Incremental Quality-adjusted Life Years Research Articles

Cost-effectiveness analysis of intravascular ultrasound-guided peripheral vascular interventions in patients with femoropopliteal peripheral artery disease.

Background: Intravascular ultrasound (IVUS)-guided percutaneous transluminal angioplasty (PTA) might offer clinical benefits compared to angiography-guided PTA in patients with peripheral artery disease (PAD). A cost-effectiveness model was developed to examine the benefits and costs of IVUS-guided PTA versus angiography-guided PTA in PAD patients with femoropopliteal (FP) occlusive disease. Methods: A two-step model (a one-year decision tree followed by a lifetime semi-Markov model) was developed from a German healthcare payer perspective to estimate the costs and outcomes over a one-year and lifetime horizon. Clinical events included target lesion revascularization (TLR), amputation, and death. Transition probabilities and utility values were derived from published literature. Healthcare costs were based on German Diagnosis Related Groups (DRG) codes. Costs and outcomes were discounted at a rate of 3% per year. The incremental cost-effectiveness ratio (ICER) was calculated, and sensitivity analyses were performed to assess the robustness of the results. Results: In the one-year horizon, IVUS-guided PTA resulted in incremental quality-adjusted life-years (QALY) and costs of 0.02 and €919 per patient respectively, with a corresponding ICER of €45,195/QALY gained versus angiography-guided PTA. In the lifetime horizon, IVUS-guided PTA outperforms angiography-guided PTA; it was associated with a cost saving of €46 per patient and incremental QALY of 0.22. Utility value for post-TLR, as well as probabilities of death and TLR had the greatest impact on the one-year ICER, while cost of TLR and probabilities of TLR and amputation influenced the lifetime ICER most. The probability of IVUS-guided PTA being cost-effective at a willingness-to-pay (WTP) threshold of €50,000/QALY was 50.4% in the one-year horizon and increased to 85.9% in the lifetime horizon. Conclusions: In this analysis IVUS-guided PTA among patients with symptomatic FP atherosclerosis was cost-saving in a lifetime horizon from the German healthcare payer perspective.

Cost-Effectiveness Analysis of Maribavir in Patients with Post-Transplant Cytomegalovirus Infection or Disease that are Refractory or Resistant to Conventional Therapy

OBJECTIVES: Cytomegalovirus (CMV) infection represents a serious post-transplantation complication, particularly among transplant recipients with resistant or refractory (R/R) infection. Despite the improvement made in CMV prevention and treatment, remains a need for new therapies that are better tolerated and safer for patients, especially for refractory or resistant (R/R) infections. This analysis investigates the cost-effectiveness of maribavir versus investigator-assigned therapy (IAT) for treatment of post-transplant R/R CMV in the Italian context.METHODS: The analysis was conducted from the National Health Service (NHS) perspective using a Markov model previously developed in Excel® to evaluate the cost-effectiveness of maribavir versus IAT for the treatment of post-transplant R/R CMV. Health event rates and transition probabilities were informed by data from observational studies and from SOLSTICE clinical trial, which compared maribavir to conventional antiviral treatments for transplant patients with R/R CMV infections. Utilities were from SOLSTICE and a utility vignette study. Costs inputs were derived from published literature and Italian national tariffs for outpatient and hospital services. Costs and outcomes were discounted by 3%.RESULTS: Maribavir compared to the IAT used for the treatment of post-transplant R/R CMV achieved an incremental cost of € 11,455 for an incremental quality adjusted life year (QALY) gain of 0.313. This resulted in an incremental cost-effectiveness ratio of € 36,626/QALY, for maribavir compared to IAT, which is below the generally accepted, national willingness-to-pay threshold for orphan drugs (€ 37,000/QALY).CONCLUSIONS: This analysis showed that maribavir, an orphan drug treatment, is cost-effective compared to the current antiviral treatments used for treatment of post-transplant R/R CMV.

