United States Payer Perspective Research Articles

Cost-Effectiveness of Betrixaban Compared with Enoxaparin for Venous Thromboembolism Prophylaxis in Nonsurgical Patients with Acute Medical Illness in the United States.

Studies show that the risk of venous thromboembolism (VTE) continues post-discharge in nonsurgical patients with acute medical illness. Betrixaban is the first anticoagulant approved in the United States (US) for VTE prophylaxis extending beyond hospitalization. The aim was to establish whether betrixaban for VTE prophylaxis in nonsurgical patients with acute medical illness at risk of VTE in the US is cost-effective compared with enoxaparin. A cost-effectiveness analysis was conducted, estimating the cost per quality-adjusted life-year (QALY) gained with betrixaban (35-42days) compared with enoxaparin (6-14days) from a US payer perspective over a lifetime horizon. A decision tree (DT) estimated primary VTE events, thrombotic events, and treatment complications in the first 3months based on data from the phase III Acute Medically Ill VTE Prevention with Extended Duration Betrixaban study. A Markov model estimated recurrent events and long-term complication risks from published literature. EuroQoL-5 Dimensions utility data and costs inflated to 2017 US dollars (US$) were from published literature. Results were discounted at 3.0% per annum. Deterministic and probabilistic sensitivity analyses explored uncertainty. Betrixaban dominated enoxaparin, with savings of US$784 and increased QALYs of 0.017 per patient. In addition, betrixaban dominated enoxaparin across all sensitivity analyses, but was most sensitive to utilities and DT probabilities. Furthermore, probabilistic sensitivity analysis found that betrixaban was more cost-effective than enoxaparin at all willingness-to-pay thresholds. Betrixaban can be considered cost-effective for nonsurgical patients with acute medical illness at risk of VTE, requiring longer VTE prophylaxis from hospitalization through post-discharge.

Cost-Effectiveness Analysis of Lenalidomide for Maintenance Therapy after Autologous Stem Cell Transplant (ASCT) in Newly Diagnosed Multiple Myeloma (NDMM) Patients: A United States Payer Perspective

