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Cyber risk affects all organizations. Cyber risk management has generally been based on the heuristics and the availability of protective tools, such as firewalls and passwords. Only recently have there been quantitative analyses of these tools’ costs and benefits. This article presents a probabilistic method based on the existing data in an organization and on their extension to assess the probabilities of new attack scenarios. The objective is to set priorities among risk management measures and to optimize the allocation of limited resources. The model is illustrated first by a statistical analysis of 60 000 incidents, such as lost or stolen laptops, over six years in a specific organization. This analysis is then expanded to the probabilistic domain to cover threats that have not occurred yet. This requires a systematic construction of new attack scenarios and an assessment both of their probability of success and of subsequent losses. The conjunction of statistics and probabilities of more extreme scenarios yields full risk curves. These curves represent the overall cyber risk for the organization and its insurers and unable assessment of the benefits of a spectrum of protective options.

The growing number of people living with dementia will result in increased costs of dementia worldwide. The e-Health intervention 'Exergaming' may improve health and quality of life of people with dementia, but the cost-effectiveness is unknown. We assessed the cost-effectiveness of exergaming compared to regular activities from a societal perspective in day-care centres (DCC) for people with dementia and their informal caregivers (IC) alongside a cluster randomised controlled trial. We included 112 dyads (person with dementia and IC) from 20 psychogeriatric DCCs (11 exergaming, 9 control) across the Netherlands. Exergaming consisted of interactive cycling at least twice a week for 6 months. Measurements were conducted at baseline (T0), after 3 (T1) and 6 (T2) months. Primary outcomes were minutes of physical activity, mobility of the participants with dementia (Short Physical Performances Battery, SPPB), and Quality-Adjusted Life-Years (QALYs) of participants with dementia and ICs. ICs filled out cost diaries to measure healthcare and informal care utilisation during the study. There were no statistically significant differences in outcomes or costs between the groups at the level of participants with dementia, the ICs or the dyad. With regard to QALYs and SPPB, the probability that exergaming is cost-effective compared to control was low for all possible willingness-to-pay (WTP) thresholds. However, for physical activity at WTP thresholds of 0, 50 and 250 Euros per additional minute of physical activity, the probability of cost-effectiveness is 0.46, 0.84 and 0.87, respectively. Exergaming in DCC was not cost-effective compared to usual activities. However, considering the small sample size and the large number of missing observations, findings should be interpreted with caution. Future studies with larger samples are recommended to obtain definitive answers on the cost-effectiveness of exergaming. This trial was registered in the Netherlands Trial Register (NTR5537/NL5420).

Background: Despite increasing global attention to the national human papillomavirus (HPV) immunization program, this program is controversial in Iran. Evidence indicate that HPV vaccination is not cost-effective in Iran. Using cost-effectiveness analysis for decision-making about public health interventions such as vaccination is debated, because its potential benefits may not fit this framework. Our objective was to evaluate economic effects of HPV vaccination by cost-benefit analysis (CBA). In this case, we used bivalent and quadrivalent in Iran in 2020. Methods: We performed a CBA from a societal perspective. We used two approaches of the vaccine's economic benefit: willingness to pay by discrete choice experiment; and cost of illness. Costs only included the vaccine cost. Results: The cost of two doses of bivalent and quadrivalent vaccines were US $ 29 and US $ 151, respectively (US $1 =IRR 42,000). The benefits of bivalent and quadrivalent vaccines were US $ -432, and US $ 380 per person using willingness to pay approach and they were US $ 7,375 and US $ 6,590 thorough cost-of-illness approach. The cost-benefit ratio (CBR) of bivalent and quadrivalent vaccines was -15.114 and 2.512 by willingness to pay approach, and it was 258.12 and 43.51, by cost of illness approach. Conclusions: This study confirms the benefit of both the national bivalent and quadrivalent vaccination program and provide reliable evidence for policy-makers when programming HPV vaccination.

Summary Background The aim of this study was to compare, in terms of cost and serious complications, the use of biosynthetic resorbable parietal mesh with biologic mesh in patients undergoing contaminated ventral hernia repair (modified Ventral Hernia Working Group grade 3). Poly-4-hydroxy-butyrate (P4HB) biosynthetic mesh has rarely been the subject of comparative studies in the context of contamination. Data are required to confirm the effects of a transition from biological mesh to biosynthetic resorbable mesh. Patients and methods A cost-effectiveness analysis was conducted. It was based on a decision analysis model built with clinical and economic data issued from a before-after study that included 94 patients hospitalized for ventral hernia repair at the University Hospital of Strasbourg (France) from June 2011 to February 2018. The effectiveness endpoint was the number of patients presenting with a serious specific complication or a general complication at 6 months. Data for surgical hospitalization stays, home hospitalizations and ambulatory care costs were included. Results We found fewer serious complications with biosynthetic mesh: 21% versus 33% with biologic mesh. A cost savings of US $5146 was determined. Deterministic sensitivity analyses and a probabilistic analysis confirmed our findings and the robustness of the model. Conclusion P4HB biosynthetic resorbable mesh appeared to be the most effective and the least costly option. Additional data will be needed to confirm the superiority of biosynthetic mesh in terms of the recurrence risk reduction over a longer period.

The aim of this systematic review is to evaluate the accuracy of waferless osteotomy procedures in orthognathic surgery with a secondary aim to determine the cost-effectiveness of the procedure. A literature search was conducted on the databases PubMed and Scopus, with PRISMA guidelines followed. An initial yield of 4149 articles were identified, ten of which met the desired inclusion criteria. The total sample of patients undergoing waferless osteotomies included in this review was 142 patients. Nine of the studies used surgical cutting guides along with customised surgical plates to eliminate the surgical wafer and one study used pre-bent locking plates instead of customised plates. The eligible articles determined their surgical accuracy by comparing the positions of bony or dental landmarks on the pre-operative and post-operative images. The articles all reported acceptable accuracy within previously established clinical parameters. The majority of authors concluded that it is an accurate surgical approach and can be cost effective which is often a barrier to novel techniques however there were studies that contrasted the view of the cost efficacy. Due to the lack of published randomised controlled trials, current evidence is not strong enough to recommend the use of surgical cutting guides and customised/pre-bent plates for orthognathic surgery.

Objectives The Kidney Team at Home program is an educational intervention aimed at patients with chronic kidney disease to assist them in their choice for kidney replacement therapy. Previous studies have shown that the intervention results in an increase in knowledge and communication on kidney replacement therapy, and eventually in an increase in the number of living donor kidney transplantations. The study assesses the cost-effectiveness of the intervention compared to standard care. Methods A dynamic probabilistic Markov model was used to estimate the monetary and health benefits of the intervention in The Netherlands over 10 years. Data on costs and health-related quality of life were derived from the literature. Transition probabilities, prevalence, and incidence rates were calculated using a large national database. An optimistic and a pessimistic implementation scenario were compared to a base case scenario with standard care. Results In both the optimistic and pessimistic scenario, the intervention is cost-effective and dominant compared to standard care: savings were €108,681,985 and €51,770,060 and the benefits were 1382 and 695 QALYs, respectively. Conclusions The superior cost-effectiveness of the intervention is caused by the superior health effects and the reduction of costs associated with transplantation, and the relatively small incremental costs of the intervention. The favorable findings of this implementation project resulted in national uptake of the intervention in The Netherlands as of 2021. This is the first time a psychosocial intervention has been implemented as part of standard care in a kidney replacement therapy program worldwide.

