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Aims: This economic model was developed to assess the budget impact of a novel radiotracer, Flurpiridaz (F 18 -PET-MPI), compared to SPECT-MPI from a US payer perspective., Materials and Methods: The model was developed comparing F 18 -PET-MPI and SPECT-MPI, with F 18 -PET-MPI modality share increasing from 0.5% to 2.5% of the total MPI modality share, over a 5-year time horizon. The model estimates the impact of diagnostic performance on downstream healthcare resource utilization (HCRU) including invasive coronary angiography (ICA), revascularization, pharmacological treatment, and cardiac outcomes (CO) such as cardiac mortality (CM) and myocardial infarction (MI). Four suspected CAD populations, including general and difficult-to-image subgroups, were analyzed. Clinical inputs used to support the parameterization of the model were sourced from a systematic literature search and included claims-based real-world evidence, observational, and multicenter registry studies to inform the rates of HCRU and CO, and head-to-head comparative clinical trial data advised diagnostic performance inputs. Reimbursement codes informed MPI modality costs. Results are reported as per-member per-month (PMPM) based on a hypothetical health plan., Results: In all suspected CAD populations analyzed, there was a nominal cost increase in the world with F 18 -PET-MPI. The 5-year average PMPM incremental budget impact ranged from $0.02 to $0.05 across all suspected CAD subgroups. Cost-savings were associated with decreased downstream CO such as CM, MI, and ICA., Limitations and Conclusion: The available literature to source all parameters in the model was limited; therefore, assumptions and additional calculations were made based on published evidence to inform the model. A one-way sensitivity analysis was performed to confirm and address uncertainty in key parameters. This comprehensive analysis illustrates that the superior diagnostic performance of F 18 -PET-MPI may result in reduced adverse CO events and associated costs, increased appropriate identification and treatment of CAD, and a minimal increase in overall costs among general and difficult-to-image patient subgroups.

Background: The Italian National Health Service (INHS) has recently reimbursed the monoclonal antibody inebilizumab as a second line monotherapy after rituximab (RTX) use for neuromyelitis optica spectrum disorders (NMOSD) patients ≥ 18 years anti-aquaporin 4 antibody-immunoglobulin G positive, who experienced a relapse in the last year or cannot receive RTX, if incident patients. Other INHS-reimbursed drugs for NMOSD treatment are satralizumab, eculizumab and, off-label, besides RTX, ocrelizumab, tocilizumab, and immunosuppressants., Research Design and Methods: A 3-year (2023-2025) prevalence-based budget impact model following the INHS viewpoint compared the costs and the NMOSD attacks without (1st scenario) and with inebilizumab (2nd scenario). The epidemiology of NMOSD, and the INHS-funded healthcare resources (drugs and their administration; specialist visits; hospitalizations due to drug-related adverse events and NMOSD attacks) were obtained from the literature. One-way, threshold value and scenario sensitivity analyses investigated the robustness of the baseline findings., Results: During 2023-2025 inebilizumab saves the INHS €8,373,125.13 (1st scenario: €176,770,028.63; 2nd scenario: €168,396,903.50) and 12.74 NMOSD attacks (1st scenario: 213.94; 2nd scenario: 201.19). Sensitivity analyses confirmed the robustness of the baseline results., Conclusion: Inebilizumab reduces the INHS expenditure for NMOSD drugs. Future research should explore the cost-effectiveness of inebilizumab vs other NMOSD-targeting drugs in Italy.

