In Singapore, patient involvement has been an integral component of health technology assessment (HTA) since 2022. Processes co-designed with local patient organizations have encouraged meaningful collaboration, reflection, and learning. This presentation discusses the impact of tailored training resources implemented by the Agency for Care Effectiveness (ACE) to build capacity and empower patients and caregivers to contribute to healthcare decision-making.

Surveys were electronically distributed to 27 local patient organizations in 2023 to identify knowledge gaps and the information needs of their members. A comprehensive review of patient resources from overseas HTA agencies and patient groups was undertaken to identify best practices for enhancing capacity building. The insights gathered informed the co-development of two interactive training workshops and a series of self-directed learning modules on HTA. Feedback was gathered from participants at the end of each workshop, and post-training surveys were conducted to assess changes in participants’ understanding of HTA, skill development, attitudes towards patient involvement, and expectations for future training resources.

Thirty-seven patient leaders attended the workshops, which introduced HTA in plain language and role-playing as decision-makers to explore factors considered for funding recommendations. Learning modules were published online and disseminated to all local patient organizations. Survey results showed that the training resources led to marked increases in patients’ self-reported confidence to participate in future HTAs, and in their knowledge of HTA concepts. The resources were viewed as a positive step toward improving the quality of patient input in HTAs, and overcoming local perceptions of tokenistic involvement, by strengthening the value of experiential knowledge over traditional scientific evidence.

Workshops and learning modules tailored specifically to the information needs of patients play an important role in building confidence and skills in HTA. ACE will continue to expand its resources to equip patients and caregivers with the skills required to meaningfully participate in healthcare decision-making. Impact evaluation of training initiatives will drive continuous improvement and ensure they remain relevant to patients’ needs.

Patients with difficult biliary stones often require repeated conventional ERCP procedures or others surgical interventions, leading to complications and unnecessary expenses for the healthcare system. ERCP with cholangioscopy allows direct visualization and use of laser or electrohydraulic lithotripsy, which are not possible without cholangioscopy. This study aimed to evaluate the cost effectiveness of ERCP with cholangioscopy for treating difficult biliary stones.

A decision tree model was developed to estimate the cost effectiveness of ERCP with cholangioscopy for direct visualization. Patients started the model with difficult biliary stones after an initial failed ERCP procedure. In the base scenario, patients underwent a second conventional ERCP procedure. In the proposed scenario, cholangioscopy was used in the same procedure immediately after the first failed ERCP. In the case of technical success with complete removal of the stones, patients exited the model, otherwise surgery was performed. Estimates of success rate, resources used, and costs were obtained from the literature or expert opinion and estimated from the supplementary health sector’s perspective.

ERCP with cholangioscopy used immediately after a failed conventional ERCP in the same procedure was cost-effective and dominant alternative. It increased success rates for removing difficult biliary stones and reduced resource use and complication rates, benefiting the Brazilian supplementary health sector. This approach offered significant advantages over repeated conventional ERCP procedures or surgical interventions.

Reducing the number of repeated attempts at endoscopic extraction of complex gallstones prevented several adverse patient outcomes and reduced healthcare resource utilization, medical costs, safety implications, and delays to more effective treatment.

The BRIGHTE study assessed the efficacy of fostemsavir tromethamine 600 mg with optimized background therapy in adults with multidrug-resistant HIV-1 and virological failure. Participants were divided into a randomized cohort, comparing fostemsavir with placebo, and an observational cohort. After eight days, all randomized participants switched to fostemsavir. This study evaluated the certainty of evidence from the BRIGHTE trial.

The GRADEpro tool was used to assess the quality of evidence. Five studies related to BRIGHTE were included, covering follow-up assessments at eight days (randomized phase) and 48, 96, and 240 weeks (observational phase). The outcomes assessed included changes in viral load, adverse events, virological response and failure, changes in CD4+ T cell count from baseline, mortality, quality of life, and treatment adherence. Differences between the randomized and observational phases were evaluated, focusing on the impact of this transition on evidence quality.

Transition to the observational phase compromised methodological robustness, increasing the risk of bias. In the randomized phase, evidence quality was reduced by the lack of details on the randomization process and uncertainties regarding double blinding. In the observational phase, confounding bias arose mainly due to intervention classification and the absence of an active comparator, hampering the assessment of fostemsavir’s relative efficacy. Intervention status influenced outcomes. Despite these limitations, fostemsavir showed consistent virological responses, indicating potential benefit for highly treatment-experienced individuals. Overall, evidence certainty was rated low for most outcomes, reflecting the combined impact of bias risks and methodological limitations.

