Public Health System Perspective Research Articles

Budget impact and cost-utility analysis of prophylactic emicizumab versus on-demand bypassing agents for adolescent severe haemophilia A patients with inhibitors in India

IntroductionSevere Haemophilia A patients with inhibitors are currently being treated with bypassing agents like activated prothrombin complex concentrates (aPCC) and recombinant factor VIIa. Emicizumab is a recombinant humanized monoclonal antibody, introduced to reduce the bleeding events, improve treatment adherence, and quality of life. However, cost-effectiveness and long-term sustainability of the intervention is not studied in a low middle income setting like India. AimThe primary objective of this study was to evaluate the cost-utility of Emicizumab compared to traditional bypassing agents in the treatment of severe haemophilia A patients with inhibitors in India. Secondary objective was to analyze the budgetary impact of introducing Emicizumab for this patient population from the perspective of public health system in India. MethodsMarkov model was created to compare the prophylactic emicizumab therapy against bypassing agents for a hypothetical cohort of 10-year-old adolescents in India. The time horizon was 10 years and model built based on health system perspective. Cost utility was expressed as costs per quality-adjusted life-years (QALYs) gained. All costs were expressed as 2021 US dollars. Probabilistic sensitivity analysis was performed to check the robustness of the estimates. ResultsProphylactic emicizumab was a cost saving intervention with negative Incremental Cost Utility Ratio (ICUR) against recombinant factor VIIa of −853,573 USD (INR -63,109,773), and negative ICUR of −211,675 USD (INR -15,650,403) against APCC. The estimated total budget for treating all the severe Haemophilia A patients with inhibitors in India was USD 59,042,000 (INR 4,365,329,312) for 10 years’ time horizon (per patient cost of USD 295,210 [INR 21,826,646.56]). ConclusionProphylactic emicizumab therapy is a cost saving intervention when compared to both the bypassing agents as it is less costly and more effective for severe Haemophilia A patients with inhibitors in India.

Examining the Relationship Between Screening for Postpartum Depression and Associated Child Health Service Utilization and Costs: A Study Using the All Our Families Cohort and Administrative Data

IntroductionDespite a recognized association between maternal postpartum depression (PPD) and adverse child health outcomes, evidence examining the relationship between PPD symptoms and associated child health service utilization and costs remains unclear. In addition, there is a paucity of evidence describing the relationship between early identification of maternal PPD and associated health service utilization and costs for children. This study aims to address this gap by describing the secondary associations of screening for maternal PPD and annual health service utilization and costs for children over their first five years of life.MethodsMothers and children enrolled in the prospective All Our Families cohort were linked to provincial administrative data in Alberta, Canada. Multivariable generalized linear models were used to estimate the average annual inpatient, outpatient, physician, and total health service utilization and costs from a public health system perspective for children of mothers screened high risk for PPD, low/moderate risk for PPD, or unscreened.ResultsTotal mean costs were greatest for children during their first year of life than other years. Those whose mothers were not screened had significantly lower costs compared to those whose mothers were screened low/moderate risk, despite equivalent health service utilization.DiscussionFindings from this study describe the secondary associations of screening for maternal PPD using a public health system perspective. More research is required to fully understand variations in health costs for children across maternal PPD screening categories.

Comparative effectiveness and cost-effectiveness of cardioprotective glucose-lowering therapies for type 2 diabetes in Brazil: a Bayesian network model