Aims: French guidelines for the management of non-muscle-invasive bladder cancer recommend that blue-light cystoscopy should be used in patients where the risk of missing residual tumor is highest. Despite evidence for its cost-effectiveness, budgetary concerns have limited uptake in France. The aim of this analysis was to model the cost-consequences of adopting the recommendations in a French urology unit., Materials and Methods: A budget impact model was developed in Excel, using a decision tree approach derived from guidelines issued by L'Academie franҫaise d'urologie. Risk profiles were derived from an analysis of studies using white-light cystoscopy; estimates for the impact of blue-light cystoscopy were derived from a published Cochrane Review. Costs were based on published tariff prices from L'Agence Technique de L'Information sur L'Hospitalisation. The model allowed results to be tailored to activity levels and projected blue-light usage in the chosen urology unit., Results: Two scenarios were evaluated, based on a 3-year time horizon. Full implementation of all recommendations within a large public hospital was estimated to yield incremental costs of €269 per procedure (∼10% increase overall); a more targeted approach within a smaller private hospital yielded incremental costs of €133 per procedure (5% increase overall)., Limitations: The basis of the model is a change in the time to first recurrence. There are no data available for subsequent recurrences or progression, both of which could have an influence on expenditure. Secondly, recurrence rates for blue-light cystoscopy were not specifically available for each patient group identified in the guidelines: extrapolation of data may have resulted in bias. Finally, the data were derived from clinical trials, which may not be generalisable to real-world clinical practice., Conclusions: The model has shown that the additional expenditure required to implement blue-light cystoscopy is modest and not disproportionate to the overall cost of care.

Aims: This study aimed to evaluate the value and affordability of insulin glargine 300 U/mL (Gla-300) in a budget impact model from a United States (U.S.) payer perspective by leveraging recent real-world evidence (RWE) studies and incorporating the recent insulin price caps where applicable., Materials and Methods: An economic model for a hypothetical one million U.S. health-plan population was developed to assess the budgetary impact of therapeutic interchanges in either direction between the two long- and longer-acting basal insulins (BIs) for patients with type 2 diabetes over a three-year model horizon. The utilization of long-acting BIs, longer-acting BIs, biosimilar BIs, and insulin degludec (IDeg-100) were informed by IQVIA data and internal forecasting at Sanofi. The DELIVER-2 and DELIVER-naïve studies provided healthcare resource utilization (HCRU) parameters. In the model base case, 24% of patients switched from long-acting BIs to insulin glargine biosimilars, IDeg-100, and other longer-acting BIs (Gla-300) by projected year 3., Results: The base case total costs were $10,145 per patient per year (PPPY) in year 3 for the cumulative population. When all patients switched to Gla-300, the total costs in year 3 were $8,799, reflecting a net savings of -$660 PPPY compared to the budget increase of $686 PPPY in the base case. However, the longer-acting to long-acting BIs reversal scenario demonstrated a budgetary decrease of $676 PPPY over the model horizon. The reduction in incremental PPPY cost of $93 was observed using net drug costs rather than wholesale acquisition costs (WAC)., Limitations: The market shares for years 1-3 were based on expectations supported by the clinicians' expert opinions and were not obtained from real-world data., Conclusions: The economic value of increased utilization of Gla-300 was driven by the reduction in HCRU, costs and market shares assumptions. Budgetary reductions were achieved by switching patients from long-acting BIs to Gla-300.

Aim: Gaucher disease (GD) is a rare autosomal recessive condition. Type 1 GD (GD1) is the most prevalent form of GD in Western countries; enzyme replacement therapy (ERT) is a treatment option for patients with GD1. To understand the economic value of the GD1 ERT velaglucerase alfa, a budget impact model (BIM) was developed from a United States (US) payer perspective., Methods: We estimated the budget impact of velaglucerase alfa for a 10-million-member US health plan by comparing the annual total costs of therapy between a scenario using current velaglucerase alfa uptake to a projected scenario with increased velaglucerase alfa uptake. Total drug costs for both scenarios were estimated as the sum of the product of the number of eligible patients on each treatment and the annual per-patient cost of each medication. Average per-patient costs for ERTs were calculated by adding the yearly drug acquisition, drug administration, and site-of-care markup costs. The budget impact was measured over years 1-3., Results: An estimated 65 patients would receive velaglucerase alfa treatment in year 1, increasing to 90 patients by year 3. Across analyses, cost savings were realized with velaglucerase alfa compared with imiglucerase ($115,909) and taliglucerase alfa ($80,401). An annual total budget savings of $8.67 million could be realized for a hypothetical 10-million-member US health plan with increased velaglucerase alfa uptake. The per-member per-month costs decreased by $0.0241 across years 1-3., Conclusions: BIM results show that increased velaglucerase alfa uptake for GD1 treatment is cost-saving for US health plans.

