Mortality among US veterans with a physician-documented diagnosis of pyruvate kinase deficiency.

Real-world studies of pyruvate kinase (PK) deficiency and estimates of mortality are lacking. This retrospective observational study aimed to identify patients with PK deficiency and compare their overall survival (OS) to that of a matched cohort without PK deficiency. Patients with ≥1 diagnosis code related to PK deficiency were selected from the US Veterans Health Administration (VHA) database (01/1995-07/2019); patients with a physician-documented diagnosis were included (PK deficiency cohort; index: date of first diagnosis code related to PK deficiency).

Burden of illness for patients with cholangiocarcinoma in the United States: a retrospective claims analysis.

Background: Advanced cholangiocarcinoma (CCA) is associated with considerable morbidity and mortality. Novel second-line treatments for advanced CCA underscore the need to understand treatment patterns and economic burden of illness in clinical practice.

Methods: This retrospective, claims-based study using Optum's de-identified Clinformatics Data Mart Database [2007-2019] selected patients with CCA who experienced failure of a line of therapy containing either gemcitabine or fluorouracil.

The Economic Value of Improved Productivity from Treatment of Chronic Hepatitis C Virus Infection: A Retrospective Analysis of Earnings, Work Loss, and Health Insurance Data.

Introduction: Patients with chronic hepatitis C virus infection (HCV) may incur significant indirect costs due to health-related work loss. However, the impact of curative HCV therapy on work productivity is not well characterized. We estimated the economic value of improved productivity following HCV treatment.

Economic Outcomes of Inflammatory Bowel Disease Patients Switching to a Second Anti-Tumor Necrosis Factor or Vedolizumab.

Background: Anti-tumor necrosis factor (TNF) therapies have been the mainstay of inflammatory bowel disease (IBD) treatment for nearly 2 decades. Therapies with novel mechanisms of action have been recently developed. This study compared healthcare resource utilization (HRU) and costs incurred while switching from an initial anti-TNF to another anti-TNF versus switching to vedolizumab.

Economic Burden of Illness Among Commercially Insured Patients with Systemic Sclerosis with Interstitial Lung Disease in the USA: A Claims Data Analysis.

Introduction: Interstitial lung disease (ILD) is a common manifestation of scleroderma/systemic sclerosis (SSc). However, the direct and indirect economic burdens of SSc-ILD remain unclear. This study assessed and compared healthcare resource utilization (HRU), direct healthcare costs, work loss, and indirect costs between patients with SSc-ILD and matched controls with neither SSc nor ILD in the USA.

Clinical and Economic Burden of Patients with Chronic Hepatitis C with Versus Without Antiviral Treatment in Japan: An Observational Cohort Study Using Hospital Claims Data.

Introduction: Japan has one of the highest prevalence rate of chronic hepatitis C (CHC) in the industrialized world. However, the burden of CHC treatment is poorly understood. Thus, the healthcare resource utilization and costs of treated versus untreated patients, and patients with early versus delayed treatment initiation, were assessed in Japan.

Economic Burden among Commercially Insured Patients with Systemic Sclerosis in the United States.

Objective: To quantify healthcare resource utilization (HRU), work loss, and annual direct and indirect healthcare costs among patients with systemic sclerosis (SSc) compared to matched controls in the United States.

Methods: Data were obtained from a large US commercial claims database. Patients were ≥ 18 years old at the index date (first SSc diagnosis) and had ≥ 1 SSc diagnosis in the inpatient (IP) or emergency room (ER) setting, or ≥ 2 SSc diagnoses on 2 different dates in the outpatient (OP) setting between January 1, 2005, and March 31, 2015; continuous enrollment was required during the followup period (12 months after the index date).

The long-term direct and indirect economic burden among Parkinson's disease caregivers in the United States.

Background: Parkinson's disease is a progressive, disabling neurodegenerative disorder associated with significant economic burden for patients and caregivers. The objective of this study was to compare the direct and indirect economic burden of Parkinson's patients' caregivers with demographically matched controls in the United States, in the 5 years after first diagnosis of Parkinson's disease.

Methods: Policyholders (18-64 years old) linked to a Parkinson's disease patient (≥2 diagnoses of Parkinson's disease; first diagnosis is the index date) from January 1, 1998 to March 31, 2014, were selected from a private-insurer claims database and categorized as Parkinson's caregivers.

Incidence of catheter-related complications among Japanese patients with central venous catheters as well as patients with short bowel syndrome.

