bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP04 The Modernized Cardiff Model: Multifaceted Modeling In The Era Of Cardiovascular-Kidney-Metabolic Syndrome
Authors: McEwan Philip , Foos Volker , Chen Jieling , Evans Marc , Roberts Geraint , Jenkins Robert , Gibson Andrew 
Published: January 2025

IntroductionIn the era of cardiovascular-kidney-metabolic syndrome, thorough evaluation of medicines with multiple treatment effects/indications demands a multifaceted modeling philosophy, despite the requirement of health technology assessment (HTA) models to focus on one disease. Using Cardiff, a model previously built for type 2 diabetes (T2D), we illustrate the changes needed to capture contemporary, holistic, patient-centered decision-making, and argue that HTA bodies should revise their approach.MethodsThe upgraded model enables therapy selection and escalation determined by HbA1c thresholds, cardiovascular risk (QRISK3), comorbidities (established cardiovascular or chronic kidney disease), and weight (body mass index ≥35 kg/m2). Risk factor trajectories were updated by incorporating UKPDS-90 equations and other relevant data sources. Clinical outcomes were predicted using new risk equations incorporating cardiovascular outcomes trial data whenever possible. The updated model was applied to assess quality-adjusted life years (QALYs) and lifetime costs in newly diagnosed T2D patients in the UK, modeled via a conventional glycemic-centric approach versus a multifactorial treatment algorithm. Extrapolation to the national level utilized estimates of annual incidence.ResultsThe updated treatment algorithm captured and quantified the impact of nuanced comorbidity management called for in guidelines. In a cohort of newly diagnosed T2D patients, 81 percent initiated an SGLT2 inhibitor within five years, predominantly due to increasing cardiovascular risk, versus zero percent when escalation was dictated by HbA1c alone. Broad, early use of SGLT2 inhibitors resulted in an additional 0.73 predicted QALYs and GBP10,757 (USD13,600) in predicted lifetime cost savings per patient versus a “traditional” approach. Cost savings were primarily due to avoided renal events; extrapolation to the national level predicted cost savings to the payer of GBP2.8 billion (USD3.5 billion), which traditional models cannot capture.ConclusionsThe modernized Cardiff model incorporates multifactorial prescribing guidelines and contemporary evidence around cardio-renal protection and is more adept at modeling costs and outcomes of multidimensional antidiabetic treatments; traditional glucose-centric modeling methods may introduce bias. Economic modeling and HTA processes must adapt to follow the complexities of modern disease management and remain relevant as healthcare systems address the cardiovascular-kidney-metabolic syndrome epidemic.
OP18 Incorporating Environmental Impacts In The Economic Evaluation Of Health Technology Assessment: State Of The Art And Challenges
Authors: Guirado-Fuentes Carmen , Sacks Analía Abt , García-Pérez Lidia , Carrion Carme , Trujillo-Martín Mar 
Published: January 2025

IntroductionEnvironmental impact has been poorly addressed in health technology assessment (HTA) processes despite its potential role in promoting more sustainable health systems. Initiatives to incorporate this dimension into economic evaluations (EE) that support HTA are few and far between. We aim to identify the state of the art and challenges for incorporating environmental impact into the EE of HTA.MethodsWe conducted a scoping review to identify publications on the assessment of the environmental dimension of health technologies from different approaches: establishment of theoretical frameworks and methods; data search strategies; identification of parameters, designs, and indicators; as well as descriptions of practical applications in HTA (literature review, EE, or budget impact analysis). The literature search was conducted through PubMed. Selected studies should provide insights to incorporate environmental impact into the EE of HTA regardless of the technology or environmental aspect considered (carbon footprint, use of resources, waste generation, etc.).ResultsFrom a total of 219 references initially identified, 22 publications meeting the selection criteria were found. The holistic approach is recognized as the most appropriate for incorporating the environmental dimension, through the evaluation of the entire life cycle of the technology, as well as the management of the disease and the use of resources throughout the care process. A large amount of information and accurate estimates about the impact of the technology are needed. Therefore, the first reported approaches have focused on particular aspects of the environmental impact of a health technology (mainly the carbon footprint).ConclusionsThe practical incorporation of the environmental dimension into the HTA is still very incipient. Foundations have begun to be established for its incorporation into economic evaluation. A consensus is required on the most appropriate methodologies and tools to collect the necessary data. It would also require a multidisciplinary approach and a framework for cooperation between all the stakeholders.
OP20 Incorporating External Information In Survival Extrapolation Using A Simplified Bayesian Approach
Authors: McConnell David , White Arthur , Lamrock Felicity , O’Callaghan Joan , McCullagh Laura , Leahy Joy , Trela-Larsen Lea 
Published: January 2025

