Patients with antiphospholipid syndrome (APS) present a significant risk of thrombotic events or morbidity during pregnancy, associated with the presence of persistently positive antiphospholipid antibodies (aPL). The risk stratification is crucial to adopt primary prophylaxis measures. With this objective, assays for the in vitro detection of anti-domain 1 β2 glycoprotein I (aD1 IgG) antibodies by chemiluminescence immunoassay (CLIA) have been developed.

We conducted a systematic review with meta-analyses on the effectiveness of incorporating aD1 IgG detection by CLIA for the identification of patients with APS at high risk of thrombosis or morbidity during pregnancy. A cost analysis was also conducted using a decision tree model from the perspective of the Spanish National Health System and a time horizon of three months. We ran extensive sensitivity analyses. Twelve diagnostic performance studies with a total of 3,570 patients were selected.

The sensitivity and specificity of aD1 IgG added to the conventional aPL criteria for obstetric and thrombotic manifestations ranged from 15 to 16 percent, and from 94 to 97 percent, respectively. The sensitivity of the detection of aD1 IgG in isolation for thrombotic and obstetric manifestations was 29 and 48 percent, and specificity was 60 percent, respectively. The GRADE quality of evidence was very low due to the risk of bias and indirectness issues. The incorporation of the aD1 IgG detection involves a higher cost per patient than the usual clinical practice, with an incremental difference of EUR24.04 (USD26.01).

Despite its potential, the incorporation of the aD1 IgG detection by CLIA as an additional test to the determination of classical aPL for the identification of patients with APS at high thrombotic or obstetric risk may not be feasible due to the very low available evidence on diagnostic performance and increased costs.

The objective of this assessment was to assess diagnostic effectiveness of phosphorylated isoforms of the tau protein (p-tau) in blood samples (plasma and/or serum) in Alzheimer’s disease diagnosis compared to clinical criteria, cerebrospinal fluid (CSF) biomarkers, positron emission tomography (PET), or post-mortem pathology imaging.

A systematic review of MEDLINE, Cochrane, and Embase was undertaken from 1 January 2000 to 1 September 2022. QUADAS-2 was used to assess the quality of the studies. The selection and quality assessment was carried out independently by two reviewers.

A total of 1,811 references were identified after eliminating duplicates, selecting five: two studies evaluated p-tau181, two others evaluated p-tau217, and one evaluated both isoforms. The studies provided information on diagnostic accuracy using ROC curves. However, none of the studies provided information on sensitivity and/or specificity. The ROC curve values ranged from 83.3 percent to 99.4 percent for p-tau181 and 84 percent to 94 percent for p-tau217. Information was presented in an unclear and heterogeneous way, so our PICO (population, intervention, comparator, outcome) questions were left unanswered. The heterogeneity was due to factors such as the type of isoform, the laboratory platform, and the classification criteria considered.

Despite technology presenting as promising in several studies, the available evidence did not provide high-quality information about its diagnostic accuracy. Diagnostic technologies are often launched and marketed without enough high-quality evidence. The generation of evidence in diagnostic studies is often based on designs that do not provide adequate health technology assessment information.

In the field of laboratory medicine, the evolution of knowledge and the innovation of technologies are the basis of analytic and diagnostic progress, leading to the development of new solutions based on innovative technologies. However, these advances must be accompanied by evidence of appropriateness, diagnostic effectiveness, and efficiency of organizational aspects, considering the impact of the test on patient outcomes.

This study is an exemplificative case of the application of health technology assessment (HTA), exploiting the EUnetHTA core model, on two analyzers able to determine the glycated hemoglobin (hemoglobin A1c, HbA1c), the Capillarys 2 Flex Piercing analyzer (by Sebia, Lisses, France), and the HLC-723G11 analyzer (by Tosoh Corporation, Shunan, Yamaguchi, Japan) in the Laboratory Medicine Service of the IRCCS San Raffaele Hospital (Milan, Italy).

The nine domains of the EUnetHTA model are assessed, comparing the two technologies of interest and highlighting their pros and cons. As described below, the main aspect to be considered is the impact of the testing results on the clinical effectiveness evidence, due to the two different detection methods while both analyzers respond equally to the clinical and organizational needs of the San Raffaele laboratory.

This study demonstrates how HTA can aid decision-makers in evaluating health technologies to achieve specific objectives of diagnosis, treatment, and prevention.

