Evidence synthesis is the foundation of health technology assessment (HTA); however, systematic literature reviews (SLRs) are highly resource intensive. While augmentation with artificial intelligence (AI) theoretically offers to make this rigorous process more efficient, it is unclear whether AI-supported SLRs will be widely accepted among HTA bodies. This study’s objective was to evaluate how HTA bodies regard the use of AI-supported SLRs.

A targeted literature review (TLR) (January 2019 to October 2024) was conducted in Embase, MEDLINE, and the gray literature. Search terms included AI, natural language processing, large language models, and machine learning. The TLR informed development of a survey to be fielded to respondents from HTA bodies and questions for qualitative interviews.

The TLR found that most HTA bodies do not address using AI for SLRs. Two that do are the National Institute for Health and Care Excellence (England) and the Institute for Quality and Efficiency in Health Care (Germany). Both suggested that AI can support human efforts across multiple SLR phases. Respondents from Europe and the USA completed the survey and qualitative interviews. Most respondents had some familiarity with using AI in SLRs but said their respective organizations doubted AI’s utility in improving the quality of SLRs. Respondents stated the primary responsibility for AI tool development and validation should not rest with manufacturers.

Most HTA bodies do not address using AI for SLRs, but respondents said that AI might improve SLR production by augmenting (not replacing) human effort. Gaining acceptance for AI use in SLRs for HTA will require multistakeholder engagement to ensure transparency and reliability. HTA bodies will need appropriate infrastructure and legal frameworks so that they can test and use AI software.

Respiratory syncytial virus (RSV) is a leading cause of severe respiratory illness in infants worldwide and is associated with significant morbidity. This study evaluated inequities in RSV-related hospitalizations among Brazilian infants under one year of age. Our objective was to evaluate how socioeconomic disparities impact RSV burden and cost-effectiveness metrics for RSV vaccination strategies.

A hybrid model combining a Markov framework and a decision tree was developed. The coefficient of hospitalization was estimated using the Live Birth Information System (Sistema de Informações sobre Nascidos Vivos) and the Hospital Information System (Sistema de Informações Hospitalares). RSV-related hospitalizations were identified using International Classification of Diseases, 10th Revision codes, and deprivation quintiles were defined by the BrazDep index. The RSV hospitalization risk reduction of 0.30 was extrapolated from the literature for the first year of life. Incremental cost-effectiveness ratios (ICERs) were calculated using Brazil’s cost-effectiveness threshold of USD6,873 (USD1=BRL5.82).

Infants in the most deprived quintile experienced 33-fold higher hospitalization rates compared with the least deprived quintile. At the current vaccine price of USD47.82, the ICER was USD26,934, exceeding the national threshold. Cost effectiveness was only achieved in the most deprived quintile, where the vaccine price threshold was USD48.29. Threshold prices for quintiles two to five were significantly lower: USD12.21, USD6.01, USD4.42, and USD2.15, respectively. Vaccination reduced hospitalization risk proportionally across quintiles, but the burden remained disproportionately high in the most deprived quintile.

At the current price, RSV vaccination was not cost effective in most scenarios in Brazil, with ICERs exceeding the national threshold. Cost effectiveness was achieved only in the most deprived quintile, where the disease burden is highest. To align with Brazil’s threshold, significant price reductions or targeted subsidies would be necessary to ensure equitable vaccine access and public health impact.

Health technology assessment (HTA) bodies published stringent criteria for cost-minimization analyses (CMA), but evidence shows that many appraisals still fail to meet their full requirements: notably, adequately powered trials showing non-inferior outcomes regarding minimal clinically important difference. Here, we report the use of CMA in recommending biological disease-modifying antirheumatic drugs (bDMARDs) in psoriatic arthritis.

A comprehensive review of HTA agency reports was conducted to identify the economic evaluations applied in the appraisal of bDMARDs for psoriatic arthritis. Data were collected from publicly available HTA databases and official websites of globally recognized agencies. The search included appraisals published up to December 2024. Evaluations were categorized based on the economic model used (e.g., CMA, cost effectiveness, cost utility, or cost benefit). Extracted data were analyzed to summarize trends and identify differences in the economic approaches used among agencies.

