Cholesteatoma is an abnormal skin growth within the middle ear that can destroy the ossicular chain and cause hearing loss. This study aimed to evaluate the cost effectiveness of canal-wall-down (CWD) mastoidectomy for cholesteatoma removal with or without hearing reconstruction using an active transcutaneous bone conduction implant (BCI) in patients with advanced cholesteatoma to support the design of a future clinical study.

A Markov model was developed to evaluate the cost effectiveness of the following strategies: CWD without hearing reconstruction (CWD only); CWD with simultaneous BCI implantation (CWD+BCI0); CWD with implantation of BCI after one year (CWD+BCI1), two years (CWD+BCI2), three years (CWD+BCI3), or five years (CWD+BCI5). The outcomes evaluated over a lifetime were quality-adjusted life years (QALYs), costs (from the perspective of the National Health Fund, Poland), and net monetary benefit (NMB), assuming a willingness-to-pay threshold of one gross domestic product (GDP) per capita in Poland (PLN92,955 [USD53,422]). One-way deterministic sensitivity analyses (DSA) were performed to evaluate the robustness of the results. Cost and NMB results were presented in 2024 PLN and USD (conversion using purchasing power parities for GDP).

The simulation indicated that CWD+BCI0 was expected to be the most effective strategy, yielding 18.35 QALYs, followed by CWD+BCI1, CWD+BCI2, CWD+BCI3, CWD+BCI5, and CWD only. The lifetime costs of performing CWD+BCI0 were expected to be lower than the costs of CWD+BCI1, CWD+BCI2, and CWD+BCI3. Moving from CWD only to CWD+BCI0 would incur an additional cost of PLN67,754 (USD38,939) over a lifetime to gain 3.13 QALYs. CWD+BCI0 resulted in the higher NMB (PLN1,595,530 [USD916,971]), followed by CWD+BCI1 (PLN1,578,976 [USD907,457]), CWD+BCI2 (PLN1,569,383 [USD901,944]), CWD+BCI3 (PLN1,560,090 [USD896,603]), CWD+BCI5 (PLN1,542,377 [USD886,424]), and CWD only (PLN1,372,572 [USD788,834]), respectively. DSA showed that the results were robust.

CWD with simultaneous hearing reconstruction using a BCI (CWD+BCI0) is expected to be the most cost effective strategy for patients with cholesteatoma in the Polish context. This result will support the design of a clinical study evaluating the surgical and audiological outcomes of the simultaneous approach.

The United Nations 2030 Agenda for Sustainable Development aims to provide a “shared blueprint for peace and prosperity for people and planet, now and into the future.” Improving the quality of patient care goes end-to-end with sustainability across surgical care pathways, including pre-, intra-, and postoperative surgical optimization and maximal patient benefit. The Green Surgery report provides initiatives and recommendations to reduce the adverse impact of surgical care on the environment.

Representatives from a consortium of 23 global healthcare professional organizations met every three months to provide different perspectives on sustainable surgical care practices and technologies. Consortium members were consulted throughout the development of the report. Real-world evidence was collected from case studies focusing on triple bottom line outcomes. Surgical care products and practices were evaluated using the following criteria: prevention, resource optimization, low carbon alternatives, patient optimization, leaner pathways, and patient empowerment. Data were grouped and synthesized to spotlight health technology environmental impact along the surgical care pathway. Recommendations to stakeholders based on short- and long-term objectives.

Surgical care is a major area of resource consumption, with the annual carbon footprint of surgical care in the UK estimated at 5.7 million tons of carbon dioxide equivalent. Carbon hotspots in the operating theatre include anesthetic gases, energy, and products (particularly single use). This report signaled the need for sustainable products and services from industry, encouraging wider systems change toward disease prevention and health promotion, alongside equitable access to high quality care. The emergence of artificial intelligence and its anticipated application within healthcare settings needs careful evaluation, as it may lead to overdiagnosis and excessive use of healthcare and associated carbon.

This report highlighted emerging evidence that can support the transition to green surgical care. Bringing about real-world change relies upon coordinated action from all individuals and organizations influencing surgical care. It is recognized that the triple bottom line framework has some limitations, including in evaluation (data for each pillar), practical application (competing healthcare priorities), and strategic investment prioritization.

