bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP16 Vial Sharing And Wastage Are Familiar Concepts, But What About Pack Sharing And Wastage?
Authors: Wilkes Sarah Kate , Turner Emily Eaton , Jiandani Neha , Thomson David 
Published: January 2025

IntroductionThe National Institute for Health and Care Excellence (NICE) conducts health technology assessment to assess cost-effectiveness and budget impact. For treatments provided in vials, NICE often considers how treatments are dispensed and adjusts the economic modeling costs accordingly. Vial sharing and wastage are likely familiar concepts to stakeholders, but the same consideration is not consistently given to tablet packs.MethodsUsing anonymized examples, NICE assessed potential implications for cost-effectiveness and budget impact of different methods for modeling oral treatments. Firstly, the cost-effectiveness and budget impact were calculated based on a cost per milligram (mg) of treatment. The per mg cost was multiplied by the number of mg for each dose and did not account for the number of tablets or packs required. Using the same example, the cost per tablet was calculated by rounding each dose to the nearest whole tablet mg dose. Finally, the example was costed based on whole packs, which included the cost for any wasted tablets.ResultsThe anonymized examples showed that costing per mg versus per tablet versus per pack can have a significant impact on cost-effectiveness and budget impact. One example showed that treatment costs per 28 days could increase by over GBP1,000 (USD1,271) when costing per whole pack compared to per mg. This led to a difference in the incremental cost-effectiveness ratio (ICER) of nearly GBP10,000 (USD12,716). Another example demonstrated a potential increase in budget impact of nearly GBP1 million (USD1.27 million) per year. This magnitude of impact on cost-effectiveness and budget has the potential to change health technology assessment decisions and affordability in the United Kingdom.ConclusionsNICE is assessing an increasing number of oral treatments provided as tablet packs, not vials. This highlights the need to consider how pack sharing and wastage should be consistently considered in economic modeling. Developing standardized methods for modeling oral treatments would help ensure consistency of cost calculations and better reflect how treatments are dispensed in clinical practice.
PP23 Machine-Learning-Based Evaluation Of Community Resilience Through Social Media During The First Post-COVID-19 Reopening In China
Authors: Zhang Shouchuang , Zhang Lanyue , Weng Jiayi , Yang Yiying , Chen Zefeng , Jian Weiyan 
Published: January 2025

IntroductionThe Chinese government lifted most COVID-19 pandemic restrictions in December 2022, triggering a spike in confirmed cases and higher demand for medications. Consequently, a significant number of residents resorted to social media to seek assistance. This study aimed to evaluate community resilience by leveraging Weibo user datasets, coupled with interpretable machine learning (ML)-based techniques, to identify important resilience characteristics.MethodsDatasets geotagged from the Sina Weibo social media platform between 8 December 2022 and 7 January 2023 were crawled using search terms of “help-seeking” and the keywords of conventional drugs. This study utilized natural language processing (NLP) to label COVID-19-related posts to identify the type of posts, stakeholders’ behaviors, and other information. We built a comprehensive evaluation model, and five ML-based algorithms were compared for analyzing community resilience. Local interpretable model-agnostic explanations (LIME) was employed to verify five models and the XGBoost algorithm showed optimal effects. Shapley Additive Explanations (SHAP) elucidated the best model’s outputs and estimated contributions for key resilience characteristics.ResultsFor this study, 199,709 posts were collected. Out of these, 48,425 posts were identified as help-seeking posts, with more than two-thirds receiving responses from community level. The area under curve (AUC) of the XGBoost model was 0.82 (95% confidence interval [CI]: 0.82, 0.83), and the values of accuracy and F1 score were 0.72 and 0.80, respectively. This result demonstrated that the model can successfully evaluate community resilience and subsequently identify the features driving this outcome. Collective efficacy in providing aid, support from official rescue guidelines, and residents’ rapid response to rescue information were identified as the most important characteristics for evaluating community resilience.ConclusionsThis study is the first to harness social media data to quantify community resilience in China based on a framework we developed. Five updated ML-based algorithms were developed to evaluate community resilience, and XGBoost showed optimal effects. Three characteristics of community resilience were found as potential predictors that can enhance decision-making support to reshape health emergency rescue activities.
PP28 The Final Arguments To Incorporate Or Not Incorporate Technologies For Ultra-Rare Diseases Into The Brazilian Public Healthcare System
Authors: de Paula de Sordi Monica Aparecida , Machado-Rugolo Juliana , Thabane Lehana , Santos Marisa , Komoda Denis Satoshi , Modelli de Andrade Luis Gustavo , da Silva Pereira Curado Daniel , Theresa Weber Silke Anna , de Almeida Cardoso Marilia Mastrocolla 
Published: January 2025

