Hemophilia A (HA) results from mutations in the F8 gene, leading to a deficiency in factor VIII (FVIII) and consequent spontaneous bleeds. Treatment options include bleeding prophylaxis with FVIII and, more recently, emicizumab (EMI), a FVIII-mimetic bispecific antibody. EMI was introduced into the Brazilian Unified Health System in 2019 for people with HA and inhibitors (PwHAi). This study compared EMI costs with prior prophylactic methods and evaluated its budgetary impact against government projections.

The National Registry of People with HA Using Emicizumab in Brazil (EMCase) is a prospective observational study initiated in 2020, conducted in 16 Brazilian hemophilia treatment centers. The analysis included consumption of FVIII, bypassing agents, and EMI. Treatment costs for the pre-EMI period and the first year of EMI use were calculated using prices paid by the Ministry of Health, as reported in the Brazilian Health Price Database. Yearly average treatment costs per kilogram of body weight were compared with government budget projections.

A total of 56 moderate to severe PwHAi were included. Before EMI treatment, the actual treatment costs were BRL31,145.19 (USD5,190.87) per kg per year. During the first year of EMI prophylaxis, the actual treatment costs were BRL22,583.79 (USD3,763.96) per kg per year, representing approximately 27.5 percent reduction in costs, compared with the previous period. In addition, the government projected cost for 2019, adjusted for inflation, was estimated at BRL19,736.84 (USD3,289.47) per kg per year for the first year of EMI use, resulting in a 14.4 percent increase in the projected budget impact.

Although the actual cost during the first year of EMI prophylaxis was higher than the government project cost, a cost reduction was observed in comparison with the pre-EMI period cost. The study highlighted the importance of real-world data-based economic analyses in the evaluation of health technologies, complementing government projections to inform decision-making.

Rare diseases, including Gaucher disease and spinal muscular atrophy, impose significant challenges in access to treatments and equitable healthcare policies. In Ecuador, patients and caregivers lack representation in health technology assessment (HTA) processes. This study aimed to amplify their voices, exploring their experiences to develop inclusive methodologies that promote equity and transparency in healthcare decision-making.

This study employed a mixed-methods approach combining qualitative and quantitative data comprising perspectives of patients with Gaucher disease and spinal muscular atrophy (as well as the perspectives of their caregivers), in-depth interviews, surveys, and observation. Collaboration occurred with patient organizations. Qualitative analysis focused on thematic coding of experiences, while quantitative surveys assessed access to treatment and socioeconomic burdens. Data triangulation ensured robust findings.

Preliminary results indicated significant challenges in equitable access to treatments for rare diseases in Ecuador. Patients and caregivers reported high emotional and financial burdens, with limited systemic support. Early findings suggested disparities in treatment availability and awareness between urban and rural settings. Engagement workshops demonstrated a strong willingness among stakeholders to participate in developing inclusive HTA processes.

Preliminary findings highlighted the urgent need for inclusive HTA processes in Ecuador. Amplifying patient and caregiver voices can improve equity in rare disease management and inform policy changes. Future research will focus on refining methodologies for participatory HTA and addressing systemic gaps in access to treatment, with emphasis on rural and vulnerable populations.

Since 2023, the Agency for Care Effectiveness (ACE) has included lived experiences from patients and caregivers in health technology assessments (HTAs) for wearable or home-based medical devices. This presentation discusses the impact of patient experience data on the HTA and subsequent subsidy recommendations for continuous glucose monitoring (CGM) systems for type 1 diabetes mellitus (T1DM) in Singapore.

All local patient organizations with an interest in CGM systems or T1DM were invited to share their views using a structured, qualitative survey or simple free-text patient journey form. Testimonials were analyzed by ACE to extract key themes and insights and incorporated into the HTA report alongside clinicians’ input and clinical and economic evidence to inform subsidy recommendations made by the Ministry of Health Medical Technology Advisory Committee. The impact of patient experience data on the Committee’s deliberations and recommendations for CGM systems was determined by reviewing the HTA report, minutes from Committee meetings, and the published HTA guidance document.