Scientific journals are essential for scientific dissemination, indirectly reflecting research interests and funding. Epidemics targeted to end by the Sustainable Development Goals (SDG) 3.3 (AIDS, malaria, tuberculosis) still have significant impacts, requiring innovative health technologies. Monitoring publications on these epidemics can guide strategies to prioritize research and funding. We investigated publication priorities about the SDG 3.3 in pharmaceutical journals and associated factors.

This cross-sectional study included journals in the pharmacology and pharmacy category of the Journal Citation Reports (JCR) in 2022. The primary outcome was publication priority on AIDS, malaria, and tuberculosis, defined as greater than or equal to three percent. Bibliometric indicators were obtained from the JCR, and the number of publications in each journal up to 2022 were collected using the Web of Science Core Collection via EndNote. The editors-in-chief countries were identified on journal websites and classified as low- and middle-income or not according to the World Bank. Prevalence ratios (PR) and 95 percent confidence intervals (CI) of the outcome and journal factors were calculated by Poisson regression.

Of 362 eligible journals, eight were excluded due to discontinuation or missing editor information. Most journals started between 1991 and 2010 (39.4%) and were open access (50.7%). Sixty-one editors-in-chief were from low- and middle-income countries (17.3%), with most of their journals belonging to the JCR’s Emerging Sources Citation Index (39.0%; p<0.001) and being open access (22.9%; p=0.005). More recent journals had more editors-in-chief from developing countries (34.5%; p<0.001) and higher publication priority on the SDG 3.3 (PR=2.32; 95% CI: 1.47, 3.66). Journals with such editors published more on the SDG 3.3 (PR=1.57, 95% CI: 1.10, 2.23) and had lower impact factor (2.33 [standard deviation 2.63] versus 4.10 [standard deviation] 7.39; p=0.066).

Journals that were founded more recently and had editors from developing countries published more articles on the SDG 3.3. Increasing the diversity of editorial boards in scientific journals could be an important strategy to promote the dissemination of health technologies aimed at addressing these neglected diseases and achieving the SDG by 2030.

The National Committee for Health Technology Incorporation (CONITEC) plays a crucial role in advising the Brazilian Ministry of Health. Evaluating medical devices (MDs) poses challenges due to gaps in clinical and economic evidence. This study aimed to: (i) describe the current landscape, highlighting advancements in specific health technology assessment (HTA) processes for medical devices; and (ii) deepen knowledge through a case study.

The study methodology included descriptive exploratory research, reviewing normative references, and conducting documentary analysis on the evaluation of technologies for incorporating medical devices into Brazil. The normative review involved consulting the CONITEC website and “Sistema Saúde Legis,” which compiles federal regulations. Document analysis used CONITEC’s “CONITEC in Numbers” panel filtered by “health products,” which tracks the progress, incorporation status, and updates on health technologies over the last 12 years. The case study selection criteria included demand with a decision on incorporation, external claimant profile, and suitability for reassessment, which allowed comparative analysis of the HTA processes.

Since its creation, CONITEC has received 122 requests, approximately 10 percent of which are related to MDs. Of the total evaluated, 65 percent came from external applicants and 35 percent from areas of the Ministry of Health. The study highlighted significant changes in the structure and configuration of CONITEC in 2022, including the establishment of subcommittees and thematic committees specifically dealing with MDs. A case study on dynamic phototherapy, developed at a Brazilian University, illustrates the evolution: initially not recommended in 2020, dynamic phototherapy received a favorable recommendation in 2023 following review. This underscores the importance of specialist involvement and real-world data collection.

CONITEC’s 12-year journey has improved MD evaluation, especially with the new specialized committees. The study demonstrates the evolution of the HTA process in Brazil, emphasizing the importance of specialized subcommittee participation and contextual data collection for comprehensive evaluation of demands.

The advent of immunobiological drugs, with special storage, transport, administration, and care requirements, adds to budget impact analysis (BIA) challenges. We analyzed BIA studies used in the recommendations of the Brazilian National Committee for Health Technology Incorporation (CONITEC) related to the incorporation of immunobiological drugs for treating immune-mediated rheumatic diseases (IMRD) between 2012 and 2023.

An analysis of the BIA records related to immunobiologicals for the treatment of IMRD was carried out and nine CONITEC reports were reviewed. All reports were assessed regarding the compliance of the national BIA guidelines (NBIAG), published by the Brazilian Ministry of Health in 2012. Additionally, five semi-structured interviews were conducted with different key players, academic experts in the field, to discuss specific aspects related to the challenges of BIA of these drugs in the Brazilian scenario.

