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Background: Chronic obstructive pulmonary disease (COPD) represents a significant public health problem in Italy, with a high prevalence and a substantial economic burden on the National Health Service (NHS). Effective management of the disease is crucial for reducing exacerbations and improving patients’ quality of life. Methods: This study employs a dynamic predictive model to assess the economic impact of implementing three specific health policies aimed at improving COPD management over a five-year horizon. The analysis is conducted from the perspective of the NHS, focusing on the direct costs associated with disease management. Results: The implementation of the proposed policies demonstrated potential significant savings in direct healthcare costs. The first policy, focused on increasing therapeutic adherence, generated an estimated savings of 202 million €. The second, concerning the wider use of triple therapies, led to savings of about 142 million €. Finally, the third policy, aimed at reducing the delay in prescribing appropriate therapies, contributed to an additional saving of over 175 million € in the time horizon considered in the analysis. Overall, these results suggest a substantial improvement opportunity in COPD management, with significant economic benefits for the Italian NHS. Conclusion: The study confirms the importance of targeted policies in the management of COPD, highlighting how strategic interventions can lead to significant resource savings while simultaneously improving patient care. These results provide a solid foundation for further research and serve as a useful guide for decision-makers in planning effective intervention strategies, aimed at optimizing resources and enhancing disease management in the Italian healthcare context.

More than 16 million Americans living with cognitive impairment warrant a diagnostic evaluation to determine the cause of this disorder. The recent availability of disease-modifying therapies for Alzheimer's disease (AD) is expected to significantly drive demand for such diagnostic testing. Accurate, accessible, and affordable methods are needed. Blood biomarkers (BBMs) offer advantages over usual care amyloid positron emission tomography (PET) and cerebrospinal fluid (CSF) biomarkers in these regards. This study used a budget impact model to assess the economic utility of the PrecivityAD® blood test, a clinically validated BBM test for the evaluation of brain amyloid, a pathological hallmark of AD. The model compared 2 scenarios: (1) baseline testing involving usual care practice, and (2) early use of a BBM test before usual care CSF and PET biomarker use. At a modest 40% adoption rate, the BBM test scenario had comparable sensitivity and specificity to the usual care scenario and showed net savings in the diagnostic work-up of $3.57 million or $0.30 per member per month in a 1 million member population, translating to over $1B when extrapolated to the US population as a whole and representing a 11.4% cost reduction. Savings were driven by reductions in the frequency and need for CSF and PET testing. Additionally, BBM testing was associated with a cost savings of $643 per AD case identified. Use of the PrecivityAD blood test in the clinical care pathway may prevent unnecessary testing, provide cost savings, and reduce the burden on both patients and health plans. [ABSTRACT FROM AUTHOR]

Aims: This economic model was developed to assess the budget impact of a novel radiotracer, Flurpiridaz (F18-PET-MPI), compared to SPECT-MPI from a US payer perspective. Materials and Methods: The model was developed comparing F18-PET-MPI and SPECT-MPI, with F18-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 F18-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 F18-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. PLAIN LANGUAGE SUMMARY: Myocardial perfusion imaging is a technique used to diagnose Coronary Artery Disease. Single-photon emission computed tomography (SPECT) and positron emission tomography (PET) are key Myocardial perfusion imaging functional heart assessments. This study compares the costs of Flurpiridaz-PET (a new PET radiotracer) to SPECT. Flurpiridaz-PET improves diagnostic accuracy, leading to better treatment and reduced cardiac outcomes, including fewer heart attacks and mortality. The overall cost difference between Flurpiridaz-PET and SPECT is minimal. [ABSTRACT FROM AUTHOR]

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. 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. 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. Inebilizumab reduces the INHS expenditure for NMOSD drugs. Future research should explore the cost-effectiveness of inebilizumab vs other NMOSD-targeting drugs in Italy. [ABSTRACT FROM AUTHOR]

Objectives: Patient Blood Management (PBM) is defined as a patient‐centered, systematic, evidence‐based approach to improve patient outcomes by managing and preserving a patient's own blood, while promoting patient safety and empowerment. As a corollary, it also reduces the utilization of allogeneic blood components. However, demonstrating cost‐effectiveness depends on the health insurance system considered. This analysis aims to estimate the one‐year budget impact of PBM in four elective surgical areas, from French National Health Insurance and hospital perspectives. Methods: A budget impact model was developed to estimate the difference in the cost of care between scenarios with and without PBM. The impact of hematopoiesis optimization (first pillar of PBM) was studied throughout the management of preoperative anemia and iron deficiency in four types of surgeries: orthopedic, cardiac & cardiovascular, vascular & thoracic, and urologic & visceral surgery. Estimation of model's parameters was based on data collected in 10 French hospitals, literature, and on data from the French national medico‐administrative database. Results: A total of 980,125 patients were modeled for all four therapeutic areas. Results shows that implementation of a PBM program could generate annual savings up to €1079 M from the French National Health Insurance perspective (€1018 M from the hospital perspective), and the sparing of 181,451 red blood cells units per year. The deterministic sensitivity analysis showed that PBM generates savings for both perspectives in most parameters tested. Conclusion: Implementing PBM programs could result in important savings for the health care system in France. [ABSTRACT FROM AUTHOR]

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. 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. 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). 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. 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. Type 2 Diabetes (T2D) is a chronic and debilitating condition that can lead to severe macro or microvascular complications. To mitigate these complications, it is crucial to effectively manage blood glucose levels. When other treatments prove ineffective in achieving adequate glucose control, insulin-based therapy becomes necessary. However, insulin-based treatments often come with the risk of hypoglycemic episodes, which can lead to increased utilization of healthcare resources (HCRU) and have a negative impact on costs. This study aimed to assess the budgetary impact of higher market shares of longer-acting basal insulins (specifically insulin glargine Gla-300) compared to long-acting basal insulins, insulin glargine biosimilars, and insulin degludec (IDeg) in the treatment of T2D. The perspective taken was that of a U.S. payer, taking into account the recent insulin price caps where applicable. The economic benefit of increased utilization of Gla-300 was driven by reductions in HCRU, costs, and market share assumptions. This resulted in a budgetary increase of $686 per patient per year (PPPY). In an alternative scenario where all patients transitioned to Gla-300, it led to a net savings of $660 PPPY. These findings provide valuable insights for decision-makers and healthcare professionals when making choices related to formulary placement and treatment utilization. [ABSTRACT FROM AUTHOR]

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. 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. 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). 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. 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. [ABSTRACT FROM AUTHOR]

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.