Cost-effectiveness and cost-utility analyses were the predominant approaches in HTA agency assessments of bDMARDs for psoriatic arthritis. Certolizumab pegol, golimumab, and secukinumab were the most frequently evaluated medications, each with nine evaluations. The Brazilian National National Committee for Health Technology Incorporation (CONITEC) was the only agency that exclusively used CMA in all its evaluations. In contrast, the National Institute for Health and Care Excellence (NICE) did not use this methodology in its assessments.

Our findings highlight the significant variation in economic evaluation methods used by HTA agencies for bDMARDs in psoriatic arthritis. The inappropriateness of superiority trials that fail to reject the null hypothesis for CMA is a key takeaway. While CMA remain relevant due to their simplicity, their use by CONITEC contrasts with broader methodologies preferred by other agencies, particularly NICE.

Horizon scanning (HS) systematically identifies emerging health technologies to support future decision-making. However, HS methods face challenges due to inconsistent terminology and lack of reporting standards. This hinders dissemination and increases research waste. Unlike PRISMA for evidence synthesis, no reporting guidelines exist to support HS. This work proposed a preliminary reporting checklist.

In July 2024, a working group comprising HS analysts, evidence synthesis researchers, and information specialists was established at the Innovation Observatory (IO), the UK’s national HS organization. The group convened three times to define objectives, refine methods, and develop the reporting checklist. In the first meeting, we outlined a prototype by adapting the PRISMA Extension for Scoping Reviews (PRISMA-ScR). We internally validated it on 25 IO HS reports (2017 to 2024). The second meeting assessed the relevance of each checklist item following our internal validation. In the third meeting, additional items were proposed based on the validation’s results, and the group reached consensus on the final checklist.

The final checklist prototype comprised a total of 35 items of which 29 were essential and six optional. It introduced new key elements absent in PRISMA-ScR: “interest holder description” in the introduction, “horizon-scanning scope” in the methods, “technology characteristics” in the results and “political, economic, societal, technological, legal and environmental (PESTLE) interpretation” in the discussion. Additionally, the checklist was tailored to promote transparency, equity, fairness, and sustainability in signal detection processes over those characteristics in evidence synthesis, aligning with the goals of HS. The checklist is available to download in the Open Science Framework.

Our checklist addressed a critical methodological gap, advancing HS research by promoting standardization and transparency of HS methods. By making the draft checklist available, we encourage HS practitioners to pilot it. Feedback is welcomed to contribute to the final checklist. Its adoption and endorsement will improve output visibility, ensuring this meets necessary methodological standards underpinning unbiased healthcare policy decision-making.

The use of real-world data (RWD) for real-world evidence (RWE) is essential for evidence-based decisions but requires robust technical, methodological, and organizational capabilities. We conducted an analysis of the Agència de Qualitat i Avaluació Sanitàries de Catalunya (AQuAS) Health Technology Assessment area to determine its capacity to generate RWE using RWD in Catalonia’s healthcare system, to identify priority areas to strengthen competencies and address emerging demands effectively.

A structured SWOT (Strengths, Weaknesses, Opportunities, Threats) analysis was conducted, evaluating internal (strengths and weaknesses) and external (opportunities and threats) aspects. Data collection involved individual reflections and participatory workshops with the Assessment area’s team involved in RWE generation, focusing on critical areas such as data access, analytical capacities, technological resources, and regulatory environments. Finally, a strategic action plan was developed to implement appropriate measures.

Strengths: Consolidated expertise in data analysis and alliances with programs such as the Data Analytics Program for Research and Innovation in Health (PADRIS) in Catalonia; and availability of technological tools and procedures for RWE generation.

Weaknesses: Dependency on third parties for data access; centralized knowledge in key individuals; training gap in advanced methodologies (emulation of clinical trials and predictive analysis); and gaps in data homogenization and quality.

Opportunities: Potential to establish strategic alliances with other entities and leverage emerging technological advancements (implementing Observational Medical Outcomes Partnership standards).

Threats: Regulatory changes and competition in RWE development.