IntroductionCost-effectiveness modeling often requires extrapolation of survival data from clinical trials over a long-term horizon. The choice of extrapolation method is often uncertain and can have a profound impact on the results. We propose a novel Bayesian approach towards incorporating external information (e.g., registry data or clinical opinion) into the extrapolation process as a means of reducing this uncertainty.Methods
Standard parametric survival curves are fitted to immature time-to-event data using maximum likelihood estimation (MLE). Separately, external information on expected cohort-level survival at a future time point is used to specify a prior probability distribution. These are combined to generate posterior distributions of survival curve extrapolations that simultaneously incorporate both observed data and external information. This is done using importance sampling and multivariate normal approximations of the likelihood and posterior distributions; it requires only summary model parameter estimates (and not patient-level data). We apply our method to analyze survival data from the KEYNOTE-426 trial of pembrolizumab+axitinib in advanced renal cell carcinoma.ResultsThe method was implemented in R, and outputs survival curve parameters that are compatible with cost-effectiveness models developed in other software (e.g., Microsoft Excel). In all examples considered, our method resulted in extrapolated survival predictions that were more closely aligned with the external information compared with the standard (MLE-based) approach. Incorporation of external information decreased between-distribution variance (reduced structural uncertainty), and generally also decreased within-distribution variance as well (reduced parameter uncertainty). Results were comparable with those obtained from the method of Cooney and White, which uses similar ideas but requires full patient-level data and is more computationally complex.ConclusionsThe extrapolation method we describe can reduce uncertainty when valid external information is available. Only MLE-based parameter estimates are required to implement our method, thus secondary model users such as HTA agencies can adjust survival extrapolations from existing cost-effectiveness models without access to patient-level data. Implementation is straightforward and computationally efficient, and outputs are easily incorporated into existing cost-effectiveness models.
OP22 Productivity Benefits Of Modifying Cardiovascular Disease Risk In Type 2 Diabetes In Australia: A 10-Year Prediction Dynamic Model
Authors: Abushanab Dina , Al-Badriyeh Daoud , Marquina Clara , Morton Jedidiah I , Lloyd Melanie , Zomer Ella , Talic Stella , Liew Danny , Ademi Zanfina 
Published: January 2025

