObjectivesThis study aimed to systematically review and summarize economic evaluations of noninvasive remote patient monitoring (RPM) for chronic diseases compared with usual care.MethodsA systematic literature search identified economic evaluations of RPM for chronic diseases, compared with usual care. Searches of PubMed, Embase, CINAHL, and EconLit using keyword synonyms for RPM and economics identified articles published from up until September 2021. Title, abstract, and full-text reviews were conducted. Data extraction of study characteristics and health economic findings was performed. Article reporting quality was assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist.ResultsThis review demonstrated that the cost-effectiveness of RPM was dependent on clinical context, capital investment, organizational processes, and willingness to pay in each specific setting. RPM was found to be highly cost-effective for hypertension and may be cost-effective for heart failure and chronic obstructive pulmonary disease. There were few studies that investigated RPM for diabetes or other chronic diseases. Studies were of high reporting quality, with an average Consolidated Health Economic Evaluation Reporting Standards score of 81%. Of the final 34 included studies, most were conducted from the healthcare system perspective. Eighteen studies used cost-utility analysis, 4 used cost-effectiveness analysis, 2 combined cost-utility analysis and a cost-effectiveness analysis, 1 used cost-consequence analysis, 1 used cost-benefit analysis, and 8 used cost-minimization analysis.ConclusionsRPM was highly cost-effective for hypertension and may achieve greater long-term cost savings from the prevention of high-cost health events. For chronic obstructive pulmonary disease and heart failure, cost-effectiveness findings differed according to disease severity and there was limited economic evidence for diabetes interventions.     

Is ultra-hypo-fractionated radiotherapy more cost-effective relative to conventional fractionation in treatment of prostate cancer? A cost–utility analysis alongside a randomized HYPO-RT-PC trial

Background

Economic evidence for comparing low fraction with ultra-hypo fractionated (UHF) radiation therapy in the treatment of intermediate-to-high-risk prostate cancer (PC) is lacking, especially in Europe. This study presents an economic evaluation performed alongside an ongoing clinical trial.

Aim

To investigate up to 6 years’ follow-up whether conventional fractionation (CF, 78.0 Gy in 39 fractions, 5 days per week for 8 weeks) is more cost-effective than UHF (42.7 Gy in 7 fractions, 3 days per week for 2.5 weeks inclusive of 2 weekends) radiotherapy in treatment for patients with intermediate-to-high-risk PC.

Method

HYPO-RT-PC trial is an open-label, randomized, multicenter (10 in Sweden; 2 in Denmark) phase-3 trial. Patients from Sweden (CF 434; UHF 445) were included in this study. The trial database was linked to the National Patient Registry (NPR). Costs for inpatient/non-primary outpatient care for each episode were retrieved. For calculating Quality-adjusted life years (QALYs), the EORTC QLQ-C30 questionnaire was mapped to the EQ-5D-3L index. Multivariable regression analyses were used to compare the difference in costs and QALYs, adjusting for age and baseline costs, and health status. The confidence interval for the difference in costs, QALYs and incremental cost-effectiveness ratio effectiveness ratio (ICER) was estimated by the bootstrap percentile method.

Results

No significant differences were found in ICER between the two arms after 6 years of follow-up.

Conclusion

The current study did not support that the ultra-hypo-fractionated treatment was more cost-effective than the conventional fraction treatment up to the sixth year of the trial.

     

Single food focus dietary guidance: lessons learned from an economic analysis of egg consumption

Background

Schizophrenia is a severe, chronic, and costly illness that adversely impacts patients' lives and health care payer budgets. Cost comparisons of treatment regimens are, therefore, important to health care payers and researchers. Pre-Post analyses ("mirror-image"), where outcomes prior to a medication switch are compared to outcomes post-switch, are commonly used in such research. However, medication changes often occur during a costly crisis event. Patients may relapse, be hospitalized, have a medication change, and then spend a period of time with intense use of costly resources (post-medication switch). While many advantages and disadvantages of Pre-Post methodology have been discussed, issues regarding the attributability of costs incurred around the time of medication switching have not been fully investigated.

