Cost-Effectiveness Analysis of Fluocinolone Acetonide Intravitreal (FAI) Implant for Chronic Noninfectious Uveitis Affecting the Posterior Segment of the Eye (NIU-PS) in China

Cost Effectiveness Analysis of Venetoclax Plus Azacitidine Versus Azacitidine in Newly Diagnosed Adult Patients with Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy from a United States Payer Perspective

From the perspective of Chinese healthcare system, this study compared the cost-utility of aripiprazole once-monthly (AOM) and paliperidone palmitate once-monthly injectable (PP1M) in the treatment of adult patients with schizophrenia in China. A 5-state Markov model was developed to evaluate the cost-utility of 10 years of long-acting injections (LAI) treatment for schizophrenia. The long-term costs and quality-adjusted life years (QALYs) were estimated, with the incremental cost-effectiveness ratio (ICER) as the primary outcome. The annual discount rate was set at 5%. A cost-effectiveness threshold (CET) of 0.51 times China's 2023 gross domestic product (GDP) (US$ 6,394.536) was used to judge the economics of intervention. The current price of AOM in China is relatively high (US$418.140). To assess its cost-effectiveness in the context of potential price negotiations with China Healthcare Security Administration (CHS) for inclusion in the National Reimbursement Drug List (NRDL), we simulated a 40% price reduction (US$257.619). At a CET of 0.51 times GDP per capita (US$6,394.536), the base-case analysis showed that the incremental costs of AOM relative to PP1M after 10 years of treatment were US$1,926.373 with an incremental gain of 0.306 QALYs. The ICER for AOM was US$6,285.303 per QALY, which is below the CET, indicating that AOM is cost-effective. One-way sensitivity analysis identified AOM's drug cost as the parameter with the greatest impact on results. Probabilistic sensitivity analysis revealed that with a 40% price reduction, the probability of AOM being cost-effective is only 41.70%. However, with a 60% price reduction, AOM became dominantly cost-effective, with the probability increasing to 100%. When the CET was relaxed to 0.90 times GDP per capita (US$11,284.476), the probability of cost-effectiveness for AOM after a 40% price reduction rose to 85.10%. Scenario analyses conducted over a time horizon extending from 10 to 30 years showed that the ICER decreased significantly with longer follow-up, gradually approaching the 0.51GDP threshold and remaining below the 0.90 GDP threshold throughout the analysis. The cost-effectiveness of AOM relative to PP1M is highly influenced by its price and the CET. Healthcare decision makers or clinical users need to balance innovation incentives and accessibility.

Sugemalimab plus chemotherapy significantly prolonged progression-free survival and overall survival in patients with advanced oesophageal squamous-cell carcinoma (ESCC). However, considering the high cost of sugemalimab, we evaluated the cost-effectiveness of sugemalimab plus chemotherapy in the first-line treatment for advanced ESCC. A partitioned survival model with a 3-week cycle length and the lifetime time horizon was constructed to assess the cost-effectiveness of sugemalimab plus chemotherapy versus placebo plus chemotherapy as first-line treatment for advanced ESCC. The key survival data, drug costs, and utility values were obtained from the GEMSTONE-304 trial, YAOZHI database and the published literature. Total costs, quality-adjusted life-years (QALYs) and incremental cost-effectiveness ratios (ICERs) were measured. One-way and probabilistic sensitivity analyses were performed to evaluate the uncertainty and model stability. In addition, subgroup and scenario analyses were conducted. The Chinese healthcare system perspective. A hypothetical Chinese cohort of patients with unresectable, locally advanced, recurrent or metastatic ESCC. Sugemalimab plus chemotherapy versus chemotherapy. Costs, QALYs, ICERs. Compared with the placebo plus chemotherapy, the sugemalimab plus chemotherapy provided an additional 0.36 QALYs, with an incremental cost of $44 376.88. The result of ICER was $119 400.69/QALY, which was significantly higher than the willingness-to-pay (WTP) threshold of three times per capita gross domestic product ($38 024.68/QALY) in 2023. Sensitivity analysis revealed that the cost of sugemalimab, the discount rate and the utility values were the most influential parameters on the base-case analysis results. Subgroup analyses showed that programmed cell death - ligand 1 subgroup ICERs exhibited dose-dependent efficacy: the ICERs for sugemalimab plus chemotherapy versus placebo plus chemotherapy were $138 739.23/QALY and $108 051.81/QALY in advanced ESCC patients with 1 ≤ combined positive score (CPS) <10 and CPS ≥10. Scenario analysis indicated that even with patient assistance programme, the total cost of the sugemalimab plus chemotherapy group ($23 474.25) would be $15 030.18 higher than that of the placebo plus chemotherapy group ($8444.07), which had an ICER of $40 440.28/QALY. When the price of sugemalimab decreased by 50% and 85%, the probabilities of sugemalimab plus chemotherapy being cost-effective were 0% and 100%, respectively. From a Chinese healthcare system perspective, sugemalimab plus chemotherapy as first-line treatment for advanced ESCC might not be a cost-effective treatment option at the WTP threshold of $38 024.68/QALY.

