Budget Impact Research Articles

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.

Budget Impact Analysis for Empagliflozin in Patients with Nondiabetic CKD

Budget Impact Analysis for Empagliflozin in Patients with Nondiabetic CKD

Letter to the Editor: “Cost‐effectiveness and budget impact analysis of the implementation of differentiated service delivery models for HIV treatment in Mozambique: a modelling study”: Resource reductions are not equal to cost savings

Letter to the Editor: “Cost‐effectiveness and budget impact analysis of the implementation of differentiated service delivery models for HIV treatment in Mozambique: a modelling study”: Resource reductions are not equal to cost savings

Budget impact analysis of high-priced orphan medicinal products intended for the treatment of rare diseases in China: evidence from a densely populated metropolis of Chengdu

BackgroundAn increasing number of orphan medicinal products (OMPs) are being included in social health insurance schemes, significantly improving access to medicines for patients with rare diseases. However, high-priced OMPs are still not covered, primarily due to health equity controversies and inadequate data systems required for economic evaluation. The aim of this study was to estimate the burden of drug expenditures and the size of the reimbursement budget required for high-priced OMPs from the perspectives of society and healthcare payers.MethodsThe study performed a budget impact analysis using data from multiple sources to estimate the reimbursement budget for high-priced OMPs in Chengdu, a densely populated metropolis in China. The budget analysis consisted of three main elements: the number of patients, the price of drugs, and the simulated policy scenario. By adjusting the combinations of these elements, the budget fluctuations for payers were estimated. Furthermore, the study predicted the budget trend for the next three years to validate its sustainability.ResultsThe analysis indicated that 98 rare disease patients in Chengdu required high-priced OMPs in 2019. This suggested a projected budget of CNY 179 million for these patients without reimbursement policies, from a societal perspective. Under six assumed policy scenarios, this budget ranged from CNY 32 million to CNY 156 million. Over the next three years, the annual budget was estimated to range from CNY 200 million to CNY 1.303 billion.ConclusionIntegration of multi-source data helps to obtain more scientifically reliable results on budget impacts. The study found that the budgetary impacts of high-priced OMPs on society and payers are relatively limited. Health policymakers can choose appropriate reimbursement strategies based on financial affordability among a diverse mix of elements. The results of related studies provided insights for optimizing the allocation of health resources and improving patient access to medications.

The Impact of Islamic Finance Development, Economic Growth, Function of Regional Government Budgets on Carbon Emissions in Indonesia

Carbon emissions are a pressing global issue, with international agreements such as the Kyoto Protocol, Paris Agreement, and COP26 aiming to limit temperature rise and achieve net zero emissions. This research investigates the impact of Islamic financial development, economic growth, and government budget functions on carbon emissions in Indonesia, using panel data from 2018 to 2022. The study reveals that Islamic finance, specifically Sharia banking financing, significantly reduces carbon emissions. Conversely, economic growth (GRDP) is positively associated with carbon emissions, while the government’s environmental budget shows a negative relationship but lacks statistical significance. These findings highlight the effectiveness of Islamic finance in promoting environmentally sustainable practices and underscore the need for more effective government budgeting and regulatory measures. Future research should explore a broader range of Islamic finance sectors and budgetary functions to provide a more comprehensive understanding of their impact on carbon emissions. This study’s insights can guide policymakers in reinforcing successful strategies and developing new approaches to achieve emission reduction goals.

Economic effects of next-generation sequencing diagnostics in unspecific sepsis patients – a budget impact analysis from the healthcare providers’ perspective in Germany

