bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP17 Methods To Measure The Environmental Impacts Of Health Technologies And Include Them In Economic Evaluations: A Scoping Review
Authors: Ardito Vittoria , Banks Helen , Cusumano Niccolò , Tarricone Rosanna 
Published: January 2025

IntroductionThe environmental footprint of the healthcare sector is widely acknowledged and increasingly impacting decision-making in health policy. This study aims to explore what methods and metrics have been proposed to measure environmental impact of health technologies, and to investigate the feasibility and consequences of its inclusion in economic evaluations. This study is part of a larger Horizon Europe project, HI-PRIX.MethodsWe conducted a PRISMA-ScR-guided scoping review of the scientific publications in PubMed, Web of Science, and Scopus between 2013 and November 2023. For the grey literature, the international HTA database and HTA agencies websites were searched to identify ongoing or published HTA dossiers. The search strategy was developed around two concepts: environment and health technology assessment. Any study or report that described a conceptual framework, methodology, or approach used or theorized to integrate environmental impacts of health technologies in an HTA was considered eligible for inclusion.ResultsStarting from 12,336 records, 16 scientific publications and 6 HTA dossiers were included for analysis. One-third of the contributions were published in 2023 and were mostly opinions, editorials, reviews, or economic evaluations. Lifecycle assessment was considered the preferred approach to investigate environmental impacts, yet difficulties exist; for example, methodologies for measuring greenhouse gas emissions are established, while for other dimensions are under development. Nevertheless, methods to incorporate environmental impact into economic models appear viable. About half of the papers analyzed cost–utility analysis, multiple-criteria decision analysis, and cost–benefit analysis as potential approaches. Fewer papers addressed budget impact, cost-effectiveness analysis, or other approaches.ConclusionsIncorporating the environmental impact of health technologies in economic evaluations is under development, but consensus is still lacking on appropriate, feasible methodologies for its uptake. Before considering it in pricing or reimbursement-related decisions, the preferred method to maximize utility levels of different stakeholders (e.g., HTA agencies, industry) needs clarification.
OP29 Determinants Of The Financial Impact Of Rare Disease Drugs In Italy: Differences Between Expected And Observed Pharmaceutical Expenditure
Authors: Mennini Francesco Saverio , Cicchetti Americo , Sciattella Paolo , Rumi Filippo , Zanuzzi Matteo , Carletto Angelica , Sammarco Annalisa , Romano Federica , Russo Pierluigi 
Published: January 2025

IntroductionIn Italy, a fixed proportion of health spending is allocated to pharmaceutical expenditure. While the main objective of setting a budget for pharmaceuticals is to control spending, the effectiveness of this ceiling is questionable. This study aims to investigate the determinants of pharmaceutical expenditure for orphan drugs and gather information for effective planning and programming of pharmaceutical spending.Methods
Data analysis relied on pharmaceutical companies’ pricing and reimbursement (P&R) dossiers submitted to the Italian Medicines Agency (AIFA) for drug-reimbursement approval, along with AIFA’s internal procedural documents. The study encompassed all rare disease drugs reimbursed from January 2013 to January 2019. For each drug, a comparison was made between the expected post-negotiation expenditure and the actual spending observed over the three years following reimbursement approval. Potential determinants of the normalized ratio between observed and expected spending were identified using univariate and multivariate beta regression models. The same methodology was replicated to identify potential determinants of the difference between expected spending before and after negotiation.Results
Fifty-two rare disease drugs admitted for reimbursement during the study period were analyzed. The median expenditure in the first three commercialization years was 7.6 percent lower than the expected post-negotiation spending. Beta regression analysis indicated a significantly lower reduction for innovative drugs (β 0.736, p-value 0.011 univariate, β 0.585, p-value 0.045 multivariate). Similar effects were observed for P&R procedures (β 0.902, p-value 0.007) and the number of indications presented (β 0.754, p-value 0.021), but only in univariate model. Beta regression analysis for the expected expenditure ratio before/after negotiation revealed a significant effect only for the payment-by-result variable (β 1.485, p-value 0.001).ConclusionsObserved expenditure for orphan drugs aligns with the expected spending post-negotiation. However, in the subgroup of innovative orphan drugs, the observed pharmaceutical spending was higher than estimated. This could be attributed to prescriber preferences and to a prevalent patient pool awaiting innovative treatment. It appears that the recognition of innovativeness favors orphan drugs that are rewarded with faster market access.
OP31 Monitoring Of The Budget Impact Determinants Of Incorporated Technologies For Rare Diseases In The Brazilian Health System
Authors: Pereira Silva Mariá Gonçalves , Lyrio Amanda Oliveira , Neiva Pantuzza Laís Lessa , Borges Thais Conceição , Ottavio Barbara Pozzi , de Freitas Lopes Ana Carolina , Bonan Luciene 
Published: January 2025

