bluuebunny/crossref_metadata_2025_split huggingface dataset:
PD112 Calcitonin Gene-Related Peptide Monoclonal Antibodies Reimbursed Under A Managed Access Protocol In Ireland
Authors: Finnigan Karen , Smith Amelia , Gorry Claire , Clarke Sarah , Barry Michael 
Published: January 2025

IntroductionThe calcitonin gene-related peptide monoclonal antibodies (CGRP MABs) erenumab, fremanezumab, and galcanezumab are reimbursed in Ireland under the High Tech Arrangement, subject to a managed access protocol (MAP), for the prophylaxis of chronic migraine in adults in whom three or more prophylactic treatments have failed. This study provides an overview of submitted reimbursement applications and the utilization of CGRP MABs.MethodsThe MAP for CGRP MABs was introduced on 1 September 2021 and is operated by the Health Service Executive (HSE) Medicines Management Programme. Individual patient reimbursement applications for CGRP MABs submitted through an online reimbursement application system between 1 September 2021 and 30 April 2023 were reviewed. Utilization data from 1 September 2021 to 30 April 2023 were extracted from the HSE Primary Care Reimbursement Service national pharmacy reimbursement claims database for the High Tech Arrangement. Analysis was performed using SAS® 9.4 software.ResultsA total of 1,517 reimbursement applications were submitted in the study period. Reimbursement was approved for 96.1 percent (n=1,458) of the applications. A total of 1,399 individual patients (mean age 45 years) were dispensed a CGRP MAB under the High Tech Arrangement between September 2021 and April 2023, the majority of whom were women (n=1,141). Almost 90 percent of patients were considered treatment adherent. In April 2023, the market share of the individual CGRP MABs on the High Tech Arrangement was 56 percent (n=599) for fremanezumab, 38.3 percent (n=409) for erenumab, and 5.7 percent (n=61) for galcanezumab.ConclusionsMAPs are part of the health technology management approach to drug reimbursement in the Irish healthcare setting, ensuring that reimbursement is in line with approved subgroups of the licensed indication. Used in conjunction with health technology assessment, MAPs enable access to high-cost drug treatments for patients with the greatest unmet need, while providing budgetary oversight and certainty for the payer.
PD135 Real-World Evidence On The Effects Of Robotic Prostatectomy In Poland
Authors: Dzik Maciej , Płusa Aneta , Zaleska Monika 
Published: January 2025

IntroductionRobot-assisted radical prostatectomy (RARP) was incorporated into the public healthcare system in Poland in April 2022. RARP quickly gained popularity among healthcare providers, constituting nearly 25 percent of all publicly financed prostatectomies by the end of 2022. The aim of this study was to evaluate the effects of RARP using early real-world data from the public reporting system.MethodsThe sample included 7,177 patients who had either RARP or conventional radical prostatectomy (CRP) between 27 March and 31 December 2022. CRP was performed as either an open or a laparoscopic procedure. Due to reporting limitations, a comparison with laparoscopic radical prostatectomy (LRP) only was carried out on a subset of 2,306 patients who had prostatectomy after 20 September 2022. Data analyzed included length of hospitalization, the percentage of patients who received transfusions of blood products or who were hospitalized within 30 days of discharge, and number of deaths.ResultsIn total 2,190 patients had RARP. Compared with both CRP and LRP, RARP was associated with a reduction in hospital stay by 1.13 days (95% confidence interval [CI]: −1.27, −0.99; p<0.0001) and 0.83 days (95% CI: −1.02, −0.64; p<0.0001), respectively, and a lower risk of needing a transfusion of blood products, with odds ratios of 0.39 (95% CI: 0.31, 0.49; p<0.0001) and 0.53 (95% CI: 0.39, 0.77; p=0.0008), respectively. There were no statistically significant differences in rates of rehospitalization. Only three hospitalizations ended due to death. By 31 December 2022 only seven patients in the RARP group and 19 in the CRP group had died.ConclusionsThe findings of this study suggest that there is a marginal, though statistically significant, benefit with RARP, compared with CRP and LRP, that may be factored into economic evaluations of RARP.
PD144 Uncertainty In Precision Medicine: The Value Of Research To Support The Value Of Personalized Intervention Policies
Authors: Rothery Claire , Escudero Fernando Alarid , Kunst Natalia , Manca Andrea 
Published: January 2025

