Health-economic evaluation of meniscus tear treatments: a systematic review.

PURPOSE: To evaluate the overall evidence of published health-economic evaluation studies on meniscus tear treatment. METHODS: Our systematic review focuses on health-economic evaluation studies of meniscus tear treatment interventions found in PubMed and Embase databases. A qualitative, descriptive approach was used to analyze the studies' results and systematically report them following PRISMA guidelines. The health-economic evaluation method for each included study was categorized following one of the four approaches: partial economic evaluation (PEE), cost-effectiveness analysis (CEA), cost-benefit analysis (CBA), or cost-utility analysis (CUA). The quality of each included study was assessed using the Consensus on Health Economic Criteria (CHEC) list. Comparisons of input variables and outcomes were made, if applicable. RESULTS: Sixteen studies were included; of these, six studies performed PEE, seven studies CUA, two studies CEA, and one study combined CBA, CUA, and CEA. The following economic comparisons were analyzed and showed the respective comparative outcomes: (1) meniscus repair was more cost-effective than arthroscopic partial meniscectomy (meniscectomy) for reparable meniscus tear; (2) non-operative treatment or physical therapy was less costly than meniscectomy for degenerative meniscus tear; (3) physical therapy with delayed meniscectomy was more cost-effective than early meniscectomy for meniscus tear with knee osteoarthritis; (4) meniscectomy without physical therapy was less costly than meniscectomy with physical therapy; (5) meniscectomy was more cost-effective than either meniscus allograft transplantation or meniscus scaffold procedure; (6) the conventional arthroscopic instrument cost was lower than laser-assisted arthroscopy in meniscectomy procedures. CONCLUSION: Results from this review suggest that meniscus repair is the most cost-effective intervention for reparable meniscus tears. Physical therapy followed by delayed meniscectomy is the most cost-effective intervention for degenerative meniscus tears. Meniscus scaffold should be avoided, especially when implemented on a large scale. LEVEL OF EVIDENCE: Systematic review of level IV studies.

"Cost-effectiveness of ACL treatment is dependent on age and activity level: a systematic review".

PURPOSE: To systematically review the literature on health-economic evaluations of anterior cruciate ligament (ACL) injury between reconstruction surgery (ACLR) and non-operative treatment (NO) and suggest the most cost-effective strategy between the two. METHODS: All economic studies related to ACLR versus NO post-ACL injury, either trial based or model based, published until April 2022, were identified using PubMed and Embase. The methodology of the health-economic analysis for each included study was categorized according to the four approaches: cost-minimization analysis (CMA), cost-effectiveness analysis (CEA), cost-benefit analysis (CBA), and cost-utility analysis (CUA). The quality of each included study was assessed using the Consensus on Health Economic Criteria (CHEC) list. RESULTS: Of the seven included studies, two compared the strategies of early ACLR and NO alone, and five compared early ACLR and NO with optional delayed ACLR. All studies performed a CUA, and one study performed a CBA additionally. The CHEC scores of the included studies can be considered good, ranging from 15 to 18 from a maximum of 19. Applying the common standard threshold of $50,000 per QALY, six studies in young people with high-activity levels or athletes showed that early ACLR would be preferred over either NO alone or delayed ACLR. Of six studies, two even showed early ACLR to be the dominant strategy over either NO alone or delayed ACLR, with per-patient cost savings of $5,164 and $1,803 and incremental per-patient QALY gains of 0.18 and 0.28, respectively. The one study in the middle-aged people with a moderate activity level showed that early ACLR was not more cost-effective than delayed ACLR, with ICER $101,939/QALY using the societal perspective and ICER $63,188/QALY using the healthcare system perspective. CONCLUSION: Early ACLR is likely the more cost-effective strategy for ACL injury cases in athletes and young populations with high-activity levels. On the other hand, non-operative treatment with optional delayed ACLR may be the more cost-effective strategy in the middle age population with moderate activity levels. LEVEL OF EVIDENCE: Systematic review of level III studies.

Exploratory Analysis of the Economically Justifiable Price of a Hypothetical RSV Vaccine for Older Adults in the Netherlands and the United Kingdom.

