Probabilistic Sensitivity Analysis Research Articles

Economic Evaluation of Transitioning to the 20-Valent Pneumococcal Conjugate Vaccine in the Dutch Paediatric National Immunisation Programme.

In 2024, the vaccination strategy against pneumococcal disease in the Dutch paediatric population was changed from the 10-valent pneumococcal conjugate vaccine (PCV) (PCV10) to a 15-valent PCV (PCV15) under a 2 + 1 schedule. We aimed to assess whether switching from PCV15 under a 2 + 1 schedule to 20-valent PCV (PCV20) under a 3 + 1 schedule in the Dutch paediatric national immunisation programme (NIP) would yield economic savings and health benefits. A multiple-cohortpopulation model with an annual cycle and 10-year time horizon was adapted for the Dutch populationfrom a societal perspective. Discounting was set at 3.0% and 1.5% for costs and benefits, respectively. Medical and societal costs were calculated, along with cases of invasive and non-invasive pneumococcal disease, and quality-adjusted life years (QALY) for PCV15 and PCV20,from which, the incremental cost-effectiveness ratio (ICER) per QALY was calculated for PCV20 versus PCV15 to determine the economic benefits of PCV20. The model assumptions were tested in probabilistic and deterministic sensitivity analyses, as well as a series of scenario analyses. Both medical and societal cost of disease were substantially lower with PCV20 versus PCV15 (incremental cost-savings of €130,113,010 and €61,593,168, respectively), with total incremental cost-savings of €29,365,696 whenconsidering the cost of the vaccination programme. With an overall QALY gain of 33,232, the ICER per QALY placed PCV20 as the dominant strategy, as it was both more effective and less costly than PCV15. PCV20 was estimated to result in 57,657 fewer pneumococcal disease cases across invasive and non-invasive disease and 1561 fewer disease-related deaths. The sensitivity and scenario analyses demonstrated the robustness of the model results. This cost-effectiveness analysis demonstrated that switching from PCV15 2 + 1 to PCV20 3 + 1 in the Dutch paediatric NIP would reduce both the clinical burden and projected costs of pneumococcal disease over 10years.

Burden of HPV-induced diseases and cost effectiveness of catch-up vaccination in Czech Republic: a model-based study

ObjectivesHuman papillomavirus (HPV) infections are highly prevalent sexually transmitted infections, notably associated with various cancers. This study analyses the health and economic impacts of HPV-associated diseases in the Czech Republic and evaluates the cost-effectiveness of a catch-up vaccination program.MethodsUtilizing a Markov multistate model, the study assesses the lifetime impacts and costs related to HPV infections. Cohorts of ages 15–21 were simulated to assess the impact of catch-up vaccination outside the 11-year-old age group.ResultsThe total quality-adjusted life years (QALYs) for the female and male cohorts (together 119,362 individuals) were higher in the vaccination scenario compared to the non-vaccination scenario. The increase in QALYs was 122,246 and 200,852 respectively, when considering the actual vaccination rates. Across both cohorts, 329 cancer-related deaths were prevented. In the probabilistic sensitivity analysis for the female population, vaccination was the dominant strategy in 99.3% of iterations. In the male population, vaccination was the dominant strategy in 80.3% of iterations. The implementation of catch-up vaccination for the 15–21 age group significantly increased QALY gains and reduced life-years-lost (LYLs). In the female cohort, all analysed rates of catch-up vaccination were the dominant strategy, while in the male cohort, the incremental cost-effectiveness ratios (ICERs) remained consistently below 42,000 CZK/QALY.ConclusionsThe catch-up vaccination program for 15-21-year-olds is cost-effective and can prevent a significant number of HPV-related cancers in both men and women.

Cost-effectiveness of silver diamine fluoride depends on caries activity: A decision analytic model.

The study sought to assess the cost-effectiveness of silver diamine fluoride (SDF) relative to sodium fluoride varnish (NaF), and direct restorations placed chairside or under general anaesthesia (GA), accounting for caries activity. An analytical decision tree model was developed with clinical data from multiple centres to evaluate the four treatment options among all children aged 1 to 6 years in Singapore (N=231,880) over a 12-month time horizon. Base-case scenarios consisted of children with low- (1 carious tooth) and high- (7 carious teeth) caries activity. Incremental cost-effectiveness ratios (ICER) and net monetary benefits (NMB) were tabulated with the outcomes set to be the control of active caries and avoidance of tooth extractions. Deterministic and probabilistic sensitivity analyses were conducted. Overall, SDF was less effective than direct restorations placed chairside but was less costly. For children with low-caries activity, the ICER for SDF was $9 per caries-controlled tooth, and $68 per extraction avoided. For children with high-caries activity, the ICERs were $267 and $1 909 respectively. At a willingness-to-pay threshold of $30, the NMB was negative (favours restorations) for caries control in children with low-caries activity and positive (favours SDF) for all other situations. In the low-caries activity group, SDF had a 41.5% probability of being cost-effective for caries control and 49.2% probability of being cost-effective for the avoidance of extraction. The corresponding probabilities for the high-caries activity group were 99.8% and 100%. The NMB of SDF increases proportionally with caries activity, suggesting utilisation of SDF should depend on caries activity. Results were most sensitive to changes in relative cost, and effectiveness of SDF/direct restorations. SDF was cost-effective in most situations except for caries control in children with low-caries activity. Direct restorations were more effective and should be considered in low-caries activity children.

