Probabilistic Sensitivity Analysis Research Articles

Cost-utility analysis of expanding the colorectal cancer screening policy in Flanders

ObjectiveSince 2013, Flanders has introduced a screening programme for colorectal cancer for all citizens aged between 50 and 74 years. The objective of this study was to assess the cost-utility of an expansion of the colorectal cancer screening policy in Flanders (Belgium) and to place these findings in the international context. MethodsCost-utility analysis using high-detail data about screening participation, screening results, and epidemiological data, a Markov cohort model has been constructed to study long-term costs and effects. A cost-utility analysis was performed as a three-way comparison between current, expanded (from age 45 years), and no screening scenarios, from a societal and healthcare perspective. Robustness was assessed by both one-way and probabilistic sensitivity analyses. ResultsAnalyses show that both current and expanded screening result in quality-adjusted life years (QALY) gains and are mostly cost-saving. Overall, 97.5% of Incremental Cost-Effectiveness Ratios (ICERs) remained well below € 2000 per QALY for all comparisons. Parameters related to the colonoscopy that follows a positive test result such as compliance and cost are especially impactful on the cost-effectiveness. ConclusionsScreening participation and screening costs have remained comparatively stable, making colorectal cancer screening a cost-effective (dominant) policy. Expanding the screen age to 45 years is also cost-effective (dominant) compared with current screening, albeit with a slimmer margin.

Cost-effectiveness analysis of tislelizumab vs. camrelizumab for the treatment of second-line locally advanced or metastatic esophageal squamous cell carcinoma

BackgroundEsophageal carcinoma is a type of cancer that occurs in the esophagus. For patients with locally advanced or metastatic esophageal squamous cell carcinoma who have either experienced disease progression following first-line standard chemotherapy or are intolerant to it, the prognosis is typically poor. Additionally, these patients often bear a substantial economic burden during the course of their treatment. Tislelizumab is a selective PD-1 inhibitor with efficacy proven in locally advanced or metastatic esophageal squamous cell carcinoma. The study aims to evaluate the cost-effectiveness of tislelizumab versus camrelizumab as the second-line treatment in locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) patients in China.MethodsFrom the perspective of China’s healthcare system, the partitioned survival model with three health states was established in a 3-week cycle and a lifetime horizon. Anchored matching adjusted indirect comparison was used for survival analyses based on individual patient data from RATIONALE 302 trial and the published ESCORT study due to the lack of head-to-head clinical trials. Only direct medical costs were included. Costs and utility values were derived from local charges, the published literature, and related databases. Sensitivity analyses and a scenario analysis were also performed to verify the robustness of the model results.ResultsCompared with camrelizumab monotherapy, tislelizumab monotherapy incurred a lower lifetime cost ($8,346 vs. $8,851) and yielded higher quality-adjusted life-years (QALYs) (0.87 vs. 0.63), which resulted in an incremental cost-effectiveness ratio (ICER) of -$2,051/QALY. Tislelizumab monotherapy is a dominant option over camrelizumab monotherapy in China. The three primary parameters upon which this result was most sensitive were the unit cost of camrelizumab, the unit cost of tislelizumab, and the duration of reactive cutaneous capillary endothelial proliferation (RCCEP). According to the probabilistic sensitivity analysis (PSA), tislelizumab monotherapy was 100% cost-effective when the WTP was 1–3 times GDP per capita in China($11,207/QALY∼$33,621/QALY). Scenario analysis showed that the result was consistent.ConclusionTislelizumab monotherapy is a dominant option compared with camrelizumab monotherapy as the second-line treatment for locally advanced or metastatic ESCC in China.

Cost-effectiveness analysis of alternative screening strategies for the detection of cervical cancer among women in rural areas of Western Kenya.

While the incidence of cervical cancer has dropped in high-income countries due to organized cytology-based screening programs, it remains the leading cause of cancer death among women in Eastern Africa. Therefore, the World Health Organization (WHO) now urges providers to transition from widely prevalent but low-performance visual inspection with acetic acid (VIA) screening to primary human papillomavirus (HPV) DNA testing. Due to high HPV prevalence, effective triage tests are needed to identify those lesions likely to progress and so avoid over-treatment. To identify the optimal cost-effective strategy, we compared the VIA screen-and-treat approach to primary HPV DNA testing with p16/Ki67 dual-stain cytology or VIA as triage. We used a Markov model to calculate the budget impact of each strategy with incremental quality-adjusted life years and incremental cost-effectiveness ratios (ICER) as the main outcome. Deterministic cost-effectiveness analyses show that the screen-and-treat approach is highly cost-effective (ICER 2469 Int$), while screen, triage, and treat with dual staining is the most effective with favorable ICER than triage with VIA (ICER 9943 Int$ compared with 13,177 Int$). One-way sensitivity analyses show that the results are most sensitive to discounting, VIA performance, and test prices. In the probabilistic sensitivity analyses, the triage option using dual stain is the optimal choice above a willingness to pay threshold of 7115 Int$ being cost-effective as per WHO standards. The result of our analysis favors the use of dual staining over VIA as triage in HPV-positive women and portends future opportunities and necessary research to improve the coverage and acceptability of cervical cancer screening programs.