One BIA was dated before the NBIAG and was excluded from the joint analysis. Within the reports, the economic adjustments had the highest degree of adequacy (100%), while the costs of introducing a new medicine were not included in any of the reports. Nine categories were identified through content analysis, including the insufficiency of the BIA carried out for the complexity of the scenario being analyzed and issues related to biosimilar drugs. All the interviewees felt that the BIA carried out were not done from the perspective of the Unified Health System (SUS) as a tripartite management and financing model.

This research pointed to the need for methodological improvements in BIA and strict compliance with the methodology currently recommended, at the risk of high uncertainty in the proposed models. The BIA should also consider the complexity of the SUS, its financing and tripartite management, the judicialization of health, and the Brazilian reindustrialization policies under development.

The assessment of a technology’s innovativeness, which indicates its capacity to transform practice, is crucial for strategic planning in health care. However, clear assessment criteria are often lacking. This abstract presents the early experience of the Agency for Care Effectiveness with the UK Government’s Medical Technology Innovation Classification Framework to identify innovative medical technologies (MedTechs) anticipated to disrupt clinical practice in Singapore.

ACE’s horizon scanning methodologies were employed to identify innovative MedTechs, based on innovation programs of regulatory bodies, such as the United States Food and Drug Administration breakthrough device designation. The UK Framework was applied, using only the technology aspects (novelty) and not the implementation elements (expected shift in clinical practice) to assess the novelty of MedTechs. Three evaluators independently classified each MedTech as incremental (exists within healthcare system), transformative (novel application of existing technology), or disruptive (first-in-class) based on desktop research. The usability of the classifications was assessed through user experience and inter-rater agreement, with consensus reached for most disagreements.

Among 228 innovative MedTechs identified based on regulatory body designations, pre-consensus inter-rater agreement was 76 percent across innovation categories, with higher consistency (89%) for incremental innovations but lower agreement (64%) for transformative and disruptive categories. The inconsistencies stemmed from limited understanding of the use of MedTechs and their expected shift in local clinical practice. Following consensus, 42 percent were classified as incremental and 58 percent as transformative or disruptive. To enhance future usability, transformative and disruptive technologies were combined into a single highly innovative category to expedite categorization. Highly innovative technologies were then prioritized for local clinician input.

The UK Framework offers a valuable approach to assess the level of innovativeness of MedTechs. By combining transformative and disruptive technologies into a highly innovative category, ACE could expediently prioritize impactful MedTechs to inform the system on upcoming innovations with disruptive potential. This fit-for-purpose approach could offer resource-constrained health technology assessment bodies a means to assess the innovativeness of MedTechs.

This study aimed to externally validate the budget impact analysis of the drug fingolimod after its incorporation into the Brazilian Unified Health System, comparing real parameters arising from its incorporation in the treatment of relapsing-remitting multiple sclerosis with the estimates presented in the 2017 incorporation report.

The study included the following:

• • A survey of the target population to validate the demand measured by drug dispensing to patients with multiple sclerosis in the Brazilian Unified Health System;

• • Validation of unit costs of drugs through purchases made by the Logistics Department of the Ministry of Health;

• • Determination of market share within the defined time horizon based on drugs purchases;

• • Estimation of direct administration costs; and

• • Monitoring of drugs to compare estimated values with the real value.

Divergences were identified between the real-world data collected in relation to the study’s defined population and the market share of fingolimod over the studied time horizon. There was a significant increase in the fingolimod market share, with the largest market shares being in 2019 and 2020. The budget impact presented in the incorporation report was USD277,431,260.28, whereas the validated impact was USD194,955,442.76.

The process of validating the 2017 budget impact analysis of fingolimod identified savings of around USD82.4 million relative to the value proposed for incorporation. The percentage of market share and drugs costs were the most divergent factors in relation to what had been proposed in the incorporation report.

Demonstrating meaningful overall survival (OS) treatment benefit in high-risk localized or locally advanced prostate cancer (HR-LPC/LAPC) remains challenging due to the prolonged natural disease evolution. There is growing interest in validating intermediate clinical endpoints (ICEs) for OS. We evaluated the predictive value of ICEs for OS in HR-LPC/LAPC using data from randomized controlled trials (RCTs) and non-randomized comparative studies (non-RCTs).