Aims: Use of comprehensive genomic profiling (CGP) in metastatic colorectal cancer (mCRC) is limited. We estimated impacts of expanded 1 L CGP, using the Tempus xT test, on detection of actionable alterations and testing budgets in a modeled US health plan over two-years., Materials and Methods: A decision analytic model was developed to estimate the impact of replacing 20% of usual testing (a mix of CGP and non-CGP) with Tempus xT CGP. Actionable alterations for matched treatments or clinical trial included KRAS , NRAS , RAF , BRAF , deficient mismatch repair (dMMR)/microsatellite instability (MSI), NTRK , RET , EGFR , HER2 , MET , PIK3CA and POLE1 . Costs included initial and repeat testing, physician-associated and administrative costs., Results: In a hypothetical five-million-member plan, 50% Medicare and 50% commercial, 1,112 new cases of mCRC were expected per year. Of these, 566 (51%) would undergo 1 L molecular testing, with 55 re-tested upon progression. Based on current testing rates, there were an expected 521 missed opportunities for genomically informed treatment (47% of new cases), with 442 missed due to lack of testing and 79 due to testing without CGP. Replacing 20% of usual testing with Tempus xT CGP was associated with up to a $0.003 per member per month testing cost increase (net total cost of $202,102 for the five-million-member plan) and 15.5 additional patients with an opportunity for genomically informed care (12.7 patients for treatment and 2.8 for clinical trial). The testing total cost (initial test, repeat test, biopsy and physician services, and administrative cost) to put one additional patient with mCRC on matched therapy or matched clinical trial was estimated to be $13,005. Number needed to test to identify one actionable alteration with Tempus xT CGP versus usual testing was 7.8 patients., Limitations: Conservative assumptions were made for inputs with limited evidence. Based on high concordance rates with dMMR/MSI status, tumor mutational burden (TMB) status was not calculated separately., Conclusions: Replacing 20% of usual testing with Tempus xT CGP was associated with a small incremental testing cost and can identify meaningfully more actionable alterations.

Aims: To assess the impact of belumosudil on the cost of care in chronic graft-versus-host disease (cGVHD) patients who have failed at least two prior lines of systemic therapy using a budget impact model., Methods: A budget impact model with a 5-year time horizon was constructed in Microsoft Excel. The base case model uses the US prevalence rate of 3 L/4L + cGVHD patients from literature and secondary sources, with the potential for user-defined inputs, including model perspectives. The model includes data for two perspectives: the national US population and a hypothetical US private payer health insurance plan with 10 million (Mil) members. Additional model inputs include market share of cGVHD treatments, their associated adverse event rates, and healthcare resource utilization., Results: The potential annual budget impact for the US national and payer plans was evaluated for cGVHD patients. Based on belumosudil utilization increasing to 55% in 3 L and 4 L + by 2026, cost savings of ∼5.5% and 6.7% ($128.8 and $4.9 Mil USD) were observed from national and payer perspectives, respectively. Cost savings in 2026 were derived from fewer AEs ($108.4 and $3.9 Mil USD, for national and payer perspectives; e.g. neutropenia, and thrombocytopenia) and reduced HCRU ($65.1 and $2.3 Mil USD, for national and payer perspectives; e.g. emergency room visits, ICU stays, etc.)., Limitations: Results from the model were dependent on the available data inputs and assumptions. Real-world values may differ from the assumed performance of treatments, market growth, and treatment dosing and duration., Conclusion: The model results suggest that the introduction of belumosudil to treat cGVHD would be associated with substantial cost savings when evaluating a scenario with versus without belumosudil from a US payer perspective.