IntroductionOne-size-fits-all policies are not always optimal. Stratified decision-making is only possible when the characteristics used to define subgroups can be identified for stratum-specific predictions of outcomes. Conditioning decisions on characteristics that are not readily known may require information (diagnostic, prognostic, predictive) to be derived, the value of which needs to be assessed to support personalized strategies.MethodsA general framework was developed to show how personalized policies can be accountably informed by characterizing uncertainty, heterogeneity, and bias in evidence. In the framework, observed heterogeneity was disentangled from random variability by conditioning the value of the model input parameters on a set of prognostic or predictive variables, while unobserved heterogeneity was quantified as the systematic variability that cannot be explained given current information. Value of information analysis was used to quantify the value of additional information for resolving decision uncertainty in model input parameters and to identify individual- or subgroup-level attributes that contribute to the degree of heterogeneity.ResultsDecision-making based on average cost effectiveness fails to account for the role that sources of outcome variability play in guiding nuanced decision-making. Conditioning on a set of known covariates to reflect observable heterogeneity may be extended to conditioning on the latent random variable for unobservable covariates to quantify unobservable heterogeneity. Quantifying the potential value of research to inform subgroup- or individual-level attributes may be used to direct further research toward the attributes expected to be of most interest because they drive the value of individualized decisions—the expected value of sample information for attributes.ConclusionsTwo distinct, but interrelated concepts for assessing the value of stratified decision-making are important: (i) the value of heterogeneity; and (ii) the value of further research to inform both heterogeneous factors and to reduce decision uncertainty in precision medicine. Assessing the value of unexplained heterogeneity and bias can be central to supporting the value of personalized intervention strategies in health technology assessment.
PD146 Can We Properly Evaluate Genetic And Genomic Applications? A Systematic Review Of Health Technology Assessment Reports
Authors: Migliara Giuseppe , Sciurti Antonio , Mussetto Ilaria , De Blasiis Maria Roberta , Di Lorenzo Giuseppe , Pierri Dr Francesco , Leone Immacolata , Marzuillo Carolina , Villari Paolo , Baccolini Valentina 
Published: January 2025

IntroductionThe last decade has witnessed a steady adoption of personalized medicine. However, the evaluation of genetic and genomic tests is not straightforward. The purpose of this systematic review was to identify health technology assessment (HTA) reports assessing genetic and genomic tests to summarize the methodologies used, the maturity level of the evidence included, and the highlighted research gaps.Methods
The PubMed, Scopus, and Web of Science databases were searched for HTA reports of genetic or genomic tests. The main national and international HTA report repositories (e.g., the international HTA database) were also searched. HTA reports that were specifically created to assess genetic or genomic technologies and included at least three core evaluation components (analytic validity, clinical validity, clinical utility, economic evaluation, organizational aspects, or ethical, legal, and social implications) were included. This study was supported by the European Commission and the Ministry for Universities and Research under the National Recovery and Resilience Plan (M4C2-I1.3 Project PE_00000019 “HEAL ITALIA”).ResultsOverall, 27,331 unique records were retrieved, 55 of which were included in the systematic review. The reports were mainly from Australia (29%), Canada (27%), and the UK (25%); focused on pharmacogenomics (36%) and oncology (35%); and investigated test use for treatment guidance (42%) or diagnosis (29%). The most reported evaluation components were economic evaluation (87%), clinical utility (76%), and clinical validity (67%). On the other hand, personal utility (7%), patients’ perspectives (27%), and ethical (15%), legal (11%), and social (24%) implications were poorly represented. Analytical validity, safety, and organizational aspects were included in about half of the reports.Conclusions
Although these are only preliminary results, the substantial lack of a shared standard in the evaluation of genetic and genomic applications is clear given the heterogeneity of the dimensions addressed among the reports. Theres is a need to strengthen evaluation of the neglected dimensions, which are often of primary importance in defining the value and risks of personalized medicine.
PD150 How Is Genetic Testing Evaluated? An Updated Systematic Review Of Assessment Frameworks
Authors: Baccolini Valentina , Migliara Giuseppe , Sciurti Antonio , Pitini Erica , Kaminska Anna Ewa , Soccodato Valentina , Iera Jessica , Leone Immacolata , Marzuillo Carolina , Villari Paolo 
Published: January 2025

