Journal of Medical Economics

Introduction Melanoma, responsible for most skin cancer deaths globally, has mortality rates expected to double by 2040. Pembrolizumab is a highly selective antibody approved for melanoma treatment and other cancers. Despite new treatments for melanoma, high treatment costs and long approval times limit patient access to new therapies. To support decision-making regarding metastatic melanoma therapies, a model was developed to calculate the number needed to treat (NNT) and the cost of preventing an event (COPE) using KEYNOTE-716 (NCT03553836) data. Method A cost-per-responder model comparing the clinical and economic impacts of pembrolizumab versus best supportive care (BSC) was developed considering a 52.8-month follow-up for recurrence-free survival (RFS) and distant metastasis-free survival (DMFS) in patients with resected high-risk melanoma. KEYNOTE-716 RFS and DMFS survival curves were used to calculate restricted mean survival time (RMST). The RMST was used to calculate NNT (NNTRMST). The NNTRMST calculates the NNT to result in a difference in mean survival time for a death or an event. NNTRMST is subsequently used to quantify COPE outcomes. Results NNT for RFS was 5.3, reflecting the number of patients needed to treat to gain the additional difference observed in the mean RFS for resected high-risk type II (IIB and IIC) melanoma patients treated with pembrolizumab. For DMFS, the NNTRMST was 7.8. The estimated COPE to prevent an RFS or DMFS event was Mexican Peso (Mex $) 9,554,593 (2024) and Mex$13,961,427, respectively. Conclusions NNT values for RFS and DMFS data were both lower than the published average NNT value for current melanoma therapies. This demonstrated that fewer additional patients need to be treated in order to avoid a recurrence or a distant metastases event, compared to currently available melanoma therapies. The NNT and COPE highlight the clinical and economic impact of introducing pembrolizumab therapy for the treatment of patients in resected high-risk stage II melanoma.

Aims Systemic corticosteroids (SCS) are used to manage asthma exacerbations. Among the broad population of patients with asthma, SCS-related risk of adverse events (AEs), health care resource utilization (HCRU), and costs remain unclear. Materials and methods This retrospective cohort study used the Optum Research Database claims to identify adults with asthma from 1/1/2017 to 6/30/2022. The index date was the earliest SCS claim for SCS users; non-SCS users were randomly selected and adjusted proportionally to SCS users by index year. SCS use was measured during the first 12 months of follow-up. Inverse probability of treatment weighting balanced the two cohorts for selected baseline demographic and clinical characteristics. SCS users were further stratified into low, medium, and high dose sub-cohorts. SCS-related AEs were assessed up to 48 months, while HCRU and costs were assessed during the first 12 months of follow-up. A generalized linear model (GLM) analyzed follow-up costs by SCS exposure. Results The 130,739 patients included 55,363 non-SCS users (42.3%), while 75,376 were SCS users stratified into 60,319 low-, 12,235 medium-, and 2,822 high-dose users. The mean age was 49.6 years; 61.8% were female and 68.9% were non-Hispanic White. SCS users had a significantly greater risk of new-onset acute and chronic SCS-related AEs, increasing incrementally with dose exposure (all p < .001) across numerous physiological systems. Follow-up HCRU and costs also rose incrementally with dose exposure (all p < .001). Compared with non-users, SCS-related costs were 1.43, 1.97, and 3.21 times higher among low-, medium-, and high-dose users, respectively. The adjusted GLM predicted a 9.9% cost increase per 100 mg of prednisone equivalents. Limitations Retrospective administrative claims studies cannot randomize patients and may not capture all patient events. Conclusions Among a broad population of adults with asthma, even low doses of SCS were associated with significantly increased risk of new-onset AEs, HCRU, and costs.

Aims Migraine leads to substantial healthcare utilization and associated costs. However, much higher costs are attributed to lost productivity. The impact of effective migraine treatment on these costs, at the individual level, has not been well established. Even less known is the impact of treatment failure. The objective of this study was to assess lost earnings as a measure of migraine-attributed burden among triptan non-responders in Denmark. Materials and methods We used data from the Danish National Prescription Register and Danish Income Statistics Register over the 27-year period 1995–2021. We identified 4,979 triptan non-responders (85.9% female) and matched them for sex, age and region of residence with 14,292 continuing users of triptans (triptan responders) and 13,592 individuals from the background population (triptan never-users). We then estimated average annual individual earnings, and compared those among triptan non-responders, from 3 years prior to and 10 years after their last triptan redemption, with those among their matched triptan responders and triptan never-users over the same periods. Results Triptan non-responders earned significantly less than both their matched triptan responders and their matched triptan never-users. The earnings gap was evident even 3 years prior to the last triptan prescription (€4,344 and €4,356 respectively). This gap widened substantially over time, so that average cumulative earnings over the 14-year period of follow-up for each triptan non-responder were €93,684 less than those of responders and €99,485 less than those of never-users. Limitations There are uncertainties with regard to the reasons for triptan discontinuation (whether non-response or otherwise), and to lack of diagnostic confirmation of migraine. Conclusions Triptan non-response represents failure of currently available acute treatment options. It is associated with substantial and cumulative lost earnings, highlighting a disproportionate economic burden. These findings underscore the potential economic benefit of recognizing, and rectifying, unmet treatment needs in migraine management.