SWOT analysis identified priority actions to enhance AQuAS’s capacity for RWE generation, including implementing training programs, formalizing strategic collaborations, and developing integrated processes for data management. This approach reinforced AQuAS’s role as a regional and international leader in using RWD for informed healthcare decision-making, highlighting the importance of capacity building and cooperation.

Central precocious puberty (CPP) is a rare condition, commonly characterized by early development of biochemical puberty and physical characteristics before the ages of eight (girls) and nine (boys) years. According to the Brazilian clinical practice guideline (CPG) the treatment of CPP includes gonadotropin-releasing hormone agonists. The objective of this study was to verify implementation of the CPP CPG using real-world drug dispensing data from the Brazilian Unified Health System.

Data from drug dispensing in the Brazilian Unified Health System between 2013 and 2023 were extracted by a platform that creates follow-up cohorts for specific diseases (Open Room for Health Intelligence Situation). Only data for patients with an International Classification of Diseases, 10th Revision category of E22.8 were selected. All records that did not have encrypted user identification or that did not have an approved quantity of medication were excluded. A descriptive analysis was performed using PostgreSQL 24.2 to model the data.

A total of 147,045 individuals with CPP were treated with the recommended drugs in the Unified Health System over the study period. Most patients were female (94%). The mean age of patients at the beginning of treatment was 8.8 years (standard deviation 5.3). The most used drugs were leuprolide (80.2%), triptorelin (17.7%), goserelin (2.0%), and cyproterone acetate (0.2%). Leuprolide and triptorelin, with a biannual frequency of use, were included in the CPG at the end of 2022. Consequently, 1,905 patients used leuprolide (45 mg) in 2023 and 1,131 patients used triptorelin (22.5 mg).

Data from the drug dispensing registry indicated that the CPP CPG is being implemented. Recently, this document was updated to recommend biannual frequency use of leuprolide and triptorelin to improve adherence to treatment. Data from the Open Room for Health Intelligence Situation platform can be an important source of information for evaluating the performance of these technologies.

The preferred regimen for initiating HIV/AIDS treatment, according to the Clinical Protocol and Therapeutic Guidelines (PCDT), is tenofovir, lamivudine, and dolutegravir (TDF/3TC/DTG). Fiocruz, through a productive development partnership (PDP), produces these antiretrovirals, strengthening the national industry. The National Committee for Health Technology Incorporation (CONITEC) evaluated the budgetary impact of incorporating bictegravir, emtricitabine, and tenofovir alafenamide (BIC/FTC/TAF) combination therapy and its effect on the sustainability of the PDP.

The analysis used data from the 2022 HIV Clinical Monitoring Report to identify the eligible population. The unit cost of the current regimen (TDF/3TC/DTG) was obtained from the Health Price Database, while the cost of the new regimen (BIC/FTC/TAF) was extracted from CONITEC Recommendation Report No. 675/2021. Using a market share analysis, a moderate 10 percent annual increase in uptake of BIC/FTC/TAF was projected, reaching 50 percent by the fifth year.

For the budget impact calculation, a total of 245,923 people aged 50 years or older who were living with HIV and/or AIDS and undergoing antiretroviral therapy were considered. The costs with and without the incorporation of the BIC/FTC/TAF regimen were analyzed. The incorporation of the new technology resulted in an estimated budget impact of BRL15,463,638.24 (USD2,693,122.18) over the analyzed period.

The BIC/FTC/TAF regimen is an effective, safe treatment approved by international agencies. It is administered in a daily dose, which simplifies treatment. However, its patent restricts national production, and its use in place of the current regimen could impact PDPs, harming the national industry and the sustainability of the health system. Therefore, incorporations require balancing clinical benefits with innovation and financial impacts.

Neonatal disorders are a significant contributor to years lost to premature mortality and years lived with disability worldwide. Healthcare cost estimates for neonatal inpatient health care and the health conditions that most affect newborns in Brazil remain unknown at the national level. We estimated the inpatient healthcare costs in Brazil and identified the most burdensome health conditions.

