Cost-effective Treatment For Patients Research Articles

An economic evaluation of eptinezumab for the preventive treatment of migraine in the UK, with consideration for natural history and work productivity

BackgroundMigraine is a highly prevalent neurological disease with a substantial societal burden due to lost productivity. From a societal perspective, we assessed the cost-effectiveness of eptinezumab for the preventive treatment of migraine.MethodsAn individual patient simulation of discrete competing events was developed to evaluate eptinezumab cost-effectiveness compared to best supportive care for adults in the United Kingdom with ≥ 4 migraine days per month and prior failure of ≥ 3 preventive migraine treatments. Individuals with sampled baseline characteristics were created to represent this population, which comprised dedicated episodic and chronic migraine subpopulations. Clinical efficacy, utility, and work productivity inputs were based on results from the DELIVER randomised controlled trial (NCT04418765). Timing of natural history events and treatment holidays—informed by the literature—were simulated to unmask any natural improvement of the disease unrelated to treatment. The primary outcomes were monthly migraine days, migraine-associated costs, quality-adjusted life years (QALYs), incremental cost-effectiveness ratio, and net monetary benefit, each evaluated over a 5-year time horizon from 2020. Secondary analyses explored a lifetime horizon and an alternative treatment stopping rule.ResultsTreatment with eptinezumab resulted in an average of 0.231 QALYs gained at a saving of £4,894 over 5 years, making eptinezumab dominant over best supportive care (i.e., better health outcomes and less costly). This result was confirmed by the probabilistic analysis and all alternative assumption scenarios under the same societal perspective. Univariate testing of inputs showed net monetary benefit was most sensitive to the number of days of productivity loss, and monthly salary.ConclusionsThis economic evaluation shows that from a societal perspective, eptinezumab is a cost-effective treatment in patients with ≥ 4 migraine days per month and for whom ≥ 3 other preventive migraine treatments have failed.Trial registrationN/A.Graphical

Decreasing alloimmunization-specific mortality in sickle cell disease in the United States: Cost-effectiveness of a shared transfusion resource.

Red blood cell alloimmunization and consequent delayed hemolytic transfusion reaction (DHTR) incidence and mortality in patients with sickle cell disease (SCD) are high. A shared transfusion resource has decreased both in other countries, while in the United States cost concerns persist. We conducted a Markov cohort simulation of a birth cohort of alloimmunized patients with SCD to estimate lifetime DHTR incidence, DHTR-specific mortality, quality-adjusted life expectancy (QALE), and costs with the implementation of a shared transfusion resource to identify antibody history versus without (i.e., status quo). We conducted our analysis using a lifetime analytic time horizon and from a United States health system perspective. Implementation of shared transfusion resource projects to decrease cumulative DHTR-specific mortality by 26% for alloimmunized patients with SCD in the United States, relative to the status quo. For an average patient population of 32 000, this intervention would generate a discounted increment of 4000 QALYs at an incremental discounted cost of $0.3 billion, resulting in an incremental cost-effectiveness ratio of $75 600/QALY [95% credible interval $70 200-81 400/QALY]. The results are most sensitive to the baseline lifetime medical expenditure of patients with SCD. Alloantibody data exchange is cost-effective in 100% of 10 000 Monte Carlo simulations. The resource would theoretically need a minimum patient population of 1819 patients or cost no more than $5.29 million annually to be cost-effective. By reducing DHTR-specific mortality, a shared transfusion resource in the United States projects to be a life-saving and cost-effective intervention for patients with SCD in the United States.

Budget Impact of RefluxStop™ as a Treatment for Patients with Refractory Gastro-oesophageal Reflux Disease in the United Kingdom.