BACKGROUND: In older adults, the burden of respiratory syncytial virus (RSV) resembles that of influenza and may even be considered worse due to the lack of preventive interventions. This study was performed to identify the available literature on RSV infection in older adults, and to provide updated exploratory results of the cost-effectiveness of a hypothetical RSV vaccine in the Netherlands and the United Kingdom. METHODS: A literature search was performed in Medline and EMBASE on 11 November 2019, which served as input for a static decision-tree model that was used to estimate the EJP, for an RSV vaccine applying different willingness-to-pay (WTP) thresholds. WTP thresholds applied were €20 000 and €50 000 per quality-adjusted life-year for the Netherlands, and £20 000 and £30 000 per quality-adjusted life-year for the United Kingdom. Analyses were-in line with country-specific guidelines-conducted from a societal perspective for the Netherlands and a third-party payer perspective for the United Kingdom. The robustness of the cost-effectiveness results was tested in sensitivity analysis. RESULTS: After screening the literature, 3 studies for the Netherlands and 6 for the United Kingdom remained to populate the country-specific models. In the base case analysis for the Netherlands (mean RSV incidence, 3.32%), justifiable vaccine prices of €16.38 and €50.03 were found, based on applying the lower and higher WTP thresholds, respectively. Similarly, for the United Kingdom (mean incidence, 7.13%), vaccine prices of £72.29 and £109.74 were found, respectively. CONCLUSION: RSV vaccination may well be cost-effective in both the Netherlands and the United Kingdom, depending on the exact RSV incidence, vaccine effectiveness and price. However, sensitivity analysis showed that the results were robust based on varying the different parameter estimates and assumptions. With RSV vaccines reaching the final stages of development, a strong need exists for cost-effectiveness studies to understand economically justifiable pricing of the vaccine.

Cost-minimisation analysis of a treat-and-extend regimen with anti-VEGFs in patients with neovascular age-related macular degeneration.

PURPOSE: Although intraocular anti-vascular endothelial growth factors (anti-VEGFs) are effective as treatment of neovascular age-related macular degeneration (nAMD), the (economic) burden on the healthcare system is considerable. A treat-and-extend (T&E) regimen is associated with a lower number of injections without compromising the effectiveness and can therefore help optimise nAMD treatment. This study investigates the per-patient costs associated with nAMD treatment, when using aflibercept, bevacizumab, or ranibizumab with a T&E regimen. METHODS: In this cost-minimisation model, the per-patient costs in the Netherlands were modelled using a healthcare payers' perspective over a 3-year time horizon with the assumption that efficacy of treatments is similar. Additionally, the break-even price of the different anti-VEGFs was calculated relative to the cheapest option and injection frequency. RESULTS: The injection frequency varied from 14.2 for aflibercept to 27.4 for bevacizumab in 3 years. Nonetheless, bevacizumab remains the cheapest treatment option (€14,215), followed by aflibercept (€18,202) and ranibizumab (€31,048). The medication covers the majority of the per-patient costs for aflibercept and ranibizumab, while administration covers the majority of the per-patient costs for bevacizumab. The break-even prices of aflibercept and ranibizumab are respectively €507 and €60.58 per injection. Brolucizumab was included in the scenario analysis and was more expensive than aflibercept (€20,446). Brolucizumab should reduce to 13.8 injections over 3 years to be as costly as aflibercept. CONCLUSION: Bevacizumab is the cheapest anti-VEGF treatment. The list prices of all anti-VEGFs should reduce to be as costly as bevacizumab. Aflibercept is the second-choice treatment and so far brolucizumab is not.

HIV care cost in England: a cross-sectional analysis of antiretroviral treatment and the impact of generic introduction.

