To estimate the cost-effectiveness of cetuximab in combination with radiotherapy compared with radiotherapy alone, for the treatment of locally advanced head and neck cancer patients in Spain. A probabilistic Markov model (second-order Monte Carlo simulation) with a five-year time horizon and quarterly Markov cycles was performed from the perspective of the Spanish National Health System (NHS). The additional cost and quality-adjusted life-year (QALY) gain per patient receiving radiotherapy in combination with cetuximab compared with radiotherapy alone was €4356 (95% CI: €4350-4362) and 0.2380 (95% CI: 0.2370-0.2391) QALY, respectively. The incremental cost per QALY gain was €18,303 (95% CI: €18,243-18,354) with a probability of cost-effectiveness of 65.4% for a willingness to pay of €30,000 per QALY gained. According to the results of this analysis, the addition of cetuximab to radiotherapy would be a cost-effective alternative to radiotherapy alone in the treatment of locally advanced head and neck cancer in Spain.

The treatment of non-small-cell lung cancer (NSCLC) has progressed from histology-oriented cytotoxic therapy to the era of molecular biology-oriented targeted therapy and immunotherapy. As the first tyrosine kinase inhibitor (TKI) targeting the pathway, crizotinib is widely used as a first-line regimen for -rearranged NSCLC. However, due to the paucity of solid data from randomized, controlled phase III clinical studies, clinicians often require more systematic, real-world data-based guidance for its optimal clinical use. As one of the leading countries of real-world research on crizotinib, China has contributed significantly to data on standardization of the therapeutic use of crizotinib, including its clinical treatment patterns, the timing and duration of treatment and drug resistance monitoring and management.

This ambispective observational study assessed the impact of Noona, an electronic patient-reported outcomes (ePRO) platform, for patients with non-small cell lung cancer (NSCLC) treated in a community oncology setting. Adults with advanced NSCLC, ECOG performance status of 0-2, who received first-line (1L) pembrolizumab (monotherapy or with chemotherapy) were eligible. Those initiating pembrolizumab from 1 July 2017 to 30 June 2019, identified retrospectively (historical cohort), were compared with those initiating pembrolizumab from 1 October 2019 to 30 September 2021 who were prospectively offered Noona (standard of care [SoC] cohort). The Kaplan-Meier method and Cox proportional hazards models were used to compare pembrolizumab real-world time on treatment (rwToT; primary outcome measure) and rw time to next treatment or death (rwTTNTD) between historical and SoC cohorts. Healthcare resource use (HCRU) was compared using generalized linear models with Poisson distribution. Analyses were repeated to compare outcomes in the SoC cohort between Noona users (created a profile and used any function ≥one-time during 1L therapy) and nonusers with >42 days on 1L pembrolizumab. Data cutoff was 30 June 2020 and 30 September 2022 for historical and SoC cohorts, respectively. Median pembrolizumab rwToT was 4.4 months (95% CI: 3.9-5.1) in the historical cohort (n = 448) versus 4.1 months (95% CI: 3.3-4.8) in the SoC cohort (n = 462; adjusted hazard ratio [aHR], 0.9; 95% CI: 0.8-1.0; p = 0.14 vs historical cohort). In the SoC cohort, 147 of 341 eligible patients (43%) established a Noona profile; 122/341 (36%) were Noona users. Median rwToT was 6.4 months (95% CI: 5.1-7.4) and 6.9 months (95% CI: 5.6-7.6) among Noona users and Noona nonusers (n = 219), respectively (aHR, 1.1; 95% CI: 0.8-1.4; p = 0.95 vs Noona users). The rwTTNTD and HCRU were comparable in historical versus SoC cohorts and for Noona users versus nonusers. During the first year after establishing a Noona profile, 92 of 147 patients (63%) used the platform; monthly use was 32-42%, and checking laboratory results was the most used function overall (by 52% of the 147). Notwithstanding the null findings of this study, positive results of ePRO use in clinical trials and observational studies support the treatment-related symptom monitoring and survival benefits of ePRO utilization.