We conducted a systematic literature review using Embase, MEDLINE, CENTRAL, and gray literature (22 March 2024) to identify RCTs and non-RCTs investigating therapeutic options for adults (≥18 years) with HR-LPC/LAPC. Eligible studies reporting treatment effects (hazard ratios [HR]) for OS (HROS) or ICEs were included. Event-free survival (EFS), metastasis-free survival (MFS), no evidence of disease (NED), and pathological complete response (pCR) were ICEs of interest. Correlation of each ICE with OS was evaluated using Bayesian bivariate random-effects meta-analysis (BRMA) with an uninformative prior distribution. The predictive value of each ICE was determined by assessing observed versus predicted treatment effects of OS via leave-one-out cross validation (LOOCV).

We selected 137 unique studies (89 RCTs and 48 non-RCTs) for meta-analysis: EFS-OS, k=85 studies (n=53,965 patients); MFS-OS, k=79 (n=51,746); NED-OS, k=100 (n=56,187); pCR-OS, k=112 (n=70,228). We observed moderate correlation estimates of 0.53 (95% credible interval [Crl]: −0.23, 0.96), 0.69 (95% CrI: −0.35, 0.98), and −0.53 (95% CrI: −0.95, 0.39) for EFS-OS, MFS-OS, and NED-OS, respectively. pCR-OS showed a weak correlation (0.06, 95%Crl: −0.89, 0.94). None of the correlation estimates were statistically significant. An RCT-only sensitivity analysis showed consistent results. The LOOCV had good predictive accuracy, with 95 percent prediction intervals capturing the observed HROS for 89.5, 87.5, 100, and 83.3 percent of studies, for respective ICEs. No ICEs met the Institute for Quality and Efficiency in Health Care (IQWiG) criteria for strong correlation.

Despite consistent direction of associations between ICEs and OS using rigorous BRMA, we could not meet the stringent surrogacy thresholds used by health technology assessment (HTA) agencies. Our findings highlight a need for further analysis to prove correlations, including analyses using real-world data and consideration of how surrogacy evidence is assessed by HTA agencies in early-stage prostate cancer.

Second-line treatment for Philadelphia-positive chronic myeloid leukemia (Ph+CML) in the Brazilian Unified Health System (SUS) currently involves the tyrosine kinase inhibitors (TKIs) dasatinib and nilotinib. Choice of TKIs should be made considering existing comorbidities and the risk of adverse events. Our objective was to estimate the proportion of patients with cardiovascular comorbidities receiving TKIs in the SUS.

A retrospective administrative database analysis was conducted of cases recorded from October 2013 to May 2024. The International Classification of Diseases, 10th Revision codes used for chronic cardiovascular diseases were identified. Two databases of the outpatient information system were analyzed, the oncology database and the individualized outpatient production report, to identify patients receiving dasatinib or nilotinib as a second-line treatment for Ph+CML who had at least one outpatient procedure with an International Classification of Diseases, 10th Revision code of cardiovascular disease. To achieve this, an inner join procedure using the cryptographed identifier was implemented. Cases were characterized by age, sex, disease stage, and geographic region.

A total of 6,556 unique patients receiving second-line treatment for Ph+CML were analyzed. Their mean age was 49.4 (standard deviation 16.2). There was a predominance of males (55.6%) and white (47.6%) people. Cases were concentrated in the southeast (48.4%), the most populous region of the country. Most cases were at the chronic phase (70.8%), followed by the accelerated (17.7%) and blast phases (11.5%). Dasatinib was the most used treatment, representing 52 percent of cases, followed by 40.5 percent using nilotinib. Other regimens included chemotherapy and combined treatments. Patients with cardiovascular disease represented 10.9 percent of the total sample; 9.8 percent of patients treated with dasatinib and 13 percent of patients receiving nilotinib.

Real-world administrative data provided information on patient profile, treatment patterns, and disease severity. Moreover, the proportion of patients with Ph+CML who had cardiovascular comorbidities was estimated at the national level. These elements are necessary to assess unmet needs and treatment patterns not compliant with the recommendations from guidelines, to underpin the design of optimized strategies of care.

CONITEC plays a central role in health technology assessment (HTA), particularly in advancing the evaluation of medical devices (MDs). Tools such as horizon scanning (HS) and the Committee on Products and Procedures enhance its methodologies. This study examined the evolution of MD evaluation over time.

