Net Budget Impact Research Articles

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

Aims This study assessed the budget impact of resmetirom as a treatment for adults with non-cirrhotic non-alcoholic steatohepatitis (NASH) with moderate-to-advanced liver fibrosis and estimated total costs for a hypothetical private payer in the United States. Materials and methods A three-year budget impact analysis based on an open cohort state transition model was developed for a hypothetical one-million-member private health plan. The comparator was Standard of Care (SOC), defined as routine care for non-cirrhotic NASH patients with moderate-to-advanced liver fibrosis. Each year, the number of resmetirom treatment-eligible patients was estimated through prevalent, incident, and diagnostic rate estimates. Costs included resources incurred by the medical and pharmacy benefits of private payers, including resmetirom drug acquisition costs, diagnosis and monitoring, other medical and other prescription costs stratified by disease progression status (i.e. non-cirrhotic vs. cirrhotic/advanced liver diseases). Resmetirom adverse event management costs were included in sensitivity analysis. Drug costs were estimated based on the average wholesale acquisition cost as of March 2024. Other costs were based on published sources and inflated to 2023 US dollars. Budget impact outcomes were presented in aggregate, net, and on a per-member per-month (PMPM) basis. Results Compared with a scenario without resmetirom, the introduction of resmetirom yielded results ranging from 50 to 238 treated patients, net budget impact of $2.2 to $9.5 million, and PMPM from $0.19 to $0.80 over years one and three. Net costs excluding resmetirom declined over time. In sensitivity analyses, results were most sensitive to diagnostic and epidemiologic inputs. Limitations Market shares are based on internal forecasts, a short time horizon, average treatment effects, and other limitations common to BIMs. Conclusion The adoption of resmetirom on the formulary for the treatment of non-cirrhotic NASH with moderate-to-advanced liver fibrosis resulted in a moderate increase in budget impact with declining costs related to NASH progression.

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

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

Cost-Effectiveness Analysis and Budget Impact Analysis of Enzalutamide for the Treatment of Metastatic Hormone-Sensitive Prostate Cancer

We conducted cost-effectiveness analysis and budget impact analysis for androgen deprivation therapy (ADT) plus enzalutamide (ENZ) on patients with metastatic hormone-sensitive prostate cancer (mHSPC) from the publicly-funded healthcare system perspective. Using a partitioned survival model, lifetime costs, quality-adjusted life years (QALYs) and incremental cost-effectiveness ratios (ICERs) of ADT＋ENZ were estimated against ADT alone, ADT plus abiraterone (ADT＋ABI), and ADT plus apalutamide (ADT＋APA). Total healthcare cost differences with and without ENZ in mHSPC therapy were estimated for the period from 2022 to 2026. Based on cost-effectiveness analysis, the ICER of ADT＋ENZ versus ADT alone was estimated as ￥7.18 million/QALY gained. ADT＋ABI and ADT＋APA were dominated options (extended dominance). Budget impact analysis showed that incorporation of ENZ had a net budget impact of ￥57.19 billion, an 8.4% increase, over these 5 years. This amounted to a budgetary impact of ￥16,000 per patient per month at year 5. However, the number of patients with disease progressed to metastatic castration-resistant prostate cancer (mCRPC) would be reduced from 79,000 (without ENZ) to 65,000 (with ENZ), resulting in a 17% cost reduction within the mCRPC phase. In conclusion, ADT＋ENZ would be a cost-effective option, at the willingness to pay threshold of ￥7.5 million/QALY gained. Introduction of ENZ in the mHSPC treatment would result in a marginal increase in the total budget. However, ENZ is also expected to provide clinical benefits in reducing the number of patients with disease that would otherwise progress to mCRPC during these 5 years, resulting in cost savings in this phase.

Introduction of Glofitamab for the Treatment of Relapsed or Refractory Diffuse Large B-Cell Lymphoma (DLBCL) after ≥2 Lines of Systemic Therapy Results in Cost Savings to the Healthcare System Based on a United States Budget Impact Analysis

