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Aims: The present analysis estimated the cost‐effectiveness of treatment with the Tandem t: slim X2 insulin pump with Control IQ technology (CIQ) in children with type 1 diabetes in Sweden. Methods: A four‐state Markov model and probabilistic sensitivity analyses (PSA) were used to assess the cost‐effectiveness of CIQ use compared with treatment with multiple daily insulin injections (MDI) or continuous subcutaneous insulin infusion (CSII) in conjunction with CGM. Data sources included clinical input data from a recent retrospective, observational study, cost data from local diabetes supply companies and government agencies, and published literature. Outcomes measures were quality adjusted life years (QALYs) at 10, 20 and 30‐year time horizons based on cost per QALY and incremental cost‐effectiveness ratio (ICER). Results: A total of 84 type 1 diabetes children were included (CIQ, n = 37; MDI, n = 19; CSII, n = 28). For all time horizons, the use of CIQ was a dominant strategy (e.g. more effective and less costly) compared with MDI or CSII use: 10‐year ICER, SEK ‐88,010.37 and SEK ‐91,723.92; 20‐year ICER, SEK −72,095.33 and SEK −87,707.79; and 30‐year ICER, SEK −65,573.01 and SEK ‐85,495.68, respectively. PSA confirmed that CIQ use was less costly compared with MDI and CSII. Conclusions: Initiation of CIQ use in children with type 1 diabetes is cost‐saving, besides previously shown improved glycaemic control, and increased quality of life. Further investigations are needed to more fully elucidate the cost‐effectiveness of these technologies in different countries with existing differences in payment models. [ABSTRACT FROM AUTHOR]

Objective: To evaluate the economic value of mepolizumab as an add-on therapy to the standard of care (SoC) for patients with severe eosinophilic asthma in China. Methods: A Markov model with three health conditions was constructed to calculate the incremental cost per quality-adjusted life year (QALY) in mepolizumab with SoC and SoC only groups from the perspective of the Chinese healthcare system throughout an entire lifespan. The model was populated with local costs, while efficacy parameters were obtained from the global Phase III MENSA trial and mortality was derived from two surveys. One-way and probabilistic sensitivity analyses were conducted. Additional scenario analysis was used to estimate the cost-effectiveness impact of changes in the price of mepolizumab. Results: Over the lifetime treatment horizon, the incremental cost-effectiveness ratio (ICER) of mepolizumab plus SoC compared to SoC alone was $170 648.73 per QALY. Sensitivity analyses focused on these results. Scenario analysis showed that mepolizumab would require a price reduction of at least 82% to reach the current willingness-to-pay (WTP=$38 223.34/QALY) threshold. Conclusion: Mepolizumab is not a cost-effective healthcare resource in China at its current pricing. [ABSTRACT FROM AUTHOR]

Abstract Objectives Since Helicopter Emergency Medical Services (HEMS) is an expensive resource in terms of unit price compared to ground-based Emergency Medical Service (EMS), it is important to further investigate which methods would allow for the optimization of these services. The aim of this study was to evaluate the cost-effectiveness of physician-staffed HEMS compared to ground-based EMS in developed scenarios with improvements in triage, aviation performance, and the inclusion of ischemic stroke patients. Methods Incremental cost-effectiveness ratio (ICER) was assessed by comparing health outcomes and costs of HEMS versus ground-based EMS across six different scenarios. Estimated 30-day mortality and quality-adjusted life years (QALYs) were used to measure health benefits. Quality-of-Life (QoL) was assessed with EuroQoL instrument, and a one-way sensitivity analysis was carried out across different patient groups. Survival estimates were evaluated from the national FinnHEMS database, with cost analysis based on the most recent financial reports. Results The best outcome was achieved in Scenario 3.1 which included a reduction in over-alerts, aviation performance enhancement, and assessment of ischemic stroke patients. This scenario yielded 1077.07–1436.09 additional QALYs with an ICER of 33,703-44,937 €/QALY. This represented a 27.72% increase in the additional QALYs and a 21.05% reduction in the ICER compared to the current practice. Conclusions The cost-effectiveness of HEMS can be highly improved by adding stroke patients into the dispatch criteria, as the overall costs are fixed, and the cost-effectiveness is determined based on the utilization rate of capacity.

Syphilis is a major sexually transmitted disease, causing a significant public health burden for countries all over the world. Since 2000, there has been a new outbreak of the syphilis epidemic in the United States. Therefore, the prevention and control of syphilis have important research significance. We have established a sex structure and ordinary differential equation model that includes men who have sex with men (MSM). Its epidemiological and biological parameters were obtained by fitting with regional monitoring data from the Centers for Disease Control and Prevention from 1984 to 2014, and the basic reproduction number ( R 0 ) of syphilis is 1.3876. Through cost-effectiveness analysis, we have found that the most cost-effective strategies in the cases of sufficient and insufficient funds are conducting syphilis screening for 50% of sexually active susceptible individuals and conducting syphilis screening for 30% of sexually active susceptible individuals while increasing the treatment rate, respectively. Therefore, in the prevention and control strategies of syphilis, measures such as increasing the coverage rate of syphilis screening for susceptible individuals and simultaneously increasing both the screening coverage rate and the treatment rate are valuable control strategy measures for reference. [ABSTRACT FROM AUTHOR]

Malaria is an old, curable vector‐borne disease that is devastating in the tropics and subtropical regions of the world. The disease has unmatched complications in the human host, especially in children. Mathematical models of infectious diseases have been the steering wheel, driving scientists towards elucidation of the dynamic behaviour of epidemics and providing tailored strategic management of diseases. With the ongoing vaccination programs for vector‐borne diseases, the research proposes a nonlinear differential equation model for the malaria disease that provides public health with a shift from the classical understanding of nonpharmaceutical preventive malaria control to pharmaceutical measures of vaccines. The asymptotic dynamic behaviour of the model is studied at the model's equilibria. The bifurcation type invoked at the disease‐free state is analysed, and the result revealed that the convention that R0<1 is the condition for eradicating the disease is not always sufficient when the system undergoes backward bifurcation. Furthermore, sensitivity analysis was investigated to quantify the amount of influence each parameter has on R0. With the Latin hypercube sampling and partial rank correlation coefficient method, the uncertainty in R0 is computed with a 95% confidence interval, with the mean, and 5th and 95th percentiles, respectively, simulated as 0.143788, 0.01545, and 0.41491. An intervention model was derived from the nonintervention model to experiment with and evaluate the respective effects of the various pairings of interventions on the dynamics of the disease. Lastly, an in‐depth cost analysis was studied to identify the most cost‐effective intervention regarding rewarding the desired outcome. From the analysis, we recommend that besides the nonpharmaceutical measure of bed nets and insecticide spray, public health should target the pharmaceutical intervention of vaccine as it can close the gap in malaria prevention. [ABSTRACT FROM AUTHOR]

Objectives: Since Helicopter Emergency Medical Services (HEMS) is an expensive resource in terms of unit price compared to ground-based Emergency Medical Service (EMS), it is important to further investigate which methods would allow for the optimization of these services. The aim of this study was to evaluate the cost-effectiveness of physician-staffed HEMS compared to ground-based EMS in developed scenarios with improvements in triage, aviation performance, and the inclusion of ischemic stroke patients. Methods: Incremental cost-effectiveness ratio (ICER) was assessed by comparing health outcomes and costs of HEMS versus ground-based EMS across six different scenarios. Estimated 30-day mortality and quality-adjusted life years (QALYs) were used to measure health benefits. Quality-of-Life (QoL) was assessed with EuroQoL instrument, and a one-way sensitivity analysis was carried out across different patient groups. Survival estimates were evaluated from the national FinnHEMS database, with cost analysis based on the most recent financial reports. Results: The best outcome was achieved in Scenario 3.1 which included a reduction in over-alerts, aviation performance enhancement, and assessment of ischemic stroke patients. This scenario yielded 1077.07–1436.09 additional QALYs with an ICER of 33,703-44,937 €/QALY. This represented a 27.72% increase in the additional QALYs and a 21.05% reduction in the ICER compared to the current practice. Conclusions: The cost-effectiveness of HEMS can be highly improved by adding stroke patients into the dispatch criteria, as the overall costs are fixed, and the cost-effectiveness is determined based on the utilization rate of capacity. [ABSTRACT FROM AUTHOR]

Strokes remain a leading cause of death and disability worldwide. The STROKE OWL study evaluated a novel case management approach for patients with stroke (modified Rankin Scale 0–4) or transient ischemic attack (TIA) who received support across healthcare settings and secondary prevention training from case managers for one year. The primary aim of this quasi-experimental study was a reduction in stroke recurrence. Here, we report the results of a health economic analysis of the STROKE OWL study, conducted in accordance with CHEERS guidelines. The calculations were based on claims data of cooperating statutory health insurance companies. In addition to a regression analysis for cost comparison, the incremental cost-effectiveness ratio was determined, and a probabilistic sensitivity analysis was carried out. In total, 1167 patients per group were included in the analysis. The intervention group incurred 32.3% higher direct costs (p < 0.001) than the control group. With a difference of EUR 1384.78 (95% CI: [1.2384–1.4143], p < 0.0001) and a 5.32% increase in hazards for the intervention group (HR = 1.0532, 95% CI: [0.7869–1.4096], p = 0.7274) resulting in an ICER of EUR 260.30, we found that the case management intervention dominated in the total stroke population, even for an arbitrarily high willingness to pay. In the TIA subgroup, however, the intervention was cost-effective even for a low willingness to pay. Our results are limited by small samples for both TIA and severe stroke patients and by claims data heterogeneity for some cost components, which had to be excluded from the analysis. Future research should investigate the cost-effectiveness of case management interventions for both severe stroke and TIA populations using appropriate data. [ABSTRACT FROM AUTHOR]

This paper is a novel attempt to analyze different aspects of a two-strain epidemic model. The paper introduces a novel approach to analyzing a two-strain epidemic model, emphasizing the efficacy of combining non-pharmaceutical interventions and vaccination, particularly against new variants. Additionally, it presents a unique spatio-temporal model to assess spatial distribution of infections, offering fresh insights into how spatial factors influence disease transmission and control. The analysis involves stability analysis(local and global) and optimal control. Time-dependent social/non-pharmaceutical interventions coupled with vaccination of the susceptible class in the presence of both strains are analyzed using Pontryagin’s Maximum Principle. The numerical section shows the behavior of the infected class with and without control, the control intensity trend for the scenario when only non-pharmaceutical interventions (NPI) are practiced for the original strain, and when both NPI and vaccination are incorporated with the emergence of the new strain. The evaluation of the Incremental average ratio (IAR) and Incremental cost-effectiveness ratio (ICER) determines that NPI and vaccination as a combination is better and ideal in terms of costs incurred and effectiveness as a whole in averting infection in the presence of a new variant. Finally, we have also proposed a spatio-temporal pattern for the new strain model to analyze patterns using the finite element method by PDE Toolbox to show the effect of different initial conditions and geometry on the density of the infected population.

