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AimTo assess, from a United States (US) payer's perspective, the cost-effectiveness of gels designed to separate the endometrial surfaces (intrauterine spacers) placed following intrauterine surgery.Materials and methodsA decision tree model was developed to estimate the cost-effectiveness of intrauterine spacers used to facilitate endometrial repair and prevent the formation (primary prevention) and reformation (secondary prevention) of intrauterine adhesions (IUAs) and associated pregnancy- and birth-related adverse outcomes. Event rates and costs were extrapolated from data available in the existing literature. Sensitivity analyses were conducted to corroborate the base case results.ResultsIn this model, using intrauterine spacers for adhesion prevention led to net cost savings for US payers of $2,905 per patient over a 3.5-year time horizon. These savings were driven by the direct benefit of preventing procedures associated with IUA formation ($2,162 net savings) and the indirect benefit of preventing pregnancy-related complications often associated with IUA formation ($3,002). These factors offset the incremental cost of intrauterine spacer use of $1,539 based on an assumed price of $1,800 and the related increase in normal deliveries of $931. Model outcomes were sensitive to the probability of preterm and normal deliveries. Budget impact analyses show overall cost savings of $19.96 per initial member within a US healthcare plan, translating to $20 million over a 5-year time horizon for a one-million-member plan.LimitationsThere are no available data on the effects of intrauterine spacers or IUAs on patients' quality of life. Resultingly, the model could not evaluate patients' utility related to treatment with or without intrauterine spacers and instead focused on costs and events avoided.ConclusionThis analysis robustly demonstrated that intrauterine spacers would be cost-saving to healthcare payers, including both per-patient and per-plan member, through a reduction in IUAs and improvements to patients' pregnancy-related outcomes.

Background: Almost 2 million people in the UK live with sight loss, and this number is predicted to double by 2050. There is increasing recognition of the need to provide counselling services to people living with sight loss. Aim: The aim of this research was to assess whether there is an unmet need for counselling services for people living with sight loss in North Wales and to estimate the social value and budget impact of the potential roll‐out of counselling services to this population. Method: Between December 2020 and May 2021, a telephone survey was completed by 192 people living with sight loss in North Wales, UK. The survey instrument was a 19‐item questionnaire, which included the Short Warwick–Edinburgh Mental Well‐being Scale (SWEMWBS). Using data from the SWEMWBS and published statistics, a social return on investment (SROI) and budget impact analysis were conducted. Results: Most respondents (84%, n = 161) had never been offered counselling in relation to sight loss. Thirty‐nine per cent (n = 74) stated that they would use a new counselling service if it were available. Fifty‐one per cent (n = 97) indicated that counselling could be especially beneficial for people who have recently been registered or diagnosed with sight loss (the timely window). Of those who completed the SWEMWBS, approximately 25% reported low mental well‐being or probable depression. Social return on investment analysis estimated that providing counselling services for people with sight loss in North Wales could generate a social value of £3.42 for every £1 invested. Conclusion: Timely counselling may improve an individual's ability to cope with sight loss and improve their quality of life over time. [ABSTRACT FROM AUTHOR]

Aim: Rectus abdominal diastasis (RAD) can cause mainly incontinence and lower-back pain. Despite its high incidence, there is no consensus regarding surgical indication. We aimed at comparing RAD repair (minimally invasive technique with mesh implant) with no treatment (standard of care – SOC) through cost-effectiveness and budget impact analyses from both National Healthcare Service (NHS) and societal perspectives in Italy. Methods: A model was developed including social costs and productivity losses derived by the online administration of a socio-economic questionnaire, including the EuroQol for the assessment of quality of life. Costs for the NHS were based on reimbursement tariffs. Results: Over a lifetime horizon, estimated costs were 64,115€ for SOC and 46,541€ for RAD repair in the societal perspective; QALYs were 19.55 and 25.75 for the two groups, respectively. Considering the NHS perspective, RAD repair showed an additional cost per patient of 5,104€ compared to SOC, leading to an ICUR of 824€. RAD repair may be either cost-saving or cost-effective compared to SOC depending on the perspective considered. Considering a current scenario of 100% SOC, an increased diffusion of RAD repair from 2 to 10% in the next 5 years would lead to an incremental cost of 184,147,624€ for the whole society (87% borne by the NHS) and to incremental 16,155 QALYs. Conclusion: In light of the lack of economic evaluations for minimally invasive RAD repair, the present study provides relevant clinical and economic evidence to help improving the decision-making process and allocating scarce resources between competing ends. [ABSTRACT FROM AUTHOR]

Objectives: We perform a cost-effectiveness analysis (CEA) and budget impact analysis (BIA) of baloxavir marboxil compared to current care in the Netherlands for patients at risk of influenza-related complications, including patients with comorbidities and the elderly. Methods: In the CEA, a decision tree model was developed to assess the cost-effectiveness of baloxavir marboxil for a cohort of 52-year-olds from a societal perspective. A lifetime horizon was taken by incorporating the quality-adjusted life expectancy. The BIA included different epidemiological scenarios, estimating different plausible epidemiological scenarios for seasonal influenza considering the whole Dutch population with an increased risk of influenza complications. Results: The base-case ICER was estimated to be €8,300 per QALY. At the willingness-to-pay threshold of €20,000 per QALY, the probability of being cost effective was 58%. The base-case expected budget impact was €5.7 million on average per year, ranging from €1.5 million to €10.5 million based on the severity of the influenza epidemic and vaccine effectiveness. Conclusion: In the Netherlands, baloxavir is a cost-effective treatment option for seasonal influenza, with a base-case ICER of €8,300 per QALY for the population aged 60 years and over and patients at high risk of influenza-related complications. For a large part, this ICER is driven by the reduction of the illness duration of influenza and productivity gains in the working population. [ABSTRACT FROM AUTHOR]

ABSTRACT Objective Design Setting Sample Methods Main Outcome Measures Results Conclusion Prenatal exome sequencing (pES) for diagnosing fetal structural anomalies commenced in the English National Health Service (NHS) in 2020. We evaluated cost‐effectiveness to the healthcare system, and costs to families, of pES in addition to standard testing, compared to standard testing alone.A cost‐effectiveness analysis combining costs, outcomes, parent and professional interview and professional survey data.The English NHS Genomic Medicine Service.413 families with fetal anomalies with a suspected genetic cause referred for pES from 01 October 2021 to 30 June 2022.We costed the incremental resource required to deliver the pES clinical pathway. We calculated the diagnostic yield (proportion of cases with pathogenic variants). We divided the total incremental cost by the number of cases with a diagnosis to calculate cost‐effectiveness. We estimated the annual NHS budget requirement based on case numbers. We determined parental costs from interviews.Incremental costs of pES to the NHS and families, incremental cost per additional diagnosis and NHS budget impact.Of 413 referred cases, 241 were tested, at a cost of £2331 (95% credibility interval £1894–£2856) per referred case or £3592 (£2959–£4250) per case that proceeded with testing. The incremental cost per diagnosis (yield 35.3%) was £11 326 (£8582–£15 361). Based on referrals data 01 October 2022 to 30 September 2023, pES costs the NHS £1.8 m annually. Family costs could not be separated from other pregnancy‐related appointments but were not considered burdensome; most appointments were concurrent or remote.pES costs the English NHS £11 326 for each additional diagnosis. Incremental costs to families are negligible. [ABSTRACT FROM AUTHOR]

Objective: To estimate the efficiency of a nurse‐led telephone program for nonadherent to treatment Type 2 diabetics with comorbid depression (TELE‐DD program). Design: Secondary analysis of cost‐consequence and budget impact, utilizing data from a randomized clinical trial conducted in the primary healthcare setting. The target population consisted of Type 2 diabetic patients with comorbid depression who were nonadherent to their pharmacological treatment. Method: The average cost per controlled patient (glycated hemoglobin < 7%) and the incremental cost‐effectiveness ratio were calculated. Similarly, the budgetary impact over 1 year of implementing this program in the region of reference of the randomized clinical trial was assessed. Results: The number of controlled patients is higher in the TELE‐DD group at 6, 12, and 18 months. The average cost per controlled patient was higher in the TELE‐DD group than in the control group at 6 months (€160.31 vs. €49.79), but lower at 12 (€150.09 vs. €179.59) and 18 months (€209.22 vs. €376.88). The incremental cost‐effectiveness ratio at 6, 12, and 18 months was €254.47, €143.65, and €177.46, respectively. The budget impact analysis revealed that implementing the TELE‐DD program would result in a reduction of €721,940.68 in expenditure for the funder in the first year of application. Conclusions: A nurse‐led telephone program for nonadherent Type 2 diabetics with comorbid depression is an efficient option in the management of healthcare resources. These results highlight the role of nursing in chronic patient management and the efficient use of healthcare resources. Trial Registration: ClinicalTrials.gov Identifier: NCT04097483. [ABSTRACT FROM AUTHOR]