OBJECTIVES: Reliable and timely HIV care cost estimates are important for policy option appraisals of HIV treatment and prevention strategies. As HIV clinical management and outcomes have changed, we aimed to update profiles of antiretroviral (ARV) usage pattern, patent/market exclusivity details and management costs in adults (≥ 18 years old) accessing HIV specialist care in England. METHODS: The data reported quarterly to the HIV and AIDS Reporting System in England was used to identify ARV usage pattern, and were combined with British National Formulary (BNF) prices, non-ARV care costs and patent/market exclusivity information to generate average survival-adjusted lifetime care costs. The cumulative budget impact from 2018 to the year in which all current ARVs were expected to lose market exclusivity was calculated for a hypothetical 85 000 (± 5000) person cohort, which provided an illustration of potential financial savings afforded by bioequivalent generic switches. Price scenarios explored BNF70 (September 2015) prices and generics at 10/20/30/50% of proprietary prices. The analyses took National Health Service (NHS) England's perspective (as the payer), and results are presented in 2016/2017 British pounds. RESULTS: By 2033, most currently available ARVs would lose market exclusivity; that is, generics could be available. Average per person lifetime HIV cost was ~£200 000 (3.5% annual discount) or ~£400 000 (undiscounted), reducing to ~£70 000 (3.5% annual discount; ~£120 000 undiscounted) with the use of generics (assuming that generics cost 10% of proprietary prices). The cumulative budget to cover 85 000 (± 5000) persons for 16 years (2018-2033) was £10.5 (± 0.6) billion, reducing to £3.6 (± 0.2) billion with the use of generics. CONCLUSIONS: HIV management costs are high but financial efficiency could be improved by optimizing generic use for treatment and prevention to mitigate the high cost of lifelong HIV treatment. Earlier implementation of generics as they become available offers the potential to maximize the scale of the financial savings.

Burden of early, advanced and metastatic breast cancer in The Netherlands.

BACKGROUND: The aim of this study was to estimate the total economic and health related burden of breast cancer in the Netherlands. METHODS: Data on incidence, prevalence, mortality and survival were extracted from the Dutch National Cancer Registry and were used to calculate the economic and health related burden of breast cancer for overall, DCIS (stage 0), early- (stage I), locally advanced- (stage II-III) and metastatic- (stage IV) breast cancer by age groups and by year (if applicable). RESULTS: The overall incidence of breast cancer increased from 103.4 up to 153.2 per 100,000 women between 1990 and 2014. The increase was driven by DCIS and early breast cancer as the incidence of locally advanced and metastatic breast cancer remained stable. Between 1990 and 2014, ten-year overall survival rates increased from 87% to 93% for early breast cancer, 41% to 62% for locally advanced- and from 6% to 9% for metastatic disease. Annually, breast cancer in the Netherlands is responsible for approximately 3100 deaths, 26,000 life years lost, 65,000 Disability Adjusted Life Years (DALYs) and an economic burden of €1.27 billion. CONCLUSIONS: This study provides a comprehensive assessment of the burden of breast cancer and subsequent trends over time in the Netherlands.

Quantifying the impact of mass vaccination programmes on notified cases in the Netherlands.

Vaccination programmes are considered a main contributor to the decline of infectious diseases over the 20th century. In recent years, the national vaccination coverage in the Netherlands has been declining, highlighting the need for continuous monitoring and evaluation of vaccination programmes. Our aim was to quantify the impact of long-standing vaccination programmes on notified cases in the Netherlands. We collected and digitised previously unavailable monthly case notifications of diphtheria, poliomyelitis, mumps and rubella in the Netherlands over the period 1919-2015. Poisson regression models accounting for seasonality, multi-year cycles, secular trends and auto-correlation were fit to pre-vaccination periods. Cases averted were calculated as the difference between observed and expected cases based on model projections. In the first 13 years of mass vaccinations, case notifications declined rapidly with 82.4% (95% credible interval (CI): 74.9-87.6) of notified cases of diphtheria averted, 92.9% (95% CI 85.0-97.2) cases of poliomyelitis, and 79.1% (95% CI 67.1-87.4) cases of mumps. Vaccination of 11-year-old girls against rubella averted 49.9% (95% CI 9.3-73.5) of cases, while universal vaccination averted 68.1% (95% CI 19.4-87.3) of cases. These findings show that vaccination programmes have contributed substantially to the reduction of infectious diseases in the Netherlands.

Health-related quality of life in HIV/AIDS patients on antiretroviral therapy at a tertiary care facility in Zimbabwe.

