Most treatment regimens for people living with HIV (PLHIV) require the intake of multiple daily pills, which can pose challenges to adherence. As an alternative, long-acting (LA) dual regimens have gained attention for their potential to simplify treatment. Given the importance of evaluating the long-term effects of LA regimens, this study aimed to synthesize evidence on their effectiveness and safety.

A systematic review was performed following the Cochrane Collaboration recommendations and the Meta-Analysis of Observational Studies in Epidemiology (MOOSE) guidelines, with searches in PubMed, Scopus, and Web of Science (July 2024) (PROSPERO-CRD42024570754). Longitudinal studies evaluating any dual-therapy antiretroviral regimen with LA agents (islatravir, cabotegravir, lenacapavir, rilpivirine, albuvirtide) for PLHIV were included. For each outcome of interest, data were pooled using bivariate meta-analysis (discontinuations due to adverse events) and network meta-analysis (NMA) (virologic failure [VF]), with results presented as risk ratios (RR) with 95 percent credibility intervals (CrIs); heterogeneity was assessed using the I-squared statistic. The surface under the cumulative rating curve analysis (SUCRA) was also conducted for the NMA.

Thirty-two studies (2016 to 2024) evaluating 11 treatments were included. Four cohorts (n=2,570) were assessed in the NMA of VF, where the rilpivirine/dolutegravir (RPV/DTG) regimen was superior to non-LA regimens dolutegravir/lamivudine (RR −1.60, 95% CrI: −4.08, 0.91) and dolutegravir/doravirine (RR −0.42, 95% CrI: −1.68, 0.68), with SUCRA probabilities of 9.6 percent, 31 percent, and 59.5 percent, respectively. The triplet regimens dolutegravir plus tenofovir/emtricitabine and dolutegravir plus abacavir/lamivudine had the highest probabilities of leading to CD4+ count increases at six to 24 months (65.6 to 85.7% and 65.8 to 84.4%, respectively), followed by RPV/DTG (59 to 60%) and dolutegravir/lamivudine (46 to 53%). However, RPV/DTG regimens were considered safer than dolutegravir/lamivudine (RR 0.53, 95% CrI: 0.32, 0.89) regarding discontinuation due to adverse events.

This updated synthetized evidence suggested that the RPV/DTG regimen is a promising alternative for simplifying the treatment of PLHIV, demonstrating comparable effectiveness to the dolutegravir/lamivudine and dolutegravir/doravirine regimens, while offering better tolerability. However, considerations of drug accessibility and cost must be carefully weighed in the decision-making process in different settings.

A legislation, regulation, and policy review of limited South African legislative framework of health technology assessment (HTA) was undertaken to identify opportunities to advocate for required legislation reform or development to entrench principles and processes for patient and citizen involvement (PCI). We reviewed and mapped the capacity, knowledge, and skills of South African patient and citizen advocacy actors to actively advocate in developing regulations and policy in the National Health Insurance landscape.

We used a literature scan and analysis of relevant national and international documentation, including reports of primary and secondary data analysis to aid in the identification of current gaps in, and opportunities for, PCI in HTA in South Africa. Data sources included resolutions, legislation, regulations, government policy, and technical reports gleaned from websites of relevant agencies and experts. An electronic survey was distributed to 213 patient and citizen actors. The survey aimed to establish the capacity, knowledge, and skills of South African patient and citizen advocacy actors and organizations across multiple disease areas, predominately non-communicable diseases.

Legislation and policy documents indicated engagement initiatives are located at “involvement” or “consultation” stages of the engagement continuum. Patient advocacy groups participated in raising awareness of disease burden, advocating for adoption of specific technology, or utilizing legal channels to compel coverage arrangements for specific therapies.

The barriers and challenges observed were:

• • lack of skills, training, and education;

• • insufficient information regarding roles and responsibilities;

• • lack of logistical or financial support; and

• • lack of integrated strategy.

Possible dimensions for capacity building include select starting points that build on research partners’ strengths, meeting the immediate needs of governments, and contributing to longer-term goals.

Results indicated non-involvement of key actors in crucial healthcare processes. Actors were adamant that they would have greater influence at the provincial or national level to make meaningful contributions. Existing legislative and policy frameworks do not include PCI capacity building strategies. We can only conclude that once healthcare processes are inclusive, PCI will become mandatory.

