Inflation-adjusted analysis of ICER's Unsupported Price Increase reports: contextualizing drug spending changes.

Objective: The Institute for Clinical and Economic Review (ICER) publishes annual Unsupported Price Increase (UPI) reports on prescription drugs, which have gained attention from policymakers and healthcare stakeholders. These reports do not adjust for inflation in their analyses of net price changes. This study aimed to evaluate the economic context of reported drug price increases in ICER's UPI reports by applying inflation adjustments to the estimated impact on healthcare spending.

Subsequent Indications in Oncology Drugs: Pathways, Timelines, and the Inflation Reduction Act.

Introduction: Recent research has raised questions about potential unintended consequences of the Inflation Reduction Act's Drug Price Negotiation Program (DPNP), suggesting that the timelines introduced by the law may reduce manufacturer incentives to invest in post-approval research towards additional indications. Given the role of multiple indications in expanding treatment options in patients with cancer, IRA-related changes to development incentives are especially relevant in oncology. This study aimed to describe heterogeneous drug-level trajectories and timelines of subsequent indications in a cohort of recently approved, multi-indication oncology drugs, including overall, across subgroups of drugs characterized by the timing and pace of additional indications, and by drug type (i.

Will the Institute for Clinical and Economic Review's Shared Savings Approach Decrease Value-Based Prices Most for the Most Severe Diseases?

Objectives: To identify the types of disease most likely to be affected by the Institute for Clinical and Economic Review's (ICER) shared savings assumptions.

Methods: For diseases with treatments that were Food and Drug Administration approved between 2019 and 2023, annual direct and indirect economic burden and characteristics of each disease were extracted from peer-reviewed literature. ICER's shared savings methodology was applied 2 ways: 50/50 shared savings and $150 000 cost-offset cap.

Cost-Effectiveness Analysis of a Prescription Digital Therapeutic in Type 2 Diabetes.

Introduction: BT-001 (AspyreRx™) prescription digital therapy, a form of personalized cognitive behavioral therapy, has demonstrated clinically meaningful and durable hemoglobin A1c reductions in patients with type 2 diabetes (T2D). The current study examined the cost-effectiveness of BT-001 plus standard of care (SoC) versus SoC alone in T2D over a lifetime horizon from a healthcare payer perspective.

Methods: We modeled the T2D pathway using an individual patient-level simulation; clinical data were sourced from the intention-to-treat subset of the BT-001 randomized clinical trial (RCT).

Clinical Events and Renal Function in the First Year Predict Long-Term Kidney Transplant Survival.

Background: Estimated glomerular filtration rate (eGFR) at 1 year post transplantation has been shown to be a strong predictor of long-term graft survival. However, intercurrent events (ICEs) may affect the relationship between eGFR and failure risk.

Methods: The OPTN and USRDS databases on single-organ kidney transplant recipients from 2012 to 2016 were linked.

Costs in the Year Following Deceased Donor Kidney Transplantation: Relationships With Renal Function and Graft Failure.

Relationships between renal function and medical costs for deceased donor kidney transplant recipients are not fully quantified post-transplant. We describe these relationships with renal function measured by estimated glomerular filtration rate (eGFR) and graft failure. The United States Renal Data System identified adults receiving single-organ deceased donor kidneys 2012-2015.

Defining a minimal clinically meaningful difference in 12-month estimated glomerular filtration rate for clinical trials in deceased donor kidney transplantation.

Background: A Minimal Clinically Meaningful Difference (MCMD) has not been defined for Estimated glomerular filtration rate (eGFR). Our goal was to define the MCMD for eGFR anchored to kidney graft failure.

Methods: A systematic review of studies with 12-month eGFR and subsequent renal graft failure was conducted.

Patency of ePTFE Arteriovenous Graft Placements in Hemodialysis Patients: Systematic Literature Review and Meta-Analysis.