The requests submitted to CONITEC for analysis were carefully identified, quantified, and analyzed in detail. Special attention was given to requests that were specifically directed to the evaluation and recommendation processes carried out by the Commission’s various committees. These requests were thoroughly reviewed to ensure that all relevant aspects were considered. Subsequently, the MD-related proposals were carefully examined, with a particular focus on the incorporation recommendations provided by CONITEC. In addition, the evaluations that led to the creation of HS analyses were closely analyzed, providing insights into the decision-making process and the development of these important evaluations.

Over the last 12 years, CONITEC received 1,082 HTA requests, with 660 originating internally and 422 externally. Of these, 832 were related to medicines, 174 to procedures, and 76 to products. Of the product and procedure proposals, 159 were recommended for incorporation, 35 were not recommended, five resulted in exclusion, one resulted in non-exclusion, and six were closed by CONITEC’s decision. Approximately 18 percent of the MD-related requests led to the creation of HS analyses, particularly in 2023 and 2024 when all demands in this area resulted in HS analyses, reflecting improvements in the evaluation process.

The evaluation of MDs is a rapidly growing field within HTA, requiring the adoption of specific methods for progress. These methods include tools and databases focused on HS as well as tailored methodological guidelines. CONITEC has continuously developed tools to enhance decision-making on the incorporation of MDs into the Unified Health System.

Choosing the optimal ulcerative colitis treatment is complex, given the range of medical and surgical options with varying side effects and effectiveness. Decision aids can improve patient choices, but current tools lack personalization. To address this, we developed a personalized decision tool using a discrete choice experiment (DCE) to help patients make informed decisions about medical or surgical treatments.

An online DCE survey was developed containing competing treatment profiles described using all important aspects of the treatment (effectiveness, side effects, family planning). Patients (n=300) with ulcerative colitis were asked to consider the benefits and disadvantages of each treatment profile and select the treatment that they would choose. The DCE data were analyzed using mixed logit and latent class models. The model results were integrated into an online decision aid using a Shiny application.

R Shiny was successfully used to enable the real-time personalization of DCE results. The developed decision aid contained two aspects of personalization. First, attribute importance scores showed the treatment characteristics that mattered most to patients based on their DCE choices. Second, a “best-match” treatment that aligned with their preferences was provided from uptake rate calculations. User testing of the developed decision aid is ongoing. However, initial feedback from patients has been positive.

A key challenge in developing personalized decision aids is providing real-time, tailored recommendations based on individual preferences. This study demonstrated the feasibility of integrating DCE methods into personalized decision aids for ulcerative colitis. By tailoring treatment recommendations to individual patient preferences, this tool has the potential to empower patients, reduce decisional conflict, and enhance shared decision-making between patients and clinicians.

Health technology assessment (HTA) of Class III implantable medical devices (MDs) in Europe is conducted at the national or regional level. Despite common clinical evidence, sometimes appraisal results and recommendations vary significantly, affecting patient access to innovative technologies. An adaptive approach to HTA for MDs will need to be consistent with current expert thinking on adaptive approaches in HTA methodology.

The websites of HTA agencies across 13 European countries were searched for HTA reports published between 1 January and 31 December 2023 on high-risk cardiovascular MDs used to treat structural heart disease. The goal was to identify the classes of technologies assessed by different HTA agencies and compare the submitted evidence and decisions. Since assessments are sometimes repeated across different years, we reviewed all available submissions for the selected innovations from the CE mark up to 2023 to follow the evolution of appraisals and decisions on the same technology.

Eighty reports from 11 countries were identified, with 23 on structural heart disease from Austria, France, Italy, and Spain. The main diseases involved heart valves, septal defects, and heart failure. In 2023, two agencies reassessed technologies for mitral valve regurgitation (MVR). Austria’s HTA gave negative recommendations to the first device CE marked in 2008 in fourth consecutive assessments, despite three randomized controlled trials (RCTs). A fifth reassessment is planned for 2026. France has conducted 26 assessments since 2015 on MVR devices (due to design changes, indication expansions, generation changes, economic assessments, new devices entering the market, and life cycle reassessment), with a positive recommendation in 2015, which was supported by two single-arm studies and a registry. A second device, CE marked in 2019, was recommended by France in 2023 based on a single-arm trial, an RCT, and two registries.