BackgroundThe escalating prevalence of type 2 diabetes (T2DM) poses an unparalleled economic catastrophe to developing countries. Cardiovascular diseases remain the primary source of costs among individuals with T2DM, incurring expenses for medications, hospitalizations, and surgical interventions. Compelling evidence suggests that the risk of cardiovascular outcomes can be reduced by three classes of glucose-lowering therapies (GLT), including SGLT2i, GLP-1A, and pioglitazone. However, an evidence-based and cost-effective protocol is still unavailable for many countries. The objective of the current study is to compare the effectiveness and cost-effectiveness of GLT in individuals with T2DM in Brazil.MethodsWe employed Bayesian Networks to calculate the incremental cost-effectiveness ratios (ICER), expressed in international dollars (Int$) per disease-adjusted life years [DALYs] averted. To determine the effectiveness of GLT, we conducted a systematic review with network meta-analysis (NMA) to provide insights for our model. Additionally, we obtained cardiovascular outcome incidence data from two real-world cohorts comprising 851 and 1337 patients in primary and secondary prevention, respectively. Our cost analysis took into account the perspective of the Brazilian public health system, and all values were converted to Int$.ResultsIn the NMA, SGLT2i [HR: 0.81 (95% CI 0.69–0.96)], GLP-1A [HR: 0.79 (95% CI 0.67–0.94)], and pioglitazone [HR: 0.73 (95% CI 0.59–0.91)] demonstrated reduced relative risks of non-fatal cardiovascular events. In the context of primary prevention, pioglitazone yielded 0.2339 DALYs averted, with an ICER of Int$7,082 (95% CI 4,521–10,770) per DALY averted when compared to standard care. SGLT2i and GLP-1A also increased effectiveness, resulting in 0.261 and 0.259 DALYs averted, respectively, but with higher ICERs of Int$12,061 (95% CI: 7,227–18,121) and Int$29,119 (95% CI: 23,811–35,367) per DALY averted. In the secondary prevention scenario, all three classes of treatments were deemed cost-effective at a maximum willingness-to-pay threshold of Int$26,700. Notably, pioglitazone consistently exhibited the highest probability of being cost-effective in both scenarios.ConclusionsIn Brazil, pioglitazone presented a higher probability of being cost-effective both in primary and secondary prevention, followed by SGLT2i and GLP-1A. Our findings support the use of cost-effectiveness models to build optimized and hierarchical therapeutic strategy in the management of T2DM.Trial registrationCRD42020194415.

Improved prognosis after cardiac resynchronization therapy over a decade.

The past decade has seen an increased delivery of cardiac resynchronization therapy (CRT) for patients with heart failure (HF). We explored whether clinical outcomes after CRT have changed from the perspective of an entire public healthcare system. A national database covering the population of England (56.3 million in 2019) was used to explore clinical outcomes after CRT from 2010 to 2019. A total of 64 698 consecutive patients (age 71.4 ± 11.7 years; 74.8% male) underwent CRT-defibrillation [n = 32 313 (49.7%)] or CRT-pacing [n = 32 655 (50.3%)] implantation. From 2010-2011 to 2018-2019, there was a 76% increase in CRT implantations. During the same period, the proportion of patients with hypertension (59.6-73.4%), diabetes (26.5-30.8%), and chronic kidney disease (8.62-22.5%) increased, as did the Charlson comorbidity index (CCI ≥ 3 from 20.0% to 25.1%) (all P < 0.001). Total mortality decreased at 30 days (1.43-1.09%) and 1 year (9.51-8.13%) after implantation (both P < 0.001). At 2 years, total mortality [hazard ratio (HR): 0.72; 95% confidence interval (CI) 0.69-0.76] and total mortality or HF hospitalization (HR: 0.59; 95% CI 0.57-0.62) decreased from 2010-2011 to 2018-2019, after correction for age, race, sex, device type (CRT-defibrillation or pacing), comorbidities (hypertension, diabetes, chronic kidney disease, and myocardial infarction), or the CCI (HR: 0.81; 95% CI 0.77-0.85). From the perspective of an entire public health system, survival has improved and HF hospitalizations have decreased after CRT implantation over the past decade. This prognostic improvement has occurred despite an increasing comorbidity burden.

EE15 Cost-Effectiveness Evaluation of Human Papilloma Virus Test in Comparison with Cytology in Uruguay from the Public Health System Perspective

To estimate the cost-effectiveness of the screening strategy with the molecular test with genotyping 16/18 compared to conventional cytology for the detection of human papilloma virus infection in Uruguay.

EE471 Cost-Effectiveness Evaluation of Human Papilloma Virus Test in Comparison with Cytology in Costa Rica from the Public Health System Perspective

To estimate the cost-effectiveness of the screening strategy with the molecular test with genotyping 16/18 compared to conventional cytology for the detection of human papilloma virus infection in Costa Rica.

