A budget impact analysis for making treatment decisions based on anti-cyclic citrullinated peptide (anti-CCP) testing in rheumatoid arthritis

Abstract

Aim: Given that rheumatoid arthritis (RA) patients with high anti-citrullinated protein antibodies (ACPA) titer values respond well to abatacept, the aim of this study was to estimate the annual budget impact of anti-cyclic citrullinated peptide (anti-CCP) testing and treatment selection based on anti-CCP test results.

Materials and methods: Budget impact analysis was conducted for patients with moderate-to-severe RA on biologic or Janus kinase inhibitor (JAKi) treatment from a hypothetical US commercial payer perspective. The following market scenarios were compared: (1) 90% of target patients receive anti-CCP testing and the results of anti-CCP testing do not impact the treatment selection; (2) 100% of target patients receive anti-CCP testing and the results of anti-CCP testing have an impact on treatment selection such that an increased proportion of patients with high titer of ACPA receive abatacept. A hypothetical assumption was made that the use of abatacept would be increased by 2% in Scenario 2 versus 1. Scenario analyses were conducted by varying the target population and rebate rates.

Results: In a hypothetical health plan with one million insured adults, 2,181 patients would be on a biologic or JAKi treatment for moderate-to-severe RA. In Scenario 1, the anti-CCP test cost was $186,155 and annual treatment cost was $101,854,295, totaling to $102,040,450. In Scenario 2, the anti-CCP test cost increased by $20,684 and treatment cost increased by $160,467, totaling an overall budget increase of $181,151. This was equivalent to a per member per month (PMPM) increase of $0.015. The budget impact results were consistently negligible across the scenario analyses.

Limitations: The analysis only considered testing and medication costs. Some parameters used in the analysis, such as the rebate rates, are not generalizable and health plan-specific.

Conclusions: Testing RA patients to learn their ACPA status and increasing use of abatacept among high-titer ACPA patients result in a small increase in the total budget (<2 cents PMPM).     

The budget impact of brodalumab for the treatment of moderate-to-severe plaque psoriasis on US commercial health plans

Introduction: Brodalumab is a new biologic approved by the US Food and Drug Administration in 2017 for the treatment of moderate-severe psoriasis. This study evaluated the impact of the introduction of brodalumab on the pharmacy budget on US commercial health plans.

Methods: An Excel-based health economic decision analytic model with a US health plan perspective was developed. The model incorporated published moderate-to-severe psoriasis prevalence data; market shares of common biologic drugs, including adalimumab, ustekinumab, secukinumab, ixekizumab, and etanercept, used for the treatment of moderate–severe psoriasis; 2017-year Wholesale Acquisition Costs for the biologic drugs; drug dispensing fee; patient co-pay; and drug contracting discount. Total annual health plan costs for the biologic drugs were estimated. Scenarios with different proportions of patients treated with brodalumab were compared to a control scenario when no brodalumab was used.

Results: In a hypothetical commercial health plan covering two million members, 7,038 moderate-to-severe psoriasis patients were estimated to be eligible for treatment with brodalumab. Prior to brodalumab approval, the proportions of patients treated by other biologics were estimated at 50.8% for adalimumab, 13.5% for ustekinumab, 14.1% for secukinumab, 4.4% for ixekizumab, and 17.2% for etanercept. With a 20% drug price discount applied to all biologics, the annual health plan costs for brodalumab, adalimumab, ustekinumab, secukinumab, ixekizumab, and etanercept were estimated at $37,224, $49,166, $55,084, $56,061, $64,396, and $57,170, respectively. When no brodalumab is used, the total annual pharmacy budget for the biologics used among these patients was estimated at $414,362,647. Among scenarios where the proportions of brodalumab usage were 3%, 8%, 16%, and 30%, the total annual pharmacy cost was estimated to be reduced by $3,698,129, $9,861,677, $19,723,355, and $36,981,290, respectively.

Conclusion: Based on the economic model, brodalumab has the potential to substantially reduce pharmacy expenditures for the treatment of patients with moderate-to-severe plaque psoriasis in the US.     

