Infective endocarditis is a leading cause of morbidity and mortality in children and adolescents with underlying CHD. Appropriate diagnostic workup and management in the inpatient setting can be challenging in this patient population due to the spectrum of disease complexity and the dynamic nature of the field. Therefore, the Paediatric Acute Care Cardiology Collaborative has undertaken the creation of this clinical practice guideline.

A panel of paediatric cardiologists, infectious disease specialists, intensivists, advanced practice practitioners, pharmacists, cardiothoracic surgeons, and a dentist was convened. The literature was systematically reviewed for relevant articles on the management of infective endocarditis in patients with CHD. Using the modified Delphi technique, recommendations were generated and put through iterative Delphi rounds to achieve consensus for inclusion.

Based on 127 articles that met the inclusion criteria, 82 recommendations were generated, 50 of which achieved consensus for inclusion and are included in this guideline. They address risk factors specific to CHD lesion type and prior interventions including implanted material, diagnostic considerations, management strategies, and recommendations on counselling other healthcare providers, patients, and families. Of the 50 consensus recommendations, 36 are strong recommendations, though 20 have low or very low quality of evidence.

A central theme in this guideline is that an individual’s specific CHD lesion and prior interventions must be carefully considered for risk stratification, diagnostic approach, and management. While most are strong recommendations, many are supported by low quality of evidence, emphasising the need for further research in this subject.

Human brucellosis, a neglected zoonotic disease, often presents as a chronic febrile illness with nonspecific symptoms like asthenia and weight loss, causing significant morbidity and socioeconomic impacts. Combination therapy involving two or more drugs has proven superior to monotherapy. This study aimed to update evidence on the efficacy and safety of therapeutic options through network meta-analysis, supporting the development of Clinical Protocols and Therapeutic Guidelines (CPTG) for human brucellosis in Brazil.

A systematic search was conducted in the MEDLINE (PubMed), Embase, CENTRAL, and Virtual Health Library databases by independent reviewers to evaluate therapeutic failure, adverse events, and time to defervescence associated with various treatments. Randomized controlled trials (RCTs) evaluating any pharmacological therapeutic intervention were included, while non-original studies, studies focused on localized forms of the disease, or those with fewer than 10 participants were excluded. The risk of bias was assessed using the revised Cochrane risk-of-bias tool, and data were analyzed using a frequentist network meta-analysis approach with a random-effects model. The certainty of evidence was evaluated using the Confidence in Network Meta-Analysis (CINeMA) framework (PROSPERO CRD42023411952).

A total of 31 RCTs involving 4,167 patients were included. Three evidence networks were identified to evaluate the outcomes of interest. Triple therapy (doxycycline plus streptomycin and hydroxychloroquine) for 42 days (risk ratio [RR] 0.08, 95% CI: 0.01, 0.76) had the lowest risk of therapeutic failure, compared with doxycycline plus streptomycin. Doxycycline plus rifampin had a higher risk of therapeutic failure than did doxycycline plus streptomycin (RR 1.96, 95% CI: 1.27, 3.01). No significant differences were observed among regimens regarding adverse event incidence or time to defervescence. Most studies had a high risk of bias. The results demonstrated very low certainty of evidence.

This review confirmed the efficacy of doxycycline with aminoglycosides for treating human brucellosis. Hydroxychloroquine showed potential for reducing therapeutic failure but requires further study. The findings support Brazil’s CPTG, including the incorporation of gentamicin sulfate with doxycycline, thereby expanding treatment options within the Brazilian Unified Health System.

Abdominal aortic aneurysm (AAA) is an abnormal dilation of the abdominal aorta, often asymptomatic but potentially fatal if ruptured. Introducing population-based ultrasound screening could support earlier detection and surgical repair. This systematic review assessed the cost effectiveness of population-based ultrasound screening for AAA in men versus no screening, and explored factors influencing cost effectiveness, as part of a health technology assessment.