EE36 Cost-Effectiveness Analysis of Taptest and Continuous Lumbar Drainage in Comparison to Standard Care in Suspected Idiopathic Normal Pressure Hydrocephalus from the Perspective of the Brazilian Unified Health System

Three symptoms — impaired gait, cognition, and incontinence — define idiopathic normal pressure hydrocephalus (iNPH). To predict whether iNPH patients will benefit from shunt surgery, the tap test (TT) or continuous lumbar drainage (CLD) may be employed. This study's goal is to evaluate the cost-effectiveness of TT and CLD in comparison to the standard of care, in which all patients with suspected iNPH underwent shunt surgery, from the perspective of the Brazilian Public Health System.

MT8 A Cost-Utility Analysis of Artificial Urinary Sphincter Versus BSC in Severe Male Postprostatectomy Incontinence - Brazilian Public Health System Perspective

Despite being considered the ''gold standard'' for the treatment of severe male postprostatectomy incontinence, the artificial urinary sphincter (AUS) is not incorporated and provided in the Brazilian public health system. The objective of the presented study was to evaluate the cost-utility of AUS in this perspective.

Cost savings and clinical outcomes in cancer patients enrolled in clinical trials from a national public health system perspective.

e18813 Background: Clinical research has led to great advances that have an impact on improving the survival and quality of life of cancer patients and should be part of the standard of care. However, there is no clear evidence on whether participation in clinical trials (CTs) is cost-efficient for the national health system. For this reason, in the present study I have tried to analyse the direct impact of participation in CTs in terms of improvement in clinical results and costs. Particularly, I have looked into CT subjects diagnosed with metastatic renal cell carcinoma (mRCC) or G1-G2 metastatic neuroendocrine tumors (mNET). In addition, the potential influence of different prognostic and demographic factors on these variables has also been evaluated. Methods: Retrospectively, clinical data of the patients related to their baseline characteristics and the efficacy of the different lines of treatment they had received for their tumor were collected, as well as the direct healthcare costs derived from the clinical approach of all patients diagnosed with mRCC or mNET and clinically followed by the Ramón y Cajal University Hospital Medical Oncology Service, in Madrid, Spain, between January 2010 and February 2017. Subsequently, a comparative statistical analysis was performed of the results obtained in patients included in a clinical trial versus those who never participated in one but instead received therapy according to the standard of care (SOC). Results: Of the 104 patients (65 mRCC and 39 mNET) who were eventually included, 39 participated in a clinical trial. Overall, the 39 CT patients presented a higher overall survival (OS) than SOC patients (52 vs. 15 months. HR: 0.41; 95% CI: [0.23; 0.71]; p &lt; 0.01), a finding that carried through in all subgroups, including age, sex and prognostic factors. Regarding healthcare costs, patients who participated in CT yielded a mean annual savings of €46,109.56 per patient (p &lt; 0.01) compared to patients treated according to SOC, mainly due to pharmacological savings (€27,406.42). Furthermore, the savings were generalized in all subgroups regardless of age, sex and prognostic factors. Additionally, CT patients required 75.81% fewer hospitalisations (p &lt; 0.01) with a lower associated mean cost (p = 0.02), and the mean annual cost corresponding to the use of the Emergency Service was lower (p &lt; 0.01). Finally, the unadjusted incremental cost-effectiveness ratio (ICER) was -€14,970.64 per year of life gained (YLG) and the adjusted ICER was -€42,656.11 per life saved. Conclusions: From the national health system perspective, the participation of patients in CTs is related to an improvement in OS and savings in clinical costs in patients diagnosed with mRCC and mNET. In short, the participation of cancer patients in CTs represents a cost-efficient therapeutic option.

Cost-Effectiveness Analysis of Monoclonal Antibodies Associated With Chemotherapy in First-Line Treatment of Metastatic Colorectal Cancer

