Probabilistic Sensitivity Analysis Research Articles

Oral aripiprazole in the treatment of tic disorders in China: a cost-effectiveness analysis based on a mapping algorithm derived from a Chinese children and adolescents population

BackgroundOral aripiprazole exhibits favorable clinical efficacy and safety in the suppression of tics in children and adolescents with tic disorders. This study aims to evaluate and compare the cost-effectiveness of high-dose and low-dose aripiprazole in children and adolescents with tic disorders from the perspective of the Chinese healthcare system.MethodsA questionnaire survey was conducted on 146 patients with tic disorders, of whom 144 completed EQ-5D-Y and YGTSS. Four models were built to convert YGTSS onto EQ-5D-Y utility using two mapping algorithms. We constructed a decision tree model containing efficacy and safety to compare the cost-effectiveness of high-dose and low-dose aripiprazole based on our mapping function.ResultsThe GLM with model 1 (YGTSS total tic scores) was selected as the preferred function in our decision tree model. The base case cost-effectiveness analysis showed that compared to low-dose aripiprazole, high-dose aripiprazole improves effectiveness by 0.001QALYs and increases the overall cost by $197.99, resulting in an ICER of $174339.22 per QALY, which exceeds three times the gross domestic product per capita. Hence, high-dose aripiprazole is not likely to be a cost-effective option for child patients with tic disorders. One-way sensitivity analysis and probabilistic sensitivity analysis showed that these results is robust.ConclusionOn the basis of currently available data, low-dose aripiprazole may be a safe, effective, and economical dosage for children and adolescents with tic disorders.LimitationsThe main limitation of our study is the lack of utility directly used for cost-effectiveness analysis. We obtained the utility of patients with tic disorders indirectly by the mapping function. This may introduce some bias and uncertainty. And it is a limitation to use the direct medical costs of Germany in our model. Although we converted it to the equivalent value of China using purchasing power parities, caution should be exercised when interpreting the results of this study.

A cost-utility analysis of cochlear implants for single sided deafness in adults and children in the Netherlands.

Cochlear Implant (CI) has been shown to improve speech comprehension, sound localization and tinnitus in adults with Single-Sided-Deafness (SSD) compared to standard treatment currently available in the Dutch setting such as a CROS (Contralateral Routing of Signals) hearing device or a BCD (Bone Conduction Device). Also, for the pediatric population with SSD, CI has shown to be clinically meaningful. Because currently no information is available on the health economic effects of CI in adults and children with SSD in the Netherlands, a cost-utility analysis was conducted. We developed a Markov cohort model, for both the adult and pediatric SSD population, with three states: implant, no implant, and dead. CI was compared with the Bone Conduction Device (BCD) treatment, requiring surgery and no specific treatment. The time horizon of the model was lifelong, costs were discounted with 3% and effects with 1.5%. A societal perspective was taken, including productivity costs in the analysis, with costing data based on publicly available prices for the Netherlands. Values for clinical outcome parameters, i.e. hearing gain, and event probabilities were based on existing literature. Deterministic and probabilistic sensitivity analyses as well as scenario analyses were performed to outline uncertainty of individual and combined parameters. Mean per patient costs for CI in the adult population were €194,051 (95%-CrI €177,274 to €211,108) compared to the total costs of €185,310 (95%-CrI €182,367 to €194,142) for BCD resulting in a cost difference of €8,826 (95%-CrI -€5,020 to €18,252). Compared to no treatment, the cost difference was -€25,089 (95%-CrI -€31,678 to -€6,003). Adults who were treated with CI gained 18.41 (95%-CrI 18.07 to 18.75) quality adjusted life years (QALY) whereas BCD patients gained 15.81 QALYs (95%-CrI 15.53 to 16.10), a difference of 2.60 QALYs (95%-CrI 2.15 to 3.05). The Incremental Cost Effectiveness Ratio (ICER) for adults with CI was determined to be €3,494/QALY gained. Patient without treatment gained 13.46 QALY (95%-CrI 13.20 to 13.73), a difference of 4.95 (95%-CrI 4.87 to 5.01) resulting in CI dominating no treatment. The ICER remained below the Dutch threshold of €20,000/QALY. The probabilistic sensitivity analyses confirmed the results. For children, CI dominated when compared to BCD and when compared to no treatment. Compared to BCD, CI led to a cost saving of €29,611 (95%-CrI -€126,800 to €54,375) and compared to no treatment, CI resulted in a cost saving of €57,658 (95%-CrI -€146,687 to €5,919). The incremental QALY gain compared to BCD was 7.22 (95%-CrI 4.19 to 8.55) and 26.03 (95%-CrI 20.82 to 31.06) compared to no treatment. Based on the results of this health economic evaluation with a Markov cohort model, it is very likely that CI is cost-effective compared to BCD and to no treatment in the Dutch adult and pediatric population with SSD. In both populations the ICER was below the Dutch cost-effectiveness threshold of €20,000/QALY.

