bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP45 Incorporation Of The Tetravalent Dengue Vaccine In The Brazilian Public Health System: A Cost–Benefit Analysis
Authors: Gomes dos Santos Ana Zulmira , Barros Gabriella Santos , Gonzalez Mosegui Gabriela Bittencourt , Martins de Freitas Gabriel Rodrigues 
Published: January 2025

IntroductionDengue, a mosquito-borne disease, is prevalent in Brazil, ranking third in the Americas. The country has confirmed all four serotypes (DENV-1, DENV-2, DENV-3, and DENV-4) of the virus. In 2023, a tetravalent vaccine, approved for individuals aged four to 60 years, opens the possibility of integration into the public health system.MethodsThe invested amount for purchasing two doses of the vaccine to immunize the entire eligible population in the year 2023 was calculated. A comprehensive analysis of direct and indirect costs was conducted regarding the integration of the tetravalent dengue virus vaccine into Brazil’s public healthcare system. Direct costs encompassed treatment expenses for mild, moderate, and severe cases, while indirect costs involved workdays lost and mortality-related expenses. Direct and indirect costs were compared in two scenarios (with and without vaccination), followed by a cost–benefit ratio calculation.ResultsThe investment for procuring vaccines for over 168 million Brazilians amounted to approximately EUR17.9 billion, resulting in savings of about EUR193.5 million. Indirect costs were particularly significant when compared to the non-vaccinated population. Considering herd immunity and reducing the vaccinated population to 70 percent of the eligible populace, the invested amount was approximately EUR12.5 billion, while savings reached EUR214.2 million. A cost-benefit ratio calculation revealed a return of one centavo (EUR0.0019) for every BRL1.00 invested (EUR0.0019), and considering herd immunity, the cost–benefit ratio was approximately 0.02.ConclusionsDespite Brazil being one of the countries with the highest prevalence of dengue in the Americas, the availability of the tetravalent dengue virus vaccine in the country’s public system does not seem to be a sustainable option, given the unfavorable cost–benefit analysis for such implementation. Nevertheless, it is imperative to conduct this analysis with due consideration for alternative scenarios.
OP63 A European Value Assessment Framework For Next-Generation Sequencing And Comprehensive Genomic Profiling Oncology Diagnostics
Authors: Augustovski Federico , Chavez Danitza , Haig Madeleine , Main Caitlin , Argento Fernando , Colaci Carla , Mills Mackenzie , Alfie Verónica , Pichon-Riviere Andres , Alcaraz Andrea , Kanavos Panos 
Published: January 2025

IntroductionAdvances in next-generation sequencing/comprehensive genomic profiling (NGS/CGP) diagnostics require a value framework (VF) for accurate assessment within cancer care in Europe. Building on a previously established VF for diagnostic technologies in Latin America by the Institute for Clinical Effectiveness and Health Policy (IECS), this study synthesizes insights from a systematic literature review and stakeholder perspectives to inform the co-creation of an NGS/CGP diagnostic framework for healthcare decision-making.MethodsThe study utilized a mixed methods approach including a systematic literature review (SLR) and web-Delphi panel. The SLR identified and mapped existing VFs against the IECS VF, extracting non-overlapping criteria relevant to NGS/CGP in Europe. A Delphi panel further adapted and validated the framework, ensuring comprehensive stakeholder contribution and engagement. The Delphi´s first round was qualitative and involved open-ended feedback from participants to adapt the indicators and domains. Rounds two through four involved Likert-scale judgments of importance of each indicator. In rounds three and four, participants were shown the distribution of responses across stakeholders and could reconsider their answers.ResultsThe SLR revealed 42 VFs with an 83 percent criterion overlap with the IECS VF, resulting in 46 indicators forming the literature-adapted framework. Thirty-four participants completed the Delphi. In round one, 14 indicators and 22 descriptions were adapted, 11 indicators were merged, 14 were deleted, and one was kept the same resulting in 29 indicators for scoring in round two. The final VF has 27 indicators: 23 essential and four complementary; two indicators did not reach consensus. The domains included clinical impact, test performance, scientific evidence quality, non-clinical impact, health system integration, economic aspects, ethical/governance concerns, and health system priorities.ConclusionsThis approach has yielded a robust, stakeholder co-created VF for NGS/CGP diagnostics in oncology, tailored to the European setting. It offers a comprehensive set of criteria that extends beyond traditional health technology assessments, incorporating novel aspects like post-test data governance. This framework sets the stage for improving patient access to high-value technologies by aligning European stakeholder values across health systems.
OP65 Focusing On What Matters Most: A Public Dialogue On How NICE Should Prioritize Topics In Health Technology Assessment
Authors: Murray Alice , Shah Koonal 
Published: January 2025