To evaluate the real-world clinical effectiveness and safety of the 0.18 mg fluocinolone acetonide intravitreal implant (FAi) in Chinese patients with macular edema (ME) secondary to non-infectious uveitis (NIU). This single-center, retrospective case series included patients diagnosed with NIU-associated ME who received an intravitreal 0.18 mg FAi injection between January and September, 2024 and completed at least 12 months of follow-up. Primary outcome measures were changes in best-corrected visual acuity (BCVA, converted to logMAR), intraocular pressure (IOP), central macular thickness (CMT), and subfoveal choroidal thickness (SFCT) from baseline to 1, 3, 6, and 12 months post-treatment, analyzed using a linear mixed model. Inflammatory recurrence and treatment-related adverse events were also documented. A total of 39 patients (50 eyes) were included, with a mean age of 54.6 ± 14.8 years. The mean baseline BCVA was 0.65 ± 0.33 logMAR, and the mean CMT was 333.4 ± 108.6 μm. BCVA demonstrated significant improvement at all postoperative time points compared to baseline (all P< 0.001), reaching 0.48 ± 0.31 logMAR at 12 months. CMT showed a significant and sustained reduction postoperatively (all P< 0.001), decreasing to 239.7 ± 54.4 μm at 12 months. SFCT also decreased significantly from baseline (all P< 0.001). Postoperative IOP was significantly higher than baseline (P= 0.008, P= 0.001, P< 0.001, P< 0.001). Postoperative IOP elevation was observed in 18 eyes (36%). Management strategies included topical anti-glaucoma medication in 12 eyes and laser therapy in 3 eyes (1 selective laser trabeculoplasty and 2 diode laser cyclophotocoagulation). Notably, 3 eyes (6%) required trabeculectomy due to uncontrolled IOP. The inflammation recurrence rate during the 12-month follow-up was 14% (7/50 eyes). Implant migration into the anterior chamber occurred in one eye. No other serious ocular complications or systemic adverse events were observed. In this real-world study, the 0.18 mg FAi effectively improved visual acuity, reduced ME, and attenuated choroidal thickening in Chinese patients with NIU-associated ME over 12 months. The primary risk was manageable IOP elevation, resulting in an overall favorable safety profile. The 0.18 mg FAi represents an effective long-term treatment option for this patient population.

ObjectivesTo assess the cost-effectiveness of aducanumab at its updated price for treating patients with mild cognitive impairment (MCI) and mild Alzheimer’s disease (AD).DesignCost-effectiveness analysis.SettingsA five-state Markov model was constructed using...

