bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP36 Real-World Data From Early Access Programs In France: A Two-Year Review
Authors: Thomassin Camille , Fernandez Judith , Galbraith Margaret , Babin Céleste , Bardin Marion , Diatta Thierno , Desbiolles Alice , Beaufils Alexandre , Pelon Floriane , Cochat Pierre 
Published: January 2025

IntroductionThe 2021 reform of early access (EA) for medicinal products in France includes a mandatory data collection for each patient. The objective of this work was to perform a two-year review of the protocols for therapeutic use and data collection (PUT-RD) validated by the health technology assessment body (Haute Autorité de Santé [HAS]) in collaboration with the national regulatory body where applicable.MethodsThe minimum dataset defined in the PUT-RD varies depending on the regulatory status of the product and may include patients’ characteristics, conditions of use, efficacy, and safety data. A descriptive review of the PUT-RDs approved between 1 July 2021 and 30 June 2023 was carried out by HAS. The following information was retrieved in each published PUT-RD: type of EA (pre- or post-marketing authorization); collection of efficacy data (yes or no) (if yes, inclusion of a patient-reported outcome measure [PROM] or no); type of data source (registry or electronic platform provided by a contract research organization [CRO]).Results
During the review period, a total of 98 PUT-RDs were validated, corresponding to 98 EA favorable decisions. EAs were authorized prior to marketing authorization in 40 percent of the cases. A collection of follow-up efficacy data was planned in 52 PUT-RDs, including integration of patients’ perspectives through a PROM in 32 PUT-RDs. Data collection through a registry was planned in six PUT-RDs (using the DESCAR-T registry), otherwise data collection was mainly planned through private electronic platforms operated by CROs (80% of the PUT-RDs).ConclusionsTo optimize the generation of relevant clinical data to be used in the regular HTA process, EAs should be requested early, prior to marketing authorization. Additionally, the use of existing data sources, in particular disease registries, is to be developed to avoid duplicate data collection and alleviate the burden for healthcare professionals.
OP37 The Spanish Health Technology Assessment Network Methodological Approach For The Utilization Of Real-World Data In The Preadoption Phase
Authors: García Guillermo Pérez , Fernandez Celia Muñoz , Remón Lucía Prieto , Orriols Carlos Tellería , Alemán Hugo Hernández , De Val Soledad Isern , Armesto Sandra García 
Published: January 2025

IntroductionPreadoption assessments are the most frequent type of evaluation conducted by the Spanish Network of Agencies for Health Technology Assessment and Services of the National Health System (RedETS). RedETS aimed to develop a framework to utilize real-world data (RWD) to better adjust its assessments to the Spanish population’s context and provide live assessments throughout the lifecycle of health technologies.MethodsA working group within RedETS was set up, which held several meetings to define the primary uses of RWD in the short term. Next, a manual review was conducted on national and international initiatives that provided guidance on the use of RWD in HTA. Common pathways for utilizing RWD in HTA were identified. The working group agreed to outline and explain the key overarching steps and provide general guidelines for working with RWD, developing as illustration a use case for an interventional technology. The Big Data project of Aragon (BIGAN) was chosen as the data source for the use case.ResultsWe formulated a case for leveraging RWD in the assessment of implantable cardiac defibrillators (ICDs) for the prevention of sudden cardiac death (SCD). Based on this scenario, we developed a methodological framework outlining a workflow consistent with RedETS practices. RWD complemented the usual process of systematic review of a technology. Crucial steps comprised the definition of data requirements through a data model specification, an exploratory data analysis, and the construction of a decision model. We presented solutions for dealing with unavailable data on essential variables and unstructured records. We discussed the main limitations to account for when working with RWD.ConclusionsThe task ahead holds great hope but requires overcoming some challenges to fully deploy RWD-driven methods. This entails fostering collaboration with health authorities and designated data holders to address data access challenges. In the short term, it is essential to include data scientists in assessment teams and provide appropriate capacity-building to encompass RWD tools and modeling techniques.
OP47 New Health Technology Assessment Framework For Digital Health Technologies
Authors: Segur-Ferrer Joan , Moltó-Puigmartí Carolina , Peiró Roland Pastells , Vivanco-Hidalgo Rosa Maria 
Published: January 2025