This paper explores national strategies for emerging countries with ambitious policy targets for future offshore wind development. A scenario-based economic cost-benefit analysis of multiple approaches to the development of offshore wind projects is conducted based on (1) who takes the initiative on financing (public sector vs. private sector), (2) whether wind turbines are supplied locally or not, and (3) whether investment plans provide managerial flexibility to respond to changing circumstances or not (real options strategy vs. traditional investment decision-making). The analysis demonstrates that a real options strategy that allows flexibility regarding future uncertainties can significantly increase the economic value of a project regardless of the type of developer or dependence on foreign wind turbine technologies. The study concludes with a recommendation that late-mover countries focus on the combination of public-sector initiatives, a real options strategy, and the purchase of domestic turbines.

Background There is a need for effective and cost-effective interprofessional care models that support older people to maintain their quality of life (QoL) and physical performance to live longer independently in their own homes. Objectives The objectives were to evaluate effectiveness, QoL and physical performance, and cost-utility of a people-centred care model (PCCM), including the contribution of clinically trained pharmacists, compared with that of usual care in primary care. Methods A randomised controlled trial (RCT) with a two-year follow-up was conducted. The participants were multimorbid community-living older people, aged ≥75 years. The intervention comprised an at-home patient interview, health review, pharmacist-led clinical medication review, an interprofessional team meeting, and nurse-led care coordination and health support. At the baseline and at the 1-year and 2-year follow-ups, QoL (SF-36, 36-Item Short-Form Health Survey) and physical performance (SPPB, Short Performance Physical Battery) were measured. Additionally, a physical dimension component summary in the SF-36 was calculated. The SF-36 data were transformed into SF-6D scores to calculate quality-adjusted life-years (QALYs). Healthcare resource use were collected and transformed into costs. A healthcare payer perspective was adopted. Incremental cost-effectiveness ratio (ICER) was calculated, and one-way sensitivity analysis was performed. Results No statistically or clinically significant differences were observed between the usual care (n = 126) and intervention group (n = 151) patients in their QoL; at the 2-year follow-up the mean difference was −0.02, (95 % CI -0.07; 0.04,p = 0.56). While the mean difference between the groups in physical performance at the 2-year follow-up was −1.02, (−1.94;-0.10,p = 0.03), between the physical component summary scores it was −7.3, (−15.2; 0.6,p = 0.07). The ICER was −73 638€/QALY, hence, the developed PCCM dominated usual care, since it was more effective and less costly. Conclusions The cost-utility analysis showed that the PCCM including pharmacist-led medication review dominated usual care. However, it had no effect on QoL and the effect towards physical performance remained unclear.

Rationale & Objective Benefits of sodium-glucose co-transporter 2 inhibitors on kidney outcomes have been demonstrated in clinical trials. Among patients with type 2 diabetes and established cardiovascular (CV) disease enrolled in EMPA-REG Outcome Study (NCT01131676), empagliflozin added to standard of care (SoC) reduced the risk of incident or worsening nephropathy compared to SoC alone. This analysis evaluated the cost-effectiveness of empagliflozin versus SoC alone in the subpopulation with diabetic kidney disease (DKD) from the perspective of United States (US) commercial insurers and Medicare. Study Design Discrete event simulation model. Setting & Population Patients with DKD in a US healthcare system. Interventions Empagliflozin 10 or 25 mg with SoC versus SoC alone. SoC included glucose-lowering therapies and medications to treat CV risk factors. Outcomes Incremental cost-effectiveness ratios (ICERs, 2020 US dollars per quality-adjusted life-year [QALY] gained). Costs and QALYs were discounted 3.0%/year. Model, Perspective, & Timeframe: Cost-effectiveness analysis, commercial insurers and Medicare perspective, lifetime horizon. Results The ICER of empagliflozin with SoC versus SoC alone was $25,974/QALY. Empagliflozin added 0.67 QALYs and $17,322/patient over a lifetime horizon. Results were driven by fewer clinical events (including CV death, heart failure [HF] hospitalization, albuminuria progression, and a composite kidney outcome) experienced by patients receiving empagliflozin with SoC versus SoC alone. Results were sensitive to rates of CV death, non-fatal MI, and HF hospitalization, as well as to drug costs, and time horizon. Probabilistic sensitivity analyses indicated 91% of simulations falling below $50,000/QALY. Limitations The EMPA-REG Outcome Study was not powered to assess treatment benefits in a subgroup and excluded patients with estimated glomerular filtration rate Conclusion Based on EMPA-REG Outcome Study, this cost-effectiveness analysis suggests that for commercial insurers and Medicare, adding empagliflozin to SoC may be a cost-effective treatment option for patients with DKD.

Importance: Septorhinoplasties are performed for functional, aesthetic, or a combination of these indications. As a nonvital intervention, cost-effectiveness may be questioned. Objective: To determ...

Objective To estimate differences in botulinum toxin type A (BoNT-A) treatment costs per patient for spasticity-injecting physicians, with a focus on physicians’ use of alternative BoNT-A agents other than onabotulinumtoxinA. Design Retrospective Cohort Study Setting 2017 National Medicare data for fee for service beneficiaries Participants Physicians who use BoNT-A treatments predominantly for spasticity chemodenervation, along with their patients Interventions Type of BoNT-A selected by physicians was the independent variable of interest. Included physicians were separated into two groups: (1) onabotulinumtoxinA only injectors; and (2) abobotulinumtoxinA and/or incobotulinumtoxinA injectors (may still utilize onabotulinumtoxinA). Main Outcome Measure Average cost per patient per year Results A total of 116 physicians, 6,829 BoNT-A procedures, and 3,051 patients were included in this analysis. Most physicians were physiatrists (84%) and utilized onabotulinumtoxinA only (82%). Total average BoNT-A cost per patient per year was significantly less for physicians who utilized abobotulinumtoxinA and/or incobotulinumtoxinA versus onabotulinumtoxinA-only physicians ($3,684 vs $4,739; p=0.01). Patients’ average annual out-of-pocket costs also reflected a similar difference ($855 vs $1,082; p=0.02) between the groups. Doses utilized and numbers of injections per patient per year were not significantly different between groups. Conclusions The present analysis demonstrated lower cost per patient for both the payer and patient when physicians utilized types of BoNT-A other than onabotulinumtoxinA for spasticity. Nevertheless, most physicians in this spasticity-focused study utilized exclusively onabotulinumtoxinA, the most expensive BoNT-A available. Reasons for this are complex and include history on the market and approved indications beyond those associated with spasticity; however, future research should continue to identify such issues with a goal of finding solutions to improve cost inefficiencies.