PurposeNext-generation sequencing (NGS) tools have clinical advantages over blood culture but are more expensive. This study assesses the budget impact and break-even point of NGS testing costs from a healthcare provider’s perspective in Germany.MethodsThe budget impact was calculated based on aggregated data of German post-operative surgery cases. Simulated cost savings were calculated based on a simulated reduction in hospital length of stay (LOS) of four or eight days with a positivity rate of 71% and compared to the costs of one (scenario A) or two tests (scenario B) per case. Furthermore, the break-even point of the cost of two tests compared to saved costs through shortened LOS was conducted.ResultsFor 9,450 cases, an average budget impact for scenario A and scenario B of €1,290.41 [95% CI €1,119.64 – €1,461.19] and - €208.59 [95% CI - €379.36 – - €37.81] was identified for gastrointestinal and kidney surgery cases, and €1,355.58 [95% CI €1,049.62 – €1,661.55] and €18.72 [95% CI - €324.69 – €287.24] for vascular artery surgery cases, respectively. The break-even analysis showed that using two tests per case could achieve a minimum positive contribution margin with an average of 1.9 tests per case across the study population.ConclusionThe results revealed a positive budget impact for one NGS test and a slightly negative budget impact for two NGS tests per case. Findings suggest that largest cost savings are generated for more severe cases and are highly dependent on the patient population.

Authors’ reply: Inadequate analysis of the budgetary impact of new label indications after initial drug registration in Brazil: A response to a published case study

Authors’ reply: Inadequate analysis of the budgetary impact of new label indications after initial drug registration in Brazil: A response to a published case study

Cost-Utility and Budget Impact Analysis of Adding SGLT-2 Inhibitors to Standard Treatment in Type 2 Diabetes Patients with Heart Failure: Utilizing National Database Insights from Thailand

Cost-Utility and Budget Impact Analysis of Adding SGLT-2 Inhibitors to Standard Treatment in Type 2 Diabetes Patients with Heart Failure: Utilizing National Database Insights from Thailand

Health impact, budget impact, and price threshold for cost-effectiveness of lenacapavir for HIV pre-exposure prophylaxis in eastern and southern Africa: a modelling analysis

Injectable lenacapavir administered every 6 months is a promising product for HIV pre-exposure prophylaxis (PrEP). We aimed to estimate the health and budget impacts and threshold price at which lenacapavir could be cost-effective in eastern and southern Africa. We adapted an agent-based network model, EMOD-HIV, to simulate lenacapavir scale-up in Zimbabwe, South Africa, and western Kenya from 2026 to 2035. Uptake assumptions were informed by a literature review of PrEP product preferences. In the main analysis, we varied lenacapavir coverage by subgroup: female sex workers (40% coverage); male clients of female sex workers (40%); adolescent girls and young women aged 15-24 years with more than one sexual partner (32%); women aged 25 years and older with more than one sexual partner (36%); and males with more than one sexual partner (32%). We also assessed a higher coverage scenario (64-76% across subgroups) and scenarios of expanding lenacapavir use, varying from concentrated among those at highest HIV risk to broader coverage including those at medium HIV risk. We estimated the maximum per-dose lenacapavir price that achieved cost-effectiveness (<US$500 per disability-adjusted life-year averted), infections averted, and 5-year budget impact, compared with daily oral PrEP only. In the main analysis, lenacapavir was projected to achieve from 1·6% (95% uncertainty interval [UI] 1·5-1·8) to 4·0% (3·4-5·1) population coverage across settings and to avert from 12·3% (5·4-19·5) to 18·0% (11·0-22·9) of infections over 10 years. The maximum price per dose was highest in South Africa ($106·28 [95% UI 95·72-115·87]), followed by Zimbabwe ($21·15 [17·70-24·89]), and lowest in western Kenya ($16·58 [15·44-17·70]). The 5-year budget impact was US$507·25 million (95% UI 436·14-585·42) in South Africa, $16·80 million (13·95-22·64) in Zimbabwe, and $4·09 million (3·86-4·30) in western Kenya. In the higher coverage scenario, lenacapavir distribution was projected to reach from 3·2% (95% UI 2·9-3·6) to 8·1% (6·8-10·5) population coverage and to avert from 21·2% (95% UI 14·7-18·5) to 33·3% (28·5-36·9) of HIV infections across settings over 10 years. Price thresholds were lower than in the main analysis: $88·34 (95% UI 83·02-94·19) in South Africa, $17·71 (15·61-20·05) in Zimbabwe, and $14·78 (14·33-15·30) in western Kenya. The 5-year budget impact was higher than the main analysis: $835·29 million (95% UI 736·98-962·98) in South Africa, $29·50 million (24·62-39·52) in Zimbabwe, and $7·45 million (7·11-7·85) in western Kenya. Expanding lenacapavir coverage resulted in higher HIV infections averted but lower price thresholds than scenarios of concentrated use among those with highest HIV risk. Our findings suggest that lenacapavir could avert substantial HIV incidence and that price thresholds and budget impacts vary by setting and coverage. These results could inform policy deliberations regarding lenacapavir pricing and resource planning. The Bill & Melinda Gates Foundation.