Cost-effectiveness of spinal muscular atrophy newborn screening based on real-world data in Belgium

The objective of the study was to assess the cost-effectiveness of real-world spinal muscular atrophy newborn screening followed by treatment. We modeled the lifetime cost-effectiveness of the spinal muscular atrophy newborn screening followed by treatment (screening) compared to treatment without screening (no screening) from the Belgian healthcare perspective. Real-world data, including quality of life, costs, and motor development data, were collected on 12 patients identified by screening and 43 patients identified by their symptoms. “Screening” was associated with slightly higher healthcare costs (€ 6,858,061 vs. € 6,738,120) but more quality-adjusted life years (QALY) (40.95 vs. 20.34) compared to “no screening”, leading to an incremental cost-effectiveness ratio of € 5,820 per QALY gained. “Screening” was dominant from a societal perspective (negative incremental costs: € -14,457; incremental QALY = 20.61), when incorporating the burden on caregivers (negative incremental costs = € -74,353; incremental QALY = 27.51), and when the treatment was chosen by the parents (negative incremental costs = € -2,596,748; incremental QALY = 20.61). Spinal muscular atrophy newborn screening coupled with early treatment is thus cost-effective compared with late treatment following clinical diagnosis and is dominant when societal perspective, caregiver burden, and treatment based on parental preference were considered.

Cost‑Effectiveness Analysis of Tapentadol PR Versus Oxycodone/Naloxone PR in Patients with Musculoskeletal Pain in Spain

Objetive: Chronic musculoskeletal pain is highly prevalent worldwide and is associated with a huge social and economic burden. Opioids are commonly prescribed to treat chronic pain as recommended by the European Pain Federation. Tapentadol prolonged release (PR), is a strong opioid that has proven being effective in treating chronic musculoskeletal pain with less adverse events than oxycodone/naloxone PR. The aim of this study was to estimate the cost-effectiveness of tapentadol PR compared to oxycodone/naloxone PR from the Spanish National Healthcare System perspective (SNHS). Methods: A Markov model was adapted analysing two arms: patients treated with tapentadol PR and patients treated with oxycodone/naloxone PR. The model assesses a cohort of 1,000 patients over a one-year period. Results: Overall, treating patients with tapentadol PR was less costly than treating patients with oxycodone/naloxone PR (€388,631.70 vs €568,168.85). In terms of cost effectiveness, the incremental cost of tapentadol PR over oxycodone/naloxone PR was -€179,537.15 and the incremental quality-adjusted life year value was 40.05. The incremental cost-effectiveness ratio was estimated at -€4,027.16 per quality-adjusted life year suggesting that tapentadol PR is an economically dominant alternative over oxycodone/naloxone PR. Conclusion: Tapentadol PR represents a cost-effective option to treat musculoskeletal chronic pain patients in Spain. Keywords: Musculoskeletal pain, Opioids, Tapentadol PR, Cost effectiveness, Spain.

The Cost-Effectiveness of Chin Tuck Against Resistance Compared to Usual Care in Citizens with Oropharyngeal Dysphagia - An Economic Evaluation.

This study aims to evaluate the cost-effectiveness of chin tuck against resistance (CTAR) for citizens suffering from dysphagia compared to the standard municipal treatment in Denmark. A cost-utility analysis, employing a municipal perspective, was conducted using cost data collected alongside clinical data of a randomized controlled trial evaluating the effect of CTAR training for citizens with dysphagia. The composition of the clinical randomized controlled trial, which included citizens with different diagnoses, means that participants had different disease courses. Ninety-two citizens from seven different Danish municipalities were enrolled, of whom 43 received standard care, and 49 received CTAR in addition to standard care. The effect outcome of the economic evaluation was quality-adjusted life years (QALY), estimated using the EQ-5D-5L questionnaire. Individual resource consumption of each citizen was determined based on the use of home care, home nursing care, physio- and occupational therapy, dietitian guidance, and hospital admissions. The incremental costs and QALYs between the intervention group (CTAR in addition to standard care) and standard care group were estimated using regression analysis, and sensitivity analyses were performed to investigate the robustness of the results. The base case analysis showed that the intervention group was dominant compared to the standard care group, with a decrease in incremental costs of £542.38 and an increase in incremental QALYs of 0.0118. All sensitivity analyses demonstrated similar findings as the base case analysis, supporting the robustness of the results. This study found that the intervention group was the dominant alternative, hence being more effective and cost-saving, compared to the standard care group in a Danish municipality perspective with a three-month time horizon. This study adds to the scarce evidence on the cost-effectiveness of CTAR in a Danish clinical setting, but further studies should focus on estimating long-term cost-effectiveness.

Economic evaluation of serplulimab plus chemotherapy as the first-line treatment of oesophageal squamous cell carcinoma in China

ObjectiveThe ASTRUM-007 study confirmed the significant efficacy and safety of serplulimab plus chemotherapy for patients with locally advanced/metastatic, programmed cell death-ligand 1 positive oesophageal squamous cell carcinoma (OSCC). The economics...