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. 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. 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.). 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. 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. [ABSTRACT FROM AUTHOR]

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. 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. 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. BIM results show that increased velaglucerase alfa uptake for GD1 treatment is cost-saving for US health plans. Type 1 Gaucher disease (GD1) is a rare inherited condition. Long-term enzyme replacement therapy (ERT) can reverse and prevent complications. Imiglucerase, taliglucerase alfa, and velaglucerase alfa are 3 ERTs used to treat GD1. In this study, we estimated how increasing uptake of velaglucerase alfa vs. the other ERTs would impact the budget of a hypothetical US healthcare plan. The results show that increased uptake of velaglucerase alfa is cost-saving for US health plans. [ABSTRACT FROM AUTHOR]

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. 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. 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. 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. Replacing 20% of usual testing with Tempus xT CGP was associated with a small incremental testing cost and can identify meaningfully more actionable alterations. [ABSTRACT FROM AUTHOR]

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 e8,620, e868,498, e868,498, e814,102, and e958,952 over years 1 to 5, respectively, leading to a cumulative 5-year cost savings of e3,518,669. Using data from clinical trials, NYVEPRIA resulted in total annual cost savings of e14,366, e1,447,496, e1,447,496, e1,356,836, and e1,598,253 over years 1 to 5, respectively, leading to a cumulative 5-year cost savings of e5,864,448. Conclusions: The introduction of a new pegfilgrastim biosimilar (NYVEPRIA) is potentially associated with substantial cost savings for the French healthcare system. [ABSTRACT FROM AUTHOR]

Purpose: Improvement in long-term outcomes through innovative, cost-effective medical technologies is a focus for endovascular procedures aimed at treating symptomatic lower-limb peripheral arterial disease (PAD). The advent of drug-eluting stents (DES) has improved symptomatic PAD treatment via a reduction in high rates of target lesion revascularisation (TLR). The present study aimed to compare the 5-year financial impact of treatment with Eluvia, a new paclitaxel-eluting stent, versus treatment with Zilver PTX, a drug-coated stent, among patients in Australia by developing a budget impact model (BIM). Methods: A BIM was developed from an Australian public hospital payer perspective using Australian national cost weights (AUD), published literature, and public hospital audit data. Clinical outcomes, including clinically driven TLRs (CD-TLRs), adverse events, and length of stay, were based on the 2-year results of the IMPERIAL trial, which compared Eluvia DES to Zilver PTX. Results: Assuming EVP eligibility rate of 80% and DES uses rate ranging from 10 to 28% (superficial femoral artery lesions only), the 5-year model forecasted a treatment population between 14,428 and 40,399 patients. The model estimated 1499–4198 fewer CD-TLRs and 16,515–46,243 fewer hospital days with Eluvia DES use. This translated to 5-year potential savings of $4.3–$12.1 million to the Australian public hospital payer attributable to reduced CD-TLRs for Eluvia DES and $33.1–$92.6 million to Australian public hospitals owing to reduced adverse events and hospital bed days. Conclusion: Eluvia DES use as treatment for symptomatic lower-limb PAD could lead to potential savings for the Australian public healthcare system based on improved patient outcomes. [ABSTRACT FROM AUTHOR]

Thitima Kongnakorn,1 Murtuza Bharmal,2 Mairead Kearney,3 Hemant Phatak,4 Agnes Benedict,5 Abhijeet Bhanegaonkar,4 Matthew Galsky6 1Evidence Synthesis, Modeling and Communication, Evidera, London, UK; 2Global Evidence and Value Development, EMD Serono, Inc. (A business of Merck KGaA, Darmstadt, Germany), Rockland, MA, USA; 3Global Evidence and Value Development, Merck KGaA, Darmstadt, Germany; 4US Health Economics and Outcomes Research, EMD Serono, Inc. (A business of Merck KGaA, Darmstadt, Germany), Rockland, MA, USA; 5Evidence Synthesis, Modeling and Communication, Evidera, Budapest, Hungary; 6Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, NY, USACorrespondence: Murtuza BharmalGlobal Evidence and Value Development, EMD Serono, Inc. (A business of Merck KGaA, Darmstadt, Germany), Rockland, MA, USAEmail murtuza.bharmal@emdserono.comObjective: To estimate the budget impact of introducing avelumab as a second-line (2L) treatment option for patients with locally advanced or metastatic urothelial cancer (mUC) from the perspective of a US third-party payer (commercial and Medicare).Methods: A budget impact model (BIM) with a three-year time horizon was developed for avelumab. Efficacy and safety data were sourced from published literature and US package inserts. The analysis was conducted in collaboration with a specialist oncologist who validated clinical assumptions. Costs were based on the number of eligible patients, time-to-treatment failure, overall survival, adverse events (AEs), and projected market shares of various treatments.Results: In a hypothetical commercial health plan of 30,000,000 members, 884 patients were estimated to be eligible for 2L treatment over a three-year time period. Without avelumab, the total cost for treating patients with mUC was estimated to be US$70,268,035. The introduction of avelumab increased total costs by $73,438 (0.10% increase). In a hypothetical Medicare health plan of 30,000,000 beneficiaries, a total of 4,705 patients were estimated to be eligible for 2L treatment. Without avelumab, the total cost for treating patients with mUC was estimated to be $292,923,098 from a Medicare perspective; however, with avelumab, there was an increase of $719,324 (0.25% increase) in total costs. Results of the sensitivity analyses demonstrated a cost-neutral impact across all tested scenarios from both perspectives.Conclusion: The BIM estimated that avelumab would have a cost-neutral impact within a US commercial and a Medicare health plan. Overall, avelumab can be an affordable and valuable treatment option for patients with locally advanced or mUC in the 2L setting. These findings demonstrate a consistently favorable budget impact in both populations. Further studies should be conducted to more comprehensively assess the clinical and economic implications of adding avelumab to the treatment armamentarium of 2L mUC.Keywords: urothelial carcinoma, budget impact model, cost analysis, economic analysis, immuno-oncology, chemotherapy