Purpose: Short bowel syndrome (SBS) is a serious rare disorder that is usually managed with parenteral nutrition. Central venous catheter (CVC)-related complications are known to occur, but their incidence rates (IRs) in Japan are unknown. The aim of this study was to estimate the incidence of complications in Japanese patients with CVCs, including patients with SBS using CVCs.

Short-term costs associated with non-medical switching in autoimmune conditions.

Objectives: To estimate short-term costs associated with non-medical switch (NMS) from originator biologics to biosimilars among stable patients with autoimmune conditions in rheumatology, gastroenterology, and dermatology from a US provider's and third-party payer's perspective.

Methods: An economic model was constructed to estimate switching costs related to physician time and healthcare resource utilisation (HRU) at the initial NMS visit and over 3 months. The proportion of patients with relevant conditions treated with originators and expected NMS rate, physician time, HRU, and payer reimbursement were derived from a physician survey.

Economic Burden of Patients with Inadequate Response to Targeted Immunomodulators for Rheumatoid Arthritis.

Background: Targeted immunomodulators (TIMs), including biologic disease-modifying antirheumatic drugs (DMARDs) and JAK/STAT inhibitors, are effective therapies for rheumatoid arthritis (RA), but some patients fail to respond or lose response over time. This study estimated the real-world prevalence of RA patients with inadequate responses to an initial TIM (nonresponders) in the United States and assessed their direct and indirect economic burden compared with treatment responders.

Methods: Administrative claims data (January 1999-March 2014) from a large private-insurer database were used, which included work-loss data from a subset of reporting companies.

Healthcare resource utilization and costs by age and joint location among osteoarthritis patients in a privately insured population.

Aims: To compare healthcare resource utilization and costs between patients aged 18-64 years with osteoarthritis (OA) and matched controls without OA in a privately insured population.

Methods: Patients with OA were selected from de-identified US-based employer claims (Q1:1999-Q3:2011). The index date was defined as the first OA diagnosis indicated by ICD-9-CM codes.

Inpatient Admissions and Costs Associated with Persistent Use of Dalfampridine Extended-Release in Multiple Sclerosis: A Claims Database Analysis.

Background: While the clinical benefits of dalfampridine extended-release (D-ER) have been established in patients with multiple sclerosis (MS) through multiple clinical trials, there is limited real-world data on D-ER use, in particular the persistent use of D-ER, and associated acute care resource utilization and costs.

Objective: To examine the real-world association of D-ER use and inpatient admissions and costs among patients with MS.

Methods: This study was a retrospective observational claims analysis of the MarketScan database (April 2009-March 2014).

Prevalence of Noninfectious Uveitis in the United States: A Claims-Based Analysis.

Importance: Noninfectious uveitis (NIU) is a collection of intraocular inflammatory disorders that may be associated with significant visual impairment. To our knowledge, few studies have investigated NIU prevalence overall or stratified by inflammation location, severity, presence of systemic conditions, age, or sex.

Objective: To estimate NIU prevalence using a large, retrospective, administrative claims database.

Direct Costs in Patients with Celiac Disease in the USA: A Retrospective Claims Analysis.

Background: Celiac disease (CeD) is an autoimmune disease triggered by gluten ingestion.

Aim: We assessed total direct costs burden associated with CeD in patients with CeD versus patients without CeD using administrative claims data.

Methods: Patients with CeD (cases) with ≥1 occurrences of CeD diagnosis were selected at a randomly chosen date (index date) from the OptumHealth Reporting and Insights database from 01/01/1998 through 03/31/2013.

Economic impact of expanded use of biologic therapy for the treatment of rheumatoid arthritis and Crohn's disease in Argentina, Brazil, Colombia, and Mexico.

Objectives: To estimate economic impact resulting from increased biologics use for treatment of rheumatoid arthritis (RA) and Crohn's disease (CD) in Argentina, Brazil, Colombia, and Mexico.

Methods: The influence of increasing biologics use for treatment of RA during 2012-2022 and for treatment of CD during 2013-2023 was modeled from a societal perspective. The economic model incorporated current and projected medical, indirect, and drug costs and epidemiologic and economic factors.

Comparison of real-world outcomes of adalimumab and infliximab for patients with ulcerative colitis in the United States.

Objective: We compared the real-world effectiveness of initiating adalimumab and infliximab among patients in the US who were naïve to tumor necrosis factor (TNF) inhibitors.