The potential cost‐effectiveness of roflumilast drug treatment in mild cognitive impairment

AbstractBackgroundEvidence is needed on the value of new (pharmacological) health technologies in their early stage of development, to support stakeholders (industry, regulators, payers, and health care providers) in decision‐making linked to their further development and introduction in routine care. Early health‐technology assessment supports stakeholders by providing insight into the size and uncertainty of the expected benefits new technologies may have. This can facilitate early guidance on positioning in clinical practice and research recommendations. Our objective was to provide a pragmatic example of the potential cost‐effectiveness of roflumilast treatment in addition to usual care versus usual care alone if prevention of progression to dementia is in part realized in a population of patients diagnosed with a mild cognitive impairment (MCI) at a memory clinic.MethodThe IPECAD open‐source decision‐analytic model was used to simulate the natural disease progression of MCI and dementia and estimate the quality‐adjusted life years (QALYs) and care costs, comparing roflumilast treatment added to usual care with usual care alone. In this model roflumilast proof of concept evidence on a visual 30‐word verbal learning test (0.39 z‐score) was translated using a published risk equation model to a relative risk of 0.93 for developing dementia. The treatment was assumed symptomatic with no effect on mortality.ResultMore lifeyears were spent in MCI in the roflumilast strategy compared with usual care, with 0.2 increment lifeyears (95%CI: 0.0 to 0.6) (see figure 1), and 0.04 incremental lifetime QALYs (95%CI: 0.00 to 0.11) and k€1.3 adjusted societal costs (95%CI: ‐6.3 to 5.5) (see figure 2). This resulted in an incremental cost‐effectiveness ratio of k€34 per QALY (see table 1). Roflumilast was estimated to have a 37% probability of being cost‐effective (net health benefit 95% interval ‐0.27 to 0.42), with uncertainty mainly driven by extrapolating the word learning surrogate outcome to the patient‐relevant outcome of dementia onset, and the efficacy estimate (see figure 3).ConclusionThe potential cost‐effectiveness of roflumilast treatment is uncertain, driven by uncertainty in treatment efficacy and extrapolation to postponing dementia onset. We believe the results indicate a potential for roflumilast and provide several recommendations to consider investing in its future research.

OP79 Gene Expression Profiling In The Diagnosis Of Aggressive Large B Cell Lymphoma: An Early Exploratory Economic Evaluation

IntroductionThe addition of gene expression profiles (GEP) to the current clinicopathological diagnosis of aggressive large B cell lymphomas may lead to the reclassification of patients, treatment changes and improved outcomes. A GEP test is in development using TempoSeq technology to distinguish Burkitt Lymphoma (BL) and Primary Mediastinal Large B Cell lymphoma (PMBCL) from Diffuse Large B Cell Lymphoma (DLBCL). This study aims to inform developers about the potential impact of the test on costs and health outcomes, and pricing and evidence generation strategies.MethodsDecision models compared current diagnosis with current plus GEP signatures over a lifetime horizon using a UK health and social care perspective. Inputs were taken from the literature and based on assumptions. Threshold estimates were made of the maximum price of the test and impact of incorrect disease classification using a threshold of GDP30,000 (USD37,155) per Quality Adjusted Life year (QALY). One way sensitivity analysis was conducted.ResultsAt base case values the BL signature delivers incremental QALYs of 0.0249 at an additional cost per patient of GBP508 (USD629). This results in a net monetary benefit (NMB) of GBP239 (USD296). The PMBCL signature delivers 0.0011 QALYs, a cost saving of GBP202 (USD250) and an NMB of GBP236 (USD292). The maximum threshold price for a combined test to be cost effective is GBP776 (USD961) (base case GBP400 (USD495)). Results are sensitive to cost differences in first line treatments and impact of false diagnoses.ConclusionsA combined test could be cost-effective in a UK context at a price around GBP750 (USD929). The developers can use this estimate to inform return on investment calculations. The number of patients who were reclassified as a result of the addition of GEP in our model was taken from small retrospective studies and the impact of false diagnoses was based on limited evidence. If the developers choose to proceed with the development, these aspects should be incorporated in evidence generation strategies.

Cost-effectiveness of Spironolactone for Adult Female Acne (SAFA): economic evaluation alongside a randomised controlled trial

ObjectiveThis study aims to estimate the cost-effectiveness of oral spironolactone plus routine topical treatment compared with routine topical treatment alone for persistent acne in adult women from a British NHS...

