bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP52 How Will European Joint Clinical Assessment Impact National Decision-Making?
Authors: Müller Elvira , Neeser Kurt 
Published: January 2025

IntroductionIn 2025, oncology drugs with new active substances and advanced therapy medicinal products will undergo joint clinical assessment (JCA). The comparative analysis of the clinical evidence as defined in the Regulation (EU) 2021/2282 on health technology assessment (HTAR) will save national/regional submissions of the same evidence. JCA will be available early supporting appraisal and decision-making, which remains within the responsibility of member states (MS).MethodsTargeted searches on JCA and statements from stakeholders were performed and analyzed. We conducted interviews with current and former national payers, as well as members of HTA agencies, across Germany, France, Italy, and Eastern Europe to explore their perspectives on the anticipated implications of JCA on decision-making processes and reimbursement strategies in Europe. Focus was on reduced/additional effort for authorities and health technology developers (HTDs), required national amendments, and potential discrepancies between JCA outcome and MS benefit evaluations.Results
Stakeholders appreciate the standardized methodology and guidance on HTA, which, especially in countries without an established HTA system, could enhance patients’ access to new treatments by considering JCA in decision-making. The comprehensive evidence compilation may also save resources in pursuing national/regional submissions. On the other hand, country-based appraisals within the MS could lead to diverse conclusions, and there is uncertainty as to which extent national authorities will adopt JCA and how its integration into decision-making will be handled. Some stakeholders challenge an impact on local patients’ access as reimbursement and pricing processes remain within MS responsibility.ConclusionsJCA is a long-desired achievement and will set the groundwork for timely access of new treatments in the MS. However, presently there are several uncertainties on how JCA will impact decision-making and whether MS appraisal could lead to contradictory value conclusions for a given treatment. Future adjustments to national/regional procedures and refinement of the JCA framework are expected.
OP62 Economic Evaluations Submitted For Reimbursement To The Brazilian Unified Health System: A Meta-Epidemiological Study
Authors: Falavigna Maicon , Dorneles Gilson , Araujo Cintia , Parahiba Suena , Stein Cinara 
Published: January 2025

IntroductionAllocating financial resources efficiently poses a significant challenge for health systems worldwide. In this context, economic evaluations (EE) are pivotal for the health technology assessment (HTA) process, particularly in standardizing approaches to enhance decision-making accuracy. This study focuses on delineating methodological nuances of EE submissions within Brazil’s Unified Health System (SUS).MethodsThis meta-epidemiological study analyzed dossiers submitted to the National Committee for Health Technology Incorporation (Conitec) for drug reimbursement from January 2022 to May 2023. We selected dossiers that included complete EE studies such as cost–utility or cost-effectiveness analyses. Our evaluation involved extracting data on the characteristics of the studies, reimbursement decisions, elements of the base case scenario, and parameters used in utility and sensitivity analyses. [Protocol: DOI 10.17605/OSF.IO/JVYEC]ResultsWe included 56 dossiers, leading to 11 favorable reimbursement decisions. EE study methods were cost-effectiveness (17) and cost–utility analyses (17), with some employing both (16). Markov chain models were used in 27 dossiers, primarily utilizing quality-adjusted life years as the health outcome measure. Additional outcomes included life years gained and frequency of avoided events. While utility assessment was reported in 33 dossiers, only six adjusted for age and seven accounted for disutility from adverse events. Thirty-two conducted deterministic or probabilistic sensitivity analyses, but only four presented parameters like credibility intervals, and 17 presented acceptability curves.ConclusionsThis study highlights the need for more standardized and refined methods in the EE submitted to Conitec. The existing EE methodological guidelines, dating from 2014, are currently under revision. This update is crucial to integrate recent advancements in health technology assessment and to better address contemporary requirements, particularly in light of the newly defined willingness-to-pay threshold.
OP64 Risk-Based Prioritization In Patients Referred For Transcatheter Aortic Valve Implantation: A Simulation Study
Authors: Miranda Rafael , Austin Peter , Naimark David , Wijeysundera Harindra 
Published: January 2025