Background: Glofitamab is a humanized CD20xCD3 T-cell engaging bispecific immunoglobulin G1 antibody that was granted accelerated approval from the United States (US) Food and Drug Administration for adult patients with relapsed or refractory (R/R) DLBCL, not otherwise specified or large B-cell lymphoma arising from follicular lymphoma, after ≥2 lines of systemic therapy. We conducted a budget impact analysis to assess the effect of introducing glofitamab as a treatment option for R/R DLBCL after ≥2 lines of systemic therapy by estimating total costs to a US health plan, with and without glofitamab. Methods: A 3-year budget impact model was developed for a hypothetical cohort of 1 million patients enrolled in a mixed commercial/Medicare health plan. Comparators were axicabtagene ciloleucel (Axi-cel), lisocabtagene maraleucel (Liso-cel), tisagenlecleucel (Tisa-cel), loncastuximab tesirine (Lonca), polatuzumab vedotin + bendamustine + rituximab (Pola-BR), rituximab + gemcitabine + oxaliplatin (R-GemOx), tafasitamab + lenalidomide, and epcoritamab. Total costs included those of the drug (based on mean treatment duration observed in pivotal trials) and its administration, as well as those arising from managing adverse reactions (including cytokine release syndrome and hematology laboratory abnormalities) and routine care. Drug costs were estimated based on the average wholesale acquisition cost reported in AnalySource ® (2023) or the Average Sales Price in the Centers for Medicare Services Pricing File (2023). All other costs were based on several US published sources. Market shares were based on Genentech internal projections and expert opinions. All costs were inflated to 2023 US dollars, where applicable. Budget impact outcomes over 3 years were presented as total cumulative costs and average per-member per-month (PMPM) costs. In a scenario analysis, we examined the budget impact when maximum treatment duration per US prescribing information (USPI) was applied across treatments. The net budget impact was the difference in costs between the projected scenario with glofitamab and the current scenario without glofitamab. Results: Among 1 million patients,approximately nine patients were projected to be eligible for treatment of R/R DLBCL after ≥2 lines of therapy.The introduction of glofitamab as a treatment option resulted in an estimated cumulative total cost saving of $728,697 over 3 years; the average PMPM net budget impact was −$0.0202 ( Figure A). The introduction of glofitamab reduced costs across all categories ( Figure A). Among the newer therapies including chimeric antigen receptor T-cell therapy and bispecific antipbodies, the total cost per treated patient was lowest for glofitamab over the 3-year period: glofitamab = $226,658; Tisa-cel = $564,113; Axi-cel = $540,002; Liso-cel = $516,272; epcoritamab = $335,293 ( Figure B). In the scenario analysis where the full treatment duration per USPI was modeled, the introduction of glofitamab as a treatment option resulted in greater cost savings over 3 years, versus the current scenario without glofitamab (cumulative: −$921,407 and PMPM: −$0.0256). Conclusions: Over 3 years, the estimated cumulative per-patient cost of glofitamab is projected to be the lowest when compared with per-patient costs of other available T-cell engaging therapies, resulting in cost savings after its formulary adoption for the treatment of R/R DLBCL after ≥2 lines of therapy.

Clinical and Economic Assessment of MyDiaCare, Digital Tools Combined With Diabetes Nurse Educator Support, for Managing Diabetes in South Africa: Observational Multicenter, Retrospective Study Associated With a Budget Impact Model.

In South Africa, diabetes prevalence is expected to reach 5.4 million by 2030. In South Africa, diabetes-related complications severely impact not only patient health and quality of life but also the economy. The Diabetes Nurse Educator (DNE) study assessed the benefit of adding the MyDiaCare program to standard of care for managing patients with type 1 and type 2 diabetes in South Africa. An economic study was also performed to estimate the budget impact of adding MyDiaCare to standard of care for patients with type 2 diabetes older than 19 years treated in the South African private health care sector. The real-world DNE study was designed as an observational, retrospective, multicenter, single-group study. Eligible patients were older than 18 years and had at least 6 months of participation in the MyDiaCare program. The MyDiaCare program combines a patient mobile app and a health care professional platform with face-to-face visits with a DNE. The benefit of MyDiaCare was assessed by the changes in glycated hemoglobin (HbA1c) levels, the proportion of patients achieving clinical and biological targets, adherence to care plans, and satisfaction after 6 months of participating in the MyDiaCare program. A budget impact model was performed using data from the DNE study and another South African cohort of the DISCOVERY study to estimate the economic impact of MyDiaCare. Between November 25, 2019, and June 30, 2020, a total of 117 patients (8 with type 1 diabetes and 109 with type 2 diabetes) were enrolled in 2 centers. After 6 months of MyDiaCare, a clinically relevant decrease in mean HbA1c levels of 0.6% from 7.8% to 7.2% was observed. Furthermore, 54% (43/79) of patients reached or maintained their HbA1c targets at 6 months. Most patients achieved their targets for blood pressure (53/79, 67% for systolic and 70/79, 89% for diastolic blood pressure) and lipid parameters (49/71, 69% for low-density-lipoprotein [LDL] cholesterol, 41/71, 58% for high-density-lipoprotein [HDL] cholesterol, and 59/71, 83% for total cholesterol), but fewer patients achieved their targets for triglycerides (32/70, 46%), waist circumference (12/68, 18%), and body weight (13/76, 17%). The mean overall adherence to the MyDiaCare care plan was 93%. Most patients (87/117, 74%) were satisfied with the MyDiaCare program. The net budget impact per patient with type 2 diabetes, older than 19 years, treated in the private sector using MyDiaCare was estimated to be approximately South African Rands (ZAR) 71,023 (US $4089) during the first year of introducing MyDiaCare. The results of using MyDiaCare program, which combines digital tools for patients and health care professionals with DNE support, suggest that it may be a clinically effective and cost-saving solution for diabetes management in the South African private health care sector.