Context Nutrition care is an effective lifestyle intervention for the treatment and prevention of many noncommunicable diseases. Primary care is a high-value setting in which to provide nutrition care. Objective The objective of this review was to evaluate the cost-effectiveness of nutrition care interventions provided in primary care settings. Data Sources Medline, Embase, the Cumulative Index to Nursing and Allied Health Literature (CINAHL), the Cochrane Central Register of Controlled Trials, EconLit, and the National Health Service Economic Evaluation Database (NHS EED) were searched from inception to May 2021. Data Extraction Data extraction was guided by the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) reporting guidelines. Randomized trials of nutrition interventions in primary care settings were included in the analysis if incremental cost-effectiveness ratios were reported. The main outcome variable incremental cost-effectiveness ratios (ICERs) and reported interpretations were used to categorize interventions by the cost-effectiveness plane quadrant. Results Of 6837 articles identified, 10 were included (representing 9 studies). Eight of the 9 included studies found nutrition care in primary care settings to be more costly and more effective than usual care. High study heterogeneity limited further conclusions. Conclusion Nutrition care in primary care settings is effective, though it requires investment; it should, therefore, be considered in primary care planning. Further studies are needed to evaluate the long-term cost-effectiveness of providing nutrition care in primary care settings. Systematic review registration PROSPERO registration no. CRD42020201146.

Background The current COVID-19 pandemic is causing significant morbidity and death worldwide and produces significant socio-economic losses. Objective To assess the cost–benefit relation of implementing point-of-care COVID-19 antigen testing (POCT) in emergency rooms (ER) of German hospitals. Methods A deterministic decision-analytic model simulated the incremental costs of using the Sofia® SARS Antigen FIA test compared to those of using clinical judgement alone to confirm or exclude COVID-19 in adult patients in German ER, prior to hospitalization. Direct and indirect costs, with and without subsequent RT-PCR confirmation, were evaluated from the hospital perspective. Results With respect to ER patients, in base-case analysis, considering a COVID-19 prevalence of 15.6% and a hospitalization rate among COVID-19 suspects of 10.1%, POCT testing reduces average costs of hospitalized patients by €213 per tested patient if nasopharyngeal swabs of patients suspected to have COVID-19 are also sent to external labs for RT-PCR testing. In probabilistic sensitivity analysis, under all reasonable assumptions, implementing the Sofia® SARS Antigen FIA saves on average about €210 as compared to applying the clinical-judgement-only strategy. The major part of cost savings, €159 or 75.9%, is due to the POC test´s high specificity resulting in a 21-fold lower proportion of unnecessary bed blocking at the first day of hospitalization. Conclusions Using highly specific rapid COVID-19 tests in COVID-19 suspects at German ER, despite of their sub-optimal sensitivity, may significantly reduce hospital expenditure.

Background: There are no established methods for pancreatic cancer (PAC) screening, but the NCI and the Pancreatic Cancer Action Network (PanCAN) are investigating risk-based screening strategies in patients with new-onset diabetes (NOD), a group with elevated PAC risk. Preliminary estimates of the cost-effectiveness of these strategies can provide insights about potential value and inform supplemental data collection. Using data from the Enriching New-Onset Diabetes for Pancreatic Cancer (END-PAC) risk model validation study, we assessed the potential value of CT screening for PAC in those determined to be at elevated risk, as is being done in a planned PanCAN Early Detection Initiative trial. Methods: We created an integrated decision tree and Markov state-transition model to assess the cost-effectiveness of PAC screening in patients aged ≥50 years with NOD using CT imaging versus no screening. PAC prevalence, sensitivity, and specificity were derived from the END-PAC validation study. PAC stage distribution in the no-screening strategy and PAC survival were derived from the SEER program. Background mortality for patients with diabetes, screening and cancer care expenditure, and health state utilities were derived from the literature. Life-years (LYs), quality-adjusted LYs (QALYs), and costs were tracked over a lifetime horizon and discounted at 3% per year. Results are presented in 2020 US dollars, and we took a limited US healthcare perspective. Results: In the base case, screening resulted in 0.0055 more LYs, 0.0045 more QALYs, and $293 in additional expenditures for a cost per QALY gained of $65,076. In probabilistic analyses, screening resulted in a cost per QALY gained of 25% of screen-detected PAC cases needed to be resectable for the cost per QALY gained with screening to be Conclusions: We found that risk-based PAC screening in patients with NOD is likely to be cost-effective in the United States if even a modest fraction (>25%) of screen-detected patients with PAC are resectable. Future studies should reassess the value of this intervention once clinical trial data become available.

PURPOSE: To investigate the cost-effectiveness of prophylactic treatments against cystoid macular edema (CME) after cataract surgery in diabetic patients.SETTING: Seven ophthalmology clinics in the Netherlands and Belgium.DESIGN: Prospective trial-based cost-effectiveness analysis using data from a European multicenter randomized clinical trial.METHODS: Diabetic patients (n=163) undergoing uncomplicated cataract surgery were randomized to perioperative subconjunctival triamcinolone acetonide (n=36), perioperative intravitreal bevacizumab (n=36), combination treatment (n=45), or no additional treatment (control group, n=46). The cost analysis was performed from a healthcare perspective within a 12-week postoperative time horizon. The main effectiveness outcome was quality-adjusted life years (QALYs). The main cost-effectiveness outcome was the incremental cost-effectiveness ratio (ICER; cost per QALY).RESULTS: Mean total healthcare costs and QALYs were: Triamcinolone € 1827 (US$ 2295) / 0.166; Bevacizumab € 2050 (US$ 2575) / 0.144; Combination € 2027 (US$ 2546) / 0.166; Control € 2041 (US$ 2564) / 0.156. Bevacizumab and control treatment were most costly and least effective. The ICER was € 321,984 (US$ 404,503) per QALY for the combination group compared to the triamcinolone group. Assuming the willingness-to-pay is € 20,000 (US$ 25,126) per QALY, the cost-effectiveness probability was 70% and 23% in the triamcinolone and combination groups, respectively. No patient that received triamcinolone developed clinically significant macular edema (CSME). A secondary cost-effectiveness analysis based on this outcome showed a clear preference for triamcinolone.CONCLUSIONS: In diabetic patients, subconjunctival triamcinolone was effective in preventing CSME after cataract surgery. The cost-effectiveness analysis showed triamcinolone is also cost-effective.