The evidence synthesis developed to inform decision-making on the use of cytoreductive surgery (CRS) and heated intraperitoneal chemotherapy (HIPEC) for pediatric peritoneal carcinomatosis showed that currently available evidence is of very low quality. As new evidence could arise within the following months, we adopted a rigorous living evidence synthesis (LES) approach to provide a timely update and favor decision-making based on actual evidence.

This LES started with a baseline synthesis about the effects of CRS and HIPEC on pediatric peritoneal carcinomatosis. On 31 August 2023, we set up the evidence monitoring for up to 12 months. Following the Living Evidence to Inform Health Decisions (LE-IHD) framework, we planned and developed the evidence monitoring, supported by technological enablers. We searched for ongoing studies in trial registries every three months. New eligible studies were assessed following a systematic and reproducible process to decide on their incorporation in the evidence summary. This process was periodically reviewed to determine the continuation/withdrawal of the living mode.

The baseline synthesis identified one systematic review suggesting that CRS and HIPEC could increase overall survival in pediatric peritoneal carcinomatosis (very low-quality evidence), but no comparative data could be obtained against usual care. To date, the evidence monitoring has not identified new relevant studies on the impact of CRS and HIPEC in overall and disease-free survival, morbidity, or quality of life in pediatric peritoneal carcinomatosis. At the time of the conference, we will report on nine months of monitoring and regular updates including key messages on any changes in the evidence synthesis conclusions.

For HTA reports based on very low-quality evidence (uncertain results), the LE approach allows for timely updating of conclusions, adding value in decision-making. The LE-IHD framework facilitates HTA developers’ tasks for planning and conducting LE synthesis to inform health decisions.

Herpes zoster (HZ), also known as shingles, is characterized by a vesicular skin rash, often associated with acute pain and itching. The safety profile of the recombinant zoster vaccine (RZV) in adults aged 50 years and older and in adults aged 18 and older who are at increased risk of HZ was assessed in this systematic review.

A comprehensive electronic search was performed in Embase, MEDLINE, the Cochrane Library, and clinical trials registries. Searches were limited to the period from 2008 to July 2023. Article screening and data extraction were carried out by two independent reviewers. Risk of bias was assessed using the Cochrane revised Risk of Bias 2 (RoB2) tool for randomized controlled trials (RCTs). The Risk of Bias in Non-Randomized Studies of Interventions (ROBINS-I) tool was used to assess the quality of non-randomized studies. An adapted version of the Newcastle–Ottawa Scale was used for the appraisal of quality of non-comparative studies.

Eighteen RCTs, four observational cohort studies, seven single-arm trials, and 11 single-arm observational studies were identified. Compared with placebo, solicited local (RZV: 74.1 to 84.0%; placebo: 7.9 to 11.9%) and systemic reactions (RZV: 53.0 to 66.1%; placebo: 6 to 11.4%) were more common in the vaccinated cohorts. Reactions were generally transient and mild to moderate in intensity. The most frequent reactions reported were pain at the reaction site, fatigue, and myalgia. The incidence of potential immune-mediated diseases (pIMDS), serious adverse events (SAEs), and fatalities was similar in vaccine and placebo groups. No SAEs, pIMDs, or deaths were reported as vaccine related.

The available data on RZV shows that while local and systemic adverse events are common with RZV, these are typically transient, and SAEs are uncommon in both the general population and those at increased risk of HZ.

While systematic reviews (SRs) are regarded as the gold standard in healthcare evidence reviewing (and a requirement of many health technology assessments [(HTAs)]), other types of review also play an important role throughout a product’s lifecycle. Drawing on more than thirty years’ experience in conducting reviews, we present key points to consider when deciding which review type might be required.

SRs are recommended when a comprehensive search and synthesis approach is required, for example HTAs. They have highly structured methods, emphasizing bias minimization, transparency, and replicability. “Rapid,” “pragmatic,” or “targeted” reviews are increasingly popular due to their accelerated timelines and reduced costs, with methodological shortcuts possible at various stages. Scoping reviews explore what is known about a topic and typically have a broad research question. “Reviews of reviews” or “overviews” identify existing SRs on an established topic. Finally, “living reviews” follow the same process as an SR or rapid review but incorporate new evidence on a continual or regular basis.