Maurizio Benucci,1 Arianna Damiani,1 Mariangela Manfredi,2 Maria Infantino,2 Valentina Grossi,2 Francesca Li Gobbi11Rheumatology Unit, S.Giovanni di Dio Hospital, Florence, Italy; 2Immunology and Allergology Laboratory Unit, S.Giovanni di Dio Hospital, Azienda USL-Toscana Centro, Florence, ItalyAbstract: Rheumatoid arthritis (RA) is a chronic inflammatory disorder that affects joints with swelling and progressive joint destruction. The pathology leads to a progressive disability with an impact on the quality of life of the patients. Strategies to reduce in-patient care costs could have a considerable impact on lowering the direct medical costs of RA in Italy. Abatacept, a selective T-cell costimulation modulator, is a valuable treatment option for patients with moderate-to-severe RA. A search using the keywords “cost-effectiveness analysis", "budget impact model", "abatacept", and "rheumatoid arthritis” was carried out on PubMed. Abatacept in the first- and second-treatment lines has been evaluated in our research. We evaluated patients with inadequate MTX response, inadequate anti-TNF agents response, switch studies and real-world data. Furthermore, in our research, we evaluated the main head-to-head studies published.Keywords: abatacept, budget impact model, cost-effectiveness analysis, rheumatoid arthritis

Murtuza Bharmal,1 Mairead Kearney,1 Ying Zheng,2 Hemant Phatak21Global Evidence and Value Development, Merck Healthcare KGaA, Darmstadt, Germany; 2US Health Economics and Outcomes Research, EMD Serono, Inc, Rockland, MA, USAObjective: To estimate the budget impact of avelumab as a treatment option for patients with treatment-naïve first-line (1L) and previously treated second-line or later (2L+) metastatic Merkel cell carcinoma (mMCC) in the US.Methods: A budget impact model was developed to evaluate the addition of avelumab for the treatment of mMCC patients using a hypothetical 30 million-member US health plan over a 3-year time horizon (2019–2021). The comparator treatments included in the analysis were pembrolizumab and nivolumab (other immuno-oncology agents); and the chemotherapies routinely used in the eligible mMCC population. Model inputs included market share uptake of avelumab and other comparators, duration of treatments, and costs (drugs, health care resource utilization, adverse events). The model was evaluated from a commercial payer perspective. Sensitivity analyses were conducted to test uncertainties arising from the input values used in the model.Results: In a hypothetical commercial health plan of 30 million members, 285 patients with mMCC were identified over 3 years; 43 patients received avelumab as a 1L treatment over 3 years. In a world without avelumab, the total health care costs of treating patients with mMCC over 3 years were estimated to be US$11,710,115 from a commercial health plan perspective. With avelumab, there were estimated savings of $2,643,173 considering the total costs related to the treatment of mMCC over 3 years (23% reduction in the budget). The incremental cost per member per month over 3 years was −$0.0025.Conclusion: The model results indicate that the adoption of avelumab as a treatment option for mMCC would likely result in minimal budget impact from a US health plan perspective. Patients with mMCC, a rare condition with a poor prognosis and high unmet need, may benefit greatly from recently approved immunotherapies.Keywords: metastatic merkel cell carcinoma, cost, budget impact model, avelumab

Abstract Background During cataract surgery, maintaining an adequate degree of mydriasis throughout the entire operation is critical to allow for visualisation of the capsulorhexis and the crystalline lens. Good anaesthesia is also essential for safe intraocular surgery. Mydrane® is a new injectable intracameral solution containing two mydriatics (tropicamide 0.02% and phenylephrine 0.31%) and one anaesthetic (lidocaine 1%) that was developed as an alternative to the conventional topical pre-operative mydriatics used in cataract surgery. This study aimed to estimate the budget impact across a one year time frame using Mydrane® instead of topical dilating eye drops, for a UK hospital performing 3,000 cataract operations a year. Methods A budget impact model (BIM) was developed to compare the economic outcomes associated with the use of Mydrane® versus topical drops (tropicamide 0.5% and phenylephrine 10%) in patients undergoing cataract surgery in a UK hospital. The outcomes of interest included costs and resource use (e.g. clinician time, mydriasis failures, operating room time, number of patients per vial of therapy etc.) associated with management of mydriasis in patients undergoing cataract surgery. All model inputs considered the UK hospital perspective without social or geographical variables. Deterministic sensitivity analyses were also performed to assess the model uncertainty. Results Introduction of Mydrane® is associated with a cost saving of £6,251 over 3,000 cataract surgeries in one year. The acquisition costs of the Mydrane® (£18,000 by year vs. £3,330 for eye drops) were balanced by substantial reductions in mainly nurses’ costs and time, plus a smaller contribution from savings in surgeons’ costs (£20,511) and lower costs associated with auxiliary dilation (£410 due to avoidance of additional dilation methods). Results of the sensitivity analyses confirmed the robustness of the model to the variation of inputs. Except for the duration of one session of eye drop instillation and the cost of Mydrane®, Mydrane® achieved an incremental cost gain compared to tropicamide/phenylephrine eye drops. Conclusions Despite a higher acquisition cost of Mydrane®, the budget impact of Mydrane® on hospital budgets is neutral. Mydrane® offers a promising alternative to traditional regimes using eye drops, allowing for a better patient flow and optimisation of the surgery schedule with neutral budget impact.

