bluuebunny/crossref_metadata_2025_split huggingface dataset:
OD29 From Listening To Lift Off: Developing A Three-Year Public Involvement And Engagement Strategy For NICE
Authors: Rasburn Mark , Crosbie Helen , Fitzpatrick Ella , Livingstone Heidi , Morgan Clare , Norburn Laura , Taylor Sally , Tonkinson Mandy , Wigmore Janine 
Published: January 2025

IntroductionInvolving patients in the health technology assessment (HTA) lifecycle is a core principle at the National Institute for Health and Care Excellence (NICE). To achieve this, NICE has adopted a mixed approach to patient and public involvement and engagement (PPIE) spanning the entire appraisal process. To ensure the PPIE approach enables meaningful involvement, NICE engaged with stakeholders to review its effectiveness and identify areas for improvement.MethodsIn 2023, an independent consultant reviewed NICE’s PPIE approach and engaged with NICE staff and external stakeholders from patient organizations, individual patient contributors, and engagement leads at national health and social care organizations. The engagement included interviews with NICE staff (n=19) and external stakeholders (n=13), and an online survey that received 83 responses from patient organizations and patient contributors. Using this feedback, NICE’s patient and public involvement program conducted four focus groups to develop a framework of improved methods and processes for PPIE with NICE staff, patient organizations, and patient contributors.ResultsThe engagement identified many positives in NICE’s approach to PPIE, including:

•lay members sitting on each HTA committee as equal members•patient organizations providing written evidence to HTA committees•patient experts providing written and verbal testimony to HTA committees•support provided by NICE.The engagement also identified areas where PPIE could have a greater impact, including:•improved methods for collecting patient evidence and insight•strengthening the role of lay members•collating and reusing previously collected patient evidence•taking a proportionate approach to involving small organizations•allocating staff resources to focus on impactful PPIE practices.

ConclusionsNICE has developed a draft framework for an improved approach to increase the impact of PPIE in HTA decision-making. In 2024, NICE will publicly consult with NICE staff and external stakeholders to review the framework, agree the strategic aims, and develop metrics for measuring success. Following this consultation, the findings and NICE’s updated approach to PPIE will be presented.
OD31 Value-Based Healthcare And Health Technology Assessment: Opportunities For Implementing A Colorectal Cancer Patient-Centered Care System
Authors: Asselin Genevieve , L’Espérance Sylvain , Nourissat Alice , Rhainds Marc 
Published: January 2025

IntroductionThe value-based healthcare (VBHC) concept links dollars spent to outcomes that matter to patients, rather than to the volume of services. A health technology assessment (HTA) was carried out to determine if the VBHC model could be effective to improve care delivery in colorectal cancer at the CHU de Québec-Université Laval.MethodsA systematic review on VBHC models implemented in oncology was conducted in indexed databases and grey literature between January 2000 and January 2022. Semistructured interviews were conducted from various key informants in our hospital (n=19), including three patients, to characterize the colorectal cancer care organization. HTA was conducted with an interdisciplinary group including surgeons, nurses, hospital managers, and patients.ResultsKey factors to consider in the implementation of VBHC include organizing care around the same medical condition, measuring outcomes and costs for every patient, and tracking data through information technology platforms. Results from case studies suggest that VBHC may have promising effects on clinical, patient-reported, and process outcomes measures. Several issues regarding the degree of agreement with the VBHC principles have been identified in our facility, including a non-uniform care trajectory, a lack of an integrated interdisciplinary team, and the absence of efficient information technologies.ConclusionsIt was recommended that the CHU de Québec-Université Laval initiates an organizational transformation in colorectal cancer care to implement a VBHC model. An effective care transformation will require a significant culture change with impacts on organization and practices but also with positive spin-offs by creating value and patient-centered
care.
OD44 Consolidated Health Economic Evaluation Reporting Standards For Interventions That Use Artificial Intelligence (CHEERS-AI)
Authors: Avsar Tuba Saygin , Elvidge Jamie , Hawksworth Claire , Knies Saskia , Zemplenyi Antal , Petykó Zsuzsanna , Siirtola Pekka , Chandra Gunjan , Srivastava Divya , Denniston Alastair , Chalkidou Anastasia , Delaye Julien , Nousios Petros , Gomes Manuel , Wang Junfeng , Petrou Stavros , Dawoud Dalia 
Published: January 2025