IntroductionThe decision-making process for health technology assessment (HTA) in ultra-rare diseases is a global challenge. Establishing a comprehensive analytical framework for these unique diseases poses difficulties. This study aims to descriptively analyze arguments reported by the Brazilian National Committee for Health Technology Incorporation (Conitec) in deciding whether to include technology for ultra-rare diseases.MethodsData from recommendation reports (2012 to 2022) were analyzed. Diseases with a prevalence of fewer than one per 50,000 inhabitants were classified as ultra-rare. Extracted variables included preliminary and final recommendation results and justifications by Conitec. Six argument categories were created (method-related issues; evidence; cost; technology effectiveness or safety; context; innovation). Word clouds were generated based on word frequency in each category to present the data.ResultsIn the analysis of 45 reports, the word clouds highlight frequent terms in favorable arguments, emphasizing evidence quality, cost reduction, and applicability in the healthcare system. Conversely, unfavorable arguments also revolve around evidence quality and cost impact. The analysis of the arguments according to categories, 16 arguments were identified: seven concern evidence issues, five cite methodological problems in presented studies, four relate to costs, and three pertain to technology effectiveness or safety. Unfavorable arguments primarily stem from evidence-related concerns. In favorable arguments, cost (seven) and safety (six) are prominent, with innovation (one) and context (three) being additional categories not found in the unfavorable group.ConclusionsWhile technology assessment processes for ultra-rare diseases have evolved, the justifications for recommending or not incorporating new technologies remain unchanged. Over time, reports have become more detailed, focusing on evidence and methodological specifics. This highlights the importance of scrutinizing evidence characteristics and determining relevant criteria and data types for this unique context.
PP32 Health Technology Assessment Regulation In Europe: Implications For A National Authority
Authors: Usher Cara , Fogarty Emer , Barry Michael , Adams Roisin 
Published: January 2025

IntroductionThe Regulation (EU) 2021/2282 on health technology assessment (HTAR) for medicines will come into effect in January 2025; initially, new oncology medicines and advanced therapy medicinal products will be assessed at EU level. How will this work in practice? What does this mean for national HTA bodies, such as the National Centre for Pharmacoeconomics (NCPE) Ireland that uses a cost-effectiveness framework to inform decision-making for medicines?MethodsJoint work to be conducted under the Regulation includes joint clinical assessments (JCA), joint scientific consultations (JSC), and production of procedures and methodological guidance. A review was undertaken of key areas that will be impacted by the HTAR in the Irish HTA process for medicines, including timing, evidence synthesis structure, capacity building, and resource implications.ResultsThe HTAR will alter the current process for medicines assessment in Ireland, from early scientific advice to cost-effectiveness assessment post-authorization. JSCs will represent an additional step. Significant training and capacity implications are associated with the JCA, which will require earlier engagement with stakeholders. The NCPE’s pragmatic HTA early triaging process, the “Rapid Review,” may be delayed due to the non-duplication clause in the HTAR. The availability of high-quality comparative effectiveness evidence may help avoid full HTAs in some cases. The benefit of the JCA will be realized if the results can directly inform treatment-effectiveness estimates in cost-effectiveness modeling.ConclusionsThe HTAR will significantly impact on medicines reimbursement procedures in Ireland. For the HTAR to be effective in achieving its aims, sufficient resources will need to be built into the EU HTA system. The balance between the extra resources needed and the resources spared will depend on the quality of the comparative effectiveness evidence available for the JCA.
PP38 Assessment Outcome By Type Of Available Evidence: Retrospective Analysis Of Oncological Early Benefit Assessment Reports
Authors: Goretzko Jonas , Pusch-Klein Annette , Wieseler Beate 
Published: January 2025