Background: NYVEPRIA, a pegfilgrastim (a long-acting granulocyte colony-stimulating factor [G-CSF]) biosimilar, was recently recommended for marketing authorization in Europe for decreasing the incidence of febrile neutropenia (FN) in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs. The present study aimed to evaluate the financial impact of introducing a new pegfilgrastim biosimilar from a French healthcare system perspective., Methods: An Excel-based budget impact model was developed to estimate the financial impact by introducing a new pegfilgrastim biosimilar (NYVEPRIA) to France over a 5-year time horizon. Comparators included existing long-acting and short-acting G-CSFs. The burden of FN was obtained from existing literature. Costs (2021 Euros) included drug acquisition and administration, estimated based on drug dosage in both clinical trial and real-world settings. Scenario analyses were conducted to examine the robustness of key model assumptions., Results: In a total French population of 67.19 million, 79,873 patients were estimated to be treated with G-CSFs annually. The annual number of patients to be treated with NYVEPRIA was estimated to be 1593, 3195, 3674, 3782, and 4052 in years 1 to 5, respectively. Using real-world data, NYVEPRIA resulted in total annual cost savings of €8,620, €868,498, €868,498, €814,102, and €958,952 over years 1 to 5, respectively, leading to a cumulative 5-year cost savings of €3,518,669. Using data from clinical trials, NYVEPRIA resulted in total annual cost savings of €14,366, €1,447,496, €1,447,496, €1,356,836, and €1,598,253 over years 1 to 5, respectively, leading to a cumulative 5-year cost savings of €5,864,448., Conclusions: The introduction of a new pegfilgrastim biosimilar (NYVEPRIA) is potentially associated with substantial cost savings for the French healthcare system.

Research aim: To model the annual value of a novel ready-to-use, room-temperature stable liquid glucagon rescue pen and prefilled syringe (GRP, G-PFS; Xeris Pharmaceuticals, Inc.) for treatment of severe hypoglycemia events (SHE) versus current lyophilized powder glucagon emergency kits (GEK). GRP is a prefilled auto-injector designed to promptly administer concentrated liquid glucagon in a simple two-step process. G-PFS is a stable liquid formulation of glucagon in a prefilled syringe. In simulated emergencies, GRP and G-PFS demonstrated high functional efficacy, where 99% of users successfully administered a full-dose of drug. Studies with currently available injectable GEK suggest very low success rates (6-31%). The high functional efficacy of GRP and G-PFS significantly reduces user errors and may reduce utilization across emergency medical services (EMS), emergency departments (ED), and inpatient and outpatient costs for SHE. Methods: To estimate the economic impact of GRP and G-PFS, we developed a one-year budget impact model from a US commercial health plan perspective. Cost offsets from successful glucagon administration incorporated EMS, ED, inpatient, and outpatient utilization. Diabetes prevalence and event probabilities were estimated from publicly-available sources and clinical expert opinion. Costs (US$) were obtained from the 2018 Medicare Fee Schedules and adjusted to represent commercial payer costs. Results: GRP and G-PFS led to fewer EMS, ED, inpatient, and outpatient costs compared to GEK and no kit, resulting in total per-patient SHE costs of $2,564, $3,606, and $3,849, respectively. Costs for 1 million covered lives were 8.2 million following the introduction of GRP and G-PFS compared to almost 9 million before GRP and G-PFS. Limitations: The model is limited by reliance on assumptions based on expert opinion for key variables, primarily the probability of: (1) ambulance calls, (2) ambulance transport to the ED, and (3) non-ambulance transport to the ED. Conclusions: A budget impact model suggests GRP and G-PFS can lead to significant annual cost savings for US commercial payers.

A number of live-attenuated varicella vaccines are produced globally that provide protection against the varicella zoster virus. In Mexico, varicella vaccination is not included in the national immunization program and is recommended for use only in high-risk subgroups. We developed a budget impact model to estimate the impact of universal childhood immunization against varicella on the national payer system in Mexico. A scenario of no varicella vaccination was compared to scenarios with vaccination with a single dose at 13 months of age, in alignment with the existing program of immunization with the measles-mumps-rubella vaccine. Nine different vaccination scenarios were envisioned, differing by vaccine type and by coverage. Varicella cases and treatment costs of each scenario were computed in a dynamic transmission model of varicella epidemiology, calibrated to the population of Mexico. Unit costs were based on Mexico sources or were from the literature. The results indicated that each of the three vaccine types increased vaccine acquisition and administration expenditures but produced overall cost savings in each of the first 10 years of the program, due to fewer cases and reduced varicella treatment costs. A highly effective vaccine at 95% coverage produced the greatest cost savings.