Cost-effectiveness of treating relapsed or refractory 3L+ follicular lymphoma with axicabtagene ciloleucel vs mosunetuzumab in the United States

IntroductionNovel therapies for 3L+ relapsed/refractory (r/r) follicular lymphoma (FL) have been approved recently by the US Food and Drug Administration including anti-CD19 CAR-T therapies such as axicabtagene ciloleucel (axi-cel) and CD20 × CD3 T-cell-engaging bispecific monoclonal antibodies such as mosunetuzumab (mosun). The objective of this study was to assess the cost-effectiveness of axi-cel compared to mosun in 3L+ r/r FL patients from a US third-party payer perspective.MethodsA three-state (progression-free, progressed disease, and death) partitioned-survival model was used to compare two treatments over a lifetime horizon in a hypothetical cohort of US adults (age ≥18) receiving 3L+ treatment for r/r FL. ZUMA-5 and GO29781 trial data were used to inform progression-free survival (PFS) and overall survival (OS). Mosun survival was modeled via hazard ratios (HRs) applied to axi-cel survival curves. The PFS HR value was estimated via a matching-adjusted indirect comparison (MAIC) based on mosun pseudo-individual patient data and adjusted axi-cel data to account for trial populations differences. One-way sensitivity analysis (OWSA) and probabilistic sensitivity analyses (PSA) were conducted. Scenario analyses included: 1) the mosun HRs were applied to the weighted (adjusted) ZUMA-5 24-month data to most exactly reflect the MAIC, 2) mosun HR values were applied to axi-cel 48-month follow-up data, and 3) recent axi-cel health state utility values in diffuse large B-cell lymphoma patients.ResultsThe analysis estimated increases of 1.82 LY and 1.89 QALY for axi-cel compared to mosun. PFS for axi-cel patients was 6.42 LY vs. 1.60 LY for mosun. Increase of $257,113 in the progression-free state was driven by one-time axi-cel treatment costs. Total incremental costs for axi-cel were $204,377, resulting in an ICER of $108,307/QALY gained. The OWSA led to ICERs ranging from $240,255 to $75,624, with all but two parameters falling below $150,000/QALY. In the PSA, axi-cel had an 64% probability of being cost-effective across 5,000 iterations using a $150,000 willingness-to-pay threshold. Scenarios one and two resulted in ICERs of $105,353 and $102,695, respectively.DiscussionThis study finds that axi-cel is cost-effective compared to mosun at the commonly cited $150,000/QALY US willingness-to-pay threshold, with robust results across a range of sensitivity analyses accounting for parameter uncertainty.

Economic evaluation of trastuzumab in HER2-positive early breast cancer in Indonesia: A cost-effectiveness analysis.

Trastuzumab has significantly enhanced the survival and prognosis of individuals diagnosed with human epidermal growth factor receptor 2 (HER2)-positive early breast cancer. Considering its relatively high costs, we aimed to examine the cost-effectiveness of trastuzumab plus chemotherapy compared with chemotherapy alone in HER2-positive early breast cancer from an Indonesian healthcare payer's perspective. A Markov model was developed to project the lifetime health benefits and costs associated with trastuzumab treatment for a cohort of women with HER2-positive early breast cancer. Efficacy data and baseline characteristics in the base-case analysis were primarily derived from the 11-year results of the HERA trial. Costs were based on verified reimbursement data from Indonesia's Health and Social Security Agency (BPJS Kesehatan) of the year 2020. A scenario analysis was conducted with efficacy data based on the joint analysis from the NSABP B-31 and NCCTG N9831 trials, allowing for subgroup analysis by age at diagnosis. Univariate and probabilistic sensitivity analyses were conducted to assess the influence of parameter uncertainty. In the base-case analysis, the results indicated that the lifetime costs for trastuzumab plus chemotherapy and chemotherapy alone were US$33,744 and US$22,720, respectively, resulting in substantial incremental savings of US$11,024 per patient for the former. Trastuzumab plus chemotherapy also led to higher total quality-adjusted life years (QALYs) and life years gained (LYG), resulting in incremental cost-effectiveness ratios (ICERs) of US$6,842 per QALY and US$5,510 per LYG. In scenario analysis, the subgroup with an age at diagnosis <40 years old reflected the most cost-effective subgroup. Both the base-case and scenario analyses demonstrated cost-effectiveness with a willingness-to-pay threshold of three-times Gross Domestic Product (GDP). Sensitivity analyses confirmed the robustness of the findings and conclusions. In Indonesia, trastuzumab plus chemotherapy can be considered cost-effective compared to chemotherapy alone at a willingness-to-pay threshold of three times GDP, and it is likely most cost-effective in women <40 years of age.