Background: Whether Endovascular Thrombectomy (EVT) is cost-effective in large ischemic core infarcts is unknown. Methods: In the prospective multicenter cohort study of imaging selection study (SELECT), large core was defined as CT ASPECTS &lt; 6 or CTP ischemic core volume (rCBF&lt;30%) ≥ 50 cc. A Markov model estimated costs, quality-adjusted life years (QALYs) and the Incremental Cost-effectiveness Ratio (ICER) of EVT compared to Medical Management (MM) over 20 years life expectancy. The lower and upper willingness to pay (WTP) per QALY were set at $50000 and $100000 and the Net Monetary Benefit (NMB) for EVT were calculated. A probabilistic sensitivity analysis (PSA) and cost-effectiveness acceptability curves (CEAC) assessed EVT cost-effective probability at WTP range values. Results: Of 361 enrolled, 105 had large core on CT or CTP (EVT 62, MM 43). 19 (31%) EVT patients achieved mRS 0-2 vs 6 (14%) MM (aOR: 3.27, 95% CI: 1.11-9.62; P = .03) with a shift towards better mRS (adj cOR: 2.12, 95% CI: 1.05-4.31, P = .04). Over 20 years EVT was associated with $26589 (C.I. $8672- $43978) incremental costs and a gain of 1.18 QALYs (C.I. 0.091- 2.2) per patient. EVT could avert 75 deaths over a theoretical cohort of 1000 patients (MM 861 vs EVT 786) thus the ICER of EVT compared to MM was $22400 per QALY (CI. $10109 - $66140), which is &lt;$50000/QALY, Tab 1. EVT has a higher NMB compared to MM at the lower and upper WTP thresholds (EVT $86,3 and 271,4 million vs MM $53,6-$179,3 million), Tab 2. The PSA confirmed the results (fig 1). The CEAC showed 94% and 97% cost-effectiveness probability of EVT at the lower and upper values respectively of the maximum WTP, fig 2. EVT ICER in SELECT large core ($22400/QALY) was higher but still comparable to those in HERMES ($16882/QALY), DAWN ($7335/QALY) and DEFUSE3 ($14673/QALY), Tab 3. Conclusion: EVT may result in better outcomes and more lives saved in large core patients with higher QALYs, NMB and an acceptable ICER. The results were comparable to other EVT RCTs.

BackgroundPosaconazole is confirmed to be more effective for preventing invasive fungal infections (IFIs) than first-generation triazoles (fluconazole and itraconazole), but its economic value has not been comprehensively evaluated in China. This study compared the cost-effectiveness of these two antifungal prophylaxis regimens in hematological-malignancy patients at high risk for IFIs from the Chinese healthcare perspective.MethodsA hybrid decision tree and Markov model were built using published data to estimate the total costs and quality-adjusted life-years (QALYs) of antifungal prophylaxis with posaconazole oral suspension and first-generation triazoles. Regimens with an incremental cost-effectiveness ratio (ICER) lower than the threshold of willingness to pay (WTP) were considered cost-effective. One-way and probabilistic sensitivity analyses were performed to assess model robustness. The regional imbalance of economic development and the tablet formulation of posaconazole were considered in the scenario analyses.ResultsIn the base-case analysis, posaconazole oral suspension provided an additional 0.109 QALYs at an incremental cost of $954.7, yielding an ICER of $8,784.4/QALY, below the national WTP threshold of $31,315/QALY. One-way and probabilistic sensitivity analyses showed that the results were robust. Scenario analyses showed that the base-case ICER was consistently below the WTP thresholds of all 31 Chinese provinces, with the likelihood of posaconazole being cost-effectiveness ranging from 78.1 to 99.0%. When the posaconazole oral suspension was replaced by the tablet formulation, the ICER increased to $29,214.1/QALY, still below the national WTP threshold and WTP thresholds of 12 provinces.ConclusionsPosaconazole oral suspension is a highly cost-effective regimen for preventing IFI in high-risk hematological-malignancy patients from the Chinese healthcare perspective. Posaconazole tablets may also be considered in some high-income regions of China.

