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.

Summary measures of health, such as quality-adjusted life expectancy (QALE), are used to monitor health inequalities by reflecting both quantity and quality of life. The Australian distribution of QALE by socioeconomic status, measured using educational attainment, is unknown. We aimed to estimate QALE stratified by sex and year of age across levels of educational attainment for the Australian population.

Educational attainment was measured as: low (secondary schooling incomplete), intermediate (completed secondary schooling and/or non-tertiary qualification), or high (completed bachelor’s degree or above). The Person-Level Integrated Data Asset linked Australian Census (2016) demographic information to Deaths Registrations (2016 to 2019). Age-sex-education specific mortality rate ratios were then applied to the 2019 population. Life tables were constructed to generate life expectancy (LE). The Household, Income and Labour Dynamics in Australia Survey (2022) was used to estimate mean Short Form 6 Dimensions (SF-6D) health utility by age, sex, and education using linear regression. Person-years lived were multiplied by utility to calculate QALE.

Both LE and QALE increased with increasing level of education. Males aged 25 years with a high level of education had a LE of 61.0 years and a QALE of 39.9 (95% confidence interval [CI]: 38.7, 41.2) years, compared with 53.7 years and 28.8 (95% CI: 27.4, 30.3) years, respectively, for those with low education, a difference of 11.1 quality-adjusted life years (39%). Females with high educational attainment had a LE of 63.1 years and a QALE of 36.9 (95% CI: 35.8, 38.1) years, compared with 59.2 years and 29.3 (95% CI: 27.9, 30.8) years, respectively, for low education, a QALE difference of 7.6 years (26%).

There were large disparities in QALE, impacting both the quantity and quality of life, associated with educational achievement in Australia. Tailored policies addressing education-related inequalities in health are needed. The QALE distribution across equity relevant groups provided in this study can be utilized in future equity informative studies that evaluate the equity impact of healthcare funding decisions.

Visualase is a minimally invasive, MRI-guided system that uses laser interstitial thermal therapy (LITT) to treat patients with brain tumors and drug resistant epilepsy. Our objective was to perform an economic analysis of LITT compared with open cranial resection for patients with brain tumors, from the perspective of the Australian healthcare system and with consideration of equity for eligible patient populations.

A literature review was carried out to gather international data on clinical outcomes of LITT and open resection for patients with brain tumors, as data in Australia are very limited. Cost data were sourced from 2024 Medicare and Pharmaceutical Benefits Scheme fees and hospital costs were sourced from the 2014 Australian Critical Care Resources Survey, costed at the 2023 consumer price index equivalent. A cost-consequence analysis was carried out to calculate the incremental cost per life year (LY) gained from the perspective of the Australian healthcare system. Subgroup analysis was carried out due to heterogeneity of the patient population.

Our analysis demonstrated that the cost per LY gained was AUD87,630 (USD56,318) for recurrent primary brain tumors, AUD24,630 (USD15,828) for recurrent brain metastases, and AUD15,948 (USD10,249) for radiation necrosis. Considering a willingness to pay threshold of approximately AUD50,000 per LY (USD32,131/LY), LITT could be cost effective for some patient groups.

The minimally invasive nature of LITT meant that hospital stay was reduced from an average of four days to one day, potentially increasing hospital throughput and reducing waiting lists. The model could be further refined with inputs from patients in the Australian setting and consideration of equity, when such data become available.

From a payer perspective, LITT can be a cost-effective option in Australia for certain patient groups. The full economic value to society could be demonstrated by quantifying the utility of patient preference for minimally invasive procedures, and the benefits of patients and carers returning to school or work. Equity considerations or the rule of rescue may be applicable for eligible patient populations.

Brazil’s health-related litigation has highlighted the need for effective communication between health technology assessment (HTA) and legal professionals. The Hospital Sírio-Libanês Center of HTA (NATS-HSL) in Brazil collaborates with the National Council of Justice to provide capacity building initiatives. This study aimed to present the development of an HTA glossary tailored to judges and legal staff.