OP14 Cost-Utility Analysis Of Regorafenib For Patients With Hepatocellular Carcinoma Who Progressed On Sorafenib Treatment

IntroductionIn the RESORCE trial, regorafenib was shown to provide overall survival (OS) benefit for patients with hepatocellular carcinoma (HCC) that has progressed on sorafenib treatment. Subsequently, it was approved by the Therapeutic Goods Administration for the treatment of patients with HCC who were previously treated with sorafenib; however, regorafenib is still not recommended by the Pharmaceutical Benefits Advisory Committee in Australia. We aimed to assess the cost effectiveness of regorafenib as a second-line therapy for patients with HCC who progressed on sorafenib from an Australian healthcare perspective.MethodsWe developed a Markov model to compare the cost effectiveness of regorafenib with best supportive care (BSC) as a second-line therapy for HCC after treatment with sorafenib. The health outcomes of life-years and quality-adjusted life-years (QALYs) were derived from the RESORCE trial. Survival benefits sourced from the RESORCE trial were fitted with the parametric model to estimate survival beyond the follow-up period. Drug costs and costs associated with adverse events (AEs) were sourced from published literature and the Independent Health and Aged Care Pricing Authority cost report. Model validity was verified using probabilistic sensitivity analyses.ResultsThe incremental monthly cost of treatment with regorafenib was AUD19,273 (USD13,374), with an incremental life-year gain of 0.38, compared with BSC. The incremental QALYs gained with regorafenib were 0.24, resulting in a base-case incremental cost-effectiveness ratio (ICER) of AUD80,511 (USD55,872) per QALY. In the probabilistic sensitivity analyses across scenarios, the ICER remained above the conventional threshold of AUD50,000 (USD34,698) per QALY, with a zero probability of being cost effective at this willingness-to-pay threshold.ConclusionsAt the current price, second-line treatment with regorafenib in patients with HCC that has progressed on sorafenib was not cost effective at the conventional willingness-to-pay threshold from an Australian health-system perspective.

Cost-effectiveness of treatment in adults with blood pressure of 130–139/80–89 mmHg and high cardiovascular risk in China: a modelling study

The most recent updated hypertension guidelines recommend individuals with systolic blood pressure (SBP)/diastolic blood pressure (DBP) of 130-139/80-89mmHg and high cardiovascular risk should receive antihypertensive drug treatment. This study aimed to assess the benefits and cost-effectiveness of medication for people aged ≥35 years with this blood pressure stratum and high cardiovascular risk in China. The benefits of drug treatment in adults aged ≥35 years with SBP/DBP of 130-139/80-89mmHg and high cardiovascular risk were evaluated in decision-analytic simulation models. Decreasing numbers of cardiovascular disease (CVD) events and premature deaths from all causes and increasing quality-adjusted life-years (QALYs) from drug treatment were estimated in 10-year and lifetime horizons. The incremental cost-effectiveness ratios (ICER) for drug treatment were calculated. Among approximately 106.60 million Chinese adults aged ≥35 years with this blood pressure stratum and high cardiovascular risk, drug treatment was estimated to prevent 2,060,000 strokes and 660,000 myocardial infarctions over a 10-year time horizon. Adults prescribed antihypertensive drugs could gain 0.034 incremental QALYs. Over a lifetime horizon, adults who start treatment earlier could benefit more in preventing CVD and gaining incremental QALYs. The medication treatment is cost-effective either over a 10-year time horizon with an ICER of Int$13321.29 per QALY gained or over the remaining lifetime. Antihypertensive treatment of adults with SBP/DBP of 130-139/80-89mmHg and high cardiovascular risk would gain substantial benefits with cost-effectiveness. The young and middle-aged population would derive the most benefit. National Natural Science Foundation of China, and Beijing Natural Science Foundation.

Superiority and cost-effectiveness of monthly extended-release buprenorphine versus daily standard of care medication: a pragmatic, parallel-group, open-label, multicentre, randomised, controlled, phase 3 trial