Although findings from the BRIGHTE study highlight fostemsavir’s clinical potential, the transition from a randomized controlled trial to an observational study reduced its evidence quality. The lack of comparators in the observational phase limited interpretations. However, a single-arm design is ethically justified for individuals with limited options to ensure access to interventions. Future studies should prioritize hybrid approaches and real-world data to improve clinical applicability.

Health technology assessment (HTA) events are essential for fostering collaboration among stakeholders, enhancing methodologies, and addressing challenges in evidence integration. These events are particularly valuable for HTA centers (HTACs), which play a crucial role in evidence-informed policymaking in Brazil. This report highlights the experiences and outcomes of the first in-person meeting of the Brazilian Coalition for Evidence (CBE).

The CBE is a network comprising over 40 institutions dedicated to evidence-informed social interventions. The meeting was held on 8 to 9 August 2024, at the University of Sorocaba, Brazil, in conjunction with the seventh International Workshop on Rational Use of Medicines and the fourth Worknowledge in Evidence-Informed Policies. The agenda featured thematic panels on integrating evidence ecosystems and the role of qualitative evidence in HTA. The target audience included representatives from academia, public administration, civil society, HTACs, and evidence centers, fostering cross-sectoral dialogue and capacity building.

Approximately 100 participants attended the meeting, sharing insights on HTA’s critical role in advancing evidence-informed policies in Brazil and Latin America. Key sessions included the plenary, which explored the integration of qualitative perspectives into HTA frameworks. Presentations from the Evidence-Informed Policy Network – Americas (EVIPNet Americas), Health Technology Assessment Network of the Americas (RedETSA), and the Brazilian Health Technology Assessment Network (REBRATS) showcased successful regional HTA initiatives, highlighting their impact on local governance and decision-making. Discussions underscored the need to expand HTA applications, addressing social challenges and emphasizing relevance to sustainable development.

The meeting highlighted HTA’s potential to transform political landscapes by connecting science with decision-making. By equipping stakeholders with advanced tools and fostering cross-sectoral collaboration, it laid a foundation for more equitable and effective public policies. These efforts underscore the importance of investment in evidence ecosystems for long-term societal benefits, and highlight the significance of collaborative networks such as CBE, REBRATS, and EVIPNet.

Patient organizations giving input to health technology assessment (HTA) often receive little information on the treatment being assessed. To promote equitable assessment and enhance trust and transparency, patient organizations need clear and accessible information. The use of plain language summaries as part of the HTA process was evaluated to examine how they can help break down barriers and empower patient involvement in HTA.

The experience of piloting the Summary Information for Patient groups template in the HTA process in England was assessed using surveys, interviews, an advisory board, and a short-life working group. These qualitative data were evaluated by the HTAi Patient and Citizen Involvement in HTA Interest Group, including the issues and challenges, lessons learned, and recommendations for future adaptation to support patient involvement.

The pilot tests reported positive feedback on the Summaries by patient organizations, regardless of their level of HTA experience. The Summaries reduced preparation time and increased confidence in providing input. Other stakeholders also appreciated them. In a public consultation, 80 percent of respondents agreed or strongly agreed that “manufacturers should provide a ‘Summary of Information for Patients’ with their evidence submission.” However, HTA bodies highlighted the need to consider additional resources, timelines for incorporating Summaries into existing processes, and the need to review completed templates to mitigate bias. Feedback was analyzed and eight recommendations were proposed for consideration by HTA bodies and other stakeholders.

Pilot tests have indicated a role for plain language summary information to support patient organization input and involvement in the HTA process, although there are issues and challenges that warrant further discussion. Nonetheless, recommendations can help establish best practice for stakeholders wanting to adapt the international Summary Information template and implement the approach to achieve more transparent and equitable assessments.

The Innovation Observatory (IO) uses horizon scanning (HS) for systematic identification of emerging medicines for the health technology assessment (HTA) process of the National Institute for Health and Care Excellence (NICE) in the UK. The IO’s HS methods have evolved with the policy and demands of HTA for medicines. We reported the evolution of HS processes to facilitate NICE’s HTA process.

