Health technology assessment (HTA) has gained increased attention and investment in low- and middle-income countries (LMICs). HTAi Interest Groups provide critical platforms for networking, information exchange, and collaborative research. To understand the challenges and advances in HTA across LMICs, the Developing Countries Interest Group (DCIG) conducted a comprehensive survey in 2024.

The survey targeted LMICs, focusing on the following dimensions of HTA: operational frameworks (geographical distribution, focus areas, patient engagement, rare disease processes) and a SWOT analysis assessing: (i) strengths—things that the country does particularly well or that distinguishes it from others; (ii) weaknesses—features, resources, systems, and procedures, including what could improve and the sort of practices should be avoided; (iii) opportunities—openings or chances for something positive to happen; and (iv) threats—any issue that can negatively affect the development. Additionally, respondents identified key challenges in implementing HTA and proposed ways the HTAi DCIG could address these barriers.

The survey received 33 responses, with 50 percent being HTAi members, 36.3 percent from Latin America, 27.2 percent from Africa, and others from Asia. Of the respondents, 57.6 percent reported having an HTA department in their ministry of health, 48.5 percent had a national policy, and 84.4 percent used HTA for assessing medicines and devices. However, 54.5 percent lacked patient engagement processes. Genuine interest in building local capacity for HTA, but weaknesses included knowledge gaps, with limited funding and awareness among stakeholders as significant threats. Opportunities arose from growing interest among health professionals. Respondents suggested the DCIG support HTA institutionalization and provide foundational training.

The findings underscored active HTAi member engagement, especially in Latin America, Asia and Africa. While many countries have established HTA departments and policies, gaps persist in patient engagement and knowledge dissemination. Key strengths included interest in capacity building, while weaknesses included knowledge gaps. Opportunities for growth exist, but limited funding poses a threat. Respondents recommended that the DCIG provide support in institutionalizing HTA and training.

Congenital cytomegalovirus (CMVc) is a major cause of neonatal morbidity, with long-term effects such as hearing loss and neurodevelopmental impairments. Screening strategies, including systematic and targeted approaches, aim to improve early detection and management. This study evaluated the cost utility of these strategies to inform decision-making in Spain’s healthcare system.

A cost-utility analysis was performed using a decision tree and Markov model over an 18-year horizon. Incremental cost-effectiveness ratios (ICERs) were calculated based on quality-adjusted life years (QALYs). The analysis compared systematic screening using a saliva polymerase chain reaction test, targeted screening linked to universal newborn hearing screening (UNHS), and symptom-based detection. Sensitivity analyses explored the robustness of results, focusing on key cost and effectiveness variables.

Systematic screening detected 1,547 CMVc cases at birth, compared with 112 cases for targeted screening and 49 cases for symptom-based detection. At 18 years, systematic screening had the lowest number of patients with hearing loss and neurodevelopmental impairments. The ICER for systematic screening was EUR33,225 per QALY, compared with symptom-based detection and EUR37,254 per QALY compared with targeted screening. Targeted screening demonstrated an ICER of EUR6,912 per QALY compared with symptom-based detection.

Targeted screening linked to UNHS is a cost-effective strategy for detecting CMVc in Spain. Systematic screening, while identifying more cases, exceeded cost-effectiveness thresholds and may not be economically viable under current conditions. Optimizing the cost of systematic screening could improve feasibility and should be a focus of future research.

The United States Food and Drug administration (FDA) Accelerated Approval (AA) pathway facilitates early access to therapies for severe conditions based on surrogate endpoints, requiring confirmatory trials to validate benefits. This study compared FDA AA outcomes with Brazilian regulatory decisions, highlighting alignment and discrepancies, and their implications for patient safety and healthcare systems, focusing on post-market surveillance and evidence-based decision-making.

Data on drugs on the FDA AA pathway were retrieved from the FDA’s publicly available repository as of 7 November 2024. Each drug was categorized by approval status: ongoing, verified clinical benefit, withdrawn, and other. Drugs were further analyzed for approval, coverage, and reimbursement decisions in Brazil using data from the Brazilian Health Regulatory Agency (ANVISA), the National Committee for Health Technology Incorporation (CONITEC), and the National Supplementary Health Agency (ANS). The analysis focused on discrepancies between FDA and ANVISA decisions and the implications for Brazilian healthcare systems.

A total of 336 drugs were analyzed: 53 percent achieved confirmed clinical benefit, 12.8 percent were withdrawn due to safety or efficacy concerns, and 30.7 percent are still undergoing confirmatory trials. The average time from AA to withdrawal was 7.35 years, highlighting extended patient exposure to unverified therapies. Of the 43 drugs withdrawn by the FDA, 12 retained active label indications by ANVISA. This regulatory lag impacts the Brazilian National and Supplementary Health Systems through potential inefficiencies in resource allocation.

