US Perspective Research Articles

Economic evaluation of Erdafitinib as a second-line treatment for advanced metastatic urothelial carcinoma: real-world data from the USA and prospective analysis from China.

Metastatic urothelial carcinoma (mUC) is challenging to treat, with 37% of patients failing first-line therapy. Effective second-line treatments, like Erdafitinib, are crucial. This study evaluates the cost-effectiveness of Erdafitinib as a second-line treatment for mUC from US and Chinese payer perspectives. A Markov model was developed to project costs, life years, and quality-adjusted life years (QALYs) over lifetime. Data were collected from December 2023 to December 2024 for up-to-date estimates and were obtained from literature, health databases, and clinical trials.. The model was run to project long-term outcomes for both the United States and China. In the United States, Erdafitinib provides an additional 0.467 QALYs at a cost of $238,294.2 per QALY, which exceeds the $150,000 per QALY willingness-to-pay threshold. For China, when the cost of Erdafitinib is below $6.9 or $14 per milligram, there is a 90% probability that its incremental cost-effectiveness ratio will be below $38,223 or $84,966 per QALY, respectively. From the perspective of U.S. payers, Erdafitinib as a second-line treatment for mUC is not cost-effective. From the perspective of China, the cost-effectiveness of Erdafitinib is highly sensitive to its price, which could provide a reference for healthcare reimbursement negotiations.

Perception of cyber attacks on automated vehicles and its influence on road sharing and ridership: Insights of the US perspective from a 2021 nationwide survey

Perception of cyber attacks on automated vehicles and its influence on road sharing and ridership: Insights of the US perspective from a 2021 nationwide survey

Cost-effectiveness analysis of pembrolizumab plus chemotherapy as first-line treatment for advanced biliary tract cancer: perspectives from US and Chinese payers

BackgroundThe KEYNOTE-966 study demonstrated that pembrolizumab combined with chemotherapy is more effective than chemotherapy alone as first-line treatment for patients with advanced biliary tract cancer (BTC). However, the cost-effectiveness of...

European strategic autonomy in the transatlantic security context: American perceptions of European security and defence integration 1998–2022

ABSTRACT European strategic autonomy has attracted a great deal of attention in recent years, but the discussion has largely been Euro-centric and overlooked the American perspective. This paper addresses this scholarly gap by reviewing European strategic autonomy from the US perspective. The paper focuses on two groups: political decision-makers (such as presidents, secretaries of state and defence) and foreign policy influencers (people working in think tanks and research institutes). By analysing their speeches, statements, and reports over nearly 25 years (1998–2022), the paper provides a review of the American debate on European strategic autonomy, its complexities, and its evolving nature. The paper concludes that the American debate on European strategic autonomy is more multivocal and complex than is often understood in Europe. The arguments for and against strategic autonomy coexist and are relatively permanent. Especially the debate among decision-makers has shown little fluctuation across the last five administrations, while policy influencers present a more varied discourse, revealing distinct perspectives. The paper demonstrates the nuanced nature of US attitudes towards European security and defence, which decision-makers in Europe should pay close attention to.

A systematic review of the cost-effectiveness of anti-VEGF drugs for the treatment of diabetic retinopathy.

