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The prevalence of heart failure (HF) and its risk factors are high in Singapore. The EMPEROR-Reduced trial demonstrated that add-on empagliflozin resulted in a reduction in the risk of cardiovascular death or hospitalization for HF compared with standard of care (SoC). This study aimed to estimate the cost-effectiveness of empagliflozin + SoC versus SoC in patients with HF with reduced ejection fraction from a Singaporean healthcare perspective. A Markov cohort model simulated progression through health states based on New York Heart Association classes over a lifetime horizon using a cycle length of 1 month. Transition probabilities, and the risk of transient events (hospitalization for HF and cardiovascular/all-cause death) were modeled based on the EMPEROR-Reduced trial. Costs for HF-related events, adverse events, and for monitoring were estimated from a combination of published literature and publicly available fees for public hospitals/polyclinics. Empagliflozin + SoC was estimated to be very cost-effective versus SoC alone with an incremental cost-effectiveness ratio of < 8000 Singapore Dollars/quality-adjusted life-year gained. The base-case results were robust as evidenced from the consistency of various scenario and sensitivity analyses performed. When using Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score quartiles as the health states, the incremental cost-effectiveness ratio reduced significantly to 4625 Singapore Dollars/quality-adjusted life-year. The use of empagliflozin on top of SoC represents a highly cost-effective solution for the treatment of patients with HF with reduced ejection fraction in Singapore when considering its efficacy, relative affordability, and the growing economic burden of HF in Singapore. • Recent large-scale clinical trials have demonstrated the efficacy of sodium-glucose cotransporter-2 inhibitors such as empagliflozin as an add-on therapy on for managing heart failure (HF) with reduced ejection fraction. • Applied to a Singaporean healthcare perspective, this economic modeling estimates that the use of empagliflozin on top of standard of care can provide incremental quality-adjusted life-year gains at an incremental cost-effectiveness ratio of 7839 Singapore Dollars/quality-adjusted life-year gained. • The use of empagliflozin represents a highly cost-effective solution for the treatment of HF patients with reduced ejection fraction in Singapore when considering the growing economic burden of HF in Singapore. [ABSTRACT FROM AUTHOR]

This study aimed to assess the cost-effectiveness of biologic disease-modifying antirheumatic drugs (bDMARDs) for treating patients with psoriatic arthritis who failed conventional synthetic disease-modifying antirheumatic drugs (csDMARDs). A decision tree and Markov model were constructed to capture long-term costs and outcomes from a societal perspective. Patients with psoriatic arthritis who failed 2 previous csDMARDs were modeled over a 3-month cycle with a lifetime horizon. Clinical probabilities were derived from a published meta-analysis. Prices of bDMARDs were proposed by pharmaceutical companies. Other costs and utilities were based on data in Thailand. All costs and outcomes were discounted at a 3% annual rate. Incremental cost-effectiveness ratio and a series of sensitivity analyses were performed. All 11 bDMARDs (3 infliximab originator and biosimilars, 2 etanercept originator and biosimilar, golimumab, 2 secukinumab 150 mg and 300 mg, 3 adalimumab biosimilars) gained better quality-adjusted life-years (QALYs) with more costly than csDMARDs. Infliximab had the highest QALYs compared with other bDMARDs. Only secukinumab 150 mg showed the incremental cost-effectiveness ratio below the Thai threshold of 5152 US dollars per QALY. Cost of bDMARDs was the most influential factor. At the current price, secukinumab 150 mg shows the value for money in the Thai context. Price negotiation is of great importance for other bDMARDs. [ABSTRACT FROM AUTHOR]

Pulmonary arterial hypertension (PAH) is a chronic and progressive disease that, if left untreated, shortens the life expectancy of patients. Endothelin receptor antagonists, such as macitentan and bosentan, play an essential role in improving the patient's symptoms, quality of life, and life expectancy. This study aimed to evaluate the cost-utility of macitentan compared with bosentan in treating PAH from the health system perspective in Iran. For evaluating the cost-effectiveness of macitentan, a Markov model consisting of 5 states, functional class (FC) I, FC II, FC III, FC IV, and death, was designed using the TreeAge software. The lifetime time horizon and a 3-month cycle length were set. Patients entered the model from FC II or FC III states based on the initial probabilities. Costs were measured in US dollars (USD), and outcomes were measured in terms of quality quality-adjusted life-years (QALYs). Consequently, the incremental cost-effectiveness ratio (ICER) was calculated. In addition, sensitivity analysis was performed to determine the robustness of the model by examining the possible effects of uncertainties on the final result. The costs of treatment with macitentan and bosentan in PAH in Iran were calculated at 19 429 and 17 246 USD, and the outcomes were 4.02 and 3.04 QALYs, respectively. Therefore, ICER was calculated at 2233.46 USD/QALY. One-way sensitivity analysis demonstrates that the model is robust; nevertheless, it is most sensitive to the price of macitentan and bosentan. Treatment with macitentan was associated with both higher costs and QALYs than bosentan. Nevertheless, it is considered the cost-effective treatment strategy in Iran given that the calculated ICER falls below the willingness to pay threshold. [ABSTRACT FROM AUTHOR]

Background: Economic evidence for comparing low fraction with ultra-hypo fractionated (UHF) radiation therapy in the treatment of intermediate-to-high-risk prostate cancer (PC) is lacking, especially in Europe. This study presents an economic evaluation performed alongside an ongoing clinical trial. Aim: To investigate up to 6 years' follow-up whether conventional fractionation (CF, 78.0 Gy in 39 fractions, 5 days per week for 8 weeks) is more cost-effective than UHF (42.7 Gy in 7 fractions, 3 days per week for 2.5 weeks inclusive of 2 weekends) radiotherapy in treatment for patients with intermediate-to-high-risk PC. Method: HYPO-RT-PC trial is an open-label, randomized, multicenter (10 in Sweden; 2 in Denmark) phase-3 trial. Patients from Sweden (CF 434; UHF 445) were included in this study. The trial database was linked to the National Patient Registry (NPR). Costs for inpatient/non-primary outpatient care for each episode were retrieved. For calculating Quality-adjusted life years (QALYs), the EORTC QLQ-C30 questionnaire was mapped to the EQ-5D-3L index. Multivariable regression analyses were used to compare the difference in costs and QALYs, adjusting for age and baseline costs, and health status. The confidence interval for the difference in costs, QALYs and incremental cost-effectiveness ratio effectiveness ratio (ICER) was estimated by the bootstrap percentile method. Results: No significant differences were found in ICER between the two arms after 6 years of follow-up. Conclusion: The current study did not support that the ultra-hypo-fractionated treatment was more cost-effective than the conventional fraction treatment up to the sixth year of the trial. [ABSTRACT FROM AUTHOR]

Introduction: The increasing incidence of prostate cancer (PC) in China leads to a significant disease burden. Although three novel androgen inhibitors (darolutamide, apalutamide, and enzalutamide) have been approved for patients with high-risk non-metastatic castration-resistant prostate cancer (nmCRPC), the economic evaluation of these novel treatments in China remains unknown. In this study, we aimed to evaluate the cost–utility of darolutamide combined with androgen deprivation therapy (ADT), comparing with apalutamide + ADT and enzalutamide + ADT, in patients with high-risk nmCRPC from a healthcare system perspective in China. Methods: A partitioned survival model was developed to capture time spent by patients in three health states: nmCRPC, metastatic CRPC (mCRPC), and death. Clinical outcomes from the ARAMIS, PROSPER, and SPARTAN studies were obtained. In the absence of head-to-head studies, indirect treatment comparisons were conducted to capture the comparative effectiveness between darolutamide + ADT, apalutamide + ADT, and enzalutamide + ADT. The prices of apalutamide and enzalutamide were assumed to be the same as the initial launch price of darolutamide, since post-negotiation prices after national reimbursement drug list (NRDL) inclusion remain confidential. Other health resources costs, baseline characteristics, treatment patterns, and utility were collected through literature or clinical expert interviews. Selected sensitivity analyses were also performed. Results: For a 20-year time horizon, darolutamide + ADT was associated with lower cost per quality-adjusted life years (QALYs) than apalutamide + ADT and enzalutamide + ADT (202,897 Chinese yuan (CNY)/QALY vs. 228,998 CNY/QALY and 221,409 CNY/QALY, respectively) (exchange rate, 1 USD = 6.7871 CNY). Darolutamide + ADT had better health outcomes and lower total costs compared to both apalutamide + ADT (+ 0.22 QALYs and − 72,818 CNY) and enzalutamide + ADT (+ 0.09 QALYs and − 67,451 CNY). Across the modelled sensitivity analyses (including hazard ratios and drug costs), darolutamide + ADT remained dominant or cost-effective. Conclusions: This economic evaluation suggested that, in comparison with apalutamide + ADT and enzalutamide + ADT, darolutamide + ADT was a dominant or cost-effective treatment option for patients with high-risk nmCRPC in China. [ABSTRACT FROM AUTHOR]

Introduction: In the randomized PARTNER 3 trial, transcatheter aortic valve implantation (TAVI) with the SAPIEN 3 device significantly reduced a composite of all-cause death, stroke, and rehospitalization, compared with surgical aortic valve replacement (SAVR), in patients with severe symptomatic aortic stenosis and low risk of surgical mortality. Furthermore, TAVI has been shown to be cost-effective in low-risk patients, compared with SAVR, in a number of countries. This study aimed to determine the cost-effectiveness of TAVI with SAPIEN 3 versus SAVR in Germany. Methods: A previously published two-stage Markov-based model that captured clinical outcomes from the PARTNER 3 trial was adapted for the German context using the German Statutory Health Insurance perspective. The model had a lifetime horizon. The cost–utility analysis estimated changes in direct healthcare costs as well as survival and health-related quality of life using TAVI with SAPIEN 3 compared with SAVR. Results: TAVI with SAPIEN 3 increased quality-adjusted life years (QALYs) by + 0.72 at an increased cost of €8664 per patient. The incremental cost-effectiveness/QALY ratio was €12,037, which fell below that of other cardiovascular interventions in use in Germany. The cost-effectiveness of TAVI over SAVR remained robust across multiple challenging scenarios and was driven by lower longer-term management costs compared with SAVR. Conclusions: TAVI with SAPIEN 3 appears to be a clinically meaningful, cost-effective treatment option over SAVR for patients with severe symptomatic aortic stenosis and low risk for surgical mortality in Germany. Clinical Trial Registration Number: www.clinicaltrials.gov identifier: NCT02675114. [ABSTRACT FROM AUTHOR]