Objective: To determine the net economic impact of switching from low-osmolar contrast media (LOCM) to iso-osmolar contrast media (IOCM; iodixanol) in patients undergoing inpatient coronary or peripheral angioplasty in the United States (US)., Methods: A budget impact model (BIM) was developed from a hospital perspective. Nationally representative procedural and contrast media prevalence rates, along with MARCE (major adverse renal cardiovascular event) incidence and episode-related cost data were derived from Premier Hospital Data (October 2014 to September 2015). A previously estimated relative risk reduction in MARCE associated with IOCM usage (9.3%) was applied. The higher cost of IOCM was included when calculating the net impact estimates at the aggregate, hospital type, and per hospital levels. One-way (±25%) and probabilistic sensitivity analyses identified the model's most important inputs., Results: Based on weighted analysis, 513,882 US inpatient angioplasties and 35,610 MARCE cases were estimated annually. Switching to an "IOCM only" strategy from a "LOCM only" strategy increases contrast media cost, but prevents 2,900 MARCE events. The annual budget impact was an estimated saving of $30.71 million, aggregated across all US hospitals, $6,316 per hospital, or $60 per procedure. Net savings were maintained across all univariate sensitivity analyses. While MARCE/event-free cost differential was the most important factor driving total net savings for hospitals in the Northeast and West, procedural volume was important in the Midwest and rural locations., Conclusions: Switching to an "IOCM only" strategy from a "LOCM only" approach yields substantial net global savings to hospitals, both at the national level and within hospital sub-groups. Hospital administrators should maintain awareness of the factors that are likely to be more influential for their hospital and recognize that purchasing on the basis of lower contrast media cost may result in higher overall costs for patients undergoing inpatient angioplasty.

Aims: To develop a budget impact model (BIM) for estimating the financial impact of formulary adoption and uptake of calcipotriene and betamethasone dipropionate (C/BD) foam (0.005%/0.064%) on the costs of biologics for treating moderate-to-severe psoriasis vulgaris in a hypothetical US healthcare plan with 1 million members., Methods: This BIM incorporated epidemiologic data, market uptake assumptions, and drug utilization costs, simulating the treatment mix for patients who are candidates for biologics before (Scenario #1) and after (Scenario #2) the introduction of C/BD foam. Predicted outcomes were expressed in terms of the annual cost of treatment (COT) and the COT per member per month (PMPM)., Results: At year 1, C/BD foam had the lowest per-patient cost ($9,913) necessary to achieve a Psoriasis Area and Severity Index (PASI)-75 response compared with etanercept ($73,773), adalimumab ($92,871), infliximab ($34,048), ustekinumab ($83,975), secukinumab ($113,858), apremilast ($47,960), and ixekizumab ($62,707). Following addition of C/BD foam to the formulary, the annual COT for moderate-to-severe psoriasis would decrease by $36,112,572 (17.91%, from $201,621,219 to $165,508,647). The COT PMPM is expected to decrease by $3.00 (17.86%, from $16.80 to $13.80)., Limitations: Drug costs were based on Medi-Span reference pricing (January 21, 2016); differences in treatment costs for drug administration, laboratory monitoring, or adverse events were not accounted for. Potentially confounding were the definition of "moderate-to-severe" and the heterogeneous efficacy data. The per-patient cost for PASI-75 response at year 1 was estimated from short-term efficacy data for C/BD foam and apremilast only., Conclusions: The introduction of C/BD foam is expected to decrease the annual COT for moderate-to-severe psoriasis treatable with biologics by $36,112,572 for a hypothetical US healthcare plan with 1 million plan members, and to lower the COT PMPM by $3.00.

Background: Advanced neuroendocrine tumors (NETs) are a rare malignancy with considerable need for effective therapies. Everolimus is a mammalian target of rapamycin (mTOR) inhibitor approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) in 2016 for treatment of adults with progressive, well-differentiated, non-functional NETs of gastrointestinal (GI) or lung origin that are unresectable, locally advanced, or metastatic., Objective: To assess the 3-year budget impact for a typical US health plan following availability of everolimus for treatment of GI and lung NETs. Methods An economic model was developed that considered two perspectives: an entire health plan and a pharmacy budget. The total budget impact included costs of drug therapies, administration, hospitalizations, physician visits, monitoring, and adverse events (AEs). The pharmacy model only considered drug costs., Results: In a US health plan with 1 million members, the model estimated 66 patients with well-differentiated, non-functional, and advanced or metastatic GI NETs and 20 with lung NETs undergoing treatment each year. Total budget impact in the first through third year after FDA approval ranged from $0.0568-$0.1443 per member per month (PMPM) for GI NETs and from $0.0181-$0.0355 PMPM for lung NETs. The total budget impact was lower than the pharmacy budget impact because it included cost offsets from administration and AE management for everolimus compared with alternative therapies (e.g. chemotherapies)., Limitations: Because GI and lung NETs are rare diseases with limited published data, several assumptions were made that may influence interpretation of results., Conclusions: The budget impact for everolimus was minimal in this rare disease area with a high unmet need, largely due to low disease prevalence. These results should be considered in the context of significant clinical benefits potentially provided by everolimus, including significantly longer progression-free survival (PFS) for advanced GI and lung NET patients.