Methods: A retrospective chart review was conducted to evaluate the real-world effectiveness among adults with ulcerative colitis (UC) initiating adalimumab or infliximab. Charts of patients with UC were abstracted by treating physicians (randomly selected from a nationally representative panel) in April 2014.

Direct and indirect resource use, healthcare costs and work force absence in patients with non-infectious intermediate, posterior or panuveitis.

Purpose: To ascertain resource use, costs and risk of workforce absence in non-infectious uveitis cases versus matched controls.

Methods: In a retrospective claims analysis of employees in the United States, prevalent (N = 705) and incident (N = 776) cases 18-64 years old with ≥2 diagnoses of non-infectious intermediate, posterior or panuveitis were matched 1:1 to controls without uveitis. Persistent prevalent cases (treated for ≥90 days, N = 112) also were analysed.

Change in forced vital capacity and associated subsequent outcomes in patients with newly diagnosed idiopathic pulmonary fibrosis.

Background: Idiopathic pulmonary fibrosis (IPF) is a rare and serious disease characterized by progressive lung-function loss. Limited evidence has been published on the impact of lung-function loss on subsequent patient outcomes. This study examined change in forced vital capacity (FVC) across IPF patients in the 6 months after diagnosis and its association with clinical and healthcare resource utilization (HRU) outcomes in a real-world setting in the U.

Association of early suspected acute exacerbations of idiopathic pulmonary fibrosis with subsequent clinical outcomes and healthcare resource utilization.

Background: Idiopathic pulmonary fibrosis (IPF) may be complicated by episodes of acute exacerbation. This study quantified the association between occurrence of suspected acute exacerbations of IPF (AEx-IPF) in the 6 months post-IPF diagnosis with clinical outcomes and IPF-related healthcare resource utilization (HRU).

Methods: U.

Impact of a Telehealth and Care Management Program on All-Cause Mortality and Healthcare Utilization in Patients with Heart Failure.

Background: Telehealth has the potential to improve chronic disease management and outcomes, but data regarding direct benefit of telehealth in patients with heart failure (HF) have been mixed. The objective of this study was to determine whether the Health Buddy Program (HBP) (Bosch Healthcare, Palo Alto, CA), a content-driven telehealth system coupled with care management, is associated with improved outcomes in Medicare beneficiaries with HF.

Materials And Methods: This was a retrospective cohort study of 623 Medicare beneficiaries with HF offered HBP enrollment compared with a propensity score-matched control group of Medicare beneficiaries with HF from the Medicare 5% sample.

Costs and Resource Utilization Associated With Anemia and Rash in Chronic Hepatitis C Patients Treated With Direct-Acting Antiviral Agents in the United States.

Purpose: The addition of 2 direct-acting antiviral (DAA) agents, telaprevir and boceprevir, to peginterferon and ribavirin therapy significantly improves sustained virologic response rates in patients treated for chronic hepatitis C virus (CHC) but is associated with a higher risk of adverse events (AEs), including anemia and rash. Using a large administrative claims database, this study compared the health care resource utilization and costs among CHC patients who developed anemia and/or rash while receiving DAA-based therapies (telaprevir and boceprevir) versus those who did not develop anemia or rash. Adjusted costs were compared by using regression analysis.

Treatment patterns, health care resource utilization, and costs in U.S. patients diagnosed with chronic hepatitis C infection who received telaprevir or boceprevir.

Background: Chronic hepatitis C (CHC) is associated with substantial morbidity and mortality, with the future burden of disease predicted to significantly increase. The recent addition of 2 direct-acting antiviral (DAA) protease inhibitors, telaprevir and boceprevir, to peginterferon alfa (PEG) and ribavirin (RBV) therapy has been shown to significantly improve sustained virologic response rates and thus has become standard of care. While the efficacy and safety of DAAs has been assessed in the clinical trial setting, less is known about real-world use of these new therapies.

Nomenclature and traceability debate for biosimilars: small-molecule surrogates lend support for distinguishable nonproprietary names.

Introduction: The purpose of the present study was to investigate the traceability of adverse events (AEs) for branded and generic drugs with identical nonproprietary names and to consider potential implications for the traceability of AEs for branded and biosimilar biologics.

Methods: Adverse event reports in the Food and Drug Administration AE Reporting System (FAERS) were compared with those in a commercial insurance claims database (Truven Health MarketScan(®)) for 2 drugs (levetiracetam and enoxaparin sodium) with manufacturing or prescribing considerations potentially analogous to those of some biosimilars. Monthly rates of branded- and generic-attributed AEs were estimated pre- and post-generic entry.