ObjectivesThis study aimed to evaluate the cost-effectiveness of anti–epidermal growth factor receptor (cetuximab and panitumumab) or anti–vascular endothelial growth factor (bevacizumab) monoclonal antibodies associated with conventional chemotherapy (CT) (fluorouracil and leucovorin with irinotecan) as a first-line treatment for unresectable metastatic colorectal cancer. MethodsA partitioned survival analysis model was adopted to simulate direct health costs and benefits comparing therapeutic options in a 10 years’ time horizon. Model data were extracted from the literature and costs were obtained from Brazilian official government databases. The analysis considered the perspective of the Brazilian Public Health System; costs were measured in local currency (BRL) and benefits in quality-adjusted life-years (QALY). A 5% discount rate was applied to costs and benefits. Alternative willingness-to-pay scenarios, varying from 3 to 5 times the cost-effectiveness threshold established in Brazil, were estimated. The results were presented incremental cost-effectiveness ratio (ICER), and both deterministic and probabilistic sensitivity analyses were performed. ResultsThe most cost-effective choice would be the association of CT with panitumumab, with an ICER of $58 330.15/QALY compared with isolated CT. The second-best option was CT with bevacizumab and panitumumab, with an ICER of $71 195.40/QALY compared with panitumumab alone. Although having higher costs, the second-best option was the most effective. Both strategies were cost-effective in part of the Monte Carlo iterations, considering the 3× threshold. ConclusionsThe therapeutic option CT + panitumumab + bevacizumab represents the most significant effectiveness gain in our study. It is the second-lowest cost-effectiveness, and this option includes monoclonal antibodies association for patients with and without KRAS mutation.

Dengue control in the state of goias-brazil using “wmel wolbachia”: a cost-effectiveness study

Context: Dengue and other arboviruses have a considerable economic impact in Brazil. There are vector control strategies for dengue: traditional control with pesticides, Incompatible Insect Technique (IIT) with “wMel Wolbachia”, and Sterile Insect Technique (SIT). Objective: To analyze the cost-effectiveness ratio of the IIT/SIT strategy, compared to traditional vector control with pesticides, for dengue prevention from the perspective of the Brazilian Public Health System (BPHS) as the payer and from the societal perspective in the state of Goiás, Brazil.

The financial impact of neonatal sepsis on the Brazilian Unified Health System

ObjectiveTo evaluate the hospital cost of newborn infants diagnosed with sepsis from the perspective of the Brazilian Public Health System over 11 years. MethodCross-sectional study that analyzed secondary data from the databases of the Hospital Information System of the Brazilian Public Health System. Infants hospitalized between 0‒29 days after birth with a diagnosis of sepsis from 2008 to 2018 were included. The diagnosis used in the study was the one that the hospital considered the main diagnosis at admission. Costs were analyzed in US dollars and reflected the amount paid by the Brazilian Public Health System to the hospitals for the informed diagnosis upon admission. The costs were evaluated as the total per admission, and they were compared among Brazilian geographic regions, among etiologic agents, and between neonates with the diagnosis of sepsis that survived or died. ResultsFrom 2008 to 2018, 47,554 newborns were hospitalized with sepsis (148.04 cases per 100,000 live births), with an average cost of US$ 3345.59 per hospitalization, ranging from US$ 2970.60 in the North region to US$ 4305.03 in the Midwest. Among sepsis with identified agents, the highest mean cost was related to Gram-negative agents, and the lowest to Streptococcus agalactiae sepsis. Patients with sepsis who died had a higher cost than the survivors (t-test; p = 0.046). ConclusionsThe evaluation of costs related to neonatal sepsis in the country during an 11-year period shows the economic impact of morbidity that may be avoided by improving the quality of neonatal care.

EE329 Public Health and Economic Impact of a Gender-Neutral Quadrivalent Human Papillomavirus Vaccination Program in El Salvador

To assess the public health and economic impact of adding males to the existing 9-10-year-old female-only vaccination (FOV) with two-dose schedule of quadrivalent HPV vaccine (4vHPV) from the El Salvador public health system perspective.

EE335 Cost-Efectiveness Analysis Between Multichambered Standarized Pediatric Parental Nutrition and Individualized Compounded Parental Nutrition, Analysis From the Mexican Public Health System Perspective

To determine, from the Mexican public health system, if the use of a pediatric standardized parental nutrition is cost-effective vs current use of individualized compounded parental nutrition.