Introduction: China has ~6 million patients with active epilepsy every year, around 60% of whom suffer from partial-onset seizures. Perampanel (PER) is a novel anti-epileptic drug for partial-onset seizures. PER has been included in the latest Chinese National Reimbursement Drug List (NRDL) in 2020. However, there is still a lack of evaluation evidence on the value of PER in China.Methods: This study selected a health system perspective. A Markov model was established to simulate the lifelong transition of different response levels and calculate the number of seizures in Chinese patients. Based on the utility value and mortality risk, the life years and quality-adjusted life years (QALYs) of patients using PER vs. lacosamide (LCM) were estimated. Efficacy data were derived from clinical trials and the literature. Cost data (in US dollars) included drug costs and medical service costs. A lifetime horizon was adopted. Health outcomes and costs were discounted at an annual discount rate of 5%. Deterministic sensitivity analysis, probability sensitivity analysis, and scenario analysis were performed. The impact of the inclusion of PER in the NRDL on the medical insurance budget over 3 years (2021–2023) was also estimated.Results: Cost-effectiveness analysis indicates that 8 mg/day of PER increases QALYs by 0.054 and saves costs by $2,390 compared with 400 mg/day of LCM. 4 mg/day of PER increases QALYs by 0.010 and saves costs by $860 compared with 200 mg/day of LCM. Deterministic sensitivity analysis reveals that utility value and the extreme discount rate are the factors with the greatest impact on the incremental cost-effectiveness ratio. Probabilistic sensitivity analysis and scenario analysis show that the results are robust. Budget impact analysis indicates that after inclusion of PER in the NRDL, the incremental budget would be $1.28, $2.83, and $4.56 million from 2021 to 2023, respectively, but covering more eligible epileptic patients in the same time (1,918, 4,287, and 8,983, respectively).Conclusion: PER (8 or 4 mg/day) is of relatively high value as an add-on therapeutic regimen for partial-onset seizures in China because of its dominate advantage of cost-effectiveness over LCM and acceptable budget impact.

IntroductionWhile the APOLLO trial confirmed the clinical efficacy of first-line anlotinib plus penpulimab in unresectable hepatocellular carcinoma (HCC), its economic impact on China’s healthcare system remains underexplored. This study was conducted to evaluate the cost-effectiveness of this novel combination vs. sorafenib from the perspective of the Chinese healthcare system.MethodsA partitioned survival model with three health states was developed to simulate economic outcomes for patients with advanced HCC. Survival data were derived from the APOLLO trial using parametric fitting. Direct medical costs and utility values were obtained from local public databases and the published literature. The primary outcomes included total costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs) evaluated against the willingness-to-pay (WTP) threshold of $40334.05/QALY. Model robustness was assessed through deterministic and probabilistic sensitivity analyses (PSA).ResultsThe base-case analysis revealed that anlotinib plus penpulimab incurred a total cost of $25681.69 and yielded 1.42 QALYs, compared with sorafenib’s total cost of $18082.48 and 1.19 QALYs. This resulted in an incremental cost of $7599.21 and an incremental effectiveness of 0.22 QALYs, resulting in an ICER of $34050.28/QALY, which is below the predefined WTP threshold. Sensitivity analyses identified anlotinib treatment duration (cycles) and progression-free survival (PFS) utility values as key drivers of model variability. The PSA indicated an 85.9% probability of cost-effectiveness at the WTP threshold.ConclusionAnlotinib plus penpulimab represents a potentially cost-effective first-line treatment for advanced HCC from a Chinese healthcare system perspective. These findings support incorporating this regimen into guidelines for selecting cost-effective immunotherapeutic strategies and provide evidence to inform decision-making about resource allocation for advanced HCC management.