Rapid reviews may be appropriate when flexibility exists regarding the scope and review methods. Any limitations due to methodological shortcuts must be acknowledged in a transparent manner. Scoping reviews are useful for pioneering research ahead of an SR, or early in a product’s development phase, when an overall understanding of the evidence base is required. Reviews of reviews are particularly useful when the size of the primary study literature means that a review of primary studies would be unfeasible. Living reviews are best suited to topics where the evidence base is changing rapidly, or the best information is needed quickly.

When considering conducting or commissioning a review, organizations should consider the intended audience for the review, the resources, time, and budget available, and the size of the existing literature. Although SRs remain the gold standard, a rapid review, scoping review, or review of reviews may offer a more suitable way to approach a given research question.

China has a high incidence of birth defects. Tandem mass spectrometry (MS/MS) screening enables rapid detection of multiple inherited metabolic disorders and has been widely promoted globally. This study aimed to conduct a budget impact analysis of replacing phenylketonuria screening with MS/MS by calculating the financial impact of reimbursing the costs of MS/MS screening.

An Excel-based budget impact analysis model for MS/MS screening was developed. The number of newborns in Shanghai from 2024 to 2026 was estimated using the birth rate trend among the permanent population of Shanghai over the past decade. By integrating clinical screening data, along with the corresponding screening costs and diagnostic fees for the gold standard test, the financial impact of replacing phenylketonuria screening with MS/MS screening was calculated. The screening data for this study was extracted from a tertiary hospital in Shanghai. Demographic data were obtained from statistical websites, while cost data were derived from literature and a tertiary hospital in Shanghai.

The fiscal expenditures for phenylketonuria screening were CNY1.75 million (USD0.25 million), CNY1.65 million (USD0.23 million), and CNY1.56 million (USD0.22 million) for 2024, 2025, and 2026, respectively. In contrast, the corresponding fiscal expenditures for MS/MS were CNY25.23 million (USD3.54 million), CNY23.78 million (USD3.33 million), and CNY22.41 million (USD3.14 million). The additional fiscal expenditure for MS/MS, compared with phenylketonuria screening, was CNY23.48 million (USD3.29 million), CNY22.13 million (USD3.10 million), and CNY20.85 million (USD2.92 million), showing a yearly decreasing trend.

The financial impact of MS/MS screening was controllable. It was recommended that the cost of MS/MS screening in Shanghai be covered by government funding. The promotion of newborn screening using MS/MS deserves priority consideration and publicity in Shanghai, China.

The decision-making process for incorporating technologies for ultrarare diseases (URD) has been a challenge for health technology assessment agencies worldwide. These challenges have been presented in debates about the budget impact of incorporating technologies for URD. This is an important issue because there are other dimensions of the economic and social impact of URD that require consideration.

Data were extracted from National Committee for Health Technology Incorporation (CONITEC) reports (2012 to 2022) on technologies for the treatment of URD in Brazil. Diseases were classified using an epidemiological criterion or Orphanet consultation (prevalence ≤1 per 50,000 inhabitants). Variables included eligible patient count, population estimation method, incremental impact values for one and five years, and diffusion rate in the first and fifth year. Univariate logistic regression was used to adjust the relationship between the budget impact analysis and the final recommendation, considering factors associated with incorporation in univariate regression and p-values less than 0.10 in a multivariate regression.

Among 53 reports, 48 percent exclusively employed the epidemiological approach for incremental impact assessment population estimation, rising to 69.5 percent when combined with measured demand. Population data were nearly evenly sourced from national and international platforms, with the UK, the USA, and multicenter studies being the most cited internationally. Notable differences were found between favorable and unfavorable CONITEC recommendations, with lower values being associated with incorporation. Market share diffusion rates favored the option of 100 percent diffusion in both the first year and the cumulative five years. The analysis highlighted the influence of demand characteristics and technology type on the budget impact value over one and five years.

The study found that budget impact data significantly influenced the final recommendation for technology incorporation, indicating a criterion favoring technologies with a lower budget impact. However, requester characteristics and technology type also played a role in the decision-making process, suggesting that additional factors influence recommendations.

The Department of Health in Ireland published a review of expenditure on state subsidized medicines in August 2021. Detailed analysis indicated exponential growth of expenditure on cancer drugs administered in the community. However, expenditure by drug group across all state subsidized schemes, including hospitals, was not explored.

Using national reimbursement claims data, total medicines expenditure on community drug schemes (CDS) was analyzed annually for the years 2018 to 2022. The total drug expenditure stratified by anatomical therapeutic class (ATC) code was calculated. Expenditure on cancer drugs (ATC code L) between 2018 and 2022, including hospital data, was further explored. Cancer drugs with the highest expenditure were identified, and trends in their expenditure were analyzed. Dates of European regulatory approval, completion of health-technology assessments by the National Centre for Pharmacoeconomics in Ireland, and reimbursement by the Health Service Executive for the identified cancer drugs were collected.