The National Institute for Health and Care Excellence (NICE) invited the manufacturer (Kyowa Kirin) of mogamulizumab (Poteligeo(R)), as part of the single technology appraisal process, to submit evidence for its clinical and cost-effectiveness for previously treated mycosis fungoides (MF) and Sezary syndrome (SS). Kleijnen Systematic Reviews Ltd, in collaboration with Maastricht University Medical Centre, was commissioned to act as the independent evidence review group (ERG). This paper summarises the company submission (CS), presents the ERG's critical review of the clinical and cost-effectiveness evidence in the CS, highlights the key methodological considerations and describes the development of the NICE guidance by the Appraisal Committee. Based on a systematic literature review, one randomised controlled trial, MAVORIC, was identified showing favourable results in patients with MF and SS. However, MAVORIC compared mogamulizumab to vorinostat, which is not standard care in the NHS, and there is uncertainty due to the study design, specifically crossover of patients. Based on a "naive comparison of results from the vorinostat arm of the MAVORIC study and the physician's choice arm (methotrexate or bexarotene i.e. United Kingdom [UK] standard treatments) of the ALCANZA study as well as comparison to Phase II bexarotene data", the company considered vorinostat to be "a reasonable proxy for current standard of care in the NHS". The ERG considered, based on the limited data available, that the comparability of vorinostat (MAVORIC) and physician's choice (ALCANZA) could not be established. In response to the Appraisal Consultation Document, the company provided an unanchored matched adjusted indirect comparison (MAIC) of mogamulizumab with UK standard care by analysing Hospital Episode Statistics (HES) data. However, given the high risk of bias of an unanchored MAIC, these results needed to be regarded with a considerable degree of caution. The economic analysis suffered from uncertainty because there was no trial evidence on the comparator in the England and Wales National Health Service (NHS), and it was unclear to what extent the trial (MAVORIC) comparator (vorinostat) was comparable to standard care, referred to as established clinical management (ECM) in the NHS. The evidence for overall survival had not reached maturity and was confounded by treatment switching, for which different crossover adjustment methods produced large variations in life years. Caregiver utilities were applied in the analysis, but there was a lack of guidance on their application and whether these were indicated in this appraisal. After consultation, the company updated the economic analysis with the MAIC. Incremental cost-effectiveness ratios comparing mogamulizumab against ECM were (depending on whether the HES or MAVORIC comparison were used) 31,030 pound or 32,634 pound per quality-adjusted life years (QALYs) gained according to the company's base case and 38,274 pound or 80,555 pound per QALY gained according to the ERG's base case. NICE did not recommend mogamulizumab for treating MF or SS in adults who have had at least one previous systemic treatment.

Objectives Cryptococcal meningitis constitutes a significant source of mortality in the developing world. Annually, approximately 625 000 deaths occur worldwide among patients with human immunodeficiency virus (HIV) infection. This study aims to assess the cost-effectiveness of implementing cryptococcal antigen lateral flow assay (CRAG-LFA) screening in Brazil compared with the current practice. Methods An economic evaluation using a Monte Carlo microsimulation was conducted, considering the perspective of the Brazilian Public Health System, to calculate the cost-effectiveness of 4 diagnosis tests: (1) CRAG-LFA, (2) the cryptococcal antigen latex agglutination (CRAG-LA) test, (3) India ink, and (4) nontracking as a baseline. The time horizon comprised 1 year for the intervention and 5 years for the budgetary impact analysis. Two primary effectiveness outcomes were considered: years of life and quality-adjusted life-years. Results CRAG-LFA has extended dominance vis a vis CRAG-LA and India ink. CRAG-LFA would cost $418.46 more than CRAG-LA for the treatment of each symptomatic patient living with HIV, with an incremental cost effectiveness ratio of $2478.75/quality-adjusted life year. The budgetary impact analysis estimated that the incorporation of CRAG-LFA would have an additional cost of $1 959 236.50 in 5 years. Conclusions These findings suggest that, for patients living with HIV in the Brazilian Public Health System, the adoption of CRAG-LFA screening is cost-effective compared with the use of CRAG-LA and India ink. It represents an opportunity to prevent cryptococcal meningitis and its mortality in Brazil.

Objective The aim of the study was to determine whether adjunctive hydrocortisone reduced healthcare expenditure and was cost-effective compared with placebo in New Zealand patients in the Adjunctive Glucocorticoid Therapy in Patients with Septic Shock (ADRENAL) trial. Design This is a health economic analysis using data linkage to New Zealand Ministry of Health databases to determine resource use, costs, and cost-effectiveness for a 24-month period. Setting The study was conducted in New Zealand. Participants and intervention Patients with septic shock were randomised to receive a 7-day continuous infusion of 200 mg of hydrocortisone or placebo in the ADRENAL trial. Main outcome measures Healthcare expenditure was associated with all hospital admissions, emergency department presentations, outpatient visits, and pharmacy expenditure. Effectiveness outcomes included mortality at 6 months and 24 months and quality of life at 6 months. Cost-effectiveness outcomes were assessed with reference to quality-adjusted life years gained at 6 months and life years gained at 24 months. Results Of 3800 patients in the ADRENAL trial, 419 (11.0%) were eligible, and 405 (96.7% of those eligible) were included. The mean total costs per patient over 24 months were $143,627 ± 100,890 and $143,772 ± 97,117 for the hydrocortisone and placebo groups, respectively (p = 0.99). Intensive care unit costs for the index admission were $50,492 and $62,288 per patient for the hydrocortisone and placebo groups, respectively (p = 0.09). The mean number of quality-adjusted life years gained at 6 months and mean number of life years gained at 24 months was not significantly different by treatment group, and the probability of hydrocortisone being cost-effective was 55% at 24 months. Conclusions In New Zealand, adjunctive hydrocortisone did not reduce total healthcare expenditure or improve outcomes compared with placebo in patients with septic shock.

Objectives Radical prostatectomy (RP) is the gold standard for the surgical treatment of localized prostate cancer, presenting better results than radiotherapy especially for high-risk patients. Although it has clinical and technical benefits compared with open and laparoscopic techniques, the robotic-assisted RP is not publicly funded in Brazil. The objective of this study was to calculate the cost-effectiveness of the robotic-assisted RP from the Brazilian public system perspective. Methods A state transition model was built to simulate the life of a patient undergoing RP. A total of 3 arms were compared: robotic-assisted, laparoscopic, and open surgeries. The assumed time horizon was 20 years; discounts were applied to both costs and health outcomes. Events and transition probabilities were obtained in the literature, and costs were obtained in official government databases. The results were reported as incremental cost-utility ratios. Results Robotic-assisted surgery was found to be costlier but more effective than both open and laparoscopic techniques, resulting in Brazilian reals 4518 per quality-adjusted life-year and Brazilian reals 3631 per quality-adjusted life-year incremental cost-effectiveness ratios, respectively. Conclusions This study gives relevant inputs for decision making regarding the inclusion of robotic-assisted RP in the Brazilian public formularies. The study demonstrates that the technology is cost-effective even when considering willingness-to-pay thresholds lower than the traditionally used ones.

