Cost effectiveness of rosuvastatin in patients at risk of cardiovascular disease based on findings from the JUPITER trial

Abstract

Objective:

This study assessed the long-term cost effectiveness of rosuvastatin therapy compared with placebo in reducing the incidence of major cardiovascular (CVD) events and mortality.

Methods:

A probabilistic Monte Carlo simulation model estimated long-term cost effectiveness of rosuvastatin therapy (20?mg daily) for the prevention of CVD mortality and morbidity. The model included three stages: (1) CVD prevention simulating the 4 years of the JUPITER trial, (2) initial CVD prevention beyond the trial, and (3) subsequent CVD event prevention. A US payer perspective was assessed reflecting direct medical costs, and up to a lifetime horizon. Sensitivity analyses tested the robustness of the model estimates.

Results:

For a hypothetical cohort of 100,000 patients at moderate and high risk of CVD events based on Framingham risk of ≥10%, estimated quality-adjusted life-years (QALYs) gained with rosuvastatin therapy compared with placebo was 33,480 over a lifetime horizon, and 25,380 and 9916 over 20-year and 10-year horizons, respectively. Approximately 12,073 events were avoided over the lifetime; 6,146 non-fatal MIs, 2905 non-fatal strokes, and 4030 CVD deaths avoided. Estimated incremental cost-effectiveness ratio (ICER) for cost per QALY was $7062 (lifetime), $10,743 (20-year horizon), and $44,466 (10-year horizon). For a hypothetical cohort similar to the overall JUPITER population, the cost per QALY ICER was $11,025 for the lifetime and $60,112 for a 10-year horizon.

Limitations:

The cost-effectiveness comparison of rosuvastatin 20?mg was against no active treatment (as opposed to an alternative statin) due to lack of comparative cardiovascular morbidity and mortality risk reduction data for other statins in a population similar to the JUPITER trial population. The analysis was conducted from the payer perspective and lack of inclusion of indirect costs limit interpretability of results from a societal perspective.

Conclusions:

Treatment with rosuvastatin 20?mg daily, is a cost-effective treatment alternative to no treatment in patients at a higher risk (Framingham risk ≥10%) of CVD.     

Budget impact model of tobramycin inhalation solution for treatment of Pseudomonas aeruginosa in cystic fibrosis patients

Abstract

Background:

Pseudomonas aeruginosa (PA) is the most common airway pathogen in cystic fibrosis (CF) patients. The objective of this analysis was to determine the costs of managing PA infection in CF patients with a chronic regimen of tobramycin inhalation solution (TIS).

Methods:

A budget impact model of CF patients was developed to evaluate the costs of TIS from a US managed-care organization (MCO) perspective. The Microsoft Excel model compared TIS treatment plus standard care with standard care alone over a 4-year time horizon and included the cost of drugs, medical care, and annual probabilities of hospitalization and IV anti-pseudomonal (anti-PA) antibiotics administration.

Results:

For an MCO with 5,000,000 members, 389 members 6 years of age or older were estimated to have CF, and 218 (56%) had PA infection. Assuming that use of TIS increased from 20% to 25%, the 1-year budget increased $231,251 or from $0.049 to $0.053 per member per month (PMPM). The net drug budget increase was $243,919, while medical costs associated with exacerbation management decreased $12,669 over the first year. Increasing utilization of TIS, from 20% to 40% over 4 years resulted in an incremental overall budget increase of $925,002, a 3% decrease in hospitalizations, and a 4% decrease in administrations of IV anti-PA antibiotics. These reductions translated to a medical care cost saving of $50,676 over 4 years. Limitations of this study include that the clinical data for the model are from clinical trials conducted in 1996 and the estimation of TIS use for CF patients with chronic PA infections can be impacted by TIS adherence.

Conclusion:

Model results suggest that increasing the use of TIS decreases medical care costs due to decreased hospital admissions and the use of IV anti-PA antibiotics at the expense of higher drug costs.     

Comparing the effectiveness of rosuvastatin and atorvastatin in preventing cardiovascular outcomes: estimates using the Archimedes model

Abstract

Objective:

This study used simulation to compare the effectiveness of rosuvastatin 20?mg vs atorvastatin 40?mg, and rosuvastatin 40?mg vs atorvastatin 80?mg in preventing MACE in a range of patient populations with varying baseline cardiovascular risk.

