US Setting Research Articles

AI‐assisted research collaboration with open data for fair and effective response to call for proposals

AbstractBuilding teams and promoting collaboration are two very common business activities. An example of these are seen in the TeamingForFunding problem, where research institutions and researchers are interested to identify collaborative opportunities when applying to funding agencies in response to latter's calls for proposals. We describe a novel deployed system to recommend teams using a variety of Artificial Intelligence (AI) methods, such that (1) each team achieves the highest possible skill coverage that is demanded by the opportunity, and (2) the workload of distributing the opportunities is balanced among the candidate members. We address these questions by extracting skills latent in open data of proposal calls (demand) and researcher profiles (supply), normalizing them using taxonomies, and creating efficient algorithms that match demand to supply. We create teams to maximize goodness along a novel metric balancing short‐ and long‐term objectives. We evaluate our system in two diverse settings in US and India of researchers and proposal calls, at two different time instants about 1 year apart (total 4 settings), to establish generality of our approach, and deploy it at a major US university. We validate the effectiveness of our algorithms (1) quantitatively, by evaluating the recommended teams using a goodness score and find that more informed methods lead to recommendations of smaller number of teams and higher goodness, and (2) qualitatively, by conducting a large‐scale user study at a college‐wide level, and demonstrate that users overall found the tool very useful and relevant.

Improving Access to Hygiene, Sanitation, and Drinking Water in King County and Beyond: Success Factors and Costs

Context: Most major urban areas in the US, including Seattle and King County, have a long-standing lack of public restrooms, handwashing stations, and drinking water, presenting public health risks. Objective: To aid decision-makers in expanding access, we review available information regarding successful hygiene programs in urban settings to identify shared characteristics and costs. Design: We reviewed 10 journal articles, 49 news articles, and 54 pieces of gray literature including reports, white papers, and online resources describing real-world hygiene, sanitation, and drinking water programs in US and global urban settings. We selected programs in 8 cities and applied a thematic analysis to identify shared success factors. We also summarized costs where available. Settings: Calgary (Canada), Denver (Colorado), London (United Kingdom), Los Angeles (California), Portland (Oregon), San Francisco (California), Seattle (Washington), and Vancouver (Canada). Results: Successful programs usually provide frequent cleaning and maintenance, are designed and operated to discourage crime and misuse, leverage existing infrastructure, and include mobile solutions. Cities can expect an initial cost of at least $133 000 per toilet and annual operating costs of at least $100 000 per toilet. Conclusions: By employing proven solutions and bringing them to scale over time, cities can promote health while improving quality of life and facilitating movement through public spaces for all. Costs should be understood in the context of expenses such as sidewalk cleaning and human waste removal that are necessitated by a lack of restrooms.

Cost-Effectiveness of Fruquintinib for Refractory Metastatic Colorectal Cancer in the USA.

The FRESCO-2 trial established the efficacy and safety of fruquintinib in patients with refractory metastatic colorectal cancer. However, its cost-effectiveness in the US context is not well documented. This study evaluates the cost-effectiveness of fruquintinib versus placebo for this patient population from the perspective of US payers. We developed a partitioned survival model on the basis of patient-level data reconstructed from the survival curves of the FRESCO-2 trial. Parametric estimation was conducted to estimate long-term clinical outcomes and medical costs over a lifetime horizon. Cost inputs and utilities were sourced from public data and previous literature. We used a discount rate of 3.0% per year for both clinical outcomes and costs. We adopted an incremental cost-effectiveness ratio (ICER) threshold of US$100,000 per quality-adjusted life-year (QALY) gained. We performed sensitivity and scenario analyses to examine the robustness of cost-effectiveness results. Fruquintinib treatment resulted in incremental gains of 0.108 life years (LYs) and 0.073 QALYs compared with the placebo, at an additional cost of US$112,294, primarily driven by medication expenses. The ICER for fruquintinib versus placebo was calculated at US$1,037,855 per LY and US$1,546,619 per QALY gained, exceeding the predefined cost-effectiveness threshold. The cost-effectiveness results were robust across all sensitivity and scenario analyses. Despite the survival benefit, fruquintinib was not cost-effective compared with the placebo in patients with refractory metastatic colorectal cancer in the US setting, on the basis of the conventional willingness-to-pay threshold. Our findings may provide a basis for informing the pricing and reimbursement decisions regarding fruquintinib.