IntroductionAssessment of the risks and benefits of genetic and genomic tests has long been addressed using ad hoc evaluation methods. They are mostly ACCE-based, focus on technical aspects, and often overlook economic and organizational considerations. The few health technology assessment (HTA) based approaches, though more comprehensive, lack validation and implementation. This review’s purpose was to identify evaluation frameworks for genetic and genomic tests and to synthesize their key aspects.MethodsPubMed, Scopus, Web of Science, Google Scholar, and Google Search were used to identify records describing any assessment framework for genetic or genomic tests. As this was an update of a previous systematic review, the search was restricted to records published from 1 October 2020. Inclusion criteria were documents describing evaluation frameworks for genetic or genomic tests that were original, specifically created, and covered at least three evaluation components (analytic validity, clinical validity, clinical utility, economic aspects, or ethical, legal, and social implications). This study was supported by the European Commission and the Ministry for Universities and Research under the National Recovery and Resilience Plan (M4C2-I1.3 Project PE_00000019 “HEAL ITALIA”).ResultsOverall, 22,862 records were retrieved and 12,546 unique records were screened, of which 67 documents were assessed for eligibility. However, none of these met the inclusion criteria and no additional framework was found. In contrast, a total of 37 studies reporting 30 different frameworks were included from the previous systematic review. The analysis of these frameworks revealed that they were published between 2000 and 2019 and were mostly based on the ACCE model (n=13), on the HTA process (n=6), or both (n=3). Others referred to the Wilson and Jungner screening criteria (n=3) or to a mixture of different criteria (n=5).ConclusionsA pressing need exists for a universally accepted evaluation framework for genetic and genomic tests. A shift from ad hoc assessments to a general HTA methodology, potentially based on the EUnetHTA Core Model®, is needed. By integrating solid theoretical and methodological principles, a validated, comprehensive, and widely shared tool for evaluating genetic tests can be realized, promoting consistency across Europe and beyond.
PD166 Artificial Intelligence, Healthcare System Budget Cuts, And Flow of New Evidence: Moving To Living Health Technology Assessment Reform
Authors: Sarri Grammati , Abogunrin Seye 
Published: January 2025

IntroductionHealth technology assessment (HTA) agencies struggle with how to ensure timely assessment of promising technologies, especially considering the volume of rapidly produced evidence using complex analytical methodologies and applications, such as artificial intelligence (AI). Furthermore, healthcare systems that are already overburdened are now dealing with issues related to sustainability and increasing budgetary constraints resulting from several public health emergencies, such as the COVID-19 pandemic.MethodsA targeted literature review of primary publications in English published during the last five years was conducted to answer the following research question: Would AI integration into health outcomes research and health economics encourage automation in the HTA process, allowing for a living model—a real-time, dynamic approach using explicit methods to determine the value of a technology at different points in its lifecycle—to be implemented? We selected publications presenting information on the following concepts: automation in evidence generation; health economics in the decision-making context; cost efficiencies from the integration of automation; and separation of concepts such as lifecycle and living HTA. A narrative synthesis was conducted.ResultsThe publications selected explored four different aspects of the living concept in decision-making: living clinical guidelines, living evidence reviews and economic evaluations, and living HTA. Automation in systematic reviews (screening and data extraction), including time efficiencies, was the most frequently reported living aspect. The value of open-source economic models was increasingly recognized. Few references were found for methods such as living meta-analyses or network meta-analyses. Adaptive HTA was another related key term. A few publications outlined how a living HTA model could be implemented in real decision-making and its operational challenges.ConclusionsSo far, HTA bodies have been slow in adopting AI and automation innovation in their practices. Pressures to evolve with the increasingly complex treatment and evidence landscape necessitate a reform in HTA methods. A living HTA model may overcome these barriers and ensure faster patient access for new, promising technologies. A set of “living” standards is needed to gain HTA trust.
PD177 The Effect Of COVID-19 On Cancer Screening In Brazil, Canada, And The USA: A Cross-National Study
Authors: Kockaya Guvenc , Erkut Yaren , Okcun Selin , Ozdemir Ekin Begum , Kurnaz Mustafa , Tibet Birol 
Published: January 2025

