Background Heart failure presents a growing clinical and economic burden in the USA. Robust cost data on the burden of illness are critical to inform economic evaluations of new therapeutic interventions. Objectives This systematic literature review of heart failure-related costs in the USA aimed to assess the quality of the published evidence and provide a narrative synthesis of current data. Methods Four electronic databases (MEDLINE, EMBASE, EconLit, and the Centre for Reviews and Dissemination York Database, including the NHS Economic Evaluation Database and Health Technology Assessment Database) were searched for journal articles published between January 2014 and March 2020. The review, registered with PROSPERO (CRD42019134201), was restricted to cost-of-illness studies in adults with heart failure events in the USA. Results Eighty-seven studies were included, 41 of which allowed a comparison of cost estimates across studies. The annual median total medical costs for heart failure care were estimated at $24,383 per patient, with heart failure-specific hospitalizations driving costs (median $15,879 per patient). Analyses of subgroups revealed that heart failure-related costs are highly sensitive to individual patient characteristics (such as the presence of comorbidities and age) with large variations even within a subgroup. Additionally, differences in study design and a lack of standardized reporting limited the ability to compare cost estimates. The finding that costs are higher for patients with heart failure with reduced ejection fraction compared with patients with preserved ejection fraction highlights the need for differentiating among different heart failure types. Conclusions The review underpins the conclusion drawn in earlier reviews, namely that hospitalization costs are the key driver of heart failure-related costs. Analyses of subgroups provide a clearer understanding of sources of heterogeneity in cost data. While current cost estimates provide useful indications of economic burden, understanding the nuances of the data is critical to support its application. Electronic supplementary material The online version of this article (10.1007/s40273-020-00952-0) contains supplementary material, which is available to authorized users.

Summary Background Hepatorenal syndrome and acute kidney injury are common complications of decompensated cirrhosis, and terlipressin is recommended as first‐line vasoconstrictor therapy. However, data on its use outside of clinical trials are lacking. Aims To assess practice patterns and outcomes around vasoconstrictor use for hepatorenal syndrome in UK hospitals. Methods This was a multicentre chart review study. Data were extracted from medical records of patients diagnosed with hepatorenal syndrome and treated by vasoconstrictor drugs between January 2013 and December 2017 at 26 hospitals in the United Kingdom. The primary outcome was improvement of kidney function, defined as complete response (serum creatinine improved to ≤1.5 mg/dL), partial response (serum creatinine reduction of ≥20% but >1.5 mg/dL) and overall response (complete or partial response). Other outcomes included need for dialysis, mortality, liver transplantation and adverse events. Results Of the 225 patients included in the analysis, 203 (90%) were treated with terlipressin (median duration, 6 days; range: 2‐24 days). Mean (±standard deviation) serum creatinine at vasopressor initiation was 3.25 ± 1.64 mg/dL. Terlipressin overall response rate was 73%. Overall response was higher in patients with mild acute kidney injury (baseline serum creatinine <2.25 mg/dL), compared to those with moderate (serum creatinine ≥2.25 mg/dL and <3.5 mg/dL) or severe (serum creatinine ≥3.5 mg/dL). Ninety‐day survival was 86% for all patients (93% for overall responders vs 66% for treatment nonresponders, P < 0.0001). Conclusion Terlipressin is the most commonly prescribed vasoconstrictor for patients with hepatorenal syndrome in the United Kingdom. Treatment with terlipressin in patients with less severe acute kidney injury (serum creatinine <2.25 mg/dL) was associated with higher treatment responses, and 90‐day survival.

BackgroundSince persistence to first biological disease modifying anti-rheumatic drugs (bDMARDs) is far from ideal in rheumatoid arthritis (RA) patients, many do receive a second and/or third bDMARD treatment. However, little is known about treatment persistence of the second-line bDMARD and it is specifically unknown whether the mode of action of such a treatment is associated with different persistence rates. We aimed to assess discontinuation-, re-initiation- or continuation-rates of a 2nd bDMARD therapy as well as switching-rates to a third biological DMARD (3rd bDMARD) therapy in RA patients.MethodAnalysis was based on German claims data (2010–2013). Patients were included if they had received at least one prescription for an anti-TNF and at least one follow-up prescription of a 2nd bDMARD different from the first anti-TNF. Patient follow-up started on the date of the first prescription for the 2nd bDMARD and lasted for 12 months or until a patient’s death.Results2667 RA patients received at least one anti-TNF prescription. Of these, 451 patients received a second bDMARD (340 anti-TNF, mean age 52.6 years; 111 non-anti-TNF, mean age 55.9 years).During the follow-up, 28.8% vs. 11.7% of the 2nd anti-TNF vs. non-anti-TNF patients (p < 0.001) switched to a 3rd bDMARD; 14.1% vs. 19.8% (p = 0.179) discontinued without re-start; 3.8% vs.1.8% (p = 0.387) re-started and 53.5 vs. 66.7% (p < 0.050) continued therapy. Patients in the non-anti-TNF group demonstrated longer drug survival (295 days) than patients in the anti-TNF group (264 days; p = 0.016).Independent variables associated with earlier discontinuation (including re-start) or switch were prescription of an anti-TNF as 2nd bDMARD (HR = 1.512) and a higher comorbidity level (CCI, HR = 1.112), whereas previous painkiller medication (HR = 0.629) was associated with later discontinuation or switch.ConclusionsOnly 56.8% of RA patients continued 2nd bDMARD treatment after 12 months; 60% if re-start was included. Non-anti-TNF patients had a higher probability of continuing 2nd bDMARD therapy.Electronic supplementary materialThe online version of this article (doi:10.1186/s12891-017-1684-0) contains supplementary material, which is available to authorized users.