Surgical site infection is common after colorectal surgery and is associated with increased costs. Prophylactic negative pressure wound therapy has previously been shown to reduce surgical site infection compared with conventional dressings. However, negative pressure wound therapy application is met with hesitancy because of its additional cost.This study aims to determine whether the application of prophylactic negative pressure wound therapy after elective colorectal surgery is cost-effective.A cost-effectiveness analysis comparing prophylactic negative pressure wound therapy versus conventional dressing was completed using a Markov microsimulation model. A publicly funded single health care payer perspective was adopted across a lifetime horizon.This study was conducted using in-hospital elective colorectal surgery.The base case was an age-, sex-, and comorbidity-standardized patient undergoing open elective colorectal surgery.Negative pressure wound therapy was applied postoperatively over closed incisions.The primary outcomes of interest were the number of surgical site infections, total costs, and quality-adjusted life-years gained. Secondary outcomes included emergency department presentation, hospital readmission, nursing wound care utilization, fascial dehiscence, incisional hernia, and non-surgical site infection-related complications.We found that prophylactic negative pressure wound therapy, standardized to 1000 patients, prevented 51 surgical site infections, 3 fascial dehiscences, 10 incisional hernias, 22 emergency department presentations, and 6 hospital readmissions. This resulted in a total cost saving of $17,066 and 92.2 quality-adjusted life-years gained ($17.07 and 0.09 quality-adjusted life-years gained on average per patient). When the patients' risk of surgical site infections was greater than 3.2%, negative pressure wound therapy was a cost-effective strategy at a willingness to pay of $50,000/quality-adjusted life-years.We did not model for societal perspective, emergent presentations of incarcerated hernias, or complications with hernia repair. The results of this model are reliant on the published negative pressure wound therapy efficacy and may change when additional data arise.The use of negative pressure wound therapy is the dominant strategy with improved outcomes and reduced costs compared with conventional dressing in patients undergoing colorectal surgery, particularly in at-risk patients. See Video Abstract at http://links.lww.com/DCR/B782.ANTECEDENTES:La infección del sitio quirúrgico es común después de la cirugía colorrectal y se asocia con un aumento de los costos. Anteriormente se demostró que la terapia profiláctica con presión negativa reduce la infección del sitio quirúrgico en comparación con los apósitos convencionales. Sin embargo, el uso de la terapia de presión negativa se encuentra en dudas debido a su costo adicional.OBJETIVO:Determinar si la aplicación de la terapia profiláctic con presión negativa después de la cirugía colorrectal electiva es rentable.DISEÑO:Se completó un análisis de costo-efectividad comparando la terapia profiláctica con presión negativa versus apósito convencional utilizando un modelo de microsimulación de Markov. Se adoptó una perspectiva de pagador único de asistencia sanitaria financiada con fondos públicos a lo largo de toda la vida.AJUSTE:Cirugía colorrectal electiva intrahospitalaria.PACIENTES:El caso base fue un paciente estandarizado por edad, sexo y comorbilidad sometido a cirugía colorrectal abierta electiva.INTERVENCIÓN:Aplicación postoperatoria de terapia de presión negativa sobre incisiones cerradas.RESULTADOS PRINCIPALES:Los resultados primarios de interés fueron el número de infecciones del sitio quirúrgico, los costos totales y los años de vida ganados ajustados por calidad. Los resultados secundarios incluyeron presentación en la sala de emergencias, reingreso al hospital, la utilización del cuidado de heridas por enfermería, dehiscencia fascial, hernia incisional y complicaciones relacionadas con infecciones del sitio no quirúrgico.RESULTADOS:Estandarizado para 1,000 pacientes, encontramos que la terapia profiláctica con presión negativa previno 51 infecciones del sitio quirúrgico, 3 dehiscencias fasciales, 10 hernias incisionales, 22 presentaciones en la sala de emergencias y 6 reingresos al hospital. Esto resultó en un ahorro total de costos de $ 17.066 y 92.2 años de vida ganados ajustados por calidad ($ 17.07 y 0.09 años de vida ganados ajustados por calidad en promedio por paciente). Cuando el riesgo de infección del sitio quirúrgico de los pacientes era superior al 3,2%, la terapia de presión negativa era una estrategia rentable con una disposición a pagar de 50.000 dólares por años de vida ajustados por calidad.LIMITACIONES:No modelamos para la perspectiva social, presentaciones emergentes de hernias encarceladas o complicaciones con la reparación de hernias. Los resultados de este modelo dependen de la eficacia publicada de la terapia de presión negativa y pueden cambiar cuando surjan más datos.CONCLUSIONES:El uso de la terapia de presión negativa es la estrategia dominante con mejores resultados y costos reducidos en comparación con el apósito convencional en pacientes sometidos a cirugía colorrectal, particularmente en pacientes de riesgo. Consulte Video Resumen en http://links.lww.com/DCR/B782. (Traducción- Dr. Francisco M. Abarca-Rendon).

Introduction and objectives Transcatheter aortic valve implant has become a widely accepted treatment for inoperable patients with aortic stenosis and patients at high surgical risk. Its indications have recently been expanded to include patients at intermediate and low surgical risk. Our aim was to evaluate the efficiency of SAPIEN 3 vs conservative medical treatment (CMT) or surgical aortic valve replacement (SAVR) in symptomatic inoperable patients at high or intermediate risk. Methods We conducted a cost-effectiveness analysis of SAPIEN 3 vs SAVR/CMT, using a Markov model (monthly cycles) with 8 states defined by the New York Heart Association and a time horizon of 15 years, including major complications and management after hospital discharge, from the perspective of the National Health System. Effectiveness parameters were based on the PARTNER trials. Costs related to the procedure, hospitalization, complications, and follow-up were included (euros in 2019). An annual discount rate of 3% was applied to both costs and benefits. Deterministic and probabilistic sensitivity analyses (Monte Carlo) were performed. Results Compared with SAVR (high and intermediate risk) and CMT (inoperable), SAPIEN 3 showed better clinical results in the 3 populations and lower hospital stay. Incremental cost-utility ratios (€/quality-adjusted life years gained) were 5471 (high risk), 8119 (intermediate risk) and 9948 (inoperable), respectively. In the probabilistic analysis, SAPIEN 3 was cost-effective in more than 75% of the simulations in the 3 profiles. Conclusions In our health system, SAPIEN 3 facilitates efficient management of severe aortic stenosis in inoperable and high- and intermediate-risk patients.

Objectives The clinical and cost-saving benefits of transcatheter aortic valve implantation (TAVI) over surgical aortic valve replacement (SAVR) in patients with severe aortic stenosis who are at high or intermediate risk of surgical mortality are supported by a growing evidence base. The PARTNER 3 trial (Placement of AoRTic TraNscathetER Valve Trial) demonstrated clinical benefits with SAPIEN 3 TAVI compared with SAVR in selected patients at low risk of surgical mortality. This study uses PARTNER 3 outcomes in combination with a French national hospital claim database to inform a cost-utility model and examine the cost implications of TAVI over SAVR in a low-risk population. Methods A 2-stage cost-utility analysis was developed to estimate changes in both direct healthcare costs and health-related quality of life using TAVI with SAPIEN 3 compared with SAVR. Early adverse events associated with TAVI were captured using the PARTNER 3 data set. These data fed into a Markov model that captured longer-term outcomes of patients, after TAVI or SAVR intervention. Results TAVI with SAPIEN 3 offers meaningful benefits over SAVR in providing both cost saving (€12 742 per patient) and generating greater quality-adjusted life-years (0.89 per patient). These results are robust with TAVI with SAPIEN 3 remaining dominant across several scenarios and deterministic and probabilistic sensitivity analyses. Conclusions This model demonstrated that TAVI with SAPIEN 3 was dominant compared with SAVR in the treatment of patients with severe symptomatic aortic stenosis who are at low risk of surgical mortality. These findings should help policy makers in developing informed approaches to intervention selection for this patient population.