IntroductionProgress and innovation in artificial intelligence (AI)-based healthcare interventions continue to develop rapidly. However, there are limitations in the published health economic evaluations (HEEs) of AI interventions, including limited reporting on characteristics and development of algorithms. We developed an extension to the existing Consolidated Health Economic Evaluation Reporting Standards (CHEERS) to improve consistency, transparency, and reliability of the reporting of HEEs of AI interventions.MethodsThe Delphi method was used, following a prespecified study protocol. A steering group with expert oversight was formed to guide the development process. A long list of potential items was defined based on two recent systematic reviews of HEEs of AI-based interventions. The steering group identified and invited 119 experts to the three-stage survey. Participants were asked to score each item on a nine-point Likert scale, and they were also able to provide free-text comments. The final checklist was piloted on a random sample of nine HEEs of AI-based interventions.ResultsThree stages of the Delphi survey were completed by 58, 42, and 31 multidisciplinary respondents, respectively, including HTA specialists, health economists, AI experts, and patient representatives. The CHEERS-AI extension includes 18 AI-specific reporting items. Ten are entirely new items, including considerations about user autonomy, validation of the AI component, and AI-specific uncertainty. In addition, elaborations on eight existing CHEERS items were added to emphasize important AI-specific nuances. Some participants highlighted that CHEERS-AI can provide key benefits; for example, it could clarify the misconception that the predictive algorithms supporting AI-driven healthcare interventions are available for use without cost.ConclusionsCHEERS-AI can aid in improved reporting quality for researchers, editors, and reviewers conducting or assessing HEEs of AI interventions.
OD46 Inequalities In Dementia: Identifying Instruments For Measurement
Authors: Hodgson Sian , Hayes Helen , Cubi-Molla Patricia , Garau Martina 
Published: January 2025

IntroductionDespite dementia being the seventh leading cause of death globally, there is relatively little discussion of the presence and impact of inequalities in this context. We explore ways to quantify the magnitude and variation over time of inequalities related to people living with dementia (PLWD) and their informal carers.MethodsWe conducted a targeted literature review to identify inequalities faced by PLWD and their informal carers regarding their access to and experience of health and social care in England, Wales, and Northern Ireland. We selected four of the identified inequalities as case studies (CS) to explore data and methods that can be used to measure and monitor progress to tackle them. The CS considered were: (CS1) timely diagnosis in rural areas; (CS2) financial pressures for informal carers; (CS3) timely diagnosis in deprived areas; and (CS4) diagnosis rates for ethnic minority groups. We use data from 2018 to 2023 in England.ResultsWe identified 110 inequalities for PLWD and 28 inequalities for carers. For CS1, we proposed two measures: the “rurality gap” (gap in diagnosis rates between the most and least rural areas) and the “concentration index” (the extent to which diagnosis rates are distributed disproportionately between less or more rural areas). The rurality gap suggests that diagnosis rates are five to eight percent lower in rural areas in England. The concentration index supports this finding. CS2 shows that 41 percent of informal carers experience financial difficulties. Due to insufficient data, it was not possible to construct robust measures for CS3 and CS4.Conclusions
Many inequalities for PLWD and their informal carers are reported in the literature. Our CS highlight the need to improve methods and data to measure a set of inequalities, including those to calculate dementia prevalence and measure timely diagnosis. Better data is crucial now to inform value assessment of the upcoming Alzheimer’s disease treatments and avoid exacerbating existing inequalities.
PP06 Incentives To Incorporate Innovation Into Care Delivery Processes: A Scoping Review And SWOT Analysis
Authors: Haig Madeleine , Main Caitlin , Kanavos Panos 
Published: January 2025