Ying Chen,1 Jiahua Pan,2 Yan Zhong,3 Bin Wu,1 Mengxia Yan,4 Ruiyun Zhang2 1Clinical Research Institute, Renji Hospital, School of Medicine, Shanghai Jiaotong University, Shanghai, 200000, People’s Republic of China; 2Department of Urology, Renji Hospital, School of Medicine, Shanghai Jiaotong University, Shanghai, 200000, People’s Republic of China; 3China Association for Promotion of Health Science and Technology, Beijing, 100000, People’s Republic of China; 4Department of Pharmacy, Ningbo Hangzhou Bay Hospital, Ningbo, 315000, People’s Republic of ChinaCorrespondence: Ruiyun Zhang, Email 19610@renji.comObjective: Prostate Cancer can be treated with various formulations of Gonadotropin-Releasing Hormone Agonists (GnRHa), but cost analyses of these treatments in China are lacking. This study aims to evaluate the differences in cost and resource utilization between various formulations of GnRHa for Prostate Cancer by conducting a resource utilization assessment and cost minimization analysis.Methods: From the perspective of society and medical healthcare, this study used the cost minimization model to generate cost and resource estimates for GnRHa drug acquisition and administration for “Current practice” and for a “Base case” scenario. In the “Base case” scenario, all of the patients who were receiving 1-monthly or 3-monthly GnRHa therapy in “Current practice” switched to a 6-monthly formulation triptorelin. Cost/Resource estimates were calculated per patient per administration and scaled to annualized population levels. Deterministic sensitivity analysis was conducted to explore the uncertainty of the model variables and applied assumptions.Results: From a societal perspective, if all 1-monthly and 3-monthly formulations of GnRHa were switched to a 6-monthly formulation triptorelin, it is conservatively estimated that the annual societal cost could be reduced by ¥ 13,382,951.13, with an average annual cost savings of ¥ 46.53 per patient. Additionally, the 6-monthly formulation could save 3,608,973.91 hours annually, translating to an average time savings of 12.55 hours per patient, reducing treatment time by 78%. From a healthcare system perspective, if the introduction of the 6-monthly formulation of GnRHa is delayed, it would lead to an annual increase of ¥ 94 million in medical costs, and require an additional 64,445.96 working days for doctors and nurses. Deterministic sensitivity analysis demonstrated the model’s robustness, showing the 6-monthly GnRHa remains cost-effective across various parameter changes, with drug price being the most influential factor.Conclusion: Compared to current 1-monthly and 3-monthly formulations, the 6-monthly GnRHa can reduce the total burden associated with prostate cancer treatment.Keywords: triptorelin, prostate cancer, cost-minimization, budget impact

Two vaccines against rotavirus diseases, Rotarix® and RotaTeq®, are being marketed in Spain; but rotavirus is not presently among the diseases covered by universal vaccination in Spain. The aim of this study was to assess the efficiency of extending Spain's current targeted rotavirus vaccination strategy including only preterm babies, to a policy of universal vaccination. A de novo cohort-based Markov model was built to evaluate the efficiency of three compared rotavirus vaccination strategies in Spain: targeted, universal, and no vaccination. Using Rotarix® or RotaTeq®, we compared the cost–utility of these strategies from both a societal perspective and Spanish National Health System (SNHS) perspective. The model represents the most important clinical events conceivably linked to rotavirus infection. Efficacy, effectiveness, safety, costs, and utilities were identified by systematic reviews. Incremental cost–utility ratio (ICUR) is EUR 23,638/QALY (Quality-Adjusted Life Year) for targeted vaccination with Rotarix® compared with no vaccination. The ICUR for the rest of the strategies evaluated are above EUR 30,000/QALY. The sensitivity analysis shows price as the only parameter that could make the universal vaccination strategy efficient. Considering a threshold of EUR 25,000/QALY, only targeted vaccination with Rotarix® would be efficient from societal perspective. Price drops of 36.9% for Rotarix® and 44.6% for RotaTeq® would make universal vaccination efficient. [ABSTRACT FROM AUTHOR]

Introduction: Financial incentives to stop smoking (FISS) programs have been implemented internationally to encourage people who smoke to quit smoking. However, such programs require that the financial reward structure and its resulting effects on smoking quit rates are considered. We analyzed a number of scenarios for FISS reward schedules for current smoking individuals in Ireland, with a view to identify the potential implications in terms of financial consequences and expected effects. Methods: Using national QuitManager services data 2021–2023, we defined smoking quit rates for smokers currently using the national Health Services Executive stop smoking services in Ireland. Smoking quit rates at 4, 12 and 52 weeks were defined, and additionally defined by sex, age and education level. Using scenarios assuming different FISS reward sizes, structures and targeted population sub-groups, we estimated the number of additional quitters, budget impact, and incremental costeffectiveness ratio. Results: A FISS program, if implemented for a cohort of 3500 smokers can result in a budget impact ranging €250000 – €870000. The cost-effectiveness trade-off between different payment schedules and the expected effect size suggested that FISS are cost-effective even at a moderate effect size. A FISS program implemented to approximately 20000 smokers nationally would cost between €2.0 million and €4.8 million, subject to the chosen reward schedule. Across social groups, FISS is more cost-effective for females, individuals in the youngest age group, and individuals with a medium level of education. Conclusions: This analysis highlights the importance of considering different FISS schedules and potential quit effects, when designing such programs. We highlight that FISS programs should be targeted at certain social groups to achieve highest long-term smoking cessation rates. We also identified important challenges that decision-makers face when designing the reward structure of FISS programs. The acceptability or otherwise of the FISS structures may differ among stakeholders and should be explored. [ABSTRACT FROM AUTHOR]

Background Drug expenditure is an important part of health expenditure. Managed Entry Agreement (MEA) is a common strategy implemented in many countries, such as the United States, the United Kingdom, and the European countries to control drug expenditures, especially for new and high-cost drugs. This study aims to explore the appropriate MEA technique for reaching the lowest cost of drug procurement under specified uncertainty of the high-cost drug.Methods The cost of drug procurement varied by the MEA techniques will be investigated in the quantitative analysis based on MEA taxonomies and uncertainty in terms of price, use, and effectiveness. Then, the content analysis will be employed to the qualitative analytical part to summarise the matching of appropriate MEA technique with the characteristics of high-cost drug to lower the cost of drug procurement and increase access to high-cost drugs.Discussion The rationales for each MEA technique selection are similar across their objectives. MEA can help reduce drug expenditures. Therefore, the budget in health care system could be sustainable and the patient access to high-cost drug could be increased. However, it might not be suitable for some circumstances and should not be implemented to determine drug price or used as regular reimbursement.

Gene therapies delivered through a single administration have revolutionized treatment possibilities for many patients living with serious or fatal conditions such as spinal muscular atrophy, hemophilia and sickle cell disease. However, shadowing the excitement about the transformational potential of many gene therapies has been widespread concern about the combination of uncertainty in the durability of their benefits over the long term and the short-term financial shock of high prices. As the healthcare payment ecosystem prepares for the growing number of gene therapies entering the market, three key interconnected challenges must be addressed: determining a fair price, managing clinical uncertainty and managing short-term budget impacts. This paper identifies specific policy reforms and market-based tools to help the US health system address these challenges to achieve more equitable and affordable access for patients to the growing number of gene therapies expected to be approved in the coming years.

Summary: Background: The high price of HPV vaccines remains a significant barrier to vaccine accessibility in China, hindering the country’s efforts toward cervical cancer elimination and exacerbating health inequity. We aimed to inform HPV vaccine price negotiations by identifying threshold prices that ensure that a government-funded HPV vaccination programme is cost-effective or cost-saving. Methods: We used a previously validated transmission model to estimate the health and economic impact of HPV vaccination over a 100-year time horizon from a healthcare payer perspective. Threshold analysis was conducted considering different settings (national, rural, and urban), cervical cancer screening scenarios (cytology-based or HPV DNA-based, with different paces of scale-up), vaccine types (four types available in China), vaccine schedules (two-dose or one-dose), mode of vaccination (routine vaccination with or without later switching to high-valency vaccines), willingness-to-pay thresholds, and decision-making criteria (cost-effective or cost-saving). Furthermore, we examined the budget impact of introducing nationwide vaccination at the identified threshold prices. Findings: Using the current market price, national routine HPV vaccination with any currently available vaccine is unlikely cost-effective. Under a two-dose schedule, the prices of the four available HPV vaccine types cannot exceed $26–$36 per dose (44.1%–80.2% reduction from current market prices) depending on vaccine type to ensure the cost-effectiveness of the national programme. Adopting vaccination at threshold prices would require an annual increase of 72.18%–96.95% of the total annual National Immunization Programme (NIP) budget in China. A cost-saving routine vaccination programme requires vaccine prices of $5–$10 per dose (depending on vaccine type), producing a 21.38%–34.23% increase in the annual NIP budget. Adding the second dose is unlikely to be cost-effective compared to a one-dose schedule, with the threshold price approaching or even falling below zero. Rural pilot vaccination programmes require lower threshold prices compared with a national programme. Interpretation: Our study could inform vaccine price negotiation and thus facilitate nationwide scale-up of current HPV vaccination pilot programmes in China. The evidence may potentially be valuable to other countries facing HPV introduction barriers due to high costs. This approach may also be adapted for other contexts that involve the introduction of a pricy vaccine. Funding: CAMS Innovation Fund for Medical Sciences (CIFMS); Bill & Melinda Gates Foundation.

Objectives. To analyze the budget impact of upadacitinib (UPA) 15 mg + methotrexate (MTX) for the treatment of moderate-to-severe rheumatoid arthritis (RA) in patients with an inadequate response to conventional disease-modifying antirheumatic drugs (cDMARD-IR) from the perspective of social security and the private health sector in Argentina. Materials and methods. A budget impact analysis model was developed for a hypothetical cohort of 100,000 adults with health insurance coverage who were diagnosed with RA over a 5-year time horizon. The model parameters were obtained through literature review and validated by local experts. The costs are expressed in 2024 US dollars (USD). Results. The introduction of UPA 15 mg + MTX for the treatment of moderate-to-severe RA and cDMARD-IR resulted in minimal increase, with a five-year total cumulative incremental cost of USD 1,855 for social security and USD 1,812 for the private health sector, representing 2% of the total budget. The acquisition cost of UPA was the most influential variable in the sensitivity analysis. Conclusions. The introduction of UPA 15 mg + MTX for the treatment of moderate-to-severe RA and cDMARD-IR can provide an effective treatment option with a minimal increase in costs for the healthcare system in Argentina, which is especially important in developing countries where health system budgets are more limited. Providing evidence-based estimates is a valuable tool for informing healthcare policies and can help policymakers make informed decisions about the allocation of healthcare resources to improve patient outcomes while also managing costs.