Restricted access to orphan drugs significantly challenges rare disease patients, impairing health outcomes and quality of life. In Türkiye, systemic barriers such as regulatory inefficiencies, economic limitations, and inadequate stakeholder collaboration persist. This study evaluated these issues through stakeholder perspectives, proposing a collaborative framework to improve access, ensure equity, and enhance systemic efficiency in addressing rare disease challenges in Türkiye.

This qualitative study employed semi-structured focus group discussions with 19 patients and caregivers and 13 in-depth interviews with healthcare providers, policymakers, industry representatives, and regulatory bodies. Thematic analysis of 1,996 coded excerpts identified key barriers to orphan drug access, categorized as direct or indirect impacts. Grounded theory was applied to synthesize these findings into a cohesive framework addressing economic, regulatory, and systemic challenges. Data triangulation enhanced validity, ensuring the perspectives of diverse stakeholders were accurately represented. Stakeholder feedback on the framework was incorporated iteratively, ensuring practicality and adaptability for policy and decision-making in Türkiye. Supported by TUBITAK 2224-A Program for participation in HTAi 2025; it had no role in the study conduct.

The study revealed multifaceted barriers to orphan drug access in Türkiye, including fragmented regulatory processes, high costs, and inadequate inter-stakeholder collaboration. Indirect factors, such as limited public awareness and insufficient professional education, exacerbated these challenges. The proposed framework emphasizes centralized regulatory mechanisms to streamline approvals, public–private partnerships to mitigate financial constraints, and enhanced communication to ensure transparency and accountability. Stakeholder feedback highlights the framework’s adaptability and potential impact, particularly in addressing disparities within Türkiye. Success metrics, such as improved regulatory efficiency and patient outcomes, were defined to support future implementation and continuous improvement.

The proposed framework addresses critical systemic barriers to orphan drug access in Türkiye by fostering collaboration, streamlining regulatory pathways, and enhancing equity. It emphasizes the importance of stakeholder alignment and sustainable policy solutions for rare disease patients. Future research may focus on piloting the framework, adapting it for broader contexts, and developing evaluation tools to measure its long-term impact.

Globally, healthcare services contribute five percent of total greenhouse gas (GHG) emissions. Advances in biomedical research have led to innovative technologies, but the use of fossil fuel reliant products is a critical environmental issue. The burden is even greater in low resource settings (LRS) with increasing waste management issues. Real world studies can shed light on the environmental footprint of health care and help prioritize assessments.

Various policies and guidance are being developed on environmental sustainability of healthcare. However, it is imperative that health technology at different levels of care is studied empirically, taking a holistic approach to the environmental impact from a planetary health perspective. By presenting research from LRS, we can understand how use of devices and services have different environmental consequences depending on established regulatory frameworks. We conducted a rapid review with snowballing to understand how LRS have adapted to unmet needs and tried to solve these using a circular economy approach.

The footprint of a typical cataract operation in a high resource setting was 182 kg of carbon dioxide equivalent. Efficient systems and equipment reuse reduces this to 6 kg in a LRS, a 30-fold reduction. Despite both having positive clinical outcomes, one phacoemulsification surgery in the UK emits 20 times more GHG than the equivalent in India. Resource scarcity necessitated circular economy practices like “repair” and “reuse” to keep products in circulation. An electrosurgical unit, typically lacking in LRS, is designed to be durable, repairable, upgradable, and reusable even during electrical failures. Planetary and human health across all settings require resource optimization, innovative materials engineering, and reusable solutions.

Urgent research is needed to understand the potential impacts of more sustainable health technology innovation across eye care in different settings. A broader range of environmental impacts beyond GHG emissions studied across the life cycle of devices is necessary. Routine inclusion of environmental outcomes in clinical studies and explicit identification, testing, and promotion of sustainable practices will help promote sustainable decisions.

Saúde e Educação (SeE) is an extension project of the Federal University of Minas Gerais (UFMG), Brazil, which aims to disseminate information and research related to health in a simplified way for the public. Based on the perception of existing knowledge gaps in society, the SeE created a thematic series to disseminate accessible information about health technology assessment (HTA).