Discrepancies between FDA and ANVISA decisions underscore the need for stronger international collaboration on post-market surveillance and harmonized criteria for drug approvals. For Brazil, addressing these gaps could enhance patient safety, optimize healthcare resources, and align regulatory practices to ensure timely, evidence-based decision-making.

Public consultations (PCs) are widely used by health technology assessment (HTA) agencies, but there is no consensus on methodological procedures for analyzing PCs. We propose a method for analyzing and synthesizing PCs, offering an agile approach, systematic and transparent steps, reproducible aspects, and reliable analyses. The aim of this study was to validate the method.

The Delphi technique was used to validate the proposed method in terms of face (applicability and relevance) and content validity (analysis results). The setting included the HTA processes conducted by the National Committee for Health Technology Incorporation (CONITEC). A group of 20 Brazilian experts was invited to participate, each of whom had experience in at least one of the following areas: HTA, social participation, IRaMuTeQ software, or qualitative research. Consensus was defined as at least 70 percent with “agree” or “strongly agree” responses or an interquartile range of one or less. Comments were summarized and categorized thematically. A pilot study was also conducted.

Fifteen experts participated in the face validity assessment and 14 in the content validity assessment. Both validations achieved over 80 percent consensus in the first round. In face validity, five methodological steps were validated, including corpus analysis, corpus preparation and organization, data mining using IRaMuTeQ software, systematization and interpretation, and synthesis of findings. In content validity, the results were deemed adequate, providing sufficient information to understand PCs. Although this method was used in the Brazilian HTA setting, it can be adapted by different countries. The method provides a way to organize data already collected in HTA PCs, contributing to processes already in use.

This study validated a method for analyzing and systematizing HTA PCs—the first strategy of this kind. The method proposes a systematic and transparent approach to organizing data collected for HTA agencies. This initial strategy for systematizing PC analyses can support more participatory, agile, and transparent processes that include public perspectives, which can be a challenge in HTA.

Over the past decade, there has been an increase in the development of innovative cancer treatments that have positively impacted upon mortality rates. However, the approval process at the local level remains lengthy. This study estimated the clinical and economic impact of the availability of innovative therapies for treating advanced lung cancer in men in five Latin American countries.

Using public data, we analyzed the relationship between available innovative therapies and age-specific mortality rates for Argentina, Brazil, Chile, Colombia, and Mexico through a regression model. By comparing the number of therapies approved by the United States Food and Drug Administration (FDA) with those approved by each local regulatory agency, we estimated the avoidable deaths (ADs) that could have been averted if innovations had been available. We calculated years of life lost (YLLs) based on life expectancy, median age of death, and ADs. Productivity loss was estimated using each country’s retirement age and the annual gross domestic product (GDP) per capita in 2022.

Between 2006 and 2021, the FDA approved 24 innovative treatments for 30 indications. By 2021, only 60 to 77 percent of these therapies were available locally. Total ADs, YLLs, and PL were USD8,694, USD114,477, and USD439,179,876, respectively. The most significant productivity losses were found in Brazil and Argentina due to the large Brazilian population and Argentina’s high GDP per capita. Despite its low population, Chile’s high GDP per capita resulted in considerable productivity loss. Colombia and Mexico showed a notable clinical impact, suggesting the advantages of early approval. Although the availability of FDA-approved therapies has increased, local time lags have recently increased.

Our results highlight the potential of improving regulatory processes to increase the availability of innovative therapies in five Latin American countries. Accelerating the introduction of these therapies for advanced lung cancer presents a major opportunity to enhance survival rates, instilling a sense of hope and optimism while avoiding substantial productivity losses.

Venous thromboembolism (VTE) prevention for women with additional risk factors during pregnancy is centered on anticoagulant therapy, with enoxaparin as the anticoagulant of fchoice. This study evaluated the efficacy, safety, and costs of enoxaparin for thromboprophylaxis in pregnant or postpartum women at risk for VTE, aiming to expand access to the Brazilian national Clinical Protocols and Therapeutic Guidelines (PCDT).

A search was performed in the MEDLINE, LILACS, and Embase databases (January 2024) using a strategy that combined target population, enoxaparin, and low molecular weight heparin; filters for systematic review (SR) with or without meta-analysis; and publications up to five years, without language restriction. Duplicate exclusion and study eligibility were performed using Rayyan. Only eight studies met the pre-established criteria. For budget impact, enoxaparin values and doses, local hospital consumption, and epidemiological data were used. The daily, average, and annual costs of hospitalization of pregnant women (n=259) with VTE in the State of Bahia were calculated.