Introduction: ASCT in multiple myeloma (MM) is associated with prolonged progression-free survival (PFS) compared with chemotherapy alone. Studies have shown that lenalidomide maintenance therapy after ASCT significantly improves PFS and overall survival (OS) in patients (pts) with NDMM. Although post-ASCT lenalidomide maintenance therapy is approved by the US FDA and the EMA for pts with MM, a small percentage of pts either use bortezomib or no maintenance in this setting. The current study aimed to assess the cost-effectiveness of lenalidomide maintenance versus no treatment or bortezomib maintenance after ASCT in pts with NDMM from a US third-party payer perspective.Methods: A partitioned survival model with a 28-day cycle was developed to estimate costs and outcomes of lenalidomide maintenance versus either no treatment or bortezomib maintenance therapy after ASCT among pts with NDMM over a lifetime time horizon. The model included four health states: PFS on treatment, PFS off treatment, progressed disease, and death. The OS and PFS for lenalidomide and no treatment arms were estimated using observed data from the phase 3 CALGB trial. Crossover adjustment was implemented to adjust for the potential diluting effects introduced by pts crossing over to lenalidomide from the no treatment arm before progression in the CALGB trial. Time on treatment for lenalidomide was estimated using the observed data in the pooled phase 3 trials (CALGB, IFM, GIMEMA). Efficacy inputs for bortezomib maintenance were estimated based on published literature. Standard parametric models were fitted to extrapolate OS and PFS for each treatment, and best fit was determined based on Akaike or Bayesian information criterion and clinical judgement. OS was adjusted using natural mortality rates in the USA. Treatment costs (including drug and drug administration costs), post-progression treatment costs, adverse event (AE) costs, and medical costs associated with health states were obtained from publicly available databases, literature, and real-world data. All costs were inflated to 2018 US dollars. Utilities for each health state and disutilities associated with AEs were obtained from the literature. Incremental costs per quality-adjusted life year (QALY) and life year (LY) gained were estimated comparing lenalidomide maintenance therapy with each comparator. Deterministic sensitivity analyses (DSAs) were performed to test the robustness of the results.Results: Over a lifetime time horizon, lenalidomide maintenance was associated with an increase of 3.64 and 2.76 in LYs compared with no treatment and bortezomib maintenance, respectively; and an increase of 2.99 and 2.42 in QALYs, respectively. Pts in the lenalidomide maintenance arm incurred higher total direct costs with an incremental cost of $476,690 and $412,872 versus no treatment and bortezomib maintenance, respectively. Initial and post-progression treatment costs comprised the majority of direct costs. The annual treatment costs for lenalidomide maintenance decreased by 49% and 86%, 3 years and 5 years after treatment initiation, respectively.Incremental cost per LY gained for lenalidomide maintenance versus no treatment and bortezomib maintenance was $130,817 and $149,411, respectively, and incremental cost per QALY gained was $159,240 and $170,408, respectively. The base-case results suggest that lenalidomide is cost-effective at a willingness-to-pay threshold of $200,000.Results from the DSA generally supported the base-case findings, with the largest variation observed when time horizon and treatment costs for lenalidomide were varied. Longer time horizons yielded greater cost-effectiveness for lenalidomide because of the reduction in treatment costs and increased effectiveness benefits. Lower initial treatment costs also yielded greater cost-effectiveness for lenalidomide.Conclusions: Over a lifetime time horizon, compared to no treatment and treatment with bortezomib, lenalidomide maintenance resulted in better effectiveness with incremental QALYs of 2.99 and 2.42, respectively, favorable incremental cost-effectiveness ratios, and an OS advantage. Annual treatment costs for lenalidomide decrease with incremental effectiveness increasing over time. Lenalidomide maintenance after ASCT offers a cost-effective strategy in the treatment of pts with NDMM from a US third-party payer perspective. DisclosuresZhou:Celgene Corporation: Research Funding; Analysis Group, Inc.: Employment. Parikh:Celgene Corporation: Employment, Equity Ownership. Chai:Celgene Corporation: Research Funding; Analysis Group, Inc.: Employment. Rokito:Analysis Group, Inc.: Employment; Celgene Corporation: Research Funding. Udeze:Celgene Corporation: Employment. Xie:Celgene Corporation: Research Funding; Analysis Group, Inc.: Employment. Agarwal:Celgene Corporation: Employment.

PCV45 - Cost Effectiveness Of Direct Oral Anticoagulants (DOACS) Compared With Warfarin: A Literature Review

Direct oral anticoagulants (DOACs), including apixaban, rivaroxaban, dabigatran, edoxaban, are alternative anticoagulant therapies to warfarin in United States (US) to prevent strokes or recurrent venous thromboembolisms (VTEs), due to their lower risk of bleeding. Since DOACs cost more than warfarin, the objective of this study was to examine the published literatures on the cost-effectiveness of DOACs compared to warfarin. A literature review was conducted in PubMed using keywords: “cost-effectiveness”, “warfarin”, “apixaban” or “rivaroxaban” or “dabigatran” or “edoxaban”. The inclusion criteria were articles (1) measured costs from US societal or US payer perspective; (2) used standard warfarin therapy as comparator; (3) with full text published in peer-reviewed journals. We excluded non-human studies and literature reviews from the final review. A total of 46 studies were identified, and 11 studies met the selection criteria. Most studies (8) accessed the anticoagulant indications for stroke prevention in nonvalvular atrial fibrillation (AF) patients, and 2 studies also included indication for systemic embolism prevention and 1 in VTE, respectively. Ten studies included patients 65 years old and above, while 1 included patients aged over 60. Dabigatran was the most commonly studied DOACs (dabigatran, 6; apixaban, 3; rivaroxaban, 3; edoxaban, 2). Only two studies included indirect costs, such as travel cost and loss of productivity, in addition to the direct medical costs. ICER of any DOAC to warfarin ranged from -$60,725 to $45,372 with a $50,000 willingness-to-pay(WTP) per QALY. All articles concluded DOACs are cost-effective alternatives to warfarin for the studied indications from either societal or US payer perspective. This literature review reveals DOACs are cost-effective alternatives to warfarin in stroke prevention for AF patients, and in secondary VTE prevention, even after adjusting indirect cost associated with therapy. The parameters were obtained from literatures, and future studies are warranted to better understand the cost-effectiveness in practice.