Eighty testimonials were received from patients and caregivers about the impact T1DM has on their lives, and their experiences using different blood glucose monitors. They also confirmed real-world CGM usage across different age groups, informing assumptions for budget impact calculations. Testimonials highlighted benefits and drawbacks of different monitoring methods, unmet needs, patient preferences, and expectations for new technologies to be more affordable and less invasive. While scientific evidence for children with T1DM was limited, testimonials confirmed CGM systems could improve outcomes for both adults and children, which influenced the subsidy criteria recommended by the Committee to cover both age groups.

Incorporating patients’ lived experiences in ACE’s HTAs validated and strengthened the scientific evidence, addressed uncertainties by filling data gaps, and led to patient-centered subsidy recommendations for CGM systems, demonstrating the value of systematic patient involvement. Post-recommendation, ACE co-developed a factsheet on CGM systems with local patient organizations to explain the subsidy recommendations in plain language and encourage HTA guidance adoption.

Monitoring elderly health via wearable electronic devices offers significant benefits but faces ethical and regulatory challenges. While these devices are useful in emergencies, such as falls, concerns about user privacy and data confidentiality persist. This study aimed to develop recommendations for developers of wearable electronic devices used in elderly health monitoring, as well as for other stakeholders, focusing on privacy and confidentiality.

A scoping review was performed by searching databases including MEDLINE (PubMed), Embase, Scopus, the Cochrane Library, Web of Science, Epistemonikos, and the Virtual Health Library through to October 2023. Studies were included if they addressed all the following criteria: (i) wearable electronic devices; (ii) elderly individuals; (iii) health monitoring; and (iv) privacy, confidentiality, or data protection. Studies that did not include recommendations for developers or other stakeholders aimed at enhancing user privacy protection and data confidentiality were excluded. The results were presented as a narrative synthesis.

A total of 1,037 records were identified in the databases, and 12 studies published between 2014 and 2022 were included. The main findings were addressed in topics, including: (i) privacy and confidentiality in monitoring the health of elderly individuals using wearable electronic devices; (ii) barriers and facilitators for the adoption of wearable devices in monitoring the health of elderly individuals; and (iii) recommendations for developers of wearable devices in monitoring the health of elderly individuals and for other stakeholders.

The use of wearable electronic devices for health monitoring in elderly individuals presents complex ethical, regulatory, and technological challenges, particularly regarding privacy and confidentiality. Collaboration between developers and stakeholders is essential for implementing robust security measures and establishing effective regulatory standards that ensure comprehensive user protection.

Scientific advice (SA) in the process of developing medical devices and technologies (MedTechs) allows manufacturers to target the profile of their production and gain a better understanding of the requirements for approval and reimbursement. The aim of this study was to evaluate the SA framework developed by National Evidence-based Healthcare Collaborating Agency (NECA) in Korea.

We developed the SA program by reviewing all available information regarding the service offered, processes, duration (in weeks), stage of development, types of technologies, and fees. The SA program supports a total of 60 MedTechs per year. By the end of 2023, a total of 150 technologies had participated. We also evaluated participant satisfaction with the SA program after their participation.

The NECA SA program offers Evidence Preview, Evidence Search Education, Evidence Development Plans with regulatory advice, and Clinical Expert Advice for clinical trial design. The process is flexible, depending on the applicant’s needs, and is completed within 24 weeks, with a final report issued after completion. Stage of development and type of technology are mainly focused on early stages of development (110/150 technologies) and innovative technologies (87/150 technologies). Participant satisfaction with the SA program was more than 90 percent for the past three years. The SAs offered by NCEA are optional, free of charge, confidential, and non-binding.

This framework helps manufacturers understand the evidence requirements for NECA and other payers. Considering that most MedTechs are in the early stages of development, it is important to develop a target technology profile (TTP) tool to prepare guidelines for MedTech development plans. In addition, there is growing international collaboration between industry and regulatory authorities. It may also be beneficial to engage with specialized consulting institutions.