Literature searches were conducted in electronic databases and gray literature sources (through to 23 July 2024). Screening, data extraction, quality appraisal (Consensus Health Economic Criteria list) and assessment of transferability (Professional Society for Health Economics and Outcomes Research questionnaire) were performed in duplicate (outlined in protocol CRD42024501141). Findings were presented with reference to the local decision context (Ireland). To facilitate comparison, incremental cost-effectiveness ratios (ICERs) were converted to 2023 EUR. Cost effectiveness was interpreted using willingness-to-pay (WTP) thresholds of EUR20,000 and EUR45,000 per quality-adjusted life year (QALY).

Of 235 reviewed publications, 20 relevant studies were identified for inclusion and analyzed in detail (17 cost-utility analyses [CUAs] and three cost-effectiveness analyses [CEAs]). Screening was found to be cost effective at a WTP threshold of EUR45,000 per QALY in 16 of 17 CUAs (ICERs: EUR200 to EUR30,600 per QALY). CEAs reported ICERs between EUR7,500 and EUR16,100 per life year gained. In sensitivity analyses, AAA screening became less cost effective with decreasing AAA prevalence. None of the studies were considered transferable to the Irish context.

International evidence suggested that AAA screening in men was cost effective. However, despite the consistency of findings in the evidence base, declining AAA prevalence contributed to uncertainty about the long-term cost effectiveness of AAA screening in men.

The objective of this assessment was to determine the benefit of using a next-generation sequencing gene panel for the clinical management of patients with non-small cell lung cancer. The aim was to assess the diagnostic performance, identify the molecular alterations of interest, and define the role of this technology in the therapeutic care of these patients.

The method used for this assessment were based on: (i) a critical analysis of systematic reviews and meta-analyses and clinical practice guidelines identified by a systematic search based on PICO criteria (Population, Intervention, Comparator, Outcome); (ii) the identification of the level of evidence of the clinical actionability of molecular targets, as set out by the European Society for Medical Oncology scale, and targeted therapies included on the list of reimbursable drugs, or drugs that have compassionate use authorizations; and (iii) stakeholder consultations, as well as public health institutions.

Assessment of the evidence and data demonstrated that next-generation sequencing gene panel testing (EGFR, ALK, ROS1, BRAF, RET, and KRAS): (i) is highly concordant with the comparators (Kappa coefficient of 0.884); (ii) can detect additional targetable molecular alterations (marginal increase in the detection capacity of 2%); (iii) makes better use of the tumor tissue; and (iv) has clinical utility, demonstrated by the benefits provided by targeted therapies.

The next-generation sequencing gene panel (EGFR, ALK, ROS1, BRAF, RET, KRAS) was recommended for patients with locally advanced or metastatic non-small cell lung cancer, following diagnosis or in cases of progression, excluding any emergency situations. The composition of the gene panel may be subject to change, in accordance with favorable assessments of new gene alterations.

Social media listening studies (SMLS) analyze online dialogue from patients, caregivers, and clinicians to gain insights into the lived experience of disease including treatment efficacy and safety, and unmet needs. This analysis aims to understand whether SMLS is a feasible method for generating real-world data that can shape pivotal trial design or supplement trial data for health technology assessment (HTA).

A targeted literature search was conducted on PubMed to identify articles published in English that describe and report the findings of SMLS specifically investigating health conditions and diseases. No limits were placed on date of publication or geographical scope. Information was extracted from these papers and used to map concepts and themes covered in SMLS to key domains of HTA submission dossiers in the UK, France, and Germany, as well as the upcoming joint clinical assessments (JCA). This analysis was used to determine the extent to which SMLS can be used to generate payer-relevant data.

A total of 19 papers were included in this analysis. All studies used a retrospective approach, and the majority (84%) analyzed data from more than one market. The most common markets included were the UK (74%), USA (58%), France, and Germany (53%). Thirty-two percent included only patient conversations; 47 percent included conversations from patients, caregivers, family, and friends; and 21 percent included physicians. The most common themes covered were quality of life (89%), burden of disease or symptomology (79%), and treatment patterns (79%), which align closest to the dossier templates for HAS (French National Authority for Health) and the JCA.

