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The overall value of treatments for chronic lymphocytic leukemia (CLL) depends on several factors, including preferences of the general population, who contributes to the financing of health systems. This study investigated societal preferences for attributes of CLL treatments in Italy. An online large-scale survey was designed using a discrete choice experiment (DCE) methodology and delivered to the Italian adult general population. Ten treatment attributes were identified, covering efficacy, safety, operational aspects and (hypothetical) out-of-pocket cost. DCE data were analyzed using a mixed logit regression model, estimating the willingness-to-pay for attribute levels' change. The general population significantly preferred more effective treatments, with shorter duration, administered orally rather than orally + intravenously. Changes in therapy duration, frequency of checkups and organ damage risk had the greatest impact on preferences. The integration of societal preferences in the value judgments of CLL therapies may help health authorities in establishing priority setting and taking pricing-reimbursement decisions.

Background: Recent practice guidelines favor direct oral anticoagulants (DOACs) over warfarin for primary stroke prevention in patients with non-valvular atrial fibrillation (NVAF). However, challenges persist in Iraq's private pharmaceutical sector. DOACs have been sold at high and inconsistent retail prices and lack insurance coverage, leading to significant out-of-pocket (OOP) costs. The objective of this study is to investigate the impact of OOP costs on oral anticoagulants (OAC) adherence among NVAF patients., Research Design and Methods: This multicenter cross-sectional study interviewed 359 eligible patients attending three private cardiology clinics within Iraq's southern region from December 2022 to February 2023. The 8-item Morisky Adherence Scale evaluated patient adherence. Statistical analyses, including descriptive analysis, ANOVA, and chi-square. p  < 0.05 was considered statistically significant., Results: The most frequently prescribed OAC were DOACs (62.8%). Patient adherence level to OAC was chiefly medium (54.6%) with no significant difference in adherence based on OAC type. Patient adherence was significantly associated with monthly income ( p  = 0.001), number of daily pills ( p  = 0.006), and OACs' average monthly cost ( p  = 0.011)., Conclusion: Addressing the issue of cost-related non-adherence to OACs requires multiple actions. These include ensuring comprehensive health insurance coverage for OACs, increasing the use of affordable generic alternatives, and establishing effective cost-related discussions between healthcare providers and patients.

Aims: To evaluate the healthcare resource utilization (HRU) and costs of patients who initiated cariprazine as their first versus subsequent atypical antipsychotic (AA) following a bipolar I disorder (BP-I) diagnosis., Methods: Adults with a BP-I diagnosis (first claim = index), commercial, Medicare Supplemental, or Medicaid insurance, and ≥1 outpatient cariprazine dispensing were identified from Merative MarketScan database. Cohorts included patients who initiated cariprazine as either their first or subsequent AA after initial BP-I diagnosis. Characteristics were balanced between cohorts using inverse probability of treatment weighting (IPTW). Outcomes evaluated post-index included all-cause and mental health (MH)-related HRU (hospitalizations, emergency department [ED] visits, outpatient visits), total healthcare costs (medical + pharmacy), and treatment patterns. HRU and healthcare costs were reported per patient-year (PPY) and compared between cohorts using rate ratios and 95% CIs estimated using nonparametric bootstrap procedures. Treatment patterns were analyzed descriptively, with standardized differences ≥10% considered important., Results: After IPTW, cohorts included 1,409 patients who initiated cariprazine first and 1,621 patients who initiated cariprazine subsequently; the average (standard deviation, SD) observation period was 678 (373) and 758 (389) days for first and subsequent initiators, respectively. Patients who initiated cariprazine first had 23% fewer all-cause hospitalizations and 28% fewer MH-related hospitalizations PPY (each comparison, p  < 0.001). Rates of all-cause and MH-related outpatient visits were significantly lower in patients who initiated cariprazine first versus subsequently (each comparison, p  < 0.001), while rates of ED visits were similar. Relative to subsequent initiators, first initiators incurred $2,587 and $2,130 lower all-cause and MH-related total healthcare costs PPY, respectively (each comparison, p  < 0.05). Before starting cariprazine, first initiators used fewer BP-I-related medications on average than subsequent initiators (2.6 vs 3.9; standardized difference = 23.9%)., Limitations: Potential coding inaccuracies and residual confounding., Conclusions: In this real-world database analysis, patients with BP-I who initiated cariprazine as their first AA had lower rates of HRU and incurred lower costs than patients who initiated cariprazine as a subsequent AA.

Aims: This study evaluated the association between provider types for patients with newly diagnosed Huntington's disease (HD) and healthcare resource utilization (HCRU), costs, and treatment patterns., Materials and Methods: This retrospective analysis used MarketScan databases (1 January 2017-31 December 2021) to identify provider types who diagnosed and managed US adult patients with HD. Patients with continuous enrollment 6 months pre- and 12 months post-diagnosis were included. Outcomes evaluated over 12 months post-diagnosis included hospitalizations, outpatient visits, antipsychotic or vesicular monoamine transporter 2 (VMAT2) inhibitor use, and total healthcare costs., Results: Three hundred and forty eligible patients had a mean age at diagnosis of 49 years. 56.5% were female; 71.5% had a Charlson Comorbidity Index of 0. Patients were diagnosed by neurologists (48.5%), primary care providers (PCPs) (35.6%), psychiatrists (3.5%), or other providers (12.4%). Patients diagnosed by PCPs or neurologists received significantly more follow-ups by the same diagnosing provider type ( p  < 0.05). All-cause and HD-related outpatient visits at 12-month follow-up had more patients diagnosed by PCPs (23.9, 5.1) than neurologists (18.0, 2.4), psychiatrists (16.7, 1.67), or others (15.3, 2.4). HD-related mean costs totaled $2,489 ($1,179 inpatient and $1,310 outpatient). Patients diagnosed by neurologists had significantly lower HD-related total non-medication costs vs. those diagnosed by PCPs (-$2,256; p  < 0.05). Among patients diagnosed by neurologists vs. PCPs, similar proportions received antipsychotics within the first year (55 vs. 52%, respectively); more patients managed by neurologists received VMAT2 inhibitors (12 vs. 7%, respectively)., Limitations: Our study includes limitations inherent to retrospective claims studies., Conclusions: Patients with HD are most often diagnosed by neurologists or PCPs; the same diagnosing provider type typically manages follow-up. Patients diagnosed by neurologists had significantly fewer HD-related outpatient visits, lower HD-related non-drug costs, and more frequently received VMAT2 inhibitors vs. those diagnosed by PCPs. Our findings show an integrated care team may provide evidence-based, personalized care for patients with HD.

Aims: To compare healthcare resource utilization (HRU) and costs between patients with or without melanoma recurrence and between patients with distant or locoregional melanoma recurrence., Methods: Patients aged ≥65 years with completely resected, stage IIB/IIC or III melanoma were identified from Surveillance, Epidemiology, and End Results-Medicare data and stratified based on whether they experienced a recurrence, and whether it was distant or locoregional (separately for each stage). The index date was the date of recurrence (recurrence group) or a randomly assigned date (non-recurrence group). Patients in the recurrence and non-recurrence groups were propensity score-matched 1:1 based on patient characteristics; HRU and healthcare costs were compared between the 2 groups and between patients with distant or locoregional recurrence during the ≤24 months following index., Results: After matching, 507 pairs of patients with recurrent or non-recurrent stage IIB/IIC melanoma (236 patients with distant recurrence, 271 with locoregional) and 141 pairs of patients with recurrent or non-recurrent stage III melanoma (50 patients with distant recurrence, 91 with locoregional) were included. During the first year following recurrence, unadjusted HRU was generally higher in patients with versus without recurrence and patients with distant versus locoregional recurrence among both stage IIB/IIC and III cohorts. Patients who experienced recurrence incurred $6,474 (stage IIB/IIC) or $6,112 (stage III) per patient per month (PPPM) more in unadjusted, all-cause, total healthcare costs than patients without recurrence (both p  < 0.001). Patients with distant recurrence incurred $7,292 (stage IIB/IIC) or $5,436 (stage III) PPPM more in unadjusted, all-cause, total healthcare costs than patients with locoregional recurrence (both p  < 0.05)., Limitations: Melanoma recurrence was identified using a claims-based algorithm., Conclusions: Economic burden is higher in patients with versus without melanoma recurrence and patients with distant versus locoregional recurrence. There is a high unmet need for adjuvant therapies that may help to prevent or delay recurrence.

Aims: In advanced Parkinson's disease (aPD), adequate 24-hour control of OFF-time may not be achievable using oral/transdermal therapies. Clinical trials of foslevodopa/foscarbidopa (LDp/CDP) demonstrate meaningful reductions in OFF-time and OFF-related sleep disturbance in aPD. Previous analyses have only considered direct medical costs: this analysis considers a broader societal perspective (direct non-medical costs, informal care, loss of earnings, productivity, and tax)., Methods: Inputs for the societal impact model were taken from a cost-utility model comparing LDp/CDp with best medical treatment (BMT), accepted by the UK National Institute of Health and Care Excellence (NICE). Quintiles of normalized OFF-time across a 16-hour waking day in each treatment group were applied to literature-based estimates for direct medical, non-medical, and indirect costs. The resulting state-specific cost estimates were applied to the modelled aPD patient population., Results: The model estimates the potential UK population for LDp/CDp at 17,505. Continuous 24-hour delivery of LDp/CDp results in greater time spent in OFF-time states 0-1 (0-4 hours of OFF-time/16-hour waking day) vs BMT alone. Net savings if all eligible patients receive LDp/CDp are £79.1 M in year 1, £235.4 M in year 2, rising to £262.2 M in year 3, declining to £222.9 M in year 4 and £153.7 M in year 5 as disease progresses and the efficacy of LDp/CDp declines. The estimated total net savings are £953 M after 5 years. Results are robust in scenario analyses (excluding costs of excessive sleepiness, earnings loss, productivity, and tax loss)., Limitations: A NICE-accepted model was used as the economic modelling basis for the societal impact model, however, much of the data was derived from Adelphi datasets, with the potential for inconsistent definitions., Conclusion: When considered from a societal perspective, the use of LDp/CDp in aPD patients inadequately controlled on oral therapy is associated with net healthcare and societal annual savings of over £79.1 M vs BMT.