Countries face challenges in paying for new drugs. High prices are driven in part by exploding drug development costs, which, in turn, are driven by essential but excessive regulation. Burdensome regulation also delays drug development, and this can translate into thousands of life-years lost. We need system-wide reform that will enable less expensive, faster drug development. The speed with which COVID-19 vaccines and AIDS therapies were developed indicates this is possible if governments prioritize it. Countries also differ in how they value drugs, and generally, those willing to pay more have better, faster access. Canada is used as an example to illustrate how “incremental cost-effectiveness ratios” (ICERs) based on measures such as gains in “quality-adjusted life-years” (QALYs) may be used to determine a drug’s value but are often problematic, imprecise assessments. Generally, ICER/QALY estimates inadequately consider the impact of patient crossover or long post-progression survival, therapy benefits in distinct subpopulations, positive impacts of the therapy on other healthcare or societal costs, how much governments willingly might pay for other things, etc. Furthermore, a QALY value should be higher for a lethal or uncommon disease than for a common, nonlethal disease. Compared to international comparators, Canada is particularly ineffective in initiating public funding for essential new medications. Addressing these disparities demands urgent reform.

Countries face challenges in paying for new drugs. High prices are driven in part by exploding drug development costs, which, in turn, are driven by essential but excessive regulation. Burdensome regulation also delays drug development, and this can translate into thousands of life-years lost. We need system-wide reform that will enable less expensive, faster drug development. The speed with which COVID-19 vaccines and AIDS therapies were developed indicates this is possible if governments prioritize it. Countries also differ in how they value drugs, and generally, those willing to pay more have better, faster access. Canada is used as an example to illustrate how "incremental cost-effectiveness ratios" (ICERs) based on measures such as gains in "quality-adjusted life-years" (QALYs) may be used to determine a drug's value but are often problematic, imprecise assessments. Generally, ICER/QALY estimates inadequately consider the impact of patient crossover or long post-progression survival, therapy benefits in distinct subpopulations, positive impacts of the therapy on other healthcare or societal costs, how much governments willingly might pay for other things, etc. Furthermore, a QALY value should be higher for a lethal or uncommon disease than for a common, nonlethal disease. Compared to international comparators, Canada is particularly ineffective in initiating public funding for essential new medications. Addressing these disparities demands urgent reform. [ABSTRACT FROM AUTHOR]