Objective von Willebrand Disease (vWD) is the most common congenital bleeding disorder, with an estimated prevalence in Spain of 0.01%. The aim was to assess the cost-utility of Haemate-P compared with present alternatives in the treatment of vWD in Spain. Methods A Markov model was developed in Microsoft Excel to estimate the cost-effectiveness of various treatments for vWD over a lifetime horizon. Transition probabilities among health states were based on age and number of bleeding events. Treatment strategies compared included Haemate-P, Fanhdi, and Wilate in long-term prophylaxis (LTP) or on-demand treatment (ODT). Costs and quality-of-life were measured based on patient age, treatment, and number of bleeding events incurred. Both costs and utilities were discounted at 3%. One-way and probabilistic sensitivity analyses were performed. Results When comparing LTP regimens, Haemate-P was less costly and numerically more effective than both Fanhdi (incremental costs = −€1,313,845; incremental quality-adjusted life-years [QALY] = 0.13) and Wilate (incremental costs = −€2,233,940; incremental QALY = 0.29). For ODT, Haemate-P was assumed to have equal effectiveness as Fanhdi and Wilate but reduced the costs by €696,857 and €1,145,780, respectively. Haemate-P prophylaxis was more effective and less costly compared with Haemate-P on-demand in the base case (incremental costs = −€633,317; incremental QALY = 0.90). Results were generally robust to sensitivity analyses. Conclusions In patients with severe vWD experiencing a high bleed rate, Haemate-P prophylaxis is a less costly and potentially more effective treatment strategy and Haemate-P is cost-saving among on-demand strategies.

Introduction A cell harvesting device for preparing non-cultured autologous skin cell suspension (ASCS) at the point-of-care is FDA-approved for repigmentation of stable depigmented vitiligo lesions in patients 18 years and older. The pivotal RSVP trial showed ≥80% repigmentation at Week-24 in 36% of lesions treated with laser ablation, ASCS, and narrowband ultraviolet B phototherapy compared to 0% with phototherapy alone (p = 0.012). The objective of this analysis was to evaluate the potential economic impact of laser ablation plus ASCS with phototherapy versus phototherapy alone for repigmentation of stable vitiligo lesions from a US payer perspective. Methods A 5-year decision-tree model was developed reflecting clinical pathways of adults with stable vitiligo lesions who had an inadequate response to prior topicals and phototherapy. Patients entering the model were treated with ASCS plus phototherapy or phototherapy alone and assessed for treatment response at Weeks-24 and 52 based on the RSVP trial’s effectiveness endpoints. Durable response for Year-2 onwards was proxied by melanocyte-keratinocyte transplantation data. Model outcomes included per-patient total and incremental healthcare costs, treatment costs and total costs, cost per-patient per-month (PPPM), and cost per-patient per-year (PPPY). One-way sensitivity analyses assessed model result robustness. Results The cumulative total per-patient cost for ASCS plus phototherapy increased from $28,177 to$92,779 between Year-1 and Year-5. Phototherapy alone increased from $21,146 to$101,518 over the same period. Compared to phototherapy alone, ASCS plus phototherapy incurred $7,030 more total per-patient cumulative costs in Year-1 and$8,738 less by Year-5 (-$146 PMPM; -$1,748 PPPY). Breakeven occurred between Years 2-3. Results were most sensitive to changes in ASCS response at Weeks-24 and 52 and healthcare costs. Conclusion Among adults with stable vitiligo with prior inadequate response to topicals or phototherapy, ASCS treatment may lead to lower all-cause direct medical costs over 5 years compared to phototherapy alone.

Objective The prevalence of chronic kidney disease (CKD) in Thailand is high and kidney disease progression remains a problem. Empagliflozin has been known to be used to slow CKD progression, but its accessibility remains limited. This study aimed to assess the cost-utility of empagliflozin for CKD progression in Thailand. Methods A state-transition model was developed consisting of eight health states: five eGFR health states (G2, G3a, G3b, G4, and G5), dialysis, kidney transplantation, and death. Empagliflozin 10 mg was assessed as an add-on treatment to standard of care (SoC). The efficacy of empagliflozin was derived from the EMPA-KIDNEY trial, while other inputs were obtained from a comprehensive literature review. The incremental cost-effectiveness ratio (ICER) per quality-adjusted life year (QALY) was calculated. A probabilistic sensitivity analysis (PSA) was performed to explore uncertainties. Results Empagliflozin could improve QALYs by 0.62 and 0.71 for patients with CKD without and with diabetes mellitus (DM) compared with SoC, respectively. However, it required higher total lifetime costs of 77,966 Thai baht (THB) and 59,454 THB for patients with CKD without and with DM, respectively. The ICER for CKD without DM was 126,201 THB/QALY, while the ICER for CKD with DM was 83,473 THB/QALY. The PSA indicated that empagliflozin had a 64.00% probability of being cost-effective for CKD without DM and an 89.18% probability for CKD with DM. Limitations An important limitation was that the treatment effects of empagliflozin were derived from the EMPA-KIDNEY, which was conducted in DM patients and assumed to be the same for non-DM patients because of the limited evidence in non-DM patients. Conclusion At the current willingness-to-pay threshold of 160,000 THB/QALY, empagliflozin was cost-effective for treating patients with CKD without or with DM.

