Health technology assessment (HTA) evaluates the cost effectiveness and clinical efficacy of health technologies to inform policy decisions. Established in 2017 by the Department of Health Research, the Office of Health Technology Assessment in India is housed within the Ministry of Health and Family Welfare; however, challenges remain. This study analyzed gaps in India’s HTA framework to aid policymakers in making improvements.

The study conducted a systematic review of HTA literature and a focus group discussion with Ministry of Health representatives. A search on PubMed using “HTA” AND “India” yielded 74 papers from the last seven years, with 12 additional papers found through Google. Using the PRISMA methodology, titles, abstracts, and full texts were evaluated, and duplicates were managed with Zotero. Data were collected in an excel sheet, and the HTA scorecard from Türkiye helped identify context-specific gaps in India, which were discussed with six focus-group members from the HTA department at the Ministry of Health and Family Welfare and the National Health Systems Resource Centre.

The desk review identified gaps in India’s data usage, including a fragmented data ecosystem, limited electronic health records, and challenges with real-world evidence that affect HTA effectiveness. A major issue was the scarcity of trained professionals for high quality HTA and insufficient public funding for HTA research. The potential of HTA remains underutilized. Barriers to effective HTA legislation include the complexity of central policies, incoherence with state administration, and fragmented insurance schemes. Focus group participants recommended capacity building and orientation programs for the HTA department and state procurement, highlighting the need for funding to support implementation research for benchmark development.

India faces challenges in data infrastructure, stakeholder involvement, funding, and policy integration for HTA. Investments in data collection and training are essential. With initiatives such as Digital India and Make in India, the country aims to establish itself as a global digital economy. Strengthening HTA institutionalization and implementing an artificial intelligence-based HTA framework can position India as a leader in HTA.

To support decision-making regarding the introduction of digital health technologies (DHTs) with added value for patients and society, health technology assessment (HTA) frameworks for DHTs are needed. However, the absence of standardized definitions and taxonomies creates a barrier to the development of such a framework. This research addressed these challenges by developing a consensus-based unified definition and taxonomy for DHTs.

The study comprised a scoping review of existing definitions of DHTs, a targeted review of taxonomies used across European countries, and a two-round Delphi process. For the scoping review, data on the source identifier, type of DHT, the definition itself, and the purpose of the definition were extracted. For identified taxonomies, data on criteria of classification (e.g., functionality, risk, user), types of DHTs, and background of the classification (development process, purpose, and policy context) were extracted. Thematic analysis of both reviews informed the development of an online Delphi survey, distributed via several HTA stakeholder groups and societies, including HTAi.

We identified 8,651 unique records from four databases (PubMed, the Cochrane Library, Embase, EconLit) and extracted 328 definitions. Analysis revealed three key components of a definition for DHT: intended purpose, users, and underlying technology. The targeted review identified nine taxonomies classifying DHTs by maturity stages, intended purpose, digital health architecture, illness severity, personalization capacity, and levels of human intervention. The first round of the Delphi survey, with 118 complete responses, achieved consensus on the three key components, using intended purpose as a classification criterion. The results of the second round will be available in January 2025.

This study provided a unified definition for DHTs, incorporating three key components: intended purpose, users, and underlying technology. The Delphi process validated intended purpose as a primary classification criterion. This standardized definition and taxonomy streamlines assessment, enhances cross-border HTA collaboration, and enables stakeholders to compare and evaluate the impact of DHTs appropriately, thus fostering informed decision-making.

Patient involvement in health technology assessment (HTA) is increasingly valued globally but remains challenging, particularly in Brazil. Reporting tools like the Guidance for Reporting Involvement of Patients and the Public Short Form (GRIPP2-SF) help assess and enhance public participation efforts. This study evaluated the reporting of HTA documents published by the National Committee for Health Technology Incorporation (CONITEC).

We conducted a retrospective analysis of technical reports published by CONITEC between 1 January 2023 and 29 November 2024. We collected characterization data from reports that included the perspective of patients (PP) or public consultation (CP) item, in addition to the challenges for social participation. We used the GRIPP2-SF reporting guide, which has five items and aims to improve the quality of reporting on patient and public involvement in health and social care research. We performed a descriptive analysis of the data, presenting them as percentages and averages.