The project followed a Design Thinking methodology with five stages: empathize, define, ideate, prototype, and test. Initially, NATS-HSL conducted an electronic survey of a sample of Brazilian judges to identify preferred terminology and glossary format (empathize stage). During the define and ideate stages, challenges and gaps in interpreting scientific findings were analyzed. The prototype glossary will be produced in print, digital, and PDF formats. These versions will be distributed to legal professionals for review, and their feedback will guide subsequent revisions before implementation.

Sources of Support: Program to Support the Institutional Development of the Unified Health System (PROADI-SUS), Ministry of Health, NUP: 25000.175715/2023-41.

The survey was answered by 144 participants through to November 2024. Among them, 99 (69%) selected an alphabetical glossary format. The most favored formats were online PDF (n=63; 44%) and website (n=50; 35%). Most respondents stated the importance of brevity, the inclusion of examples and “learn more” links (n=97; 67%), the use of informal language (n=93; 65%), and comprehensive terminology (n=90; 63%). The terms most frequently suggested were related to medicines, cost-effectiveness analysis, technical terms, and economic evaluations. The pilot glossary currently contains 210 terms.

The next stages of the glossary development process are prototype distribution, feedback gathering, re-evaluation, and implementation of the final version. This knowledge translation initiative aims to enhance the capacity of legal decision-makers to interpret HTA reports. A better-informed judicial decision may contribute to the sustainability of the healthcare system.

Mexico has an institutionalized health technology assessment (HTA) process. However, structural changes in the system and new trends make it necessary to rethink strategies for strengthening and institutionalizing HTA. For this reason, the National Center for Technological Excellence in Health (CENETEC), through the Inter-institutional HTA Working Group, in which the main institutions of the sector participate, coordinates sectoral efforts and has implemented an instrument to collect information to achieve this objective. This study aimed to provide a comprehensive overview of current HTA practices in Mexico to inform future capacity building initiatives within the health system.

The study used a structured survey to assess the state of HTA across healthcare institutions in Mexico. The survey included 115 participants from all institutions and diverse fields, including medicine, biomedical engineering, economics, and health sciences. The survey questions addressed awareness, implementation, and perceived gaps in HTA. Data collection focused on institutional participation, existing frameworks, and areas requiring improvement. Responses were analyzed to identify trends, challenges, and opportunities for HTA enhancement.

The survey received 115 responses, predominantly from medical professionals, researchers, and economists. Findings revealed widespread awareness of HTA but noted limited institutional implementation. Key challenges included insufficient training, lack of sensitization among decision-makers, and the need for defined expert groups in health institutions. Notable areas requiring focus were cost effectiveness, clinical safety, and broader societal impacts. Participants emphasized the importance of developing consistent HTA methodologies and fostering collaboration across institutions. Despite challenges, the study highlighted significant interest and expertise among stakeholders, providing a solid foundation for advancing HTA practices in Mexico.

The study underscored the importance of strengthening HTA practices in Mexico. While awareness of HTA exists, consistent implementation and capacity building are needed to optimize decision-making. Developing expert groups and enhancing training among stakeholders are crucial steps. These findings offer a roadmap for institutional collaboration and the advancement of equitable, evidence-based HTA frameworks to improve Mexico’s healthcare outcomes.

Health priority setting is a complex process, especially in governmental decision-making, for improving access and addressing unmet needs. To review its technological roadmap, Bio-Manguinhos (BM)/Fiocruz has improved its strategy. The objective of this approach was to incorporate research institutions and medical and scientific experts into the technology analysis processes of a public pharmaceutical organization.

The first stage of this roadmap review consisted of workshops in the areas of pneumology, infectious diseases, oncology, hematology, gastroenterology, dermatology, rheumatology, cardiology, and neurology. At each workshop, a scientific leader, researchers, and specialists discussed the main diseases and their short-, medium- and long-term health demands. These observations were translated into products and lines of health research by BM’s technical team. Epidemiological data were collected, and in-depth analysis was conducted that considered the type of technology, development status, and correlation with existing public policies. The products and lines of research were categorized, and a database was built for further examination in subsequent stages.

