One-year budget impact of InTandem™: a novel neurorehabilitation system for individuals with chronic stroke walking impairment.

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.

Cost and budget impact of mass drug administration compared to expanded school-based targeted preventive chemotherapy for soil-transmitted helminth control in Zamboanga Peninsula, the Philippines.

Background: School-based targeted preventive chemotherapy (PC), the primary strategy for soil-transmitted helminth (STH) control, typically focusing on primary schoolchildren, was expanded to secondary school students in the Philippines in 2016. This program still excludes adults, who may also suffer from considerable morbidity and can be a significant reservoir of infection. Mass drug administration (MDA), where the entire population is treated, would bring additional health benefits but will also increase implementation costs. The incremental cost of implementing MDA for STH control compared to expanded school-based targeted PC, however, is unknown. Methods: A cost survey was conducted in Zamboanga Peninsula region in 2021 to estimate the economic and financial cost of implementing MDA compared to the expanded school-based targeted PC from a government payer perspective. A budget impact analysis was conducted to estimate the financial cost to the government of implementing MDA over a five-year timeframe. Monte Carlo simulation accounted for uncertainty in cost estimates. Costs were reported in 2021 United States Dollars ($). Findings: The economic cost of MDA was $809,000 per year (95% CI: $679,000-$950,000) or $0.22 per person targeted (95% CI: $0.19-$0.26), while the expanded school-based targeted PC would cost $625,000 (95% CI: $549,000-$706,000) or $0.57 per person targeted (95% CI: $0.50-$0.64). Over five years, the financial cost to the government for MDA would be $3,113,000 (95% CI: $2,475,000-$3,810,000); $740,000 (95% CI: $486,000-$1,019,000) higher than expanded school-based targeted PC. Interpretation: Implementing MDA in the region will increase the economic and financial costs by 29% and 31%, respectively, when compared to expanded school-based targeted PC. Implementing MDA would require the Department of Health to increase their total expenditure for STH control by 0.2% and could be key in addressing the ongoing STH burden. Funding: The project was funded by the Australian Centre for the Control and Elimination of Neglected Tropical Diseases (NHMRC GA19028), and JPCDT was supported by a UNSW Scientia PhD Scholarship. SVN is funded by an NHMRC Investigator Grant (APP 2018220).

The budget impact of implementing atrial fibrillation-screening in European countries.

A budget impact analysis estimates the short-term difference between the cost of the current treatment strategy and a new treatment strategy, in this case to implement population screening for atrial fibrillation (AF). The aim of this study is to estimate the financial impact of implementing population-based AF-screening of 75-year-olds compared with the current setting of no screening from a healthcare payer perspective in eight European countries. The net budget impact of AF-screening was estimated in country-specific settings for Denmark, Germany, Ireland, Italy, Netherlands, Serbia, Spain, and Sweden. Country-specific parameters were used to allow for variations in healthcare systems and to reflect the healthcare sector in the country of interest. Similar results can be seen in all countries AF-screening incurs savings of stroke-related costs since AF treatment reduces the number of strokes. However, the increased number of detected AF and higher drug acquisition will increase the drug costs as well as the costs of physician- and control visits. The net budget impact per invited varied from €10 in Ireland to €122 in the Netherlands. The results showed the increased costs of implementing AF-screening were mainly driven by increased drug costs and screening costs. In conclusion, across Europe, though the initial cost of screening and more frequent use of oral anti-coagulants will increase the healthcare payers' costs, introducing population screening for AF will result in savings of stroke-related costs.

Budget impact of resmetirom for the treatment of adults with non-cirrhotic non-alcoholic steatohepatitis (NASH) with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis).

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.

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.

Economic Evaluation of an Integrated Care Program Compared to Conventional Care for Patients With Chronic Kidney Disease in Rural Communities of Thailand.

Introduction: An integrated care program for chronic kidney disease (CKD) in Thailand has shown its effectiveness in delaying the decline in kidney function, as evidenced by the Effectiveness of Integrated Care on Delaying Progression of Stages 3 to 4 CKD in Rural Communities of Thailand (ESCORT-1) randomized control trial and the ESCORT-2 prospective cohort study. Designed for sustainability within the primary healthcare system, the program optimizes the use of the existing workforce by fostering collaboration among local multidisciplinary care teams (MDCTs) and community care networks (CCNs). Methods: A Markov model with a lifetime horizon was used to conduct a cost-utility analysis from a societal perspective. Individual participant level data from ESCORT studies, national registries, and relevant literature were used to estimate model parameters. A budget impact analysis from the payer's perspective was also assessed over a 5-year period. Results: The integrated care program yielded a dominant result with 1.84 quality-adjusted life years (QALYs) gained with "less" lifetime cost, resulting in a negative incremental cost-effectiveness ratio (ICER). Probabilistic analysis showed that the intervention being cost-effective almost 100% of the time at the local willingness-to-pay threshold. The intervention maximized cost-effectiveness when delivered as early as possible, both in terms of age and stage. The budget impact analysis estimated that the introduction of the intervention could save about 7% of the Thai government's total health expenditure or 205 billion Thai-Baht ($5.9 billion) over 5 years with cost savings beginning from the third year onwards. Conclusion: The integrated care program for CKD offers potential benefits and cost savings for patients, caregivers, and payers. Future efforts should focus on the screening and implementation processes across various regions and healthcare settings.