Therapeutic ranking (TR) is a process used in health technology assessment (HTA) by the Institute for Health Technology Assessment (IETS) in Colombia to optimize drug prescription through analyses of effectiveness, safety, costs, and post-marketing surveillance. However, decision-making relying on clinical expert preferences may introduce biases, since they are often influenced by subjective values. This study presented an alternative methodology by IETS that emphasizes a structured, evidence-based approach to improve decision-making in TR.

The standard TR process involved forming an interdisciplinary group to assess the effectiveness, safety, costs, and post-marketing surveillance of medications, followed by evidence synthesis and informed voting using the Borda method to prioritize technologies based on consensus-driven preferences. To enhance informed voting, a methodological strategy was introduced that incorporated structured comparative tables and relative position scales derived from specific quantitative analyses for each parameter (e.g., surface under the cumulative ranking curve [SUCRA], costs, number of adverse events). This approach visually displayed performance patterns for integrated analysis by clinical experts.

This adaptation to TR consolidated the results for effectiveness, safety, costs, and economic evaluations of the assessed technologies. The structured format facilitated the identification of performance patterns by technology and enabled the integration of these results into a visual framework that supported deliberations during informed voting. This methodology promoted the integration of multiple evaluated dimensions, ensuring that inputs from epidemiology, pharmacology, health economics, and other specialized analyses were not overshadowed by the clinical experience of the expert panel. Instead, each input contributed to the collective decision-making process.

This methodological approach enhanced TR as an HTA process by fostering evidence-based decisions. Optimizing the process could involve assigning weights to each analysis component, including clinical expertise, to balance their influence on prioritization. Explicit weighting would improve transparency and reproducibility. This model is replicable in other settings, maximizing HTA’s impact on global health systems.

Neonatal sepsis remains a major cause of morbidity and mortality in neonatology. While blood culture is considered the gold standard for detecting sepsis, its low sensitivity significantly limits its utility. The identification of biomarkers, such as procalcitonin (PCT), is essential for improving diagnostic accuracy. This Scientific Technical Opinion critically evaluated the available evidence on the role of PCT in diagnosing neonatal sepsis and clinical practice.

This study followed Joanna Briggs institute and Cochrane Collaboration guidelines and was registered with PROSPERO. Systematic searches were conducted in Embase and MEDLINE. Eligible studies included randomized, quasi-randomized, cohort, case-control, observational, and cross-sectional designs that evaluated PCT diagnostic accuracy for neonatal early or late sepsis, microbiologically or clinically documented. Studies had to perform testing at the onset of sepsis symptoms, prior to antimicrobial therapy. Data collected included true positive, true negative, false positive, and false negative results. Sensitivity and specificity meta-analyses were performed, and summary receiver operating characteristic curves were generated using Meta-DiSc 2.0 software, with results reported as 95 percent confidence intervals (CIs).

A total of 111 studies were analyzed, involving 19,175 neonates. The sensitivity of PCT in diagnosing sepsis was 0.787 (95% CI: 0.757, 0.815) and the specificity was 0.853 (95% CI: 0.822, 0.879). In the “time of collection” group analysis, we obtained the following results: at zero hours, sensitivity was 0.753 (95% CI: 0.695, 0.802) and specificity was 0.803 (95% CI: 0.739, 0.854); up to 12 hours, sensitivity was 0.912 (95% CI: 0.748, 0.973) and specificity was 0.827 (95% CI: 0.643, 0.927).

The findings suggested that PCT demonstrates high diagnostic accuracy for neonatal sepsis. Thus, incorporating PCT could enhance diagnostic precision and help minimize the unnecessary use of antimicrobials, ultimately supporting better outcomes in antimicrobial stewardship programs within neonatal intensive care units.

The dynamic nature of the prehospital environment poses a challenge for maintaining optimal storage conditions for medicines and other products. Failure to ensure adequate temperature control can impact drug efficacy, potentially compromising effective prehospital care. This study aims to monitor temperature variations in vehicles operated by a helicopter Emergency Medical Service (EMS) in the east of England to evaluate temperature variation and the potential exposure of therapeutic products to extreme temperatures.