Background Severe acute respiratory syndrome coronavirus 2 is the virus that causes coronavirus disease 2019. Over six million deaths worldwide have been associated with coronavirus disease 2019. Objective To assess the cost-effectiveness of treatments used for the treatment of coronavirus disease 2019 in hospital or used in the community in patients with coronavirus disease 2019 at high risk of hospitalisation. Setting Treatments provided in United Kingdom hospital and community settings. Methods Clinical effectiveness estimates were taken from the coronavirus disease-network meta-analyses initiative and the metaEvidence initiative. A mathematical model was constructed to explore how the interventions impacted on patient health, measured in quality-adjusted life-years gained. The costs associated with treatment, including those of hospital care, were also estimated and used to form a cost per quality-adjusted life-year gained value which was compared with thresholds published by the National Institute for Health and Care Excellence. Estimates of cost-effectiveness compared against current standard of care were produced in both the hospital and community settings at three different levels of efficacy: mean, low and high. Public list prices were used for interventions with neither confidential patient access schemes nor confidential list prices considered. Results incorporating confidential pricing data were provided to the National Institute for Health and Care Excellence appraisal committee. Results The treatments were estimated to be clinically effective although not all reached statistical significance. All treatments in the hospital setting, or community, were estimated to plausibly have a cost per quality-adjusted life-year gained value below National Institute for Health and Care Excellence’s thresholds when compared with standard of care. However, almost all drugs could plausibly have cost per quality-adjusted life-years above National Institute for Health and Care Excellence’s thresholds. However, there is considerable uncertainty in the results as the prevalent severe acute respiratory syndrome coronavirus 2 variant, vaccination status, history of being infected with severe acute respiratory syndrome coronavirus 2 and standard of care have all evolved since the pivotal studies were conducted which could have significant impact on the efficacy of each drug. For drugs used in high-risk patients in the community setting, the proportion of people at high risk who need hospital admission was a large driver of the cost per quality-adjusted life-year. Limitations No studies were identified that were conducted in current conditions. This may be a large limitation as the severe acute respiratory syndrome coronavirus 2 variant changes. No head-to-head studies of interventions were identified. Conclusions The results produced could be informative to decision-makers, although conclusions regarding the most clinical – and cost-effectiveness of each intervention should be tentative due to the evolving nature of the decision problem and, in this report, the use of list prices only. Comparisons between interventions should also be treated with caution due to potentially large heterogeneity between studies. Future work Research assessing the relative clinical effectiveness of interventions within head-to-head studies in current conditions would be beneficial. Contemporary information related to the probability of hospital admission and death for patients at high risk in the community would improve the precision of the estimates generated. Funding This project was funded by the National Institute for Health and Care Research (NIHR) Evidence Synthesis programme (NIHR135564) and will be published in full in Health Technology Assessment; Vol. 27, No. 14. See the NIHR Journals Library website for further project information. Plain language summary Coronavirus disease 2019 is an infectious disease that can cause death and long-term ill-health. Treatments exist that can be provided in hospital to reduce the number of deaths from coronavirus disease 2019. Treatments also exist which can be provided in the community for people at high risk of needing to be admitted to hospital to reduce the number of admissions and to reduce the number of deaths from coronavirus disease 2019. However, the value for money of these treatments has not been estimated. We took the clinical effectiveness of nine treatments from published literature sources and built a model that estimated the value for money of six treatments compared with care without these treatments. Three treatments were excluded due to confidential prices. The results of the model showed that many treatments in a hospital setting had estimates of cost-effectiveness that would normally be seen to be good value for money using the thresholds published by the National Institute of Health and Care Excellence. The same was true for some treatments in a community setting. However, it is also possible that these treatments are not good value for money. The benefit of the drugs and value for money is highly uncertain as studies trying to estimate the gain have been done with (1) previous variants of the virus causing coronavirus disease 2019 being widespread, (2) where the proportion of people who have had vaccinations or who had previously had coronavirus disease 2019 is low and (3) where standard treatment was that when coronavirus disease 2019 was first identified, and not the drugs used now. Because of these differences, and the unknown price of some interventions, we cannot confidently say which (if any) treatments help patients the most, or which treatment represents the best value for money. Further research, in current conditions, would improve the accuracy of our answers. Scientific summary Background Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) is the virus that causes coronavirus disease 2019 (COVID-19). At the time of writing (January 2023) there had been over 620 million confirmed cases and over six-and-a-half million deaths worldwide associated with COVID-19. For the UK, these values are more than 24 million cases and nearly 200,000 deaths. In addition to the widespread vaccination programme, treatments exist that can help people who have been hospitalised due to COVID-19 (casirivimab and imdevimab (henceforth casirivimab/imdevimab), tocilizumab, remdesivir, baricitinib, and baricitinib with remdesivir) or be used in patients who have COVID-19 and are at high risk of needing hospitalisation [casirivimab/imdevimab, molnupiravir, nirmatrelvir and ritonavir (henceforth nirmatrelvir/ritonavir), remdesivir, sotrovimab, and tixagevimab and cilgavimab (henceforth tixagevimab/cilgavimab)]. For reasons related to urgency, these treatments, unlike interventions in other disease areas, have not received positive guidance from the National Institute of Health and Care Excellence (NICE) before being routinely used. As the pandemic subsides there is more need for a formal evaluation of the clinical and cost-effectiveness of these treatments. Objectives The objective of this study is to summarise the current knowledge related to the clinical efficacy of the interventions and to conduct an economic evaluation that estimates the cost-effectiveness of each intervention against standard of care (SoC), as of January 2023. A full incremental analysis is performed while noting the caveats in the comparison of all interventions simultaneously. Methods Given the timescale of the project, where there were ˂ 3 months between the publication of the final scope and the deadline of a report for NICE and the consultation process, a literature review following best practice was not possible. Instead, a pragmatic, alternative approach was undertaken where evidence was taken from two living systematic reviews (supported by the COVID-network meta-analyses (NMA) initiative and the metaEvidence initiative) in line with current best practice guidelines. For interventions related to use in hospitals, data were extracted on time to death, clinical improvement and time to discharge. For interventions that are used in the community for patients at high risk of hospitalisation, data were extracted on the risks of hospitalisation or death, and the risks of death. These measures of efficacy were assumed generalisable to January 2023 despite changes in background conditions which include the SoC, the percentage of people who have been vaccinated and a change in the dominant SARS-CoV-2 variant. This is noted as a very large limitation as drugs that have looked effective in previous variants have not worked as well in later variants and sensitivity analysis on the efficacy of the interventions has been conducted. A mathematical model was constructed that used the data from the living systematic reviews to simulate the experiences of patients in hospital, and requirement for supplemental oxygen, until discharge or death in hospital. Due to the (conditional) marketing authorisations of the interventions, the model was developed such that results could be produced for the supplemental oxygen group and the non-supplemental oxygen group separately. The model structure used an eight-point ordinal scale that was used in clinical trials to categorise patients during their admissions. Outputs from this model included the costs associated with interventions and care, and the quality-adjusted life-years (QALYs) gained by the patient both within the hospital episode and after discharge, incorporating decrements in health-related quality of life associated with the lasting impact of COVID-19. For interventions used in the hospital, these values allowed a cost per QALY gained to be calculated for each treatment compared with SoC, and for completeness, a full incremental analysis to be conducted although the External Assessment Group (EAG) cautions against comparisons between treatments due to the heterogenous conditions when pivotal studies were undertaken. The costs of each intervention were taken from public sources where available. However, baricitinib, sotrovimab and tocilizumab have confidential patient access schemes agreed, which discount the price of the intervention, and are not considered in this document, but were provided to the NICE Appraisal Committee in a separate confidential appendix. The price of three treatments (casirivimab/imdevimab, molnupiravir and tixagevimab/cilgavimab) were not publicly available at the time of writing and the cost-effectiveness results for these three drugs are contained in a confidential appendix. For patients at high risk of hospitalisation treated in the community, a decision tree was put before the hospital model, which simulated the reduced need for hospitalisation associated with early treatment. The total costs and QALYs associated with treatment options were estimated to allow an evaluation of the cost per QALY of each treatment against SoC and for completeness, a full incremental analysis to be undertaken, noting the same caveat as for interventions used in hospital when comparing treatments. The modelling did not assess the logistical aspects of treatment in the community, but the EAG notes that this could be a large factor in deciding which treatments could be preferred, as oral treatments could be more acceptable to patients and healthcare systems than treatments that are given intravenously or subcutaneously. The costs of providing treatment within the community were provided by National Health Service (NHS) England. Three scenarios were run changing the efficacy of interventions. The ‘mean efficacy’ estimate used the mean of each distribution extracted from the living systematic reviews, the ‘high efficacy’ estimate used the most favourable limits of the 95% confidence intervals (CIs) and the ‘low efficacy’ estimate used the least favourable limits of the 95% CIs. The EAG has acknowledged a limitation that the CI is influenced by the number of observed events and the sample size, such that two identical treatments could have markedly different confident intervals purely due to the size of the pivotal study. Seven scenario analyses were performed, explored the impact of changing: (1) the duration of long COVID (ranging from half to double that of the base case); (2) changing the rate of hospital admission in the community with people being at ‘high risk’ of hospitalisation from a value of 2.79% to 1.00%, 5.00% and 10.00%; (3) changing the average age of patients at high risk of hospitalisation in the community from 55 years to 50 and 60 years; (4) using a hazard ratio (HR) of unity for all interventions in relation to time to hospital discharge and time to clinical improvement; (5) changing the baseline distribution of supplemental oxygen requirements from that associated with SoC (19% no supplemental oxygen, 55% high-flow oxygen, 16% non-invasive ventilation and 10% invasive ventilation) to an arbitrarily less severe baseline distribution (25% no supplemental oxygen, 60% high-flow oxygen, 10% non-invasive ventilation and 5% invasive ventilation) for patients who have received an intervention in the community; (6) assuming a utility decrement of 0.02 per day for patients receiving intravenous (i.v.) treatment in the community; and (7) changing the standardised mortality ratio for people during the period of long COVID from 7.7 to 5.0 and 10.0. Two scenario analyses were conducted that explored the use of different efficacy measures based on the Solidarity study for remdesivir and the ‘Efficacy and safety of intramuscular administration of tixagevimab–cilgavimab for early outpatient treatment of COVID-19’ (TACKLE) study for tixagevimab/cilgavimab. Results were presented in terms of incremental cost-effectiveness ratios (ICERs) measured in cost per QALYs gained and also using incremental net monetary benefit (NMB). An advantage of NMB is that interventions can be compared using different assumptions on efficacy for different interventions, and interventions can be omitted without the need to recalculate efficiency frontiers. Results Due to changes between the conditions when the pivotal studies were undertaken and the current conditions in terms of the SoC, the percentage of people who have been vaccinated and a change in the dominant SARS-CoV-2 variant all results should be treated with caution. Caution should also be applied when comparing between interventions. The results also do not incorporate confidential price discounts for baricitinib, sotrovimab and tocilizumab, nor were any cost-effectiveness results presented for casirivimab/imdevimab, molnupiravir and tixagevimab/cilgavimab which had confidential list prices. These analyses were seen by the NICE appraisal committee in a confidential appendix. All treatments used for hospitalised patients, had a median HR for death below one, indicating a benefit, although all CIs crossed unity apart from those for baricitinib, casirivimab/imdevimab and tocilizumab. The overlapping CIs and heterogeneous studies meant that no firm conclusions could be made regarding the relative efficacy of these treatments. There was less data relating to the relative risks (RRs) of clinical improvement at 28 days and the HRs for the time to discharge, although these were generally close to unity and had CIs that crossed unity. No clear conclusions could be made on the relative efficacy of treatments for these two measures compared with SoC. All treatments used in the community had favourable median RRs for hospitalisation and death at 28 days with the upper limit of the CI being below 1 for all drugs except molnupiravir. The median RRs associated with death at 28 days were favourable for all interventions, except for remdesivir where the median estimate was unity as no deaths were observed in the study within COVID-NMA. The CIs were wide and spanned one for all treatments except for molnupiravir and nirmatrelvir/ritonavir. For hospitalised patients requiring supplemental oxygen, all treatments had estimated ICERs compared with SoC below £12,000 in both the mean efficacy and high efficacy scenarios. However, in the low efficacy scenario only baricitinib and tocilizumab generated more QALYs than SoC. Baricitinib had an estimated ICER under £9000, while tocilizumab had an estimated ICER under £29,000. For hospitalised patients not requiring supplemental oxygen, all treatments had estimated ICERs compared with SoC below £12,000 in both the mean efficacy and high efficacy scenarios. However, in the low efficacy scenario, only baricitinib generated more QALYs than SoC with an estimated ICER below £6000. For interventions used in the community, the estimated ICERs compared with SoC were more varied. In the mean efficacy scenario, the estimated ICERs were below £7000 for nirmatrelvir/ritonavir, below £35,000 for sotrovimab and below £91,000 for remdesivir. In the high efficacy scenario, the estimated ICERs were below £5000 for nirmatrelvir/ritonavir, below £19,000 for sotrovimab and below £25,000 for remdesivir. In the low efficacy scenario, the estimated ICER was below £12,000 for nirmatrelvir/ritonavir, with remdesivir and sotrovimab having ICERs in excess of £10,000. Only one of the scenario analyses noticeably changed the ICERs for all interventions, which was changing the proportion of people with COVID-19 in the community at high risk of hospitalisation who are hospitalised when treated with SoC. Treatments became more cost-effective as the admission proportion increased at the mean and high efficacy scenarios. The ranges in the ICERs assuming mean efficacy for the drugs, when using 1%, 10% and 20%, rather than 2.82% as assumed in the base case, were: nirmatrelvir/ritonavir (£25,544, dominant and dominant), remdesivir (£280,819, £16,170 and £1512) and sotrovimab (£111,318, £4870 and dominant). If data from Solidarity are included, the low efficacy scenarios for remdesivir had a positive NMB regardless of the willingness-to-pay threshold and oxygen status assumed. For patients requiring supplemental oxygen the ICER was £25,903; the corresponding ICER was £34,550 for those not requiring supplementary oxygen. Conclusions There is considerable uncertainty in the efficacy of treatments compared to SoC observed in the studies due to the small number of events, which results in wide CIs for HRs and RRs. Some treatments (baricitinib and tocilizumab in the hospitalised setting and casirivimab/imdevimab, molnupiravir and nirmatrelvir/ritonavir in the community setting) were estimated to have a statistically significant benefit related to death due to COVID-19, however, this may also have been shown for other treatments if the pivotal studies had had larger sample sizes. However, the dominant SARS-CoV-2 variant, the SoC and the percentage of people who have had a vaccination, have all changed since the pivotal studies were undertaken meaning that the efficacies for treatments are highly uncertain. This is demonstrated by sotrovimab having favourable median and mean efficacies in prevention hospitalisation, but this drug is not authorised in the USA, as it is unlikely to be effective against the Omicron BA.2 subvariant. Further the World Health Organization has made strong recommendations against the use of sotrovimab. Given potential further changes in the variant, the results presented in this report, and within the confidential appendix, should be treated with caution. Funding This project was funded by the National Institute for Health and Care Research (NIHR) Evidence Synthesis programme (NIHR135564) and will be published in full in Health Technology Assessment; Vol. 27, No. 14. See the NIHR Journals Library website for further project information.