Non-proliferative and proliferative diabetic retinopathy are common complications of diabetes and a major cause of sight loss. Anti-vascular endothelial growth factor drugs represent a treatment option for people with diabetic retinopathy and are routinely used to treat various other eye conditions. However, anti-vascular endothelial growth factor drugs are expensive relative to current care options, and it is unclear whether this additional cost is justified when the immediate risk of vision loss is lower compared to patients with more aggressive ophthalmological conditions. To systematically review the evidence supporting the cost-effectiveness of alternative treatments for diabetic retinopathy. A systematic review of all comparative cost-effectiveness studies evaluating any treatment for diabetic retinopathy was conducted. Bibliographic searches were carried out to identify studies reporting on the cost-effectiveness of treatments for diabetic retinopathy; the latest searches were conducted on 28 April 2023. Included studies were synthesised narratively and evaluated with reference to UK decision-making. Studies were grouped by population into non-proliferative diabetic retinopathy and proliferative diabetic retinopathy. The review identified five studies in the proliferative diabetic retinopathy population, all of which examined the cost-effectiveness of anti-vascular endothelial growth factor treatments compared to pan-retinal photocoagulation. Results of these studies suggest that anti-vascular endothelial growth factor treatments offer some additional benefits in terms of preserved visual acuity but also incur substantial additional costs relative to pan-retinal photocoagulation. Most authors agreed that the additional costs outweigh the limited benefits, especially in certain patient subgroups without pre-existing oedema. As most of the identified evidence considered a US perspective, it is unclear how these results would translate to a UK setting. Two studies were identified in the non-proliferative diabetic retinopathy population. There was limited evidence to support the early use of anti-vascular endothelial growth factor treatment. However, one UK study suggested that early treatment of non-proliferative diabetic retinopathy with pan-retinal photocoagulation is cost-effective compared to delayed pan-retinal photocoagulation. Overall, there is a dearth of cost-effectiveness evidence considering the UK context. The identified studies raised doubts about the cost-effectiveness of anti-vascular endothelial growth factor treatments for proliferative diabetic retinopathy. No conclusions can be made regarding the cost-effectiveness of anti-vascular endothelial growth factor treatments for non-proliferative diabetic retinopathy. Future research should focus on developing rigorous model-based cost-effectiveness analyses integrating all available evidence. This article presents independent research funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme as award number NIHR132948.

The Role of Continuous Glucose Monitoring in People without Diabetes: A US Perspective

The Role of Continuous Glucose Monitoring in People without Diabetes: A US Perspective

Humanity on the final frontier: Challenges in applying international humanitarian law to modern military space operations

Abstract This article provides the personal perspectives of US military operational attorneys and analyzes three significant challenges in applying international humanitarian law (IHL) to modern military space operations: the lack of clear standards for assessing when IHL rules govern particular military activities in outer space; the challenges of effectively distinguishing between civilian objects and military objectives when targeting space systems; and the difficulties of applying IHL rules of proportionality when attacking space systems. To address these challenges, the article argues that States should take steps to develop non-binding norms for military space operations that contribute to broader understanding of States’ views on how IHL applies in space.

Economic Evaluation of Multilayer Silicone-Adhesive Polyurethane Foam Dressing for the Prevention of Pressure Ulcers in at-risk Hospitalized Patients: US and Italian Perspective.

Hospital-acquired pressure ulcers (HAPUs) constitute an important source of concern for healthcare systems due to their negative consequences on patient quality of life and hospital costs. This phenomenon is increasing worldwide, driven by an aging population and increasing prevalence of chronic conditions. This economic evaluation aimed to determine whether using a multilayer, silicone-adhesive polyurethane foam dressing shaped for the sacrum area, alongside standard prevention (SP), is cost-effective in preventing HAPUs for hospitalized patients compared to SP alone. We developed a decision-analytic model to estimate the expected costs and clinical benefits of using the polyurethane foam dressing from Italian and US payor perspectives. Model inputs were taken from published studies, and uncertainty was assessed using one-way and probabilistic sensitivity analyses (PSA). From both US and Italian perspectives, using a foam dressing in addition to SP was found to be cost-saving in all hospital settings. That is, it reduced the incidence of HAPUs at a lower cost overall. The estimated savings were €179 per patient and $305 per patient from Italian and US perspectives. Following sensitivity analysis, the results remained cost-saving, suggesting that our findings are robust. This is the first economic analysis investigating the cost-effectiveness of preventive dressings and standard prevention for avoiding sacral pressure ulcers for at-risk hospitalized patients. This analysis suggests that using a multilayer polyurethane foam dressing to prevent sacral HAPUs in at-risk hospitalized patients is a cost-effective strategy compared with standard prevention alone and, therefore, should be considered as a strategy for PU prevention in hospital settings.

Lifestyle Factors and Stroke Prevention: From the Individual to the Community.

The overwhelming majority of stroke burden can be prevented through the pillars of lifestyle medicine: diet, exercise, sleep, substance abuse, stress management, and healthy relationships. Among these, diet confers the greatest attributable risk. Despite abundant data and integration of lifestyle medicine within major stroke prevention guidelines, several barriers to effective implementation remain. These include lack of emphasis in medical education, integration in hospital certification metrics, reimbursement from medical insurance, and health policy that inadequately addresses social determinants of health. However, both top-down and bottom-up solutions introduced within the last few years are helping to break down these barriers. This review highlights recent literature and interventions that are closing the gap between the theory and practice of stroke prevention through lifestyle risk factors from a US perspective. By strategically targeting the various institutional barriers, it is possible and essential to substantially reduce stroke burden.