The health and budget impact of sodium-glucose co-transporter-2 inhibitors (SGLT2is) in The Netherlands

ObjectivesType-2 Diabetes mellitus (T2DM) increases both the patient risk of cardiovascular disease (CVD) and renal outcomes, such as chronic kidney disease (CKD). Recent clinical trials of the glucose-lowering drug-class of sodium-glucose co-transporter-2 inhibitors (SGLT2is) have shown benefits in preventing CVD events and progression of CKD, leading to an update of the Dutch T2DM treatment guideline for patients at risk. The aim of this study is to assess the health and economic impact of the guideline-recommended utilization of SGLT2is in the Netherlands.MethodsThe patient population at risk was determined by multiplying Dutch T2DM prevalence rates with the total numbers of inhabitants of the Netherlands in 2020. Subsequently, two analyses, comparing a treatment setting before and after implementation of the new guideline for SGLT2is, were conducted. Clinical and adverse event rates in both settings as well as direct healthcare costs were sourced from the literature. Total costs were calculated by multiplying disease prevalence, event rates and costs associated to outcomes. One-time disutilities per event were included to estimate the health impact. The potential health and economic impact of implementing the updated guideline was calculated.ResultsUsing a 5-year time horizon, the guideline-suggested utilization of SGLT2is resulted in a health impact equal to 4835 quality adjusted life years gained (0.0031 per patient per year) and €461 million cost-savings. The costs of treatment with SGLT2is were €813 million. Hence the net budget impact was €352 million for the total Dutch T2DM population, which translated to €0,57 per patient per day.ConclusionSGLT2is offer an option to reduce the number of CVD and CKD related events and associated healthcare costs and health losses in the Netherlands. Further research is needed to include the benefits of improved T2DM management options from a broader societal perspective.HighlightsThe glucose-lowering drug-class of sodium-glucose co-transporter-2 inhibitors (SGLT2is) has shown benefits in preventing cardiovascular events and progression of kidney disease in patients with type-2 diabetes leading to a revision of the respective Dutch treatment guideline.The 5-year budget impact of the adoption of SGLT2is in the new treatment guideline was equal to €352 million or €0.57 per patient per day, with a total of 4385 quality adjusted life years gained.The introduction of SGLT2is for Dutch type-2 diabetes patients has the potential to substantially reduce the number of cardiovascular as well as renal disease events and related healthcare costs while also delivering a health benefit.

Budget impact analysis of daratumumab for light-chain amyloidosis in Cyprus

ABSTRACT Objective The aim of this study is to assess the budget impact of daratumumab for light-chain amyloidosis in Cyprus Methods A budget impact model assessed the cost prior and after the introduction of daratumumab for light-chain amyloidosis. All related costs were set from the perspective of Cyprus NHS. Clinical data were extracted from the published trials. One-way sensitivity analysis was conducted. We reported incremental budget impact, per member per month, per year, and per treated member per month Results The introduction of D-VCd led to a net budget impact of €254,264 in the first year, which escalated to €497,007 by fifth year. The PMPY was estimated at €0.2893 in the first year, reaching €0.5246 at fifth year, the PMPM were at €0.0241 at the first year escalating to €0.0437 at the fifth year, and the PTMPM costs were €2,379 at the first year and gauged to €4,435 by fifth year. Our results were sensitive to incidence of the disease, percentage of patients without cardiac involvement and daratumumab cost. Conclusions The introduction of daratumumab for AL amyloidosis, with a 90% annual uptake over 5 years, leads to a substantial budget impact. Managed entry agreement schemes can be considered in order to mitigate the impact.