IntroductionFor early benefit assessment in Germany, drugs must be assessed at market entry as to whether they provide an added benefit versus the current standard therapy. This retrospective analysis focuses on assessment reports prepared by the independent Institute for Quality and Efficiency in Health Care (IQWiG). The objective was to describe the relationship between available evidence and assessment outcome.MethodsData were retrospectively extracted from IQWiG’s early benefit assessment reports of oncological drugs that entered the market between June 2021 and December 2021. The underlying evidence for the benefit assessments was divided into three groups: direct comparison (based on a randomized controlled trial [RCT]), indirect comparison, and other sources (e. g., only a single-arm trial was submitted). Afterwards, the outcome of the assessment was described by type of available evidence.ResultsIn the period under review, IQWiG worked on 27 oncology projects addressing 40 different oncological research questions resulting in 46 conclusions. About half of the available evidence in the benefit assessments was a direct comparison with standard therapy based on an RCT (n=22/46). From this level of evidence, an added benefit was derived for the drug under assessment in 64 percent of cases. Indirect comparisons (n=7/46) mostly led to an added benefit not proven (86%). If only other sources were submitted (n=17/46), the added benefit was assessed as not proven in all projects examined.ConclusionsThis retrospective analysis highlights the pivotal role of RCTs in providing strong evidence for health technology assessment. Conversely, single-arm and observational studies fail to yield substantial evidence. In the future, RCTs should be made simpler, faster, and cheaper; for example, by integrating them into existing infrastructures in routine practice (e.g., registry-based RCTs) or by conducting randomized adaptive platform trials.
PP42 Implementing A Sustainable Enhanced Recovery From Surgery Pathway After Hip And Knee Arthroplasty: A Budget Impact Analysis
Authors: Lloyd Melanie , Ackerman Ilana , Harris Ian , Naylor Justine , Lewis Peter , de Steiger Richard , Wan Anthony , Buchbinder Rachelle , Ademi Zanfina 
Published: January 2025

IntroductionIncreasing resources are devoted to osteoarthritis surgical care in Australia annually, with significant expenditure attributed to hip and knee arthroplasties. Safe, efficient, and sustainable models of care are required. This study aimed to determine the impact on healthcare costs of implementing an enhanced short-stay model of care (ESS-MOC) for arthroplasty at a national level.MethodsBudget impact analysis was conducted for hospitals providing arthroplasty surgery over the years 2023 to 2030. Population-based sample projections obtained from clinical registry and administrative datasets of individuals receiving hip or knee arthroplasty for osteoarthritis were applied. The ESS-MOC assigned 30 percent of eligible patients to a shortened acute-ward-stay pathway and outpatient rehabilitation. The remaining 70 percent received a current practice pathway. The primary outcome was total healthcare cost savings post-implementation of the ESS-MOC, with return on investment (ROI) ratio and hospital bed-days utilized also estimated. Costs are presented in Australian dollars (AUD) and United States dollars (USD), at 2023 prices.ResultsEstimated hospital cost savings for the years 2023 to 2030 from implementing the ESS-MOC were AUD641 million (USD427 million) (95% CI: AUD99 million [USD66 million] to AUD1,250 million) [USD834 million]). This corresponds to a ROI ratio of 8.88 (1.3 to 17.9) dollars returned for each dollar invested in implementing the care model. For the period 2023 to 2030, an estimated 337,000 (261,000 to 412,000) acute surgical ward bed-days, and 721,000 (471,000 to 1,028,000) rehabilitation bed-days could be saved. Total implementation costs for the ESS-MOC were estimated at AUD72 million (USD46 million) over eight years.ConclusionsImplementation of an ESS-MOC for eligible arthroplasty patients in Australia would generate significant cost and healthcare resource savings. This budget impact analysis demonstrates a best practice approach to comprehensively assessing value, at a national level, of implementing sustainable models of care in high-burden healthcare contexts. Findings are relevant to other settings where hospital stay following joint arthroplasty remains excessively long.
PP43 Budget Impact Analysis Of Next-Generation Sequencing Coverage In Taiwan’s National Health Insurance
Authors: Chang Hui-Ru , Li Ying Huang Grace , Lin Shou-Yu , Lin Shyr-Yi 
Published: January 2025