IntroductionBudget impact analyses for the treatment of rare diseases are especially important for the sustainability of health systems due to high treatment costs and uncertainties in target population estimates. The objective of this work is to analyze the elements that influence discrepancies between predicted and observed budget impacts for enzyme replacement therapies for rare diseases in Brazil’s public health system.MethodsAll enzyme replacement therapies for rare diseases evaluated by the National Committee for Health Technology Incorporation in the Brazilian Public Health System (Conitec) and with at least one year of use were included. For each technology, the following were identified: number of patients, median patient weight, annual quantity of medication, unit price, and budget impact. The attributes were compared between previous estimates and real-world observation after use. The data sources were publicly accessible administrative databases and Conitec technical reports.ResultsFive technologies were selected: elosulfase alfa, alglucosidase alfa, idursulfase, laronidase, and galsulfase. In the first year, the difference between the estimated and the observed number of patients treated was up to 15 percent lower or higher for four technologies, but with monthly fluctuation throughout the year. The median weight of users was between 23 percent and 468 percent higher for three technologies. The observed price was as expected, with variations between three percent lower and 14 percent higher. The quantity of medicines used was lower (between 39% and 46%) than expected for all technologies. The observed budget impact was 37 percent to 47 percent lower than estimated.ConclusionsReal-world budget impact was lower than expected for all technologies. The main cause of discrepancies was the estimate of the annual amount of medication, which did not consider gradual adherence and discontinuation of treatment. This highlights the need to review the budget impact methodology for rare diseases, forecasting monthly market share and treatment discontinuation rate.
OP42 A Budget Impact Analysis Of The Introduction Of Noninvasive Prenatal Testing To Korean National Health Insurance
Authors: Seo Hyerin , Ahn Jeonghoon , Jin Doyeon , Lee Hyorim 
Published: January 2025

IntroductionThis study assessed the budget impact (BI) of introducing noninvasive perinatal testing (NIPT) to the Korean National Health Insurance (KNHI). It aims to provide useful economic evidence on reimbursement decisions on prenatal screening. Since NIPT is widely used in Korea as a noncovered service, BI can be reliably estimated from real-world data (RWD).MethodsIn this BI analysis, we derived the total number of pregnant women from 2017 birth statistics (n=358,000) and its distribution of singleton births (96.1%) and multiples (3.9%). The age distribution of pregnant women was 29.4 percent for 35 and above, and 70.6 percent for below 35, according to the 2017 statistics. For NIPT costs, we considered a price range of KRW357,000 (USD260) to KRW715,000 (USD522) from a 2018 survey. To evaluate the BI of introducing NIPT, we examined eight scenarios based on NIPT cost sharing of 30 percent and 70 percent, NIPT price, and whether to include pregnant women aged less than 35 years old.Results
BI for KNHI was estimated as KRW6 trillion (USD4 billion) to approximately KRW56 trillion (USD40 billion). Scenario seven, targeting older pregnant women with serum screening high risk with NIPT price at the lower end and payer coverage of 30 percent, shows the lowest annual BI of KRW6,115,813,866 (USD4,469,516); scenario two, covering all older pregnant women and younger high-risk cases with NIPT price at the higher end and payer coverage of 70 percent, exhibits the highest burden at KRW56,240,636,611 (USD41,091,993).ConclusionsOur BI analysis of introducing NIPT to KNHI can serve as essential data for estimating insurer burdens during the potential transition of NIPT to provisional or selective coverage. In the absence of prenatal diagnostics cost-effectiveness research in Korea, our findings provide crucial evidence for establishing relevant reimbursement criteria, addressing a gap in current research and supporting evidence-based policymaking.
OP57 Comparing Institute For Clinical And Economic Review Comparative Effectiveness Assessments And Federal Joint Committee Added Benefit Assessments
Authors: DiStefano Michael J. , Pearson Steven D. , Rind David M. , Zemplenyi Antal 
Published: January 2025