IntroductionDemand for transcatheter aortic valve implantation (TAVI) has increased in the last decade and has outpaced system capacity, impacting wait times and bringing undesirable health outcomes such as waitlist mortality and number of urgent procedures. Risk-based prioritization can improve equitable access to patients. In this study, we assess the impacts of different classifications and wait times for each risk group on health outcomes.MethodsWe developed decision-analytic models that simulate the patient trajectory from referral to completion of TAVI. Using prediction models that can classify patients based on their risk of adverse events on the waitlist, we assessed the impacts of (i) the number of risk groups, (ii) size of the risk groups, and (iii) recommended wait times for each risk group, on waitlist mortality, hospitalization, and the proportion of urgent TAVIs. All scenarios were modeled under the same resource constraints, allowing us to explore the trade-offs between faster access to prioritized patients and deferred access to nonprioritized groups.ResultsIncreasing the number of risk groups from two to three, increasing the sizes of the higher-risk groups from five percent to 30 percent of the cohort each, and providing faster access to the higher-risk groups (five to three weeks for high-risk and 11 to five weeks for medium-risk) achieved the greatest reductions in mortality, hospitalizations, and urgent TAVIs (relative reductions of up to 29%, 23%, and 38%, respectively). However, this occurs at the expense of excessive wait times in the nonprioritized group (up to 25 weeks). The reduction in adverse events was lower when the nonprioritized group had more reasonable wait times.ConclusionsWhen developing and implementing waitlist prioritization strategies, it is important to consider the resource constraints of the system and the patient profile, as the benefits of providing faster access to prioritized patients can lead to unreasonable wait times for nonprioritized ones. In settings with long wait times, prioritization initiatives must be followed by expansion of supply to achieve optimal improvements in health outcomes.
OP70 Implementation Of An Online Consultation Hub To Facilitate Consumer Engagement In Health Technology Assessment Processes
Authors: Trowman Rebecca , Watson Jo , Beach-Mills Bella 
Published: January 2025

Introduction
In 2021, the Australian Government Department of Health and Aged Care’s Consumer Evidence and Engagement Unit (CEEU) launched an online consultation hub to provide a centralized pathway for consumer engagement in health technology assessment (HTA) processes. The hub enables consumers (patients, carers, health professionals, and citizens) to provide commentary on items being considered by HTA committees for subsidization.MethodsA survey was developed by the CEEU, committee members, and consumer representatives to facilitate consultation on applications assessed by the Pharmaceutical Benefits Advisory Committee (PBAC)—a principal Australian HTA committee. Questions were designed as simple and engaging, including guidance on the type of information required. Responses are summarized thematically by efficacy, safety, accessibility, and quality-of-life impacts of the proposed health technology. New surveys are launched to coincide with each PBAC meeting agenda publication and allow a ten-week consultation period. Awareness of consultations is supported by the CEEU’s HTA Engage e-newsletter, which alerts the public and targeted stakeholders.ResultsThe hub surveys have enabled streamlined consumer commentary to be provided for PBAC considerations. It has also allowed increased time for quality consultation with stakeholders. The success of the hub is further demonstrated in the current development of a similar survey for another principal committee, the Medical Services Advisory Committee (MSAC), to transition its consultation processes to the hub. Of note, while consumers’ feedback on the hub is positive, there remains a desire for educational resources and face-to-face interactions to support consumer engagement in HTA processes. The CEEU are developing materials to address and further support this need.ConclusionsIn a time when technological communication can be optimized to complement face-to-face conversations, it is vital consumer engagement in HTA processes follows suit. To facilitate continued engagement that is sustainable for both present and future Australians, the CEEU continues to evolve a strategy regarding virtual consultations to increase consumer awareness and education and promote effective participation in Australian HTA.
OP71 Patient Disease Strategy: A New Operational Framework For Collecting And Applying Patient Experience Data Into Clinical Development Programs
Authors: Coulouvrat Catherine , DiBiaso Victoria , Bascle Stephanie , Lucats Laurence , Largeron Nathalie , Tomme Sophie Van , Arnould Benoit 
Published: January 2025