Background Biosimilars to originator bevacizumab (Avastin), such as bevacizumab-bvzr (Zirabev), can deliver substantial savings and/or expanded access to biologic therapy for patients with metastatic colorectal (mCRC) and non-squamous non-small cell lung cancer (mNSCLC). The objective of this study is to explore the cost-efficiency and budget-neutral expanded access of bevacizumab-bvzr in mCRC and mNSCLC in Medicare. Methods We developed a Medicare payer perspective simulation model of patients treated for mCRC and mNSCLC to estimate cost-savings from converting bevacizumab (originator) to bevacizumab-bvzr or alternative biosimilars such as bevacizumab-awwb, -maly, and -abcd. The target patient population receiving annual first-line systemic therapy was calculated using Medicare enrollment data, SEER cancer incidence rates in patients age ≥65, and an assumption that 39.3% and 77.2% of new diagnoses receive systemic therapy for mCRC and mNSCLC respectively based on recent evidence. 76.0% of systemically treated mCRC patients and 11.4% of incident mNSCLC patients were expected to be treated with bevacizumab-based regimens based on recent evidence. Costs were derived from the 2024 Average Sales Price (ASP). Results include per-patient per-month (PPPM) cost savings (vs. originator), total monthly savings in the cohort, and number needed to convert (NNC) to biosimilar to fund the treatment of an additional 100 patients. Results PPPM savings from conversion to bevacizumab-bvzr were $4,205 in mCRC and$8,410 in mNSCLC. In 100% conversion scenarios, full cohort monthly savings were $27,664,432 in mCRC (n = 6,579) and$32,319,323 in mNSCLC (n = 3,843), respectively. At 100% conversion, monthly savings from biosimilar conversion could fund up to 13,887 additional mCRC patient-months of treatment with bevacizumab-bvzr + FOLFOX, and up to 8,959 additional mNSCLC patient-months of treatment with bevacizumab-bvzr + paclitaxel + carboplatin. In mCRC and mNSCLC the biosimilar NNC from the originator was 47 and 43, respectively. The biosimilar NNC from other biosimilars ranged from 60-4,564 and 55-4,422 for mCRC and NSCLC, respectively. Conclusion In the first cost-efficiency and expanded access study of biosimilar bevacizumab in mCRC and mNSCLC, we find that bevacizumab-bvzr-based regimens can result in substantial cost savings relative to originator-based first line treatment in Medicare. These cost savings could be reinvested to treat a substantial number of additional patients with mCRC or mNSCLC, or fund other costs of care in Medicare, on a budget-neutral basis.

Aim The aim of this study is to elucidate the prevalence, incidence, patient characteristics, and recent treatment trends of valvular heart disease (VHD) in Japan using a comprehensive claims database. Methods We conducted a cross-sectional study using the national database of health insurance claims in Japan (April 2009—June 2021), which contains data from the entire Japanese population, regardless of the type of medical facility. Descriptive analyses were conducted to examine the prevalence, incidence, and patient characteristics of each valve disease in 2020 based on diagnoses, and the treatment trends from 2009 to 2021. Results We identified 28,366,924 patients with VHD over the entire data period, and 2,473,070 patients in 2020, including 711,876 newly diagnosed. The prevalence and annual incidence in the entire Japanese population were 1.96% (1.88% in men and 2.04% in women) and 0.56% (0.53 and 0.60%), respectively, and increased with age in adults. Among the 8 types of VHD in combination with a disordered valve (aortic, mitral, tricuspid, or pulmonic) and type of valve disease (stenosis or regurgitation), mitral regurgitation had the highest prevalence followed by aortic regurgitation and tricuspid regurgitation. Heart failure was diagnosed in ≥50% of patients with aortic, mitral, or tricuspid disease, with the highest rate in mitral stenosis. The number of open-heart surgeries remained constant, while the number of transcatheter surgeries increased over time, particularly between 2016 and 2021. Aortic stenosis prevalence in transcatheter surgeries rose to ≥60% in 2014 and ≥80% in 2016. Limitations Diagnoses of VHD and comorbidity were based on claims data, so diagnostic criteria and disease severity are unknown, and misclassification of VHD types might have occurred. Incidence rates were based on the initial VHD diagnosis only, excluding any subsequent diagnoses of different VHD type. Conclusions: We presented basic information, which may provide an understanding of the clinical status of VHD in Japan.

