Management of Vulnerable Patients Hospitalized for COVID-19 With Remdesivir: A Retrospective Comparative Effectiveness Study of Mortality in US Hospitals.

Background: Coronavirus disease 2019 (COVID-19) remains a major public health concern, with continued resurgences of cases and substantial risk of mortality for hospitalized patients. Remdesivir has become standard-of-care for hospitalized COVID-19 patients. Given the continued evolution of the disease, clinical management of COVID-19 relies on evidence from the current endemic period.

Assessing the Combined Public Health Impact of Pharmaceutical Interventions on Pandemic Transmission and Mortality: An Example in SARS CoV-2.

To assess the combined role of anti-viral monoclonal antibodies (mAbs) and vaccines in reducing severe acute respiratory syndrome-coronavirus 2 (SARS-CoV-2) transmission and mortality in the United States, an agent-based model was developed that accounted for social contacts, movement/travel, disease progression, and viral shedding. The model was calibrated to coronavirus disease 2019 (COVID-19) mortality between October 2020 and April 2021 (aggressive pandemic phase), and projected an extended outlook to estimate mortality during a less aggressive phase (April-August 2021). Simulated scenarios evaluated mAbs for averting infections and deaths in addition to vaccines and aggregated non-pharmaceutical interventions.

Remdesivir Treatment in Hospitalized Patients With Coronavirus Disease 2019 (COVID-19): A Comparative Analysis of In-hospital All-cause Mortality in a Large Multicenter Observational Cohort.

Background: Remdesivir (RDV) improved clinical outcomes among hospitalized patients with coronavirus disease 2019 (COVID-19) in randomized trials, but data from clinical practice are limited.

Methods: We examined survival outcomes for US patients hospitalized with COVID-19 between August and November 2020 and treated with RDV within 2 days of hospitalization vs those not receiving RDV during their hospitalization using the Premier Healthcare Database. Preferential within-hospital propensity score matching with replacement was used.

Practice patterns and incidence of adenovirus infection in allogeneic hematopoietic cell transplant recipients: Multicenter survey of transplant centers in the United States.

Background: Adenovirus (AdV) is increasingly recognized as a threat to successful outcomes after allogeneic hematopoietic cell transplantation (allo-HCT). Guidelines have been developed to inform AdV screening and treatment practices, but the extent to which they are followed in clinical practice in the United States is still unknown. The incidence of AdV in the United States is also not well documented.

Treatment Patterns and Health Resource Use Among Patients with Metastatic Gastroenteropancreatic Neuroendocrine Tumors Treated at a Tertiary Referral Center.

Background: Although an increasing number of treatments have become available for patients with advanced gastroenteropancreatic neuroendocrine tumors (GEP-NETs), there remains little consensus on treatment sequence and its impact on health care resource use (HRU). We sought to describe treatment patterns and HRU, in a cohort of patients with metastatic GEP-NETs treated at a tertiary referral center in the U.S.

Moving Towards Accountability for Reasonableness - A Systematic Exploration of the Features of Legitimate Healthcare Coverage Decision-Making Processes Using Rare Diseases and Regenerative Therapies as a Case Study.

Background: The accountability for reasonableness (A4R) framework defines 4 conditions for legitimate healthcare coverage decision processes: Relevance, Publicity, Appeals, and Enforcement. The aim of this study was to reflect on how the diverse features of decision-making processes can be aligned with A4R conditions to guide decision-making towards legitimacy. Rare disease and regenerative therapies (RDRTs) pose special decision-making challenges and offer therefore a useful case study.

Second-line cabozantinib versus nivolumab in advanced renal cell carcinoma: Systematic review and indirect treatment comparison.

Background: Nivolumab and cabozantinib, two new treatment options for previously-treated advanced/metastatic renal cell carcinoma (aRCC), have recently been approved.

Methods: Two independent reviewers performed study selection, data extraction, and risk of bias assessment. Indirect treatment comparisons were carried out by directly assessing HR differences and statistical modeling of Kaplan-Meier curves from these two trials.

Cost-effectiveness comparison of cabozantinib with everolimus, axitinib, and nivolumab in the treatment of advanced renal cell carcinoma following the failure of prior therapy in England.