The number of HTAs performed on MDs remains low, particularly for high-risk device classes. There was no consistency in the number of HTAs performed between countries, nor in the evidence required for a successful assessment. These differences lead to unequal access to specialized treatment for patients in different countries and highlight the need for a unified European HTA approach.

The Cancer Drugs Fund (CDF) is a type of managed entry agreement used in England to allow patient access while undertaking evidence generation followed by re-evaluation to address uncertainty. We assessed the impact of changes to the National Institute for Health and Care Excellence (NICE) health technology assessment (HTA) methods in 2022 plus the publication of a national commercial framework on recommendation outcomes.

NICE staff, in conjunction with clinical stakeholders, assess all cancer medicines for their suitability for entry into the CDF. Cancer topics looked at by NICE from 2016 to 2024 were identified, and the type of evidence uncertainties and the final recommendation outcomes were recorded. Types of evidence uncertainty were assigned to one of nine different categories: survival data, relevant patient population, lack of relevant comparators, trial design, quality-of-life data, cost estimates, treatment duration, cost-effectiveness model, and adverse events. Recommendation outcomes included entry to the CDF, full reimbursement, reimbursement for optimized population, no reimbursement, or research only.

The types of uncertainties identified for appraisals using pre-2022 methods (n=103) and post-2022 methods (n=35) showed the same trends in most common uncertainties. Immature survival data was the most common, with lack of relevant comparators the second most common. There was an increase in the average number of uncertainties seen in post-2022 topics (2.05), compared with pre-2022 topics (1.75). The number of CDF recommendations has been decreasing in recent years, with a peak between 2017 and 2019. For appraisals that did not enter the CDF, the likelihood of a positive recommendation was lower in topics where entry to the CDF had been considered a possibility.

There were no clear changes in trends of uncertainties seen in cancer topics following the 2022 methods changes, though there was a small increase in overall uncertainty. There was a decline in CDF entries, which correlates to payers offering a more flexible commercial environment giving alternative reimbursement options to evidence generation with re-evaluation via the CDF.

Instituto Dara, a Brazilian non-profit organization, supports vulnerable families through multidimensional interventions addressing poverty and chronic illness. While validated quality-of-life instruments are essential for program evaluation, few target disadvantaged populations with chronically ill children in Brazil. This study evaluated the psychometric properties of the EuroQol Health and Wellbeing Short Version (EQ-HWB-S) questionnaire, including its convergent validity and test-retest reliability.

The study included 99 individuals representing families supported by Instituto Dara. Data collection used face-to-face interviews with EQ-HWB-S and additional validated instruments (EuroQol-5D and the Warwick-Edinburgh Mental Wellbeing Scale [WEMWBS]) to assess convergent validity. Test-retest reliability was assessed in 45 participants over a two-week period. Statistical analyses included descriptive methods and correlation analyses to summarize EQ-HWB-S applicability and performance in this population.

Physical domains (mobility and activities) showed minimal impairment, with most participants at level one (>75%). In contrast, psychosocial domains showed significant burden: over 50 percent reported moderate to extreme levels of exhaustion, loneliness, and anxiety or depression. Test-retest reliability showed moderate agreement (r=0.568; p<0.001). The EQ-HWB-S yielded an overall score of 0.707. Construct validity analyses demonstrated moderate correlations with established measures (r=0.33 to 0.45), particularly with the physical well-being domains of the EQ-5D and alignment with the WEMWBS for psychological dimensions.

The results highlighted the significance of EQ-HWB-S in assessing domains such as loneliness, exhaustion, and control. The instrument demonstrated robust psychometric properties with moderate test-retest reliability and strong convergent validity, though its discriminatory ability warrants further investigation. These findings support its use for assessing quality-of-life outcomes in vulnerable populations with chronically ill children.

During the health technology assessment (HTA) process it is possible to identify unmet medical needs (UMN) that can be used to guide research and development investments. This study aimed to develop a methodology to identify and prioritize research and development themes using UMN identification in the HTA process of a teaching hospital.

We reviewed and looked for UMN in all the HTA technical reports related to technology incorporation solicitations submitted between 2018 and 2023. The UMN definition used was based on that published by the US Food and Drug Administration in 2014. A prioritization process was adopted using criteria based on the World Health Organization and the Brazilian Unified Health System list of priorities for the year. The UMN were categorized by characteristics such as indication (diagnostic, therapeutic, preventive, rehabilitation) and type of diseases.