Abstract Background Indonesia has the world’s second-highest tuberculosis (TB) burden, with 969,000 annual TB infections. In 2017, Indonesia faced significant challenges in TB care, with 18% of cases missed, 29% of diagnosed cases unreported, and 55.4% of positive results not notified. The government is exploring a new approach called “strategic purchasing” to improve TB detection and treatment rates and offer cost-effective service delivery. Objectives We aimed to analyze the financial impact of implementing a TB purchasing pilot in the city of Medan and assess the project’s affordability and value for money. Methods We developed a budget impact model to estimate the cost-effectiveness of using strategic purchasing to improve TB reporting and treatment success rates. We used using data from Medan’s budget impact model and the Ministry of Health’s guidelines to predict the total cost and the cost per patient. Results The model showed that strategic purchasing would improve TB reporting by 63% and successful treatments by 64%. While this would lead to a rise in total spending on TB care by 60%, the cost per patient would decrease by 3%. This is because more care would be provided in primary healthcare settings, which are more cost-effective than hospitals. Conclusions While strategic purchasing may increase overall spending, it could improve TB care in Indonesia by identifying more cases, treating them more effectively, and reducing the cost per patient. This could potentially lead to long-term cost savings and improved health outcomes.

Bjoern Schwander,1 Katharina Klesper,2 Siegbert Rossol,3 Ken Herrmann,4 York Francis Zoellner5 1Department of Health Economics & Outcomes Research, AHEAD GmbH, Bietigheim-Bissingen, Baden-Württemberg, Germany; 2Department of Health Economics & Epidemiology, ECON-EPI, Meerbusch, Nordrhein-Westfalen, Germany; 3Medical Clinic, Krankenhaus Nordwest, Frankfurt a.M., Hessen, Germany; 4Department of Nuclear Medicine, University of Duisburg-Essen, and German Cancer Consortium (DKTK)-University Hospital Essen, Essen, Nordrhein-Westfalen, Germany; 5Department of Health Sciences, University of Applied Sciences Hamburg, Hamburg, GermanyCorrespondence: Bjoern Schwander, AHEAD Gmbh, Wilhelm-Leibl-Straße 7, Bietigheim-Bissingen, D-74321, Germany, Tel + 49 1577 194 8125, Email bjoern.schwander@ahead-net.dePurpose: To compare personalized dosimetry with yttrium-90 (90Y)-loaded glass microspheres (SIRT) vs atezolizumab and bevacizumab (A+B) in hepatocellular carcinoma (HCC) treatment in terms of cost-effectiveness and budget impact from a German statutory health insurance (SHI) perspective.Patients and Methods: Cost-effectiveness analysis (CEA) and budget impact analysis (BIA) models were developed in MS Excel. The available key studies (IMbrave150 and DOSISPHERE-01) suggest that both strategies are comparable in terms of progression-free survival and overall survival in HCC, but a difference in severe adverse events (SAE) in favor of SIRT was observed. Accordingly, the CEA model investigates the endpoints “cost per SAE avoided” and “cost per quality-adjusted life year (QALY) gained”, whereas the BIA simulates the impact of a stepwise re-allocation of current market share to the option which emerges as more cost-effective from the CEA.Results: The model suite estimated a mean annual total per-patient costs of € 29,984 for SIRT, compared to € 75,725 for A+B. SIRT was associated with a lower number of SAE and a higher number of QALYs compared to A+B. Switching additionally 25% of the eligible patients (&ap;500) from systemic therapy to SIRT could generate annual savings of approximately € 22.6 million Euros to the SHI.Conclusion: SIRT was identified as dominant treatment strategy. SIRT use not only saves SHI expenditure compared to systemic immunotherapy but also yields extra QALYs. This positions SIRT as the dominant and more cost-effective treatment strategy for patients with HCC. The savings to the SHI system, derived from the BIA conducted, become increasingly significant with rising adoption rates of SIRT.Keywords: selective internal radiation therapy, atezolizumab, bevacizumab, hepatocellular carcinoma, cost-effectiveness, budget impact

Background: Indonesia has the world's second-highest tuberculosis (TB) burden, with 969,000 annual TB infections. In 2017, Indonesia faced significant challenges in TB care, with 18% of cases missed, 29% of diagnosed cases unreported, and 55.4% of positive results not notified. The government is exploring a new approach called "strategic purchasing" to improve TB detection and treatment rates and offer cost-effective service delivery. Objectives: We aimed to analyze the financial impact of implementing a TB purchasing pilot in the city of Medan and assess the project's affordability and value for money. Methods: We developed a budget impact model to estimate the cost-effectiveness of using strategic purchasing to improve TB reporting and treatment success rates. We used using data from Medan's budget impact model and the Ministry of Health's guidelines to predict the total cost and the cost per patient. Results: The model showed that strategic purchasing would improve TB reporting by 63% and successful treatments by 64%. While this would lead to a rise in total spending on TB care by 60%, the cost per patient would decrease by 3%. This is because more care would be provided in primary healthcare settings, which are more cost-effective than hospitals. Conclusions: While strategic purchasing may increase overall spending, it could improve TB care in Indonesia by identifying more cases, treating them more effectively, and reducing the cost per patient. This could potentially lead to long-term cost savings and improved health outcomes. [ABSTRACT FROM AUTHOR]

Primary demyelinating disorders of the central nervous system (CNS) include multiple sclerosis and the orphan conditions neuromyelitis optica spectrum disorder (NMOSD) and myelin oligodendrocyte glycoprotein IgG-associated disease (MOGAD). Curative technologies under development aim to selectively block autoimmune reactions against specific autoantigens while preserving the responsiveness of the immune system to other antigens. Our analysis focused on target patient selection for such developments, carefully considering the relevant clinical, regulatory, and market-related aspects. We found that the selection of patients with orphan conditions as target populations offers several advantages. Treatments for orphan conditions are associated with limited production capacity, qualify for regulatory incentives, and may require significantly shorter and lower-scale clinical programs. Furthermore, they may meet a higher acceptable cost-effectiveness threshold in order to compensate for the low numbers of patients to be treated. Finally, curative technologies targeting orphan indications could enter less competitive markets with lower risk of generic price erosion and would benefit from additional market protection measures available only for orphan products. These advantages position orphan conditions and subgroups as the most attractive target indications among primary demyelinating disorders of the CNS. The authors believe that after successful proof-of-principle demonstrations in orphan conditions, broader autoimmune patient populations may also benefit from the success of these pioneering developments. [ABSTRACT FROM AUTHOR]

Objective: The price of pharmaceuticals is important from the economic and industrial perspectives but as well as patients' access to treatment. This study aimed to analyze the variables affecting the prices of new drugs in South Korea's pricing system. Methods: Data on 192 new drugs listed in South Korea from 2012 to 2022 were collected from the official website of the Health Insurance Review and Assessment Service. The independent variables included drugs for severe diseases, alternatives, number of patients, number of advanced 7 countries listed, budget impact, and listing period. The dependent variables included annual treatment cost and the price ratio to the advanced 7 country's average adjusted price. Descriptive statistics of variables, linear correlations between quantitative independent and dependent variables, and associations between independent and dependent variables were analyzed. Results: The mean annual treatment cost and price ratio to the advanced 7 country's average adjusted price were higher for drugs for severe diseases and those with no alternatives. Annual treatment cost and price ratio to the advanced 7 country's average adjusted price were negatively correlated with the number of patients and positively correlated with the number of advanced 7 countries listed. Annual treatment cost was affected by the variables drugs for severe diseases, alternatives, number of patients, number of advanced 7 countries listed, and budget impact. The price ratio to the advanced 7 country's average adjusted price was affected by drugs for severe diseases, alternatives, and the number of patients. Conclusion: This study revealed the effect of different variables on the prices of new drugs in South Korea, allowing for the development of a more effective assessment system to evaluate the prices of new drugs while ensuring profitability for pharmaceutical companies, sustainability of public insurance, and accessibility to drugs by patients. [ABSTRACT FROM AUTHOR]

Background: Prediabetes management is a priority for policymakers globally, to avoid/delay type 2 diabetes (T2D) and reduce severe, costly health consequences. Countries moving from low to middle income are most at risk from the T2D "epidemic" and may find implementing preventative measures challenging; yet prevention has largely been evaluated in developed countries. Methods: Markov cohort simulations explored costs and benefits of various prediabetes management approaches, expressed as "savings" to the public health care system, for three countries with high prediabetes prevalence and contrasting economic status (Poland, Saudi Arabia, Vietnam). Two scenarios were compared up to 15 y: "inaction" (no prediabetes intervention) and "intervention" with metformin extended release (ER), intensive lifestyle change (ILC), ILC with metformin (ER), or ILC with metformin (ER) "titration." Results: T2D was the highest‐cost health state at all time horizons due to resource use, and inaction produced the highest T2D costs, ranging from 9% to 34% of total health care resource costs. All interventions reduced T2D versus inaction, the most effective being ILC + metformin (ER) "titration" (39% reduction at 5 y). Metformin (ER) was the only strategy that produced net saving across the time horizon; however, relative total health care system costs of other interventions vs inaction declined over time up to 15 y. Viet Nam was most sensitive to cost and parameter changes via a one‐way sensitivity analysis. Conclusions: Metformin (ER) and lifestyle interventions for prediabetes offer promise for reducing T2D incidence. Metformin (ER) could reduce T2D patient numbers and health care costs, given concerns regarding adherence in the context of funding/reimbursement challenges for lifestyle interventions. [ABSTRACT FROM AUTHOR]

Objectives. To analyze the budget impact of upadacitinib (UPA) 15 mg + methotrexate (MTX) for the treatment of moderate-to-severe rheumatoid arthritis (RA) in patients with an inadequate response to conventional disease-modifying antirheumatic drugs (cDMARD-IR) from the perspective of social security and the private health sector in Argentina. Materials and methods. A budget impact analysis model was developed for a hypothetical cohort of 100,000 adults with health insurance coverage who were diagnosed with RA over a 5-year time horizon. The model parameters were obtained through literature review and validated by local experts. The costs are expressed in 2024 US dollars (USD). Results. The introduction of UPA 15 mg + MTX for the treatment of moderate-to-severe RA and cDMARD-IR resulted in minimal increase, with a five-year total cumulative incremental cost of USD 1,855 for social security and USD 1,812 for the private health sector, representing 2% of the total budget. The acquisition cost of UPA was the most influential variable in the sensitivity analysis. Conclusions. The introduction of UPA 15 mg + MTX for the treatment of moderate-to-severe RA and cDMARD-IR can provide an effective treatment option with a minimal increase in costs for the healthcare system in Argentina, which is especially important in developing countries where health system budgets are more limited. Providing evidence-based estimates is a valuable tool for informing healthcare policies and can help policymakers make informed decisions about the allocation of healthcare resources to improve patient outcomes while also managing costs. [ABSTRACT FROM AUTHOR]

