First-line cyclin-dependent kinase 4 and 6 inhibitors in combination with an aromatase inhibitor for HR+/HER2- metastatic breast cancer: A real-world cost-effectiveness assessment in a US Medicare-eligible population.

Background: In hormone receptor-positive, human epidermal growth factor 2-negative (HR+/HER2-) metastatic breast cancer (mBC), cyclin-dependent kinase 4 and 6 inhibitors (CDK4/6is) in combination with an aromatase inhibitor (AI) are the preferred first-line (1L) treatment. Although prior cost-effectiveness models comparing CDK4/6is palbociclib, ribociclib, and abemaciclib have used data from placebo-controlled clinical trials, no analyses in the United States have been conducted using real-world evidence (RWE) for a US Medicare-eligible population.

Objective: To estimate the long-term clinical outcomes and health care costs of 1L CDK4/6i treatment in patients aged 65 years and older using RWE.

Comprehensive Access to Semaglutide: Clinical and Economic Implications for Medicare.

Objectives: The objective of this study was to estimate comprehensive value of semaglutide in the Medicare population for current and future FDA-approved indications.

Methods: We used data from government reports, published clinical studies, and real-world claims to model clinical outcomes and costs in patients with diabetes, overweight/obesity, and metabolic-associated steatohepatitis over a 10-year period (2026 to 2035). We estimated the financial impact to the Medicare program by comparing the impact of costs of averted clinical events and anticipated treatment costs under various treatment utilization and price assumptions with a scenario in which no patients were treated with semaglutide.

Estimated True Out-of-Pocket Cost Changes From the Inflation Reduction Act on Medicare Part D Beneficiaries With Cancer.

Objectives: To estimate changes in true out-of-pocket (TrOOP) spend from implementation of a $2000 cap for outpatient prescriptions as authorized by the 2022 Inflation Reduction Act (IRA) among Medicare Part D beneficiaries who received a diagnosis of cancer.

Methods: Medicare beneficiaries who received a diagnosis of cancer and at least one Part D claim for a prescription drug to treat cancer in 2021 were identified from a 5% random sample of beneficiaries. Part D drug expenditures were extracted and adjusted to 2025 expenditures using previously published methods.

Referencing Drug Prices of Other Countries May Not Sustainably Lower Prices in the United States: Lessons From Europe.

The Trump Administration issued an Executive Order on May 12, 2025, with the intent to "equalize pricing across developed countries" through a "Most-Favored Nation" approach with international reference pricing (IRP). IRP has been used extensively in Europe and resulted in short-term budget savings. However, list prices quickly converged to those of major high-income markets, such as Germany and France, without evidence of reduced prices.

The consequences of pharmaceutical tariffs in the United States.

The Trump Administration has threatened to impose tariffs on imported branded, generic, and biosimilar pharmaceutical products. Although specific details regarding the exact rates and implementation timeline remain unclear, the administration has indicated that these tariffs will be substantial. Tariffs can create supply chain disruptions, increase costs, limit patient access to essential medications, and negatively impact research and innovation.

The financial value of improving patient access to COVID-19 antiviral therapy in Medicare Part D: A simulation study.

Background: Nirmatrelvir-ritonavir is an approved treatment for mild to moderate COVID-19 in adults who are at high risk for progression to severe COVID-19, including hospitalization or death. Age is the leading risk factor for severe COVID-19, making treatment access particularly important for the Medicare population. Part D plans must include COVID-19 antivirals on formularies.

The Price Effects of Biosimilars in the United States.

Objectives: This study evaluates the impact of biosimilar competition on the price of reference products and biosimilars in the US pharmaceutical market. We focus on average sales price (ASP) dynamics since 2015 in Medicare Part B because Medicare is a critical payer stakeholder in biosimilar price dynamics.

Methods: Using Centers for Medicare and Medicaid Services data over 34 quarters beginning in 2015, we analyzed the price effects of biosimilar competition in 8 biologic product markets.

Cost-effectiveness of caplacizumab in immune thrombotic thrombocytopenic purpura in the United States.

Background: Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening thrombotic microangiopathy. Caplacizumab is the only treatment approved by the European Medicines Agency and the US Food and Drug Administration for iTTP, to be given in combination with plasma exchange therapy (PEX) and immunosuppression (IS). The National Institute for Health and Care Excellence's independent appraisal committee assessed the cost-effectiveness of caplacizumab and concluded that the addition of caplacizumab to PEX+IS is cost-effective under a patient access scheme in the United Kingdom.

Defining a Framework for Sustainable Global Biosimilars Markets Using Findings from a Targeted Literature Review.