PLAIN LANGUAGE SUMMARY What is already known about the topic? Respiratory syncytial virus (RSV) is an important cause of respiratory illness in children, and, in the worst cases, can require hospital care. To maximize the effectiveness of drugs designed to prevent RSV by temporarily boosting the child’s immune system (e.g. palivizumab and nirsevimab), their use needs to be matched to RSV activity in the community. Wastewater and environmental monitoring (WEM) is a technology that can detect the presence of viruses in sewage samples to show when specific diseases are increasing. What does the paper add to existing knowledge? A key question to answer for any investment in a new technology is whether the expected benefits justify the costs associated with setup and use. Cost-utility analysis sets the financial cost of a new technology against any savings it may produce plus the value of any improvements to people’s everyday life. This first assessment of the cost-utility of WEM compares it to clinical surveillance (CS), which is the current standard for assessing RSV activity. The results showed WEM to be a cost-saving approach over CS to guide a Canadian all-infant immunization program with nirsevimab. This means that the costs of implementing the WEM program can be offset by the savings from reducing the need for medical care. What insights does the paper provide for informing healthcare-related decision making? Our cost-utility analysis on the integration of RSV-WEM provides practical information for both provincial and local public health authorities to utilize and supports the implementation of such resources in Canada and other countries. Adoption of RSV-WEM has the potential to precisely guide the initiation of RSV immunization programs which will help reduce avoidable illness and hospitalization, creating cost savings and improving the lives of the protected children and their parents.

Background In contrast to clinical effectiveness of resective epilepsy surgery (RES) for patients with drug-resistant epilepsy, societal costs of RES is still unclear. The aim of this study was to report on total societal costs up until two years after surgery and analyse the trend of post-surgical costs over time. Secondary objectives included assessing quality of life (QoL) changes and identifying determinants of post-surgical costs. Methods Data were derived from the patients’ entire medical history based on hospital files and accompanied by validated questionnaires before and 3-, 6-, 12-, and 24-months post-surgery to additionally include medical consumption outside of the hospital, productivity losses and gains, and QoL. To explore the trend of post-surgical costs over time and identify determinants of post-surgical costs, linear mixed effects and linear regression models were performed. Results The study included 44 patients. Mean complete costs from diagnostics and treatment strategies in the period before referral for pre-surgical evaluation up until two years after RES were €121,856 (Interquartile range = €76,058−€137,027). Post-surgical costs significantly decreased 12 months (mean 3-month difference = €−6,675, p = 0.000) and 24 months (mean 3-month difference = €−7,690, p = 0.000) after surgery compared to 3 months before surgery. Higher post-surgical costs were associated with a clinically relevant increase in disease-specific QoL after RES (p = 0.000), previous ketogenic diet (p = 0.005), RES in the left hemisphere (p = 0.014), previous RES (p = 0.007), and higher diagnostics and treatment strategies costs before referral for pre-surgical evaluation (p = 0.021). For disease-specific and generic QoL, 20 (45%) patients reached a clinically relevant QoL increase two years after surgery compared to before RES. Conclusion In conclusion, RES leads to significant reduction in costs 2 years post-surgery. History of RES and ketogenic diet, clinically relevant disease-specific QoL increase, surgery in the left hemisphere, and higher costs of diagnostics and treatment strategies before referral for pre-surgical evaluation were significant determinants for higher post-surgical costs after RES.

Aim We assessed the relationship between hospital septal reduction therapy (SRT) procedural volume and clinical outcomes, healthcare resource utilization, and hospital costs. Methods This cross-sectional study used 2012–2022 US hospital data from the PINC AI Healthcare Database for adults with hypertrophic cardiomyopathy (HCM) undergoing alcohol septal ablation (ASA) or septal myectomy (SM; with or without mitral valve repair or replacement [MVRR]). We categorized hospital procedural volume into tertiles according to the numbers of procedures performed and made pairwise comparisons of patient characteristics, clinical events, healthcare utilization, and hospital costs between tertiles. We conducted multivariable analyses (adjusted for patient, clinical, and hospital characteristics) for index hospitalization length of stay, cost, and 30-day readmission rates. Results Overall, 3,068 patients with HCM (across 315 hospitals) underwent SRT (ASA: 1,400; SM: 1,668). Index visit in-hospital mortality was 1.1–1.5% among individuals undergoing ASA, 3.2–7.4% for SM with MVRR, and 2.8–3.8% for SM without MVRR. There were no significant differences in in-hospital mortality or stroke/transient ischemic attack at index visits between the hospital procedural volume tertiles for ASA or SM. Adjusted hospital length of stay, costs, and readmission rates were significantly greater in low-volume than high-volume hospitals for ASA (p < 0.001). Similar trends were reported for SM for length of stay and costs (p < 0.001). Limitations This study relied upon accurate and complete reporting of diagnoses and procedures by hospitals. Patients were not randomly assigned, potentially leading to selection bias. Only in-hospital costs were evaluated. Follow-up events were only captured if they occurred in the same healthcare facility. Conclusions Resource utilization and in-hospital costs for patients undergoing SRT are lower in high procedural volume hospitals than low procedural volume hospitals. SRT procedure volume remains low even in hospitals with the highest relative procedural volumes, highlighting a need for globally accessible therapies that improve outcomes.