This population-based descriptive study was conducted from the perspective of the Brazilian public health system. We used secondary nationwide data from the Hospital Information System. Inpatient healthcare records between 2011 and 2022 for newborns from zero to 27 days of life were included. Inpatient healthcare costs consisted of reimbursements for hospital procedures and daily hospital stays, including neonatal intensive care unit (NICU) admissions. Costs were adjusted for the 2023 inflation rate and presented in USD considering the parity index of 2023. The main health conditions are presented as International Classification of Diseases, 10th Revision categories.

A total of 3,835,128 neonatal hospital admissions accounted for approximately USD6 billion, of which 26.7 percent were for NICU admissions. The ten costliest health condition categories accounted for 75.6 percent of total inpatient healthcare costs. Although the most prevalent neonatal health condition was neonatal jaundice (n=652,314), the category of disorders related to prematurity accounted for most inpatient neonatal care costs at USD1.7 billion (USD1.1 billion for NICU), followed by neonatal respiratory distress syndrome at USD1.6 billion (USD1.2 billion for NICU), and the category of other respiratory disorders at USD316.1 million (USD238.3 million for NICU).

This study revealed the economic burden of neonatal inpatient healthcare in Brazil, driven predominantly by NICU admissions for prematurity and respiratory conditions. The findings highlight the importance of targeted health interventions and policies to improve neonatal outcomes and optimize resource allocation.

Clinical trial registries provide relevant information to address publication bias in systematic reviews. However, monitoring these registries is a complex process. Results are rarely posted in registries, and finding publications in journals requires a laborious search across multiple resources. We aimed to monitor registries of randomized clinical trials, results posted on platforms, and articles published in journals in the context of a rapid review to inform decision-makers.

We searched ClinicalTrials.gov and the World Health Organization International Clinical Trials Registry Platform from April 2020 to November 2024 as part of a systematic search on the use of vitamin D in COVID-19, including the MEDLINE, Embase, Web of Science, and the Cochrane Library databases. We collected data such as the study start date, sample size, principal trial characteristics, population, intervention, control, and main outcome. We reviewed new register monitoring results published in databases or platforms and assessed each report’s update frequency until November 2024.

We found 54 unique register numbers with this different status information: 29 trials were completed, two terminated, four active (one recruiting and three not yet recruiting), two were ongoing, one had recruitment and regulatory approvals pending, 11 had unknown status, one was prematurely ended, and two were withdrawn. Two clinical trials were matched with one study published. In total, 26 trials had results published in journals. However, only two trials posted results on the platform. In the last review, 25 trials remained without results, and related publications were not found.

Searching clinical trial registries as part of a systematic review strategy is a necessary source for addressing publication bias. Monitoring clinical trial registries provides relevant information on the development and length of trials, identifying gaps and failures to assist research. Lack of updated information on registries, and finding accurate and worthy information about trials, can result in a time consuming and sometimes unsuccessful process.

Argentina’s healthcare system is fragmented, comprising public, private, and social security sectors. The guidance for incorporating a new health technology into the healthcare system suggests a single threshold value (0.016% of total health expenditure). This study analyzed the influence of cost heterogeneity on budget impact using the reimbursement of talazoparib for the treatment of metastatic prostate cancer as an example.

A budget impact analysis (BIA) was adapted for the National Institute of Social Services for Retirees and Pensioners and SURGE (union social security sector) perspectives. To be able to estimate the impact of medical costs, other parameters such as population and market share were fixed. Drug costs were estimated using reimbursement values, while the costs of healthcare resources and management of adverse events were obtained from the 3Eff SA cost database (3val), which is based on tariffs of institutions within the subsectors of the Argentine healthcare system.

For direct medical resources (medication, medical consultations, laboratory tests, etc.) associated with metastatic prostate cancer treatment, cost heterogeneity across health sectors was 79 percent. The lowest variability was observed in laboratory tests (18%), while the highest was in medical consultations (310%). The cost of managing adverse events varied by 38 percent across sectors, with the highest variability in managing fatigue (118%) and the lowest in thrombocytopenia (2.4%). As a result, the average annual budget impact varied across sectors by just over 15 percent.

Our results showed a significant heterogeneity in costs among Argentina’s healthcare subsectors. This heterogeneity might lead to different reimbursement decisions based on the BIA results. Variability in costs underscores the need for sector-specific guidelines or thresholds for reimbursement decisions to ensure equitable and efficient resource allocation.