IntroductionThis study investigated the various incentives employed in Organisation for Economic Co-operation and Development (OECD) and European Economic Area (EEA) countries to enhance access to innovative medical technologies. The literature review encompasses real and theoretical models, offering an overview of strategies to bridge the gap between innovation and access. A strengths, weaknesses, opportunities, and threats (SWOT) analysis was conducted to analyze incentives in health system and therapeutic area context.MethodsThe review methodically examined peer-reviewed articles, reports, and policy documents published between 2000 and 2023. Databases searched include PubMed, Scopus, Web of Science, and EconLit. Grey literature was searched from international organizations’ websites, including the World Bank, World Health Organization (WHO), OECD, Pan American Health Organization (PAHO), and European Commission. Inclusion criteria focused on relevance of financial and non-financial mechanisms to effective implementation of innovative medical technologies, and their application within OECD and EEA countries. COVID-19 research, vaccines, cost-effectiveness studies, and studies that did not discuss implementation were excluded. A SWOT analysis was utilized to categorize the mechanisms by therapeutic area and health system design.ResultsThe review identified diverse mechanisms, including reinsurance, impact bonds, outcomes-based agreements, annuity payments, and risk-sharing agreements. Financial mechanisms, such as outcomes-based agreements, were prominent but highlighted implementation obstacles, including a lack of data infrastructure capable of linking outcomes to payments, which ultimately undermines the effectiveness of these strategies. Non-financial mechanisms, such as population health management, were also identified. The effectiveness varied, with some models showing significant improvement in technology accessibility, while others faced implementation and affordability challenges. Comparative analysis highlighted differences in efficacy dependent on the therapeutic area and type of health system in which the incentive is applied.ConclusionsThe review underscores multifaceted approaches to improve access to innovative medical technologies. While financial incentives play a crucial role, non-financial strategies are also vital. This study provides insights into which incentives are most effective in certain health systems and therapeutic areas. Policymakers can benefit from these insights, leveraging successful models and addressing challenges to ensure equitable access to medical innovations.
PP08 Prognostic Factors For Survival In Adults With Burkitt Lymphoma: A Systematic Review And Meta-Analysis
Authors: De Armas-Castellano Aythami , Infante-Ventura Diego , González-Hernández Yadira , Pino-Sedeño Tasmania del , Leon-Salas Beatríz , Quirós-López Raúl , Trujillo-Martín Mar ,   EuroBloodNet Working Team
Published: January 2025

IntroductionBurkitt lymphoma (BL) is a rare and highly aggressive subtype of non-Hodgkin’s lymphoma. Several studies have identified prognostic factors (PFs) for disease progression and mortality among adults with BL. However, there is no consensus on risk stratification based on PFs. This study aims to identify, critically assess, and synthesize the available evidence on PFs for survival in adults with BL.MethodsA systematic review of the literature was conducted. MEDLINE, Embase, and CENTRAL were searched from inception to 22 February 2022. Randomized or non-randomized clinical trials and longitudinal observational studies were eligible for inclusion. Reference screening, data extraction, and risk-of-bias assessment using the Quality in Prognosis Studies (QUIPS) tool for prognostic factor studies were conducted independently and in duplicate. Publication bias was examined by visual inspection of funnel plots. Effect measures and the corresponding 95% confidence intervals were pooled with an indirect variance estimation in meta-analyses using Review Manager 5, and sensitivity analyses were conducted. Certainty of evidence was assessed using GRADE.ResultsThe search identified 1,119 references after duplicate removal. Of these, 76 potentially relevant papers were selected for full-text assessment and 36 studies (N=10,882) reported in 39 articles were eligible for inclusion. Older age, higher performance status, and central nervous system involvement were associated with poorer overall survival (OS) and progression-free survival (PFS). Black patients exhibited significantly lower OS and relative survival. Bone marrow involvement and higher albumin levels were associated with poorer OS. Treatment with rituximab and treatment with methotrexate were associated with better OS and PFS. No significant differences in survival were found for HIV status, sex, and risk stratification.ConclusionsThis study, framed within a collaboration with European Reference Network EuroBloodNet, provides a comprehensive and methodologically rigorous evidence review on PFs in adults with BL. Several significant associations of PFs and survival estimates were observed, providing data to inform treatment decisions and to improve patient care.
PP09 Fecal Immunochemical Tests For Patients With Symptoms Suggestive Of Colorectal Cancer: A Systematic Review And Multiple-Threshold Meta-Analysis
Authors: Harnan Sue , Hamilton Jean , Simpson Emma , Clowes Mark , Biz Aline Navega , Whyte Sophie , Ren Shijie , Cooper Katy , Ball Alex , Benton Sally , Carten Rachel , Kurien Matt , Monahan Kevin , Heathcote Laura , Stevenson Matt 
Published: January 2025