Aims To quantify preferences for an adult combination vaccine for influenza and COVID-19 (flu + COVID) compared with standalone influenza and COVID-19 vaccines. Materials and methods This survey study used a series of direct-elicitation questions to assess preferences for a single-shot combination flu + COVID, standalone influenza, and standalone COVID-19 vaccines among US consumers (N = 601) and healthcare professionals (HCPs) (N = 299). Response frequencies described the proportion of each sample that would prefer a flu + COVID vaccine to standalone influenza and COVID-19 vaccines. A multivariate logit regression model explored how certain characteristics influenced the odds of selecting the flu + COVID vaccine over a standalone influenza vaccine. Results Most consumers (398/601; 66.2%) and HCPs (250/298; 83.9%) preferred a flu + COVID vaccine to a standalone influenza vaccine. When not forced to choose between flu + COVID and standalone influenza vaccines, most consumers again selected the flu + COVID vaccine (62.3%); 14.7% would prefer separate standalone influenza and COVID-19 vaccines, 8.3% a standalone influenza vaccine only, 7.3% a COVID-19 vaccine only, and 7.4% neither vaccine. Consumers aged ≥50 years with a body mass index ≥40, those aged ≥65 years who previously received a COVID-19 vaccine, and those who had previously experienced severe impacts from influenza were more likely to choose a flu + COVID vaccine over a standalone influenza vaccine than were consumers without these characteristics. HCPs whose practice stocks high-dose influenza vaccines were more likely to choose the flu + COVID vaccine for patients aged ≥65 with no risk factors and patients aged 18–64 with ≥1 risk factor over the standalone influenza vaccine. Limitations Results are subject to potential hypothetical, responder, selection, and information biases. Conclusions Most US consumers and HCPs would likely prefer a single-shot combination flu + COVID vaccine compared with standalone influenza and COVID-19 vaccines. Given the low COVID-19 vaccination coverage rates in the US, the availability of a combination flu + COVID vaccine could help increase COVID-19 vaccine coverage.

Aim To compare all-cause and mental health (MH)–related short-term and long-term disability leaves and associated costs among patients in the United States with bipolar disorder (BP), major depressive disorder (MDD), or schizophrenia spectrum disorders (SCZ) before versus after cariprazine initiation. Methods Merative MarketScan Commercial and Health and Productivity Management (HPM) databases (January 2016 to December 2021) were utilized to identify adults diagnosed with BP, MDD, or SCZ with ≥2 pharmacy cariprazine claims (first claim = index), ≥3 months of cariprazine use (adjunctively for MDD), and continuous commercial insurance coverage and HPM eligibility during baseline (12 months pre-index) and ≥3 months post-index. Observation continued until cariprazine discontinuation, insurance or HPM eligibility end, 1 year post-index, or HPM data availability end. All-cause and MH-related disability claims, days, and costs were evaluated. Baseline versus post-index rates of disability claims (events) and days were compared using rate ratios (RR); costs were compared using mean cost differences. Comparisons were calculated from generalized estimating equation models. Analyses were replicated separately across indications. Results There were 489 patients overall (BP = 238, MDD = 233, SCZ = 18; mean age = 43.3 years; 60.7% female; mean follow-up = 7.6 months). All-cause rates of disability events and days following cariprazine initiation were 29% (RR = 0.71 [95% CI = 0.57, 0.86]) and 28% (0.72 [0.53, 0.94]) lower than baseline, respectively (both p < .05). MH-related rates of disability events and days were 40% (0.60 [0.43, 0.80]) and 43% (0.57 [0.34, 0.84]) lower, respectively (both p < .01). All-cause disability costs were $2,917 lower and MH-related disability costs were$2,482 lower than baseline (40% and 51% decrease, respectively; both p < .01). Results were similar for indication-specific analyses. Limitations Limited generalizability to patients who are unemployed, uninsured, or have public insurance. Conclusions Rates of disability events, days, and mean costs were significantly lower after versus before cariprazine initiation. These results can help contextualize cariprazine’s role in managing disability for these patients.

Aims To predict the budget impact of Symvess (Symvess is a trademark of Humacyte Global, Inc.) (acellular tissue engineered vessel-tyod [ATEV]) for extremity arterial trauma repair when autologous vein repair is not feasible. Materials and methods The 3-year budget impact of adding ATEV as a repair option alongside autologous vein, prosthetic graft, and “non-autologous other” grafts was evaluated from the perspectives of a Level I trauma center and third-party commercial payers. Conduit-specific complication rates were obtained from two clinical studies for ATEV and from the published literature and analysis of the PROOVIT registry for other conduits. Costs were compared pre- and post-ATEV availability. Conduit-related costs and complications included conduit infections, amputations, vein harvest site infection, surgical re-interventions, rehabilitation after amputation, and 12-month post-discharge costs. Impact on operating room (OR) time and readmissions was evaluated. A sensitivity analysis was conducted to evaluate parameter uncertainty. Results With introduction of ATEV, there was a 29.8% reduction in amputations and a 29.5% reduction in graft infections over 3 years. From a Level I trauma center perspective, seven patients were expected to receive an ATEV over 3 years, with cumulative cost savings of $80,650 (2.3$0.08 per member per month after 3 years. For trauma centers, sensitivity analysis showed that cost drivers were amputation risk associated with “non-autologous other” graft types and market share of autologous vein (short ischemia time). Limitations Uncertainty surrounding model parameters. Conclusions ATEV was projected to be cost-saving over 3 years for both trauma centers and third-party payers due to reductions in the costs related to amputations and conduit infections.