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. 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. 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. The introduction of a new pegfilgrastim biosimilar (NYVEPRIA) is potentially associated with substantial cost savings for the French healthcare system. [ABSTRACT FROM AUTHOR]

Jesse D Ortendahl,1 Sonia J Pulgar,2 Beloo Mirakhur,3 David Cox,3 Tanya GK Bentley,1 Alexandria T Phan4 1Health Economics, Partnership for Health, LLC, Beverly Hills, CA, USA; 2Health Economics and Outcomes Research, Ipsen Biopharmaceuticals, Basking Ridge, NJ, USA; 3Medical Affairs, Oncology, Ipsen Biopharmaceuticals, Basking Ridge, NJ, USA; 4GI Medical Oncology, University of New Mexico Comprehensive Cancer Center, Albuquerque, NM, USA Objective: With the introduction of new therapies, hospitals have to plan spending limited resources in a cost-effective manner. To assist in identifying the optimal treatment for patients with locally advanced or metastatic gastroenteropancreatic neuroendocrine tumors, budget impact modeling was used to estimate the financial implications of adoption and diffusion of somatostatin analogs (SSAs). Patients and methods: A hypothetical cohort of 500 gastroenteropancreatic neuroendocrine tumor patients was assessed in an economic model, with the proportion with metastatic disease treated with an SSA estimated using published data. Drug acquisition, preparation, and administration costs were based on national pricing databases and published literature. Octreotide dosing was based on published estimates of real-world data, whereas for lanreotide, real-world dosing was unavailable and we therefore used the highest indicated dosing. Alternative scenarios reflecting the proportion of patients receiving lanreotide or octreotide were considered to estimate the incremental budget impact to the hospital. Results: In the base case, 313 of the initial 500 gastroenteropancreatic neuroendocrine tumor patients were treated with an SSA. The model-predicted per-patient cost was US$83,473 for lanreotide and US$89,673 for octreotide. With a hypothetical increase in lanreotide utilization from 5% to 30% of this population, the annual model-projected hospital costs decreased by US$488,615. When varying the inputs in one-way sensitivity analyses, the results were most sensitive to changes in dosing assumptions. Conclusion: Results suggest that factors beyond drug acquisition cost can influence the budget impact to a hospital. When considering preparation and administration time, and real-world dosing, use of lanreotide has the potential to reduce health care expenditures associated with metastatic gastroenteropancreatic neuroendocrine tumor treatments. Keywords: health economics, oncology, model, costs, real-world evidence, lanreotide

BACKGROUND: Vedolizumab is a biologic drug approved by the US Food and Drug Administration (FDA) for the treatment of adults with moderately to severely active Crohn's disease (CD) or ulcerative colitis (UC) who have had inadequate response to, lost response to, or were intolerant of immunomodulators or tumor necrosis factor (TNF) blocker therapy, or who had an inadequate response with, were intolerant to, or demonstrated dependence on corticosteroid therapy. The biologics approved by the FDA for CD and/or UC include adalimumab, infliximab, golimumab, certolizumab, and ustekinumab. OBJECTIVE: To assess the budget impact of including vedolizumab in a health plan formulary among current options as a preferred first-line biologic therapy for UC and CD rather than only for patients who failed anti-TNF therapy. METHODS: We developed a 3-year budget impact model for a 1-million-member health plan. Comparators included all currently approved brand-name biologic and biosimilar agents for the treatment of UC (ie, adalimumab, infliximab, and golimumab) and CD (ie, adalimumab, certolizumab, infliximab, and ustekinumab). Clinical inputs included therapy response probabilities, disease remission, and surgery risk. Given the lack of head-to-head clinical trials, we estimated indirect comparisons of treatment efficacy based on clinical trial data using the Bucher method. The drug and medical costs were obtained from published literature. The model compared hypothetical health plan costs for 2 scenarios--(1) a market mix with vedolizumab included on the formulary with currently existing first- and second-line preferred treatments, and (2) vedolizumab included only with existing preferred second-line treatments on the hypothetical formulary. These scenarios were compared in the context of 3 hypothetical health plan formulary cases. RESULTS: Including vedolizumab in a hypothetical formulary with currently preferred first-line biologic treatment options (Scenario 1) resulted in cost-savings compared with vedolizumab as a preferred second- line biologic option (Scenario 2). The total cost-savings were from $0.13 million to $1.63 million in year 1, and from $0.38 million to $4.68 million in year 3. The per-member per-month cost-savings were from $0.01 to $0.14 in year 1 and from $0.03 to $0.39 in year 3. CONCLUSION: Based on our model's results, including vedolizumab among the current health plan formulary biologic options as a preferred first-line treatment for UC and CD can result in substantial cost-savings compared with including vedolizumab as a preferred second-line treatment only. [ABSTRACT FROM AUTHOR]

Purpose: Diabetic macular edema (DME) is a leading cause of vision loss and blindness. The aim of this study was to evaluate the economic benefits of introducing additional alternative technologies (Dexamethasone intravitreal implant – DEX – and Aflibercept injections), compared with the historical scenario of Ranibizumab intravitreal injections. Methods: A 3‐year budget impact model was developed, taking into consideration the perspective of the Lombardy Region Healthcare Service (LRHS). Total administration costs (real‐life data retrieved from clinical practice at three Departments of Ophthalmology) as well as costs related to the management of potential adverse events (information collected from the literature) were analysed. Results: Over a 36‐month horizon, the results showed that a higher consumption of DEX could lead to significant economic savings for the Regional Healthcare Service, ranging from a minimum of −4.35% (if DEX were used only in the second‐line of treatment) to a maximum of −12.97% (if DEX were used in both the first‐line and second‐line), including the potential impact of adverse events. Therapy costs with Aflibercept and Ranibizumab were similar. Conclusions: This study demonstrates that concentrating all eligible patients within the Ranibizumab regimen is unlikely to represent a cost‐effective strategy. Indeed, significant economic advantages would be achieved by introducing the other licensed alternatives, Dexamethasone implant and Aflibercept, thus optimising DME Italian healthcare expenditure. The results demonstrate DEX as an advantageous technological alternative for the target population affected by DME, both as a first‐ and second‐line treatment option, reducing the economic burden of the pathology for the Regional/National Health Service. [ABSTRACT FROM AUTHOR]

