bluuebunny/crossref_metadata_2025_split huggingface dataset:
OD43 The Growing Clinical And Economical Burden Of Chronic Kidney Disease In Brazil: An IMPACT CKD Analysis
Authors: Moreira Ricardo , Regis Cassio , Grizolli Beatriz , Montenegro Cinthia , Gobbi Talita , Rao Naveen , Priest Stacey , Brown Stephen , Guiang Hannah , Moura Ana Flavia , Zhao Ming-Hui , Chadban Steven 
Published: January 2025

IntroductionThe growing burden of chronic kidney disease (CKD) in Brazil is increasingly evident, marked by its significant contributions to mortality rates and healthcare costs. Managing CKD, especially through renal replacement therapy (RRT), demands substantial resources. To enhance healthcare decision-making, a thorough examination of the relationship between the rising prevalence of CKD and its clinical and economic impacts is crucial.Methods
We developed a patient-level simulation model to project the natural history of CKD, defined as the IMPACT CKD. This model integrated factors such as acute kidney injury, cardiovascular events, and comorbidities, and aimed to assess CKD’s clinical, humanistic, and economic impact on the healthcare system. It forecasted the burden of CKD over the next decade (2023 to 2032). This projection is pivotal to derive the burden of CKD for health technology assessment (HTA) evaluations. Validation was conducted against Brazil’s demographic data and cross-validated with the Inside CKD model.ResultsThe IMPACT CKD forecast a rapid increase of CKD population in Brazil, outpacing the growth of the general population. Specifically, there is an expected 6.9 percent increase in stages 3 to 5 CKD, leading to a higher demand for dialysis (projected 370,000 cases in 2032) and transplants (projected 115,000 cases in 2032). A significant increase in cardiovascular CKD-related events (+100.6%) and mortality (+67.8%) is expected. In 2032, it is projected 15 million CKD patients will be in stages 1 to 2, and 12.7 million in stages 3 to 5. CKD-related healthcare costs will represent 25.7 percent of Brazil’s healthcare budget, and dialysis will reach USD2.7 billion in annual costs.ConclusionsIMPACT CKD predicts an increasing CKD prevalence and an alarming rise in stages 3 to 5 and RRT, including thousands of premature deaths, and a substantial economic burden on the Brazilian healthcare system. This data could be informative for healthcare decision-makers when choosing strategy to reduce the impact of CKD in Brazil.
PP01 Early Health Technology Assessments Of Health And Well-Being Returns On Investment In The Biobanks
Authors: Marquina Clara , Lloyd Melanie , Ng Wayne , Hess Jonas , Evans Sue , Ademi Zanfina 
Published: January 2025

IntroductionHuman tissue biobanks provide vital infrastructure to support both basic science and clinical research, but their economic value in terms of attributable population health gains is unclear. We evaluated the population health returns from investment in the Victorian Cancer Biobank (VCB). The VCB comprises five hospital-integrated sample repositories and a central lead agency located in Melbourne, Australia.Methods
This evaluation assigned monetary values to the health gains attributable to VCB-supported public-funded research. These were then compared to the total investment in VCB infrastructure since inception (2006 to 2022) to determine the return on investment (ROI). A time lag of 40 years was incorporated, recognizing the delay from investment to impact in scientific research. Health gains were therefore measured for the years 2046 to 2066, with a three percent discount rate applied. Health gains were measured in terms of disability-adjusted life years (DALYs) attributable to VCB-associated research, with monetary cost assigned via the standardized value of a statistical life year (AUD227,000 [USD149,883]).Results
The age-standardized DALY rate attributable to cancer was modeled for two standpoints: (i) extrapolating the current decreasing trajectory and (ii) assuming nil future improvement from current rates, with 33 percent of the difference attributed to scientific innovation. The proportion of the aggregate health gain attributable to VCB-supported research was estimated from the number of VCB-credited scientific publications as a proportion of total oncology publications over the same period. The AUD32,628,016 [USD21,554,571] of public funding invested in VCB activities over the years 2006 to 2022 generated AUD84,561,373 [USD55,868,539] total savings. Return on investment was AUD1.59 [USD1.05] for each AUD1 [USD0.66] invested.Conclusions
The VCB offers a strong return on investment in terms of population health impacts, justifying the use of public funds and supporting the use of biobanks to advance scientific research. Future health technology assessments could capture the total impact of research on the role of the biobanks attributed to research outputs.
PP02 The Application Of Care Pathway Analysis And Economic Modeling In Early Health Technology Assessment: Learnings From Two Projects
Authors: Gregg Emily , Butler Karin , McCool Rachael , Graziadio Sara 
Published: January 2025