Aim: Chronic stroke walking impairment is associated with high healthcare resource utilization (HCRU) costs. InTandem™ is a neurorehabilitation system that autonomously delivers a rhythmic auditory stimulation (RAS)-based intervention for the at-home rehabilitation of walking impairment in adults in the chronic phase of stroke recovery. This study was conducted to estimate the budget impact of InTandem in comparison with currently available intervention strategies for improvement of gait/ambulation in individuals with chronic stroke walking impairment. Methods & materials: A budget impact analysis (BIA) for InTandem was conducted based on a 1-million-member US third-party payer perspective over a 1- year time horizon. Key inputs for the budget impact model were: costs for each intervention strategy (InTandem, physical therapy, self-directed walking and no treatment), HCRU costs for persons with chronic stroke and anticipated HCRU cost offsets due to improvements in gait/ambulatory status as measured by self-selected comfortable walking speed (based on functional ability). In addition to the reference case analysis, a sensitivity analysis was conducted. Results: Based on the reference case, introduction of InTandem was projected to result in overall cost savings of $439,954 in one year. Reduction of HCRU costs (-$2,411,778) resulting from improved walking speeds with InTandem offset an increase in intervention costs (+$1,971,824). Demonstrations of cost savings associated with InTandem were robust and were consistently evident in nearly all scenarios evaluated in the sensitivity analysis (e.g., with increased/decreased patient shares, increased HCRU cost or increased InTandem rental duration). Conclusion: The InTandem system is demonstrated to improve walking and ambulation in adults in the chronic phase of stroke recovery after a five-week intervention period. The BIA predicts that introduction of InTandem will be associated with overall cost savings to the payer.

• The use of vacuum-induced hemorrhage control (VHC) devices potentially saves costs. • Patients treated with uterine balloon tamponade (UBT) have highest saving potential. • In Germany, 7,129 and 49,523 patients had (main or secondary) diagnosis of PPH. This study aimed to assess the budget impact of vacuum-induced hemorrhage control (VHC) devices for treating postpartum hemorrhage (PPH) from the perspective of the German statutory health insurance (SHI). Evidence shows that treating PPH with VHC instead of uterine balloon tamponade (UBT) can reduce resource consumption (e.g., reduced number of blood transfusions and length of stay). A budget impact model combining aggregated German real-world reimbursement data of PPH cases with the assumption of resource reduction due to VHC usage was developed. Diagnosis-related groups (DRG) of PPH cases and their frequencies were collected using a publicly available database. A "downgrading mechanism" was performed, leading to a less resource-intensive DRG, i.e., resulting in a lower flat fee to be paid by SHI. Four subgroups were differentiated based on coded diagnoses and procedures: 1) PPH (O72.-) as main diagnosis, 2) PPH as secondary diagnosis, 3) UBT procedure coded, and 4) UBT or standard tamponade coded. Weighted averages of cost savings per case were calculated. Data from 7,129 (subgroup 1), 49,523 (subgroup 2), 1,668 (subgroup 3), and 3,406 (subgroup 4) cases were retrieved. After applying the downgrading mechanism, cost savings (weighted average) resulted in 184.09 €, 210.50 €, 921.33 €, and 633.74 € for subgroups 1–4, respectively, This is the first German budget impact analysis of VHC for the treatment of PPH. Results showed the highest cost-saving potential for cases currently treated with UBT. Demonstrating not only clinical but also financial consequences of innovative treatments is crucial for the adoption into clinical practice. [ABSTRACT FROM AUTHOR]

Introduction: Anemia and transfusion of blood products are risk factors associated with poor patient outcomes across all elective surgeries. Patient blood management (PBM) is a patient-centered approach to optimize patient's endogenous red cell mass, to minimize blood loss in patients undergoing surgery, and to harness and optimize patient-specific physiological tolerance to anemia. This study aimed to assess (1) the impact of PBM on blood product usage in cardiovascular surgeries in a state hospital setting, (2) cost-effectiveness of PBM with a model based on transfusion of red blood cells (RBCs) in cardiovascular surgeries, and (3) the budget impact of PBM implementation based on transfusion of RBCs. Methods: Cost-effectiveness and budget impact models, based on the numbers of avoided transfusions and avoided complications after implementation of the PBM program, were compared between pre- and post-PBM periods at the cardiovascular surgery department of Ankara Bilkent City Hospital between February 11, 2019 and July 24, 2022. The probabilities of transfusions and complications with and without PBM were taken from recent meta-analyses. Data from the Ankara Bilkent City Hospital transfusion center informed the pre- and post-PBM calculations. Costs were calculated from the Social Security Institution's perspective. Results: There was a 21% decrease in the use of RBCs and a 23.7% decrease in use of all blood products after the implementation of PBM. The number of RBC packs per patient reduced by 0.88 packs (21%). The cost saving from reduction of RBC transfusions per patient was 518.68 Turkish lira (TRY) and for the hospital it was 1,635,948 TRY. Fewer complications and lower costs in favor of the post-PBM arm were demonstrated in the cost-effectiveness analysis. On the basis of the budget impact model, in 20 months, the hospital's cardiovascular surgery department saved 6,596,934 TRY (€342,302). Conclusion: This hospital-based study demonstrated that PBM is a budget-saving and cost-effective option in Turkey. Plain Language Summary: When undergoing elective surgery, patients who develop anemia or who need a transfusion of blood products may develop further complications. This study aimed to understand the impact of patient blood management on blood usage in cardiovascular surgeries. Patient blood management is a patient-centered approach that aims to optimize a patient's red cell mass, minimize blood loss in patients undergoing surgery, and maximize a patient's tolerance to anemia. In addition, this study assessed the cost-effectiveness of patient blood management using an economic model based on red blood cells in cardiovascular surgeries and assessed the impact of patient blood management on budget. The patient blood management program resulted in a 21% decrease in use of red blood cells and 23.7% decrease in use of all blood products. The cost savings from reduction of red blood cells transfusions per patient were 518.68 Turkish lira and cost savings for the hospital were 1,635,948 Turkish lira. Fewer complications and lower costs were demonstrated after implementation of patient blood management in the cost-effectiveness analysis. Between December 2020 and July 2022, the hospital's cardiovascular surgery department saved 6,596,934 Turkish lira (€342,302). Overall, this hospital-based study has shown that patient blood management is a budget-saving and cost-effective option in Turkey. [ABSTRACT FROM AUTHOR]

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). [ABSTRACT FROM AUTHOR]

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. PLAIN LANGUAGE SUMMARY: Dostarlimab with carboplatin–paclitaxel is a recently approved treatment for newly diagnosed advanced or recurrent endometrial cancer. This analysis was done to estimate the added costs that US commercial and Medicare health plans would have over 3 years if this treatment was covered. This analysis found that the budget increase for covering dostarlimab with carboplatin–paclitaxel was small ($0.02–$0.06 per commercial plan member per month; $0.07–$0.22 per Medicare plan member per month). [ABSTRACT FROM AUTHOR]

Aims: This study assessed the budget impact of resmetirom as a treatment for adults with non-cirrhotic non-alcoholic steatohepatitis (NASH) with moderate-to-advanced liver fibrosis and estimated total costs for a hypothetical private payer in the United States. Materials and methods: A three-year budget impact analysis based on an open cohort state transition model was developed for a hypothetical one-million-member private health plan. The comparator was Standard of Care (SOC), defined as routine care for non-cirrhotic NASH patients with moderate-to-advanced liver fibrosis. Each year, the number of resmetirom treatment-eligible patients was estimated through prevalent, incident, and diagnostic rate estimates. Costs included resources incurred by the medical and pharmacy benefits of private payers, including resmetirom drug acquisition costs, diagnosis and monitoring, other medical and other prescription costs stratified by disease progression status (i.e. non-cirrhotic vs. cirrhotic/advanced liver diseases). Resmetirom adverse event management costs were included in sensitivity analysis. Drug costs were estimated based on the average wholesale acquisition cost as of March 2024. Other costs were based on published sources and inflated to 2023 US dollars. Budget impact outcomes were presented in aggregate, net, and on a per-member per-month (PMPM) basis. Results: Compared with a scenario without resmetirom, the introduction of resmetirom yielded results ranging from 50 to 238 treated patients, net budget impact of $2.2 to $9.5 million, and PMPM from $0.19 to $0.80 over years one and three. Net costs excluding resmetirom declined over time. In sensitivity analyses, results were most sensitive to diagnostic and epidemiologic inputs. Limitations: Market shares are based on internal forecasts, a short time horizon, average treatment effects, and other limitations common to BIMs. Conclusion: The adoption of resmetirom on the formulary for the treatment of non-cirrhotic NASH with moderate-to-advanced liver fibrosis resulted in a moderate increase in budget impact with declining costs related to NASH progression. PLAIN LANGUAGE SUMMARY: Non-alcoholic steatohepatitis (NASH) is a serious liver disease that can lead to significant liver damage, other health complications, and increased healthcare costs. As the disease progresses, patients typically experience worsening health outcomes. Until recently, there were no Food and Drug Administration (FDA) approved treatments for NASH in the United States. However, in March 2024, the FDA approved REZDIFFRA, a new drug specifically designed to treat NASH patients with moderate-to-advanced liver fibrosis (i.e. NASH with moderate-to-advanced scarring of the liver). Clinical trials have shown that REZDIFFRA can improve health outcomes in these patients. To identify patients who could benefit from REZDIFFRA and to estimate the associated costs, we developed a budget impact model. In this study, we detail the development of this model and present its findings. Our analysis revealed that, while REZDIFFRA is associated with higher overall costs, primarily due to the price of the drug itself, there are potential cost savings when considering the drug's ability to slow disease progression. [ABSTRACT FROM AUTHOR]