Comparing the effectiveness and cost-effectiveness of alternative Type 2 Diabetes monitoring intervals in resource limited settings.

Type 2 Diabetes (T2D) represents a growing disease burden in South Africa. While glycated haemoglobin (HbA1c) testing is the gold standard for long-term blood glucose management, recommendations for HbA1c monitoring frequency are based on expert opinion. This study investigates the effectiveness and cost effectiveness of alternative HbA1c monitoring intervals in the management of T2D. A Markov model with three health states (HbA1c <7%, HbA1c ≥ 7%, Dead) was used to estimate lifetime costs and quality-adjusted-life-years (QALYs) of alternative HbA1c monitoring intervals among patients with T2D, using a provider's perspective and a 3% discount rate. HbA1c monitoring strategies (three-monthly, four-monthly, six-monthly, and annual tests) were evaluated with respect to the incremental cost-effectiveness ratio (ICER) assessing each comparator against a less costly, undominated alternative. The scope of costs included the direct medical costs of managing diabetes. Transition probabilities were obtained from routinely collected public sector HbA1c data, while health service utilization and health-related-quality-of-life (HRQoL) data were obtained from a local cluster randomized controlled trial. Other parameters were obtained from published studies. Robustness of findings was evaluated using one-way and probabilistic sensitivity analyses. A South African indicative cost-effectiveness threshold of USD2,665 was adopted. Annual and lifetime costs of managing diabetes increased with HbA1c monitoring, while increased monitoring provides higher QALYs and Life Years. For the overall cohort, the ICER for six-monthly vs annual monitoring was cost-effective (USD 2,322.37 per QALY gained), whereas the ICER of moving from six-monthly to three-monthly monitoring was not cost-effective(USD 6,437.79 per QALY gained). The ICER for four-monthly vs six-monthly monitoring was extended dominated. The sensitivity analysis showed that the ICERs were most sensitive to health service utilization rates. While the factors influencing glycaemic control are multifactorial, six-monthly monitoring is potentially cost-effective while more frequent monitoring could further improve patient HrQoL.

Evaluating the cost-effectiveness of Cy-Tb for LTBI in India: a comprehensive economic modelling analysis.

Latent tuberculosis infection (LTBI) remains a significant challenge, as there is no gold standard diagnostic test. Current methods used for identifying LTBI are the interferon-γ release assay (IGRA), which is based on a blood test, and the tuberculin skin test (TST), which has low sensitivity. Both these tests are inadequate, primarily because they have limitations with the low bacterial burden characteristic of LTBI. This highlights the need for the development and adoption of more specific and accurate diagnostic tests to effectively identify LTBI. Herein we estimate the cost-effectiveness of the Cy-Tb test as compared with the TST for LTBI diagnosis. An economic modelling study was conducted from a health system perspective using decision tree analysis, which is most widely used for cost-effectiveness analysis using transition probabilities. Our goal was to estimate the incremental cost and number of TB cases prevented from LTBI using the Cy-Tb diagnostic test along with TB preventive therapy (TPT). Secondary data such as demographic characteristics, treatment outcome, diagnostic test results and cost data for the TST and Cy-Tb tests were collected from the published literature. The incremental cost-effectiveness ratio was calculated for the Cy-Tb test as compared with the TST. The uncertainty in the model was evaluated using one-way sensitivity analysis and probability sensitivity analysis. The study findings indicate that for diagnosing an additional LTBI case with the Cy-Tb test and to prevent a TB case by providing TPT prophylaxis, an additional cost of 18658 Indian rupees (US${\$}$223.5) is required. The probabilistic sensitivity analysis indicated that using the Cy-Tb test for diagnosing LTBI was cost-effective as compared with TST testing. If the cost of the Cy-Tb test is reduced, it becomes a cost-saving strategy. The Cy-Tb test for diagnosing LTBI is cost-effective at the current price, and price negotiations could further change it into a cost-saving strategy. This finding emphasizes the need for healthcare providers and policymakers to consider implementing the Cy-Tb test to maximize economic benefits. Bulk procurements can also be considered to further reduce costs and increase savings.