Background: Gastro-oesophageal reflux disease (GORD) is a common condition associated with heartburn and regurgitation. Standard of care for GORD patients in the UK involves initial treatment with proton pump inhibitors (PPIs) and laparoscopic antireflux surgery in patients unwilling to continue or intolerant of long-term PPI treatment. Recently, RefluxStop™, a novel, implantable medical device, has proven to be an efficacious and cost-effective treatment for patients with GORD. The current analysis aimed to describe the budget impact of introducing RefluxStop™ within National Health Service (NHS) England and Wales. Objectives: To estimate the more immediate, short-term clinical and economic effects of introducing RefluxStop™ as a therapeutic option for patients with GORD treated within NHS England and Wales. Methods: A model adherent to international best practice guidelines was developed to estimate the budget impact of introducing RefluxStop™ over a 5-year time horizon, from an NHS perspective. Two hypothetical scenarios were considered, one without RefluxStop™ (comprising PPI treatment, laparoscopic Nissen fundoplication, and magnetic sphincter augmentation using the LINX® system) and one with RefluxStop™ (adding RefluxStop™ to the aforementioned treatment options). Clinical benefits and costs associated with each intervention were included in the analysis. Results: Over 5 years, introducing RefluxStop™ allowed the avoidance of 347 surgical failures, 39 reoperations, and 239 endoscopic esophageal dilations. The financial impact of introducing RefluxStop™ was £3 029 702 in year 5, corresponding to a 1.68% increase in annual NHS spending on GORD treatment in England and Wales. Discussion: While the time horizon was too short to capture some of the adverse events of PPIs and complications of GORD, such as the development of Barrett's esophagus or esophageal cancer, the use of RefluxStop™ was associated with a substantial reduction in surgical complications, including surgical failures, reoperations, and endoscopic esophageal dilations. This favorable clinical profile resulted in cost offsets for the NHS and contributed to the marginal budget impact of RefluxStop™ estimated in the current analysis. Conclusions: Introducing RefluxStop™ as a treatment option for patients with GORD in England and Wales may be associated with clinical benefits at the expense of a marginal budget impact on the NHS.

Budget Impact of RefluxStopTM as a Treatment for Patients with Refractory Gastro-oesophageal Reflux Disease in the United Kingdom

Background: Gastro-oesophageal reflux disease (GORD) is a common condition associated with heartburn and regurgitation. Standard of care for GORD patients in the UK involves initial treatment with proton pump inhibitors (PPIs) and laparoscopic antireflux surgery in patients unwilling to continue or intolerant of long-term PPI treatment. Recently, RefluxStop™, a novel, implantable medical device, has proven to be an efficacious and cost-effective treatment for patients with GORD. The current analysis aimed to describe the budget impact of introducing RefluxStop™ within National Health Service (NHS) England and Wales. Objectives: To estimate the more immediate, short-term clinical and economic effects of introducing RefluxStop™ as a therapeutic option for patients with GORD treated within NHS England and Wales. Methods: A model adherent to international best practice guidelines was developed to estimate the budget impact of introducing RefluxStop™ over a 5-year time horizon, from an NHS perspective. Two hypothetical scenarios were considered, one without RefluxStop™ (comprising PPI treatment, laparoscopic Nissen fundoplication, and magnetic sphincter augmentation using the LINX® system) and one with RefluxStop™ (adding RefluxStop™ to the aforementioned treatment options). Clinical benefits and costs associated with each intervention were included in the analysis. Results: Over 5 years, introducing RefluxStop™ allowed the avoidance of 347 surgical failures, 39 reoperations, and 239 endoscopic esophageal dilations. The financial impact of introducing RefluxStop™ was £3 029 702 in year 5, corresponding to a 1.68% increase in annual NHS spending on GORD treatment in England and Wales. Discussion: While the time horizon was too short to capture some of the adverse events of PPIs and complications of GORD, such as the development of Barrett’s esophagus or esophageal cancer, the use of RefluxStop™ was associated with a substantial reduction in surgical complications, including surgical failures, reoperations, and endoscopic esophageal dilations. This favorable clinical profile resulted in cost offsets for the NHS and contributed to the marginal budget impact of RefluxStop™ estimated in the current analysis. Conclusions: Introducing RefluxStop™ as a treatment option for patients with GORD in England and Wales may be associated with clinical benefits at the expense of a marginal budget impact on the NHS.

Olaparib plus bevacizumab as a first-line maintenance treatment for patients with advanced ovarian cancer by molecular status: an updated PAOLA-1 based cost-effectiveness analysis.