Cost-effectiveness of nivolumab plus chemotherapy for advanced gastric/gastroesophageal junction/esophageal adenocarcinoma in the United States

Nivolumab, in combination with chemotherapy, significantly improves survival in patients with gastric/gastroesophageal junction (G/GEJ)/esophageal adenocarcinoma. The study evaluated the cost-effectiveness of nivolumab plus chemotherapy for the treatment of G/GEJ/esophageal adenocarcinoma. A Markov model was developed from the perspective of United States healthcare payers. We estimated costs and summarized effectiveness in terms of quality-adjusted life-years (QALYs). One-way and probabilistic sensitivity analyses were conducted to assess the impact of uncertainties on the cost-effectiveness results. The incremental cost-effectiveness ratio for nivolumab plus chemotherapy ($149,636.97, 1.24 QALYs) compared to chemotherapy alone ($13,941.06, 0.75 QALYs) is $135,695.91, with a difference of 0.49 QALYs. Evidence suggests that nivolumab plus chemotherapy for the first-line treatment of locally advanced or metastatic G/GEJ/esophageal adenocarcinoma may not represent a cost-effective option within the United States healthcare system.

Dapagliflozin for the treatment of heart failure with reduced ejection fraction in Brazil: a cost-effectiveness analysis.

Heart failure, a complex clinical syndrome with high morbidity and mortality, has become a significant burden on public health. Recently, a new class of antidiabetic agents-the sodium-glucose cotransporter 2 (SGLT2) inhibitors-was associated with a significant reduction on mortality and hospitalization in HF with reduced ejection fraction (HFrEF) when added to standard pharmacological treatment. Considering the lack of data on its cost-effectiveness, the present study aims to estimate the incremental cost-effectiveness ratio of add-on dapagliflozin treatment for HFrEF from the Brazilian public healthcare system perspective. We built a Markov model to estimate the clinical outcomes and costs of 1,000 hypothetical subjects with established HFrEF in a lifetime horizon. The model inputs were based on the Dapagliflozin and Prevention of Adverse Outcomes in Heart Failure (DAPA-HF) trial and local data. The main outcome was the incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) gained. Deterministic and probabilistic sensitivity analyses, as well as scenario analyses, were performed. The addition of dapagliflozin to standard care treatment in 1,000 HFrEF patients yielded an expected value of 366.99 additional QALYs at an incremental cost of US$ 1,517,878.49, resulting in an ICER of US$ 4,136.08 per QALY gained, being a cost-effective strategy considering the Brazilian official cost-effectiveness threshold (US$ 8,000/QALY). In probabilistic sensitivity analyses, 96.60% of the simulations were also cost-effective. In the scenario analyses, results were similar for individuals with and without diabetes. Dapagliflozin is likely to be cost-effective when added to standard HFrEF therapy in Brazil. This study was supported by the National Institute of Science and Technology for Health Technology Assessment (Instituto de Avaliação de Tecnologias em Saúde-IATS).

Cost-effectiveness of NALIRIFOX compared to other first-line treatments for metastatic pancreatic cancer.

733 Background: NALIRIFOX was recently approved for first-line treatment of metastatic pancreatic cancer, where combination chemotherapy remains the main treatment modality. We conducted a cost-effectiveness analysis of NALIRIFOX compared to both FOLFIRINOX and gemcitabine/protein-bound paclitaxel (GP). Methods: A partitioned survival model with three health states (progression-free, post-progression and death) was constructed to model the disease course. Using published data from NAPOLI-3, parametric survival curves were generated for NALIRIFOX, from which survival curves for FOLFIRINOX and GP were extrapolated using pooled efficacy data reported by a network meta-analysis of clinical trials. Model inputs were retrieved from the literature or other publicly available resources. Specifically, drug acquisition costs were from average wholesale prices in the Red Book while administration, disease monitoring and supportive care costs were from the CMS Physician Fee Schedule. Utility values to estimate quality-adjusted life years (QALY) were from the CALGB80303 trial. Adverse effects that were considered included anemia, neutropenia, febrile neutropenia, thrombocytopenia, diarrhea, peripheral neuropathy, nausea and vomiting, and fatigue. Adverse effect rates were based on data pooled from clinical trials while costs and disutility were obtained from a secondary analysis of claims data and a clinician survey, respectively. The time horizon was 5 years and utilized a third-party payer perspective. Both costs ($USD 2024) and outcomes were discounted at 3% annually. A probabilistic sensitivity analysis was conducted by varying model parameters based on 95% confidence intervals if known or 10% in both directions otherwise. Results: The overall cost associated with NALIRIFOX was $129,314, followed by GP ($104,593) and FOLFIRINOX ($41,528). Among the 3 regimens, NALIRIFOX incurred the highest treatment-related cost ($105,533) but the lowest adverse effects management cost ($16,541). On average, patients on NALIRIFOX gained a total of 1.14 life years (LY) or after adjusting for utility, 0.86 QALY. FOLFIRINOX led to 1.09 LY or 0.82 QALY while GP resulted in 0.99 LY or 0.75 QALY. Due to its higher cost and lower survival benefit, GP was dominated by FOLFIRINOX. Compared to FOLFIRINOX, the incremental cost-effectiveness ratio (ICER) of NALIRIFOX was $1,952,062/QALY. In our sensitivity analysis, the probability of NALIRIFOX being cost-effective was 0% at all conventional ICER thresholds up to $200,000/QALY. Conclusions: Due to the high drug acquisition cost, the ICER of NALIRIFOX far exceeds currently acceptable thresholds for economic value and NALIRIFOX is considered not cost-effective. This is despite that in our model among first line treatments for metastatic pancreatic cancer, NALIRIFOX was associated with reduced costs for managing adverse effects.