Thirty-seven Structured Technical Reports—formal documents submitted for evaluation of health technologies—were reviewed by two investigators. Among these, 37 UMN were identified, most of which were characterized as therapeutic needs (59.5%) or related to neurological diseases (16.2%). According to established criteria, 81.1 percent of the UMN warranted investment in research and technology development.

The proposed methodology can reliably identify UMN by analyzing technical reports. Further improvements can make it possible to fit the process to diverse HTA systems to support research and development decisions and investments.

The objective of this assessment was to determine the benefit of using a next-generation sequencing (NGS) gene panel for the clinical management of gastrointestinal stromal tumor (GIST) among patients in routine clinical practice. The aim was to assess the clinical utility of this procedure, the somatic molecular alterations of specific interest, and to define its role in the therapeutic care of patients with GIST.

The method used for this fast-track assessment were based on: (i) a critical analysis of systematic reviews, meta-analyses, and clinical practice guidelines identified by a systematic literature search; (ii) identification of the level of evidence of molecular alteration clinical actionability as set out by the European Society for Medical Oncology Scale, of targeted therapies included on the list of reimbursable drugs assessed by the French National Authority for Health, or drugs that have compassionate use authorizations issued by the French National Agency for Medicines and Health Products Safety; and (iii) stakeholder consultations and observations by public health institutions.

Assessment of the evidence and data demonstrated that NGS gene panel testing has: (i) superior diagnostic performance for detecting KIT and PDGFRA molecular alterations, compared with Sanger sequencing; (ii) superior diagnostic performance for detecting NTRK1/2/3 fusion, compared with immunohistochemistry; and (iii) evidence of clinical utility of the targeted gene panel considering the benefits provided by targeted therapies.

The composition of the NGS gene panel may be subject to change, in accordance with favorable assessments of new gene alterations. New assessments will be conducted in a dynamic manner in response to developments in scientific knowledge.

The French National Authority for Health deemed that funding the NGS gene panel for GIST was justified for: (i) KIT and PDGFRA genes in cases of locally advanced or metastatic GIST at an intermediate or high risk of recurrence, and in cases of suspected GIST when histology is inconclusive for diagnosis; and (ii) the NTRK1/2/3 gene in cases of refractory or relapsed locally advanced or metastatic pediatric wild-type GIST.

Diffuse large B-cell lymphoma (DLBCL) is the most common hematological neoplasm in adults globally and in Colombia, but there are limited data on its local economic impact. This study analyzed the relationship between clinical variables and healthcare resource utilization of patients with DLBCL in a Colombian health maintenance organization.

This analytical observational study included patients with histopathologically confirmed DLBCL treated at the Luis Carlos Sarmiento Angulo Cancer Treatment and Research Center (CTIC) between July 2022 and September 2025. We reported an interim analysis with a data cut-off in July 2024. Clinical data from CTIC’s institutional registry were merged with the information provided by the administrative database, covering healthcare resource use and costs for drugs, procedures, and medical supplies. A multivariate linear regression estimated the effect of prognosis (revised International Prognostic Index [R-IPI] score) on total costs, adjusting for confounding (e.g., sex, comorbidities, and history of neoplasm).

Sixty-two patients were included (mean 66 years [standard deviation 14]; 58% men). R-IPI scores at baseline indicated that 34 percent were at high-intermediate risk, 21 percent had low and low-intermediate risk, and 23 percent were at high risk. R-IPI scores at baseline showed that 17.7 percent patients had very good prognosis, while 24.2 and 41.9 percent had good or poor prognosis, respectively (16.1% missing). The R-CHOP chemotherapy regimen was the most common first-line (1L) treatment (73%), and fifteen percent of patients had disease progression. The total 1L treatment cost was COP1,386,178,104 (USD320,500) per patient. Thirteen patients received second-line (2L) treatment, costing COP3,203,033,513 (USD740,577) per patient. Regression analysis showed that patients with poor prognosis, per R-IPI score, incurred 8.9 times higher costs than those with very good R-IPI scores.

Our study showed the association between clinical variables and the cost of treatment in patients with DLBCL. Regression analysis indicated that higher R-IPI scores and advanced disease stages significantly increased costs. The cost per patient for 2L treatment was 2.3 times higher than for 1L treatment, highlighting the importance of timely detection and treatment.

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.