Methods

Medical resource use data, including medications and acute-care services (hospitalizations, partial hospitalizations, emergency department) were collected for patients with schizophrenia who switched antipsychotics (n = 105) during a 1-year randomized, naturalistic, antipsychotic cost-effectiveness schizophrenia trial. Within-patient changes in total costs per day were computed during the pre- and post-medication change periods. In addition to the standard Pre-Post analysis comparing costs pre- and post-medication change, we investigated the sensitivity of results to varying assumptions regarding the attributability of acute care service costs occurring just after a medication switch that were likely due to initial medication failure.

Results

Fifty-six percent of all costs incurred during the first week on the newly initiated antipsychotic were likely due to treatment failure with the previous antipsychotic. Standard analyses suggested an average increase in cost-per-day for each patient of $2.40 after switching medications. However, sensitivity analyses removing costs incurred post-switch that were potentially due to the failure of the initial medication suggested decreases in costs in the range of $4.77 to $9.69 per day post-switch.

Conclusion

Pre-Post cost analyses are sensitive to the approach used to handle acute-service costs occurring just after a medication change. Given the importance of quality economic research on the cost of switching treatments, thorough sensitivity analyses should be performed to identify the impact of crisis events around the time of medication change.     

Cost-Effectiveness of Providing the Depression Care for People With Cancer Program to Patients With Prostate Cancer in the United States

ObjectivesThe Depression Care for People with Cancer program (DCPC) is a cost-effective depression care model for UK patients with cancer. However, DCPC’s cost-effectiveness in the United States is unknown, particularly for patients with prostate cancer in the United States. This study evaluates the health and economic impact of providing DCPC to patients with prostate cancer.MethodsDCPC was compared with usual care in a mathematical model that simulates depression and its outcomes in a hypothetical cohort of US patients with prostate cancer. DCPC was modeled as a sequential combination of universal depression screening, post-screening evaluations, and first-line combination therapy. Primary outcomes were lifetime direct costs of depression care, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios. Secondary outcomes included life expectancy, number of depression-free months and lifetime depressive episodes, duration of depressive episodes, cumulative incidence of depression, lifetime depression diagnoses/misdiagnoses, and the cumulative incidence of maintenance therapy for depression. Sensitivity analyses were used to examine uncertainty.ResultsIn the base case, DCPC dominated usual care by offering 0.11 more QALYs for $2500 less per patient (from averted misdiagnoses). DCPC also offered 5 extra depression-free months, shorter depressive episodes, and a lower chance of maintenance therapy. DCPC’s trade-offs were a higher cumulative incidence of depression and more lifetime depressive episodes. Life expectancy was identical under usual care and DCPC. Sensitivity analyses indicate that DCPC was almost always preferable to usual care.ConclusionCompared with usual care, DCPC may offer more value to US patients with prostate cancer. DCPC should be considered for inclusion in prostate cancer survivorship care guidelines.     

Economic evaluation of return-to-work interventions for mental disorder-related sickness absence: two years follow-up of a randomized clinical trial

ObjectiveThe objective was to (i) assess the long-term cost-effectiveness of acceptance and commitment therapy (ACT), a workplace dialog intervention (WDI), and ACT+WDI compared to treatment as usual (TAU) for common mental disorders and (ii) investigate any differences in cost-effectiveness between diagnostic groups.MethodsAn economic evaluation from the healthcare and limited welfare perspectives was conducted alongside a randomized clinical trial with a two-year follow-up period. Persons with common mental disorders receiving sickness benefits were invited to the trial. We used registry data for cost analysis alongside participant data collected during the trial and the reduction in sickness absence days as treatment effect. A total of 264 participants with a diagnosis of depression, anxiety, or stress-induced exhaustion disorder participated in a two-year follow-up of a four-arm trial: ACT (N=74), WDI (N=60), ACT+WDI (N=70), and TAU (N=60).ResultsFor all patients in general, there were no statistically significant differences between interventions in terms of costs or effect. The subgroup analyses suggested that from a healthcare perspective, ACT was a cost-effective option for depression or anxiety disorders and ACT+WDI for stress-induced exhaustion disorder. With a two-year time horizon, the probability of WDI to be cost-saving in terms of sickness benefits costs was 80% compared with TAU.ConclusionsACT had a high probability of cost-effectiveness from a healthcare perspective for employees on sick leave due to depression or anxiety disorders. For participants with stress-induced exhaustion disorder, adding WDI to ACT seems to reduce healthcare costs, while WDI as a stand-alone intervention seems to reduce welfare costs.     