The remit applied by the IO to identify relevant information has evolved to become inclusive of trials in Australia, Japan, New Zealand, and Singapore to accommodate changes in regulatory procedures in the UK. Novel processes have been introduced to gather information from additional sources since inception of the IO in 2017. Semi-automation has been introduced alongside manual processes to gather, sift, and triangulate intelligence from several sources, to then populate a bespoke internal database called the Medicines Innovation Database (MInD). Additionally, a bespoke live, confidential, and searchable dashboard was developed in 2019 to improve collaboration with NICE.

Initially, information was shared with NICE via an early awareness form, a filtration form, and then a technology briefing (TB). Since 2019, NICE receives monthly TBs along with a live dashboard. TBs are sent to NICE 24 months from estimated approval in the UK, which is the first step in the HTA process. Since inception of the IO in April 2017, over 1,200 TBs have been submitted to NICE to kickstart the HTA process. The live dashboard provides NICE with real-time information on medicines on record being monitored in MInD, allowing better transparency, efficiency, and resource planning.

HS systems need to be systematic and agile, to allow adaptation to the needs of HTA stakeholders for timely decision-making regarding emerging medicines that might reach the market. The IO HS methods are a potential blueprint for constructing a robust and flexible early awareness system using HS to support HTA processes. Progressive additional automation will be supported imminently.

Although Brazil has a public and universal health system, the Unified Health System (SUS), more than 50 million people also have private health insurance. This can generate health inequalities between citizens with and without private health insurance. We aimed to identify potential health inequalities by mapping the differences between drugs covered in the SUS and in the private health sector in Brazil.

We compiled a list of new drugs, including biologics and advanced therapy products approved in Brazil (2010 to 2020) and analyzed drug-indication pairs from their labels. Oncology drugs were excluded. Coverage in the SUS and private health system was assessed through official documents (until 2022). Potential health inequalities were identified when pairs available to private health had no therapeutic alternatives in SUS care pathways.

A total of 206 drug-indication pairs were evaluated: 126 (61%) were not incorporated into either the SUS or private health insurance; 38 (18%) were exclusive to the SUS; 18 (9%) were exclusive to private health insurance; and 24 (12%) were in both. The SUS provided treatments for chronic non-communicable diseases, immune-mediated inflammatory diseases (IMIDs), rare diseases, and infectious diseases, while private health insurance primarily focused on IMIDs, particularly severe forms. Potential health inequalities were identified in four of the 18 pairs covered only by private health insurance, which were related to macular edema, asthma, thromboembolic disease, and anemia. Despite this, the SUS offered broader care pathways than private health insurance in most cases.

Although some potential health inequalities were identified, the SUS incorporated more drugs for a wider variety of diseases than did private health insurance. Guided by the basic principles of universality, integrality, and equity, the SUS offers more comprehensive care than private health insurance and, in most cases, addresses gaps in care left by the private sector.

Aortic valves for transcatheter aortic valve implantation (TAVI) vary in design. Traditional models without a skirt, used at the Amosov National Institute of Cardiovascular Surgery, have notable disadvantages. This study conducted a hospital-based health technology assessment (HB-HTA) to evaluate the feasibility of implementing skirted aortic valves to optimize resource utilization within the facility.

The Amosov National Institute of Cardiovascular Surgery of the National Academy of Medical Sciences of Ukraine (the Institute) conducted this project as part of a national pilot to introduce health technology assessment (HTA) in hospitals. The institute formed a multidisciplinary team of specialists with clearly defined roles and responsibilities. The team relied on Ukrainian methodological guidelines for implementing HTA in hospitals that were approved in 2023 and were derived from recommendations from Adopting Hospital-Based Health Technology Assessment (AdHopHTA) and the Danish Centre for Evaluation and Health Technology Assessment.

The analysis of clinical effectiveness and safety showed that aortic valves with a skirt have advantages over those without a skirt for aortic valve replacement in patients with aortic stenosis. A systematic review of literature from PubMed and the Cochrane Library found reduced rates of paravalvular leakage (10 to 20%) and complications with skirted valves, minimizing the need for corrective interventions and further monitoring. Budget impact analysis revealed that using skirted valves reduced cost overruns and optimized resource use. Transitioning to this model required no structural changes and offered significant strategic and economic benefits.