Health-related quality of life (HRQoL) is a broad concept reflecting a patient's general subjective perception of the effect of an illness or intervention on physical, psychological and social aspects of their daily life. HRQoL among patients infected with HIV has become an important indicator of impact of disease and treatment outcomes. A cross-sectional survey was carried out at Chitungwiza Central Hospital, Zimbabwe, to assess HRQoL in patients with HIV/AIDS receiving antiretroviral therapy (ART), using two validated instruments. The HIV/AIDS-targeted quality of life (HAT-QoL) and EuroQoL Five-dimensions-Three-level (EQ-5D-3L) instruments were used to assess HRQoL. Internal consistency reliability and convergent validity of the two instruments were also evaluated. For construct validity, the relationships between HRQoL scores and socio-economic and HIV/AIDS-related characteristics were explored. The median scores for the HAT-QoL dimensions ranged from 33.3 (financial worries) to 100 (HIV mastery). A considerably low HAT-QoL dimension score of 50.0 was observed for sexual function. There were ceiling effects for all HAT-QoL dimension scores except for financial worries and disclosure worries. Floor effects were observed for financial worries and sexual function. The median of the EQ-5D-3L index and visual analogue scale (VAS) was 0.81 and 79.0, respectively. There were no floor or ceiling effects for both the EQ-5D-3L index and VAS. The overall scale Cronbach's alpha was 0.83 for HAT-Qol and 0.67 for EQ-5D-3L. HAT-QoL demonstrated good convergent validity with EQ-5D index (0.58) and VAS (0.40). A higher level of HRQoL was positively and significantly related to income, education and employment. The patients' self-reported HRQoL was generally satisfactory in all the HAT-QoL dimensions as well as the two components on the EQ-5D-3L instrument. The two instruments demonstrated good measurement properties in HIV/AIDS patients receiving ART and have potential for use, alongside biomarkers, in monitoring outcomes of interventions.

Costs and benefits of individuals conceived after IVF: a net tax evaluation in The Netherlands.

This study evaluated the lifetime future net tax revenues from individuals conceived after IVF relative to those naturally conceived. A model based on the method of generational accounting was developed to evaluate investments in IVF. Calculations were based on average investments paid and received from the government by an individual. All costs were discounted to their net present values and adjusted for survival. The lifetime net present value of IVF-conceived individuals was -€81,374 (the minus sign reflecting negative net present value). The lifetime net present value of IVF-conceived men and women were -€47,091 and -€123,177, respectively. The lifetime net present value of naturally conceived individuals was -€70,392; respective amounts for men and women were -€36,109 and -€112,195. The model was most sensitive to changes in the growth of healthcare costs, economic growth and the discount rate. Therefore, it is concluded that, similarly to naturally conceived individuals in the Netherlands, IVF-conceived individuals have negative discounted net tax revenue at the end of life. The analytic framework described here undervalues the incremental value of an additional birth because it only considers the fiscal consequences of life and does not take into consideration broader macroeconomic benefits. This study evaluated the lifetime future net tax revenues from individuals conceived after IVF relative those naturally conceived. A model based on the method of generational accounting to evaluate investments in IVF was used. Calculations were based on average investments paid and received from the government by an individual. The lifetime net present value of IVF-conceived individuals was -€81,374 (the minus sign reflecting negative net present value). The lifetime net present value of IVF-conceived men and women were -€47,091 and -€123,177, respectively. The lifetime net present value of naturally conceived individuals was -€70,392; respective amounts for men and women were -€36,109 and -€112,195. The model was most sensitive for changes in the growth in healthcare costs, economic growth and the discount rate. Just as naturally conceived individuals in the Netherlands, IVF-conceived individuals have negative discounted net tax revenue at the end of life. The analytic framework described here undervalues the incremental value of an additional birth because it only considers the fiscal consequences of life and does not take into consideration broader macroeconomic benefits.

Can Cyprus Afford Luspatercept? A Budget Impact Analysis of the Reimbursement of Luspatercept for the Management of Thalassaemia in Cyprus.

OBJECTIVE: This study aims to estimate the budget impact of luspatercept reimbursement as an adjuvant to the standard management of ß-thalassaemia major in Cyprus, from a societal perspective, and assess the financial feasibility of its inclusion in the ß-thalassaemia armamentarium. METHODS: A 5-year horizon budget impact model was developed to determine the budget impact of reimbursing luspatercept for the management of ß-thalassaemia major in Cyprus. Two treatment discontinuation scenarios were elaborated. In the first scenario, luspatercept is reimbursed complementary to best supportive care, and a dropout rate of 40% is assumed based on published real-world data, while for the second scenario a dropout rate of 25%, is assumed as per the clinical trial data. Input parameters were retrieved from the phase III clinical trial of luspatercept, literature, and expert opinion consensus. One-way sensitivity analyses were conducted for both scenarios. RESULTS: The addition of luspatercept to the standard management of ß-thalassaemia major in Cyprus imparted an incremental budget impact ranging from €21,300,643 to €25,834,368, depending on the drop-out rate scenario assumed. Results were sensitive to the number of eligible patients and dose per patient. CONCLUSION: The potential reimbursement of luspatercept will wield a substantial impact on Cyprus total pharmaceutical expenditure and it is therefore imperative to affix a reimbursement framework that will allow the payer to mitigate uncertainty stemming out of the scarce clinical data and the inherently complex therapeutic landscape of ß-thalassemia management.