To compare the efficacy and safety of lanadelumab versus other approved long-term prophylaxis (LTP) treatments in patients with pediatric hereditary angioedema (HAE) aged <12 years. A systematic literature review was conducted to identify studies of LTP in patients with HAE aged <12 years. Two studies met the inclusion criteria in an indirect treatment comparison of efficacy and safety data in pediatric HAE patients. These were for lanadelumab (SPRING, NCT04070326) and intravenous-C1-esterase inhibitor (C1-INH[IV], NCT02052141). A propensity score analysis used individual patient-level data from both studies in a logistic regression model to estimate inverse probability weights. To avoid convergence issues and an underpowered analysis due to the small sample size (n = 29), the base case was defined as Poisson regression analyses on monthly attack rate adjusting for one covariate (baseline attack rate). Model selection among unadjusted, adjusted and weighted regression models was conducted through the Akaike and Bayesian Information Criteria. Lanadelumab 150 mg every 2 weeks (Q2W) reduced the monthly HAE attack rate by 82.1% versus C1-INH(IV) 1000 IU twice weekly (every 3-4 days [BIW]; rate ratio [RR], 0.1792 [95% CI: 0.0296-1.0853]) and by 88.9% versus C1-INH(IV) 500 IU BIW (RR: 0.1107 [95% CI: 0.0234-0.5239]). Treatment with lanadelumab Q2W reduced the risk of total adverse events by 56.2% versus C1-INH(IV) 1000 IU BIW (RR:0.4377 [95% CI: 0.1536-1.2469]) and by 66.0% versus C1-INH(IV) 500 IU BIW (RR: 0.3401 [95% CI: 0.1234-0.9371]). This exploratory analysis suggested a trend toward greater efficacy and fewer adverse events with lanadelumab 150 mg Q2W compared with C1-INH(IV) BIW 1000 IU and 500 IU in pediatric patients with HAE. Future studies could potentially assess larger samples over longer periods of time for the long-term preventative efficacy, safety and tolerability of lanadelumab and C1-INH(IV).

To compare the safety and efficacy of antidepressants (AD) among older adults with major depressive disorder (MDD) by assessing treatment change, augmentation and hospitalization rates. This retrospective study analyzed data from the Veterans Affairs (VA) database, including 142,138 patients aged ≥60 years diagnosed with MDD. Patients prescribed bupropion, citalopram, duloxetine, escitalopram, fluoxetine, mirtazapine, paroxetine, sertraline, or venlafaxine were included. Outcomes were treatment change, augmentation and hospitalization rates. Hazard ratios (aHRs) were calculated using sertraline as the reference. Of the patients, 39.6% required augmentation, 18.1% changed antidepressant treatment and 13.3% were hospitalized. The corresponding incidence rate was 544, 124 and 122 events per 1000 person-years. Compared with sertraline, mirtazapine users had the highest AD change risk (aHR 1.34, 95% CI: 1.29-1.40), while duloxetine users had the lowest (aHR 0.87, 95% CI: 0.83-0.92). Duloxetine also had the lowest augmentation risk (aHR 0.89, 95% CI: 0.86-0.92). Mirtazapine users also had the highest risks of augmentation (aHR 1.15, 95% CI: 1.12-1.18) and hospitalization (aHR 1.14, 95% CI: 1.07-1.23). Bupropion had the lowest hospitalization risk (aHR 0.77, 95% CI: 0.71-0.84). Antidepressant choice significantly influences treatment outcomes in older adults with MDD. Duloxetine demonstrated the best profile with the lowest risks of AD change and augmentation, while mirtazapine posed the highest risks of all three outcomes. Personalized treatment strategies are crucial to improving outcomes in this population.