Budget impact of netupitant/palonosetron for the prevention of chemotherapy-induced nausea and vomiting

Background: Chemotherapy-induced nausea and vomiting (CINV) are among the most common and debilitating side-effects patients experience during chemotherapy, and are associated with considerable acute care use and healthcare cost. It is estimated that 70–80% of CINV could be prevented through appropriate use of CINV prophylaxis; however, suboptimal CINV compliance and control remains an issue in clinical practice. Netupitant/palonosetron (NEPA) is a fixed combination of serotonin-3 (5-HT₃) and neurokinin-1 (NK₁) receptor antagonists (RAs), respectively, indicated for the prevention of acute and delayed nausea and vomiting associated with highly emetogenic chemotherapy (HEC) and moderately emetogenic chemotherapy (MEC). Phase 3 clinical trials showed a significantly higher complete response rate in both acute and delayed CINV in chemotherapy-naïve patients receiving NEPA compared to patients receiving palonosetron.

Objective: The objective of this study was to estimate the budgetary impact of adding NEPA to a US payer or practice formulary for CINV prophylaxis.

Methods: A model was developed to estimate the impact of adding NEPA to the formulary of a hypothetical US payer with 1.15 million members, including 150,000 (13%) Medicare beneficiaries. The model compared the annual total costs of CINV-related events and CINV prophylaxis in two scenarios: base year (no NEPA) and comparator year (10% and 5% NEPA usage in HEC and MEC patients, respectively). A univariate sensitivity analysis was conducted to explore the effect of variability in model parameters on the budget impact.

Results: A total of 2,021 patients were eligible to receive CINV prophylaxis. With NEPA, CINV prophylaxis costs increased by 0.7% ($3,493,630 vs $3,518,760) while medical costs associated with CINV events decreased by 3.9% ($15,118,639 vs $14,532,442), resulting in a net cost saving of $561,067 (3.0%) for the health plan ($18,612,269 vs $18,051,202), or Background: Chemotherapy-induced nausea and vomiting (CINV) are among the most common and debilitating side-effects patients experience during chemotherapy, and are associated with considerable acute care use and healthcare cost. It is estimated that 70–80% of CINV could be prevented through appropriate use of CINV prophylaxis; however, suboptimal CINV compliance and control remains an issue in clinical practice. Netupitant/palonosetron (NEPA) is a fixed combination of serotonin-3 (5-HT₃) and neurokinin-1 (NK₁) receptor antagonists (RAs), respectively, indicated for the prevention of acute and delayed nausea and vomiting associated with highly emetogenic chemotherapy (HEC) and moderately emetogenic chemotherapy (MEC). Phase 3 clinical trials showed a significantly higher complete response rate in both acute and delayed CINV in chemotherapy-naïve patients receiving NEPA compared to patients receiving palonosetron.

Objective: The objective of this study was to estimate the budgetary impact of adding NEPA to a US payer or practice formulary for CINV prophylaxis.

Methods: A model was developed to estimate the impact of adding NEPA to the formulary of a hypothetical US payer with 1.15 million members, including 150,000 (13%) Medicare beneficiaries. The model compared the annual total costs of CINV-related events and CINV prophylaxis in two scenarios: base year (no NEPA) and comparator year (10% and 5% NEPA usage in HEC and MEC patients, respectively). A univariate sensitivity analysis was conducted to explore the effect of variability in model parameters on the budget impact.

Results: A total of 2,021 patients were eligible to receive CINV prophylaxis. With NEPA, CINV prophylaxis costs increased by 0.7% ($3,493,630 vs $3,518,760) while medical costs associated with CINV events decreased by 3.9% ($15,118,639 vs $14,532,442), resulting in a net cost saving of $561,067 (3.0%) for the health plan ($18,612,269 vs $18,051,202), or $0.04 per member per month. This was equivalent to saving $5,011 per patient moved to NEPA. Among all 5-HT₃ RA?+?NK₁ RA regimens, NEPA was associated with the lowest CINV-related costs, leading to the lowest total cost of care.

Conclusions: Adding NEPA to a payer or practice formulary results in a net decrease in the total budget due to a substantial reduction in CINV event-related resource utilization and medical costs, and an increase in pharmacy costs <1%, saving over $5,000 per patient.     

Budget impact analysis of bioabsorbable drug-eluting sinus implants following endoscopic sinus surgery

Objective: Propel is a bioabsorbable drug-eluting sinus implant inserted following an endoscopic sinus surgery (ESS) for chronic rhinosinusitis (CRS). The objective of this study was to estimate the budget impact of incorporating Propel post-ESS for CRS patients from a self-insured employer or third-party payer perspective.