IPECAD Modeling Workshop 2023 Cross-Comparison Challenge on Cost-Effectiveness Models in Alzheimer’s Disease

ObjectivesDecision-analytic models assessing the value of emerging Alzheimer’s disease (AD) treatments are challenged by limited evidence on short-term trial outcomes and uncertainty in extrapolating long-term patient-relevant outcomes. To improve understanding and foster transparency and credibility in modeling methods, we cross-compared AD decision models in a hypothetical context of disease-modifying treatment for mild cognitive impairment (MCI) due to AD. MethodsA benchmark scenario (US setting) was used with target population MCI due to AD and a set of synthetically generated hypothetical trial efficacy estimates. Treatment costs were excluded. Model predictions (10-year horizon) were assessed and discussed during a 2-day workshop. ResultsNine modeling groups provided model predictions. Implementation of treatment effectiveness varied across models based on trial efficacy outcome selection (clinical dementia rating – sum of boxes, clinical dementia rating – global, mini-mental state examination, functional activities questionnaire) and analysis method (observed severity transitions, change from baseline, progression hazard ratio, or calibration to these). Predicted mean time in MCI ranged from 2.6 to 5.2 years for control strategy and from 0.1 to 1.0 years for difference between intervention and control strategies. Predicted quality-adjusted life-year gains ranged from 0.0 to 0.6 and incremental costs (excluding treatment costs) from −US$66 897 to US$11 896. ConclusionsTrial data can be implemented in different ways across health-economic models leading to large variation in model predictions. We recommend (1) addressing the choice of outcome measure and treatment effectiveness assumptions in sensitivity analysis, (2) a standardized reporting table for model predictions, and (3) exploring the use of registries for future AD treatments measuring long-term disease progression to reduce uncertainty of extrapolating short-term trial results by health-economic models.

Caregivers of children with asthma alarmed by climate change: a cross-sectional study.

Climate change poses significant health risks, with children being particularly vulnerable to its adverse health effects. Children with asthma are expected to have worsening disease due to increased exposure to heat, air pollution, mold from flooding, and pollen. Understanding caregiver perspectives on these health harms is crucial for informing public health policy and education. Therefore, we aimed to explore caregiver perceptions of climate change-related health risks to children with asthma. In this cross-sectional study, a survey instrument was created and distributed to caregivers of children with asthma during their visits to pulmonology clinics located in an urban northeastern US setting and via email. Among 198 completed surveys, 78% of participants reported high levels of concern about climate change, with most respondents agreeing that climate change has already impacted their child's health. Examples provided by respondents included worsening asthma control due to air pollution, wildfire events, pollen exposure, and rapid changes in weather. Respondents who self-identified as female had greater concern. Most respondents agreed that these topics should be further discussed with their child's doctor. Although, barriers to such discussions were noted by the respondents. Caregivers of children with asthma have high levels of concern regarding climate change and report adverse impacts on their child's asthma. Clinicians caring for children with asthma should consider discussing the respiratory health impacts of climate change with caregivers. However, barriers to these discussions need further examination.