The total expenditure on drugs on CDS rose from EUR1.74 billion (EUR1.61 billion excluding value-added tax [VAT]) in 2018 to EUR2.2 billion (EUR2.03 billion excluding VAT) in 2022. Expenditure on antineoplastic and immunomodulatory agents (ATC code L) rose from 34 percent in 2018 to 38 percent in 2022. The cancer drugs with the highest cumulative expenditure on hospital schemes were pembrolizumab (EUR98.41 million excluding VAT), nivolumab (EUR75.15 million), daratumumab (EUR54.05 million), and trastuzumab (EUR41.48 million). All the aforementioned drugs demonstrated year-on-year increases in annual expenditure apart from nivolumab. Expenditure on pembrolizumab increased by 48 percent in 2022, compared with 2021.

Expenditure on state subsidized medicines is increasing annually, although confidential discounts may reduce budget impact. Increasing expenditure may be attributed to the expansion of existing indications and increasing patient volume. Expenditure on monoclonal antibodies is substantially larger than expenditure on other drugs. Pembrolizumab was reimbursed for six additional indications in 2021, contributing to the sharp increase in expenditure between 2021 and 2022.

Economic evaluation using decision analytical models (DAMs) plays a limited role in shaping healthcare resource optimization and reimbursement decisions in the Middle East. This review aimed to systematically examine economic evaluation studies focusing on DAMs of medicines in the Middle East, defining methodological characteristics and appraising the quality of the identified models.

Six databases were searched (MEDLINE, Embase, EconLit, Web of Science, the Global Health Cost-Effectiveness Analysis Registry, and the Global Index Medicus) from 1998 to September 2023 to identify published DAMs of medicines in the Middle East. Studies meeting the inclusion criteria—full economic evaluations of medicines using a model-based method in the Middle East—were included. Data were extracted and tabulated to include study characteristics and methodological specifications. The results were analyzed narratively. The Philips checklist was used to assess the quality of the studies.

Sixty-two DAM studies of medicines were identified from nine Middle Eastern countries, the majority of which (76%) were conducted in Iran, Turkey, and Saudi Arabia. The cost effectiveness of medications for non-communicable diseases was explored in 70 percent of the models. Cost-effectiveness thresholds based on gross domestic product were commonly used. International sources provided data on intervention effectiveness and health outcomes, while national sources were mainly used for the costs of resource use. Most models incorporated an assessment of parameter uncertainty, whereas other types of uncertainty were not explored. Studies from high-income countries were generally of higher quality than those from middle-income countries.

The number of published DAMs was low considering the available medicines and disease burden. Key aspects of high quality DAMs regarding model structure, input sources, and uncertainty assessment were not consistently fulfilled. Recommendations for future studies and policies included strengthening existing health economic capacities, establishing country-specific health technology assessment systems, and initiating collaborations to generate national cost and outcome data.

Deployment of an electronic automated advisory vital signs monitoring and notification system to signal clinical deterioration is associated with significant improvement in clinical outcomes. This study aimed to estimate the incremental cost per quality-adjusted life-year (QALY) gained with an electronic automated advisory notification system, compared with standard care.

A decision analytic model was developed to estimate the cost effectiveness of an electronic automated advisory notification system, compared with standard care, in adults admitted to a district general hospital. Analyses considered the following: (i) cost effectiveness (cost/event avoided) based on a before-and-after study (n=3,787) that recorded rates of acute myocardial infarction, pulmonary embolism, acute pulmonary edema, respiratory failure, stroke, severe sepsis, acute renal failure, cardiopulmonary arrest, admission to the intensive care unit, and death; and (ii) the cost utility (cost per QALY) over a lifetime horizon extrapolated using published data. The analysis was conducted from the perspective of the National Health Services (NHS) in the UK.