Cost-utility and budget impact analysis of CPAP therapy compared to no treatment in the management of moderate to severe obstructive sleep apnea in Colombia from a third-party payer perspective

ABSTRACT Objectives To conduct cost-utility and budget impact analysis of providing Continuous Positive Airway Pressure (CPAP) therapy versus no treatment for moderate to severe obstructive sleep apnea (OSA) in Colombia from a third-party payer perspective. Methods We used a Markov model to assess the cost-utility and budget impact analysis of CPAP in patients over 40 years old with moderate to severe OSA. Data on effectiveness and utility values were obtained from published literature. A discount rate of 5% was applied for outcomes and costs. ICER was calculated and compared against the threshold estimated for Colombia, which is 86% of the GDP per capita. Results Over a lifetime horizon, the base case analysis showed the incremental cost per quality-adjusted life-years (QALYs) gained with CPAP therapy was COP$3,503,804 (USD$1,011 in 2020 prices). The budget impact analysis showed that the adoption of CPAP therapy in the target population would lead to a cumulative net budget impact of COP$411,722 million (USD$118,784,412 in, 2020 prices) over five years of time horizon. Conclusions CPAP was cost-effective compared to no-treatment in OSA. According to the budget impact analysis, adopting this technology would require a budget allocation that is partially offset by reduced number of strokes and traffic accident events.

Budget impact analysis of a home-based colorectal cancer screening programme in Malaysia

ObjectivesThe 2020–2022 research project ‘Colorectal Cancer Screening Intervention for Malaysia’ (CRC-SIM) evaluated the implementation of a home-based CRC screening pilot in Segamat District. This budget impact analysis (BIA) assessed the...

PP74 Taking A Lifecycle Approach To Scottish Medicines Consortium Budget Impact Analysis

IntroductionThe Scottish Medicines Consortium (SMC) conducts early health technology assessment of new medicines in Scotland. While budget impact is not a factor in decisions on reimbursement, budget impact information is provided initially through horizon scanning reports for high impact medicines (estimated net budget impact >GBP 500,000 [EUR 585,710] per annum) to aid financial planning and implementation of advice at the local level, and later through budget impact templates from the submitting company issued alongside SMC advice. This research aimed to understand how the information is used and to evaluate the benefits of a lifecycle approach to budget impact analysis.MethodsHealth Board users of the budget impact templates were surveyed to explore the degree of utilization and identify areas for improvement, including the need to cross-validate the horizon scanning estimates with those of the submitting company. Responses were analyzed quantitatively and qualitatively, with comments coded in Nvivo (QSR International) and themes established through thematic analysis.ResultsThe initial responses received (n=17) provided representation from 57 percent of Health Boards (i.e., payers) covering 79 percent of the population. Preliminary results showed that while the budget impact templates were valued, 69 percent of respondents found them ‘somewhat useful’, suggesting scope for improvement. Almost half (48%) of the respondents used the templates for high impact medicines, with only 30 percent using them for all medicines. The majority (76%) of those surveyed thought there would be value in linking budget impact information throughout the SMC process. An emerging theme was that some users found the templates complex and inflexible, and that a simpler, more adaptable tool to aid the planning process would be welcomed. Priorities identified for improving template included adapting them to the local population and adjusting medicine prices to reflect confidential discounts.ConclusionsThis research suggests that budget impact information is valued by Health Boards and that there is strong support for linking budget impact estimates and engaging stakeholders throughout a medicine’s lifecycle. Simplifying the templates, increasing their adaptability, and providing guidance and training in their use will be key steps in improving this important part of SMC process.

Budget Impact Analysis of the Introduction of a Trastuzumab Biosimilar for HER2-Positive Breast Cancer in China.

Biosimilars provide the possibility to reduce the high expenditure on biologic drugs and expand access to effective but less expensive treatments. Biosimilars of trastuzumab showed significant cost savings from the payer's perspective in the USA and Europe. After 2020, with the first approval of a trastuzumab biosimilar in China, it became feasible for biosimilar switching for trastuzumab. However, the economic impact of switching to a trastuzumab biosimilar was not evaluated. A budget impact model was constructed from a payer's perspective of China to demonstrate the economic impact of the introduction of a biosimilar trastuzumab in the treatment of human epidermal growth factor receptor 2-positive breast cancer. This budget impact model was based on disease incidence to estimate the net budget impact using epidemiological data from the literature, financial reports from manufacturers on the market shares of originator trastuzumab (Herceptin®) or the biosimilar, and localized direct costs. The budget impact was estimated for 5 years after the introduction of the first-approved trastuzumab biosimilar in China. Furthermore, two scenarios were simulated in this study to estimate the budget impact of biosimilars within: (1) real-world practice and (2) the policy of volume-based procurement. Analyses of the base-case and scenario results implied that adoption of a trastuzumab biosimilar would lead to an expenditure decrease. The average total cost savings over 5 years was estimated to be US$46,651,348, with a range from $10,306,611 in year 1 to $60,821,822 in year 5. The cost savings could benefit an additional 654-3858 patients with breast cancer. If utilizing costs from real-world practice, the introduction of a trastuzumab biosimilar could help an additional 2237-13,203 patients get access to human epidermal growth factor receptor 2-positive targeted therapy. When volume-based procurement was carried out after year 4, $672,366,180 could be saved annually. This budget impact analysis emphasized the positive effects of adopting a trastuzumab biosimilar in the healthcare system of China. However, cost savings still have a large potential to decrease by regulating pricing and by the procurement policy of biosimilars.