Background Renal cell carcinoma is the most common type of kidney cancer, comprising approximately 85% of all renal malignancies. Patients with advanced renal cell carcinoma are the focus of this National Institute for Health and Care Excellence multiple technology appraisal. A patient’s risk of disease progression depends on a number of prognostic risk factors; patients are categorised as having intermediate/poor risk or favourable risk of disease progression. Objectives The objectives of this multiple technology appraisal were to appraise the clinical effectiveness and cost-effectiveness of lenvatinib plus pembrolizumab versus relevant comparators listed in the final scope issued by the National Institute for Health and Care Excellence: sunitinib, pazopanib, tivozanib, cabozantinib and nivolumab plus ipilimumab. Methods The assessment group carried out clinical and economic systematic reviews and assessed the clinical and cost-effectiveness evidence submitted by Eisai, Hatfield, Hertfordshire, UK (the manufacturer of lenvatinib) and Merck Sharp & Dohme, Whitehouse Station, NJ, USA (the manufacturer of pembrolizumab). The assessment group carried out fixed-effects network meta-analyses using a Bayesian framework to generate evidence for clinical effectiveness. As convergence issues occurred due to sparse data, random-effects network meta-analysis results were unusable. The assessment group did not develop a de novo economic model, but instead modified the partitioned survival model provided by Merck Sharp & Dohme. Results The assessment group clinical systematic review identified one relevant randomised controlled trial (CLEAR trial). The CLEAR trial is a good-quality, phase III, multicentre, open-label trial that provided evidence for the efficacy and safety of lenvatinib plus pembrolizumab compared with sunitinib. The assessment group progression-free survival network meta-analysis results for all three risk groups should not be used to infer any statistically significant difference (or lack of statistically significant difference) for any of the treatment comparisons owing to within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption. The assessment group overall survival network meta-analysis results for the intermediate-/poor-risk subgroup suggested that there was a numerical, but not statistically significant, improvement in the overall survival for patients treated with lenvatinib plus pembrolizumab compared with patients treated with cabozantinib or nivolumab plus ipilimumab. Because of within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption, the assessment group overall survival network meta-analysis results for the favourable-risk subgroup and the all-risk population should not be used to infer any statistically significant difference (or lack of statistically significant difference) for any of the treatment comparisons. Only one cost-effectiveness study was included in the assessment group review of cost-effectiveness evidence. The study was limited to the all-risk population, undertaken from the perspective of the US healthcare system and included comparators that are not recommended by the National Institute for Health and Care Excellence for patients with untreated advanced renal cell carcinoma. Therefore, the extent to which resource use and results are generalisable to the NHS is unclear. The assessment group cost-effectiveness results from the modified partitioned survival model focused on the intermediate-/poor-risk and favourable-risk subgroups. The assessment group cost-effectiveness results, generated using list prices for all drugs, showed that, for all comparisons in the favourable-risk subgroup, treatment with lenvatinib plus pembrolizumab costs more and generated fewer benefits than all other treatments available to NHS patients. For the intermediate-/poor-risk subgroup, treatment with lenvatinib plus pembrolizumab costs more and generated more benefits than treatment with cabozantinib and nivolumab plus ipilimumab. Conclusions Good-quality clinical effectiveness evidence for the comparison of lenvatinib plus pembrolizumab with sunitinib is available from the CLEAR trial. For most of the assessment group Bayesian hazard ratio network meta-analysis comparisons, it is difficult to reach conclusions due to within-trial proportional hazards violations or uncertainty regarding the validity of the proportional hazards assumption. However, the data (clinical effectiveness and cost-effectiveness) used to populate the economic model are relevant to NHS clinical practice and can be used to inform National Institute for Health and Care Excellence decision-making. The assessment group cost-effectiveness results, generated using list prices for all drugs, show that lenvatinib plus pembrolizumab is less cost-effective than all other treatment options. Study registration This study is registered as PROSPERO CRD4202128587. Funding This award was funded by the National Institute for Health and Care Research (NIHR) Evidence Synthesis Programme (NIHR award ref: NIHR134985) and is published in full in Health Technology Assessment; Vol. 28, No. 49. See the NIHR Funding and Awards website for further award information. Plain language summary What was the problem? Renal cell carcinoma is the most common type of kidney cancer. Several drug treatment options are available for NHS patients with advanced or metastatic disease, and the choice of treatment varies depending on a patient’s risk of disease progression. A new drug combination, lenvatinib plus pembrolizumab, may soon become available to treat NHS patients. This review explored whether treatment with lenvatinib plus pembrolizumab offered value for money to the NHS. What did we do? We reviewed the effectiveness of treatment with lenvatinib plus pembrolizumab versus other NHS treatment options. We also estimated the costs and benefits of treatment with lenvatinib plus pembrolizumab versus current NHS treatments for patients with higher and lower risks of disease progression. What did we find? Compared with current NHS treatments, treatment with lenvatinib plus pembrolizumab may increase the time that people with a higher risk of disease progression (i.e. worsening disease) were alive. However, for patients with a lower risk of disease progression, the available evidence is limited and only shows that treatment with lenvatinib plus pembrolizumab may prolong the time that patients have a stable level of disease. For all patients, compared to all current NHS treatments, treatment with lenvatinib plus pembrolizumab is very expensive. What does this mean? Compared with current NHS treatments for untreated renal cell carcinoma, using published prices (which do not include any discounts that are offered to the NHS), treatment with lenvatinib plus pembrolizumab may not provide good value for money to the NHS. Scientific summary Background Renal cell carcinoma (RCC) is the most common type of kidney cancer, comprising approximately 85% of all renal malignancies. Patients with advanced RCC (aRCC) have Stage 3 (locally advanced) or Stage 4 (metastatic) disease. A patient’s risk of disease progression depends on a number of prognostic risk factors. The International Metastatic Renal Cell Carcinoma Database Consortium (IMDC) model is used in NHS clinical practice to categorise patients into one of two groups, namely favourable risk or intermediate/poor risk. This systematic review and cost-effectiveness analysis has been conducted to inform the following National Institute for Health and Care Excellence (NICE) multiple technology appraisal: lenvatinib with pembrolizumab for untreated aRCC (ID3760). In November 2021, the Medicines and Healthcare products Regulatory Agency approved the use of lenvatinib plus pembrolizumab as a treatment for all patients with untreated aRCC. Objectives The comparators listed in the final scope issued by NICE differ depending on the risk of disease progression. The objectives of this assessment were to appraise the clinical effectiveness and cost-effectiveness of lenvatinib plus pembrolizumab versus: cabozantinib and nivolumab plus ipilimumab in the intermediate-/poor-risk subgroup sunitinib, pazopanib and tivozanib in the favourable-risk subgroup sunitinib, pazopanib and tivozanib in the all-risk population. Clinical and economic systematic review methods The assessment group (AG) carried out a systematic review of clinical effectiveness evidence following the general principles outlined by the Centre for Reviews and Dissemination (CRD). The review was reported using the criteria recommended in the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement. Searches were conducted between 11 October 2021 and 22 November 2021 in accordance with the general principles recommended by the European Network for Health Technology Assessment. The protocol is registered with PROSPERO (registration number: CRD42021285879). The AG reviewed only randomised controlled trials (RCTs) and full economic analyses identified by the searches. However, the group also considered evidence provided by the manufacturers of lenvatinib (Eisai Ltd) and pembrolizumab (Merck Sharp & Dohme, Whitehouse Station, NJ, USA) provided in submissions to NICE; company submission (CS) reference lists were searched for relevant RCTs. In line with the final scope issued by NICE, the outcomes considered by the AG were overall survival (OS), progression-free survival (PFS), objective tumour response rate, adverse events (AEs), health-related quality of life (HRQoL), incremental cost per life-year gained and incremental cost per quality-adjusted life-year (QALY) gained. Clinical effectiveness results Direct clinical effectiveness evidence (CLEAR trial) The AG systematic review included one RCT, the CLEAR trial. The CLEAR trial was a good-quality, phase III, multicentre, open-label RCT (with an ongoing extension phase) that provided evidence for the comparison of the efficacy of lenvatinib plus pembrolizumab versus sunitinib. Results for all outcomes were assessed at the third interim analysis (August 2020, median OS follow-up of 26.6 months), that is the final data cut-off for PFS. The companies also presented OS results from an updated OS analysis (March 2021, median OS follow-up of approximately 33 months). At the time of the third interim analysis, the CLEAR trial hazard ratio (HR) results showed statistically significant improvements in PFS and objective tumour response rate for patients treated with lenvatinib plus pembrolizumab versus patients treated with sunitinib for the intermediate-/poor-risk subgroup, the favourable-risk subgroup and the all-risk population. The HR results from the updated OS analysis showed a statistically significant improvement for patients treated with lenvatinib plus pembrolizumab versus patients treated with sunitinib for the intermediate-/poor-risk subgroup and the all-risk population; there were too few events in the favourable-risk subgroup for robust OS conclusions to be drawn. Eisai carried out a treatment-switching analysis to test whether adjusting for the effect of subsequent treatments affected OS results. Results were generated only for the all-risk population and were marked as academic-in-confidence. Nearly all the patients in the CLEAR trial lenvatinib plus pembrolizumab and sunitinib arms experienced at least one all-grade AE, with more Grade ≥ 3 AEs reported in the lenvatinib plus pembrolizumab arm than in the sunitinib arm. The proportion of patients who discontinued treatment of either lenvatinib or pembrolizumab due to AEs was approximately twice as high as patients who discontinued treatment of sunitinib; the proportion of patients who withdrew treatment of both lenvatinib and pembrolizumab due to AEs was approximately the same as the proportion of patients who withdrew treatment with sunitinib. Health-related quality of life was measured using three tools, including the EuroQol-5 Dimensions, three-level version questionnaire. When compared with treatment with sunitinib, treatment with lenvatinib plus pembrolizumab did not result in any clinically meaningful differences (as measured by predefined minimally important differences) in HRQoL measured using any of the three tools. Indirect clinical effectiveness evidence To compare the effectiveness of lenvatinib plus pembrolizumab versus relevant comparators other than sunitinib, the AG carried out Bayesian HR network meta-analyses. It was decided not to undertake a flexible modelling approach for network meta-analysis (NMA), which relaxes the proportional hazards (PH) assumption, such as fractional polynomial network meta-analyses because interpretation of the estimates provided by these complex modelling techniques can be difficult and results are often not intuitive. While deviance information criterion (DIC) statistics provide an approach to compare the fit of different models, they do not provide information about whether a model is a good fit to the data or whether the estimates generated by the model, including projections of results beyond the follow-up times of trials included in the NMA, are clinically plausible. Furthermore, flexible models, which appear similar according to model fit (i.e. according to DIC statistics), may generate very different long-term survival estimates. The AG assessed the feasibility of conducting Bayesian HR NMAs for the three population risk groups (intermediate-/poor-risk subgroup, favourable-risk subgroup and all-risk population) for all outcomes listed in the final scope issued by NICE. However, due to limited data availability, it was not possible to carry out NMAs for all outcomes for all three patient risk groups. Further, as networks were sparse, it was only possible to generate results using fixed-effect NMAs. The AG PFS NMA results for the intermediate-/poor-risk subgroup, the favourable-risk subgroup and the all-risk population should not be used to infer any statistically significant difference (or lack of statistically significant difference) for any of the treatment comparisons because of within-trial PH violations or uncertainty regarding the validity of the PHs assumption. The AG OS NMA results for the intermediate-/poor-risk subgroup suggested that there was a numerical, but not statistically significant, improvement in the OS for patients treated with lenvatinib plus pembrolizumab compared with patients treated with cabozantinib or nivolumab plus ipilimumab. Because of within-trial PH violations or uncertainty regarding the validity of the PH assumption, the AG OS NMA results for the favourable-risk subgroup and the all-risk population should not be used to infer any statistically significant difference (or lack of statistically significant difference) for any of the treatment comparisons. The AG objective tumour response rate NMA results for the intermediate-/poor-risk subgroup suggested that, although treatment with lenvatinib plus pembrolizumab led to a statistically significant improvement in objective tumour response rate compared to treatment nivolumab plus ipilimumab, it did not lead to a statistically significant improvement in objective tumour response rate for the comparison of lenvatinib plus pembrolizumab versus cabozantinib. It was not possible to generate results for the IMDC/MSKCC (Memorial Sloan-Kettering Cancer Center) favourable-risk subgroup due to data limitations. The AG objective tumour response rate NMA results for the all-risk population suggest that treatment with lenvatinib plus pembrolizumab led to a statistically significant improvement in objective tumour response rate versus treatment with sunitinib and versus treatment with pazopanib. The AG Grade ≥ 3 AE NMA results for the intermediate-/poor-risk subgroup suggested that treatment with lenvatinib plus pembrolizumab led to statistically significantly more Grade ≥ 3 AEs versus treatment with cabozantinib. It was not possible to generate results for the IMDC/MSKCC favourable-risk subgroup. The AG Grade ≥ 3 AE NMA results for the all-risk population suggested that treatment with lenvatinib led to statistically significantly more Grade ≥ 3 AEs versus treatment with sunitinib and versus treatment with pazopanib. Economic systematic review results The AG systematic review identified one relevant cost-effectiveness study. This study compared the cost-effectiveness of lenvatinib plus pembrolizumab versus sunitinib (and vs. other treatments). However, the study was undertaken from the perspective of the US healthcare system and generated results only for the all-risk population and included comparators that are not recommended by NICE as treatment options for patients with aRCC. Therefore, the extent to which these results were generalisable to the NHS was unclear. Cost-effectiveness analysis methods The Eisai and Merck Sharp & Dohme CSs to NICE included partitioned survival models built in Microsoft Excel. The AG considered that results from both models could be used to inform decision-making but that, in some instances, the companies could have made more appropriate assumptions and parameter choices. The AG did not develop a de novo economic model; instead, it modified the model provided by Merck Sharp & Dohme [referred to as the Merck Sharp & Dohme/Assessment Group (MSD/AG) model]. Neither of the companies produced cost-effectiveness results for the comparison of lenvatinib plus pembrolizumab versus nivolumab plus ipilimumab (intermediate-/poor-risk subgroup) despite both models having the functionality for this comparison. Furthermore, Eisai did not generate any cost-effectiveness results for the favourable-risk subgroup. The MSD/AG model was populated with OS, PFS and time to treatment discontinuation (TTD) data from the CLEAR trial (lenvatinib plus pembrolizumab versus sunitinib for favourable-risk subgroup and the all-risk population). The AG PFS and OS NMA results were used to estimate effectiveness for the comparison of lenvatinib plus pembrolizumab versus cabozantinib and versus nivolumab plus ipilimumab for the intermediate-/poor-risk population. NICE appraisal committees have concluded that sunitinib and pazopanib are of equivalent effectiveness and that, at best, tivozanib may have a similar effect to sunitinib or pazopanib. These conclusions were based on all-risk population data; the AG has assumed that this assumption holds for the favourable-risk population. The most important changes made by the AG to the Merck Sharp & Dohme model were different choices for estimating PFS, OS and TTD for the intervention and comparator treatments and for modelling two lines, rather than one line, of subsequent treatment. Cost-effectiveness analysis results The AG cost-effectiveness results presented in this report were estimated using list prices. Also, the AG cost-effectiveness results generated using confidential discounted prices were supplied to NICE in a confidential appendix, but cannot be presented here. For the intermediate-/poor-risk subgroup, the AG base-case cost-effectiveness results suggested that treatment with lenvatinib plus pembrolizumab generated more QALYs versus treatment with cabozantinib and versus nivolumab plus ipilimumab, but at a greater overall cost than either of these two treatments. Using list prices, the incremental cost-effectiveness ratios per QALY gained for the comparison of lenvatinib plus pembrolizumab versus cabozantinib and versus nivolumab plus ipilimumab exceed £100,000. For the favourable-risk subgroup, the AG base-case cost-effectiveness results suggested that treatment with sunitinib generated more QALYs than treatment with lenvatinib plus pembrolizumab at a lower overall cost, that is treatment with lenvatinib plus pembrolizumab was dominated by treatment with sunitinib (and, using the assumption of equivalent effectiveness, by pazopanib and tivozanib). The AG carried out extensive one-way sensitivity analyses, scenario analyses and probabilistic sensitivity analyses. Results from these analyses demonstrate that the AG base-case cost-effectiveness results are robust. Clinical and cost-effectiveness conclusions Good-quality clinical effectiveness evidence for the comparison of lenvatinib plus pembrolizumab versus sunitinib was available from the CLEAR trial. For most of the AG Bayesian HR NMA comparisons, it was difficult to reach conclusions due to within-trial PH violations or uncertainty regarding the validity of the PHs assumption. However, the data (clinical effectiveness and cost-effectiveness) used to populate the MSD/AG model are relevant to NHS clinical practice and can be used to inform NICE decision-making. The all-risk population comprises patients with intermediate-/poor-risk and patients with favourable-risk disease. The AG cost-effectiveness analyses have focused on the two subgroups, and the AG cost-effectiveness results, generated using list prices for all drugs, show that lenvatinib plus pembrolizumab is less cost-effective than all other treatment options. Study registration This study is registered as PROSPERO CRD4202128587. Funding This award was funded by the National Institute for Health and Care Research (NIHR) Evidence Synthesis Programme (NIHR award ref: NIHR134985) and is published in full in Health Technology Assessment; Vol. 28, No. 49. See the NIHR Funding and Awards website for further award information.

