Cost-Effectiveness of Voncento Prophylaxis Versus On-Demand Treatment in von Willebrand Disease in the United Kingdom.

von Willebrand factor (VWF) concentrates may be required for on-demand treatment (ODT) or long-term prophylaxis (LTP) in von Willebrand disease (VWD). This study assesses the cost-effectiveness of LTP compared with ODT in VWD patients treated with Voncento in the United Kingdom (UK). A Markov structure was developed to estimate quality-adjusted life years (QALYs) and costs of VWD treatment over a lifetime horizon.

Analysis of long-term clinical and cost impact of etranacogene dezaparvovec for the treatment of hemophilia B population in the United States.

Introduction: Etranacogene dezaparvovec (EDZ), Hemgenix, is a gene therapy recently approved for people with hemophilia B (PwHB).

Objective: To estimate long-term clinical impact and cost of EDZ in the United States (US).

Methods: A decision-analytic model was developed to evaluate the long-term impact of introducing EDZ for PwHB over a 20-year time horizon.

Assessing health plan payer's budget impact of etranacogene dezaparvovec for the treatment of hemophilia B in the United States.

Background: Etranacogene dezaparvovec is a recently approved gene therapy for people with hemophilia B (PwHB). Current standard of care is prophylaxis with factor IX (FIX) to prevent bleeding. Etranacogene dezaparvovec increases blood FIX levels such that FIX prophylaxis could be eliminated.

Assessing Public Health Impact of Four Pediatric Pneumococcal Conjugate Vaccination Strategies in the Netherlands.

Introduction: The 10-valent pneumococcal conjugate vaccine (PCV10, Synflorix) was introduced into the Dutch pediatric national immunization program (NIP) starting in 2011. However, there is substantial pneumococcal disease burden due to increases in non-PCV10 covered serotypes. Higher-valent vaccines for pediatrics (PCV13, PCV15, and PCV20) may alleviate much of the remaining disease burden upon implementation through broader serotype coverage.

Historical Population-Level Impact of Infant 13-Valent Pneumococcal Conjugate Vaccine (PCV13) National Immunization Programs on Invasive Pneumococcal Disease in Australia, Canada, England and Wales, Israel, and the United States.

Introduction: This study estimates the annual population-level impact of 13-valent pneumococcal conjugate vaccine (PCV13) infant national immunization programs (NIPs) on vaccine-type and non-vaccine type invasive pneumococcal disease (IPD) incidence across all ages using national surveillance data.

Methods: We identified countries (Australia, Canada, England and Wales, Israel, and the US) with national IPD active surveillance data that introduced the seven-valent PCV (PCV7) followed by PCV13, which also reported annual serotype- and age group-specific incidence. We extracted IPD incidence by serotype groupings [PCV13 minus PCV7 (PCV13-7) serotypes; PCV13-7 serotypes excluding serotype 3; non-PCV13 serotypes; and the 20-valent (PCV20) minus PCV13 (PCV20-13) serotypes] and by age groups (< 2 years, 2-4 years, 5-17 years, 18-34 years, 35-49 years, 50-64 years, and ≥ 65 years).

Cost-Effectiveness Analysis of the South African Infant National Immunization Program for the Prevention of Pneumococcal Disease.

Introduction: Pneumococcal disease, which presents a substantial health and economic burden, is prevented through pneumococcal vaccination programs. We assessed the impact of switching from a 13-valent-based (PCV13) to lower 10-valent-based (PCV10-GlaxoSmithKline [GSK] or PCV10-Serum Institute of India [SII]) or higher-valent (PCV15 or PCV20) vaccination programs in South Africa.

Methods: A previously published decision-analytic model was adapted to a South African setting.

Cost-Effectiveness of Blood-Based Brain Biomarkers for Screening Adults with Mild Traumatic Brain Injury in the French Health Care Setting.