The PAOLA-1 trial (NCT02477644) reported final survival benefit associated with olaparib plus bevacizumab maintenance treatment of patients with advanced ovarian cancer (AOC) based on molecular status. Our aimed to compare the cost-effectiveness of olaparib plus bevacizumab for overall patients, patients with a breast cancer susceptibility genes (BRCA) mutation, homologous recombination deficiency (HRD), or HRD without BRCA mutations AOC from the context of the American healthcare system. Analysis of health outcomes in life-years (LYs), quality-adjusted life-years (QALYs), and the incremental cost-effectiveness ratio (ICER) in various molecular status-based AOC patient at a $150,000/QALY of willingness-to-pay was performed using a state-transitioned Markov model with a 20-year time horizon. Meanwhile, sensitivity analyses assessments were also used to gauge the model's stability. The ICERs of olaparib plus bevacizumab versus bevacizumab alone were $487,428 ($374,758), $249,579 ($191,649), $258,859 ($198,739), and $270,736 ($206,640) per QALY (LY) in the overall patients, patients with BRCA mutations, patients with HRD, and patients with HRD without BRCA mutations AOC, respectively, which indicated that The ICERs was higher than $150,000/QALY in the US. Progression-free survival (PFS) value and olaparib cost emerged as the primary influencing factors of these findings in the sensitivity analysis. At current cost levels, olaparib plus bevacizumab treatment is not a cost-effective treatment for patients with AOC regardless of their molecular status in the US. However, this maintenance treatment may be more favorable health advantages for patients with BRAC mutations AOC.

The general public's attitude towards accepting payment for kidney donation.

Kidney transplantation has become the most cost-effective treatment for patients with end-stage kidney disease (ESKD) and offers them the highest quality of life. Yet, kidney donation is often inaccessible due to cultural and traditional beliefs about organ donation. The goal of our study is to assess the value of kidney donation using the Willingness to Accept (WTA) technique. We also aim to understand the factors influencing an individual's willingness to donate an organ. A self-administered survey was completed by 985 participants from the general public. The quantitative method and survey design that were chosen used descriptive, correlational, nonparametric, and multivariate statistical tests. Most of the respondents, 895 (90.9%) are not willing to donate a kidney while alive. Four hundred and five (41.1%) of the respondents are not willing to donate a kidney after their death, while the rest are willing to donate their kidney after their death without financial compensation. The same attitude applies to the donation of a kidney from their relatives. Significant predictors from the results of the logistic regression model in predicting the lowest (minimal) amount that will encourage donation of one kidney after death were: Marital status; Nationality; Adi card holder; Knowing people who need a kidney donation; confidence in the medical staff; and consideration of the family's opinions regarding organ donation. Using cost benefit analysis (CBA), with the aim of evaluating the willingness of individuals to accept payment for innovative medical procedures, such as kidney donation, allows an assessment of the perceived value of the medical procedure and enables policymakers to decide whether to allocate funds or offer subsidies for kidney donation, given the limited healthcare resources available. During our research, we found that most participants did not support the commercialization of organs. Our recommendation for policymakers and health professionals is to continue providing adequate funding for kidney donations and to implement educational programs aimed at improving attitudes towards organ donation.

Applying Artificial Intelligence to Identify Common Targets for Treatment of Asthma, Eczema, and Food Allergy

Introduction: Allergic disorders are common diseases marked by the abnormal immune response toward foreign antigens that are not pathogens. Often patients with food allergy also suffer from asthma and eczema. Given the similarities of these diseases and a shortage of effective treatments, developing novel therapeutics against common targets of multiple allergies would offer an efficient and cost-effective treatment for patients. Methods: We employed the artificial intelligence-driven target discovery platform, PandaOmics, to identify common targets for treating asthma, eczema, and food allergy. Thirty-two case-control comparisons were generated from 15, 11, and 6 transcriptomics datasets related to asthma (558 cases, 315 controls), eczema (441 cases, 371 controls), and food allergy (208 cases, 106 controls), respectively, and allocated into three meta-analyses for target identification. Top-100 high-confidence targets and Top-100 novel targets were prioritized by PandaOmics for each allergic disease. Results: Six common high-confidence targets (i.e., IL4R, IL5, JAK1, JAK2, JAK3, and NR3C1) across all three allergic diseases have approved drugs for treating asthma and eczema. Based on the targets’ dysregulated expression profiles and their mechanism of action in allergic diseases, three potential therapeutic targets were proposed. IL5 was selected as a high-confidence target due to its strong involvement in allergies. PTAFR was identified for drug repurposing, while RNF19B was selected as a novel target for therapeutic innovation. Analysis of the dysregulated pathways commonly identified across asthma, eczema, and food allergy revealed the well-characterized disease signature and novel biological processes that may underlie the pathophysiology of allergies. Conclusion: Altogether, our study dissects the shared pathophysiology of allergic disorders and reveals the power of artificial intelligence in the exploration of novel therapeutic targets.

Healthcare Resource Utilization and Costs Among Commercially Insured Patients With Advanced or Recurrent Endometrial Cancer Initiating First-Line Therapy in the United States.