To estimate time-to-progression milestones in people with amyotrophic lateral sclerosis (PALS) treated versus not treated with intravenous (IV) edaravone (Radicava IV, Mitsubishi Tanabe Pharma America [MTPA], hereafter "IV edaravone") in a real-world setting. IV edaravone is US FDA approved for the treatment of ALS and was shown in clinical trials to slow the rate of physical functional decline. This retrospective observational analysis included PALS continuously enrolled in Optum's Clinformatics Data Mart between 8 August 2017 and 31 December 2021. Cases treated with IV edaravone and controls not treated with IV edaravone were propensity score matched for: age, sex, race, US region of residence, pre-index disease duration, insurance, riluzole prescription; and pre-index claims for cardiovascular disease, artificial nutrition/gastrostomy tube, noninvasive ventilation and all-cause hospitalization. The index date was the first IV edaravone claim for cases; for controls, the index date was randomly assigned after IV edaravone market availability. Restricted mean time lost was calculated for the following disease progression milestones: new use of canes/walkers/wheelchairs, artificial nutrition, noninvasive ventilation, invasive ventilation, speech-generating devices and hospice. Cases (n = 395) were matched to controls (n = 395). Cases had less restricted mean time lost, indicating longer disease progression milestone-free time, for all disease progression milestones. From 0 to 24 months post index, more cases (n = 129) than controls (n = 103) reported no milestones and more controls (n = 232) than cases (n = 131) reported deaths. In a US-based real-world setting, IV edaravone-treated PALS had a longer time to disease progression milestone events and fewer deaths in 2 years compared with PALS not treated with IV edaravone.

exon 14 ex14) skipping occurs in 3-4% of non-small-cell lung cancer (NSCLC) cases. Low frequency of this alteration necessitated open-label, single-arm trials to investigate MET inhibitors. Since broad MET biomarker testing was only recently introduced in many countries, there is a lack of historical real-world data from patients with ex14 skipping NSCLC receiving conventional therapies. Given the rarity of this population and limitations of existing real-world data sources, the MOMENT registry aims to prospectively collect uniform, comprehensive, high-quality data from patients with ex14 skipping advanced NSCLC treated in routine clinical practice, which can support clinical and regulatory decision making. MOMENT is a multinational, non-interventional disease registry collecting data on patients with ex14 skipping advanced NSCLC receiving any systemic anticancer therapy. Newly diagnosed patients and those already receiving treatment are eligible. Patients with previous participation in a clinical trial can be included if they receive at least one subsequent therapy line in a routine clinical setting. Eligible systemic treatment includes all available anticancer therapies (approved, conditionally approved or provided through Early Access). Data collection includes biomarker testing results, demographics, baseline clinical characteristics, treatment details and effectiveness, safety information and imaging. Registry site inclusion is dependent on confirmation that local ex14 skipping detection methods are sufficient to confirm ex14 skipping status. MOMENT is currently active at more than 60 sites across Europe and North America and approximately 700 patients are expected to be enrolled within the next 4 years. The first patient was enrolled on 4 October 2022. After completion of data collection, MOMENT data can be shared with external parties to conduct non-interventional studies. The MOMENT registry collects comprehensive, high-quality real-world data from patients with ex14 skipping advanced NSCLC receiving systemic anticancer treatment in a routine clinical setting, to enable future studies informing regulatory decisions and optimal care for this rare population. NCT05376891 (ClinicalTrials.gov); EUPAS47602 (EU PAS register no.).

Alagille syndrome (ALGS) is a rare, cholestatic multiorgan disease associated with bile duct paucity, leading to cholestasis. Clinical symptoms of cholestasis include debilitating pruritus, xanthomas, fat-soluble vitamin deficiencies, growth failure, renal disease and impaired health-related quality of life (HRQoL). The main objective was to review the current literature on the epidemiological, clinical, psychosocial and economic burden of ALGS in view of the development of ileal bile acid transporter (IBAT) inhibitors. Electronic literature databases were searched in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses checklist. 330 publications were screened, 119 were relevant: 11 randomized controlled trials (RCTs), 21 non-RCTs, 10 HRQoL studies, two studies assessing cost/resource use and 77 epidemiological studies across several databases through 31 July 2024. Studies confirm that patients with ALGS experience cardiac anomalies, impaired growth, renal disease, poor HRQoL, fat-soluble vitamin deficiencies and debilitating pruritus; until the approval of IBAT inhibitors for the treatment of cholestatic pruritus in patients with ALGS, supportive management was the standard of care. This review confirms the substantial clinical, economic and HRQoL burden associated with ALGS and consolidates current treatment evidence. Data from recent trials in ALGS demonstrate the potential impact of IBAT inhibitors to transform lives by improving cholestatic pruritus symptoms, HRQoL and native liver survival.