Research design and methods:

The Archimedes Model was used to simulate head-to-head clinical trials in nine patient populations: Framingham Risk Score (FRS)?≥?5%, 5–10%, 10–20%,?>?20%, EURO-SCORE?≥?5% and >10%, diagnosed diabetes, secondary prevention (history of myocardial infarction or stroke, CVD), and acute coronary syndrome (ACS). Simulated patients, aged 45–70 at trial start, were based on the NHANES 1999–2006. Treatments were modeled using results from the STELLAR, JUPITER, CARDS, ASCOT-LLA, and TNT trials. Treatment models were confirmed using trial validations.

Results:

Comparing rosuvastatin 20?mg vs atorvastatin 40?mg, the 5-year numbers needed to treat to prevent one MACE event (NNT) were 525, 70, and 55 for the FRS?≥?5%, CVD, and ACS groups, respectively. Comparing rosuvastatin 40?mg vs atorvastatin 80?mg the corresponding NNT values were 468, 63, and 51. The 20-year relative risks of MACE in the FRS?≥?5% population were 0.907 (0.901–0.913) for rosuvastatin 20?mg vs atorvastatin 40?mg and 0.892 (0.884–0.901) for rosuvastatin 40?mg vs atorvastatin 80?mg. The relative risks were similar for the remaining populations.

Conclusions:

This study found that rosuvastatin 20?mg and 40?mg lowers the risk of MACE more than atorvastatin 40?mg and atorvastatin 80?mg. While simulation models cannot replace real-world clinical trials, this study bridges gaps in the evidence, and identifies high risk cohorts that would likely see additional benefit from treatment with rosuvastatin rather than atorvastatin.     

Budget impact analysis of niraparib and olaparib for maintenance treatment of platinum-sensitive,recurrent ovarian cancer in the US

Aims: This study aimed to evaluate the budget impact of niraparib and olaparib in patients with platinum-sensitive, recurrent ovarian cancer from a US third party payer perspective.

Materials and methods: A budget impact model was constructed to assess the additional per member per month (PMPM) costs associated with the introduction of niraparib and olaparib, two poly ADP-ribose polymerase ribose polymerase (PARP) inhibitors recently approved to be used in platinum-sensitive, recurrent ovarian cancer patients with and without a gBRCA mutation. The model assessed both pharmacy costs and medical costs. Pharmacy costs included adjusted drug costs, coinsurance, and dispensing fees. Medical costs included costs associated with disease monitoring and management of adverse events from the treatment. Epidemiological data from the literature were used to estimate the target population size. The analysis used 1-year time frame, and patients were assumed on treatment until disease progression or death. All costs were computed in 2017 USD. One-way sensitivity analyses were conducted to evaluate the model robustness.

Results: In a hypothetical plan of 1,000,000 members, 206 patients were estimated to be potential candidates for niraparib or olaparib maintenance treatment after applying all epidemiological parameters. At listed 30-day supply WAC prices of $14,750 for niraparib and $13,482 for olaparib, budget impacts of these two drugs were $0.169 PMPM and $0.156 PMPM, respectively, most of which were contributed by pharmacy costs. Sensitivity analyses suggested that assumptions around market share, platinum-sensitive rate after first treatment, and WAC prices affected results the most.

Limitations: In this model, it was assumed that adopting niraparib and olaparib would not affect utilization of existing medications. Also, the estimated clinical parameters from clinical trials could differ from real-world data.     

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 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.     

Budget impact model of a 5-grass sublingual immunotherapy tablet for the treatment of grass pollen-induced allergic rhinitis

Abstract

Objective:

Allergic rhinitis (AR) is a chronic disease with a substantial clinical and economic burden. This study estimated the potential budget impact (BI) associated with market entry of Sweet Vernal, Orchard, Perennial Rye, Timothy, and Kentucky Blue Grass Mixed Pollens Allergen Extract Tablet for Sublingual Use (‘5-grass SLIT tablet’) for patients aged 10–65 with grass pollen-induced AR.     

Budget impact analysis of darbepoetin alfa every 3 weeks versus epoetin alfa every week for the treatment of chemotherapy-induced anaemia from a US payer's perspective

Abstract

Objective: This analysis was conducted to compare the direct medical costs of treatment with darbepoetin alfa every 3 weeks (Q3W) and epoetin alfa every week (QW) in patients with chemotherapy-induced anaemia (CIA) from the payer's perspective.

Methods: An analysis was conducted from a US health plan perspective to compare the annual budget impact for CIA with darbepoetin alfa Q3W and epoetin alfa QW over a 16-week treatment period. Dosing regimens were obtained from registration clinical trials.