Arteriovenous grafts (AVGs) are an appropriate option for vascular access in certain hemodialysis patients. Expanded polytetrafluoroethylene (ePTFE) has become the dominant material for such grafts, due in part to innovations in graft design and surgical interventions to reduce complications and improve patency rates. Comprehensive evidence syntheses have not been conducted to update AVG performance in an era in which both access choice and ePTFE graft functioning may have changed.

Healthcare resource utilization among commercially insured patients with cold agglutinin disease in the United States.

Cold agglutinin disease (CAD) is a rare subtype of autoimmune hemolytic anemia associated with increased thromboembolism risk and early mortality. Healthcare resource utilization (HRU) in CAD has not been reported. We aimed to compare HRU of patients with CAD with a matched non-CAD cohort in the United States.

Toward Patient-Centered Innovation: A Conceptual Framework for Patient-Reported Outcome Measures for Transformative Kidney Replacement Devices.

Individuals with dialysis-dependent kidney failure experience considerable disease- and treatment-related decline in functional status and overall well-being. Despite these experiences, there have been few substantive technological advances in KRT in decades. As such, new federal initiatives seek to accelerate innovation.

Angiotensin II for the treatment of distributive shock in the intensive care unit: A US cost-effectiveness analysis.

Background: Patients with distributive shock who are unresponsive to traditional vasopressors are commonly considered to have severe distributive shock and are at high mortality risk. Here, we assess the cost-effectiveness of adding angiotensin II to the standard of care (SOC) for severe distributive shock in the US critical care setting from a US payer perspective.

Methods: Short-term mortality outcomes were based on 28-day survival rates from the ATHOS-3 study.

Vascular access-specific health-related quality of life impacts among hemodialysis patients: qualitative development of the hemodialysis access-related quality of life (HARQ) instrument.

Background: End stage kidney disease and hemodialysis dependence are associated with impairments in health-related quality of life (HRQOL), which may be related to vascular access (VA). Few HRQOL measures are VA-specific and none differentiate HRQOL impact by VA type. We developed a VA-targeted HRQOL measure to distinguish the impact of fistulas, grafts and catheters.

Estimating the Impact of Novel Digital Therapeutics in Type 2 Diabetes and Hypertension: Health Economic Analysis.

Background: Behavioral interventions can meaningfully improve cardiometabolic conditions. Digital therapeutics (DTxs) delivering these interventions may provide benefits comparable to pharmacologic therapies, displacing medications for some patients.

Objective: Our objective was to estimate the economic impact of a digital behavioral intervention in type 2 diabetes mellitus (T2DM) and hypertension (HTN) and estimate the impact of clinical inertia on deprescribing medications.

Costs Attributable to Arteriovenous Fistula and Arteriovenous Graft Placements in Hemodialysis Patients with Medicare coverage.

Introduction: Hemodialysis (HD) in end-stage renal disease (ESRD) patients requires vascular access (VA) through an arteriovenous fistula (AVF), a prosthetic arteriovenous graft (AVG), or a central venous catheter. While AVF or AVG is commonly used for HD, the economic implications of AVF versus AVG use have not been fully established. We describe the healthcare resource utilization and costs of AVF and AVG use for incident ESRD patients in the United States.

Clinical Outcomes of Arteriovenous Access in Incident Hemodialysis Patients with Medicare Coverage, 2012-2014.

Background: Chronic hemodialysis requires a mode of vascular access through an arteriovenous fistula (AVF), a prosthetic arteriovenous graft (AVG), or a central venous catheter (CVC). AVF is recommended over AVG or CVC due to increased patency and decreased intervention rates for those that mature. AVG are preferred over CVC due to decreased infection and mortality risk.

Patient-level costs of major cardiovascular conditions: a review of the international literature.

Objective: Robust cost estimates of cardiovascular (CV) events are required for assessing health care interventions aimed at reducing the economic burden of major adverse CV events. This review synthesizes international cost estimates of CV events.