Cost-effectiveness of the MitraClip device in German heart failure patients with secondary mitral regurgitation

Aim

To evaluate the cost-effectiveness of the MitraClip device (MitraClip) in addition to optimal medical therapy (OMT) in patients with heart failure and secondary mitral regurgitation in Germany.

Methods and results

A model-based economic evaluation was performed to estimate the incremental cost per quality-adjusted life-years (QALYs) for patients with a moderate-to-severe or severe secondary mitral regurgitation receiving MitraClip plus OMT compared with OMT alone from the statutory health insurance (SHI) perspective. Transition probabilities, data on survival rates, and hospitalization rates were obtained from the COAPT trial, a randomized-controlled multicenter trial. Data on health utility and costs were taken from published evidence. To assess parameter uncertainty, several deterministic and probabilistic sensitivity analyses were performed. The incremental costs per QALY gained were € 59,728 (costs/incremental life years gained: € 42,360). The results were most sensitive to the transition probabilities and the hospitalization rates. The probabilistic sensitivity analysis showed that the MitraClip strategy was cost-effective with a probability of 80% at a willingness-to-pay threshold of € 67,000/QALY.

Conclusions

Depending on the willingness-to-pay threshold, for patients with heart failure and a moderate-to-severe or severe secondary mitral regurgitation the MitraClip can be cost-effective from the perspective of the German SHI.

Graphical abstract

     

Impact of Adding Oseltamivir to Usual Care on Quality-Adjusted Life-Years During Influenza-Like Illness

ObjectivesThe ALIC⁴E trial has shown that oseltamivir reduces recovery time while increasing the risk of nausea. This secondary analysis of the ALIC⁴E trial aimed to determine the gain in quality-adjusted life-years (QALYs) associated with adding oseltamivir to usual primary care in patients presenting with influenza-like illness (ILI).MethodsPatients with ILI were recruited during the influenza season (2015-2018) in 15 European countries. Patients were assigned to usual care with or without oseltamivir through stratified randomization (age, severity, comorbidities, and symptom onset). Patients’ health status was valued with the EQ-5D and visual analog scale (VAS) for up to 28 days. Average EQ-5D and VAS scores over time were estimated for both treatment groups using one-inflated beta regression in children (<13 years old) and adults (≥13 years old). QALY gain was calculated as the difference between the groups. Sensitivity analysis considered the value set to convert EQ-5D answers to summary scores and the follow-up period.ResultsIn adults, oseltamivir gained 0.0006 (95% confidence interval 0.0002-0.0010) QALYs, whereas no statistically significant gain was found in children (14-day follow-up, EQ-5D). QALY gains were statistically significant in patients aged ≥65 years, patients without relevant comorbidities, or patients experiencing symptoms for ≤48 hours. Using VAS and accounting for 28-day follow-up resulted in higher QALY gain.ConclusionsQALY gain owing to oseltamivir is limited compared with other diseases, and its clinical meaningfulness remains to be determined. Further analysis is needed to evaluate whether QALY gain and its impact on ILI treatment cost render oseltamivir cost-effective.     

Value of Triage Treatment Strategies to Distribute Hepatitis C Direct-Acting Antiviral Agents in an Integrated Healthcare System: A Cost-Effectiveness Analysis