Introduction
Experts emphasize the necessity of reconsidering the approaches used in the health technology assessment (HTA) of digital health technologies (DHTs) to adequately address their particular characteristics. In this context, the Agency for Health Quality and Assessment of Catalonia was commissioned to develop a specific framework for the assessment of DHTs in collaboration with the agencies that constitute the Spanish Network of Agencies for Assessing National Health System Technologies and Performance (RedETS).MethodsTo accomplish the assignment, a combination of methodologies was used. To establish the domains and dimensions, an international survey, a scoping review of the literature, and a thematic analysis were executed. To determine the evidence standards that DHTs must achieve according to the risk classification derived from their use, the updated version of the evidence standards framework (ESF) from the National Institute for Health and Care Excellence (NICE) was adapted to the Spanish context. National experts in the fields of HTA and DHTs collaborated on different tasks to develop the framework.ResultsThe framework encompasses three distinct sections. The first section provides an introduction, detailing a DHT classification system that is based on the system proposed by NICE in their ESF. Following this, Section A elaborates on a comprehensive set of 13 domains, 41 dimensions, and nine sub-dimensions, offering a detailed description of each. Section B consists of the adaptation of the 21 evidence standards from the NICE ESF to the Spanish context. Of note, a clear link was established between the classification system, the domains, dimensions, sub-dimensions, and the evidence standards.ConclusionsThe resulting framework was published in November 2023. Since its publication, it has become the reference framework for the HTA of DHTs within the Spanish National Health System, primarily by the HTA agencies that constitute RedETS. The document will be periodically reviewed and, if necessary, adapted to align with emerging technologies and changes in legislation.
OP48 Establishing The Implant Subsidy List In Singapore: A Collaborative Approach Between Health Technology Assessment And National Procurement Agencies
Authors: Ee Wong Yen , Ding Wayne , Ho Yan Teck , Soon Swee Sung , Ng Kwong 
Published: January 2025

IntroductionPublic healthcare institutions (PHIs) experience rising and discordant implant costs. Implementing subsidies for implants without concurrent price harmonization through national procurement contracts impedes equitable patient access. This abstract describes the collaboration between the Agency for Care Effectiveness (ACE), Singapore’s national health technology assessment (HTA) agency, and the public healthcare supply chain agency (ALPS) to establish the Implant Subsidy List (ISL).MethodsThe alignment between ISL and concurrent national procurement contracts for implants required close coordination and co-development of streamlined and scalable processes. To achieve this, ACE and ALPS collaborated to understand the complex implant landscape by leveraging on known vendors and products and pre-empting upcoming ones, identifying key opinion leaders, and sharing technical expertise. Both agencies also developed common-use templates and streamlined vendor submission processes. Of note, joint request-for-proposal (RFP) exercises for national subsidy and procurement listings combined negotiation efforts to achieve lower implant prices. Regular cross-agency discussions enabled continuous knowledge sharing and process refinement in an evolving implants landscape.ResultsFor phase one rollout of ISL, 15 implant topics across eight clinical functions were evaluated, supported by concurrent establishment of 15 national procurement contracts. As of December 2023, ACE listed 22,689 ISL implants for implementation, with more topics onboarded on a rolling basis. ACE and ALPS streamlined process flows, standardized communications channels and information, and utilized contractual tools. Vendors could submit for both subsidy and procurement through the secure ALPS portal, reducing duplication of work and errors while allowing procurement and subsidy consideration processes to proceed concurrently. Combined negotiation efforts have resulted in an average price reduction of 19 percent.ConclusionsThe alignment between national HTA and procurement agencies is important for successful scaling. The ACE–ALPS collaboration has synergized both agencies’ understanding of the complex implant landscape and established streamlined workplans and processes to support initial establishment of ISL and its maintenance. Challenges include navigating strict procurement rules and continuous cross-agency improvement efforts to ensure applicability in an evolving landscape.
OP51 Strategies For Training Laypeople To Participate In Health Technology Assessment: A Scoping Review
Authors: Dias Morais Quenia Cristina , Itaborahy Alex , Frossard Leny , Mateus Iandy , Leite Bianca , Santos Marisa 
Published: January 2025

IntroductionThis study aimed to map strategies for educating laypeople about health technology assessment (HTA). Although integrating community is challenging, the engagement of patients/public in the processes of HTA has garnered support and endorsement from international network agencies. Dissemination of information, educational empowerment, and training are vital to give individuals capacity to partake in the intricate web of processes actively.MethodsThis review considered studies addressing educative strategies to train laypeople on HTA, additionally mapping and summarizing relevant methodological papers from any international HTA agency. Four databases were searched for qualitative, quantitative, and mixed methods study designs. The grey literature search included policy and practice documents from HTA and health organization websites. Two reviewers independently completed title and abstract screening before the full-text review and data extraction.ResultsThe main contributors to the production of knowledge about educating laypeople in HTA were the United Kingdom (40%), Spain (20%), and Canada (13%). Most studies included were conducted in the context of the United Kingdom (27%), followed by Spain (20%), and international networks context (20%). The main strategies included conference-like events (21%), the production of educational materials (18%), training (11%), and the use of plain language (8%). Furthermore, international HTA and health agencies have offered courses, and online training produced and made available online guidance materials for increasing laypeople’s participation in the HTA process.ConclusionsDespite the global efforts to educate laypeople on HTA, jurisdictional variations underscore the need for a more inclusive approach. Strategies like events, educational material production, training, and clear-language use offer diverse avenues for public engagement. International agencies’ commitment to courses, online training, and guidance reflects a collective effort to enhance public involvement.
OP53 An Actionable And Legible Toolbox For The Appraisal Of Healthcare Innovations Developed Through Nationwide Stakeholder Collaboration
Authors: Plamondon Geneviève , Ganache Isabelle , Martin Mélanie , Lehoux Pascale 
Published: January 2025

