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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

The Hospital-Based Health Technology Assessment (HB-HTA) Unit at the third-level pediatric Hospital Garrahan has had a specific Drug Assessment Sector since 2019 for mandatory evaluation of all incorporation requests for high-cost or high-risk medications. The aim of this study was to analyze its production and response times, the annual cost per patient of drugs assessed, the proportion of recommendations to reject, management acceptance, and patient results of drugs incorporated during the last five years.

All medication incorporation requests follow a standardized procedure. The Pharmacy and Therapeutics Committee makes an initial appraisal of relevance. Drugs with a high cost, safety risk, or any significant organizational impact are submitted to the HB-HTA Unit for systematic review. A non-binding report is sent to the Executive Director to support decision-making. The Drug Sector also carries out proactive assessments like yearly analyses of pharmacy medication expenses, drug use variability, overutilization, trends in consumption, interchangeability, and pharmacovigilance. The proportion of full and rapid reports, rejection rate, response time, and annual cost per patient were analyzed.

In five years, the Drug Assessment Sector at the HB-HTA Unit produced 24 reports (56% were full reports and 44% were brief or rapid). Of these, 76 percent were for high-cost drugs and 96 percent were accepted by management. The median response time was 106 days (range 5 to 511). The recommendations included approvals for restricted conditions (36%) and rejections for safety issues, lack of evidence of effectiveness, or high budget impact (21%). The annual estimated cost per patient of assessed drugs ranged from ARS10,528 to ARS304,837,050 (USD1,757 to USD314,265). Follow-up for a minimum of two years included six drugs (44 patients), which showed compliance with restricted use recommendations and no clinical results justifying disinvestment.

The creation of a specific Drug Assessment Sector in the HB-HTA Unit at our hospital has produced a more rational incorporation and utilization of high-cost drugs. Variable response times result from urgency and coexisting requests. Despite the non-binding recommendations, management acceptance was high. Follow-up and clinician reports of patient results enable review of the decisions made.

Innovation in medications creates challenges for access to health care, making the rational use of drugs essential. The Health Technology Assessment Committee (HTAC) help to adapt technologies to hospital settings and align recommendations with institutional needs. We aimed to describe the role of the HTAC in a high-complexity hospital by quantifying the requests received and approved.

The study was conducted at the Hospital Italiano de Buenos Aires. The primary objective was to provide a descriptive analysis of the functioning of the HTAC. The secondary objective was a retrospective observational study of the quick reports produced by the HTAC between January 2017 and December 2023. Variables analyzed included the total number of drug evaluation requests received and the proportion of requests incorporated or not incorporated annually during the study period.

The HTAC at our institution began in 2011, initially consisting of one physician, one pharmacist, and one administrative staff member. It now includes two coordinators and 11 evaluators.

The drug incorporation process starts with a request from the prescribing service, followed by a literature review and specialist meetings. A technical report is produced that covers efficacy, safety, costs, and final recommendations. During the study period, 111 drug incorporation requests were received, with 90 (81.1%) being included in the formulary. In the 2022 to 2023 biennium, 50 requests were received, representing 45 percent of the total requests during the study period.

Standardizing the HTAC’s structure was essential for managing the increasing number of new drug approvals. Adapting evidence on new molecules to the hospital setting was crucial to support recommendations for inclusion in the therapeutic formulary and to optimize the use of technologies, enabling more efficient and structured decision-making.

Futile treatment is defined as maintenance of organ function without achieving meaningful goals of care. Poland is characterized by low prevalence of introducing limitations of treatment in intensive care units (ICUs). The aim of the study was to conduct a questionnaire study to evaluate the approach of Polish medical personnel to futile treatment in the ICUs.

We conducted an anonymous questionnaire study during a national intensive care conference in April 2023. We collected data on participants’ experiences with limiting futile treatment and their demographics. Statistical analysis comparing the responses between respondents with shorter (less than 10 years) or longer (10 years or more) work experience was conducted with a chi-squared test with residual analysis and Bonferroni correction.

354 respondents completed the questionnaire. Most participants (94.5%) found discussing end-of-life care with patients important. Additionally, 81.6% believed that the medical personnel should be more decisive than the patient’s family regarding end-of-life care decisions. While 81% were aware of the existence of futile treatment protocol, only 35% used it regularly. Fear of legal consequences (61.9%) or family’s reaction (55.6%) were the most common reasons for not adhering to existing guidelines. Improving hospital procedures (83.6%) and proper legislation (67.2%) were commonly suggested measures to improve end-of-life care. Respondents with shorter work experience more often reported no awareness of futile treatment protocol (28.7% vs. 6.9%, p < 0.001) as well as no experience discussing treatment limitations with patients (24.6% vs. 8.2%, p < 0.001) or their families (20.0% vs. 3.8%) compared to the clinicians with longer work experience.

Despite widespread recognition of the unethical nature of futile treatment, it remains controversial among Polish ICU clinicians. Improvement of legislation and hospital procedures could contribute to improvement of clinicians’ and patients’ well-being when facing end-of-life care decisions.

Rheumatoid arthritis (RA) is a chronic inflammatory autoimmune disease affecting the joints. Biological disease-modifying antirheumatic drugs (bDMARDs) and targeted synthetic disease-modifying antirheumatic drugs (tsDMARDs) have expanded the therapeutic armamentarium for RA and significantly changed its management. However, there are still unmet needs for patients who do not respond to treatment. This cohort study aimed to evaluate the effectiveness of DMARDs in real-world settings.

The study compared the effectiveness of Janus kinase inhibitors (iJAK; a type of tsDMARD) and anti-tumor necrosis factor (TNF) and non-anti-TNF drugs (bDMARDs) through treatment persistence in first- and second-line therapy. Access to these drugs requires an administrative approval process in the Brazilian public health system (BPHS). The cohort included patients with RA who requested their first drug between June 2018 and September 2022. The study was conducted in BPHS (Minas Gerais State) pharmacies and used drug dispensing records and clinical and sociodemographic data. Kaplan-Meier curves were used to compare treatment persistence at 18 months’ follow-up.

Among 763 patients, 85.1 percent were female and 33.0 percent white—mean age of 55.4 years and 6.5 years since initial diagnosis. Initially, 68.2 percent used anti-TNF drugs (adalimumab, etanercept, infliximab, certolizumab pegol, golimumab), 7.2 percent used non-anti-TNF drugs (abatacept, tocilizumab), and 24.6 percent used iJAK agents (tofacitinib, baricitinib, upadacitinib). The non-anti-TNF group had the lowest treatment persistence (p<0.05). Etanercept and baricitinib showed higher persistence. In the second line, 46.2 percent of patients used anti-TNF drugs, 23.9 percent used non-anti-TNF drugs, and 29.9 percent used iJAK agents (p>0.05). The baricitinib and tocilizumab groups showed higher persistence. In the second line, a general decrease in treatment persistence occurred.

First- and second-line iJAK agents had the best persistence; there was no difference between drug groups in the second line. However, the rates of discontinuation and drug switching suggested treatment failure. The results suggested that patients respond less to second-line treatment. These results are useful for future reassessment decisions.