Introduction
Early health technology assessment (eHTA) can help to explore the potential value of a technology in the early stages of development. Care pathway analysis (CPA) is a method to identify and map clinical decisions in the current and new care pathways (including the new intervention). This work provides examples of applying CPA within the context of eHTA for medical interventions.Methods
CPA usually involves a pragmatic review to identify and synthesize national/international guidelines that describe the care pathway for the condition of interest. This is typically followed by a qualitative evaluation that can include semistructured interviews with thematic analysis. Interviews with experts are undertaken to understand where (and why) real-world practices differ from published guidance and to validate the care pathway. They also help to evaluate the strengths/weaknesses of the new technology, potential population and role in the pathway, and barriers/facilitators to adoption. The CPA forms the basis of economic modeling that helps assess the monetary value of the new technology.ResultsThe application of CPA from two recent projects will be presented: an innovative diagnostic test for respiratory tract infections and a medical device for treating cataracts. Additionally, the value of CPA in eHTA will be described from the technology developers’ perspective. In both projects, CPA was used to inform the potential value propositions of the new technology and its positioning in the care pathway. It also helped to optimize the structure of the early economic model and to identify evidence generation needs. The early model identified the pathway that was more likely to be cost effective in the future.ConclusionsCPA is a valuable method within the context of eHTA. Alongside identifying the potential role and positioning of the new technology, test developers found the assessment useful for informing internal strategy decisions and discussions with potential external investors. The developers were able to demonstrate the clinical perspective around the value of the test, elicited through an independent and rigorous methodology.
PP10 Addressing The Challenge Of Environmental Sustainability Between Management And Health Technology Assessment At The Hospital Level
Authors: Bobini Michela , Di Bidino Rossella , Lipska Iga , Cicchetti Americo 
Published: January 2025

IntroductionSeveral organizations specializing in health technology assessment (HTA), at various levels, are exploring avenues to integrate the dimension of environmental sustainability (ES) within their assessments. However, evidence remains scant on how best to do this. Across the different levels, hospital-based HTA (HB-HTA) also needs to comply with the leadership and strategies of the hospital and adapt to the existing resources and established partnerships.MethodsThe purpose of this study is threefold: to provide an overview of the progress achieved by hospitals with respect to the integration of ES into HB-HTA; to detect consistencies between HB-HTA activities and hospital ES strategies; and to explore possible approaches and methods for integrating ES into HB-HTA. The methodology is structured in two steps: (i) a literature review on HB-HTA and environmental impact/sustainability to identify emerging approaches and methods for integrating environmental sustainability into HB-HTA; (ii) data collection by means of a questionnaire submitted to hospitals/organizations based on their membership of Health Technology Assessment International.ResultsPreliminary results highlight a widespread awareness of the importance of integrating ES issues into HTA at the different levels at which it is performed. The incorporation of ES issues into HB-HTA is still in its infancy. There are some factors hindering the development of methods, such as the absence of scientific evidence on emerging approaches and the scarce availability of data or difficulty in tracing data back to a specific technology.ConclusionsThe integration of the dimension of ES into HB-HTA still appears to be very immature. Coordination between the different actors in the system seems necessary to overcome the obstacles identified.
PP11 Evaluating Environmental Sustainability In Health Technology Assessment: A Multisectoral Systematic Review
Authors: Pegg Melissa , Bouttell Janet , Taylor Matthew 
Published: January 2025