Implementation of collaborative care for depression in VA HIV clinics: Translating Initiatives for Depression into Effective Solutions (HITIDES): protocol for a cluster-randomized type 3 hybrid effectiveness-implementation trial

BackgroundDepression is the most diagnosed mental health condition among people living with HIV. Collaborative care is an effective intervention for depression, typically delivered in primary care settings. The HIV Translating Initiatives for Depression into Effective Solutions (HITIDES) clinical intervention involves a depression care team housed off-site that supports depression care delivery by HIV care providers. In a randomized controlled trial, HITIDES significantly improved depression symptoms for veterans living with HIV and delivered cost savings. However, no HIV clinics in the Veterans Health Administration (VHA) have implemented HITIDES; as such, it is unclear what implementation strategies are necessary to launch and sustain this intervention.MethodsThis hybrid type-3 effectiveness-implementation trial examines the implementation and effectiveness of HITIDES in 8 VHA HIV clinics randomly assigned to one of two implementation arms. Each arm uses a different implementation strategy package. Arm 1 includes an intervention operations guide; an on-site clinical champion who, with the help of a peer community of practice, will work with local clinicians and leadership to implement HITIDES at their site; and patient engagement in implementation tools. Arm 2 includes all strategies from Arm 1 with assistance from an external facilitator. The primary implementation outcomes is reach; secondary outcomes include adoption, implementation dose, depressive symptoms, and suicidal ideation. We will conduct a budget impact analysis of the implementation strategy packages. We hypothesize that Arm 2 will be associated with greater reach and adoption and that Arm 1 will be less costly.DiscussionPreliminary work identified implementation strategies acceptable to veterans living with HIV and HIV care providers; however, the effectiveness and cost of these strategies are unknown. While the depression care team can deliver services consistently with high quality, the ability of the depression care team to engage with HIV care providers at sites is unknown. Findings from this study will be used to inform selection of implementation strategies for a broad rollout to enhance depression and suicide care for people living with HIV.Trial registrationClinicalTrials.gov ID: NCT05901272, Registered 10 May 2023, https://clinicaltrials.gov/study/NCT05901272

A Analysis Of Central Government Ministry Pro-Poor Budgeting Program Before And After The Pandemic

Pro-poor budgeting analysis is an approach used to ensure that government budget allocations favor and favor the interests of the poor and vulnerable. This study uses a qualitative approach to determine the differences in pro-poor budgeting budget allocations in the Central Government. The data collection techniques used in this study are using secondary data from the Central Government Financial Statements for the 2019 and 2022 fiscal years. The data analysis technique in this study uses descriptive analysis. This analysis is important to see budget allocations that are right on target and effective in reducing poverty levels in Indonesia. The results of this study indicate that the three Central Governments have differences in pro-poor budgeting priorities between the pre-COVID-19 period and the post-COVID-19 period, and other results show differences in the effectiveness of pro-poor budgeting between Ministries. The implications of this research are related to the impact of pro-poor budgeting and can be used as a basis for more effective policy-making related to pro-poor budgeting in the Central Government. This research has significant benefits as a literature reference for students exploring related fields. This study can be used as an important reference by providing secondary data and in-depth analysis. In addition, the results of this study open up opportunities for further development by future researchers. Thus, this study enriches the academic literature and triggers future innovation and continuous research, ultimately contributing to the development of science and practical application in related fields.