The automated notification system was more effective (2.7 fewer events per 100 patients) and provided cost savings of −GBP12.17 [−EUR14.07] per patient admission (95% CI: −GBP182.07 [−EUR211.20], GBP154.80 [EUR179.57]). The automated notification system was dominant over a lifetime horizon, demonstrating a positive incremental QALY gain (0.0287 QALYs, equivalent to approximately 10 days of perfect health) and a cost saving of −GBP55.35 (−EUR64.02). At a threshold of GBP20,000 per QALY (EUR23,126), the probability of automated monitoring being cost effective in the NHS was 0.81. The increased use of cableless sensors may reduce cost-savings, but the intervention remained cost effective at 100 percent usage (incremental cost-effectiveness ratio GBP3,107 per QALY [EUR3,594 per QALY]).

An automated notification system for adult patients admitted to general wards appears to be a cost-effective strategy in the NHS. The analysis suggests that adopting this technology could be good use of scarce resources. The impact of automated monitoring solutions on staffing warrants further exploration and may show additional value in adopting such technology.

The effectiveness and cost saving advantages of colorectal cancer (CRC) screening have gained widespread scientific consensus. However, the rising incidence of early-onset CRC has challenged Germany’s current screening program, which focuses on individuals aged 50 years or older. This study evaluated the potential cost effectiveness of initiating CRC screening in Germany at the age of 45 years.

The cost-effectiveness analysis utilized a validated discrete-event-simulation model, DECAS, which incorporates both adenomatous and serrated polyp pathways in CRC development. This model has been validated using German CRC epidemiological data and simulates the effects of screening interventions. It was used to compare four new CRC screening strategies starting at age 45 years (10-yearly colonoscopy, annual or biennial fecal immunochemical testing [FIT], or both) with the current screening strategy starting at age 50 years. The simulation, assuming perfect adherence, included a cohort of 100,000 individuals with an average CRC risk from age 20 to 90 years or death, applying a three percent discount with costs in 2023 Euros.

The model outcomes included quality-adjusted life-years (QALYs) gained and total incremental costs, considering both CRC treatment and screening costs. Initiating 10-yearly colonoscopy only or FIT plus colonoscopy strategies at age 45 years yielded incremental gains of seven to 28 QALYs, with incremental costs of EUR28,360 to EUR71,759 per 1,000 individuals, compared with the current strategy. The incremental cost-effectiveness ratios varied between EUR1,029 and EUR9,763 per QALY gained. The FIT-only strategy was dominated by the current screening strategy. These findings remained consistent throughout the probabilistic sensitivity analyses.

The cost-effectiveness findings support initiating CRC screening at age 45 years with either colonoscopy alone or colonoscopy plus FIT, demonstrating substantial gains in QALYs and a modest increase in costs. Our findings emphasize the importance of implementing CRC screening five years earlier than the current practice to achieve more significant health and economic benefits.

There is a need to consider whether treatments included in essential medicine lists, standard treatment guidelines, and health benefits packages are cost effective, improve financial sustainability, and increase equitable access to health care. This assessment evaluated the cost effectiveness of selected antidiabetic medicines for inclusion on Ghana’s Essential Medicines List and updated standard treatment guidelines, and reimbursement by the National Health Insurance Authority.

This study was produced in line with the broad steps of the Ghana health technology assessment (HTA) process guideline using an adaptive HTA (aHTA) approach and following the process used by the National Cancer Grid of India. High quality HTA evidence was sourced from four HTA agencies based on the population (children aged two years or older and adults older than 18 years using antidiabetics), intervention, comparator, and outcomes framework using an adaptability checklist developed by the researchers. A price benchmarking analysis was conducted to generate context relevant evidence on medicine prices in terms of local value for money.

The study found that all medicines evaluated (sitagliptin, vildagliptin, saxagliptin, insulin detemir, insulin degludec, insulin glargine, insulin glusiline, insulin aspart, and insulin lispro) were efficacious. The price benchmark analysis showed that insulin detemir, glargine, and degludec had higher price ratios than their comparators, and an annual drug cost per patient that was approximately two to four times higher. Insulin lispro, aspart, and glulisine had price ratios of 0.22 to 0.44 and an estimated annual cost of GHS1,894 to GHS3,552 (USD163.3 to USD306.2), which was two to five times higher per patient than the comparators. The cost of saxagliptin and vildagliptin were four and three times lower than those in the benchmark country (the UK).

The study revealed that all medicines included are efficacious and potentially cost effective. The price benchmark analysis showed that, except for gliclazide 80 mg, Ghana is paying less for antidiabetic medications than the UK. Cost effectiveness may not be a sufficient basis to include or exclude medicines for reimbursement because they have a potentially significant budget impact for the payer.