Aim: This study assessed the cost–utility of mogamulizumab, a novel monoclonal antibody, versus established clinical management (ECM) in UK patients in previously treated advanced mycosis fungoides (MF)/S ´ ezary syndrome (SS). Materials & methods: Lifetime partitioned survival model based on overall survival, next treatment-free survival and the use of allogeneic stem cell transplant was developed. Inputs were from the pivotal MAVORIC trial, real-world evidence and published literature. Extensive sensitivity analyses were conducted. Results: Discounted incremental quality-adjusted life years (QALYs), costs and incremental cost–effectiveness ratio were 3.08, £86,998 and £28,233. Results were most sensitive to the survival extrapolations, utilities and costs after loss of disease control. Conclusion: Mogamulizumab is a cost-effective alternative to ECM in UK patients with previously treated advanced MF/SS.

Evidence on the cost-effectiveness of eHealth in palliative care is scarce. Oncokompas, a fully automated behavioral intervention technology, aims to support self-management in cancer patients. This study aimed to assess the cost-utility of the eHealth application Oncokompas among incurably ill cancer patients, compared to care as usual. In this randomized controlled trial, patients were randomized into the intervention group (access to Oncokompas) or the waiting-list control group (access after three months). Healthcare costs, productivity losses, and health status were measured at baseline and three months. Intervention costs were also taken into account. Non-parametric bootstrapping with 5000 replications was used to obtain 95% confidence intervals around the incremental costs and quality-adjusted life years (QALYs). A probabilistic approach was used because of the skewness of cost data. Altogether, 138 patients completed the baseline questionnaire and were randomly assigned to the intervention group (69) or the control group (69). In the base case analysis, mean total costs and mean total effects were non-significantly lower in the intervention group (−€806 and −0.01 QALYs). The probability that the intervention was more effective and less costly was 4%, whereas the probability of being less effective and less costly was 74%. Among patients with incurable cancer, Oncokompas does not impact incremental costs and seems slightly less effective in terms of QALYs, compared to care as usual. Future research on the costs of eHealth in palliative cancer care is warranted to assess the generalizability of the findings of this study.

Saowalak Turongkaravee,1 Naiyana Praditsitthikorn,2 Thundon Ngamprasertchai,3 Jiraphun Jittikoon,4 Surakameth Mahasirimongkol,5 Chonlaphat Sukasem,6– 9 Wanvisa Udomsinprasert,4 Olivia Wu,10 Usa Chaikledkaew11,12 1Social, Economic and Administrative Pharmacy (SEAP) Graduate Program, Faculty of Pharmacy, Mahidol University, Bangkok, Thailand; 2Department of Disease Control, Ministry of Public Health, Nonthaburi, Thailand; 3Department of Clinical Tropical Medicine, Faculty of Tropical Medicine; Mahidol University, Bangkok, Thailand; 4Department of Biochemistry, Faculty of Pharmacy, Mahidol University, Bangkok, Thailand; 5Department of Medical Sciences, Medical Genetics Center, Medical Life Sciences Institute, Ministry of Public Health, Nonthaburi, Thailand; 6Division of Pharmacogenomics and Personalized Medicine, Department of Pathology, Faculty of Medicine Ramathibodi Hospital, Mahidol University, Bangkok, Thailand; 7Laboratory for Pharmacogenomics, Somdech Phra Debaratana Medical Center (SDMC), Ramathibodi Hospital, Bangkok, Thailand; 8Pharmacogenomics and Precision Medicine, The Preventive Genomics & Family Check-Up Services Center, Bumrungrad International Hospital, Bangkok, Thailand; 9MRC Centre for Drug Safety Science, Department of Pharmacology and Therapeutics, Institute of Systems, Molecular and Integrative Biology, University of Liverpool, Liverpool, L69 3GL, UK; 10Health Economics and Health Technology Assessment (HEHTA), Institute of Health and Wellbeing, University of Glasgow, Glasgow, UK; 11Social and Administrative Pharmacy Division, Department of Pharmacy, Faculty of Pharmacy, Mahidol University, Bangkok, Thailand; 12Mahidol University Health Technology Assessment (MUHTA) Graduate Program, Mahidol University, Bangkok, ThailandCorrespondence: Usa Chaikledkaew, Social and Administrative Pharmacy Division, Department of Pharmacy, Faculty of Pharmacy, Mahidol University, 447 Sri-Ayudhaya Road, Rajathevi, Bangkok, 10400, Thailand, Tel +662-644-8679 ext 5317, Fax +662-644-8694, Email usa.chi@mahidol.ac.thPurpose: Pharmacogenetics (PGx) testing is one of the methods for determining whether individuals are at risk of adverse drug reactions (ADRs). It has been reported that multiple-PGx testing, a sequencing technology, has a higher predictive value than single-PGx testing. Therefore, this study aimed to determine the most cost-effective PGx testing strategies for preventing drug-induced serious ADRs in human immunodeficiency virus (HIV)-infected patients.Patients and Methods: Potential strategies, including 1) single-PGx esting (ie, HLA-B&ast;57:01 testing before prescribing abacavir, HLA-B&ast;13:01 testing before prescribing co-trimoxazole and dapsone, and NAT2 testing before prescribing isoniazid) and 2) multiple-PGx testing as a combination of four single-gene PGx tests in one panel, were all compared to no PGx testing (current practice). To evaluate total cost in Thai baht (THB) and quality-adjusted life years (QALYs) for each strategy-based approach to a societal perspective, a hybrid decision tree and Markov model was constructed. Incremental cost-effectiveness ratios (ICERs) were estimated. Uncertainty, threshold, and scenario analyses were all performed.Results: Before prescribing HIV therapy, providing single or multiple-PGx testing might save roughly 68 serious ADRs per year, and the number needed to screen (NNS) to avoid one serious ADR was 40. Consequently, approximately 35% and 40% of the cost of ADR treatment could be avoided by the implementation of single- and multiple-PGx testing, respectively. Compared with no PGx testing strategy, the ICERs were 146,319 THB/QALY gained for single-PGx testing and 152,014 THB/QALY gained for multiple-PGx testing. Moreover, the probability of multiple-PGx testing being cost-effective was 45% at the Thai willingness to pay threshold of 160,000 THB per QALY. Threshold analyses showed that multiple-PGx testing remained cost-effective under the range of cost, sensitivity at 0.95– 1.00 and specificity at 0.98– 1.00.Conclusion: Single and multiple-PGx testing might be cost-effective options for reducing the incidence of drug-induced serious ADRs in people living with HIV.Keywords: pharmacogenetic, adverse drug reactions, HIV, cost-utility analysis, economic evaluation

Introduction. Intraoperative control of bleeding during surgical treatment of juvenile nasopharyngeal angiofibroma (JAN) remains controversial. There is no consensus as to the routine use of preoperative embolization, given its high cost and inconsistent availability of the procedure in care centers. There were no publications devoted to the comprehensive pharmacoeconomic analysis of preoperative vascular embolization (PVE) occlusion classes on the surgical treatment of JAN by the time of publication Aim. To evaluate pharmacoeconomic value of preoperative vascular embolization (PVE) occlusion classes (non-embolized, incomplete and complete) on the surgical treatment of JAN.Materials and methods. A total of 139 patients who underwent JNA surgical resection in 2013-2021 time period were identified. All of these patients were treated in National Medical Research Center for Children's Hematology, Oncology and Immunology named after Dmitry Rogachev. We evaluated JNA surgical resection outcomes, length of stay (LOS) with prognostic factors using a multiple linear regression (MLR), cost-per-day (CPD), cost-minimization analysis (CMA), cost-utility ratio (CUR), willingness to pay (WTP) with purchasing power parity (PWP).Results and discusion. Surgical transnasal endoscopic treatment of JAN in the quaternary healthcare center, regardless of the degree of embolization and the presence/absence of intraoperative blood transfusion, is at the willingness-to-pay threshold for the healthcare system of the Russian Federation. The most cost-utility method of treatment remains the absence of embolization (within clips placed in the larger diameter vessels). and the absence of blood transfusion, the least cost-utility method is the incomplete embolization, regardless of the presence/absence of blood transfusion.Conclusion. The results of the pharmacoeconomic study emphasize the high significance of the rejection of routine preoperative vascular embolization of JAN and implementation of standard risk-adapted treatment algorithm for indications of preoperative vascular embolization. Which is particularly important for resource allocations and health policy decision making to increase the efficacy and safety of the JNA treatment.

Aims: To compare the cost-utility of removable brace compared with cast in the management of adult patients with ankle fracture. Methods: A within-trial economic evaluation conducted from the UK NHS and personnel social services (PSS) perspective. Health resources and quality-of-life data were collected as part of the Ankle Injury Rehabilitation (AIR) multicentre, randomized controlled trial over a 12-month period using trial case report forms and patient-completed questionnaires. Cost-utility analysis was estimated in terms of the incremental cost per quality adjusted life year (QALY) gained. Estimate uncertainty was explored by bootstrapping, visualized on the incremental cost-effectiveness ratio plane. Net monetary benefit and probability of cost-effectiveness were evaluated at a range of willingness-to-pay thresholds and visualized graphically. Results: The incremental cost and QALYs of using brace over a 12-month period were £46.73 (95% confidence interval (CI) £-9 to £147) and 0.0141 (95% CI -0.005 to 0.033), respectively. The cost per QALY gained was £3,318. The probability of brace being cost-effective at a £30,000 per QALY willingness-to-pay threshold was 88%. The results remained robust to a range of sensitivity analyses. Conclusion: This within-trial economic evaluation found that it is probable that using a removable brace provides good value to the NHS when compared to cast, in the management of adults with ankle fracture. Cite this article: Bone Jt Open 2022;3(6):455–462.