The role of Streptococcus pneumoniae in community-acquired pneumonia among adults in Europe: a meta-analysis.

The primary objective of this meta-analysis was to estimate the prevalence of adult community-acquired pneumonia (CAP) caused by Streptococcus pneumoniae in Europe, adjusted for possible independent covariates. Two reviewers conducted a systematic literature search using PubMed on English-language articles that involved human subjects with CAP during the period from January 1990 to November 2011 across European countries. A mixed-effects meta-regression model was developed and populated with 24,410 patients obtained from 77 articles that met the inclusion criteria. The model showed that the observed prevalence of S. pneumoniae in CAP significantly varies between European regions, even after adjusting for explanatory covariates, including patient characteristics, diagnostic tests, antibiotic resistance, and health-care setting. The probability of detecting S. pneumoniae was substantially higher in studies that performed more frequently a diagnostic polymerase chain reaction assay compared to all the other diagnostic tests included. Furthermore, S. pneumoniae was more likely to be confirmed as the cause of a CAP in studies with intensive care unit patients as compared to those with hospital- or community-treated patients. This study provides estimates of the average observed prevalence of S. pneumoniae, which could be used for projecting the health and economic benefits of pneumococcal immunization.

Evaluating interventions to improve somatic health in severe mental illness: a systematic review.

OBJECTIVE: To present a systematic review of the evaluation of randomized interventions directed toward improving somatic health for patients with severe mental illness (SMI). METHOD: A systematic search in PubMed, Embase, Cinahl, and PsycInfo was performed. The scope of the search was prospective studies for patients aged 18-70, published from January 2000 till June 2011. Randomized interventions directed toward improving somatic health for patients with SMI were selected. We excluded studies on elderly, children, and studies performed before 2000. Information on population, type of intervention, follow-up, outcome measures, and on authors' conclusions were drawn from the original articles. RESULTS: Twenty-two original studies were included, presenting four types of interventions: health education (n = 9), exercise (n = 6), smoking cessation (n = 5), and changes in health care organization (n = 2). To evaluate the effect of these studies 93 different outcome measures were used in 16 categories. CONCLUSION: Many interventions directed toward improving somatic health for patients with SMI have been started. These studies did not apply similar evaluations, and did not use uniform outcome measures of the effect of their interventions. Valuable comparisons on effectiveness are therefore almost impossible.

COPD in the working age population: the economic impact on both patients and government.

OBJECTIVES: To explore the full economic impact, on both patients and government, as a result of COPD in the working age population. METHODS: The economic impact of COPD due to medical treatment, impaired productivity and early retirement was assessed in a cross sectional cost analysis of the Dutch COPD population aged 45-64 years. The costing year was 2009 and input parameters were derived from both national data sources and the international COPD uncovered survey. RESULTS: While direct medical costs for COPD patients of working age were relatively low (€ 91 million), the amount of lost productivity (income) due to early retirement (€ 223 million) exceeded over two times their medical costs. In addition, costs for the government were considerable because of lost tax revenues (€ 77 million) and COPD related disability pensions paid (€ 180 million). Apart from lost productivity due to early retirement, costs due to impaired productivity for working COPD patients were € 63 million. CONCLUSIONS: The costs of COPD for both patients of working age as for the government were considerable, making this population a priority for prevention and intervention programs of healthcare providers, employers and government.

Implementing a Managed Entry Agreement Framework in Cyprus.