IntroductionExtending fecal immunochemical tests for hemoglobin (FITs) to primary care patients with high-risk symptoms suggestive of colorectal cancer (CRC) could reduce colonoscopy waiting lists, enabling earlier treatment. Higher FIT thresholds could decrease referrals but increase missed disease compared with lower thresholds. We aimed to systematically review and synthesize test accuracy data across thresholds for use in a cost-effectiveness analysis.MethodsSearches across ten sources were conducted (December 2022). Included were diagnostic accuracy studies of HM-JACKarc, OC-Sensor, FOB Gold, QuikRead go, NS-Prime, and four Immunodiagnostik (IDK) tests in patients presenting to, or referred from, primary care with symptoms suggestive of CRC using any reference standard. Risk of bias was assessed with QUADAS-2. Syntheses of sensitivity and specificity at all reported thresholds were planned for each test to provide summary estimates at all possible thresholds within the observed range. Sensitivity analyses investigating population type and reference standard, and subgroup analyses by patient characteristics (e.g., anemia, age, sex, ethnicity) were conducted.ResultsHM-JACKarc (n=16 studies) sensitivity ranged from 95.9 percent (95 percent credible interval [95% CrI]: 92.7, 97.9) to 46.3 percent (95% CrI: 37.4, 54.9) and specificity from 65.1 percent (95% CrI: 55.6, 74.8) to 97.7 percent (95% CrI: 94.7, 99.2) (thresholds 2 and 400 μg hemoglobin/g feces [μg/g], respectively). OC-Sensor (n=11) sensitivity ranged from 94.2 percent (95% CrI: 91.2, 96.7) to 54.2 percent (95% CrI: 48.4, 60.2) and specificity from 62.7 percent (95% CrI: 47.4, 77.2) to 97.3 percent (95% CrI: 92.9, 99.3) (thresholds 4 and 200 μg/g, respectively). FOB Gold (n=3) sensitivity ranged from 91.4 percent (95% CrI: 71.6, 99.6) to 73.9 percent (95% CrI: 53.8, 91.2) and specificity from 78.1 percent (95% CrI: 70.0, 86.0) to 96.4 percent (95% CrI: 92.6, 98.9) (thresholds 2 and 150 μg/g, respectively). There were limited or no data on the other tests.ConclusionsSensitivity and specificity were synthesized for three tests only, since data for the remaining tests were extremely limited or absent. Even at the lowest threshold, none of the tests had perfect sensitivity. Future studies should further investigate comparative accuracy and the impact of patient characteristics, patient recruitment criteria, and the reference standard on estimates of diagnostic test accuracy.
PP14 Recombinant Human Erythropoietin For Sickle Cell Disease And Brazilian Healthcare System Sustainability: Cost-Effectiveness And Budget Impact Analysis
Authors: Gargano Ludmila , Lobo Clarisse , De Oliveira Haliton Alves , Lucchetta Rosa 
Published: January 2025