IntroductionResearch widely reports healthcare technologies contribute substantial environmental impacts. Timely health technology assessment (HTA) environmental sustainability (ES) framework development is critical. A lack of multisectoral expertise, resource constraints, and consensus to assess environmental impact is delaying methodological progression. The study objective was to identify and critically evaluate methods supporting ES in HTA and formulate recommendations to underpin the development of an ES framework in HTA.MethodsThis multisectoral systematic review followed PRISMA guidelines. Maximizing the opportunity to evaluate applied environmental assessment methods, the databases Web of Science, Embase, PubMed, IEEE Xplore, EBSCOhost GreenFILE, Cochrane Library, and International Network of Agencies for Health Technology Assessment (INAHTA) were searched between 2008 and 2023. Full-text published studies applying both qualitative and quantitative methods to evaluate technology ES were included. Frameworks were critiqued for their comprehensiveness based on sustainability scope and in challenging barriers to decision-making. Studies were ranked according to how transparent and feasible frameworks were in their ability to assess technology ES and alignment with HTA principles.ResultsA total of 10 studies were identified and ranked in order of suitability in assessing technology ES in HTA. All studies applied a combination of methods to overcome issues such as data and resource constraints, expert knowledge, consensus in decision-making, and multiple criteria trade-off. Ranked highest, the One Health “extended” life cycle assessment (LCA) framework that utilized SimaPro LCA advanced software addressed the greatest methodological needs for HTA. Ranked second, the circular economy (CE) framework used in conjunction with an analytical hierarchy process (AHP) makes use of weighting and expert consultation to support multiple-criteria decision-making (MCDM).ConclusionsThis is the first systematic review to identify multidisciplinary frameworks, supporting evaluation of ES and decision-making in HTA. Providing valuable insight into potential methodological solutions where there is limited research, this study facilitates ES policy development in HTA. This study challenges limitations to methodological development highlighted by previous research. Further research should apply these recommendations in an HTA setting.
PP20 Health Technology Assessment To Sustain The Health Insurance Scheme: A Case Study On Anemia Treatment In Ghana
Authors: Amankwah Ivy , Heupink Lieke , Mensah Annan Ruby Aileen , Asare Brian , Owusu Richmond , Abassah-Konadu Emmanuella , Asante Hannah , Chola Lumbwe , Frønsdal Katrine 
Published: January 2025

IntroductionIn Ghana, the reimbursement of iron polymaltose complex (IPC) for iron-deficiency anemia treatment raises concerns as a potential cost driver for the National Health Insurance Scheme (NHIS). Prioritizing value for money is crucial for NHIS sustainability. With Ghana’s established health technology assessment (HTA) framework, we provide insights and preliminary findings in this case study on anemia treatment.MethodsTo further support institutionalization, we follow the Ghana HTA process guidelines and Ghana HTA reference case. Simultaneously, to build wider capacity for HTA in Ghana, relatively new trained staff was engaged while anchoring the team at the HTA Secretariat from the Ministry of Health. We started a situational analysis, which includes desktop review and key-stakeholder interviews. An umbrella review was commenced simultaneously to identify systematic reviews assessing clinical effectiveness of IPC compared to other medicines on the essential medicine lists for all population groups. We will assess cost-effectiveness to ultimately inform coverage decisions and possible implications for organizational structures.ResultsAnemia is a persistent public health issue in Ghana, impacting children under five, young women, and expectant mothers. The essential medicine list includes various treatments, but ambiguity exists in the standard treatment guidelines regarding when to administer IPC versus alternative medicines. Under the NHIS, the intravenous formulation of IPC stands out as the most expensive treatment. A systematic review identified three papers, focusing on infants, children, adults, and pregnant women. Preliminary findings suggest weak overall evidence supporting IPC’s superiority, although adults may exhibit higher tolerance for IPC compared to alternative treatments.ConclusionsConducting an HTA on anemia treatments, despite the low treatment costs, is still significant as technical efficiencies can be achieved with high-volume drugs by analyzing prescription patterns and generating evidence to support potential changes. Findings from this HTA can be used elsewhere, especially in resource-constrained settings. Next steps involve finalizing the HTA and disseminating results to stakeholders.
PP21 Examining Clinical Evidence: Incorporation Of Medications For Ultra-Rare Diseases – Descriptive Analysis Of Conitec Reports (2012 to 2022) – Brazil
Authors: Machado-Rugolo Juliana , da Silva Pereira Curado Daniel , Komoda Denis Satoshi , Modelli de Andrade Luis Gustavo , de Paula De Sordi Mônica Aparecida , Fonseca Alan Francisco , Coneglian de Almeida Juliana Tereza , Lima Silvana Molina , Theresa Weber Silke Anna , Santos Marisa , Tarride Jean-Eric , Thabane Lehana , de Almeida Cardoso Marilia Mastrocolla 
Published: January 2025