INTRODUCTION: The aim of this study is to explore the current practice in Cyprus regarding the introduction and reimbursement of innovative pharmaceuticals through Managed Entry Agreements (MEA), assess its operational context, and suggest approaches toward spanning the knowledge gap consequential to these efforts, especially the barriers of a small country context. AREAS COVERED: The recent introduction of a National Health System (NHS), brought about fundamental reforms in Cyprus' Healthcare sector. Among such reforms, of particular interest, has been the introduction of a Managed Entry Agreements (MEA) mechanism. The first preliminary results indicate that despite being a small and unattractive market, Cyprus can apply a substantial MEA program. Concomitantly, it annotates the need to design an operational framework which should include, the definition of important technical parameters, clear demarcation of the scope, cooperation principles ensuring the effective operation of scientific committees, and clear delineation of what 'value' is. Moreover, in the context of the unified healthcare market, budget transfers should be considered, which could alleviate the inordinate budget impact of new products, which nevertheless will cut down on hospital expenditures. Narrative synthesis and health policy analysis-related resources were used. EXPERT OPINION: The implementation of MEA in Cyprus provides an ideal testing ground for innovative reimbursement approaches. This will streamline the country's efforts toward reimbursement of innovation, while concomitantly add to the collective MEA experience.

Clinical pharmacy services for tuberculosis management: a systematic review.

Objective: This study aims to systematically review the content and potential effects of clinical pharmacy services in tuberculosis (TB) care management. Methods: Searches were performed in PubMed, Embase, Cochrane, Scopus, and Web of Science databases following Preferred Reporting Items for Systematic Reviews and Meta-Analysis (PRISMA) guidelines. Study characteristics and outcomes were extracted, and clinical pharmacy service components were characterized using the Descriptive Elements of Pharmacist Intervention Characterization Tool. Results: Twenty articles were included for full-text assessment, of which 10 fulfilled inclusion criteria, comprising 1,168 patients (N = 39 to 258 per study). These articles included five prospective cohort studies, two case-control studies, two quasi-experimental studies, and one cross-sectional study. Intervention foci within clinical pharmacy services were medication adherence (50%), medication safety (40%), education to patients/caregivers regarding needs/beliefs (30%), optimizing medication/therapy effectiveness (30%), emphasizing HRQoL (10%), and drug selections (10%). The three most frequently applied interventions were drug information/patient counseling (80%), adverse drug reaction monitoring (50%), and drug use evaluation (20%). Based on the World Health Organization (WHO) outcome classification, treatment success ranged from 72% to 93%, with higher cure outcomes (53%-86%) than treatment completion (7%-19%). Other outcomes, including isoniazid metabolites, medication counts, sputum conversion, adherence/compliance, knowledge, and quality of life, were better in the intervention group than those in comparator groups, and/or they improved over time. Risk of bias analysis indicated that the included studies were not comparable to a randomized clinical trial. Conclusion: Clinical pharmacy services as single or composite interventions potentially improve TB outcomes, but its evidence is still inconsistent and limited due to the lack of randomized controlled studies using the WHO outcome classification. Systematic review registration: https://www.crd.york.ac.uk/prospero/display_record.php?RecordID=199028, identifier CRD42020199028.

Cost-effectiveness of ravulizumab compared with eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria in the Netherlands.

OBJECTIVES: The aim of this study was to evaluate the cost-effectiveness of ravulizumab compared with eculizumab for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) in the Netherlands. METHODS: A cost-effectiveness analysis was conducted based on a Markov cohort model simulating the course of patients with PNH with clinical symptom(s) indicative of high disease activity, or who are clinically stable after having been treated with eculizumab for at least the past six months. Costs, quality of life, and the incremental cost-effectiveness ratio (ICER) were estimated over a lifetime horizon from a Dutch societal perspective. Several additional analyses were performed, including a one-way sensitivity analysis, a probabilistic sensitivity analysis, and scenario analysis. RESULTS: When compared with eculizumab, ravulizumab saves €266,833 and 1.57 quality adjusted life years (QALYs) are gained, resulting in a dominant ICER. Drug costs account for the majority of the total costs in both intervention groups. Cost savings were driven by the difference in total treatment costs of ravulizumab compared with eculizumab caused by the reduced administration frequency, accounting for 98% of the total cost savings. The QALY gain with ravulizumab is largely attributable to the improved quality of life associated with less frequent infusions and BTH events. At a willingness-to-pay threshold of €20,000/QALY, there is a 76.6% probability that ravulizumab would be cost-effective. CONCLUSIONS: The cost reduction and QALY gain associated with the lower rates of BTH and less frequent administration make ravulizumab a cost-saving and clinically beneficial substitute for eculizumab for adults with PNH in the Netherlands.

Model and Empirical Data-Based Cost-Utility Studies of Continuous Glucose Monitoring in Individuals with Type 1 Diabetes: A Protocol of a Systematic Review on Methodology and Quality.