Budget impact analysis of introducing fruquintinib for metastatic colorectal cancer previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy and biologics in the United States from the payer perspective.

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.

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.

Budget impact and cost per responder analysis of upadacitinib for the treatment of moderate to severe atopic dermatitis from the perspective of the social security and the private sector in Argentina.

OBJECTIVES: Our study assessed the budget impact and cost per responder of upadacitinib15mg and 30 mg for moderate to severe atopic dermatitis (MS-AD) treatment from social security and private health sector perspective in Argentina. METHODS: A budget impact model was adapted to depict clinical and economic aspects of treatment over a 5-years horizon time. Scenario analyses and deterministic sensitivity analyses were performed. A 16-weeks cost per responder model was adapted based on a network meta-analysis. Primary analyses assessed the cost per Eczema Area and Severity Index 50, 75 and 90 at week 16. RESULTS: The inclusion of upadacitinib 15 mg and 30 mg in the biological treatment mix for MS-AD was associated with an average budget saving per-member per-month ofU$S0.062 (social security) and U$S0.064 (private sector). Percentage of patients with access to treatment, acquisition cost of upadacitinib 30 mg and prevalence of MS-AD were the most influential parameters in the budget impact results. At week 16, upadacitinib 30 mg was associated with the lowest number needed to treat and the lowest cost per responder for all outcomes. CONCLUSION: The introduction of upadacitinib in MS-AD treatment was associated with modest savings for the social security and private payer budget in Argentina.

Updating and Refining of Economic Evaluation of Rotavirus Vaccination in Spain: A Cost-Utility and Budget Impact Analysis.

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.

Budget impact analysis of introducing digital breast tomosynthesis in breast cancer screening in Italy.

PURPOSE: This study quantifies the impact on budget and cost per health benefit of implementing digital breast tomosynthesis (DBT) in place of digital mammography (DM) for breast cancer screening among asymptomatic women in Italy. METHODS: A budget impact analysis and a cost consequence analysis were conducted using parameters from the MAITA project and literature. The study considered four scenarios for DBT implementation, i.e., DBT for all women, DBT for women aged 45-49 years, DBT based on breast density (BI-RADS C + D or D only), and compared these to the current DM screening. Healthcare provider's perspective was adopted, including screening, diagnosis, and cancer treatment costs. RESULTS: Introducing DBT for all women would increase overall screening costs by 20%. Targeting DBT to women aged 45-49 years or with dense breasts would result in smaller cost increases (3.2% for age-based and 1.4-10.7% for density-based scenarios). The cost per avoided interval cancer was significantly higher when DBT was applied to all women compared to targeted approaches. The cost per gained early-detected cancer slightly increases in targeted approaches, while the assumptions on the clinical significance and overdiagnosis of cancers detected by DBT and not by DM have a strong impact. CONCLUSIONS: Implementing DBT as a primary breast cancer test in screening programs in Italy would lead to a substantial increase in costs. Tailoring DBT use to women aged 45-49 or with dense breasts could enhance the feasibility and sustainability of the intervention. Further research is needed to clarify the impact of DBT on overdiagnosis and the long-term outcomes.

Financial incentives to stop smoking: Potential financial consequences of different reward schedules.

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 cost-effectiveness 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.

U.S. payer budget impact of using an AI-augmented cancer risk discrimination digital histopathology platform to identify high-risk of recurrence in women with early-stage invasive breast cancer.

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.

Budget impact analysis of the novel PD-1 inhibitor toripalimab versus pembrolizumab in recurrent or metastatic nasopharyngeal carcinoma.