Cost-effectiveness of atrial fibrillation detection with insertable cardiac monitors in large-artery or small-vessel disease stroke in the United Kingdom

Abstract Background The randomised STROKE-AF trial demonstrated clinical effectiveness of continuous cardiac monitoring to detect atrial fibrillation (AF) with insertable cardiac monitors (ICMs) for patients with ischemic stroke attributed to large-artery or small-vessel disease (LAD/SVD). ICMs were ten-fold more effective at detecting AF compared to the standard of care (SoC). However, cost-effectiveness of this monitoring strategy is unknown. Purpose The aim of this economic evaluation was to assess the cost-effectiveness of long-term continuous cardiac monitoring via ICMs, for detection of AF (and, in event of AF diagnosis, subsequent anticoagulation), in patients with ischemic stroke attributed to large-or-small vessel disease compared to the SoC of conventional follow up in the United Kingdom. Methods We constructed a Markov model with a cycle length of three months and a lifetime horizon to conduct the cost-effectiveness analysis, from the National Health Service (NHS) perspective. Costs and health-related benefits were discounted at an annual rate of 3.5%. We expressed outcomes in terms of quality-adjusted life-years (QALYs). Patient characteristics in the analysis and detection probabilities were based on the STROKE-AF trial. Detection of AF resulted in a switch in treatment from antiplatelet to direct oral anticoagulant (DOAC). Risk of cerebrovascular events were dependent on a patient’s AF detection status and subsequent treatment. Scenario, deterministic, and probabilistic sensitivity analyses were conducted to test the robustness of the base-case incremental cost-effectiveness ratio (ICER) and account for uncertainties in the model. We stratified patients into risk groups based on CHA2DS2-VASc scores and risk of AF detection. Results The base case incremental cost-effectiveness ratio of the implantation of an ICM was £15,485 per QALY, substantially under the NICE willingness-to-pay threshold of £20,000-£30,000 per QALY. ICM implantations resulted in 0.139 QALYs gained per patient at an incremental cost of £2,147. There were 0.054 fewer stroke events per patient in the ICM arm versus SoC. Results were robust under sensitivity analysis. Conclusion ICMs were cost-effective compared to SoC for detection of AF in patients with LAD/SVD stroke in the United Kingdom driven largely by more stroke events avoided. Subgroup analysis showed that ICMs were more cost-effective in patients with higher CHA2DS2-VASc scores and those with a higher individual risk of AF.Markov Model SchematicSummary ICER Results and Events avoided

An economic evaluation of first-line cryoballoon ablation versus antiarrhythmic drug therapy for the treatment of paroxysmal atrial fibrillation from a Danish healthcare perspective