First-Line Treatment with Atezolizumab plus Bevacizumab and Chemotherapy for US Patients with Metastatic, Persistent, or Recurrent Cervical Cancer: A Cost-Effectiveness Analysis

ObjectiveThe BETAcc clinical trial demonstrated that chemotherapy combined with bevacizumab plus atezolizumab (CBA) significantly prolonged progression-free survival (PFS) and overall survival (OS) in patients with metastatic, persistent, or recurrent cervical cancer. However, to our knowledge, the economic value of using this new therapy for this indication is currently unknown. Therefore, our study aims to evaluate the cost-effectiveness of CBA for the first-line treatment of metastatic, persistent, or recurrent cervical cancer from the United States healthcare payers perspective. MethodsA state-transition Markov model over a 10-year lifetime horizon was developed to compare the cost and effectiveness of CBA versus chemotherapy plus bevacizumab (CB). The primary outcomes of our study included costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs). One-way sensitivity analysis and probabilistic sensitivity analysis were performed to assess the robustness of the results. ResultsCBA was associated with an additional 0.58 QALY at an extra cost of $172,495.90 compared to CB. The ICER was $295,972.43/QALY, significantly higher than the willingness-to-pay (WTP) threshold value of $150,000/QALY. One-way sensitivity analyses revealed that results were most sensitive to the PFD utility, the unit cost of atezolizumab, and PD utility. Probabilistic sensitivity analysis indicated that CBA achieved a 4.3% probability of cost-effectiveness at a $150,000/QALY threshold. To achieve cost-effectiveness, the unit price of atezolizumab must be reduced by approximately 56.6%. ConclusionsCBA treatment is unlikely to be a cost-effective option compared with CB for patients with persistent, recurrent, or metastatic cervical cancer in the United States.

Cost-Effectiveness of Low-Dose Computed Tomography Screenings for Lung Cancer in High-Risk Populations: A Markov Model.

Domestic and foreign studies on lung cancer have been oriented to the medical efficacy of low-dose computed tomography (LDCT), but there is a lack of studies on the costs, value and cost-effectiveness of the treatment. There is a scarcity of conclusive evidence regarding the cost-effectiveness of LDCT within the specific context of Taiwan. This study is designed to address this gap by conducting a comprehensive analysis of the cost-effectiveness of LDCT and chest X-ray (CXR) as screening methods for lung cancer. Markov decision model simulation was used to estimate the cost-effectiveness of biennial screening with LDCT and CXR based on a health provider perspective. Inputs are based on probabilities, health status utility (quality-adjusted life years (QALYs)), costs of lung cancer screening, diagnosis, and treatment from the literatures, and expert opinion. A total of 1,000 simulations and five cycles of Markov bootstrapping simulations were performed to compare the incremental cost-utility ratio (ICUR) of these two screening strategies. Probability and one-way sensitivity analyses were also performed. The ICUR of early lung cancer screening compared LDCT to CXR is $-24,757.65/QALYs, and 100% of the probability agree to adopt it under a willingness-to-pay (WTP) threshold of the Taiwan gross domestic product (GDP) per capita ($35,513). The one-way sensitivity analysis also showed that ICUR depends heavily on recall rate. Based on the prevalence rate of 39.7 lung cancer cases per 100,000 people in 2020, it could be estimated that LDCT screening for high-risk populations could save $17,154,115. LDCT can detect more early lung cancers, reduce mortality and is cost-saving than CXR in a long-term simulation of Taiwan's healthcare system. This study provides valuable insights for healthcare decision-makers and suggests analyzing cost-effectiveness for additional variables in future research.

Cost-Effectiveness of an Ultrasound-First Strategy in the Diagnostic Evaluation of Noncalcified Lesions Recalled From Screening Digital Breast Tomosynthesis.

Background: Ultrasound may be sufficient in the diagnostic evaluation of many noncalcified lesions recalled from screening digital breast tomosynthesis (DBT). In some scenarios, omission of diagnostic mammography can save healthcare costs. Purpose: To evaluate the cost-effectiveness of a strategy of ultrasound first versus of diagnostic mammography first in the diagnostic evaluation of noncalcified lesions recalled from screening DBT. Methods: Decision tree analysis was performed to compare ultrasound first versus diagnostic mammography first in the diagnostic evaluation of DBT-recalled noncalcified lesions from a U.S. healthcare system perspective with a 40-year-horizon. The analysis used probabilities and prevalence information from published single-institution prospective data, additional literature-derived estimates of diagnostic test performance, and Medicare allowable reimbursement rates. Health states were represented in a Markov chain model. Each strategy's total cost and effectiveness [in quality-adjusted life years (QALY)] were estimated. Cost-effectiveness was assessed through incremental cost-effectiveness ratios (ICER) and incremental net monetary benefit, using a willingness-to-pay (WTP) threshold of $100,000 per QALY gained. Deterministic sensitivity analyses were performed to estimate the impact of different input parameters; probabilistic sensitivity analysis with Monte Carlo simulations was performed to estimate the impact of combined uncertainty across parameters. Results: In the base-case scenario, for diagnostic evaluation of DBT-recalled noncalcified lesions, a strategy of ultrasound first, versus a strategy of diagnostic mammography first, was more cost-saving (total cost of $17,672 vs $18,323) and more effective (QALYs of 23.1309 vs 23.1306), over the 40-year horizon. Ultrasound first resulted in an ICER of +2,170,2000 and incremental net monetary benefit of $681 versus diagnostic mammography first. Therefore, ultrasound first was deemed the more cost-effective strategy at the WTP threshold. In deterministic sensitivity analyses, the most important driver of cost-effectiveness was the lost utility from delayed diagnosis, followed by relative sensitivities of ultrasound and diagnostic mammography. In probabilistic sensitivity analysis, ultrasound first was the better strategy in 93.0% of iterations. Conclusion: A strategy of ultrasound first, with or without diagnostic mammography, is more cost effective compared to a traditional strategy of diagnostic mammography first. Clinical Impact: This cost-effectiveness analysis supports growing prioritization of ultrasound as the primary method for evaluating DBT-recalled noncalcified lesions.