Aims: Fruquintinib is a selective small molecule tyrosine kinase inhibitor of vascular endothelial growth factor receptor (VEGFR)-1, -2, and -3 recently approved in the United States (US) for the treatment of adult patients with metastatic colorectal cancer (CRC) who have previously been treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF biological therapy, and if RAS wild-type and medically appropriate, anti-epidermal growth factor receptor therapy. This study aimed to estimate the 5-year budget impact of fruquintinib from a US payer perspective (commercial and Medicare). Materials and methods: A budget impact model was developed to compare two scenarios: a reference scenario in which patients received regorafenib, trifluridine/tipiracil, or trifluridine/tipiracil with bevacizumab and an alternative scenario in which patients received reference scenario treatments or fruquintinib. Market shares were evenly divided across available options. A 5-year time horizon and a hypothetical health plan of 1 million members was assumed. The model included epidemiological inputs to estimate the eligible population; clinical inputs for treatment duration, progression-free survival, overall survival, and adverse event (AE) frequency; and cost inputs for treatment, AEs, disease management, subsequent therapy, and terminal care costs. Budget impact was reported as total, per member per year (PMPY), and per member per month (PMPM). Results: The model estimated an eligible population of 194 patients (39 per year) over 5 years. In the base case, the estimated 5-year budget impact of fruquintinib was $4,077,073 ($0.82 PMPY and 0.07 PMPM) for a commercial health plan. During the first year, the estimated budget impact was $627,570 ($0.63 PMPY and 0.05 PMPM). Results were robust across sensitivity analyses. PMPM costs from the Medicare perspective were greater than the base-case (commercial) ($0.17 vs. $0.07) due to higher incidence of CRC in that population. Conclusions: Fruquintinib is associated with a low budget impact for payers based on proposed thresholds in the US. PLAIN LANGUAGE SUMMARY: Fruquintinib is a treatment for metastatic colorectal cancer that has progressed after or not responded to multiple guideline-recommended therapies. This budget impact analysis was conducted to estimate the added costs a health plan would incur over a 5-year period if it chose to cover this therapy. The analysis found that the per plan member per month cost of covering fruquintinib was $0.07 for a United States commercial health plan and $0.17 for Medicare. [ABSTRACT FROM AUTHOR]

Aims: Use of gene expression signatures to predict adjuvant chemotherapy benefit in women with early-stage breast cancer is increasing. However, high cost, limited access, and eligibility for these tests results in the adoption of less precise assessment approaches. This study evaluates the cost impact of PreciseDx Breast (PDxBr), an AI-augmented histopathology platform that assesses the 6-year risk of recurrence in early-stage invasive breast cancer patients to help improve informed use of adjuvant chemotherapy. Materials and methods: A decision-tree Markov model was developed to compare the costs of treatment guided by standard of care (SOC) risk assessment (i.e. clinical diagnostic workup with or without Oncotype DX) versus PDxBr with SOC in a hypothetical cohort of U.S. women with early-stage invasive breast cancer. A commercial payer perspective compares costs of testing, adjuvant therapy, recurrence, adverse events, surveillance, and end-of-life care. Results: PDxBr use in prognostic evaluation resulted in savings of $4 million (M) in year one compared to current SOC in 1 M females members. Over 6-years, savings increased to $12.5 M. The per-treated patient costs in year one amounted to $19.5 thousand (K) for SOC and $16.9K for PDxBr. Limitations: For simplicity, recurrence was not specified. We performed scenario analyses to account for variations in rates for local, regional, and distant recurrence. Second, a recurrent patient incurs the total cost of treated recurrence in the first year and goes back to remission or death. Third, CDK4/6i treatment is only incorporated in the recurrence costs but not in the first line of treatment for early-stage breast cancer due to limited data. Conclusions: Sensitivity analyses demonstrated robust overall savings to changes in all variables in the model. The use of PDxBr to assess breast cancer recurrence risk has the potential to fill gaps in care and reduce costs when gene expression signatures are not available. [ABSTRACT FROM AUTHOR]

Objective: To estimate the potential budget impact on US third party payers (commercial or Medicare) associated with addition of selpercatinib as a tumor-agnostic treatment for patients with Rearranged during Transfection (RET)-altered solid tumors. Methods: An integrated budget impact model (iBIM) with 3-year (Y) time horizon was developed for 19 RET-altered tumors. It is referred to as an integrated model because it is a single model that integrated results across multiple tumor types (as opposed to tumor-specific models developed traditionally). The model estimated eligible patient populations and included tumor-specific comparator treatments for each tumor type. Estimated annual total costs (2022USD, $) included costs of drug, administration, supportive care, and toxicity. For a one-million-member plan, the number of patients with RET-altered tumors eligible for treatment, incremental total costs, and incremental per-member per-month (PMPM) costs associated with introduction of selpercatinib treatment were estimated. Uncertainty associated with model parameters was assessed using various sensitivity analyses. Results: Commercial perspective estimated 11.68 patients/million with RET-altered tumors as treatment-eligible annually, of which 7.59 (Y1), 8.17 (Y2), and 8.76 (Y3) patients would be selpercatinib-treated (based on forecasted market share). The associated incremental total and PMPM costs (commercial) were estimated to be: $873,099 and $0.073 (Y1), $2,160,525 and $0.180 (Y2), and $2,561,281 and $0.213 (Y3), respectively. The Medicare perspective estimated 55.82 patients/million with RET-altered tumors as treatment-eligible annually, of which 36.29 (Y1), 39.08 (Y2), and 41.87 (Y3) patients would be selpercatinib-treated. The associated incremental total and PMPM costs (Medicare) were estimated to be: $4,447,832 and $0.371 (Y1), $11,076,422 and $0.923 (Y2), and $12,637,458 and $1.053 (Y3), respectively. One-way sensitivity analyses across both perspectives identified drug costs, selpercatinib market share, incidence of RET, and treatment duration as significant drivers of incremental costs. Conclusions: Three-year incremental PMPM cost estimates suggest a modest impact on payer-budgets associated with introduction of tumor-agnostic selpercatinib treatment. [ABSTRACT FROM AUTHOR]

Abstract Background Prediabetes management is a priority for policymakers globally, to avoid/delay type 2 diabetes (T2D) and reduce severe, costly health consequences. Countries moving from low to middle income are most at risk from the T2D “epidemic” and may find implementing preventative measures challenging; yet prevention has largely been evaluated in developed countries. Methods Markov cohort simulations explored costs and benefits of various prediabetes management approaches, expressed as “savings” to the public health care system, for three countries with high prediabetes prevalence and contrasting economic status (Poland, Saudi Arabia, Vietnam). Two scenarios were compared up to 15 y: “inaction” (no prediabetes intervention) and “intervention” with metformin extended release (ER), intensive lifestyle change (ILC), ILC with metformin (ER), or ILC with metformin (ER) “titration.” Results T2D was the highest‐cost health state at all time horizons due to resource use, and inaction produced the highest T2D costs, ranging from 9% to 34% of total health care resource costs. All interventions reduced T2D versus inaction, the most effective being ILC + metformin (ER) “titration” (39% reduction at 5 y). Metformin (ER) was the only strategy that produced net saving across the time horizon; however, relative total health care system costs of other interventions vs inaction declined over time up to 15 y. Viet Nam was most sensitive to cost and parameter changes via a one‐way sensitivity analysis. Conclusions Metformin (ER) and lifestyle interventions for prediabetes offer promise for reducing T2D incidence. Metformin (ER) could reduce T2D patient numbers and health care costs, given concerns regarding adherence in the context of funding/reimbursement challenges for lifestyle interventions.

Abstract Introduction Cervical cancer ranks as the third most prevalent cancer among women in Thailand. However, the effectiveness of cervical cancer screening programs is limited by several factors that impede the screening rate. The utilization of self-collected samples for screening purposes has the potential to alleviate barriers to screening in Thai women. This study assessed the cost-utility and budget impact of implementing cervical cancer screening using self-collected samples for human papillomavirus (HPV) deoxyribonucleic acid (DNA) testing in Thailand. Materials and methods We employed a decision tree integrated with a Markov model to estimate the lifetime costs and health benefits associated with the cervical cancer screening program for women aged 25–65. The analysis was conducted from a societal perspective. Four screening policy options were compared: (1) additional self-collected samples for HPV DNA testing, (2) clinician-collected samples for HPV DNA testing only, (3) clinician-collected samples for cytology test (i.e., status quo), and (4) no screening. The model inputs were based on unvaccinated women. The screening strategies and management in those with positive results were assumed followed to the Thai clinical practice guideline. Costs were reported in 2022 Thai baht. Sensitivity analyses were conducted. The ten-year budget impacts of the additional self-collected samples for HPV DNA testing were calculated from a payer perspective. Results All screening policies were cost-saving compared to no screening. When comparing the additional self-collected samples for HPV DNA testing with the clinician-collected samples policy, it emerged as the dominant strategy. The incremental benefit in cervical cancer prevention achieved by incorporating self-collected samples for screening was observed at any additional screening rate that could be achieved through their use. Sensitivity analyses yielded consistently favorable results for the screening policies. The average annual budget impact of the additional self-collected samples for screening policy amounted to 681 million Thai baht. This budget allocation could facilitate cervical cancer screening for over 10 million women. Conclusions An addition of self-collected samples for HPV DNA testing into the cervical cancer screening program is cost-saving. The benefits of this screening policy outweigh the associated incremental costs. Policymakers should consider this evidence during the policy optimization process.