Abstract Background Olanzapine has been shown to have an additive effect on the three-drug antiemetic therapy consisting of aprepitant, palonosetron, and dexamethasone, in a highly emetogenic cisplatin-containing chemotherapy. Although olanzapine may be more economical than aprepitant or palonosetron, an adequate cost-efficacy analysis has not been conducted. Methods We conducted a cost-utility analysis to evaluate the cost-effectiveness of olanzapine use in four-drug antiemetic therapy among Japanese patients. We simulated model patients treated with highly emetogenic cisplatin-containing chemotherapy and developed a decision-analytical model of patients receiving triple antiemetic therapy with or without olanzapine in an inpatient setting. The cost and probabilities of each treatment were calculated from the perspective of the Japanese healthcare payer. The probabilities, utility value, and other costs were obtained from published sources. One-way and probabilistic sensitivity analyses were conducted to examine the influence of each parameter on the model and the robustness of a base-case analysis. Threshold analysis was conducted to determine the cost of olanzapine that would make the incremental cost-effectiveness ratio (ICER) equivalent to the threshold ICER). The threshold incremental cost-effectiveness ratio was set at 5 million Japanese Yen (JPY) per quality-adjusted life-year (QALY) gained. Results The cost was 10,238 JPY in the olanzapine regimen and 9719 JPY in the non-olanzapine regimen. The QALY gained were 0.01065 QALYs and 0.01029 QALYs in the olanzapine and non-olanzapine regimen, respectively. The incremental cost of the olanzapine regimen relative to the non-olanzapine regimen was 519 JPY, and the incremental QALYs were 0.00036 QALY, resulting in an ICER of 1,428,675 JPY per QALY gained. In the one-way sensitivity analysis, the results were most sensitive to the utility value of incomplete control. The probabilistic sensitivity analysis revealed the probability that the ICER was below the willingness-to-pay, and the incremental QALYs was positive was 96.2%. The calculated cost of olanzapine per 5 mg that would make the incremental cost-effectiveness ratio equivalent to the threshold incremental cost-effectiveness ratio was calculated to be 475 JPY. Conclusions Olanzapine was cost-effective in the four-drug antiemetic therapy for Japanese patients treated with highly emetogenic cisplatin-containing chemotherapy.

Background: Hernia surgery procedures are among the most frequently performed in Bulgaria. An open, mesh-based repair is a standard method for hernia repair. From a societal perspective, a cost-utility analysis of open hernia surgical procedures performed in Bulgaria is necessary in light of the economic and social burden that poses this health issue. The aim of the study was to perform an economic evaluation of the quality of health results after a conventional elective hernia operation with implanted light and standard meshes.

IntroductionSince September 2020, Chinese populations aged > 3 years have been encouraged to receive a two-dose inoculation with vaccines against coronavirus disease 2019 (COVID-19). This study aims to evaluate the cost-effectiveness of the current vaccination strategy amongst the general population in mainland China from a societal perspective.MethodsIn this study, we construct a decision tree with Markov models to compare the economic and health consequences of the current vaccination strategy versus a no-vaccination scenario, over a time horizon of one year and an annual discount rate of 5%. Transition probabilities, health utilities, healthcare costs, and productivity losses are estimated from literature. Outcome measures include infection rates, death rates, quality-adjusted life years (QALYs), and costs. The incremental cost-effectiveness ratio (ICER) is then calculated to evaluate the cost-effectiveness of the current vaccination strategy, and both one-way deterministic sensitivity analysis and probabilistic sensitivity analysis (PSA) are applied to assess the impact of uncertainties on results.ResultsOur simulation indicates that compared with a no-vaccination scenario, vaccination amongst the general population in mainland China would reduce the infection rate from 100% to 45.3% and decrease the death rate from 6.8% to 3.1%. Consequently, the strategy will lead to a saving of 37,664.77 CNY (US$5,256.70) and a gain of 0.50 QALYs per person per year on average (lifetime QALY and productivity loss due to immature death are included). The cost-saving for each QALY gain is 74,895.69 CNY (US$10,452.85). Result of the PSA indicates that vaccination is the dominating strategy with a probability of 97.9%, and the strategy is cost-effective with a probability of 98.5% when the willingness-to-pay (WTP) is 72,000 CNY (US$10,048.71) per QALY.ConclusionCompared with a no-vaccination scenario, vaccination among the general population in mainland China is the dominating strategy from a societal perspective. The conclusion is considered robust in the sensitivity analyses.

Background: Lumbar interbody fusion techniques treat degenerative lumbar diseases effectively. Minimally invasive lateral lumbar interbody fusion (LLIF) decreases soft tissue disruption and accelerates recovery better than standard open posterior lumbar interbody fusion (PLIF). However, the material cost of LLIF is high, especially in Thailand. The cost-effectiveness of LLIF and PLIF in developing countries is unclear. This study compared the cost-utility and clinical outcomes of LLIF and PLIF in Thailand. Methods: Data from patients with lumbar spondylosis who underwent single-level LLIF and PLIF between 2014 and 2020 were retrospectively reviewed. Preoperative and 1-year follow-up EuroQol-5D-5L and healthcare costs were collected. A cost-utility analysis with a lifetime time horizon was performed using a societal perspective. Outcomes are reported as the incremental cost-effectiveness ratio (ICER) and quality-adjusted life-year (QALY) gained. A Thai willingness-to-pay threshold of 5003 US dollars (USD) per QALY gained was used. Results: The 136 enrolled patients had a mean age of 62.26 ± 11.66 years. Fifty-nine patients underwent LLIF, while 77 underwent PLIF. The PLIF group experienced greater estimated blood loss (458.96 vs 167.03 ml; P < 0.001), but the LLIF group had a longer operative time (222.80 vs 194.62 min; P = 0.007). One year postoperatively, the groups' Oswestry Disability Index and EuroQol-Visual Analog Scale scores were improved without statistical significance. The PLIF group had a significantly better utility score than the LLIF group (0.89 vs 0.84; P = 0.023). LLIF's total lifetime cost was less than that of PLIF (30,124 and 33,003 USD). Relative to PLIF, LLIF was not cost-effective according to the Thai willingness-to-pay threshold, with an ICER of 19,359 USD per QALY gained. Conclusions: LLIF demonstrated lower total lifetime cost from a societal perspective. Regard to our data, at the 1-year follow-up, the improvement in patient quality of life was less with LLIF than with PLIF. Additionally, economic evaluation modeling based on the context of Thailand showed that LLIF was not cost-effective compared with PLIF. A strategy that facilitates the selection of patients for LLIF is required to optimize patient benefits. [ABSTRACT FROM AUTHOR]

In many countries, intervention costs are not covered by public health care. A critical basis for deciding whether an intervention is covered or not is to analyse the relation between benefits and costs of the intervention, and to quantify the consequential costs. In this study, a cost‐utility analysis was computed to investigate the costs of individualized dyslexia intervention while quantifying the benefit in terms of health‐related quality of life in a sample of 36 individuals with dyslexia. In addition, educational outcomes and costs of untreated dyslexia for the society were estimated using information for class repetition, school success, and unemployment rates from previous studies and official statistics. A significant increase in quality of life with medium effect sizes was found across all quality‐of‐life measures. Increases in quality of life were domain‐specific, thus occurring specifically in those domains that are affected by learning disorders. The cost‐utility ratio was 9,782 Euros per quality adjusted life years (QALYs), which is in line with similar therapy forms, such as speech therapy. The loss of productivity for untreated dyslexia in the German population was estimated for class repetition and reduced income due to lower school degrees. The cost‐utility analysis and the calculation of consequential costs suggest that the dyslexia intervention is cost‐effective. [ABSTRACT FROM AUTHOR]

Background: Multiple sclerosis (MS) is an inflammatory, neurodegenerative disease of the central nervous system which results in disability over time and reduced quality of life. To increase the sensitivity of the EQ-5D-5L for psychosocial health, four bolt-on items from the AQoL-8D were used to create the nine-item EQ-5D-5L-Psychosocial. We aimed to externally validate the EQ-5D-5L-Psychosocial in a large cohort of people with MS (pwMS) and explore the discriminatory power of the new instrument with EQ-5D-5L/AQoL-8D. Methods: A large representative sample from the Australian MS Longitudinal Study completed the AQoL-8D and EQ-5D-5L (including EQ VAS) and both instruments health state utilities (HSUs) were scored using Australian tariffs. Sociodemographic/clinical data were also collected. External validity of EQ-5D-5L-Psychosocial scoring algorithm was assessed with mean absolute errors (MAE) and Spearman's correlation coefficient. Discriminatory sensitivity was assessed with an examination of ceiling/floor effects, and disability severity classifications. Results: Among 1683 participants (mean age: 58.6 years; 80% female), over half (55%) had moderate or severe disability. MAE (0.063) and the distribution of the prediction error were similar to the original development study. Mean (± standard deviation) HSUs were EQ-5D-5L: 0.58 ± 0.32, EQ-5D-5L-Psychosocial 0.62 ± 0.29, and AQoL-8D: 0.63 ± 0.20. N = 157 (10%) scored perfect health (i.e. HSU = 1.0) on the EQ-5D-5L, but reported a mean HSU of 0.90 on the alternative instruments. The Sleep bolt-on dimension was particularly important for pwMS. Conclusions: The EQ-5D-5L-Psychosocial is more sensitive than the EQ-5D-5L in pwMS whose HSUs approach those reflecting full health. When respondent burden is taken into account, the EQ-5D-5L-Psychosocial is preferential to the AQoL-8D. We suggest a larger confirmatory study comparing all prevalent multi-attribute utility instruments for pwMS. [ABSTRACT FROM AUTHOR]

Introduction: The main research purpose is to compare the long‐term cost‐effectiveness of semaglutide (SEMA) with that of dulaglutide (DULA) for patients with inadequately controlled type 2 diabetes throughout their lifetime. If necessary, the second aim is to investigate a further price cut for SEMA to provide sound advice for government drug price adjustments. Methods: Cost‐utility analysis was performed by the United Kingdom Prospective Diabetes Study Outcomes Model 2 (UKPDS OM2) from the perspective of health care providers in China. Baseline characteristics and clinical efficacy of SEMA and DULA were sourced from the high‐dose comparison in the SUSTAIN‐7 trial. A binary search was used to identify the scope for further reduction in the price of SEMA. The impact of individual parameters was assessed with sensitivity analyses. Results: Main analysis (SEMA vs. DULA) revealed a mean difference in quality‐adjusted life years (QALYs) of 0.04 QALYs and costs of $1132.29. The incremental cost‐utility ratio was $26 957.44/QALY, showing that SEMA was a better option compared with DULA. In sensitivity analyses, the discount rate made the greatest contribution to the incremental cost‐utility ratio. In the binary search, there was still scope to reduce the SEMA cost further by approximately 6.83% to be cost‐effective, taking DULA as a reference. Conclusion: After its addition to the National Medical Insurance System in China, SEMA is expected to be a cost‐effective choice compared with DULA for patients with type 2 diabetes with inadequately controlled from the cost‐utility analysis. However, there is still scope to reduce the annual cost of SEMA further. [ABSTRACT FROM AUTHOR]

Study Design.: Retrospective analysis of prospectively collected data. Objective.: Assess the cost-utility of anterior cervical discectomy and fusion (ACDF) performed in the ambulatory surgery center (ASC) versus inpatient hospital setting for Medicare and privately insured patients at one-year follow-up. Summary of Background Data.: Outpatient ACDF has gained popularity due to improved safety and reduced costs. Formal cost-utility studies for ambulatory versus inpatient ACDF are lacking, precluding an accurate assessment of cost-effectiveness. Materials and Methods.: A total of 6504 patients enrolled in the Quality Outcomes Database (QOD) undergoing one-level to two-level ACDF at a single ASC (520) or the inpatient hospital setting (5984) were compared. Propensity matching generated 748 patients for analysis (374 per cohort). Demographic data, resource utilization, patient-reported outcome measures, and quality-adjusted life-years (QALYs) were assessed. Direct costs (1-year resource use×unit costs based on Medicare national allowable payment amounts) and indirect costs (missed workdays×average US daily wage) were recorded. Incremental cost-effectiveness ratios were calculated. Results.: Complication rates and improvements in patient-reported outcome measures and QALYs were similar between groups. Ambulatory ACDF was associated with significantly lower total costs at 1 year for Medicare ($5879.46) and privately insured ($12,873.97) patients, respectively. The incremental cost-effectiveness ratios for inpatient ACDF was $3,674,662 and $8,046,231 for Medicare and privately insured patients, respectively, reflecting unacceptably poor cost-utility. Conclusion.: Inpatient ACDF is associated with significant increases in total costs compared to the ASC setting without a safety, outcome, or QALY benefit. The ASC setting is a dominant option from a health economy perspective for first-time one-l to two-level ACDF in select patients compared to the inpatient hospital setting. [ABSTRACT FROM AUTHOR]