A partnership was established between the Collaborating Centre for Health Technology Assessment and Excellence (CCATES) and the UFMG. Members of CCATES were invited by the SeE to voluntarily produce informative texts about HTA to be published and disseminated by the SeE. The SeE editors reviewed the texts and formatted them to have a layout that motivates reading and facilitates understanding. The materials were published on the SeE website and disseminated on the project’s social networks. Strategic days were selected for publication. Partnerships with patient associations, for example, were formed to facilitate dissemination. The project had no funding.

In 2024, five texts by seven collaborators were produced, published, and disseminated. Three are in the process of preparation or review. To date, the texts have received 154 views on the website, and the advertisements have received more than 1,622 views on social media. Challenges encountered include problems with knowledge translation and reaching the target audience.

The SeE has been disseminating knowledge about HTA through digital means. This is important to ensure that information is accessible to the public. It can promote more inclusive and participatory decision-making, contribute to equity in access to health innovations, strengthen social control, and improve the implementation of technologies that meet the population’s needs.

The Australian Government Department of Health and Aged Care (Department) and Medicines Australia (the peak body for the pharmaceutical industry in Australia) have entered into a five-year strategic agreement. A core objective of this agreement was to develop a consumer-led, co-designed process to enhance consumer engagement earlier in the Australian health technology assessment (HTA) system.

A co-design proposal was developed by a consumer reference group and included guidance on “consumer-led” processes such as establishing a Co-design Working Group (Group) of consumer, industry, and Department members weighted toward a greater number of consumers. Selection of consumer members was by a consumer-peer selection panel. An independent facilitator led the co-design process, founded on a work schedule and design principles developed by the Group. Recent consultation reports on consumer engagement in HTA processes guided the Group and enabled it to be “solution focused.” The Group sought open consultation on draft recommendations prior to finalizing a report of recommendations.

The Enhance HTA report describes recommendations for consideration by the Australian Minister for Health and Aged Care. The Group established a vision where “Australians’ diverse health care experiences and needs are understood, and consumer engagement is integral in HTA decision-making.” Enhance HTA includes 10 recommendations related to themes of partnerships, transparency, and evidence and input. The report notes the need for alignment with reformative HTA policies and methods, investment in systemic change, and development of a framework to embed the proposed recommendations along the health technology pathway. The co-design process and outcomes will be discussed if accepted for presentation.

The consumer-led, co-designed process is a strong signal of the desire to engage consumers earlier in Australian HTA processes and in a more meaningful way. The expectations are high that the recommendations emerging from this process may be implemented to further embed consumer engagement into HTA processes, ensuring the diverse healthcare needs of Australians are integral to HTA decision-making.

Health systems in Australia and globally struggle to keep up with disruptive health technologies like gene therapies, gene editing, regenerative medicine, genomics, and artificial intelligence. These technologies promise better health outcomes but pose challenges due to rapid advancements, information gaps, and lack of a unified national approach. Solutions require new legal frameworks, workforce training, infrastructure investments, and government funding, supported by horizon scanning (HS) to anticipate future needs.

We will provide a review of the history of HS efforts to date in Australia and outline our proposal for the establishment of a new HS function to transform Australia’s approach to new technologies from reactive to proactive. This function will integrate with post-reform health technology assessment (HTA) processes to support planning and priority setting across all levels of government, service providers, product developers, and consumers by identifying emerging technologies and outlining their benefits, risks, and operational implications over the short and long term.

Australia has a 17-year history of HS devices and diagnostic health technologies for HTA, investment, implementation, and disinvestment purposes, although such activity ceased during the pandemic. There is growing support for re-establishing HS, although stakeholders have diverse views on its use. A proposed HS approach aims to support planning and priority setting across federal and state/territory governments, service providers, product developers, and consumers by identifying emerging technologies and articulating the likely benefits, risks, and operational implications, including resource requirements, over near- and longer-term horizons.

With respect to HTA, following the completion of the HTA Policy and Methods Review, the Australian government established an Implementation Advisory Group to develop a roadmap in 2025 for the government’s response to the review recommendations. We will discuss how our proposed HS function can integrate with the HS-related reforms that have been proposed under this review.