IntroductionTo meet the needs of an evolving health and care system, the National Institute of Health and Care Excellence (NICE) is changing its approach to topic prioritization so it can focus on what matters most. To support this, NICE ran a public dialogue to gather informed opinion on how it should select topics for guidance, including for some technology evaluation programs.MethodsFifty-five general public participants from across England took part in two face-to-face and three online deliberative workshops (each lasting two or three hours, held over four weeks in 2023). Participants were asked to consider the following criteria in the context of prioritization: health and care need, evidence availability, system impact, budget impact, health inequalities, and environmental sustainability. The workshops were designed to understand whether any aspects were more important than others and explore the reasons why. They used deliberative engagement methods and included trade-off exercises, role-play, group discussion, ranking tasks, and interactions with specialists.ResultsEmerging findings show that the participants think NICE should consider several aspects when prioritizing topics for guidance. Health and care need was of primary importance for people, followed by evidence availability, budget impact, and system impact. Health inequalities and environmental sustainability were generally considered to be less important, though participants still felt these were areas that should inform NICE’s prioritization decisions. Participants identified relevant interactions between the criteria, suggesting that each criterion cannot be considered in isolation. Full results will be available to present at HTAi 2024.ConclusionsDeliberative public engagement is a meaningful way to involve the public in complex policy decisions with a social value element. Broad public agreement was found with the criteria NICE has proposed to consider when prioritizing topics for guidance, and some criteria are more important to people than others. The findings will feed into NICE’s new approach to topic prioritization.
OP80 Generating Patient Preference Evidence For Health Technology Assessment: A Sustainable Roadmap
Authors: Fifer Simon , Joshi Maya , Keen Brittany 
Published: January 2025

IntroductionResearch with members of health technology assessment (HTA) bodies has uncovered key barriers to integrating patient preference (PP) data into HTA, including concerns about resources/time constraints and a lack of clarity around who is responsible for data generation. We sought to develop a roadmap that addresses these issues, outlining the roles and responsibilities of different stakeholders to foster more sustainable PP data generation.MethodsBased on a forthcoming article to be published in The Patient, this roadmap consists of a step-by-step approach for PP evidence generation. Real-world case studies and literature will be used to illustrate each stage, from identifying priority treatment areas and evidence gaps, forming a steering committee and engaging HTA members, to securing syndicated funding and disseminating results with full transparency.ResultsIn contrast to standard approaches to data generation, this roadmap focuses on proactive data collection, collaborating with those who will ultimately use the data (HTA), and pooling resources to mitigate costs and the risk of bias. The roadmap can be applied to all preference-sensitive treatment areas and across health systems/countries. It is designed to be a continuous process, whereby preferences are regularly updated to align with changes to the treatment landscape. A graphic summary of the roadmap is available for viewing at this link: https://cappre.info/images/HTAprocess.pdfConclusionsPatient preference data has the potential to make healthcare decision-making more informed, socially legitimate, transparent, and accountable to the patient community. However, current approaches to capturing PP data can be resource intensive with narrow applicability in their findings. The present roadmap offers an alternative, sustainable solution.
OD01 Delays In Funded Access To Medicines: A Global Perspective
Authors: Laka Mah , Gao Yuan , Carter Drew , Merlin Tracy 
Published: January 2025