STN1013001 is an innovative latanoprost cationic emulsion for open-angle glaucoma/ocular hypertension (OAG/OHT) and ocular surface disease (OSD). A 5-year, 7 health states, 1-year cycle early Markov model-supported cost-utility analysis (CUA) of STN1013001 vs. other latanoprost formulations (Latanoprost) followed the Italian National Health Service (INHS) perspective. One-way, probabilistic and scenario sensitivity analyses tested the uncertainty of the baseline results. Value of information analysis (VOIA) investigated the potential cost-effectiveness of collecting further evidence. Over 5 years, the Markov model-supported CUA predicts STN1013001 to be potentially highly cost-effective vs. Latanoprost (+€57.60 cost at €2020 values; +0.089 Quality-Adjusted Life Years). The Incremental Cost-Utility Ratio (€647.65) falls well below the lower limit of the acceptability range proposed for Italy (€25,000-€40,000). Sensitivity analyses confirmed the robustness of the baseline findings. VOIA highlighted that further information might only be cost-effective for OAG/OHT utilities and OSD-related disutility. STN1013001 is potentially highly cost-effective and strongly dominant vs. Latanoprost for OAG/OHT+OSD patients from the INHS perspective. These findings should be re-assessed using the data from the ongoing Phase III trial (NCT04133311) comparing the efficacy and safety of STN1013001 vs. Latanoprost and with future real-world CUAs upon the availability of STN1013001 on the Italian market. [ABSTRACT FROM AUTHOR]

Purpose: To evaluate the cost-effectiveness of Platelet Rich Plasma (PRP), Plasma Rich in Growth Factors (PRGF), Hyaluronic Acid (HA) and ozone as effective treatment approaches in knee osteoarthritis management from Iran Health care perspective. Methods: A decision tree model was conducted to assess the cost-effectiveness of four common intra-articular treatment approaches in patients with mild and moderate knee osteoarthritis. The data on clinical effectiveness was obtained from a randomized controlled trial (RCT) conducted in Iran and used to estimate utility values. The direct medical costs were estimated according to tariffs for public medical centers and hospitals, approved by the Iran Ministry of Health and Medical Education in 2021. The incremental cost-effectiveness ratio (ICER) and the net monetary benefit (NMB) were used to evaluate the cost-utility analysis. Deterministic and probabilistic sensitivity analyses are performed to investigate the robustness of the results and account for the different sources of uncertainty. Results: In this study, HA intra-articular injection-related costs ($581.67/patient) were defined as the highest cost, followed by PRGF ($328.10/patient), PRP (318.58/patient), and Ozone (103.20/patient). According to the utility value, PRP and PRGF (0.68) have the same and the most utility among Intra-articular injections in knee osteoarthritis management. However, the PRP injection method was identified as the most cost-effective intervention due to its high NMB and ICER estimates. Based on the Monte Carlo Simulation, PR intervention, compared to other ones, was introduced as the dominant strategy regarding knee OA management, with a WTP of $10,000 for 100% of cases. Conclusion: The study result demonstrated that intra-articular injection of PRP, compare to other injections, is a cost-effective treatment option for patients with mild and moderate knee osteoarthritis. In addition, intra-articular injection of PRP was identified as the best injection, with the highest level of net monetary benefit, for knee OA management. [ABSTRACT FROM AUTHOR]

To analyse the cost-effectiveness (CE) of implementing different newborn hearing screening protocols in a low- to middle-income country. A decision analytical model with a 78-year time horizon. Direct medical, direct non-medical and indirect costs were collected from 126 subjects in southern Thailand. Various protocols involving universal newborn hearing screening (UNHS) and targeted newborn hearing screening (TNHS), using two technologies, namely automated otoacoustic emissions (aOAEs) and automated auditory brainstem responses (aABRs), were evaluated. Incremental cost-effectiveness ratios (ICERs) were calculated for all protocols in United States dollars (US$)/quality-adjusted life year (QALY) gained. Also, probabilistic sensitivity analyses with 1000 trials for each specific protocol were performed. The ICERs of UNHS with aOAE, UNHS with aABR, TNHS with aABR and UNHS with optimised baseline parameters were 3702, 3545, 1545 and 2483 US$/QALY gained, respectively. With the CE threshold of 5000 US$/QALY gained, the chances of ICERs to be cost-effective for UNHS with aOAE, UNHS with aABR, TNHS with aABR and UNHS with optimised baseline parameters were 72, 77, 93 and 94%, respectively. All screening protocols were considered as cost-effective, and a very high chance of being cost-effective for UNHS could be achieved when certain baseline parameters were optimised. [ABSTRACT FROM AUTHOR]

Chronic kidney disease is ranked fourth among the top 10 causes of death in Saudi Arabia. Renal transplantation has been recognized as the treatment of choice compared with long-term dialysis to maintain graft survival and prolong a patient's healthy living. Immunosuppressants (ISs) must be administered lifelong. The choice between IS therapies can be challenging because of the similarity in efficacy with some differences in adverse events profile. The objective of this study was to assess the cost-effectiveness of different IS regimens in Saudi Arabia. A 25-year Markov model was developed based on a previously published study from the Saudi Ministry of Health payer perspective. Efficacy parameters were driven from the literature, whereas cost data were estimated from the Ministry of Health database. A Monte Carlo simulation was conducted to test the base-case model results' robustness. All comparators resulted in 6.2 quality-adjusted life-years (QALYs) except for Advagraf® treatment (5.5 QALYs). Generic tacrolimus plus mycophenolate mofetil (MMF) will cost 70 701.45 US dollars ($) (Saudi riyal 265 130.44) per patient to gain 6.2 QALYs over 25 years' time horizon. In the improved adherence scenario, Envarsus® plus generic MMF generated 9.6 QALYs with a cost of $59 849 per patient. Monte Carlo simulation results have shown that generic tacrolimus is still the cheapest treatment option compared with other treatment arms. The current analysis suggested that all IS options are not cost-effective strategies relative to the willingness-to-pay threshold of $20 000. Nevertheless, Envarsus plus generic MMF regimen could become the most cost-effective regimen at different willingness-to-pay thresholds. • To the best of our knowledge, this is the first economic evaluation conducted on renal transplant patients in the Kingdom of Saudi Arabia. The current analysis studied the cost and outcomes of different Immunosuppressant (IS) regimens from a payer perspective for 25 years' time horizon with an annual cycle length. • The comparators included in the analysis were IS regimes used in the Saudi Ministry of Health (MOH) clinical practice. The outcome measures were majorly derived from the literature; nevertheless, the cost of drugs was obtained from National Unified Procurement Company and resource utilization from the MOH services price list. • The current analysis demonstrated that IS therapy is not cost-effective at the US dollar 20 000 willingness-to-pay threshold, although they were associated with substantial clinical and patient-reported outcomes benefits, which is not necessarily advised against the use of ISs for life; nevertheless, the prices of medications might be revised for resubmission at the MOH level. Further studies are required to reconduct the analysis by adopting local patients and outcomes data to reflect real-world clinical practice in the Kingdom of Saudi Arabia. [ABSTRACT FROM AUTHOR]

The advantage of tenecteplase (TNK) over alteplase (ALT) in managing acute ischemic stroke (AIS) has been reported, but the cost-effectiveness of these two strategies has not received as much attention. The objective of this study was to compare TNK and ALT for the management of AIS patients in Iran in terms of cost-effectiveness. This study was carried out from the payer's perspective in Iran, with a lifetime horizon. A full economic evaluation model was designed as a decision tree and a Markov model. After defining different Markov states, each health state was assigned a utility value, and quality-adjusted life year (QALY) was estimated using that value. The incremental cost-effectiveness ratio (ICER) was ultimately used for evaluating the comparative cost-effectiveness. Both deterministic and probabilistic sensitivity analyses were carried out. Compared to ALT, TNK can save approximately 4333.81 USD, and is able to increase one unit of QALY while saving approximately 17,450.29 USD. So, Base-case results showed that TNK strongly dominates ALT. Moreover, the base case results were strongly confirmed by deterministic and probabilistic sensitivity analysis. Base-case and sensitivity analysis showed that TNK is the dominant strategy compared to ALT for the management of AIS patients. [ABSTRACT FROM AUTHOR]

To assess the cost-effectiveness of tezepelumab as add-on maintenance therapy compared with standard of care (SoC) for the treatment of patients with severe asthma in Canada. A cost utility analysis was conducted using a Markov cohort model with five health states ("controlled asthma", "uncontrolled asthma", "previously controlled asthma with exacerbation", "previously uncontrolled asthma with exacerbation", and "death"). Tezepelumab plus SoC was compared to SoC (high-dose inhaled corticosteroids plus long-acting beta agonist) using efficacy estimates derived from the NAVIGATOR (NCT03347279) and SOURCE (NCT03406078) trials. The model included the costs of therapy, administration, resource use for disease management, and adverse events. Utility estimates were calculated using a mixed-effects regression analysis of the NAVIGATOR and SOURCE trials. A Canadian public payer perspective was used with a 50-year time horizon, a 1.5% annual discount rate, and the base case analysis was conducted probabilistically. A key scenario analysis assessed the cost-effectiveness of tezepelumab compared with currently reimbursed biologics informed by an indirect treatment comparison. The base case analysis suggested that tezepelumab plus SoC was associated with a quality-adjusted life-year (QALY) gain of 1.077 compared with SoC alone at an incremental cost of $207,101 (2022 Canadian dollars), resulting in an incremental cost-utility ratio of $192,357/QALY. The key scenario analysis demonstrated that tezepelumab was dominant against all currently reimbursed biologics, with higher incremental QALYs (ranging from 0.062 to 0.407) and lower incremental costs (ranging from −$6,878 to −$1,974). Additionally, when compared against currently reimbursed biologics in Canada, tezepelumab had the highest probability of being cost-effective across all willingness-to-pay (WTP) thresholds. Tezepelumab provided additional life years and QALYs at additional cost compared with SoC in Canada. In addition, tezepelumab dominated (i.e. more effective, less costly) the other currently reimbursed biologics. [ABSTRACT FROM AUTHOR]

Paediatric growth hormone deficiency (pGHD) manifests as growth failure associated with inadequate growth hormone (GH) production. Daily injections of recombinant human GH (dGH) [somatropin] is the current standard of care, which has been shown to be well tolerated and effective, but associated with suboptimal adherence, leading to reduced effectiveness. Somatrogon, a once-weekly injectable long-acting human GH, has demonstrated clinical non-inferiority and significantly lower life interference (i.e. treatment burden) vs. somatropin in two Phase 3 studies. This work evaluated cost-effectiveness and cost-utility of somatrogon vs dGHs from an Irish payer perspective. A Markov model was developed for patients starting somatrogon or dGHs treatment at 3–12 years and continuing up to achievement of near adult height (NAH), with growth driven by trial-based height velocity (HV) and treatment-specific adherence. Patients could discontinue treatment at the end of Year 1 (4%). DGH adherence (95.3%–65% over treatment duration) and adherence-growth relationship were based on published evidence. Higher Year 1 adherence of 4%, tapering over time, for somatrogon vs. dGHs was based on clinical consultation. Treatment costs, monitoring costs and costs due to different wastage types (device setting and adherence) were sourced from local data. Health utilities based on height and injection frequency were derived from published literature. Scenario analysis, deterministic and probabilistic sensitivity analysis were performed. Somatrogon treatment led to 1.87–3.66 cm greater NAH gain and 0.21–0.50 higher quality adjusted life years (QALYs) vs. dGHs, across the base case and scenarios evaluated. Somatrogon treatment was associated with cost savings of €5,699–€21,974 and lower cost per cm gained vs. dGHs (€197–€527), per patient. Somatrogon was cost-effective vs. dGHs, with the result consistent across the sensitivity analyses conducted. Somatrogon weekly injections were estimated to result in higher NAH, higher QALYs, lower overall costs and lower costs per cm gained than dGHs, in pGHD. [ABSTRACT FROM AUTHOR]

Background: Understanding what an economic evaluation is, how to interpret it, and what it means for making choices in a health delivery context is necessary to contribute to decisions about healthcare resource allocation. The aim of this paper to demystify the working parts of a health economic evaluation, and explain to clinicians and clinical researchers how to read and interpret cost-effectiveness research. Main body: This primer distils key content and constructs of economic evaluation studies, and explains health economic evaluation in plain language. We use the PICOT (participant, intervention, comparison, outcome, timeframe) clinical trial framework familiar to clinicians, clinical decision-makers, and clinical researchers, who may be unfamiliar with economics, as an aide to reading and interpreting cost-effectiveness research. We provide examples, primarily of physiotherapy interventions for osteoarthritis. Conclusions: Economic evaluation studies are essential to improve decisions about allocating resources, whether those resources be your time, the capacity of your service, or the available funding across the entire healthcare system. The PICOT framework can be used to understand and interpret cost-effectiveness research. [ABSTRACT FROM AUTHOR]