Government emergency budgeting and disaster impact: Evidence from China

AbstractEmergency budgets have become indispensable fiscal tools for governments around the world to cope with disasters, but how governments determine the size of emergency budgets and to what extent such budgets are effective remain unclear. Drawing on organizational learning theory and using data collected from 30 provinces in China, we conduct a series of panel regression analysis and robustness checks to investigate the determinants and impact of emergency budgets. We find that both prior disaster experience and government fiscal ability as measured by budget surplus are positively related to the size of subnational governments' emergency budgets, but the interaction effect between the two is negative. Moreover, while emergency budgets can help maintain the level of government budget surplus, they only play a limited role in moderating the negative impact of disaster damage on governments' fiscal ability. In particular, this study reveals the importance of socioeconomic environment and organizational capacity for the decision‐making process and outcomes of emergency budgets, suggesting that subnational governments should always adopt a holistic approach and improve their financial preparedness for future emergencies by incorporating more information from past disasters and considering diverse drivers of the complex dynamics among emergency budgets, disaster experience, and government fiscal ability.

Abstract B018: UroPanc: A large prospective observational study for validation of urinary biomarker test for the earlier detection of pancreatic adenocarcinoma

Abstract Introduction. A bleak prognosis of Pancreatic ductal adenocarcinoma (PDAC) is largely due to late diagnosis and highly accurate, non-invasive diagnostic tests are urgently needed to improve patients’ survival. Our group has previously reported on urinary biomarker panel, comprising of LYVE1, REG1B and TFF1 (1-2), which distinguished controls from PDAC stages I-II with AUC 0.936 and sensitivity/specificity (SN/SP)&amp;gt;85%. We constructed the logistic regression based algorithm for interpretation of the data, PancRISK (3), which enables the stratification of high- risk patients into those with ‘average’ or ‘elevated’ risk of developing PDAC. Recently, we showed that our panel combined with CA19-9 can detect PDAC up to 2 years before clinical diagnosis (4). Aim. Here, we report on the design and interim analysis of the UroPanc Trial (ClinicalTrials.gov NCT04449406), which aims to evaluate the accuracy of the urinary biomarker panel and the affiliated PancRISK. We plan to recruit two cohorts: the first one comprises asymptomatic individuals that have high risk of developing PDAC due to familial history and genetic syndroms - these will be recruited through the EUropean Registry Of familial PAncreatic Cancer and hereditary pancreatitis (EUROPAC) (PI: Prof W Greenhalf and team, Liverpool, UK). The second cohort comprises patients with symptoms suggestive of PDAC – such patients are recruited at gastrointestinal clinics at University College London Hospitals (UCLH, PI: Prof S Pereira and team), and The Royal London Hospital (PI: Dr P Wilson).Methods. The urinary proteins are assayed by commercially available ELISAs. Plasma CA19-9 is measured at The Doctors Laboratory in London, UK using Cobas601E, Roche platform. The statistical analysis and accuracy of the test is measured by c-statistics, SN/SP and positive/negative predictive value. The results are compared to imaging and histopathology records (where available). Human factor and budget impact analysis of the future inclusion of the urine test into the diagnostic and surveillance pathways for PDAC patients is ongoing (PI: Dr M Z Ni and the team, Imperial College London, UK). Results and conclusions. At present, 1067 patients have been recruited, and urine samples from 825 patients have been assayed. An interim analysis has shown promising results with SP of 91% and SN of 86%, and accuracy of cancer detection of 90%. Combined with the health economic analysis, we currently work towards the future implementation of this test into clinical practice, with the potential to significantly improve the current diagnostic pathway for individuals at risk of pancreatic cancer. 1. Radon, TP, et al. Clin Cancer Res, 2015; 21:3512-21. 2. Debernardi, S, et al. PLOSMed, 17(12); e1003489. 3. Blyuss, O, et al. Br J Cancer, 2020; 122:692-6. 4. Debernardi, S, et al. Int J Cancer, 2023; 152:769–80. Citation Format: Silvana Debernardi, Evelyn Kurotova, Nurshad Ali, Mariam Mahamood, Ismita Chhetri, Steve Pereira, Patrick Wilson, Bill Greenhalf, Melody Z Ni, Oleg Blyuss, Tatjana Crnogorac-Jurcevic. UroPanc: A large prospective observational study for validation of urinary biomarker test for the earlier detection of pancreatic adenocarcinoma [abstract]. In: Proceedings of the AACR Special Conference in Cancer Research: Advances in Pancreatic Cancer Research; 2024 Sep 15-18; Boston, MA. Philadelphia (PA): AACR; Cancer Res 2024;84(17 Suppl_2):Abstract nr B018.