Economic evaluation of diagnosis and treatment for latent tuberculosis infection among contacts of pulmonary tuberculosis patients in Thailand

Currently, interferon-gamma release assay (IGRA) is costly and not included as latent tuberculosis infection (LTBI) screening test strategy in Thailand’s Universal Coverage Scheme (UCS) benefit package. The objective of this study was to assess the cost-utility of LTBI screening strategies among tuberculosis (TB) contacts in Thailand. A hybrid decision tree and Markov model was developed to compare the lifetime costs and health outcomes of tuberculin skin test (TST) and IGRA, in comparison to no screening, based on a societal perspective. Health outcomes were the total number of TB cases averted and quality-adjusted life years (QALYs), with results presented as an incremental cost-effectiveness ratio (ICER). One-way and probabilistic sensitivity analyses were performed to explore uncertainties in all parameters. The ICER of TST compared with no screening was 27,645 baht per QALY gained, while that of IGRA compared to TST was 851,030 baht per QALY gained. In a cohort of 1000 TB contacts, both TST and IGRA strategies could avert 282 and 283 TB cases, respectively. At the Thai societal willingness-to-pay threshold of 160,000 baht per QALY gained, TST was deemed cost-effective, whereas IGRA would not be cost-effective, unless the cost of IGRA was reduced to 1,434 baht per test.

Economic evaluation of the "paramedics and palliative care: bringing vital services to Canadians" program compared to the status quo.

Based on programs implemented in 2011-2013 in three Canadian provinces to improve the support paramedics provide to people receiving palliative care, the Canadian Partnership Against Cancer and Healthcare Excellence Canada provided support and funding from 2018 to 2022 to spread this approach in Canada. The study objectives were to conduct an economic evaluation of "the Program" compared to the status quo. A probabilistic decision analytic model was used to compare the expected costs, the quality-adjusted life years (QALYs) and the return on investment associated with the Program compared to the status quo from a publicly funded healthcare payer perspective. Effectiveness data and Program costs, expressed in 2022 Canadian dollars, from each jurisdiction were supplemented with literature data. Probabilistic sensitivity analyses varying key model assumptions were conducted. Analyses of 5416 9-1-1 calls from five jurisdictions where paramedics provided support to people with palliative care needs between April 1, 2020 and March 31, 2022 indicated that 60% of the 9-1-1 calls under the Program enabled people to avoid transport to the emergency department and receive palliative care at home. Treating people at home saved paramedics an average of 31min (range from 15 to 67). The Program was associated with cost savings of $2773 (95% confidence interval [CI] $1539-$4352) and an additional 0.00069 QALYs (95% CI 0.00024-0.00137) per 9-1-1 palliative care call. The Program return on investment was $4.6 for every $1 invested. Threshold analyses indicated that in order to be cost saving, 33% of 9-1-1 calls should be treated at home under the Program, the Program should generate a minimum of 97 calls per year with each call costing no more than $2773. The Program was cost-effective in the majority of the scenarios examined. These results support the implementation of paramedic-based palliative care at home programs in Canada.

Cost‑effectiveness analysis of tislelizumab plus chemotherapy in Chinese patients with advanced or metastatic oesophageal squamous cell carcinoma

The RATIONALE-306 study revealed that patients with advanced or metastatic oesophageal squamous cell carcinoma (OSCC) could benefit from treatment with tislelizumab plus chemotherapy. This study aimed to evaluate the cost-effectiveness of tislelizumab plus chemotherapy for treating OSCC from the perspective of the Chinese healthcare system. Partitioned survival model estimated the cost-effectiveness of tislelizumab plus chemotherapy compared with chemotherapy alone for treating OSCC using RATIONALE-306 data. Costs and utilities were obtained from local databases and published studies. Costs, quality-adjusted life-years (QALYs), life-years, incremental cost-effectiveness ratios (ICER), incremental net health benefits (INHB), and incremental net monetary benefits (INMB) were outcomes. Price simulation were conducted at the willingness-to-pay (WTP) threshold. Sensitivity and subgroup analyses were performed to assess model robustness. Compared with chemotherapy alone, tislelizumab plus chemotherapy yielded an ICER of USD 27,896/QALY, gained an additional 0.414 QALYs and 0.751 life-years, and increased the cost by USD 11,560. Probabilistic sensitivity analysis revealed that tislelizumab plus chemotherapy was cost-effective at the WTP of USD 38,258/QALY with probability of 94.43%. When the price in China was less than USD 3.714 per mg, the price simulation results indicated that tislelizumab plus chemotherapy was cost-effective at a WTP threshold of USD 38,258. Tislelizumab plus chemotherapy yielded an INHB of 0.112 QALYs and an INMB of USD 4,279 compared with chemotherapy alone at a WTP threshold of USD 38,258. Based on the sensitivity analyses, the above results were stable. A general trend was observed for subgroups with better survival benefits related to a higher probability of cost-effectiveness. From the Chinese healthcare perspective, tislelizumab plus chemotherapy is more cost-effective than chemotherapy alone as a first-line therapy for OSCC. These findings can help clinicians make optimal clinical decisions and assist decision-makers in evaluating the cost-effectiveness of tislelizumab in clinical practice.