Impact of endoscopic ultrasound-guided gallbladder drainage on reducing costs of reintervention and unplanned readmission: a budget impact analysis.

Background and study aims Endoscopic ultrasound-guided gallbladder drainage (EUS-GBD) is the preferred treatment for patients with acute calculous cholecystitis who are unfit for surgery. The aim of this study was to perform a cost-effective analysis (CEA) comparing EUS-GBD with percutaneous gallbladder drainage (PT-GBD). Patients and methods CEA was performed on patients recruited for our prior randomized controlled trial. A budget impact model was developed to compare the base-case and scenario of EUS-GBD applications. The costs including peri-procedure and intra-procedure, reinterventions, expenses associated with treatment of adverse events (AEs), costs of hospital stay, subsequent clinic follow-up, and unplanned readmission were included. Results PT-GBD had a lower total procedure cost per patient (USD$4,375.00) than EUS-GBD (USD$9,397.44). For EUS-GBD, the cost of cautery-enhanced lumen-apposing stent accounted for the major part of the expense (USD$4,910.26). EUS-GBD resulted in a lower expected cost (USD$108.26 vs USD$1,601.54) for a re-procedure. The expected cost per patient in unplanned readmissions in the EUS-GBD group (USD$450.00) was lower than that in the PT-GBD group (USD$1,717.56). Based on the budget impact analysis, the net budget impact per year of introducing EUS-GBD to replace PT-GBD was higher (USD$16,424.10 vs USD$11,433.08). The net budget impact was most sensitive to the cost of stent and linear echoendoscope used in EUS-GBD. Conclusions The net budget impact per year was higher for introducing EUS-GBD. The cost of the stent accounted for the major cost difference between the two procedures. EUS-GBD saved on the cost in management of AEs, reinterventions, and unplanned readmissions but these did not offset the cost of the stent.

Budget impact analysis of providing hospital inpatient care at home virtually, starting with two specific surgical patient groups

ObjectiveTo determine the budget impact of virtual care.MethodsWe conducted a budget impact analysis of virtual care from the perspective of a large teaching hospital in the Netherlands. Virtual care included...

Ex ante budget impact analysis (BIA) of PD-1 inhibitor toripalimab plus gemcitabine-cisplatin (T+GC) versus a simulated pembrolizumab regimen in first line (1L) treatment of recurrent or metastatic nasopharyngeal cancer (rmNPC).