IntroductionThe COVID-19 pandemic strained hospital systems and diverted resources, prompting a reorientation of healthcare priorities. This shift disrupted patient access to preventive cancer screenings and curtailed interactions between medical professionals and patients. This study aimed to examine changes in cancer screening during the COVID-19 pandemic period (2019 to 2021) in Brazil, Canada, and the USA.MethodsThe study included a literature review of academic articles, health reports, and government data that focused on the impact of the pandemic on cancer screening. Official health data in Brazil, Canada, and the USA were collected from medical records, national health databases, and screening statistics. A comparative analysis was conducted to unveil the changes in access to screening services for colorectal cancer (CRC), breast cancer, hepatocellular carcinoma (HCC), and cervical cancer.ResultsDuring the COVID-19 period, significant declines in cancer screening were observed globally. In Canada, CRC diagnoses dropped by 55 percent and remained 20 percent lower than averages from previous years, with an estimated 467 cases undiagnosed by August 2020. In the USA, HCC screenings were reduced by 44 percent, while cervical cancer screenings for women aged 21 to 29 years plummeted by 78 percent. Additionally, mammography screenings fell drastically from 180,724 in March to May 2019 to just 1,681 in the same period of 2020, leading to fewer breast cancers detected and a surge in symptomatic, aggressive tumors. Similarly, Brazil saw a 39 percent drop in breast cancer screenings.ConclusionsThe COVID-19 pandemic significantly disrupted cancer screening programs across Brazil, Canada, and the USA, resulting in marked declines in the number of diagnoses of various cancers. This reduction highlights the extensive impact of the pandemic on preventive health care, necessitating strategies to address the backlog and ensure timely cancer detection and treatment in the post-pandemic
era.
PD186 Treat-To-Target In Gout: Are Private General Practitioners In Malaysia Riding The Evidence-Based Wave?
Authors: binti Mohtar Siti Mariam , Ismail Maslinor , Anhar ‘Aziezah Norul , Mohamed Ghazali Izzuna Mudla 
Published: January 2025

IntroductionGout is an inflammatory disease that can cause severe pain and permanent joint destruction if it is not treated appropriately. Despite readily available treatments, the management of gout in primary care is suboptimal. Hence, this study aimed to investigate gout knowledge and compliance with evidence-based clinical practice guidelines (CPGs) among private general practitioners (GPs) in Malaysia.MethodsA cross-sectional study using stratified random sampling was conducted among private GPs in Selangor, Putrajaya, and Kuala Lumpur from October to December 2023. The survey consisted of four parts: inclusion and exclusion criteria, sociodemographic data, knowledge, and practice domain. A total of 15 questions were posed in the knowledge domain and nine questions in the practice domain. Statistical analysis was performed using R software (version 4.3.2). Descriptive statistics were used for initial analyses. Proportions were calculated for categorical data, which were tested using a chi-square test. A p-value of less than 0.05 was considered statistically significant.ResultsThe preliminary results from 203 private GPs showed that 64 percent had moderate knowledge of the management of gout. About two-thirds of the GPs were aware of local evidence-based CPGs on gout, but only 20 percent had good knowledge of gout. In the practice domain, only 65.5 percent of private GPs were aiming for a serum urate level of less than six milligrams per deciliter when starting urate-lowering therapy for gout.ConclusionsAlthough most private GPs had moderate or good knowledge of gout, clinical practice was not fully concordant with the recommendations of local evidence-based CPGs. Our findings provide a foundation for local CPG developers and professional societies to enhance their strategies for implementing evidence-based CPGs and encouraging GPs in Malaysia to incorporate evidence-based practices into their clinical decisions.
PD194 The Scenario Of Hospital-Based Health Technology Assessment In Brazil, Italy, And Poland
Authors: Di Bidino Rossella , Lipska Iga , Kukla Monika , de Almeida Cardoso Marilia Mastrocolla 
Published: January 2025