Point-of-care Testing HbA1c screening for type 2 diabetes in urban and rural areas of China: a cost-effectiveness analysis

BackgroundPoint-of-care Testing (POCT) glycosylated hemoglobin (HbA1c) is a convenient, cheap, effective and accessible screening method for type 2 diabetes in rural areas and community settings that is widely used in the European region and Japan, but not yet widespread in China. The study is the first to evaluate the cost-effectiveness of POCT HbA1c, fasting capillary glucose (FCG), and venous blood HbA1c to screen for type 2 diabetes in urban and rural areas of China, and to identify the best socio-economically beneficial screening strategy.MethodsBased on urban and rural areas in China, economic models for type 2 diabetes screening were constructed from a social perspective. The subjects of this study were adults aged 18–80 years with undiagnosed type 2 diabetes. Three screening strategies were established for venous blood HbA1c, FCG and POCT HbA1c, and cost-effectiveness analysis was performed by Markov models. One-way sensitivity analysis and probabilistic sensitivity analysis were performed on all parameters of the model to verify the stability of the results.ResultsCompared with FCG, POCT HbA1c was cost-effective with an incremental cost-utility ratio (ICUR) of $500.06/quality-adjusted life year (QALY) in urban areas and an ICUR of $185.10/QALY in rural areas, within the willingness-to-pay threshold (WTP = $37,653). POCT HbA1c was cost-effective with lower cost and higher utility compared with venous blood HbA1c in both urban and rural areas. In the comparison of venous blood HbA1c and FCG, venous blood HbA1c was cost-effective (ICUR = $20,833/QALY) in urban areas but not in rural areas (ICUR = $41,858/QALY). Sensitivity analyses showed that the results of the study were stable and credible.ConclusionsPOCT HbA1c was cost-effective for type 2 diabetes screening in both urban and rural areas of China, which could be considered for future clinical practice in China. Factors such as geographic location, local financial situation and resident compliance needed to be considered when making the choice of venous blood HbA1c or FCG.

Evaluating Cost-Effectiveness of Antiretroviral Therapy over Time: A Cohort and Cost-Effectiveness Study.

To estimate the costs and cost-effectiveness of introducing highly active antiretroviral therapy (HAART) in Denmark based on real-world evidence for the three treatment eras pre-HAART (1985-1995), early HAART (1996-2005), and late HAART (2006-2017). We performed a cohort study using Danish clinical and administrative registries to estimate costs, quality-adjusted life-years (QALYs), and life-years (LY) gained per person living with human immunodeficiency virus (PLHIV) in three treatment eras. The study utilized Markov modeling for a health economic evaluation, which summarized inputs from real-world evidence and estimated the cost-effectiveness in 2017 prices of the introduction of HAART in Denmark. We performed deterministic and probabilistic sensitivity analyses to assess the robustness of the results. The total annual costs per PLHIV increased with the introduction of HAART for the index year but decreased in the incremental years and the last year of life. The total lifetime discounted (and undiscounted) cost for an average PLHIV was €91,010 (€128,981) in pre-HAART, €103,130 (€199,062) in early HAART, and €126,317 (€254,964) in late HAART. The estimated incremental cost-effectiveness ratios showed that early HAART was cost-effective compared with pre-HAART with an incremental cost-effectiveness ratio (ICER) of €1378 per QALY, and that late HAART was cost-effective compared with early HAART with an ICER of €7385 per QALY. Sensitivity analyses confirmed cost-effectiveness in all scenarios. The introduction and implementation of HAART in Danish healthcare was cost-effective, and in some scenarios, even disruptive, i.e., led to both cheaper and more effective care of PLHIV.