Methods: An Excel-based budget impact model was developed. Estimates of the prevalence of CRS, rates of ESS, and effectiveness outcomes, along with direct and indirect costs from CRS were obtained from published literature. A total population of 1.5 million members was hypothesized for the analysis. All cost data were adjusted to October 2015 US dollars using the Medical Care Component of the Consumer Price Index. The cost and clinical/economic characteristics of Propel were compared to other treatments commonly used to minimize post-operative complications. The primary outcome was the incremental budget impact reported using per-member-per-month (PMPM) costs. Scenario-based, probabilistic, and one-way sensitivity analyses were performed to gauge the robustness of the results and identify the parameters with the most influence on the results.

Results: For a US self-insured employer or a commercial health plan of 1.5 million members, the incremental PMPM impact of incorporating Propel was estimated to range from ?Objective: Propel is a bioabsorbable drug-eluting sinus implant inserted following an endoscopic sinus surgery (ESS) for chronic rhinosinusitis (CRS). The objective of this study was to estimate the budget impact of incorporating Propel post-ESS for CRS patients from a self-insured employer or third-party payer perspective.

Methods: An Excel-based budget impact model was developed. Estimates of the prevalence of CRS, rates of ESS, and effectiveness outcomes, along with direct and indirect costs from CRS were obtained from published literature. A total population of 1.5 million members was hypothesized for the analysis. All cost data were adjusted to October 2015 US dollars using the Medical Care Component of the Consumer Price Index. The cost and clinical/economic characteristics of Propel were compared to other treatments commonly used to minimize post-operative complications. The primary outcome was the incremental budget impact reported using per-member-per-month (PMPM) costs. Scenario-based, probabilistic, and one-way sensitivity analyses were performed to gauge the robustness of the results and identify the parameters with the most influence on the results.

Results: For a US self-insured employer or a commercial health plan of 1.5 million members, the incremental PMPM impact of incorporating Propel was estimated to range from ?$0.003 to $0.036, respectively, for all members in the health plan. Sensitivity analyses identified the cost of Propel, probability of polyposis recurrence requiring medical intervention, probability of adhesion formation requiring surgical intervention, and the treatment costs for polyposis as the primary parameters influencing the results.

Conclusion: This study has demonstrated the use of Propel following ESS procedures has a negligible impact on the budget of a US self-insured employer or payer. The upfront cost of Propel was offset by savings associated with reduced probability for polyp recurrence, adhesion formation, and their subsequent treatment.     

Budget impact of erlotinib for maintenance therapy in advanced non-small cell lung cancer

Abstract

Objective:

Assess the budgetary impact of adding erlotinib for maintenance therapy (MTx) in advanced non-small cell lung cancer (NSCLC) from a US health plan perspective.

Methods:

A budget impact model was developed to analyze the costs (drug, administration, adverse events) associated with adding erlotinib MTx to a hypothetical 500,000 member US health plan. Treatment durations and dosing were derived from randomized controlled trials, FDA labeling, and National Comprehensive Cancer Network guidelines. Treatment patterns and assumptions were based on market research data, the SEER registry, and published literature. Cost data were obtained from Centers for Medicare and Medicaid Services payment rates and a drug pricing database. Sensitivity analyses were conducted to assess uncertainty.

Results:

Overall health plan expenditures increased by $0.010 per member per month (PMPM). The main driver of additional cost was the erlotinib drug cost (～$66,000) with the administration ($464) and side-effect ($47) costs being relatively modest. One-way sensitivity analyses showed that the results were most sensitive to the proportion of members receiving MTx; however, the PMPM did not exceed $0.013.

Conclusions:

The overall budget impact to a health plan of expanding the use of erlotinib from the 2nd/3rd-line advanced NSCLC setting to include the maintenance setting was relatively small. This was primarily due to the proportion of patients who would receive erlotinib MTx, the low cost of side-effects and minimal cost of drug administration. Additional research may be warranted to estimate the relative clinical and economic impacts of erlotinib MTx versus alternative MTx treatments.     

Budget impact analysis of comprehensive genomic profiling in patients with advanced non-small cell lung cancer

Aims: Broad molecular profiling of patients with advanced non-small cell lung cancer (NSCLC) is strongly advised to optimize genomic matching with available targeted treatment options or investigational agents. Unlike conventional molecular diagnostic testing, or smaller hotspot panels, comprehensive genomic profiling (CGP) identifies genomic alterations across hundreds of clinically relevant cancer genes from a single tissue specimen. The present study sought to estimate the budget impact of increased use of CGP using a 324-gene panel (FoundationOne) vs non-CGP (represented by a mix of conventional molecular diagnostic testing and smaller NGS hotspot panels) and the number needed to test with CGP to gain 1 life year.