Abstract Background To estimate the economic impact of preventing urinary tract infections (UTI) by increasing water intake among women with recurrent UTI and low fluid intake across seven countries: France, United Kingdom, Spain, United States of America, Mexico, China and Australia. Methods A Markov model was developed to compare costs and outcomes of UTIs associated with low fluid intake in women versus a strategy of primary prevention by increasing water intake. Model inputs were based on randomized controlled trial data which found that increasing water intake by 1.5 L/day decreased the risk of developing cystitis by 48% in women with low fluid intake and recurrent UTI. A time horizon of 10 years was used; outcomes were from the payer perspective and included both direct and indirect costs, reported in 2019 United States dollars ($). Discounting rates varied by country. Scenarios of increasing levels of compliance to the increased water intake strategy were evaluated. Results The total cost of one UTI episode, including diagnostics, treatment and complications, ranged from $2164 (Mexico) to $7671 (Australia). Assuming 80% compliance with the increased water intake strategy over a 10-year time horizon, the number of UTIs prevented ranged from 435,845 (Australia) to 24150,272 (China), resulting in total savings of 286 million (Australia) to $4.4 billion (China). Across all countries, increased water intake resulted in lower cost and fewer UTIs compared with low water intake. Conclusion Preventing recurrent UTIs by increasing water intake would reduce both the clinical and economic burden associated with UTI. Public, healthcare professionals and patients should be made aware about the preventive positive impact of appropriate water intake on UTIs.

Two vaccines against rotavirus diseases, Rotarix® and RotaTeq®, are being marketed in Spain; but rotavirus is not presently among the diseases covered by universal vaccination in Spain. The aim of this study was to assess the efficiency of extending Spain’s current targeted rotavirus vaccination strategy including only preterm babies, to a policy of universal vaccination. A de novo cohort-based Markov model was built to evaluate the efficiency of three compared rotavirus vaccination strategies in Spain: targeted, universal, and no vaccination. Using Rotarix® or RotaTeq®, we compared the cost–utility of these strategies from both a societal perspective and Spanish National Health System (SNHS) perspective. The model represents the most important clinical events conceivably linked to rotavirus infection. Efficacy, effectiveness, safety, costs, and utilities were identified by systematic reviews. Incremental cost–utility ratio (ICUR) is EUR 23,638/QALY (Quality-Adjusted Life Year) for targeted vaccination with Rotarix® compared with no vaccination. The ICUR for the rest of the strategies evaluated are above EUR 30,000/QALY. The sensitivity analysis shows price as the only parameter that could make the universal vaccination strategy efficient. Considering a threshold of EUR 25,000/QALY, only targeted vaccination with Rotarix® would be efficient from societal perspective. Price drops of 36.9% for Rotarix® and 44.6% for RotaTeq® would make universal vaccination efficient.

Abstract Background India had an estimated 2.9 million tuberculosis cases and 506 thousand deaths in 2021. Novel vaccines effective in adolescents and adults could reduce this burden. M72/AS01E and BCG-revaccination have recently completed phase IIb trials and estimates of their population-level impact are needed. We estimated the potential health and economic impact of M72/AS01E and BCG-revaccination in India and investigated the impact of variation in vaccine characteristics and delivery strategies. Methods We developed an age-stratified compartmental tuberculosis transmission model for India calibrated to country-specific epidemiology. We projected baseline epidemiology to 2050 assuming no-new-vaccine introduction, and M72/AS01E and BCG-revaccination scenarios over 2025–2050 exploring uncertainty in product characteristics (vaccine efficacy, mechanism of effect, infection status required for vaccine efficacy, duration of protection) and implementation (achieved vaccine coverage and ages targeted). We estimated reductions in tuberculosis cases and deaths by each scenario compared to the no-new-vaccine baseline, as well as costs and cost-effectiveness from health-system and societal perspectives. Results M72/AS01E scenarios were predicted to avert 40% more tuberculosis cases and deaths by 2050 compared to BCG-revaccination scenarios. Cost-effectiveness ratios for M72/AS01E vaccines were around seven times higher than BCG-revaccination, but nearly all scenarios were cost-effective. The estimated average incremental cost was US$190 million for M72/AS01E and US$23 million for BCG-revaccination per year. Sources of uncertainty included whether M72/AS01E was efficacious in uninfected individuals at vaccination, and if BCG-revaccination could prevent disease. Conclusions M72/AS01E and BCG-revaccination could be impactful and cost-effective in India. However, there is great uncertainty in impact, especially given the unknowns surrounding the mechanism of effect and infection status required for vaccine efficacy. Greater investment in vaccine development and delivery is needed to resolve these unknowns in vaccine product characteristics.

BackgroundRefractory angina (RA) is a chronic condition characterized by the presence of debilitating angina symptoms due to established reversible ischemia in the presence of obstructive coronary artery disease (CAD). Treatments for this condition have undergone major developments in recent decades; however, the treatment for RA remains a challenge for medicine. In this sense, the Coronary Sinus Reducer System (CSRS) stands as the last line of therapy for ineligible patients for revascularization with reversible ischemia. The purpose of this report is to evaluate the potential burden on the National Health Service (NHS) and measure the health effects in terms of both quantity (life years) and quality-of-life aspects related to the reducer.MethodsTwo different economic evaluation models were developed as part of the analysis. The budget impact was developed to estimate the potential burden on the NHS from incremental uptake of the use of the reducer in the target population. The utility cost analysis compares and evaluates the quality of life and health resource use and costs between the two alternatives, based on the research of Gallone et al. A deterministic and probabilistic sensitivity analysis was carried out to characterize the uncertainty around the parameters of the model.ResultsIn the budget impact analysis (BIA), the reducer is shown to be more expensive in the first 2 years of the model, due to the gradual uptake in the market and the cost of the device. Starting from the third year, assuming maintenance of effectiveness, there are savings in terms of resource absorption in direct healthcare costs arising from hospitalizations, emergency department accesses, coronarography, and visits avoided.ConclusionThe BIA and cost-effectiveness model show that the reducer device, despite an increase in resources absorbed in the first years of implementation and use, has the potential to result in increased quality of life in patients with RA. These costs are largely offset in the short term by the improved clinical outcomes achievable leading to savings from the third year onward in the BIA and a dominance ratio in the cost–utility analysis.

Aim: Posterior cranial fossa (PCF) surgery is associated with complications, including cerebrospinal fluid (CSF) leakage. Dural sealants such as polyethylene glycol (PEG)-based hydrogels and fibrin glue can prevent CSF leaks, with evidence suggesting PEG hydrogels may outperform fibrin glue. However, the budget impact of using PEG hydrogels in PCF surgeries in Europe is unclear. Materials & methods: A decision tree was developed based on a previous US model, to assess the budget impact of switching from fibrin glue to PEG hydrogel in PCF surgery across five European countries. Input costs were derived from published sources for the financial year 2022/2023. Health outcomes, including CSF leaks, were considered. Results: The model predicted that using PEG hydrogel instead of fibrin glue in PCF surgery can lead to cost savings in five European countries. Cost savings per patient ranged from EUR 419 to EUR 1279, depending on the country. Sensitivity analysis showed that the incidence of CSF leaks and pseudomeningoceles had a substantial impact on the model’s results. Conclusion: PEG hydrogels may be a cost-effective alternative to fibrin glue in PCF surgery. The model predicted that cost savings would be mainly driven by a reduction in the incidence of postoperative CSF leaks, resulting in reduced reliance on lumbar drains, reparative surgery and shortened hospital stays.

South Korea has adopted a unique approach to address drug shortages by increasing reimbursement prices within its National Health Insurance Service. This study aims to analyze the characteristics, increase rates, affecting factors, and budget impacts of products that have increased price through the negotiation system. Between 2007 and 2022, there were price increase negotiations over 244 items. Of these, price increase negotiations were successful for 217 items, resulting in an agreement rate of 89%. The average rate of price increase for the agreed-upon products was 37.8%, and the overall budget increase for drugs with price increases (n = 217) was approximately 24.5%. Budget impact of each variable of the negotiated agreements showed that the number of negotiated agreement items was smaller after 2015 than before 2015, but each total budget impact (initial budget, increased budget, and final budget) and the average budget impact were higher. Although domestic companies accounted for a larger overall budget, the average budget per item was larger for multinational companies. The correlation analysis of the ratio of price increase and budget impact variables showed that the ratio of price increase was positively and significantly correlated with the increased budget, while it was negatively but not significantly correlated with the initial and final budgets. The South Korean model of increasing reimbursement prices in public insurance for drugs at risk of shortages serves as an exemplary case for not only securing patient access but also considering budget management. [ABSTRACT FROM AUTHOR]

Introduction: Cervical cancer ranks as the third most prevalent cancer among women in Thailand. However, the effectiveness of cervical cancer screening programs is limited by several factors that impede the screening rate. The utilization of self-collected samples for screening purposes has the potential to alleviate barriers to screening in Thai women. This study assessed the cost-utility and budget impact of implementing cervical cancer screening using self-collected samples for human papillomavirus (HPV) deoxyribonucleic acid (DNA) testing in Thailand. Materials and methods: We employed a decision tree integrated with a Markov model to estimate the lifetime costs and health benefits associated with the cervical cancer screening program for women aged 25–65. The analysis was conducted from a societal perspective. Four screening policy options were compared: (1) additional self-collected samples for HPV DNA testing, (2) clinician-collected samples for HPV DNA testing only, (3) clinician-collected samples for cytology test (i.e., status quo), and (4) no screening. The model inputs were based on unvaccinated women. The screening strategies and management in those with positive results were assumed followed to the Thai clinical practice guideline. Costs were reported in 2022 Thai baht. Sensitivity analyses were conducted. The ten-year budget impacts of the additional self-collected samples for HPV DNA testing were calculated from a payer perspective. Results: All screening policies were cost-saving compared to no screening. When comparing the additional self-collected samples for HPV DNA testing with the clinician-collected samples policy, it emerged as the dominant strategy. The incremental benefit in cervical cancer prevention achieved by incorporating self-collected samples for screening was observed at any additional screening rate that could be achieved through their use. Sensitivity analyses yielded consistently favorable results for the screening policies. The average annual budget impact of the additional self-collected samples for screening policy amounted to 681 million Thai baht. This budget allocation could facilitate cervical cancer screening for over 10 million women. Conclusions: An addition of self-collected samples for HPV DNA testing into the cervical cancer screening program is cost-saving. The benefits of this screening policy outweigh the associated incremental costs. Policymakers should consider this evidence during the policy optimization process. [ABSTRACT FROM AUTHOR]