Aim Although liver biopsy is considered the most reliable diagnostic tool for metabolic dysfunction–associated steatohepatitis (MASH), it is invasive and can be costly. Clinicians are increasingly relying on routine biomarkers and other noninvasive tests (NITs) for diagnosis. We examined real-world diagnostic pathways for patients newly diagnosed with MASH with a primary focus on NITs. Materials and methods This retrospective, observational study analyzed healthcare claims data (Merative MarketScan Commercial and Medicare Databases) from patients in the United States newly diagnosed with MASH from October 1, 2016, to March 31, 2023. Patients ≥18 years old with ≥12 months of continuous enrollment with medical and pharmacy benefits prior to diagnosis were included. Diagnostic pathways leading up to MASH diagnosis, including NITs (blood-based and imaging-based tests) and liver biopsies were assessed. Prevalence of comorbid conditions, MASH-associated medication use, and the diagnosing physician specialty were also examined. Results A total of 18,396 patients were included in the analysis. Routine laboratory tests (alanine aminotransferase [ALT], albumin, aspartate aminotransferase [AST], cholesterol, complete blood count, and hemoglobin A1c) were performed among ≥70% of patients prior to MASH diagnosis, including 89% of patients with a liver enzyme test (ALT and/or AST). More than 75% of patients had necessary laboratory tests to calculate AST to platelet ratio index (APRI) and fibrosis-4 index (FIB-4) scores. The most common imaging performed was ultrasound (62%); liver biopsy was only performed in 10% of patients. There was a high prevalence of cardio metabolic risk factors such as hyperlipidemia (66%), hypertension (62%), obesity (58%), type 2 diabetes (40%), and cardiovascular disease (21%). Nearly half of the patients (49%) were diagnosed by a primary care physician. Limitations and conclusions This study highlights real-world diagnostic pathways among patients newly diagnosed with MASH, supporting previous findings that liver biopsies are infrequently used in favor of noninvasive methods.

Objective This study aimed to investigate the willingness to pay (WTP) of the Japanese population for the transmission prevention function of SARS-CoV-2 antiviral treatments and identify the attributes associated with higher WTP. Methods A web-based survey (registration number: UMIN000054955) was conducted from May 17 to June 1, 2024, targeting a general population using a survey company panel. We aimed to obtain around 3,000 valid responses. Respondents were randomly divided into two groups: one assuming a COVID-19 infection (infection-assumed group) and the other without this assumption (non-infection-assumed group). WTP was assessed using an open-ended question format, asking how much they would be willing to pay out-of-pocket for a hypothetical antiviral drug that reduces the risk of transmitting COVID-19 to others by half. The survey also collected demographic information, COVID-19 related attributes, empathy levels using the Multidimensional Empathy Scale (MES), and health literacy using the Communicative and Critical Health Literacy scale. The mean WTP for COVID-19 treatment was calculated for all respondents and for the infection-assumed and non-infection-assumed groups. Subgroup analyses examined the effects of respondent attributes on WTP. A linear regression model with stepwise selection identified factors associated with WTP. Results Responses were obtained from 3,657 individuals, with 3,131 valid responses analyzed. The mean WTP among all respondents was JPY 3,205 (USD 20.85) (standard error: JPY 84 [USD 0.55]). The infection-assumed group showed a 21% higher WTP than the non-infection-assumed group (p < 0.001). Subgroup analyses indicated that WTP varied based on attributes such as co-residing children, occupation, empathy levels, and health literacy. Higher WTP was significantly associated with being aged 65 years and older, higher household income, absence of co-residing children, being a company employee, executive, or public servant, fear of COVID-19 infection, higher other-oriented emotional reactivity (a factor of MES), and higher health literacy. Conclusion We presented the WTP of the Japanese population for the transmission prevention function of COVID-19 treatments as an actual monetary value. Factors such as empathy, health literacy, and some attributes were significantly associated with WTP. These findings might help inform policymakers in developing health policies based on the universal health insurance system in Japan.