Organizational and budget impact model (OBIM) of SAME a new autotransfusion medical device

Objectives This study aimed to assess the budget and organizational impact of progressively replacing the intraoperative cell salvage centrifugation-based systems currently installed in French hospitals with the SAME system, a new autotransfusion medical device. Methods An Organizational and Budget Impact Model (OBIM) was developed based on two methodological guidelines issued by the French Health Authority (Haute Autorité de Santé, HAS). The OBIM was also developed based on a pragmatic literature review, hospital data and hospital pharmacists’ expertise. Results Considering an average hospital cohort of 600 cardio-thoracic surgery patients, with 57% experiencing mild hemorrhages, 23% moderate hemorrhages, and 20% massive hemorrhages, and a SAME market share of 33% in Year 1, the implementation of SAME resulted in significant savings. With an average allogeneic transfusion of 4.19 packed red blood cells (RBC) and 0.62 platelet concentrate per patient based on National Hemovigilance Report of the ANSM (French National Agency for the Safety of Medicines and Health Products) and when using SAME system a reduction of 45% of RBC transfusion associated with a reduction of 60% and 90% of platelet use for moderate and massive hemorrhages respectively, the first year annual saving amounted to €44,601 and the cumulated saving over 5 years to €535,206. Discussion This model structure was developed based on overall hospitals’ needs and acknowledged guidelines, with inputs based on French literatures and hospital data, so findings were specifics to a context. Among the inputs, the number of annual SAME procedure is not based on device capability but rather on hospital capability with the number of operating rooms used for cardio-thoracic surgery equipped with the device. Conclusions The results of this OBIM demonstrated the economic and organizational benefits of SAME for hospitals. This benefit results mainly of a reduction in the use of allogeneic blood products (RBCs, platelets).

Artificial-Intelligence Cloud-Based Platform to Support Shared Decision-Making in the Locoregional Treatment of Breast Cancer: Protocol for a Multidimensional Evaluation Embedded in the CINDERELLA Clinical Trial.

Shared decision-making (SDM) plays a crucial role in breast cancer care by empowering patients and reducing decision regret. Patient decision aids (PtDAs) are valuable tools for facilitating SDM, now available in digital and artificial intelligence (AI)-powered formats to offer increasingly personalized contents. The ongoing CINDERELLA clinical trial (ClinicalTrials.gov: NCT05196269) evaluates an innovative AI cloud-based approach using a web platform and a mobile application (CINDERELLA APProach) versus the conventional approach to support SDM in breast cancer patients undergoing locoregional treatment. This protocol outlines a trial-based multidimensional evaluation, encompassing economic, financial, implementability, and environmental considerations associated with the CINDERELLA APProach. A within-trial cost-consequence and cost-utility analysis from a societal perspective will be performed using patient-level data on outcomes and resource use. The latter will be valued in monetary terms using country-specific unit costs or patient valuations. A budget impact analysis will be performed over 1 and 5 years from the budget holder perspectives. The CINDERELLA APProach implementability will be assessed through an evaluation of its usability, acceptability, organizational impact, and overall feasibility. The environmental impact will be quantitatively assessed across several dimensions, such as quantity, appropriateness, and emissions, supplemented by qualitative insights. Overall, data for the evaluation will be gathered from patient questionnaires, interviews with patients and managers, focus groups with healthcare professionals, and app electronic data. A thorough understanding of the broad consequences of the CINDERELLA APProach may foster its successful translation into real-world settings, hopefully benefiting breast cancer patients and clinical practice.