Background: Endometrial cancer (EC) represents a substantial economic burden for patients in the United States. Patients with advanced or recurrent EC have a much poorer prognosis than patients with early-stage EC. Data on healthcare resource utilization (HCRU) and costs for patients with advanced or recurrent EC specifically are lacking. Objectives: To describe HCRU and costs associated with first-line (1L) therapy for commercially insured patients with advanced or recurrent EC in the United States. Methods: This was a retrospective cohort study of adult patients with advanced or recurrent EC using the MarketScan® database. Treatment characteristics, HCRU, and costs were assessed from the first claim in the patient record for 1L therapy for advanced or recurrent EC (index) until initiation of a new anti-cancer therapy, disenrollment from the database, or the end of data availability. Baseline demographics were determined during the 12 months before the patient's index date. Results: A total of 7932 patients were eligible for inclusion. Overall, mean age at index was 61 years, most patients (77.3%) had received prior surgery for EC, and the most common 1L regimen was carboplatin/paclitaxel (59.1%). During the observation period, most patients had at least one healthcare visit (all-cause, 99.9%; EC-related, 82.8%), most commonly outpatient visits (all-cause, 91.4%; EC-related, 68.7%). The highest mean (SD) costs (US dollars) were for inpatient hospitalization for both all-cause and EC-related events ($8396 [$15,130] and $9436 [$16,784], respectively). Total costs were higher for patients with a diagnosis of metastasis at baseline than for those without a diagnosis of metastasis. Discussion: For patients with advanced or recurrent EC in the United States, 1L therapy is associated with considerable HCRU and economic burden. They are particularly high for patients with metastatic disease. Conclusions: This study highlights the need for new cost-effective treatments for patients with newly diagnosed advanced or recurrent EC.

Cost-effectiveness analysis of pembrolizumab versus chemotherapy as first-line treatment for mismatch-repair-deficient (dMMR) or microsatellite-instability-high (MSI-H) advanced or metastatic colorectal cancer from the perspective of the Chinese health-care system

BackgroundPembrolizumab is superior to chemotherapy as a first-line treatment for patients with mismatch-repair-deficient (dMMR) or microsatellite-instability-high (MSI-H) advanced or metastatic colorectal cancer (CRC), with a significant long-term survival benefit according to the KEYNOTE-177 trial. The current study aimed to determine whether pembrolizumab is a cost-effective treatment for patients with dMMR/MSI-H advanced or metastatic CRC in China.MethodsA partitioned survival model (PSM) was developed to simulate patients with dMMR/MSI-H advanced or metastatic CRC based on progression-free survival (PFS), progressive disease (PD) and death. The model was designed using a lifetime horizon, a 6-week cycle, and a 5% discount rate. The patients in the model had metastatic dMMR/MSI-H CRC and had not previously received treatment; these characteristics were similar to those of patients in KEYNOTE-177, a phase 3, open-label randomized clinical trial. The health outcomes and utilities were based on the KEYNOTE-177 trial and published data, respectively. Costs were calculated based on local charges (2022) and published literature. A treatment was deemed cost-effective in China if the incremental cost-effectiveness ratio (ICER) value was less than U.S.$38,142.56 per quality-adjusted life-year (QALY). The robustness of the results was assessed via one-way deterministic and probabilistic sensitivity analyses.ResultsBaseline analysis revealed that pembrolizumab provided an additional 2.58 QALYs (3.00 life-year) at an incremental cost of U.S.$78,286.04, resulting in an ICER of U.S.$30,330.15 per QALY, which was below the willingness-to-pay threshold of U.S.$38,142.56 per QALY. When the patient assistance program (PAP) was considered, the ICER became U.S.$1,730.67 per QALY, manifesting absolute cost-effectiveness. The results of sensitivity analyses demonstrated that pembrolizumab was cost-effective in most cases.ConclusionsPembrolizumab is a cost-effective first-line treatment for dMMR/MSI-H advanced or metastatic CRC patients in China, especially considering the PAP.

Cost-effectiveness of neoadjuvant pembrolizumab plus chemotherapy with adjuvant pembrolizumab for early-stage non-small cell lung cancer in the United States.