Continuous glucose monitoring for self-management of diabetes in people living with type 2 diabetes mellitus on basal insulin therapy: A microsimulation model and cost-effectiveness analysis from a US perspective with relevance to Medicaid.

Reducing the risks of complications is a primary goal of diabetes management, with effective glycemic control a key factor. Glucose monitoring using continuous glucose monitoring (CGM) technology is an important part of diabetes self-management, helping patients reach and maintain targeted glucose and glycated hemoglobin (HbA1c) levels. Although clinical guidelines recommended CGM use, coverage by Medicaid is limited, likely because of cost concerns. To assess the cost-effectiveness of FreeStyle Libre CGM systems, compared with capillary-based self-monitoring of blood glucose (SMBG), in US individuals with type 2 diabetes mellitus using basal insulin. A patient-level microsimulation model was used to compare CGM with SMBG for a population of 10,000 patients. A 10-year horizon was used, with an annual discount rate of 3.0% for costs and utilities. Model population characteristics were based on US national epidemiology data. Patient outcomes were based on published clinical trials and real-world studies. Annual costs, reflective of 2023 values, included CGM and SMBG acquisition costs and the costs of treating diabetic ketoacidosis, severe hypoglycemia, and diabetes complications. The effect of CGM was modeled as a persistent 1.1% reduction in HbA1c relative to SMBG based on US real-world evidence. Disutilities were based on published clinical trials and other relevant literature. The primary outcome was cost per quality-adjusted life-year (QALY) gained. Sensitivity analyses were performed to test the validity of the model results when accounting for a plausible variation of inputs. In the base case analysis, CGM was dominant to SMBG, providing more QALYs (6.18 vs 5.97) at a lower cost ($70,137 vs $71,809) over the 10-year time horizon. A $10,456 increase in glucose monitoring costs was offset by a $12,127 reduction in treatment costs. Cost savings reflected avoidance of acute diabetic events (savings owing to reductions in severe hypoglycemia and diabetic ketoacidosis were $271 and $2,159, respectively) and a reduced cumulative incidence of diabetes complications, particularly renal failure (saving $5,292), myocardial infarction (saving $1,996), and congestive heart failure (saving $1,061). Scenario analyses were consistent with the base case results, and the incremental cost-effectiveness ratio for CGM vs SMBG ranged from dominant to cost-effective. In probabilistic analysis, CGM was 100% likely to be cost-effective at a willingness-to-pay threshold of $50,000/QALY. CGM is cost-effective compared with SMBG for US patients with type 2 diabetes mellitus receiving basal insulin therapy. This suggests that state Medicaid programs could benefit from broader coverage of CGM.

Diversity, equity and inclusiveness in healthcare: A primary care perspective.

Addressing Diversity, Equity and Inclusion in health care is a multidimensional challenge. From a US perspective, the third-party payment system has disempowered and depersonalized health-care delivery. The net result is wasteful and inefficient use of human and financial resources, burnout among providers, as well as care inequities. Financial integration at the point of patient care is essential to aligning the needs of patients with advances in medical technology. Complexity theory offers valuable insights into the roles of government, intermediaries and patients. The government must focus on equity as a rule compiler and referee of the system. Patient and providers who are actively engaged in shared decision-making will naturally address the diverse needs of multitudinous communities. Intermediaries address inclusion by connecting resources with the point of care. In a dynamic, emerging health-care system that serves diverse communities, patient and community-based financing, vouchers and defined contributions are necessary first steps in addressing cultural diversity, inclusion and equity.