Benmelstobart and anlotinib plus etoposide-carboplatin (EC) group has demonstrated substantial clinical efficacy in improving survival outcomes for patients with extensive-stage small-cell lung cancer (ES-SCLC). However, the high treatment cost raises concerns regarding its affordability and cost-effectiveness across healthcare systems with heterogeneous pricing and reimbursement mechanisms. This study aimed to evaluate the cost-effectiveness of benmelstobart and anlotinib plus EC group compared to EC alone group and anlotinib plus EC group from both US and Chinese payer perspectives. The findings are intended to inform value-based pricing strategies and evidence-based reimbursement decision-making. A partitioned survival model (PSM) with a lifetime horizon and 21-day cycles was constructed using clinical data from the ETER701 trial. Direct medical costs and health utility inputs were obtained from national databases, local hospitals, and published literature. The primary outcome was the incremental cost-effectiveness ratio (ICER), calculated by comparing costs and quality-adjusted life years (QALYs) between treatment strategies. Scenario analyses, including drug price simulations and deterministic and probabilistic sensitivity analyses, were conducted to evaluate model robustness. Willingness-to-pay (WTP) thresholds were set at $100,000/QALY and $150,000/QALY (US) and $40,011/QALY (China). In the US, the benmelstobart and anlotinib plus EC group yielded ICER of $121,560.40/QALY versus EC alone group and $127,579.09/QALY versus anlotinib plus EC group, both below the $150,000/QALY threshold. However, at the $100,000/QALY threshold, cost-effectiveness would require reducing benmelstobart's price to $1316.12/600mg. In China, the ICER of $117,667.17/QALY exceeded the local threshold. Price simulations suggested that cost-effectiveness could be achieved if prices were reduced below $2230.60/600mg (US) and $328.47/600mg (China). Sensitivity analyses identified progression-free survival (PFS) utility and benmelstobart pricing as major cost drivers. Probabilistic analysis indicated a 75.1% probability of cost-effectiveness at $150,000/QALY in the US. However, the probability of cost-effectiveness is 0% at WTP thresholds of $100,000/QALY in the US and $40,011/QALY in China. Benmelstobart plus anlotinib and EC group is likely to be cost-effective in the US at a WTP threshold of $150,000/QALY, but not in China at current prices. An 80% price reduction in China would be necessary to align with its WTP threshold, emphasizing the need for policy interventions in drug pricing and reimbursement to improve patient access.

Invasive mold diseases (IMD) is associated with high mortality and a substantial economic burden. For high-risk patients, fever drive or diagnostic drive therapy is usually initiated prior to the differential diagnosis of the pathogen. This study evaluated the cost-effectiveness of isavuconazole, posaconazole, vs. voriconazole in the treatment of IMD from the perspective of the Chinese healthcare system, informing healthcare decision-making and resource allocation. A decision analytic model was constructed using TreeAge Pro 2011 software to evaluate the cost-effectiveness of the entire disease course. We assumed that the prevalence of mucormycosis in the patients entering the model was 7.8%. Efficacy, cost, adverse events, and other data included in the model were mainly derived from clinical studies, published literature, and publicly available databases. The primary outcomes of the model output were total cost, quality-adjusted life years (QALYs), life years (Lys), and incremental cost-effectiveness ratio (ICER). The willing-to-pay (WTP) threshold was defined as one to three times China's GDP per capita in 2022. One-way sensitivity analysis and probability sensitivity analysis were used to determine the robustness of the model. At the same time, the cost-effectiveness of three triazole antifungal agents under a broader range of mucormycosis prevalence, when voriconazole was covered by medical insurance reimbursement, and after the price reduction of posaconazole was discussed. Compared with voriconazole, isavuconazole provided an additional 0.38 Lys (9.29 vs. 8.91 LYs) and 0.31 QALYs (7.62 vs. 7.31 QALYs); ICER was $15,702.46/QALY, well-below the WTP threshold ($38,223/QALY). However, posaconazole did not provide a significant economic advantage over voriconazole (9.40 vs. 9.36 Lys; 7.71 vs. 7.68 QALYs; ICER $64,466.57/QALY). One-way sensitivity analysis found that ICER was highly sensitive to the mortality of patients with invasive aspergillus infection. In the probabilistic sensitivity analysis, when the WTP threshold was $38,223/QALY, the probability of isavuconazole being cost-effective was 72.9%. The scenario analysis results indicated that posaconazole would become cost-effective when the price was reduced by 15% or the prevalence of mucormycosis was 14%. Isavuconazole represents a cost-effective initial option for treating IMD in high-risk hematological patients prior to the differential diagnosis of pathogens. It will also be economical when a 15% reduction in posaconazole cost is achieved.