Considering the clinical efficacy, safety, and economic feasibility of aortic valves with a skirt, their introduction into the Institute’s practice is highly justified. These valves offer the potential to enhance long-term treatment outcomes, reduce the incidence of complications such as paravalvular leakage, and optimize resource utilization. This implementation represents a strategic step toward improving patient care and healthcare efficiency.

A fundamental stage of any evidence synthesis is the description of the study selection process. This description is ideally represented through flowcharts, which should contain the results of the searches, the number of studies eliminated in the initial phase of title and abstract screening, the number of studies excluded in the subsequent phase of full-text review, and the final number of studies included.

This is a descriptive study of the direct_flow program, developed using the ado programming language within Stata software. This program was developed in the context of two projects conducted by the Center of Health Technology Assessment, Hospital Sírio-Libanês (NATS/NEv-HSL), São Paulo, Brazil and various governmental health organizations, including the Ministry of Health, the National Supplementary Health Agency, and the National Council of Justice, as part of the Support Program for Institutional Development of the Unified Health System (PROADI-SUS). The direct_flow program was used in both projects and aims to enhance the efficiency and consistency of evidence summaries by automating flowchart generation.

The direct_flow program operates using pre-programed templates, which can be chosen by the researcher. Eleven templates were created: seven tailored for systematic reviews and four for overviews of systematic reviews. The researcher needs to specify the number of references at each stage of the selection process through standardized commands. The program automatically populates the flowchart boxes and generates high resolution figures. All templates are available in Portuguese and English and can be downloaded via Stata. More details and access to the templates can be found at https://rlpacheco.github.io/direct_flow/.

Drawing up evidence summaries can be complex and time consuming. Automating the generation of figures and other steps using direct_flow can reduce workload, while ensuring high quality and consistency across different syntheses. Although developed as part of two projects funded by PROADI-SUS, direct_flow is freely available for all HTA researchers.

In 2024, 24 therapeutic sites were expanded within the universal coverage provided by the National Resources Fund (FNR) for all users of the National Integrated Health System (SNS), with an annual investment of USD30 million, mainly for the expansion of immunotherapy coverage. The FNR promotes the development of financial coverage regulations, discouraging the use of widely used drugs that could be subject to partial disinvestment within the SNS.

An increase in the FNR’s benefits basket was proposed in 2024, focusing on technologies with proven evidence and innovation. This initiative linked health technology evaluations, social and judicial demands, and budgetary impact analyses to determine the required investment. The proposed methodology targeted therapeutic areas where older comparators within the benefits basket could have their indications reduced in consultation with clinical experts, leading to a decrease in investment costs. The relationship between the cost of investment and the cost of disinvesting current technologies was calculated, enabling comparisons between technologies for disinvestment.

Within the SNS, the FNR’s high cost benefits basket proposed the modification of 12 treatments across 24 therapeutic sites for 2024. Of these 12 treatments, eight had active ingredients already funded by the FNR. In developing the regulations and conducting the budgetary impact analysis, a partial disinvestment of previously funded active ingredients was considered. The cost variation associated with disinvestment was quantified by comparing the costs of investment to the costs of disinvestment. The budgetary quantification showed variations ranging from 1.37 percent of the investment value in lung cancer to 24.5 percent in hepatocarcinoma, with significant heterogeneity among the different services.

To ensure the sustainability of healthcare systems, it is necessary to have dynamic benefits baskets that facilitate a constant flow of disinvestment alongside investments. The quantification of technologies to be disinvested in the FNR’s experience should not be considered a marginal expense. It is prudent to implement methodologies that encourage disinvestment, thus ensuring the system’s sustainability.

Medical device technology evaluation has advanced significantly in recent years. This study was conducted with the aim of identifying the main domains used globally for the evaluation of these technologies and considering the perspective of technology adoption in healthcare systems.

Structured searches were conducted in MEDLINE, Embase, the Virtual Health Library, the Cochrane Library, and Web of Science databases using the terms “medical technology” OR “medical device” OR “equipment and supplies” AND “health technology assessment” OR “biomedical technology assessments” for studies published between January 2017 and May 2023, excluding those on specific technologies or audiences. Documents from the International Network of Agencies for Health Technology Assessment and databases from the World Health Organization were also included. Study selection, extraction, and quality assessment were performed by two independent reviewers, with discrepancies resolved by a third reviewer. The term “domain” in this systematic review was inspired by the EUnetHTA Core Model.