Results: Mean doses, including dose adjustments, were 375.6 μg Q3W for darbepoetin alfa and 43,187 U QW for epoetin alfa. Costs of medical resources included drug acquisition and administration costs. The base case analysis resulted in a per-patient budget impact of $8,544 and $8,667 for darbepoetin alfa and epoetin alfa, respectively. Per member per month cost was $0.90 for darbepoetin alfa and $0.91 for epoetin alfa, based on an estimate of 2,735 CIA patients in a health plan population of 2.17 million. The analysis was most sensitive to drug dose, treatment period and drug price.

Conclusions: Results suggest that per-patient direct medical costs of CIA treatment, when initiated at labelled starting doses, are comparable for darbepoetin alfa Q3W and epoetin alfa QW.     

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 analysis of demineralized bone matrix in combination with autograft in lumbar spinal fusion procedures for the treatment of lumbar degenerative disc disease in Spain

Objective: To estimate the budget impact (BI) of introducing local autograft (LA) combined with demineralized bone matrix (LA?+?DBM) in lumbar spinal fusion (LSF) procedures to treat lumbar degenerative disc disease (LDDD) in Spain.

Methods: A decision tree model was developed to evaluate the 4-year BI associated with introducing LA?+?DBM putty to replace currently available grafting methods, including iliac crest bone graft (ICBG), LA alone, and LA combined with beta-tricalcium phosphate (LA?+?ceramics), with 30%, 40%, and 30% market shares, respectively. The analysis was conducted for a hypothetical cohort of 100 patients with LDDD receiving LSF, assuming LA?+?DBM would replace 100% of the standard of care mix. The fusion rates extracted from the literature were validated by an expert panel. Costs (€2017) were obtained from different Spanish sources. Budget impact and incremental cost per successful fusion were calculated from the perspective of the Spanish National Health System (NHS).

Results: Over 4 years, replacing currently available options with LA?+?DBM for 100 patients resulted in an additional cost of €12,330 (€123/patient), and an additional 14 successful fusions, implying a cost of €881 per additional successful fusion. When costs of productivity loss were included, the introduction of LA?+?DBM resulted in cost savings of €70,294 (€703/patient).

Limitations: The lack of high-quality, homogeneous, head-to-head research studying the efficacy of grafting procedures available to patients undergoing LSF, in addition to a lack of long-term follow-up in existing studies. Therefore, the number of fusions occurring within the model’s time horizon may be underestimated.

Conclusions: Acquisition costs of DBM were partially offset by costs of failed fusions, adverse events and reoperation when switching 100 hypothetical LDDD patients undergoing LSF procedures from standard of care grafting methods to LA?+?DBM from the perspective of the Spanish NHS. DBM cost was entirely offset when costs of lost productivity were considered.     

Cost-effectiveness of prasugrel in a US managed care population

Abstract

Objective:

Decision-makers in the US may be interested in the applicability to their populations of cost-effectiveness results generated from clinical trial populations.

Methods:

An economic model estimating the cost-effectiveness of prasugrel plus aspirin relative to clopidogrel plus aspirin for patients with acute coronary syndromes (ACS) undergoing percutaneous coronary intervention (PCI) was developed from a managed care organization (MCO) perspective. The model estimated 15-month cardiovascular events or bleeding-related outcomes, life expectancy, and costs for patients who received thienopyridine treatment during and after a PCI following a diagnosis of ACS. Post-ACS event rates for patients treated with clopidogrel were from an MCO. The relative risks of these events with prasugrel compared with clopidogrel were from a head-to-head clinical trial.

Results:

The results of the base-case analysis indicated that, in an MCO population, use of prasugrel-based therapy rather than clopidogrel-based therapy at current prices resulted in cost-savings and fewer clinical events over the 15 months after an ACS diagnosis followed by PCI. At possible lower prices for generic clopidogrel-based therapy, the cost-effectiveness ratio for prasugrel-based therapy compared with clopidogrel-based therapy was between $6643 and $13,906 per life-year gained. The results were most sensitive to the relative costs of the two treatments and the cost for hospital stays.

Limitations:

Limitations of the study included lack of follow-up of patients disenrolling from the MCO before the end of the 15-month observation period, the assumption of equal relative risks of events in an MCO as in the clinical trial, and the lack of information on the ratio of cost to charges in the MCO database.

Conclusions:

Use of prasugrel-based therapy compared with clopidogrel-based therapy in ACS patients having a PCI resulted in cost-savings at current prices and favorable cost-effective ratios at likely generic prices for clopidogrel-based therapy because of offsetting savings in the costs of rehospitalization.     