Modeling the population health impact of incorporating a multi-cancer early detection (MCED) test to existing cancer screening among immunocompromised individuals

Objective: To assess the screening efficiency of an MCED test added to SoC screening, compared to SoC screening alone, among immunocompromised individuals, and to estimate the diagnostic workup costs associated with positive screening results. Methods: We estimated the potential impact of cancer screening among immunocompromised individuals aged 50-79 years within the University of Utah Health system who underwent a stem cell/solid organ transplant or were diagnosed with a primary or secondary immunodeficiency disorder between January 2000 and February 2018. We derived cancer incidence rates from the Huntsman Cancer Institute Tumor Registry, and screening performance of SoC screening and an MCED test from published literature. Outcomes of screening efficiency included the true-positive to false-positive (TP:FP) ratio, diagnostic yield (DY), and cancer detection rate (CDR) for SoC screening alone and an incremental MCED test. Scenario and probabilistic sensitivity analyses were conducted. Results: Among 4932 immunocompromised individuals aged 50-79 years, we estimated that 2595 tests would be done under SoC screening and assumed that all individuals received an additional MCED test. Adding an MCED test to SoC screening substantially improved screening efficiency (TP:FP 1:1, DY 5.15/1000 tests, CDR 42.0%), compared to SoC screening alone (TP:FP 1:99, DY 1.23/1000 tests, CDR 5.3%), assuming an MCED test with 100% uptake. Our findings were also robust to parameter uncertainty. Conclusion: Adding an MCED test to complement existing screening may be a highly efficient strategy to increase the detection of cancers among immunocompromised individuals. These results could help to improve cancer prevention and detection efforts among individuals with multiple cancer risk factors.

Cost-effectiveness analysis of budesonide/formoterol SMART therapy versus salmeterol/fluticasone plus as-needed SABA among patients ≥12 years with moderate asthma from the Chinese societal perspective

Objectives: To evaluate the cost-effectiveness of budesonide/formoterol reliever and maintenance therapy compared with salmeterol/fluticasone plus salbutamol as reliever therapy for asthma patients ≥12 years from the societal perspective in China. Methods: A Markov model was developed with three health states (non-exacerbation, exacerbation, and death) with a lifetime horizon. The exacerbation rates were obtained from a prospective cohort study conducted in Chinese asthma patients. Healthcare resources utilization data were estimated based on current clinical asthma management guidelines. Asthma-related mortality, cost input and utility values were derived from public database and literature. Model robustness was assessed with one-way sensitivity and probabilistic sensitivity analyses. Results: Compared with salmeterol/fluticasone plus salbutamol, budesonide/formoterol reliever and maintenance therapy led to fewer exacerbation events (13.6 vs. 15.9) and 0.0077 quality-adjusted life years (QALY) gain at an additional cost of ¥196.38 over lifetime. The base case incremental cost-effectiveness ratio (ICER) was ¥25,409.98 per QALY gained. The variables that had most impact on the model output included drug costs and medication adherence. At a willingness-to-pay threshold of ¥257,094/QALY (3 times of gross domestic product per capita in China in 2022), the probability of budesonide/formoterol maintenance and reliever therapy being cost-effective versus salmeterol/fluticasone plus as-needed salbutamol was 83.00%. Conclusion: From the societal perspective, budesonide/formoterol reliever and maintenance therapy is likely to be a cost-effective option compared with salmeterol/fluticasone plus as-needed salbutamol for Chinese asthma patients ≥12 years.

Methods to Quantify the Importance of Parameters for Model Updating and Distributional Adaptation.

Decision models are time-consuming to develop; therefore, adapting previously developed models for new purposes may be advantageous. We provide methods to prioritize efforts to 1) update parameter values in existing models and 2) adapt existing models for distributional cost-effectiveness analysis (DCEA). Methods exist to assess the influence of different input parameters on the results of a decision models, including value of information (VOI) and 1-way sensitivity analysis (OWSA). We apply 1) VOI to prioritize searches for additional information to update parameter values and 2) OWSA to prioritize searches for parameters that may vary by socioeconomic characteristics. We highlight the assumptions required and propose metrics that quantify the extent to which parameters in a model have been updated or adapted. We provide R code to quickly carry out the analysis given inputs from a probabilistic sensitivity analysis (PSA) and demonstrate our methods using an oncology case study. In our case study, updating 2 of 21 probabilistic model parameters addressed 71.5% of the total VOI and updating 3 addressed approximately 100% of the uncertainty. Our proposed approach suggests that these are the 3 parameters that should be prioritized. For model adaptation for DCEA, 46.3% of the total OWSA variation came from a single parameter, while the top 10 input parameters were found to account for more than 95% of the total variation, suggesting efforts should be aimed toward these. These methods offer a systematic approach to guide research efforts in updating models with new data or adapting models to undertake DCEA. The case study demonstrated only very small gains from updating more than 3 parameters or adapting more than 10 parameters. It can require considerable analyst time to search for evidence to update a model or to adapt a model to take account of equity concerns.In this article, we provide a quantitative method to prioritze parameters to 1) update existing models to reflect potential new evidence and 2) adapt existing models to estimate distributional outcomes.We define metrics that quantify the extent to which the parameters in a model have been updated or adapted.We provide R code that can quickly rank parameter importance and calculate quality metrics using only the results of a standard probabilistic sensitivity analysis.