Ovarian cancer (OC) is the most lethal gynecological tumor worldwide. This study aimed to describe the profile of patients with OC and estimate the indirect costs of OC within the Brazilian Unified Health System (SUS).

This retrospective observational study used data from the Department of Informatics of the SUS and included patients aged 18 years or older with at least one record of International Classification of Diseases, 10th Revision code C56 from 2014 to 2023. Indirect costs were estimated by calculating years of potential life lost (YPLL) based on a 2022 life expectancy of 79 years for Brazilian women. The age threshold of years of potential productivity lost (YPPLL) was set at 62 years. The cost of productivity loss (CPL) was calculated using the gross domestic product (GDP) per capita in USD from the World Bank, converted to BRL (USD1=BRL5.66).

The study included a total of 36,141 patients with OC, with an average age of 56.1 years (standard deviation 13.4) at the time of their first claim. Most of the patients (51.2%) were white, lived in the southeast region of Brazil (44.6%), and had their first claim at advanced cancer stages (78.1%). The estimated indirect costs for OC were 22.1 YPLL per patient. The estimated YPPLL was 7.3 years, and the CPL was USD65,705.30 (BRL374,040.56) per patient.

This study provided valuable insights into the profile and indirect costs of OC. By analyzing real-world data, we highlighted the burden of OC, which impacts not only the health and longevity of patients but also places a significant financial burden on society due to productivity losses.

Treatment of HIV-1 infection is based on antiretroviral therapy. A pharmaceutical innovativeness index (PII) was proposed as a tool to evaluate the degree of innovativeness of technologies, initially for cancer treatment. This study aimed to adapt the PII for assessing therapies used in the treatment of people living with multidrug-resistant HIV-1 (MDR-HIV), based on an assessment of lenacapavir.

The PII is structured into four domains: unmet therapeutic need (UTN), added therapeutic value (ATV), study design (SD), and methodological quality (MQ). The second domain required adaptation to better fit the outcomes typically evaluated in pivotal studies of antiretrovirals for the treatment of MDR-HIV patients. To adapt the PII for antiretroviral therapy innovations, we assessed the innovativeness of lenacapavir as a case study, based on the phase two/three study comparing it to fostemsavir trometamol and its pivotal phase three study.

In this adaptation, virological response rate ratio replaced relative risk of mortality in the ATV domain. The proposed scale suggested that a ratio less than one corresponded to “absent” ATV; a ratio between 1.01 and 1.24 to “poor” ATV; between 1.25 and 1.99 to “moderate” ATV; and a ratio of at least two to “maximum” ATV. Lenacapavir was classified as “moderate” for both UTN and ATV domains, as “inadequate” for study design (single-arm trial), and as low risk of bias in the MQ domain. Thus, an innovativeness score of 50 was assigned to lenacapavir.

This preliminary case study adaptation provided an important contribution to evaluating the degree of innovativeness of antiretrovirals used in the treatment of people living with MDR-HIV. Maintaining the weights assigned to each domain proposed by the PII, led to an innovation score of 50 for lenacapavir.

Duchenne muscular dystrophy (DMD) is a rare, X-linked recessive disease in which dystrophin deficiency leads to progressive muscular degeneration. Ataluren is a mutation-specific therapy that delays disease progression. Our study aimed to assess the cost-utility of ataluren plus standard care (SC), compared with SC alone, in patients with nonsense mutations DMD from a Brazilian Unified Health System perspective.

A semi-Markov model with five health states based on ambulatory status was developed to assess the incremental cost-effectiveness ratio (ICER) of ataluren plus SC versus SC alone, which was expressed as BRL per quality-adjusted life year (QALYs). A 50-year time horizon was used in the base case analysis, considering annual cycles. Both costs and QALYs were discounted five percent. Transition probabilities and utility data were taken from published literature. Costs were based on data from a micro-costing study and the Brazilian Chamber of Drug Market Regulation database. Parameter uncertainties were tested using deterministic and probabilistic sensitivity analysis.