e18024 Background: Interim results of the JUPITER-02 randomized double-blind phase 3 trial demonstrated a PFS benefit for T+GC (11.7 m) vs placebo+GC (8m) in 1L treatment of rmNPC. The KEYNOTE-028 nonrandomized (single group assignment) phase 1b trial showed the potential benefit of pembrolizumab in previously treated NPC. The trials were not comparable in population and design, precluding a direct comparison. We performed an ex ante BIA comparing T+GC to a simulated pembrolizumab+GC (sP+GC) regimen under an assumed equivalence in efficacy and safety. Methods: An ex ante budget impact model (budget holder perspective; accrual approach) compared a treatment mix “without” T+GC (only sP+GC) to one “with” T+GC in an annual incident US population of 1200 patients (year 1), a 5y time horizon, and a fixed 50/50 market share split. Population adjustments were for discontinuation, death, and progression with corresponding medication adjustments to estimate fully treated patient equivalents. Cost inputs included: drugs (average sales price), drug administration, and grade 3/4 adverse event (AE) management. Three scenarios were modeled: toripalimab priced at 70%, 80% and 90% of pembrolizumab. Results: In the “without” scenario, the 5y costs of sP+GC treatment totaled $2,062,509,042 and the 5y cost of managing AEs $210,382,926, for a total of $2,272,891,967. In the “with” scenario at 50% market share, the corresponding 5y costs for sP+GC declined to $1,031,254,521 for treatment and $105,191,463 in AE costs, for a total of $1,136,445,984. The corresponding costs for T+GC priced at 70%, 80%, 90% of pembrolizumab were $746,834,887, $841,641,432 and $936,447,976 (treatment) and $105,191,463 (AE management), for 5y gross budget impacts of $852,026,350, $946,832,895 and $1,041,639,439. Net savings “with” T+GC were $49,331,430, $32,887,620 and $16,443,810 in 2022 rising to $64,974,264, $43,316,176 and $21,658,088 by 2026. The total 5y net budget impact of adding T+GC to the treatment mix totaled $284,419,634, $189,613,089 and $94,806,545 in savings. Conclusions: Citing the findings from the JUPITER-02 trial of T+GC, regimens including a PD-1 inhibitor, gemcitabine and cisplatin are now a NCCN category 2A recommendation for 1L treatment of rmNPC. This ex ante BIA demonstrates the substantial savings potential of toripalimab (at a discounted cost compared to pembrolizumab) in rmNPC. With forthcoming final JUPITER-02 data showing a PFS benefit of 21.4m, the value of an available, lower-priced and efficacious anti-PD1 agent is highly significant to patients, providers and payers in terms of both clinical efficacy and cost efficiency. Emerging data suggest that toripalimab may be effective in more frequently occurring tumor types and the savings from such expanded access are likely to be multifold.

The Use of Evidence-Informed Deliberative Processes for Health Insurance Benefit Package Revision in Iran.

Iran considers the revision of its health insurance benefit package (HIBP) as a means to achieve universal health coverage (UHC). Yet, its decision-making process has been criticised for being weak in terms of accountability and transparency. This paper reports on the development and implementation of the HIBP revision in Iran in the period 2019-2021, employing evidence-informed deliberative processes (EDPs), a framework for benefit package design with the explicit aim of optimising the legitimacy of decision-making. The High Council for Health Insurance (HCHI) is coordinating the HIBP revision: it planned the six steps of the EDP framework with support from World Health Organization (WHO) and Radboudumc in 2019, and conducted a pilot project on multiple sclerosis (MS) diagnosis and treatment in 2020. Implementation of the MS pilot project concerned the installation of advisory committees (involving some 60 stakeholders in supportive task forces, a technical working group [TWG] and a national advisory committee [NAC]), the selection of decision criteria (relating to quality of care, necessity, and sustainability), the inclusion of services for evaluation (nine in total), and the assessment and appraisal of these services. Implementation of the priority setting process for MS diagnosis and treatment services has likely improved the legitimacy of decision-making by involving stakeholders who engaged in deliberation based on available evidence in a stepwise, transparent process. It is expected to improve the quality of care for MS patients as well as its financial accessibility, at a zero net budget impact. The pilot project has served to help Iran's health system move faster toward UHC for a broader range of essential health services.

Budget impact analysis of trilaciclib for decreasing the incidence of chemotherapy-induced myelosuppression in patients with extensive-stage small cell lung cancer in the United States.