Cost-effectiveness of cemiplimab plus chemotherapy vs pembrolizumab plus chemotherapy as first-line treatment for advanced non-small cell lung cancer.

In 2022, the US Food and Drug Administration approved cemiplimab in combination with chemotherapy (CCT) as a first-line treatment for advanced non-small cell lung cancer (aNSCLC). However, whether CCT presents a cost-effective alternative to the previously preferred first-line treatment, pembrolizumab plus chemotherapy (PCT), remains uncertain. To evaluate the cost-effectiveness of CCT vs PCT as the first-line treatment for aNSCLC from a US health care payer perspective. A 3-state partitioned survival model with a 10-year horizon was constructed. Clinical data were derived from the EMPOWER-Lung 3, KEYNOTE-407, and KEYNOTE-189 trials. Costs and quality of life were obtained from published 2024 US list prices and literature. The cost, quality-adjusted life-years (QALYs) gained, and incremental cost-effectiveness ratio (ICER) were calculated. All outcomes were discounted at a rate of 3% per year. Scenario analyses, deterministic and probabilistic sensitivity analyses, and subgroup analyses were performed for patients with different programmed death ligand 1 (PD-L1) levels. In the base-case analysis, the total cost of PCT was $207,926 with 1.609 QALYs, whereas CCT had a total cost of $175,247 with 1.657 QALYs. Results from the scenario analyses were consistent with the base-case analysis, indicating that CCT was a dominant treatment strategy over PCT (ICER =-$675,304 per QALY). The cost of pembrolizumab highly impacted the ICER. At a willingness-to-pay threshold of $150,000 per QALY, CCT would be accepted as a cost-effective option 96.9% of the time. In subgroup analyses, CCT remained a dominant alternative to PCT for patients with PD-L1 levels of at least 50% and 1%-49%. This cost-effectiveness analysis suggests that CCT is a dominant first-line treatment option for aNSCLC with PD-L1 levels of at least 1% compared with PCT.

24-week, all-oral regimens for pulmonary rifampicin-resistant tuberculosis in TB-PRACTECAL trial sites: an economic evaluation.

New 6-month rifampicin-resistant tuberculosis treatment regimens containing bedaquiline, pretomanid, and linezolid (BPaL) with or without moxifloxacin or clofazimine, could improve treatment efficacy, safety, and tolerability, and free up resources within the health system. Following a change to WHO rifampicin-resistant tuberculosis treatment guidelines, countries are facing difficult decisions about when and how to incorporate new drug regimens into national guidelines. We aimed to assess the probability of BPaL-based regimens being cost-saving using data collected in the TB-PRACTECAL trial. This economic evaluation using a cost-utility analysis was embedded in five TB-PRACTECAL trial sites in Belarus, Uzbekistan, and South Africa. Between Nov 19, 2020, and Sept 27, 2022, we collected detailed primary unit cost data in six hospitals and four ambulatory health facilities and collected data on patient-incurred costs from 73 trial participants. The primary efficacy endpoint of the main trial, a composite of unfavourable outcomes (death, disease recurrence, treatment failure, early discontinuation of therapy, withdrawal, or loss to follow-up) and clinically important safety outcomes by 72 weeks of follow-up were incorporated into the analysis. Societal perspective cost data and effect outcome data were input into a Markov model to estimate the cost per disability-adjusted life-year (DALY) averted by BPaL-based regimens compared with the standard of care over a 20-year time horizon. We conducted a range of univariate and probabilistic sensitivity analyses to test our findings. BPaL-based regimens averted a mean of 1·28 DALYs and saved a mean of US$14 868 (SD 291) per person from the provider perspective compared with standard-of-care regimens over 20 years. Patient-incurred costs were reduced by a mean of $172 (SD 0·84) in BPaL-based regimen groups compared with standard of care. The main cost drivers for both providers and patients were inpatient bed-days; the duration of the inpatient period varied across countries. Varying a range of model parameters affected the degree of cost savings but did not change the finding that BPaL-based regimens are cost-saving compared with standard of care. This trial-based evidence adds to consistent indications from modelling studies that BPaL-based regimens are cost-saving for both the patient and health system. Urgent implementation of BPaL-based regimens in countries with a high burden of tuberculosis could improve treatment of rifampicin-resistant tuberculosis, reduce pill burden, and free up desperately needed resources within the health system. Médecins Sans Frontières.