Abstract Background As demonstrated in three randomized clinical trials (RCTs), initial rhythm control with first-line pulmonary vein isolation (PVI) using cryoballoon ablation decreases atrial arrhythmia recurrence compared to antiarrhythmic drug (AAD) therapy in patients with symptomatic paroxysmal atrial fibrillation (PAF). Purpose To assess the cost-effectiveness of first-line cryoablation versus first-line AADs in a Danish healthcare setting. Methods Data from 703 participants with symptomatic PAF enrolled into Cryo-FIRST, STOP AF First and EARLY-AF RCTs were used to estimate risk equations (rate of ablation, AF recurrence and resolution, AF-related hospital attendance in addition to health-related quality-of-life (HRQoL) utilities). These were then incorporated into a cost-effectiveness model (CEM) . The Danish cost data was derived from published Danish literature, DRG tariffs and pharmaceutical list prices in Denmark. Where parameters could not be derived, inputs were sourced from published literature or expert opinion. The CEM consisted of a decision tree (one-year time horizon) and a Markov model (three-month cycle length) hybrid, with a lifetime (40 years) time horizon. The CEM is from the perspective of the Danish healthcare system. Health benefits were expressed in quality-adjusted life-years (QALYs), and all costs and benefits were discounted at 3%. As there is no official cost-effectiveness threshold in Denmark, the UK threshold of £20,000 (~€23,200) was assumed. Input uncertainty within the CEM was explored using probabilistic sensitivity analysis. The results presented include data from the initial 12-week blanking period for all studies. Results The three-monthly rate of AF symptom recurrence had a reduction, on average, of 46.7% (p&amp;lt;0.001) in those treated with cryoablation. In addition, the cryoablation arm had a 72.8% (p&amp;lt;0.001) reduction in the monthly rate of receiving an ablation following initial treatment. The average cryoablation patient was also associated with a 4.3% (p=0.025) increase in their HRQoL. Finally, there was no difference in the rate of AF symptom resolution in those who failed initial treatment. Base case results per patient are presented in Table 1. Cryoablation was shown to be dominant when compared to AADs (0.159 more QALYs and €2,519 less costly), with a 99.96% probability of being cost-effective at a willingness-to-pay threshold of ~€23,200 per QALY gained. Individuals in both arms of the model were also expected to receive ~1.2 ablations over a lifetime horizon. Nonetheless, there was a 45% relative reduction in time spent in symptomatic AF states for individuals who were initially treated with cryoablation. Scenario analysis results are presented in Table 2. Cryoablation remained cost-effective over AADs in all scenarios conducted. Conclusions Initial rhythm control with cryoballoon ablation is cost-effective (dominant) when compared to AADs in the Danish healthcare system.Table 1:Key results (per patient)Table 2.Deterministic scenario analysis

Public Health Impact and Cost-Effectiveness Analysis of 4-Component Meningococcal Serotype B Vaccination for Infants in France.

In France, meningococcal serogroup B (MenB) is the most common serogroup causing invasive meningococcal disease (IMD) in infants and young children. Our objective was to illustrate the impact of model choices on health outcomes and the cost-effectiveness of infant vaccination with the multicomponent meningococcal serogroup B vaccine (4CMenB) versus no vaccine in France. A previously published dynamic transmission-based cost-effectiveness model was adapted for the French context using updated, French-specific demographic, epidemiological, and cost data. IMD incidence and long-term sequelae were derived through analysis of French healthcare and surveillance databases. A collective perspective over a 100-year time horizon was adopted, with a discount rate of 2.5%, reduced to 1.5% after the first 30 years. Deterministic and probabilistic sensitivity and scenario analyses were performed. In the base case analysis, infant vaccination with 4CMenB avoided 3101 MenB IMD cases in infants aged <1 year (-54%) and 6845 cases in all age groups (-21%). The estimated incremental cost-effectiveness ratio was €316,272/quality-adjusted life-year (QALY) but was highly sensitive to the types of sequelae included, MenB incidence, vaccine effectiveness parameters, and consideration of life-expectancy in IMD survivors (range: €65,272/QALY to €493,218/QALY). Using economic models compliant with French methodology guidelines, 4CMenB does not seem cost-effective; however, results are sensitive to model choices and 4CMenB immunization is an effective strategy to prevent MenB IMD cases and to improve quality of life and economic burden associated with MenB IMD treatment, especially with regard to long-term sequelae.

Pharmacoeconomic study of anti-influenza virus drugs in Japan based on a network meta-analysis.

An analysis was conducted in Japan to determine the most cost-effective neuraminidase inhibitor for the treatment of influenza virus infections from the healthcare payer's standpoint. This study reanalysed the findings of a previous study that had some limitations (no probabilistic sensitivity analysis and quality of life scores measured by the EQ-5D-3L instead of the EQ-5D-5L) and used a decision tree model with only three health conditions. This study incorporated new data from a network meta-analysis study into the first examination. The second examination involved constructing a new decision tree model encompassing seven health conditions and identifying costs, which consisted of medical costs and drug prices based on the 2020 version of the Japanese medical fee index. Effectiveness outcomes were measured using EQ-5D-5L questionnaires for adult patients with a history of influenza virus infections within a 14-day time horizon. Deterministic and probabilistic sensitivity analyses were performed to examine the uncertainty. In the first examination, the base-case cost-effectiveness analysis confirmed that oseltamivir outperformed laninamivir, zanamivir and peramivir, making it the most cost-effective neuraminidase inhibitor. The second examination revealed that oseltamivir dominated the other agents. Both deterministic and probabilistic sensitivity analyses showed robust results that validated oseltamivir as the most cost effective among the four neuraminidase inhibitors. This study thus reaffirms oseltamivir's position as the most cost-effective neuraminidase inhibitor for the treatment of influenza virus infections in Japan from the perspective of healthcare payment. These findings can help decision makers and healthcare providers in Japan.