Background and Objectives New treatments and interventions are in development to address clinical needs in heart failure. To support decision making on reimbursement, cost-effectiveness analyses are frequently required. A systematic literature review was conducted to identify and summarize heart failure utility values for use in economic evaluations. Methods Databases were searched for articles published until June 2019 that reported health utility values for patients with heart failure. Publications were reviewed with specific attention to study design; reported values were categorized according to the health states, 'chronic heart failure', 'hospitalized', and 'other acute heart failure'. Interquartile limits (25th percentile 'Q1', 75th percentile 'Q3') were calculated for health states and heart failure subgroups where there were sufficient data. Results The systematic literature review identified 161 publications based on data from 142 studies. Utility values for chronic heart failure were reported by 128 publications; 39 publications published values for hospitalized and three for other acute heart failure. There was substantial heterogeneity in the specifics of the study populations, methods of elicitation, and summary statistics, which is reflected in the wide range of utility values reported. EQ-5D was the most used instrument; the interquartile limit for mean EQ-5D values for chronic heart failure was 0.64-0.72. Conclusions There is a wealth of published utility values for heart failure to support economic evaluations. Data are heterogenous owing to specificities of the study population and methodology of utility value elicitation and analysis. Choice of value(s) to support economic models must be carefully justified to ensure a robust economic analysis.

Rivaroxaban treatment of patients with venous thromboembolism results in health gains and cost savings compared to LMWH/VKA therapy. This conclusion holds for the Dutch setting, both for the societal perspective, as well as the healthcare perspective.

The current cost-minimization analysis demonstrates that, from the Dutch healthcare perspective, treating active RRMS patients with alemtuzumab results in cost savings compared to second-line alternatives fingolimod and natalizumab from ∼3 years since treatment initiation onwards. After 5 years, alemtuzumab's cost savings are estimated at €30k compared to fingolimod and €43k compared to natalizumab.

Objectives: Non-reconstituted, hexavalent vaccines (HV-NRs) can facilitate clinical practice by shortening vaccine preparation and administration time and by reducing the risk of vaccination errors compared to combination vaccines requiring reconstitution. The aim of this study was to determine the budget impact of introducing an HV-NR into the United Kingdom's (UK) pediatric immunization program, which currently uses a hexavalent vaccine requiring reconstitution (HV-R). Methods: Abudget impact model covering a 10-year time horizon was developed. The target population constituted closed UK birth cohorts from 2020 to 2029. Total direct costs from the payer's perspective consisted of four main categories: vaccine acquisition and management, healthcare provider's service provision, (non-)contaminated needle-stick and sharps injury (NSI), and non-NSI vaccination error costs. The net budget impact was calculated by comparing the costs in two different market share scenarios. Results: The use of HV-NR instead of HV-R was estimated to save £9,079,927 over a 10-year time horizon (i.e. £907,993 per year). Assuming all other vaccine criteria are equivalent the budget impact was most sensitive to changes in time spent by the healthcare provider and management costs. Conclusion: Results suggest, introducing an HV-NR into the UK's pediatric immunization program is potentially cost saving for the healthcare system.

Introduction Standard valuation methods, such as TTO and DCE are inefficient. They require data from hundreds if not thousands of participants to generate value sets. Here, we present the Online elicitation of Personal Utility Functions (OPUF) tool; a new type of online survey for valuing EQ-5D-5L health states using more efficient, compositional elicitation methods, which even allow estimating value sets on the individual level. The aims of this study are to report on the development of the tool, and to test the feasibility of using it to obtain individual-level value sets for the EQ-5D-5L. Methods We applied an iterative design approach to adapt the PUF method, previously developed by Devlin et al., for use as a standalone online tool. Five rounds of qualitative interviews, and one quantitative pre-pilot were conducted to get feedback on the different tasks. After each round, the tool was refined and re-evaluated. The final version was piloted in a sample of 50 participants from the UK. A demo of the EQ-5D-5L OPUF survey is available at: https://eq5d5l.me Results On average, it took participants about seven minutes to complete the OPUF Tool. Based on the responses, we were able to construct a personal EQ-5D-5L value set for each of the 50 participants. These value sets predicted a participants' choices in a discrete choice experiment with an accuracy of 80%. Overall, the results revealed that health state preferences vary considerably on the individual-level. Nevertheless, we were able to estimate a group-level value set for all 50 participants with reasonable precision. Discussion We successfully piloted the OPUF Tool and showed that it can be used to derive a group-level as well as personal value sets for the EQ-5D-5L. Although the development of the online tool is still in an early stage, there are multiple potential avenues for further research.