Background: Tyrosine kinase inhibitors (TKI) have dramatically improved survival in chronic myeloid leukemia in chronic phase (CML‐CP), with a high percentage of patients reaching a major molecular response (MMR). Recently, several clinical trials demonstrated that some patients with CML-CP who achieve a sustained MMR on tyrosine kinase inhibitor (TKI) therapy can safely discontinue their therapy and attempt treatment-free remission (TFR). Objective: The aim of the study was to evaluate the clinical and economic impact of TFR in naïve patients with CML-CP who start treatment with nilotinib, imatinib or dasatinib as first-line therapy, from the perspective of the Italian National Health Service (NHS). Methods: An Excel-based budget impact model was developed, in order to estimate the costs of the patients in first-line pharmacological treatment with CML. A specific Markov model was built, to simulate seven years of treatment with different TKIs. A systematic literature review was carried out, to identify the epidemiological and economic data, which were subsequently used to inform the model. The model considers two scenarios: 1) a Standard of Care (SoC) scenario, with the current estimated distribution of patients over the various TKI treatment, versus 2) an innovative scenario, characterized by an increase in the use of nilotinib (+28%) and generic imatinib (+35%) and a decrease in the use of dasatinib (-17%). A one-way deterministic sensitivity analysis was performed, in order to consider the variability of the results as a function of the main parameters considered in the model. Results: The model estimated that 775 patients with CML-CP could be treated with a TKI as first-line drug. The innovative scenario could increase TFR patients by approximately 60% and reduce the costs by more than € 30 million over 7 years. The increase in the use of nilotinib and the generic imatinib would generate a significant expenditure reduction. Conclusions: This study demonstrates the economic effects of discontinuing TKIs in CML-CP patients. The increase in the use of nilotinib and the generic imatinib could generate an increase in the number of patients who achieve TFR, as well as an actual cost reduction.

BACKGROUND: Nutrition interventions can alleviate the burden of malnutrition by improving patient outcomes; however, evidence on the economic impact of medical nutrition intervention remains limited. A previously published nutrition-focused quality improvement program targeting malnourished hospitalized patients showed that screening patients with a validated screening tool at admission, rapidly administering oral nutritional supplements, and educating patients on supplement adherence result in significant reductions in 30-day unplanned readmissions and hospital length of stay. OBJECTIVES: To assess the potential cost-savings associated with decreased 30-day readmissions and hospital length of stay in malnourished inpatients through a nutrition-focused quality improvement program using a web-based budget impact model, and to demonstrate the clinical and fiscal value of the intervention. METHODS: The reduction in readmission rate and length of stay for 1269 patients enrolled in the quality improvement program (between October 13, 2014, and April 2, 2015) were compared with the pre-quality improvement program baseline and validation cohorts (4611 patients vs 1319 patients, respectively) to calculate potential cost-savings as well as to inform the design of the budget impact model. Readmission rate and length-of-stay reductions were calculated by determining the change from baseline to post-quality improvement program as well as the difference between the validation cohort and the post-quality improvement program, respectively. RESULTS: As a result of improved health outcomes for the treated patients, the nutrition-focused quality improvement program led to a reduction in 30-day hospital readmissions and length of stay. The avoided hospital readmissions and reduced number of days in the hospital for the patients in the quality improvement program resulted in cost-savings of $1,902,933 versus the pre-quality improvement program baseline cohort, and $4,896,758 versus the pre-quality improvement program in the validation cohort. When these costs were assessed across the entire patient population enrolled in the quality improvement program, per-patient net savings of $1499 when using the baseline cohort as the comparator and savings per patient treated of $3858 when using the validated cohort as the comparator were achieved. CONCLUSION: The nutrition-focused quality improvement program reduced the per-patient healthcare costs by avoiding 30-day readmissions and through reduced length of hospital stay. These clinical and economic outcomes provide a rationale for merging patient care and financial modeling to advance the delivery of value-based medicine in a malnourished hospitalized population. The use of a novel web-based budget impact model supports the integration of comparative effectiveness analytics and healthcare resource management in the hospital setting to provide optimal quality of care at a reduced overall cost. [ABSTRACT FROM AUTHOR]

Objective: to estimate the impact of lapatinib utilization within the Italian National Health Service (NHS) resources consumption. Lapatinib is an oral inhibitor of kinase protein, approved as dual therapy with capecitabine for the treatment of metastatic breast cancer patients with HER2 overexpression who experience disease progression despite trastuzumab treatment. Methods: the analysis is based on a model, which structure can be summarized as follows: a) national cancer registries-based estimate of the yearly number of HER2+ breast cancer patients who develop metastatic disease in Italy; b) literature-based identification of the rate of patients eligible to receive lapatinib; c) identification of the current therapeutic strategy-mix; d) costing of the alternatives, and e) calculation of budget impact. Direct NHS costs (drug acquisition and administration, and monitoring for 8 cycles of 21 days) are estimated based on current Italian prices and tariffs. Results: the annual number of patients eligible for lapatinib-based therapy can vary from 1,676 to 2,172, according to the expected extent of the trastuzumab use as adjuvant therapy. The current strategy-mix beyond progression is based on drugs used in the clinical practice, with a portion of patients continuing trastuzumab. Pharmaceutical cost of lapatinib results higher than the average cost of the current pattern of treatments. This cost increase would be partially offset by the reduction of laboratory tests and hospital personnel work for the oral administration of lapatinib, as compared to intravenous strategies. Furthermore, a risk sharing agreement has been adopted by NHS and manufacturer, according to which the NHS pays only for responding patients. As a consequence, lapatinib-based therapy would increase yearly NHS expenditure by about 3.8-4.9 millions of euro. Conclusions: lapatinib is the only treatment option specifically indicated for the management of HER2+, metastatic breast cancer in patients who received prior treatments including trastuzumab and is estimated to induce a low budget impact for the Italian NHS.