In this update, we discuss an article covering the implementation challenges of the upcoming European Union Health Technology Assessment regulation, particularly focusing on the complexity of population, intervention, comparator and outcomes requirements across member states; a user guide to applying generalized cost-effectiveness analysis for broader value assessment and finally highlight an ongoing debate surrounding National Institute for Health and Care Excellence's severity modifier implementation.

Screening and monitoring of diabetes or dyslipidemia frequently involves a multi-step process requiring patients to obtain test requisitions from their primary care physician (PCP), followed by a laboratory visit and re-consultation. Point-of-care testing (POCT) for hemoglobin A (HbA) and lipid panel can streamline the patient care pathway. This study assessed the budget impact of introducing Afinion™ 2 POCT (Abbott Rapid Diagnostics) from the Canadian and Italian societal perspectives. Budget impact models were developed for Canada and Italy over a 5-year time horizon (2025 to 2029). The analyses considered the screening and monitoring of diabetes or dyslipidemia for patients utilizing the public healthcare system and attending primary care, and included direct costs (testing, consultations) and indirect costs (productivity loss, transportation) based on published sources. The budget impact (BI) was calculated by comparing scenarios with and without POCT. All costs were adjusted to Canadian dollars ($) or 2024 Euros (€). Scenario analyses were conducted to explore the impact of alternative assumptions. The 5-year cumulative BI was -$758,006,692 (-$50,709,964 direct, -$707,296,728 indirect) for HbA POCT and -$726,452,755 ($2,684,011 direct, -$729,136,766 indirect) for lipid panel POCT in Canada and -€1,380,658,764 (-€6,391,954 direct, -€1,374,266,809 indirect) for HbA POCT and -€851,792,115 (€55,962,879 direct, -€907,754,993 indirect) for lipid panel POCT in Italy. In both countries, cost savings for both the healthcare payer and patients were observed for HbA POCT, while costs savings were derived from patient indirect costs for lipid panel POCT. The analyses estimated that 1,558,062 and 1,501,260 PCP consultations in Canada, 4,962,338 and 1,951,026 PCP consultations in Italy were avoided with implementation of POCT for HbA and lipid panel, respectively. Scenario analyses demonstrated potential further cost savings with implementation of POCT in pharmacies. This study demonstrates that the adoption of Afinion 2 POCT for HbA and lipid panel can provide efficiencies to different types of healthcare systems through reducing PCP consultations, saving time and money for patients and providing cost savings for payers.

Vaginitis and other vaginal discharge syndromes lead to high healthcare utilization. Molecular tests like syndromic multiplex real-time (RT) polymerase chain reaction (PCR)-based tests are highly sensitive and specific at diagnosing the infectious causes of vaginitis. This study compared the healthcare resource utilization (HCRU) and direct all-cause healthcare costs among patients with vaginitis in the US receiving next-day syndromic multiplex RT-PCR tests with those receiving other PCR tests or no diagnostic test of interest. This retrospective study utilized claims data from IQVIA PharMetrics Plus database to identify adult patients with a diagnosis for vaginitis (first claim = index) from January 2021 to April 2023, with 6 months of continuous enrollment prior to (baseline) and after index (follow-up). Pairwise comparisons were conducted between RT-PCR and 1:1 propensity matched Other PCR and No Test subcohorts for all-cause HCRU and costs during follow-up. Each of the RT-PCR, Other PCR and No Test subcohorts included 1946 matched patients. Mean(SD) follow-up total cost was significantly lower for the RT-PCR than the No Test subcohort ($5607 [$15,122] vs $6680 [$20,751], p = 0.0023). Mean(SD) overall outpatient and other medical service costs were lower for RT-PCR versus Other PCR (outpatient: $2964 [$9666] vs $3174 [$7113], p = 0.0110; other medical: $1961 [$9244] vs $2099 [$6475], p = 0.0002) and No Test subcohorts (outpatient: $2964 [$9666] vs $4067 [$12,341], p < 0.0001; other medical: $1961 [$9244] vs $2973 [$11,685]; p < 0.0001). A lower proportion had any outpatient service HCRU in RT-PCR versus Other PCR subcohort (92.6% vs 94.2%, p = 0.0349). A lower proportion had any other medical service claim in RT-PCR versus Other PCR (78.3% vs 83.2%, p < 0.0001) and No Test subcohorts (78.3% vs 83.0%, p = 0.0001). Physician office, emergency room (ER), prescription use and costs were similar between the subcohorts. The use of syndromic multiplex RT-PCR diagnostics with next day test results in patients with vaginitis was associated with lower outpatient costs and total healthcare costs than those in the no test cohort over 6 months. These findings indicate that use of syndromic multiplex RT-PCR diagnostics may contribute to improved patient management compared with clinical diagnosis alone.