Aims The cardioprotective effects of semaglutide 2.4 mg reported in the SELECT cardiovascular (CV) outcomes trial (ClinicalTrials.gov NCT03574597) provide clinical benefit for subjects with overweight or obesity and established CV disease without type 2 diabetes (T2D). We assessed cost-effectiveness of semaglutide 2.4 mg in this population against the American College of Cardiology/American Heart Association value framework. Materials and methods A cohort-level Markov-state cost-effectiveness model using trial-derived data with outcomes from a healthcare sector perspective measured over a lifetime horizon was developed. Treatment costs were based on US list prices; scenario analyses used literature-reported estimated rebates. Healthcare costs and benefits were discounted at 3.0%. A simulated cohort of 100,000 subjects was aligned to the SELECT trial population baseline characteristics and time-on-treatment. Subjects received either semaglutide 2.4 mg or placebo in addition to standard of care (SoC). Modelled outcomes included clinical events (CV events, progression to T2D, chronic kidney disease [CKD]) and health economic measures, including direct costs and quality-adjusted life years (QALYs). Results Mean semaglutide 2.4 mg treatment duration was 2.79 years. Per 100,000 subjects, treatment avoided 2,791 non-fatal myocardial infarctions, 3,000 coronary revascularizations, 487 non-fatal strokes, and 115 CV deaths over the modeled lifetime horizon. Average per-subject lifetime treatment costs were $47,353; savings arose from avoided T2D ($14,431), CKD ($2,074), and CV events ($1,512). Semaglutide 2.4 mg was associated with increased lifetime costs ($29,767), additional QALYs gained (0.218) and an incremental cost-effectiveness ratio of$136,271/QALY at list price; a scenario using an empirically estimated 48% rebate predicted $32,219/QALY. Limitations The generalizability of observations from SELECT to a broader US population is unknown. Our model does not capture all outcomes nor costs that may be affected by weight loss. Modeling assumptions may present limitations. Conclusions Semaglutide 2.4 mg use as in SELECT is cost-effective at list price, using a$150,000/QALY willingness-to-pay threshold.

Objective/Aim In 2009, dronedarone was approved by the United States Food and Drug Administration based on results from the ATHENA trial (NCT00174785), which showed significant reduction of cardiovascular (CV) hospitalization and death in patients with atrial fibrillation (AF) randomized to dronedarone versus placebo. In 2020, a retrospective study by Goehring et al. showed CV hospitalizations and deaths were lower in clinical practice following initiation of dronedarone compared to other antiarrhythmic drugs (AADs) in patients with AF and atrial flutter. However, the economic impact associated with dronedarone use has not been fully assessed. The objective of this study was to estimate the cost associated with CV outcomes reported by Goehring et al. (2020). Methods National average Medicare payments in the Centers for Medicare and Medicaid Services (CMS) database (www.data.CMS.gov) were used to assign cost estimates to CV outcomes evaluated in Goehring et al. (2020) by diagnosis-related grouping. When costs were unavailable in the CMS database, a literature search was performed to identify publications reporting hospitalization costs. Results The weighted average cost for CV hospitalization was calculated to be $20,508. When multiplied by the event rate reported in Goehring et al. (2020), cost per person year for CV hospitalization was 14$3,679 vs $4,272, respectively). For hospitalizations due to heart failure, cost was 31$324 vs $472, respectively). Limitations Costs have been calculated based on national averages reported by CMS (Medicare perspective) and are estimates. Regional differences may be present. Conclusions Patients with AF taking dronedarone had lower costs associated with CV hospitalization compared with patients taking other AADs.

Background Pneumococcal diseases (PD) caused by Streptococcus pneumoniae include invasive PD (IPD) and non-bacteremia pneumococcal pneumonia (NBPP). Current French vaccination guidelines focus on patients with underlying medical conditions (UMC) who are at a higher risk of PD. This study describes the healthcare resource utilization (HCRU) and economic burden of inpatient PD in French adults, to inform vaccination guidelines, especially among vulnerable subpopulations at increased risk of PD. Methods A retrospective study utilizing the French administrative healthcare database (SNDS) was conducted among adults with an inpatient PD episode between 2015 and 2018. HCRU and costs were described per inpatient PD episode, according to patient risk level, type of PD, and age group. Results Between 2015 and 2018, 42,466 inpatient PD episodes were identified. Most of the inpatient PD episodes (73.7%) occurred in patients with UMCs. The median (Q1–Q3) cost per inpatient PD episode seemed higher among medium-risk patients (€14,863 (€7,875–€30,434)) than among low-risk (€11,034 (€5,803–€23,098)) and high-risk patients (€13,258 (€7,143–€26,815)). Cost per inpatient PD episode did not seem to increase steadily with age, however, ≥65 patients represented 59.5% of all inpatient PD episodes and 52.3% of the overall inpatient PD episodes cost (€548,224,569 out of €1,049,214,069). Limitations Due to constraints of the SNDS dataset, results are limited to inpatient episodes of PD and may not be representative of all PD episodes in France. Conclusion This large, retrospective study highlights a substantial economic burden associated with inpatient PD in France, especially among individuals with UMCs and those aged 65 years or over. These results emphasize the need to improve prevention strategies, especially among older patients, regardless of their risk level.