Cost-utility analysis of ropeginterferon alfa-2b to manage low-risk patients with polycythemia vera as compared to phlebotomy only in the Austrian healthcare system.

Treatment of polycythemia vera (PV) aims to maintain hematocrit on target to reduce risk of thrombotic complications, while preventing disease progression to myelofibrosis (MF) and acute myeloid leukemia (AML). This analysis evaluated cost-effectiveness of adding ropeginterferon alfa-2b (ropegIFNα) to phlebotomy in patients with low-risk PV (those younger than 60 years without prior thrombosis), compared to phlebotomy alone. We combined a 12-month decision tree with a semi-Markov cohort model comparing ropegIFNα to the standard treatment from the Austrian healthcare system perspective over 30 years. Outcomes were quality adjusted life years (QALYs), costs, and incremental cost-utility ratio (ICUR). Model inputs were obtained from the phase 2 Low-PV study, additional published literature and from Austrian-specific cost databases. One-way and probabilistic sensitivity analyses (SA) assessed the robustness of findings. RopegIFNα led to 1,43 higher QALYs and 50.960 EUR overall higher costs compared to phlebotomy alone, with an ICUR of 35.525 EUR/QALY. Thrombosis, MF, and AML costs decreased for the ropegIFNα group by 12%, 30% and 16% respectively, due to the delayed complications onset and disease progression. In the one-way SA, ropegIFNα costs and discount rates had the greatest impact on results. The probabilistic SA showed a 100% probability of cost-effectiveness at willingness-to-pay threshold aligned to the Austrian GDP per capita. RopegIFNα is a cost-effective treatment option for patients with low-risk PV. These findings suggest that early treatment with ropegIFNα could ensure optimal resource allocation by preventing costly thrombotic events and progression to MF whilst increasing patient quality of life.

P-28. A Clinical and Economic Comparison of Non-Egg Influenza Vaccines in Adults 18-64 Years in the U.S

Abstract Background Influenza vaccination with non-egg-based vaccines had improved the antibody response to circulating flu viruses over that of traditional egg-based vaccines, which are the most common flu vaccines worldwide. Two non-egg-based flu vaccines— the cell-based flu vaccine (QIVc) and the recombinant flu vaccine (QIVr) —were introduced in the United States during the 2013-14 flu season. The objective of this analysis was to determine the cost-effectiveness of QIVc compared to QIVr administered to people between 18-64 years of age from U.S. commercial and societal perspectives.Table 1.Summary of Health and Economic Outcomes from the Commercial Perspective (Season 2018-19 – Vaccine Effectiveness) Methods A compartmental dynamic transmission model, calibrated to match infection data from the U.S., was used to estimate the clinical and economic impact of vaccination over one influenza season. Epidemiological data and vaccination coverage rate were obtained from CDC official databases. Vaccine effectiveness data against outpatient visits and hospitalizations, were obtained from published U.S. observational studies including outpatient and hospital setting outcomes (2018/19 and 2019/20 seasons). Associated direct medical costs, health state utilities and disutilities, and productivity loss costs were obtained from recent economic literature. Deterministic and probabilistic sensitivity analysis were conducted. Results From the U.S. commercial perspective of an equal number of vaccinated adults in both groups, QIVc compared to QIVr showed fewer outpatient visits (585,936) and emergency department visits (12,492), and a comparable number of hospitalizations (503) (see Table). QIVc compared to QIVr resulted in a higher overall number of quality-adjusted life years (QALYs) gained (2,546) and a reduction in total associated costs by US$2.9B. Cost savings were driven mainly by the lower 2023/24 acquisition costs, for QIVc vs QIVr (≈-50%). All sensitivity analyses produced similar results, confirming the robustness of the model. Results were consistent from the societal perspective. Conclusion The cell-based vaccine was cost saving compared to the recombinant vaccine for subjects 18-64 years in the U.S. and achieved comparable health outcomes results at a significant reduction in cost. Disclosures Myron J. Levin, MD, CSL Seqirus USA: Advisor/Consultant|Curevo: Advisory Board|GSK: Grant/Research Support|GSK: Advisory Board|Moderna: Advisory Board|Pfizer: Advisory Board Neda Al Rawashdh, n/a, CSL Seqirus USA: Advisor/Consultant Liliane Mofor, n/a, CSL Seqirus USA: Stocks/Bonds (Private Company) Pablo Anaya, n/a, CSL Seqirus USA: Advisor/Consultant Richard M. Zur, n/a, CSL Seqirus USA: Advisor/Consultant Emily B. Kahn, n/a, CSL Seqirus USA: Advisor/Consultant Daniel Yu, n/a, CSL Seqirus Australia: Stocks/Bonds (Private Company) Joaquin F. Mould-Quevedo, PhD, CSL Seqirus USA: Stocks/Bonds (Private Company)

Cost-effectiveness analysis for implementation of smoking cessation strategies at primary health care settings in Tamil Nadu.