A total of 131 reports were evaluated, with 12,449 registered participants. Four documents did not include any social participation. Of the reports, 83 (63.3%) addressed the PP item and 48 (36.6%) addressed the PC item. The average duration of the public call was 13 days for PP and 18 days for PC. The experience report for the PP item was collected by consensus or random selection among patients. Challenges to social participation included lack of registration in the public call and failure to meet the inclusion criteria. Only the GRIPP2-SF “objectives, methods and results” items were described.

Application of GRIPP-2F in the critical evaluation of patient involvement highlighted the importance of continuing to investigate dynamic and simple approaches to patient integration into HTA reports. Including patients after the report is completed should not be the end point but a trigger for incorporating them throughout the process in future projects.

Drug out-referral occurs when patients must obtain prescribed medications from external pharmacies due to unavailability or unaffordability at healthcare facility pharmacies. This practice may burden patients financially, lead to health complications, and disrupt the revenue and workflow of healthcare facilities. The study evaluated the economic costs incurred by patients and the potential revenue loss for the University of Ghana Medical Centre (UGMC) due to out-referrals for antihypertensive medications.

This cross-sectional study took place from September to October 2023. Data on prescribed and dispensed drugs and their prices were collected from the UGMC electronic database and compared with prices from selected local pharmacies. Cost analyses were conducted from both patient and healthcare provider perspectives. Exit interviews with 367 outpatients who experienced drug out-referrals captured financial and productivity cost burden data. Microsoft Excel was used for data analysis, presenting results as frequencies and percentages.

The rate of drug out-referral at UGMC was significant, with 48 percent (5,378/11,220) of prescribed drugs not dispensed at the UGMC pharmacy, a reduction from 54 percent from 2022. Over two months, UGMC’s revenue loss on undispensed antihypertensive drugs alone was GHS43,009.61 (USD3,669.76). Patients referred for antihypertensive drugs incurred a mean direct cost of GHS18.32 (USD1.56) and an indirect cost of GHS26.15 (USD2.23), totaling GHS44.47 (USD3.79) in economic costs, alongside intangible costs such as disappointment (54%), frustration (51%), and stress (54%).

Addressing drug out-referral is essential for enhancing patient care and healthcare facility efficiency. That is, evaluating and highlighting the economic impact of this practice can drive economic growth and innovation to identify cost-effective solutions and improve resource allocation. These insights can strengthen global health systems by ensuring that healthcare practices are both economically viable and beneficial to patient outcomes.

The aim of this study was to explore the acceptability of an educational video among primary care clinicians as a tool to promote the use of stigma-free language in interactions with individuals with type 2 diabetes (T2D).

The language used by primary care clinicians in interactions with adults living with T2D can contribute to perceptions and experiences of diabetes-related stigma and be a barrier to achieving and sustaining glycaemic targets. In 2017, the American Diabetes Association (ADA) and the Association for Diabetes Care & Education Specialists (ADCES) issued a guidance paper with recommendations to promote stigma-free communication about diabetes.

The educational video, developed by the research team, presents two versions of a vignette in which a nurse practitioner interacts with an adult with T2D in a primary care setting. The first version of the vignette features the nurse practitioner using stigmatizing language as outlined in the ADA and ADCES guidance paper; the second demonstrates the use of stigma-free language by the nurse practitioner. A narrator highlights the linguistic differences. The study participants, comprising physicians (n = 8), nurse practitioners (n = 9), and physician assistants (n = 1), were recruited through professional networks and via online forums and listservs for healthcare professionals. Participants viewed the educational video and were interviewed via Zoom by a research team member using a semi-structured interview guide. The transcripts of the interviews were analysed using a qualitative descriptive approach.

Three main themes emerged from the data: aligning video content with existing attitudes and beliefs, reducing the use of stigmatizing language, and increasing the use of stigma-free language. Findings suggest that an educational video promoting the use of stigma-free language in interactions with adults with T2D is acceptable among primary care clinicians.