Two blood-based brain biomarker tests such as the combination of glial fibrillary acidic protein and ubiquitin C-terminal hydrolase-L1 (GFAP+UCH-L1) or S100B have potential to reduce the need for head computed tomography (CT) scanning in patients with mild traumatic brain injury (mTBI). We assessed the clinical and economic impact of using GFAP+UCH-L1 versus CT scan and GFAP+UCH-L1 versus S100B to screen adults with suspected mTBI presenting to an emergency department (ED). A decision model was developed to estimate costs and health outcomes of GFAP+UCH-L1, CT scan, and S100B associated with these screening protocols.

Real-World Financial and Clinical Impact of Diagnostic-Driven and Empirical-Treatment Strategies in High-Risk Immunocompromised Patients with Suspected Aspergillus Infection in the United Kingdom.

A diagnostic-driven (DD) treatment strategy has proven successful for treating invasive fungal infections (IFIs) caused by Aspergillus. However, uptake of this treatment strategy is not fully embraced. This study compares the economic and clinical impact of DD and empirical-treatment (ET) strategies used within hospitals.

Clinical and economic impacts of large volume delayed sampling and pathogen reduction technology platelet processing strategies in the United States.

Background: Large volume delayed sampling (LVDS) and pathogen reduction technology (PRT) are strategies for platelet processing to minimize transfusion of contaminated platelet components (PCs). This study holistically compares the economic and clinical impact of LVDS and PRT in the United States.

Study Design And Methods: A decision model was constructed to simulate collection, processing, and use of PCs and to compare processing strategies: PRT with 5-day shelf life, LVDS with 7-day shelf life (LVDS7), and LVDS with 5-day shelf life extended to 7 days with secondary testing (LVDS5/2).

Validation of a Novel Forecasting Method for Estimating the Impact of Switching Pneumococcal Conjugate Programs: Evidence from Belgium.

Introduction: Since 2010, 10-valent (PCV10) and 13-valent pneumococcal conjugate vaccines (PCV13) have been available as part of infant national immunization programs. Belgium is as one of the few countries that implemented PCV13 (2007-2015), switched to PCV10 (2015-2018) and then switched back to PCV13 (2018-present) after observing increases in disease. We assessed the impacts of both historical and prospective PCV choice in the context of the Belgian health care system and used this experience to validate previously developed economic models.

Conversational analysis of consciousness during seizures.

Objective: The objectives of the study were to 1) investigate how patients with epilepsy describe the subjective, conscious experience of having a seizure and 2) determine whether certain themes and descriptions correspond to specific types of epilepsy.

Methods: We interviewed thirteen patients with electroencephalographically confirmed epilepsy about their subjective experience of having a seizure and used conversational analysis (CA) to analyze the language they used to describe this experience.

Results: Seven patients had focal to bilateral tonic-clonic seizures (FBTCS), 7 had focal impaired awareness seizures (FIAS), 1 had focal aware seizures (FAS), and one had generalized onset tonic-clonic (GTC) seizures.

Health and Economic Impact of Routine Pediatric Pneumococcal Immunization Programs in Canada: A Retrospective Analysis.

Objective: A model was developed to estimate the historical impact (including total societal health and economic benefit) of pneumococcal conjugate vaccine (PCV) programs in the overall Canadian population between 2005 and 2015, inclusively.

Methods: Historical incidence of invasive pneumococcal disease (IPD), pneumonia, and acute otitis media (AOM) were obtained from epidemiologic databases supplemented with published and unpublished data. Two scenarios were considered: (1) the observed historical incidence from 2005 to 2015 in the setting of PCV use; (2) a hypothetical scenario in which we estimated the number of disease cases assuming no PCV use.

Comment on Gomez et. al. "Response to article by Wasserman et. al. (2018) 'Modelling the sustained use of the 13-valent pneumococcal conjugate vaccine compared to switching to the 10-valent vaccine in Mexico'".

In a recent Letter, Gomez et. al. provided a critique of our original analysis estimating the clinical and economic impact of switching from the 13-valent (PCV13) to the 10-valent (PCV10) pneumococcal conjugate vaccine in Mexico.

Modeling the sustained use of the 13-valent pneumococcal conjugate vaccine compared to switching to the 10-valent vaccine in Mexico.