• Late-life depression can impact health (QALYs), retirement, and consumer spending. • We use panel VAR model of mental and physical health, labor supply, and consumption. • Late-life depression costs 0.85 to 2.1 quality-adjusted life years per person. • Total willingness-to-pay cost of depression estimated at $180–360 billion annually. • Cost of late-life depression is falling over birth cohorts but growing more unequal. We quantify the welfare cost of depression among older Americans by estimating a panel VAR model of mental and physical health, labor supply, and consumption using data from the Health and Retirement Study. We use the estimated model and age sixty joint distribution of outcomes to simulate life-cycle paths with and without prevalence of depressive symptoms after age sixty. We estimate that the prevalence of late-life depressive symptoms costs an average of between 0.85 and 2.1 years in quality-adjusted life expectancy per person. Moreover, depression may result in an average loss of labor supply of up to 1.1 months and lifetime consumption of up to $16,000. Combining into a single compensating variation welfare metric, we estimate a bound on the average welfare cost of depression of 8–15% of annual consumption after age sixty. On aggregate, this amounts to roughly $180–360 billion annually. We also project that while the average welfare cost of late-life depression is declining slightly over birth cohorts, the welfare burden is becoming significantly more unequal. [ABSTRACT FROM AUTHOR]

Summary: During the COVID‐19 pandemic, ICU bed shortages sparked a discussion about resource allocation. We aimed to analyse the value of ICU treatment of COVID‐19 from a patient‐centred health economic perspective. We prospectively included 49 patients with severe COVID‐19 and calculated direct medical treatment costs. Quality of life was converted into aggregated quality‐adjusted life years using the statistical remaining life expectancy. Costs for non‐treatment as the comparator were estimated using the value of statistical life year approach. We used multivariable linear or logistic regression to identify predictors of treatment costs, quality of life and survival. Mean (SD) direct medical treatment costs were higher in patients in ICU with COVID‐19 compared with those without (£60,866 (£42,533) vs. £8282 (£14,870), respectively; p < 0.001). This was not solely attributable to prolonged ICU length of stay, as costs per day were also higher (£3115 (£1374) vs. £1490 (£713), respectively; p < 0.001), independent of overall disease severity. We observed a beneficial cost‐utility value of £7511 per quality‐adjusted life‐year gained, even with a more pessimistic assumption towards the remaining life expectancy. Extracorporeal membrane oxygenation therapy provided no additional quality‐adjusted life‐year benefit. Compared with non‐treatment (costs per lost life year, £106,085), ICU treatment (costs per quality‐adjusted life‐year, £7511) was economically preferable, even with a pessimistic interpretation of patient preferences for survival (sensitivity analysis of the value of statistical life year, £48,848). Length of ICU stay was a positive and extracorporeal membrane oxygenation a negative predictor for quality of life, whereas costs per day were a positive predictor for mortality. These data suggest that despite high costs, ICU treatment for severe COVID‐19 may be cost‐effective for quality‐adjusted life‐years gained. [ABSTRACT FROM AUTHOR]

Objective: To evaluate the economical effect of zoledronic acid and ibandronate sodium in the treatment of patients with bone metastases of malignant tumors. Methods: A retrospective analysis of 86 cases of bone metastases of malignant tumors in × Hospital from January 2020 to January 2022, of which 46 cases were treated with zoledronic acid (group A) and 40 cases were treated with ibandronate sodium (Group B), compare the pain control effect, adverse reactions and the SF-36 score between the two groups, and summarize the cost of the two treatment options, and use the principles of pharmaco economics to conduct a cost-utility analysis. Results: The pain control rate in group A was 82.61%, which was not statistically different from 85.00% in group B (P>0.05); after treatment, the physiological function (PF) and physiological function (RP) in the SF-36 score of the two groups, body pain (BP), general health (GH), social function (SF), mental health (MH), emotional function (RE), and vitality (VT) were significantly higher than those before treatment (P<0.05), but the group There was no statistically significant difference between groups (P>0.05); the incidence of adverse reactions in group A and group B was similar, and the difference was not statistically significant (P>0.05); the drug cost (C1) and total cost (C) of group A were respectively (4052.50±80.50) yuan and (4453.87±123.56) yuan, which were higher than (2025.50±70.15) yuan and (2395.44±109.17) yuan in group B, and the differences were statistically significant (P<0.05); group A cost- The utility ratio (CUR) was judged by the SF-36 scale to be 4189.94, and that of group B was 2829.17. Conclusion: Considering the effectiveness and economy of the drug, the use of ibandronate sodium in the treatment of bone metastases of malignant tumors. [ABSTRACT FROM AUTHOR]

This study aims to systematically identify and review published economic evaluations related to the treatment of mHSPC and assess their quality. These datas will help to provide a better understanding of these treatments and a better use of healthcare resources. The management of patients with metastatic hormone-sensitive prostate cancer (mHSPC) has been significantly modified by the availability of innovative but expensive treatments, increasing the economic burden of prostate cancer. Here, we aimed to systematically identify and review published economic evaluations (EEs) related to the treatment of mHSPC and assess their quality. A systematic search was performed of the PubMed and Cochrane databases. Three reviewers independently selected EEs by defined inclusion and exclusion cr iter ia. They extracted all data from each EE (general information, study population, data about the EE, interventions and comparators, and outcomes). They also assessed the quality of the selected EEs according to Drummond's checklist. Fourteen EEs published between 2016 and 2021 were eligible for the systematic review. The EEs found ADT + docetaxel to be the most cost-effective of all available treatments as a first-line strategy for mHSPC (abiraterone acetate plus prednisone, enzalutamide, and apalutamide). Five EEs showed that a simple price reduction of abiraterone acetate of 50% to 75% could change the results to render this treatment also cost-effective relative to that with docetaxel. Twelve EEs were of high quality, with a Drummond score ≤ 7. Analysis of the 14 EEs identified by our systematic review, amongst which 78.6% met high quality standards, showed that ADT + docetaxel tends to be the most cost-effective alternative for mHSPC. These results were assessed by sensitivity analysis. The data provided by this systematic review help to provide a better understanding of these treatments and the better use of healthcare resources. [ABSTRACT FROM AUTHOR]

Introduction: In light of the lack of an agreed international standard for how to conduct cost-effectiveness analyses (CEAs), including cost–utility analyses (CUAs) from a societal perspective, there is uncertainty regarding to what extent the inclusion of productivity losses/gains in economic evaluations can affect cost-effectiveness results and subsequently decisions on whether to recommend new health technologies. To investigate this, we conducted a systematic review of CEAs and CUAs of drug-based therapies for a set of chronic immune-mediated disorders to understand how cost elements and calculation methods related to productivity losses/gains are used, examine the impact on the incremental cost-effectiveness ratio (ICER) of including productivity costs, and explore factors that affect the inclusion of productivity loss. Methods: Databases (MEDLINE® In-process, MEDLINE, Embase and Cochrane Library) were searched from January 2010 to October 2020 by two independent reviewers for all CEAs and CUAs in adults with any of the following conditions: ankylosing spondylitis, chronic idiopathic urticaria, Crohn's disease, fibromyalgia, juvenile idiopathic arthritis, psoriasis, rheumatoid arthritis, systemic lupus erythematosus and ulcerative colitis. Relevant study data were extracted and evidence was synthesized for both qualitative and quantitative analysis. Productivity cost elements including absenteeism, presenteeism, unemployment/early retirement, premature mortality and informal care were extracted, along with the method used to determine them. A multivariate analysis was performed to identify factors associated with the inclusion of productivity loss. Results: Our searches identified 5016 records, culminating in 198 unique studies from 234 publications following screening. Most of the studies investigated rheumatoid arthritis (37.0%) or psoriasis (32.0%). The majority were CUAs, with some including both a CEA and a CUA (73.0%). Most studies used a payer perspective only (28.5%) or a societal perspective only (21.0%). Of the 49 studies incorporating productivity losses/gains, 42 reported the type of cost element used; all of these used patient absenteeism, either alone or in addition with other elements. Only 16 studies reported the method used to value productivity changes, of which eight used a human capital approach, four used a friction cost approach and four used both approaches. Twenty-eight of the 49 studies (57.1%) reported inclusion of productivity losses/gains as contributing to more favourable cost-effectiveness outcomes and ICERs, while 12 (24.5%) reported no substantial impact. On the basis of a multivariate analysis, rheumatoid arthritis as the target disease had a statistically significant association with the inclusion of productivity loss compared with psoriasis and inflammatory bowel disease. Conclusions: The results of our review suggest that incorporating productivity cost elements may positively affect cost-effectiveness outcomes in evaluations of therapeutics for immune-mediated disorders. Our work highlights the continued need for clarity when reporting how CEAs and CUAs in this disease area are conducted, in order to better inform healthcare decision-making. [ABSTRACT FROM AUTHOR]

Abstract Background Effective symptom control in painful knee osteoarthritis (OA) may improve patient quality of life. In a randomised crossover trial (NCT03381248), COOLIEF* cooled radiofrequency ablation (CRFA) reduced pain and stiffness and improved physical function and quality of life compared with intra-articular hyaluronan (HA) injections. The present study aimed to establish the cost effectiveness of CRFA versus intra-articular HA injections for treating moderate-to-severe OA knee pain from a US Medicare perspective. Methods We conducted a cost-effectiveness analysis using utility data (EQ-5D) from the randomised crossover trial of CRFA versus intra-articular HA injections, which had follow-ups at 1, 3, 6, and 12 months. Patients in the HA group with unsatisfactory outcomes (e.g., continued pain) at 6 months could cross over to CRFA. Economic analysis outcomes included quality-adjusted life-years (QALYs), costs, and cost effectiveness (cost per QALY gained). Base-case analyses were modelled on a 6-month time horizon (to trial crossover). Due to limited trial data in the HA arm beyond 6 months, scenarios explored potential outcomes to 12 months if: 1) Utility with HA persisted for a further 6 months; 2) A second HA injection was received at 6 months and achieved the same utility change for the second 6 months. In both scenarios, the CRFA arm used trial data for patients who received CRFA from baseline to 12 months. Alternative costing scenarios were also explored. Results CRFA resulted in an incremental QALY gain of 0.020 at an incremental cost of US$1707, equating to an incremental cost-effectiveness ratio (ICER) of US$84,392 per QALY over 6 months, versus intra-articular HA injections. Extending the analysis to 12 months and assuming persistence in utility in the HA arm resulted in a larger utility gain for CRFA (0.056 QALYs) and a lower ICER of US$30,275 per QALY. If patients received a second HA injection, the incremental benefit of CRFA out to 12 months was reduced (QALY gain 0.043) but was offset by the costs of the second HA injection (incremental cost US$832). This resulted in an ICER of US$19,316 per QALY. Conclusions CRFA is a cost-effective treatment option for patients with OA-related knee pain considering the typical US threshold of US$100,000/QALY.

