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BACKGROUND: Budget impact models (BIMs) forecast the financial implications of adopting new technologies and the potential need for budget reallocation, thus playing a crucial role in reimbursement decisions. Despite the importance of accurate forecasts, studies indicate large discrepancies between estimates and reality. We are developing an artificial intelligence-based clinical decision tool to identify patients with non-small cell lung cancer who are most likely to benefit from immunotherapy. OBJECTIVE: To evaluate the budgetary implications and describe a systematic literature review of published lung cancer BIMs. METHODS: We searched PubMed and EMBASE for studies published between 2010 and 2023 that include BIMs that describe lung cancer interventions. Forward and backward reference searches were performed for all qualifying studies. We extracted author and publication year, country, interventions, disease stages, time horizon, analytical perspective, modeling methods used, types of costs included, sensitivity analyses conducted, and data sources used. We then evaluated adherence to the Professional Society for Health Economics and Pharmacoeconomics Research best-practice guidelines. RESULTS: A total of 25 BIMs were identified, spanning 14 different countries. Model structure could not be ascertained definitively for nearly half of the models. The cost calculator approach was most common among the others. Time horizons ranged from 1 to 5 years, in line with recommendations. Most models compared drugs, 4 compared nondrug interventions, and 7 compared diagnostic technologies. Assumptions about market uptake were poorly documented and poorly motivated. Inclusion of cancer-related costs was rare. Adherence to best practices was variable and did not appear to improve over time. CONCLUSIONS: The number of published BIMs for lung cancer exceeded expectations. There were modest trends toward publication frequency and model quality over time. Our analysis revealed variability across the models, as well as their adherence to best practices, indicating substantial room for improvement. Although none of the models were individually suitable for the purpose of evaluating an artificial intelligence-based treatment selection tool, some models provided valuable insights.
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Presupuestos , Neoplasias Pulmonares , Modelos Económicos , Humanos , Neoplasias Pulmonares/economía , Neoplasias Pulmonares/terapia , Carcinoma de Pulmón de Células no Pequeñas/economía , Carcinoma de Pulmón de Células no Pequeñas/terapia , Carcinoma de Pulmón de Células no Pequeñas/tratamiento farmacológico , Inmunoterapia/economía , Análisis Costo-BeneficioAsunto(s)
Inteligencia Artificial , Presupuestos , Unión Europea , Gobierno Federal , Liderazgo , Ciencia , Inteligencia Artificial/legislación & jurisprudencia , Política , Apoyo a la Investigación como Asunto/legislación & jurisprudencia , Apoyo a la Investigación como Asunto/tendencias , Ciencia/legislación & jurisprudencia , Ciencia/tendencias , Reino UnidoRESUMEN
INTRODUCTION: As a result of the success of Maryland's full risk capitated payment model experiment (Global Budget Revenue) in constraining healthcare costs, there is momentum for expanding the reach of such models. However, as these models are implemented, studies analyzing their long-term effects suggest unintended spillover effects that may ultimately influence patient experiences. The aim of this study was to determine whether implementation of the GBR was associated with changes in patient experience. METHODS: Cross-sectional study using a difference-in-difference analysis to examine changes in patient experiences according to the Hospital Consumer Assessment of Healthcare Providers and Systems (HCAHPS) domains before and after implementation of the GBR model. Acute care hospitals from 2010-2016 with completed HCAHPS surveys were included. Hospitals identified for inclusion were then matched, based on county location, to area level characteristics using the Area Health Resource File. RESULTS: A total of 844 hospitals were included. Compared to hospitals in non-GBR states, hospitals in GBR states experienced significant declines in the following HCAHPS domains: "would definitely recommend the hospital to others" [Average treatment effect (ATT) = -1.19, 95% CI = -1.97, -0.41)] and 9-10 rating of the hospital (ATT = -0.93, 95% CI = -1.71, -0.15). Results also showed significant increases in the HCAHPS domains: "if patient's rooms and bathroom were always kept clean" (ATT = 1.10, 95% CI = 0.20, 2.00). There were no significant differences in changes for the other domains, including no improvements in: nursing communication, doctor communication, help from hospital staff, pain control, communication on medicines, discharge information, and quietness of the patient environment. CONCLUSION: These findings suggest there should be efforts made to ascertain and mitigate potential adverse effects of care transformation initiatives on patient experience. Patients are stakeholders and their inputs should be sought and incorporated in care transformation efforts to ensure that these models align with improved patient experiences.
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Presupuestos , Satisfacción del Paciente , Humanos , Maryland , Estudios Transversales , Hospitales , Economía HospitalariaRESUMEN
PURPOSE: This study quantifies the impact on budget and cost per health benefit of implementing digital breast tomosynthesis (DBT) in place of digital mammography (DM) for breast cancer screening among asymptomatic women in Italy. METHODS: A budget impact analysis and a cost consequence analysis were conducted using parameters from the MAITA project and literature. The study considered four scenarios for DBT implementation, i.e., DBT for all women, DBT for women aged 45-49 years, DBT based on breast density (BI-RADS C + D or D only), and compared these to the current DM screening. Healthcare provider's perspective was adopted, including screening, diagnosis, and cancer treatment costs. RESULTS: Introducing DBT for all women would increase overall screening costs by 20%. Targeting DBT to women aged 45-49 years or with dense breasts would result in smaller cost increases (3.2% for age-based and 1.4-10.7% for density-based scenarios). The cost per avoided interval cancer was significantly higher when DBT was applied to all women compared to targeted approaches. The cost per gained early-detected cancer slightly increases in targeted approaches, while the assumptions on the clinical significance and overdiagnosis of cancers detected by DBT and not by DM have a strong impact. CONCLUSIONS: Implementing DBT as a primary breast cancer test in screening programs in Italy would lead to a substantial increase in costs. Tailoring DBT use to women aged 45-49 or with dense breasts could enhance the feasibility and sustainability of the intervention. Further research is needed to clarify the impact of DBT on overdiagnosis and the long-term outcomes.