Aims: To understand treatment patterns, healthcare resource utilization (HCRU), and the economic burden of diffuse large B-cell lymphoma (DLBCL) in elderly adults in the US., Materials and Methods: This retrospective database analysis utilized US Centers for Medicare and Medicaid Services Medicare fee-for-service administrative claims data from 2015 to 2020 to describe DLBCL patient characteristics, treatment patterns, HCRU, and costs among patients aged ≥66 years. Patients were indexed at DLBCL diagnosis and required to have continuous enrollment from 12 months pre-index until 3 months post-index. HCRU and costs (USD 2022) are reported as per-patient per-month (PPPM) estimates., Results: A total of 11,893 patients received ≥1-line (L) therapy; 1,633 and 391 received ≥2 L and ≥3 L therapies, respectively. Median (Q1, Q3) age at 1 L, 2 L, and 3 L initiation, respectively, was 76 (71, 81), 77 (72, 82), and 77 (72, 82) years. The most common therapy was R-CHOP (70.9%) for 1 L and bendamustine ± rituximab for 2 L (18.7%) and 3 L (17.4%). CAR T was used by 14.8% of patients in 3 L. Overall, 39.6% (1 L), 42.1% (2 L), and 47.8% (3 L) of patients had all-cause hospitalizations. All-cause mean (median [Q1-Q3]) costs PPPM during each line were $22,060 ($20,121 [$16,676-$24,597]) in 1 L, $30,027 ($20,868 [$13,416-$31,016]) in 2 L, and $47,064 ($25,689 [$15,555-$44,149]) in 3 L, with increasing costs driven primarily by inpatient expenses. Total all-cause 3 L mean (median [Q1-Q3]) costs PPPM for patients with and without CAR T were $153,847 ($100,768 [$26,534-$253,630]) and $28,466 ($23,696 [$15,466-$39,107]), respectively., Conclusions: No clear standard of care exists in 3 L therapy for older adults with relapsed/refractory DLBCL. The economic burden of DLBCL intensifies with each progressing line of therapy, thus underscoring the need for additional therapeutic options.

Aims: Patients with inborn errors of immunity (IEI) are predisposed to severe recurrent/chronic infections, and often require hospitalization, resulting in substantial burden to patients/healthcare systems. While immunoglobulin replacement therapies (IgRTs) are the standard first-line treatment for most forms of IEI, limited real-world data exist regarding clinical characteristics and treatment costs for patients with IEI initiating such treatment. This retrospective analysis examined infection and treatment characteristics in US patients with IEI initiating IgRT with immune globulin infusion (human), 10% (IG10%). Healthcare resource utilization (HCRU) and associated costs before and after treatment initiation were compared. Additionally, the impact of COVID-19 on infection diagnoses was evaluated., Methods: Patients with IEI initiating IG10% between July 2012 and August 2019 were selected from Merative MarketScan Databases using diagnosis/prescription codes. Patients were followed 6 months before and after first IG10% claim date. Demographic and clinical characteristics were described. Treatment characteristics and HCRU before and after IG10% initiation were compared. Infection diagnoses during 2020 and 2019 (March-December) were compared., Results: The study included 1,497 patients with IEI diagnoses (mean age = 43.4 years) initiating IG10%, with frequently reported comorbidities like asthma (32.1%). Following IG10% initiation, fewer severe infection diagnoses (11.6% vs 19.9%), fewer infection-related inpatient (10.8% vs 19.5%) and outpatient services (71.6% vs 79.9%), and lower infection-related total healthcare costs ($7,849 vs $13,995; p  < 0.001)-driven by lower inpatient costs ($2,746 vs $9,900)-were observed than before. Fewer patients had infection diagnoses during COVID-19 (22.8%) than the prior year (31.2%)., Conclusion: Patients with IEI are susceptible to severe infections leading to high disease burden and treatment costs. Following IG10% initiation, we observed fewer infections, lower infection-related treatment costs, and shift in care (inpatient to outpatient) leading to significant cost savings. Among patients with IEI, 27% fewer infection diagnoses were observed during the early COVID-19 lockdown period than the prior year.

Aim: To quantify the economic burden associated with tobacco smoking among smokers aged 30-69 years, and second-hand smokers (SHS) aged 15-69 years in Jordan., Materials and Methods: A prevalence-based analysis was conducted in alignment with the Economics of Tobacco Toolkit developed by the WHO. The time-horizon of the analysis was one year (2019). Direct and indirect costs were estimated using data from the 2019 Global Burden of Diseases study. The analysis targeted the Jordanian population of smokers aged 30-69 years, and SHS aged 15-69 years. Adjustments were applied for age, gender, and smoking-related diseases. Direct costs were estimated using the smoking-attributable fraction (SAF) and national health expenditures. Indirect costs were divided into morbidity and mortality components. A discount rate of 3.0% and an annual productivity growth rate of 1.0% were assumed in modelling future economic losses. A sensitivity analysis was conducted on the lower and upper estimates of data used in this study., Results: The cost of tobacco smoking and SHS exposure was estimated at US$2,108 million (95% confidence interval [CI] = US$2,003 million-US$2,245 million). This represents 4.7% (95%CI = 4.5%-5.0%) of national gross domestic product (GDP). Direct costs accounted for 3.1% of national GDP. Tobacco smoking accounted for 85.0% of total cost and SHS exposure accounted for 15.0% of total cost. Direct costs accounted for 67.0% of total cost, while indirect morbidity and mortality costs accounted for 9.0% and 24.0% of total cost, respectively. Non-communicable diseases accounted for 96.0% of total direct costs compared to communicable diseases (4.0% of total direct costs)., Conclusions: Smoking cessation interventions such as raising taxes on cigarettes, protecting people from tobacco smoke, warning labels, plain packaging, and bans on advertising, are crucial for controlling national expenditures for treating smoking-related diseases and for averting future economic losses.

Aims: There are multiple recently approved treatments and a lack of clear standard-of-care therapies for relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL). While total cost of care (TCC) by the number of lines of therapy (LoTs) has been evaluated, more recent cost estimates using real-world data are needed. This analysis assessed real-world TCC of R/R DLBCL therapies by LoT using the IQVIA PharMetrics Plus database (1 January 2015-31 December 2021), in US patients aged ≥18 years treated with rituximab plus cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP) or an R-CHOP-like regimen as first-line therapy., Methods: Treatment costs and resources in the R/R setting were assessed by LoT. A sensitivity analysis identified any potential confounding of the results caused by the impact of the COVID-19 pandemic on healthcare utilization and costs. Overall, 310 patients receiving a second- or later-line treatment were included; baseline characteristics were similar across LoTs. Inpatient costs represented the highest percentage of total costs, followed by outpatient and pharmacy costs., Results: Mean TCC per-patient-per-month generally increased by LoT ($40,604, $48,630, and $59,499 for second-, third- and fourth-line treatments, respectively). Costs were highest for fourth-line treatment for all healthcare resource utilization categories. Sensitivity analysis findings were consistent with the overall analysis, indicating results were not confounded by the COVID-19 pandemic., Limitations: There was potential misclassification of LoT; claims data were processed through an algorithm, possibly introducing errors. A low number of patients met the inclusion criteria. Patients who switched insurance plans, had insurance terminated, or whose enrollment period met the end of data availability may have had truncated follow-up, potentially resulting in underestimated costs., Conclusion: Total healthcare costs increased with each additional LoT in the R/R DLBCL setting. Further improvements of first-line treatments that reduce the need for subsequent LoTs would potentially lessen the economic burden of DLBCL.

Aims: Non-cystic fibrosis bronchiectasis (NCFB) is a chronic progressive respiratory disorder occurring at a rate ranging from 4.2 to 278.1 cases per 100,000 persons, depending on age, in the United States. For many patients with NCFB, the presence of Pseudomonas aeruginosa (PA) makes treatment more complicated and typically has worse outcomes. Management of NCFB can be challenging, warranting a better understanding of the burden of illness for NCFB, treatments applied, healthcare resources used, and subsequent treatment costs. Comparing patients diagnosed with exacerbated NCFB, with or without PA on antibiotic utilization, treatments, and healthcare resources utilization and costs was the purpose of this study., Materials and Methods: This was a retrospective cohort study of commercial claims from IQVIA's PharMetrics Plus database (January 1,2006-December 31, 2020). Study patients with a diagnosis of NCFB were stratified into two groups based on the presence or absence of PA, then followed to identify demographic characteristics, comorbid conditions, antibiotic treatment regimen prescribed, healthcare resources utilized, and costs of care., Results: The results showed that patients with exacerbated NCFB who were PA + had significantly more oral antibiotic fills per patient per year, more inpatient admissions with a longer length of stay, and more outpatient encounters than those who were PA - . For costs, PA + patients also had significantly greater total healthcare costs per patient when compared to those who were PA - ., Conclusion: Exacerbated NCFB with PA + was associated with increased antibiotic usage, greater resource utilization, and increased costs. The major contributor to the cost differences was the use of inpatient services. Treatment strategies aimed at reducing the need for inpatient treatment could lessen the disparities observed in patients with NCFB.

Aim: To estimate the budget impact of adding a toripalimab regimen as a treatment option to the existing pembrolizumab regimen, both including gemcitabine and cisplatin, in untreated recurrent/metastatic nasopharyngeal carcinoma (R/M NPC) using the published wholesale acquisition cost (WAC) and average sales price (ASP)., Methods: Budget impact analysis comparing a treatment mix "without" versus "with" the toripalimab regimen in the US eligible annual incident R/M NPC population, a 3-year time horizon, toripalimab/pembrolizumab market splits of 60/40 (Y1) and 80/20 (Y2/3), and medication adjustments for discontinuation or progression. Cost inputs included drugs, administration, and adverse event (AE) management. The models were replicated for a hypothetical 1-million-member health plan in which costs per-member-per-month (PMPM) and per-member-per-year (PMPY) were estimated. One-way (OWSA) and probabilistic sensitivity analyses (PSA) as well as scenario analyses were performed., Results: In the "without" scenario, the 3-year WAC-based costs for the pembrolizumab regimen total $1,449,695,333 ($1,305,632,448 for treatment and $144,062,885 for managing AEs). In the "with" scenario, total 3-year costs for pembrolizumab decline to $380,012,135 with toripalimab adding $885,505,900 ($779,206,567 for treatment and $106,299,333 for AE management). Annual net savings range from $46,526,152 in 2024 to $71,194,214 in 2026, for 3-year savings of $184,177,298. Associated net savings in a 1-million-member health plan are $543,068 over 3 years with savings of $0.045 PMPM and $0.543 PMPY. The ASP-based model shows similar patterns with 3-year net savings of $174,235,983 in the US incident population and savings of $0.043 PMPM and $0.514 PMPY in a 1-million-member health plan. The PSA support base case findings; OWSA and scenario analyses reveal how parameter variability impacts results., Conclusion: Savings between $174 million and $184 million can be achieved from treating 60% of R/M NPC patients in year 1 and 80% in years 2 and 3 with the toripalimab regimen over a similar pembrolizumab regimen.