IntroductionThe decision-making process for health technology assessment (HTA) in ultra-rare diseases faces a significant challenge for agencies worldwide. This study sought to offer an analytical overview of the clinical evidence outlined in the recommendations of the Brazilian National Committee for Health Technology Incorporation (Conitec) in ultra-rare diseases.Methods
Data were extracted from recommendation reports for the ultra-rare diseases evaluated between 2012 and 2022. To classify a disease as ultra-rare, the epidemiological criterion or a consultation with the Orphanet platform was used (prevalence of ≤1/50,000 inhabitants). The extracted variables included the type of evidence synthesis, type of studies, instrument, the result of the assessment of the methodological quality of the studies, the format of evidence synthesis presentation, whether the evidence was graded, and the result.ResultsAmong 53 analyzed reports, 70 percent relied on randomized controlled trials, followed by systematic reviews (SR), and observational studies. Reports with positive recommendations based on SR comprised 63 percent. GRADE applied to 27 reports and indicated low or very low results for the first two outcomes (62% and 65%). No clear link between evidence quality and final recommendations was observed. Meta-analysis-based reports had 83 percent positive recommendation rate, compared to 55 percent without meta-analysis. Surrogate outcomes were predominant. Clinical characteristics significantly influenced final decisions, especially when new data emerged in public consultation or had the potential to alter disease progression, reduce severe events, or enhance survival.ConclusionsUltra-rare diseases pose challenges in evidence quantity and quality. Traditional HTA frameworks seem inadequate, lacking robust evidence for these conditions. The difficulties in ultra-rare disease HTA underscore the need for specialized frameworks. This analysis acknowledges limitations, notably the heterogeneity in older report structures compared to recent ones, reflecting evolving HTA methodologies in Brazil.
PP24 Creating A Comprehensive Open-Access Health Technology Assessment (HTA) Policy Research Database Through Automated Data Extraction From HTA Reports
Authors: Versteeg Jan-Willem , Bloem Lourens , Leopold Christine , De Bruin Marieke , Mantel-Teeuwisse Aukje , Goettsch Wim 
Published: January 2025

IntroductionFindable, structured, and understandable data from health technology assessment (HTA) reports is the core of HTA policy research. Available databases with this information, such as the International Network of Agencies for Health Technology Assessment (INAHTA) database, may be incomplete and their common manual data collection is time-consuming. Automated data extraction may offer a solution by creating a standardized, real-time-updating, comprehensive, open-access HTA database.MethodsIn this research, we explore the possibilities of automated data extraction in the context of creating a standardized and comprehensive HTA policy research database. Data points were extracted from publicly available guidance reports of the National Institute for Health and Care Excellence (NICE) using different text extraction techniques such as natural language processing (NLP) and generative pre-trained transformers (GPTs). Future efforts are aiming to expand the database to other HTA bodies and link it to the European Medicines Regulatory Database (EMRD) that is also being developed.ResultsPreliminary results of our research show that it is possible to use existing text extraction techniques to extract relevant information from publicly available HTA recommendations. Scaling the system to include more HTA bodies and data points is challenging as extraction based on document structure is complicated by heterogeneity in document structure within HTA bodies and between HTA bodies. Future results will focus on finding the best data extraction approach for each data point and on validating the system.ConclusionsUsing automated data extraction to extract data from HTA reports can be a viable option for creating a comprehensive database that can be used to enhance comparative HTA policy research. Challenges remain in scaling the system to include more HTA bodies and data points. Results regarding best-performing extraction techniques and data validation of the system are expected soon.
PP29 Social Preferences In Health Technology Assessments For Rare Diseases: A Systematic Literature Review Of New Analytic Approaches
Authors: Vasquez Paola , Hall Lisa , Merlo Gregory 
Published: January 2025