• We reviewed 49 CUA articles in substance use disorder. • There is a lack of justification for selected health utilities in CUA. • Health economics modellers can use the HAT to assess health utility applicability. • We provide several suggestions and feedback for the HAT team to refine the tool. We aim to identify within-trial and modelled Cost-Utility Analysis (CUA) in substance use disorders (SUD) and review the applicability assessment associated with health utility used in modelled CUA. We searched Medline, Embase, EconLit and the Pharmaceutical Benefits Advisory Committee (PBAC) databases. A global systematic literature search was undertaken to determine the CUA of SUD interventions. Key characteristics of the studies and use of health utility were described. The applicability assessment associated with health utility used in modelled CUA was reviewed using The Health Utility Application Tool (HAT). The final review retrieved 49 CUA (14 within-trial and 35 modelled CUA). Three major health utility measurements were used - standard gamble, EQ-5D-5L and SF-6D. EQ-5D-5L was mainly used in within-trial CUA, whereas standard gamble, EQ-5D-5L and SF-6D were equally cited in modelled CUA and within-trial CUA. Twenty-nine articles using modelled CUA citing health utilities from published literature were assessed. Only half and one-third of CUA studies described the type of quality-of-life measure and value sets used in health utility studies, respectively. Only two-thirds showed the authors addressed questions about the similarities in clinical conditions, and health state description between health utility studies and economic evaluation studies. Justifications for chosen health utilities in modelled CUA studies were mostly absent in SUD. We suggested health economists use the HAT to make judgements when assessing health utility from published estimates. The use of this tool will increase the reliability of economic evaluation carried out to assist government and policymakers in making informed decisions around health topics. [ABSTRACT FROM AUTHOR]

Chemotherapy has helped prolong survival in patients with malignant lymphoma, enhancing their quality of life (QOL). Despite the eventual decline in the QOL of patients, the impact of initial chemotherapy remains poorly understood. A prospective patient-reported QOL survey among patients with malignant lymphoma receiving initial chemotherapy was conducted, targeting those treated at Gifu Municipal Hospital (Gifu, Japan) between January 2021 and December 2022. Surveys were conducted pre- and post-chemotherapy based on the EuroQol 5 dimensions. Drug costs were calculated using official prices and analyzed from the cost payer's perspective via cost-utility analysis. Among the 60 patients included in the present study, 28 had diffuse large B-cell lymphoma. Cyclophosphamide, doxorubicin, vincristine, prednisolone ± rituximab therapy was the most common treatment (38 patients) and demonstrated superior cost-effectiveness due to its lowest cost and change in utility value. Initial chemotherapy for patients with malignant lymphoma generally improved the QOL. Clinical trial registration: UMIN000042868 (registered on December 28, 2020). [ABSTRACT FROM AUTHOR]

Osteoarthritis (OA) is the degeneration of cartilage in joints that results in bones rubbing against each other; it causes uncomfortable symptoms such as pain, swelling, and stiffness and can lead to disability. It usually occurs in the elderly and causes a large medical burden. The aim of this study is to evaluate the cost-effectiveness between the standard treatment for osteoarthritis and standard treatment with added crystalline glucosamine sulfate at various stages. Markov analysis modeling was applied to evaluate the effect of both adding glucosamine compared to standard treatment from a societal perspective during whole patients’ lifetimes. Data input was collected from reviews in previous studies. The outcome was measured in quality-adjusted life years (QALYs), and the Incremental Cost-Effectiveness Ratio (ICER) from a societal perspective was applied with 3% and discounted for all costs and outcomes. One-way analysis via the Tornado diagram was performed to investigate the change in factors in the model. In general, adding glucosamine into the standard treatment proved to be more cost-effective compared to the standard treatment. Particularly, the early-stage addition of glucosamine in the treatment was cost-effective compared to the post-stage addition of glucosamine. The addition of supplementing crystalline glucosamine sulfate to the whole regimen at any stage was cost-effective at the willingness-to-pay (WTP) threshold.

Carlo Lazzaro,1 Cécile van Steen,2 Stephan Billeit,3 Heinrich Frauenknecht,4 Christopher Kallen,5 Stefan Pfennigsdorf,6 Ulrich Thelen,7,8 Luigi Angelillo2 1Health Economist and Research Director, Studio di Economia Sanitaria, Milan, Italy; 2Santen GmbH, München, Germany; 3Private Practicing Ophthalmologist, Lübeck, Germany; 4Private Practicing Ophthalmologist, Treuchtlingen, Germany; 5Private Practicing Ophthalmologist, Krefeld, Germany; 6Private Practicing Ophthalmologist, Polch, Germany; 7Private Practicing Ophthalmologist, Münster, Germany; 8University Hospital Muenster, Department of Ophthalmology, Münster, GermanyCorrespondence: Carlo LazzaroHealth Economist and Research Director, Studio di Economia Sanitaria, Via Stefanardo da Vimercate, 19, Milan, I-20128, Italy, Tel/Fax +39 02 2600 0516, Email carlo.lazzaro@tiscalinet.itPurpose: This study aimed to estimate the cost–utility and economic value of STN1013001, a latanoprost cationic emulsion vs other latanoprost formulations (henceforth latanoprost) in patients with open-angle glaucoma (OAG) or ocular hypertension (OHT) and concomitant ocular surface disease (OSD) in Germany.Methods: An early 5-year Markov model-supported cost–utility analysis was performed to estimate costs, quality-adjusted life years (QALYs) and life-years saved (LYS) for STN1013001 vs latanoprost from the German health system perspective. The model included seven mutually exclusive health states and adopted a 1-year cycle length. The model was populated with pooled data derived, by means of a questionnaire, from a convenience sample of five German glaucoma specialists. Remaining data were derived from published sources. Data provided by the ophthalmologists included annual treatment adherence probabilities, utility values and resource utilization. The half-cycle correction as well as a discount rate of 3.0% per year were applied to costs (expressed in € 2020), life-year saved (LYS) and QALYs. The incremental cost–utility ratio (ICUR) was contrasted against the informal willingness-to-pay (WTP) threshold for incremental LYS saved or QALY gained (€ 30,000) proposed for Germany. One-way and probabilistic sensitivity analyses (OWSA; PSA) tested the robustness of the base case ICUR.Results: Over the 5-year time horizon, STN1013001 strongly dominates latanoprost as it is less costly (€ 1003.65 vs € 1145.37; − 12.37%) and produces more QALYs (2.612 vs 2.365; +10.44%) per notional patient. Baseline findings were robust against all the variations included in OWSA. PSA shows that STN1013001 has a 100% probability of being cost-effective vs Latanoprost at each WTP threshold for incremental QALY gained.Conclusion: Once on the market, STN1013001 will provide a cost-effective and possibly strongly dominant therapy vs latanoprost for OAG/OHT+OSD patients from a German health system perspective. Future empirical research should confirm these findings.Keywords: open-angle glaucoma, ocular surface disease, STN1013001, latanoprost, cost–utility analysis, Germany

Salvatore Russo,1,&ast; Stefano Landi,2,&ast; Stephane Courric3 1Department of Management, University of Venice, Venezia, Italy; 2Department of Business Administration and Management, University of Verona, Verona, Italy; 3Hays Pharma, Paris, France&ast;These authors contributed equally to this workCorrespondence: Stefano LandiDepartment of Business Administration and Management, University of Verona, Via Cantarane, 24, Verona, 37129, ItalyTel +39 3463287572Email stefano.landi@univr.itIntroduction: Diabetic chronic foot ulcers (DFU) lead to pain, reduced quality of life and represent a severe economic burden for patients and health systems. The clinical results of PRP effectiveness in the treatment of DFU are promising; on the other hand, the costs associated with treating DFUs with PRP are higher than those using standard therapy. Therefore, this study aims to determine the cost-effectiveness of platelet-rich plasma (PRP) therapy compared to standard therapy from the French healthcare system perspective.Methods: A cost-effectiveness analysis (CEA) was performed using a decision Markov model with a cohort of patients with chronic DFU (duration of > 3 weeks) with high orthopaedic risk and with ulcers graded 3A according to University of Texas classification. The effectiveness outcomes are reported in terms of quality adjusted life year (QALY). The costs are reported in euro (€) currency evaluated in 2019. A micro-costing approach alongside a clinical study was used to assess resource use. Deterministic sensibility analyses are reported to evaluate the robustness of the results. The analyses were carried out in the French setting.Results: The incremental cost-effectiveness ratio (ICER) of PRP treatment is –€ 613/ QALY, which, being lower than zero, indicates the dominance of the PRP therapy. Deterministic and probabilistic sensitivity analysis underlines the main parameter affecting CE results. Lowest number of standard of care weekly medications (from 5 to 3) leads to a € 622/QALY while increasing PRP weekly medication (from 1 to 4) has an ICER of € 732/QALY.Discussion: PRP is a cost-effective or even a cost-saving alternative in the French setting. PRP has higher cost for the complete medication, but, in the absence of wound complications, has the potential to involve lower consumption of resources in the form of routine medication over a 1-year time horizon.Keywords: platelet-rich plasma, cost-effectiveness analysis, cost-utility analysis, diabetic foot ulcer

Abstract Background Only two-thirds of patients admitted to psychiatric wards return to their previous jobs. Return-to-work interventions in Germany are investigated for their effectiveness, but information regarding cost-effectiveness is lacking. This study investigates the cost-utility of a return-to-work intervention for patients with mental disorders compared to treatment as usual (TAU). Methods We used data from a cluster-randomised controlled trial including 166 patients from 28 inpatient psychiatric wards providing data at 6- and 12-month follow-ups. Health and social care service use was measured with the Client Sociodemographic and Service Receipt Inventory. Quality of life was measured with the EQ-5D-3L questionnaire. Cost-utility analysis was performed by calculating additional costs per one additional QALY (Quality-Adjusted Life Years) gained by receiving the support of return-to-work experts, in comparison to TAU. Results No significant cost or QALY difference between the intervention and control groups has been detected. The return-to-work intervention cannot be identified as cost-effective in comparison to TAU. Conclusions The employment of return-to-work experts could not reach the threshold of providing good value for money. TAU, therefore, seems to be sufficient support for the target group.

Background: Biologics are a good therapeutic option for severe, chronic plaque psoriasis; however, they come with significant cost to the health care system. Objective: To conduct a cost-utility analysis of outpatient biologics (adalimumab, etanercept, guselkumab, ixekizumab, risankizumab, secukinumab, tildrakizumab, and ustekinumab) available to adults with severe, chronic plaque psoriasis from the perspective of the Australian health care system. Methods: A Markov cohort model was constructed to estimate the quality-adjusted life years (QALYs) and costs accrued for treatment pathways commencing with different first-line biologics, over a 96-week time horizon. The model adhered to the Australian Pharmaceutical Benefits Scheme eligibility criteria and guidelines. Results: A biologic treatment pathway commencing on tildrakizumab was the most cost-effective first-line treatment (Australian dollar 39,930; total utility of 1.57 QALYs over 96 weeks). First-line secukinumab and risankizumab had incremental cost-utility ratios of Australian dollar 194,524/QALY and Australian dollar 479,834/QALY, respectively, when compared with first-line tildrakizumab. Limitations: The efficacy and utility input parameters were derived from international randomized control trials and patients from the United Kingdom, respectively. Findings from this study cannot be generalized beyond Australia. Conclusion: Tildrakizumab may be considered as first-line treatment for adult patients with severe, chronic plaque psoriasis embarking on biologic therapy, from the economic perspective of the Australian health care system.

ObjectiveTo evaluate the economics of sugemalimab plus chemotherapy in the first-line treatment of metastatic non-small cell lung cancer, and to provide a reference for the formulation of relevant medical insurance policies and rational drug use.MethodsFrom the perspective of the Chinese health system, a three-state partitioned survival model was constructed based on data from a phase III randomized clinical trial (GEMSTONE 302) to evaluate the cost-utility of sugemalimab plus chemotherapy compared with chemotherapy in first-line treatment of metastatic non-small cell lung cancer. Model results were expressed as total cost, life years, quality-adjusted life years, and incremental cost-effectiveness ratio. The robustness of the underlying analysis results was verified using one-way sensitivity analysis and probabilistic sensitivity analysis.ResultsThe results of the base-case analysis showed that sugemalimab plus chemotherapy yielded 1.63 QALYs at a total cost of 130,667.70 USD, chemotherapy yielded 1.04 QALYs at a total cost of 64,001.02 USD, and the ICER was 113,155.52 USD/QALY, which was well above the current willingness-to-pay threshold in China (3 times 2021 per capita GDP) (36,203.88 USD).ConclusionThis study suggests that sugemalimab in combination with a chemotherapy regimen is more effective but not economical for patients with metastatic non-small cell lung cancer receiving first-line therapy in China and that a reasonable reduction in drug prices could improve the probability of it being economical.

