Journal of Health Economics and Outcomes Research最新文献

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 $895\hspace{0.17em}202\hspace{0.17em}311percohortand$ 6460 per patient, across all patients. Scenario analysis resulted in cost savings of $890\hspace{0.17em}829\hspace{0.17em}889percohort,and$ 6429 per patient. Similarly, savings of $378\hspace{0.17em}689\hspace{0.17em}020percohortor$ 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 $376\hspace{0.17em}902\hspace{0.17em}203percohortand$ 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

Background: Attention-deficit/hyperactivity disorder (ADHD) affects approximately 4.4% of US adults. ADHD is associated with high-risk driving behavior and costly motor vehicle accidents. DYANAVEL XR (DXR) (Tris Pharma, Inc.) is a once-daily fast-acting amphetamine developed for ADHD treatment. A randomized controlled trial showed that DXR patients were 43% less likely to crash during a driving simulation than individuals taking placebo. Study outcomes suggest a DXR crash rate similar to that of a driver without ADHD, while patients treated with the current standard of care (SOC) have a 52% higher crash risk than non-ADHD drivers. Objective: The aim was to evaluate the economic benefits attributable to improved driving abilities and avoided crashes in DXR patients compared with patients treated with the SOC or those who are untreated. Methods: A cost-impact model estimated 1-year crash-related cost outcomes for DXR-treated patients compared with SOC-treated and untreated ADHD patients. SOC was assumed to consist of a combination of short-, intermediate-, and long-acting ADHD stimulant and non-stimulant medications. DXR crash risk was assumed equivalent to the non-ADHD population risk, as supported by trial data. Crash risk for untreated and SOC-treated ADHD patients were assumed to be 99% and 52% higher than the general US population, respectively. Model outcomes included the cost impact (medication- and crash-related costs) and the number of crashes, injuries, and fatalities avoided with DXR. Results: Treatment with DXR would avoid 0.82 crashes, 0.016 injuries, and 0.036 fatalities per year compared with untreated patients, and 0.036 crashes, 0.007 injuries, and 0.0001 fatalities per year compared with SOC-treated patients. Compared with a population of 25% SOC-treated patients and 75% untreated patients, DXR use would save an average of $4581perpersonperyearacrossallagegroupswhenpricedat$ 80 per month, assuming all SOC-treated and untreated patients utilized DXR. When the value of quality-of-life improvement is considered, savings increase over 7-fold. Discussion: Outcomes suggest that DXR may be an economically beneficial treatment compared with SOC for ADHD patients. Conclusions: The economic model showed that DXR is cost-saving compared with no treatment and SOC by reducing the number of motor vehicle crashes in the ADHD population.

Background: Individuals with type 2 diabetes (T2D) show high risk of heart failure (HF). Left ventricular ejection fraction is a major factor for disease progression. In Germany, no recent longitudinal data are available. Objectives: To (1) measure the proportion of individuals with T2D who acquire HF over 2 years and (2) categorize ejection fraction using routine data and an algorithm, and (3) understand progression of HF in 5-year follow-up. Methods: This descriptive, retrospective study used longitudinal data from German statutory health insurance claims. A model using coded data classified the patients with HF into ejection fraction (EF) categories. Individuals were selected during 2013, with an inclusion period from 2014 to 2015 and a follow-up from 2016 to 2020. Baseline characteristics included demographic data, disease stage, comorbidities, and risk factors. Follow-up criteria included major adverse cardiac events (MACEs), EF category, and mortality. Disease progression was visualized by Sankey plots. Results: Among the 173 195 individuals with T2D identified in 2013, 6725 (median age, 74 years) developed HF in 2014 or 2015. 34.4% of individuals had MACEs, and 42.9% died over 5 years. Myocardial infarction (42%) was the most common event, followed by stroke (32%) and hospitalization (28%). A total of 5282 (78.54%) patients were classified into preserved EF and 1443 (21.46%) into reduced EF. Survival after 5 years was 71% in HF for preserved EF patients, and 29% in the HF for those with reduced EF. Conclusion: Heart failure is relevant in individuals with diabetes. A high number of patients may likely not survive a 5-year period. Validation of the model with German data is highly desirable. New ways of close monitoring could help improve outcomes.