INTRODUCTION: This review aims to critically appraise differences in methodology and quality of model-based and empirical-data-based cost-utility studies to address key limitations, opportunities, and challenges to inform future cost-utility analyses of continuous glucose monitoring (CGM) in type 1 diabetes. This protocol is registered at PROSPERO (CRD42023391284). METHODS: The review will be conducted in accordance with the PRISMA guideline for systematic reviews. Searches will be conducted in MEDLINE, Embase, Web of Science, Cochrane Library, and Econlit from 2000 to January 2023. Model and empirical data-based studies evaluating the cost-utility of any CGM system in type 1 diabetes will be considered for inclusion. Studies that only report on cost per life year or any other clinical outcome, or reporting only costs or only clinical outcomes studies in type 2 diabetes populations, and studies on bi-hormonal closed loops and do-it-yourself hybrid closed loop devices will be excluded. Two reviewers will independently screen each study for inclusion. Data on the intervention, population, model settings (such as perspective, time horizon), model type and structure, clinical outcomes used to populate the model, validation, and uncertainty will be extracted and qualitatively synthesised. Quality will be assessed using the Philips et al. 2006 (model-based studies) or Consensus Health Economic Criteria (empirical data-based studies) checklists. Model validation will be assessed using the AdViSHE checklist. DISCUSSION: Now that CGM is being used more broadly in practice, critical appraisal of existing cost-utility methodology and quality is important to inform future cost-utility analyses of CGM in type 1 diabetes in various settings.

Modelling the impact of extended vaccination strategies on the epidemiology of pertussis.

The aim of this study was to investigate the optimal pertussis booster vaccination strategy for The Netherlands. A realistic age-structured deterministic model was designed. Assuming a steady-state situation and correcting for underreporting, the model was calibrated using notification data from the period 1996-2000. Several sensitivity analyses were performed to explore the impact of different assumptions for parameters surrounded by uncertainty (e.g. duration of protection after natural infection, underreporting factors, and transmission probabilities). The optimal age of an additional booster dose is in the range of 10-15 years, and implementation of this booster dose will reduce both symptomatic and asymptomatic infections, although the incidence of symptomatic infections in older age groups will increase. The impact of the different assumptions used in the model was in general limited. We conclude that over a wide range of assumptions, an additional booster dose can reduce the incidence of pertussis in the population.

Cost-Effectiveness of Bosutinib for the Treatment of Adult Patients with Chronic Phase Chronic Myeloid Leukemia in the Second-Line Setting.

BACKGROUND: A recently conducted matching-adjusted indirect comparison demonstrated that bosutinib improved progression-free survival, and delayed progression to advanced disease compared with dasatinib and nilotinib in patients with second line (2L) chronic-phase chronic myeloid leukemia (CP-CML). However, the long-term clinical and economic impact of using bosutinib versus dasatinib and nilotinib has not been evaluated. The objective was to determine the cost-effectiveness of bosutinib compared with dasatinib and bosutinib compared with nilotinib from a US payer perspective. METHODS: A cost-effectiveness model was developed using partition survival methods and three health states: progression-free, progression, and death. Trial data (individual patient-level and aggregate-level data) informed the progression-free and overall survival estimates. Costs included drugs and medical resource use. Utility values were obtained from literature. Sensitivity analyses (SAs) included one-way and probabilistic sensitivity analyses (PSAs). RESULTS: Comparing bosutinib versus dasatinib resulted in a gain of 0.4 discounted life years, 1.5 quality-adjusted life years (QALYs), and incremental costs of $28,459 (values in 2020 US dollars), for an incremental cost-effectiveness ratio (ICER) of $19,811/QALY gained. Comparing bosutinib versus nilotinib resulted in a gain of 0.8 discounted life-years, 1.8 QALYs, and incremental costs of $76,563, for an ICER of $41,932/QALY gained. Drug costs and extrapolation distribution type were the main drivers of the model in the one-way SAs. In the PSAs, bosutinib had >90% and >80% probabilities of being cost-effective at a willingness-to-pay threshold of $100,000/QALY versus dasatinib and nilotinib, respectively. CONCLUSIONS: Our results suggest that compared with dasatinib and nilotinib, bosutinib may represent good value for money for treating 2L CP-CML patients.