Cost-effectiveness analysis of a digital diabetes-prevention programme versus an in-person diabetes-prevention programme in people with prediabetes in the United States.

To assess the cost-effectiveness of a digital diabetes prevention programme (d-DPP) compared with a diabetes prevention programme (DPP) for preventing type 2 diabetes (T2D) in individuals with prediabetes in the United States. A Markov cohort model was constructed, simulating a 10-year period starting at the age of 45 years, with a societal and healthcare sector perspective. The effectiveness of the d-DPP intervention was evaluated using a meta-analysis, with that of the DPP as the comparator. The initial cycle represented the treatment period, and transition probabilities for the post-treatment period were derived from a long-term lifestyle intervention meta-analysis. The onset of T2D complications was estimated using microsimulation. Quality-adjusted life years (QALYs) were calculated based on health utility measured by short form (SF)-12 scores, and a willingness-to-pay threshold of $100 000 per QALY gained was applied. The d-DPP intervention resulted in cost savings of $3,672 from a societal perspective and $2,990 from a healthcare sector perspectiveand a gain of 0.08 QALYs compared with the DPP. The dropout rate was identified as a significant factor influencing the results. Probabilistic sensitivity analysis showed that the d-DPP intervention was preferred in 85.8% in the societal perspective and 85.2% in the healthcare sector perspective. The d-DPP is a cost-effective alternative to in-person lifestyle interventions for preventing the development of T2D among individuals with prediabetes in the United States.

Cost-Utility Analysis of Bilateral Cochlear Implants for Children With Severe-to-Profound Sensorineural Hearing Loss in Taiwan.

Cochlear implants are an option for children with sensorineural hearing loss who do not benefit from hearing aids. Although bilateral cochlear implantation (CI) has been shown to enhance hearing performance and quality of life, its cost-effectiveness remains unclear. This study aimed to evaluate the cost-effectiveness of bilateral CI compared with bimodal hearing for children with sensorineural hearing loss in Taiwan from both the perspectives of patients and Taiwan's National Health Insurance Administration (TNHIA). A four-state Markov model was utilized in the study, including "use the first internal device," "use the second internal device," "use the third internal device," and "death." Health utility values were obtained from a local survey of health professionals and then adjusted by a scale to reflect both the negative impact of aging on hearing and the time needed to develop the full benefit of treatment in the earliest years of life. The cost data were derived from a caregiver survey, hospital databases, clinical experts, and the TNHIA. The incremental cost-effectiveness ratio (ICER) was calculated over the lifetime horizon and presented as cost per quality-adjusted life year (QALY) to evaluate the cost-effectiveness of simultaneous bilateral CI, sequential bilateral CI, and bimodal hearing. In addition, one-way sensitivity analyses and probabilistic sensitivity analyses were conducted to investigate the impact of uncertainty and the robustness of the model. The base-case analysis showed that children with bilateral CI gained more QALYs while incurring more costs when compared with those with bimodal hearing. From the TNHIA perspective, compared with bimodal hearing, the ICER of simultaneous bilateral CI was New Taiwan Dollars 232,662 per QALY whereas from the patient perspective, the ICER was New Taiwan Dollars 1,006,965 per QALY. Moreover, simultaneous bilateral CI dominated sequential bilateral CI from both perspectives. Compared with bimodal hearing, the ICER of sequential bilateral CI did not exceed twice the gross domestic product per capita in Taiwan from either perspective. One-way sensitivity analysis demonstrated that the utility gain of bilateral CI compared with bimodal hearing was the most impactful parameter from both perspectives. Probabilistic sensitivity analysis confirmed the robustness of the base-case analysis results. Our findings reveal that bilateral CI was cost-effective when using the threshold of one to three times the 2022 gross domestic product per capita in Taiwan from both the TNHIA and patient perspectives. Future research incorporating cost and effectiveness data from other dimensions is needed to help decision-makers assess the cost-effectiveness of bilateral CI more comprehensively.

Optimal malarial screening strategy in Australian blood donors: A cost-effectiveness analysis.

The risk of transfusion-transmitted malaria (TTM) infections is extremely low in Australia, and the cost-effectiveness of the current screening strategy has not been assessed. This study aims to conduct a cost-effectiveness analysis of different malaria screening strategies in blood donors as part of the risk-based decision-making framework. A decision tree model was developed to assess the cost-effectiveness of five alternative malaria screening strategies from a healthcare sector perspective. Screening strategies combining total or partial removal of malaria testing with different deferral periods were considered. The probabilities of developing severe and uncomplicated TTM were based on a literature review of cases in non-endemic areas since 2000. The health outcomes were quantified using disability-adjusted life years. The costs of non-returning donors due to deferral were also included. Deterministic and probabilistic sensitivity analyses were conducted to account for data uncertainty. The residual risks for all strategies were so low that the costs, mortality and morbidity associated with TTM are almost negligible. The overall costs were predominantly influenced by the costs of non-returning blood donors. As a result, removal of malaria testing and applying a 28-day deferral for at-risk donors were the least costly and most cost-effective of all the options considered. The current screening strategy for malaria in blood donors in Australia is not an efficient use of healthcare resources. Partial or total removal of malaria testing would bring significant cost savings without significantly compromising blood safety.