Background: For patients with locally advanced or metastatic urothelial carcinoma (la/mUC), prognosis is poor and effective treatment options are limited. Erdafitinib is an oral fibroblast growth factor receptor (FGFR) kinase inhibitor approved by the FDA for the treatment of adults with la/mUC harboring FGFR alterations whose disease progressed following at least 1 prior line of therapy, including a PD-1 or PD-L(1) inhibitor, based on the phase 3, randomized THOR trial (NCT03390504, Cohort 1). Objective: To compare the efficacy and safety of erdafitinib vs enfortumab vedotin-ejfv (EV) in the absence of head-to-head comparison via an anchored matching-adjusted indirect comparison (MAIC). Methods: An anchored MAIC was conducted according to the National Institute for Health and Care Excellence Decision Support Unit guidance, with physician's choice of chemotherapy (docetaxel/paclitaxel and vinflunine) as the common comparator. Individual patient data from THOR were adjusted to match published key eligibility criteria and average baseline characteristics of EV-301, such as Bellmunt risk score, liver or visceral metastases, primary site, among others. Erdafitinib was then indirectly compared with EV using the relative treatment effects for the reweighted THOR population and those published for EV-301. Results: After matching, the effective sample size for THOR was 126 patients. The MAIC-recalculated hazard ratio (95% credible interval) for erdafitinib vs EV was 0.92 (0.54, 1.57) for overall survival and 0.93 (0.55, 1.56) for progression-free survival, yielding Bayesian probabilities of erdafitinib being better than EV of 62.1% and 60.5%, respectively. For response outcomes, the MAIC-recalculated risk ratio was 1.49 (0.56, 3.90) for confirmed objective response rate and 2.89 (0.27, 30.33) for confirmed complete response with probabilities of 72.6% and 81.3% for erdafitinib being better than EV, respectively. For safety, MAIC-yielded risk ratios of 1.09 (0.99, 1.21) for any treatment-related adverse events, 0.86 (0.57, 1.28) for grade 3+ TRAEs, and 1.02 (0.98, 1.06) for any treatment-emergent adverse events. Conclusion: The MAIC indicates comparable efficacy of erdafitinib vs EV for overall survival and progression-free survival, with erdafitinib showing a higher probability of achieving deep responses. While erdafitinib is associated with slightly more adverse events compared with EV, these events seem to be less severe.

Background: The use of androgen receptor signaling inhibitors, including apalutamide, in combination with androgen deprivation therapy is recommended for the treatment of metastatic castration-sensitive prostate cancer (mCSPC) and non-metastatic castration-resistant prostate cancer (nmCRPC). Objective: To describe real-world treatment patterns and clinical outcomes among patients with mCSPC or nmCRPC who initiated apalutamide in the United States. Methods: A retrospective cohort study of patients with mCSPC or nmCRPC who initiated apalutamide was conducted using electronic medical record data from US community-based urology practices (Feb. 1, 2017-April 1, 2022). Persistence with apalutamide was reported at 6-, 12-, and 18-months post treatment initiation. Clinical outcomes described up to 24 months after apalutamide initiation using Kaplan-Meier analyses included progression to castration resistance, castration resistance-free survival (CRFS), and metastasis-free survival (MFS). Outcomes were reported separately based on mCSPC or nmCRPC status and race (ie, Black or non-Black). Results: This study included 589 patients with mCSPC (mean age, 75.9 years) and 406 patients with nmCRPC (mean age, 78.8 years). Using a treatment gap of >90 days, persistence with apalutamide at 12 months remained high for both the mCSPC (94.9%) and nmCRPC (92.7%) cohorts, and results were descriptively similar among Black and non-Black patients, and when a treatment gap of >60 days was considered. In patients with mCSPC, overall progression to castration resistance rates at 12 and 24 months were 20.9% and 33.5%, and overall CRFS rates were 76.2% and 62.0%, respectively. In patients with nmCRPC, overall MFS rates at 12 and 24 months were 89.7% and 75.4%, respectively. Rates of these clinical outcomes were descriptively similar between Black and non-Black patients. Discussion: While clinical trials have demonstrated the efficacy and safety of apalutamide, there is limited real-world data describing treatment persistence and clinical outcomes among patients with mCSPC and nmCRPC who initiated apalutamide. Conclusions: In this real-world study of patients with mCSPC or nmCRPC initiated on apalutamide, treatment persistence was high and apalutamide demonstrated robust real-world effectiveness with respect to progression to castration resistance, CRFS, and MFS, overall and among Black and non-Black patients.