The 83 invited specialists highlighted 1,045 demands, of which 56 percent were long term, 39 percent short term, and 5 percent medium term. Market available products comprised 36 percent, while 51 percent were innovative solutions. Biotechnological demands represented 65 percent of the total, with 43 percent being biopharmaceuticals. Of those, 35 percent pertained to in vitro diagnostics, nine percent to vaccines, and 13 percent to other technologies. Monoclonal antibodies stood out at 24 percent. Cellular therapies made up six percent, gene therapies two percent, chimeric antigen receptor T-cell therapies two percent, and antibody-drug conjugates one percent. Rheumatology had the highest number of biotechnological demands and accounted for 24 percent.

The importance of recognizing genetic and economic patterns in the diseases that most affect the population, with a focus on personalized medicine, was consistently emphasized. Health demand assessment is a form of social participation in the decision-making process. Combined with other data sources, it allows for integrated and synergistic mapping to analyze existing biotechnological products and innovative solutions.

Post-prostatectomy urinary incontinence (PPUI) greatly impacts self-esteem and social, work, and sexual activities. Artificial urinary sphincter (AUS) is the gold standard treatment for severe PPUI according to medical societies, the National Institute for Health and Care Excellence (NICE) and the Haute Autorité de santé (HAS). In Brazil, AUS has been reimbursed in the supplementary health sector since 2014, but it is not yet available in the Brazilian Unified Healthcare System (SUS).

A search of the PubMed, Embase, LILACS, and Cochrane Library databases was conducted for literature published through to September 2024. This retrieved 1,480 titles (duplicates excluded), of which 75 were read in full. The study selection criteria included patients with severe PPUI and a comparator of no treatment or pelvic physiotherapy, the only options available in the SUS to date. Risk of bias and quality of evidence by outcome were assessed using the ROBINS-I (observational studies), AMSTAR-2 (systematic reviews), and GRADE tools accordingly. Studies with less than 100 participants adhering to the proposed PICO population were excluded. The results were critically analyzed.

Two systematic reviews and four observational studies were selected. AUS significantly reduced pads-per-day use in patients with severe PPUI, with 43.5 to 58 percent of patients reaching total continence (zero pads/day) and 79 to 83 percent reaching social continence (up to 1 pad/day). The International Consultation on Incontinence Questionnaire-Urinary Incontinence Short Form (ICIQ-SF) score ranged from 20 before to five after the procedure (21 being the worst health status). For Incontinence Quality of Life Questionnaire (IQoL) score, patients reached 86.4 after AUS implantation, compared with 35.5 before (100 being the best health status). GRADE indicated moderate quality evidence for all outcomes. Only one study had critical risk of bias related to the period in which it was undertaken.

As pelvic physiotherapy has been demonstrated to be ineffective for severe PPUI, no head-to-head studies comparing it with AUS have been or are expected to be conducted. Therefore, making AUS available in the SUS is not just filling an important treatment gap, it is a big step toward healthcare access equity between patients treated in the private sector and the public health system in Brazil.

Individuals with moderate to severe sensorineural hearing loss (SNHL), who are unable to use conventional hearing aids (CHA) for medical reasons, have no publicly financed treatment options in Brazil. This study aimed to evaluate the cost effectiveness of active middle ear implants (aMEI) for Brazilian adults with this health condition.

A Markov model with cohort simulation over lifetime was developed. To populate the model, a systematic review was conducted to identify implant-related complications and utilities. Cost of aMEI was based on the price proposed for public reimbursement. Pre-, peri-, and postoperative costs were obtained from the Brazilian public healthcare system list of procedures (SIGTAP). Costs of untreated SNHL in Brazilian adults were obtained from the World Health Organization report (2015) and adjusted for 2024 BRL using purchasing power parities (PPP) for gross domestic product (GDP). Probabilistic sensitivity analyses (PSA) were performed to evaluate the robustness of the results. Cost results were presented in 2024 BRL and USD (conversion using PPP for GDP).