Ataluren plus SC led to incremental 0.503 QALYs and incremental costs of BRL32,453,742.94 (USD13,298,877.45), resulting in an ICER of BRL64,463,723.25 (USD26,415,910.08) per QALY. The deterministic sensitivity analysis showed that early ambulatory state utility was the most significant for the ICER, followed by costs of ataluren plus SC related to early ambulatory state. All estimates in the probabilistic sensitivity analysis were above the willingness-to-pay threshold of BRL120,000.00 (USD49,180.33) per QALY, which takes into consideration the rarity of the disease.

The ICER value was 537 times greater than the cost-effectiveness threshold of BRL120,000.00 (USD49,180.33). Therefore, ataluren plus SC is not a cost-effective therapy for patients with nonsense mutations DMD. However, it is essential to discuss cost-effectiveness threshold, value-based prices, and commercial agreement for pricing of orphan drugs in order to provide more effective therapies and prevent judicialization.

Rates of loneliness have increased over the past several decades worldwide, particularly among people with serious mental illnesses. A better understanding of the neurocognitive mechanisms underlying loneliness could provide useful information for the efforts to address this public health problem.

To investigate these mechanisms, a functional magnetic resonance imaging (fMRI) study was conducted which accounted for known cognitive biases associated with loneliness. Participants with (n = 40) and without (n = 60) psychotic disorders (PD) viewed images of faces that appeared to approach or withdraw from the participants while fMRI data were collected. Following the scanning, participants rated the trustworthiness of the faces, and these ratings were included as weights in the fMRI analyses. Neural responses to approaching versus withdrawing faces were measured, and whole-brain regression analyses, with loneliness as the regressor, were performed.

In the PD and full samples, a higher level of loneliness was significantly associated with greater responses of the hippocampus and areas of the basal ganglia to withdrawing (versus approaching) face stimuli. Moreover, the effects in the hippocampus, but not the basal ganglia, remained significant after controlling for potential confounds such as social activity levels, depression and social anhedonia. Finally, in a subset of the full sample (n = 66), greater hippocampal responses to withdrawing faces predicted greater loneliness 1 year later.

Heightened responses of the hippocampus to withdrawing faces may represent a candidate neurobiological marker of loneliness that could be modified by interventions targeting loneliness.

There are big challenges in Latin America and Argentina in terms of access to health. There is also consensus on the importance of managing access, coverage of health technology assessment (HTA), and the achievement of more efficient health expenditure. Our aim was to describe the creation and implementation of an Argentinian government HTA agency.

A local HTA agency project was implemented in Mendoza, Argentina based on: (i) the experience of an active local government HTA committee; and (ii) local and national consultation with decision-makers, health funders, and providers. An institutionalization plan for HTA was implemented based on a set of interventions that included legal framework, resources, and institutional agreements. It was then submitted for legislative and citizen debate. Following achievement of the legal framework, HTA agency work began.

The Agencia de Evaluación de Tecnologías Sanitarias de Mendoza (AETS Mendoza) was created by Law 9547 (1 July 2024), regulatory decree (Dec.1777/24). HTA good practice recommendations were achieved: autonomous body with legal and budgetary independence and functions to evaluate and issue recommendations on the value of technologies in key aspects of their life cycle (efficacy, clinical relevance, cost effectiveness, budgetary impact, equity). A multidisciplinary technical team was constituted. Recommendations that emerged from the evaluations are mandatory for public sector and provincial social security funders. The HTA reports are planned through annual prioritization and published on the official website.

HTA agencies are regulatory bodies that assess the value of health technologies and give local health systems responses to innovation, incremental spending, and equity. AETS Mendoza is the first government HTA agency in Argentina. This experience is innovative given the fragmentation and complexity of the Argentine health system. This model could be adopted by other health systems.

Canada’s Drug Agency (CDA-AMC) conducted a health technology assessment of RapidAI for detecting ischemic stroke and hemorrhagic stroke to test and learn from its first comprehensive assessment of an artificial intelligence (AI)-enabled health technology.

The assessment included a review evaluating the effectiveness, accuracy, and cost-effectiveness of RapidAI for detecting ischemic and hemorrhagic stroke, alongside an implementation review capturing digital infrastructure considerations. Ethics and equity considerations were integrated throughout, informed by literature, patient engagement, and expert input. Checklists and other AI or digital health tools were applied. The Health Technology Expert Review Panel (HTERP), an advisory body to CDA-AMC, reviewed the evidence and developed recommendations on the appropriate use of RapidAI for stroke detection, considering the following domains: unmet clinical need, clinical value, economic considerations, impacts on health systems, and distinct social and ethical considerations.