Emerging regions require equitable training processes to implement health technology assessment (HTA) effectively. This work presents the initial adaptive curriculum designed for a professional master’s degree in HTA, tailored to the regional context in Brazil, particularly for areas with evolving infrastructure. The objective was to foster HTA expertise by enhancing methodological skills, technical knowledge, and collaborative abilities among students.

The curricular adaptation was developed through collaboration between students and course coordinators, utilizing a cooperative methodology grounded in the first four stages of the Cooperation Pedagogy framework.

The class consisted of 20 students: 75 percent were women, 60 percent from the Central-West region, 30 percent from the Northeast, and 10 percent from the North. Ten students worked in HTA units in hospitals and two worked in legal units. Students’ topics of interest included vaccination in remote areas, judicialization, post-incorporation assessments, and budget impact analyses. The results of the four stages were: (i) a coexistence contract developed based on students’ expectations; (ii) a “World Café” dynamic that facilitated the creation of “hot questions”; (iii) the curricular adaptation integrating all relevant subjects; and (iv) the affective environment fostering mutual learning and collaboration.

The next steps involve co-creating solutions to the students’ hot questions, which were: (i) Does decision-making overturn an HTA report?; (ii) Is the HTA language understandable to stakeholders?; (iii) How to raise awareness among judges?; (iv) How to stimulate the creation of HTA units in emerging regions?; and (v) What is the future of HTA?

Including drugs in the National Essential Medicines List is a complex process that considers population, budget, and socioeconomic factors. In Ecuador, these decisions rely on efficacy and safety data from existing studies without estimating the real impact and clinical benefit of the drugs. This study proposed a multicriteria model using minimal clinically important difference (MCID) values to evaluate oncologic drugs for inclusion.

Safety and efficacy were assessed using MCID values for primary outcomes, incorporating measures such as overall survival (OS), disease-free survival (DFS), and complete remission, following the Magnitude of Clinical Benefit Scale (v1.1) of the European Society for Medical Oncology. Epidemiological and demographic data were obtained from the National Institute of Statistics and quality of life was evaluated using EuroQol-5D scores. Information on oncologic drugs and regulatory considerations was sourced from authoritative organizations. All parameters were systematically organized and weighted, ensuring the total criteria groups accounted for 100 percent.

Seven criteria groups were established, each containing parameters evaluated on a scale from zero to five, designed to reward or penalize based on assessment goals. The groups were weighted from five to 30 percent to reflect their relative importance or impact on decision-making. The criteria and weights were as follows: (i) disease burden (30%); (ii) efficacy (30%); (iii) safety (15%); (iv) quality of life impact (5%); (v) availability and accessibility (5%); (vi) therapeutic alternatives and international recommendations (10%); and (vii) other important criteria (5%). Consequently, scores were weighted differently across groups.

Accurately estimating the impact of oncologic drugs is critically important for public health. Implementing this multicriteria model will provide a more efficient, objective, and systematic framework for evaluating the inclusion, modification, or exclusion of oncologic drugs with respect to the National Essential Medicines List. This approach enhances decision-making processes and ensures the optimal selection of anti-cancer therapies, ultimately improving patient outcomes and resource allocation in Ecuador.

In Brazil’s Unified Health System (SUS), initial treatment for follicular lymphoma (FL) is generally effective, but many patients relapse and continue treatment with rituximab plus chemotherapy. Lenalidomide plus rituximab (LR) has been shown to improve overall survival (OS) and progression-free survival (PFS) in patients with FL. We assessed the cost utility and budget impact of LR for previously treated patients with FL from the SUS perspective.

A three-state partitioned survival model was built using rituximab OS and PFS curves from the AUGMENT clinical trial, which were extrapolated over a 40-year horizon using parametric distributions and adjusted with hazard ratios (HRs) from the comparison with LR (OS: HR 0.45, 95% confidence interval [CI]: 0.22, 0.92; PFS: HR 0.40, 95% CI: 0.29, 0.56). Curve selection was based on the Akaike and Bayesian information criterion and visual inspection. Utility values were obtained from international literature. Direct medical costs, including the cost of drugs, monitoring, and pre- and post-progression were considered. For the budget impact analysis, eligible patients were estimated based on measured demand, and two different market shares were compared with the reference scenario (without LR).