Radiofrequency (RF) catheter ablation (CA) is a mainstay treatment for atrial fibrillation (AF). RF catheters with contact force (CF) sensing technology and electroanatomical mapping systems enable real-time assessment of catheter tip-tissue interface CF, facilitating individualized and precise CA. This study examined inpatient hospital readmissions in patients with AF treated with THERMOCOOL™ ST/ THERMOCOOL™ STSF catheter with the CARTO™ 3 System versus TactiCath™ catheter with the EnSite™ System. Patients undergoing CA for AF between 1 July 2019 to 30 November 2021 were identified from the Premier Healthcare Database and grouped based on use of THERMOCOOL ST/STSF or TactiCath™. Study outcomes were all-cause, cardiovascular (CV)-, and AF-related inpatient readmission at 91-365-day post-CA. Inverse probability of treatment weighting of propensity scores balanced baseline patient, comorbidity and hospital characteristics. A weighted generalized estimating equation (GEE) model examined differences in readmission outcomes. A total of 15,518 patients met inclusion criteria (THERMOCOOL ST/STSF, n = 13,001; TactiCath™, n = 2517). Patient characteristics were generally well-balanced after weighting. Patients treated with THERMOCOOL ST/STSF + CARTO 3 had a 20% lower likelihood of all-cause inpatient readmissions (7.8 vs 9.3%, chi-square p = 0.041; odds ratio [OR]: 0.80, 95% confidence interval [CI]: 0.66-0.96, GEE p = 0.019) and a 21% lower likelihood of CV-related inpatient readmission (5.2 vs 6.2%, chi-square p = 0.133, OR: 0.79, 95% CI: 0.62-0.99, GEE p = 0.043) in 91-365-days post-CA versus those treated with TactiCath™ + Ensite. No significant differences were observed for AF-related readmissions. Patients undergoing CA for AF treated with THERMOCOOL ST/STSF + CARTO 3 had a significantly lower risk of all-cause and CV-related inpatient hospital readmission versus those treated with TactiCath™ + Ensite.

Neurofibromatosis type 1 (also called NF1) is a rare genetic condition. It causes a range of symptoms that develop from childhood onwards and worsen over time. Some children with NF1 develop non-cancerous nerve tumors called plexiform neurofibromas. Plexiform neurofibromas can grow large and compress nearby tissues. This can cause severe pain, reduced movement, vision and hearing loss, and other medical problems. Some children can have plexiform neurofibromas removed surgically. Most children have tumors that cannot be removed by surgery (known as inoperable tumors). Children with inoperable plexiform neurofibromas can receive a medicine called selumetinib. This plain language summary includes important findings from two selumetinib studies in children with NF1 and inoperable plexiform neurofibromas: The SPRINT selumetinib studies are part of a clinical study program that looked at how well selumetinib works in treating children with symptomatic, inoperable plexiform neurofibromas. The SPRINT studies program included the first studies of this medicine done in children, called phase 1 and phase 2 studies. For the phase 2 study, some children had severe symptoms and some children did not. The group of children with severe symptoms is called group 1, and their results are included in this summary. The researchers monitored the participating children for up to 5 years in a long-term study to better understand how the treatment works over time. The NF1 caregivers experience study is a related study where caregivers shared their experiences of caring for children with NF1and plexiform neurofibromas.