IntroductionDiabetes has been shown to influence the individual’s work productivity in terms of both presenteeism (reduced productivity while at work) and absenteeism (absence from work because of illness). We sought to estimate the potential productivity gains associated with the modification of cardiovascular disease (CVD) risk in type 2 diabetes (T2D) over the next 10 years in Australia, from 2023 to 2032.MethodsDynamic models were structured to estimate productivity-adjusted life years (PALYs) lived by Australians aged 20 to 69 years. The model simulation was first undertaken assuming currently expected trends in the incidence of myocardial infarction (MI) and stroke in T2D (original cohort), as calculated using the 2013 PCE-ASCVD algorithm. Subsequent models were then re-simulated using hypothetical scenarios that reflected the potential benefits of CVD reduction using published trials. The model was also repeated assuming that the original cohort has no CVD. Differences in PALYs lived by the “original cohort” and the different cohorts with reduced CVD risk reflected the PALYs gained. Sensitivity analyses were conducted.Results
Using data from published studies, the model assumed a reduction of 50 percent in systolic blood pressure (SBP), a reduction of 50 percent in smoking, 50 percent increase in high-density lipoprotein cholesterol (HDL), and a reduction of 35 percent in incidence of T2D. Over the working lifetime, from 2023 to 2032, reducing SBP, smoking, and incidence of T2D led to the gain of 140,105, 333,127, and 998,805 PALYs, respectively. Further, increasing HDL and assuming the original cohort with T2D has no CVD are expected to lead to the gain of 71,623 and 889,455 PALYs, respectively. Sensitivity analyses confirmed the robustness of study findings.ConclusionsThe impact of CVD as a complication from T2D on work productivity is significant. Screening and prevention strategies tailored early in life are likely to exert a positive impact on health and work productivity.
OP23 Key Challenges For The Appraisal Of Disease-Modifying Dementia Drugs
Authors: Joshi Roshni , Dawoud Dalia , Salih Fatima , Avsar Tuba Saygin , Longson Carole , Crabb Nick 
Published: January 2025

IntroductionDisease-modifying dementia treatments (DMDTs) target amyloid beta or tau proteins and have the potential to change disease progression, representing a step change in the management and treatment of Alzheimer’s disease. Given the novel mechanism of action and impact on health care, the NICE Health Technology Assessment Innovation Laboratory (HTA Lab) sought to identify and contextualize the key issues for future appraisals.MethodsWe reviewed published assessment reports of DMDTs from international HTA agencies and conducted a scoping review of published economic models of pharmacological treatments to understand the challenges associated with evaluating the cost-effectiveness of dementia treatments. The HTA Lab held an engagement workshop with 27 external stakeholders, including expert clinicians, implementation partners, health economists, and representatives from international agencies to discuss and confirm the key issues and considerations likely to emerge during an appraisal of DMDTs.ResultsKey clinical and cost-effectiveness issues were identified and discussed. We concluded that consideration needs to be given to the diagnostic methods to identify the DMDT-eligible population in the UK, the validity of the surrogate outcomes used in the DMDT clinical trials, treatment effectiveness in different populations, and the incidence of DMDT-associated adverse events. Economic considerations include the type of economic model used in the appraisal, modeling the natural history of the disease, paucity of quality-of-life data in the treatment population, the inclusion of societal impact, treatment duration, stopping rules, and long-term effectiveness beyond the clinical trials.ConclusionsDMDTs could have the potential to transform Alzheimer’s disease care. With multiple treatments on the horizon, the appropriateness and acceptability of the new mechanism of action underpinning these treatments should be considered. We have identified areas of uncertainty that are likely to arise during an appraisal process, to facilitate the timely approval of these medicines and patient access.
OP33 Advancing Patient Experience Data Implementation In Reimbursement Decision-Making: Insights On Challenges And Opportunities From Multistakeholder Interviews
Authors: Vanneste Alice , Wens Io , Adriaenssens Tom , Janssens Rosanne , Barbier Liese , Huys Isabelle 
Published: January 2025