†Real-World Effectiveness and Safety of Evinacumab in Patients with Homozygous Familial Hypercholesterolemia: A Multi-site US Perspective

†Real-World Effectiveness and Safety of Evinacumab in Patients with Homozygous Familial Hypercholesterolemia: A Multi-site US Perspective

EE54 A Framework for Evaluating the Economic Impact of Artificial Intelligence for Screening Mammography: Implications for Facilities and Payers from the US Perspective

EE54 A Framework for Evaluating the Economic Impact of Artificial Intelligence for Screening Mammography: Implications for Facilities and Payers from the US Perspective

Comparison of zanubrutinib (zanu) and acalabrutinib (acala) in B-cell malignancies: An adverse event (AE)-based analysis.

e19049 Background: Acala and zanu are 2nd generation Bruton tyrosine kinase inhibitors (BTKis) that have demonstrated improved safety profiles vs 1st generation BTKi ibrutinib in clinical trials. While head-to-head comparison of zanu vs acala is lacking, a recent meta-analysis of clinical trials provided a comprehensive, indirect comparison of the AE profiles in B-cell malignancies (Hwang, Hemasphere. 2023;7(S3):1134). This study aims to evaluate costs and impacts on quality of life (QoL) for zanu vs acala using their AE profiles. Methods: A health economic model was developed to determine cost and QoL associated with AE management for zanu and acala. The incidence rates ( IRs) of all grade and grade ≥3 AEs of interest (n=21, including bleeding events, hypertension, atrial fibrillation, cytopenias, infections, headache, arthralgia, diarrhea) were taken from the meta-analysis. Additional model inputs, such as disutility, average duration of AE, and unit cost of each AE were sourced from published literature and publicly available cost databases in consultation with clinical experts. The model was developed from the US perspective, considering Medicare costs, inflated to 2023 USD. Model outcomes were AE management cost (= IR * unit cost) and quality-adjusted life years (QALYs) lost due to AE (= IR * Disutility * Duration/365.25). Results: In the base-case scenario (considering all AEs), treating a hypothetical cohort of 1000 patients (pts) with zanu instead of acala yielded cost savings of $124K and 3.7 QALY gains (i.e., 3.7 years extra in full health). Subgroup analysis for severe (grade ≥3) and non-severe AEs (grade 1-2) demonstrated similar trends. A sensitivity analysis limited to AEs significantly different between zanu and acala (n=13) yielded consistent results. Additionally, a probabilistic sensitivity analysis, conducted with 1000 iterations to account for uncertainty in model parameters, confirmed robustness of the model, indicating stable conclusions across a wide range of parameter uncertainties. Conclusions: This analysis suggested that zanu is cost-saving and associated with added health benefits compared to acala in terms of AE management in pts with B-cell malignancies. Should these results derived from meta-analysis of clinical trial data be applicable to real-world pt populations across different indications, the potential cost savings could be considerable. These results could be further improved if the differences in efficacy of zanu vs acala are also incorporated. [Table: see text]

EJRR Special Issue: The Future of Food Law after and Beyond the Farm to Fork Strategy

AbstractThis Special Issue stems from some of the insights at the “Future of Food Law” Conference held at Wageningen University and Research (WUR) in 2023, prompting an examination of the Farm to Fork (F2F) Strategy’s achievements and shortcomings. With the von der Leyen Commission’s term concluding, the paper critically assesses key aspects of the F2F Strategy, laying the foundation for an in-depth discussion presented in six contributions. The exploration extends from US and EU perspectives to national considerations, moving even further and beyond the boundaries of the Strategy. It encompasses emerging views on food safety, fostering fair and sustainable agri-food production models, encouraging healthier and democratic food choices, and reevaluating decision-making distribution from EU to Member States in sustainability regulatory actions. This issue aims to probe how agri-food regulatory frameworks should adapt to current challenges, acknowledging new economic, social, and environmental expectations. As a frame to the six contributions, this paper addresses the substantial delay in implementing crucial food law interventions, with a focus on the legislative Framework for Sustainable Food Systems (SFSF). The paper concludes by outlining potential scenarios for the future of EU food law, emphasising the necessity for establishing a guiding principle of sustainability for food systems.

Cost-effectiveness analysis of immune checkpoint inhibitors as first-line therapy in advanced biliary tract cancer.

Aim: To assess the cost-effectiveness of immune checkpoint inhibitors as first-line treatments for advanced biliary tract cancer (BTC).Methods: This pharmacoeconomic evaluation employed the fractional polynomial network meta-analysis and partitioned survival model. Costs and utilities were collected from the literature and databases. Sensitivity analyses were used to examine uncertainties.Results: The incremental cost-effectiveness ratios (ICERs) of first-line treatment strategies were $761,371.37 per quality-adjusted life-year (QALY) or $206,222.53/QALY in the US and $354,678.79 /QALY or $213,874.22/QALY in China, respectively. The sensitivity analysis results were largely consistent with the base case.Conclusion: From the US and Chinese payer perspectives, adding durvalumab or pembrolizumab to chemotherapy is unlikely to be cost effective in the first-line setting for advanced BTC.