Materials and methods: A decision analytic model was developed to assess the budget impact of increased CGP in advanced NSCLC from a US private payer perspective. Model inputs were based on published literature (epidemiology and treatment outcomes), real-world data (testing and rates, medical service costs), list prices for CGP and anti-cancer drugs, and assumptions for clinical trial participation.

Results: Among 2 million covered lives, 532 had advanced NSCLC; 266 underwent molecular diagnostic testing. An increase in CGP among those tested, from 2% to 10%, was associated with $0.02 per member per month budget impact, of which $0.013 was attributable to costs of prolonged drug treatment and survival and $0.005 to testing cost. Approximately 12 patients would need to be tested with CGP to add 1 life year.

Limitations: The model incorporated certain assumptions to account for inputs with a limited evidence profile and simplify the possible post-CGP treatments.

Conclusions: An increase in CGP utilization from 2% to 10% among patients with advanced NSCLC undergoing molecular diagnostic testing was associated with a modest budget impact, most of which was attributable to increased use of more effective treatments and prolonged survival.     

Clinical and economic benefits of professional CGM among people with type 2 diabetes in the United States: analysis of claims and lab data

Background: It is estimated that one in 10 people in the US have a diagnosis of diabetes. Type 2 diabetes accounts for 95% of all cases in the US, with annual costs estimated to be $246 billion per year. This study investigated the impact of a glucose-measuring intervention to the burden of type 2 diabetes.

Objective: This analysis seeks to understand how professional continuous glucose monitoring (professional CGM) impacts clinical and economic outcomes when compared to patients who are not prescribed professional CGM.

Methods: This study utilized a large healthcare claims and lab dataset from the US, and identified a cohort of patients who were prescribed professional CGM as identified by CPT codes 95250 and 95251. It calculated economic and clinical outcomes 1 year before and 1 year after the use of professional CGM, using a generalized linear model.

Results: Patients who utilized professional CGM saw an improvement in hemoglobin A1C. The “difference-in-difference” calculation for A1C was shown to be –0.44%. There was no statistically significant difference in growth of total annual costs for people who used professional CGM compared to those who did not ($1,270, p?=?.08). Patients using professional CGM more than once per year had a –$3,376 difference in the growth of total costs (p?=?.05). Patients who used professional CGM while changing their diabetes treatment regimen also had a difference of –$3,327 in growth of total costs (p?=?.0023).

Conclusion: Significant clinical benefits were observed for patients who used professional CGM. Economic benefits were observed for patients who utilized professional CGM more than once within a 1-year period or who used it during a change of diabetes therapy. This suggests that professional CGM may help decrease rising trends in healthcare costs for people with type 2 diabetes, while also improving clinical outcomes.     

Cost-impact of cardiac magnetic resonance imaging with Fast-SENC compared to SPECT in the diagnosis of coronary artery disease in the U.S

Aims: The purpose of this study is to assess the economic cost differences and the associated treatment resource changes between the developing coronary artery disease (CAD) diagnostic tool fast strain-encoded cardiac imaging (Fast-SENC) and the current commonly used stress test single-photon emission computed tomography (SPECT).

Materials and methods: A “payer perspective” model was created first, consisting of long-term and short-term components that used a hypothetical cohort of patients of average age (60.8?years) presenting with chest pain and suspected CAD to assess cost-impact. A cost impact model was then built that assessed likely savings from a “hospital perspective” from substituting Fast-SENC for a portion of SPECTs assuming an average number of annual SPECT tests performed in US hospitals.

Results: In the payer model, using Fast-SENC followed by coronary angiography (CA) and percutaneous coronary intervention (PCI) treatment when necessary is less costly than the SPECT method when considering both direct and indirect costs of testing. Expected costs of the Fast-SENC were between $2,510 and $2,632 per correct diagnosis, while expected costs for the SPECT were between $3,157 and $4,078. Fast-SENC reduced false positives by 50% and false negatives by 86%, generating additional cost savings. The hospital model showed total costs per CAD patient visit of $825 for SPECT and $376 for Fast-SENC.

Limitations: Limitations of this study are that clinical data are sourced from other published clinical trials on how CAD diagnostic strategies impact clinical outcome, and that necessary assumptions were made which impact health outcomes.