The base case result showed that, over a lifetime, the expected cost of aMEI treatment was BRL62,177 (USD26,951), compared with BRL5,806 (USD2,517) in an untreated adult. The expected quality-adjusted life years (QALYs) for a patient treated with aMEI was 10.53, compared with 9.03 in a patient left untreated. The incremental cost-effectiveness ratio was BRL37,699 (USD16,341) per QALY gained, which is cost effective considering the current willingness-to-pay threshold adopted in Brazil of one GDP per capita (BRL55,400 [USD24,014] in 2024). PSA showed that results of the intervention were robust and cost effective in most of the simulations.

aMEI is a cost-effective hearing solution for adults with moderate to severe SNHL in Brazil considering the perspective of the healthcare system. Assuring the access of aMEI to adults with moderate to severe SNHL who are unable to use CHA for medical reasons is important to promote health equity in Brazil.

Health technology assessments (HTAs) demand exhaustive evidence retrieval, encompassing both peer-reviewed research and often elusive and poorly indexed grey literature, to guide informed healthcare decisions. This case study investigates Perplexity, a generative artificial intelligence (AI)-powered search engine, as a novel tool for uncovering diverse, often overlooked grey literature sources crucial for comprehensive HTA.

Iterative searches in Perplexity were conducted utilizing a diverse range of tailored prompts to identify evidence sources, including organizational reports, guidelines, epidemiological data, statistical analyses, and white papers. Search results were evaluated for relevance and uniqueness, with comparisons made against traditional retrieval methods. Feedback from participating information professionals provided additional insights into Perplexity’s strengths, limitations, and overall usability. Furthermore, the characteristics of effective prompt engineering strategies were explored and methods for integrating Perplexity into evidence retrieval workflows for HTA were examined.

The results obtained through the Perplexity approach revealed several unique sources that were not identified using traditional search methods. Furthermore, the time required to locate and retrieve these distinctive sources was significantly reduced, making the process more efficient while broadening the scope of information gathered.

This study showcased Perplexity’s potential to transform gray literature searches for HTAs. By efficiently uncovering diverse, hard-to-find sources, including those often missed by traditional methods, Perplexity enhanced both relevance and uniqueness, while also saving significant time in the search process. Insights from this study will underscore its value in optimizing evidence retrieval workflows, making HTAs more comprehensive, efficient, and impactful.

Bevacizumab combined with chemotherapy is used in the first-line treatment of advanced non-small cell lung and metastatic colorectal cancer. Bevacizumab biosimilars have been developed and marketed after the expiration of the reference bevacizumab patent. Equivalence studies have demonstrated that biosimilars are comparable with their reference medicine, but synthesis of the effects and safety is not available to improve clinical decision-making.

We performed a Cochrane systematic review and searched main bibliographic (CENTRAL, MEDLINE, Embase, Web of Science) and clinical trials databases up to February 2024. We included randomized controlled trials that directly compared adults with non-small cell lung or colorectal cancer receiving either bevacizumab biosimilars or the reference product. When applicable, we combined the results for outcomes using random-effects meta-analyses, including progression-free survival, duration of response, overall survival, mortality, objective response rate, and serious adverse events. We applied the revised Cochrane risk-of-bias tool. To assess the certainty of evidence for critical and important outcomes, we utilized the GRADE approach.

We included 23 studies (n=10,639 participants). Most had a high risk of bias due to issues with discontinuation and patient selection. The bevacizumab biosimilar was likely similar to the reference product in progression-free survival (hazard ratio [HR] 1.00, 95% confidence interval [CI]: 0.91, 1.09; 5 studies, n=2,660), duration of response (HR 1.05, 95% CI: 0.81, 1.37; 1 study, n=762), overall survival (HR 1.06, 95% CI: 0.94, 1.19; 5 studies, n=2,783), mortality (RR 1.03, 95% CI: 0.97, 1.09; 19 studies, n=9,231), objective response (risk ratio [RR] 0.96, 95% CI: 0.93, 1.00; 23 studies, n=10,054), and serious adverse events (RR 0.98, 95% CI: 0.93, 1.03; 23 studies, n=10,619). The certainty of evidence for all outcomes was moderate, mainly due to imprecision.

Moderate certainty evidence indicated that bevacizumab biosimilars are likely similar to the reference biologic drug in terms of efficacy and safety.