Cost-effectiveness of ivosidenib versus chemotherapy for previously treated IDH1-mutant advanced intrahepatic cholangiocarcinoma in Taiwan

BackgroundInternational guidelines recommend ivosidenib followed by modified FOLFOX (mFOLFOX) for advanced intrahepatic cholangiocarcinoma (ICC) with isocitrate dehydrogenase 1 (IDH1) mutations. Taiwan National Health Insurance covers only fluorouracil/leucovorin (5-FU/LV) chemotherapy for this ICC group, and there has been no prior economic evaluation of ivosidenib. Therefore, we aimed to assess ivosidenib’s cost-effectiveness in previously treated, advanced ICC-presenting IDH1 mutations compared with mFOLFOX or 5-FU/LV.MethodsA 3-state partitioned survival model was employed to assess ivosidenib’s cost-effectiveness over a 10-year horizon with a 3% discount rate, setting the willingness-to-pay threshold at 3 times the 2022 GDP per capita. Efficacy data for Ivosidenib, mFOLFOX, and 5-FU/LV were sourced from the ClarIDHy, ABC06, and NIFTY trials, respectively. Ivosidenib’s cost was assumed to be NT$10,402/500 mg. Primary outcomes included incremental cost-effectiveness ratios (ICERs) and net monetary benefit. Deterministic sensitivity analyses (DSA) and probabilistic sensitivity analyses (PSA) were employed to evaluate uncertainty and explore price reduction scenarios.ResultsIvosidenib exhibited ICERs of NT$6,268,528 and NT$5,670,555 compared with mFOLFOX and 5-FU/LV, respectively, both exceeding the established threshold. PSA revealed that ivosidenib was unlikely to be cost-effective, except when it was reduced to NT$4,161 and NT$5,201/500 mg when compared with mFOLFOX and 5-FU/LV, respectively. DSA underscored the significant influence of ivosidenib’s cost and utility values on estimate uncertainty.ConclusionsAt NT$10,402/500 mg, ivosidenib was not cost-effective for IDH1-mutant ICC patients compared with mFOLFOX or 5-FU/LV, indicating that a 50–60% price reduction is necessary for ivosidenib to be cost-effective in this patient group.

Cost-effectiveness analysis of atezolizumab plus bevacizumab compared with sorafenib as first-line treatment in advanced hepatocellular carcinoma in Singapore

ABSTRACT Objectives This study aims to explore the cost-effectiveness of atezolizumab plus bevacizumab against sorafenib for first-line treatment of locally advanced or metastatic hepatocellular carcinoma (HCC) in Singapore. Methods A partitioned survival model was developed from a healthcare system perspective, with a 10-year lifetime horizon. Clinical inputs and utilities were obtained from the IMbrave150 trial. Healthcare resource use costs were obtained from published local sources; drug costs reflected the most recent public hospital selling prices. Outcomes included life years, quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs). Deterministic and probabilistic sensitivity analyses were performed to assess the model’s robustness. Results Atezolizumab plus bevacizumab offered an additional 1.42 life years and 1.09 QALYs, with an additional cost of S$111,847; the ICER was S$102,988/QALY. The World Health Organization considers interventions with ICERs <1 gross domestic product (GDP)/capita to be highly cost-effective. At a willingness-to-pay (WTP) threshold of S$114,165/QALY (Singapore’s 2022 GDP/capita), atezolizumab plus bevacizumab is cost-effective compared with sorafenib. The ICER was most sensitive to variations in utilities, but all parameter variations had no significant impact on the model outcomes. Conclusion At a WTP threshold of Singapore’s GDP/capita, atezolizumab plus bevacizumab is cost-effective compared with sorafenib.

Evaluating the economic efficiency of open, laparoscopic, and robotic distal pancreatectomy: an updated systematic review and network meta-analysis.