Background: To estimate the economic impact of preventing urinary tract infections (UTI) by increasing water intake among women with recurrent UTI and low fluid intake across seven countries: France, United Kingdom, Spain, United States of America, Mexico, China and Australia. Methods: A Markov model was developed to compare costs and outcomes of UTIs associated with low fluid intake in women versus a strategy of primary prevention by increasing water intake. Model inputs were based on randomized controlled trial data which found that increasing water intake by 1.5 L/day decreased the risk of developing cystitis by 48% in women with low fluid intake and recurrent UTI. A time horizon of 10 years was used; outcomes were from the payer perspective and included both direct and indirect costs, reported in 2019 United States dollars ($). Discounting rates varied by country. Scenarios of increasing levels of compliance to the increased water intake strategy were evaluated. Results: The total cost of one UTI episode, including diagnostics, treatment and complications, ranged from $2164 (Mexico) to $7671 (Australia). Assuming 80% compliance with the increased water intake strategy over a 10-year time horizon, the number of UTIs prevented ranged from 435,845 (Australia) to 24150,272 (China), resulting in total savings of 286 million (Australia) to $4.4 billion (China). Across all countries, increased water intake resulted in lower cost and fewer UTIs compared with low water intake. Conclusion: Preventing recurrent UTIs by increasing water intake would reduce both the clinical and economic burden associated with UTI. Public, healthcare professionals and patients should be made aware about the preventive positive impact of appropriate water intake on UTIs. [ABSTRACT FROM AUTHOR]

ContextSeveral liquid- and tissue-based biomarker tests (LTBTs) are available to inform the need for prostate biopsies and treatment of localised prostate cancer (PCa) through risk stratification, but translation into routine practice requires evidence of their clinical utility and economic impact.ObjectiveTo review and summarise the health economic evidence on the ability of LTBTs to inform decisions on prostate biopsies and treatment of localised PCa through risk stratification.Evidence acquisitionA systematic search was performed in the EMBASE, MEDLINE, Health Technology Assessment, and National Health Service Health Economic Evaluation databases. Eligible publications were those presenting health economic evaluations of an LTBT to select individuals for biopsy or risk-stratify PCa patients for treatment. Data on the study objectives, context, methodology, clinical utility, and outcomes were extracted and summarised.Evidence synthesisOf the 22 studies included, 14 were focused on test-informed biopsies and eight on treatment selection. Most studies performed cost-effectiveness analyses (n = 7), followed by costing (n = 4) or budget impact analyses (n = 3). Most (18 of 22) studies concluded that biomarker tests could decrease health care costs or would be cost-effective. However, downstream consequences and long-term outcomes were typically not included in studies that evaluated LTBT to inform biopsies. Long-term effectiveness was modelled by linking evidence from different sources instead of using data from prospective studies.ConclusionsAlthough studies concluded that LTBTs would probably be cost-saving or -effective, the strength of this evidence is disputable because of concerns around the validity and transparency of the assumptions made. This warrants prospective interventional trials to inform health economic analyses to ensure collection of direct evidence of clinical outcomes based on LTBT use.Patient summaryWe reviewed studies that evaluated whether blood, urine, and tissue tests can reduce the health and economic burden of prostate cancer. Results indicate that these tests could be cost-effective, but clinical studies of long-term outcomes are needed to confirm the findings.

Abstract Background Chronic obstructive pulmonary disease (COPD) is a progressive debilitating condition with frequent exacerbations that have a high burden for patients and society. Digital tools may help to reduce the economic burden for patients and payers by improving outcomes. The Propeller platform is a digital self-management tool that facilitates passive monitoring of inhaler medication utilization, potentially assisting the healthcare team to identify patients at risk of a COPD exacerbation who may require further intervention. This study estimated the budget impact of Propeller from commercial payer and Medicare fee-for-service payer perspectives. Methods An Excel-based model was used to estimate the budget impact of Propeller for COPD patients in commercial and Medicare population sizes of 5 million members. Data on prevalence, baseline healthcare resource utilization (HCRU), and baseline use of rescue and controller inhaler medications with unit costs (adjusted to 2020 US dollars) were obtained from peer-reviewed literature. Data on reductions in HCRU during Propeller usage were based on direct evidence. Estimates for costs of remote monitoring were obtained from publicly available information. All patients were assumed to have insurance claims related to ongoing remote monitoring. Results The estimated number of annual eligible COPD patients for commercial and Medicare was 212,200 and 606,600, respectively. Propeller decreased costs by an estimated $2,475 (commercial) and $915 (Medicare) per enrolled patient. The greatest increase in expenditure was for remote monitoring related expenses. After accounting for estimated reductions in hospitalizations, emergency department visits and short-acting beta-agonist use, total net savings were approximately $1.60 and $1.70 per-member per-month for commercial and Medicare payers, respectively. Conclusion Propeller is projected to be cost saving from both the commercial and Medicare payer perspectives.

Costs of hemophilia A treatment are increasing. Waste of clotting products should be avoided. To estimate the first-year waste of emicizumab prophylaxis for people with hemophilia A and inhibitors (PwHAi) who failed immune tolerance induction (ITI), in Brazil. We evaluated the manufacturer and the Brazilian Ministry of Health (MoH) protocol-recommended regimens in a budget impact model. The loading dose consisted of 3.0 mg/kg/Q1W for 4 weeks, for both recommendations. The manufacturer maintenance regimens comprised 1.5 mg/kg/Q1W, 3.0 mg/kg/Q2W, and 6.0 mg/kg/Q4W. The MoH protocol maintenance regimen encompassed a hybrid Q1W/Q2W administration, depending on the body weight. The Q4W regimen was not recommended by the MoH protocol. Analyses were performed to estimate waste given its expense based on the World Health Organization body weight range (percentiles [P] 15, 50, and 85). The first-year emicizumab waste was estimated individually and for the disclosed PwHAi who failed ITI (n = 114). The highest emicizumab waste was estimated for the lowest body weights and the Q1W regimen. The Q4W regimen resulted in the lowest emicizumab waste, followed by the MoH protocol regimen. The total reconstituted costs estimated for the PwHAi who failed ITI according to the hybrid MoH protocol ranged from US$32,858,777 (P15) to US$47,186,858 (P85), with emicizumab waste ranging from 7.9 % (US$2,594,515) to 3.7 % (US$1,738,750), respectively. Lost resources due to current protocols for emicizumab prophylaxis for PwHAi who failed ITI in Brazil are considerable. Waste was more pronounced due to lower body weight and shorter administration intervals.

Background: India had an estimated 2.9 million tuberculosis cases and 506 thousand deaths in 2021. Novel vaccines effective in adolescents and adults could reduce this burden. M72/AS01E and BCG-revaccination have recently completed phase IIb trials and estimates of their population-level impact are needed. We estimated the potential health and economic impact of M72/AS01E and BCG-revaccination in India and investigated the impact of variation in vaccine characteristics and delivery strategies. Methods: We developed an age-stratified compartmental tuberculosis transmission model for India calibrated to country-specific epidemiology. We projected baseline epidemiology to 2050 assuming no-new-vaccine introduction, and M72/AS01E and BCG-revaccination scenarios over 2025–2050 exploring uncertainty in product characteristics (vaccine efficacy, mechanism of effect, infection status required for vaccine efficacy, duration of protection) and implementation (achieved vaccine coverage and ages targeted). We estimated reductions in tuberculosis cases and deaths by each scenario compared to the no-new-vaccine baseline, as well as costs and cost-effectiveness from health-system and societal perspectives. Results: M72/AS01E scenarios were predicted to avert 40% more tuberculosis cases and deaths by 2050 compared to BCG-revaccination scenarios. Cost-effectiveness ratios for M72/AS01E vaccines were around seven times higher than BCG-revaccination, but nearly all scenarios were cost-effective. The estimated average incremental cost was US$190 million for M72/AS01E and US$23 million for BCG-revaccination per year. Sources of uncertainty included whether M72/AS01E was efficacious in uninfected individuals at vaccination, and if BCG-revaccination could prevent disease. Conclusions: M72/AS01E and BCG-revaccination could be impactful and cost-effective in India. However, there is great uncertainty in impact, especially given the unknowns surrounding the mechanism of effect and infection status required for vaccine efficacy. Greater investment in vaccine development and delivery is needed to resolve these unknowns in vaccine product characteristics. [ABSTRACT FROM AUTHOR]

Minimally invasive glaucoma surgery devices fill an unmet need in the treatment paradigm between topical intraocular pressure medicines and more invasive filtration procedures. This study evaluated the adoption of The OMNI® Surgical System with or without cataract surgery in primary open-angle glaucoma patients. A budget impact analysis estimated costs before and after adoption of OMNI® to a hypothetical US health plan with 1 million Medicare-covered lives over two years. Model input data were derived from published sources and development of the model included primary research with key opinion leaders and payers. The model compared total annual direct costs for OMNI® versus other treatment options (medications, other minimally invasive surgical procedures, selective laser trabeculoplasty) to calculate budget impact. A one-way sensitivity analysis was conducted to assess parameter uncertainty. Increased adoption of OMNI® resulted in budget neutrality over the two years with a decrease in total costs of $35,362. Per member per month incremental costs were $0.00 when used without cataract surgery and yielded cost savings of -$0.01 when used with cataract surgery. Sensitivity analysis confirmed model robustness and identified surgical center fee variability as a key driver of costs. OMNI® is budgetary efficient from a US payer perspective. [ABSTRACT FROM AUTHOR]