Smoking is a major public health concern in Tamil Nadu, as it is in many parts of the world. It is a leading cause of preventable diseases and deaths, with a significant economic burden on healthcare systems and society as a whole. Recognizing the need to address this issue, the implementation of smoking cessation strategies at primary health care (PHC) settings has gained attention. Conducting a cost-effectiveness analysis in this context can help policymakers and healthcare providers make informed decisions about the allocation of resources for such interventions. To compare the cost-effectiveness of the smoking cessation of proposed strategies (PSs), PS1: enhanced counselling (EC) + nicotine replacement therapy (NRT) + bupropion tablet; PS2: behavioural intervention (BI) + NRT + promotion of bupropion sustained release (SR); PS3: EC + NRT + promotion of bupropion SR with the current strategy (BI +NRT+ Bupropion) in a population of smokers aged ≥15 years attending the PHC in Tamil Nadu. In this hypothetical cohort of 100,000 individuals using the decision tree analysis, a cost-effectiveness assessment was conducted for both proposed and existing strategies. The results were evaluated in terms of incremental cost-effectiveness ratios (ICERs) per person quitting smoking. To assess the robustness of the findings, one-way sensitivity analysis and probabilistic sensitivity analysis were performed which aims to explore and address the uncertainties associated with the outcomes. The cost of the current strategy (CS) was higher (₹359 or $4.28 million) when compared with PS1 (₹327 or $3.90 million) and PS3 (₹327 or $3.90 million) strategies. The PS2 with BI + bupropion SR + NRT was found to be more cost (₹2,720,571 or $ 32,414.76) as compared to current strategy. ICER values indicates that compared to the current strategy, the PS1 and PS3 were found to be cost-saving, whereas the PS2 was found to be cost-effective. The cost-effectiveness acceptability curve demonstrated that the PS1 and PS3 indicates 100% probability of the intervention being cost-saving. After excluding dominated interventions (PS2 and CS), the remaining strategies (PS1 and PS3) were compared. The PS3, with an incremental cost of ₹462,497 ($5,510) for 131 additional quitters, resulted in an ICER of ₹3,531 ($42) per quitter, making it a cost-effective option compared to PS1. Our study findings indicate that the need for healthcare providers and policymakers to implement PS3 with EC, NRT, Bupropion SR, as which was found to be cost-saving compared to current practices.

Modelling the cost effectiveness and budget impact of uterine botulinum toxin injections versus conventional treatment in severe dysmenorrhoea: A French Perspective.

To assess the cost-effectiveness sand the budgetary impact of the combination of botulinum toxin (BT) + conventional treatment (CT) (hormonal treatments + analgesics) compared with CT alone in patients suffering from severe dysmenorrhoea, using a Markov model. A Markov model was developed to estimate, from the perspective of French Health Insurance (HI), the cost effectiveness and the budgetary impact of BT+CT compared with CT alone. The main health states in the model were based on Visual Analogue Scale (VAS) scores and expert opinion. All model parameters were derived from a cohort of patients treated for 12 months at the Centre de Recherche de la Santé et de la Femme (CRSF) for severe dysmenorrhoea in 2021. A Cost-Utility Analysis (CUA) was carried out to assess the quality of life of patients, crucial in this context, in which the direct healthcare costs were considered in and Budget Impact Analysis (BIA). The main decision-making criteria were the Incremental Cost-Utility Ratio (ICUR) for the CUA and the net impact for the BIA. Deterministic and probabilistic univariate sensitivity analyses were performed to assess the robustness of our results. Over the 1-year time horizon (main analysis), the costs and quality-adjusted life year (QALY) of BT+CT versus CT alone were equal to €1895.65 vs €3055.20 and 2.03 QALYs vs 1.23 QALYs, respectively. Consequently, the ICUR equalled -€1651.5/QALY, which shows that, although the initial costs of BT are higher than those of CT, the reduced follow-up costs associated with the long-term efficacy of BT make it the most effective and economically dominant option at 1, 5 and 10 years. Sensitivity analyses show that 100% of Monte Carlo iterations are below the willingness-to-pay threshold of €30,0001/QALY, making BT+CT an efficient strategy that could be adopted and reimbursed. In the absence of a reference treatment for the management of severe dysmenorrhoea, BT+CT offering an improvement in quality of life, as well as a reduction in follow-up costs. It is therefore the most cost-effective strategy over 10 years.