This was a prospective observational study over a 12-month period (September 2022 through August 2023). ALTA Industrial Wireless temperature sensors were deployed across five Volvo XC90 rapid response vehicles (RRVs) and an AgustaWestland 169 (AW169) helicopter operated by Essex & Herts Air Ambulance (EHAAT). One RRV (RRV02) remained permanently outside for the period of observation. Sensors recorded at 30-minute intervals, triggering alerts if temperatures exceeded 25°C or dropped below 2°C. Ambient data were obtained from the Writtle Weather Station, via the Met Office Integrated Data Archive System (MIDAS). The study did not involve any patients and ethical board review was not required.

A total of 102,524 readings were recorded; temperatures ranged from −9°C to 46.8°C. The RRV02 recorded the lowest monthly mean (7.9°C) while the AW169 recorded the highest (24.9°C). Overall, daily maximum temperatures ranged from 17.7°C to 46.8°C, with the AW169 reaching 46.8°C in May. Daily minimum values ranged from −9°C to 19.6°C, with the RRV02 recording −9°C in December.

Temperatures inside the vehicles and aircraft frequently exceed recommended limits for environmental temperatures. Indoor RRV storage provides some protection from extreme temperatures, particularly mitigating against low temperatures. The AW169 aircraft demonstrated the poorest overall temperature control. These results can be used to guide effective storage protocols and climate control strategies to ensure the integrity of therapeutic products.

Countries face the challenge of advancing access to innovative health technologies. There is growing interest in managed entry agreements (MEAs) in Latin America, but very few publications. A recent cross-country comparison of Argentina, Brazil, Chile, Colombia, Costa Rica, Ecuador, Mexico, and Peru analyzed state-of-the-art MEAs in these settings. After collecting information from 69 relevant stakeholders through focus groups, interviews, and surveys, a series of recommendations for MEA scalability were provided.

Qualitative methods were used. A desk review of recent regulations and publications of successful MEAs in the eight countries of interest was performed, followed by eight focus groups with 63 key informants. Also, three key informants from local authorities and three representatives from the local pharmaceutical federations of Argentina, Brazil, and Chile were invited to participate in a survey. The qualitative methods used served to provide relevant information to inform a set of recommendations.

In total, 69 key informants participated in the study. The level of knowledge and understanding of the use and limitations of MEAs was moderate to high in all countries. There seemed to be positive or moderate political will and policy commitment to implementing MEAs in the region.

The most common barriers to implementing MEAs were a lack of clear rules, roles, and responsibilities; the lack of enabling legal and policy frameworks; and the scarcity of available information on successful implementation of MEAs in neighboring countries.

The Regional Database of Health Technology Assessment Reports of the Americas (BRISA) provides free access to bibliographic information on health technology assessment (HTA) prepared and published by member institutions of the HTA Network of the Americas (RedETSA) and strategic partners. BRISA is the first open regional virtual library that centralizes HTA reports which would otherwise be scattered or inaccessible to the public.

BRISA was developed in collaboration with the Latin American and Caribbean Center on Health Sciences Information (BIREME) through the Virtual Health Library. The platform and the methodology for registering, organizing, and disseminating reports were developed by BIREME, in coordination with the Pan American Health Organization. HTA reports are shared in Spanish, Portuguese, French, and English. A retrospective analysis of BRISA’s development and achievements since its launch was conducted. The analysis included the number of countries and institutions providing HTA reports, the number of HTA reports published over time, the number of users per country, and the total number of page views since its creation.

Since its launch in 2017, the number of contributors to BRISA has reached 14 countries and more than 40 institutions. BRISA is constantly growing and in 2024, it reached more than 4,000 reports. In 2024, Brazil, Mexico, and Argentina had the highest number of users (with 16,620, 12,750, and 9,338 users, respectively). The number of page views increased from 4,833 in 2018 to more than 840,000 in 2024. Since 2024, BRISA also includes HTA reports from partner networks like the Spanish Network of Agencies for HTA and Services of the National Health System (RedETS) in Spain.