To assess the clinical effectiveness and cost-effectiveness of cardiac resynchronisation therapy (CRT) for people with heart failure and evidence of dyssynchrony by comparing cardiac resynchronisation therapy devices, CRT-P and CRT with defibrillation (CRT-D), each with optimal pharmaceutical therapy (OPT), and with each other. Electronic databases were searched up to June 2006. Manufacturer submissions to the National Institute for Health and Clinical Excellence (NICE) were also searched for additional evidence. Relevant data from selected studies were extracted, narrative reviews were undertaken and meta-analyses of the clinical trial data were conducted. A Markov model was developed. Incremental costs and quality-adjusted life-years (QALYs) were calculated. Extensive one-way sensitivity analyses, threshold analyses, probabilistic sensitivity analyses and value of information analyses were carried out. Five randomised controlled trials met the inclusion criteria, recruiting 3434 participants. Quality was good to moderate. Meta-analyses showed that both CRT-P and CRT-D devices significantly reduced the mortality and level of heart failure hospitalisations and they improved health-related quality of life in people with New York Heart Association (NYHA) class III and IV heart failure and evidence of dyssynchrony (QRS interval >120 ms) who were also receiving OPT. A single direct comparison indicated that the effects of CRT-P and CRT-D were similar, with the exception of an additional reduction in sudden cardiac death (SCD) associated with CRT-D. On average, implanting a CRT device in 13 people would result in the saving of one additional life over a 3-year period compared with OPT. The NHS device and procedure cost of implanting a new CRT-P system (pulse generator unit and required leads) was estimated to be 5074 British pounds and that of a CRT-D system 17,266 British pounds. The discounted lifetime costs of OPT, CRT-P and CRT-D were estimated as 9375 British pounds, 20,804 British pounds and 32,689 British pounds, respectively. The industry submissions to NICE contained four cost-effectiveness analyses, of which two were more appropriate as reference cases. One used a discrete event simulation model that gave estimated incremental cost-effectiveness ratios (ICERs) of CRT-P vs OPT of 15,645 British pounds per QALY. The other analysis was based on the results of the COMPANION trial and estimated an ICER of 2818 British pounds per QALY gained by CRT-P vs OPT and a cost per QALY gained of 22,384 British pounds for CRT-D vs OPT. Compared with OPT, the Markov model base case analysis estimated that CRT-P conferred an additional 0.70 QALYs for an additional 11,630 British pounds per person, giving an estimated ICER of 16,735 British pounds per QALY gained for a mixed age cohort (range 14,630-20,333 British pounds). CRT-D vs CRT-P conferred an additional 0.29 QALYs for an additional 11,689 British pounds per person, giving an ICER of 40,160 British pounds per QALY for a mixed age cohort (range 26,645-59,391 British pounds). The authors' ICERs are higher than those from the industry-submitted analysis. Probabilistic sensitivity analysis based on 1000 simulated trials showed that, at a willingness-to-pay (WTP) threshold of 30,000 British pounds per QALY, in CRT-P versus OPT, CRT-P was likely to be cost-effective in 91.3% of simulations and that CRT-P was negatively dominated in 0.4% of simulations. It also showed that in CRT-P versus CRT-D, CRT-D was likely to be cost-effective in 26.3% of simulations and that CRT-P dominated CRT-D in 7.8% of simulations. The relative risk for SCD when CRT-D is compared with OPT is 0.44 in the base case. This treatment becomes cost-ineffective at a WTP threshold of 30,000 British pounds when this value is greater than 0.65. When both CRT-P and CRT-D were considered as competing technologies with each other and OPT (three-way probabilistic analysis), and at the same WTP, there was a 68% probability that CRT-P provided the highest expected net benefit. The WTP threshold would need to be above 40,000 British pounds before CRT-D provided the highest expected net benefit. The study found that CRT-P and CRT-D devices reduce mortality and hospitalisations due to heart failure, improve quality of life and reduce SCD in people with heart failure NYHA classes III and IV, and evidence of dyssynchrony. When measured using a lifetime time horizon and compared with optimal medical therapy, the devices are estimated to be cost-effective at a WTP threshold of 30,000 British pounds per QALY; CRT-P is cost-effective at a WTP threshold of 20,000 British pounds per QALY. When the cost and effectiveness of all three treatment strategies are compared, the estimated net benefit from CRT-D is less than with the other two strategies, until the WTP threshold exceeds 40,160 British pounds/QALY. Further research is needed into the identification of those patients unlikely to benefit from this therapy, the appropriate use of CRT-D devices, the differences in mortality and heart failure hospitalisation for NYHA classes I and II, as well as the long-term implications of using this therapy.