Economic Evaluation of a Novel Lung Cancer Diagnostic in a Population of Patients with a Positive Low-Dose Computed Tomography Result

Background: Early detection of lung cancer is crucial for improving patient outcomes. Although advances in diagnostic technologies have significantly enhanced the ability to identify lung cancer in earlier stages, there are still limitations. The alarming rate of false positives has resulted in unnecessary utilization of medical resources and increased risk of adverse events from invasive procedures. Consequently, there is a critical need for advanced diagnostics after an initial low-dose computed tomography (LDCT) scan. Objectives: This study evaluated the potential cost savings for US payers of CyPath® Lung, a novel diagnostic tool utilizing flow cytometry and machine learning for the early detection of lung cancer, in patients with positive LDCT scans with indeterminate pulmonary nodules (IPNs) ranging from 6 to 29 mm. Methods: A cost offset model was developed to evaluate the net expected savings associated with the use of CyPath® Lung relative to the current standard of care for individuals whose IPNs range from 6 to 29 mm. Perspectives from both Medicare and private payers in a US setting are included, with a 1-year time horizon. Cost calculations included procedure expenses, complication costs, and diagnostic assessment costs per patient. Primary outcomes of this analysis include cost savings per cohort and cost savings per patient. Results: Our analysis showed positive cost savings from a private payer’s perspective, with expected savings of $895 202 311 per cohort and $6460 per patient, across all patients. Scenario analysis resulted in cost savings of $890 829 889 per cohort, and $6429 per patient. Similarly, savings of $378 689 020 per cohort or $2733 per patient were yielded for Medicare payers, across all patients. In addition, scenario analysis accounting for false negative patients from a Medicare payer perspective yielded savings of $376 902 203 per cohort and $2720 per patient. Discussion: The results suggest substantial cost savings, primarily due to reductions in follow-up diagnostic assessments and procedures, and highlight the importance of accurate diagnostic tools in reducing unnecessary healthcare expenditures. Conclusion: CyPath® Lung utilization yields savings for private and Medicare payers relative to the current standard of care in a US setting for individuals with 6 to 20 mm IPNs.

Heterogeneity in the integration of ESG measures in executive compensation: Determinants, contracting details and outcomes

Corporate social responsibility (CSR) contracting incorporates environmental, social, and governance (ESG) related measures in executive compensation plans. Current research on this practice is limited to a US setting, despite global adoption. We investigate heterogeneity in CSR contracting using data from 59 countries between 2002 and 2019. We find that besides firm-level past ESG performance and the industry-level adoption rate, country-level ESG regulations have significant explanatory power in firms’ tendencies to adopt CSR contracting. Hand-collected data reveal significant cross-country differences in CSR contracting details. Finally, CSR contracting is positively associated with subsequent financial performance only in countries with more stringent ESG regulations and stronger legal enforcement. In contrast, CSR contracting is associated with subsequent ESG performance regardless of country-level factors.

HSD98 Benefits and Barriers to Same-Day Long-Acting Reversible Contraceptive Insertion: Evidence and Its Implications from US Settings

HSD98 Benefits and Barriers to Same-Day Long-Acting Reversible Contraceptive Insertion: Evidence and Its Implications from US Settings

Outcomes of concomitant antiobesity medication use with endoscopic sleeve gastroplasty in clinical US settings

BackgroundTo evaluate the weight loss outcomes of the large US cohort of patients undergoing endoscopic sleeve gastroplasty (ESG) with or without concomitant anti-obesity (AOM) use. MethodsWe performed a retrospective analysis of adult patients who underwent ESG from seven different sites, from January 1, 2020 to November 30, 2022. Percent total body weight loss (%TBWL) and %excess weight loss (%EWL) were calculated based on baseline weight at the procedure. Medication use was considered if the subject received a prescribed AOM during the study period. SPSS (version 29.0) was used for statistical analyses. ResultsA total of 1506 patients were included (1359 (90.2 %) no AOM use and 147 (9.8 %) AOM use). Patients who were on an active AOM at the time of the procedure had a significantly lower TBWL% as compared to patients not on AOMs at 6 months. At the 24-month visit, patients who were prescribed AOMs after the 12-month visit had a significantly higher TBWL% and EWL% as compared to patients who were on active AOM at the time of the procedure. There was no significant difference between classes of medications at any time point, however, patients on a GLP-1RA had a trend towards improved weight loss at 18 and 24 months. ConclusionIn this large, real-world cohort of patients from the United States, data signal that with the use of pharmacotherapy at the appropriate time, patients can achieve optimal results.