Budget impact analysis of everolimus for the treatment of hormone receptor positive,human epidermal growth factor receptor-2 negative (HER2-) advanced breast cancer in the United States

Abstract

Objective:

To estimate the budget impact of everolimus as the first and second treatment option after letrozole or anastrozole (L/A) failure for post-menopausal women with hormone receptor positive (HR+), human epidermal growth factor receptor-2 negative (HER2-) advanced breast cancer (ABC).     

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.     

A budget impact analysis of budesonide/formoterol in patients with mild asthma in Egypt

Abstract

Background: The aim of this study is to estimate the budget impact of budesonide/formoterol fixed dose combination (FDC) vs salbutamol, both used as needed, in mild asthma patients, from the perspective of the Health Insurance Organization (HIO).

Methods: A static budget impact model was developed to assess the impact of budesonide/formoterol FDC entry on HIO budget over a 3-year period in Egyptian settings. Direct medical costs, including the costs of asthma medications, exacerbations, and management of side-effects, were obtained from HIO cost data. Population data were obtained from the World Bank and supplemented with local studies, and the rates of exacerbations, adverse effects, and number of sick leave days were elicited from the SYGMA 1 trial. Scenario analyses from a societal perspective and deterministic sensitivity analyses were conducted.

Results: The total costs (drug and non-drug costs) for managing mild asthma patients from the HIO perspective were estimated to be EGP8.563 billion before budesonide/formoterol entry compared to EGP5.525 billion post-entry, leading to a total budget savings of EGP3.038 billion after 3?years. This total budget saving included an increase in drug costs (EGP104 million) and a decrease in non-drug costs (EGP3.143 billion). Drug costs were higher in the budesonide/formoterol group than in the salbutamol group, but this cost was offset by reductions in non-drug costs, resulting in a reduction in the total costs of healthcare resources. At the societal level, the total budget savings after including the indirect costs was expected to be EGP5.976 billion after 3?years of budesonide/formoterol entry.

Conclusion: Budesonide/formoterol in mild asthma instead of salbutamol produces better patient outcomes and decreases total costs, with increases in drug cost offset by reductions in non-drug costs due to fewer exacerbations. Budesonide/formoterol is a budget saving option for guideline-directed treatment, from the economic perspective of the payer and the health perspective of the patient.     

The budget impact and cost-effectiveness of defibrotide for treatment of veno-occlusive disease with multi-organ dysfunction in patients post-hematopoietic stem cell transplant

Background: A Phase-3 study of defibrotide compared with historical controls demonstrated a 23% improvement in 100-day survival post-hematopoietic stem cell transplantation (HSCT) among patients with veno-occlusive disease with multi-organ dysfunction (VOD with MOD).

Aim: To estimate the budget impact and cost-effectiveness of introducing defibrotide to a transplant center.

Methods: The authors developed a budget impact model from the perspective of a bone-marrow transplant center. It was estimated that 2.3% of adults and 4.2% of children would develop VOD with MOD following HSCT based on a retrospective hospital database analysis and the effect that treating patients with defibrotide would have on costs for adult and pediatric centers was estimated. A cost-utility analysis (CUA) was also developed to capture the long-term cost-effectiveness of defibrotide. Projected life expectancies in the two groups were estimated based on trial data, transplant registry data, studies of long-term survival among HSCT patients, and US population life-tables.

Results: There was an estimated 3% increase ($330,706) per year in total adult transplantation center costs associated with adopting defibrotide, and a <1% increase ($106,385) for pediatric transplant centers, assuming 100 transplants per year. In the CUA, the lifetime increase in cost per patient was $106,928, life expectancy increased by 3.74 years, and quality-adjusted life-years (QALYs) increased by 2.24. The incremental cost-effectiveness ratio (ICER) was $47,736 per QALY gained; 88% probability defibrotide was cost-effective at a $100,000/QALY threshold.

Conclusion: The budget impact of defibrotide for a transplant center is relatively modest compared to the overall cost of transplantation. Defibrotide provides an important survival advantage for VOD with MOD patients, and the life years gained lead to defibrotide being highly cost-effective.     

Healthcare utilization and costs among chronic bronchitis patients treated with maintenance medications from a US managed care population

Abstract

Objectives:

This study aimed to examine the real-world healthcare resource utilization (HCRU) and direct costs among chronic bronchitis (CB) patients treated with chronic obstructive pulmonary disease (COPD) maintenance medications.