Examining macroeconomic factors and environmental sustainability: An OLS regression analysis of the impact of exports, imports, governmental budget, and oil products on CO2 emissions in Jordan

This study aims to investigate the relationship between macroeconomic factors and CO2 emissions. The study used a sample of data covering the period from 1991 to 2019 and employed ordinary least squares (OLS) method to estimate the relationship. The study found that there is a strong relationship between CO2 emissions and the macroeconomic factors of exports, imports, governmental budget, and oil products. The R-squared value of 0.717668 suggests that these variables explain approximately 72% of the variation in CO2 emissions. The coefficients for exports, imports and oil products are statistically significant at the 5% level. A negative coefficient for exports indicates that an increase in exports is associated with a decrease in CO2 emissions. On the other hand, a positive coefficient for imports suggests that an increase in imports is associated with an increase in CO2 emissions. Additionally, the positive coefficient for oil products indicates that an increase in oil product consumption is associated with a significant increase in CO2 emissions. The coefficient for the government budget variable is not statistically significant, which suggests that changes in the government budget do not have a significant impact on CO2 emissions. The results are robust and reliable as the assumptions of linearity, normality and homoscedasticity of errors were met. The study results could help policy makers in taking potential actions to mitigate CO2 emissions in the country.

Budget impact of dostarlimab plus carboplatin-paclitaxel for primary advanced or recurrent endometrial cancer from a third-party US payer perspective

Aim Dostarlimab plus carboplatin-paclitaxel (CP) significantly increased progression-free survival in patients with primary advanced or recurrent endometrial cancer (pA/rEC) vs CP alone in the RUBY trial (NCT03981796). This analysis estimated the per-member-per-month (PMPM) costs of introducing dostarlimab + CP as a treatment alternative from a third-party US payer perspective. Materials and methods A budget impact model was developed to estimate the costs of introducing dostarlimab + CP into commercial and Medicare health plans over a 3-year time horizon (2023–2025). Costs were sourced from relevant literature and US-specific databases and were calculated using epidemiology data, clinical inputs, treatment costs, and market share estimates. Clinical inputs were sourced from primary clinical trials for each respective treatment (i.e. dostarlimab + CP, CP, pembrolizumab, pembrolizumab plus lenvatinib, bevacizumab + CP, and pembrolizumab + CP). Current and future market shares assumed dostarlimab + CP reduced the market share of CP only. Analyses were performed in mismatch repair–deficient/microsatellite instability–high (dMMR/MSI-H) and overall populations using a US 2023 cost year. Results For a commercial plan, the model estimated (dMMR/MSI-H and overall populations) that 7 and 26 patients would be treated with dostarlimab + CP, respectively; average annual budget impacts per patient treated were $118,257 and $116,094; average budget impacts per patient treated per month (PPPM) were $9,855 and $9,675; average budget impacts PMPM were $0.02 and $0.06. For a Medicare plan, the model estimated that 28 and 93 patients, respectively, would be treated with dostarlimab + CP. Average annual budget impacts per patient treated and PPPM were the same as those for the commercial plan in both populations; average budget impacts PMPM were $0.07 and $0.22, respectively. Conclusions Introducing dostarlimab + CP as a first-line treatment for patients with pA/rEC results in minimal budget impact PMPM from a US third-party payer’s perspective. Together with the efficacy and safety results from RUBY, these results support the use of dostarlimab + CP as a treatment option.

Budget impact analysis on the Italian healthcare system of roxadustat for the treatment of anemia due to chronic kidney disease

Objective: The aim of this study was to evaluate the economic consequences of the introduction of roxadustat on the market as treatment for chronic kidney disease (CKD)-related anaemia in Italy. Method: A budget impact model was developed to assess the economic impact of the introduction of roxadustat for the management of patient with CKD-related anaemia. The analysis was conducted considering National Health Service (NHS) perspective and three years’ time horizon. Eligible population for the treatment with roxadustat was estimated through published literature and clinicians’ support. Direct healthcare costs included drug acquisition (list prices for base case) and administration costs, monitoring costs, cost per blood transfusion, costs related to the management of adverse events and dialysis costs. Results: The model estimated a three years expenditure associated with the management of patients with CKD-related incident anaemia in Italy of about € 260 and € 44.6 million for non-dialysis dependent (NDD) and dialysis-dependent (DD) patients respectively. The introduction of roxadustat on the market could generate a reduction in the expenditure related to the iron supplementation both for NDD and for DD patients (−€ 397,305 and −€ 50,135 over three years of the analysis, respectively) and a reduction in the pharmaceutical expenditure for NDD patients (−€ 2.3 million, −€ 7.5 million and −€ 13 million at first, second and third year of the analysis). Overall, the introduction of roxadustat could generate a saving for NHS approximately equal to € 2.3, € 7.5 and € 13 million at first, second and third year of the analysis. Conclusion: The introduction of roxadustat could generate a reduction in the expenditure for the management of patients with CKD anaemia. A scenario analysis which used tender prices for ESAs and hypothesis of confidential discount for roxadustat showed that the saving for NHS will be kept if roxadustat’s discount is greater than 60.3%.