Aim Inadequate response to antidepressant therapy (ADT) is common in major depressive disorder (MDD); atypical antipsychotic (AA) adjunctive therapy may be effective for these patients. This study aimed to compare healthcare resource utilization (HRU) and costs between patients initiating the AA cariprazine as their first adjunctive therapy vs those initiating cariprazine subsequently. Methods The Merative MarketScan Commercial Database (January 1, 2015, to June 30, 2021) was used to identify US adults with MDD and ≥1 pharmacy claim for cariprazine adjunctive to ADT in 2018 or after. Rates of mental health (MH)‑related and all‑cause HRU per patient-year (PPY) and mean healthcare costs per-patient-per-year (PPPY) were assessed after patients first initiated adjunctive therapy. HRU and costs were compared between cohorts using rate ratios (RRs) and mean cost differences, respectively, estimated from multivariable regression models. Results Of 838 patients receiving cariprazine, 44.7% initiated cariprazine as their first adjunctive therapy to ADT, and 55.3% initiated it subsequently. Those initiating cariprazine first had significantly lower rates of MH‑related hospitalizations (RR [95% confidence interval] = 0.55 [0.30, 0.90], p = .020) and outpatient (OP) visits (0.67 [0.57, 0.82], p < .001) PPY than those initiating cariprazine subsequently. Moreover, patients initiating cariprazine as their first adjunctive therapy had lower annual total MH‑related healthcare costs (mean cost difference [95% confidence interval] −$2,182 [−$4,206, −$69], p = .040), driven primarily by lower OP visit costs (−$1,511 [−$2,330, −$615], p < .001). Similar trends were observed for all-cause HRU and costs. Limitations This was a retrospective analysis of secondary data with limited follow-up. Claims were a proxy for cariprazine use. Conclusions Results from this real‑world study of commercially insured US adults suggest that initiating cariprazine as the first adjunctive therapy rather than a subsequent therapy could help mitigate the considerable economic burden of MDD for appropriate patients.

Aims Direct-acting oral anticoagulants (DOACs) have emerged as the preferred treatment for nonvalvular atrial fibrillation (NVAF). However, evidence concerning the economic outcomes of DOAC switching remains limited. This study aimed to assess the economic outcomes of DOAC switching in the US and Germany, two countries with a high AF prevalence and DOAC utilization. Methods A decision model was developed to assess the incidence and cost of stroke/systemic embolism (SE) and major bleeding (MB) associated with switching from apixaban to rivaroxaban in patients with NVAF. The model compared two scenarios: continuers (patients continuing apixaban) and switchers (patients switching from apixaban to rivaroxaban). Model inputs on clinical event rates were sourced from a published real-world study, cost inputs were from a standard costing database and published literature. The analysis was conducted over a 1-year time horizon from US Medicare fee-for-service and German public healthcare payer perspectives. Results Over one year, 47,036 patients among a hypothetical plan size of 1,000,000 US Medicare fee-for-service members and 1,019,079 patients among the German adult population size of 70,107,122 were estimated to be treated for NVAF with apixaban. Switching all patients from apixaban to rivaroxaban resulted in 1,498 and 32,447 additional clinical events (stroke/SE and MB) and deaths in the US and Germany, respectively, compared to continuing with apixaban. This led to a total incremental cost of $17.3 million and €153 million from Medicare fee-for-service and German public healthcare perspectives, respectively. Limitations The incidence and hazard ratios of clinical events informing this analysis were based on a US commercial and Medicare Advantage population and may not be generalizable to other populations. Conclusions Switching from apixaban to rivaroxaban was associated with increased clinical events, deaths, and higher medical care costs, potentially representing a less favorable strategy economically compared to continuing apixaban among patients with NVAF.

Background Psoriasis is a chronic, systemic, inflammatory skin disease, with increasing prevalence; however, few studies have reported real-world prescription patterns and healthcare burden. Objectives This retrospective, observational cohort study used statutory health insurance claims data (January 2014–December 2019) to estimate prevalence/incidence of moderate-to-severe psoriasis in Germany. Patient characteristics, treatment patterns/compliance, and healthcare resource utilization (HCRU)/costs were evaluated, focusing on apremilast and anti-interleukin (IL), and anti-tumor necrosis factor (TNF) biologics. Methods The epidemiology population included adults with psoriasis; 1-year prevalence/incidence rates were extrapolated to the statutory health insurance population. The HCRU/costs population included adults with psoriasis and a first prescription for a drug of interest (index date). Baseline periods were 12 or 48 months before the index date, with 12‑month follow-up. Results In 2019, the estimated psoriasis prevalence/incidence was 2,672.9 per 100,000 individuals/508.7 per 100,000 person-years. Of 2,809 patients in the HCRU/costs population, 3.6% (n = 101) received index drug apremilast, 10.2% (n = 287) anti-IL, 6.8% (n = 191) anti-TNF, and 79.4% (n = 2,230) traditional/other systemic therapy. Patients initiating apremilast were older and were more often biologic-naïve than those initiating anti-IL/TNF biologics. Twelve months after treatment initiation, drug adherence (medication possession rate >80%) and persistence (<60 days between prescriptions/no switch) were lower for apremilast vs. anti-IL and anti-TNF groups (24.8% vs. 59.6% and 53.9%; 36.6% vs. 66.9% and 57.6%, respectively). During a 12-month baseline period, psoriasis-related hospitalization was lower for apremilast vs. anti-IL and anti-TNF groups (4.95% vs. 15.68% and 14.14%) and higher during 12 months’ follow-up (5.94% vs. 2.44% and 3.14%). Adjusted index drug costs during follow-up were €4,105, €3,498, and €13,777 higher for adalimumab, other anti-TNF and anti-IL biologics vs. apremilast, respectively, and the main driver for lower overall apremilast costs. Conclusion Given variation in treatment adherence/persistence, HCRU, and costs between apremilast and biologics, these findings could be key considerations during treatment selection.