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Neoplasias de la Mama , Presupuestos , Análisis Costo-Beneficio , Detección Precoz del Cáncer , Mamografía , Humanos , Neoplasias de la Mama/diagnóstico por imagen , Femenino , Italia , Mamografía/economía , Mamografía/métodos , Persona de Mediana Edad , Detección Precoz del Cáncer/economía , Detección Precoz del Cáncer/métodos , AncianoRESUMEN
AIMS: Fruquintinib is a selective small molecule tyrosine kinase inhibitor of vascular endothelial growth factor receptor (VEGFR)-1, -2, and -3 recently approved in the United States (US) for the treatment of adult patients with metastatic colorectal cancer (CRC) who have previously been treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, an anti-VEGF biological therapy, and if RAS wild-type and medically appropriate, anti-epidermal growth factor receptor therapy. This study aimed to estimate the 5-year budget impact of fruquintinib from a US payer perspective (commercial and Medicare). MATERIALS AND METHODS: A budget impact model was developed to compare two scenarios: a reference scenario in which patients received regorafenib, trifluridine/tipiracil, or trifluridine/tipiracil with bevacizumab and an alternative scenario in which patients received reference scenario treatments or fruquintinib. Market shares were evenly divided across available options. A 5-year time horizon and a hypothetical health plan of 1 million members was assumed. The model included epidemiological inputs to estimate the eligible population; clinical inputs for treatment duration, progression-free survival, overall survival, and adverse event (AE) frequency; and cost inputs for treatment, AEs, disease management, subsequent therapy, and terminal care costs. Budget impact was reported as total, per member per year (PMPY), and per member per month (PMPM). RESULTS: The model estimated an eligible population of 194 patients (39 per year) over 5 years. In the base case, the estimated 5-year budget impact of fruquintinib was $4,077,073 ($0.82 PMPY and 0.07 PMPM) for a commercial health plan. During the first year, the estimated budget impact was $627,570 ($0.63 PMPY and 0.05 PMPM). Results were robust across sensitivity analyses. PMPM costs from the Medicare perspective were greater than the base-case (commercial) ($0.17 vs. $0.07) due to higher incidence of CRC in that population. CONCLUSIONS: Fruquintinib is associated with a low budget impact for payers based on proposed thresholds in the US.
Fruquintinib is a treatment for metastatic colorectal cancer that has progressed after or not responded to multiple guideline-recommended therapies. This budget impact analysis was conducted to estimate the added costs a health plan would incur over a 5-year period if it chose to cover this therapy. The analysis found that the per plan member per month cost of covering fruquintinib was $0.07 for a United States commercial health plan and $0.17 for Medicare.
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Protocolos de Quimioterapia Combinada Antineoplásica , Benzofuranos , Bevacizumab , Neoplasias Colorrectales , Piridinas , Timina , Humanos , Neoplasias Colorrectales/tratamiento farmacológico , Benzofuranos/uso terapéutico , Benzofuranos/economía , Estados Unidos , Bevacizumab/uso terapéutico , Bevacizumab/economía , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Protocolos de Quimioterapia Combinada Antineoplásica/economía , Piridinas/uso terapéutico , Piridinas/economía , Trifluridina/uso terapéutico , Trifluridina/economía , Presupuestos , Quinazolinas/uso terapéutico , Quinazolinas/economía , Compuestos de Fenilurea/uso terapéutico , Compuestos de Fenilurea/economía , Uracilo/análogos & derivados , Uracilo/uso terapéutico , Uracilo/economía , Compuestos Organoplatinos/uso terapéutico , Compuestos Organoplatinos/economía , Análisis Costo-Beneficio , Camptotecina/análogos & derivados , Camptotecina/uso terapéutico , Camptotecina/economía , Antineoplásicos/economía , Antineoplásicos/uso terapéutico , Irinotecán/uso terapéutico , Irinotecán/economía , Medicare , Fluorouracilo/uso terapéutico , Fluorouracilo/economía , Oxaliplatino/uso terapéutico , Oxaliplatino/economía , Receptores de Factores de Crecimiento Endotelial Vascular , Modelos Económicos , Combinación de Medicamentos , PirrolidinasRESUMEN
BACKGROUND AND OBJECTIVE: Treatment of sickle cell disease (SCD) has traditionally focused on symptomatic and preventative care. Recent advances in novel therapeutic developments, likely to be orphan-designated, are anticipated to carry a substantial price tag. This study assesses the potential budget impact of adopting disease-modifying treatments, crizanlizumab and voxelotor, and pioneering CRISPR gene-edited therapy, CTX001, in the Belgian healthcare system. METHODS: The perspective of the Belgian healthcare payer (RIZIV-INAMI including patient copayments), a 5-year horizon with a 2-10% uptake of disease-modifying interventions, and a 2% uptake of CTX001 were considered. Data, encompassing target population, current (chronic and acute management, curative hematopoietic stem cell transplantation) and new (crizanlizumab, voxelotor, and CTX001) interventions, clinical effectiveness, adverse events, healthcare resource utilization, and associated costs, were gathered through a comprehensive literature review (first phase) and two Delphi panels involving hematologists (second phase). The cost difference between a "world with and without crizanlizumab, voxelotor, and CTX001" was calculated to obtain the budget impact. Three scenario analyses were conducted: a 5-13% and 4% uptake analysis, a 10-18% and 8% uptake analysis, respectively for disease-modifying treatments (crizanlizumab and voxelotor) and CTX001, and a 0% crizanlizumab uptake and managed entry agreements analysis . A ± 20% univariate sensitivity analysis was performed to test the robustness of the analysis. RESULTS: The total