A total of 5,790 studies were retrieved, with 41 meeting the inclusion criteria. Findings were grouped into eight domains, with percentages based on the selected articles (n=41; 100%): (i) safety (n=17; 41%); (ii) efficacy (n=18; 44%); (iii) health problem, current use of technology, and innovation (n=17; 41%); (iv) legal aspects (n=28; 68%); (iv) organizational benefits (n=17; 41%); (vi) description and technical characteristics of the technology (n=30; 73%); (vii) costs and economic evaluation (n=24; 58%); and (viii) social participation (n=11; 27%). Most articles (80%) were of European origin.

Due to the diversity of medical devices, a single methodological guideline may not cover all their specificities. Studies suggested grouping by technological characteristics to standardize the process. We highlight the importance of exploring less discussed domains, such as social participation and organizational benefits, which represent future topics.

Medical devices represent a challenge for health systems due to their complexity in decision-making analysis. In 2019, as part of the initiatives of the Health Technology Assessment Network of the Americas (RedETSA), the Working Group on Health Technology Assessment (HTA) for Medical Devices (MD) was established to strengthen capacities, explore methodologies, and share experiences among member countries. This group comprises 16 institutions from nine countries and serves as a collaborative platform to identify and address common areas of interest for the region.

Since its formation, the working group has met quarterly virtually. Progress is presented at the annual RedETSA meeting, and a common agenda has been established and coordinated by two countries. Initially, the group conducted a systematic literature review (SLR) to identify needs and new challenges in HTA for MD. Subsequently, the group collaborated on a critical analysis of the World Health Organization’s HTA of MD draft. The latest initiative started with periodic presentations on the status of HTA in MD by each member.

There is significant regional interest in addressing MD. The SLR results highlighted the need to establish methodologies across clinical, economic, organizational, ethical, and social dimensions. Collaboration among members has been vital in providing regional perspectives on technical documents and discovering methodologies developed in some countries that offer advancements and examples. Despite common needs, maintaining collaboration is challenging due to a lack of exclusive resources, staff turnover, and diverse health system structures. Coordinated efforts are essential to develop new methodologies to aid decision-making in health systems with limited resources.

Despite common needs, maintaining collaboration is challenging due to limited dedicated resources, staff turnover in member countries, and diversity in health system structures. This initiative aimed to strengthen HTA processes for medical devices in Latin America through collaborative efforts, innovative methodologies, and shared experiences, ultimately ensuring the effective and safe implementation of medical technologies in Latin American health systems.

In Brazil, the databases of the informatics department of the Brazilian Unified Health System (DATASUS) are key sources of oncology real-world data (RWD). Despite challenges like data quality, transparent methods ensure robust real-world evidence for cancer care. This study proposed a reproducible and open-sourced methodology to count cancer cases in the Unified Health System (SUS).

The methodology counts cancer cases treated in the SUS using claims data available from DATASUS. The unique patient counting system employs patient ID, with deduplication for patients with more than one ID, using a key based on sex, ZIP code, International Classification of Diseases, 10th Revision (ICD-10) codes, diagnosis date, and calculated birth year. This new methodology, which includes data download, was implemented in an R software package. Data from DATASUS outpatient records (chemotherapy, immunotherapy, or radiotherapy) for patients aged 18 to 99 years with pancreatic cancer (ICD-10 code C25) diagnosed between 2008 and 2022 were used to validate the method.

A total of 254,240 outpatient claim records were analyzed: counting 31,425 unique patients by the proposed methodology and 1.3 percent of records where the patient ID represented duplication. Patients had a mean age of 60.9 years, 50.2 percent were female, and 82.8 percent were aged 50 years or older. Regarding the cancer stage, 3.5 percent had in situ, whereas 3.4 percent, 9.3 percent, 17.6 percent, and 66.2 percent had stages I, II, III, and IV, respectively.

The study highlighted how a database created for administrative purposes can be used to describe cancer care. The open-source availability of the methodology (via an R package) ensures transparency and reproducibility in RWD analysis.

The need for reintervention peaks one to three years after an index endovascular intervention. Updating the Australian budget impact model becomes imperative to understand the impact of Eluvia™ drug-eluting stents (DES) over Zilver® PTX® drug-coated stents. This study forecasted the economic impact of Eluvia stents across various time horizons.