Adaptive designs are frequently used in drug randomized controlled trials (RCTs). However, their use in medical device RCTs remains unclear. We aimed to characterize medical device RCTs with adaptive designs. We searched for adaptive RCTs in the following databases: ClinicalTrials.gov, International Clinical Trials Registry Platform and the International Standard Randomised Controlled Trial Number registry. Adaptive design keywords and medical device corporation names were used as terms to search the trial records registered between 1 January 2000 and 18 October 2024 in the databases. The annual number and proportions of adaptive trials were analyzed, and characteristics such as design type, sponsor, therapeutic area, trial stage and regulatory status were summarized. Overall, 105 adaptive RCTs were identified from ClinicalTrials.gov, accounting for 2.112 per 1000 trials in 49,721 medical device clinical trials registered in ClinicalTrials.gov during the period. The average annual number of adaptive RCTs per 1000 clinical trials was the highest (8.55 ± 11.65) during 2005-2010, reduced to 3.33 ± 2.35 during 2011-2016, and significantly decreased to 1.29 ± 0.85 during 2017-2024 (p = 0.011). The most common adaptive designs were group sequential design (GSD, 50.5%), sample size reassessment (SSR, 17.1%) and investigating both superiority and non-inferiority (10.5%). Most RCTs were sponsored by the private sector (62.9%), conducted in Europe/North America (95.2%), in the field of heart disease (46.7%) and post-market trials (76.2%). Compared with pre-market RCTs, post-market RCTs showed more diverse adaptive designs such as response-adaptive randomization and adaptive enrichment. The average annual proportions of adaptive medical device RCTs in ClinicalTrials.gov has reduced in the last 10 years. The most-used adaptive designs in medical device RCTs are GSD, SSR and investigating both superiority and non-inferiority.

To understand the impact of the niraparib individualized starting dose (ISD), compared with fixed starting dose (FSD), on the cost of hematologic adverse event (AE) management from a US payer perspective. The frequencies of grade ≥3 hematologic AEs that occurred in >1% of patients treated with niraparib were obtained from the primary analysis results of the phase III PRIMA/ENGOT-OV26/GOG-3012 trial. US unit costs for each grade ≥3 AE in the base case were obtained from the 2017 Agency for Healthcare Research and Quality Healthcare Cost and Utilization Project database; unit costs were adjusted to 2020 US dollars. AE management costs per patient were calculated by multiplying AE unit cost by the frequency of each AE by niraparib starting dose. Because AEs were assumed to occur independently of one another, costs were added to derive the total cost. For niraparib, the estimated AE management cost per patient was lower for the ISD than the FSD for all hematologic AEs (FSD vs ISD: thrombocytopenia, $4701.87 vs $1921.89; anemia, $2784.00 vs $1760.59; platelet count decreased, $2103.47 vs $922.51; neutropenia, $2112.50 vs $1369.56; neutrophil count decreased, $1285.87 vs $770.38). The total mean calculated AE management cost per patient was $12,987.71 with the FSD and $6744.93 with the ISD. For niraparib, the cost of managing hematologic AEs in the US was reduced by almost half with the ISD compared with the FSD. The cost reduction and improvements in safety associated with the niraparib ISD support its use in clinical practice.