The Malaria epidemic is a serious public health issue that is worrying to the affected people in the tropics and subtropical parts of the world. The alarming consequences of the disease have prompted the scientific communities to seek better ways to manage the disease. In Ghana, the accurate statistical estimate of the Malaria transmission parameters is paramount to the public Health to embark on a transmission reduction program but rarely exists. The study’s purpose is to undertake a comprehensive mathematical analysis of a newly designed compartmentalized vector-host Malaria model capturing the aquatic mosquito vector compartment and vaccinated human host for the goal of estimating the model’s parameters and the threshold R0 computation and examining the associated bifurcation type that invokes at the Malaria-free state. The study thoroughly analyses the asymptomatic stability of the model to classify the respective behaviours at the equilibria. A sensitivity study of LHS-PRCC was undertaken to measure the variability in the threshold R0. Further, the model was subjected to a comprehensive analysis to assess the impact of vaccines on the dynamics of the disease. The analysis showed that vaccines have a substantial impact in reducing the Malaria cases in Ghana. To have a more reliable and accurate estimation of the model parameters of Ghana and robust mitigating intervention protocols for public health officials, the Least square fitting approach is applied to data of Ghana for an extended period of 10 years, starting from 2010 to 2020, to estimate the model’s parameters. The Malaria model was further structured into an intervention model to provide tailored eradication strategies for curtailing the disease. To exemplify the cost-effective procedure for assessing the cost related to the identified interventions, the ACER and ICER were used to analyse the cost of each intervention per the benefit. The results from the analyses suggest that increasing accessibility and executing a control intervention of 8 as identified by the cost analysis by Public Health will assist in mitigating the disease with a comparatively minimal cost.

Microvascular complications can occur due to type 2 diabetes mellitus (DM). The cost of therapy increases due to the use of various antidiabetic drugs over a long period. This research is a pharmacoeconomic study that uses the Markov Model to compare costs and utilities in type 2 DM patients who receive metformin+glimepiride and metformin+vildagliptin therapy. Primary data was collected through the EQ5D questionnaire at Cirebon Harbor Hospital and secondary data (transition probability) was collected through a literature study. Characteristics of type 2 DM patients show that the majority of people suffering from this condition are aged between 55 and 59 years. Primary data from the hospital shows that the cost of therapy for controlled type 2 DM patients who receive combination therapy of metformin + glimepiride, and metformin + vildagliptin is Rp. 1,699,400, Rp. 5,807,786, and Rp. 10,100,426, respectively. Secondary data from articles treating type 2 DM shows that the 55-59 year age group is the largest. With an ICER of 61,493,943/QALYS, microvascular prevention in T2DM patients receiving the metformin+vildagliptin combination in Indonesia is considered effective and able to reduce the number of microvascular cases. This is based on the ICER value compared to the Indonesian Government's GDP, namely Rp. 71,030,183.3. This study shows that replacing glimepiride with vildagliptin for the prevention of microvascular complications is cost-effective and has better utility and effectiveness in reducing the incidence of microvascular complications. The use of vildagliptin can be recommended to reduce costs and the risk of microvascular complications.

Introduction Studies on economic evaluations of the 13-valent pneumococcal conjugate vaccine (PCV13) have been increasing over the last decade. No systematic reviews have synthesized the evidence of economic evaluations of the PCV13. Areas covered We systematically searched the literature which published on peer-reviewed journals from January 2010 to June 2022. The literature search was conducted in the following electronic databases: PubMed, Web of Science, Embase, the Cochrane Library, CNKI, Wanfang database, VIP database. We identified 1827 records from the database search. After excluding 511 duplicates, 1314 records were screened, of which 156 records were retained for the full-text reviews. A total of 44 studies were included in the review. Among the included studies, 33 studies were economic evaluations of PCV13 among children, and 11 studies were conducted among adults. The literature search initiated in April, 2022, and updated in June 2022. Expert opinion Vaccination with PCV13 was found to significantly reduce the mortality and morbidity of pneumococcal diseases and was cost-effective compared to no vaccine or several other pneumococcal vaccines (e.g. PCV10, PPV23). Future research is advised to expand economic evaluations of PCV13 combined with dynamic model to enhance methodologic rigor and prediction accuracy.

Purpose: Arthroscopic Bankart repair (ABR) may be more effective than nonoperative management for patients with anterior shoulder instability following first-time dislocation. The purpose of the study was to determine the most cost-effective treatment strategy by evaluating the incremental cost-effectiveness ratio (ICER) for ABR versus nonoperative treatment. Methods: This cost-effectiveness study utilized a Markov decision chain and Monte Carlo simulation. Probabilities, health utility values, and outcome data regarding ABR and nonoperative management of first-time shoulder instability derived from level I/II evidence. Costs were tabulated from Centers for Medicaid & Medicare Services. Probabilistic sensitivity analysis was performed using >100,000 repetitions of the Monte Carlo simulation. A willingness-to-pay (WTP) threshold was set at $50,000. Results: The expected cost for operative management higher than nonoperative management ($32,765 vs $29,343). However, ABR (5.48 quality-adjusted life years (QALYs)) was the more effective treatment strategy compared to nonoperative management (4.61 QALYs). The ICER for ABR was $3943. Probabilistic sensitivity analysis showed that ABR was the most cost-effective strategy in 100% of simulations. Discussion: ABR is more cost-effective than nonoperative management for first-time anterior shoulder dislocation. The threshold analysis demonstrated that when accounting for WTP, ABR was found to be the more cost-effective strategy. [ABSTRACT FROM AUTHOR]

Background: Cervical cancer is a common malignancy causing cancer-related deaths worldwide. Evidence suggests that adequate screening can reduce cervical cancer-related deaths. Screening for cervical cancer can be done using Visual Inspection of Acetic Acid (VIA), HPV DNA and cytology. The economic burden of cancer patients is substantial ranging from US$8,066 to 22,888 per patient. Health cost effectiveness was comparative in the analysis of costs and consequences for the three screening methods. VIA is the most cost-effective primary screening test for cervical cancer. Purpose: To review the research results of a full economic evaluation to encourage the specific cervical cancer screening methods used to enhance the detection of precancerous lesions in women's cervix. Method: This systematic review used the PRISMA ScR framework with a literature search using 3 databases, namely Pubmed, Wiley, and Google Scholar, with years of publication from 2009 – 2023. The data that has been obtained is carried out by critical appraisal using the Consolidated Health Economic Evaluation Reporting Standards (CHEERS). Results: Based on the search for 3,973 selected articles, 7 match the inclusion criteria. This systematic review discovered three major themes: the utility value of health status, cost and analysis. Conclusion: The most cost-effective cervical cancer screening is by VIA. HPV DNA and pap smear based cytology techniques have been reported to show high sensitivity and specificity respectively including being expensive and resource intensive. In contrast the VIA technique has moderate sensitivity and specificity but is less expensive Various developed countries have used Pap or HPV DNA cytology tests as the main screening method, which can lead to a 50-70% reduction in cervical cancer incidence, while in developing countries using VIA tests as a cervical cancer screening method.

Strokes remain a leading cause of death and disability worldwide. The STROKE OWL study evaluated a novel case management approach for patients with stroke (modified Rankin Scale 0–4) or transient ischemic attack (TIA) who received support across healthcare settings and secondary prevention training from case managers for one year. The primary aim of this quasi-experimental study was a reduction in stroke recurrence. Here, we report the results of a health economic analysis of the STROKE OWL study, conducted in accordance with CHEERS guidelines. The calculations were based on claims data of cooperating statutory health insurance companies. In addition to a regression analysis for cost comparison, the incremental cost-effectiveness ratio was determined, and a probabilistic sensitivity analysis was carried out. In total, 1167 patients per group were included in the analysis. The intervention group incurred 32.3% higher direct costs (p < 0.001) than the control group. With a difference of EUR 1384.78 (95% CI: [1.2384–1.4143], p < 0.0001) and a 5.32% increase in hazards for the intervention group (HR = 1.0532, 95% CI: [0.7869–1.4096], p = 0.7274) resulting in an ICER of EUR 260.30, we found that the case management intervention dominated in the total stroke population, even for an arbitrarily high willingness to pay. In the TIA subgroup, however, the intervention was cost-effective even for a low willingness to pay. Our results are limited by small samples for both TIA and severe stroke patients and by claims data heterogeneity for some cost components, which had to be excluded from the analysis. Future research should investigate the cost-effectiveness of case management interventions for both severe stroke and TIA populations using appropriate data.

In this latest update, we explore some of the key updates in market access over recent months including the UK’s voluntary scheme for branded medicines pricing, access and growth (VPAG), the first drugs funded by the Innovative Medicines Fund in the UK and the Direct Access Scheme in France, and, finally, the new Institute for Clinical and Economic Review (ICER) value assessment framework in the USA.

Background: Prolactinoma, the most common pituitary adenoma, is usually treated with dopamine agonist (DA) therapy like cabergoline. Surgery is second-line therapy, and radiotherapy is used if surgical treatment fails or in relapsing macroprolactinoma. Objective: This study aimed to provide economic evidence for the management of prolactinoma in Italy, using a cost-of-illness and cost-utility analysis that considered various treatment options, including cabergoline, bromocriptine, temozolomide, radiation therapy, and surgical strategies. Methods: The researchers conducted a systematic literature review for each research question on scientific databases and surveyed a panel of experts for each therapeutic procedure’s specific drivers that contributed to its total cost. Results: The average cost of the first year of treatment was €2,558.91 and €3,287.40 for subjects with microprolactinoma and macroprolactinoma, respectively. Follow-up costs from the second to the fifth year after initial treatment were €798.13 and €1,084.59 per year in both groups. Cabergoline had an adequate cost-utility profile, with an incremental cost-effectiveness ratio (ICER) of €3,201.15 compared to bromocriptine, based on a willingness-to-pay of €40,000 per quality-adjusted life year (QALY) in the reference economy. Endoscopic surgery was more cost-effective than cabergoline, with an ICER of €44,846.64. Considering a willingness-to-pay of €40,000/QALY, the baseline findings show cabergoline to have high cost utility and endoscopic surgery just a tad above that. Conclusions: Due to the favorable cost-utility profile and safety of surgical treatment, pituitary surgery should be considered more frequently as the initial therapeutic approach. This management choice could lead to better outcomes and an appropriate allocation of healthcare resources.