A biosimilar is a biologic medication that is highly similar to and has no clinically meaningful differences from an existing approved biologic referred to as "reference product." From the introduction of the first biosimilar in 2006 to today, a variety of challenges to biosimilar development and uptake have arisen across global markets, threatening sustainability. Consequences of an unsustainable market can include drug shortages, limited competition, and less innovation.

Cost-effectiveness of finerenone therapy for patients with chronic kidney disease and type 2 diabetes in England & Wales: results of the FINE-CKD model.

Objective: Chronic kidney disease (CKD) is the leading cause of kidney failure, end-stage kidney disease (ESKD), and cardiovascular (CV) events in patients with type 2 diabetes (T2D). The FIDELIO-DKD trial demonstrated that finerenone lowered the risk of renal and CV events in patients with CKD and T2D, regardless of cardiovascular disease history. This study evaluated the cost-effectiveness of finerenone added to background treatment (finerenone + BT) versus background treatment (BT) alone in patients with CKD and T2D from the perspective of the National Health Service in England and Wales.

Drugs anticipated to be selected for the Medicare Drug Price Negotiation Program in 2025.

Background: The Centers for Medicare and Medicaid Services (CMS) recently announced the Maximum Fair Price for the first 10 Medicare Part D drugs selected for price negotiation. By February 2025, CMS should announce the list of Part D drugs to be negotiated with implementation of the negotiated prices in 2027.

Objective: To identify up to 15 Medicare Part D single-source drugs anticipated to be selected by CMS for price negotiation in 2025.

A systematic literature review of the clinical and socioeconomic burden of bronchiectasis.

Background: The overall burden of bronchiectasis on patients and healthcare systems has not been comprehensively described. Here, we present the findings of a systematic literature review that assessed the clinical and socioeconomic burden of bronchiectasis with subanalyses by aetiology (PROSPERO registration: CRD42023404162).

Methods: Embase, MEDLINE and the Cochrane Library were searched for publications relating to bronchiectasis disease burden (December 2017-December 2022).

Long-Term Outcomes of Heart Failure With Preserved or Mid-Range Ejection Fraction in the United States.

Background: Approximately one-half of all heart failure (HF) consists of heart failure with preserved ejection fraction (HFpEF) or heart failure with mid-range ejection fraction (HFmrEF). Although several recent trials have investigated treatments for HFpEF/HFmrEF, there is limited insight on the long-term clinical trajectory of this population.

Objectives: The purpose of this study was to model clinical outcomes in patients with symptomatic (NYHA functional class II-IV) HFpEF/HFmrEF over 10 years.

Stakeholder perspectives on the sustainability of the United States biosimilars market.

Biologic therapies play a critical role in modern medical practice but also present challenges for payers, patients, and other stakeholders because of their high cost. Biosimilars can mitigate the cost pressures of reference biologic therapy because they are typically priced at least 25% lower, providing a means to administer cutting-edge biologic therapy while also managing cost of care. In fact, the US health care system has saved an estimated $23.

Cheaper is not always better: Drug shortages in the United States and a value-based solution to alleviate them.

Drug shortages threaten patients' access to medications and are associated with adverse health outcomes and increased costs. Drug shortages disproportionately occur among generic drugs of limited profitability, most notably drugs administered by injection. In this perspective, we discuss how reimbursement and purchasing practices that were meant to create an efficient marketplace for generics have generated strong price pressure that threatens profitability in certain markets.

Price benchmarks of drugs selected for Medicare price negotiation and their therapeutic alternatives.

Background: The Centers for Medicare and Medicaid Services (CMS) are currently negotiating prices with pharmaceutical manufacturers for the first 10 Part D drugs selected for Medicare drug price negotiation. Non-publicly available data, including the net prices of selected drugs and their therapeutic alternatives, will play a central role in the determination of the maximum fair prices (MFPs).

Objective: To estimate price benchmarks involved in the derivation of the starting point of the CMS initial price offer for the 10 drugs selected for Medicare price negotiation.

Projecting long-term clinical outcomes with larotrectinib compared with immune checkpoint inhibitors in metastatic nonsmall cell lung cancer and differentiated thyroid cancer.

Background: Larotrectinib is approved for patients with advanced gene fusion-positive solid tumors. Prior studies demonstrated promising results with larotrectinib compared with other systemic therapy. However, comparisons to checkpoint inhibitors, such as nivolumab or pembrolizumab, have not been done.

Net prices of new antiobesity medications.

Objective: Glucagon-like peptide-1 receptor agonists (GLP1s) are effective antiobesity drugs and the subject of intense debate around insurance coverage due to the large prevalence of obesity and overweight. The estimation of the budget impact associated with GLP1 insurance coverage requires estimates of GLP1 prices that account for manufacturer discounts. The authors applied a peer-reviewed method to estimate the net prices of GLP1s after manufacturer discounts.