Daily methadone maintenance or buprenorphine treatment is the standard-of-care (SoC) medication for opioid use disorder (OUD). Subcutaneously injected, extended-release buprenorphine (BUP-XR) may be more effective-but there has been no superiority evaluation. This pragmatic, parallel-group, open-label, multi-centre, effectiveness superiority randomised, controlled, phase 3 trial was conducted at five National Health Service community-based treatment clinics in England and Scotland. Participants (adults aged ≥ 18 years; all meeting DSM-5 diagnostic criteria for moderate or severe OUD at admission to their current maintenance treatment episode) were randomly assigned (1:1) to receive continued daily SoC (liquid methadone (usual dose range: 60-120mg) or sublingual/transmucosal buprenorphine (usual dose range: 8-24mg) for 24 weeks; or monthly BUP-XR (Sublocade;® two injections of 300mg, then four maintenance injections of 100mg or 300mg, with maintenance dose selected by response and preference) for 24 weeks. In the intent-to-treat population (senior statistician blinded to blinded to treatment group allocation), and with a seven-day grace period after randomisation, the primary endpoint was the count of days abstinent from non-medical opioids between days 8-168 (i.e., weeks 2-24; range: 0-161 days). Safety was reported for the intention-to- treat population. Adopting a broad societal perspective inclusive of criminal justice, NHS and personal social service costs, a trial-based cost-utility analysis estimated the Incremental Cost-effectiveness Ratio (ICER) per quality-adjusted life year (QALY) of BUP-XR versus SoC at the National Institute for Health and Care Excellence threshold. The study was registered EudraCT (2018-004460-63) and ClinicalTrials.gov (NCT05164549), and is completed. Between Aug 9, 2019 and Nov 2, 2021, 314 participants were randomly allocated to receive SoC (n=156) or BUP-XR (n=158). Participants were abstinent from opioids for an adjusted mean of 104.37 days (standard error [SE] 9.89; range: 0-161 days) in the SoC group and an adjusted mean of 123.43 days (SE 4.76; range: 24-161 days) in the BUP-XR group (adjusted incident rate ratio [IRR] 1.18, 95% confidence interval [CI] 1.05-1.33; p-value 0.004). The incidence of any adverse event was higher in the BUP-XR group than the SoC group (128 [81.0%] of 158 participants versus 67 [42.9%] of 156 participants, respectively-most commonly rapidly-resolving (mild-moderate range) pain from drug administration in the BUP-XR group (121 [26.9%] of 450 adverse events). There were 11 serious adverse events (7.0%) in the 158 participants in the BUP-XR group, and 18 serious adverse events (11.5%) in the 156 participants in the SoC group-none judged to be related to study treatment. The BUP-XR treatment group had a mean incremental cost of £1033 (95% central range [CR]-1189 to 3225) and was associated with a mean incremental QALY of 0.02 (95% CR 0.00-0.05), and an ICER of £47,540 (0.37 probability of being cost-effective at the £30,000/QALY gained willingness-to-pay threshold). However, BUP-XR dominated the SoC among participants who were rated more severe at study baseline, and among participants in maintenance treatment for more that 28 days at study enrolment. Evaluated against the daily oral SoC, monthly BUP-XR is clinically superior, delivering greater abstinence from opioids, and with a comparable safety profile. BUP-XR was not cost-effective in a base case cost-utility analysis using the societal perspective, but it was more effective and less costly (dominant) among participants with more severe OUD, or those whose current treatment episode was longer than 28 days. Further trials are needed to evaluate if BUP-XR is associated with better clinical and health economic outcomes over the longer term. Indivior.

Early modelling of the effects and healthcare costs of the Dutch citizen-rescuer system for out-of-hospital cardiac arrests.

1) to analyse the total average healthcare costs of a patient with an out-of-hospital cardiac arrest (OHCA), as well as estimating the operational costs of the citizen-rescuer system (CRS); 2) to conduct an early modelling of the effects and healthcare costs of the Dutch CRS in comparison to no CRS. A health economic modelling study was conducted. Adult patients with OHCA from cardiac causes in the province of Limburg (the Netherlands) were included. The time horizon was from OHCA occurrence up to one year after hospital discharge. First, the total average healthcare costs of OHCA patients were analysed as well as the yearly operating costs of the CRS. Second, an early modelling was conducted to compare from the healthcare perspective the healthcare costs of OHCA patients with the CRS being activated but no responders attended (CRS-NV) versus the CRS being activated with attendance of ≥1 responder(s) (CRS-V). The total average healthcare costs per patient are €42,533. The yearly operating costs for the CRS are approximately €1.5 million per year in the Netherlands. The early modelling of costs and effects showed that the incremental healthcare costs per patient thus were €4,131 in the CRS-V versus the CRS-NV group (€25,184 in the CRS-V group and €21,053 in the CRS-NV group). Incremental quality-adjusted life years (QALYs) was 5 per 100 patients (16 per 100 patients in the CRS-V group versus 11 per 100 patients in the CRS-NV group). The incremental cost-effectiveness ratio (ICER) was €79,662 per QALY for the CRS-V group. This study shows that patients in the CSR-V group had additional health care costs of €4,131 per patient compared to patients in the CRS-NV group. This increase is caused by patients surviving more often, who then continue to utilise health services, which results in a (logic) increase in healthcare costs. For future research, accurate and up-to-date data on effectiveness and costs of the CRS needs to be collected.