Cost-Effectiveness of Olaratumab in Combination with Doxorubicin for Patients with Soft Tissue Sarcoma in the United States.

Background Standard first-line treatments for advanced soft tissue sarcoma (STS) have changed little for 40 years, and outcomes have been poor. Recently, the United States (US) Food and Drug Administration conditionally approved olaratumab in combination with doxorubicin (Olara + Dox) based on a randomized phase II trial that reported a significant 11.8-month improvement in median survival versus single-agent doxorubicin (Dox). The present study investigated the cost-effectiveness of Olara + Dox compared with Dox and five other standard-of-care regimens from the US payer perspective. Methods An economic model was constructed to estimate costs and outcomes over patients' lifetimes from start of therapy. Progression-free and overall survival were based on survival analysis of patient-level data and a meta-analysis. Adverse-event rates were based on trials. Costs were from published sources. Results Olara + Dox resulted in an estimated additional 1.27 life-years (LYs) compared with Dox, with an increase in total expected lifetime costs of $133,653. The incremental cost-effectiveness ratio (ICER) was estimated at $105,408 per LY gained; in a fully incremental analysis, all other regimens were dominated (higher costs and lower LYs or a higher ICER). Conclusion Olara + Dox is cost-effective for STS treatment compared with Dox and other standard-of-care regimens at willingness-to-pay thresholds of $150,000 per LY and above.

Abstract 16597: Economic Evaluations of PCSK9 Inhibitors in Reducing Cardiovascular Risk From Societal & United States Payer Perspective

Introduction: We aim to evaluate cost-effectiveness of recent FDA advisory committee recommended PCSK9 inhibitors for reducing cardiovascular disease (CVD) risk from societal & U.S. third-party payer perspective. Methods: A Markov model was used to evaluate costs and effects associated with addition of PCSK9 inhibitor to standard of care (SOC) based on a) estimated 53% 1-year relative risk reduction (SOC 1 year CVD event rate: 2.18%, PCSK9 inhibitor+SOC: 0.95%), b) indirect annual CVD reduction probability (using Framingham risk) via mean 61% LDL lowering among at CV risk patients. The model considers cycles of 1 year. Post CVD events last 5 years, after that time individuals go back to treatment state if no subsequent CVD observed. Health state costs were derived from published evidence. Survival probabilities >1 year were transformed to annual probabilities assumed on survival models. From societal perspective, cost per Quality-Adjusted Life Years (QALYs) gained are presented. We estimated payer’s perspective on net benefit balancing treatment vs. avoided cost and premium revenues over patient lifespan. Results: The table highlights societal gains and payer’s net benefit across range of estimated annual drug cost. At willingness-to-pay threshold of $100,000/QALY, PCSK9 inhibitors appear cost-effective if priced annually at $2500. At annual cost of $10,000, cost/QALY gained with PCSK9 inhibitor is nearly $400,000. From payer’s perspective, approximate net benefit was -$11,000 to -$51,000 per patient treated across annual drug cost range. Net benefits were only positive if treatment priced around $500/year. Conclusion: This study provides insights to facilitate discussion among healthcare stakeholders for considering future PCSK9 inhibitor’s utility balancing estimated benefits while accounting need for additional financial resources. Gaps between social & private benefits need to be aligned for appropriate PCSK9 inhibitors adoption in U.S.