five-year cumulative budget impact was estimated at 30,024,968, with 91% attributed to drug acquisition costs. The largest budget impact share was for CTX001 (25,575,150), while crizanlizumab (2,301,095) and voxelotor (2,148,723) was relatively small. In scenarios one and three, a two-fold increase of the cumulative budget impact to 60,731,772 and a four-fold increase to 120,846,256 from the base case was observed. In scenario three, this budget impact decreased by 63% to 11,212,766. Patient population size, number of treated patients, and drug costs influenced the analysis the most, while discontinuation, acute crisis, and adverse event rates had comparatively minimal impact. CONCLUSIONS: Belgian decision-makers may consider alternative financing models, such as outcome-based risk-sharing agreements or annuities, to ensure sustainable coverage of these treatments. This study adheres to recommended practices for assessing budget impact of orphan drugs, distinguishing it from earlier studies with potentially weaker methodologies.
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Anemia de Células Falciformes , Anticuerpos Monoclonales Humanizados , Presupuestos , Humanos , Anemia de Células Falciformes/genética , Anemia de Células Falciformes/tratamiento farmacológico , Anemia de Células Falciformes/economía , Anemia de Células Falciformes/terapia , Anticuerpos Monoclonales Humanizados/economía , Anticuerpos Monoclonales Humanizados/uso terapéutico , Bélgica , Edición Génica/métodos , Edición Génica/economía , Sistemas CRISPR-Cas , Terapia Genética/economía , Terapia Genética/métodos , Análisis Costo-BeneficioRESUMEN
Two vaccines against rotavirus diseases, Rotarix® and RotaTeq®, are being marketed in Spain; but rotavirus is not presently among the diseases covered by universal vaccination in Spain. The aim of this study was to assess the efficiency of extending Spain's current targeted rotavirus vaccination strategy including only preterm babies, to a policy of universal vaccination. A de novo cohort-based Markov model was built to evaluate the efficiency of three compared rotavirus vaccination strategies in Spain: targeted, universal, and no vaccination. Using Rotarix® or RotaTeq®, we compared the cost-utility of these strategies from both a societal perspective and Spanish National Health System (SNHS) perspective. The model represents the most important clinical events conceivably linked to rotavirus infection. Efficacy, effectiveness, safety, costs, and utilities were identified by systematic reviews. Incremental cost-utility ratio (ICUR) is EUR 23,638/QALY (Quality-Adjusted Life Year) for targeted vaccination with Rotarix® compared with no vaccination. The ICUR for the rest of the strategies evaluated are above EUR 30,000/QALY. The sensitivity analysis shows price as the only parameter that could make the universal vaccination strategy efficient. Considering a threshold of EUR 25,000/QALY, only targeted vaccination with Rotarix® would be efficient from societal perspective. Price drops of 36.9% for Rotarix® and 44.6% for RotaTeq® would make universal vaccination efficient.
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Análisis Costo-Beneficio , Años de Vida Ajustados por Calidad de Vida , Infecciones por Rotavirus , Vacunas contra Rotavirus , Vacunación , Vacunas Atenuadas , Vacunas contra Rotavirus/economía , Vacunas contra Rotavirus/administración & dosificación , Vacunas contra Rotavirus/inmunología , España , Humanos , Infecciones por Rotavirus/prevención & control , Infecciones por Rotavirus/economía , Vacunación/economía , Vacunas Atenuadas/economía , Vacunas Atenuadas/administración & dosificación , Vacunas Atenuadas/inmunología , Lactante , Rotavirus/inmunología , Cadenas de Markov , Preescolar , Recién Nacido , PresupuestosRESUMEN
AIMS: This study assessed the budget impact of resmetirom as a treatment for adults with non-cirrhotic non-alcoholic steatohepatitis (NASH) with moderate-to-advanced liver fibrosis and estimated total costs for a hypothetical private payer in the United States. MATERIALS AND METHODS: A three-year budget impact analysis based on an open cohort state transition model was developed for a hypothetical one-million-member private health plan. The comparator was Standard of Care (SOC), defined as routine care for non-cirrhotic NASH patients with moderate-to-advanced liver fibrosis. Each year, the number of resmetirom treatment-eligible patients was estimated through prevalent, incident, and diagnostic rate estimates. Costs included resources incurred by the medical and pharmacy benefits of private payers, including resmetirom drug acquisition costs, diagnosis and monitoring, other medical and other prescription costs stratified by disease progression status (i.e. non-cirrhotic vs. cirrhotic/advanced liver diseases). Resmetirom adverse event management costs were included in sensitivity analysis. Drug costs were estimated based on the average wholesale acquisition cost as of March 2024. Other costs were based on published sources and inflated to 2023 US dollars. Budget impact outcomes were presented in aggregate, net, and on a per-member per-month (PMPM) basis. RESULTS: Compared with a scenario without resmetirom, the introduction of resmetirom yielded results ranging from 50 to 238 treated patients, net budget impact of $2.2 to $9.5 million, and PMPM from $0.19 to $0.80 over years one and three. Net costs excluding resmetirom declined over time. In sensitivity analyses, results were most sensitive to diagnostic and epidemiologic inputs. LIMITATIONS: Market shares are based on internal forecasts, a short time horizon, average treatment effects, and other limitations common to BIMs. CONCLUSION: The adoption of resmetirom on the formulary for the treatment of non-cirrhotic NASH with moderate-to-advanced liver fibrosis resulted in a moderate increase in budget impact with declining costs related to NASH progression.