Model inputs for clinical endpoints were obtained from the IMPERIAL trial results from three to five years, published sources, and publicly available data. A public healthcare payer perspective was adopted. Cost inputs were obtained from national cost averages in the National Hospital Cost Data Collection Public Sector Report 2020–21, to reflect post-COVID-19 cost of care. Population statistics were obtained from the Australian Bureau of Statistics to reflect the evolving demographics in Australia. The original model assumptions were unchanged, except for the annual procedure growth rate (3.2%).

Assuming an 80 percent endovascular procedural eligibility rate and a DES use rate of 10 to 28 percent (superficial femoral artery lesions), cumulative savings from avoided reinterventions for Eluvia DES were as follows: one-year, USD0.41 to 1.14 million; two-year, USD1.06 to 2.95 million; three-year, USD1.12 to 3.12 million; four-year, USD1.35 to 3.76 million; and five-year, USD1.58 to 4.39 million. When considering non-significant secondary trial endpoints, the total net savings were: one-year USD0.20 to 0.55 million; two-year USD0.37 to 1.03 million; three-year, USD0.68 to 1.88 million; four-year, USD1.16 to 3.22 million; and five-year, USD0.90 to 2.48 million. The cost savings from avoided reinterventions for Eluvia DES group, as a ratio of total net healthcare cost savings, was the highest during two-year horizon.

Using Eluvia DES for treating peripheral artery disease offers substantial early savings to healthcare payers through avoided reinterventions. As such, a focus on clinical data during the reintervention risk peak at one to three years; one- to two-year budget cycles; and the use of high-quality devices upfront may improve patient outcomes and healthcare efficiencies.

The use of apps represents a revolution in mental health because they are fast, versatile, manageable, available twenty-four seven, empower patients and professionals, and can reduce stigmatization. However, there is not yet a standardized method to assess effectiveness and safety. The objective of the EvalDepApps project is to develop an evidence-based tool to evaluate apps whose main aim is to manage depression.

The EvalDepApps project followed several stages: (i) identification of health apps for depression through systematic mapping of the marketplaces; (ii) analysis of effectiveness of mobile health interventions for treating depression through a meta-analysis; (iii) identification by healthcare professionals and users (n=30) of a set of criteria to specifically evaluate apps for depression through a two-round modified Delphi study; and (iv) identification of requirements to be implemented in the EvalDepApps tool through co-creation workshops (design thinking methodology) in three settings (17 healthcare professionals and 13 patients). Currently, the app is being piloted by 15 healthcare professionals and 15 patients.

Thirty apps were identified in marketplaces. Twenty-nine randomized controlled trials were included in the meta-analysis. The analysis showed the most common elements in digital health interventions, the significant effect of mobile health interventions in reducing symptoms (95% confidence interval [CI]: −0.87, −0.37; I2=87%), and that hybrid interventions (mobile health plus face-to-face sessions) were the most effective. The Delphi study was based on 51 criteria and reached consensus in 28 criteria; co-creation workshops identified elements of interest for end users (ranking of the apps and recommendation systems). All these elements have been implemented in the EvalDepApps tool, which is being piloted currently.

Mobile health interventions can be effective in reducing depressive symptoms. It is important to standardize evaluation tools to identify which are the most effective. A human-centered approach improves app engagement and effectiveness. Thanks to inputs given by professionals and patients, together with existing evidence, EvalDepApps will be a tool to assess health apps based on a robust methodological approach.

Glucagon-like peptide-1 receptor agonist (GLP-1RA/pA) drugs are a breakthrough in obesity management. They produce nearly twice the weight loss achieved by previous drugs and have raised clinical expectations. At least nine GLP-1RA/pA drugs are currently approved or in phase three evaluation. This report aimed to provide an overview of evidence on the clinical outcomes of GLP-1RA/pA drugs to assist topic selection for a complete health technology assessment.

Exploratory searches were conducted in PubMed, combining the validated database systematic review filter with a structured string for patients with severe obesity (population) and GLP-1RA/pA as the intervention of interest. Inclusion criteria were: (i) systematic reviews with a meta-analysis (SRMA); (ii) severely obese individuals; and (iii) GLP-1RA/pAs as the active intervention. We excluded studies with: (i) children or adolescents only; (ii) any other therapeutic drug class used as an active intervention; (iii) systematic reviews without meta-analyses and other study designs; and (iv) studies in which obesity was not an inclusion criterion.