LR showed an incremental benefit (3.10 quality-adjusted life years [QALYs]) and incremental costs (USD76,718 [BRL187,192]). The incremental cost-effectiveness ratio (ICER) suggested that LR was cost effective (USD24,712 [BRL60,298]/QALY), considering the Brazilian threshold of USD49,180 (BRL120,000) per QALY. In sensitivity analyses, LR remained cost effective in most scenarios except when the OS HR was adjusted to the upper limit of the 95 percent CI. Budget impact analysis showed that listing LR in the SUS would increase the budget by USD12,523,280 (BRL30,557,046) in five years if market share reached 50 percent by the fifth year (approximately 648 to 737 eligible patients per year).

Standard treatment options for relapsed FL are limited. Previously treated individuals with FL may benefit more from LR than from standard treatment with rituximab and chemotherapy. The ICER showed that lenalidomide could be cost effective for the Brazilian health system.

The reallocation of resources to efficacious and safe health technologies (HT) using disinvestment strategies to identify technologies considered to have low health value is relevant to ensure efficient use of resources. The objective of this work was to identify HT disinvestment strategies in Latin American countries and to describe the procedures used.

Participants in the 15th meeting of the Health Technology Assessment Network of the Americas (RedETSA), held in November 2024, were interviewed. Representatives of agencies from 10 Latin American countries (Argentina, Chile, Colombia, Cuba, Dominican Republic, Ecuador, El Salvador, Mexico, Peru, Uruguay) answered the following open questions: (i) Is disinvestment performed in your country?; (ii) Do you have established procedures for disinvestment?; (iii) How do you select HT to be disinvested?; and (iv) What type of HT are remove from the coverage of benefits?

Argentina and Colombia have established procedures and have used them to implement HT disinvestment. Mexico also has procedures, but they have been used rarely. El Salvador does not have explicit procedures, but mechanisms for disinvestment have been used following requests from different stakeholders. The rest of the countries do not have established procedures but have excluded HT due to obsolescence or occurrence of adverse events that were previously unknown. Health technology assessment has been used to remove coverage for obsolete technologies and indications not previously assessed. Most technologies disinvested using health technology assessment were high-priced oncological medications.

In Latin America, very few countries have explicit procedures to identify and disinvest inefficient HT. Since the reallocation of resources is a well-known tool to improve the efficiency of health systems, it is necessary to establish well defined procedures to identify potential technologies for disinvestment and to remove their funding in the different health systems.

Intensive care unit (ICU) hospitalization costs are relevant due to their high impact on the healthcare system. ICU admissions financed by the Brazilian government through the Unified Health System totaled USD2.260 billion in 2023. Given the magnitude of these expenditures, economic evaluations of innovations in ICU care are critical. Because of the limitations of conventional sources of cost data, this study aimed to develop a cost estimation tool for health technology assessments.

Data were extracted from the IMPACTO-MR Project, a collaborative research platform coordinated by Brazilian hospitals, including Hospital Israelita Albert Einstein, partnering with the Ministry of Health and the National Health Surveillance Agency (ANVISA). Patient-level data were collected from 15 Brazilian ICUs from October 2019 to October 2024. Costs were categorized into fixed costs (doctors, nurses, physiotherapists, nursing assistants, administrative assistants, other workers, depreciation, electricity, water, telephone, internet, indirect costs, office supplies) and variable costs (drugs, materials, hemodialysis, blood transfusions, laboratory and imaging tests, medical gases). ICU costs were estimated by identifying consumption patterns using machine learning and considering clinical variables. Costs were adjusted for purchasing power parity and inflation.

ICU costs per patient showed substantial variability. Fixed costs were more related to the structure of the ICU, management practices, and local and regional characteristics, did not vary in the short term, and had a strong correlation with length of stay. Variable costs were more related to the patient characteristics, reflected direct consumption, and varied in the short term. Estimating costs for health technology comparisons was possible with this calculator. Machine learning can be used to identify consumption patterns in ICUs and help estimate costs while taking into account clinical variables.