Do customers matter in currency hedging policies? Evidence from product warranties

AbstractUsing a data set of US manufacturing firms with sales from foreign operations, we find that firms' currency hedging activities vary with their warranty obligations. The positive link of warranty obligations to currency hedging policies prevails in financially more constrained firms, companies facing fiercer product market competition and corporations producing more unique products. Our results suggest that firms are likely to incorporate their contractual commitments to customers into their currency hedging activities, especially when their failures to honor these commitments are more likely or costly.

Scopes, Specula, the Speculative: Histories of Medical Experimentation and Looking in African American Art and Fiction

This article traces a speculative and critical engagement with histories of health care disparity and medical exploitation shared across fictions by Ralph Ellison and Toni Morrison, and the artwork of Ellen Gallagher. It argues that insistent returns to racialized experimentation and scientific modes of looking form a significant interrogation of a wider set of US promises and attritions. Specifically, it asks how postwar African American culture takes up scopic questions to address dominant accounts of progress and the modern, both via reference to visual orders and technologies, and via formal choices regarding iteration, perspective and scale.

Use of Recommended Neurodiagnostic Evaluation Among Patients With Drug-Resistant Epilepsy

Interdisciplinary practice parameters recommend that patients with drug-resistant epilepsy (DRE) undergo comprehensive neurodiagnostic evaluation, including presurgical assessment. Reporting from specialized centers suggests long delays to referral and underuse of surgery; however, longitudinal data are limited to characterize neurodiagnostic evaluation among patients with DRE in more diverse US settings and populations. To examine the rate and factors associated with neurodiagnostic studies and comprehensive evaluation among patients with DRE within 3 US cohorts. A retrospective cross-sectional study was conducted using the Observational Medical Outcomes Partnership Common Data Model including US multistate Medicaid data, commercial claims data, and Columbia University Medical Center (CUMC) electronic health record data. Patients meeting a validated computable phenotype algorithm for DRE between January 1, 2015, and April 1, 2020, were included. No eligible participants were excluded. Demographic and clinical variables were queried. The proportion of patients receiving a composite proxy for comprehensive neurodiagnostic evaluation, including (1) magnetic resonance or other advanced brain imaging, (2) video electroencephalography, and (3) neuropsychological evaluation within 2 years of meeting the inclusion criteria. A total of 33 542 patients with DRE were included in the Medicaid cohort, 22 496 in the commercial insurance cohort, and 2741 in the CUMC database. A total of 31 516 patients (53.6%) were women. The proportion of patients meeting the comprehensive evaluation main outcome in the Medicaid cohort was 4.5% (n = 1520); in the commercial insurance cohort, 8.0% (n = 1796); and in the CUMC cohort, 14.3% (n = 393). Video electroencephalography (24.9% Medicaid, 28.4% commercial, 63.2% CUMC) and magnetic resonance imaging of the brain (35.6% Medicaid, 43.4% commercial, 52.6% CUMC) were performed more regularly than neuropsychological evaluation (13.0% Medicaid, 16.6% commercial, 19.2% CUMC) or advanced imaging (3.2% Medicaid, 5.4% commercial, 13.1% CUMC). Factors independently associated with greater odds of evaluation across all 3 data sets included the number of inpatient and outpatient nonemergency epilepsy visits and focal rather than generalized epilepsy. The findings of this study suggest there is a gap in the use of diagnostic studies to evaluate patients with DRE. Care setting, insurance type, frequency of nonemergency visits, and epilepsy type are all associated with evaluation. A common data model can be used to measure adherence with best practices across a variety of observational data sources.