IntroductionHospital-based health technology assessment (HB-HTA) is evolving constantly. In 2023, a survey was conducted by the HB-HTA Interest Group of Health Technology Assessment International with the aim of collecting data on HB-HTA activities, including perceptions of the role of, potential for, and barriers to HB-HTA. Overall, 87 responses were collected: 41 from hospitals performing health technology assessment (HTA), 18 from hospitals not conducting HTA, and 28 from policymakers.MethodsThe survey collected data from 28 countries. Italy, Poland, Brazil provided the highest number of responses. We conducted a descriptive comparative analysis focusing on these three countries to investigate whether and how the policies for HB-HTA, the activities of HB-HTA, and the perceptions of HB-HTA differed among policymakers and hospitals not performing HTA.ResultsOverall, 19 responses were collected from Italy and 10 each were collected from Brazil and Poland. While Italy (n=10) and Brazil (n=8) had a high number of responses from hospitals performing HB-HTA, most of the responses from Poland (n=9) were from hospitals not performing HTA. There was a lack of policies for HB-HTA in all three countries. HTA was performed in big hospitals (≥500 beds) in Italy and Brazil. HB-HTA covered all kinds of technologies, including digital health. In Poland, hospitals not conducting HTA recognized the ability of HB-HTA to promote cost containment. Policymakers were open to including HB-HTA in their activities.ConclusionsInterpreting the diffusion of HB-HTA, its integration with other HTA processes, and the perceptions of its pros and cons should be both country specific and be conducted from an international perspective. The analysis demonstrated the need for specific policies and for better dissemination and promotion of HB-HTA activities and collaborations with HTA agencies.
PD197 Improving The Impact Of Health Technology Assessment Reports: Experience Of The Synthesis In Catalonia
Authors: Lleixà Berta Mestre , Vivanco-Hidalgo Rosa Maria 
Published: January 2025

IntroductionTo enhance the impact of health technology assessment (HTA) on Catalonia’s health system, a summary product called “synthesis” was developed. It presents the key information from HTA reports. After initial development and pilot testing, we developed a final version. Our aim was to evaluate its impact on decision-making at different levels.MethodsWe prepared an eight-question survey to assess the impact of the 15 syntheses that were published on the Agency for Health Quality and Assessment of Catalonia (AQuAS) website in 2023. We asked about the role of the healthcare professionals answering the questions, why they used the information of the syntheses, and whether they read the associated HTA report later and recommend it. We invited the professionals of the Catalan health system, providers, and payers to answer the survey by email. The survey also was published on the AQuAS website.ResultsWe obtained 31 responses. The roles of the professionals who answered the questions were mainly directors and managers of the institutions of the Catalan health system (45.2%), healthcare professionals (32.3%), and care managers (12.9%). Most of them consulted the synthesis to update their knowledge about a specific topic (71.0%), obtain reliable data (35.5%), or support a strategic or professional decision (29.0%). The HTA report related to each synthesis was only consulted in some cases (38.7%) but was recommended in almost all cases (93.5%).ConclusionsSummarizing the most important information of an HTA report in a new product can be a useful tool for improving the impact of the HTA on the Catalan health system. Professionals who answered the survey agreed that the syntheses helped them in making decisions related to the health technologies.