Exploring the Cost-Effectiveness of Newborn Screening for Metachromatic Leukodystrophy (MLD) in the UK.

Metachromatic leukodystrophy (MLD) is a fatal inherited lysosomal storage disease that can be detected through newborn bloodspot screening. The feasibility of the screening assay and the clinical rationale for screening for MLD have been previously demonstrated, so the aim of this study is to determine whether the addition of screening for MLD to the routine newborn screening program in the UK is a cost-effective use of National Health Service (NHS) resources. A health economic analysis from the perspective of the NHS and Personal Social Services was developed based on a decision-tree framework for each MLD subtype using long-term outcomes derived from a previously presented partitioned survival and Markov economic model.

A cost-consequence analysis of the Xpert Xpress CoV-2/Flu/RSV plus test strategy for the diagnosis of influenza-like illnesses.

Aims: Influenza-like illnesses (ILI) affect millions each year in the United States (US). Determining definitively the cause of symptoms is important for patient management. Xpert Xpress CoV-2/Flu/RSV plus (Xpert Xpress) is a rapid, point-of-care (POC), multiplex real-time polymerase chain reaction (RT-PCR) test intended for the simultaneous qualitative detection and differentiation of SARS-CoV-2, influenza A/B, and respiratory syncytial virus (RSV).

Health Economics of Selective Early Rescue Surfactant Administration and Standard Surfactant Administration for Newborns With Respiratory Distress Syndrome.

Objective: To quantify the effect of early rescue surfactant administration techniques for preterm infants with respiratory distress syndrome (RDS) from a health care delivery system perspective.

Methods: A cost-consequence model was developed based on previously published literature to compare the health economic impact of implementing early surfactant administration strategies vs standard surfactant administration via endotracheal intubation and mechanical ventilation (MV).

Results: Early rescue surfactant treatment strategies are associated with a decrease in the number of patients requiring MV, cumulative MV days, and rate of neonatal complications.

Bleeding in patients with hemophilia who have inhibitors: Modeling US medical system utilization and cost avoidance between recombinant factor VIIa products with different clinical dosing requirements.

A mainstay of treatment in patients with hemophilia with inhibitors (PWIs) is the use of a recombinant factor VIIa (rFVIIa) bypassing agent. A new rFVIIa product may allow reduced rFVIIa utilization for on-demand treatment of bleeding episodes (BEs). A decision analytic health economic model was developed to compare the utilization and consequent need for bleed-related clinical encounters of 2 rFVIIa products, with the International Nomenclature Name of eptacog alfa (EA) and eptacog beta (EB).

The impact of increasing multitarget stool DNA use among colorectal cancer screeners in a self-insured US employer population.

In the United States (US), colorectal cancer (CRC) is the second leading cause of cancer-related deaths. With the majority of the US population covered by employer-based health plans, employers can play a critical role in increasing CRC screening adherence, which may help avert CRC-related deaths. Therefore, it is important for self-insured employers to consider the impact of appropriate utilization of CRC screening options.

Cost-Consequence Analysis of Using Cangrelor in High Angiographic Risk Percutaneous Coronary Intervention Patients: A US Hospital Perspective.

Objectives: The objective of this study was to evaluate a US hospital's cost implications and outcomes of cangrelor use in percutaneous coronary intervention (PCI) patients with two or more angiographic high-risk features (HRFs), including avoidance of oral P2Y inhibitor pretreatment in patients requiring cardiac surgery. Intravenous cangrelor provides direct, immediate onset and rapid-offset P2Y inhibition, which may reduce the necessity for oral P2Y pretreatment.

Methods: A decision analytic model was developed, estimating the annual impact over 3 years of cangrelor availability.

The health economic impact of varying levels of adherence to colorectal screening on providers and payers.

Aims: To examine the impact of increasing multi-target stool DNA test (mt-sDNA [Cologuard]) utilization for colorectal cancer (CRC) screening in cohorts aged 50-75 and 45-75 years old with varying levels of adherence from the perspectives of integrated delivery networks (IDNs) and payers.