This study compared the cost-effectiveness of open (ODP), laparoscopic (LDP), and robotic (RDP) distal pancreatectomy (DP). Studies reporting the costs of DP were included in a literature search until August 2023. Bayesian network meta-analysis was conducted, and surface under cumulative ranking area (SUCRA) values, mean difference (MD), odds ratio (OR), and 95% credible intervals (CrIs) were calculated for outcomes of interest. Cluster analysis was performed to examine the similarity and classification of DP approaches into homogeneous clusters. A decision model-based cost-utility analysis was conducted for the cost-effectiveness analysis of DP strategies. Twenty-six studies with 29,164 patients were included in the analysis. Among the three groups, LDP had the lowest overall costs, while ODP had the highest overall costs (LDP vs. ODP: MD - 3521.36, 95% CrI - 6172.91 to - 1228.59). RDP had the highest procedural costs (ODP vs. RDP: MD - 4311.15, 95% CrI - 6005.40 to - 2599.16; LDP vs. RDP: MD - 3772.25, 95% CrI - 4989.50 to - 2535.16), but incurred the lowest hospitalization costs. Both LDP (MD - 3663.82, 95% CrI - 6906.52 to - 747.69) and RDP (MD - 6678.42, 95% CrI - 11,434.30 to - 2972.89) had significantly reduced hospitalization costs compared to ODP. LDP and RDP demonstrated a superior profile regarding costs-morbidity, costs-mortality, costs-efficacy, and costs-utility compared to ODP. Compared to ODP, LDP and RDP cost $3110 and $817 less per patient, resulting in 0.03 and 0.05 additional quality-adjusted life years (QALYs), respectively, with positive incremental net monetary benefit (NMB). RDP costs $2293 more than LDP with a negative incremental NMB but generates 0.02 additional QALYs with improved postoperative morbidity and spleen preservation. Probabilistic sensitivity analysis suggests that LDP and RDP are more cost-effective options compared to ODP at various willingness-to-pay thresholds. LDP and RDP are more cost-effective than ODP, with LDP exhibiting better cost savings and RDP demonstrating superior surgical outcomes and improved QALYs.

Protocol for cost-effectiveness analysis of a randomised trial of mHealth coaching (Bump2Baby and Me) compared with usual care for healthy gestational weight gain and postnatal outcomes in at-risk women and their offspring in the UK, Australia, Ireland and Spain

IntroductionGestational diabetes mellitus and overweight are associated with an increased likelihood of complications during birth and for the newborn baby. These complications lead to increased immediate and long-term healthcare costs...

Decision tree-Markov model of perinatal depression screening: a cost–utility analysis

BackgroundPerinatal depression affects the physical and mental health of pregnant women. It also has a negative effect on children, families, and society, and the incidence is high. We constructed a cost–utility analysis model for perinatal depression screening in China and evaluated the model from the perspective of health economics.MethodsWe constructed a Markov model that was consistent with the screening strategy for perinatal depression in China, and two screening strategies (screening and non-screening) were constructed. Each strategy was set as a cycle of 3 months, corresponding to the first trimester, second trimester, third trimester, and postpartum. The state outcome parameters required for the model were obtained based on data from the National Prospective Cohort Study on the Mental Health of Chinese Pregnant Women from August 2015 to October 2016. The cost parameters were obtained from a field investigation on costs and screening effects conducted in maternal and child health care institutions in 2020. The cost–utility ratio and incremental cost–utility ratio of different screening strategies were obtained by multiplicative analysis to evaluate the health economic value of the two screening strategies. Finally, deterministic and probabilistic sensitivity analyses were conducted on the uncertain parameters in the model to explore the sensitivity factors that affected the selection of screening strategies.ResultsThe cost–utility analysis showed that the per capita cost of the screening strategy was 129.54 yuan, 0.85 quality-adjusted life years (QALYs) could be obtained, and the average cost per QALY gained was 152.17 yuan. In the non-screening (routine health care) group, the average cost was 171.80 CNY per person, 0.84 QALYs could be obtained, and the average cost per QALY gained was 205.05 CNY. Using one gross domestic product per capita in 2021 as the willingness to pay threshold, the incremental cost–utility ratio of screening versus no screening (routine health care) was about −3,126.77 yuan, which was lower than one gross domestic product per capita. Therefore, the screening strategy was more cost-effective than no screening (routine health care). Sensitivity analysis was performed by adjusting the parameters in the model, and the results were stable and consistent, which did not affect the choice of the optimal strategy.ConclusionCompared with no screening (routine health care), the recommended perinatal depression screening strategy in China is cost-effective. In the future, it is necessary to continue to standardize screening and explore different screening modalities and tools suitable for specific regions.