Conclusion: The lower cost, higher sensitivity and specificity rates, and faster, less burdensome process for detecting CAD patients make Fast-SENC a more capable and economically beneficial stress test than SPECT. The payer model and hospital model demonstrate an alignment between payer and provider economics as Fast-SENC provides monetary savings for patients and resource benefits for hospitals.     

Burden of venous leg ulcers in the United States

Objective:

To estimate the annual incremental per-patient and overall payer burden (2012USD) of venous leg ulcers (VLU) in the US.

Methods:

Beneficiaries with and without VLU were identified using two de-identified insurance claims databases: aged 65+ from a 5% random sample of Medicare beneficiaries (2007–2010: n?～?2.3 million); and aged 18–64 from a privately-insured population (2007–2011: n?～?8.4 million). The index date was selected as the date of a VLU claim with no other VLU diagnoses in the preceding 12 months for the VLU cohort and as the date of a random medical claim for the non-VLU patients. These groups were matched using propensity scores to account for differences in demographics, comorbidities, resource utilization, and costs in the 12 month pre-index period. Medical resource use and costs incurred during the 12 month follow-up period were calculated for both payers. Drug costs and indirect work-loss due to disability and medically-related absenteeism were estimated for the privately-insured sample only. Annual VLU incidence rates were also estimated for both payers.

Results:

Data for 58,672 matched VLU/non-VLU pairs of Medicare and 22,476 matched pairs of privately-insured patients were analyzed. Relative to matched non-VLU patients, VLU patients used more medical resources and incurred annual incremental medical costs of $6391 in Medicare ($18,986 vs $12,595), and $7030 ($13,653 vs $6623) in private insurance ($7086 including drug costs). Compared with non-VLU patients, privately-insured VLU patients had more days missed from work (14.0 vs 10.0), resulting in 29% higher work-loss costs (comparisons significant at p?Limitations:

Findings did not account for out-of-pocket payments or other indirect costs (e.g., lost productivity), and relied on accuracy of diagnosis and procedure codes contained in claims data.

Conclusion:

These findings suggest an annual US payer burden of $14.9 billion.     

Lifetime cost of everolimus vs axitinib in patients with advanced renal cell carcinoma who failed prior sunitinib therapy in the US

Abstract

Objective:

Everolimus and axitinib are approved in the US to treat patients with advanced renal cell carcinoma (RCC) after failure on sunitinib or sorafenib, and one prior systemic therapy (e.g., sunitinib), respectively. Two indirect comparisons performed to evaluate progression-free survival in patients treated with everolimus vs axitinib suggested similar efficacy between the two treatments. Therefore, this analysis compares the lifetime costs of these two therapies among sunitinib-refractory advanced RCC patients from a US payer perspective.

Research design and methods:

A Markov model was developed to simulate a cohort of sunitinib-refractory advanced RCC patients and estimate the cost of treating patients with everolimus vs axitinib. The following health states were included: stable disease without adverse events (AEs), stable disease with AEs, disease progression (PD), and death. The model included the following resources: active treatments, post-progression treatments, adverse events, physician and nurse visits, scans and tests, and palliative care. Resource utilization inputs were derived from a US claims database analysis. Additionally, a 3% annual discount rate was applied to costs, and the robustness of the model results was tested by conducting sensitivity analyses, including those on dosing scheme and post-progression treatment costs.

Results:

Base case results demonstrated that patients treated with everolimus cost an average of $12,985 (11%) less over their lifetimes than patients treated with axitinib. The primary difference in costs was related to active treatment, which was largely driven by axitinib’s higher dose intensity. Results remained consistent across sensitivity analyses for AE and PD treatment costs, as well as dose intensity and discount rates.

Conclusion:

The results suggest that everolimus likely leads to lower lifetime costs than axitinib for sunitinib-refractory advanced RCC patients in the US.     

Budget impact of everolimus for the treatment of progressive,well-differentiated,non-functional neuroendocrine tumors of gastrointestinal or lung origin that are advanced or metastatic

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.     

Insulin degludec versus insulin glargine U100 for patients with type 1 or type 2 diabetes in the US: a budget impact analysis with rebate tables

Background and aims: Drug rebates are almost universally negotiated privately between the manufacturer and the payer in the US. The aim of the present study was to illustrate the use of a “rebate table” to improve the transparency and utility of published budget impact analyses in the US by modeling ranges of hypothetical rebates for two comparators. Worked examples were conducted to illustrate the budgetary implications of using insulin degludec (IDeg) relative to insulin glargine (IGlar) U100 in patients with type 1 or 2 diabetes.