Single-nucleotide polymorphisms (SNPs) can severely impact individual drug response and health outcomes in cancer patients. Genetic tests to screen for marker SNPs are available to adjust the drug dose of oncologicals to the patient's needs. However, it is unclear whether the positive effects outbalance the increased costs or even lead to an overall cost reduction. This very pragmatic analysis used three frequently used oncologicals for the treatment of breast cancer to evaluate whether preemptive pharmacogenetic testing may have a cost-reducing impact on health care spending in the Swiss health care system. Our results indicate that oncopharmacogenetics might help to reduce health care costs (ie, by avoiding adverse drug effects) and to increase efficiency of drugs in oncologic patients. [ABSTRACT FROM AUTHOR]

BACKGROUND: Chronic venous disease is a common disorder in the United States. The manifestations of chronic venous disease include varicosities and related sequelae that are frequent contributors to the morbidity and high costs associated with the disease. The interventional treatment options for chronic venous disease have expanded greatly in recent years and include various surgical and vein ablation techniques. Polidocanol injectable foam (also known as polidocanol endovenous microfoam 1%), a chemical ablation agent, is the most recent entrant to the market. OBJECTIVE: To evaluate the expected patient-level total treatment costs and health plan--level budgetary impact of polidocanol injectable foam compared with the currently available interventional treatment options from a third-party US payer perspective. METHODS: A Microsoft Excel--based budget impact model was designed to compare the costs of polidocanol injectable foam with other interventional treatments (ie, laser ablation, radiofrequency ablation, surgery, and multimodality treatment). The model included drug acquisition, medical procedure, administration, additional treatment, and disease progression costs. The treatment patterns and rates of additional treatment were incorporated from a recent retrospective claims analysis for established treatment modalities and from the clinical trials for polidocanol injectable foam. The model estimates the 1-year total estimated costs and the health plan budget impact assuming an 8-week treatment time frame. RESULTS: The total expected 8-week treatment costs were $2165 for polidocanol injectable foam, $1827 for endovenous laser ablation, $2106 for radiofrequency ablation, $2374 for surgery, and $2844 for multimodality treatment. The initial treatment costs were higher for surgery and multimodality treatment compared with polidocanol injectable foam and were lower for endovenous laser ablation and radiofrequency ablation treatments. Polidocanol injectable foam is projected to have a relatively small budget impact ($0.01 per member per month) at an initial 5% market share. CONCLUSION: Polidocanol injectable foam offers an alternative to other interventional options for the treatment of varicose veins and is projected to have a relatively small budget impact. From a health plan perspective, this drug is likely to have a relatively low budget impact as it becomes more widely used. [ABSTRACT FROM AUTHOR]

: Evidence on the favorable efficacy, safety, and cost effectiveness of lumbar total disc replacement (TDR) compared with fusion for lumbar degenerative disc disease is mounting; however, a key barrier identified for TDR utilization is lack of coverage by US health insurers. Although economic considerations in a fee-for-service model should not be a determining factor in patient access, concerns regarding the budget impact of lumbar TDR surgery may unfortunately underlie coverage decisions. On the basis of the data available and economic modeling, the panel agreed that there is no indication that there would be a dramatic increase in patients seeking lumbar TDR. Considering several possible scenarios on potential growth in TDR utilization with coverage, as well as growth in the overall surgical pool of patients, economic modeling demonstrated that adoption of lumbar TDR would result in minimal or no budget impact for commercial insurance plans. Considering these model results and the economic literature, the panel concluded that adopting lumbar TDR within a coverage policy is expected to remain cost neutral for the insurer. [ABSTRACT FROM AUTHOR]

Purpose: The Zilver PTX drug-eluting stent (Cook Ireland Ltd., Limerick, Ireland) represents an advance in endovascular treatments for atherosclerotic superficial femoral artery (SFA) disease. Clinical data demonstrate improved clinical outcomes compared to bare-metal stents (BMS). This analysis assessed the likely impact on the French public health care budget of introducing reimbursement for the Zilver PTX stent. Methods: A model was developed in Microsoft Excel to estimate the impact of a progressive transition from BMS to Zilver PTX over a 5-year horizon. The number of patients undergoing SFA stenting was estimated on the basis of hospital episode data. The analysis from the payer perspective used French reimbursement tariffs. Target lesion revascularization (TLR) after primary stent placement was the primary outcome. TLR rates were based on 2-year data from the Zilver PTX single-arm study (6 and 9 %) and BMS rates reported in the literature (average 16 and 22 %) and extrapolated to 5 years. Net budget impact was expressed as the difference in total costs (primary stenting and reinterventions) for a scenario where BMS is progressively replaced by Zilver PTX compared to a scenario of BMS only. Results: The model estimated a net cumulative 5-year budget reduction of €6,807,202 for a projected population of 82,316 patients (21,361 receiving Zilver PTX). Base case results were confirmed in sensitivity analyses. Conclusion: Adoption of Zilver PTX could lead to important savings for the French public health care payer. Despite higher initial reimbursement for the Zilver PTX stent, fewer expected SFA reinterventions after the primary stenting procedure result in net savings. [ABSTRACT FROM AUTHOR]