Interprofessional practice and person-centred care: moral contexts and ethical conflicts in the US and Canada

Health and social care services and systems increasingly promote both interprofessional practice and person-centred care. This discussion first examines the clinical and community-based contexts for providing interprofessional practice and person-centred care through the lens of personal, interpersonal, interprofessional and organisational values. Emerging conflicts among values are highlighted using the concept of moral distress, with examples from US and Canadian settings. The comparison then moves to the systems level, examining different US and Canadian contexts for improving the quality and reducing the cost of care. Finally, implications and applications are presented, focusing on interrelationships among the individual, interprofessional team and organisation.

Promoting Research Collaboration with Open Data Driven Team Recommendation in Response to Call for Proposals

Building teams and promoting collaboration are two very common business activities. An example of these are seen in the TeamingForFunding problem, where research institutions and researchers are interested to identify collaborative opportunities when applying to funding agencies in response to latter's calls for proposals. We describe a novel deployed system to recommend teams using a variety of AI methods, such that (1) each team achieves the highest possible skill coverage that is demanded by the opportunity, and (2) the workload of distributing the opportunities is balanced amongst the candidate members. We address these questions by extracting skills latent in open data of proposal calls (demand) and researcher profiles (supply), normalizing them using taxonomies, and creating efficient algorithms that match demand to supply. We create teams to maximize goodness along a novel metric balancing short- and long-term objectives. We validate the success of our algorithms (1) quantitatively, by evaluating the recommended teams using a goodness score and find that more informed methods lead to recommendations of smaller number of teams but higher goodness, and (2) qualitatively, by conducting a large-scale user study at a college-wide level, and demonstrate that users overall found the tool very useful and relevant. Lastly, we evaluate our system in two diverse settings in US and India (of researchers and proposal calls) to establish generality of our approach, and deploy it at a major US university for routine use.

Audit partner style and financial statement comparability: New evidence from the US market

AbstractWe explore the influence of individual audit partner style on financial statement comparability in a US setting using newly available data. We find evidence of an audit partner style effect on comparability incremental to audit firm and audit office effects. Our results are consistent across several comparability measures and are economically significant. For example, the audit partner effect is associated with a 12.59% decrease in the mean difference in abnormal accruals compared with a 4.4% decrease for firm pairs audited by the same Big 4 audit office. We also find that audit partner expertise and experience have a greater influence on comparability than audit office expertise and experience. Our results are consistent in endogeneity and robustness tests including (1) auditor switching tests, (2) entropy balancing, (3) placebo tests and (4) controlling for clients’ economic similarity. Our results suggest that the association between audit partner style and financial statement comparability persists in highly regulatory environments and corroborate the PCAOB's intuition that the role of the engagement partner “is of singular importance” to the outcome of the audit (PCAOB 2011).

Individual and Neighborhood Level Predictors of Children’s Exposure to Residential Greenspace

Inequities in urban greenspace have been identified, though patterns by race and socioeconomic status vary across US settings. We estimated the magnitude of the relationship between a broad mixture of neighborhood-level factors and residential greenspace using weighted quantile sum (WQS) regression, and compared predictive models of greenspace using only neighborhood-level, only individual-level, or multi-level predictors. Greenspace measures included the Normalized Difference Vegetation Index (NDVI), tree canopy, and proximity of the nearest park, for residential locations in Shelby County, Tennessee of children in the CANDLE cohort. Neighborhood measures include socioeconomic and education resources, as well as racial composition and racial residential segregation. In this sample of 1012 mother–child dyads, neighborhood factors were associated with higher NDVI and tree canopy (0.021 unit higher NDVI [95% CI: 0.014, 0.028] per quintile increase in WQS index); homeownership rate, proximity of and enrollment at early childhood education centers, and racial composition, were highly weighted in the WQS index. In models constrained in the opposite direction (0.028 unit lower NDVI [95% CI: − 0.036, − 0.020]), high school graduation rate and teacher experience were highly weighted. In prediction models, adding individual-level predictors to the suite of neighborhood characteristics did not meaningfully improve prediction accuracy for greenspace measures. Our findings highlight disparities in greenspace for families by neighborhood socioeconomic and early education factors, and by race, suggesting several neighborhood indicators for consideration both as potential confounders in studies of greenspace and pediatric health as well as in the development of policies and programs to improve equity in greenspace access.