ObjectivesThis study aimed to assess the cost-effectiveness of fibrosis-based direct-acting antiviral treatment policies for patients with chronic hepatitis C virus at the Kaiser Permanente Mid-Atlantic States health system.MethodsWe used a Markov model to compare the lifetime costs and effects of treating patients with chronic hepatitis C virus at different stages of disease severity, or all stages simultaneously, based on a fibrosis score from the US healthcare sector perspective and societal perspective. The initial distribution of patients across fibrosis scores, the effectiveness of direct-acting antiviral therapy, and follow-up and monitoring protocols were specific to the Kaiser Permanente Mid-Atlantic States health system. Direct and indirect costs, transition probabilities, and utilities were derived from the literature. Deterministic and probabilistic sensitivity analyses were performed to assess the robustness of our results.ResultsThe “Treat All” option was dominant from both the societal and healthcare sector perspectives. The conclusion was robust in deterministic sensitivity analysis. The range of incremental costs between the less restrictive policies was small—the difference between the “Treat F1+” and the “Treat All” option was only $111 per person. Probabilistic sensitivity analyses showed, at both the $100 000/quality-adjusted life-year and $150 000/quality-adjusted life-year thresholds, there was a 70% chance that the “Treat All” option was more cost-effective than the “Treat F1+” option.ConclusionsWe found that expanded treatment access is cost-effective and, in many cases, cost saving. Although our results are primarily applicable to a regional integrated healthcare system, it offers some direction to any healthcare setting faced with resource constraints in the face of highly priced drugs.     

Modeling the potential efficiency of a blood biomarker-based tool to guide pre-hospital thrombolytic therapy in stroke patients

Objectives

Stroke treatment with intravenous tissue-type plasminogen activator (tPA) is effective and efficient, but as its benefits are highly time dependent, it is essential to treat the patient promptly after symptom onset. This study evaluates the cost-effectiveness of a blood biomarker test to differentiate ischemic and hemorrhagic stroke to guide pre-hospital treatment with tPA in patients with suspected stroke, compared with standard hospital management. The standard care for patients suffering stroke consists mainly in diagnosis, treatment, hospitalization and monitoring.

Methods

A Markov model was built with four health states according to the modified Rankin scale, in adult patients with suspected moderate to severe stroke (NIHSS 4-22) within 4.5 hours after symptom onset. A Spanish Health System perspective was used. The time horizon was 15 years. Quality-adjusted life-years (QALYs) and life-years gained (LYGs) were used as a measure of effectiveness. Short- and long-term direct health costs were included. Costs were expressed in Euros (2022). A discount rate of 3% was used. Probabilistic sensitivity analysis and several one-way sensitivity analyses were conducted.

Results

The use of a blood-test biomarker compared with standard care was associated with more QALYs (4.87 vs. 4.77), more LYGs (7.18 vs. 7.07), and greater costs (12,807€ vs. 12,713€). The ICER was 881€/QALY. Probabilistic sensitivity analysis showed that the biomarker test was cost-effective in 82% of iterations using a threshold of 24,000€/QALY.

Conclusions

The use of a blood biomarker test to guide pre-hospital thrombolysis is cost-effective compared with standard hospital care in patients with ischemic stroke.

     

The economic burden of pulmonary arterial hypertension (PAH) in the US on payers and patients

Background

Pulmonary arterial hypertension (PAH) is a rare condition that can ultimately lead to right heart failure and death. In this study we estimated the health care costs and resource utilization associated with PAH in a large US managed care health plan.

Methods

Subjects with claims-based evidence of PAH from 1/1/2004 to 6/30/2010 (identification period) were selected. To be included in the final PAH study sample, subjects were required to have ≥2 claims with a primary PH diagnosis; ≥2 claims with a PAH related-diagnosis (connective tissue diseases, congenital heart diseases, portal hypertension); and ≥1 claim with evidence of a PAH-indicated medication. The earliest date of a claim with evidence of PAH-indicated medication during the identification period was set as the index date. Health care costs and resource utilization were compared between an annualized baseline period and a 12 month follow-up period.

Results

504 PAH subjects were selected for the final study cohort. Estimated average total health care costs were approximately 16% lower in the follow-up period compared to the baseline period (follow-up costs?=?$98,243 [SD?=?110,615] vs. baseline costs?=?$116,681 [SD?=?368,094], p?<?0.001), but substantively high in each period relative to costs reported for other chronic diseases. Pharmacy costs were significantly higher in the follow-up period vs. the baseline period, ($38,514 [SD?=?34,817] vs. $6,440 [SD?=?12,186], p?<?0.001) but medical costs were significantly lower in the follow-up vs. baseline ($59,729 [SD?=?106,683] vs. $110,241 [SD?=?368,725], p?<?0.001). These costs were mirrored in health-care resource utilization estimates. The average counts of ambulatory visits and inpatient stays were lower in the follow-up vs. the baseline (both p?<?0.001). Results varied in exploratory analyses when less restrictive subject identification algorithms were used.