Objective: Allergic rhinitis (AR) is a chronic disease with a substantial clinical and economic burden. This study estimated the potential budget impact (BI) associated with market entry of Sweet Vernal, Orchard, Perennial Rye, Timothy, and Kentucky Blue Grass Mixed Pollens Allergen Extract Tablet for Sublingual Use ('5-grass SLIT tablet') for patients aged 10-65 with grass pollen-induced AR., Methods: A budget impact model was constructed to estimate the potential BI from a US payer perspective. The model calculated pharmacy, medical, and total (pharmacy + medical) costs per-member-per-month (PMPM) with and without market entry of the 5-grass SLIT tablet, considering a 3-year time horizon. The target population was determined using an epidemiological approach and existing literature. The treatment market shares without 5-grass SLIT tablet entry were derived from an analysis of de-identified insurance claims data. Pharmacy costs and medical utilization rates and costs were obtained from the claims data analysis and existing literature. One-way sensitivities were conducted for key model inputs., Results: Using an illustrative example of a hypothetical health plan with one million members, the estimated target population of AR patients aged 10-65 was 26,320. On a PMPM basis, pharmacy costs increased by $0.36, $0.44, and $0.51, while total costs (after medical cost offsets) increased by $0.15, $0.18, and $0.22 in the first, second, and third years following entry of the 5-grass SLIT tablet, respectively. Results were most sensitive to changes in the compliance rate, treatment duration, and price. The BI will vary from the base case example when alternative, payer-specific inputs are used., Conclusions: Using base case inputs, use of the 5-grass SLIT tablet to treat grass pollen-induced AR increased the pharmacy budget for a hypothetical third-party payer. Higher pharmacy costs were partially offset by lower medical budget due to reduced resource use compared with existing treatments.

Objectives: The buprenorphine/naloxone combination for the treatment of opioid dependence is available in a film or tablet formulation. Recent retrospective studies demonstrated that treatment with the sublingual film formulation is associated with improved treatment retention and lower healthcare costs. In March 2013, generic buprenorphine/naloxone tablets were approved in the US. A budget impact model was built to compare healthcare expenditures for different market shares of sublingual film and tablet., Methods: A Markov model was developed to track a cohort of opioid dependent patients treated with sublingual film or tablet through the following treatment phases: initiation, maintenance, discontinuation, off-treatment and reinitiation. Transition probabilities and costs for each phase were estimated from the MarketScan Medicaid database for the period between 1 March 2010 and 30 June 2012. The total expenditure for the plan and expenditure per plan member per month were predicted over 5 years. Two market share scenarios were considered: 1) sublingual film is progressively replaced by generic tablet (current situation) and 2) the sublingual film holds a market share of 100%., Results: Predicted total costs over 5 years were $6400 million when the sublingual film holds a market share of 100% (as per Scenario 2) which is lower than when sublingual film is progressively replaced by generic tablet (current situation as per Scenario 1) by $64 million. These savings were mostly driven by inpatient care ($56 million saved over 5 years), followed by emergency room care ($27 million) and pharmaceutical costs ($24 million). Costs of outpatient care attenuated the difference as they were predicted to be higher by $44 million in Scenario 2. The reduction in total cost per member per month reached $0.027 in the fifth year. Results were most sensitive to price rebates and to the probability of non-psychiatric hospitalization., Conclusions: While using the sublingual film formulation for more patients treated with buprenorphine/naloxone is predicted to increase outpatient care costs, it would generate savings in emergency care and hospitalizations. In the treatment of opioid dependence, total direct medical costs for Medicaid would be lower for sublingual film treated patients, at current drug prices.