Abstract 14830: Comparative Cost-Effectiveness of Cardioprotective Glucose-Lowering Therapies for Type 2 Diabetes in a Middle-Income Country: A Bayesian Network Model

Introduction: Unprecedented economic and social burden has expanded with the steady increase in Type 2 Diabetes (T2D) prevalence, particularly in low- and middle-income countries. Hypothesis: We hypothesize that novel anti-diabetic therapies such as SGLT2i or GLP1-RA would figure as more cost-effective among T2D subjects. Methods: Electronic databases were searched between August and April 2021. Inclusion criteria were: randomized clinical trials with T2D; articles published in English; double-blind, phase 3 or 4 trials; ≥100 patients per arm; follow-up &gt;24 weeks; and the pre-specified endpoints: death from any cause, major adverse cardiovascular events, and hospitalization for heart failure. Two prospective cohorts of T2D patients were used to feed the Markov Influence Diagram transition probabilities. Bayesian Networks and Markov Influence Diagram were used to estimate the incremental cost-effectiveness ratios (ICER) per disease-adjusted life years [DALYs] averted. The perspective of the Brazilian public health system was considered for costs and values were converted to international dollars (Int$). Results: We selected a total of 157 trials generating a sample size of 684,389 patient-years. Modeling was based on a primary and secondary prevention cohort of 851 and 1,337 patients, respectively. SGLT2i [HR:0.81 (95%CI: 0.69; 0.96)], GLP-1RA [HR:0.79 (95%CI: 0.67; 0.94)], and pioglitazone [HR:0.73 (95%CI: 0.59; 0.91)] reduced the relative risk of non-fatal cardiovascular events. In primary prevention, pioglitazone resulted in 0.2339 DALYs averted, with ICER of Int$7,082 (95%CI: 4,521; 10,770) per DALY averted, when compared to standard care. SGLT2i and GLP-1RA also increased the effectiveness (0.261 and 0.259 DALYs averted, respectively) but with higher ICERs [Int$12,061 (95%CI: 7,227; 18,121) and Int$29,119 (95% CI:23,811; 35,367) per DALY averted, respectively]. In secondary prevention, the three classes were cost-effective at a maximum willingness to pay threshold of Int$26,700. In both scenarios, however, pioglitazone had the highest probability of being cost-effective. Conclusions: In a middle-income country, pioglitazone presented a higher probability of being cost-effective followed by SGLT2i and then GLP-1RA.

EE243 Economic Evaluation of Lidocaine Patches for the Treatment of Localized Neuropathic Pain from Mexican Public Health System Perspective

EE243 Economic Evaluation of Lidocaine Patches for the Treatment of Localized Neuropathic Pain from Mexican Public Health System Perspective

Cost-Utility of the CDK 4/6 Inhibitors for Postmenopausal Women With Luminal Advanced Breast Cancer in Brazil

Several trials have demonstrated the benefit of the CDK 4/6 inhibitors for postmenopausal women with luminal advanced breast cancer. This research aims to compare the cost-utility of the CDK 4/6 inhibitors in patients with no history of resistance to endocrine therapy. A Markov model was constructed to estimate the incremental cost per quality-adjusted life-years (QALYs) of treatments from the Brazilian public health system perspective over a lifetime horizon (30 years) with 5% annual discount rate for both benefits and costs. Efficacy parameters were extracted from the pivotal studies. Costs were based on open data from the Brazilian Ministry of Health. The utilities were calculated according to the overall population preferences from a British study. Deterministic and probabilistic sensitivity analyses evaluated the robustness of the results. The most cost-effective drug was ribociclib (US$50 748/QALY), followed by abemaciclib (US$64 052/QALY) and palbociclib (US$65 289/QALY). The univariate analysis showed that the incremental cost-utility ratio (ICUR) was mainly sensitive to the overall survival hazard ratio. The one thousand-probabilistic simulation showed that all ICUR values were above classical thresholds such as 1 to 3 gross domestic product (GDP) per capita per QALY. Even though there is no established willingness to pay threshold in Brazil, the estimated ICUR for CDK 4/6 inhibitors is >6 times the Brazilian GDP per capita (GDP per capita= US$5694.73), which might be a barrier to their inclusion in the Brazilian public health system.

Real-life quantitative G6PD screening in Plasmodium vivax patients in the Brazilian Amazon: A cost-effectiveness analysis.