AIM: To estimate the budget impact of adding a toripalimab regimen as a treatment option to the existing pembrolizumab regimen, both including gemcitabine and cisplatin, in untreated recurrent/metastatic nasopharyngeal carcinoma (R/M NPC) using the published wholesale acquisition cost (WAC) and average sales price (ASP). METHODS: Budget impact analysis comparing a treatment mix "without" versus "with" the toripalimab regimen in the US eligible annual incident R/M NPC population, a 3-year time horizon, toripalimab/pembrolizumab market splits of 60/40 (Y1) and 80/20 (Y2/3), and medication adjustments for discontinuation or progression. Cost inputs included drugs, administration, and adverse event (AE) management. The models were replicated for a hypothetical 1-million-member health plan in which costs per-member-per-month (PMPM) and per-member-per-year (PMPY) were estimated. One-way (OWSA) and probabilistic sensitivity analyses (PSA) as well as scenario analyses were performed. RESULTS: In the "without" scenario, the 3-year WAC-based costs for the pembrolizumab regimen total $1,449,695,333 ($1,305,632,448 for treatment and $144,062,885 for managing AEs). In the "with" scenario, total 3-year costs for pembrolizumab decline to $380,012,135 with toripalimab adding $885,505,900 ($779,206,567 for treatment and $106,299,333 for AE management). Annual net savings range from $46,526,152 in 2024 to $71,194,214 in 2026, for 3-year savings of $184,177,298. Associated net savings in a 1-million-member health plan are $543,068 over 3 years with savings of $0.045 PMPM and $0.543 PMPY. The ASP-based model shows similar patterns with 3-year net savings of $174,235,983 in the US incident population and savings of $0.043 PMPM and $0.514 PMPY in a 1-million-member health plan. The PSA support base case findings; OWSA and scenario analyses reveal how parameter variability impacts results. CONCLUSION: Savings between $174 million and $184 million can be achieved from treating 60% of R/M NPC patients in year 1 and 80% in years 2 and 3 with the toripalimab regimen over a similar pembrolizumab regimen.

Toripalimab, a human monoclonal anti-body that targets PD-1, was recently approved by the US Food and Drug Administration (FDA) for the first-line treatment of adults with metastatic or recurrent, locally advanced nasopharyngeal carcinoma (NPC), in combination with gemcitabine and cisplatin. We evaluated how much it would cost a payor to cover the FDA-approved toripalimab plus gemcitabine and cisplatin regimen (the toripalimab regimen) to a non-FDA-approved pembrolizumab plus gemcitabine and cisplatin regimen (the pembrolizumab regimen). With no trial data available for such pembrolizumab regimen, we assumed that it would be comparable to the toripalimab regimen in efficacy and safety. Our model adopted a 3-year time horizon and assumed a 60/40 market share split in year 1 and an 80/20 market split in years 2 and 3. It included two US cost inputs: the wholesale acquisition cost (WAC) or "list price" at market entry and, as no average sales price (ASP) will be available for toripalimab for several quarters, a toripalimab price point of 80% of the pembrolizumab ASP. We adjusted for patients whose cancer progressed or who discontinued treatment to determine the number of fully-treated-patient-equivalents. We found that treating 60% of NPC patients in year 1 and 80% in years 2 and 3 with the toripalimab regimen instead of the pembrolizumab regimen generates, for the entire adjusted patient population, savings ranging from $174 million when using ASP to $184 million when using WAC.

A budget impact analysis of a digital monitoring solution in patients treated with oral anticancer agents: a medico-economic analysis of the randomized phase 3 CAPRI trial.

BACKGROUND/OBJECTIVES: Remote patient monitoring (RPM) has demonstrated numerous benefits in cancer care, including improved quality of life, overall survival, and reduced medical resource use. This study presents a budget impact analysis of a nurse navigator-led RPM program, based on the CAPRI trial, from the perspective of the French national health insurance (NHI). The study aimed to assess the impact of the program on medical resource utilization and costs. METHODS: Medical resource utilization data were collected from both medico-administrative sources and patient-reported questionnaires. Costs were calculated by applying unit costs to resource utilization and estimating the average monthly cost per patient. Sensitivity analyses were conducted to explore different perspectives and varying resource consumption. RESULTS: The analysis included 559 cancer patients participating in the CAPRI program. From the NHI perspective, the program resulted in average savings of €377 per patient over the 4.58-month follow-up period, mainly due to reduced hospitalizations. The all-payers perspective yielded even greater savings of €504 per patient. Sensitivity analyses supported the robustness of the findings. CONCLUSION: The budget impact analysis demonstrated that the CAPRI RPM program was associated with cost savings from the perspective of the NHI. The program's positive impact on reducing hospitalizations outweighed the additional costs associated with remote monitoring. These findings highlight the potential economic benefits of implementing RPM programs in cancer care. Further research is warranted to assess the long-term cost-effectiveness and scalability of such programs in the real-world settings.

Development Perspectives for Curative Technologies in Primary Demyelinating Disorders of the Central Nervous System with Neuromyelitis Optica Spectrum Disorder (NMOSD) and Myelin Oligodendrocyte Glycoprotein Antibody-Associated Disease (MOGAD) at the Forefront.

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.

Estimating the Economic Impact of Levalbuterol's Potential Transition From the National Reimbursement Drug List for the Treatment of Pediatric Asthma in China: A Budget Impact Analysis.