Treatment guidelines are key drivers of prescribing practice in the management of Clostridioides difficile infection (CDI), but recommendations on best practice can vary. We conducted a cost-utility analysis to compare the treatment pathway recommended by the European Society of Clinical Microbiology and Infectious Diseases (ESCMID) guideline with the pathway proposed by the National Institute for Health and Care Excellence (NICE) guideline, from the perspective of the UK National Health Service. A decision tree modelling approach was adopted to reflect the treatment pathway for CDI as outlined in ESCMID and NICE guidelines. Patients experiencing a CDI infection received up to three treatments per infection to achieve a response and could subsequently experience up to two recurrences. Data on patient demographics, treatment response, recurrence, utilities, CDI-related mortality, and costs were taken from published literature. The ESCMID treatment pathway was cost-effective versus the NICE treatment pathway at a threshold of £20 000 per quality-adjusted life year gained, with an incremental cost-effectiveness ratio of £4931. Cost-effectiveness was driven by differences in index infection recommendations (ESCMID recommends fidaxomicin as first-line treatment whereas NICE recommends vancomycin). The model results were robust to variations in inputs investigated in scenarios and sensitivity analyses, and probabilistic sensitivity analysis demonstrated that the ESCMID guideline treatment strategy had a 100% likelihood of being cost-effective versus the NICE treatment strategy. Compared with the NICE guideline, the ESCMID guideline recommendations for treating an index CDI represent the most cost-effective use of healthcare resources from the perspective of the UK National Health Service. [ABSTRACT FROM AUTHOR]

Background & aimsPatients with solid tumors who undergo chemotherapy have an increased risk of hepatitis B virus (HBV) reactivation, but a low proportion of these patients are screened for HBV infection and guidelines make conflicting recommendations. Further, the cost-effectiveness of newer treatments for HBV prophylaxis has not been examined for this population. We aimed to analyze the cost-effectiveness of HBV screening before chemotherapy for patients with solid tumors.MethodsWe compared 3 HBV screening strategies (screen all, screen only high-risk patients, or screen none) using a Markov model of a population of adults in the United States who initiated chemotherapy for a solid tumor. We modeled use of entecavir prophylaxis for HB surface antigen (HBsAg)-positive patients and surveillance for HBsAg-negative patients who are positive for HBV core antibody. The Markov cycle length was 1 year, with model simulation for up to 5 years.ResultsThe screen all strategy was the most cost effective, with an incremental cost-effectiveness ratio of $42,761 compared to screening only high-risk patients. The screen none strategy was less effective and less costly than screening all patients or only high-risk patients. The screen-all strategy was the most cost effective for all estimates of prevalence of HBsAg-positive patients and estimates of HBV reactivation in HBsAg-positive patients. Screening only high-risk patients was the most cost-effective strategy when more than 25% of high-risk patients were screened for HBV infection.ConclusionsIn a Markov model analysis, we found screening all patients with solid tumors for HBV infection before chemotherapy to be the most cost-effective strategy. Guidelines should consider recommending HBV tests for patients initiating chemotherapy.

Background: The rising cost of cancer drug therapy threatens the long-term sustainability of Taiwan National Health Insurance. Cost savings can be achieved through various strategies, e.g., using smaller vial sizes, sharing vials, weight-based dosing, or switching to biosimilars. Here we aimed to examine the cost-effectiveness of a trastuzumab biosimilar combined with docetaxel (TDbiol) for treatment-naïve HER2+ metastatic breast cancer (MBC), and the financial impact of drug wastage. Methods: A Markov model with three health states was developed to assess the cost-effectiveness of trastuzumab biosimilars plus docetaxel over a 40-month time horizon in patients with HER2+ MBC. Based on the literature and our expert opinion, we assumed similar efficacy between the trastuzumab biosimilar and its reference product. The primary clinical input for the biosimilar was the same as for the reference product in the Catastrophic Patient Database (HV). Health state utilities were derived from the literature, and direct medical costs were obtained from the National Health Insurance Administration (NHIA). Results: In the base-case scenario, the incremental cost-effectiveness ratio (ICER) was NTD 811,050 per QALY gained. One-way sensitivity analyses showed that the model was sensitive to utilities and transition probabilities, but not particularly sensitive to the wastage assumption. In scenario analyses, the ICER was higher when applying the price for trastuzumab reference biologic (branded), than for trastuzumab biosimilar. Conclusion: The trastuzumab biosimilar combination regimen is cost-effective and offers significant drug cost savings in Taiwan.

Background: Severe pediatric allergic asthma (SPAA) induces a huge economic burden in terms of direct, indirect, and intangible costs. The use of omalizumab for the treatment of these patients has produced a significant improvement in several clinical outcomes, but at the same time, the cost for the management of the disease has also increased. The aim of this report was to evaluate whether the use of omalizumab is cost‐effective. Methods: A sample of 426 children with SPAA from the ANCHORS (Asthma iN CHildren: Omalizumab in Real‐life in Spain) study was used to calculate the incremental cost‐effectiveness ratio (ICER) for the avoidance of moderate‐to‐severe exacerbations (MSE) and also for the improvement in childhood Asthma Control Test (c‐ACT) or the Asthma Control Questionnaire (ACQ5). We retrospectively collected data on health encounters and drug consumption before and up to 6 years after the beginning of the treatment with omalizumab. Results: The ICER per avoided MSE was €2107 after 1 year, and it consistently decreased to €656 in those followed up to 6 years. Similarly, the ICER for the minimally important difference in control tests showed a decrease from €2059 to €380 per each 0.5 points of improvement in ACQ5 and from €3141 to €2322 per each 3 points improvement in c‐ACT, at years 1 and 6, respectively. Conclusion: The use of OMZ is a cost‐effective option for most children with uncontrolled SPAA, especially those who have frequent exacerbations; the costs are progressively reduced in successive years of treatment. [ABSTRACT FROM AUTHOR]

The global impact of exclusive versus inclusive nursing on particular baby mortalities and morbidities from conception to 6 months is examined in this study. Exclusive breastfeeding practices are more crucial and effective in preventing illness outbreaks when there is no access to appropriate medications or vaccinations. Additionally, this study takes optimal control theory into account, applying it to a system of differential equations that uses Pontryagin's Maximum Principle to describe a bimodal pneumonia transmission behavior in a vulnerable compartment. The proposed pneumonia transmission model was then updated to include two control variables. These include preventing illness exposure in susceptible children through various preventative measures and treating infected children through antibiotics, hospital care, and other treatments. If the threshold number (ℜ0) is less than one, then treatment and prevention rates are increased, and the disease will be wiped out of the population. However, when (ℜ0) is greater than one, then the disease persists in the population, which indicates that prevention and treatment rates are low. To evaluate the cost-effectiveness of all potential control techniques and their combinations, the incremental cost-effectiveness ratio (ICER) was determined. The simulation results of the identified model show that the interventions of prevention and treatment scenarios were the most successful in eradicating the dynamics of the pneumonia disease's propagation during the epidemic, but they were ineffective from a cost-saving perspective. Therefore, limiting pneumonia transmission to prevention alone during an outbreak is the most economical course of action.

Chandra Bala,1 Paul Athanasiov,2 Jason Holland,3 Mukesh Dhariwal,4 Amit Gupta,5 Hemant Rathi6 1personalEYES Pty Ltd, Sydney, NSW, Australia; 2Eye Surgeons SA, North Adelaide, SA, Australia; 3The Eye Health Centre, Brisbane, QLD, Australia; 4Alcon Vision LLC, Fort Worth, TX, USA; 5Skyward Analytics Pvt Ltd, Gurgaon, India; 6Skyward Analytics Pte Ltd, SingaporeCorrespondence: Chandra Bala, personalEYES Pty Ltd, Level 2, 33 York Street, Sydney, NSW, 2000, Australia, Tel +61-2-88337111, Fax +61-2-88337112, Email Bala@personaleyes.com.auPurpose: AcrySof IQ Vivity is a unique non-diffractive extended depth of focus intraocular lens with wavefront-shaping X-WAVE technology. This study evaluated the cost-effectiveness of AcrySof IQ Vivity intraocular lens (DFT015) compared with standard aspheric monofocal intraocular lens (SN60WF), from a private health fund perspective in Australia.Methods: A Markov model was developed using the following health states: well, need for spectacles (near/distance/bifocal/varifocal), very bothersome visual disturbances (glare/haloes/starbursts) – with/without spectacles, and death. Model inputs were sourced from a randomized clinical study (NCT03010254), published literature, prostheses list and clinical opinion. A lifetime horizon (up to 30 years) was considered, and cost and health outcomes were discounted at 5% per annum. Model outcomes included incremental cost–effectiveness ratio defined as incremental cost per quality adjusted life year gain. Sensitivity and scenario analyses were also conducted.Results: Bilateral implantation of DFT015 intraocular lens provided quality adjusted life year gain of 0.16 at an incremental cost of AU$307 compared to bilateral SN60WF, leading to an incremental cost-effectiveness ratio of AU$1908/quality adjusted life year, well below the cost-effectiveness threshold (Range: AU$45,000-AU$75,000) typically used by Medical Services Advisory Committee in Australia. Results were most sensitive to intraocular lens costs, post-operative spectacle dependence, and disutility due to wearing glasses. Robustness of the results was further confirmed by probabilistic sensitivity analysis and scenario analyses.Conclusion: AcrySof IQ Vivity intraocular lens is a highly cost-effective treatment strategy with improved vision-related quality of life outcomes for presbyopic cataract surgery patients.Keywords: cataract surgery, ATIOL, EDoF, presbyopia, ICER, quality of life