Methods: A short-term (1-year) budget impact model was developed to evaluate the costs of switching to IDeg from IGlar in patients with type 1 or 2 diabetes on basal-bolus and basal-only insulin, respectively. The analysis used insulin dose and hypoglycemia data from recent randomized trials, data on the prevalence of diabetes, and estimates of the proportion of patients using each insulin regimen. The model was configured to run multiple rebate scenarios to generate a rebate table in a hypothetical 1 million member commercial plan.

Results: Relative to IGlar, IDeg resulted in reductions in non-severe and severe hypoglycemia incidence and costs both in patients with type 1 and patients with type 2 diabetes. Insulin acquisition costs were higher, and respective rebates of 7.3% and 10.6% were required for IDeg to break-even with IGlar at the full list price. Incremental per member per month IDeg costs without a rebate were USD 0.04 in type 1 diabetes and USD 0.80 in type 2 diabetes.

Conclusions: Using IDeg instead of IGlar at list price could result in a modest increase in costs when considering insulin and hypoglycemia costs alone, but modest incremental rebates with IDeg would result in cost neutrality relative to IGlar. In addition, IDeg would result in reduced incidence of severe and non-severe hypoglycemia.     

Budget impact analysis for dapagliflozin in type 2 diabetes in Egypt

Abstract

Introduction: Type 2 diabetes mellitus (T2DM) is a major health problem in Egypt with a high impact on morbidity, mortality, and healthcare resources. This study evaluated the budget impact and the long-term consequences of dapagliflozin versus other conventional medications, as monotherapy, from both the societal and health insurance perspectives in Egypt.

Methods: A static budget impact model was developed to estimate the financial consequences of adopting dapagliflozin on the healthcare payer budget. We measured the direct medical costs of dapagliflozin (new scenario) as monotherapy, compared to metformin, insulin, sulphonylurea, dipeptidyl peptidase-4 (DPP-4) inhibitors, thiazolidinedione, and repaglinide (old scenarios) over a time horizon of 3 years. Myocardial infarction (MI), ischemic stroke, hospitalization for heart failure (HHF), and initiation of renal replacement therapy (RRT) rates were captured from DECLARE TIMI 58 trial. One-way sensitivity analyses were conducted.

Results: The budget impact model estimated 2,053,908 patients eligible for treatment with dapagliflozin from a societal perspective and 1,207,698 patients from the health insurance (HI) perspective. The new scenario allows for an initial savings of EGP121 million in the first year, which increased to EGP243 and EGP365 million in the second and third years, respectively. The total cumulative savings from a societal perspective were estimated at EGP731 million. Dapagliflozin allows for savings of EGP71, EGP143, and EGP215 million in the first, second and third years respectively, from the HI perspective, with total cumulative savings of EGP430 million over the 3 years.

Conclusion: Treating T2DM patients using dapagliflozin instead of conventional medications, maximizes patients’ benefits and decreases total costs due to drug cost offsets from fewer cardiovascular and renal events. The adoption of dapagliflozin is a budget-saving treatment option, resulting in substantial population-level health gains due to reduced event rate and cost savings from the perspective of the national healthcare system.     

Budget impact analysis of adjunctive therapy with lacosamide for partial-onset epileptic seizures in Belgium

Summary

Objective:

This study aims to compute the budget impact of lacosamide, a new adjunctive therapy for partial-onset seizures in epilepsy patients from 16 years of age who are uncontrolled and having previously used at least three anti-epileptic drugs from a Belgian healthcare payer perspective.

Methods:

The budget impact analysis compared the ‘world with lacosamide’ to the ‘world without lacosamide’ and calculated how a change in the mix of anti-epileptic drugs used to treat uncontrolled epilepsy would impact drug spending from 2008 to 2013. Data on the number of patients and on the market shares of anti-epileptic drugs were taken from Belgian sources and from the literature. Unit costs of anti-epileptic drugs originated from Belgian sources. The budget impact was calculated from two scenarios about the market uptake of lacosamide.