The IMPACTO-MR Project is conducting several health technology assessments in ICUs in Brazil. With this calculator, it is possible to perform cost-effectiveness analyses in ICUs when cost data are not available.

Economic restrictions can affect breast cancer healthcare attention; therefore, it is essential to identify the main economic factors that impact the health system’s budget. This study identified and quantified monetarily the events generating direct costs associated with the care of HER2+ breast cancer according to stage of the disease in a Colombian healthcare institution.

This retrospective observational study included women over 18 years of age with a diagnosis of HER2+ breast cancer identified through International Classification of Diseases, 10th Revision codes present in medical records between January 2018 and December 2022. Resources were organized into the categories of laboratory tests, medical consultations, procedures, diagnostic imaging, supplies, radiotherapy, and medications. The monetary valuation was carried out using the UPC Sufficiency, RIPS, SOAT, and SISMED prices databases for 2023. A micro-costing technique and a third-payer perspective were employed. The average and total care costs by baseline stage were evaluated.

During the study period, 289 patients with HER2+ breast cancer were identified (age 53 years, standard deviation 11.7) with the following disease stages: in situ (n=2 patients), early (n=68), locally advanced (n=165), metastatic (n=41), and not reported (n=13). The total cost for managing the cohort was USD13,012,354, with the cost for the in situ stage being USD11,953 versus USD7,465,033 for locally advanced. Compared with treating patients in the situ stage, the average treatment cost was 4.8 times higher for early cancer, 7.6 times higher for locally advanced cancer, and 13.8 times for metastatic disease. The costs associated with medications represented 83 percent of the total, followed by procedures with eight percent.

The comprehensive care of patients with HER2+ breast cancer reflects a significant economic burden for the healthcare system, showing an increase in average costs in the advanced stages of the disease. These results highlight the importance of early diagnosis from an economic perspective on resources used for patients with HER2+ breast cancer to guide decision-making and achieve efficient resource allocation.

The rising costs of rare disease therapies pose significant challenges for health technology assessment (HTA) decision-makers and payers, particularly given the limited clinical evidence available at the time of appraisal. This has created a growing need for outcome-based managed entry agreements (OBMEAs) that link post-launch evidence generation to appropriate payment models. This study examined the implementation of OBMEAs in Belgium and offers best practices to optimize their use globally.

A document analysis was conducted of managed entry agreements (MEAs) for all rare disease therapies reimbursed between January 2012 and August 2024. Through a standardized template, OBMEAs were identified and classified based on type of uncertainties included and outcomes to be collected. HTA reports were analyzed to assess the impact of clinical and real-world evidence (RWE) on the reassessment of these therapies at the end of the OBMEA.

The Belgian payer implemented outcome-based managed entry agreements (OBMEAs) for 57 orphan drugs, including five advanced therapy medicinal products. All OBMEAs followed a coverage with evidence development scheme, primarily addressing budget impact (32%) and efficacy (27%). The median duration was 24 months, with some agreements extended or renewed up to five times. Only eight percent of OBMEAs resulted in a definitive listing. RWE was frequently rejected during reassessment due to concerns about validity and incomplete registries. Key recommendations include establishing clearer data collection protocols and improving transparency at multiple levels to enhance the effectiveness of these agreements.

The findings highlight the challenges of implementing outcome-based MEAs in a manner that truly addresses long-term uncertainties. Adopting the best practices outlined in this study could support OBMEAs to fulfill their promise of balancing sustainability with timely patient access to innovative therapies.

Constraints on surgical capacity due to budgetary and workforce shortages necessitate prioritization. Lessons learned from the COVID-19 pandemic emphasize the societal debate around these decisions and stress the need to align decisions with societal preferences. This study examined societal preferences for prioritizing patients with three different conditions—breast cancer, deafness, or knee arthrosis—for scarce surgical capacity.

We conducted a labeled discrete choice experiment among 1,046 members of the Dutch public. Respondents completed 14 choice tasks in which they prioritized patients for surgery, based on condition, age, health-related quality of life (HRQoL) before and after surgery, and waiting time until surgery.