Purpose: The aim of this study was to compare the cost-effectiveness of cabozantinib with the standard of care in England in adult patients with advanced renal cell carcinoma (aRCC), following prior vascular endothelial growth factor receptor (VEGFR)-targeted therapy.

Methods: We developed a partitioned-survival model with three health states to assess the cost-effectiveness of cabozantinib and its comparators. The model time horizon was 30 years.

Effect of Diabetes Treatment-Related Attributes on Costs to Type 2 Diabetes Patients in a Real-World Population.

Background: Type 2 diabetes mellitus (T2DM) results in a substantial economic burden on patients, health care systems, and society. Most literature assessing the cost of T2DM focuses on the long-term complications of the disease, the association between glucose control and cost, and patient characteristics resulting in poor and costly outcomes. However, it is likely that attributes specific to diabetes therapy can affect the use of costly resources.

An Indirect Comparison of Everolimus Versus Axitinib in US Patients With Advanced Renal Cell Carcinoma in Whom Prior Sunitinib Therapy Failed.

Purpose: The purpose of this study was to perform a weight-adjusted indirect comparison to approximate the relative efficacy of everolimus versus axitinib among patients with second-line metastatic renal cell carcinoma in whom sunitinib therapy previously failed.

Methods: Individual patient data from the RECORD-1 (Renal Cell Cancer Treatment With Oral RAD001 Given Daily) Phase III clinical trial provided information for patients taking everolimus. Summary baseline clinical and demographic characteristics and progression-free survival (PFS) outcomes were available for patients taking axitinib who were included in the AXIS (axitinib versus sorafenib) Phase III clinical trial.

Lifetime cost of everolimus vs axitinib in patients with advanced renal cell carcinoma who failed prior sunitinib therapy in the US.

Objective: Everolimus and axitinib are approved in the US to treat patients with advanced renal cell carcinoma (RCC) after failure on sunitinib or sorafenib, and one prior systemic therapy (e.g., sunitinib), respectively.

United States-based practice patterns and resource utilization in advanced neuroendocrine tumor treatment.

Aim: To assess advanced neuroendocrine tumor (NET) treatment patterns and resource utilization by tumor progression stage and tumor site in the United States.

Methods: United States Physicians meeting eligibility criteria were provided with online data extraction forms to collect patient chart data on recent NET patients. Resource utilization and treatment pattern data were collected over a baseline period (after diagnosis and before tumor progression), as well as initial and secondary progression periods, with progression defined according to measureable radiographic evidence of tumor progression.

International practice patterns and resource utilization in the treatment of neuroendocrine tumors.

Objectives: This study compared resource use and practice patterns in patients with advanced neuroendocrine tumors (NETs) on disease progression, across countries, and by tumor type.

Methods: Physicians in the United States, United Kingdom, Germany, France, Brazil, and Italy completed data extraction forms to extract chart data of patients with NET relating to health care resource utilization and treatment practice. Data were assessed in a cross-sectional manner, by country, and by NET subtype.

Cost-effectiveness of everolimus vs sunitinib in treating patients with advanced, progressive pancreatic neuroendocrine tumors in the United States.

Background: Everolimus (Afinitor) and sunitinib (Sutent) were recently approved to treat patients with advanced, progressive pancreatic neuroendocrine tumors (pNETs). (Afinitor is a registered trademark of Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA; Sutent is a registered trademark of Pfizer Inc., New York, NY, USA.

Economic evaluation of everolimus versus sorafenib for the treatment of metastatic renal cell carcinoma after failure of first-line sunitinib.

Background: A recent indirect comparison study showed that sunitinib-refractory metastatic renal cell carcinoma (mRCC) patients treated with everolimus are expected to have improved overall survival outcomes compared to patients treated with sorafenib. This analysis examines the likely cost-effectiveness of everolimus versus sorafenib in this setting from a US payer perspective.

Methods: A Markov model was developed to simulate a cohort of sunitinib-refractory mRCC patients and to estimate the cost per incremental life-years gained (LYG) and quality-adjusted life-years (QALYs) gained.