Immunoglobulin G (IgG) is a high-cost drug, leading our hospital pharmacy expense ranking since 2015, with a rising utilization trend from 16 percent of expenses in 2018 to 27 percent in 2023. The health technology assessment (HTA) unit at Hospital Garrahan proactively undertook sequential interventions to control variability, overutilization, costs, and resource use in the administration of IgG to pediatric patients with immune deficiencies. We report the results of a guideline implementation and a cost-minimization analysis.

An evidence-based guideline on adequate IgG use was developed, indicating similar effectiveness for subcutaneous (SCIG) and intravenous IgG (IVIG) in replacement therapy for pediatric immune deficiencies. As the switch to SCIG was rejected due to higher cost, a sequential cost-minimization analysis was performed as SCIG hospital purchase prices decreased over time. Real-world data were collected from the hospital system on IgG units consumed, acquisition prices, and percentage of drug expenses from 2018 to 2023. Daycare bed occupation was calculated assuming monthly four-hour infusions for supplementary IVIG for all registered patients. Costs are expressed in Argentine pesos and equivalent American dollars.

In 2022, switching from IVIG to SCIG had an estimated annual budget impact of ARS51,189,073 (USD392,795); the proposal was rejected on cost grounds. The switch was recommended again in 2023 when prices of alternatives became equivalent. By 2024, the initial price situation reversed, and the change implied annual estimated savings of ARS4,413,487,361 (USD4.931.270). Sensitivity analysis considering price variations determined minimum annual savings of ARS1,408,175,937 (USD1,573,380). The switch was also estimated to free 432 daycare beds per year (1.8 beds/day) and 2,400 annual nurse work hours. The recommendation was finally accepted by managers and communicated to clinicians and specialists.

Switching from IVIG to SCIG in pediatric immune deficiency patients can reduce hospital expenses, bed occupation, and nursing hours; additionally, it can prevent monthly hospital visits and subsequent school absenteeism. Hospital-based HTA can have a positive impact on cost reductions without sacrificing healthcare quality, especially by controlling adequate utilization of high-cost drugs and disinvesting in outdated technologies.

Telemedicine capacity has evolved and currently offers excellent tools for fortifying health screening systems, particularly for building a cost-effective screening network for the early diagnosis of breast cancer. In this context, a telemedicine driven approach should be directed toward improving breast cancer screening innovation in underserved hospitals. This study evaluated the results of a telemedicine-driven breast cancer diagnostic network in remote public hospitals in Paraguay.

This is a descriptive study, where the results using telemedicine-driven breast cancer diagnosis innovations in remote public hospitals were evaluated for their ability to improve accessibility and equity to specialized diagnostic services countrywide. For this purpose, the type and frequency of diagnoses performed through a telemedicine platform was determined.

During the study, a digital telemedicine-driven breast cancer screening network was implemented in 34 hospitals countrywide. The screening procedure involved analyzing a digitally acquired mammogram for masses, calcifications, or areas of abnormal density that may indicate the presence of cancer. The implemented cancer screening network performed 840 tests between October and November 2024. This resulted in the following diagnoses: normal (60%); cyst (33%); and fibroadenoma, macrocalcifications, and abnormal area (7%). If there is a significant suspicion of cancer, then tissue will be removed for a biopsy and examined by a pathologist.

According to our results, the telemedicine-driven breast cancer screening innovation network facilitated a timely, pertinent, high-quality tertiary level diagnostic service with equitable access for patients in remote underserved public hospitals in Paraguay. A widespread use assessment is necessary before this breast cancer screening method can be implemented.

Evidence synthesis is the foundation of health technology assessment (HTA); however, systematic literature reviews (SLRs) are highly resource intensive. While augmentation with artificial intelligence (AI) theoretically offers to make this rigorous process more efficient, it is unclear whether AI-supported SLRs will be widely accepted among HTA bodies. This study’s objective was to evaluate how HTA bodies regard the use of AI-supported SLRs.

A targeted literature review (TLR) (January 2019 to October 2024) was conducted in Embase, MEDLINE, and the gray literature. Search terms included AI, natural language processing, large language models, and machine learning. The TLR informed development of a survey to be fielded to respondents from HTA bodies and questions for qualitative interviews.