Respondents were more likely to prioritize patients suffering from breast cancer over patients suffering from knee arthrosis or deafness. Respondents were also more likely to prioritize patients with lower levels of HRQoL before surgery, larger surgery-related increases in HRQoL, and longer waiting times until surgery. They were less likely to prioritize patients who were relatively older, although the opposite held true for patients suffering from deafness. Observed preference heterogeneity largely resulted from differences in preference strength, rather than preference direction.

Our results provided insight into societal preferences for prioritizing patients with different conditions for surgery. This insight aids in understanding public outcry that may follow decisions deviating from societal preferences. Aligning prioritization decisions with societal preferences may increase their legitimacy. Further research may examine the relevance of these preferences for physicians and their willingness to be guided by this.

Atrial fibrillation (AF) has a high economic burden for the healthcare system. Due to the emboligenic profile of AF, stroke prevention with oral anticoagulants (OAC) is usually recommended. For patients with AF at risk of bleeding who are contraindicated for OAC, non-pharmacological thromboembolism prevention therapies such as left atrial appendage closure (LAAC) is recommended. We aimed to incorporate the LAAC procedure into the Brazilian Supplementary Health Sector (SHS).

A scientific technical opinion (STO) including a literature review and economic and budget impact analysis was submitted to the National Supplementary Health Agency (ANS). The STO was technically evaluated and a health technology assessment based ANS decision rite was followed. The rite was composed of an applicant defense meeting, publication of an ANS critical report, a preliminary decision, public consultation, a public hearing, presentation of the public consultation results, and a final decision. Applicants could participate in the defense meeting and all stages of public participation.

Arguing insufficient randomized controlled trials specifically addressing the OAC-contraindicated AF population and some concerns regarding the economic modeling, ANS preliminary decision was unfavorable to the incorporation. The ANS full critical report was published and received extensive responses from the applicants in the public consultation, which received 919 contributions. The main points addressed during public debates, including the public hearing were the ethical implications of allocating OAC-contraindicated patients to an OAC treatment arm; the lack of treatment available for this population in the Brazilian SHS; the importance of real-world evidence; the impact of stroke on patients, families, and society; and clarifications on the economic model. After considering all the contributions, the ANS reversed its verdict.

The LAAC procedure has been available in the Brazilian SHS since 1 July 2024. The ANS’ final favorable decision was only possible thanks to the broad space for public debate made available during the ANS decision rite, added to extensive popular engagement throughout the process.

Severe scoliosis affects three percent of the population, with 0.3 percent requiring surgery that often results in significant blood loss. The cell saver technique collects and reinfuses lost blood, potentially reducing the need for transfusions. This study evaluated its clinical outcomes and feasibility in scoliosis surgeries performed between January 2022 and March 2024 in a Brazilian Health Maintenance Organization.

A non-concurrent cohort study analyzed the use of cell savers in scoliosis surgeries performed between January 2022 and March 2024 within a non-profit health assistance organization in Brazil. Patients were divided into two groups: those who underwent intraoperative use of the cell saver technique and those who did not. Data on patient demographics, procedural details, and clinical outcomes were collected from institutional databases. Primary outcomes included the length of hospital stay, the need for blood transfusion, and rates of major complications. Statistical comparisons were performed using chi-square tests and t-tests (statistical significance set at p<0.05).

Among 311 patients, nine received the cell saver technique. The mean age was 14.8 years, with 66 percent being younger than 19 years. In the non-cell saver group (302 patients), 36 percent required blood transfusions (average two units/patient) and the mean hospital stay was 7.52 days. In the cell saver group, 44 percent required transfusions (average 1.25 units/patient), and the mean hospital stay was 6.89 days. The results suggested trends toward reduced transfusion needs, use of fewer blood units, and shorter hospital stays in the cell saver group, although the sample size was limited.

This study identified potential benefits of intraoperative cell saver use in scoliosis surgeries, including reduced transfusion needs, use of fewer blood units, and shorter hospital stays. However, the findings require validation in larger, more diverse samples to establish clinical significance and cost effectiveness in routine practice.