Materials And Methods: We developed a budget impact model that simulates CRC screening with colonoscopy over a 10-year time horizon, fecal immunochemical test (FIT), and mt-sDNA according to the United States Preventive Services Task Force and American Cancer Society guidelines for average risk adults. We evaluated varying levels of screening adherence for a status quo scenario and for an increased mt-sDNA utilization scenario, from the IDN and payer perspectives.

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER).

: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results. : To compare the manufacturer's proprietary CUA model to the model published by the Institute for Clinical and Economic Review (ICER), and to update the manufacturer's model with long-term follow-up data and some key ICER assumptions. : We updated a recent CUA evaluating value for money in cost per incremental Quality-adjusted Life Year (QALY) of onasemnogene abeparvovec versus nusinersen (Spinraza®) or best supportive care (BSC) in symptomatic SMA1 patients, and compared it to the ICER model.

Cost-consequence analysis of F-fluciclovine for the staging of recurrent prostate cancer.

: Current detection methodologies are often unable to identify the location and extent of recurrent prostate cancer (PCa) leading potentially to 'futile' local therapies in the presence of metastatic disease. The use of  F-fluciclovine PET/CT may lead to better patient management. : The aim of this study was to quantify the economic impact and cost-consequence of using  F-fluciclovine PET/CT in PCa recurrence.

Projecting total costs and health consequences of increasing mt-sDNA utilization for colorectal cancer screening from the payer and integrated delivery network perspectives.

To evaluate total costs and health consequences of a colorectal cancer (CRC) screening program with colonoscopy, fecal immunochemical tests (FIT), and expanded use of multitarget stool DNA (mt-sDNA) from the perspectives of Integrated Delivery Networks (IDNs) and payers in the United States. We developed a budget impact and cost-consequence model that simulates CRC screening for eligible 50- to 75-year-old adults. A status quo scenario and an increased mt-sDNA scenario were modeled.

Cost-effectiveness analysis of using onasemnogene abeparvocec (AVXS-101) in spinal muscular atrophy type 1 patients.

: Spinal muscular atrophy type 1 (SMA1) is a devastating genetic disease for which gene-replacement therapy may bring substantial survival and quality of life benefits. : This study investigated the cost-effectiveness of onasemnogene abeparvovec (AVXS-101) gene-replacement therapy for SMA1. : A Markov model was used to estimate the incremental cost-effectiveness ratio (ICER), expressed as cost/quality-adjusted life year ($/QALY), of AVXS-101 versus nusinersen over a lifetime.

Budget Impact Analysis of Using Daunorubicin-Cytarabine Liposome in Patients with Newly Diagnosed Therapy-Related AML or AML and Myelodysplasia-Related Changes.

Background: Current national estimates for acute myelogenous leukemia (AML) indicate this disease accounts for 1.1% of new cancer diagnoses and 1.8% of cancer deaths in the United States.

Evaluating cost benefits of combination therapies for advanced melanoma.

Background: Although a number of monoimmunotherapies and targeted therapies are available to treat BRAF+ advanced melanoma, response rates remain relatively low in the range of 22-53% with progression-free survival (PFS) in the range of 4.8-8.8 months.

Use of Oritavancin in Moderate-to-Severe ABSSSI Patients Requiring IV Antibiotics: A U.S. Payer Budget Impact Analysis.

Background: It is estimated that acute bacterial skin and skin structure infections (ABSSSI) account for nearly 10% of hospital admissions and 3.4-3.8 million emergency department visits per year in the United States.

Use of Oritavancin in Acute Bacterial Skin and Skin Structure Infections Patients Receiving Intravenous Antibiotics: A US Hospital Budget Impact Analysis.

Background And Objective: Nearly 10% of all US hospital admissions are attributed to acute bacterial skin and skin structure infections (ABSSSIs). While most antibacterials used to treat these infections require multi-day and multi-dose regimens, a single-dose treatment is now available. The objective of this analysis is to estimate the annual budget impact of using single-dose oritavancin in patients with moderate to severe ABSSSIs receiving intravenous methicillin-resistant Staphylococcus aureus (MRSA)-active antibacterials from a US hospital perspective.