Conclusions

Subjects with evidence of PAH had substantively high health care costs. Medical costs appeared to decrease following PAH medication use, but with a concomitant increase in pharmacy costs.

     

Assessing the Value of Cemiplimab for Adults With Advanced Cutaneous Squamous Cell Carcinoma: A Cost-Effectiveness Analysis

ObjectivesTo evaluate the cost-effectiveness of cemiplimab in patients with advanced cutaneous squamous cell carcinoma (CSCC) from a payer perspective in the United States.MethodsA partitioned survival model was developed to assess the cost-effectiveness of cemiplimab versus historical standard of care (SOC). All inputs were identified based on a systematic literature review, supplemented by expert opinion where necessary. Clinical inputs for cemiplimab were based on individual patient data from a cemiplimab phase 2 single-arm trial (NCT27060498). For SOC, analysis was based on a pooled analysis of single-arm clinical trials and retrospective studies evaluating chemotherapy and epidermal growth factor receptor inhibitors (cetuximab, erlotinib, and gefitinib) identified via a systematic literature review (6 of the 27 included studies). Overall survival and progression-free survival were extrapolated over a lifetime horizon. Costs were included for drug acquisition, drug administration, management of adverse events, subsequent therapy, disease management, and terminal care. Unit costs were based on published 2019 US list prices.ResultsIn the base case, cemiplimab versus SOC resulted in an incremental cost-effectiveness ratio of $99 447 per quality adjusted-life year (QALY), where incremental costs and QALYs were $372 108 and 3.74, respectively. At a willingness-to-pay threshold of $150 000/QALY, the probabilistic sensitivity analysis suggests a 90% probability that cemiplimab is cost-effective compared to SOC. Scenario analyses resulted in incremental cost-effectiveness ratios ranging from $90 590 to $148 738.ConclusionsCompared with historical SOC, cemiplimab is a cost-effective use of US payer resources for the treatment of advanced CSCC and is expected to provide value for money.     

Cost-Effectiveness of Mirabegron Compared with Antimuscarinic Agents for the Treatment of Adults with Overactive Bladder in the United Kingdom

BackgroundMirabegron, a first-in-class selective oral β₃-adrenoceptor agonist, has similar efficacy to most antimuscarinic agents and a lower incidence of dry mouth in patients with overactive bladder (OAB).ObjectivesTo evaluate the cost-effectiveness of mirabegron 50 mg compared with oral antimuscarinic agents in adults with OAB from a UK National Health Service perspective.MethodsA Markov model including health states for symptom severity, treatment status, and adverse events was developed. Cycle length was 1 month, and the time horizon was 5 years. Antimuscarinic comparators were tolterodine extended release, solifenacin, fesoterodine, oxybutynin extended release and immediate release (IR), darifenacin, and trospium chloride modified release. Transition probabilities for symptom severity levels and adverse events were estimated from a mirabegron trial and a mixed treatment comparison. Estimates for other inputs were obtained from published literature or expert opinion. Quality-adjusted life-years (QALYs) and total health care costs, including costs of drug acquisition, physician visits, incontinence pad use, and botox injections, were modeled. Deterministic and probabilistic sensitivity analyses were performed.ResultsBase-case incremental cost-effectiveness ratios ranged from £367 (vs. solifenacin 10 mg) to £15,593 (vs. oxybutynin IR 10 mg) per QALY gained. Probabilistic sensitivity analyses showed that at a willingness-to-pay threshold of £20,000/QALY gained, the probability of mirabegron 50 mg being cost-effective ranged from 70.2% versus oxybutynin IR 10 mg to 97.8% versus darifenacin 15 mg. A limitation of our analysis is the uncertainty due to the lack of direct comparisons of mirabegron with other agents; a mixed treatment comparison using rigorous methodology provided the data for the analysis, but the studies involved showed heterogeneity.ConclusionsMirabegron 50 mg appears to be cost-effective compared with standard oral antimuscarinic agents for the treatment of adults with OAB from a UK National Health Service perspective.