Cost-effectiveness of tirofiban for acute ischemic stroke without large or medium-sized vessel occlusion: A Markov modelling analysis from the Chinese and United States perspectives.

The RESCUE BT2 trial recently showcased the efficacy of tirofiban in treating acute ischemic stroke (AIS) without large or medium-sized vessel occlusion. To further assess the value of tirofiban from the perspectives of Chinese and US healthcare system, a study was conducted to evaluate its cost-effectiveness. A hybrid model, integrating a short-term decision tree with a long-term Markov model, was developed to assess cost-effectiveness between tirofiban and aspirin for stroke patients without large or medium-sized vessel occlusion. Efficacy data for tirofiban was sourced from the RESCUE BT2 trial, while cost information was derived from published papers. Outcomes measured included respective cost, effectiveness, and incremental cost-effectiveness ratio (ICER). We conducted a one-way sensitivity analysis to assess the robustness of the results. Additionally, we performed probabilistic sensitivity analysis (PSA) through 10,000 Monte Carlo simulations to evaluate the uncertainties associated with the results. The study revealed that tirofiban treatment in AIS patients without large or medium-sized vessel occlusion led to a considerable reduction of 2141 Chinese Yuan (CNY) in total cost, along with a lifetime gain of 0.14 quality-adjusted life years (QALYs). In the US settings, tirofiban also exhibited a lower cost ($197,055 versus $201,984) and higher effectiveness (4.15 QALYs versus 4.06 QALYs) compared to aspirin. One-way sensitivity analysis revealed that post-stroke care costs and stroke utility had the greatest impact on ICER fluctuation in both Chinese and US settings. However, these variations did not exceed the willingness-to-pay threshold. PSA demonstrated tirofiban's superior acceptability over aspirin in over 95% of potential scenarios. Tirofiban treatment for AIS without large or medium-sized vessel occlusion appeared dominant compared to aspirin in both China and the US.

High prevalence of hepatitis B virus susceptibility among persons undergoing community-based hepatitis C virus treatment

BackgroundDue to shared modes of transmission, coinfection with hepatitis B virus (HBV) and hepatitis C virus (HCV) is common, and HBV vaccination is recommended for all persons with HCV who remain susceptible to HBV. To identify potential gaps in HBV vaccination among this high-risk population, we aimed to determine the patterns of HBV susceptibility in persons undergoing community-based HCV treatment.MethodsWe performed a cross-sectional study within two community-based HCV treatment programs in an urban US setting. Participants were identified for HCV screening and confirmatory testing via street-outreach recruitment directed at persons experiencing homelessness and currently using drugs. Participants were excluded if HBsAg was reactive. Cohort characteristics were obtained via intake surveys and descriptive analysis was performed by exposure status.ResultsAmong 150 participants without chronic HBV receiving community-based HCV treatment, 43% had evidence of prior HBV infection, 26% were immune from vaccination, and 31% were non-immune. Among the subset of the cohort reporting current injection drug use (IDU) (N = 100), 31% (n = 10) of those aged 24–40 and 47% (n = 23) of those aged 41–57 remained susceptible to HBV infection. By contrast only two participants aged 58–74 were HBV non-immune (11%), with 84% immune due to prior exposure.ConclusionsOur data reflect a high prevalence of HBV susceptibility among persons undergoing community-based HCV treatment. Although younger patients were more likely to be immune due to vaccination, a high proportion remained non-immune to HBV, particularly among those reporting current IDU. Our data reflect a gap in HBV vaccination among younger persons with HCV and suggest a potential role for co-localizing HBV vaccination with community-based HCV screening and treatment.

In Support of Breast-/Chestfeeding by People With HIV in High-Income Settings.