BRISA is a collaborative network that facilitates learning from the experiences of other countries, helps avoid duplication of effort, and promotes the incorporation of cost-effective health technologies and their appropriate use. With the BRISA database, RedETSA seeks to promote the use of HTA to improve the decision-making process for incorporating technologies in health systems, improving access and equity.

One way of communicating the effect of interventions is by converting relative effects, such as relative risk, into absolute effects. Guidelines from the GRADE working group and the Cochrane Handbook for Systematic Reviews of Interventions recommend this conversion for summarizing and concluding evidence syntheses. However, this conversion can lead to errors, and previous research has indicated that inadequacies are frequent in Cochrane reviews that convert hazard ratios to absolute effect measures.

A descriptive study outlined the development of the abseff program, created using the ado programming language of the Stata software and R. This program was developed in the context of two projects conducted by the Center of Health Technology Assessment, Hospital Sírio-Libanês, São Paulo-SP, Brazil in collaboration with the Ministry of Health, the National Supplementary Health Agency, and the National Council of Justice through the Support Program for Institutional Development of the Unified Health System (PROADI-SUS). Both projects involve drawing up evidence summaries, and the abseff program aims to convert relative effect into corresponding absolute effect measures.

Abseff converts data on relative effects (relative risks, odds ratios, hazard ratios) into absolute effects, including 95 percent confidence intervals (CI). Researchers input a relative effect and its confidence interval with a baseline risk for the calculation. For example, the command abseff rr 0.83 0.63 1.03 50 130 converts a relative risk of 0.83 (95% CI: 0.63, 1.03) with a baseline risk of 50/130 (38%). The result is presented in absolute anticipated effects and absolute risk difference, and calculations account for bases of 100 and 1,000. The program is freely available via Stata and R. For more details access https://rlpacheco.github.io/abseff/; https://github.com/rlpacheco/abseff_R.

Effective communication of evidence can be challenging, and translating scientific knowledge can be facilitated by presenting absolute effect measures to the decision maker. The abseff program standardizes these conversions, minimizing errors and ensuring reproducibility in analyses. Originally created as part of two projects funded by PROADI-SUS, abseff is now available for use by all health technology assessment researchers.

Life cycle health technology assessment (LC-HTA) refers to continuous methodologies used to evaluate, reassess, and de-adopt technologies. In LC-HTA, the consequences of using a technology are assessed throughout its life cycle, from development to obsolescence. This study examined the experience of the National Committee for Health Technology Incorporation (CONITEC), the Brazilian Health Technology Agency, in applying LC-HTA at various stages of the health technology life cycle.

This study analyzed CONITEC’s implementation of the LC-HTA by reviewing publicly available recommendation reports and other data published by the Committee. A systematic analysis of these documents was conducted to identify and categorize activities performed across the health technology life cycle: pre-commercialization, post-commercialization, and disinvestment. The analysis aimed to identify when each LC-HTA activity was first implemented and to understand how these processes are operationalized within the Brazilian public health system.

CONITEC has implemented LC-HTA activities across various life cycle stages, including pre-commercialization, post-commercialization, and disinvestment. Since 2014, early awareness syntheses have evaluated technologies with potential health system impacts or emerging technologies for priority diseases. Horizon scanning identifies new and emerging technologies that could compete with the technology under evaluation by CONITEC and has been included in every report since 2017. Implementation monitoring, consistently conducted since 2020, prioritizes technologies with uncertainties regarding safety, effectiveness, or economic outcomes. Disinvestment activities, initiated in 2013, target technologies no longer used in the Brazilian public healthcare system, optimizing resources and improving decision-making.

LC-HTA activities were independently implemented by CONITEC, but not all health technologies are routinely evaluated across all phases of their life cycle. LC-HTA implementation is most beneficial in specific contexts, such as during a public health crisis (e.g., a pandemic) or for technologies deemed strategically important for the country. However, each phase-specific activity has yielded valuable results, contributing to evidence-based decision-making.

The New Zealand health system provides universal health coverage to a population of 5.2 million, delivered through a range of public, private, and voluntary providers. New Zealand has extensive experience applying health technology assessment (HTA) to pharmaceuticals and vaccines. These models could potentially be adapted and applied to innovative precision health technologies that offer substantive potential health and equity benefits for patients and whānau (families), and health system financial sustainability.