IntroductionSickle cell disease (SCD) is a rare disease, including renal complications. Erythropoiesis-stimulating agents (i.e., recombinant human erythropoietin, rHuEPO) are recommended for SCD and renal impairment. Evidence suggests its effectiveness in raising hemoglobin (Hb), which may reduce the need for regular blood transfusions. Cost-effectiveness analysis (CEA) and budget impact analysis (BIA) of rHuEPO were performed from the Brazilian Unified Health System (SUS) perspective.MethodsA decision tree was created to assess rHuEPO’s impact on reducing blood transfusions. Quality-adjusted life year (QALY) for transfusion dependency and direct medical costs related to rHuEPO and transfusion were considered. SCD patients with worsening kidney function and Hb levels enter the model with an indication of receiving regular transfusions; those who proceed to the rHuEPO+standard care arm are likely to have clinically relevant elevation in Hb levels (i.e., <1.5 g/dL), suspending transfusions, and those who do not respond to treatment continue to receive regular transfusions. BIA population was estimated based on epidemiological data, considering direct costs over a five-year horizon.ResultsrHuEPO+standard care compared to standard care generated 36.8 percent less need for transfusions, resulting in an increase of 0.033 QALY and a saving of BRL11,564 (USD2,362) per patient/year. rHuEPO was the dominant alternative; that is, there was greater clinical benefit and lower total cost. At BIA, the eligible population was 5,274 patients per year, on average. The direct cost of acquiring rHuEPO for the total eligible population summed BRL13,737,129 (USD2,806,016) in five years. However, considering the estimated effectiveness of CEA in reducing transfusions, BIA demonstrated savings of BRL96,545,791 (USD19,720,936) accumulated over five years.ConclusionsHealth technology assessments showing the new alternative as dominant are uncommon, especially in rare diseases, where patented orphan drugs usually have increased costs, which is not the case of rHuEPO. In this analysis, rHuEPO remained the dominant alternative and its incorporation would result in savings that may contribute to the sustainability of the Sistema Único de Saúde, Brazil’s national health system.
PP18 Red Flags For The Early Diagnosis Of Rare And Complex Connective Tissue And Musculoskeletal Diseases
Authors: García Guillermo Pérez , Vicente Edo Maria José , Valero Montserrat Salas , Remón Lucía Prieto , Benavent Patricia Gavin 
Published: January 2025

IntroductionIn collaboration with a European Reference Network for rare diseases, we aimed to identify red flags for the diagnosis of rare and complex connective tissue and musculoskeletal diseases (rCTDs). Some indicators, presented as red flags, might raise clinicians’ awareness about the presence of rCTDs. Their identification is critical in primary care, where they are most likely to be first observed.MethodsFirstly, we conducted a scoping review to identify red flags already published in the scientific literature. We included studies about people with rCTDs that described red flags, warning signs, alarm symptoms, and pathognomonic signs identifiable in a primary care setting. Then, we conducted a systematic review of evidence pointing out which signs and symptoms should arouse suspicion specifically for IgG4-related disease. We included studies providing estimates of diagnostic precision or prevalence of signs and symptoms, and we assessed their quality and applicability to the review question. We conducted systematic searches in major medical databases and manual searches in rare disease resources.ResultsFor the scoping review, 49 studies out of 1,656 records met the inclusion criteria. Two reported red flags for autoimmune diseases altogether, and 14 described red flags for systemic sclerosis. For the systematic review, seven studies out of 4,477 records met the criteria, comprising five diagnostic precision studies and two large case series. These were generally rated as having a high risk of bias and were included as indirect evidence. We identified 32 potential IgG4-related disease red flags, 10 related to clinical history findings and basic signs or symptoms, and eight belonging to common laboratory findings and basic imaging techniques.ConclusionsRed flags for rCTDs have generally been established through expert consensus and lack valid indicators for diagnosis, such as sensitivity, specificity, or predictive values. They frequently overlap among different rCTDs. Potential red flags are prone to change as further evidence emerges. This shows the need to collaborate with reference networks to address rare diseases where the evidence is still scarce.