Aims: The increasing prevalence of end-stage renal disease (ESRD) in the United States (US) represents a considerable economic burden due to the high cost of dialysis treatment. This review examines data from real-world studies to identify cost drivers and explore areas where dialysis costs could be reduced., Methods: We identified and synthesized evidence published from 2016-2023 reporting direct dialysis costs in adult US patients from a comprehensive literature search of MEDLINE, Embase, and grey literature sources (e.g. US Renal Data System reports)., Results: Most identified data related to Medicare expenditures. Overall Medicare spending in 2020 was $29B for hemodialysis and $2.8B for peritoneal dialysis (PD). Dialysis costs accounted for almost 80% of total Medicare expenditures on ESRD beneficiaries. Private insurance payers consistently pay more for dialysis; for example, per person per month spending by private insurers on outpatient dialysis was estimated at $10,149 compared with Medicare spending of $3,364. Dialysis costs were higher in specific high-risk patient groups (e.g. type 2 diabetes, hepatitis C). Spending on hemodialysis was higher than on PD, but the gap in spending between PD and hemodialysis is closing. Vascular access costs accounted for a substantial proportion of dialysis costs., Limitations: Insufficient detail in the identified studies, especially related to outpatient costs, limits opportunities to identify key drivers. Differences between the studies in methods of measuring dialysis costs make generalization of these results difficult., Conclusions: These findings indicate that prevention of or delay in progression to ESRD could have considerable cost savings for Medicare and private payers, particularly in patients with high-risk conditions such as type 2 diabetes. More efficient use of resources is needed, including low-cost medication, to improve clinical outcomes and lower overall costs, especially in high-risk groups. Widening access to PD where it is safe and appropriate may help to reduce dialysis costs.

Aim: To investigate hepatitis A-related healthcare resource use and costs in the US., Methods: The Merative Marketscan Commercial Claims and Encounters database was retrospectively analyzed for hepatitis A-related inpatient, outpatient, and emergency department (ED) claims from January 1, 2012 to December 31, 2018. We calculated the hepatitis A incidence proportion per 100,000 enrollees, healthcare resource utilization, and costs (in 2020 USD). Results were stratified by age, gender, and select comorbidities., Results: The overall hepatitis A incidence proportion was 6.1 per 100,000 enrollees. Among individuals with ≥1 hepatitis A-related claim, the majority (92.6%) had ≥1 outpatient visit related to hepatitis A; 9.1% were hospitalized and 4.2% had ≥1 ED visit. The mean (standard deviation [SD]) length of hospital stay was 5.2 (8.1) days; the mean (SD) number of outpatient and ED visits were 1.3 (1.3) and 1.1 (0.6), respectively. The incidence proportion per 100,000 was higher among adults than children (7.5 vs. 1.5), individuals with HIV than those without (126.7 vs. 5.9), and individuals with chronic liver disease than those without (143.6 vs. 3.8). The total mean (SD)/median (interquartile range, IQR) per-patient cost for hepatitis A-related care was $2,520 ($10,899)/$156 ($74-$529) and the mean cost of hospitalization was 18.7 times higher than that of outpatient care ($17,373 vs. $928)., Limitations: The study data included only a commercially insured population and may not be representative of all individuals., Conclusions: In conclusion, hepatitis A is associated with a substantial economic burden among privately insured individuals in the US.

Background: Bioprostheses with RESILIA tissue demonstrate a reduction in calcification and improve health outcomes in pre-clinical and clinical studies. Prior economic analyses which relied on 5 years of evidence from the COMMENCE trial demonstrate financial savings for RESILIA tissue valves relative to mechanical valves after surgical aortic valve replacement (SAVR). Given the recent release of 7-year COMMENCE data, this economic evaluation updates the estimate for long-run savings of bioprosthetic valves with RESILIA., Methods: Simulation models estimated disease progression across two hypothetical SAVR cohorts (tissue vs. mechanical) of 10,000 patients each in the US. The primary comparison calculated the SAVR-related expenditures associated with each valve type ($US, 2023). Health outcome probabilities were based on the COMMENCE trial though year 7 and projected for an additional 8 years based on prior studies of tissue and mechanical SAVR. Costs for key outcomes (mortality, reoperation, bleeding, thromboembolism, endocarditis) and anticoagulant monitoring were sourced from the literature. Incidence rates of health outcomes associated with mechanical valves relied on relative risks of tissue valve versus mechanical valve patients., Results: Seven-year savings are $13,415 (95% CI = $10,472-$17,321) per patient when comparing RESILIA versus mechanical SAVR. Projected 15-year savings were $23,001 ($US, 2023; 95% CI = $17,802-$30,421). Most of the 15-year savings are primarily attributed to lower anti-coagulation monitoring costs ($21,073 in ACM savings over 15 years), but lower bleeding cost (savings: $2,294) and thromboembolism-related expenditures (savings: $852) also contribute. Reoperation and endocarditis expenditures were slightly larger in the RESILIA cohort. If reoperation relative risk reverts from 1.1 to 2.2 (the level in legacy tissue valves) after year 7, savings are $18,064. RESILIA SAVR also reduce costs relative to legacy tissue valves., Conclusion: Patients receiving RESILIA tissue valves are projected to have lower SAVR-related health expenditures relative to mechanical and legacy tissue valves.

Aims: This study aimed to assess and compare the health care resource utilization (HCRU) and medical cost of metabolic dysfunction-associated steatohepatitis (MASH) by disease severity based on Fibrosis-4 Index (FIB-4) score among US adults in a real-world setting., Materials and Methods: This observational cohort study used claims data from the Healthcare Integrated Research Database (HIRD) to compare all-cause, cardiovascular (CV)-related, and liver-related HCRU, including hospitalization, and medical costs stratified by FIB-4 score among patients with MASH (identified by International Classification of Diseases, Tenth Revision, Clinical Modification [ICD-10-CM] code K75.81). Hospitalization and medical costs were compared by FIB-4 score using generalized linear regression with negative binomial and gamma distribution models, respectively, while controlling for confounders., Results: The cohort included a total of 5,104 patients with MASH and comprised 3,162, 1,343, and 599 patients with low, indeterminate, and high FIB-4 scores, respectively. All-cause hospitalization was significantly higher in the high FIB-4 cohort when compared with the low FIB-4 reference after covariate adjustment (rate ratio, 1.63; 95% CI, 1.32-2.02; p  < .0001). CV-related hospitalization was similar across all cohorts; however, CV-related costs were 1.26 times higher (95% CI, 1.11-1.45; p  < .001) in the indeterminate cohort and 2.15 times higher (95% CI, 1.77-2.62; p  < .0001) in the high FIB-4 cohort when compared with the low FIB-4 cohort. Patients with indeterminate and high FIB-4 scores had 2.97 (95% CI, 1.78-4.95) and 12.08 (95% CI, 7.35-19.88) times the rate of liver-related hospitalization and were 3.68 (95% CI, 3.11-4.34) and 33.73 (95% CI, 27.39-41.55) times more likely to incur liver-related costs, respectively ( p  < .0001 for all)., Limitations: This claims-based analysis relied on diagnostic coding accuracy, which may not capture the presence of all diseases or all care received., Conclusions: High and indeterminate FIB-4 scores were associated with significantly higher liver-related clinical and economic burdens than low FIB-4 scores among patients with MASH.

Aims: Respiratory syncytial virus (RSV) causes severe lower respiratory tract infections (LRTI) in infants and adults. While the clinical burden was recently estimated in adults in Germany, little is known about the economic burden. To fill this gap, this study aimed to assess hospital and outpatient healthcare resource utilization (HRU) and costs of RSV infections in adults in Germany., Methods: In this retrospective, observational study on nationwide, representative, anonymized claims data (2015-2018), we identified patients ≥18 years with ICD-10-GM-codes specific to RSV ("RSV-specific"). To increase sensitivity, patients with unspecified LRTIs (including unspecified bronchitis, bronchiolitis, bronchopneumonia, and pneumonia) during RSV seasons were also included as cases potentially caused by RSV ("RSV-possible"). RSV-related HRU (hospital days, ICU and ventilation treatment, drug dispensation) and direct costs were estimated per episode. Excess costs per episode and for follow-up periods were compared to a matched control cohort. All outcomes were reported per healthcare sector and stratified by age and risk groups as well as disease severity (ICU admission/ventilation)., Results: Direct inpatient and outpatient mean episode costs were 3,473€ and 82€, respectively, with substantially higher costs for severe cases requiring intensive care and/or ventilation (10,801€). Direct costs for RSV-specific cases were higher than for RSV-possible cases (inpatients: 6,247€ vs. 3,450€; outpatients: 127€ vs. 82€). Moreover, costs were significantly higher for RSV patients than for controls and increased over time (inpatients: 5,140€ per episode vs 10,093€ per year; outpatients: 46€ per quarter vs 114€ per year)., Limitations: While the number of RSV-specific cases was low, inclusion of seasonal LRTI cases likely increased the sensitivity to detect RSV cases and allowed a better estimation of the total costs of RSV., Conclusions: The economic burden of RSV-LRTI in adults in Germany is substantial, persists long-term, and is particularly high in the elderly. This highlights the need for cost-effective prevention measures.

Aims: We aimed to describe the clinical, economic, and societal burdens of cystic fibrosis (CF) and impact of CF transmembrane conductance regulator modulator (CFTRm) treatment on people with CF, caregivers, and healthcare systems., Material and Methods: This retrospective study used linked real-world data from Swedish national population-based registries and the Swedish CF Quality Registry to assess clinical, economic, and societal burden and CFTR impact in CF. Records from people with CF and a ten-fold control population without CF matched by sex, birth year, and location were compared during 2019. Outcomes for a subset aged >6 years initiating lumacaftor/ivacaftor (LUM/IVA) in 2018 were compared 12 months pre- and post-treatment initiation., Results: People with CF ( n  = 743) had >10 times more inpatient and outpatient specialist visits annually vs controls ( n  = 7406). Those aged >18 had an additional 77·7 (95% CI: 70·3, 85·1) days of work absence, at a societal cost of €11,563 (95% CI: 10,463, 12,662), while caregivers of those aged <18 missed an additional 6.1 (5.0, 7.2) workdays. With LUM/IVA treatment, people with CF ( n  = 100) had significantly increased lung function (mean change in ppFEV 1 [3·8 points; 95% CI: 1·1, 6·6]), on average 0·5 (95% CI: -0·8, -0·2) fewer pulmonary exacerbations and 45·2 (95% CI: 13·3, 77·2) fewer days of antibiotics. Days of work lost by caregivers of people with CF aged <18 decreased by 5·4 days (95% CI: 2·9, 7·9)., Conclusion: CF is associated with a high clinical economic and societal burden in Sweden. Improvements in clinical status observed in people with CF treated with LUM/IVA were reflected in reduced caregiver and societal burden.