Cost-effectiveness and budget impact of covering Burkitt lymphoma in children under Ghana’s National Health Insurance Scheme

BackgroundChildhood cancer is not a high priority in health care financing for many countries, including in Ghana. Delayed care seeking and treatment abandonment, often due to the financial burden of care seeking to families, are common reasons for a relatively low overall survival (OS) in low-and middle-income countries. In this study, we analyzed the cost-effectiveness of extending health insurance coverage to children with Burkitt lymphoma (BL) in Ghana.MethodsWe developed a Markov model in Microsoft Excel to estimate the costs and effects of BL treatment when National Health Insurance Scheme (NHIS) was provided compared to the status quo where NHIS does not cover care for childhood cancer. The analysis was undertaken from the societal and health system (payer) perspective. Both costs (measured in $) and effects, measured using disability adjusted life years (DALYs), were discounted at a rate of 3%. The time horizon was a lifetime. Probabilistic sensitivity analysis was done to assess uncertainty in the measurement of the incremental cost-effectiveness ratio (ICER). A budget impact analysis was undertaken from the perspective of the NHIS.ResultsIn the base-case analysis, the intervention (NHIS reimbursed treatment) was less costly than current practice ($8,302 vs $9,558). The intervention was also more effective with less DALYs per patient than the standard of care (17.6 vs 23.33). The ICER was -$219 per DALY averted from societal perspective and $113 per DALY averted from health system perspective. The probabilistic sensitivity analysis showed that the intervention is likely to be both less costly and more effective than current practice in 100% of the 1,000 simulations undertaken.ConclusionProviding health insurance coverage to children with BL is potentially cost-effective. The effectiveness and cost-savings relating to this strategy is driven by its positive impact on treatment initiation and retention. Based on this evidence, there has been a policy change where Ghana’s NHIS has prioritized financing for cancer treatment in children.

Therapeutic drug monitoring versus Bayesian AUC-based dosing for vancomycin in routine practice: a cost-benefit analysis.

AUC-based dosing with validated Bayesian software is recommended as a good approach to guide bedside vancomycin dosing. To compare treatment and vancomycin-associated acute kidney injury (AKI) costs between Bayesian AUC-based dosing and conventional therapeutic drug monitoring (TDM) using steady-state plasma concentrations of vancomycin administered as continuous infusion in hospitalized non-critically ill patients with severe Gram-positive infection. A cost-benefit analysis presented as a return on investment (ROI) analysis from a hospital perspective was conducted using a decision tree model (TDM versus AUC-based dosing) to simulate treatment cost (personnel, serum sampling and drug cost), vancomycin-associated AKI risk and cost up to 14 days. ROI was calculated against AUC-based software cost. One-way and probabilistic sensitivity analyses (respectively OWSA and PSA) were performed to check for robustness. In base case, an overall cost per patient of €621.0 with TDM and €543.6 with AUC-based dosing resulted in a treatment saving of €77.4 per patient when applying AUC-based dosing. This saving against the software cost (€26.9/patient) generated an ROI per patient of €1.9 per invested € in software [€1.9 (95% CI 1.6-2.2) in PSA]. Enrolling 900 AUC-based dosed patients annually translated to a net saving of €45 469. Software break-even was reached after 313 patients. In OWSA, a higher AKI risk with TDM strongly contributed to a positive ROI. AUC-based dosing appeared a cost-saving strategy compared with conventional TDM when applying base-case settings of vancomycin-associated AKI risk, treatment and AKI costs.

Cost-effectiveness analysis of three surgical approaches for parathyroidectomy in secondary hyperparathyroidism patients

BackgroundThere are three main surgical treatment options for secondary hyperparathyroidism (SHPT): subtotal parathyroidectomy (sPTX), total parathyroidectomy with auto-transplantation (tPTX+AT), and total parathyroidectomy (tPTX). However, a debate regarding which of these surgical methods is optimal has been ongoing. Aim of this study is to compare medical costs and final outcomes associated with the three surgical approaches for the entire treatment duration, aiming to identify the most cost-effective surgical method.MethodsBased on previous research data from domestic and international studies, as well as data from on-site surveys, TreeAge Pro 2022 software was used to construct a Markov model for the surgical treatment of SHPT patients. The model was run using data from the 2022 registered population of end-stage renal disease dialysis patients in China (1 million) as baseline cohort. Main indicators for this analysis are total cost, quality-adjusted life years, and incremental cost-effectiveness ratio (ICER). The study period is 10 years post-surgery, with a discount rate of 5% per year. Uncertainty in the model was assessed using one-way sensitivity analysis and probabilistic sensitivity analysis (PSA).ResultsThe costs incurred by SHPT patients undergoing sPTX, tPTX, and tPTX+AT within 10 years post-surgery are $7042.54, $9983.00, and $11435.60, respectively, with total utilities generated being 13.23 QALYs, 18.76 QALYs, and 18.69 QALYs. Compared to sPTX, the incremental costs and incremental effects of tPTX and tPTX+AT are $2,924.71 and $4,456.66, with 5.53 QALYs and 5.46 QALYs, respectively. The ICER for tPTX and tPTX+AT groups are $532.13/QALY and $805.10/QALY, respectively, which are well below our set willingness-to-pay (WTP) threshold. Sensitivity analysis results indicate that varying any parameter within a certain range over the given time interval will not cause the ICER to exceed the WTP threshold and will not reverse the primary analysis results.ConclusionIn the Chinese healthcare system, tPTX is considered the most cost-effective treatment for refractory hyperparathyroidism, when compared to tPTX+AT and sPTX.