Studies on economic evaluations of the 13-valent pneumococcal conjugate vaccine (PCV13) have been increasing over the last decade. No systematic reviews have synthesized the evidence of economic evaluations of the PCV13. We systematically searched the literature which published on peer-reviewed journals from January 2010 to June 2022. The literature search was conducted in the following electronic databases: PubMed, Web of Science, Embase, the Cochrane Library, CNKI, Wanfang database, VIP database. We identified 1827 records from the database search. After excluding 511 duplicates, 1314 records were screened, of which 156 records were retained for the full-text reviews. A total of 44 studies were included in the review. Among the included studies, 33 studies were economic evaluations of PCV13 among children, and 11 studies were conducted among adults. The literature search initiated in April, 2022, and updated in June 2022. Vaccination with PCV13 was found to significantly reduce the mortality and morbidity of pneumococcal diseases and was cost-effective compared to no vaccine or several other pneumococcal vaccines (e.g. PCV10, PPV23). Future research is advised to expand economic evaluations of PCV13 combined with dynamic model to enhance methodologic rigor and prediction accuracy. [ABSTRACT FROM AUTHOR]

Objective: The study aimed to assess the cost-effectiveness of sintilimab combined with cisplatin plus paclitaxel versus chemotherapy alone as firstline treatment in patients with advanced or metastatic esophageal squamous cell carcinoma from the Chinese healthcare system. Materials and methods: A partitioned survival model was developed based on the ORIENT-15 clinical trial. Drug costs and health state utility were obtained from the literature. Outcomes included the health outcomes in life-years, quality-adjusted life-years (QALYs), and the incremental cost-effectiveness ratio. One-way and probabilistic sensitivity analyses were performed to evaluate the model uncertainty. Result: In overall population, patients given sintilimab plus chemotherapy gained more health benefits (0.90 QALYs vs. 0.61 QALYs), and the cost was more (15,399.21 US$ VS. 7475.58 US$) than that for patients in the chemotherapy group. In the subgroup, patients given sintilimab plus chemotherapy gained more health benefits (0.89 QALYs vs. 0.68 QALYs), and the cost was more (15,656.19 US$ vs. 9,162.77 US$) than that for patients in the chemotherapy group. Compared with chemotherapy, patients receiving sintilimab plus chemotherapy had ICERs of $26,773.68/QALY in the overall population and $30,065.50/QALY in the subgroup, which was above the threshold of WTP. Conclusion: Sintilimab plus chemotherapy was more cost-effective than chemotherapy alone for patients with advanced esophageal cancer from the perspective of the Chinese healthcare system. [ABSTRACT FROM AUTHOR]

Co-circulation of influenza and SARS-CoV-2 has the potential to place considerable strain on health-care services. We estimate the cost-effectiveness and health-care resource utilization impacts of influenza vaccination of low-risk 50–64-y-olds in the United Kingdom (UK) against a background SARS-CoV-2 circulation. A dynamic susceptible-exposed-infected-recovered model was used to simulate influenza transmission, with varying rates of vaccine coverage in the low-risk 50–64 y age-group. Four scenarios were evaluated: no vaccination (baseline), 40%, 50%, and 60% coverage. For the 50% and 60% coverage, this rate was also applied to high-risk 50–64-y-olds, whereas 48.6% was used for the baseline and 40% coverage scenarios. Cost-effectiveness was estimated in terms of humanistic outcomes and incremental cost-effectiveness ratio (ICER), with discounting applied at 3%. Overall, influenza vaccination of 50–64-y-olds resulted in reductions in GP visits, hospitalizations, and deaths, with a reduction in influenza-related mortality of 34%, 41%, and 52% for 40%, 50%, and 60% coverage, respectively. All four scenarios resulted in acute and intensive care unit (ICU) bed occupancy levels above available capacity, although vaccination of low-risk 50–64-y-olds resulted in a 35–54% and 16–25% decrease in excess acute and ICU bed requirements, respectively. Vaccination of this group against influenza was highly cost-effective from the payer perspective, with ICERs of £2,200–£2,343/quality-adjusted life year across the coverage rates evaluated. In conclusion, in the UK, vaccination of low-risk 50–64-y-olds against influenza is cost-effective and can aid in alleviating bed shortages in a situation where influenza and SARS-CoV-2 are co-circulating.

Introduction: Remote telemonitoring (RTM) for patients with chronic heart failure (HF) holds promise to improve prognosis and well-being beyond the standard of care (SoC). The CardioBBEAT trial assessed the health economic and clinical impact of an interactive bidirectional RTM system (Motiva®) versus SoC for patients with HF and a reduced ejection fraction (HFrEF), in Germany. Methods: This multicenter, randomized controlled trial enrolled 621 patients with HFrEF (mean age 63.0 ± 11.5 years, 88% men). The primary endpoint was the integrated effect of the intervention on total costs and nonhospitalized days alive after 12 months, reported as incremental cost-effectiveness ratio (ICER). Costs (in k€) were based on actual charges of patients' statutory health insurance. Among secondary outcome measures were mortality and disease-specific quality of life. Results: We found a neutral effect on nonhospitalized days alive (RTM mean 341 ± 59 days, SoC 346 ± 45 days; p = 0.298) associated with increased total costs (RTM 18.5 ± 39.5 k€, SoC 12.8 ± 22.0 k€; p = 0.046). This yielded an ICER of −1.15 k€/day. RTM did not impact mortality risk. All quality of life scales were consistently and meaningfully improved in the RTM group at 12 months compared to SoC (all p < 0.01). Conclusions: The first 12 months of RTM were not cost-effective compared to SoC in patients with HFrEF, but associated with a relevant improvement in disease-specific quality of life. The balanced assessment of the potential benefit of RTM requires integration of both the societal and patient perspective. ClinTrials.gov (NCT02293252). [ABSTRACT FROM AUTHOR]

We aimed to report our 10-year experience of treating short children born small for gestational age (SGA) by comparing the long-term growth, metabolic safety, and cost-effectiveness of recombinant human growth hormone (rhGH) therapy in short children born SGA with those in rhGH-treated children with growth hormone deficiency (GHD) and Turner syndrome. We performed a 10-year retrospective cohort study at King Saud University Medical City. We included children aged 3–16 years who received rhGH for GHD, SGA, or Turner syndrome for >1 year. A total of 166 children received rhGH therapy for GHD, 58 for SGA, and 16 for Turner syndrome. During the last study visit, the average height change was 21 cm for GHD children and 14 cm for children born SGA (p-value <0.001). The height SDS change was 0.84 for GHD children and 0.55 for SGA children (p-value=0.004). The average cost-effectiveness ratios for treating GHD and SGA children were USD 1,717.22 and USD 1,157.19 per centimeter gained, respectively. Moreover, the mean incremental cost-effectiveness ratio for GHD vs. SGA patients was USD 2,820.39 per centimeter gained. Dysglycemia developed in 70 patients: 43 (36.44%), 22 (40.74%), and 5 (13%) in the GHD, SGA, and Turner syndrome groups, respectively. rhGH is effective in height improvement of short children. However, pursuing rhGH treatment for children born SGA requires a shared decision-making approach to balance the modest benefit of final adult height gain with the long-term metabolic effects, considering the acceptable costs on the Saudi healthcare system. [ABSTRACT FROM AUTHOR]

The rising cost of cancer drug therapy threatens the long-term sustainability of Taiwan National Health Insurance. Cost savings can be achieved through various strategies, e.g., using smaller vial sizes, sharing vials, weight-based dosing, or switching to biosimilars. Here we aimed to examine the cost-effectiveness of a trastuzumab biosimilar combined with docetaxel (TDbiol) for treatment-naïve HER2+ metastatic breast cancer (MBC), and the financial impact of drug wastage. A Markov model with three health states was developed to assess the cost-effectiveness of trastuzumab biosimilars plus docetaxel over a 40-month time horizon in patients with HER2+ MBC. Based on the literature and our expert opinion, we assumed similar efficacy between the trastuzumab biosimilar and its reference product. The primary clinical input for the biosimilar was the same as for the reference product in the Catastrophic Patient Database (HV). Health state utilities were derived from the literature, and direct medical costs were obtained from the National Health Insurance Administration (NHIA). In the base-case scenario, the incremental cost-effectiveness ratio (ICER) was NTD 811,050 per QALY gained. One-way sensitivity analyses showed that the model was sensitive to utilities and transition probabilities, but not particularly sensitive to the wastage assumption. In scenario analyses, the ICER was higher when applying the price for trastuzumab reference biologic (branded), than for trastuzumab biosimilar. The trastuzumab biosimilar combination regimen is cost-effective and offers significant drug cost savings in Taiwan. • The conversion of biosimilar combination regimen for cancer treatment is cost-effective and cost savings compared to the reference branded drugs. • The introduction of biosimilars and weight-based administration has resulted in significant drug cost savings, minimized drug waste, and facilitated the evaluation and/or clinical use of new therapies and drugs. • Cancer drug waste may be considered a hidden cost to the overall cost of the cancer care setting in the past decade. • To overcome the increasing annual healthcare expenditures, it will be essential in the near future to examine waste of high-cost target drugs, improve waste management practices, and explore cost-effective dosing regimens. [ABSTRACT FROM AUTHOR]

Drug reimbursement decisions that spark public controversy are potential signals that processes used to reach such decisions do not adequately reflect society's goals. Such controversial decisions appear to be a characteristic of Quality-Adjusted Life Year (QALY)-based Incremental Cost Effectiveness Ratio (ICER)-dominated decision-making systems. QALY-based ICER-heavy systems have several known weaknesses that lead to individual and societal preferences being either ignored or considered in an unsystematic and inconsistent manner. We reprise some of the key inadequacies of QALY-based ICER analyses and suggest that there are other means including multicriteria decision analysis (MCDA) and cost-benefit analysis based on willingness to pay (WTP) measures by which to partially mitigate these weaknesses. For long, the inadequacies of QALY-based ICER-heavy decision-making systems have been rationalized with the answer: 'while the method is a second best, it is the best we currently have.' In light of the equally well-developed and widely utilized alternatives available, this resistance to improve assessment processes should not be accepted by policy makers. Health technology assessment bodies should consider and, with appropriate modifications, adopt these alternatives as they have the potential to result in more comprehensive, systematic, and accountable decision-making. [ABSTRACT FROM AUTHOR]