Perioperative (neoadjuvant and adjuvant) pembrolizumab has shown favorable efficacy in patients with early-stage non-small cell lung cancer (NSCLC). This study aims to evaluate the cost-effectiveness of this treatment from the perspective of the United States healthcare payers. We established a Markov model to compare the cost-effectiveness of perioperative pembrolizumab with that of neoadjuvant chemotherapy in 21-day cycles, utilizing data from the phase 3 KEYNOTE-671 trial. Additional data were extracted from other publications or online sources. Sensitivity analyses were conducted to evaluate the robustness of the findings. A willingness-to-pay threshold of $150,000 per quality-adjusted life-years (QALYs) gained was established. The main outcomes of this study were the measurement of QALYs, overall costs, incremental cost-effectiveness ratio (ICER), and net monetary benefit (NMB). During a 10-year time horizon, the total costs of perioperative pembrolizumab and the control treatment were $224,779.1 and $110,026.3, respectively. The QALYs were 4.19 and 2.97 for the two treatments, respectively, which led to an ICER of $94,222.29 per QALY gained. The NMB at the WTP threshold at $150,000 per QALY gained was $67,931.3. One-way sensitivity analysis identified the cost of pembrolizumab as the primary factor influencing cost-effectiveness. Probabilistic sensitivity analysis indicated a 97.7% probability of perioperative pembrolizumab being cost-effective at the WTP threshold. From the perspective of the United States healthcare payers, perioperative pembrolizumab is a cost-effective treatment for patients with early-stage NSCLC.

Development and Characterization of a AuNP-Genipin-Viscoelastic Collagen Material.

Lower back pain is one of the leading causes of disability, affecting 11.9% of the population worldwide and studies have shown that intervertebral disc degeneration is a common cause for chronic lower back pain. We have explored the combination of three components, viscoelastic collagen, genipin, and gold nanoparticles to determine its potential to promote regeneration of the intervertebral disc, specifically for nucleus pulposus regeneration. The goal of this study was to develop, fabricate and characterize different formulations of viscoelastic collagen conjugated with gold nanoparticles and genipin to assess the feasibility as a tissue template. Results demonstrated the successful attachment of gold nanoparticles to the viscoelastic collagen utilizing the genipin crosslinker. For each of the viscoelastic collagen compositions examined, cell biocompatibility was achieved. The results also demonstrated an increase in stiffness of the material with different sizes and concentrations of AuNPs. Results from the TEM and STEM also demonstrated that the viscoelastic collagen that was developed did not display the characteristic D banding pattern of polymerized collagen. The findings from this study could lead to the development of a more efficient and cost-effective treatment for patients with chronic back pain caused by IVD degeneration.

The cost-effectiveness analysis of serplulimab versus regorafenib for treating previously treated unresectable or metastatic microsatellite instability-high or deficient mismatch repair colorectal cancer in China.

The aim of this study was to investigate the cost-effectiveness of serplulimab versus regorafenib in previously treated unresectable or metastatic microsatellite instability-high (MSI-H)/deficient mismatch repair (dMMR) colorectal cancer in China. From the perspective of China's health-care system, a Markov model with three health states (progression free, progression, death) was developed for estimating the costs and health outcomes of serplulimab and regorafenib. Data for unanchored matching-adjusted indirect comparison (MAIC), standard parametric survival analysis, the mixed cure model, and transition probabilities calculation were obtained from clinical trials (ASTRUM-010 and CONCUR). Health-care resource utilization and costs were derived from government-published data and expert interviews. Utilities used to calculate quality-adjusted life years (QALYs) were obtained from clinical trials and literature reviews. The primary outcome was the incremental cost-effectiveness ratio (ICER) expressed as cost/QALY gained. Four scenarios were considered in scenario analysis: (a) using original survival data without conducting MAIC; (b) limiting the time horizon to the follow-up time of the clinical trial of serplulimab; (c) adopting a fourfold increase in the risk of death; and (d) applying utilities from two other sources. One-way sensitivity analysis and probabilistic sensitivity analysis were also performed to assess the uncertainty of the results. In the base-case analysis, serplulimab provided 6.00 QALYs at a cost of $68,722, whereas regorafenib provided 0.69 QALYs at a cost of $40,106. Compared with that for treatment with regorafenib, the ICER for treatment with serplulimab was $5,386/QALY, which was significantly lower than the triple GDP per capita of China in 2021 ($30,036), which was the threshold used to define the cost-effectiveness. In the scenario analysis, the ICERs were $6,369/QALY, $20,613/QALY, $6,037/QALY, $4,783/QALY, and $6,167/QALY, respectively. In the probabilistic sensitivity analysis, the probability of serplulimab being cost-effective was 100% at the threshold of $30,036/QALY. Compared with regorafenib, serplulimab is a cost-effective treatment for patients with previously treated unresectable or metastatic MSI-H/dMMR colorectal cancer in China.