Aims: Food allergies impose a large clinical and financial burden on patients and the health care system. However, little is known about the factors associated with health care resource use and costs. The aim of this study was to investigate health care resource use and costs in individuals with food allergies utilizing health care in the United States., Methods: We conducted a retrospective analysis of insurance claims data from the Merative MarketScan Research Databases (indexed from 1 January 2015 to 30 June 2022). All-cause and food allergy-related health care resource use, direct medical, and out-of-pocket costs for medical services were estimated for 12 months post-index using International Classification of Diseases [ICD] codes., Results: Of 355,520 individuals with food allergies continuously enrolled in a health insurance plan for ≥12 months pre- and post-index, 17% had a food allergy-related emergency department visit and 0.9% were hospitalized. The top patient characteristic associated with all-cause and food allergy-related hospitalizations, all-cause costs, and food allergy-related outpatient visit costs was a Charlson Comorbidity Index score of ≥2. Food allergy-related direct medical and out-of-pocket costs were high among patients with a food allergy-related visit. Out-of-pocket cost per patient per year for outpatient visits, emergency department visits, and hospitalizations had an estimated mean of $1631 for patients with food allergy-related visits, which is ∼11% of the total costs for these services ($14,395 per patient per year)., Limitations: Study limitations are primarily related to the nature of claims databases, including generalizability and reliance on ICD codes. Nevertheless, MarketScan databases provide robust patient-level insights into health care resource use and costs from a large, commercially insured patient population., Conclusion: The health care resource use of patients with food allergies imposes a burden on both the health care system and on patients and their families, especially if patients had comorbidities.

Introduction: To estimate the direct and indirect costs of bladder cancer prior to and following cystectomy in a U.S. sample of patients., Methods: This retrospective, observational analysis of de-identified patients with bladder cancer utilized the MarketScan Commercial Claims & Encounters and Health & Productivity Management databases. Adult patients with bladder cancer plus ≥ 1 claim for partial or radical cystectomy between 1 October 2015 and 31 December 2020 (date of the cystectomy = index date) and who were continuously enrolled for 6 months pre- (baseline) and post-index (follow-up) were included in the sample. All-cause total healthcare costs and indirect costs associated with short-term and long-term disability (STD and LTD) employer claims were assessed during each of the 6-month baseline and follow-up periods., Results: The study included N  = 142 patients; mean age 56 ± 6 years, 76% (male), and 42% had a baseline Deyo-Charlson Comorbidity Index ≥ 2. Baseline mean total all-cause direct healthcare costs were $51,473 ± $48,560 (median: $36,202), and $99,524 ± 86,839 (median: $75,444) during follow-up. At baseline, 32% of patients had ≥ 1 STD claim, equating to a mean 134 ± 303 h lost and $2,353 ± $6,445 in total payments per patient. Follow up STD claims increased 23.4% equating to a mean 218 ± 324 h lost and $3,679 ± $7,795 per patient. Patient LTD claims increased from baseline to follow-up (1-3%), with post-cystectomy LTD claims resulting in 574 ± 490 h lost, and $1,636 ± $1,429 in total payments. Over 85% of the population had a cystectomy related complication, the most common were genitourinary-related (47.9%) and infection/sepsis (33.1%)., Conclusions: Cystectomy was associated with complications and decreased work productivity post-surgery. Findings may aid to inform decisions regarding cystectomy vs. bladder preservation approaches, and underscores an ongoing need to further develop bladder preservation therapies within the bladder cancer treatment landscape.

Objective: Attention-deficit/hyperactivity disorder (ADHD) medication is frequently associated with adverse events (AEs), but limited real-world data exist regarding their costs from a payer's perspective. Therefore, this study evaluated the healthcare costs associated with common AEs among adult patients treated for ADHD in the US., Methods: Eligible adults treated for ADHD were identified from a large US claims database (1 October 2015-30 September 2021). A retrospective cohort study design was used to assess excess healthcare costs and costs directly related to AE-specific claims per-patient-per-month (PPPM) associated with 10 selected AEs during ADHD treatment. To account for all costs associated with the AE, treatment episodes with a given AE were compared to similar treatment episodes without this AE. Entropy balancing was used to create cohorts with similar characteristics. Studied AEs were selected based on their prevalence in clinical trials for common ADHD medications and were identified from ICD-10-CM diagnosis codes recorded in claims., Results: Among the 461,464 patients included (mean age: 34.2 years; 45.5% males), 49.4% had ≥1 AE during their treatment episode. Treatment episodes with AEs were associated with statistically significant AE-specific medical costs (erectile dysfunction: $57; fatigue: $82; dry mouth: $90; diarrhea: $162; insomnia: $147; anxiety: $281; nausea: $299; constipation: $356; urinary hesitation: $491; feeling jittery: $723) and excess healthcare costs PPPM (erectile dysfunction: $120, fatigue: $248, insomnia: $265, anxiety: $380, diarrhea: $441, dry mouth: $485, nausea: $709, constipation: $802, urinary hesitation: $1,105, feeling jittery: $1,160; p  < .05)., Limitations: AEs were identified based on recorded diagnosis on medical claims and likely represent more severe AEs. Therefore, costs may not be representative of milder AEs., Conclusions: This study found that AEs occurring during ADHD treatment episodes are associated with significant healthcare costs. This highlights the potential of treatments with favorable safety profiles to alleviate the burden experienced by patients and the healthcare system.

Background: Triple Negative Breast Cancer (TNBC) is an aggressive subtype of breast cancer that can impact patients' employment and workforce participation. This study estimates how the employment effects of TNBC impact government tax revenue and public benefits expenditure in Switzerland, representing the fiscal burden of disease (FBoD), and likely consequences of introducing new treatment options., Methods: A four-state cohort model was used to calculate fiscal effects for two treatments: Neoadjuvant pembrolizumab plus chemotherapy followed by adjuvant pembrolizumab monotherapy (P + C→P) and neoadjuvant chemotherapy alone (C). Lifetime present values of tax revenue, social benefit payments, and healthcare costs were calculated for the average population and those undergoing treatment to assess the FBoD., Results: An average TNBC patient treated with C and P + C→P is expected to generate CHF128,999 and CHF97,008 less tax than the average population, respectively, and require increased social benefit payments. Compared to C, 75% of the incremental healthcare costs of P + C→P are estimated to be offset through tax revenue gains., Conclusions: This analysis demonstrates that 75% of the additional costs of a new TNBC treatment option can be offset by gains in tax revenue. Fiscal analysis can be a useful tool to complement existing methods for evaluating new treatments.

Aims: Thrombotic thrombocytopenic purpura (TTP) is an ultra-rare blood disorder, characterized by severe ADAMTS13 deficiency. Affected individuals present with potentially life-threatening acute events and may experience sub-acute and chronic TTP manifestations often resulting in long-term organ damage. Incremental symptom prevalence before, during, and after an acute event as well as healthcare resource utilization (HCRU) and costs during and after an acute event were compared between people with TTP and matched non-TTP controls., Methods: This retrospective, matched study used data from Merative MarketScan Commercial Database and Medicare Supplemental Database (from January 1, 2008, through September 30, 2021) to identify people with TTP (inpatient diagnosis for "thrombotic microangiopathy (TMA)" or "congenital TTP," and ≥1 claim for plasma exchange or infusion). People with TTP were matched (1:2) with non-TTP controls on age, sex, geographic region, index year, and select Elixhauser comorbidities., Results: 255 people with TTP were matched with 510 non-TTP controls. Both cohorts had a mean age of 43.9 years; 71% were female. Overall, more people with TTP reported symptoms compared with non-TTP controls prior to (51% vs 43%), during (99% vs 52%), and after an acute event (85% vs 50%; p  < 0.05 for all periods). Symptom prevalence decreased following an acute event compared with during an acute event, but remained high-85% of people with TTP experienced symptoms compared with 50% of non-TTP controls. HCRU and mean costs per patient per month were significantly higher in all care settings among people with TTP compared with non-TTP controls ( p  < 0.05)., Limitations: Identification of patient populations may have been limited due to coding errors, as the data were obtained from an administrative claims database., Conclusions: TTP is associated with a substantial symptom burden and increased costs and HCRU during and up to almost a year after acute events, demonstrating the longitudinal burden of this disease.

Aim: This study aimed to obtain estimates for the direct medical charges associated with hospitalizations and emergency department visits of validated SLE cases in a diverse Systemic Lupus Erythematosus (SLE) population., Methods: The Georgians Organized Against Lupus (GOAL) cohort is a population-based cohort of adult SLE patients from metropolitan Atlanta, GA USA, an area having a diverse SLE population. The GOAL cohort aims to study the impact of social determinants of health (SDoH) on outcomes relevant to patients, healthcare providers, and policymakers. For this study, survey data collected during 2011-2012 was linked to the Georgia Hospital Discharge Database (HDD) to capture hospital admissions (HAs) and emergency department visits (EDVs) throughout Georgia from 2012 through 2013. Direct medical charges were summarized by HCU type among all patients, among those with actual visits, and by socio-demographics and healthcare factors., Results: Among 829 patients (94% women, 78% Black, 64% non-private insurance, 64% not-employed, mean age of 46), 170 (20.5%) and 300 (36.2%) participants had at least one HA and one EDV in 1-year of follow-up, respectively, with 111(13.4%) having both HA and EDV. On average, each patient experienced 0.38 HAs and 0.91 EDVs, with per-patient direct medical charges of $14,968 for HAs & $3,022 for EDVs, and $39,645 per HA & $3,305 per EDV. Patients with higher social vulnerability or more severe disease had higher charges for both HA and EDV ( p  < 0.01), likely due to the delayed care and neglected health needs leading to more advanced and costly medical treatments. Living below the federal poverty level was associated with higher charges for EDVs ( p  < 0.001) but with lower charges for HAs ( p  = 0.036)., Conclusions: This study underscores the economic burden of SLE on vulnerable populations, emphasizing the importance of including socio-economic factors in healthcare planning. Policy efforts should prioritize reducing disparities in access to care and implementing preventive strategies.