An extended Bayesian network model for calculating dam failure probability based on fuzzy sets and dynamic evidential reasoning

Effective risk analysis is crucial for ensuring the safe operation of hydropower dams. Existing methods typically rely solely on subjective or objective data. The subjectivity of expert opinions and insufficient objective data affect the reliability of results. To reduce subjectivity in the risk analysis process and enhance the utilization of subjective and objective data, this study proposes an extended Bayesian network (BN) model for calculating the dam failure probability based on fuzzy sets (FSs) and dynamic evidential reasoning. To address the fuzziness and uncertainty of expert opinions, a Gaussian FS was introduced to transform expert opinions into fuzzy numbers. Additionally, a dynamic evidential reasoning approach was proposed to combine subjective and objective data, considering their differences, subjectivity, and potential conflicts. To maintain consistency in the fuzzification and defuzzification processes, an improved Onisawa formula was proposed. Finally, the BN was employed for probability prediction and sensitivity analyses. The model was applied to the Z hydropower dam in China. The results showed that the failure probability was 5.2 × 10−4, and the main controllable risk factors were identified. A comparative analysis was conducted and the results showed that the proposed method had a better ability to handle uncertainty, reduce subjectivity, and mitigate conflicts.

Cost-effectiveness analysis of procalcitonin and lung ultrasonography guided antibiotic prescriptions in primary care.

Antimicrobial resistance comes with high morbidity and mortality burden, and ultimately high impact on healthcare and social costs. Efficient strategies are needed to limit antibiotic overuse. This paper investigates the cost-effectiveness of testing patients with lower respiratory tract infection with procalcitonin, either at the point-of-care only or combined with lung ultrasonography. These diagnostic tools help detect the presence of bacterial pneumonia, guiding prescription decisions. The clinical responses of these strategies were studied in the primary care setting. Evidence is needed on their cost-effectiveness. We used data from a cluster-randomized bi-centric clinical trial conducted in Switzerland and estimated patient-level costs using data on resource use to which we applied Swiss tariffs. Combining the incremental costs of the two strategies and the reduction in the 28-days antibiotic prescription rate (APR) compared to usual care, we calculated Incremental Cost-Effectiveness Ratios (ICER). We also used the Cost-Effectiveness Acceptability Curve as an analytical decision-making tool. The robustness of the findings is ensured by Probabilistic Sensitivity Analysis and scenario analysis. In the base case scenario, the ICER compared to usual care is $2.3 per percentage point (pp) reduction in APR for the procalcitonin group, and $4.4 for procalcitonin-ultrasound combined. Furthermore, we found that for a willingness to pay per patient of more than $2 per pp reduction in the APR, procalcitonin is the strategy with the highest probability to be cost-effective. Our findings suggest that testing patients with respiratory symptoms with procalcitonin to guide antibiotic prescription in the primary care setting represents good value for money.

Health economic evaluation of 2-dose and 3-dose rotavirus vaccines in children below 5 years of age in Morocco

ABSTRACT Following the introduction of rotavirus vaccination into the Moroccan National Immunization Program, the prevalence of the disease has decreased by nearly 50%. However, evidence on the economic value of rotavirus vaccinations in Morocco is limited. This health economic analysis evaluated, from both country payer and societal perspectives, the costs and the cost-effectiveness of three rotavirus vaccines using a static, deterministic, population model in children aged < 5 years in Morocco. Included vaccines were HRV (2-dose schedule), HBRV (3-dose schedule) and BRV-PV 1-dose vial (3-dose schedule). One-way and probabilistic sensitivity analyses were conducted to assess the impact of uncertainty in model inputs. The model predicted that vaccination with HRV was estimated to result in fewer rotavirus gastroenteritis events (−194 homecare events, −57 medical visits, −8 hospitalizations) versus the 3-dose vaccines, translating into 7 discounted quality-adjusted life years gained over the model time horizon. HRV was associated with lower costs versus HBRV from both the country payer (−$1.8 M) and societal (−$4.1 M) perspectives, and versus BRV-PV 1-dose vial from the societal perspective (−$187,000), dominating those options in the cost-effectiveness analysis. However, costs of BRV-PV 1-dose vial were lower than HRV from the payer perspective, resulting in an ICER of approximately $328,376 per QALY, above the assumed cost effectiveness threshold of $3,500. Vaccination with a 2-dose schedule of HRV may be a cost-saving option and could lead to better health outcomes for children in Morocco versus 3-dose schedule rotavirus vaccines.