Compared with uncomplicated urinary tract infections (UTIs), complicated UTIs (cUTIs) including acute pyelonephritis (AP) present with significant morbidity, a higher risk of treatment failure and typically require longer courses of treatment, or alternative antibiotics. The emergence of drug-resistant organisms represents a considerable challenge in the treatment of patients with cUTIs/AP and has limited antibiotic options. Carbapenems are considered the current last line of therapy, however, carbapenem resistance represents a growing problem. Although several established and novel treatment options are available, direct comparative evidence is lacking. Randomized controlled trials (RCTs) were identified by systematic literature review of Embase, MEDLINE and Cochrane databases (database inception to 15th June 2022). Relevant conference proceedings (2020-2022) were also reviewed. Following feasibility assessment to verify network connectivity at an overall level, outcome specific networks were prepared. Bayesian network meta-analysis (NMA) was performed (using R version 4.2.1) to determine the relative efficacy of various treatments for cUTI/AP, including cefepime + enmetazobactam. Convergence was assessed by visual inspection of trace plots. The accuracy of the posterior estimates was assessed using the Monte Carlo error for each parameter. Published study results were included in the synthesis of the relative risk (RR) of efficacy end points, using a logit link with binomial likelihood distribution. Feasibility assessment was conducted for 40 RCTs identified, to assess the viability of constructing a network of interlinked RCTs. Of those, 28 studies were included in the master NMA network. A fixed effects model (FEM) was selected due to low statistical heterogeneity, according to I values. For composite outcome at test of cure (TOC), ceftolozane + tazobactam, cefepime + enmetazobactam, cefiderocol, levofloxacin and plazomicin demonstrated significantly higher RRs versus carbapenems. For microbiological eradication at TOC, cefepime + enmetazobactam, plazomicin, cefiderocol, fosfomycin, meropenem + vaborbactam and ceftazidime + avibactam demonstrated significantly higher RRs versus carbapenems. RRs for cefepime + enmetazobactam were also significantly higher versus several established and novel treatment options for composite outcome, microbiological eradication and clinical cure. Against the backdrop of increasing bacterial resistance, these findings suggest that cefepime + enmetazobactam may represent an effective carbapenem-sparing treatment option in patients with cUTI including AP.

Percutaneous transluminal angioplasty (PTA) for peripheral artery disease (PAD) commonly leads to dissections which are associated with higher target lesion revascularization (TLR) rates. Clinical and economic consequences of dissection management in the femoropopliteal artery following PTA, and specifically the potential economic benefit of focal dissection repair using the novel Tack Endovascular System, remain unknown. A decision-analytic model was used to estimate 24-month clinical events, costs and quality-adjusted life year (QALY) gain for a Tack-supported versus status-quo PTA strategy. Patient and lesion characteristics and TLR rates were derived from the PTA cohort of the TOBA II clinical trial, an observational cohort, and literature. Cost-effectiveness was determined from a US payer and provider perspective separately for the non-severe (grade A or B), severe (grade C and higher) and the entire dissection cohort. TLR rates were lower for the Tack-supported strategy compared with PTA (7.7 vs 27.4% in the non-severe, 13.9 vs 25.8% in the severe and 12.0 vs 26.3% in the entire dissection cohort). Cost and QALY differences were +$297/ + 0.0110 in the non-severe dissection cohort and -$1602/ + 0.0067 in the severe dissection cohort, resulting in an incremental cost-effectiveness ratio (ICER) of $25,622 in the non-severe cohort and dominance in the severe cohort and the entire cohort. Compared with a 'status-quo' approach, proactive focal stenting may lead to fewer reinterventions and improved quality of life. There appears to be a graded economic benefit of focal dissection treatment, being cost-effective in non-severe dissections and even cost saving in severe dissections.