IntroductionThere are significant delays in the funded access to medicines. Studies indicate that in many countries it takes more than a year for patients to have funded access to medicines after market authorization. This study aimed to understand the disparities in timelines for funded access to medicines across different countries and to identify underlying reasons for this access gap.MethodsWe conducted a scoping review to examine the nature of health technology assessment (HTA) processes, current methods, and policies for medicines in ten jurisdictions. The jurisdictions included in this study are Australia, Canada, France, Germany, South Korea, the Netherlands, United Kingdom (divided into England, Scotland and Wales), and United States of America. The information was extracted from the websites of International Network of Agencies for Health Technology Assessment (INAHTA) member agencies in the selected jurisdictions, grey literature from governments’ websites, and peer-reviewed literature.ResultsOverall median time from submission of the evidence dossier to HTA recommendations for most jurisdictions is 22 weeks. Although there are similarities in the time taken to reach a funding decision, there are considerable variations in the time taken for patients to have funded access to medicines after HTA recommendations. Only a few countries mentioned a specific timeline within which medicines approved for funding should be listed. Time taken for price negotiations and other arrangements (i.e., risk-sharing agreements) may contribute to varying timelines for listing medicines for funding. Mostly, such negotiations are confidential and may not be time limited.ConclusionsThere was surprising consistency, globally, in the time it takes for funding decisions after medicines registration. The causes of delays in the medicines’ listing decisions are multifactorial and mostly occur after HTA recommendations. The parallel regulatory-assessment process and prioritization tend to reduce the time to a funding decision. However, transparency is needed in the listing process to improve overall timeliness.
OD09 How Are Population/Intervention/Comparator/Outcomes Criteria For Pharmaceutical Assessments Determined Around The World?
Authors: Newton Skye , Hill Hayley , Merlin Tracy 
Published: January 2025

IntroductionDefining the population, intervention, comparator, outcomes (PICO) criteria is an essential step prior to performing a health technology assessment (HTA), but variations exist in how this step is performed.MethodsA scoping review was performed to compare the processes and guidance provided for developing the PICO criteria for the assessment of new medicines across Australia, the UK, Canada, the US, European Union (as a single jurisdiction), Germany, France, the Netherlands, South Korea, and Taiwan. The websites of HTA agencies in these jurisdictions were searched for methodological guidance, and PubMed, Embase, and the HTA database were also searched for published literature on the topic of the process or methods for developing the PICO criteria.ResultsTwo main approaches are used for developing the PICO criteria. In the UK, US, and European Union, a separate scoping process is used; in the remaining countries, the pharmaceutical manufacturer defines the PICO criteria as part of developing their dossier for submission. Guidance on PICO elements were similar in content but highly varied in the degree of guidance provided. The largest differences were in whether outcomes for people beyond the treated individual were recommended to be assessed.ConclusionsA separate scoping phase allows stakeholder input into the criteria, which is important with the shift to incorporating more patient input into each phase of HTA. It can come at the cost of timeliness, so requires manufacturers to engage with the HTA systems earlier in the process.
OD15 Multiparametric Magnetic Resonance Imaging In Biopsy-Naive Patients: A Real-World Evidence For Decision-Making In Prostate Cancer Pathway
Authors: Asselin Genevieve , Tremblay-Boily Melanie , Fradet Yves , Rhainds Marc 
Published: January 2025