Community-acquired pneumonia: a US perspective on the guideline gap.

Community-acquired pneumonia continues to be one of the most common causes of morbidity and mortality due to infectious disease. The aetiologies, clinical presentations, diagnostic modalities and therapeutic options are changing and outpacing the creation of management guidelines. This educational article summarizes a roundtable activity sponsored by an unrestricted educational grant by Paratek that included US experts discussing these changes and identifying gaps in the current guidelines.

A US perspective on closing the carbon cycle to defossilize difficult-to-electrify segments of our economy.

Electrification to reduce or eliminate greenhouse gas emissions is essential to mitigate climate change. However, a substantial portion of our manufacturing and transportation infrastructure will be difficult to electrify and/or will continue to use carbon as a key component, including areas in aviation, heavy-duty and marine transportation, and the chemical industry. In this Roadmap, we explore how multidisciplinary approaches will enable us to close the carbon cycle and create a circular economy by defossilizing these difficult-to-electrify areas and those that will continue to need carbon. We discuss two approaches for this: developing carbon alternatives and improving our ability to reuse carbon, enabled by separations. Furthermore, we posit that co-design and use-driven fundamental science are essential to reach aggressive greenhouse gas reduction targets.

Practical use of DAT SPECT imaging in diagnosing dementia with Lewy bodies: a US perspective of current guidelines and future directions.

Diagnosing Dementia with Lewy Bodies (DLB) remains a challenge in clinical practice. The use of 123I-ioflupane (DaTscan™) SPECT imaging, which detects reduced dopamine transporter (DAT) uptake-a key biomarker in DLB diagnosis-could improve diagnostic accuracy. However, DAT imaging is underutilized despite its potential, contributing to delays and suboptimal patient management. This review evaluates DLB diagnostic practices and challenges faced within the U.S. by synthesizing information from current literature, consensus guidelines, expert opinions, and recent updates on DaTscan FDA filings. It contrasts DAT SPECT with alternative biomarkers, provides recommendations for when DAT SPECT imaging may be indicated and discusses the potential of emerging biomarkers in enhancing diagnostic approaches. The radiopharmaceutical 123I-ioflupane for SPECT imaging was initially approved in Europe (2000) and later in the US (2011) for Parkinsonism/Essential Tremor. Its application was extended in 2022 to include the diagnosis of DLB. DaTscan's diagnostic efficacy for DLB, with its sensitivity, specificity, and predictive values, confirms its clinical utility. However, US implementation faces challenges such as insurance barriers, costs, access issues, and regional availability disparities. 123I-ioflupane SPECT Imaging is indicated for DLB diagnosis and differential diagnosis of Alzheimer's Disease, particularly in uncertain cases. Addressing diagnostic obstacles and enhancing physician-patient education could improve and expedite DLB diagnosis. Collaborative efforts among neurologists, geriatric psychiatrists, psychologists, and memory clinic staff are key to increasing diagnostic accuracy and care in DLB management.

Employer as an AI System Operator and Tortious Liability for Damage Caused by AI Systems: European and US Perspectives

Abstract The article examines if the standard of protecting parties injured by artificial intelligence (AI) systems used by professional operators is high in the European Union (EU) as compared to the USA—that is, whether the liability model of an operator, as applicable in the EU, ensures that injured parties have effective protection. For the purposes of this article, the term ‘effective protection’ should be understood as referring to a liability model guaranteeing to injured parties a real possibility of obtaining due compensation without the burden of proof that a claimant cannot fulfil. The EU legislature has proposed amendments in the area of non-contractual civil liability of AI system users, which also includes professional operators of such systems. An employer using an AI system for professional or commercial purposes and deriving profits from such use qualifies, in a legal sense, as a professional operator of such a system. Therefore, it is worth considering the question whether the EU, compared to the USA, is really in the vanguard when it comes to drafting legislation tailored to the challenges of AI technology and if the currently proposed legislative amendments ensure effective legal protection to persons injured by AI systems used by a professional operator.