A scoping review of the role of managed entry agreements in upcoming drugs for amyotrophic lateral sclerosis: learning from the case of spinal muscular atrophy

Introduction: The therapeutic options for spinal muscular atrophy (SMA) are encouraging. However, there is currently no cure for amyotrophic lateral sclerosis (ALS). The clinical and economic uncertainty surrounding innovative treatments for rare neurodegenerative diseases makes it necessary to understand managed entry agreements (MEAs). The aim of this study was to review whether models of MEAs in SMA could be extrapolated to ALS. Methods: We performed a scoping review with information on MEAs on SMA in Web of Science (WOS), PubMed, Lyfegen Library, the National Institute for Health and Care Excellence (NICE), and the Canadian Agency for Drugs and Technologies in Health (CADTH). Results: We found 45 results in WOS and PubMed. After an initial survey, 10 were reviewed to assess eligibility, and three were selected. We obtained 44 results from Lyfegen Library, and three results each from NICE and CADTH. Conclusion: The main objective of MEAs is to reduce uncertainty in the financing of drugs with a high budgetary impact and clinical concerns, as is the case with drugs for SMA and ALS. While the information available on MEAs in SMA is scarce, some conceptual models are publicly available. MEAs for long-term treatments for SMA could be used for the design of MEAs in ALS because of their similarities in economic and clinical uncertainty.

Budget Impact Analysis of Dapagliflozin in Treating Patients With Heart Failure With Reduced Ejection Fraction From the Perspective of Malaysian Public Healthcare System

PurposeThis is a budget impact analysis that compared the scenario of treating heart failure with reduced ejection fraction (HFrEF) using dapagliflozin plus standard of care (SoC) versus a scenario without dapagliflozin, from the perspective of Ministry of Health (MOH) Malaysia over a 5-year time horizon. MethodsA Microsoft Excel-based cost calculator was developed for such comparison. The estimated size of eligible population, uptake rates for dapagliflozin, as well as costs related to drugs, clinical events, and adverse events were based on published data, official tariffs, and databases, and expert opinion. Clinical data from the DAPA-HF trial were used to inform efficacy and safety inputs (i.e., hospitalization for heart failure (hHF), cardiovascular death, and adverse events). Results were reported as total annual and cumulative costs (in 2023 Malaysian Ringgits [RM], United States Dollars [USD], and European Union Euros, [EUR]; with exchange rates of 1 USD = RM 4.40 and 1 EUR = RM 4.90]), as well as the number of clinical events. Sensitivity and scenario analyses were also conducted. FindingsThe base-case analysis estimated that over a five-year period, the adoption of dapagliflozin for HFrEF treatment would result in a cumulative cost-saving of RM 2.6 million (USD 0.6 million/EUR 0.5 million), representing a 0.3% reduction in costs, driven primarily by reduced expenditure on hHF. Moreover, dapagliflozin treatment would lead to 731 fewer hHF and 366 fewer cardiovascular deaths. Sensitivity and scenario analyses revealed that the results were most sensitive to assumptions regarding loop diuretic requirements and the cost of dapagliflozin. Although cost savings or a net-zero balance were projected for the first four years, an anticipated 2.5% annual increase in dapagliflozin uptake in the longer term would lead to additional costs for the MOH, starting from the fifth year. ImplicationsIncorporating dapagliflozin into the SoC can improve health outcomes for HFrEF patients and may generate cost savings, potentially easing the economic strain of HFrEF management on Malaysia's public healthcare system in the short term. Nonetheless, a modest increase in budget should be anticipated as more patients gain access to the treatment over time.