IntroductionUse of multiparametric magnetic resonance imaging (mpMRI) in the prostate cancer (PCa) diagnostic pathway could reduce prostate biopsy (Bx) in Bx-naive patients. The objective was to assess the feasibility of implementing a new PCa diagnostic pathway with the addition of mpMRI in a real care environment.MethodsFollowing an HTA report published in 2019 by our team, a committee involving stakeholders (e.g., urologists, radiologists, hospital managers) was created to review the PCa diagnostic pathway including mpMRI in Bx-naive patients and both systematic and targeted 3DTRUS-MRI fusion Bx when Bx was recommended. Data in the new PCa diagnostic pathway were collected between September 2021 and June 2022. The comparison group is a cohort of 629 men who underwent an initial systematic transrectal ultrasound Bx in 2017 when prostate mpMRI was not available. Clinically significant PCa (csPCa) was defined as Grade Group ≥2.ResultsIn 2021 and 2022, 1,336 Bx-naive patients were referred to a urologist. Recommendations were: 703 (53%) for follow-up in six to 12 months, 254 (19%) directly to systematic Bx, and 379 (28%) to mpMRI. Overall, csPCa was diagnosed in 246/427 (58%) patients referred to mpMRI or Bx in the 2021 to 2022 cohort compared to 274/629 (44%) patients in the 2017 cohort (p<0.05). The new diagnostic pathway prevented 33 percent of patients from having Bx. Shorter delays between initial consultation with urologists and transmission of Bx results were observed for patients referred directly for prostate Bx compared to mpMRI before Bx (mean: 2.8 vs 9.1 months).ConclusionsExperimentation in a real care setting has highlighted the added value of the early involvement of urologists in the diagnostic pathway of prostate cancer for the triage of patients. Integration of mpMRI was associated with a lower number of patients referred for prostate Bx and a higher csPCa detection rate.
OD24 Scalability Analysis Of Multimodal Prehabilitation For Frail Elderly Patients Before Elective Surgery In Germany
Authors: Felgner Susanne , Eckhardt Helene , Weber Zoe , Quentin Wilm , Rombey Tanja 
Published: January 2025

IntroductionMultimodal prehabilitation, including interventions like physiotherapy, combined with frailty screening and a shared decision-making conference for frail elderly patients before elective surgery is an innovative approach currently under investigation (PRAEP-GO RCT, NCT04418271). The PRAEP-GO intervention aims to enhance postoperative outcomes and prevent care dependency. Our aim is to systematically assess the scale-up potential of PRAEP-GO within the German healthcare system.Methods
We are conducting a scalability analysis using the Intervention Scalability Assessment Tool (ISAT). The ISAT questionnaire comprises two parts: (A) “Setting the scene,” describing the current health service situation (e.g., intervention characteristics, political context), and (B) “Intervention implementation planning,” outlining future requirements (e.g., workforce, infrastructure), with open-ended questions and a scalability readiness assessment using a scale. Our analysis involves three stages: (i) health economists from the PRAEP-GO research team individually answering ISAT questions, (ii) trialists from the PRAEP-GO research team interviewed in a group, and (iii) external experts representing relevant stakeholders for future implementation interviewed in an advisory board meeting.ResultsData collection for stage (i) and (ii) has been completed, while data collection for stage (iii) is expected to be completed in February 2024. The preliminary findings for part (A) highlight the need for a sustainable approach to manage an aging and increasingly frail patient population requiring surgery. There is no clinical guideline available for the management of this population group. Regarding part (B), the current infrastructure (e.g., therapy facilities) and personnel structures might need to be adapted and should be expanded for large-scale application. Employing professionals to coordinate the patient pathway was recommended, along with adjustments to reimbursement structures.ConclusionsIn PRAEP-GO, we are pursuing a multidisciplinary process with the aim of supporting health decisions that promote an equitable, efficient, and high-quality healthcare system meeting the challenges of an aging population due to demographic change. The PRAEP-GO trial is currently exploring this approach on a small scale. Existing infrastructure and personnel structures would need to be adapted and expanded for scale-up.
OD37 How The PICOTS-ComTeC Framework For Digital Health Interventions Can Be Useful For Health Technology Assessment
Authors: Zrubka Zsombor , Champion Annette , Di Bidino Rossella , Holtorf Anke-Peggy , Earla Jagadeswara R , Boltyenkov Artem , Tabata-Kelly Masami , Asche Carl , Burrell Anita 
Published: January 2025