Background: An orally disintegrating film (ODF) formulation of sildenafil was recently developed in Korea. This formulation is expected to enhance dosing convenience and increase patient compliance while yielding pharmacokinetics comparable to those of the conventional film-coated tablet (FCT) formulation. Objective: The goal of this study was to compare the pharmacokinetic profiles of a newly developed ODF formulation with those of a FCT formulation of sildenafil in healthy Korean male volunteers. Methods: This was a randomized, open-label, single- dose, 2-period crossover study conducted in 2 parts. Eligible subjects were between the ages of 20 and 50 years and within 20% of their ideal weight; subjects were equally divided into parts 1 and 2. Each subject received a single dose of the ODF and FCT formulations of sildenafil orally in a fasted state (part 1, 50 mg; part 2, 100 mg), with a 7-day washout period between the formulations. Blood samples were collected up to 24 hours postdosing. Pharmacokinetic parameters were determined for sildenafil and its active metabolite (N-desmethyl sildenafil). Adverse events (AEs) were evaluated based on subject interviews and physical examinations. Results: Among the 120 enrolled subjects (60 subjects for each part), 110 completed the study (part 1, n = 53; part 2, n = 57). In both parts, all the primary pharmacokinetic parameters were included in the range for assumed bioequivalence in sildenafil, yielding 90% CI ratios of 91.07% to 120.67% for AUC0-last and 86.68% to 122.93% for Cmax in part 1, and 101.68% to 114.78% for AUC0-last and 93.76% to 109.76% for Cmax in part 2. In part 1, headache was the most frequently noted AE, occurring in 3 subjects with both the test and the reference formulations. All other AEs occurred in <3 subjects. In part 2, nasal congestion was the most frequently observed AE (7 with the test formulation and 1 with the reference formulation), followed by abnormal vision (4 with the test formulation and 4 with the reference formulation), headache (4 with the test formulation and 4 with the reference formulation), and rhinorrhea (5 with the test formulation). All other AEs occurred in <3 subjects. Conclusions: The study findings suggest that the pharmacokinetics of the ODF formulation of sildenafil do not differ significantly from those of the conventional FCT formulation (50 and 100 mg) in these healthy Korean male subjects. The 2 formulations were well tolerated in both parts of the study, with no serious AEs observed. ClinicalTrials.gov identifier: NCT01769638 [ABSTRACT FROM AUTHOR]

SUMMARY Objective The objective of this study is to evaluate the economic benefits of immunoglobulin replacement therapy achieved subcutaneously (subcutaneous immunoglobulin, SCIG) by the rapid push method compared to intravenous infusion therapy (intravenous immunoglobulin, IVIG) in primary immune deficiency ( PID) patients from the healthcare system perspective in the context of the adult SCIG home infusion program based at St Paul's Hospital, Vancouver, Canada. Materials and methods SCIG and IVIG options were compared in cost-minimisation and budget impact models (BIMs) over 3 years. Sensitivity analyses were performed for both models to evaluate the impact of varying modality of IVIG treatments and proportion of patients switching from IVIG to SCIG. Results The cost-minimisation model estimated that SCIG treatment reduced cost to the healthcare system per patient of $5736 over 3 years, principally because of less use of hospital personnel. This figure varied between $5035 and $8739 depending on modality of IVIG therapy. Assuming 50% of patients receiving IVIG switched to SCIG, the BIM estimated cost savings for the first 3 years at $1·308 million or 37% of the personnel and supply budget. These figures varied between $1·148 million and $2·454 million (36 and 42%) with varying modalities of IVIG therapy. If 75% of patients switched to SCIG, the reduced costs reached $1·962 million or 56% of total budget. Conclusion This study demonstrated that from the health system perspective, rapid push home-based SCIG was less costly than hospital-based IVIG for immunoglobulin replacement therapy in adult PID patients in the Canadian context. [ABSTRACT FROM AUTHOR]

The purpose of the study was to conduct a cost-effectiveness analysis and budget impact analysis comparing lopinavir with ritonavir (LPV/r) and atazanavir plus ritonavir (ATV+RTV) for antiretroviral-naïve patients with a baseline CD4+ T-cell distribution and total cholesterol (TC) profile as reported in the CASTLE study. This decision analysis study used a previously published Markov model of HIV disease, incorporating coronary heart disease (CHD) events to compare the short- and long-term budget impacts and CHD consequences expected for the two regimens. Patients were assumed to have a baseline CHD risk of 4.6% (based on demographic data) and it was also assumed that 50% of the population in the CASTLE study were smokers. The CHD risk differences (based on percent of patients with TC >240 mg/dL) in favor of ATV+RTV resulted in an average improvement in life expectancy of 0.031 quality-adjusted life years (QALYs) (11 days), and an incremental cost-effectiveness ratio of $1,409,734/QALY. Use of the LPV/r regimen saved $24,518 and $36,651 at 5 and 10 years, respectively, with lifetime cost savings estimated at $38,490. A sensitivity analysis using a cohort of all smokers on antihypertensive medication estimated an average improvement in life expectancy of 31 quality-adjusted days in favor of ATV+RTV, and a cost-effectiveness ratio of $520,861/QALY: a ratio that is still above the acceptable limit within the US. The use of an LPV/r-based regimen in antiretroviral-naïve patients with a baseline CHD risk similar to patients in the CASTLE study appears to be a more cost-effective use of resources compared with an ATV+RTV-based regimen. The very small added CHD risk predicted by LPV/r treatment is more than offset by the substantial short- and long-term cost savings expected with the use of LPV/r in antiretroviral-naïve individuals with average to moderately elevated CHD risk. [ABSTRACT FROM AUTHOR]

A budget impact model was used to estimate the effect of introducing inhaled salmeterol/fluticasone propionate combination on asthma drug-related costs in France. The model is based on disease prevalence, drug use, drug acquisition cost and sales forecasting data specific to France. It takes the perspective of social security and has a time horizon of 1 year. All drug acquisition costs are adjusted for the average rate of reimbursement of asthma medications in France (77.3%). All costs are expressed in euros. The model shows that if patients receiving concurrent salmeterol plus fluticasone propionate (or budesonide plus formoterol switch to combined salmeterol/fluticasone propionate, the anticipated annual savings would be 2,691,580 and 1,916,966 euros, respectively. On a fixed budget 4067 additional patients could be treated if salmeterol/fluticasone propionate is substituted for concurrent salmeterol plus fluticasone propionate and an additional 2939 patients if the combination is used to replace concurrent budesonide plus formoterol. Overall, using sales forecasting data to estimate how many patients will switch to the new combination from their current therapy, it is estimated that the introduction of salmeterol/fluticasone propionate will increase the national expenditure in France on asthma medications by a maximum of 3%. This budget impact model shows that the introduction of inhaled salmeterol/fluticasone propionate combination is likely to have minimal impact on asthma-related medication costs in France. Moreover, the available data on the salmeterol/fluticasone propionate combination suggest that it is clinically effective, cost-effective and affordable to the French healthcare system. [ABSTRACT FROM AUTHOR]

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.

