Opportunities and Barriers to the Development and Use of Open Source Health Economic Models: A Survey.

Objectives: Health economic (HE) models are routinely used to support health policy and resource allocation decisions but are often considered "black boxes" that may be prone to error and bias. Open source models (OSMs) have been advocated to increase the transparency, credibility, and reuse of HE models. Previous studies have demonstrated interest in OSMs among the health economics and outcomes research community, but the number of OSMs remains low.

Cost-effectiveness of adding Endocuff to standard colonoscopies for interval colorectal cancer screening.

Higher screening colonoscopy adenoma detection rates (ADRs) correlate with reduced risk of interval colorectal cancer (CRC). The Endocuff device has been shown to improve ADRs compared to standard colonoscopy (SC). This cost-effectiveness analysis compared interval CRC screening using Endocuff-assisted colonoscopy (EC) vs SC.

Transparency in Decision Modelling: What, Why, Who and How?

Transparency in decision modelling is an evolving concept. Recently, discussion has moved from reporting standards to open-source implementation of decision analytic models. However, in the debate about the supposed advantages and disadvantages of greater transparency, there is a lack of definition.

Budget Impact of Next-Generation Sequencing for Molecular Assessment of Advanced Non-Small Cell Lung Cancer.

Background: Genetic testing for nonsquamous advanced non-small cell lung cancer (aNSCLC) is recommended to guide first-line therapy. Activating mutations can be identified via single-gene testing or next-generation sequencing (NGS).

Objectives: To evaluate the budget impact of NGS instead of single-gene testing for tissue-based molecular assessment of aNSCLC from the US health care payer perspective.

Budget impact analysis of ocriplasmin for the treatment of symptomatic vitreomacular adhesion in the USA.

Background: Vitreomacular traction (VMT) treatment options include watchful waiting, vitrectomy and intravitreal ocriplasmin injection (Jetrea). This analysis used results from the recently completed OASIS randomized clinical trial to evaluate the 2-year budget impact of ocriplasmin injection availability for treatment of Stage I or II VMT without epiretinal membrane formation in a modeled US health plan.

Materials & Methods: VMT prevalence, treatment patterns and disease resolution rates were from literature, a US retinal-specialist survey and the OASIS trial.

Impact of Definitive Drug-Drug Interaction Testing on Medication Management and Patient Care.

Background And Objective: Aegis Sciences Corporation developed a test (InterACT Rx) that objectively and definitively identifies substances known to interact with drug-drug interaction-prone medications commonly prescribed in the treatment of chronic pain and behavioral health disorders. The objective of this study was to assess the severity of identified drug-drug interactions, the reduction in the frequency and severity of identified drug-drug interactions, and the impact of the test on healthcare utilization.

Methods: Patients with chronic pain, behavioral health disorders, or both who had one or more drug-drug interaction tests and one or more drug-drug interactions identified in the study period were included.

Comparison between Surgical and Endovascular Hemodialysis Arteriovenous Fistula Interventions and Associated Costs.

Purpose: To compare: (i) rate of arteriovenous fistula (AVF) interventions in both incident and prevalent end-stage kidney disease patients; (ii) their associated costs; and (iii) intervention-free survival between patients with surgical hemodialysis arteriovenous fistula (SAVF) versus those with an endovascularly created fistula (endoAVF).

Materials And Methods: Data from the United States Renal Data System (USRDS) were abstracted to determine the rate of AVF interventions performed in the first year and associated costs (based on Medicare payment rates) for SAVFs created from 2011 to 2013 in the incident and prevalent patient cohorts. Comparative data for endoAVF were obtained from the Novel Endovascular Access Trial (NEAT).

Cost impact of monitoring exhaled nitric oxide in asthma management.

Background: Asthma guidelines recommend periodic assessment of impairment and risk factors to prevent exacerbations, which can lead to hospitalization and increased health care utilization and cost. According to recent meta-analysis data, fractional exhaled nitric oxide (FeNO) monitoring is associated with a 40-50% reduction in the risk of exacerbations.

Objective: Cost modeling of these data indicates the potential for significant cost savings with FeNO use.

A Review of the Utility and Cost Effectiveness of Monitoring Fractional Exhaled Nitric Oxide (FeNO) in Asthma Management.

Unlabelled: Asthma is a common chronic respiratory disease affecting nearly 8% of the U.S.

Population: It results in substantially higher direct and indirect costs as well as an increased mortality risk and poorer quality of life, particularly among patients with difficult-to-control asthma.

Association of Inadequately Controlled Disease and Disease Severity With Patient-Reported Disease Burden in Adults With Atopic Dermatitis.

Importance: Real-world data are limited on the patient-reported burden of adult atopic dermatitis (AD).

Objective: To characterize the patient-reported burden of AD with regard to impact of disease severity and inadequate control in adults from clinical settings.

Design, Setting, And Participants: In this cross-sectional study using data from 6 academic medical centers in the United States collected by a self-administered internet-based questionnaire, 1519 adult patients with AD were stratified by AD severity as mild or moderate/severe using the Patient-Oriented Scoring Atopic Dermatitis (PO-SCORAD).

Assessment of the relationship between diabetes treatment intensification and quality measure performance using electronic medical records.

Aims: Assess the relationship between timely treatment intensification and hemoglobin A1C (HbA1C) control quality-of-care performance measures, i.e., HbA1C levels, among patients with uncontrolled type 2 diabetes.

Patient-Reported Outcomes From the United States Clinical Trial for a Hybrid Cochlear Implant.

Objective: To assess patient-reported outcomes (PROs) in individuals with significant residual low-frequency hearing and severe-to-profound high-frequency sensorineural hearing loss (SNHL) who received the hybrid cochlear implant (CI).

Study Design: Prospective, multicenter, nonrandomized, single-arm repeated measures, single-subject design.