The TLR found that most HTA bodies do not address using AI for SLRs. Two that do are the National Institute for Health and Care Excellence (England) and the Institute for Quality and Efficiency in Health Care (Germany). Both suggested that AI can support human efforts across multiple SLR phases. Respondents from Europe and the USA completed the survey and qualitative interviews. Most respondents had some familiarity with using AI in SLRs but said their respective organizations doubted AI’s utility in improving the quality of SLRs. Respondents stated the primary responsibility for AI tool development and validation should not rest with manufacturers.

Most HTA bodies do not address using AI for SLRs, but respondents said that AI might improve SLR production by augmenting (not replacing) human effort. Gaining acceptance for AI use in SLRs for HTA will require multistakeholder engagement to ensure transparency and reliability. HTA bodies will need appropriate infrastructure and legal frameworks so that they can test and use AI software.

Respiratory syncytial virus (RSV) is a leading cause of severe respiratory illness in infants worldwide and is associated with significant morbidity. This study evaluated inequities in RSV-related hospitalizations among Brazilian infants under one year of age. Our objective was to evaluate how socioeconomic disparities impact RSV burden and cost-effectiveness metrics for RSV vaccination strategies.

A hybrid model combining a Markov framework and a decision tree was developed. The coefficient of hospitalization was estimated using the Live Birth Information System (Sistema de Informações sobre Nascidos Vivos) and the Hospital Information System (Sistema de Informações Hospitalares). RSV-related hospitalizations were identified using International Classification of Diseases, 10th Revision codes, and deprivation quintiles were defined by the BrazDep index. The RSV hospitalization risk reduction of 0.30 was extrapolated from the literature for the first year of life. Incremental cost-effectiveness ratios (ICERs) were calculated using Brazil’s cost-effectiveness threshold of USD6,873 (USD1=BRL5.82).

Infants in the most deprived quintile experienced 33-fold higher hospitalization rates compared with the least deprived quintile. At the current vaccine price of USD47.82, the ICER was USD26,934, exceeding the national threshold. Cost effectiveness was only achieved in the most deprived quintile, where the vaccine price threshold was USD48.29. Threshold prices for quintiles two to five were significantly lower: USD12.21, USD6.01, USD4.42, and USD2.15, respectively. Vaccination reduced hospitalization risk proportionally across quintiles, but the burden remained disproportionately high in the most deprived quintile.

At the current price, RSV vaccination was not cost effective in most scenarios in Brazil, with ICERs exceeding the national threshold. Cost effectiveness was achieved only in the most deprived quintile, where the disease burden is highest. To align with Brazil’s threshold, significant price reductions or targeted subsidies would be necessary to ensure equitable vaccine access and public health impact.

Health technology assessment (HTA) bodies published stringent criteria for cost-minimization analyses (CMA), but evidence shows that many appraisals still fail to meet their full requirements: notably, adequately powered trials showing non-inferior outcomes regarding minimal clinically important difference. Here, we report the use of CMA in recommending biological disease-modifying antirheumatic drugs (bDMARDs) in psoriatic arthritis.

A comprehensive review of HTA agency reports was conducted to identify the economic evaluations applied in the appraisal of bDMARDs for psoriatic arthritis. Data were collected from publicly available HTA databases and official websites of globally recognized agencies. The search included appraisals published up to December 2024. Evaluations were categorized based on the economic model used (e.g., CMA, cost effectiveness, cost utility, or cost benefit). Extracted data were analyzed to summarize trends and identify differences in the economic approaches used among agencies.

Cost-effectiveness and cost-utility analyses were the predominant approaches in HTA agency assessments of bDMARDs for psoriatic arthritis. Certolizumab pegol, golimumab, and secukinumab were the most frequently evaluated medications, each with nine evaluations. The Brazilian National National Committee for Health Technology Incorporation (CONITEC) was the only agency that exclusively used CMA in all its evaluations. In contrast, the National Institute for Health and Care Excellence (NICE) did not use this methodology in its assessments.

Our findings highlight the significant variation in economic evaluation methods used by HTA agencies for bDMARDs in psoriatic arthritis. The inappropriateness of superiority trials that fail to reject the null hypothesis for CMA is a key takeaway. While CMA remain relevant due to their simplicity, their use by CONITEC contrasts with broader methodologies preferred by other agencies, particularly NICE.