BackgroundAs quantitative glucose 6-phosphate dehydrogenase deficiency (G6PDd) screening tools are evaluated in operational studies, questions remain as to whether they are cost-effective. Here, a cost-effectiveness analysis (CEA) was performed to estimate the Incremental Cost-effectiveness Ratio (ICER) of the introduction of quantitative screening test to detect G6PDd among P. vivax carriers in two municipalities in the Brazilian Amazon.Methodology/Principal findingsThis cost-effectiveness analysis evaluated the use of the Standard G6PD quantitative screening test in vivax malaria treatment units in two municipalities of the Brazilian Amazon. Using the perspective of the Brazilian public health system, the analysis was performed for the outcome ‘PQ-associated hospitalization avoided’, based on a decision tree model. The results indicated that the G6PDd screening strategy compared with the routine strategy was highly cost-effective, with an ICER of US$495 per additional hospitalization avoided, which represented less than 8% of one Brazilian gross domestic product per capita (US$6,822). The uncertainties evaluated in the sensitivity analysis did not significantly affect the ICER identified in the base-case.Conclusions/SignificanceThis cost-effectiveness analysis showed the quantitative G6PD testing was effective in avoiding PQ-associated hospitalizations. The incorporation of G6PD screening is of paramount importance towards P. vivax malaria elimination in the Amazon to promote the safe use of primaquine and tafenoquine.

Cost Utility of Vaccination Against COVID-19 in Brazil

ObjectivesThe objective of this study was to evaluate the cost-utility of the Oxford, CoronaVac, and Janssen COVID-19 vaccines from the perspective of the Brazilian public health system. MethodsA total of 3 microsimulation models were constructed with individual data to evaluate the 3 vaccines. The simulation contains 7 transition states that are related to the natural history of the disease. The model with a daily cycle has a time horizon of 1 year and uses data from 289 days of the pandemic. The analysis was conducted from the perspective of the Brazilian public health system considering direct medical costs. For the model inputs, outpatient and hospital databases were used with information on treated patients stratified by age. Information on mortality was also stratified based on patients' age in the mortality database (SIM). The efficacy of vaccines to reduce the likelihood of patients becoming ill was evaluated independently for each vaccine. Information on the quality of life of patients in outpatient or hospital treatment and the sequelae resulting from the disease were extracted from the literature. The main outcome of the analysis was quality-adjusted life-years (QALYs). ResultsThe vaccines showed incremental cost-utility ratios ranging from R$−23 161.3/QALY (Oxford) to R$17 757.85/QALY (CoronaVac). The older the population, the lower was the incremental cost-utility ratio. Given a willingness-to-pay threshold of R$17 586/QALY, all the vaccines were considered cost-effective in the probabilistic sensitivity analysis. ConclusionsThe results of the analysis by age group can help in the preparation of a vaccination prioritization plan.

Cost-effectiveness analysis: fluticasone furoate/umeclidinium/vilanterol for the treatment of moderate to severe chronic obstructive pulmonary disease from the perspective of the Chilean public health system

ABSTRACT Background Chronic obstructive pulmonary disease (COPD) is an inflammatory lung disease characterized by long-term breathing problems and airflow limitations. International guidelines recommend using bronchodilators like long-acting beta- and muscarinic antagonists, and inhalational corticosteroids. Objectives The cost-effectiveness of single-inhaler triple therapy containing fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI) was compared to the treatments Fluticasone Furoate/Vilanterol (FF/VI), Umeclidinio/Vilanterol (UMEC/VI) and Fluticasone Propionate 250 mcg/Salmeterol 25mcg + Tiotropio 18 mcg (FP/SAL/TIO) for patients with COPD from the Chilean public health system perspective. Methods A cost-effectiveness analysis was performed, including a deterministic and probabilistic sensitivity analysis over a 25-year time horizon. Two scenarios were assessed to study the effect of a 3%-discount for costs and outcomes on FF/UMEC/VI. Results The incremental cost-effectiveness (ICER) of FF/UMEC/VI versus FF/VI was $10,076/QALY, being a cost-effective alternative to a threshold of one Gross Domestic Product per capita (GDPpc), while versus FP/SAL/TIO the ICER increased to $50,288/QALY, showing to be a non-cost effective alternative to 1 GDPpc, but at a threshold of 3 GDPpc. Conclusion FF/UMEC/VI appears to be a cost-effective intervention for treating COPD compared to FF/VI. However, FF/UMEC/VI compared to FP/SAL/TIO showed an ICER above the threshold of 1 GDPpc, but, in comparison with lower price, the ICER was below 3 GDPpc.