Patient input highlighted speed and accuracy in stroke diagnosis. Low certainty clinical evidence suggested that using the AI functionalities of RapidAI to assist diagnoses may result in clinically important time reductions. Its effects on other clinical outcomes were very uncertain. Ethical and equity considerations have implications across the technology life cycle when using RapidAI for detecting stroke; however, little relevant information was identified from the literature. We found no relevant economic evaluations. The implementation review identified key considerations for AI-enabled health technologies for decision-makers. Given the evidence gaps and uncertainty, HTERP could not recommend for or against the use of RapidAI for stroke detection.

Our appraisal and deliberative processes identified evidence limitations that may be common across many AI-enabled health technologies, identifying challenges that need to be addressed in their evaluation. Based on this experience, for AI evaluations CDA-AMC plans to add AI-specific implementation and other considerations to its evidence reviews and to consider a broader range of information sources.

Axicabtagene ciloleucel (axi-cel) is a chimeric antigen receptor T-cell (CAR-T) therapy approved for relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) and r/r primary mediastinal B-cell lymphoma (PMBCL). CAR-T therapy is a type of personalized medicine that offers potentially curative options for patients with limited treatment alternatives. This study evaluated the cost effectiveness of axi-cel as a third-line therapy compared with standard care (SoC) in Switzerland.

A cost-utility analysis was conducted using a healthcare payer perspective. Only direct medical costs in 2023 CHF were considered. Health outcomes were measured in life years and quality-adjusted life years. Both costs and outcomes were discounted at three percent per annum. A lifetime horizon was used to capture differences in costs and outcomes. A hybrid decision tree and three-state partitioned survival model simulated outcomes using survival data from single-arm studies and extrapolated Kaplan-Meier curves. Comparators included SoC options such as salvage chemotherapy. Sensitivity analyses explored uncertainty related to survival assumptions, time horizons, and product costs. The research project was funded by the Swiss Federal Office of Public Health.

Axi-cel demonstrated significant long-term survival benefits for DLBCL and PMBCL, compared with SoC, with improved overall survival and progression-free survival. However, the high costs of CAR-T therapies, particularly the product itself, posed substantial economic challenges. Adverse events such as cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome were prevalent and contributed to treatment costs. Sensitivity analyses confirmed the cost effectiveness was highly dependent on survival extrapolations and pricing assumptions.

Axi-cel provided substantial survival benefits in DLBCL and PMBCL; however, its high economic burden necessitates careful cost management and monitoring. Due to limited comparative evidence, naïve comparisons were relied upon to estimate the incremental benefit of axi-cel, introducing high levels of uncertainty into the results. Further research should refine long-term survival projections and optimize pricing models to enhance affordability. These findings guide healthcare policy on axi-cel reimbursement and adoption for r/r DLBCL and PMBCL.

Patient involvement is a cornerstone of the Brazilian Unified Health System (SUS), legitimizing decisions on the reimbursement of new health technologies. However, the complexity of these processes hinders public engagement. To overcome this, the Participa SUS/ATS project developed a gamified digital platform to educate laypeople about health technology assessment (HTA), empowering them with knowledge to engage effectively in public consultations.

The platform has individualized access and comprises ten modules of educational videos and interactive games. Joana, a fictional SUS user, embarks on a journey reflecting common patient experiences, learning about the health system, HTA, and technology reimbursement processes. Content was informed by research into challenges faced by patient associations and validated through online questionnaires with pilot users. Videos used simplified language, illustrations, and Brazilian Sign Language (Libras) to enhance accessibility. Games reinforced learning by aligning with Joana’s narrative. Upon completing modules, users receive certificates. The approach prioritized inclusivity, creating an engaging and relatable environment for diverse learning needs.