Horizon scanning (HS) systematically identifies emerging health technologies to support future decision-making. However, HS methods face challenges due to inconsistent terminology and lack of reporting standards. This hinders dissemination and increases research waste. Unlike PRISMA for evidence synthesis, no reporting guidelines exist to support HS. This work proposed a preliminary reporting checklist.

In July 2024, a working group comprising HS analysts, evidence synthesis researchers, and information specialists was established at the Innovation Observatory (IO), the UK’s national HS organization. The group convened three times to define objectives, refine methods, and develop the reporting checklist. In the first meeting, we outlined a prototype by adapting the PRISMA Extension for Scoping Reviews (PRISMA-ScR). We internally validated it on 25 IO HS reports (2017 to 2024). The second meeting assessed the relevance of each checklist item following our internal validation. In the third meeting, additional items were proposed based on the validation’s results, and the group reached consensus on the final checklist.

The final checklist prototype comprised a total of 35 items of which 29 were essential and six optional. It introduced new key elements absent in PRISMA-ScR: “interest holder description” in the introduction, “horizon-scanning scope” in the methods, “technology characteristics” in the results and “political, economic, societal, technological, legal and environmental (PESTLE) interpretation” in the discussion. Additionally, the checklist was tailored to promote transparency, equity, fairness, and sustainability in signal detection processes over those characteristics in evidence synthesis, aligning with the goals of HS. The checklist is available to download in the Open Science Framework.

Our checklist addressed a critical methodological gap, advancing HS research by promoting standardization and transparency of HS methods. By making the draft checklist available, we encourage HS practitioners to pilot it. Feedback is welcomed to contribute to the final checklist. Its adoption and endorsement will improve output visibility, ensuring this meets necessary methodological standards underpinning unbiased healthcare policy decision-making.

The use of real-world data (RWD) for real-world evidence (RWE) is essential for evidence-based decisions but requires robust technical, methodological, and organizational capabilities. We conducted an analysis of the Agència de Qualitat i Avaluació Sanitàries de Catalunya (AQuAS) Health Technology Assessment area to determine its capacity to generate RWE using RWD in Catalonia’s healthcare system, to identify priority areas to strengthen competencies and address emerging demands effectively.

A structured SWOT (Strengths, Weaknesses, Opportunities, Threats) analysis was conducted, evaluating internal (strengths and weaknesses) and external (opportunities and threats) aspects. Data collection involved individual reflections and participatory workshops with the Assessment area’s team involved in RWE generation, focusing on critical areas such as data access, analytical capacities, technological resources, and regulatory environments. Finally, a strategic action plan was developed to implement appropriate measures.

Strengths: Consolidated expertise in data analysis and alliances with programs such as the Data Analytics Program for Research and Innovation in Health (PADRIS) in Catalonia; and availability of technological tools and procedures for RWE generation.

Weaknesses: Dependency on third parties for data access; centralized knowledge in key individuals; training gap in advanced methodologies (emulation of clinical trials and predictive analysis); and gaps in data homogenization and quality.

Opportunities: Potential to establish strategic alliances with other entities and leverage emerging technological advancements (implementing Observational Medical Outcomes Partnership standards).

Threats: Regulatory changes and competition in RWE development.

SWOT analysis identified priority actions to enhance AQuAS’s capacity for RWE generation, including implementing training programs, formalizing strategic collaborations, and developing integrated processes for data management. This approach reinforced AQuAS’s role as a regional and international leader in using RWD for informed healthcare decision-making, highlighting the importance of capacity building and cooperation.

Central precocious puberty (CPP) is a rare condition, commonly characterized by early development of biochemical puberty and physical characteristics before the ages of eight (girls) and nine (boys) years. According to the Brazilian clinical practice guideline (CPG) the treatment of CPP includes gonadotropin-releasing hormone agonists. The objective of this study was to verify implementation of the CPP CPG using real-world drug dispensing data from the Brazilian Unified Health System.

Data from drug dispensing in the Brazilian Unified Health System between 2013 and 2023 were extracted by a platform that creates follow-up cohorts for specific diseases (Open Room for Health Intelligence Situation). Only data for patients with an International Classification of Diseases, 10th Revision category of E22.8 were selected. All records that did not have encrypted user identification or that did not have an approved quantity of medication were excluded. A descriptive analysis was performed using PostgreSQL 24.2 to model the data.