Introduction: Patients with metastatic triple-negative breast cancer (mTNBC) have poor prognosis and survival outcomes. Sacituzumab govitecan was newly approved into Chinese market for mTNBC. However, whether its price matches the survival benefit still needs exploring. Here, this study aimed to evaluate the cost-effectiveness of sacituzumab govitecan versus chemotherapy in patients with mTNBC from the perspective of Chinese healthcare system., Methods: A partitioned survival model consisting of three discrete health states was constructed to assess the cost-effectiveness of sacituzumab govitecan versus single-agent chemotherapy. The key clinical data in the model were from the ASCENT trial. Costs and utility inputs were collected from published literatures. Life-years gained, quality adjusted life-years (QALYs), incremental cost-effectiveness ratio (ICER), incremental net health benefits, and incremental net monetary benefits were calculated between 2 treatment strategies. One-way and probabilistic sensitivity analyses were conducted to account for uncertainty and verify model robustness. Subgroup and cost-threshold analysis were also performed., Results: Sacituzumab govitecan provided an additional 0.25 QALYs and an incremental cost of $ 81,778.61 compared with chemotherapy, which was associated with an ICER of $ 323,603.84/QALY. One-way sensitivity analysis revealed that the model was most sensitive to the cost of sacituzumab govitecan, weight, and utility of progression-free survival. The probabilistic sensitivity analysis indicated that the probability of sacituzumab govitecan being cost-effective was 0%. Considering a willingness-to-pay (WTP) of 3 times GDP, the maximum cost of sacituzumab govitecan that would make it cost-effective was $155.65 per unit (180 mg)., Conclusions: Sacituzumab govitecan was not cost-effective for patients with mTNBC compared with chemotherapy at the commonly adopted WTP threshold of 3 times GDP per capita per QALY in China., Competing Interests: Disclosure The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (Copyright © 2024 Elsevier Inc. All rights reserved.)

Objectives: To identify the types of disease most likely to be affected by the Institute for Clinical and Economic Review's (ICER) shared savings assumptions., Methods: For diseases with treatments that were Food and Drug Administration approved between 2019 and 2023, annual direct and indirect economic burden and characteristics of each disease were extracted from peer-reviewed literature. ICER's shared savings methodology was applied 2 ways: 50/50 shared savings and $150 000 cost-offset cap. The primary outcome was the difference in eligible cost savings provided by a hypothetical disease cure under ICER's 2 shared savings methods. Characteristics of diseases most impacted by these 2 methods were evaluated descriptively., Results: Food and Drug Administration approved 260 therapies for 89 unique diseases between 2019 and 2023. Shared savings reduced value of a hypothetical cure for hemophilia A most (50/50 method: -$367 670 per year; cap method: -$585 340 per year), followed by acute hepatic porphyria (50/50 method: -$333 948; cap method: -$517 896) and paroxysmal nocturnal hemoglobinuria (50/50 method: -$291 997; cap method: -$433 993). Compared with diseases with annual burdens <$150 000, those ≥$150 000 had earlier disease onset by 22.0 years (age 12.3 vs 34.3), lower life expectancy by 10.6 years (55.8 vs 66.4 years), and lower disease prevalence (4.7 vs 1981.5 per 100 000). Shared savings' impact on health-benefit price benchmarks was projected to be larger for diseases with shorter life expectancy (ρ = -0.319; p =.005), worse quality of life (ρ = -0.263; P =.020), and lower prevalence (ρ = -0.418; P < .001)., Conclusions: ICER's shared savings assumptions would most likely have the largest negative impact on health-benefit price benchmarks for rare, severe, and pediatric diseases., Competing Interests: Author Disclosures Author disclosure forms can be accessed below in the Supplemental Material section., (Copyright © 2024. Published by Elsevier Inc.)

Yasinta Rakanita,1,2 Mas Rizky AA Syamsunarno,3 Rano K Sinuraya,1,4 Eka W Suradji,5,6 Rizky Abdulah,1,4 Auliya A Suwantika1,4,7 1Department of Pharmacology and Clinical Pharmacy, Faculty of Pharmacy, Universitas Padjadjaran, Bandung, Indonesia; 2College of Pharmaceutical Sciences Pelita Mas, Palu, Indonesia; 3Department of Biomedical Sciences, Faculty of Medicine, Universitas Padjadjaran, Bandung, Indonesia; 4Center of Excellence in Higher Education for Pharmaceutical Care Innovation, Universitas Padjadjaran, Bandung, Indonesia; 5Department of Public Health, Faculty of Medicine, Krida Wacana University, Jakarta, Indonesia; 6UKRIDA Hospital, Jakarta, Indonesia; 7Center for Health Technology Assessment, Universitas Padjadjaran, Bandung, IndonesiaCorrespondence: Auliya A SuwantikaDepartment of Pharmacology and Clinical Pharmacy, Faculty of Pharmacy, Universitas Padjadjaran, Bandung, IndonesiaEmail auliya@unpad.ac.idBackground: Up to now, the combinations of ferrous fumarate–folic acid (FF-FA) and ferrous gluconate–multivitamins (FG-MV) have been implemented by the local government in the province of Papua. Nevertheless, there is no a specific economic evaluation that has been applied to investigate the cost-effectiveness of FF-FA and FG-MV.Objective: This study aimed to investigate the cost-effectiveness of FF-FA and FG-MV to be implemented in Teluk Bintuni, as one of the districts with the highest prevalence of iron deficiency anemia in Papua by taking the healthcare perspective into account.Methods: A prospective observational study was applied by considering two groups of women (15– 49 years old) with iron deficiency anemia who received FF-FA and FG-MV from September to November 2018. Applying a purposive sampling method, respondents were selected from 875 targeted women in six sub-districts, who met inclusion criteria. To estimate the total cost, we applied a healthcare perspective that considered direct medical cost only (eg, the procurement cost of iron tablets, cost of Hb test, and cost of healthcare visit). To estimate the effectiveness of intervention, we applied two major parameters, such as Hb level and utility score in quality-adjusted life year (QALY). The cost-effectiveness values were evaluated by using the criteria on the cost-effectiveness of healthcare intervention according to the threshold of gross domestic product (GDP) per capita (cost per QALY gained).Results: From 875 targeted women in six sub-districts who met inclusion criteria, we found approximately 222 women with moderate–severe iron deficiency anemia and 110 women with complete data in the group of FF-FA (n=69) and FG-MV (n-41). The results showed that there were significant differences (p-value < 0.05) on the number of respondents, age, oral iron cost, total healthcare cost and utility score in both intervention groups. Comparing the use of FG-MV with FF-FA, we estimated the incremental cost-effectiveness ratios (ICERs) would be $255.77 per controlled patient, $142.09 per patient with Hb increment > 2.00 g/dL, $79.93 per patient with Hb increment > 1.00 g/dL, and $11.59 per QALY gained.Conclusion: The ICER was estimated to be $11.59 per QALY gained, which was highly cost-effective, according to GDP-based cost-effectiveness threshold. In addition, the utility score of women with iron deficiency anemia was considered to be the most influential factor impacting the cost-effectiveness value.Keywords: ICER, cost per controlled patient, cost per patient with Hb increment > 2.00 g/dL, cost per patient with Hb increment > 1.00 g/dL, cost per QALY gained

The study aims to estimate the MitraClip system’s cost-effectiveness compared with optimal medical therapy in adult patients with inoperable mitral regurgitation and assess its impact on the budget of the Russian Federation health system.Materials and methods. The cost-effectiveness analysis of the MitraClip system was carried out using the Markov model. The time horizon was three & five years. The budget impact analysis (BIA) model compared the costs of treating patients distributed across different management practices. Standard management practice included only optimal medical therapy. Expected management practice included different patient allocation between the MitraClip system and optimal medical therapy. The time horizon for the budget impact analysis was five years. Results. The incremental cost-effectiveness ratio (ICER) per additional quality-adjusted life-year (QALY) gained of the MitraClip system in comparison with optimal medical therapy was 6,271,657 rubles in three years and 3,451,342 rubles in five years. Based on the BIA results of the MitraClip system, its use would lead to an increase in costs by 12.6 billion rubles (+6.09%, minimal scenario), by 37.8 billion rubles (+18.28%, optimal scenario) or by 63 billion rubles (+30.47%, maximum scenario).Conclusion. As a result of the analysis performed, it was found that economic efficiency is noted with an increasing time horizon of up to five years. The obtained ICER values are comparable with the average values obtained in other foreign cost-effectiveness studies. The use of this technology will lead to an increase in direct medical costs by 6.09% over five years compared to the current management practice. In more comprehensive MitraClip system implementation (maximum scenario), direct medical costs will increase by 30.47% compared to the current management practice.

A genetic test is a test for the presence or absence of a genetic mutation. A positive test outcome that reveals a mutation associated with increased risk for a disease may lead a patient to seek preventive treatment provided that the penetrance (probability of developing the disease given the mutation) is sufficiently high. We derive the test threshold and the test-treatment threshold, which confine the mutation probability interval for the use of the genetic test. Test and treatment costs as well as a low penetrance rate of the mutation narrow this interval. We illustrate the model with parameters of the test for BRCA1 and BRCA2 genes as well as of preventive treatment options for breast cancer. [ABSTRACT FROM AUTHOR]

In Canada, approximately 12,000 people annually are hospitalized with influenza. While vaccination is the most effective method for reducing the burden of seasonal influenza, the propagation of vaccine virus strains in eggs can result in egg adaption, resulting in reduced antigenic similarity to circulating strains and thus lower vaccine effectiveness (VE). Cell-based propagation methods avoid these alterations and therefore may be more effective than egg-propagation vaccines. We evaluated three different scenarios: (1) egg-based quadrivalent influenza vaccine (QIVe) for individuals <65 years and adjuvanted trivalent influenza vaccine (aTIV) for ≥65 years; (2) QIVe (<65 years) and high-dose QIV (HD −; QIV; ≥65 years); and (3) cell-based derived QIV (QIVc; <65 years) and aTIV (≥65 years) compared with a baseline scenario of QIVe for all age groups. Modelling was performed using a dynamic age-structured SEIR model, which assessed each strain individually using data from the 2012–2019 seasons. Probabilistic sensitivity analysis assessed the robustness of the results with respect to variation in absolute VE, relative VE, number of egg-adapted seasons, and economic parameters. QIVe + aTIV was cost-saving compared with the baseline scenario (QIVe for all), and QIVe + HD − QIV was not cost-effective in the majority of simulations, reflecting the high acquisition cost of HD − QIV. Overall, while the incremental benefits may vary by influenza season, QIVc + aTIV resulted in the greatest reductions in cases, hospitalizations, and mortality, and was cost-effective (ICER < CAD 50,000) in all simulations. [ABSTRACT FROM AUTHOR]