IntroductionThe exploration of molecular characteristics has emerged as a prominent trend to advance precision medicine. The utilization of genetic testing to guide therapy is integral to precision medicine. This study aims to investigate the potential patient populations for the reimbursement of next-generation sequencing (NGS) and assess the budget impact from the perspective of Taiwan’s single insurer, the National Health Insurance Administration.MethodsTo comprehend the scope for medicines with companion diagnostics (CDx) involved, we analyze the U.S. Food and Drug Administration-approved/cleared diagnostic tests, conduct a literature review to identify medicines approved by the European Medicines Agency that require a CDx, and identify the medicines with CDx involved covered by the National Health Insurance (NHI) in Taiwan. Subsequently, we explore the potential reimbursement indications for NGS testing and conduct a budget impact analysis to evaluate the expected financial impact for the NHI over a five-year period. Furthermore, sensitivity analyses are conducted to deal with uncertainty.ResultsWe have compiled 13 cancer types for which NGS can serve as a companion diagnostic. These encompass non-small-cell lung cancer, colorectal cancer, breast cancer, ovarian cancer, biliary tract cancer, acute myeloid leukemia, acute lymphoblastic leukemia, melanoma, cholangiocarcinoma, prostate cancer, pancreatic cancer, gastrointestinal stromal tumor, and thyroid cancer/medullary thyroid cancer. The implementation of NGS reimbursement in NHI will benefit 25,000 to 30,000 patients undergoing targeted therapies. The projected incremental budget impact ranges from TWD570 million to TWD650 million (USD19 million to USD22 million) over five years.ConclusionsThis study focuses on evaluating the financial impact of incorporating NGS testing into NHI reimbursement for relevant cancer drug indications. The findings can serve as references for the planning of reimbursement policies. However, with the advancement of precision medicine, it is foreseeable that there will be a broader range of applications for NGS, and its cost will gradually decrease.
PP54 Towards Equitable Pricing: Unraveling The Complexities Of External Reference Pricing In Medical Devices
Authors: Tibet Birol , Kilic Ayse Gokcen , Atashi Zahra Ahangar , Kockaya Guvenc 
Published: January 2025

IntroductionExternal reference pricing (ERP) serves as a widely adopted method for establishing authorization and reimbursement prices for pharmaceuticals in Europe. However, its application within the medical device (MD) industry remains shrouded in uncertainty. This study seeks to investigate the feasibility of integrating ERP into the realm of MDs, identify pertinent challenges and ambiguities, and propose solutions to bolster affordability and accessibility.MethodsThe methodology encompassed a systematic search across diverse databases (Google Scholar, Google, ResearchGate, PubMed), utilizing combinations of relevant keywords, like “medical device,” “external reference pricing,” “price,” “reimbursement,” “affordability,” “challenge,” and “implement.” Articles were thoroughly evaluated by each researcher independently, followed by a consensus-based approach for final assessment. Rigorous scrutiny of relevance, potential insights, study design, and methodology ensured a comprehensive review, maintaining a high standard of research quality. This meticulous process guaranteed the inclusion of pertinent literature on ERP and MD pricing, enhancing the study’s depth and breadth.ResultsGlobal ERP application in the MD sector exhibits significant variability in country selection, pricing methods, and adaptations to market changes. Pricing and reimbursement policies across international contexts contribute to this diversity, involving authorization procedures, regulatory oversight, technology appraisals, and state financing limitations. Challenges in establishing equitable pricing frameworks underscore the need for clear guidelines, regulatory empowerment, regular audits, and international collaborations. Transparent methodologies for reference price calculation are essential. Addressing observed shortcomings involves negotiating lower prices, implementing subsidies, promoting generic competition and local production, and tailoring reimbursement policies to treatment needs, enhancing affordability and accessibility on a global scale.ConclusionsAmbiguities in implementing ERP for MDs underscore the necessity of equitable pricing frameworks. The highlighted multidimensional strategies and consideration of market dynamics are deemed essential for the feasibility of ERP in MDs. These insights emphasize the cruciality of a structured and transparent roadmap for optimizing ERP in the realm of MDs, holding significant potential for ensuring affordability and accessibility.
PP58 Enhancing Health Technology Assessment Understanding Through Targeted Educational Programs
Authors: Chitan Elena , Timotin Alina , Anisei Angela , Volovei Ilie , Lozan Oleg 
Published: January 2025