IntroductionIn Québec, Canada, decisions about implementing innovations are taken both centrally for province-wide access and locally by healthcare institutions. There is no systematic evaluation process and various stakeholders are involved, notably within a new nationwide governance structure. There was a wish to increase consistency and clarity with the principles and methods used by various bodies across the innovation lifecycle.MethodsThe starting point was the Institut national d’excellence en santé et services sociaux (INESSS) multidimensional framework, which focuses on the population-level, clinical, economic, organizational, and sociocultural value of drugs, technologies, and interventions. The framework, already under evolution drawing on Responsible Innovation in Health (RIH), evolved through collaborative work between INESSS’ methodological and scientific teams, but also and foremost with diverse groups and institutions within the provincial innovation ecosystem (e.g., university-based incubators, regional hospitals). The first steps were to capture current concepts and practices from different stakeholders, as well as their operational needs in terms of assessment tools.ResultsThis multistakeholder taskforce resulted in the development of an operational toolbox meant to guide the value appraisal of innovations through a lifecycle approach. First aimed at stakeholders involved locally in healthcare institutions, the work conducted was equally beneficial to INESSS by enabling its evaluation teams to contribute to the operational tools needed to enhance clarity and legibility of the agency’s processes and methods. The level of collaboration with stakeholders across the province was also unique and has strengthened the understandability and actionability of the toolbox developed. Some challenges were faced, and related actions will be discussed.ConclusionsBoth the taskforce process and its output contributed to improving consistency in the assessment of innovations across the province. They made more explicit what may sometimes be perceived as the HTA “black box.” The INESSS value appraisal framework also evolved considering key elements of responsibility from RIH and through this collaboration with stakeholders, and its applicability in different contexts was reinforced.
OP55 Transferability Of Economic Models Within Health Technology Assessment In Central And Eastern Europe: Bridging The Gap
Authors: Hołownia-Voloskova Malwina , Obrzut Grzegorz , Walczak Jacek 
Published: January 2025

IntroductionHealth technology assessment (HTA) plays a pivotal role in healthcare decision-making, evaluating the cost-effectiveness of emerging technologies. Central and Eastern Europe (CEE) presents a unique context for HTA, marked by diverse healthcare systems, economic variations, and regulatory frameworks. This study addresses the critical issue of transferability of economic models within HTA in CEE, aiming to bridge existing gaps and enhance the region’s capacity for informed decision-making.MethodsA comprehensive and systematic approach was employed to assess the transferability of economic models in the context of HTA across CEE. We conducted an extensive literature review, analyzed HTA reports, and engaged in expert consultations to understand the nuances of the healthcare systems in the region. Key factors influencing the transferability of economic models, such as healthcare infrastructure, economic disparities, and regulatory landscapes, were systematically evaluated. The study focused on a range of economic models commonly used in HTA, including cost-effectiveness analysis, budget impact analysis, and multiple-criteria decision analysis.ResultsOur findings highlight the intricate dynamics influencing the transferability of economic models within HTA in CEE. While certain economic models exhibit a degree of generalizability across the region, there are notable variations based on specific contextual factors. Economic models designed for Western healthcare systems may not seamlessly translate to the CEE context due to differences in healthcare delivery, patient populations, and policy frameworks. The study identifies critical determinants of transferability, including the level of healthcare infrastructure development, economic disparities among CEE countries, and the diversity in regulatory approaches.ConclusionsIn conclusion, this study emphasizes the need for a nuanced and context-specific approach to the transferability of economic models within HTA in CEE. Bridging the gap in transferability enhances the region’s capacity for evidence-based healthcare resource allocation and contributes to the overall efficiency and sustainability of healthcare systems in CEE.
OP56 Hospital-Based Health Technology Assessment: Barriers And Facilitators In France, Hungary, Italy, Kazakhstan, Poland, Switzerland, And Ukraine
Authors: Di Bidino Rossella , Lipska Iga , Kukla Monika , Nemeth Bertalan , Hren Rok 
Published: January 2025