Non-alcoholic steatohepatitis (NASH) is a serious liver disease that can lead to significant liver damage, other health complications, and increased healthcare costs. As the disease progresses, patients typically experience worsening health outcomes. Until recently, there were no Food and Drug Administration (FDA) approved treatments for NASH in the United States. However, in March 2024, the FDA approved REZDIFFRA, a new drug specifically designed to treat NASH patients with moderate-to-advanced liver fibrosis (i.e. NASH with moderate-to-advanced scarring of the liver). Clinical trials have shown that REZDIFFRA can improve health outcomes in these patients.To identify patients who could benefit from REZDIFFRA and to estimate the associated costs, we developed a budget impact model. In this study, we detail the development of this model and present its findings. Our analysis revealed that, while REZDIFFRA is associated with higher overall costs, primarily due to the price of the drug itself, there are potential cost savings when considering the drug's ability to slow disease progression.
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Presupuestos , Cirrosis Hepática , Enfermedad del Hígado Graso no Alcohólico , Humanos , Enfermedad del Hígado Graso no Alcohólico/economía , Enfermedad del Hígado Graso no Alcohólico/tratamiento farmacológico , Cirrosis Hepática/economía , Estados Unidos , Índice de Severidad de la Enfermedad , Adulto , Progresión de la Enfermedad , Modelos Econométricos , Masculino , Análisis Costo-Beneficio , FemeninoRESUMEN
OBJECTIVES.: To analyze the budget impact of upadacitinib (UPA) 15 mg + methotrexate (MTX) for the treatment of moderate-to-severe rheumatoid arthritis (RA) in patients with an inadequate response to conventional disease-modifying antirheumatic drugs (cDMARD-IR) from the perspective of social security and the private health sector in Argentina. MATERIALS AND METHODS.: A budget impact analysis model was developed for a hypothetical cohort of 100,000 adults with health insurance coverage who were diagnosed with RA over a 5-year time horizon. The model parameters were obtained through literature review and validated by local experts. The costs are expressed in 2024 US dollars (USD). RESULTS.: The introduction of UPA 15 mg + MTX for the treatment of moderate-to-severe RA and cDMARD-IR resulted in minimal increase, with a five-year total cumulative incremental cost of USD 1,855 for social security and USD 1,812 for the private health sector, representing 2% of the total budget. The acquisition cost of UPA was the most influential variable in the sensitivity analysis. CONCLUSIONS.: The introduction of UPA 15 mg + MTX for the treatment of moderate-to-severe RA and cDMARD-IR can provide an effective treatment option with a minimal increase in costs for the healthcare system in Argentina, which is especially important in developing countries where health system budgets are more limited. Providing evidence-based estimates is a valuable tool for informing healthcare policies and can help policymakers make informed decisions about the allocation of healthcare resources to improve patient outcomes while also managing costs.Motivation for the study. Rheumatoid arthritis (RA) is a disease that hasn't cure, so it's important to know the budget impact of treatment with upadacitinib (UPA) 15 mg + methotrexate (MTX) in patients with moderate to severe RA who didn't respond well to conventional antirheumatic drugs. Main findings. UPA + MTX would entail a minimal increase in costs for the healthcare system in Argentina, potentially making this effective treatment option more accessible to patients with RA. Access to this treatment can improve the outcome of patients with RA. Public health implications. In resource-constrained settings such as Argentina, providing evidence-based cost estimates can help healthcare managers allocate resources efficiently while improving patient outcomes. This study provides evidence to inform healthcare policies and decisions regarding the inclusion of UPA + MTX in treatment guidelines or formularies for RA management.