IntroductionRare diseases (RD) can be severe and can dramatically reduce life expectancy and quality of life. RD therapies, mostly orphan drugs (OD), fail to meet the standard criteria for public reimbursement due to uncertainty in cost-effectiveness estimates limiting healthcare access. To address this issue, experts have suggested the integration of social preferences into health technology assessments (HTAs) by implementing different methods.MethodsThis systematic literature review, performed in 2021, aimed to explore worldwide experiences of social preferences integration into HTAs for RD and OD through the implementation of multiple-criteria decision analysis (MCDA), discrete choice experiments (DCE), and person trade-off (PTO), among other methods. A systematic search of the literature was conducted using PubMed, Cochrane, Embase, and Scopus databases. The PRISMA approach was used for the review phases. Finally, the Promoting Action on Research Implementation in Health Services (PARIHS) framework was used to discuss the implementation of these instruments in the RD context.ResultsThirty-three articles met the inclusion criteria. The studies measured social preferences for RD and OD as part of HTA using MCDA (n=17), DCE (n=8), and PTO (n=4), among other methods (n=4). These found that patients and clinicians do not prioritize funding based on rarity. The public is willing to allocate funds only if OD demonstrates effectiveness and improves the quality of life (QoL), considering as relevant factors disease severity, unmet needs, and QoL. Conversely, HTA agency experts preferred their current approach and placed more weight on cost-effectiveness and evidence quality even though they expressed concern about the fairness of the drug review process.Conclusions
MCDA, PTO, and DCE, among others, are helpful and transparent methods for assessing social preferences in HTAs for RD and OD. However, their methodological limitations, such as arbitrary criteria selection, subjective scoring methods, framing effects, weighting adaptation, and value measurement models, could be hurdles to implementation. Further research is needed to tailor these methods’ applicability and impact in different social contexts.
PP30 The Health System Impact Of Returning Rare Disease Variants As Secondary Findings From Genomic Sequencing: A Population-Based Model
Authors: Dragojlovic Nick , Kopac Nicola , Llorian Elisabet Rodriguez , Lehman Anna , Lynd Larry D. 
Published: January 2025

IntroductionThe American College of Medical Genetics and Genomics (ACMG) recommends that pathogenic variants linked to 37 genetic diseases be disclosed as secondary findings (SFs) to patients undergoing genome-wide sequencing (GWS), including rare diseases (RDs) treated with enzyme replacement or gene silencing/replacement therapies. We estimated the potential budget impact of treating these RD patients in the US and Canada.MethodsA population-based model was used to estimate the number of patients that would be identified in a given jurisdiction for the 11 diseases on the ACMG’s SFv3.2 list classified as “inborn errors of metabolism” or “miscellaneous phenotypes.” Genetic and clinically diagnosed prevalence was estimated for each gene-phenotype pair based on the scientific literature. Demographic and GWS utilization data were obtained from government websites and payer reports. Drug costs were obtained from the literature and payer websites and reported in 2023 US dollars. A range of one-way sensitivity and scenario analyses were conducted, and province- and state-level estimates were also generated.ResultsBased on an estimated current annual GWS utilization rate of 77 per million in the US, 237 RD SFs would be identified annually (0.9% of 25,663 patients tested), out of which 107 (45%) would be variants linked to hereditary transthyretin amyloidosis (hATTR). In Canada (GWS utilization rate of 169 per million), 45 patients would be identified per year, of which 11 (24%) would be hATTR. Treating 50 percent of hATTR SF patients with patisiran would cost approximately USD26.0 million in the USA and USD2.8 million in Canada annually. In contrast, no cases of RPE65-related retinopathy would be detected at current utilization rates.ConclusionsThe addition of hATTR (with an estimated genetic prevalence of one in 240 in the US) to the ACMG SF list may result in a significant number of additional cases diagnosed per year. While there is an ongoing debate about initiating presymptomatic treatment, reimbursement requests for high-cost drugs like patisiran are likely to increase if GWS utilization continues to grow.