Early lowering of LDL-C is a more cost-effective strategy than lowering LDL-C later in life: a cost-effectiveness analysis using Mendelian Randomisation

Abstract Background Early control of low-density lipoprotein-cholesterol (LDL-C) is an effective way to reduce the risk of coronary heart disease (CHD). However, a comprehensive assessment of cost-effectiveness of different lipid-lowering strategies (LLS) for primary prevention of CHD across different ages is lacking. Objective To assess the cost-effectiveness of four different LLS initiated at ages 30, 40, 50, and 60 years on the primary prevention of CHD from the UK National Health Service perspective. Design, setting and participants: We developed a microsimulation model, based on the causal effect of LDL-C on myocardial infarction (MI) or coronary death derived from Mendelian randomisation, comparing initiation of an LLS to current standard of care (control). Data were primarily drawn from 458,727 participants of the UK Biobank study with available data on date of birth, LDL-C, and who were free of MI at first assessment (between 2006 and 2010), with follow-up until 2021. Interventions: The four LLS were: 1) low/moderate intensity statins; 2) high intensity statins; 3) low/moderate intensity statins and ezetimibe; and 4) inclisiran. Main outcomes measures: The incremental cost-effectiveness ratio (ICER), defined as the incremental healthcare costs divided by the incremental quality-adjusted life years (QALYs) for each LLS compared to control, with 3.5% annual discounting. Results The most effective intervention, low/moderate intensity statins and ezetimibe, was projected to lead to a gain in QALYs of 0.068 per person at age 30 and 0.026 at age 60 compared to control (Figure). The age of intervention with the lowest ICER was age 40, with ICERs of £2,524 (95% uncertainty interval: 1,383, 3,796), £4,466 (3,163, 6,134) £11,118 (8,632, 14,177), and £1,405,123 (1,117,1113, 1,789,012) per QALY gained for strategies 1-4 from age 40 years, respectively (Table). Conclusions LDL-C lowering from early ages is a more cost-effective strategy than late intervention. The approach to primary prevention of CHD may improve with a shift to early and sustained lowering of LDL-C. Figure – Results of 1,000 probabilistic sensitivity analyses presented in a common cost-effectiveness plane, by age of intervention. Inclisiran excluded due to very high costs. Solid line: £20,000 per quality-adjusted life year (QALY) willingness-to-pay threshold; dashed line: £30,000 per QALY willingness-to-pay threshold. Any dot to the right of these lines is considered cost-effective. Table – Summary of LLS interventions by age of intervention. Abbreviations: MI – Myocardial infarction; YLL – Years of life lived; QALYs – Quality-adjusted life-years; ICER – Incremental cost-effectiveness ratio.FigureTable

Cost-Utility Analysis of Mirabegron Compared to Solifenacin in the Treatment of Overactive Bladder (OAB) in Iran

Background: Overactive bladder (OAB) is a symptomatic condition characterized by urinary urgency with or without incontinence, usually associated with frequent daytime urination, enuresis, and nocturia. Objectives: This economic evaluation was aimed at assessing the cost-effectiveness of mirabegron versus solifenacin in the treatment of OAB patients from a payer’s perspective in Iran. Methods: A Markov model with a 5-year time horizon was used. The model consisted of five health states, and OAB patients with an average age of 60 years entered the cycle from the persistent state. Transition probabilities were based on published trials, clinical judgments, and expert opinions. Resource use and costs, including those for medications and adverse events, were extracted from the literature and tariff book, and all costs are presented in 2019 US dollars with a 5% discount rate for the costs and utilities. The incremental cost-effectiveness ratio (ICER) and quality-adjusted life-years (QALYs) were computed for medications, and sensitivity analyses were used to test the robustness of the results. Results: Average per-patient treatment costs were $24,720.7 and $24,668.6 for mirabegron and solifenacin, respectively. Mirabegron was expected to produce higher QALYs than solifenacin (3.20 vs. 3.19). Mirabegron had an ICER of $531.3 over solifenacin, lower than the willingness-to-pay (WTP) threshold. The probabilistic analysis showed mirabegron cost-effectiveness in 80% of simulations at the WTP of $2709/QALY. Conclusions: Compared to solifenacin, mirabegron was more cost-effective in OAB patients in the Iranian healthcare system.