Background: The Reduction of Cardiovascular Events with Icosapent Ethyl (IE)-Intervention Trial (REDUCE-IT) demonstrated the efficacy of IE among patients with elevated triglyceride levels despite the use of statins. This study aimed to examine the cost-effectiveness (CE) of IE among US adults using both in-trial and lifetime time horizons. Methods: US patients in REDUCE-IT were included in the in-trial analysis. We included all cardiovascular and serious adverse events from the REDUCE-IT database where rates differed between the study arms; we used patient-level data from REDUCE-IT USA based on 2019 US costs and $4.16/day for IE. The lifetime analysis used a microsimulation Markov model. Both analyses considered value from a US health sector perspective. The primary result is the incremental CE ratio (ICER), measured as incremental costs divided by incremental quality-adjusted life-years (QALY) of IE compared with placebo. We performed univariate and probabilistic sensitivity analyses (PSA) to capture the uncertainties involved in the estimation of costs and QALYs. Results: Based on 3146 REDUCE-IT USA participants, there was an incremental gain in QALYs with IE compared with placebo using in-trial (3.28 vs. 3.13) and lifetime (10.36 vs. 9.83) time horizons. Total healthcare costs were lower with IE compared with placebo for both in-trial ($20,221 vs. $20,357) and lifetime ($201,842 vs. $204,701). IE was a dominant strategy compared to placebo using a lifetime time horizon with a 74.8% probability of being more effective and costing less and had a 99.6% probability of costing below $50,000 per QALY. The lifetime PSA showed that IE was a dominant strategy in 65.6% of simulations and cost-effective in 98.8%, 99.6%, and 99.9% of simulations at the $50,000, $100,000, and $150,000 per QALY gained thresholds, respectively. Conclusions: The REDUCE-IT USA cost-effectiveness analysis has shown that IE provides better outcomes with lower costs, dominant both in-trial and lifetime as well in the majority of sensitivity analyses and subgroups, both in primary and secondary prevention. These results, with the clinical evidence of efficacy, suggest that at $4.16 per day, IE therapy should be strongly considered in patients similar to those enrolled in REDUCE-IT USA.