Methods:

This retrospective analysis utilized administrative claims data from 14 US commercial managed care plans. Eligible patients were ≥40 years old, had ≥2 years of continuous enrollment, ≥1 CB (ICD-9-CM code 491.xx) hospitalization or emergency department (ED) visit or ≥2 office visits between 1/1/2004 and 5/31/2011, and had ≥2 pharmacy fills for COPD medications during follow-up (first fill served as the index date). All-cause and COPD-related HCRU and costs were assessed during follow-up. Multivariate models were utilized to identify predictors of total costs.

Results:

Treated CB patients (n?=?17,382; 50.6% female; mean age 66.7 (SD?=?11.4) years) had a mean of 7.6 (SD?=?6.3) COPD maintenance medication fills during follow-up. Overall, 32.6% of patients had ≥1 COPD-related inpatient hospitalizations, 12.9% had ≥1 ED visit, and 81.8% had ≥1 office visit. Mean all-cause and COPD-related total costs were $25,747 (SD?=?$51,105) and $12,609 (SD?=?$36,801), respectively, during follow-up. Among the sub-group with ≥1 exacerbation during baseline year, 42.3% had ≥1 COPD-related inpatient hospitalization, 18.5% had ≥1 ED visit, and 88.2% had ≥1 office visit. Mean follow-up all-cause and COPD-related total costs were $29,861 (SD?=?$49,799) and $16,784 (SD?=?$34,170), respectively. The number of baseline exacerbations was a significant predictor of all-cause and COPD-related total costs during follow-up.

Limitations:

This study lacked standard measures of CB severity; however, severity proxies were utilized.

Conclusion:

HCRU and costs among CB patients were substantial during follow-up, despite treatment with COPD maintenance medications. Additional interventions aiming to prevent or reduce HCRU and costs among CB patients warrant exploration.     

Targeted erlotinib for first-line treatment of advanced non-small cell lung cancer: a budget impact analysis

Objectives:

A recent phase III trial showed that patients with advanced non-small cell lung cancer (NSCLC) whose tumors harbor specific EGFR mutations significantly benefit from first-line treatment with erlotinib compared to chemotherapy. This study sought to estimate the budget impact if coverage for EGFR testing and erlotinib as first-line therapy were provided in a hypothetical 500,000-member managed care plan.

Methods:

The budget impact model was developed from a US health plan perspective to evaluate administration of the EGFR test and treatment with erlotinib for EGFR-positive patients, compared to non-targeted treatment with chemotherapy. The eligible patient population was estimated from age-stratified SEER incidence data. Clinical data were derived from key randomized controlled trials. Costs related to drug, administration, and adverse events were included. Sensitivity analyses were conducted to assess uncertainty.

Results:

In a plan of 500,000 members, it was estimated there would be 91 newly diagnosed advanced NSCLC patients annually; 11 are expected to be EGFR-positive. Based on the testing and treatment assumptions, it was estimated that 3 patients in Scenario 1 and 6 patients in Scenario 2 receive erlotinib. Overall health plan expenditures would increase by $0.013 per member per month (PMPM). This increase is largely attributable to erlotinib drug costs, in part due to lengthened progression-free survival and treatment periods experienced in erlotinib-treated patients. EGFR testing contributes slightly, whereas adverse event costs mitigate the budget impact. The budget impact did not exceed $0.019 PMPM in sensitivity analyses.

Conclusions:

Coverage for targeted first-line erlotinib therapy in NSCLC likely results in a small budget impact for US health plans. The estimated impact may vary by plan, or if second-line or maintenance therapy, dose changes/interruptions, or impact on patients’ quality-of-life were included.     

Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013

Objective: This article aims to calculate the impact of orphan drugs on the Belgian drug budget in 2008 and to forecast its impact over the following 5 years.

Method: The 2008 budget impact was calculated by triangulating information derived from multiple Belgian data sources. The 2008–2013 budget impact analysis was based on three scenarios reflecting different levels of growth in the number of registered orphan drugs in the European Union, the number of drugs reimbursed in Belgium, and the average annual cost per patient per drug in Belgium.

Results: The orphan drug budget impact amounted to €66.2 million (or 5% of the Belgian hospital drug budget) in 2008. The impact would increase to €130–204 million in 2013, depending on the scenario.

Conclusions: This static analysis measured orphan drug costs only, assuming that other components of health expenditure do not change over time. The analysis showed that the budget impact of orphan drugs in Belgium is substantial and rising, thereby putting pressure on total drug expenditure. Policy options to address the rising budget impact include pricing linked to return on investment, risk-sharing arrangements and re-appraisal of orphan drug status if additional indications are approved.