Transcarotid arterial revascularization (TCAR) has gained popularity as an alternative to carotid endarterectomy (CEA) and transfemoral carotid artery stenting (TFCAS), potentially combining the benefits of a minimally invasive approach with a lower risk of procedural stroke compared with TFCAS. Emerging evidence shows TCAR to have excellent perioperative outcomes. However, the cost-effectiveness of TCAR is not well-understood.Incorporating data from Carotid Revascularization Endarterectomy vs Stenting Trial (CREST), the Vascular Quality Initiative Surveillance Project, and local cost data, we compared the cost-effectiveness of these three treatment modalities (TFCAS, CEA, and TCAR) for both symptomatic and asymptomatic carotid stenosis using a Markov state-transition model to quantify lifetime costs in United States dollars and effectiveness in quality-adjusted life-years (QALYs). We accounted for perioperative stroke and myocardial infarction, as well as long-term risks of stroke and restenosis. Based on CREST, we assumed a start age of 69 years and a cost-effectiveness acceptability threshold of $100,000/QALYs gained. Sensitivity analyses were performed.In the base-case scenario, TCAR cost $160,642/QALY gained compared with CEA, greater than the frequently cited $100,000/QALY gained threshold. TFCAS was more expensive and less effective than other strategies, largely due to a greater periprocedural stroke risk. In one-way sensitivity analysis, if TCAR stroke risk was 0.9% (base-case risk, 1.4%), than it was economically favorable compared with CEA at its current procedural cost. Alternatively, if TCAR procedural costs were reduced by approximately $2000 (base-case cost, $15,182), it would also become economically favorable. In a probabilistic sensitivity analysis, varying all parameters simultaneously over distributions, CEA was favored in 80% of model iterations at $100,000/QALY, with TCAR favored in 19%.At current cost and outcomes, TCAR does not meet a traditional cost-effectiveness threshold to replace CEA as the primary treatment modality for carotid stenosis. TFCAS is the least cost-effective strategy for carotid revascularization. Given these observations, TCAR should be limited to select patients, specifically those at high physiologic and anatomic risk from CEA. However, TCAR can become cost-effective if its cost is reduced. Given the current outcomes and cost, CEA remains the most cost-effective treatment for carotid revascularization.

Rationale: The objective was to assess the cost-effectiveness of staging positron emission tomography/computed tomography (PET/CT) in early-stage follicular lymphoma from the Canadian health care system perspective. Methods: The study population was FL patients staged as early-stage using conventional CT imaging and planned for curative-intent radiation therapy (RT). A decision analytic model simulated the management after adding a staging PET/CT vs. using staging CT alone. In the no-PET/CT strategy, all patients proceeded to curative-intent RT as planned. In the PET/CT strategy, PET/CT information could result in an increased RT volume, switching to a non-curative approach, or no change in RT treatment as planned. Subsequent disease course was described using a state-transition cohort model over a 30-year time horizon. Diagnostic characteristics, probabilities, utilities and costs were derived from the literature. Baseline analysis was performed using quality-adjusted life years (QALYs), costs (2019 Canadian dollars, CAD$) and the incremental cost-effectiveness ratio. Deterministic sensitivity analyses were conducted, evaluating net monetary benefit at a willingness-to-pay threshold of $100,000/QALY. Probabilistic sensitivity analysis using 10,000 simulations was performed. Costs and QALYs were discounted at a rate of 1.5%. Results: In the reference case scenario, staging PET/CT was the dominant strategy, resulting in an average lifetime cost saving of $3,165 and a gain of 0.32 QALYs. In deterministic sensitivity analyses, the PET/CT strategy remained the preferred strategy for all scenarios supported by available data. In probabilistic sensitivity analysis, the PET/CT strategy was strongly dominant in 77% of simulations (i.e., reduced cost and increased QALYs), and was cost-effective in 89% of simulations (i.e., either cost-saving or with an incremental cost-effectiveness ratio below $100,000/QALY). Conclusion: Our analysis shows that the use of PET/CT to stage early-stage FL patients reduces cost and improves QALYs. Patients with early-stage FL should undergo PET/CT prior to curative-intent RT.

The interacting components of open innovation are complex to assess empirically and theoretically. Open innovation represents a high-potential scheme to share knowledge, and collaborate; however, it needs to be carefully contextualized to succeed. The challenges that open innovation platform design encounters are as follows: attraction motivation of all actors involved; intellectual property management; costs and benefits to keep the stakeholders´ interest; information structure; governance; quality assurance; trust and reliability; and finally associated risks. Built on these challenges, our contribution offers an open innovation platform design framework, with three components: 1) ecosystems context providing foundation, purpose, and alignment amongst all actors involved; 2) innovation management integrating three stages: creation of reference framework to set the basis and governance policies; definition of support framework to technology, processes, and quality assurance; and planning and leading delivery framework, to ensure the continuity of the platform; and 3) cocreation support consolidating the resource integration and cocreation processes. Additionally, we discuss the findings from the analysis of an exploratory multiple-case study regarding the level of approach of the processes and strategies defined in the framework and the designs of open innovation platforms located at Kenya, Mexico, Nicaragua, and United States.

Introduction Objective Structured Clinical Examinations (OSCEs) are an accepted technique for evaluation of clinical competence in healthcare. However, the economic imperative requires faculty to control cost, using innovative educational strategies such as virtual simulation. The objective of this study was to evaluate the cost implications of implementing an online interactive learning module [Monash OSCE Virtual Experience (MOVE)]. Methods All fourth-year pharmacy students enrolled in Monash University in 2017 were provided access to MOVE. Cost-minimization analyses were performed to evaluate the cost of introducing MOVE in the pharmacy course using the smallest cohort size (Malaysia campus) of 40 students as the base case. We also determined under what circumstances MOVE would be more cost-effective, considering the different operational situations such as when student numbers increased or when the number of simulation modules created were increased. Results The overall cost of setup and implementation of MOVE in the first year of implementation among 40 students was US $94.38 per student. In comparison, the face-to-face workshop cost was US $64.14 per student. On the second year of implementation, the ongoing cost of operation of MOVE was US $32.86 per student compared with US $58.97 per student using face-to-face workshop. A net benefit using MOVE was observed after the third year of implementation. Larger savings were noted when the cohort size extends larger than 100 students. Conclusions Monash OSCE Virtual Experience was a flexible and cost-effective approach to aid students in preparation for an OSCE and enhanced students' learning experience. The wider applicability of these findings will need to be explored in other settings.