Marco Bellone,1 Lorenzo Pradelli,1 Stefano Molica,2 Adele Emanuela De Francesco,3 Daniela Ghislieri,4 Emanuele Guardalben,5 Antonietta Caputo4 1Department of Health Economics and Outcome Research, AdRes Health Economics and Outcomes Research, Turin, Italy; 2Dipartimento Onco-ematologico, Azienda Ospedaliera Pugliese-Ciaccio, Catanzaro, Italy; 3Hospital Pharmacy, Mater Domini Azienda Ospedaliero Universitaria, Catanzaro, Italy; 4Market Access Department, Roche SpA, Monza, Italy; 5Medical Department, Roche SpA, Monza, ItalyCorrespondence: Marco BelloneAdRes Health Economics and Outcomes Research, 17 Via Vittorio Alfieri, Turin 10121, ItalyTel +39 011 765-1239Email m.bellone@adreshe.comObjective: To assess the cost-effectiveness of obinutuzumab (O-chemo) in comparison to rituximab (R-chemo) in patients with untreated advanced follicular lymphoma (FL) at intermediate or high risk from an Italian National Health Service (NHS) perspective.Methods: A previously developed four-state Markov model was adapted to estimate lifetime clinical outcomes and costs of Italian patients with advanced FL and an FL international predictive index score ≥ 2 in treatment with O-chemo and R-chemo. Life expectancy was derived from the GALLIUM and PRIMA clinical trials. Progression-free survival (PFS), early progressive disease (PD), and treatment duration were extrapolated by fitting parametric distributions to empirical data in GALLIUM and late PD to data in PRIMA. Expected survival was weighed by published utilities. Costs updated to 2020 Euros and health gains occurring after the first year were discounted at an annual 3% rate. Probabilistic sensitivity analysis (PSA) was carried out.Results: O-chemo was associated with an incremental survival increase (0.97 life-years [LYs]), even when weighted for quality (0.88 quality-adjusted LYs [QALYs]), and incremental costs (around € 15,000), driven by longer treatment during PFS state relative to R-chemo. The incremental cost-effectiveness ratio and incremental cost-utility ratio are both widely accepted by the Italian NHS (around € 15,500/LY and € 17,000/QALY gained, respectively). PSA simulations confirmed the robustness of results given sensible variations in assumptions.Conclusion: O-chemo has superior clinical efficacy compared to rituximab, and should be considered a cost-effective option in first-line treatment of patients with advanced FL at intermediate or high risk in Italy. Incremental cost-effectiveness ratios are below the threshold considered affordable by developed countries.Keywords: economic evaluation, oncology, FLIPI score, ICER, PFS, QALY

Introduction: Cervical cancer is a leading cause of cancer among women in Mongolia with an age-standardized incidence rate of 23.5 per 100,000. HPV vaccination has not been introduced nationally and Gavi co-financing support is not available in Mongolia. Extended Gavi pricing for HPV vaccine may be available from vaccine manufacturers for a number of years. To inform introduction decision-making, we evaluated the potential cost-effectiveness of HPV vaccination among girls and young women in Mongolia. Methods: We used UNIVAC (version 1.4), a static decision model, to evaluate the health and economic outcomes of single-cohort vaccination among females from the government perspective compared to no vaccination. We modeled vaccine introduction over 10 birth cohorts starting in 2022 comparing quadrivalent or bivalent vaccine selection and vaccine pricing variations. We used locally-specific data for cancer incidence, mortality, treatment and costs. Model outcomes included cancer cases, hospitalizations, deaths, disability-adjusted life years (DALY), and costs presented in 2018 USD. Incremental costs and health outcomes were discounted at 3% and aggregated into an Incremental Cost-Effectiveness Ratio (ICER). Results: The base-case scenario of HPV vaccination among 9 year-old girls was projected to avert 5,692 cervical cancer cases, 3,240 deaths, and 11,886 DALYs and incur $2.4–3.1M more costs compared to no vaccination. At prices of ($4.50-$4.60/dose), we estimated an ICER of $166-$265/DALY averted among 9-year-olds. When price per dose was increased to reported mean vaccine purchase price for non-Gavi LMICs ($14.17/dose), the ICER ranged from $556–820/DALY averted. Conclusion: HPV vaccination among girls is highly likely to be a cost-effective investment in Mongolia compared to no vaccination with projected ICERs less than 20% of the 2018 GDP per capita of $3,735.

Research and development of innovative antimicrobials is paramount to addressing the antimicrobial resistance threat. Although antimicrobial discovery and development has increased, difficulties have emerged in the pharmaceutical industry after market approval. In this minireview, we summarize clinical trial data on recently approved antibiotics, calculate incremental cost-effectiveness ratio (ICER) values, and explore ways to adapt ICER calculations to the limitations of antimicrobial clinical trial design. We provide a systematic review and analysis of randomized, controlled studies of antibiotics approved from 2014 – 2022 and extracted the relevant clinical data. Adapted-ICER (aICER) calculations were conducted using the primary condition-specific outcome that was reported in each study (percent mortality or percent cure rate). The literature search identified 18 studies for the 8 total antibiotics which met inclusion criteria and contained data required for aICER calculation. aICER values ranged from -$17,374 to $4,966 per percent mortality and -$43,931 to $2,529 per percent cure rate. With regards to mortality, ceftolozane/tazobactam and imipenem/cilastatin/relebactam proved cost efficacious, with aICER values of $4,965 per percent mortality and $1,955 per percent mortality respectively. Finding value in novel antibiotic agents is imperative to further justifying their development, and aICER values are the most common method of determining value in healthcare. The current outcomes of clinical trials are difficult to translate to aICER, which most effectively use Quality-Adjusted Life Years (QALY) as the quality standard in other fields such as oncology. Future antimicrobial trials should consider introducing methods of assessing measures of health gain such as QALY to better translate the value of novel antimicrobials in healthcare. [ABSTRACT FROM AUTHOR]

Gianni Ghetti,1 Maria Claudia D’Avella,2 Lorenzo Pradelli1 1Department of Health Economics and Outcome Research, AdRes, Turin, Italy; 2Department of Market Access, Sanofi, Milan, ItalyCorrespondence: Gianni GhettiDepartment of Health Economics and Outcome Research, AdRes, Via Vittorio Alfieri 17, Turin, 10121, ItalyTel +39 011 765 1239Email g.ghetti@adreshe.comPurpose: Cutaneous squamous cell carcinoma (CSCC) is a common cancer that in most cases is curable with surgery. About 3– 5% of patients develop advanced CSCC (aCSCC) and are no longer responsive to surgery or radiation therapy. The aim of this study was to assess the cost-effectiveness and cost-utility of cemiplimab, the first systemic therapy approved in Italy for patients with aCSCC, vs platinum-based chemotherapy from the Italian National Health Service (SSN) perspective.Methods: A partitioned survival model, which included three mutually exclusive health states, was developed to estimate costs and outcomes for patients with aCSCC, over a 30-year time horizon (lifetime). No direct evidence of the comparative efficacy and safety of cemiplimab versus other therapies currently exists. Therefore, a simulated treatment comparison (STC) was conducted to estimate the comparative efficacy of cemiplimab versus chemotherapy. Individual patient data for cemiplimab were collected from the EMPOWER-CSCC 1 trial whereas chemotherapy data were derived from a retrospective study. In the STC a regression model was used to predict outcomes for cemiplimab in the population observed in the comparator study. Costs of drug acquisition/administration and management of adverse events were included. Costs and outcomes were discounted at 3% per year. Incremental cost-effectiveness ratio (ICER) and incremental cost-utility ratio (ICUR) were calculated; sensitivity and scenario analyses were performed to assess the robustness of results.Results: In the base-case, treatment with cemiplimab was associated with a gain of 4.89 LYs and 3.99 QALYs, compared with a platinum-based chemotherapy regimen, resulting in an estimated ICER of 27,821 €/LY gained and an ICUR of 34,110 €/QALY gained. Both ICER and ICUR were below the commonly used Italian SSN willingness to pay thresholds.Conclusion: The use of cemiplimab, compared with a platinum-based chemotherapy regimen, can be considered a cost-effective option for the treatment of aCSCC patients in Italy.Keywords: Italian National Health Service, ICER, partitioned survival model, non-melanoma skin cancers

Abstract Background Hybrid emergency room (ER) systems, consisting of an angiography-computed tomography (CT) machine in a trauma resuscitation room, are reported to be effective for reducing death from exsanguination in trauma patients. We aimed to investigate the cost-effectiveness of a hybrid ER system in severe trauma patients without severe traumatic brain injury (TBI). Methods We conducted a cost-utility analysis comparing the hybrid ER system to the conventional ER system from the perspective of the third-party healthcare payer in Japan. A short-term decision tree and a long-term Markov model using a lifetime time horizon were constructed to estimate quality-adjusted life years (QALYs) and associated lifetime healthcare costs. Short-term mortality and healthcare costs were derived from medical records and claims data in a tertiary care hospital with a hybrid ER. Long-term mortality and utilities were extrapolated from the literature. The willingness-to-pay threshold was set at $47,619 per QALY gained and the discount rate was 2%. Deterministic and probabilistic sensitivity analyses were conducted. Results The hybrid ER system was associated with a gain of 1.03 QALYs and an increment of $33,591 lifetime costs compared to the conventional ER system, resulting in an ICER of $32,522 per QALY gained. The ICER was lower than the willingness-to-pay threshold if the odds ratio of 28-day mortality was < 0.66. Probabilistic sensitivity analysis indicated that the hybrid ER system was cost-effective with a 79.3% probability. Conclusion The present study suggested that the hybrid ER system is a likely cost-effective strategy for treating severe trauma patients without severe TBI.