Methods: MEDLINE database was searched electronically for English language studies published during 2007-2012, with cost estimates for CV events of interest - unstable angina, myocardial infarction, heart failure, stroke, and CV revascularization.

Systematic Review of Low-Density Lipoprotein Cholesterol Apheresis for the Treatment of Familial Hypercholesterolemia.

Background: Apheresis is an important treatment for reducing low-density lipoprotein cholesterol (LDL-C) in patients with familial hypercholesterolemia (FH). We systematically reviewed the current literature surrounding LDL-C apheresis for FH.

Methods And Results: Electronic databases were searched for publications of LDL-C apheresis in patients with FH.

Cost-effectiveness of hepatitis B vaccination using HEPLISAV™ in selected adult populations compared to Engerix-B® vaccine.

Objective: HEPLISAV™ is an adult hepatitis B vaccine that requires fewer doses over a shorter period of time and elicits higher and earlier seroprotection compared to Engerix-B to reduce the risk of hepatitis B infection. The objective of this analysis was to evaluate the cost-effectiveness of vaccination with HEPLISAV vs. Engerix-B(®) to prevent hepatitis B infection in select populations.

Hemoglobin decline in chemotherapy patients prior to and after policy changes affecting use of erythropoiesis-stimulating agents: 2006-2009.

Objective: Since 2007, the use of erythropoiesis-stimulating agents (ESAs) to treat anemia in cancer patients receiving chemotherapy has been increasingly restricted in the USA. This study assessed hemoglobin (Hb) decline over time among chemotherapy patients.

Methods: Episodes of chemotherapy care were identified in a large US-oncology electronic medical record database; weekly Hb levels were computed in the first 8 weeks.

US Payer Perspectives on Evidence for Formulary Decision Making.

Purpose: The perspective of commercial payers on comparative effectiveness research (CER) has not been well researched. This study aims to describe how US commercial payers use and value CER for formulary decision making in different disease states.

Methods: We recruited 20 medical and pharmaceutical directors from national and regional plans who are involved in pharmaceutical and therapeutics committees to participate in the study.

US payer perspectives on evidence for formulary decision making.

Purpose: The perspective of commercial payers on comparative effectiveness research (CER) has not been well researched. This study aims to describe how US commercial payers use and value CER for formulary decision making in different disease states.

Methods: We recruited 20 medical and pharmaceutical directors from national and regional plans who are involved in pharmaceutical and therapeutics committees to participate in the study.

Use of electronic medical records (EMR) for oncology outcomes research: assessing the comparability of EMR information to patient registry and health claims data.

Unlabelled: Electronic medical records (EMRs) are used increasingly for research in clinical oncology, epidemiology, and comparative effectiveness research (CER).

Objective: To assess the utility of using EMR data in population-based cancer research by comparing a database of EMRs from community oncology clinics against Surveillance Epidemiology and End Results (SEER) cancer registry data and two claims databases (Medicare and commercial claims).

Study Design And Setting: DEMOGRAPHIC, CLINICAL, AND TREATMENT PATTERNS IN THE EMR, SEER, MEDICARE, AND COMMERCIAL CLAIMS DATA WERE COMPARED USING SIX TUMOR SITES: breast, lung/bronchus, head/neck, colorectal, prostate, and non-Hodgkin's lymphoma (NHL).

Effect of reimbursement changes on erythropoiesis-stimulating agent utilization and transfusions.

Cancer patients frequently develop chemotherapy-induced anemia, which can be treated with erythropoiesis-stimulating agents. These agents have shifted the standard of chemotherapy-induced anemia treatment away from the previous mainstay of red blood cell transfusions. In July 2007, the Centers for Medicare and Medicaid Services issued a National Coverage Decision restricting reimbursement for erythropoiesis-stimulating agents to those chemotherapy patients who have hemoglobin levels <10 g/dL at initiation of therapy.