IntroductionPatient experience data (PED), encompassing patient preferences (PP), patient-reported outcomes (PROs), and patient input, play a pivotal role in understanding patient needs and informing healthcare decision-making, including reimbursement decisions. This study aimed to assess the current barriers hindering the integration of PED into practice and its particular challenges, opportunities, and concrete policy actions for the systematic implementation of PED.MethodsSemistructured interviews (n=38) were conducted with industry (n=12), non-profit organizations and academia (n=4), regulatory authorities (n=6), health technology assessment (HTA) bodies and reimbursement agencies (n=6), and patient organizations (n=10) in Europe. A thematic analysis was conducted to explore stakeholders’ perspectives and to gain a comprehensive understanding of challenges and opportunities related to the systematic implementation of PED. Interview transcripts were analyzed using the thematic framework analysis to extract and elucidate the insights provided by the diverse stakeholders.ResultsHTA and reimbursement interviewees agreed on the value of including quality-of-life data, particularly when assessed using validated PRO measures. Despite acknowledging the potential of PP, there remained reluctance to integrate PP into reimbursement decision-making. Participants expressed divergent opinions regarding who should collect PED, with some regulators favoring industry, while HTA and reimbursement agencies emphasized transparency and independent PED collection. Limited experience in assessing PED also contributed to hesitancy, underscoring the need for more guidelines, especially at the national reimbursement level. Stakeholders endorsed collaboration through joint scientific consultations, expressing optimism about the impact of the Regulation (EU) 2021/2282 on health technology assessment (HTAR).ConclusionsThis study emphasizes the high potential of PED in informing reimbursement decision-making, fostering a more patient-centered approach. Stakeholder disparities highlight the complexity, necessitating more guidance, scientific robustness, transparency, and collaboration. In light of these stakeholder considerations, the upcoming HTAR holds promise to enhance the systematic implementation of PED, aligning healthcare decision-making with patients’ needs and preferences.
OP41 Pharmaceutical Technologies Conditionally Approved By The National Institute For Health And Care Excellence: A Critical Analysis
Authors: Perez Yankier Pijeira , Hughes Dyfrig 
Published: January 2025

IntroductionThere are concerns with the National Institute for Health and Care Excellence (NICE) conditional approval process with respect to the quality and reliability of the methods of evidence generation, analysis, and reporting; and on the increasing focus on OwR (only with research) in preference to OiR (only in research). Our study critically appraises the methods, quality, and risk of bias of the evidence generated in response to NICE conditionally approved technologies.MethodsOur study reviewed pharmaceutical technologies appraised by NICE: technology appraisals approved between March 2000 and September 2022 and highly specialized technologies approved up to October 2023. From those, we identified appraisals with OiR and OwR conditional recommendations, and summarized the evidence requested by NICE as part of conditional approval. Then, evidence resubmitted to NICE for reappraisal was analyzed for its compliance with NICE’s initial recommendations for further research and assessed using the Cochrane Collaboration’s tools for risk of bias in randomized trials and the ROBINS-I tool for the non-randomized evidence.ResultsNICE made 54 conditional recommendations relating to technology appraisals (TAs) (13 OiR and 41 OwR) and five highly specialized technologies (HSTs). From those, 16 TAs presented additional evidence for reappraisal [nine OiR and seven OwR] and three HSTs [three OwR]. Two of the nine reappraised TAs with OiR recommendation and four of the seven OwR fully complied with NICE’s request for further evidence. All three HSTs complied in full. However, the majority of reappraised TAs and HSTs included evidence that was deemed to be at serious, high, moderate, or unclear risk of bias.ConclusionsThe quality of evidence presented to NICE following conditional approval varies considerably. There is often widespread noncompliance with requests for further research when conditionally approving a pharmaceutical technology. Complying with NICE’s requests, however, does not necessarily guarantee high-quality evidence when the technology is reappraised. Evidence generated in response to NICE conditional approval recommendations should be subject to quality standards.
OP44 Cost-Of-Illness And Cost–Consequence Analysis Of Dementia In Italy
Authors: Mennini Francesco Saverio , Bini Chiara , Sciattella Paolo , Vanacore Nicola 
Published: January 2025