Background: Cataract surgery is an effective and commonly utilized procedure and can significantly improve quality of life and restore economic productivity. Certificate of need (CON) laws aim to regulate healthcare facility expansion and equipment acquisition to curtail costs, enhance quality, and ensure equitable access to care. However, little is known about the impact of CON laws on cataract surgery utilization and reimbursement. Objectives: To compare utilization and reimbursement for non-complex cataract surgery in CON and non-CON states. Methods: This retrospective database review analyzed publicly available data from the Centers for Medicare and Medicaid Services from 2017 to 2021 to identify the Medicare beneficiaries who underwent non-complex cataract surgery using Current Procedural Terminology code 66984 in Medicare outpatient hospitals. Utilization and reimbursement patterns were analyzed in states with and without CON laws using the compound annual growth rate, with reimbursement adjusted by the US Bureau of Labor Statistics Consumer Price Index. Results: The Centers for Medicare and Medicaid Services reported 893 682 non-complex cataract surgeries in the study period; of these, 609 237 were in CON and 280 215 in non-CON states. Inflation-adjusted reimbursement increased in both CON (1.17%) and non-CON (1.83%) states, while the reimbursement in non-CON states was greater than the national average adjusted reimbursement (1.67%). Utilization of non-complex cataract surgery declined during the study period in both CON and non-CON states. A larger decline in utilization was observed in CON states (-7.32%) than in non-CON states (-6.49%). Utilization was slightly higher in non-CON than in CON states for each year except 2019. Discussion: Utilization of non-complex cataract surgery by Medicare beneficiaries declined over the study period in both CON and non-CON states, possibly impacted by the COVID-19 pandemic. Inflation-adjusted reimbursement adjusted for Consumer Price Index increased more in non-CON than CON states, possibly reflecting shifts in market dynamics in CON-regulated states. Conclusions: Surgeons and policymakers should consider the implications of CON laws on the utilization and reimbursement of cataract surgery. Further study is necessary to ascertain whether these trends persist beyond 2021.

Background: Postoperative urinary retention (POUR) is a common and distressing surgical complication that may be associated with the pharmacological reversal technique of neuromuscular blockade (NMB). Objective: This study aimed to investigate the impact that POUR has on medical charges. Methods: This was a retrospective observational study of adult patients undergoing select surgeries who were administered neuromuscular blockade agent (NMBA), which was pharmacologically reversed between February 2017 and November 2021 using data from the PINC-AI™ Healthcare Database. Patients were divided into 2 groups: those experiencing POUR (composite of retention of urine, insertion of temporary indwelling bladder catheter, insertion of non-indwelling bladder catheter) during index hospitalization following surgery and those without POUR. Surgeries in inpatient and outpatient settings were analyzed separately. A cross-sectional comparison was performed to report total hospital charges for the 2 groups. Furthermore, patients experiencing subsequent POUR events within three days after discharge from index hospitalization were studied. Results: A total of 330 838 inpatients and 437 063 outpatients were included. POUR developed in 13 020 inpatients and 2756 outpatients. Unadjusted results showed that POUR was associated with greater charges in both inpatient ( $92 529withPOURvs$ 78 556 without POUR, p < .001) and outpatient ( $48 996withPOURvs$ 35 433 without POUR, p < .001) settings. After adjusting for confounders, POUR was found to be associated with greater charges with an overall mean adjusted difference of $10 668(95$ 95 760- $11 760,p\hspace{0.17em}<\hspace{0.17em}.001)ininpatientand$ 13 160 (95% CI $11 750-$ 14  571, p < .001) in outpatient settings. Charges associated with subsequent POUR events following discharge ranged from $9418inpatientchargesto$ 1694 outpatient charges. Conclusions: Surgical patients who were pharmacologically reversed for NMB and developed a POUR event incurred greater charges than p