Hemophilia B (HB) is a rare bleeding disorder caused by clotting factor IX (FIX) deficiency. Regular FIX replacement (prophylaxis) is the mainstream treatment for preventing bleeding in people with HB. Etranacogene dezaparvovec, also known as AMT-061, is a new gene therapy approved in many countries for people with HB. This study evaluated the cost effectiveness of conventional FIX prophylaxis compared with AMT-061 for people with HB.

A cost-effectiveness decision tree was constructed based on current literature and national guidelines on HB treatment. The model was based on a Brazilian Ministry of Health perspective, assumed that people with severe HB were 18 years or older, and used a time horizon of three years. The Brazilian Ministry of Health distributes plasma-derived FIX (pdFIX) for prophylaxis. The cost of pdFIX was obtained from data on public purchases and AMT-061 cost was based on the price of a currently approved gene therapy in Brazil. Sensitivity analyses were performed to assess uncertainty and to identify which variables most affected the incremental costs.

The cost of prophylaxis with pdFIX for three years was BRL233,309 (USD41,236) with an annualized bleeding rate (ABR) of 10.95, while the cost of gene therapy for three years was BRL10,379,041 (USD1,834,466) with an ABR of 2.86. This resulted in an incremental cost-effectiveness ratio (ICER) of BRL1,253,674 (USD221,583) per bleeding episode. Compared with pdFIX, AMT-061 was not cost effective since there was a threshold of BRL30,000 (USD5,302) per bleeding episode avoided. In the deterministic sensitivity analysis, treatment time had a major impact on the ICER, followed by AMT-061 cost.

In this model, AMT-061 was not cost effective compared with pdFIX prophylaxis. In the long term or with a lower AMT-061 cost, gene therapy may be cost effective. AMT-061 demonstrated better clinical results, but its economic viability must be considered in terms of access and sustainability in the Brazilian public healthcare system.

Inclisiran is approved in Singapore as an add-on to maximally tolerated statins for the treatment of primary hypercholesterolemia or mixed dyslipidemia. This study evaluated the cost effectiveness of inclisiran compared with standard care (SC), evolocumab, or alirocumab to treat primary hypercholesterolemia and mixed dyslipidemia.

A lifetime Markov model was used to evaluate the cost effectiveness of inclisiran as an add-on to SC (inclisiran+SC) versus SC alone, evolocumab+SC, or alirocumab+SC in four subpopulations: atherosclerotic cardiovascular disease (ASCVD), secondary prevention of heterozygous familial hypercholesterolemia (HeFH), primary prevention of HeFH, and primary prevention in patients with an elevated risk of ASCVD Baseline cardiovascular event risks were estimated from databases and published literature from the Netherlands and the UK. Efficacy data were obtained from the ORION trials and other comparative trials. Costs were obtained from public healthcare institutions and local publications. A willingness-to-pay (WTP) threshold of SGD45,000 (USD33,280) per quality-adjusted life-year (QALY) was selected, and a one-way deterministic sensitivity analysis (DSA) was performed.

Inclisiran+SC resulted in higher QALYs and higher total costs than SC alone in all four subpopulations, with incremental cost-effectiveness ratios (ICERs) ranging from SGD34,654 to SGD163,158 (USD25,630 to USD120,673) per QALY gained. At the selected WTP threshold, inclisiran+SC was cost effective, compared with SC, in patients with ASCVD and for secondary prevention of HeFH. Compared with evolocumab+SC and alirocumab+SC, inclisiran+SC achieved higher total QALYs at a lower total cost in all four subpopulations. The ICER was most sensitive to the price and efficacy of inclisiran and the rate ratios translating reductions in low-density lipoprotein cholesterol levels to the risk of cardiovascular death.

Inclisiran+SC resulted in greater QALYs and higher costs, compared with SC alone, and higher QALYs at lower costs, compared with evolocumab+SC and alirocumab+SC, in adults with primary hypercholesterolemia and mixed dyslipidemia. At the selected WTP threshold, inclisiran+SC was cost effective, compared with SC alone, in patients with ASCVD and for secondary prevention of HeFH. These findings can help inform future funding decisions in Singapore.

Non-small cell lung cancer (NSCLC) is the most prevalent malignant tumor in China. This study aimed to compare the cost effectiveness of combined nivolumab and ipilimumab with chemotherapy as a first-line treatment for advanced NSCLC. The findings will contribute to the economic evidence for making clinical and health policy decisions.