IntroductionIn Italy, there is a lack of evidence regarding the care and management of patients with dementia, as well as the associated costs. This study aims to fill this informational gap by utilizing data from both literature and national surveys.MethodsA prevalence-based cost-of-illness (COI) model was developed to assess dementia-related costs from a societal perspective. The resources utilization for management and treatment of patients with dementia was derived from both the literature and the analysis of surveys conducted by the National Institute of Health on the social-health structures dedicated to dementia. Indirect costs from informal caregiving were evaluated through a human capital approach. Additionally, a cost–consequence analysis (CCA) was conducted to assess the economic impact of healthcare resource utilization changes.ResultsBased on an estimated 1,150,691 dementia cases in Italy, with approximately 12 percent institutionalized, the COI model estimated an annual expenditure of around EUR23.6 billion (USD25.7 billion) for dementia patient management, with 63 percent attributed to out-of-pocket expenses. CCA indicated that if all affiliated with Centers for Cognitive Disorders and Dementia (CDCD) received nonpharmacological interventions (versus the surveyed 25.5 percent), there would be a direct cost increase of approximately EUR4.3 million (USD4.7 million).ConclusionsThis analysis provides an updated overview of current dementia patient management in Italy, offering valuable insights for decision-makers to prioritize health policies and interventions for patients and their caregivers.
OP46 A New Modular Approach To Updating National Institute For Health And Care Excellence Manuals
Authors: Walker Lizzie , Flight Laura , Prescott Carl , Shah Koonal 
Published: January 2025

Introduction
Methods and processes for health technology assessment (HTA) must be sufficiently flexible to reflect emerging best practice and adapt to changes in the health and care landscape. They must also be clearly documented and subject to review, assessment, and consultation. This requires a framework to flexibly update HTA manuals, while providing stability and predictability for stakeholders.MethodsThe National Institute for Health and Care Excellence (NICE) is introducing a modular approach to updating its manuals. Previously, NICE completed full, but infrequent, updates to its manuals. A modular update is a review of the process and/or methods informing NICE’s guidance in a specific subject area, which can then be updated in the appropriate manual(s) if required. We are developing a modular updates framework that sets out NICE’s approach for identifying and prioritizing modular updates to review, reviewing the evidence and proposing changes to the manual(s), engaging and consulting with stakeholders, and implementing the modular updates.ResultsThe proposed approach allows external stakeholders to contribute to topic identification and the content of updates, helping to ensure that the manuals meet the needs of users. The first modular update to the NICE manual for health technology evaluations was published in October 2023. This included updated processes for carrying out cost comparisons, streamlining committee decision-making and handling confidential information. Potential future modular updates may include updated methods to consider health inequalities.ConclusionsThe introduction of a modular approach will enable NICE to be more agile and responsive in monitoring, reviewing, and improving our methods and processes, making sure they remain cutting edge as the health and care landscape continues to evolve.
OP50 NICE Listens: Engaging The Public On How Environmental Sustainability Should Be Considered In Health Technology Assessment
Authors: Murray Alice , Shah Koonal 
Published: January 2025

IntroductionInvolving the public is essential to building trust in health technology assessment (HTA) organizations. The National Institute for Health and Care Excellence (NICE) runs a deliberative public engagement program, NICE Listens. It was used to explore informed public opinion on how environmental sustainability should be taken into account in HTA.MethodsTwenty-three general public participants from across England took part in three iterative online workshops (each lasting two or three hours, held three weeks apart in 2022). The workshops included trade-off exercises, role-play, group discussion, and video clips from interviews with sustainable healthcare experts.Results
Strong support was found for NICE taking action to make healthcare more environmentally sustainable. Support increased as participants learned that sustainable healthcare offers co-benefits, such as reduced burden on the National Health Service through better self-management of conditions. Participants did not want health outcomes to be compromised in pursuit of sustainability. We identified some circumstances where they found it acceptable to consider the environmental impact of interventions in decision-making: when effective treatments already exist; when the condition is not severe; when the alternative is equally cost effective; and when greener options are marginally higher in cost but as clinically effective as the alternative.Conclusions
The findings demonstrate that environmental sustainability is clearly considered a relevant element of value. They also offer insight into how the environmental impact of health interventions should be considered in HTA. Further research should focus on methods for consistent measurement of the environmental impacts of health interventions and the incorporation of those impacts into decision-making.