IntroductionAs health technology assessment (HTA) becomes vital in healthcare decision-making, the demand for specialized education grows. A new HTA course, tailored to the specific needs of the Republic of Moldova’s health system , was launched under a joint project—a collaboration between the World Bank, the Swiss Cooperation Office, Radboud University Medical Center, and the School of Public Health Management. The course targets first- and second-year master’s students, and health professionals enrolled in continuing education.MethodsThe course aimed to introduce the fundamentals of HTA. Participants included 49 master’s students and 26 health professionals. A pre- and post-test model was employed, with participants completing a 10-(multiple) question HTA knowledge assessment at both the start and end of the course.ResultsInitial pre-test results showed an average score of 30 percent, reflecting limited prior HTA knowledge. Following course completion, the post-test average escalated to 80 percent. This 50 percent increase in knowledge was consistent across student and professional groups. While a significant 79 percent of participants accurately answered questions about the use of HTA reports from other jurisdictions, identification of stakeholders, and the elements of establishing PICO (population, intervention, comparator, outcome), they found it more challenging to understand the deeper aspects of HTA. Notably, about 72 percent had difficulties with questions related to the main goals of HTA, evaluating the broader impacts of health technologies, and starting the evidence-based deliberative decision-making process.ConclusionsThe HTA course successfully covered basic concepts, yet it also highlighted the need for more comprehensive teaching of complex topics. The participants showed varying levels of understanding. This underscores the necessity for an HTA curriculum that equally emphasizes fundamental knowledge and in-depth analysis, preparing future healthcare professionals for complex decision-making in their roles.
PP59 Do Patient Contributions Matter? A Thematic Document Analysis Of NICE Ultra-Rare Disease Appraisals
Authors: Gentilini Arianna , Rana Alina 
Published: January 2025

IntroductionPatient organizations play a crucial role in health technology assessment (HTA), especially for rare diseases. Despite their important role, the contributions and impact of patient organizations have been overlooked in the literature. This study aims to address this gap by examining the contributions of patient organizations and their nominated experts in National Institute for Health Care and Excellence (NICE) highly specialised technology (HST) appraisals.MethodsWe thematically analyzed the scope and frequency of contributions from patient organizations and experts associated with 10 NICE HST appraisals completed between January 2022 and September 2023. First, to allow for a representation of what patient contributions focus on, their written submissions were categorized according to themes following a deductive/inductive approach, employing a tiered system for disease-, technology-, or submission-specific themes. Second, we compared the themes identified in written submissions with those found in the final NICE recommendations, included in the final evaluation determination (FED), to understand whether and to which extent patients’ contributions were considered.ResultsFrom 2013 to 2023, 22 drugs underwent HST assessment, with nearly half appraised during 2022 and 2023. All technologies received positive recommendations. A total of 475 unique patient contributions—from both patient organizations and their nominated experts—were identified in their written submission in support of the 10 HST appraisal assessed, predominantly emphasizing disease-specific themes (53%), such as quality of life. While 42 percent of raised themes aligned with FED content, 52 percent did not. When looking at individual appraisals, the share of themes mentioned in patients’ written submission explicitly considered in the FEDs ranged from nine percent to 73 percent, with a median of 50 percent.ConclusionsDespite progress in integrating patient inputs into HTA, this study reveals a discrepancy between patient priorities and explicit consideration in NICE’s final recommendations. While NICE consistently adheres to its methodology, certain patient-raised aspects are overlooked. Further research is important to discern the optimal areas and timing for patient contribution, refining NICE’s involvement strategies in their decision-making processes.