Background: In French hospitals, the supply and management of drugs are missions of pharmacists. The aim of this study is to assess the use of efficacy and economics opinions of the Haute Autorite de santé (HAS) in hospital referencing of drugs in France in 2017., Methods: A questionnaire for hospital pharmacists was developed to establish their knowledge and their uses of Transparency Commission and Economic and Public Health Evaluation Committee opinions. This survey was distributed by the ADIPH association in July 2017. This questionnaire included 35 questions., Results: Despite the health professional are more and more interesting by economics analysis, only 30 % of hospitals pharmacists of this panel declared to use HAS economic opinions (versus 80% for Transparency Commission opinions). Among these pharmacists, 86% used this report in the hospital referencing of drugs., Conclusion: Through this analysis, some prospects for improvement can be seen in the health professional formation, cost-effectiveness report publication and the use of cost-effectiveness analysis. This study is an overview of the use of opinions provided by the HAS. This thesis established a basis to reflection on the use of economics reports and models in the hospital decisions., (Copyright © 2018 Académie Nationale de Pharmacie. Published by Elsevier Masson SAS. All rights reserved.)

Background: Omadacycline is an oral and intravenous (IV) once-daily aminomethylcycline antibiotic that is approved in the United States for the treatment of adults with acute bacterial skin and skin structure infections (ABSSSI). It has broad-spectrum activity against common causative pathogens of ABSSSI, including methicillin-resistant Staphylococcus aureus . Omadacycline has been shown to be noninferior to linezolid for the treatment of adults with ABSSSI across 2 phase 3 clinical trials. To date, no studies have assessed the budget impact for omadacycline in the treatment of ABSSSI., Objectives: To estimate the potential budget impact of introducing omadacycline as a treatment option in patients who present to the emergency department (ED) with ABSSSI from the hospital perspective (Medicare payer) in the United States. The ED's and observation units were assumed to be hospital-owned., Methods: The base case of this decision model-based analysis was conducted from the perspective of a hospital for a theoretical cohort of 1 million covered Medicare members over a 3-year time horizon. Scenario analyses included the economic impact of (1) shifting inpatient care to the outpatient setting with omadacycline and (2) reducing hospital length of stay (LOS) among hospitalized patients with omadacycline IV to oral therapy relative to the current inpatient standard of care. Costs are presented in 2017 US dollars with no adjustments for inflation, based on the cost model estimates., Results: The annual total incremental cost following the introduction of omadacycline as a treatment of ABSSSI was $11,168, $39,918, and $88,777 in years 1, 2, and 3, respectively. The incremental cost per member treated (cost per case) rose by $0.49, $1.74, and $3.86 over 3 years. Reducing hospital LOS by 1 day among hospitalized patients with omadacycline resulted in incremental costs of $4311, $15,231, and $33,919 in years 1, 2, and 3, respectively. Under the assumption that patients may be discharged sooner when an oral formulation of the same drug with which they are being treated is available, reducing hospital LOS by 2 days reduced costs by $2546, $9455, and $20,939 in years 1, 2, and 3, respectively. Shifting inpatient care to the outpatient setting with omadacycline reduced costs by $38,777, $139,885, and $310,784 in years 1, 2, and 3, respectively., Conclusion: This hypothetical, model-based study determined that omadacycline would result in a modest increase in total cost over 3 years when introduced as a treatment for ABSSSI in adults who present to the ED for their care.

Background: During cataract surgery, maintaining an adequate degree of mydriasis throughout the entire operation is critical to allow for visualisation of the capsulorhexis and the crystalline lens. Good anaesthesia is also essential for safe intraocular surgery. Mydrane® is a new injectable intracameral solution containing two mydriatics (tropicamide 0.02% and phenylephrine 0.31%) and one anaesthetic (lidocaine 1%) that was developed as an alternative to the conventional topical pre-operative mydriatics used in cataract surgery. This study aimed to estimate the budget impact across a one year time frame using Mydrane® instead of topical dilating eye drops, for a UK hospital performing 3,000 cataract operations a year.Methods: A budget impact model (BIM) was developed to compare the economic outcomes associated with the use of Mydrane® versus topical drops (tropicamide 0.5% and phenylephrine 10%) in patients undergoing cataract surgery in a UK hospital. The outcomes of interest included costs and resource use (e.g. clinician time, mydriasis failures, operating room time, number of patients per vial of therapy etc.) associated with management of mydriasis in patients undergoing cataract surgery. All model inputs considered the UK hospital perspective without social or geographical variables. Deterministic sensitivity analyses were also performed to assess the model uncertainty.Results: Introduction of Mydrane® is associated with a cost saving of £6,251 over 3,000 cataract surgeries in one year. The acquisition costs of the Mydrane® (£18,000 by year vs. £3,330 for eye drops) were balanced by substantial reductions in mainly nurses' costs and time, plus a smaller contribution from savings in surgeons' costs (£20,511) and lower costs associated with auxiliary dilation (£410 due to avoidance of additional dilation methods). Results of the sensitivity analyses confirmed the robustness of the model to the variation of inputs. Except for the duration of one session of eye drop instillation and the cost of Mydrane®, Mydrane® achieved an incremental cost gain compared to tropicamide/phenylephrine eye drops.Conclusions: Despite a higher acquisition cost of Mydrane®, the budget impact of Mydrane® on hospital budgets is neutral. Mydrane® offers a promising alternative to traditional regimes using eye drops, allowing for a better patient flow and optimisation of the surgery schedule with neutral budget impact. [ABSTRACT FROM AUTHOR]

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.