BACKGROUND: Chemotherapy-induced myelosuppression, which commonly manifests as neutropenia, anemia, and/or thrombocytopenia, is a frequent and severe complication of standard treatment regimens for patients with extensive-stage small cell lung cancer (ES-SCLC). Trilaciclib is a first-in-class myeloprotective therapy indicated to decrease the incidence of chemotherapy-induced myelosuppression when administered prior to a platinum-/etoposide-containing regimen or topotecan-containing regimen for ES-SCLC. OBJECTIVE: To estimate the budget impact of administering trilaciclib prior to chemotherapy to manage chemotherapy-induced myelosuppression in adults with ES-SCLC from a US payer perspective. METHODS: A budget impact model was developed to assess the impact of introducing trilaciclib to a hypothetical 1 million-member health insurance plan. The model compared 2 market scenarios: a current scenario of standard treatments for ES-SCLC without trilaciclib, and an alternative scenario of standard treatment plus trilaciclib. Population, clinical, and cost inputs were derived from published literature and trilaciclib clinical trial data. Model outcomes included the number of myelosuppressive adverse events (AEs), costs of treatment, costs of AE management, total cost, and per-member per-month (PMPM) costs. The budget impact of trilaciclib was calculated as the difference in cost (2021 US dollars) between the 2 scenarios over a 1- to 5-year time horizon. Scenario and deterministic sensitivity analyses were conducted to assess uncertainty around key model inputs. RESULTS: An estimated total of 301 patients were eligible for treatment with trilaciclib over a 5-year period. The use of trilaciclib was estimated to reduce the number of myelosuppressive AEs over a 5-year period (events avoided included 108 for neutropenia, 7 for febrile neutropenia, 23 for anemia, and 46 for thrombocytopenia) compared with the scenario without trilaciclib. The adoption of trilaciclib was associated with a cost saving of $801,254 ($0.013 PMPM) over 5 years. The acquisition cost for trilaciclib ($3,704,199) was offset by the reduction in AE management cost ($4,282,748) and reduction in prophylactic granulocyte colony-stimulating factor use ($222,704). The cost savings associated with trilaciclib began in year 1 (total $34,388; $0.003 PMPM) and accrued over time. CONCLUSIONS: The acquisition cost of trilaciclib is projected to be offset by a reduction in the costs of managing AEs related to myelosuppression when added to standard chemotherapy regimens for ES-SCLC. The net budget impact of trilaciclib is estimated to be a cost saving. DISCLOSURES: This research was funded by G1 Therapeutics, Inc., and implemented by ZS Associates, an independent consultancy that collated the model inputs and performed the budget impact analysis. The study sponsor was involved in the study design; collection, analysis, and interpretation of data; writing of the report; and the decision to submit the report for publication. The journal open access fee was funded by G1 Therapeutics, Inc. Moran, Chioda, and Huang are employed by G1 Therapeutics, Inc. Chioda and Huang report stocks and stock options for G1 Therapeutics, Inc. Goyal and Deniz are employed by ZS Associates. Goyal reports consulting fees from G1 Therapeutics, Inc. Abraham reports consulting fees from Coherus, G1 Therapeutics, Inc. (unrelated to this study and manuscript), Mylan/Viatris, and Sandoz and participation on a data safety monitoring board or advisory board for G1 Therapeutics, Inc. MacDonald reports consulting fees from Coherus, G1 Therapeutics, Inc. (unrelated to this study and manuscript), Mylan/Viatris, and Sandoz. Deniz reports no disclosures. A synopsis of the current study was presented in poster format at the Virtual AMCP Annual Meeting, April 12-16, 2021.

Economic implications of FDA platelet bacterial guidance compliance options: Comparison of single-step strategies.

BACKGROUNDBloodborne pathogens pose a major safety risk in transfusion medicine. To mitigate the risk of bacterial contamination in platelet units, FDA issues updated guidance materials on various bacterial risk control strategies (BRCS). This analysis presents results of a budget impact model updated to include 5‐ and 7‐day pathogen reduced (PR) and large volumed delayed sampling (LVDS) BRCS.STUDY DESIGN AND METHODSModel base‐case parameter inputs were based on scientific literature, a survey distributed to 27 US hospitals, and transfusion experts' opinion. The outputs include hospital budget and shelf‐life impacts for 5‐ and 7‐day LVDS, and 5‐ and 7‐day PR units under three different scenarios: (1) 100% LVDS, (2) 100% PR, and (3) mix of 50% LVDS ‐ and 50% PR.RESULTSTotal annual costs from the hospital perspective were highest for 100% LVDS platelets (US$2.325M) and lowest for 100% PR‐7 units (US$2.170M). Net budget impact after offsetting annual costs by outpatient reimbursements was 5.5% lower for 5‐day PR platelets as compared to 5‐day LVDS (US$1.663 vs. US$1.760M). A mix of 7‐day LVDS and 5‐day PR platelets had net annual costs that were 1.3% lower than for 100% 7‐day LVDS, but 1.3% higher than for 100% 5‐day PR. 7‐day PR platelets had the longest shelf life (4.63 days), while 5‐day LVDS had the shortest (2.00 days).DISCUSSIONThe model identifies opportunities to minimize transfusion center costs for 5‐ and 7‐day platelets. Budget impact models such as this are important for understanding the financial implications of evolving FDA guidance and new platelet technologies.

The affordability of adding a direct-acting oral anticoagulant to the national list of essential medicine for patients with non-valvular atrial fibrillation in Thailand: a budget impact analysis

ABSTRACT Background Atrial fibrillation (AF) can lead to a significant health and economic burden to society. This study aimed to assess the net budget impact of direct-acting oral anticoagulants (DOACs) instead of warfarin for stroke prevention in patients with non-valvular AF from the payer’s perspective. Methods A budget model over a 5-year period was used. Dabigatran 150 mg, dabigatran 110 mg, apixaban 5 mg, rivaroxaban 20 mg, edoxaban 60 mg, and edoxaban 30 mg were included. Inputs were retrieved from published literature. Adoption rate of DOACs started at 5% and subsequently had a 5% increase in each year. Net budget impact (NBI) and sensitivity analyses were performed. Results The average NBI over the 5-year horizon for all DOACs ranged from 12.3 M USD to 13.9 M USD. Dabigatran 150 mg had the highest NBI, while edoxaban 30 mg had the lowest NBI. The average NBI/patient/year ranged from 63.03 USD – 70.75 USD. Conclusions Of all DOACs, edoxaban 30 mg, apixaban 5 mg, and edoxaban 60 mg are the top 3 lowest NBI. Together with cost-effectiveness evidence, those DOACs should be considered to be listed on the National List of Essential Medicine in Thailand.