A total of 147,045 individuals with CPP were treated with the recommended drugs in the Unified Health System over the study period. Most patients were female (94%). The mean age of patients at the beginning of treatment was 8.8 years (standard deviation 5.3). The most used drugs were leuprolide (80.2%), triptorelin (17.7%), goserelin (2.0%), and cyproterone acetate (0.2%). Leuprolide and triptorelin, with a biannual frequency of use, were included in the CPG at the end of 2022. Consequently, 1,905 patients used leuprolide (45 mg) in 2023 and 1,131 patients used triptorelin (22.5 mg).

Data from the drug dispensing registry indicated that the CPP CPG is being implemented. Recently, this document was updated to recommend biannual frequency use of leuprolide and triptorelin to improve adherence to treatment. Data from the Open Room for Health Intelligence Situation platform can be an important source of information for evaluating the performance of these technologies.

The preferred regimen for initiating HIV/AIDS treatment, according to the Clinical Protocol and Therapeutic Guidelines (PCDT), is tenofovir, lamivudine, and dolutegravir (TDF/3TC/DTG). Fiocruz, through a productive development partnership (PDP), produces these antiretrovirals, strengthening the national industry. The National Committee for Health Technology Incorporation (CONITEC) evaluated the budgetary impact of incorporating bictegravir, emtricitabine, and tenofovir alafenamide (BIC/FTC/TAF) combination therapy and its effect on the sustainability of the PDP.

The analysis used data from the 2022 HIV Clinical Monitoring Report to identify the eligible population. The unit cost of the current regimen (TDF/3TC/DTG) was obtained from the Health Price Database, while the cost of the new regimen (BIC/FTC/TAF) was extracted from CONITEC Recommendation Report No. 675/2021. Using a market share analysis, a moderate 10 percent annual increase in uptake of BIC/FTC/TAF was projected, reaching 50 percent by the fifth year.

For the budget impact calculation, a total of 245,923 people aged 50 years or older who were living with HIV and/or AIDS and undergoing antiretroviral therapy were considered. The costs with and without the incorporation of the BIC/FTC/TAF regimen were analyzed. The incorporation of the new technology resulted in an estimated budget impact of BRL15,463,638.24 (USD2,693,122.18) over the analyzed period.

The BIC/FTC/TAF regimen is an effective, safe treatment approved by international agencies. It is administered in a daily dose, which simplifies treatment. However, its patent restricts national production, and its use in place of the current regimen could impact PDPs, harming the national industry and the sustainability of the health system. Therefore, incorporations require balancing clinical benefits with innovation and financial impacts.

Neonatal disorders are a significant contributor to years lost to premature mortality and years lived with disability worldwide. Healthcare cost estimates for neonatal inpatient health care and the health conditions that most affect newborns in Brazil remain unknown at the national level. We estimated the inpatient healthcare costs in Brazil and identified the most burdensome health conditions.

This population-based descriptive study was conducted from the perspective of the Brazilian public health system. We used secondary nationwide data from the Hospital Information System. Inpatient healthcare records between 2011 and 2022 for newborns from zero to 27 days of life were included. Inpatient healthcare costs consisted of reimbursements for hospital procedures and daily hospital stays, including neonatal intensive care unit (NICU) admissions. Costs were adjusted for the 2023 inflation rate and presented in USD considering the parity index of 2023. The main health conditions are presented as International Classification of Diseases, 10th Revision categories.

A total of 3,835,128 neonatal hospital admissions accounted for approximately USD6 billion, of which 26.7 percent were for NICU admissions. The ten costliest health condition categories accounted for 75.6 percent of total inpatient healthcare costs. Although the most prevalent neonatal health condition was neonatal jaundice (n=652,314), the category of disorders related to prematurity accounted for most inpatient neonatal care costs at USD1.7 billion (USD1.1 billion for NICU), followed by neonatal respiratory distress syndrome at USD1.6 billion (USD1.2 billion for NICU), and the category of other respiratory disorders at USD316.1 million (USD238.3 million for NICU).