The New Zealand Ministry of Health developed a Long-Term Insights Briefing on Precision Health to highlight future trends, risks, and opportunities represented by genomics and artificial intelligence-enabled technologies. The briefing was developed collaboratively with consultation with patients and the public, including deliberative consultation with Māori and Pacific peoples. Organizational input was provided from across the sector including from government agencies, clinicians, researchers, industry, and patient organizations. The briefing was structured around key themes identified through engagement.

The briefing identified multiple themes with important implications for any future design and approach to the conduct and delivery of HTA for genomic and artificial-intelligence-enabled technologies in New Zealand. Core issues raised by stakeholders included the need for sustained and coordinated investment pathways, societal-wide consideration of the impact of new technology adoption, specific incorporation of multiple dimensions of equity, and consideration of impacts on the efficiency and financial sustainability of the health system. The importance of maintaining data sovereignty was a major element of the briefing, including the conduct of research and collection and use of information.

This policymaker-led session detailed the incremental progress New Zealand has made toward realizing and managing the opportunities and risks of precision health. HTA processes and methodology are useful tools that may be adapted to ensure appropriate consideration of identified objectives and trade-offs associated with expanded investment and use of precision health technologies in New Zealand’s publicly funded health system.

Climate change is a global concern. Medical technology, from production to disposal, has diverse environmental impacts. Many healthcare systems use value frameworks to inform transparent decision-making. Our objective was to review how healthcare systems currently consider environmental sustainability in health technology assessment (HTA) value frameworks.

A scoping review was conducted to identify value frameworks focused on the assessment of any type of medical technology from 2000 to 2024. We examined main literature databases, HTA agency websites, and gray literature, with no language restrictions. Two researchers extracted dimensions from the surveyed frameworks. A wide range of value frameworks were included, and we surveyed whether environmental impact (broad term) was included within the dimensions of value. Value framework characteristics extracted included variables like origin, intended use, and the presence of evaluation or explicit weighting methods for a technology’s environmental impact.

Forty-eight value frameworks were identified. Sixteen percent (n=8) mentioned the environmental impact of the technology. Three were developed by HTA agencies (Australia, Canada, and the UK) and the rest were from other stakeholders in Europe, Latin America, the USA, and a global alliance. Except for two frameworks focused on diagnostic technologies, most were geared toward healthcare technologies more broadly. Only one framework suggested an assessment tool for environmental impact. Four frameworks provided a conceptual definition of this dimension without tools or metrics for assessing it, while the remaining three solely listed this dimension.

Despite the increasing global relevance of the effect that human activities and natural events have on the environment, only a minority of value frameworks considered the incorporation of environmental impact of the healthcare technologies they assess. This study’s key contribution is surveying current consideration of environmental impact in health systems, providing a starting point for future actions.

The study aims to understand the perceptions of deprescribing in primary care for patients with a limited life expectancy.

In the setting of limited life expectancy, medications may become inappropriate when the possible harms of use outweigh the benefits. Whilst the cessation of potentially inappropriate medications is associated with improved patient outcomes, incorporating this process into routine primary care is poorly enacted.

Qualitative interview study performed in primary care settings in the Netherlands, including primary care health professionals, patients with limited life expectancy, and their caregivers. Semi-structured interviews were conducted and analysed using inductive thematic analysis.

Three key themes emerged: (1) facilitating well-being, (2) preventing harm, and (3) dealing with uncertainty. A key goal of mediation use is to facilitate well-being, although the perceptions of this effect may not always match the reality due to changed clinical circumstances. The decision to continue or stop medication is influenced by the wish to prevent harm and to what extent participants find ways to deal with the uncertainties facing them.

Reluctance to deprescribe medications is often related to uncertainties around ceasing medications, lack of clear clinical guidance, and the evolving situation of advanced illness. Integrating these discussions into routine primary care for patients with chronic and incurable illnesses may assist patients and healthcare professionals to address issues around medication use in a proactive manner and promote advance care planning discussions.