Forty SRMAs were included. Liraglutide was the most assessed drug, while beinaglutide was the least commonly assessed. Included patients (mean age 12.7 to 75 years) had a mean body mass index of 23 to 34.4 kg/m²; follow-up times ranged from four to 160 weeks. Of the SRMAs, 39 reported efficacy and safety outcomes, while one reported safety outcomes only. Two SRMAs reported other outcomes (economic outcomes and ethnic diversity of clinical trials). Thirty-four SRMAs included randomized controlled trials and five included observational studies that assessed bariatric surgery poor responders (BSPR). Three reviews performed network comparisons. Specific subgroups of obese populations were assessed in eight reviews (BSPR, ethnically diverse people, and obese patients using antipsychotic drugs).

While some reviews have explored specific subgroups, there is a gap regarding severe and super obese patients. Few SRMAs have focused on cardiovascular outcomes. Systematic reviews based on real-world evidence and long-term outcomes are limited. Our findings highlight the need for further research to address these gaps and provide more robust evidence for clinical practice.

The incorporation of a new technology in the health system requires extensive planning, actions towards its implementation, and monitoring of its effectiveness and safety in the population. The objective of this study was to evaluate the safety profile of miltefosine in the treatment of cutaneous leishmaniasis during the initial implementation phase.

Data from all patients who used miltefosine in the state of Minas Gerais between May 2021 and July 2023 were analyzed. Patient data were collected from the reporting and clinical monitoring form. Adverse events were classified according to the Medical Dictionary for Regulatory Activities; severity was categorized according to World Health Organization criteria and intensity according to the Division of AIDS. Descriptive analyses presenting measures of central tendency and dispersion for events were used, as well as multivariate analysis for explanatory variables and outcomes of interest.

The frequency of adverse events was 77.1 percent, 3.8 percent of which were severe, resulting in six patients requiring hospitalization. Gastrointestinal manifestations were the most frequent clinical adverse event, followed by musculoskeletal manifestations. The multivariate analysis indicated an association between diabetes and the occurrence of adverse clinical events. The most common laboratory alteration was elevated serum creatinine, which was significantly associated with arterial pressure, age, and mucosal clinical form. There were no records of pregnancy among the treated women. The rate of early treatment discontinuation was 11.8 percent, which was associated with age and an alteration in baseline serum creatinine level.

This study demonstrated the high frequency of adverse events that occur with miltefosine, predominantly gastrointestinal and renal function alterations, highlighting that pharmacovigilance strategies need to be implemented routinely. These data are essential for managing health technologies and supporting new decisions, thus contributing to patient safety.

Countries face challenges in prioritizing scarce health resources, often relying on evaluations based on quality-adjusted life years (QALYs). However, QALY gains may not fully capture intervention value, especially for rare disease treatments, which are often perceived as more valuable. This study examined whether Chinese health insurance decision-makers prioritize rare disease treatments and sought to quantify any additional value assigned.

We conducted two sequential discrete choice experiments (DCEs). The first, labeled DCE, compared drugs for common and rare disease using five attributes—disease severity, childhood onset, catastrophic expenditure, treatment novelty, and clinical benefit—to explore conditions for prioritizing rare disease coverage. The second, unlabeled DCE, compared rare disease scenarios, adding QALY gains and social insurance premium increments to estimate willingness-to-pay per QALY and derive incremental cost-effectiveness ratio (ICER) thresholds. A D-efficiency design produced 16 and 20 choice sets, blocked into two versions. In 2023, 120 decision-makers were invited for online data collection via interviews. Analysis used conditional logit models to assess preferences.

In total, 101 eligible decision-makers provided complete responses and were included in the analysis. Nearly half of the respondents were female (47.5%), with 58.4 percent having over 10 years of professional experience. Many participants had expertise in health insurance system research (48.5%) and pharmacoeconomics (77.2%). The first DCE found rarity alone did not add value; decision-makers prioritized disease severity, drug innovation, and health benefits, especially the first two. The second DCE estimated an ICER threshold for rare diseases of 1.9 times the gross domestic product per capita—around three times China’s current baseline threshold—reflecting equity considerations.

Chinese decision-makers demonstrated a willingness to assign higher value to rare disease treatments, particularly when considering severity and novelty. This study provided empirical evidence that supports higher ICER thresholds for rare disease treatments to account for equity considerations. The findings offer valuable insights for policymakers seeking to refine health insurance reimbursement strategies and ensure a more equitable allocation of resources.