IntroductionUnderstanding patient experience and needs is crucial to develop high-value therapies. Patient experience data (PED) inform trial design and evidence generation plans. The U.S. Food and Drug Administration’s roadmap to patient-focused outcome measurement advocates integrating PED into product development. We adapted this theoretical framework to include the health technology assessment (HTA) perspective and operationalized it as a patient disease strategy (PDS) framework.MethodsThe PDS framework is a methodology that systematically integrates patient-informed activities to reflect the patient health value of a new treatment. A PDS is developed per indication, initiated in the preclinical phase, applied in clinical development, and continuously adapted throughout the product development lifecycle. The three PDS phases include: (i) development of patient profile, including epidemiology, demographics, patient journey, disease, and treatment burden for patients and caregivers; (ii) PED gap analysis, focusing on identification of patient priorities, unmet needs, preferences, and expectations for new therapies; and (iii) translation into actions, such as diversity and inclusion (D&I) plans and outcomes strategy.ResultsOut of 58 indications, 31 percent have endorsed PDS and 67 percent are in progress. Patient-relevant label opportunities increased by over 50 percent. Each indication was informed on average by patients from three different countries. The PDS framework helped to identify factors that impacted health outcomes for integration into trial designs and D&I plans. Early understanding of heterogeneous patient populations, unmet needs, benefit/risk trade-offs, and patient experiences ensured development programs measured the most meaningful outcomes while also addressing evidence gaps. Early understanding of patient priorities and barriers to participation optimized the studies by reducing burden and identifying proactive support needed to complete the trial.ConclusionsThe PDS framework systematically identified health value opportunities for a target population and integrated the patient needs into the overall development plan. PED informs clinical trial design and endpoint strategy optimization, including factors that influence diversity and data integrity. We anticipate that the PDS will enable HTA decisions to reflect patients’ health value and ultimately improve access to innovative therapies.
OP79 Innovations In Measuring And Valuing Outcomes: Generating Preferences For The EQ Health And Wellbeing Short
Authors: Mukuria Clara 
Published: January 2025

IntroductionThe value of health technologies may not be fully reflected in existing generic preference-based measures (GPBMs). The nine-dimension EQ Health and Wellbeing Short (EQ-HWB-S) is a new GPBM developed to assess health, informal carer, and social-care-related quality of life. The objective was to compare standard and new approaches used to generate preferences for the EQ-HWB-S.Methods
Three feasibility studies that included qualitative work have valued the EQ-HWB-S with members of the public in the United Kingdom: (i) videoconferencing interviews using time trade-off (TTO) and discrete choice experiments (DCEs) from the EuroQol Valuation Technology (EQ-VT) protocol (n=600); (ii) online DCEs using the Potentially All Pairwise RanKings of all possible Alternatives (PAPRIKA) method (n=300), an adaptive DCE with a binary search to locate “dead”; and (iii) Online elicitation of Personal Utility Functions (OPUF) (n=300), which is a compositional method with dimension weighting, response level rating and anchoring on dead. Participant/interviewer feedback, data quality, and the weights were compared.ResultsSelf/interviewer reported understanding was high (>70%) across all studies. Qualitative findings indicated misunderstanding for some OPUF steps (e.g., the anchoring on dead). Inconsistencies or illogical answers were small in the EQ-VT study (7%) and OPUF (16%). The PAPRIKA study had a priori exclusions criteria (e.g., time taken) that resulted in 44 percent exclusions. Pain, activity, mobility, and sadness/depression were the most important in all the studies. The value of the worst state was −0.384, −0.51, and −0.15 for EQ-PVT, PAPRIKA, and OPUF, respectively, and there were differences in dimension weights (e.g., PAPRIKA gave less weight to mobility but more to cognition).ConclusionsEQ-HWB-S is a GPBM that provides an innovative approach to measuring and valuing outcomes. Standard and new approaches to eliciting preferences are feasible, but there are differences in the resultant weights. PAPRIKA and OPUF may improve attribute attendance and be more cost effective as they are administered online, but there is scope for improvement to ensure understanding and engagement.
OP81 Experience Matters: A Discrete Choice Experiment Exploring Patient Preferences For Heart Valve Procedures
Authors: Fifer Simon , Keen Brittany , Guilbert‐Wright Polo , Murdoch Dale 
Published: January 2025