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Antirreumáticos , Artritis Reumatoide , Presupuestos , Compuestos Heterocíclicos con 3 Anillos , Metotrexato , Argentina , Humanos , Artritis Reumatoide/tratamiento farmacológico , Artritis Reumatoide/economía , Antirreumáticos/economía , Antirreumáticos/uso terapéutico , Metotrexato/economía , Metotrexato/uso terapéutico , Compuestos Heterocíclicos con 3 Anillos/economía , Compuestos Heterocíclicos con 3 Anillos/uso terapéutico , Índice de Severidad de la Enfermedad , Quimioterapia CombinadaRESUMEN
AIM: To estimate the budget impact of adding a toripalimab regimen as a treatment option to the existing pembrolizumab regimen, both including gemcitabine and cisplatin, in untreated recurrent/metastatic nasopharyngeal carcinoma (R/M NPC) using the published wholesale acquisition cost (WAC) and average sales price (ASP). METHODS: Budget impact analysis comparing a treatment mix "without" versus "with" the toripalimab regimen in the US eligible annual incident R/M NPC population, a 3-year time horizon, toripalimab/pembrolizumab market splits of 60/40 (Y1) and 80/20 (Y2/3), and medication adjustments for discontinuation or progression. Cost inputs included drugs, administration, and adverse event (AE) management. The models were replicated for a hypothetical 1-million-member health plan in which costs per-member-per-month (PMPM) and per-member-per-year (PMPY) were estimated. One-way (OWSA) and probabilistic sensitivity analyses (PSA) as well as scenario analyses were performed. RESULTS: In the "without" scenario, the 3-year WAC-based costs for the pembrolizumab regimen total $1,449,695,333 ($1,305,632,448 for treatment and $144,062,885 for managing AEs). In the "with" scenario, total 3-year costs for pembrolizumab decline to $380,012,135 with toripalimab adding $885,505,900 ($779,206,567 for treatment and $106,299,333 for AE management). Annual net savings range from $46,526,152 in 2024 to $71,194,214 in 2026, for 3-year savings of $184,177,298. Associated net savings in a 1-million-member health plan are $543,068 over 3 years with savings of $0.045 PMPM and $0.543 PMPY. The ASP-based model shows similar patterns with 3-year net savings of $174,235,983 in the US incident population and savings of $0.043 PMPM and $0.514 PMPY in a 1-million-member health plan. The PSA support base case findings; OWSA and scenario analyses reveal how parameter variability impacts results. CONCLUSION: Savings between $174 million and $184 million can be achieved from treating 60% of R/M NPC patients in year 1 and 80% in years 2 and 3 with the toripalimab regimen over a similar pembrolizumab regimen.
Toripalimab, a human monoclonal anti-body that targets PD-1, was recently approved by the US Food and Drug Administration (FDA) for the first-line treatment of adults with metastatic or recurrent, locally advanced nasopharyngeal carcinoma (NPC), in combination with gemcitabine and cisplatin. We evaluated how much it would cost a payor to cover the FDA-approved toripalimab plus gemcitabine and cisplatin regimen (the toripalimab regimen) to a non-FDA-approved pembrolizumab plus gemcitabine and cisplatin regimen (the pembrolizumab regimen). With no trial data available for such pembrolizumab regimen, we assumed that it would be comparable to the toripalimab regimen in efficacy and safety. Our model adopted a 3-year time horizon and assumed a 60/40 market share split in year 1 and an 80/20 market split in years 2 and 3. It included two US cost inputs: the wholesale acquisition cost (WAC) or "list price" at market entry and, as no average sales price (ASP) will be available for toripalimab for several quarters, a toripalimab price point of 80% of the pembrolizumab ASP. We adjusted for patients whose cancer progressed or who discontinued treatment to determine the number of fully-treated-patient-equivalents. We found that treating 60% of NPC patients in year 1 and 80% in years 2 and 3 with the toripalimab regimen instead of the pembrolizumab regimen generates, for the entire adjusted patient population, savings ranging from $174 million when using ASP to $184 million when using WAC.
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Anticuerpos Monoclonales Humanizados , Carcinoma Nasofaríngeo , Neoplasias Nasofaríngeas , Anticuerpos Monoclonales Humanizados/uso terapéutico , Anticuerpos Monoclonales Humanizados/economía , Humanos , Carcinoma Nasofaríngeo/tratamiento farmacológico , Carcinoma Nasofaríngeo/economía , Neoplasias Nasofaríngeas/tratamiento farmacológico , Neoplasias Nasofaríngeas/economía , Neoplasias Nasofaríngeas/patología , Recurrencia Local de Neoplasia/tratamiento farmacológico , Análisis Costo-Beneficio , Protocolos de Quimioterapia Combinada Antineoplásica/economía , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Antineoplásicos Inmunológicos/economía , Antineoplásicos Inmunológicos/uso terapéutico , Inhibidores de Puntos de Control Inmunológico/uso terapéutico , Inhibidores de Puntos de Control Inmunológico/economía , Desoxicitidina/análogos & derivados , Desoxicitidina/uso terapéutico , Desoxicitidina/economía , Modelos Econométricos , Presupuestos , Gemcitabina , Metástasis de la Neoplasia , Estados Unidos , Gastos en Salud/estadística & datos numéricosRESUMEN
OBJECTIVES: Biosimilars provide an opportunity for a more sustainable and cost-effective treatment for multiple sclerosis (MS). This study evaluated the potential financial impact of implementing a formulary change from reference to biosimilar natalizumab (NTZ) from the US commercial payer perspective. STUDY DESIGN: The budget impact of transitioning to biosimilar NTZ for the treatment of relapsing-remitting MS (RRMS) was estimated over a 3-year time horizon based on real-world dosing. Additional scenario analyses were conducted by varying the price differential of biosimilar NTZ. METHODS: The target population was estimated from a 1-million-member hypothetical commercial health plan. Model inputs were drug acquisition costs and treatment-related and patient coinsurance costs. Budget impact and cost savings per member per year were calculated by assuming a biosimilar uptake of 10% in year 1 to 20% in year 3. RESULTS: Over 3 years, 255 patients were estimated to be treated with high-efficacy disease-modifying therapies for RRMS. The inclusion of biosimilar NTZ onto a formulary would result in cumulative cost savings to payers of $452,611 over 3 years, with mean savings per treated member per year of $1179, $1769, and $2359 in years 1, 2, and 3, respectively. One-way sensitivity analyses indicated that budget impact results were most sensitive to drug acquisition costs of both reference and biosimilar NTZ. CONCLUSION: Adoption of biosimilar NTZ can yield considerable cost savings to US health plans that could result in increased treatment access for patients with RRMS.