An adjusted indirect comparison of everolimus and sorafenib therapy in sunitinib-refractory metastatic renal cell carcinoma patients using repeated matched samples.

Objective: To date, no trial data exist comparing treatment outcomes for everolimus versus sorafenib. The current analysis indirectly compares the overall survival (OS) benefit of everolimus and sorafenib as second-line treatment options.

Research Design And Methods: A single-arm sorafenib study is selected as a basis to match an everolimus sunitinib-refractory subpopulation of the RECORD-1 trial.

Economic consequences of severe bleeding in patients with acute coronary syndrome in the USA.

Introduction: Previous studies have demonstrated increased costs associated with bleeding in clinical trials, but none have yet examined the association of bleeding with costs/charges in a real-world setting. This study examines the association between health care charges and severe bleeding events among patients with acute coronary syndrome (ACS) in a real-world US setting.

Methods: This retrospective study of ACS patients enrolled in a regional, 570,000-member commercial health plan evaluated resource utilization for patients with and without severe bleeding using medical encounter data in health care administrative records.

The effectiveness of adjustable gastric banding: a retrospective 6-year U.S. follow-up study.

Background: This study aimed to assess the efficacy of laparoscopic adjustable gastric banding (LAGB) during a 6-year follow-up period.

Methods: A retrospective database analysis of patients who underwent LAGB at New York University Medical Center between 1 January 2000 and 29 February 2008 was conducted. Patients were included for the efficacy analysis if they were 18 years old or older at the surgery date and had one or more visits with a recorded weight after surgery.

Economic consequences of ACS-related rehospitalizations in the US.

Objective: To examine economic consequences related to rehospitalization following initial acute coronary syndrome (ACS) treatment in United States managed care settings.

Study Design: Retrospective observational studies.

Research Design And Methods: Retrospective observational studies were conducted on two managed care populations to examine medical encounter insurance claims and charges for ACS-related rehospitalizations following an index hospitalization for new onset ACS (2002-2007).

Clinical impact of early clopidogrel discontinuation following acute myocardial infarction hospitalization or stent implantation: analysis in a nationally representative managed-care population.

Objectives: To evaluate the association between discontinuation of clopidogrel therapy and risk of acute myocardial infarction (AMI) hospitalization or cardiac revascularization in a nationally-representative patient population following hospitalization for an AMI or coronary stent insertion.

Research Design And Methods: This observational cohort study was performed using data on patients from the PharMetrics Anonymous Patient-Centric Database who were hospitalized for an AMI or coronary stent insertion and subsequently treated with clopidogrel. Cox proportional hazard modeling was used to evaluate the association between clopidogrel discontinuation prior to 1 year post-initial AMI hospitalization and the primary endpoint of repeat AMI hospitalization or coronary intervention defined as percutaneous coronary intervention (PCI) with or without stent, or coronary artery bypass graft (CABG).

Clinical impact of early clopidogrel discontinuation following acute myocardial infarction hospitalization or stent implantation: analysis in a single integrated health network.

Objective: To determine the association between the discontinuation of clopidogrel therapy prior to 1 year and the risk of acute myocardial infarction (AMI) hospitalization, coronary intervention or all-cause mortality in a cohort of managed-care patients following AMI hospitalization or stent insertion.

Research Design And Methods: This observational cohort study included 1152 patients enrolled in the Health Alliance Plan who were hospitalized for AMI, or who underwent coronary stent placement. Clopidogrel use was assessed using pharmacy claims data.

The impact of indirect (herd) protection on the cost-effectiveness of pneumococcal conjugate vaccine.

Background: The heptavalent pneumococcal polysaccharide-protein conjugate vaccine (PCV7) confers protection against invasive pneumococcal disease (IPD) caused by serotypes that are responsible for substantial morbidity and mortality throughout the world. In 2000, the 7 serotypes covered by PCV7 accounted for 80% to 90% of serotypes isolated from the blood or cerebrospinal fluid of children aged <6 years in the United States. A previous review of the literature spanning the years 1998 to 2005 pertaining to the incidence of IPD among nonimmunized individuals in countries with universal PCV7 immunization suggested an indirect protective effect (herd protection, or community immunity) after widespread vaccination.