Taking a healthcare system perspective, this study used a partitioned survival analysis model to simulate the disease trajectory of advanced NSCLC during first-line treatment over a model cycle of three weeks. The simulation extended over a span of 12 years. A five percent discount was incorporated for both costs and health outcomes. Published clinical efficacy and cost data were extracted from the CheckMate 9LA study (NCT03215706) and drug pricing information was gathered from the YaoZhi website. Utility values were derived from 13 tertiary hospitals in five provinces of China. Base case and sensitivity analyses were also conducted.

The combination of nivolumab and ipilimumab resulted in a lifetime cost of CNY850,068 (USD119,127) and 1.796 quality-adjusted life-years (QALYs), whereas chemotherapy incurred a lifetime cost of CNY276,313 (USD38,722) and a gain of 1.206 QALYs. The incremental cost-effectiveness ratio (ICER) for combination therapy was CNY971,955 (USD136,208) per QALY gained, which was more than three times the average gross domestic product per capita in China (CNY85,698 [USD12,010] in 2022) and indicated that the therapy was not cost effective. Probabilistic sensitivity analysis indicated that the likelihood of nivolumab plus ipilimumab being cost effective, compared with chemotherapy, was 0.02.

Nivolumab plus ipilimumab demonstrated enhanced health outcomes for patients with advanced NSCLC, compared with standard chemotherapy, but the ICER exceeded the acceptable threshold, suggesting that the treatment is not cost effective.

Antimicrobial resistance is an important public health problem with a strong epidemiological, economic, and social impact. The objective of this analysis was to compare the cost effectiveness of imipenem-cilastatin-relebactam (IMI/CIL/REL) therapy with colistin-imipenem for the treatment of hospitalized patients with Gram-negative bacterial infection caused by imipenem-resistant pathogens.

The population comprised patients with hospital-acquired bacterial pneumonia or ventilator-associated bacterial pneumonia that was complicated with an intra-abdominal or urinary tract infection caused by carbapenem-resistant Gram-negative pathogens. The model started with a short-term decision tree describing possible treatment routes and outcomes for patients during hospitalization. After treatment, patients were classified as cured, not cured, or dead. Patients who had not responded to the initial treatment received another line of therapy. Successfully treated patients were entered into the long-term Markov model, which captured follow-up costs and health-related quality of life over their lifetimes.

The analysis was conducted on a hypothetical cohort of 1,000 patients and demonstrated that IMI/CIL/REL therapy was advantageous in terms of diagnosis and treatment in the short term as well as cost effectiveness. In fact, IMI/CIL/REL therapy was dominant, compared with colistin-imipenem, from the National Health System and the societal perspective, providing an average saving of EUR2,800.15 and EUR3,174.63, respectively, and gains of 4.76 years of life and 4.12 quality-adjusted life-years per patient.

Thanks to its economic and societal value, IMI/CIL/REL therapy represents an investment in health that is lifesaving in critically ill patients and is a valuable public health tool in the fight against antimicrobial resistance.

Although it is reported that robot-assisted gait training (RAGT) with conventional physiotherapy for stroke patients increases the odds of walking independence at the end of the intervention, compared with conventional physiotherapy alone, it is not clear how long the benefit lasts or whether it is cost effective. This research aimed to clarify the duration of benefit of RAGT and its cost effectiveness.

A cost-utility analysis was conducted that compared RAGT plus conventional physiotherapy with conventional physiotherapy alone from a public healthcare payer’s perspective. The population comprised patients with subacute hemiplegic stroke who had a modified Rankin Scale of three to five (severe) and were treated in Japanese hospitals. The time horizon was half a year after admission to a convalescent hospital, since no additional benefit has been proved beyond that point. A decision tree model was used for the analysis. The effect of RAGT on walking independence and the durability of the benefit were estimated based on a literature review of randomized controlled trials (RCTs) and a meta-analysis. Costs and utility values were estimated from the literature.

The literature review identified 14 RCTs. A meta-analysis of RCTs with more than two months’ follow-up after the intervention showed a significantly higher rate of independence in walking for the RAGT group at the end of follow-up (risk ratio 1.52, 95% confidence interval: 1.20, 1.93), whereas there was no significant difference between the groups more than three months after treatment. The incremental quality-adjusted life-years (QALYs) was 0.004 and the incremental cost was -USD287, indicating that RAGT with conventional physiotherapy was dominant. The incremental cost-effectiveness ratio was USD5,509 per QALY when it was assumed that the length of stay was not reduced by achieving early independence in walking.