BackgroundDetailed information on the efficiency of health services targeting opioid use disorder (OUD) and treatment with opioid agonist treatment (OAT) is sparse. Many countries, including Norway, are still falling short of universal health coverage (UHC) of OAT. This study aims to evaluate the incremental lifetime costs and effects of treating OUD with OAT as compared to no OAT in Norway and scaling up the treatment to a universal coverage level using equity-adjusted health economic evaluations.MethodsWe conducted cost-utility and budget impact analyses and constructed a two-state Markov model to compare the lifetime costs and outcomes among patients with OUD with and without OAT. Model inputs were derived from routine health information systems and the literature, with costs reported in 2023 Norwegian Kroner (NOK). The analyses were conducted from a Norwegian extended health-service and societal perspectives, with a lifetime time horizon. Quality-adjusted life years (QALYs) was the metric of health benefits. Outcomes were reported as incremental cost-effectiveness ratios (ICERs). The willingness-to-pay (WTP) threshold was equity-adjusted according to the future prognostic healthy life year loss method in Norway (severity of disease criterion), which is sensitive to the size of future undiscounted healthy life year loss due to the affected conditions. The WTP threshold is NOK 825,000 per QALY gained in Norwegian policy for conditions with undiscounted future QALY loss > 20. Uncertainty in the parameters and robustness of the results were assessed with one-way and probabilistic sensitivity analyses and scenario analyses.FindingsThe mean results from probabilistic sensitivity analysis estimated that OAT was associated with 3.03 additional discounted QALYs gain and incremental lifetime discounted cost of NOK 1.45 million, leading to an ICER of NOK 479,099 per QALY gained when compared with not providing OAT, with the extended health-service perspective. From a societal perspective, OAT was cost-saving, i.e. OAT produced greater health benefits while resulting in lower overall societal costs compared to no OAT. The mean undiscounted future health loss was estimated to be 21.34 QALYs for the Norwegian patient group with OUD. A total 5-year budget increase of NOK 1.208 billion was estimated if OAT was going to be scaled up from the current coverage level of 70% to UHC. Compared with the current coverage, 100% coverage of OAT was associated with an additional lifetime cost of NOK 4.332 billion but also an additional 6760 QALYs gained.ConclusionOur analysis suggests that OAT is cost-effective in Norway and has the potential to be cost-saving from a societal perspective. Therefore, Norwegian policy should consider scaling up treatment to extend the coverage of OAT.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40273-024-01442-3.

Aims To evaluate the cost-effectiveness of vibegron compared with other oral pharmacologic therapies as treatment for overactive bladder (OAB). Methods A semi-Markov model with monthly cycles was developed to support a lifetime horizon of vibegron 75 mg from a US commercial payor or Medicare perspective. The model incorporated efficacy (reductions in daily micturitions and urinary incontinence episodes), adverse events, OAB-related comorbidities, drug–drug interactions, anticholinergic burden, and treatment persistence. Direct costs and quality-adjusted life years (QALY) were accumulated over time. The primary outcome was the cost per QALY incremental cost-effectiveness ratio (ICER). One-way (OWSA) and probabilistic sensitivity analyses (PSA) were performed. Results For commercial payors, vibegron was cost-effective at a willingness-to-pay (WTP) threshold of $50,000/QALY versus mirabegron 50 mg (ICER, $9,311) and at a WTP threshold of $150,000/QALY versus mirabegron 25 mg (ICER, $141,957) and versus an anticholinergic basket based on market share (ICER, $118,121). For Medicare, vibegron was cost-effective at a WTP threshold of $50,000/QALY versus mirabegron 50 mg (ICER, $12,154) and at a WTP threshold of $100,000/QALY versus mirabegron 25 mg (ICER, $99,150) and versus an anticholinergic market basket (ICER, $60,756). For commercial payors and Medicare, OWSAs for vibegron versus mirabegron indicated cost-effectiveness was most sensitive to vibegron persistence at 1 and 12 months. PSAs indicated that vibegron was cost-effective versus mirabegron 50 mg 98.6% and 100% of the time at $50,000/QALY for commercial payors and Medicare payors, respectively. Limitations Due to lack of real-world data available on persistence, vibegron was assumed to have the same persistence as mirabegron 50 mg. Long-term efficacy was assumed to be sustained beyond 52 weeks in the absence of clinical trials longer than 52 weeks. Conclusions Vibegron is cost-effective from a commercial payor (WTP threshold $150,000/QALY) and Medicare (WTP threshold $100,000/QALY) perspective when compared with other oral pharmacologic treatments for OAB.