Atrial fibrillation (AF) is a common arrhythmia, contributing to ≥15% of strokes in the United States (US). Its often asymptomatic nature makes finding cases challenging, and 24.6% of AF cases remain undiagnosed. Research has focused on screening for AF to prevent stroke via timely intervention.

Background Patients with homozygous familial hypercholesterolemia are at high risk of cardiovascular disease due to high low-density lipoprotein (LDL)-cholesterol levels. Cardiovascular disease outcome studies are impossible to conduct, due to the rarity of homozygous familial hypercholesterolemia. We modelled the potential efficacy of lomitapide, a microsomal transfer protein inhibitor, on major adverse cardiovascular events (MACEs) and survival. Design We calculated the effect on cardiovascular outcomes of a 38% plasma LDL-cholesterol reduction induced by lomitapide. Methods Age-dependent hazards and treatment-dependent hazard ratios for mortality and time to first MACE were calculated from an observational study of 149 South African homozygous familial hypercholesterolemia patients. Cardiovascular-related mortality hazards were derived by adjusting for general population non-cardiovascular-related mortality. For every mmol/L LDL-cholesterol reduction, a relative risk reductions of 23% (mortality) and 15% (major adverse cardiovascular events) were observed. Results For the most robust model, baseline median survival with current treatments (LDL-cholesterol 8.7 mmol/L) was 48 years. In the survival benefit analysis, starting lomitapide at age 18 years and reducing LDL-cholesterol by 3.3 mmol/L from baseline would increase life expectancy by 11.2 years and delay the time to first MACE by 5.7 years. Analysis suggested lifetime lomitapide treatment could increase median life expectancy by 11.7 years and time to first MACE by 6.7 years. Conclusion Our modelling analyses show that additional LDL-cholesterol lowering by lomitapide may increase life expectancy in patients with homozygous familial hypercholesterolemia. Further clinical studies are warranted to determine the cardiovascular morbidity and mortality benefits of lomitapide.

CPX-351 is a dual-drug liposomal encapsulation of cytarabine and daunorubicin that delivers a synergistic drug ratio. In a phase 3 trial (NCT01696084) in elderly patients with newly diagnosed tAML or AML-MRC, CPX-351 demonstrated superior overall survival (9.56 vs 5.95 months; HR = 0.69; P= 0.005) and significantly improved complete remission rate (38% vs 26%; P= 0.040) compared to the 7+3 regimen of cytarabine and daunorubicin, the current standard of care for remission induction. These clinical improvements may increase the cost of caring for patients with AML through higher drug costs, increased numbers of patients receiving hematopoietic cell transplant (HCT), and the costs of disease management during extended survival. In an era of increased scrutiny of drug costs and health care budgets, it is essential to understand the balance of clinical benefits and costs associated with treatment of AML using CPX-351.

Prior analyses beyond clinical trials are yet to evaluate the projected lifetime benefit of apixaban treatment compared to low-molecular-weight heparin (LMWH)/vitamin K antagonist (VKA) for treatment of venous thromboembolism (VTE) and prevention of recurrences. The objective of this study is to assess the cost-effectiveness of initial plus extended treatment with apixaban versus LMWH/VKA for either initial treatment only or initial plus extended treatment.

Purpose: To assess the cost-effectiveness of apixaban versus rivaroxaban, low-molecular-weight heparin (LMWH)/dabigatran, and LMWH/vitamin K antagonist (VKA) for the initial treatment and prevention of recurrent thromboembolic events in patients with venous thromboembolism (VTE).Methods: A Markov model was developed to evaluate the pharmacoeconomic effect of 6 months of treatment with apixaban versus other anticoagulants over a lifetime horizon. Network meta-analyses were conducted using the results of the Apixaban after the Initial Management of Pulmonary Embolism and Deep Vein Thrombosis with First-Line Therapy (AMPLIFY), EINSTEIN-pooled, and RE-COVER I and II trials for the following end points: recurrent VTE, major bleeds, clinically relevant non-major bleeds, and treatment discontinuations. The analysis was conducted from the perspective of the United Kingdom National Health Service. The outcomes evaluated were the number of events avoided in a 1000-patient cohort, total costs, life years, quality-adjusted life years (QALYs), and cost per QALY gained over a patient's lifetime.Findings: Treatment for 6 months with apixaban was projected to result in fewer recurrent VTE and bleeding events in comparison to rivaroxaban, LMWH/dabigatran, and LMWH/VKA. Apixaban was cost-effective compared with LMWH/VKA at an incremental cost-effectiveness ratio of 2520 per QALY gained and was a dominant (ie, lower costs and higher QALYs) alternative to either rivaroxaban or LMWH/dabigatran. Sensitivity analysis indicated that results were robust over a wide range of inputs.Implications: The assessment of the effects and costs of apixaban in this study predicted that apixaban is a dominant alternative to rivaroxaban and LMWH/dabigatran and a cost-effective alternative to LMWH/VKA for 6 months of treatment of VTE and the prevention of recurrence. (C) 2016 Elsevier HS Journals, Inc. All rights reserved.