Cost-Effectiveness of Carbidopa-Levodopa Enteral Suspension for Advanced Parkinson's Disease in the United States.

Carbidopa/levodopa enteral suspension (CLES) is indicated for the treatment of advanced Parkinson's disease (aPD) with severe motor fluctuations. To determine the cost, quality-adjusted life years (QALY), and cost-effectiveness of CLES compared to the standard-of-care (SoC) for aPD patients in the United States (US), using real-world data. A published Markov model, comprising of 25 health states and a death state, (defined by a combination of the Hoehn and Yahr scale and waking time spent in OFF-time) was adapted to estimate the benefits for CLES versus oral SoC over a patient's lifetime in the US healthcare setting. Clinical inputs were based on a clinical trial and a registry study; utility inputs were sourced from the Adelphi-Disease Specific Programmes. CLES compared to SoC was associated with incremental costs ($1,031,791 vs. $1,025,180) and QALY gain (4.61 vs. 3.76), resulting in an incremental cost-effectiveness ratio of $7711/QALY. CLES is a cost-effective treatment for aPD patients with medication resistant motor fluctuations. © 2023 AbbVie, Inc and The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

Endovascular treatment for basilar artery occlusion: a cost-effectiveness analysis based on a meta-analysis.

This study aimed to investigate the efficacy and economic effect of endovascular treatment (EVT) combined with standard medical treatment (SMT) vs. SMT alone in Chinese patients with basilar artery occlusion (BAO) from the perspective of the Chinese healthcare system. We conducted a cost-effectiveness analysis using the results from a meta-analysis comparing EVT and SMT efficacy in Chinese patients with BAO-induced stroke using direct medical costs from the China National Stroke Registry. The meta-analysis's primary outcome was excellent functional outcome (mRS scores of 0-2), with secondary outcomes being poor functional outcome (mRS scores of 3-5) and death (mRS score of 6). To compare EVT plus SMT's cost-effectiveness with that of SMT alone, we constructed a combined decision tree and Markov model with a lifetime duration and a 3-month cycle length. The primary cost-effectiveness outcome was the incremental cost-effectiveness ratio (ICER), representing the incremental cost per incremental quality-adjusted life year (QALY). EVT was considered cost-effective if the ICER was lower than the willingness-to-pay (WTP) threshold of three times the per capita gross domestic product (GDP) in 2021 in China; otherwise, it would not be cost-effective. The meta-analysis results indicated that EVT could increase the incidence of excellent functional outcomes, with a risk ratio (RR) of 2.23 (95% confidence interval, CI, 1.18-4.21), p = 0.01. Simultaneously, EVT reduced the risk of poor functional outcome and mortality in the EVT group, with RRs of 0.83 (95% CI, 0.67-1.03), p = 0.09, and 0.71 (95% CI, 0.59-0.85), p = 0.0002, respectively. The study also found that EVT plus SMT resulted in a lifetime effectiveness of 2.15 QALY (3.88 life years) for 32,213 international dollars (Intl.$) per patient with BAO. In contrast, SMT alone achieved an effectiveness of 1.46 QALY (3.03 life years) with a total cost of Intl.$ 13,592 per patient. The ICER was Intl.$ 27,265 per QALY (Intl.$ 22,098 per life-year), which fell below the WTP threshold. Compared to SMT, EVT improves the prognosis of BAO-induced stroke. Considering the Chinese healthcare system, adding EVT to SMT proves to be cost-effective for patients with BAO compared to SMT alone.

Evaluating the cost-effectiveness of HPV vaccination for adolescent girls in Japan: A comparison of 2-valent, 4-valent, and 9-valent HPV vaccines with consideration of cross-protection