Setting: Tertiary centers, ambulatory care.

Comparison of post-creation procedures and costs between surgical and an endovascular approach to arteriovenous fistula creation.

Introduction: Due to early and late failures that may occur with surgically created hemodialysis arteriovenous fistulas (SAVF), post-creation procedures are commonly required to facilitate AVF maturation and maintain patency. This study compared AVF post-creation procedures and their associated costs in patients with SAVF to patients with a new endovascularly created AVF (endoAVF).

Methods: A 5% random sample from Medicare Standard Analytical Files was abstracted to determine post-creation procedures and associated costs for SAVF created from 2011 to 2013.

Assessing the Impact of Medication Adherence on Long-Term Cardiovascular Outcomes.

Background: Although guideline-recommended therapies reduce major adverse cardiovascular events (MACE) in patients after myocardial infarction (MI) or those with atherosclerotic disease (ATH), adherence is poor.

Objectives: The goal of this study was to determine the association between medication adherence levels and long-term MACE in these patients.

Methods: We queried the claims database of a large health insurer for patients hospitalized for MI or with ATH.

Cost-effectiveness of minimally invasive sacroiliac joint fusion.

Background: Sacroiliac joint (SIJ) disorders are common in patients with chronic lower back pain. Minimally invasive surgical options have been shown to be effective for the treatment of chronic SIJ dysfunction.

Objective: To determine the cost-effectiveness of minimally invasive SIJ fusion.

A brief review of recent Charcot-Marie-Tooth research and priorities.

This brief review of current research progress on Charcot-Marie-Tooth (CMT) disease is a summary of discussions initiated at the Hereditary Neuropathy Foundation (HNF) scientific advisory board meeting on November 7, 2014. It covers recent published and unpublished in vitro and in vivo research. We discuss recent promising preclinical work for CMT1A, the development of new biomarkers, the characterization of different animal models, and the analysis of the frequency of gene mutations in patients with CMT.

The role of globalization in drug development and access to orphan drugs: orphan drug legislation in the US/EU and in Latin America.

Compared to a decade ago, nearly three times as many drugs for rare diseases are slated for development. This article addresses the market access issues associated with orphan drug status in Europe and the United States in contrast to the legislation in five Latin American (LA) countries that have made strides in this regard--Mexico, Brazil, Colombia, Chile and Argentina. Based on the success of orphan drug legislation in the EU and US, LA countries should strive to adopt similar strategies with regard to rare diseases and drug development.

Cost Analysis and Clinical Outcomes of Ambulatory Care Monitoring in Medicare Patients: Describing the Diagnostic Odyssey.

The diagnostic sequence and costs for arrhythmia detection utilizing Holter ambulatory ECG monitoring have not been well studied. The objective of the current study was to characterize the number of patients and associated costs incurred in the diagnosis, additional monitoring, clinical events and sequelae after an initial Holter monitor in Medicare patients with arrhythmia-the diagnostic odyssey. We performed a retrospective, longitudinal claims analysis using a 5% random sample of Medicare beneficiaries' claims from the Fee-for-Service (FFS) Standard Analytic Files.

Recommendations to enable drug development for inherited neuropathies: Charcot-Marie-Tooth and Giant Axonal Neuropathy.

Approximately 1 in 2500 Americans suffer from Charcot-Marie-Tooth (CMT) disease. The underlying disease mechanisms are unique in most forms of CMT, with many point mutations on various genes causing a toxic accumulation of misfolded proteins. Symptoms of the disease often present within the first two decades of life, with CMT1A patients having reduced compound muscle and sensory action potentials, slow nerve conduction velocities, sensory loss, progressive distal weakness, foot and hand deformities, decreased reflexes, bilateral foot drop and about 5% become wheelchair bound.

A Retrospective Patient Chart Review and Survey in Patients with Cryopyrin-associated Periodic Syndromes Treated with Anakinra.

Cryopyrin-associated periodic syndromes (CAPS) is a group of rare autoinflammatory diseases. Little is known about the burden of disease, patients' views on treatment, and adverse events (AEs) with current therapy. The main study objective was to quantify the patients' burden of disease in terms of flares and resource use and to characterize patient symptomatology and tolerability of treatment with anakinra.

Comorbid anxiety and neurocognitive dysfunctions in children with ADHD.

Previous research established that children with ADHD and comorbid anxiety have a later age of ADHD onset, show less off-task and hyperactive behavior, and have more school problems than children with ADHD alone. Comorbid anxiety appears to ameliorate behavioral inhibition deficits, worsen working memory problems, and lengthen reaction times in ADHD. This study investigated the effect of comorbid anxiety on a broad range of neurocognitive functions and includes child-, parent- and teacher reports of anxiety.

Computer-assisted school-based asthma management: a pilot study.

Background: The high prevalence of asthma among children continues to be a major public health issue. In particular, low-income African-American and Hispanic children often receive asthma care in the emergency department and lack access to continuity of care.

Objective: The aim of the current study was to test the feasibility of implementing a computerized program for empowering low-income children with asthma to manage their own disease.

A retrospective randomized study of asthma control in the US: results of the CHARIOT study.

Background: The third version of the National Asthma Education and Prevention Program (NAEPP) Expert Panel Report (EPR-3): Guidelines on the Diagnosis and Management of Asthma emphasizes the need to use asthma control rather than patient severity to base adjustments to treatment and ultimately improve patient outcomes. The objectives of the current study were to assess control of patients with moderate-to-severe asthma, examine the natural history of the disease, practice patterns and resource utilization in specialty community practices according to recently reviewed NAEPP guidelines.

Research Design And Methods: This analysis represents a retrospective, multicenter, randomized study of 1009 patient charts in sixty United States allergy and pulmonary medicine community practices.