To assess the predictive validity of apixaban cost-effectiveness model by comparing key event rates from model to those seen in AMPLIFY and AMPLIFY-EXT trials in patients with venous thromboembolism (VTE). Markov model was developed to extrapolate the observed clinical impact of apixaban versus

BACKGROUND:The economic burden of treating end-stage renal disease (ESRD) continues to grow. As one response, effective January 1, 2011, Medicare implemented a bundled prospective payment system (PPS, including injectable drugs) for dialysis patients. This study investigated the 5-year budget impact on Medicare under the new PPS of changes in the distribution of patients undergoing peritoneal dialysis (PD), in-center hemodialysis (ICHD), and home hemodialysis (HHD).METHODS:An Excel-based budget impact model was created to assess dialysis-associated Medicare costs. The model accounted for dialysis access establishment, the current monthly capitation physician payment for ESRD, Medicare dialysis payments (including start-up costs), training, oral drug costs, and the costs and probabilities of adverse events including access failure, hospitalization for access infection, pneumonia, septicemia, and cardiovascular events. United States Renal Data System (USRDS) data were used to project the US Medicare dialysis patient population across time. The baseline scenario assumed a stable distribution of PD (7.7%), HHD (1.3%) and ICHD (91.0%) over 5 years. Three comparison scenarios raised the proportions of PD and HHD by (1) 1% and 0.5%, (2) 2% and 0.75%, and (3) 3% and 1% each year; a fourth scenario held HHD constant and lowered PD by 1% per year.RESULTS:Under the bundled PPS, scenarios that increased PD and HHD from 7.7% and 1.3% over 5 years resulted in cumulative savings to Medicare of $114.8M (Scenario 1, 11.7% PD and 3.3% HHD at year 5), $232.9M (Scenario 2, 15.7% PD and 4.3% HHD at year 5), and $350.9M (Scenario 3, 19.7% PD and 5.3% HHD at year 5). When the PD population was decreased from 7.7% in 2013 to 3.7% by 2017 with a constant HHD population, the total Medicare payment for dialysis patients increased by over $121.2M.CONCLUSIONS:Under Medicare bundled PPS, increasing the proportion of patients on PD and HHD vs ICHD could generate substantial savings in dialysis-associated costs to Medicare.

Economic models are developed to provide decision makers with information related to the real-world effectiveness of therapeutics, screening and diagnostic regimens. Although compliance with these regimens often has a significant impact on real-world clinical outcomes and costs, compliance and persistence have historically been addressed in a relatively superficial fashion in economic models. In this review, we present a discussion of the current state of economic modelling as it relates to the consideration of compliance and persistence. We discuss the challenges associated with the inclusion of compliance and persistence in economic models and provide an in-depth review of recent modelling literature that considers compliance or persistence, including a brief summary of previous reviews on this topic and a survey of published models from 2005 to 2012. We review the recent literature in detail, providing a therapeutic-area-specific discussion of the approaches and conclusions drawn from the inclusion of compliance or persistence in economic models. In virtually all publications, variation of model parameters related to compliance and persistence was shown to have a significant impact on predictions of economic outcomes. Growing recognition of the importance of compliance and persistence in the context of economic evaluations has led to an increasing number of economic models that consider these factors, as well as the use of more sophisticated modelling techniques such as individual simulations that provide an avenue for more rigorous consideration of compliance and persistence than is possible with more traditional methods. However, we note areas of continuing concern cited by previous reviews, including inconsistent definitions, documentation and tenuous assumptions required to estimate the effect of compliance and persistence. Finally, we discuss potential means to surmount these challenges via more focused efforts to collect compliance and persistence data.