ObjectiveIn April 2023, the Japanese Health Ministry panel approved the inclusion of the 9-valent human papillomavirus (9vHPV) vaccine in the National Immunization Program, alongside the 2-valent (2vHPV) and 4-valent HPV (4vHPV) vaccines. In response to this, we evaluated the cost-effectiveness of these three vaccines for routine immunization of girls aged 12–16 in Japan, considering the cross-protection of 2vHPV and 4vHPV vaccines. MethodsWe constructed an age-structured mathematical model for HPV transmission, aiming to quantify the economic and epidemiological effects of various HPV vaccination strategies over a 70-year period in Japan. We determined incremental costs and quality-adjusted life years (QALYs) for each strategy, applying a 3% annual discount. Univariate and probabilistic sensitivity analyses were conducted to assess the uncertainty of our model results, with all evaluations done in 2023. ResultsOur projections indicate that the HPV vaccination program in Japan will significantly reduce the incidence of HPV-related diseases. All HPV vaccination strategies, using the 2vHPV, 4vHPV, and 9vHPV vaccines, were found to be cost-effective compared to no vaccination, with incremental cost-effectiveness ratios of ¥971,447/QALY, ¥1,237,297/QALY, and ¥742,084/QALY, respectively. Direct comparisons between vaccines demonstrated that the 9vHPV vaccination was more cost-effective than the 2vHPV vaccination, whereas 4vHPV vaccination was dominated by 2vHPV vaccination. ConclusionsOur study validates the cost-effectiveness of implementing the 9vHPV vaccine as the primary option over the 2vHPV or 4vHPV vaccine for girls in Japan. These findings underscore the need to improve the acceptance rate and coverage of HPV vaccinations in the country.

Cost-effectiveness of left atrial appendage closure with Watchman for non-valvular atrial fibrillation patients in Japan

AimsLeft atrial appendage closure (LAAC) has been demonstrated to be cost-saving relative to oral anticoagulants for stroke prophylaxis in patients with non-valvular atrial fibrillation (NVAF) in the United States and Europe. This study assessed the cost-effectiveness of LAAC with the Watchman device relative to warfarin and direct oral anticoagulants (DOACs) for stroke risk reduction in NVAF from a Japanese public healthcare payer perspective.MethodsA Markov model was developed with 70-year-old patients using a lifetime time horizon. LAAC clinical inputs were from pooled, 5-year PROTECT AF and PREVAIL trials; warfarin and DOAC inputs were from published meta-analyses. Baseline stroke and bleeding risks were from the SALUTE trial on LAAC. Cost inputs were from the Japanese Medical Data Vision database. Probabilistic and one-way sensitivity analyses were performed.ResultsOver the lifetime time horizon, LAAC was less costly than warfarin (savings of JPY 1,878,335, equivalent to US $17,600) and DOACs (savings of JPY 1,198,096, equivalent to US $11,226). LAAC also provided 1.500 more incremental quality-adjusted life years (QALYs) than warfarin and 0.996 more than DOACs. In probabilistic sensitivity analysis, LAAC was cost-effective relative to warfarin and DOACs in 99.98% and 99.73% of simulations, respectively. LAAC dominated (had higher cumulative QALYs and was less costly than) warfarin and DOACs in 89.94% and 83.35% of simulations, respectively.ConclusionsOver a lifetime time horizon, LAAC is cost-saving relative to warfarin and DOACs for stroke risk reduction in NVAF patients in Japan and is associated with improved quality-of-life.

Cost-Effectiveness of Ibrutinib as First-line Treatment for Older Patients With Chronic Lymphocytic Leukemia in Iran

ObjectivesWe aimed to evaluate the cost-effectiveness of ibrutinib versus chemoimmunotherapy for frontline treatment of elderly patients with chronic lymphocytic leukemia in Iran. MethodsWe developed a partitioned survival model with 3 health states (progression-free survival, post-progression survival, and death) and a lifetime horizon. State memberships were determined by parametric survival analysis of the ALLIANCE (A041202) randomized controlled trial’s results, comparing first-line ibrutinib with bendamustine plus rituximab. Direct medical costs were calculated from an Iranian health system perspective. Utility values were extracted from the literature to calculate the incremental costs and quality-adjusted life-years (QALYs) associated with each strategy. To address parameter uncertainties, deterministic and probabilistic sensitivity analyses were also performed. ResultsIn the base-case analysis, ibrutinib and bendamustine plus rituximab were associated with $3739.72 and $3991.20 costs per patient as the first-line treatment strategy, respectively. They resulted in an average of 2.86 and 2.66 QALYs per patient. Thus, first-line ibrutinib was associated with 0.20 incremental QALY and $251.48 cost-saving per patient and was therefore the “dominant” strategy. In deterministic sensitivity analysis, drug prices were the key drivers of model outputs. However, none of the resulting incremental cost-effectiveness ratios exceeded the currently accepted threshold by the Iranian Food and Drug Administration ($1550 per QALY). In probabilistic sensitivity analysis, 63.3% of iterations were cost-saving and 77.4% were cost-effective. ConclusionsOur findings suggest that ibrutinib as a first-line treatment appears to be the dominant strategy, compared with the standard of care, for unselected older adults with chronic lymphocytic leukemia in Iran.