Cost-effectiveness analysis of endoscopic dacryocystorhinostomy using Markov modelling

ObjectifDéterminer le rapport coût-efficacité de la dacryocystorhinostomie (DCR) endoscopique. MéthodesNous avons mis au point un modèle de Markov selon lequel des patients qui présentaient une obstruction du canal nasolacrymal ont été répartis en 2 groupes : DCR endoscopique ou absence de traitement chirurgical. Le rapport coût-efficacité différentiel, l'analyse de sensibilité à un critère et l'analyse de sensibilité probabiliste ont permis d’évaluer la sensibilité du modèle en présence de multiples intrants. RésultatsOn a pu conclure que la DCR endoscopique était rentable, son coût-efficacité différentiel s’établissant à 2162 $US par année de vie ajustée en fonction de la qualité. Le modèle s'est révélé particulièrement sensible à la déduction de l'utilité au chapitre de la santé en lien avec l’épiphora. L'analyse de sensibilité probabiliste a permis de conclure que 93,7 % du temps, la DCR endoscopique offre un bon rapport coût-efficacité comparativement à l'absence de traitement chirurgical. ConclusionsLa DCR endoscopique est un traitement rentable en présence d’épiphora. Le modèle est très sensible à l'effet négatif de l’épiphora sur la qualité de vie. Compte tenu des progrès technologiques et chirurgicaux en matière de soins de santé, les taux de réussite de la DCR endoscopique continuent de s'améliorer, ce qui en fait un traitement encore plus efficace et économique de l'obstruction du canal nasolacrymal.

Cemiplimab as Second-Line Therapy for Patients with Recurrent Cervical Cancer: A United States-based Cost-effectiveness Analysis.

The efficacy of cemiplimab in recurrent cervical cancer has been demonstrated in the clinical trial EMPOWER-Cervical 1.However, its high price makes patients and clinicians hesitate to use it. Therefore, we designed a study to evaluate its cost-effectiveness. We developed a Markov model based on phase III clinical trials to calculate the cost, life years (LYs), quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER) over 20years with a willingness-to-pay (WTP) threshold of $150,000/QALY. The economic data included were obtained from official US government websites and published literature. Sensitivity analysis was used to determine the uncertainties associated with the model, and a subgroup analysis was performed. Compared with chemotherapy, cemiplimab produced an additional 0.597 QALYs (0.751 LYs), resulting in an ICER of $111,211.471/QALY in the United States.The cost of cemiplimab is the most influential factor in the model. The results of these models were robust in all sensitivity analyses. From the American public payers' perspective, subgroup analysis showed cemiplimab was a cost-effective regimen in patients with squamous cell carcinoma, adenocarcinoma, or programmed cell death ligand 1 (PD-L1)≥1%. From the American public payers' perspective, cemiplimab is a cost-effective treatment option for second-line treatment of recurrent cervical cancer. Meanwhile, cemiplimab was a cost-effective treatment for patients with PD-L1≥1 and all histological types.

Cost-Effectiveness of Empagliflozin in Combination with Standard Care versus Standard Care Only in the Treatment of Heart Failure Patients in Finland

Sodium-glucose cotransporter-2 (SGLT2) inhibitor empagliflozin has recently been shown to improve the outcomes of heart failure (HF) patients regardless of patient's left ventricular ejection fraction by reducing the combined risk of cardiovascular death or hospitalization for worsening HF. The aim of this study was to assess the cost-effectiveness of adding empagliflozin to the standard care (SC) in comparison to SC only in the treatment of HF in Finland. The assessment was performed in the cost-utility framework using two Markov cohort state-transition models, one for HF with reduced ejection fraction (HFrEF) and one for HF with preserved ejection fraction (HFpEF). The models have been primarily developed based on the EMPEROR-Reduced and EMPEROR-Preserved trials which informed the modelled patient characteristics, efficacy of treatments in terms of associated risks for heart failure hospitalizations, cardiovascular (CV) and non-CV death, treatment related adverse events (AE), and state- and event-specific health-related quality of life weights (EQ-5D). Direct health care costs were estimated from Finnish published references. Cost-effectiveness was assessed from health care payer perspective based on incremental cost-effectiveness ratio (ICER; cost per quality adjusted life-year [QALY] gained) and probability of cost-effectiveness (at willingness-to-pay [WTP] of 35,000 euros/QALY). The ICER was reported as the weighted (HFrEF, 43.5%; HFpEF, 56.5%) average result of the two models. Empagliflozin + SC treatment increased the average quality-adjusted life-expectancy, and treatment costs of HF patients by 0.15 QALYs and 1,594 euros, respectively, when compared to SC. An additional QALY with empagliflozin was thus gained at a cost of 10,621 euros. The probability of empagliflozin + SC being cost-effective compared to placebo + SC was 77.6% and 83.5% with WTP of 35,000 and 100,000 euros/QALY, respectively. Empagliflozin is a cost-effective treatment for patients with HF in the Finnish health care setting.