PCN117 - A Health Technology Assessment of a Proposed Biosimilar for the Treatment of Chemotherapy Induced Febrile Neutropenia: A United States Payer Perspective

PCN117 - A Health Technology Assessment of a Proposed Biosimilar for the Treatment of Chemotherapy Induced Febrile Neutropenia: A United States Payer Perspective

PCN50 - Clinical Outcomes and Budget Impact of Cobas® Egfr Mutation Test Versus Sanger Sequencing in the Treatment of Locally Advanced or Metastatic Nsclc: A United States Payer Perspective

PCN50 - Clinical Outcomes and Budget Impact of Cobas® Egfr Mutation Test Versus Sanger Sequencing in the Treatment of Locally Advanced or Metastatic Nsclc: A United States Payer Perspective

PIH7 - Budget Impact of HPV16/18 Genotyping Tests for the Management of Non-concordant Cotesting Cervical Cancer Screening Results: A United States Payer Perspective

PIH7 - Budget Impact of HPV16/18 Genotyping Tests for the Management of Non-concordant Cotesting Cervical Cancer Screening Results: A United States Payer Perspective

PHS18 Economic Burden of Atopic Dermatitis from a United States Payer Perspective

PHS18 Economic Burden of Atopic Dermatitis from a United States Payer Perspective

Cost‐Utility Analysis of Biologic Treatments for Moderate‐to‐Severe Crohn's Disease

To compare the cost versus utility of four guideline-recommended biologic treatments-infliximab, adalimumab, certolizumab pegol, and natalizumab-for the treatment of Crohn's disease from a United States payer perspective. Cost-utility decision analytic model using a Monte Carlo simulation of 10,000 cases. Published primary and tertiary literature. Patients with moderate-to-severe Crohn's disease, as categorized by the Crohn's Disease Activity Index, that failed to respond to standard therapy and who were treatment naive to biologics. The decision analytic model base case was a 35-year-old patient weighing 70 kg with moderate-to-severe Crohn's disease. Therapeutic efficacy data were obtained from published clinical trials. Online prescription drug, hospitalization data, and Current Procedural Terminology codes were used to estimate cost. Utility measures were estimated using published data obtained from patients with Crohn's disease. The time end point used for the model was 54weeks. The analysis demonstrated considerable overlap in quality-adjusted life years gained with the four agents (i.e., mean values across four treatments0.79-0.80). Although infliximab had the lowest median cost, there was overlap in the 95% confidence intervals (CIs) compared with the other three biologic products: mean (95% CI) cost for infliximab $22,663 ($19,105-26,433), adalimumab $27,515 ($23,796-31,584), certolizumab pegol $29,062 ($24,952-33,882), and natalizumab $31,166 ($25,915-37,195). However, the cost-effectiveness acceptability curve demonstrated that infliximab had the most scenarios (95.2%) when it was the most cost-effective biologic therapy using a willingness-to-pay threshold of $100,000/quality-adjusted life years gained. Patients with moderate-to-severe Crohn's disease that failed to respond to standard treatment should preferentially receive infliximab as their initial biologic treatment, since this agent had the highest probability of being the most cost-effective therapy compared with the other biologic treatment options.

A cost-effectiveness analysis of using azacitidine vs. decitabine in treating patients with myelodysplastic syndromes