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Biosimilares Farmacéuticos , Presupuestos , Natalizumab , Humanos , Natalizumab/uso terapéutico , Natalizumab/economía , Estados Unidos , Biosimilares Farmacéuticos/economía , Biosimilares Farmacéuticos/uso terapéutico , Anticuerpos Monoclonales Humanizados/uso terapéutico , Anticuerpos Monoclonales Humanizados/economía , Esclerosis Múltiple Recurrente-Remitente/tratamiento farmacológico , Esclerosis Múltiple Recurrente-Remitente/economía , Análisis Costo-Beneficio , Ahorro de Costo , Costos de los Medicamentos/estadística & datos numéricosRESUMEN
This study aimed to assess the cost-utility and budget impact of dual to single HER2 targeted neoadjuvant therapy for HER2-positive breast cancer in Sri Lanka. A five-health state Markov model with lifetime horizon was used to assess the cost-utility of neoadjuvant trastuzumab (T) plus pertuzumab (P) or lapatinib (L) compared to single therapy of T with chemotherapy (C), in public healthcare system and societal perspectives. Input parameters were estimated using local data, network meta-analysis, published reports and literature. Costs were adjusted to year 2021 (1USD = LKR194.78). Five-year budget impact for public healthcare system was assessed. Incremental cost-effectiveness ratios in societal perspective for neoadjuvantLTC plus adjuvantT (strategy 3), neoadjuvantPTC plus adjuvantT (strategy 2), neoadjuvantLTC plus adjuvantLT (strategy 5), and neoadjuvantPTC plus adjuvantPT (strategy 4) compared to neoadjuvantTC plus adjuvantT (strategy 1) were USD2716, USD5600, USD6878, and USD12127 per QALY gained, respectively. One GDP per-capita (USD3815) was considered as the cost-effectiveness threshold for the analysis. Even though only the ICER for strategy 3 was cost-effective, uncertainty of efficacy parameter was revealed. For strategy 2 neoadjuvant PTC plus adjuvant T, a 25% reduction of neoadjuvant regimen cost was required to be cost effective for use in early HER2 positive breast cancer.
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Neoplasias de la Mama , Análisis Costo-Beneficio , Lapatinib , Terapia Neoadyuvante , Receptor ErbB-2 , Trastuzumab , Humanos , Neoplasias de la Mama/tratamiento farmacológico , Neoplasias de la Mama/economía , Neoplasias de la Mama/metabolismo , Femenino , Receptor ErbB-2/metabolismo , Terapia Neoadyuvante/economía , Trastuzumab/uso terapéutico , Trastuzumab/economía , Sri Lanka , Lapatinib/uso terapéutico , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Protocolos de Quimioterapia Combinada Antineoplásica/economía , Cadenas de Markov , Anticuerpos Monoclonales Humanizados/uso terapéutico , Anticuerpos Monoclonales Humanizados/economía , Años de Vida Ajustados por Calidad de Vida , Presupuestos , Persona de Mediana EdadRESUMEN
Adults with a learning disability who receive social care are legally entitled to a personal budget. Personal budgets were introduced to promote choice and control in support. Individual Service Funds were introduced as a flexible way that personal budgets can be managed by a provider while maintaining choice and control for the individual. Individual Service Funds have been shown to improve quality of life for individuals and efficiency in support. Despite this, only 20% of local authorities offer them to adults with a learning disability, demonstrating the need for resources to be developed to support their delivery. This protocol described a co-production study with key stakeholders to develop and refine Individual Service Fund resources. Our primary aim is to co-produce two actionable resources: to support development, delivery, consistency, and sustained provision of ISFs; and to support uptake and optimal use of ISFs by recipients. We also aim to user-test and evaluate these resources with three Local Authorities. The result of this study will be two resources that will support the uptake of Individual Service Funds for adults with a learning disability that will be freely available online.