This study revealed the economic burden of neonatal inpatient healthcare in Brazil, driven predominantly by NICU admissions for prematurity and respiratory conditions. The findings highlight the importance of targeted health interventions and policies to improve neonatal outcomes and optimize resource allocation.

Clinical trial registries provide relevant information to address publication bias in systematic reviews. However, monitoring these registries is a complex process. Results are rarely posted in registries, and finding publications in journals requires a laborious search across multiple resources. We aimed to monitor registries of randomized clinical trials, results posted on platforms, and articles published in journals in the context of a rapid review to inform decision-makers.

We searched ClinicalTrials.gov and the World Health Organization International Clinical Trials Registry Platform from April 2020 to November 2024 as part of a systematic search on the use of vitamin D in COVID-19, including the MEDLINE, Embase, Web of Science, and the Cochrane Library databases. We collected data such as the study start date, sample size, principal trial characteristics, population, intervention, control, and main outcome. We reviewed new register monitoring results published in databases or platforms and assessed each report’s update frequency until November 2024.

We found 54 unique register numbers with this different status information: 29 trials were completed, two terminated, four active (one recruiting and three not yet recruiting), two were ongoing, one had recruitment and regulatory approvals pending, 11 had unknown status, one was prematurely ended, and two were withdrawn. Two clinical trials were matched with one study published. In total, 26 trials had results published in journals. However, only two trials posted results on the platform. In the last review, 25 trials remained without results, and related publications were not found.

Searching clinical trial registries as part of a systematic review strategy is a necessary source for addressing publication bias. Monitoring clinical trial registries provides relevant information on the development and length of trials, identifying gaps and failures to assist research. Lack of updated information on registries, and finding accurate and worthy information about trials, can result in a time consuming and sometimes unsuccessful process.

Argentina’s healthcare system is fragmented, comprising public, private, and social security sectors. The guidance for incorporating a new health technology into the healthcare system suggests a single threshold value (0.016% of total health expenditure). This study analyzed the influence of cost heterogeneity on budget impact using the reimbursement of talazoparib for the treatment of metastatic prostate cancer as an example.

A budget impact analysis (BIA) was adapted for the National Institute of Social Services for Retirees and Pensioners and SURGE (union social security sector) perspectives. To be able to estimate the impact of medical costs, other parameters such as population and market share were fixed. Drug costs were estimated using reimbursement values, while the costs of healthcare resources and management of adverse events were obtained from the 3Eff SA cost database (3val), which is based on tariffs of institutions within the subsectors of the Argentine healthcare system.

For direct medical resources (medication, medical consultations, laboratory tests, etc.) associated with metastatic prostate cancer treatment, cost heterogeneity across health sectors was 79 percent. The lowest variability was observed in laboratory tests (18%), while the highest was in medical consultations (310%). The cost of managing adverse events varied by 38 percent across sectors, with the highest variability in managing fatigue (118%) and the lowest in thrombocytopenia (2.4%). As a result, the average annual budget impact varied across sectors by just over 15 percent.

Our results showed a significant heterogeneity in costs among Argentina’s healthcare subsectors. This heterogeneity might lead to different reimbursement decisions based on the BIA results. Variability in costs underscores the need for sector-specific guidelines or thresholds for reimbursement decisions to ensure equitable and efficient resource allocation.

Emerging regions require equitable training processes to implement health technology assessment (HTA) effectively. This work presents the initial adaptive curriculum designed for a professional master’s degree in HTA, tailored to the regional context in Brazil, particularly for areas with evolving infrastructure. The objective was to foster HTA expertise by enhancing methodological skills, technical knowledge, and collaborative abilities among students.

The curricular adaptation was developed through collaboration between students and course coordinators, utilizing a cooperative methodology grounded in the first four stages of the Cooperation Pedagogy framework.

The class consisted of 20 students: 75 percent were women, 60 percent from the Central-West region, 30 percent from the Northeast, and 10 percent from the North. Ten students worked in HTA units in hospitals and two worked in legal units. Students’ topics of interest included vaccination in remote areas, judicialization, post-incorporation assessments, and budget impact analyses. The results of the four stages were: (i) a coexistence contract developed based on students’ expectations; (ii) a “World Café” dynamic that facilitated the creation of “hot questions”; (iii) the curricular adaptation integrating all relevant subjects; and (iv) the affective environment fostering mutual learning and collaboration.