Background Levalbuterol is a short-acting ß2-agonist (SABA) indicated for treating or preventing asthma exacerbation. It was included in the 2020 Chinese National Reimbursement Drug List (NRDL). This study estimates the economic impact of levalbuterol's status change within and withdrawal from the NRDL in treating pediatric asthma from a publicly funded medical insurance perspective. Methodology A prevalence-based budget impact model was developed. The analysis compared a world with a levalbuterol scenario to a world without levalbuterol. Epidemiological data were obtained from the existing literature. Cost data were estimated based on the drug dosage in clinical trials, real-world settings, and expert opinions. Scenario analysis considered the same length of stay (LOS) in the two groups. One-way sensitivity analyses were carried out to show the impact of varying individual parameters. Results In the base-case analysis, compared to the world without scenario, the preservation of levalbuterol resulted in cost savings of ¥82.8 million in China over three years. In the scenario analysis, savings decreased to ¥76.1 million over three years. Sensitivity analysis showed that, for the most part, the results were robust to changes in input parameter values. Conclusions Using levalbuterol may lead to substantial cost savings for Chinese society.

Estimating the budget impact of a Tuberculosis strategic purchasing pilot study in Medan, Indonesia (2018-2019).

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.

Cost-Effectiveness and Budget Impact Analyses of Selective Internal Radiation Therapy versus Atezolizumab Plus Bevacizumab from a German Statutory Health Insurance Perspective.

Purpose: 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 (≈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.

Informing evidence-based medicine for opioid use disorder using pharmacoeconomic studies.

INTRODUCTION: The health and economic consequences of inadequately treated opioid use disorder (OUD) are substantial. Healthcare systems in the United States (US) and other countries are facing a growing healthcare crisis due to opioids. Although effective medications for OUD exist, relying solely on clinical information is insufficient for addressing the opioid crisis. AREAS COVERED: In this review, the role of pharmacoeconomic studies in informing evidence-based medication treatment for OUD is discussed, with a particular emphasis on the US healthcare system, where the economic burden is significantly higher than the global average. The scope/objective of pharmacoeconomics as a distinct scientific research program is briefly defined, followed by a discussion of existing evidence informed by data from systematic reviews, in addition to a convenience sample of recently published pharmacoeconomic studies and protocols. The review also explores the need for methodological advancements in the field. EXPERT OPINION: Despite the potential of pharmacoeconomic research in shaping evidence-based medicine for OUD, significant challenges limiting its real-world application remain. How to address these challenges are explored, including how to combine cost-effectiveness and budget impact analyses to address the needs of the healthcare system as a whole and specific stakeholders interested in adopting new OUD treatment strategies.

Cost-Effectiveness and Budget Impact Analysis of the Implementation of Differentiated Service Delivery Models for HIV Treatment in Mozambique: a Modelling Study.

INTRODUCTION: In 2018, the Mozambique Ministry of Health launched guidelines for implementing differentiated service delivery models (DSDMs) to optimize HIV service delivery, improve retention in care, and ultimately reduce HIV-associated mortality. The models were fast-track, 3-month antiretrovirals dispensing, community antiretroviral therapy groups, adherence clubs, family approach and three one-stop shop models: adolescent-friendly health services, maternal and child health, and tuberculosis. We conducted a cost-effectiveness analysis and budget impact analysis to compare these models to conventional services. METHODS: We constructed a decision tree model based on the percentage of enrolment in each model and the probability of the outcome (12-month retention in treatment) for each year of the study period-three for the cost-effectiveness analysis (2019-2021) and three for the budget impact analysis (2022-2024). Costs for these analyses were primarily estimated per client-year from the health system perspective. A secondary cost-effectiveness analysis was conducted from the societal perspective. Budget impact analysis costs included antiretrovirals, laboratory tests and service provision interactions. Cost-effectiveness analysis additionally included start-up, training and clients' opportunity costs. Effectiveness was estimated using an uncontrolled interrupted time series analysis comparing the outcome before and after the implementation of the differentiated models. A one-way sensitivity analysis was conducted to identify drivers of uncertainty. RESULTS: After implementation of the DSDMs, there was a mean increase of 14.9 percentage points (95% CI: 12.2, 17.8) in 12-month retention, from 47.6% (95% CI, 44.9-50.2) to 62.5% (95% CI, 60.9-64.1). The mean cost difference comparing DSDMs and conventional care was US$ -6 million (173,391,277 vs. 179,461,668) and -32.5 million (394,705,618 vs. 433,232,289) from the health system and the societal perspective, respectively. Therefore, DSDMs dominated conventional care. Results were most sensitive to conventional care interaction costs in the one-way sensitivity analysis. For a population of 1.5 million, the base-case 3-year financial costs associated with the DSDMs was US$550 million, compared with US$564 million for conventional care. CONCLUSIONS: DSDMs were less expensive and more effective in retaining clients 12 months after antiretroviral therapy initiation and were estimated to save approximately US$14 million for the health system from 2022 to 2024.