The results showed excellent cost effectiveness for RAGT plus conventional physiotherapy in patients with subacute severe hemiplegic stroke in the Japanese setting when considering a reference value of USD34,000 (JPY5,000,000) per QALY in the cost-effectiveness evaluation. Although the incremental QALY gains were relatively small, cost savings could be expected from achieving early independence in walking.

Dengue virus is a significant public health threat in Brazil. It is transmitted by Aedes aegypti mosquitoes and causes severe symptoms such as hemorrhagic fever and dengue shock syndrome. This study explored the economic evaluation of Wolbachia mosquito replacement as a promising dengue virus control strategy.

The model considered Wolbachia mosquito replacement in seven Brazilian cities: Belo Horizonte, Campo Grande, Fortaleza, Goiânia, Manaus, Niterói, and São Paulo. A mathematical microsimulation model tracked 23 million residents over 20 years and considered transitions between five health states (susceptible, asymptomatic, ambulatory, hospitalized, and death). The current dengue control strategy and the incorporation of Wolbachia mosquito replacement were analyzed from both the health sector (public and private) and the Unified Health System perspectives. Direct costs included local dengue control program resources, Wolbachia replacement implementation, and care of patients with dengue virus. The primary outcome was disability-adjusted life-years (DALYs) averted. Sensitivity analyses were also performed.

The model projected 1,762,688 dengue cases over 20 years without Wolbachia replacement. Implementing Wolbachia replacement would prevent at least 1,295,566 cases, demonstrating a high benefit in all simulated cities. Except for Manaus and São Paulo, Wolbachia replacement was dominant (lower costs and higher effectiveness) over current control strategies. Nevertheless, estimated incremental cost-effectiveness ratios for Manaus and São Paulo, which ranged from BRL1,747.11 to BRL5,072.21 (USD309.51 to USD898.56), were well below the Brazilian cost-effectiveness threshold of BRL120,000 (USD21,258.50) for neglected diseases. Furthermore, all incremental net monetary benefit values remained positive for both scenarios in all cities—from BRL41.37 to BRL1,852.42 (USD7.33 to USD328.16).

Wolbachia replacement is a highly cost-effective option in the Brazilian context, aligning with previous international and diverse perspective studies. Sensitivity analysis and alternative scenarios confirmed the robustness of the findings.

Cytomegalovirus (CMV) infections are usually mild but can lead to serious consequences in immunocompromised patients, such as those infected with human immunodeficiency virus (HIV). Valganciclovir, which is approved for treating CMV, may be a cost-effective prophylactic for CMV disease in immunocompromised patients. Cost-effectiveness and budget impact analyses were conducted to compare valganciclovir prophylaxis with no prophylaxis in immunosuppressed patients with HIV.

Immunosuppressed patients with HIV who were seropositive for CMV were modeled in a one-year horizon decision tree. Outcomes were occurrence of CMV end-organ disease (EOD)–retinitis and gastroenteritis (85 and 15 percent of cases, respectively)–and survival probability after EOD. Direct medical costs related to prophylaxis (induction and maintenance phases) and treatment of EOD were considered. The budget impact analysis (BIA) used the clinical and cost parameters of the cost-effectiveness analysis to compare scenarios with valganciclovir prophylaxis and no prophylaxis in the public health system over a five-year time horizon. The annual market share adoption rate used was 10 percent. The number of eligible patients was calculated from HIV and acquired immunodeficiency syndrome cases reported in the country and epidemiological estimates.

Valganciclovir prophylaxis had an incremental cost of BRL31,781 (USD6,531) and added 0.69 percent in one-year survival, resulting in an incremental cost-effectiveness ratio (ICER) of BRL4,610,022 (USD947,414) per death avoided in one year. The annual budget impact ranged from BRL35,660,931 (USD7,328,743) in the first year to BRL457,707,796 (USD94,064,365) in the fifth year, totaling BRL1,230,400,576 (USD252,861,870) over five years. In sensitivity analyses, the lack of a maximum treatment time for the maintenance phase with valganciclovir (50 to 365 days per year) resulted in a wide variation in the ICER and BIA results.

Despite being recommended for the treatment of CMV retinitis, the use of valganciclovir in immunocompromised patients with HIV who are seropositive for CMV but have not developed CMV disease has a higher ICER when compared with no prophylaxis. For these patients, appropriate use of standard antiretroviral therapy to improve the immune system may be more appropriate.