Increasing rates of simultaneous heart-kidney (SHK) transplant in the United States exacerbate the overall shortage of deceased donor kidneys (DDK). Current allocation policy does not impose constraints on SHK eligibility, and how best to do so remains unknown. We apply a decision-analytic model to evaluate options for heart transplant (HT) candidates with comorbid kidney dysfunction. We compare SHK with a "Safety Net" strategy, in which DDK transplant is performed 6 months after HT, only if native kidneys do not recover. We identify patient subsets for whom SHK using a DDK is efficient, considering the quality-adjusted life year (QALY) gains from DDKs instead allocated for kidney transplant-only. For an average-aged candidate with a 50% probability of kidney recovery after HT-only, SHK produces 0.64 more QALYs than Safety Net at a cost of 0.58 more kidneys used. SHK is inefficient in this scenario, producing fewer QALYs per DDK used (1.1) than a DDK allocated for KT-only (2.2). SHK is preferred to Safety Net only for candidates with a lower probability of native kidney recovery (24%-38%, varying by recipient age). This finding favors the implementation of a Safety Net provision and should inform the establishment of objective criteria for SHK transplant eligibility.

Japan recently approved a quadrivalent human papillomavirus (HPV) vaccine for males, but the 9-valent vaccine has only been approved for females. Given the low female vaccination rate due to concerns about adverse events in Japan, quantifying the risks and benefits of the HPV vaccination for males may help in deciding whether to vaccinate adolescent boys in Japan. Using quality-adjusted life years, the risk-benefit ratio for an adolescent boy receiving the 9-valent HPV vaccination was calculated. The male HPV vaccination reduced the QALYs gained due to head and neck cancer, anal cancer, penile cancer, genital warts, and recurrent respiratory papillomatosis by 401.63, 20.38, 9.40, 28.79, and 69.13 per 100,000 vaccinated persons, respectively. The total risk of vaccination was 11.85. The risk-benefit ratio for a 12-year-old boy who received the HPV vaccination series was found to be 0.022 (benefit-risk ratio, 44.670). In the sensitivity analysis, the risk-benefit ratio ranged from 0.0001 to 0.11 for all scenarios. Hence, much larger benefits than risks linked with the male HPV vaccination were observed from an individual perspective. The results support the inclusion of sex-neutral HPV vaccinations into the national immunization program as well as strengthens the decision for adolescent boys to receive the vaccination.

Duloxetine is a treatment approved by the US Food and Drug Administration for both osteoarthritis (OA) pain and depression, though uptake of duloxetine in knee OA management varies. We examined the cost-effectiveness of adding duloxetine to knee OA care in the absence or presence of depression screening.We used the Osteoarthritis Policy Model, a validated computer microsimulation of knee OA, to examine the value of duloxetine for patients with knee OA who have moderate pain by comparing 3 strategies: 1) usual care, 2) usual care plus duloxetine for patients who screen positive for depression on the Patient Health Questionnaire 9 (PHQ-9), and 3) usual care plus universal duloxetine. Outcome measures included quality-adjusted life years (QALYs), lifetime direct medical costs, and incremental cost-effectiveness ratios (ICERs), discounted at 3% annually. Model inputs, drawn from the published literature and national databases, included annual cost of duloxetine ($721-937); average pain reduction for duloxetine (17.5 points on the Western Ontario and McMaster Universities Osteoarthritis Index pain scale [0-100]), and likelihood of depression remission with duloxetine (27.4%). We considered 2 willingness-to-pay (WTP) thresholds of $50,000/QALY and $100,000/QALY. We varied parameters related to the PHQ-9 and the cost of duloxetine, efficacy, and toxicities to address uncertainty in model inputs.The screening strategy led to an additional 17 QALYs per 1,000 subjects and increased costs by $289/subject (ICER = $17,000/QALY). Universal duloxetine led to an additional 31 QALYs per 1,000 subjects and $1,205 per subject (ICER = $39,300/QALY). Under the majority of sensitivity analyses, universal duloxetine was cost-effective at the $100,000/QALY threshold.The addition of duloxetine to usual care for knee OA patients with moderate pain, regardless of depressive symptoms, is cost-effective at frequently used WTP thresholds.

Objective: Musculoskeletal conditions of the foot and ankle are common, yet the cost‐effectiveness of the variety of treatments available is not well defined. The aim of this systematic review was therefore to identify, appraise and synthesise the literature pertaining to the cost‐effectiveness of interventions for musculoskeletal foot and ankle conditions. Methods: Electronic databases were searched for studies presenting economic evaluations of non‐surgical and surgical treatments for acute or chronic musculoskeletal conditions of the foot and ankle. Data on cost, incremental cost‐effectiveness and quality‐adjusted life years for each intervention and comparison were extracted. Risk of bias was assessed using the Drummond checklist for economic studies (range 0‐35). Results: Thirty‐six studies were identified reporting non‐surgical interventions (n=10), non‐surgical versus surgical interventions (n=14) and surgical interventions (n=12). The most common conditions were osteoarthritis, ankle fracture and Achilles tendon rupture. The strongest economic evaluations were for interventions managing end‐stage ankle osteoarthritis, ankle sprain, ankle fracture, calcaneal fracture, and Achilles tendon rupture. Total ankle replacement and ankle arthrodesis for end‐stage ankle osteoarthritis, in particular, have been demonstrated through high‐quality studies to be cost‐effective compared to the non‐surgical alternative. Conclusion: Selected interventions for musculoskeletal foot and ankle conditions dominate comparators, whereas others require thoughtful consideration as they provide better clinical improvements, but at an increased cost. Researchers should consider measuring and reporting costs alongside clinical outcome to provide context when determining the appropriateness of interventions for other foot and ankle complaints to best inform future clinical practice guidelines. Refereed/Peer-reviewed

Adjuvant chemotherapy is not recommended for patients with average-risk stage II (T3N0) colon cancer. Nevertheless, a subgroup of these patients who are CDX2-negative might benefit from adjuvant chemotherapy. We evaluated the cost-effectiveness of testing for the absence of CDX2 expression followed by adjuvant chemotherapy (fluorouracil combined with oxaliplatin [FOLFOX]) for patients with stage II colon cancer.We developed a decision model to simulate a hypothetical cohort of 65-year-old patients with average-risk stage II colon cancer with 7.2% of these patients being CDX2-negative under 2 different interventions: (1) test for the absence of CDX2 expression followed by adjuvant chemotherapy for CDX2-negative patients and (2) no CDX2 testing and no adjuvant chemotherapy for any patient. We derived disease progression parameters, adjuvant chemotherapy effectiveness and utilities from published analyses, and cancer care costs from the Surveillance, Epidemiology, and End Results (SEER)-Medicare data. Sensitivity analyses were conducted.Testing for CDX2 followed by FOLFOX for CDX2-negative patients had an incremental cost-effectiveness ratio of $5500/quality-adjusted life-years (QALYs) compared with no CDX2 testing and no FOLFOX (6.874 vs 6.838 discounted QALYs and $89 991 vs $89 797 discounted US dollar lifetime costs). In sensitivity analyses, considering a cost-effectiveness threshold of $100 000/QALY, testing for CDX2 followed by FOLFOX on CDX2-negative patients remains cost-effective for hazard ratios of0.975 of the effectiveness of FOLFOX in CDX2-negative patients in reducing the rate of developing a metastatic recurrence.Testing tumors of patients with stage II colon cancer for CDX2 and administration of adjuvant treatment to the subgroup found CDX2-negative is a cost-effective and high-value management strategy across a broad range of plausible assumptions.