Background: The phase 3 clinical trial KEYNOTE-426 suggested a higher efficacy regarding overall survival (OS) and progression-free survival (PFS) of pembrolizumab+axitinib compared to sunitinib as a first-line treatment for patients with advanced renal cell carcinoma. In this analysis, the potential cost-effectiveness of this combination treatment versus sunitinib for patients with advanced clear-cell renal cell carcinoma (accRCC) was examined from the societal perspective in the Netherlands. Methods: For this analysis, a partitioned survival model was constructed. Clinical data were obtained from the published KEYNOTE-426 trial reports; data on costs and (dis-)utilities were derived from published literature. Costs outside of the healthcare sector included treatment-related travel, informal care and productivity loss. Next to a probabilistic scenario analysis, various scenario analyses were performed that aimed at survival extrapolation, different utility values, treatment duration and drug pricing, as well as restricting the cohort to patients with an intermediate or poor prognosis. Further, a budget impact analysis over three years was conducted, in which a sensitivity analysis concerning ranges in costs and the number of patients was applied. Moreover, a scenario concerning increasing market penetration of pembrolizumab+axitinib up to a market share of 80% in the third year was analyzed. Results: The incremental cost-effectiveness ratio (ICER) of pembrolizumab +axitinib was estimated at €368,396/quality-adjusted life year (QALY) gained, with an incremental QALY gain of 0.55 over sunitinib. The probability of costeffectiveness at a willingness-to-pay threshold of €80,000/QALY was estimated at 0%, a 50% probability was estimated at €340,000/QALY. Cost-effectiveness was not achieved in any of the applied scenarios. The budget impact over three years amounted to €417.3 million upon instantaneous and full replacement of sunitinib, and to €214.9 million with increasing market penetration. Conclusion: Pembrolizumab+axitinib was not estimated to be cost-effective compared to sunitinib as a first-line treatment for patients with accRCC in the Netherlands from a societal perspective. In none of the analyzed scenarios, costeffectiveness was achieved. However, price reductions and shorter treatment durations might lead to a more favorable ICER. [ABSTRACT FROM AUTHOR]

This study aimed to test (official) evaluation criteria including the potential role of budget impact (BI) on health technology assessment (HTA) outcomes published by the Federal Joint Committee (Gemeinsamer Bundesausschuss [GBA]) and the Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen [IQWiG]) in Germany as well as the National Institute for Health and Care Excellence (NICE) in England. Data were extracted from all publicly available GBA decisions and IQWiG assessments as well as NICE single technology appraisals between January 2011 and June 2018, and information with regard to evaluation criteria used by these agencies was collected. Data were analyzed using logistic regression to estimate the effect of the BI on the HTA outcomes while controlling for criteria used by GBA/IQWiG and NICE. NICE recommendations are largely driven by the incremental cost-effectiveness ratio and, if applicable, by end-of-life criteria (P <.01). While IQWiG assessments are significantly affected by the availability of randomized controlled trials and patient-relevant endpoints (P <.01), GBA appraisals primarily focus on endpoints (P <.01). The BI correlated with NICE single technology appraisals (inverted-U relationship, P <.1) and IQWiG recommendations (increasing linear relationship, P <.05), but not with GBA decisions (P >.1). Nevertheless, given that IQWiG assessments seem to be more rigorous than GBA appraisals regarding the consideration of evidence-based evaluation criteria, decisions by GBA might be negatively associated with the BI. Results reveal that GBA/IQWiG and NICE follow their official evaluation criteria consistently. After controlling for all significant variables, the BI seems to have an (independent) effect on HTA outcomes as well. • Empirical studies usually focus on the effect of evaluation criteria on recommendations by health technology assessment (HTA) agencies or compare assessment outcomes and underlying regulations. • Findings add to the existing evidence that both the Federal Joint Committee (Gemeinsamer Bundesausschuss [GBA])/Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen [IQWiG]) and the National Institute for Health and Care Excellence (NICE) follow their evaluation criteria consistently, as also reflected in the frequent occurrence of variation in recommendations. Furthermore, budget impact seems to have an effect on HTAs by GBA/IQWiG and NICE. • This article will contribute to health policy making in Europe, in particular to the debate whether and, if so, to what extent budget impact analyses shall be included in HTA to inform decision makers in England and Germany. [ABSTRACT FROM AUTHOR]

This study aimed to estimate the financial and economic impact of sacubitril/valsartan compared with enalapril for the treatment and prevention of hospitalization/rehospitalization because of heart failure with reduced ejection fraction (HFrEF). The budget impact analysis was guided by the Philippine Reference Case and ISPOR's Principles of Good Practice for Budget Impact Analysis. A government-funded healthcare payer perspective and a societal perspective were considered. Data collection was guided by the pathways of disease progression and care. Collection of costing data followed a bottom-up approach. The model was based on a Markov model used in a study in Thailand. Over the next 5 years, there will be 17 625 less hospitalizations (∼5.1% less than enalapril arm) and 7968 less cardiovascular-related deaths (∼7.0% less than enalapril arm). In 5 years, the total cost of treating patients with HFrEF with sacubitril/valsartan at current market coverage and annual growth conditions is ₱15.430 billion, which is ₱11.077 billion higher than fully treating with enalapril only. The total required additional investment with treatment of sacubitril/valsartan compared with the full enalapril arm are ₱407 million (at 30-day coverage), ₱800 million (at 60-day coverage), and ₱1.181 billion (at 90-day coverage). If hospitalizations costs alone are considered, only the 30-day coverage is cost-saving. If a societal perspective is considered, all options are cost-saving where at least ₱4.003 billion is saved by the economy. The initial investment required to treat patients with HFrEF with sacubitril/valsartan is high; nevertheless, the year-on-year cost deficit shrinks in favor of investing in sacubitril/valsartan treatment. • There is no study, to date, demonstrating the budget impact of covering sacubitril/valsartan in the Philippines, which is needed to guide decision makers in assessing whether the adoption of a new health technology is affordable, given the budget constraints. • This study showed that although the initial cost to treat patients with heart failure with reduced ejection fraction (HFrEF) with sacubitril/valsartan in the Philippines is high, its additional cost shrinks over time particularly if a societal perspective is adopted, favoring its coverage for treating HFrEF. • Coverage of sacubitril/valsartan in the Philippines could be considered to prevent hospitalization or rehospitalization because of HFrEF, and this could be affordable if the increased productivity is considered. [ABSTRACT FROM AUTHOR]

Background: Correct diagnosis of maturity-onset diabetes of the young (MODY), which is often misdiagnosed as Type 1 or 2 diabetes, is important for providing appropriate treatment. Materials & Methods: A diabetes model was adapted to Hungary, the Netherlands, and the UK to analyse the cost–effectiveness and budget impact of different screening strategies for MODY with 20 years time horizon. Results: Compared with no screening, screening with the MODY calculator then genetic testing is considered cost-effective with respect to each country's willingness to pay threshold. The addition of autoantibody testing dominated the no screening strategy. The budget impact of the strategies ranges between 0.001 and 0.025% of annual public healthcare spending. Conclusion: The analysed strategies are considered good value for money with potential cost savings in the long term. [ABSTRACT FROM AUTHOR]

Abstract Introduction Low- and middle-income countries (LMICs) are committed to achieving the Sustainable Development Goal 3.1 to reduce maternal mortality. The Ministry of Health and Family Welfare of India recommends prophylactic uterotonic administration to every woman following delivery to reduce the risk of postpartum hemorrhage (PPH), as PPH is the leading cause of maternal mortality in LMICs, including India. In 2018, the World Health Organization first recognized heat-stable carbetocin for PPH prevention. Governments are now considering its introduction into their public health systems. Methods A decision-tree model was developed from the public healthcare system perspective to compare the value of heat-stable carbetocin versus oxytocin and misoprostol among women giving birth in public sector healthcare facilities in India. The model accounted for differences in PPH risk and costs based on mode of delivery and healthcare setting, as well as provider behavior to mitigate quality concerns of oxytocin. Model outcomes for each prophylactic uterotonic included the number of PPH events, DALYs due to PPH, deaths due to PPH, and direct medical care costs. The budget impact was estimated based on projected uterotonic uptake between 2022–2026. Results Compared to oxytocin, heat-stable carbetocin avoided 5,468 additional PPH events, 5 deaths, and 244 DALYs per 100,000 births. Projected direct medical costs to the public healthcare system were lowered by US $171,700 (₹12.8 million; exchange rate of ₹74.65 = US$1 from 2 Feb 2022) per 100,000 births. Benefits were even greater when compared to misoprostol (7,032 fewer PPH events, 10 fewer deaths, 470 fewer DALYs, and $230,248 saved per 100,000 births). In the budget impact analysis, India’s public health system is projected to save US$11.4 million (₹849 million) over the next five years if the market share for heat-stable carbetocin grows to 19% of prophylactic uterotonics administered. Conclusions Heat-stable carbetocin is expected to reduce the number of PPH events and deaths, avoid more DALYs, and reduce costs to the public healthcare system of India. Greater adoption of heat-stable carbetocin for the prevention of PPH could advance India’s efforts to achieve its maternal health goals and increase efficiency of its public health spending.

Vaccination is one of the most powerful and affordable means in the history of public health. It is the most effective method of preventing infectious diseases, as immunizations save millions of people from disease, disability and death each year. Vaccines are also now available against a number of non-infectious, autoimmune and even oncological diseases. A significant number of combination vaccines (mainly for pediatric use) have also been developed, which save parents’ time and are less traumatic for the child compared to the administration of several individual vaccines. Public healthcare authorities are involved in controlling and investigating each case of vaccine-related adverse effects or complications. Lifelong immunization (also known as life course immunization) is a process involving immunization with mandatory and recommended vaccines from birth throughout a person’s life, including vaccines that are recommended for adult patients. Better use of existing vaccines and the development of new ones are successful solutions to reduce the incidence of preventable diseases and subsequent deaths.