The next steps involve co-creating solutions to the students’ hot questions, which were: (i) Does decision-making overturn an HTA report?; (ii) Is the HTA language understandable to stakeholders?; (iii) How to raise awareness among judges?; (iv) How to stimulate the creation of HTA units in emerging regions?; and (v) What is the future of HTA?

Including drugs in the National Essential Medicines List is a complex process that considers population, budget, and socioeconomic factors. In Ecuador, these decisions rely on efficacy and safety data from existing studies without estimating the real impact and clinical benefit of the drugs. This study proposed a multicriteria model using minimal clinically important difference (MCID) values to evaluate oncologic drugs for inclusion.

Safety and efficacy were assessed using MCID values for primary outcomes, incorporating measures such as overall survival (OS), disease-free survival (DFS), and complete remission, following the Magnitude of Clinical Benefit Scale (v1.1) of the European Society for Medical Oncology. Epidemiological and demographic data were obtained from the National Institute of Statistics and quality of life was evaluated using EuroQol-5D scores. Information on oncologic drugs and regulatory considerations was sourced from authoritative organizations. All parameters were systematically organized and weighted, ensuring the total criteria groups accounted for 100 percent.

Seven criteria groups were established, each containing parameters evaluated on a scale from zero to five, designed to reward or penalize based on assessment goals. The groups were weighted from five to 30 percent to reflect their relative importance or impact on decision-making. The criteria and weights were as follows: (i) disease burden (30%); (ii) efficacy (30%); (iii) safety (15%); (iv) quality of life impact (5%); (v) availability and accessibility (5%); (vi) therapeutic alternatives and international recommendations (10%); and (vii) other important criteria (5%). Consequently, scores were weighted differently across groups.

Accurately estimating the impact of oncologic drugs is critically important for public health. Implementing this multicriteria model will provide a more efficient, objective, and systematic framework for evaluating the inclusion, modification, or exclusion of oncologic drugs with respect to the National Essential Medicines List. This approach enhances decision-making processes and ensures the optimal selection of anti-cancer therapies, ultimately improving patient outcomes and resource allocation in Ecuador.

In Brazil’s Unified Health System (SUS), initial treatment for follicular lymphoma (FL) is generally effective, but many patients relapse and continue treatment with rituximab plus chemotherapy. Lenalidomide plus rituximab (LR) has been shown to improve overall survival (OS) and progression-free survival (PFS) in patients with FL. We assessed the cost utility and budget impact of LR for previously treated patients with FL from the SUS perspective.

A three-state partitioned survival model was built using rituximab OS and PFS curves from the AUGMENT clinical trial, which were extrapolated over a 40-year horizon using parametric distributions and adjusted with hazard ratios (HRs) from the comparison with LR (OS: HR 0.45, 95% confidence interval [CI]: 0.22, 0.92; PFS: HR 0.40, 95% CI: 0.29, 0.56). Curve selection was based on the Akaike and Bayesian information criterion and visual inspection. Utility values were obtained from international literature. Direct medical costs, including the cost of drugs, monitoring, and pre- and post-progression were considered. For the budget impact analysis, eligible patients were estimated based on measured demand, and two different market shares were compared with the reference scenario (without LR).

LR showed an incremental benefit (3.10 quality-adjusted life years [QALYs]) and incremental costs (USD76,718 [BRL187,192]). The incremental cost-effectiveness ratio (ICER) suggested that LR was cost effective (USD24,712 [BRL60,298]/QALY), considering the Brazilian threshold of USD49,180 (BRL120,000) per QALY. In sensitivity analyses, LR remained cost effective in most scenarios except when the OS HR was adjusted to the upper limit of the 95 percent CI. Budget impact analysis showed that listing LR in the SUS would increase the budget by USD12,523,280 (BRL30,557,046) in five years if market share reached 50 percent by the fifth year (approximately 648 to 737 eligible patients per year).

Standard treatment options for relapsed FL are limited. Previously treated individuals with FL may benefit more from LR than from standard treatment with rituximab and chemotherapy. The ICER showed that lenalidomide could be cost effective for the Brazilian health system.