Objective:Azacitidine and decitabine are used to treat patients with myelodysplastic syndromes (MDS) in the United States (US). This study sought to assess their relative cost-effectiveness.Design and methods:The authors developed a cost-effectiveness Markov model (1-month cycles) tracking hypothetical cohorts of MDS patients treated with azacitidine or decitabine over 2 years. The model used a US payer perspective and 2009 costs. Health states modeled included MDS with Transfusion Dependence, MDS with Transfusion Independence, Progression to Acute Myelogenous Leukemia (AML), and Death. Incremental cost-effectiveness outcomes included cost per quality-adjusted life year (QALY), cost per life year (LY), cost per patient-month of transfusion independence, and cost per case of AML progression avoided. One-way sensitivity analyses were performed on key model parameters.Results:Compared to decitabine, azacitidine was associated with better survival (1.512 LYs vs 1.292), more QALYs gained (1.041 vs 0.870), more patient-months with transfusion independence (8.328 vs 6.224), and a greater proportion of patients avoiding progression to AML (50.9% vs 28.5%). Total per-patient costs over 2 years for azacitidine were lower than for decitabine ($150,322 vs $166, 212).Limitations:To inform and update the model over time, it will be important that randomized or observational clinical studies be conducted to directly compare azacitidine and decitabine, provide new information on how these medicines are used, and on their relative clinical effectiveness.Conclusion:Results demonstrate that azacitidine provides greater clinical benefit and costs less than decitabine across all key outcomes. These results accentuate the positive role of azacitidine in providing cost-effective care for MDS.

Cost analysis of four major drug-eluting stents in diabetic populations

To use an indirect comparisons approach and conduct a cost analysis comparing four drug-eluting stents (DES) from a United States (US) payer (i.e., fixed-fee reimbursement) perspective. Studies were chosen that randomised two or more DES in diabetic patients. A one-year target lesion revascularisation (TLR) risk for Taxus was first derived. Risk Ratios (RRs) for each DES versus Taxus were calculated through meta-analyses. The RRs were multiplied by the average TLR risk for Taxus to estimate DES TLR risks. Estimates were added to a budget-impact model, along with utilisation and reimbursement rates for diagnosis-related groups. Budgets were calculated, assuming 100% stent use and 200,000 diabetic beneficiaries. One-year TLR risks were estimated to be 3.2%, 7.1%, 6.9% and 7.9% for Cypher, Endeavor, Taxus and Xience respectively. By substituting Cypher for DES with higher TLR, results predicted annual cost-savings greater than $146 million per population ($ 733 per patient). Results were comparable when assuming no difference in TLR risk between Endeavor, Taxus and Xience. When outcomes from trials of diabetic populations are analysed and used in a budget-impact model from a US payer perspective, the use of Cypher is associated with lower TLR rates, which translates into large potential cost savings.

Cost-Effectiveness of Pemetrexed as First-Line Maintenance Therapy for Advanced Nonsquamous Non-small Cell Lung Cancer

The primary objective was to estimate the cost-effectiveness of maintenance therapy with pemetrexed (Pem) compared with observation, each with best supportive care, in patients with advanced non-small cell lung cancer (NSCLC) who have completed, without progression, at least four cycles of first-line platinum chemotherapy, particularly in those with nonsquamous cell histology. Secondary comparisons included Pem with erlotinib (Erl) or Pem with bevacizumab (Bev). A semi-Markov model was developed to compare the 3-year impact of Pem with three other alternatives for maintenance therapy from a United States payer perspective. Data from randomized controlled clinical trials provided clinical inputs. Medicare reimbursement rates were used to determine drug costs. A retrospective claims database analysis was used to obtain estimates of other direct NSCLC-related costs. In the prespecified subset of patients with nonsquamous cell histology only, the incremental cost per life-year gained was $122,371 for Pem to observation and $150,260 for Pem to Erl, and Bev was dominated by Pem. In all patients with advanced NSCLC regardless of histologic subtype, using Pem as maintenance therapy led to an incremental cost per life-year gained of $205,597 compared with observation and $312,341 compared with Erl. Compared with observation and other agents used and/or reimbursed for maintenance therapy in advanced NSCLC, Pem may be considered cost-effective, particularly in patients with nonsquamous cell histology. This analysis is the first to evaluate the cost-effectiveness of maintenance therapy in advanced NSCLC and emphasizes the importance of histology in identifying the appropriate patient for Pem maintenance therapy.