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Discapacidades para el Aprendizaje , Humanos , Discapacidades para el Aprendizaje/economía , Discapacidades para el Aprendizaje/terapia , Presupuestos , Adulto , Calidad de VidaRESUMEN
AIMS: To estimate in a panel of patients with locally advanced/metastatic nonsquamous non-small cell lung cancer (NSCLC) treated with a programmed death receptor-1 inhibitor in the US in 2024 (1) the cost-efficiency of toripalimab regimens compared to pembrolizumab regimens; and (2) the budget-neutral expanded access to additional toripalimab cycles and regimens from accrued savings. METHODS: Simulation modeling of toripalimab + pemetrexed + carboplatin in nonsquamous NSCLC to a similar pembrolizumab regimen in a panel of 49,647 patients; utilizing two cost inputs (wholesale acquisition cost (WAC) at market entry and an estimated ex ante toripalimab price point of 80% of pembrolizumab average sales price (ASP)) plus administration costs over one and two years of treatment with treatment rates from 1%-10%. Scenario analyses with treatment durations equivalent to toripalimab and pembrolizumab trials' median PFS were also conducted. RESULTS: In the WAC-based models, toripalimab saves $2,223 per patient per cycle and $40,014 over 1 year of treatment ($77,805 over 2 years). Extrapolated to the 49,647-patient panel, estimated 1-year savings range from $19,865,840 (1% treatment rate) to $198,658,399 (10% rate). Reallocating these savings permits budget-neutral expanded access to an additional 1,753 (1% rate) to 17,533 (10% rate) toripalimab maintenance cycles or to an additional 97 (1% rate) to 972 (10%) full 1-year toripalimab regimens with all agents. Two-year savings range from $38,628,022 (1% rate) to $386,280,221 (10%). Reallocating these efficiencies provides expanded access ranging from 3,409 (1% rate) to 34,093 (10%) additional toripalimab cycles or to 97 to 973 full 2-year regimens. The ex ante ASP model showed similar results as did the scenario analyses but at a lower magnitude than the base case. CONCLUSION: Toripalimab generates significant savings that enable budget-neutral funding for up to 17,533 [34,093] additional maintenance cycles over one year [two years] with toripalimab + pemetrexed in nonsquamous NSCLC, or 972 [973] full one-year [two-year] regimens.
An estimated 49,647 patients with advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) will be treated with a PD-1 inhibitor in the US in 2024. Toripalimab, a PD-1 inhibitor recently approved by the US Food and Drug Administration for the treatment of nasopharyngeal carcinoma, has also been found to be beneficial in patients with nonsquamous NSCLC when used in combination with chemotherapy. We conducted an economic simulation of the costs of toripalimab + pemetrexed + carboplatin versus the costs of a similar regimen with the PD-1 inhibitor pembrolizumab in the treatment of patients with nonsquamous NSCLC. Our simulation models used two US cost inputs for toripalimab: the wholesale acquisition cost or "list price" at market entry and, as no average sales price (ASP) will be available for toripalimab for several quarters, a hypothetical toripalimab price point of 80% of the pembrolizumab ASP. We compared the savings in each scenario when between 1% and 10% of the 49,647 nonsquamous NSCLC patients are treated with the toripalimab regimen. We then evaluated how these savings could be re-allocated, without requiring extra funding, to provide more patients with access to toripalimab treatment on a budget-neutral basis. We found that, if 1% of new cases of advanced/metastatic nonsquamous NSCLC were treated with toripalimab for 1 year, these savings are enough to purchase up to 1,753 additional toripalimab maintenance cycles; or these savings could provide up to an additional 97 patients with full one-year regimens with all agents (toripalimab + chemotherapy). If 10% of new cases were treated with toripalimab for 1 year, the savings are enough to purchase up to 17,533 additional toripalimab maintenance cycles; or these savings could provide up to an additional 972 patients with full one-year regimens with all agents.
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Anticuerpos Monoclonales Humanizados , Protocolos de Quimioterapia Combinada Antineoplásica , Carcinoma de Pulmón de Células no Pequeñas , Análisis Costo-Beneficio , Neoplasias Pulmonares , Pemetrexed , Anticuerpos Monoclonales Humanizados/uso terapéutico , Anticuerpos Monoclonales Humanizados/economía , Humanos , Carcinoma de Pulmón de Células no Pequeñas/tratamiento farmacológico , Carcinoma de Pulmón de Células no Pequeñas/patología , Neoplasias Pulmonares/tratamiento farmacológico , Neoplasias Pulmonares/patología , Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Protocolos de Quimioterapia Combinada Antineoplásica/economía , Pemetrexed/uso terapéutico , Pemetrexed/economía , Carboplatino/uso terapéutico , Carboplatino/economía , Carboplatino/administración & dosificación , Inhibidores de Puntos de Control Inmunológico/uso terapéutico , Inhibidores de Puntos de Control Inmunológico/economía , Presupuestos , Modelos Econométricos , FemeninoRESUMEN
BACKGROUND: Durvalumab, used as consolidation immunotherapy, has shown to improve survival in patients with stage III non-small cell lung cancer who respond to chemoradiotherapy, based on the most recent follow-up of PACIFIC. The Chilean healthcare system provides access to certain immunotherapies for this condition. The present study sought to estimate the budget impact of durvalumab versus standard of care in the context of the Chilean healthcare system. RESEARCH DESIGN AND METHODS: A partitioned survival model was adapted to compare two strategies: durvalumab as consolidation therapy and standard of care for treating stage III NSCLC. The number of patients eligible for treatment was estimated using published incidence data and modeled for a 5-year time horizon. Model inputs were based on published literature, and the duration of treatment was estimated using survival curves obtained from PACIFIC. Costs were estimated in Chilean pesos (CLP) and converted to USD dollars using an exchange rate of USD 1 = CLP 827. Scenario analyses were performed to assess different subsequent therapy splits, variations in the target population and dosage of durvalumab. RESULTS: Durvalumab uptake projected total costs ranging from USD 1.27 in Year 1 to 8.5 million in Year 5 from the public perspective. From the private perspective, the budget impact for the first year is USD 1.3 million to USD 3 million for 2028. This difference relies mostly on the lower number of patients treated. Both perspectives anticipated cost savings over the time horizon through reduced monitoring, adverse events, and end-of-life expenses. CONCLUSIONS: This study demonstrates that the inclusion of Durvalumab for NSCLC in Chile represents an investment in the Chilean health system. The incremental costs align with clinical benefits and potential savings in healthcare resource utilization. However, a comprehensive cost-effectiveness analysis is needed to evaluate its economic value thoroughly.