This study assessed the economic impact of reducing one postoperative visit following inflatable penile prosthesis (IPP) implantation. Scenario analyses were used to model the effects of eliminating one 30-min IPP postoperative visit from the expected 2.5 visits accounted for by the American Medical Association resource-based relative value scale data. The reduction was attributed to simplified teaching with a modified device. The recaptured time was applied to: the most frequent in-office CPT codes utilized by IPP implanters; evaluation and management of new ED patients pursuing/receiving IPPs; and in-office vasectomy. Physician work time and reimbursement were conservatively estimated using the 2024 Medicare Physician Fee Schedule and an alternative scenario where Advanced Practice Providers conducted IPP teaching was also modeled. Annually, reducing one 30-min IPP postoperative visit for practices performing 25/50/100 IPP implants recaptured 750/1500/3000 min, respectively. This recaptured time translates into as much as $18,325 additional annual Medicare reimbursement. At 25 implants yearly, urologists could help an additional 13-25 patients with office visits and observe an additional $2049-$2270 reimbursement. At 50 implants yearly, office evaluation and counseling for 7 ED patients who progress to IPP implantation results in an additional $4125 reimbursement, excluding any diagnostic procedures and/or downstream surgical cases. At 100 implants yearly, recaptured schedule capacity can facilitate 37 in-office vasectomies, which translates to a $12,563 reimbursement. Achieving fewer IPP postoperative visits can optimize postoperative care and open schedule capacity that improves access to care for patients with urological needs.

Warfarin and direct-acting oral anticoagulants (DOACs) are widely prescribed to patients with nonvalvular atrial fibrillation (NVAF) to reduce risk of stroke and systemic embolism (SE). This study aimed to assess the cost-effectiveness of apixaban compared with warfarin, dabigatran and rivaroxaban, for patients with NVAF from a US healthcare payer (Medicare) perspective. A cohort-level Markov model was developed based on a previously published model, for the US setting, factoring in anticipated price decreases due to market entry of generic drugs. Two retrospective cohort studies in US Medicare patients provided inputs to quantify clinical events in the base case setting and in a scenario analysis. For this study, equal value of life-years (evLYs) and health years in total (HYT) were used. Cost-effectiveness was assessed based on a willingness-to-pay threshold of $100,000 per evLY gained (evLYG) or HYT gained (HYTG). Apixaban treatment was associated with gains of 2.23, 1.08 and 1.72 evLYs and 2.26, 1.08 and 1.73 HYTs, compared with warfarin, dabigatran and rivaroxaban, respectively. In the base case analysis from a Medicare perspective, apixaban was cost-effective (i.e., value for money) compared with warfarin, dabigatran and rivaroxaban, with corresponding incremental cost-effectiveness ratio (ICER) per evLYG (and HYTG) of $10,501 ($10,350), $7809 ($7769) and $758 ($768), respectively. When a societal perspective was included, and in a scenario analysis using US Medicare data from the Ray study to quantify treatment effects, apixaban dominated rivaroxaban (i.e., less expensive and more effective) in terms of ICER per evLYG (and HYTG). Using dynamic pricing assumptions, treatment with apixaban compared with warfarin, dabigatran and rivaroxaban was associated with incremental evLYs and HYT and represents a cost-effective treatment option in patients with NVAF, from a US healthcare payer (Medicare) perspective.

To examine contributions of a patient advisory board (PAB) to the design and conduct of The Pulmonary Embolism Prevention after Hip and Knee Replacement (PEPPER) Trial (NCT02810704) and compare perceptions of PAB members and researchers on the Trial. This evaluation of the PAB was conducted by Clinical Coordinating Center (CCC) members who first discussed PAB contributions, leading to the design of a semi-structured WebEx interview individually querying PAB members on their experience. Two study team members analyzed transcriptions of the interviews for common themes, which were discussed and affirmed at an in-person meeting with PAB members. The contribution most frequently cited as meaningful by PAB members was the creation of a recruitment video. In contrast, the research team considered the most impactful PAB recommendation to be omission of pneumatic compression boots as a study variable. PAB members spoke highly of their involvement in the trial and emphasized shared decision-making in the patient-physician relationship. Researchers and PAB members had different opinions about which PAB contributions were most impactful to the study. This likely derives from differences in perspective; PAB members focused on patient experience and the patient-surgeon relationship while researchers focused primarily on trial outcomes. PAB contributions led to two major protocol changes that had a substantial positive effect on trial design, recruitment and enrollment. This evaluation adds to the engagement literature, which contains little on what patients think of their involvement in the design and conduct of clinical research studies and will aid in encouraging treatment preference discussions between patient and surgeon, thereby supporting the goal of improved patient outcomes.