Cost-effectiveness of Pembrolizumab in Combination with Chemotherapy as Neoadjuvant Treatment and Continued as a Single Agent Adjuvant Treatment for High-Risk Early-Stage Triple-Negative Breast Cancer in Hong Kong.

The phaseIII randomized KEYNOTE-522 trial demonstrated that pembrolizumab in combination with chemotherapy as neoadjuvant treatment followed by adjuvant pembrolizumab (pembrolizumab + chemotherapy) provided significant improvements in event-free survival (EFS) and overall survival (OS) for patients with high-risk early-stage triple-negative breast cancer (eTNBC). The objective was to assess the cost-effectiveness of pembrolizumab + chemotherapy compared to neoadjuvant chemotherapy alone (chemotherapy) in patients with high-risk eTNBC from a Hong Kong third-party payer perspective. A multistate transition model with four health states (event-free), locoregional recurrence, distant metastases, and death) was developed to assess the lifetime medical costs and health outcomes (3% annual discount), along with incremental cost-effectiveness ratios (ICERs) using efficacy and safety data from the KEYNOTE-522 trial. The health state utilities were derived from KEYNOTE-522 Euro-QoL-five-dimension five-level questionnaire (EQ-5D-5L) data. Costs were expressed in 2022 Hong Kong dollars (HKD).Scenario and sensitivity analyses were performed to assess the robustness of results. Over a 32-year time horizon, base case results showed that pembrolizumab + chemotherapy was associated with a 3.42year longer EFS and expected gains of 3.05 lifeyears (LYs) and 2.45 quality-adjusted lifeyears (QALYs) compared to chemotherapy. The resultant ICERs were HKD135,200 per QALY gained and HKD108,463 per LY gained, which were lower than the World Health Organization (WHO) cost-effectiveness threshold of three times gross domestic product (GDP) per capita for Hong Kong of HKD1,171,308 per QALY. The one-way sensitivity analyses (OWSA) and probabilistic sensitivity analysis (PSA) showed the results were robust across various inputs and alternative scenarios. On the basis of the analysis conducted for a 56-year-old cohort with high-risk eTNBC and assumptions in the model, pembrolizumab + chemotherapy represents a cost-effective proposition (as the ICER is approximately 35% of the GDP per capita in Hong Kong) for patients with high-risk eTNBC in Hong Kong.

Cost-Effectiveness Analysis of Hepatic Arterial Chemotherapy for Advanced Hepatocellular Carcinoma in China: A Comparative Analysis of HAIC-FO and Sorafenib.

BACKGROUND The FOHAIC-1 trial showed hepatic arterial infusion chemotherapy with infusional fluorouracil, leucovorin, and oxaliplatin (HAIC-FO) improved survival, compared with sorafenib, in patients with advanced hepatocellular carcinoma (HCC). The aim of this study was to conduct a cost-effectiveness comparison between HAIC-FO and sorafenib from the perspective of the Chinese healthcare system. MATERIAL AND METHODS The economic evaluation was conducted between July 2023 and February 2024, spanning a 10-year investment horizon. A Markov model was developed to perform a cost-effectiveness analysis of HAIC-FO vs sorafenib. Health states incorporated in the model comprised progression-free disease, progressed disease, and death. Transition probabilities were derived from data obtained from the FOHAIC-1 trial. Incremental cost-effectiveness ratio (ICER) was calculated to evaluate cost-effectiveness. Additionally, one-way and probabilistic sensitivity analyses assessed the model's robustness. RESULTS The HAIC-FO group accrued a total cost of $22,781, whereas the sorafenib group totaled $18,795. In terms of effectiveness, the HAIC-FO group achieved 1.06 quality-adjusted life years (QALYs), whereas the sorafenib group attained 0.65 QALYs. Compared with sorafenib, HAIC-FO yielded an additional 0.41 QALYs at a cost of additional $3,985, resulting in an incremental cost of $9,720 per QALY gained. The one-way sensitivity analysis revealed the final ICER remained below the willingness-to-pay (WTP) threshold of $30,492 per QALY, when considering parameter fluctuations. Additionally, probabilistic sensitivity analysis indicated a 99.8% probability that the ICER for HAIC-FO compared with sorafenib would fall below the WTP threshold. CONCLUSIONS Compared with sorafenib, HAIC-FO emerged as a cost-effective first-line treatment option for patients facing advanced HCC in China.