PURPOSE: To discuss the challenges and potential improvement strategies of cost-effectiveness analyses performed for therapeutics targeting inherited retinal diseases. DESIGN: Perspective METHODS: Literature review with discussion of current limitations and improvement recommendations. RESULTS: Cost-effectiveness analysis (CEA) performed for inherited retinal diseases (IRD) therapeutics has multiple limitations. First, the available methods used to measure health-related quality of life and health utilities can be inaccurate when used in IRDs. Second, the financial burden to patients and society by vision impairment associated with IRDs has been inadequately studied and includes a variety of expenditures ranging from direct costs of IRD specialty healthcare to indirect expenses associated with daily living activities. Third, our collective understanding is limited in the areas of IRD natural history and health benefits gained from new IRD treatments (e.g. gene therapies). Additionally, the therapeutic effect from a patient perspective and its duration of action is not fully understood. Due to the scarcity of data, CEA for newly approved therapies has relied on assumptions and creations of predictive models for both costs and health benefits for these new therapeutics in order to calculate the incremental cost-effectiveness ratio (ICER). CONCLUSIONS: CEA studies performed for IRD therapeutics have been limited by the currently established health utilities in ophthalmology and the lack of disease-specific information. The assumptions and extrapolations in these studies create substantial uncertainty in ICER results. An improved framework is required for CEA of IRD therapeutics in order to determine the cost-effectiveness of each therapy brought from clinical trials to clinical practice. TABLE OF CONTENTS STATEMENT: Cost-effectiveness analysis (CEA) studies performed for inherited retinal disease (IRD) therapeutics have been limited by the currently established health utilities in ophthalmology which lack disease specific information. The assumptions and extrapolations made in these studies are susceptible to large amounts of uncertainty in incremental cost-effectiveness ratio calculations. An improved framework for CEA of IRD therapeutics would better inform policy makers and other stakeholders, as these therapies progress from preclinical testing to clinical trials, and finally to clinical practice

Introduction Glycerol-preserved skin allograft (GPA) plays a vital role, especially in the management of burns injury. Where it is utilized as temporary wound closure, the considerably cost-effective, simpler handling and storage of GPA makes it preferable in almost all clinical indications. The GPA was first introduced to the Hospital Universiti Sains Malaysia in 2001. The supply was imported from Euro Skin Bank, Beverwijk, The Netherlands. In the year 2013, our center had started maintaining an in-house glycerolized skin bank. Method We preserved donor skin grafts from patients who underwent plastic surgery-related procedures in 85% glycerol and stored them at +2 °C to +10 °C. Cost estimation of the GPA per cm2 was calculated to analyze the effectiveness of its preservation technique. Results The cost of GPA from our skin bank is estimated to be almost 90% reduction in cost as compared to the supply from Euro Skin Bank. Conclusion The selective and strategic use of the GPA in major burn patients assure effective advantages in the treatment of burns. The clinical significance of skin allograft usage is very high. The cost-effectiveness of maintaining an in-house skin bank made it possible for various centers for skin allograft usage.

Background Tolvaptan (TLV) is effective for acute heart failure (HF) with congestion, but its long-term administration in patients with chronic HF (CHF) remains controversial. Moreover, the cost-effectiveness of TLV for CHF treatment has not yet been investigated. Thus, we sought to validate the cost-effectiveness of TLV for CHF treatment in Japan. Methods A Markov model was developed to compare total costs, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER) between long-term TLV strategy and the standard strategy using furosemide for CHF. The target population included 75-year-old patients with CHF. The effectiveness of the TLV strategy for CHF treatment was determined based on a systematic review and meta-analysis. We used a 10-year horizon, with sensitivity analyses for significant variables and a scenario analysis for patients with CHF receiving high-dose furosemide (≥60 mg per day). Results In the base case analysis, the total cost of the long-term TLV strategy was higher than that of the standard strategy (\3,243,779 vs. \1,179,964). The total QALYs of the long-term TLV strategy were lower than those of the standard strategy (4.52 vs 4.59). Thus, a standard TLV prescription for CHF treatment has no clinical or economic benefit. In the scenario analysis (i.e. in patients with CHF receiving high-dose furosemide), the long-term TLV strategy was more effective (total QALYs, 5.10 vs. 4.41) but more expensive (total costs, \3,540,558 vs. \1,272,208) than the standard strategy. The ICER of the TLV strategy against the standard strategy (¥3,289,579/QALY) was below the willingness-to-pay of \5,000,000, which suggests that the long-term TLV strategy is cost-effective relative to the standard strategy in patients with CHF receiving high-dose furosemide. Conclusions Long-term TLV administration did not provide a clear benefit for all patients with CHF. However, this treatment strategy may be a cost-effective therapeutic option for patients who require high-dose furosemide.

Objectives In 2017, the Argentine Ministry of Health incorporated a sequential 13-valent pneumococcal conjugate vaccine (PCV13)-23-valent pneumococcal polysaccharide vaccine (PPSV23) regimen for adults aged ≥65 years to reduce pneumococcal disease burden. Cost-effectiveness analysis of PCV13-PPSV23 schedule for adults aged ≥65 years in Argentina was performed compared with PPSV23 only. Methods Markov model was developed. Local data were incorporated for costs and disease burden analysis. Vaccine efficacy or effectiveness was obtained from a systematic review adjusted to current local vaccine serotype circulation and vaccines coverage. A total of 3 scenarios were evaluated: main scenario according to published literature of pneumonia incidence, epidemiologic surveillance scenario based on Argentine Ministry of Health data, and an alternative scenario assuming a 50% hypothetical pneumonia incidence reduction resulting from herd immunity induced by childhood vaccination. Sensitivity analyses were done. Results Sequential PCV13-PPSV23 schedule showed cost-savings results in the main scenario with −$1 667 742.23 saved and 716 life-years gained (LYG). The epidemiologic surveillance scenario showed an incremental cost-effectiveness ratio of $2141.92 per LYG and an alternative scenario with $3740.30 per LYG. Tornado diagram shows widest bars related to adjustment for vaccine-type pneumococcal pneumonia (urine analysis) pneumonia at risk cost and pneumonia incidence rate. Monte Carlo simulation shows that >98% of simulations were cost-saving for the main scenario. Conclusions In the main scenario, cost-saving results were obtained considering only reduction of vaccine serotype coverage after the introduction of childhood PCV13 vaccination. In the epidemiologic surveillance and alternative scenarios, assuming a hypothetical incidence reduction, highly cost-effective results were observed.