Healthcare Resource Utilization and Costs Among Commercially Insured Patients With Advanced or Recurrent Endometrial Cancer Initiating First-Line Therapy in the United States

Background: Endometrial cancer (EC) represents a substantial economic burden for patients in the United States. Patients with advanced or recurrent EC have a much poorer prognosis than patients with early-stage EC. Data on healthcare resource utilization (HCRU) and costs for patients with advanced or recurrent EC specifically are lacking. Objectives: To describe HCRU and costs associated with first-line (1L) therapy for commercially insured patients with advanced or recurrent EC in the United States. Methods: This was a retrospective cohort study of adult patients with advanced or recurrent EC using the MarketScan® database. Treatment characteristics, HCRU, and costs were assessed from the first claim in the patient record for 1L therapy for advanced or recurrent EC (index) until initiation of a new anti-cancer therapy, disenrollment from the database, or the end of data availability. Baseline demographics were determined during the 12 months before the patient’s index date. Results: A total of 7932 patients were eligible for inclusion. Overall, mean age at index was 61 years, most patients (77.3%) had received prior surgery for EC, and the most common 1L regimen was carboplatin/paclitaxel (59.1%). During the observation period, most patients had at least one healthcare visit (all-cause, 99.9%; EC-related, 82.8%), most commonly outpatient visits (all-cause, 91.4%; EC-related, 68.7%). The highest mean (SD) costs (US dollars) were for inpatient hospitalization for both all-cause and EC-related events ($8396 [$15,130] and $9436 [$16,784], respectively). Total costs were higher for patients with a diagnosis of metastasis at baseline than for those without a diagnosis of metastasis. Discussion: For patients with advanced or recurrent EC in the United States, 1L therapy is associated with considerable HCRU and economic burden. They are particularly high for patients with metastatic disease. Conclusions: This study highlights the need for new cost-effective treatments for patients with newly diagnosed advanced or recurrent EC.

425 THE IMPACT OF NURSING EDUCATION ON THE QUALITY OF LIFE IN PATIENTS WITH HEART FAILURE DURING CARDIAC REHABILITATION: A SYSTEMATIC REVIEW

Abstract Introduction Heart failure (HF) occurs in 26 million people worldwide and is one of the most common causes of hospitalization, hospital readmission and death. To overcome this, ESC guidelines recommend the cardiologic rehabilitation pathway, which places the patient with HF at the center of his or her care pathway with the aim of improving quality of life through the implementation of a personalized multidisciplinary program. In the cardiology rehabilitation pathway, the nurse plays a key role of education and support, encouraging the patient to change. The aim of the review is to explore the quality of life of the elderly patient with HF and the educational role of nursing in the cardiac rehabilitation setting. Materials and Methods A systematic search was conducted using PUBMED, CINAHL until June 2022, according to REA methodology. The quality of the study was assessed by three reviewers using the DIXONWOOD checklist. Results and discussion: Analysis of the results suggests that multiple aspects need to be considered to improve the management and quality of life of patients with HF. Among these, the literature cites the implementation of a network of nurse case managers, standardization of operative protocols, mental and physical pain management, and cardiac rehabilitation units after hospital discharge. It also shows how physical activity, according to the ERIC-HF program, can improve the ability to perform ADL activities (Kang Y. et all, 2020). An additional study found that home cardiac rehabilitation intervention results in clinically significant improvement in disease-related quality of life in patients with HF. Cost-utility analyses support that home cardiac rehabilitation programs are cost-effective treatments for patients with HF and that nurses play a key role in educating and managing their condition (Rod s. et all, 2019). They should encourage patients with heart failure to practice cardiac rehabilitation for a longer period to improve their quality of life. One study shows that rehabilitation intervention for caregivers of patients with HF is helpful in supporting their role and improving their confidence (Wingham J. et all, 2019). Conclusions Cardiac rehabilitation in which nurses are present proves to be crucial in both psychological (Suzanne H et al, 2018) and exercise-enhancing projects to improve long-term health conditions, as shown by Kentaro, K., this approach produces benefits on patients with HF and their caregivers. It induces in the patient greater autonomy in daily activities, medication adherence and self-care of the disease, delaying its progression. At the same time, for caregivers there is a reduction in caregiver burden resulting in economic, emotional and social improvement. For the National Health Service, there is a reduction in length of stay in intensive care and emergency departments and consequently, a decrease in costs.