Cost-effectiveness of empagliflozin as add-on to standard of care for chronic kidney disease management in the United Kingdom

ABSTRACT Objective The sodium-glucose co-transporter-2 inhibitor empagliflozin was approved for treatment of adults with chronic kidney disease (CKD) on the basis of its demonstrated ability to slow CKD progression and reduce the risk of cardiovascular death. This analysis was performed to assess the cost effectiveness of empagliflozin plus standard of care (SoC) versus SoC alone in the treatment of CKD in the UK. Methods A comprehensive, patient-level CKD progression model that simulates the evolution of risk factors for disease progression based on CKD-specific equations and clinical data was used to project a broad range of CKD-related complications. Patient baseline characteristics, distribution across Kidney Disease Improving Global Outcomes (KDIGO) health states, and changes in estimated glomerular filtration rate (eGFR), urine albumin-creatinine ratio (uACR), and other parameters while on treatment, were derived from the EMPA-KIDNEY trial. UK cost and utilities/disutilities were sourced from the literature. Univariate and probabilistic sensitivity analyses were conducted. Annual discounting of 3.5% was applied on costs and outcomes. Results Over a 50-year horizon, SoC resulted in per-patient costs, life years, and QALYs of £95,930, 8.55, and 6.28, respectively. Empagliflozin plus SoC resulted in an incremental gain in life years (+1.04) and QALYs (+0.84), while decreasing per-patient costs by £6,019. Empagliflozin was more effective and less costly (dominant) with a net monetary benefit of £22,849 at the willingness-to-pay threshold of £20,000. Although treatment cost was higher for empagliflozin, this was more than offset by savings in kidney replacement therapy. Empagliflozin remained highly cost-effective in patients with and without diabetes, and across scenario and sensitivity analyses. Limitations This analysis is limited by reliance on short-term clinical trial data and by uncertainties in modelling CKD progression. Conclusions Empagliflozin as an add-on to SoC for treatment of adults with CKD represents cost-effective use of UK National Health Service (NHS) resources.

Cost Effectiveness of Adding Fenfluramine to Standard of Care for Patients with Dravet Syndrome in Sweden.

This study evaluated, in a Swedish setting, the cost effectiveness of fenfluramine (FFA) as an add-on to standard of care (SoC) for reducing seizure frequency in Dravet syndrome, a severe developmental epileptic encephalopathy. Cost effectiveness of FFA+SoC compared with SoC only was evaluated using a patient-level simulation model with a lifetime horizon. Patient characteristics and treatment effects, including convulsive seizures, seizure-free days and mortality, were derived from FFA clinical trials. Resource use and costs included cost of drug acquisition, routine care and monitoring, as well as ongoing and emergency resources. Quality of life (QoL) estimates for patients and their caregivers were derived from clinical trial data. Robustness was evaluated by one-way sensitivity analysis, probabilistic sensitivity analysis and scenario analyses. Lifetime cost of FFA+SoC was ~3 million SEK per patient compared with ~1.5 million SEK for SoC only. FFA+SoC generated 15% more QALYs than SoC only (21.2 vs 18.5 over a lifetime), resulting in an incremental cost-effectiveness ratio (ICER) of ~540,000SEK. Moreover, FFA+SoC had a higher probability of being cost effective than SoC only from a willingness-to-pay threshold of 710,000SEK. Results remained generally consistent across scenario analyses, with only few exceptions (exclusions of carer utility or FFA effect on sudden unexpected death in epilepsy). Due to better seizure control, FFA is a clinically meaningful add-on therapy and was estimated to be a cost-effective addition to current SoC for patients with this rare disease in Sweden at a willingness-to-pay threshold of 1,000,000 SEK.

Cost-effectiveness analysis of tislelizumab plus chemotherapy as the first-line treatment for advanced or metastatic oesophageal squamous cell carcinoma in China.

We aimed to investigate the cost-effectiveness of tislelizumab plus chemotherapy compared to chemotherapy alone as a first-line treatment for advanced or metastatic oesophageal squamous cell carcinoma (OSCC). A partitioned survival model was developed to evaluate the cost-effectiveness of tislelizumab plus chemotherapy versus chemotherapy alone in patients with advanced or metastatic OSCC over a 10-year lifetime horizon from the perspective of the Chinese healthcare system. Costs and utilities were derived from the drug procurement platform and published literature. The model outcomes comprised of costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER). One-way and probabilistic sensitivity analyses were conducted to address uncertainty and ensure the robustness of the model. Tislelizumab plus chemotherapy yielded an additional 0.337 QALYs and incremental costs of $7,117.007 compared with placebo plus chemotherapy, generating an ICER of $21,116.75 per QALY, which was between 1 time ($12,674.89/QALY) and 3 times GDP ($38,024.67/QALY) per capita. In one-way sensitivity analysis, the ICER is most affected by the cost of oxaliplatin, paclitaxel and tislelizumab. In the probabilistic sensitivity analysis, when the willingness-to-pay threshold was set as 1 or 3 times GDP per capita, the probability of tislelizumab plus chemotherapy being cost-effective was 1% and 100%, respectively. Tislelizumab plus chemotherapy was probably cost-effective compared with chemotherapy alone as the first-line treatment for advanced or metastatic OSCC in China.