Given that HIV can be transmitted through breastfeeding, historically, breastfeeding among women with HIV in the US and other resource-rich settings was discouraged. Formula feeding was the mandated feeding option out of concern for breast-milk transmission of HIV, which occurred in 16-24% of cases pre-antiretroviral therapy (pre-ART) use. In January 2023, the US Department of Health and Human Services' Perinatal Guidelines were revised to support shared decision-making for infant feeding choices. Updated clinical trials' data from resource-limited settings suggest the actual breastmilk HIV transmission rate in the context of maternal ART or neonatal postexposure prophylaxis is 0.3-1%. High-income countries are reporting more people with HIV breastfeeding their infants without cases of HIV transmission. We present the reasons for fully embracing breast-/chestfeeding as a viable, safe infant feeding option for HIV-exposed infants in high-income settings, while acknowledging unanswered questions and the need to continually craft more nuanced clinical guidance.

Cost-Effectiveness of Caplacizumab in the Warranty Program in Immune Thrombotic Thrombocytopenic Purpura in the USA

Background Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening thrombotic microangiopathy. Caplacizumab is a humanized nanobody that inhibits formation of microthrombi in iTTP by disrupting binding of von Willebrand factor to platelets. It is the only treatment approved in combination with plasma exchange therapy (PEX) and immunosuppression (IS) for the treatment of iTTP. The United Kingdom National Institute for Health and Care Excellence (NICE) committee reviewed cost-effectiveness of caplacizumab. The committee concluded that addition of caplacizumab to PEX+IS is cost-effective and recommended that the drug be reimbursed in the UK. The Sanofi Promise Warranty Program ® (SPWP) was introduced in January 2023 in the US. It refunds the full cost of caplacizumab to the hospital when treatment is discontinued due to non-response defined as refractoriness (patient with platelet counts <50 × 10 9/L after 4 days of combined treatment with caplacizumab and PEX+IS; up to 6 doses are refunded), or exacerbation (new drop in platelet count after initial platelet count normalization [>150 × 10 9/L], necessitating re-initiation of PEX after ruling out any causes for the drop in platelet count other than iTTP pathophysiology, up to 12 pre-exacerbation doses are refunded). Objectives To evaluate the cost-effectiveness of adding caplacizumab to PEX+IS from a US perspective within the context of SPWP. Methods The NICE model was adapted to the US setting by replacing UK costs with US costs and discount rates. All clinical assumptions and structure remain unchanged. Clinical inputs were derived from the HERCULES trial (NCT02553317) intent-to-treat population (mean age: 46 years; females: 69%, exacerbation rates: 4.17% during double blind phase and 12.50% in total; refractoriness: 0%). The model consisted of a 3-month decision tree, which captured patient treatment and outcomes over the acute iTTP episode, followed by a long-term Markov model (time horizon: up to 55 years; 3-monthly cycles; Figure 1). An annual discount rate of 3% was applied for both costs and quality-adjusted life-years (QALYs). Under the SPWP, the cost of caplacizumab was assumed to be $0 for refractory patients and those who exacerbate early, no discount was applied for patients who experience exacerbations after drug discontinuation, however, clinical outcomes were captured. Results Prior to the SPWP, caplacizumab addition to PEX+IS delivered a gain of 1.75 QALYs (2.96 life-years [LYs]) as compared with PEX+IS alone, at an increased lifetime cost of $256,000. The incremental cost effectiveness ratio (ICER) was $146,350 per QALY and $86,420 per LY gained ( Table 1). An estimated 4.17% of treated patients qualified for the SPWP. With this outcomes-based program, the ICER improved to $138,480 per QALY and $81,780 per LY gained, respectively ( Table 1). Conclusion Based on these analyses using the UK NICE model adapted with US costs, caplacizumab is cost-effective considering WTP threshold of $150,000 to $200,000 and has improved cost-effectiveness when the warranty program is taken into account. Outcomes based programs can improve the cost effectiveness of a treatment in addition to mitigating concerns of perceived uncertainty of efficacy in treating patients with iTTP in the US.