Introduction: Pneumococcal diseases caused by Streptococcus pneumoniae represent a significant health and economic burden. Mexico has benefited from the inclusion of the 7-valent (PCV7) and 13-valent pneumococcal conjugate vaccines (PCV13) since their inclusion in the National Immunization Program (NIP) in 2006 and 2010, respectively. The objective of this study is to estimate the impact of the existing program and predict future implications of a change in the current program.

Clinical and Economic Impact of a Potential Switch from 13-Valent to 10-Valent Pneumococcal Conjugate Infant Vaccination in Canada.

Introduction: Pneumococcal conjugate vaccines (PCVs) have been available in Canada since 2001, with 13-valent PCV (PCV13) added to the infant routine immunization program throughout all Canadian provinces by 2011. The use of PCVs has dramatically reduced the burden of pneumococcal disease in Canada. As a result, decision-makers may consider switching from a more costly, higher-valent vaccine to a lower-cost, lower-valent vaccine in an attempt to allocate funds for other vaccine programs.

Cost-effectiveness analysis of umeclidinium/vilanterol for the management of patients with moderate to very severe COPD using an economic model.

Background: Bronchodilators such as long-acting muscarinic antagonists (LAMAs) and long-acting β-agonists (LABAs) are central to the pharmacological management of COPD. Dual bronchodilation with umeclidinium/vilanterol (UMEC/VI; 62.5/25 μg) is a novel LAMA/LABA combination approved for maintenance treatment for patients with COPD.

Cost-effectiveness of Maintaining Daily Intake of Oat β-Glucan for Coronary Heart Disease Primary Prevention.

Purpose: Oat β-glucan reduces cholesterol levels and thus reduces the risk for coronary heart disease (CHD). However, its economic impact has not been well studied. We examined the economic impact of daily intake of ≥3 g of oat β-glucan in primary prevention of CHD in patients receiving statins or no pharmacologic treatment.

Economic analysis of empiric versus diagnostic-driven strategies for immunocompromised patients with suspected fungal infections in the People's Republic of China.

Invasive fungal infections (IFIs) require rapid diagnosis and treatment. A decision-analytic model was used to estimate total costs and survival associated with a diagnostic-driven (DD) or an empiric treatment approach in neutropenic patients with hematological malignancies receiving chemotherapy or autologous/allogeneic stem cell transplants in Shanghai, Beijing, Chengdu, and Guangzhou, the People's Republic of China. Treatment initiation for the empiric approach occurred after clinical suspicion of an IFI; treatment initiation for the DD approach occurred after clinical suspicion and a positive IFI diagnostic test result.

Economic Impact of Alvimopan Considering Varying Definitions of Postoperative Ileus.

Background: Prolonged postoperative ileus (POI) is the predominant cause of extended hospitalization after bowel resection surgery. Alvimopan accelerates gastrointestinal recovery, potentially reducing health care costs. We examined the value of alvimopan in reducing prolonged POI and length of stay for patients undergoing abdominal surgery using different definitions of POI.

Unraveling the sources of ground level ozone in the Intermountain Western United States using Pb isotopes.

Ozone as an atmospheric pollutant is largely produced by anthropogenic precursors and can significantly impact human and ecosystem health, and climate. The U.S.

Economic Comparison of an Empirical Versus Diagnostic-Driven Strategy for Treating Invasive Fungal Disease in Immunocompromised Patients.

Purpose: Patients with persistent or recurrent neutropenic fevers at risk of invasive fungal disease (IFD) are treated empirically with antifungal therapy (AFT). Early treatment using a diagnostic-driven (DD) strategy may reduce clinical and economic burdens. We compared costs and outcomes of both strategies from a UK perspective.

Factor VIII alloantibody inhibitors: cost analysis of immune tolerance induction vs. prophylaxis and on-demand with bypass treatment.

Development of inhibitors (alloantibodies to exogenous factor VIII) is the most significant treatment complication in patients with haemophilia A. The only proven way to eradicate inhibitors is through immune tolerance induction (ITI), while bypassing agents are typically employed to treat or prevent bleeds in patients with high titre inhibitors. Costs of these approaches have not been well studied.