Aims: Suboptimal treatment indicators, including treatment switch, are common among patients with Crohn's disease (CD), but little is known about their associated healthcare resource utilization (HRU) and costs. This study assessed the impact of suboptimal treatment indicators on HRU and costs among adults with CD newly treated with a first-line biologic., Methods: Adult patients with CD were identified in the IBM MarketScan Commercial Subset (10/01/2015-03/31/2020). The index date was defined as initiation of the first-line biologic, and the study period was defined as the 12 months following the index date. Patients were classified into Suboptimal Treatment and Optimal Treatment cohorts based on observed indicators of suboptimal treatment during the study period. Patients in the Suboptimal Treatment Cohort with a treatment switch were classified into the Treatment Switch Cohort and compared to patients with no treatment switch. All-cause HRU and costs were measured during the study period and assessed for patients with suboptimal vs optimal treatment and patients with vs without a treatment switch., Results: The study included 4,006 patients (Suboptimal Treatment: 2,091, Optimal Treatment: 1,915). Treatment switch was a common indicator of suboptimal treatment (Treatment Switch: 640, No Treatment Switch: 3,366). HRU and costs were significantly higher among patients with suboptimal treatment than those with optimal treatment (annual costs: $92,043 vs $73,764; p  < 0.01), and among those with a treatment switch than those with no treatment switch (annual costs: $95,689 vs $81,027; p  < 0.01). Increases in the number of suboptimal treatment indicators were associated with increased costs., Limitations: Claims data were used to identify suboptimal treatment indicators based on observed treatment patterns; reasons for treatment decisions could not be assessed., Conclusion: This study demonstrates that patients with suboptimal treatment indicators, including treatment switch, incur substantially higher HRU and costs compared to patients receiving optimal treatment and those that do not switch treatments.

Aims: To compare all-cause claims associated with the LATERA Absorbable Nasal Implant and surgical repair of nasal vestibular stenosis in patients with nasal valve collapse., Methods: This retrospective cohort study utilized data from STATinMED RWD Insights. A defined set of HCPCS, ICD-10-CM and CPT codes were used to identify patients with ≥1 claim for a LATERA procedure, and patients with ≥1 claim for surgical repair between June 1, 2015- March 31, 2023. Patients with continuous capture for at least 12 months before and at least 6 months after the index date were selected. The index date was defined as earliest date of encounter for a LATERA or surgical repair procedure. Inverse probability of treatment weighting (IPTW) was used to ensure balance between cohorts. Descriptive analyses were provided for all claims data using standard summary statistics. All-cause claims were assessed during the baseline, index date, and follow-up period. Chi-squared tests and independent sample t-tests were used to assess differences in cohorts for categorical and continuous variables, respectively., Results: The study population included 5,032 LATERA patients and 26,553 surgical repair patients. During the baseline and follow-up periods, the matched cohorts exhibited similar all-cause claims. On the index date, LATERA patients incurred lower claims vs. surgical repair, likely due to LATERA's ability to be implanted in the physician office setting. LATERA patients and surgical repair patients mean (SD) total costs were $9,612 [$14,930] vs $11,846 [$17,037] ( p  ≤ 0.0001), respectively., Conclusions: Treatment with the LATERA Absorbable Nasal Implant is a potentially cost saving option for payers on the index date compared to traditional surgical repair in patients with nasal valve collapse due to the ability to be performed in the office. All-cause claims were similar in the baseline and follow-up periods. When performed with concomitant procedures, all-cause claims during follow-up were similar between groups.

The COVID-19 pandemic caused the United States to hit record numbers of COVID-19 cases: peak unemployment of 14.7%, an increase in $4 trillion in national debt, and an estimated 3.4% GDP decline. The current socio-economic environment the pandemic created is just an earthquake that can create a tsunami that is bound to hit the healthcare system and can be felt around the globe. This tsunami is composed of a post-pandemic increase in healthcare facilities admission of indigent patients, decrease in medical reimbursement, and high operating costs to maintain healthcare workers, which can cause a synergistic effect that can lead to healthcare facilities experiencing significant negative total revenue. Time is of the essence, and it is imperative to make a collective effort from all healthcare professionals and legislatures to shift the nation's attention to the issue at hand that can threaten the closure of many healthcare facilities post-pandemic.

Background: Current health economic analysis guidelines emphasize the importance of using nationally appropriate cost and valuation inputs. However, some countries lack national data, and some analyses focus on interventions with costs and benefits at regional or global scales., Methods: Recognizing the need for better estimates of appropriate values for application at these levels than those used in the past, we characterize population-weighted dollar per disability-adjusted life year (DALY) averted by World Bank Income Level based on available national estimates of the marginal productivity of the healthcare system., Results: The defaults suggested here reflect health opportunity costs across countries more consistent with existing evidence than those previously used or recommended. As countries change income levels and healthcare spending, and as additional or updated marginal productivity of healthcare expenditure estimates become available, we expect the defaults to change., Conclusion: The best option for informing decisions around resource allocation in health care such that they improve health outcomes overall remains the use of time-appropriate country-specific estimates of the marginal productivity of the healthcare system. Instead of single, time-invariant defaults, health economists should seek to develop valuation inputs that better account for health opportunity costs and do so over time.

Background : Multiple sclerosis (MS) has a significant healthcare burden. This study examined the medical cost and out-of-pocket (OOP) expenses, and quantified the impact factors contributing to the costs. Methods : This is a retrospective study in 77 Chinese urban cites from 2013 to 2015. The data included the details of the utilization of medical resources, cost, and reimbursement ratio of all patients with a diagnosis of MS. A generalized estimating equation model was used to estimate the factors influencing the direct medical cost and OOP expenses for in- and outpatients. Results : A total of 267 patients with MS were identified. The mean cost per in- and outpatient was respectively 16996.2 and 2294.2 renminbi ($2768.12 and $373.65, €2087.16 and €281.73). Approximately 27% of the expenses were paid by the patients OOP. Factors contributing to high cost and high OOP expenses for inpatients were tertiary hospital admission, length of stay and residence in the east regions of China. Females and outpatients with resident insurance paid more OOP. Conclusion : This study illustrates the medical costs and burden of MS in Chinese patients and provides real-world data on MS that are essential for the improvement of health policies.

Objectives: The aim of this study is to assess the cost-effectiveness of fruquintinib compared to regorafenib as third-line treatment for patients with metastatic colorectal cancer (mCRC) in China., Methods: A three-state Markov model with monthly cycle was constructed to estimate lifetime incremental cost-effectiveness ratio (ICER) of fruquintinib versus regorafenib as third-line treatment for patients with mCRC from Chinese health care perspective. Survival analysis was applied to calculate transition probabilities using the data from the clinical trials FRESCO and CONCUR, which were also the data sources accessing probabilities of adverse events. Background mortality rate and drug costs were derived from government published data. Costs for medical services were obtained from real-world data and published literatures. Utilities applied to calculate the quality-adjusted life years (QALYs) were obtained from literature review. One-way sensitivity analysis and probabilistic sensitivity analysis were adopted to verify the robustness of the results., Results: Fruquintinib provided 0.74 QALYs at a cost of CNY 151,058 (USD 22,888), whereas regorafenib provided 0.79 QALYs at a cost of CNY 226,657 (USD 32,224). Compared to fruquintinib, the ICER of regorafenib was CNY 1,529,197/QALY (USD 231,697/QALY) from Chinese health care perspective, which was above the triple GDP per capita of China in 2019 (CNY 212,676) (USD 32,224) as the threshold to define the cost-effectiveness. One-way sensitivity analysis showed the results were generally robust. Cost-effectiveness acceptability curves derived from probabilistic sensitivity analysis demonstrated the probability that fruquintinib was more cost-effective was 100% when the threshold was the triple GDP per capita of China., Conclusions: Compared to regorafenib, fruquintinib, which leads to forego about 0.05 QALYs and save about CNY 75,599 (USD 11,454), is a cost-effective choice as the third-line treatment for patients with mCRC in China.

Background: Considering clinical benefits of new combination therapies for metastatic renal-cell carcinoma (mRCC), this study aims to calculate the number needed to treat (NTT) and the cost of preventing an event (COPE) for pembrolizumab plus axitinib (P + A), and nivolumab plus ipilimumab (N + I) as first-line treatments, from the Brazilian private perspective., Methods: Overall survival (OS) and progression-free survival (PFS) data for intermediate- and poor-risk groups were obtained from KEYNOTE-426 and CHECKMATE-214 trials for P + A and N + I, respectively, versus sunitinib as mRCC first-line treatment., Results: Considering a 12-month time horizon, 6 patients should be treated with P + A to prevent one death with sunitinib use, resulting in a COPE of 3,773,865 BRL. Using N + I, NNT for 12-month OS rate was 13 compared to sunitinib, with a COPE of 6,357,965 BRL. Regarding PFS data, NNT was also 6 when comparing P + A versus sunitinib, with an estimated COPE of 3,773,865 BRL. Estimated NNT was 20 comparing N + I and sunitinib, resulting in a COPE of 10,172,744 BRL. Cost differences between two treatment options, reached more than 6 million BRL for PFS, and 2 million BRL for OS., Conclusion: At the 12-month landmark, P + A suggests better economic scenario versus N + I as first-line mRCC treatment option for intermediate- and poor-risk groups, through an indirect comparison using sunitinib as a common comparator.

Background: Non-small cell lung cancer (NSCLC) with brain metastases (BM) is difficult to treat and associated with poor survival. This study assessed the impact of BM on healthcare-related utilization and costs (HRUC) among patients receiving epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs)., Patients and Methods: Adults newly-diagnosed with metastatic NSCLC, initiating first-/second-generation EGFR-TKI treatment, with BM or no BM (NBM), were identified retrospectively from IBM MarketScan healthcare claims databases (2013-2017). HRUC were measured during the variable-length follow-up period. Generalized linear models assessed the impact of BM on total healthcare costs, standardized to 2017 US$., Results: Overall, 222 BM and 280 NBM patients were included, with a mean duration of follow-up of 14 months. Adjusted NSCLC-related and all-cause costs over average follow-up were 1.2 times higher among BM patients (Δ$5,640 and Δ$6,366, respectively; p  <0.05); differences were driven primarily by radiation treatment and radiology. More than two times more BM than NBM patients received NSCLC-related radiation treatment, in both inpatient (15.3% vs 6.8%; p  <0.05) and outpatient settings (87.8% vs 37.5%; p  <0.05). Per-patient per-month (PPPM) radiation costs were also higher among BM patients, both inpatient ($796 vs $464, p  =0.172) and outpatient ($2,443 vs $747, p  <0.05). All-cause PPPM radiology visits (2.0 vs 1.3) and associated costs ($3,824 vs $1,621) were higher among BM patients (both p  <0.05)., Conclusion: NSCLC-related HRUC, especially those attributable to radiation treatment, were higher among patients with BM. Future research should compare the potential for CNS-active EGFR-TKIs vs first-/second-generation EGFR-TKIs combined with radiotherapy to reduce HRUC.