Introduction: IDegLira (brand name Xultophy) is a novel fixed ratio combination of insulin degludec and liraglutide for type 2 diabetes (T2D) patients. This study aimed to investigate the lifetime cost-effective value of IDegLira compared with its single component (Degludec or Liraglutide) and to explore the suitable annual cost of IDegLira if necessary. Methods: UKPDS OM2 was applied to determine the long-term qualityadjusted life years (QALYs) and total costs. The efficacy data that were inputted into the model were synthesized from 6 randomized clinical trials (RCTs) that directly assessed the clinical benefit of IDegLira and its components in the treatment of uncontrolled T2D patients. The economic results were examined by one-way sensitivity analysis (OSA) and probabilistic sensitivity analysis (PSA). Further price reduction of IDegLira was investigated by binary search. Results: The IDegLira, IDeg, and Lira yielded 11.79 QALYs, 11.62 QALYs, and 11.73 QALYs and total cost of $20281.61, $3726.76, and $11941.26, respectively. The incremental cost-utility ratio (ICUR) of IDegLira versus IDeg was $99464.12/QALYs, and the ICUR of IDegLira versus Lira was $143348.26/QALYs, which indicated that IDegLira was not a cost-effective therapy for T2D patients compared with its components at the current price from a Chinese national healthcare system perspective. Base case results were robust to OSA and PSA. A further binary search showed that IDegLira appears to only be cost-effective if the annual cost of IDegLira is decreased by 58% when IDeg is considered as a reference, or by 30.57% when Lira is considered as a reference. Conclusion: In conclusion, IDegLira appears to not be cost-effective when compared with the current prices of IDeg or Lira for T2D patients in China. However, after the binary search, IDegLira appears to only be cost-effective if the annual cost of IDegLira is decreased 58% when IDeg is considered as a reference, or by 30.57% when Lira is considered as a reference. [ABSTRACT FROM AUTHOR]

Diabetes mellitus (DM), as one of the most common metabolic diseases, is the ninth leading cause of death globally and imposes heavy costs on the health systems including both costs of treatment and management of secondary complications. This study intended to investigate the cost-effectiveness of dulaglutide compared with liraglutide in the management of patients with type 2 DM in Iran. We conducted a cost-utility analysis using a 5-state Markov model from the health system perspective, over a 10-year time horizon, in 2018 in Iran. Sensitivity of the model has been evaluated through tornado diagram and using one-way sensitivity analysis. In addition, probabilistic sensitivity analysis has been accomplished using Monte Carlo simulation. The average costs of treatment of patients with type 2 DM using the dulaglutide and liraglutide treatment regimens are 17 577.09 and 18 517.54 US dollars per patient, respectively, over a 10-year time horizon. In terms of effectiveness, the average discounted quality-adjusted life-year rates are estimated at 5.560 and 5.403 for the dulaglutide and liraglutide treatment regimens, respectively. The model is mostly sensitive to the price of dulaglutide and liraglutide, the hemoglobin A1c reduction of liraglutide, and the utility resulting from less injection frequency of dulaglutide, respectively. Dulaglutide, in addition to being more effective, providing 0.156 more quality-adjusted life-years for the patients, reduces costs by 940.45 US dollars per patient over a 10-year time horizon. Therefore, due to the greater effectiveness and lower cost, it is concludable that dulaglutide is the cost-effective (incremental cost-effectiveness ratio = −6028.52) treatment alternative from the health system perspective. • Dulaglutide was associated with both less cost and more efficacy; therefore, by proposing a negative incremental cost-effectiveness ratio, it is selected as the cost-effective treatment strategy from the health system perspective in comparison with liraglutide in Iran's healthcare setting. • Although this result is generally similar to other performed studies in this regard in different countries, the results are different when the comparison arm changes. • Sensitivity analysis shows that the result is most sensitive to the prices of dulaglutide and liraglutide. [ABSTRACT FROM AUTHOR]

Objective: To review quality of life (QOL) instruments for chronic limb-threatening ischemia (CLTI) patients and informal carers, and their use in QOL and cost-utility analysis (CUA) studies. Background: CLTI is a global health problem with significant morbidity affecting patients and informal carers. QOL is increasingly measured for holistic outcomes assessment and CUA. However, measurement instruments in CLTI are poorly understood. Methods: MEDLINE, EMBASE, PsycINFO, CINAHL, COSMIN, PROQOLID, CEA registry, and NHS EED databases were searched for all English language studies up to May 2021. Features of instruments, evidence of measurement property appraisal, and trends in use were assessed. Prospective protocol registration (Open Science Framework: https://doi.org/10.17605/OSF.IO/KNG9U). Results: A total of 146 studies on QOL instruments (n=43), QOL outcomes (n=97), and CUA (n=9) were included. Four disease-specific QOL instruments are available for lower extremity arterial disease (intermittent claudication or CLTI). VascuQoL-25 and VascuQoL-6 have been used in CLTI. There is no CLTI-specific instrument. Of 14 generic instruments, SF-36, EQ-5D-3L, NHP, and WHOQOL-BREF were most common. Studies reporting partial measurement property appraisal favored VascuQoL-25, VascuQoL-6, and SF-36. Feasibility considerations include mode of administration and responder burden. None of 4 available carer-specific instruments have been used in CLTI. Since 1992, the number of QOL studies has increased considerably, but CUA studies are scarce. Informal carers have not been assessed. Conclusions: This review provides a comprehensive reference for QOL measurement in CLTI that helps end-users with instrument selection, use, and interpretation. However, a CLTI-specific instrument is needed. There is an opportunity to benefit society through future CUA studies and evaluation of QOL in informal carers. [ABSTRACT FROM AUTHOR]

Objective: EMPEROR-Reduced and EMPEROR-Preserved studies showed the benefits of empagliflozin along with a reduction in cardiovascular death or hospitalisation for heart failure (HF). Our aim was to evaluate the economics and effectiveness of adding empagliflozin to the standard therapy for HF with reduced ejection fraction (HFrEF) and HF preserved ejection fraction (HFpEF) in China. Methods: A multistate Markov model was constructed to yield the clinical and economic outcomes of adding empagliflozin to the standard therapy for 65-year-old patients with HFrEF and HFpEF. A cost-utility analysis was conducted, mostly derived from the EMPEROR-Reduced study, EMPEROR-Preserved study, and national statistical database. All costs and outcomes were discounted at the rate of 5% per annum. The primary outcomes were total and incremental costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER). Sensitivity analyses were also performed. Results: In the HFrEF population, the 10-year incremental cost was $827.52 and the 10-year incremental QALY was 0.15 QALYs, resulting in an ICER of $5,612.06/QALY, which was below the WTP of $12,652.5/QALY. In the HFpEF population, compared with the control group, the incremental cost was $1,271.27, and the incremental QALY was 0.11 QALYs, yielding an ICER of 11,312.65 $/QALY, which was also below the WTP of $12,652.5/QALY. In the HFrEF and HFpEF populations, the results of a one-way sensitivity analysis showed that the risk of cardiovascular death in both groups was the most influential parameter. In the HFrEF population, a probability sensitivity analysis (PSA) revealed that when the WTP thresholds were $12,652.5/QALY and $37,957.5/QALY, the probabilities of being cost-effective with empagliflozin as an add-on were 59.4% and 72.6%, respectively. In the HFpEF population, the PSA results revealed that the probabilities of being cost-effective with empagliflozin as an add-on were 53.1% and 72.2%, respectively. Conclusion: Considering that the WTP threshold was $12,652.5/QALY, adding empagliflozin to standard therapy was proven to be a slightly more cost-effective option for the treatment of HFrEF and HFpEF from a Chinese healthcare system perspective, which promoted the rational use of empagliflozin for HF. [ABSTRACT FROM AUTHOR]

Treatment for acute myeloid leukemia (AML) typically involves intensive chemotherapy (IC); however, there is an unmet need for approximately 50% of AML patients who are deemed unfit or ineligible for IC. The purpose of this study was to evaluate, from a Canadian perspective, the economic impact of venetoclax in combination with azacitidine (Ven+Aza) for the treatment of patients with newly diagnosed AML who are 75 years or older or who have comorbidities that preclude using IC. A lifetime partitioned survival model was developed to assess the cost-effectiveness of Ven+Aza compared with Aza. Health states included event-free survival, progressive/relapsed disease, and death. Efficacy parameters were based on the VIALE-A trial. Analyses were conducted from Ministry of Health (MoH) and societal perspectives. Over a lifetime horizon, Ven+Aza was associated with a gain of 1.65 quality-adjusted life years (QALYs) compared with Aza. From an MoH perspective, Ven+Aza and Aza were associated with total costs of $204,305 and $82,333, respectively, resulting in an incremental cost–utility ratio of $73,841/QALY. Results were similar from a societal perspective. This economic evaluation demonstrates that, in comparison with Aza, Ven+Aza is a cost-effective strategy for the treatment of patients with newly diagnosed AML who are deemed unfit for IC. [ABSTRACT FROM AUTHOR]

Endovascular coiling (EC) has been identified in systematic reviews and meta-analyses to produce more favourable clinical outcomes in comparison to neurosurgical clipping (NC) when surgically treating a subarachnoid haemorrhage from a ruptured aneurysm. Cost-effectiveness analyses between both interventions have been done, but no cost-utility analysis has yet been published. This systematic review aims to perform an economic analysis of the relative utility outcomes and costs from both treatments in the UK. A cost-utility analysis was performed from the perspective of the National Health Service (NHS), over a 1-year analytic horizon. Outcomes were obtained from the randomised International Subarachnoid Aneurysm Trial (ISAT) and measured in terms of the patient's modified Rankin scale (mRS) grade, a 6-point disability scale that aims to quantify a patient's functional outcome following a stroke. The mRS score was weighted against the Euro-QoL 5-dimension (EQ-5D), with each state assigned a weighted utility value which was then converted into quality-adjusted life years (QALYs). A sensitivity analysis using different utility dimensions was performed to identify any variation in incremental cost-effectiveness ratio (ICER) if different input variables were used. Costs were measured in pounds sterling (£) and discounted by 3.5% to 2020/2021 prices. The cost-utility analysis showed an ICER of − £144,004 incurred for every QALY gained when EC was utilised over NC. At NICE's upper willingness-to-pay (WTP) threshold of £30,000, EC offered a monetary net benefit (MNB) of £7934.63 and health net benefit (HNB) of 0.264 higher than NC. At NICE's lower WTP threshold of £20,000, EC offered an MNB of £7478.63 and HNB of 0.374 higher than NC. EC was found to be more 'cost-effective' than NC, with an ICER in the bottom right quadrant of the cost-effectiveness plane—indicating that it offers greater benefits at lower costs. This is supported by the ICER being below the NICE's threshold of £20,000–£30,000 per QALY, and both MNB and HNB having positive values (> 0). [ABSTRACT FROM AUTHOR]

Objective: The objective of this study is to evaluate the cost-effectiveness of different knee OA care sequences compared to standard treatment reimbursed by the major health insurance payer in Thailand. Method: We used decision analytical modeling to evaluate the effect of either adding etoricoxib or crystalline glucosamine sulfate compared to standard treatment from a societal perspective over patients' lifetimes. Data were analyzed based on efficacy, whereas adverse events were considered as a substate. Model input data were retrieved from relevant published literature and the Standard Cost Lists for Health Technology Assessment, Thailand. All health outcomes were measured in a unit of quality-adjusted life-year (QALY). An incremental cost-effectiveness ratio (ICER) was applied to examine the costs and QALYs. Sensitivity analysis was performed to investigate the robustness of the model. Result: The results demonstrated that adding crystalline glucosamine sulfate (before diclofenac plus proton pump inhibitors, PPI) into the standard care sequence was a dominant strategy compared to the standard care sequence. Adding etoricoxib alone or including crystalline glucosamine sulfate (after diclofenac plus PPI) was dominated by adding crystalline glucosamine sulfate (before diclofenac plus PPI), whereas in a willingness-to-pay (WTP) threshold in Thailand, adding of both crystalline glucosamine sulfate (before diclofenac plus PPI) and etoricoxib were cost-effective when compared to adding crystalline glucosamine sulfate alone with ICER of 125,547 Thai baht/ QALY (3,472 US dollars/QALY). Conclusion: The addition of crystalline glucosamine sulfate and etoricoxib into standard knee OA treatment were cost-effective at the WTP threshold in Thailand. In addition, early initiation of crystalline glucosamine sulfate would be less costly andmore effective than delayed treatment or the use of standard treatment alone. [ABSTRACT FROM AUTHOR]