Results showed a reduction of thromboembolic events; a reduction of VTE in pregnant women at high perinatal risk as well as postpartum women who did not use anticoagulation in the antepartum period; an increased risk of hemorrhagic events in pregnant women, worsening during in vitro fertilization; a reduction in the risk of miscarriage and an increase in the rate of live births; and a significant increase in allergic reactions. Only one SR converged with the national guideline. The annual avoidable hospitalization cost was USD239,725.80, while the budget impact was USD163,207.47 in the first year. A group of experts was established to develop the state protocol with the expanded use of enoxaparin.

The economic analysis showed a high average cost of prolonged hospitalization in pregnant women receiving enoxaparin. Thus, considering the High-Risk Pregnancy Manual after the PCDT, vulnerability, prevention of maternal morbidity and mortality, turnover of obstetric beds, reduction of hospitalization costs, and qualification of care, it was decided to develop a state protocol with expansion of anticoagulation indications.

Decisions to not reimburse or discontinue reimbursement of health technologies from public funding are considered increasingly necessary. However, such decisions remain politically sensitive and often provoke public opposition. Such opposition may be considerable and puts pressure on decision-makers to revoke or revise a decision; however, the specifics of this opposition remain largely unclear. Our aim was to obtain insight into these specifics by mapping the scientific literature on this topic.

We performed a systematic review following PRISMA guidelines. The electronic search was performed in Embase and using the Google Scholar, Google, and Startpage search engines and supplemented with a hand search in 2021. Both the electronic searches and the hand search were updated in 2022 and 2024.

Based on 81 articles, we developed a framework of 27 categories grouped under the ‘who, what, when, where, and why’ of public opposition to negative reimbursement decisions. Our findings indicated that patient representatives, physicians, and citizens are the primary actors opposing such decisions—often influenced by industry and politicians. These actors typically challenge the outcomes and arguments underlying decisions, driven by unrealistically high expectations of technology effectiveness and the assumption that decision-makers prioritize cost containment above all else. Additionally, our findings indicate increasing distrust among these actors toward decision-makers and evidence-based processes, leaving them vulnerable to commercial exploitation and misinformation.

Understanding the dynamics between actors is crucial for understanding public opposition to negative reimbursement decisions in health care. Aligning decision-making processes with public values, addressing misconceptions, and countering misinformation may enhance the legitimacy of reimbursement decisions. Further research should explore strategies to mitigate distrust and foster evidence-based engagement among actors to ensure more informed and equitable healthcare decision-making.

For the technology incorporating process, indirect comparisons (IC) may be presented as alternatives when head-to-head studies are unavailable. However, health technology assessment (HTA) reports often fail to address the methodological fragilities of network meta-analysis. This interim analysis examined the frequency of IC methods in technology assessments for incorporation into the Brazilian supplementary health system and inclusion in the National Agency of Supplementary Health (ANS) catalog.

This meta-research study was conducted at the Center of HTA, Hospital Sírio-Libanês, São Paulo, Brazil. A search on the ANS website identified critical analysis reports of technologies incorporated by ANS in 2023 and 2024. Eligible reports were analyzed to identify the frequency of IC in plaintiff’s submissions and in ANS analyses. The incremental budget impact of incorporating technologies with IC in HTA reports was also evaluated.

A total of 47 reports were evaluated. Of these, 36.2 percent of the submissions and 25.5 percent of the ANS analyses included IC, mostly using network meta-analysis. Among 32 favorable recommendations, IC appeared in 31.3 percent of submissions and 25 percent of ANS analyses. From 2023 to 2024, the use of IC increased by 12.7 percent in submissions and by 21.8 percent in ANS analyses. Within incorporated technologies containing IC, the incremental budget impact analysis predicted savings of BRL61,385,084.02 to BRL174,217,905.44 for savings scenarios, and increased spending of BRL179,524,075.54 to BRL488,084,349.61 for increased investment scenarios.

IC methods were present in approximately 25 to 30 percent of all technology assessments for incorporation into the Brazilian supplementary health system, with an increased number observed from 2023 to 2024. Within technology assessments employing IC, projected financial outcomes indicated savings in some scenarios, while others suggested substantial increases in budget expenditure.

Adults with intellectual disability (ID) experience marked mental health inequities, yet population-wide estimates that capture both primary- and specialist-care diagnoses remain scarce.

Using nationwide Swedish registries, including primary care, specialist, inpatient, prescription drug, criminal, and suspicion registers, we included all individuals born in Sweden between 1958 and 1997 (N = 3,970,600), including 38,818 individuals with ID diagnoses (0.98%; 49.1% mild, 13.3% moderate, and 9.6% severe/profound). Hazard ratios (HR) were calculated using Cox proportional hazards regression to estimate the relative risk of lifetime diagnoses of major depression, anxiety disorders (ANX), obsessive-compulsive disorder (OCD), bipolar disorder (BD), attention-deficit/hyperactivity disorder (ADHD), drug use disorder, alcohol use disorder, schizophrenia, and other nonaffective psychosis. Additionally, cohort effects on psychiatric diagnosis risks in adults with intellectual disabilities versus the general population were evaluated.