Background Hypertension requires life-long medical care, which may cause economic burden and even lead to catastrophic health expenditure. Objective To estimate the extent of out-of-pocket expenditure for hypertension care at a population level and its impact on households' budgets in a low-income urban setting in Colombia. Methods We conducted a cross-sectional survey in Santa Cruz, a commune in the city of Medellin. In 410 randomly selected households with a hypertensive adult, we estimated annual basic household expenditure and hypertension-attributable out-of-pocket expenditure. For socioeconomic stratification, we categorised households according to basic expenditure quintiles. Catastrophic hypertension-attributable expenditure was defined as out-of-pocket expenditure above 10% of total household expenditure. Results The average annual basic household expenditure was US dollars at purchasing power parity (USD-PPP) $12,255.59. The average annual hypertension-attributable out-of-pocket expenditure was USD-PPP $147.75 (95% CI 120.93-174.52). It was incurred by 73.9% (95% CI 69.4%-78.1%) of patients, and consisted mainly of direct non-medical expenses (76.7%), predominantly for dietary requirements prescribed as non-pharmacological treatment and for transport to attend health care consultations. Medical out-of-pocket expenditure (23.3%) was for the most part incurred for pharmacological treatment. Hypertension-attributable out-of-pocket expenditure represented on average 1.6% (95% CI 1.3%-1.9%) of the total annual basic household expenditure. Eight households (2.0%; 95% CI 1.0%-3.8%) had catastrophic health expenditure; six of them belonged to the two lowest expenditure quintiles. Payments related to dietary requirements and transport to consultations were critical determinants of their catastrophic expenditure. Conclusions Out-of-pocket expenditure for hypertension care is moderate on average, but frequent, and mainly made up of direct non-medical expenses. Catastrophic health expenditure is uncommon and affects primarily households in the bottom socioeconomic quintiles. Financial protection should be strengthened by covering the costs of chronic diseases-related dietary requirements and transport to health services in the most deprived households. Abbreviations NCDs: Non-communicable diseases; LMICs: Low and middle-income countries; WHO: World Health Organization; HTN: hypertension; CVDs: Cardiovascular diseases; OOPE: out-of-pocket expenditure; USD-PPP: US dollars at purchasing power parity; CI: Confidence interval.

Objective : To estimate all-claims-all-conditions expenditures paid for by health plans for patients suffering from Parkinson´s disease (PD). Methods : Using administrative claims data from two health maintenance organizations for 2014 and 2015 in Colombia, we identified 2,917 patients with PD by applying an algorithm that uses International Statistical Classification of Diseases and Related Health Problems and Anatomical Therapeutic Chemical Classification System codes. Descriptive statistics were applied to compute unadjusted all-cause median costs. A generalized linear model was used to estimate adjusted and attributable direct costs of advanced PD. Results : Approximately 30% of the all-cause direct costs were associated with technologies not included in universal health coverage benefit packages. In 2015, the annual median interquartile range per patient all-cause direct costs to insurers was USD1,576 (605-3,617). About 16% of patients had advanced PD. Regression analysis estimated that additional costs attributable to advanced PD was USD3,416 (p = 0.000). Multimorbidity was highly prevalent, and 96% of PD patients had at least one other chronic condition. Conclusions : In the context of high judicialization, patients suffering from PD must increasingly use the judicial system to access treatment. To promote more equitable and efficient access benefit packages, developing countries must consider more thoroughly the needs of these patients.

Background: Patients with relapsed or refractory diffuse large B-cell lymphoma (r/r DLBCL) have limited treatment options and poor prognoses. Tisagenlecleucel, a chimeric antigen receptor (CAR) T-cell therapy has shown early promise in improving survival outcomes, but at a high upfront cost. This study evaluated the cost-effectiveness of tisagenlecleucel versus salvage chemotherapy for treating patients with r/r DLBCL who have failed at least 2 lines of systemic therapies., Methods: A hybrid decision tree and three-state partitioned survival model (progression-free (PF), progressive disease and death) was developed from the Singapore healthcare payer perspective. Survival curves from JULIET trial and CORAL-1 extension study were extrapolated beyond trial period over a 15-year time horizon to estimate the underlying progression-free survival and overall survival parametric distributions for both arms. Health state utilities were retrieved from the literature, and direct costs were sourced from public healthcare institutions in Singapore. One-way probabilistic sensitivity analyses and scenario analyses were conducted to explore the impact of uncertainties and assumptions on cost-effectiveness results., Results: Compared with salvage chemotherapy, tisagenlecleucel was associated with a base-case incremental cost-effectiveness ratio (ICER) US$508,530 (S$686,516) per quality adjusted life year (QALY) gained and US$320,200 (S$432,269) per life year (LY) gained. One-way sensitivity analysis showed the ICER was most sensitive to time horizon, PF utility and cost of tisagenlecleucel. Scenario analyses confirmed that the ICERs remained high under favorable assumptions and substantial price reduction was required to reduce the ICER., Conclusions: Our analysis showed tisagenlecleucel use in r/r DLBCL patients who failed at least 2 prior lines of systemic therapies was associated with exceedingly high ICER, which is unlikely to represent good use of healthcare resources. Comparative clinical evidence from the ongoing trials might provide more insight into future evaluations.

Aims: To evaluate the prevalence and risk factors of migraine progression and to assess the incremental burden of migraine progression on healthcare systems., Materials and Methods: Adult patients were required to have a migraine diagnosis in IQVIA's US adjudicated claims database between 1 January 2012 and 30 June 2016, continuous enrollment ≥12 months before and after the index date (i.e. the first observed migraine diagnosis), and ≥1 additional migraine diagnosis claim during the 12-month post-index period. A previously-developed algorithm identified patients with prevention-eligible episodic migraine (EM). All-cause healthcare resource utilization (HCRU) and costs were evaluated at baseline, over the follow-up period and pre/post progression from prevention-eligible EM to chronic migraine. Cox proportional hazards models were used to evaluate risk factors associated with progression., Results, Limitations, and Conclusions: Of the 125,436 patients with prevention-eligible EM that were initially identified, 5,790 (4.6%) were further identified as progressed. Patients who progressed had higher healthcare costs and higher medication use at baseline compared to patients that did not progress. Mean (SD) all-cause total costs per patient per month were $1,790 ($3,788), significantly higher in the post-progression period compared to $1,414 ($2,456) in the pre-progression period in patients who progressed ( p  < .0001). Younger age, female sex, initial diagnosis by a neurologist, chronic pain, and use of triptans and/or non-specific acute medications were all significant progression risk factors. Results are limited by the use of a heterogeneous population (incident, prevalent, treated, and untreated patients), coding biases, and lack of information on non-prescription drug utilization and plan limits. Limitations aside, there are substantial HCRU and cost burden associated with migraine progression. Younger age, female sex, and the use of specific drug classes are likely to increase migraine disease progression risk.

Objectives: To provide the most current assessment of real-world healthcare resource utilization (HRU) and costs among patients with non-valvular atrial fibrillation (NVAF) who newly initiated rivaroxaban and apixaban using a large US database., Material and Methods: A retrospective weighted cohort design was used with healthcare insurance claims from the Optum Clinformatics Data Mart databases (January 2012-December 2018). The index date was defined as the first dispensing of rivaroxaban or apixaban. Adult NVAF patients with an index date on or after 1 January 2016, ≥ 12 months of continuous eligibility before the index date and ≥ 1 month after, and without prior use of oral anticoagulant were included. The observation period spanned from the index date to the earliest of the end of data availability, end of insurance coverage, or death. Inverse probability of treatment weighting (IPTW) was used to adjust for differences in baseline characteristics between cohorts. All-cause healthcare resource utilization (HRU), including hospitalization, emergency room, and outpatient visits, and healthcare costs, including medical and pharmacy costs, were evaluated from the payer's perspective during the observation period up to 18 and 24 months, separately., Results: In total, 23,822 rivaroxaban and 53,666 apixaban users were included. After weighting, all baseline characteristics were well balanced between cohorts (mean age: 73.8 years, female: 46.6% in both cohorts). Up to 18 months of follow-up, rivaroxaban users incurred significantly lower total healthcare costs compared to apixaban users (cost difference = -$1,121; p  = 0.020), driven by significantly lower rates of outpatient hospital visits and associated costs (cost difference = -$1,579; p  < 0.001). Similar results were found in the analysis conducted for up to 24 months of follow-up (total cost difference = ‒$1,111; p  = 0.020)., Conclusions: In this large retrospective analysis, patients with NVAF initiated on rivaroxaban incurred significantly lower healthcare costs compared to those initiated on apixaban, which were primarily driven by significantly lower outpatient visits and costs during the 18- and 24-month follow-up periods.

Objective: Non-small cell lung cancer (NSCLC) accounts for 80-90% of all lung cancer cases and is usually associated with a poor prognosis. However, targeted therapy with first and second generation tyrosine kinase inhibitors (TKIs) has so far improved progression-free survival of epidermal growth factor receptor (EGFR) mutant NSCLC patients. Osimertinib, a third generation EGFR TKI has recently shown improved overall survival of 6.8 months in previously untreated EGFR mutant NSCLC patients. We assessed the cost-effectiveness of osimertinib versus standard EGFR TKIs (gefitinib or erlotinib) as first-line treatment for advanced or metastatic EGFR mutant NSCLC patients in Singapore., Methods: A partitioned survival model with three health states (progression-free, progressive disease, and death) was developed from the Singapore healthcare payer perspective. Survival curves based on the overall trial population from the FLAURA trial were extrapolated beyond trial period over a 10-year time horizon to estimate the underlying progression-free survival and overall survival parametric distributions. Health state utilities were derived from the literature and direct costs were sourced from public healthcare institutions in Singapore. Deterministic and probabilistic sensitivity analyses were conducted to explore the impact of uncertainties and assumptions on cost-effectiveness results., Results: Compared with first or second generation TKI, osimertinib had a base-case incremental cost-effectiveness ratio (ICER) of SG$418,839 (US$304,277) per quality-adjusted life year gained. One-way sensitivity analysis showed the ICER was most sensitive to time horizon and variations in progression-free utility values. Scenario analyses showed that a 50% reduction in the cost of osimertinib was still associated with a high ICER that was unlikely to be deemed cost effective., Conclusions: Osimertinib is not cost effective as a first-line treatment compared to standard EGFR TKIs in advanced EGFR mutant NSCLC patients in Singapore. The findings from our evaluation, alongside other considerations including the lack of survival benefit in the Asian subgroup of the FLAURA trial, will be useful to inform policy makers on funding decisions for NSCLC treatments in Singapore.