HTA60 Icosapent Ethyl in Addition to Statins Is a Cost-Effective Treatment in Patients at High Cardiovascular Risk With Elevated Triglycerides and Established Cardiovascular Disease or Diabetes With Risk Factor in the Netherlands

HTA60 Icosapent Ethyl in Addition to Statins Is a Cost-Effective Treatment in Patients at High Cardiovascular Risk With Elevated Triglycerides and Established Cardiovascular Disease or Diabetes With Risk Factor in the Netherlands

Exercise Rehabilitation Training in Patients With Pulmonary Hypertension: A Review.

Pulmonary hypertension (PH) has a high morbidity and mortality. Despite the existing disease-targeted therapy, most patients with PH continue to suffer from difficulty in breathing, exercise intolerance, and reduced quality of life. Recently, a large body of research results has suggested that exercise rehabilitation training (ERT) seems to be a beneficial, safe, and cost-effective treatment for patients with PH. However, knowledge gaps still exist for a uniformly accepted ERT protocol, the modality, duration, intensity, and frequency of ERT in PH. The purpose of this review is to summarise the existing research evidence and knowledge, aiming to strengthen clinicians' awareness of the application of ERT in patients with PH. PubMed databases were systematically searched for eligible studies. Twelve (12) randomised controlled trials and other important studies documenting effectiveness, safety, and adverse events of ERT are summarised. Additionally, the modality, duration, intensity, and frequency of various types of ERT and future research directions are discussed. In summary, ERT is generally effective and safe for PH patients as an adjuvant treatment to disease-specific therapy. It can improve the exercise capacity and tolerance, skeletal and respiratory muscle performance, cardiopulmonary function, and quality of life of PH patients. In view of the occurrence of a small number of adverse events, we currently recommend a combination of in-hospital and home-based ERT under close supervision. In the future, more multi-centre randomised controlled studies are needed to evaluate the effectiveness and feasibility of long-term, community, or home-based ERT, as well as to explore the molecular mechanism behind it.

Economic Evaluation of a Tumour-Agnostic Therapy: Dutch Economic Value of Larotrectinib in TRK Fusion-Positive Cancers.

BackgroundLarotrectinib is the first tumour-agnostic therapy that has been approved by the European Medicines Agency. Tumour-agnostic therapies are indicated for a multitude of tumour types. The economic models supporting reimbursement submissions of tumour-agnostic therapies are complex because of the multitude of indications per model.ObjectiveThe objective of this paper was to evaluate the cost effectiveness of larotrectinib compared with standard of care in patients with cancer with tropomyosin receptor kinase fusion-positive tumour types in the Netherlands.MethodsA previously constructed cost-effectiveness model with a partitioned survival approach was adapted to the Dutch setting, simulating costs and effects of treatment in patients with tropomyosin receptor kinase fusion-positive cancer. The cost-effectiveness model conducts a naïve comparison of larotrectinib to a weighted comparator standard-of-care arm. Dutch specific resource use and costs were implemented and inflated to reflect 2019 euros. The analysis includes a lifetime horizon and a societal perspective.ResultsLarotrectinib versus Dutch standard of care resulted in 5.61 incremental (QALYs) and €232,260 incremental costs, leading to an incremental cost-effectivenes ratio of €41,424/QALY. The probabilistic sensitivity analysis reveals a 88% chance of larotrectinib being cost effective compared with the pooled comparator standard-of-care arm at the applicable €80,000/QALY willingness-to-pay threshold in the Netherlands.ConclusionsThe incremental cost-effectivenes ratio was well below the applicable threshold for diseases with a high burden of disease in the Netherlands (€80,000). At this threshold, larotrectinib was estimated to be a cost-effective treatment for patients with tropomyosin receptor kinase fusion-positive cancer compared with current standard of care in the Netherlands.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40258-022-00740-1.