Introducing Rotavirus Vaccination in Nigeria: Economic Evaluation and Implications.

BackgroundAs Nigeria prepares to introduce a rotavirus vaccine, the Gavi board has approved the extension of the transition period for the country until 2028. The current position of the country on Gavi’s funding profile calls for a pragmatic step in planning and implementation so that sustainability at the fully self-financing phase will be feasible.ObjectiveThis study aimed to inform the decisions of the country’s health policymakers on the costs, benefits, and implications of the introduction of rotavirus vaccine.MethodsThis study was an economic evaluation using a simulation-based Markov model. It compared four approaches: ‘no vaccination’ and vaccination with ROTARIX, ROTAVAC, or ROTASIIL. Ten cohorts from the year 2021 to 2030 were used in the analysis. Primary measures were the benefit-cost ratio (BCR) and the incremental cost-effectiveness ratio (ICER). Future costs and outcomes were discounted to 2019 values.ResultsThe adjusted vaccine cost of ROTARIX was the highest, followed by ROTAVAC and ROTASIIL, whereas the immunization delivery cost was in the reverse order. All the vaccines were very cost effective, with ROTARIX being the optimal choice for the 10-year period, having a BCR of 27 and an ICER of $US100 (95% confidence interval [CI] 71–130)/disability-adjusted life-year averted. Adopting ROTARIX was the optimal choice from 2021 to 2027, whereas ROTAVAC was optimal from 2028 to 2030. The net budget impact of the programme was $US76.9 million for the 10-year period. The opportunity cost of a late introduction was about $US8 million per annum from 2021 to 2028.ConclusionsThe rotavirus vaccine ROTARIX should be implemented in Nigeria at the earliest opportunity. A switch to ROTAVAC should be considered from the year 2028. Cost-minimization measures are imperative to ensure the sustainability of the programme after the transition out of Gavi support.Supplementary InformationThe online version contains supplementary material available at 10.1007/s41669-020-00251-6.

Budget impact of tepotinib in the treatment of adult patients with metastatic non-small cell lung cancer harboring METex14 skipping alterations in the United States

Aims To estimate the budget impact of adding tepotinib to United States (US) health plans for treating adult patients with metastatic non-small cell lung cancer (mNSCLC) harboring mesenchymal–epithelial transition exon 14 (METex14) skipping alterations. Methods The base-case analysis was conducted from the perspective of a hypothetical Medicare plan of 1 million members. Scenarios were analysed for other US health plans. Treatments included tepotinib, capmatinib, crizotinib, and standard of care (SoC). Patients eligible for tepotinib were estimated from published epidemiological data and literature, and real-world evidence. Clinical inputs were derived from the phase II VISION trial, US prescribing information, and published literature. Tepotinib uptake and projected testing rates for METex14 skipping alterations were based on market research. Unit costs (2020 US dollars (USD)) and resource utilization associated with drug acquisition and administration, treatment monitoring, disease and adverse event (AE) management, and subsequent treatment were derived primarily from public sources. Results In the base-case, 38–65 patients were eligible for tepotinib each year over the three-year time horizon. The cumulative net budgetary impact of tepotinib was –$692,541 (–2.6%); $26,531,670 in the scenario without tepotinib and $25,839,129 in the scenario with tepotinib. A negligible net budget impact was observed per member per month (PMPM) at $0.2457 and $0.2393, respectively, before and after tepotinib’s introduction. Results were most sensitive to variability in unit costs of capmatinib and tepotinib and their corresponding median treatment durations. Sensitivity and scenario analyses support the conclusion that introducing tepotinib will have minimal budgetary impact for Medicare health plans. Similar results were obtained for other US health plans. Limitations Assumptions and expert opinion were applied to address data gaps in key model inputs. Conclusions The estimated budgetary impact of tepotinib for the treatment of adult patients with mNSCLC harboring METex14 skipping alterations is minimal from the perspective of US health plans.