All studies in this analysis included data that would be relevant for HTA submission, and the majority provided data that would speak to multiple payer concerns. While there are no published guidelines for leveraging SMLS for HTA, this analysis indicated that this methodology could potentially be used alongside trial data to highlight the patient experience within reimbursement submissions.

Patient and citizen perspectives are a cornerstone of health technology assessments (HTA), ensuring transparency and alignment with societal needs. This study examined the role of public opinion on the evaluation of button feeding tubes for recommendation in Brazil’s Unified Health System (SUS). The objective was to assess how public opinion shaped the final recommendation ensuring equitable access to this technology.

This was a qualitative review of the contributions received by the Brazilian Ministry of Health during the public consultation process about button feeding tubes in 2021. Data sources included contributions from various stakeholders, which were analyzed for their alignment with the preliminary recommendation and their impact on the final deliberation.

A total of 425 contributions from patients, caregivers, healthcare professionals, and industry representatives were analyzed. All of them supported the recommendation of button feeding tubes for pediatric patients in need of long-term enteral nutrition. Overall, they highlighted the advantages of the technology compared to long gastrostomy tubes such as reduced complications, cost effectiveness, ease of use, and improved quality of life. Their impact on patient dignity and social inclusion was also mentioned. Transparent communication throughout the process fostered trust and encouraged meaningful participation. Stakeholder input significantly influenced decision-making, particularly in prioritizing equitable access and designing implementation strategies.

Patient and citizen involvement proved crucial to the unanimous agreement to recommend the inclusion of button feeding tubes in the SUS. Their perspective on the technology’s potential to improve patient care and resource allocation was an important component to decision-making. This case highlighted the value of public engagement in fostering transparent and equitable HTA processes.

The development and implementation of the health technology assessment process by the Superintendence of Health and Labor Risks (SISALRIL) optimizes the inclusion of technologies in the Basic Health Plan, based on scientific evidence. This process (known as EVTESA) strengthens the social security system in the Dominican Republic by promoting informed decision-making, transparency, and spending efficiency; ensuring access to safe and effective technologies; and addressing historical challenges in the analysis and incorporation of technologies into the healthcare system.

SISALRIL, an autonomous entity created by Law 87-01, evaluates and updates the impact of the Basic Health Plan. Since 2020, it has been part of RedETSA, the Health Technology Assessment Network of the Americas, and leads EVTESA, which was institutionalized in 2022 through Resolution No. 0010-2022. EVTESA ensures evidence-based decisions for including technologies in the Dominican Family Health Insurance, promoting transparency, participation, and financial sustainability, while optimizing resources and improving healthcare quality. EVTESA represents a key methodological framework for evaluating and prioritizing healthcare technologies in the Dominican Republic, transforming the evaluation of technology inclusion and exclusion, and modernizing the historical approach to analyzing benefits for the population.

The implementation of EVTESA has established a structured framework with key documents, such as Resolution No. 0010-2022, which institutionalizes the process, and the methodological manuals for prioritizing and evaluating health technologies. The process includes the reception and verification of requests, evaluation using standardized methodologies, report approval after consensus with medical societies and patients, and decision-making by the National Health Insurance Council regarding the inclusion of technologies in the Dominican Social Security System. The prior analysis for the inclusion of coverage in the plan has strengthened coverage policies, resulting in the allocation of benefits to populations where the greatest clinical impact is achieved and healthcare spending is used more efficiently.

EVTESA marks a pivotal advancement in the Dominican Republic’s social security system by optimizing resources and ensuring access to effective health technologies. This evidence-based, equitable model enhances healthcare quality while promoting sustainability, transparency, and public trust. It also serves as a benchmark for other developing countries, demonstrating how systematic, transparent decision-making can strengthen health systems.