Background and Aims: The ReFLeCT study demonstrated that switching to insulin degludec from other basal insulins was associated with reductions in glycated hemoglobin and hypoglycemic events in type 1 (T1D) and type 2 diabetes (T2D), and reductions in insulin doses in T1D. The aim of the present analysis was to assess the short- and long-term cost-effectiveness of switching to insulin degludec in Sweden., Methods: Short-term outcomes were evaluated over 1 year in a Microsoft Excel model, while long-term outcomes were projected over patient lifetimes using the IQVIA CORE Diabetes Model. Cohort characteristics and treatment effects were sourced from the ReFLeCT study. Costs (in 2018 Swedish krona [SEK]) encompassed direct medical expenditure and indirect costs from loss of workplace productivity. In the long-term analyses, patients were assumed to receive insulin degludec or continue prior insulin therapy (primarily insulin glargine U100) for 5 years, before all patients intensified to once-daily degludec and mealtime aspart., Results: Switching to insulin degludec was associated with improved quality-adjusted life expectancy of 0.04 and 0.02 quality-adjusted life years (QALYs) over 1 year, and 0.16 and 0.08 QALYs over patient lifetimes, in T1D and T2D. Combined costs in T1D and T2D were estimated to be SEK 1,249 lower and SEK 1,181 higher over the short-term, and SEK 157,258 and SEK 2,114 lower over the long-term. Benefits were due to lower insulin doses in T1D, reduced rates of hypoglycemia, and lower incidences of diabetes-related complications. Insulin degludec was associated with an incremental cost-effectiveness ratio of SEK 64,298 per QALY gained for T2D over 1 year and considered dominant for T1D and T2D in all other comparisons., Conclusions: Insulin degludec was projected to be cost-effective or dominant versus other basal insulins for the treatment of T1D and T2D in Sweden.

Aims: The aim of this health economics study was to estimate the cost-utility of an aseptically processed, dehydrated human amnion and chorion allograft (dHACA) plus standard of care (SOC) (group 1) versus SOC alone (group 2) based on a published randomized controlled trial in which patients who had an eligible Wagner 1 diabetic foot ulcer wound were randomized to either of these treatments., Materials and Methods: A Markov microsimulation was used to project trial results out to a 1-year horizon time with a third-party payer perspective. The starting health state was an unhealed non-infected ulcer with other health states of healed ulcer, infected non-healed ulcer, cellulitis, osteomyelitis, and absorbing states of dead or amputation. All patients started with unhealed non-infected ulcers at cycle 0. Costs were incurred by patients for procedures at hospital outpatient wound care provider-based departments (PBDs) and hospitals (if complications occurred) and were calculated using time-based activity costing methods. Effectiveness units were quality-adjusted life years (QALYs) computed from literature utility values. One-way and probabilistic sensitivity analysis (PSA) were also conducted., Results: After 1 year, the calculated incremental cost-effectiveness ratio (ICER) for group 1 versus group 2 was -$4,373 with group 1 (dHACA) being dominant over group 2 (SOC). PSA demonstrated that group 1 had 69.2% lower cost values with increased positive incremental effectiveness for 94.9% of values. A willingness to pay (WTP) curve showed that about 92% of interventions were cost effective for group 1 when $50,000 was paid., Conclusions: The results of this study demonstrated that dHACA added to SOC compared to SOC alone was extremely cost-effective in the defined trial population.

Aims: To quantify the long-term direct and indirect costs among patients with Crohn's disease (CD) and specific subgroups of these patients in the United States from the private payer's perspective., Materials and Methods: This retrospective study used the OptumHealth Care Solutions, Inc database (01 January 1999-31 March 2017) to match (1:5) adult patients with ≥2 claims for CD to patients without inflammatory bowel disease (IBD). Patterns observed during follow-up (i.e. biologics, opioids, or steroids; CD-related surgery; moderate-to-severe disease; and comorbidities) were used to identify CD subgroups. Comparisons of healthcare resource utilization, work loss days, and direct and indirect work loss-related costs were made between matched cohorts. Descriptive analyses of costs were conducted within each CD subgroup., Results: There were 6,715 and 33,575 patients in the CD and non-IBD cohorts, respectively. The direct burden was significantly higher in the CD cohort compared to the non-IBD cohort, with 0.34 inpatient admissions per patient per year (PPPY) versus 0.12 (217% increase; p  < .001), and $24,500 direct healthcare costs PPPY versus $7,037 ($17,463 increase; p  < .001). The trend was similar for the indirect burden, with work loss-related costs PPPY of $5,490 in the CD cohort versus $3,322 in the non-IBD cohort ($2,168 increase; p  < .001). The burden was numerically higher in the CD subgroups, with direct healthcare costs reaching $101,013 PPPY in the surgery subgroup., Limitations: Severity of CD was determined based on claims-based algorithms due to the lack of access to medical files. Absenteeism was imputed based on claims data, and presenteeism was not assessed., Conclusions: The direct healthcare and indirect work loss-related costs of patients with CD was significantly higher compared to patients without IBD over an average follow-up of 5 years.

Aims: This study's objectives were to examine and compare the cost-effectiveness of biologic and non-biologic therapies in the improvement of the health-related quality of life (HRQoL) of patients with inflammatory bowel disease (IBD) in Saudi Arabia., Materials and Methods: This retrospective cohort study analyzed data from the medical records of patients with IBD treated at a tertiary-care hospital in Riyadh, Saudi Arabia. Drug utilization costs and HRQoL scores were evaluated at baseline and after six months of treatment. Patients' HRQoL was measured using the Arabic version of the standardized EuroQol 5 Dimensional 3 Level (EQ-5D-3L) questionnaire with a visual analog scale (VAS)., Results: Eighty-seven patients with Crohn's disease (CD) and 69 patients with ulcerative colitis (UC) were included in the study ( N  = 156), and 59 (37.82%) were treated with biologics. Similar effects of both types of medications were found on the HRQoL domains of mobility, usual activities, and pain and discomfort, while biologics outperformed non-biologics on the self-care domain. The mean utilization cost of a biologic-based treatment over a six-month period was SAR 25,690.46 (USD 6,850.79) higher than that of the non-biologic treatment (95% confidence interval (CI): 24,548.55-27,465.11), and the change in the ED-5D-3L VAS score from baseline to follow-up was 4.78 points (95% CI: 1.96-14.00). A probabilistic sensitivity analysis demonstrated that IBD therapy with biologic-based treatment is always more expensive, but also more effective in improving HRQoL 99.45% of the time. Adalimumab was found to be less cost effective than infliximab in the management of CD., Limitations: Information bias cannot be ruled out, as this investigation was a retrospective cohort study with a relatively small sample that was not randomized., Conclusions: The results of this analysis can serve as a foundation to introduce HRQoL-based recommendations for the use of biologics in the management of IBD in Saudi Arabia.

Background: This study aimed to understand the clinical and economic burden associated with postsurgical complications in high-risk surgeries in Thailand., Methods: A cost and outcome study was conducted using a retrospective cohort database from four tertiary hospitals. All patients with high-risk surgeries visiting the hospitals from 2011 to 2017 were included. Outcomes included major postsurgical complications, length of stay (LOS), in-hospital death, and total healthcare costs. Multivariate regression analyses were performed to identify risk factors of postsurgical outcomes., Results: A total of 14,930 patients were identified with an average age of 57.7 ± 17.0 years and 34.9% being male. Gastrointestinal (GI) procedures were the most common high-risk procedures, accounting for 54.9% of the patients, followed by cardiovascular (CV) procedures (25.2%). Approximately 27.2% of the patients experienced major postsurgical complications. The top three complications were respiratory failure (14.0%), renal failure (3.5%), and myocardial infarction (3.4%). In-hospital death was 10.0%. The median LOS was 9 days. The median total costs of all included patients were 2,592 US$(IQR: 1,399-6,168 US$). The patients, who received high-risk GI surgeries and experienced major complications, had significantly increased risk of in-hospital death (OR: 4.53; 95%CI: 3.81-5.38), longer LOS (6.53 days; 95%CI: 2.60-10.46 days) and higher median total costs (2,465 US$; 95%CI: 1,945-2,984 US$), compared to those without major complications. Besides, the patients, who underwent high-risk CV surgeries and developed major complications, resulted in significantly elevated risk of in-hospital death (OR: 2.22; 95%CI: 1.74-2.84) and increased median total costs (2,719 US$; 95%CI: 2,129-3,310 US$), compared to those without major complications., Conclusions: Postsurgical complications are a serious problem in Thailand, as they are associated with worsening mortality risk, LOS, and healthcare costs. Clinicians should develop interventions to prevent or effectively treat postsurgical complications to mitigate such burdens.

Aims: Obinutuzumab (GA101; G) is a new treatment for follicular lymphoma (FL) that is anticipated to have greater efficacy than the current treatment, rituximab (R). The aim of this study was to evaluate the cost-effectiveness of G plus chemotherapy (G + Chemo) against that of R plus chemotherapy (R + Chemo) for patients in Japan with previously untreated FL., Materials and Methods: We localized a previously reported cost-effectiveness model using the Japanese cost data. For estimating costs adapted in the model, FL patients treated with R were identified from Japanese hospital-based claims database and classified into three treatment regimen groups according to chemotherapies used with R: CHOP, CVP, and bendamustine (B). Based on services per patient and cost items, parameters determining the cost amounts were derived using a multivariate generalized linear mixed model to estimate the direct medical costs for each treatment regimen group for G and R. For the utility, same values in the previous model were used. Lifetime cost and quality-adjusted life year (QALY) were estimated under the payer's perspective., Results: The calculated incremental cost-effectiveness ratios (ICERs) in million JPY per QALY for G-CHOP vs. R-CHOP, G-CVP vs. R-CVP, and G-B vs. R-B were 5.4, 4.3, and 4.8, respectively. ICERs were lower than 7.5 million JPY per QALY, which is the cost-effectiveness threshold for cancer treatments, and showed that G + Chemo is a cost-effective treatment regimen for Japanese patients with previously untreated FL. Lifetime direct medical costs were lowest in the R-B group because hospitalization costs that accounted for most of the total cost were lowest in this group. Limitations and conclusions: The cost-effectiveness of G + Chemo is acceptable in Japan. Differences in the direct medical costs among treatment regimen groups were mostly due to hospitalization costs. This is probably because many Japanese hematologists choose inpatient treatments over outpatient treatments in CHOP-based induction therapy.

Aims: To characterize a US population of patients with irritable bowel syndrome with constipation (IBS-C) or chronic idiopathic constipation (CIC) using CONTOR, a real-world longitudinal research platform that deterministically linked administrative claims data with patient-reported outcomes data among patients with these conditions., Methods: Patients with IBS-C or CIC were identified using diagnosis and treatment codes from administrative claims. Potential respondents received a mailed survey followed by 12 monthly online follow-up surveys and 2 mailed diaries. Surveys collected symptom severity, treatment use, quality of life, productivity, and condition/treatment history. Comorbidities and healthcare costs/utilization were captured from claims data. Diaries collected symptoms, treatments, and clinical outcomes at baseline and 12 months. Data were linked to create a patient-centric research platform., Results: Baseline surveys were returned by 2,052 respondents (16.8% response rate) and retention rates throughout the study were high (64.8%-70.8%). Most participants reported burdensome symptoms despite having complex treatment histories that included multiple treatments over many years. More than half (55.3%) were dissatisfied with their treatment regimen; however, a higher proportion of those treated with prescription medications were satisfied., Limitations: The study sample may have been biased by patients with difficult-to-treat symptoms as a result of prior authorization processes for IBS-C/CIC prescriptions. Results may not be generalizable to uninsured or older populations because all participants had commercial insurance coverage., Conclusions: By combining administrative claims and patient-reported data over time, CONTOR afforded a deeper understanding of the IBS-C/CIC patient experience than could be achieved with 1 data source alone; for example, participants self-reported burdensome symptoms and treatment dissatisfaction despite making few treatment changes, highlighting an opportunity to improve patient management. This patient-centric approach to understanding real-world experience and management of a chronic condition could be leveraged for other conditions in which the patient experience is not adequately captured by standardized data sources.