The Budget Impact of Adopting Oral Pre-Exposure Prophylaxis for HIV Prevention: A Rapid Review

The World Health Organization (WHO) recommended the use of oral pre-exposure prophylaxis (PrEP) for HIV prevention in 2015. Although the number of countries with national PrEP recommendations increased from 2 in 2015 to 121 in 2019, there has been slow progress in Africa. The reason for this slow progress is that developing countries have budgetary constraints. Budget impact analysis (BIA) is an economic analysis that can help explore the affordability of oral PrEP. A rapid review was conducted to summarise the existing literature on the budget impact of adopting oral PrEP for HIV prevention. PubMed and Google Scholar databases were searched for relevant studies. Studies included in the review utilised primary data. Eleven studies met the inclusion criteria. This review reveals that most studies took a healthcare service provider perspective, targeted men who have sex with men (MSM), had time horizons of more than 10 years, used macro-costing, and performed univariate sensitivity analysis and discounting. If countries wish to perform a budget impact analysis of adopting oral PrEP for HIV prevention, we recommend that they select a target population that is most at risk of acquiring HIV. In addition, a time horizon of ten years or more should be used, and accurate values of the cost of oral PrEP and antiretroviral therapy (ART) and adherence to oral PrEP should be researched. Furthermore, deterministic sensitivity analysis should be carried out instead of probabilistic sensitivity analysis (PSA), as the degree of variability and the extent of the correlation among the parameters may not be known.

Cost-Utility Analysis of LifeVest® in Post-Myocardial Infarction Patients at Risk of Sudden Cardiac Death in England.

Patients with a left ventricular ejection fraction ≤ 35% are at increased risk of sudden cardiac death (SCD) within the first months after a myocardial infarction (MI). The wearable cardioverter defibrillator (WCD) is an established, safe and effective solution which can protect patients from SCD during the first months after an MI, when the risk of SCD is at its peak. This study aimed to evaluate the cost-effectiveness of WCD combined with guideline-directed medical therapy (GDMT) compared to GDMT alone, after MI in the English National Health Service (NHS). A multi-state Markov model, with a hypothetical cohort of 1000 patients, was developed to evaluate WCD + GDMT compared with GDMT alone, over a lifetime time horizon. Model input parameters were obtained from the pivotal randomised controlled trial and literature. The outcomes were costs and quality-adjusted life-years (QALYs), discounted at 3.5% annually, with overall results presented as an incremental cost-effectiveness ratio (ICER). The cost-effectiveness analysis demonstrated that WCD + GDMT is potentially a cost-effective option with an ICER of £23,024 per QALY gained, which is in the acceptable willingness to pay threshold (WTP) range of £20,000-£30,000 set by the National Institute for Health and Care Excellence (NICE) in England. Results of probabilistic sensitivity analysis (PSA) indicated that WCD + GDMT has 89.3% and 23% probability of being cost-effective at WTP thresholds of £30,000 and £20,000, respectively. Implementation of WCD in patients post-MI is potentially a cost-effective use of resources for the NHS and improves clinical outcomes amongst adherent patients and in circumstances where implantable cardioverter defibrillators are not indicated by the guidelines.

Semaglutide 2.4 mg versus Liraglutide 3 mg for the Treatment of Obesity in Greece: A Short-Term Cost-Effectiveness Analysis.