Results:

The Belgian target population is expected to increase from 5333 patients in 2008 to 5522 patients in 2013. Assuming that the market share of lacosamide increases linearly over time and is taken evenly from all other anti-epileptic drugs (AEDs), the budget impact of adopting adjunctive therapy with lacosamide increases from €5249 (0.1% of reference drug budget) in 2008 to €242,700 (4.7% of reference drug budget) in 2013. Assuming that 10% of patients use standard AED therapy plus lacosamide, the budget impact of adopting adjunctive therapy with lacosamide is around €800,000–900,000 per year (or 16.7% of the reference drug budget).

Conclusions:

Adjunctive therapy with lacosamide would raise drug spending for this patient population by as much as 16.7% per year. However, this budget impact analysis did not consider the fact that lacosamide reduces costs of seizure management and withdrawal. The literature suggests that, if savings in other healthcare costs are taken into account, adjunctive therapy with lacosamide may be cost saving.     

Implications of Real-world Adherence on Cost-effectiveness Analysis in Multiple Sclerosis

Abstract

Objectives:

Adherence to medication is essential for optimal outcomes, especially for chronic diseases such as multiple sclerosis (MS). Studies in MS indicate that lower adherence is associated with an increased risk of relapse, hospitalization or emergency room (ER) visits, and higher medical costs. A previous investigation assessed the cost per relapse avoided for patients with MS receiving first-line disease modifying therapies (DMTs); however, the model assumed 100% adherence.

Methods:

Because real-world utilization patterns influence the actual effectiveness of medications, this analysis assessed the impact of real-world adherence from a US commercial payer perspective, using updated costs.

Results:

As was seen in the original study, in this revised model, fingolimod was associated with the lowest cost per relapse avoided ($90,566), followed by SC IFN β-1b (Extavia: $127,024), SC IFN β-1b (Betaseron: $137,492), SC IFN β-1a ($144,016), glatiramer acetate ($160,314), and IM IFN β-1a ($312,629). The model inputs that had the greatest impact on the results were adherence-adjusted relative relapse rate reduction (RRR) of fingolimod, the wholesale acquisition costs of fingolimod, and the average number of relapses in untreated patients with MS.

Limitations:

The estimates of DMT adherence are from a single claims database study of a large national pharmacy benefit manager that only measured adherence, not actual relapses, and the model does not incorporate manufacturer discounts and rebates, which are not publicly available.

Conclusion:

These results suggest that economic analyses of MS therapies should incorporate real-world adherence rates where available, rather than relying exclusively on trial-based efficacy estimates when considering the economic value of treatment alternatives, and that highly efficacious therapies with low adherence may yield real-world efficacy that is substantially lower than that observed in closely monitored clinical trials.     

Budget impact of pasireotide LAR for the treatment of acromegaly,a rare endocrine disorder

Background:

Acromegaly is a rare disorder characterized by the over-production of growth hormone (GH). Patients often experience a range of chronic comorbidities including hypertension, cardiac dysfunction, diabetes, osteoarthropathy, and obstructive sleep apnea. Untreated or inadequately controlled patients incur substantial healthcare costs, while normalization of GH levels may reduce morbidity and mortality rates to be comparable to the general population.

Objective:

To assess the 3-year budget impact of pasireotide LAR on a US managed care health plan following pasireotide LAR availability.

Methods:

Two separate economic models were developed: one from the perspective of an entire health plan and another from the perspective of a pharmacy budget. The total budget impact model includes costs of drug therapies and other costs for treatment, monitoring, management of adverse events, and comorbidities. The pharmacy cost calculator only considers drug costs.

Results:

The total estimated budget impact associated with the introduction of pasireotide LAR is 0.31 cents ($0.0031) per member per month (PMPM) in the first year, 0.78 cents ($0.0078) in the second year, and 1.42 cents ($0.0142) in the third year following FDA approval. Costs were similar or lower from a pharmacy budget impact perspective. For each patient achieving disease control, cost savings from reduced comorbidities amounted to $10,240 per year.

Limitations:

Published data on comorbidities for acromegaly are limited. In the absence of data on acromegaly-related costs for some comorbidities, comorbidity costs for the general population were used (may be under-estimates).

Conclusions:

The budget impact of pasireotide LAR is expected to be modest, with an expected increase of 1.42 cents PMPM on the total health plan budget in the third year after FDA approval. The efficacy of pasireotide LAR in acromegaly, as demonstrated in head-to-head trials compared with currently available treatment options, is expected to be associated with a reduction of the prevalence of comorbidities.