IntroductionTreatment for heart valve diseases (HVD) typically involves surgery, but less invasive procedures are becoming more common. Although the two procedures have similar outcomes, the risk–benefit profiles differ, indicating patients should be included in treatment decisions so they align with their values/preferences. This study aimed to determine patients’ preferences for HVD procedures, and the relative importance of treatment attributes.MethodsAn online survey with discrete choice experiment (DCE) was disseminated to patients with aortic stenosis, mitral valve regurgitation, and tricuspid valve regurgitation. Participants were presented with several choice sets, each comprising two hypothetical treatment procedures (labeled “invasive procedure” and “minimally invasive procedure”) as well as an opt-out. DCE attributes were selected based on a literature review, qualitative interviews with patients and specialist doctors, and steering committee consultation (patients, patient organization representatives, and cardiac physicians). Responses were collected via healthcare recruiters, online panels, and patient organizations. DCE data from 143 Australian patients was analyzed using a mixed multinomial logit (MMNL) model.ResultsResults indicate an “experience effect” whereby patients preferred the same type of treatment they had undergone previously. For example, patients who had undergone a transcatheter procedure were more likely to choose the minimally invasive procedure in the experiment and vice versa for those who had undergone invasive procedures like open-heart surgery. Patients were willing to switch procedures based on its risk–benefit profile, and most patients preferred the minimally invasive procedure when it reflected the profile of transcatheter aortic valve replacement (TAVI), even if they had previous invasive procedures experience. Key attributes driving choice were valve durability and regaining independence.ConclusionsThere is a great deal of heterogeneity in HVD patient preferences, even when treatment outcomes appear similar. Patients preferred a minimally invasive procedure over an invasive procedure, irrespective of prior treatment experience with valve durability and independence driving choice. These results can inform healthcare decision-makers about what features of HVD procedures patients value most, taking into consideration patients’ prior experiences.
OD02 Developing Components For A National Strategy For Heart Valve Disease In Canada
Authors: Sproule John M , Warkentin Lindsey , Messika-Zeitoun David 
Published: January 2025

IntroductionThe research included a rapid review of current literature to describe epidemiology, management, and system impact of heart valve disease (HVD) in adult populations. Key issues were identified in consultation with expert focus groups and were framed across the continuum of care and systemic policy issues. The groupings were adapted and adjusted during deliberations.MethodsA rapid literature review was conducted on HVD key interventions and evidence of effectiveness along with expert interviews to identify high-level themes for reform. This served as the evidentiary backgrounder. Two virtual policy engagements with clinical leaders, patients, and health system managers were conducted. The focus was their collective drafting of recommendations. These workshops identified nine thematic areas and developed associated recommendations for action under each theme. The success of the process is evident as the report has been taken up as a roadmap for ongoing research and policy work.ResultsA comprehensive grouping of recommendations for improving HVD detection, management, and treatment in Canada was produced. It was designed to be comprehensive to then allow more targeted work to proceed under an evidence-informed and clinically endorsed agenda.ConclusionsHeart valve conditions are increasingly treatable, especially if detected early. Innovation in treatments as well as detection and management to address gaps in care were identified as the most urgent priorities. The key result was formation of formal working groups with a professional society to explore spoke–hub-and node models for care delivery and to launch awareness programs for early detection.
OD08 Health Technology Assessment Timelines And Outcomes Of Common Compounds In France, Germany, Sweden, And Poland From 2014 To 2022
Authors: Sola-Barrado Belen , Wang Tina , McAuslane Neil 
Published: January 2025