IntroductionThe adoption and development of health technology assessment (HTA) in the hospital setting (HB-HTA) differs among countries, but in all cases, it encounters barriers and facilitators. An analysis was conducted to promote international cooperation to develop strategies both to enforce common facilitators and overcome common barriers. HTA specialists from seven countries (at least two per country) contribute to the study.MethodsHTA experts from countries in Western Europe (three), Central and Eastern Europe (three), and Central Asia (one) voluntarily participated in the project. They provided a description of the scenario that HB-HTA faces in their country, then a two-round Delphi study was conducted. The survey was based on twelve statements that were categorized into external and internal barriers and external and internal facilitators. Next, panel experts ranked statements on a seven-point Likert scale with a median agreement score greater than or equal to six and an interquartile range (IQR) greater than or equal to one accepted as reaching consensus. The goal was to identify similarities and differences in the HB-HTA scenarios among countries.ResultsFifteen experts from France, Hungary, Italy, Kazakhstan, Poland, Switzerland, and Ukraine contributed to the analysis. Among the twelve statements, six were ranked as reaching consensus (two barriers, four facilitators). One external and one internal barrier, which reached consensus, were (i) lack of formal recognition of the role of HB-HTA in national/regional legislations and (ii) limited human resources. Two external facilitators were (iii) creation of a network among hospitals performing HB-HTA and (iv) dissemination of HB-HTA methods and activities, while two internal facilitators were (v) top hospital management support in evidence-based decision-making and (vi) training initiatives dedicated to HB-HTA.ConclusionsThe analysis showed a consensus on the barriers and facilitators for HB-HTA. This creates the opportunity for internationally developed strategies to enforce facilitators and support the adoption and sustainability of HB-HTA. Future research should extend the analysis to other countries and include the results of the HB-HTA survey conducted in 2023 by the HB-HTA Interest Group.
OP61 Target Trial Emulation To Determine The Population-Level Cost-Effectiveness Of Multigene Panel Sequencing For Advanced Melanoma
Authors: Krebs Emanuel , Weymann Deirdre , Pollard Samantha , Regier Dean 
Published: January 2025

IntroductionCompared to single-gene BRAF testing to guide targeted treatment with BRAF/MEK inhibitors for advanced melanoma, multigene panels can identify additional gene mutations with known therapeutic or prognostic relevance. Implementation of multigene panels remains uneven across healthcare systems given an uncertain clinical and economic evidence base. We determined the population-level cost-effectiveness of multigene panels compared to single-gene BRAF testing for advanced melanoma.Methods
Our population-based retrospective study emulated a hypothetical pragmatic trial comparing multigene panel sequencing to single-gene BRAF testing. We drew on comprehensive patient-level clinical and health administrative data between September 2016 and December 2018 in British Columbia, Canada. To emulate random treatment assignment, we 1:1 matched multigene panel patients to contemporaneous single-gene tested controls using genetic algorithm-based matching. We estimated three-year overall survival and healthcare costs (2021 CAD), and incremental net monetary benefit (INMB) for life years gained (LYG) using inverse probability of censoring weighted linear regression and nonparametric bootstrapping. We also estimated overall survival using Weibull regression and Kaplan–Meier survival analysis.ResultsWe matched 147 patients with advanced melanoma receiving multigene panel sequencing to contemporaneous single-gene-tested controls, achieving good balance for all 15 baseline clinical and sociodemographic covariates. After matching, mean incremental costs were CAD19,447 (USD14,217) (95% confidence interval [CI]: −CAD18,517 [−USD13,537], CAD76,006 [USD55,565]; p=0.41) and mean incremental LYG were 0.22 (95% CI: −0.05, 0.49; p=0.12). We found uncertain differences on overall survival using Kaplan–Meier (stratified Log-rank test p=0.11) and Weibull regression (HR: 0.73 [95% CI: 0.51, 1.03]; p=0.07) survival analysis. Cost differences were driven by systemic therapy (∆C: CAD8,665 [USD6,334]; 95% CI: −CAD36,387 [−USD26,600], CAD53,716 [USD39,268]; p=0.71). The INMB at CAD100,000(USD73,104)/LYG was CAD2,646 (USD1,934) (95% CI: −CAD30,044 [−USD21,963], CAD43,416 [USD31,739]; p=0.89), with a 52.8 percent probability of being cost effective.Conclusions
There were clinically relevant but uncertain differences in improved survival associated with multigene panel sequencing for advanced melanoma, and the cost-effectiveness of panel-based testing was finely balanced. This real-world evidence generated using randomized trial design principles can support jurisdictions’ deliberations on the reimbursement of precision oncology interventions.