IntroductionThe diffusion of digital health interventions (DHIs) requires agreement on a minimum information framework to define them. The ISPOR Digital Health (DH) Special Interest Group (SIG) developed the PICOTS-ComTeC framework based upon a systematic review and a Delphi study. It is an enriched version of the traditional PICOTS widely adopted in health technology assessment (HTA). ComTeC stands for communication, technology, and context.MethodsThe PICOTS-ComTeC is based upon a review that included the Shannon–Weaver model of communication, AHRQ Quality Measures, technology, geography, and the World Health Organization classification of DHIs followed by a Delphi panel. The development process adhered to the EQUATOR guidelines. The PICOTS-ComTeC aims to be a flexible and versatile tool tested on different DHIs. The results of the testing are discussed from the HTA perspective considering the tool’s additional value, utility for and applicability to HTA. The additional value is strictly linked to the actual need for a dedicated PICOTS for DHIs and its implications for HTA assessments of DHIs.ResultsThe PICOTS-ComTeC was tested internally and externally to the ISPOR DH-SIG on four DHIs for breast cancer surgery/management/patient education, one DHI for obesity, and one DHI for patients with heart failure. The testing phase demonstrated the level of detail required to use the tool, hitherto available evidence to cover all domains, and opened up discussion on implications of the PICOTS-ComTeC framework for HTA related activities (i.e., scoping, literature search, comparator selection). It emerged that there is a diffuse lack of homogeneity and details when DHIs are defined in the literature with significant implications for conducting appropriate HTAs.ConclusionsThe diffuse adoption of the PICOTS-ComTeC for patient-facing DHIs will promote a greater level of detail in order to define homogenous DHI groups. The implications for HTA range from the definition of relevant research questions to the selection of the most appropriate comparator so that assessments are geared to fulfill the needs of decision-makers.
OD41 Conducting Systematic Reviews On Rare Diseases: Lessons Learned From The European Reference Networks Guidelines Programme
Authors: Martín-Gómez Carmen , Isabel-Gómez Rebeca , García Guillermo Pérez , Vicente Edo Maria José , De Armas-Castellano Aythami , Trujillo-Martín Mar , Pla Anna Godo , Estrada Sabadell Maria Dolors , García Josune Domínguez , Jausoro Ainhoa 
Published: January 2025

IntroductionA consortium of five Spanish health technology assessment (HTA) agencies conducted the European Reference Networks Guidelines Programme for the development, appraisal, and implementation of clinical practice guidelines aiming to support clinical decision-making in the field of rare diseases (RDs). In response to this objective, methodologists and information specialists conducted systematic reviews (SRs). This study aims to explore the barriers/facilitators they encountered.MethodsA survey was designed to elicit HTA agencies’ experience in developing SRs on RDs. Information was collected on the number of SRs conducted and the types of RDs and clinical questions addressed. In addition, they were asked to identify barriers and facilitators for each stage of the review (from the definition of PICO [population, intervention, comparator, outcome] components of the question to the issuing of recommendations). Finally, they were asked for process improvement suggestions. The survey was distributed by email and completed online. A thematic analysis was conducted to identify the issues identified at each stage of SR.ResultsA total of 111 SRs were conducted on 35 RDs. Most clinical questions were about diagnosis and treatment. The main barriers identified were lack of MesH (Medical Subject Headings) terms associated with the conditions, non-representative abstracts and keywords, lack of relevant information in the body of the articles, and reported data not allowing for quantitative syntheses or recommendations to be made. Facilitating aspects included Orphanet’s specific source of RD documents and having expert clinicians in the working groups who were also involved in all steps of the SR.ConclusionsConducting SRs in the field of RDs is challenging. Authors of primary studies are encouraged to be more exhaustive in reporting the results. More research focused on the SR methodology in RDs is necessary to address their particular characteristics and obtain robust results. It is crucial to collaborate with reference networks to address RDs, where the evidence is scarce.