IntroductionThe Regulation (EU) 2021/2282 on health technology assessment (HTAR) takes effect in January 2025 and aims to improve and accelerate patients’ access to new health technologies. We examine HTA timelines and outcomes for new active substances (NASs) commonly appraised in all of France, Germany, Poland, and Sweden from 2014 to 2022, providing insights into the European landscape before HTAR.MethodsPublic data was extracted from the HTA reports from the corresponding health authorities from France (HAS), Germany (IQWiG), Poland (AOTMiT), and Sweden (TLV) published between 1 January 2014 to 31 December 2022. NASs appraised by HTA in the four jurisdictions were referred to as common compounds. Time to recommendation was calculated as European Medicines Agency approval to HTA recommendation in the corresponding country. Differences in time parameters were assessed using the Kruskal–Wallis test. HTA recommendations were categorized as positive, positive with restrictions, and negative.ResultsA total of 97 common compounds (75% chemical entities and 25% biotechnology products) were identified (388 HTA appraisals). Notably, 47/97 (48%) NASs were associated with anticancer drugs (ATC-code=L). The median (interquartile range) time to recommendation differed by jurisdiction: 128 (113, 169) days in Germany; 203 (151, 308) days in Sweden; France, 209 (162, 294) days; Poland, 479 (330, 738) days (p-value <2.2e-16). In addition, 62/97 (63.9%) products received their initial HTA recommendation in Germany, followed by 25/97 (25.8%) products in Sweden, and 10/97 (10.3%) in France. Only 7/97 (7%) of products exhibited unanimous HTA outcomes across jurisdictions.ConclusionsDiscrepancies in the time to HTA recommendation were identified across four prominent European jurisdictions, and consensus in HTA outcomes was notably limited across countries, underscoring the intricate landscape preceding the enforcement of the HTAR. The future implementation of HTAR holds the potential to address these disparities, fostering greater harmonization in HTA processes and outcomes across the European healthcare landscape.
OD11 Application Of Natural Language Processing To Predict Final Recommendation Of Brazilian Health Technology Assessment Reports
Authors: de Almeida Cardoso Marilia Mastrocolla , Machado-Rugolo Juliana , Thabane Lehana , da Rocha Naila Camila , Barbosa Abner , Almeida Juliana Tereza , da Silva Pereira Curado Daniel , Komoda Denis Satoshi , Theresa Weber Silke Anna , Modelli de Andrade Luis Gustavo 
Published: January 2025

Introduction
Several countries established health technology assessment (HTA) processes to support decision-making. Considering the high volume of submissions processed by HTA agencies, approaches to determine factors associated with the approval would be beneficial. This study aimed to predict the final recommendation of the National Committee for Health Technology Incorporation (Conitec) using a natural language processing (NLP) algorithm for text extraction.MethodsConitec’s 2012 to 2022 reports (n=389) were split into 75 percent training and 25 percent testing data. Tokenization enabled NLP models: Least Absolute Shrinkage and Selection Operator (LASSO), logistic regression, support vector machine (SVM), random forest, neural network, and Extreme Gradient Boosting (XGBOOST). Evaluation criteria included accuracy, area under the receiver operating characteristic curve (ROC AUC) score, precision, and recall. Cluster analysis with k-modes identified two clusters (group 0 = approved, group 1 = rejected).ResultsThe neural network model demonstrated the best accuracy metrics with a precision of 0.815, accuracy of 0.769, ROC AUC of 0.871, and a recall of 0.746. Some tokenization identified that linguistic markers could contribute to the prediction of incorporation decision by the Brazilian HTA Committee, such as international HTA agencies’ experience and the government as the main requester. Cluster and XGBOOST analysis identified similar results with approved technologies with a predominance of drugs assessment, mainly requested by the government, and not approved mostly assessing drugs, the industry as the main requester.ConclusionsThe NLP model could identify predictors for the final decision process on the incorporation of health technologies in Brazil’s Unified Health System, opening paths for future work using HTA reports coming from other agencies. This model could potentially improve the throughput of HTA systems by supporting experts with prediction/factors/criteria for approval or nonapproval as an earlier step.