Background: Pre-exposure prophylaxis (PrEP) for COVID-19 provides additional protection, beyond vaccines alone, for individuals who are immunocompromised (IC). This may reduce the need for preventative behavioral modification, such as shielding-a behavioral restriction limiting an IC individual to minimize face-to-face interactions and/or crowded places. Therefore, PrEP may improve psychosocial well-being and health-related quality of life (HRQoL) for individuals with IC conditions. Objective: To estimate the potential HRQoL and utility benefit of PrEP for prevention of COVID-19 in individuals with IC conditions who may not have an adequate response of full vaccination (and therefore are at "highest risk" of severe COVID-19) that can be used in future economic evaluations of preventative therapies against COVID-19. Methods: Vignettes describing HRQoL associated with 2 pre-PrEP states (shielding and semi-shielding behavioral restrictions) and a post-PrEP state were developed from a literature review and tested through interviews with clinicians (n = 4) and individuals with IC conditions (n = 10). Vignettes were valued by a general population sample (N = 100) using a visual analog scale (VAS), time trade-off (TTO), and EQ-5D-5L. A sample of individuals with IC conditions (n = 48) valued their current HRQoL and a post-PrEP vignette using VAS and EQ-5D-5L. Results: Individuals with IC conditions reported a mean current EQ-5D-5L score of 0.574, and 0.656 for post-PrEP based on the vignette. PrEP would lead to behavior changes for 75% (30/40) of individuals with IC conditions and an emotional benefit for 93% (37/40) of individuals with IC conditions. Mean values from the general population valuation based on EQ-5D-5L ranged from 0.606 ("shielding") to 0.932 ("post-PrEP"). Conclusion: This study quantified the expected health state utility benefit of reduced psychosocial burden and behavioral restriction. PrEP would potentially result in a utility gain between 0.082 and 0.326, dependent on valuation approach and expected change in behavioral restrictions, leading to improvements in daily activities and emotional well-being.

Background: The second most common hematologic cancer worldwide is multiple myeloma (MM), with incidence and mortality rates that have more than doubled over the past 30 years. The safety and efficacy of daratumumab regimens in the treatment of newly diagnosed MM (NDMM) is demonstrated in clinical trials. Objective: To assess the financial effects of the adoption of subcutaneous daratumumab (dara-SC) rather than intravenous daratumumab (dara-IV) for the treatment of NDMM in three Gulf countries (Qatar, Oman and the United Arab Emirates; UAE), a cost-minimization model was constructed. Methods: We performed static cost minimization analyses from a societal perspective to evaluate the costs and possible reductions in resource utilization associated with a shift from dara-IV infusion to dara-SC injection for NDMM patients over a 5-year time horizon. The model included 2 scenarios: the current scenario in which 100% of patients with NDMM are treated with dara-IV infusion and a future scenario in which dara-SC injection is gradually adopted over the modeled time horizon. The model differentiated precisely between autologous stem cell transplantation (ASCT)-eligible and ASCT-ineligible NDMM patients in terms of their number in each group and the associated therapeutic regimens. One-way sensitivity analyses were also conducted. Results: The model showed that the use of dara-SC in NDMM patients who were eligible or ineligible for ASCT resulted in lower non-drug costs, including premedication drug costs, adverse-effect costs, administration costs, medical staff costs, and indirect costs. The resulting total savings over the 5-year time horizon of the model for Hamad Medical Corporation, Sultan Qaboos University Hospital/Royal Hospital, Sheikh Shakhbout Medical City (SSMC), and Tawam Hospital were QAR -2 522 686, OMR -143 214, AED -30 010 627, and AED -5 003 471, respectively. Conclusion: The introduction of dara-SC as a front-line treatment for NDMM patients in Qatar (Hamad Medical Corporation), Oman (Sultan Qaboos University Hospital, Royal Hospital-MOH), and the UAE (SSMC and Tawam Hospital) can help save resources and minimize constraints on the healthcare system.

Background: Understanding how population density affected the transmission of COVID-19 is vitally important, since crowded cities were the epicenters for the disease. Since human contact was the main cause of the spread, population-weighted densities have been shown to be a better measure than conventional densities, since the variation in density across subareas matters more than the density in the total area. Objectives: This study investigates the impact of population-weighted density and other demographics on the rate of COVID-19 spread in the United States. Methods: The study considers population-weighted density and many other demographics. The population-weighted density index is the weighted average of density across the tracts, where tracts are weighted by population. Multivariate analysis has been used to determine the elasticity of the spread. Results: Using U.S. county-level data, we calculated the elasticity of COVID-19 spread with respect to population-weighted density to be 0.085 after controlling for other factors. In addition to the density, the proportion of people over 65 years of age, the number of total healthcare workers, and average temperature in each county positively contributed to the case numbers, while education level and income per capita had a negative effect. Discussion: For the spread, understanding the population characteristics and dynamics is as important as understanding the infectious disease itself. This will help policy makers to utilize and reallocate the resources more effectively. If the spread is successfully contained early, there will be less stress placed upon the healthcare system, resulting in better healthcare access for those who are sick. Conclusions: Our analysis suggests that population-weighted density can be a useful tool to control and manage outbreaks, especially within the early stage of the spread. We presented the early dynamics of the spread and recommended a policy measure on how to transfer healthcare workers from low-spread-risk areas to high-spread-risk areas to utilize resources better.