People with ID were at higher risk for all psychiatric and substance use disorders, with HRs ranging from 1.7–2.0 for major depression and anxiety, drug and alcohol use disorders, 3.5–5.8 for BD, OCD and ADHD, and 10.9–12.7 for schizophrenia and other nonaffective psychosis. Higher prevalence was consistently seen among those with mild versus moderate or severe/profound intellectual disability. Relative risks narrowed modestly in successive birth cohorts, although absolute differences remained substantial.

Across six decades of follow-up, adults with ID faced markedly higher psychiatric and substance-use morbidity – most pronounced for psychotic disorders – than the general population. Whole-system mental-health screening and tailored interventions are required to address this persistent disparity.

Understanding health technology assessment (HTA) is essential for effective public participation in Brazil’s Unified Health System (SUS). The Participa SUS/ATS project simplifies the concepts of HTA to foster training and knowledge dissemination. Through scientific marketing on social media, it provides interactive posts, making HTA accessible and empowering lay audiences to actively engage in decision-making processes.

The project leveraged social media platforms (LinkedIn, YouTube, and Instagram) to disseminate knowledge on HTA and train lay audiences of the SUS. Educational videos, including versions with Brazilian Sign Language, illustrations for inclusivity, whiteboards, and live sessions carried out by the project team and guests addressing topics of social participation, were shared on YouTube. A logo was created as part of its visual identity. Instagram featured weekly polls, stories, and personalized posts using accessible and engaging language. LinkedIn promoted broad professional dialogue. This multiplatform approach combined multiple formats to increase reach and understanding, and to promote public interaction and empowerment in HTA processes.

By 2024, the initiative garnered 518 Instagram followers, 218 YouTube subscribers, and 1,202 LinkedIn followers. LinkedIn promoted conversations among healthcare professionals, Instagram’s engaging weekly posts connected with audiences, and the most-watched YouTube video garnered 323 views. Real-time interaction was encouraged with three live sessions. A specialized logo improved brand recognition and connection with the intended audience. Carousel postings were a user-friendly format, particularly on mobile devices. Users praised the content’s clarity and emphasized how well it demystified HTA contents. Posting schedule optimization raised exposure and interaction. Feedback highlighted improved understanding, empowering users to contribute more effectively to decision-making processes.

The Participa SUS/ATS project showcases an innovative use of social media to demystify HTA for diverse audiences, fostering inclusivity and active public engagement in health decisions. Its scalable, multiplatform approach offers a replicable model for enhancing citizen participation and equity in healthcare systems worldwide, making it highly relevant for global public health initiatives.

Anti-neutrophil cytoplasmic antibodies (ANCA)-associated vasculitis is a rare disease with high morbidity and mortality rates, posing challenges for incorporating treatments due to limited high-quality evidence. Brazil recently developed a Clinical Protocol and Therapeutic Guideline (PCDT) after incorporating rituximab into the public healthcare system. This study explored the development of a Technical Scientific Report (TSR) addressing rituximab’s effectiveness and costs as well as stakeholder participation in shaping equitable, evidence-based recommendations.

A TSR was initially developed that included an evaluation of evidence, an economic model, and a budget impact analysis. Cyclophosphamide, which is already available in the public health system, was used as the comparator. Based on this analysis, the National Committee for Health Technology Incorporation (CONITEC) issued a preliminary recommendation against incorporating rituximab for ANCA-associated vasculitis. This TSR was submitted for public consultation (6 to 25 April 2023), during which community contributions were received. All submissions were thematically analyzed, and relevant contributions were re-evaluated. The updated findings were then presented to CONITEC for a final decision, incorporating new perspectives and refined results.

During the public consultation, 169 contributions were received: 56 from patients and caregivers unanimously supporting rituximab’s incorporation and 113 technical submissions highlighting cyclophosphamide’s toxicity and lack of response in 25 percent of patients, and rituximab’s economic benefits. Revised economic analyses found a reduced incremental cost-effectiveness ratio when comparing rituximab with intravenous cyclophosphamide and clarified the five-year budget impact. These updates, along with strong patient participation, influenced the reversal of CONITEC’s initial recommendation, leading to rituximab’s approval in June 2023 and the development of the PCDT.

The incorporation of rituximab into Brazil’s public health system and the creation of the PCDT for ANCA-associated vasculitis represents a significant advancement in treating rare diseases and may help promote remission, reduce relapses, and improve patient quality of life. By integrating patient perspectives and evidence-based practices, the PCDT strengthens equity and access, fostering comprehensive care for rare diseases in Brazil.

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.