Aims: Healthcare resource utilization (HRU) and costs in post-transplant lymphoproliferative disease (PTLD) patients following allogeneic hematopoietic stem cell transplant (HCT) were evaluated in the USA., Methods: MarketScan Commercial and Medicare Supplemental database claims from 01 July 2010 to 31 December 2017 were analyzed. Patients eligible for analysis received allogeneic HCT between 01 January 2011 to 31 December 2015, had ≥6 months of continuous enrollment before HCT, and had ≥1 claim for PTLD or ≥1 inpatient or ≥2 outpatient claims for a clinically-relevant lymphoma within 1 year following HCT (PTLD index = first claim of diagnosis). Patients with clinically-relevant lymphomas within 6 months before HCT were excluded. HRU and total paid amounts were assessed from the week before the HCT through 1-day pre-PTLD index (HCT to PTLD) and monthly from PTLD index through 1-year post-PTLD index. HRU is reported as mean (SD). Results were also provided by survival status., Results: Overall, 92 patients were eligible for analysis. From HCT to PTLD, 98.9% of patients were hospitalized, with 1.7 (1.2) hospitalizations/patient. The average length of stay was 25.3 (22.2) days/patient. From HCT to PTLD, 98.9% of patients had outpatient services with 233.7 (261.1) services/patient and 91.3% of patients had a prescription fill with 32.9 (26.0) prescriptions/patient. In the first month post-PTLD index, 51.2% of patients were hospitalized. Mean paid amounts were $399,470/patient (range $7542-$1.7 M) from HCT to PTLD. Cumulative mean paid amounts 1-year post-PTLD were $429,043/patient. Total cost/patient/month was ∼7 times higher in patients who died ( n  = 49; $232,591) than those who lived ( n  = 43; $33,677). Costs were mainly driven by hospitalizations., Limitations: Limitations include those inherent to retrospective analyses (i.e. miscoding, lack of clinical detail)., Conclusions: HRU and costs from HCT to PTLD were high and more than doubled within 1-year post-PTLD. PTLD patients who died had ∼7 times higher costs than those who lived, driven by hospitalizations. Effective treatments are needed to reduce the burden of PTLD.

Aims: This study estimated the total costs associated with tisagenlecleucel treatment in adult patients with relapsed/refractory (r/r) diffuse large B-cell lymphoma (DLBCL) based on the JULIET trial from a United States hospital's perspective. Methods: An economic model was developed to assess the total costs associated with tisagenlecleucel treatment (from leukapheresis to two months post-infusion) in adults (aged ≥18 years) with r/r DLBCL using a fee-for-service approach. Costs were considered during the pre-treatment, tisagenlecleucel infusion, and follow-up periods, and were estimated based on the health resource utilization and safety data from the JULIET trial. Cost components included leukapheresis, lymphodepleting chemotherapy, tisagenlecleucel infusion/administration, inpatient and intensive care unit (ICU) admission, medical professional visits, lab tests/procedures, and management of adverse events (AEs). The base-case model estimated the total costs using observed hospitalization, ICU, and AE data from JULIET, while scenario analyses varied key assumptions related to AEs and hospitalization. Results: The estimated overall cost associated with tisagenlecleucel treatment from leukapheresis to two months post-infusion was $437,927/patient, of which $64,784 (14.8%) was additional to tisagenlecleucel's list price ($373,000) and the associated administration cost ($143). The top three key drivers of the additional cost were AE management ($30,594; 47.2%), inpatient/ICU not attributed to AEs ($24,285; 37.5%), and lab tests/procedures ($5,443; 8.4%). In the scenario analyses, total costs ranged from $382,702 (no AEs, no hospitalization) to $469,006 (cytokine release syndrome and B-cell aplasia, hospitalization). Limitations : This analysis was limited to two months of follow-up after tisagenlecleucel infusion, which cannot capture long-term safety outcomes associated with the treatment and may underestimate AE costs. Conclusions: The total cost of tisagenlecleucel administration from leukapheresis to two months was estimated at $437,927. In addition to tisagenlecleucel's price, the main drivers were AE management costs and inpatient/ICU costs. Future studies based on real-world, long-term use of tisagenlecleucel are warranted.

Aims: Pembrolizumab demonstrated significantly prolonged overall survival (OS) vs. chemotherapy in the Phase III KEYNOTE-045 trial, and is approved in the US for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who progressed after platinum-based chemotherapy. Using longer follow-up and individual patient-data from KEYNOTE-045, this study evaluates the cost-effectiveness of pembrolizumab vs. chemotherapy or atezolizumab from a US payer perspective. Materials and methods: A partitioned-survival model was developed over a 20-year time horizon. Progression-free survival (PFS) and OS for pembrolizumab and chemotherapy were extrapolated using a piecewise modelling approach, where patient-level data from KEYNOTE-045 were used for the initial period followed by parametric distributions. OS of atezolizumab was estimated by indirect treatment comparisons based on KEYNOTE-045 and IMvigor211. Different scenarios were explored in the absence of indirect comparisons on PFS and time-on-treatment (ToT) between pembrolizumab and atezolizumab. Drug acquisition/administration, disease management, adverse events, and terminal care costs were considered. Results: Compared with chemotherapy, pembrolizumab resulted in a mean gain of 1.33 life-years and 1.14 quality-adjusted life-years (QALYs) and an incremental cost of $106,299, yielding an incremental cost-effectiveness ratio of $93,481/QALY gained. Pembrolizumab dominated atezolizumab in extending patients' life by 0.89 years and 0.76 QALYs, while reducing costs by $26,458. Key drivers of cost-effectiveness included survival extrapolation, OS hazard ratio of pembrolizumab vs. atezolizumab, and time horizon. Pembrolizumab had a 66% and 100% probability of being cost-effective vs. chemotherapy and atezolizumab, respectively, at a $100,000 willingness-to-pay threshold. Limitations and conclusions: Uncertainties remain with extrapolated PFS and OS for pembrolizumab, OS indirect comparison, and ToT for atezolizumab. Despite these limitations, the model used robust methods to estimate key clinical endpoints with patient-level data from longer follow-up of KEYNOTE-045. Pembrolizumab dominates atezolizumab and is very likely cost-effective vs. chemotherapy in 2 L mUC at a $100,000 willingness-to-pay threshold.

Background: Stress urinary incontinence (SUI) is a debilitating and highly prevalent condition in the UK. The condition is associated with a significant economic burden for affected patients and society. Current treatment options for SUI include minimally invasive therapies, medication and surgical intervention for the most serious cases. Electrical Muscle Stimulator with Multipath technology is a recently developed device for the treatment of SUI that relies on neuromuscular external electrical stimulation (NEES) technology. The clinical efficacy of the device has been proven in previous studies, but existing evidence surrounding its economic viability is limited. Objectives: To assess the cost-utility of the Electrical Muscle Stimulator with Multipath technology Therapy device for the treatment of SUI amongst women in a UK setting. Methods: An economic model was developed to consider the cost-utility (cost per quality-adjusted life-year [QALY] gained) of Electrical Muscle Stimulator with Multipath technology compared with current practice. A Markov model was developed, with costs and health effects estimated over the lifetime of the patient in the base-case analysis. The model was developed to reflect the treatment pathways typically followed by patients with SUI in the UK. Parameter uncertainty was explored in deterministic and probabilistic sensitivity analyses. Results: Base-case results indicate that Electrical Muscle Stimulator with Multipath technology results in cost savings and QALY gains over the patient's lifetime. In the "cure" analysis, the intervention is £250 less costly and leads to a 0.03 QALY gain per patient, while in the "improvement analysis", the intervention is £327 less costly and leads to a 0.13 QALY gain per patient. Results from the probabilistic sensitivity analyses show that the likelihood of Electrical Muscle Stimulator with Multipath technology being cost-effective is greater than 74% across all willingness-to-pay thresholds in the two analyses presented. Conclusions: Electrical Muscle Stimulator with Multipath technology is a potentially cost-effective treatment option for patients with SUI who have failed first-line treatment. It could reduce costs to the health care service and improve quality-of-life for selected patients over their lifetime.

Background: Neuroblastoma (NB) is notorious in childhood cancer because of its high incidence and poor prognosis. The Children's Oncology Group reported that the 3-year OS in the high-risk (HR) group is 50%, and the HR-NB with bone marrow metastasis in our center is 43.1%. Thousands of families in China suffer from the cost of NB, but the true costs of therapy are unknown; to date, no study has ever performed a detailed therapy costs analysis for NB. The objective of this study was to assess the economic burden of NB treatment in children to the family, to ultimately reduce related expenses for patients and promote the establishment of NB management policy., Materials and Methods: Data in this cross-sectional study were collected via questionnaires completed by parents at the outpatient clinic and was verified via a computer system. Therapy costs of children with NB of differing risks were analyzed through descriptive statistics (1 CNY ≈ 0.1412 USD)., Results: Median direct medical costs of low risk (LR), middle risk (MR), and HR NB during treatment were 180.0 (120.0, 300.0), 200.0 (166.0, 300.0), and 650.0 (415.5, 850.0) thousand Chinese yuan (CNY), respectively. Direct non-medical costs including transportation, food, and accommodation were 60.0 (37.0, 100.0), 80.0 (60.0, 120.0), and 100.0 (80.0, 157.5) thousand CNY in the LR, MR, and HR groups, respectively. Additionally, parents accrued work absences to attend treatment, and lost a total of 100.0 (50.0, 150.0) thousand CNY in indirect costs., Limitations: Families whose children had relapsed or died were excluded from this analysis and therefore limited the conclusions drawn. Parents were asked to recall costs since initial diagnosis (1-6 years in the past), but this extended time period may have introduced recall bias., Conclusions: Direct non-medical and indirect costs play an important role in the total treatment costs of NB. Children with NB treated in local hospitals and followed up in hospital specialized in childhood oncology may save many unnecessary expenses. China's healthcare system should establish mechanisms and provide financial support for children with NB.