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Anticuerpos Monoclonales , Carcinoma de Pulmón de Células no Pequeñas , Quimioradioterapia , Neoplasias Pulmonares , Carcinoma de Pulmón de Células no Pequeñas/tratamiento farmacológico , Carcinoma de Pulmón de Células no Pequeñas/terapia , Carcinoma de Pulmón de Células no Pequeñas/economía , Carcinoma de Pulmón de Células no Pequeñas/patología , Humanos , Anticuerpos Monoclonales/uso terapéutico , Anticuerpos Monoclonales/economía , Neoplasias Pulmonares/tratamiento farmacológico , Neoplasias Pulmonares/terapia , Neoplasias Pulmonares/economía , Neoplasias Pulmonares/patología , Quimioradioterapia/economía , Quimioradioterapia/métodos , Chile , Estadificación de Neoplasias , Femenino , Masculino , Quimioterapia de Consolidación/economía , Análisis Costo-Beneficio , Presupuestos , Persona de Mediana Edad , Anciano , Atención a la Salud/economíaRESUMEN
INTRODUCTION: The treatment of patients with cardiogenic shock (CS) encompasses several health technologies including Impella pumps and venoarterial extracorporeal membrane oxygenation (VA-ECMO). However, while they are widely used in clinical practice, information on resource use and quality of life (QoL) associated with these devices is scarce. The aim of this study is, therefore, to collect and comparatively assess clinical and socioeconomic data of Impella versus VA-ECMO for the treatment of patients with severe CS, to ultimately conduct both a cost-effectiveness (CEA) and budget impact (BIA) analyses. METHODS AND ANALYSIS: This is a prospective plus retrospective, multicentre study conducted under the scientific coordination of the Center for Research on Health and Social Care Management of SDA Bocconi School of Management and clinical coordination of Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Raffaele Scientific Institute in Milan. The Impella Network stemmed for the purposes of this study and comprises 17 Italian clinical centres from Northern to Southern Regions in Italy. The Italian network qualifies as a subgroup of the international Impella Cardiac Surgery Registry. Patients with CS treated with Impella pumps (CP, 5.0 or 5.5) will be prospectively recruited, and information on clinical outcomes, resource use and QoL collected. Economic data will be retrospectively matched with data from comparable patients treated with VA-ECMO. Both CEA and BIA will be conducted adopting the societal perspective in Italy. This study will contribute to generate new socioeconomic evidence to inform future coverage decisions. ETHICS AND DISSEMINATION: As of May 2024, most of the clinical centres submitted the documentation to their ethical committee (N=13; 76%), six centres received ethical approval and two centres started to enrol patients. Study results will be published in peer-reviewed publications and disseminated through conference presentations.
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Análisis Costo-Beneficio , Oxigenación por Membrana Extracorpórea , Corazón Auxiliar , Choque Cardiogénico , Humanos , Choque Cardiogénico/terapia , Choque Cardiogénico/economía , Oxigenación por Membrana Extracorpórea/economía , Oxigenación por Membrana Extracorpórea/métodos , Corazón Auxiliar/economía , Estudios Prospectivos , Estudios Retrospectivos , Italia , Calidad de Vida , Estudios Multicéntricos como Asunto , Presupuestos , Estudios Observacionales como AsuntoRESUMEN
OBJECTIVES: In many countries, the healthcare sector is dealing with important challenges such as increased demand for healthcare services, capacity problems in hospitals and rising healthcare costs. Therefore, one of the aims of the Dutch government is to move care from in-hospital to out-of-hospital care settings. An example of an innovation where care is moved from a more specialised setting to a less specialised setting is the performance of an antenatal cardiotocography (aCTG) in primary midwife-led care. The aim of this study was to assess the budget impact of implementing aCTG for healthy pregnant women in midwife-led care compared with usual obstetrician-led care in the Netherlands. METHODS: A budget impact analysis was conducted to estimate the actual costs and reimbursement of aCTG performed in midwife-led care and obstetrician-led care (ie, base-case analysis) from the Dutch healthcare perspective. Epidemiological and healthcare utilisation data describing both care pathways were obtained from a prospective cohort, survey and national databases. Different implementation rates of aCTG in midwife-led care were explored. A probabilistic sensitivity analysis was conducted to estimate the uncertainty surrounding the budget impact estimates. RESULTS: Shifting aCTG from obstetrician-led care to midwife-led-care would increase actual costs with 311 763 (97.5% CI 188 574 to 426 072) and 1 247 052 (97.5% CI 754 296 to 1 704 290) for implementation rates of 25% and 100%, respectively, while it would decrease reimbursement with -7 538 335 (97.5% CI -10 302 306 to -4 559 661) and -30 153 342 (97.5% CI -41 209 225 to -18 238 645) for implementation rates of 25% and 100%, respectively. The sensitivity analysis results were consistent with those of the main analysis. CONCLUSIONS: From the Dutch healthcare perspective, we estimated that implementing aCTG in midwife-led care may increase the associated actual costs. At the same time, it might lower the healthcare reimbursement.