Obesity is a global health issue with significant economic implications for health systems. Pharmacotherapy, including semaglutide 2.4 mg and liraglutide 3 mg, offers a treatment option for weight management; however, its cost-effectiveness requires evaluation. This study assesses the short-term cost-effectiveness of semaglutide 2.4 mg versus liraglutide 3 mg in achieving clinically relevant weight loss targets at 68 weeks in Greece. A short-term cost-effectiveness analysis was conducted from the perspective of the Greek third-party payer [National Organization for the Provision of Health Services (EOPYY)], comparing costs and outcomes for semaglutide 2.4 mg and liraglutide 3 mg over a 68-week horizon. Effectiveness was measured by the proportion of patients achieving weight loss targets of ≥ 5%, ≥ 10%, ≥ 15%, and ≥ 20%, using efficacy data from the STEP-8 head-to-head trial, a 68-week, randomized, double-blind study conducted in the USA, comparing semaglutide 2.4 mg versus liraglutide 3 mg in adults who were overweight or had obesity without diabetes. Only direct medical costs were included, reflecting the payer perspective, and no discounting was applied owing to the short time horizon. Deterministic and probabilistic sensitivity analyses assessed the results' robustness. Semaglutide 2.4 mg had higher treatment costs (€3285.55) compared with liraglutide 3 mg (€2742.47) but demonstrated greater efficacy and a lower cost of control across all weight loss targets. The cost per patient achieving ≥ 5% weight loss was €3768.72 for semaglutide and €4718.66 for liraglutide, corresponding to a difference of €949.95 per patient. The cost difference widened at higher weight loss targets, with semaglutide showing differences of €6064.20 for ≥ 10% weight loss, €17,005.23 for ≥ 15%, and €37,296.00 for ≥ 20%. These findings were consistent across sensitivity analyses. Semaglutide 2.4 mg is likely to be a short-term, cost-effective treatment option for adults who are overweight or have obesity without diabetes in Greece.

Which is the most cost-effective antiseizure medication for initial monotherapy for focal epilepsy?

An increasing number of antiseizure medications (ASMs) are approved for monotherapy for focal epilepsy, but direct comparisons of the lifetime cost-effectiveness of all existing treatment strategies are lacking. This study aims to compare the cost-effectiveness of new ASMs and traditional ASMs as first-line monotherapy for newly diagnosed focal epilepsy. We used a Markov model to evaluate the lifetime cost-effectiveness of 10 ASMs in the treatment of focal epilepsy, with lacosamide (LCM) as a control, from the perspective of society in the United States. Effectiveness, cost data, and health state utilities were obtained from published literature. The cycle of the model is 6 months. Willingness to pay was defined as $150 000 per quality-adjusted life year (QALY). One-way and probabilistic sensitivity analyses were conducted to evaluate parameter uncertainty, and several scenario analyses were also conducted. The base case analysis showed that carbamazepine (CBZ) was the least costly ASM and more effective than valproic acid (VPA), levetiracetam (LEV), gabapentin (GBP), topiramate (TPM), and lamotrigine (LTG) from an American social perspective. In contrast, oxcarbazepine (OXC), phenytoin (PHT), phenobarbitone (PHB), LCM, and zonisamide (ZNS) were more effective than CBZ, with incremental cost-effectiveness ratios of $334 703.50, $325 610.99, $3 037 148.62, $1 178 954.91, and $108 153 360.85/QALY, respectively. The traditional ASMs were ranked as CBZ, PHT, VPA, and PHB; the new ASMs were ranked as OXC, LEV, LCM, LTG, TPM, GBP, and ZNS. When generic drugs are used, PHT, OXC, and CBZ remain the three most cost-effective options. In terms of cost-effectiveness, CBZ monotherapy is the best option for newly diagnosed focal epilepsy, followed by OXC, PHT, VPA, LEV, PHB, LCM, LTG, TPM, GBP, and ZNS. Most traditional ASMs are more cost-effective than new ASMs; OXC is an exception.

Association of Admission Nutritional Status Evaluated by Prognostic Nutritional Index with One-Year Walking Independence after Primary Total Hip Arthroplasty for Osteonecrosis of the Femoral Head: A Retrospective Matched Cohort Study Based on 1,152 Patients.

Malnutrition is significantly associated with unfavorable outcomes, but the relevant prognostic assessment is not emphasized in patients who have osteonecrosis of the femoral head (ONFH). This study aimed to assess the association between preoperative nutritional status evaluated by the prognostic nutritional index (PNI) and walking independence at one year postoperatively in patients who have ONFH undergoing primary total hip arthroplasty (THA). This was a retrospective analysis of prospectively collected data from patients admitted to a tertiary referral hospital who had ONFH and underwent primary unilateral THA from October 30, 2014 to April 26, 2019. The restricted cubic spline was used to assess the dose-effect relationship between PNI and recovery of walking independence. Propensity score matching was performed to balance potential preoperative confounders, and multivariable conditional logistic regression analysis was applied to assess the association between malnutrition and losing walking independence (LWI) with perioperative and follow-up factors for further adjustment. Inverse probability treatment weighting and sensitivity analyses were performed to test the robustness of the results. Subgroup analyses were used to determine potential population heterogeneity. We found a negative relationship between admission PNI levels and recovery of walking independence at one year postoperatively, and that low PNI (< 42.2) was independently associated with a 2.35-fold (95% CI [confidence interval], 1.16 to 4.77; P = 0.018) increased risk of LWI. The results were overall robust. Significant heterogeneities were observed in the subgroups of age, labor intensity, American Society of Anesthesiologists class, Charlson's comorbidity index, and Association Research Circulation Osseous stage (P for interaction < 0.05). Preoperative low-level PNI is a significant risk factor for LWI after THA in patients who have ONFH. This finding supports nutritional screening and interventions for surgeons in decision-making for elective surgery and postoperative rehabilitation management.