Gamification, centered on Joana’s relatable journey, effectively engaged SUS users. Participants reported improved understanding of HTA concepts. Evaluative games reinforced knowledge, and over 300 certificates were issued upon module completion, marking participant progress. Joana’s narrative resonated deeply, fostering accessibility and identification. Inclusive features, such as Libras, simplified language, and illustrations, broadened engagement and reflected Brazil’s socioeconomic and cultural diversity. The project demonstrated that narrative-driven gamification is a powerful tool for translating complex technical topics into practical knowledge, empowering lay audiences to participate actively in decision-making processes.

The Participa SUS/ATS platform demonstrates that gamification, combined with relatable narratives, can demystify technical topics like HTA, fostering informed public participation. This initiative enhanced social participation in the SUS, empowering users to contribute actively and knowledgeably to decision-making. This model can be adapted to other topics, broadening its impact by promoting citizenship and fostering engagement in public health initiatives.

Brazil faces a triple burden of disease—infectious diseases, chronic non-communicable diseases, and external causes—that demand effective public health policies and access to the healthcare system. Analyzing the health technology assessment process allows us to determine whether the health needs of the Brazilian population are being met by new technologies or whether they contribute to increasing health inequalities.

This descriptive, exploratory study was based on an evaluation of the National Committee for Health Technology Incorporation (CONITEC) recommendations on technologies from 2012 to 2023, carried out year by year using the burden of diseases reference. The aim was to evaluate and classify each recommendation according to the triple burden of disease groups, such as infectious diseases (group one), chronic non-communicable diseases (group two), and external causes (group three), plus one more (group four) that was adapted for this study from group one to emphasize neglected tropical diseases (NTD).

Each of CONITEC’s 822 recommendations was evaluated and classified into one of the groups: 151 technologies for group one, especially HIV/AIDS, hepatitis, tuberculosis, COVID-19 and life cycles (18% of total); 645 technologies for group two, especially in the medical specialties of oncology, rheumatology, neurology, pulmonology, medical genetics, and cardiology (78% of total) and 41 percent of which were for rare diseases; 10 technologies for group three; and 12 technologies for group four, covering only four diseases (leprosy, leishmaniasis, Chagas disease, and dengue fever).

Although 18 percent of CONITEC’s recommendations included priority infectious diseases in Brazil, less than two percent related to NTD. For chronic diseases, 41 percent were for rare diseases, without treatment for a long time, but with a high budgetary impact that affects the financial sustainability of the SUS. This may reflect the interests of manufacturers rather than health policies.

Rare diseases affect up to 65 per 100,000 individuals, presenting challenges for health technology assessment (HTA). Key issues include evidence quality and incremental cost-utility ratio (ICUR), which influence decisions on incorporating treatments to assist patients with rare diseases in Brazil. This study aimed to evaluate incorporations into the Brazilian Unified Health System (SUS), considering clinical and economic aspects.

This was a retrospective study conducted from January 2020 to November 2024. Data were collected from the public dossiers available on the Brazilian Ministry of Health (MoH) website, focusing on drugs whose indications were for the treatment of rare diseases. The study included only dossiers with available information on cost-utility analyses that demonstrated incremental effectiveness and incremental cost to the healthcare system; the quality of evidence was evaluated using the GRADE tool. Dossiers with content that did not meet these criteria were excluded from the analysis.

Between January 2020 and November 2024, the Brazilian MoH received 465 technology evaluation requests, with 38 percent (n=176) resulting in decisions of incorporation or expanded use, of which 30 percent (n=53) related to rare diseases. From the analysis of these reports, n=22 matched the inclusion criteria reported above. The ICURs ranged from BRL4,287.24 (USD714.54) to BRL75,938,549.34 (USD12,656,424.90), with an average of BRL4,890,006.53 (USD29,340,039.17); of those, 68 percent exceeded the SUS cost-effectiveness threshold of BRL120,000 (USD20,000) per quality-adjusted life year. Nevertheless, the quality of the evidence was considered low in 70 percent of these evaluations, based on the GRADE tool.

Technologies for rare diseases are often incorporated within the SUS with a high ICUR despite low quality evidence. This is due to the challenging and complex nature of rare diseases, requiring in-depth discussions on adapting HTA methods to these conditions. Developing robust frameworks is essential to ensure equitable access while addressing evidence gaps and decision-making complexities.