IntroductionWe compared the Institute for Clinical and Economic Review’s (ICER) ratings of comparative clinical effectiveness with the German Federal Joint Committee’s (G-BA) added benefit ratings, and explored what factors, including the evidence base, may explain disagreement between the two organizations.MethodsDrugs were included if they were assessed by ICER under its 2020–2023 Value Assessment Framework and had a corresponding assessment by G-BA as of March 2023 for the same indication, patient population, and comparator drug. To compare assessments, we modified ICER’s proposed crosswalk between G-BA and ICER benefit ratings to account for G-BA’s extent and certainty ratings. We also determined whether each assessment pair was based on similar or dissimilar evidence. Assessment pairs exhibiting disagreement based on the modified crosswalk despite a similar evidence base were qualitatively analyzed to identify reasons for disagreement.ResultsWe identified 15 assessment pairs and seven out of fifteen were based on similar evidence. G-BA and ICER assessments disagreed for each of these drugs. For 4/7 drugs, G-BA (but not ICER) determined the evidence was unsuitable for assessment: for 2/4 drugs, G-BA concluded the key trials did not appropriately assess the comparator therapy; for 1/4, G-BA did not accept results of a before-and-after study due to non-comparable study settings; for 1/4, G-BA determined follow-up in the key trial was too short. Among assessment pairs where both organizations assessed the evidence, reasons for disagreement included concerns about long-term safety, generalizability, and study design.Conclusions
This study underscores the role of value judgments within assessments of clinical effectiveness. These judgments are not always transparently presented in assessment summaries. The lack of clarity regarding these value-based decisions underscores the need for improvements in transparency and communication, which are essential for promoting a more robust health technology assessment process and supporting transferability of assessments across jurisdictions.
OP58 Diagnostics And Treatments For COVID-19: Update From A Living Systematic Review Of Economic Evaluations
Authors: Hopkin Gareth , Elvidge Jamie , Narayanan Nithin , Nicholls David , Dawoud Dalia 
Published: January 2025

IntroductionThe COVID-19 pandemic put substantial strain on healthcare systems globally. Early decision-making about diagnostic tests and treatments was driven by the need for rapid responses with a focus on reducing clinical burden. As COVID-19 continues its transition into an endemic state, health technology assessment (HTA) agencies will need to consider the clinical- and cost-effectiveness of tests and treatments, as with other conditions.MethodsWe first conducted a systematic literature review in July 2021 and updated the search in July 2023. The review aimed to identify economic evaluations of diagnostics for SARS-CoV-2 and treatments for COVID-19 using predefined search strategy across journal databases and sources of grey literature. In the update, an additional targeted search was completed with terms relating to novel treatments. Search results were screened by title and abstract, and full texts of potentially relevant studies were reviewed against selection criteria. Studies with very serious methodological limitations were excluded. Findings from studies were synthesized narratively due to high levels of heterogeneity.Results
The database search identified 8,287 unique records, of which 54 full texts were reviewed, 28 were quality assessed, and 15 were included. Three further studies were included through HTA sources and citation checking. Of the 18 studies ultimately included, 16 evaluated pharmacological treatments including corticosteroids, antivirals, and immunotherapies. Two studies in lower-income settings evaluated the cost-effectiveness of rapid antigen tests and critical care provision. In most studies, a healthcare or payer perspective was used, and the comparator was standard care. There were 17 modeling analyses and one trial-based evaluation. Cost–utility analyses using QALYs were the most common analysis type.ConclusionsThis update indicates that there are cost-effective treatments for COVID-19, with repurposed pharmacological treatments like dexamethasone presenting best value. There also appear to be promising options for people with severe disease alongside standard care. Future economic evaluations would benefit from reflecting the changing context around COVID-19 with parameters that reflect current circumstances, and fully incremental analyses comparing different treatment options.
OP69 Are Artificial-Intelligence-Based Literature Reviews Accepted By Health Technology Assessment Bodies?
Authors: Singh-Mangat Gautamjeet , Sharma Sugandh , Bergemann Rito 
Published: January 2025

IntroductionLiterature reviews (LR) play a crucial role in all health technology assessment (HTA) dossiers, presenting evidence-based value of interventions. There is global exploration of artificial intelligence (AI) to expedite and enhance the efficiency of literature reviews. Our research aimed to identify any existing guidance from HTA bodies regarding the use of AI for conducting literature reviews.MethodsWe conducted a comprehensive search and review of any published guidance from prominent HTA bodies, including the National Institute for Health and Care Excellence (NICE, England), Scottish Medicines Consortium (SMC, Scotland), National Centre for Pharmacoeconomics (NCPE, Ireland), National Authority for Health (HAS, France), Federal Joint Committee (G-BA, Germany), Institute for Quality and Efficiency in Health Care (IQWiG, Germany), Canadian Agency for Drugs and Technologies in Health (CADTH, Canada), and Pharmaceutical Benefits Advisory Committee (PBAC, Australia). This was done to gain insights into their views regarding the utilization of AI in literature reviews. Additionally, we engaged with HTA representatives, such as NICE, to gain a deeper understanding of their perspectives.ResultsWe found a lack of clear guidance on the use of AI for conducting LRs. NICE has recommended a priority screening technique using machine learning (ML) for identification of a higher proportion of relevant papers at an earlier stage. NICE is currently in the process of developing guidance and is updating its manual in this area. SMC refers readers to NICE methodologies. In its HRB-CICER report, NCPE only acknowledges the potential of ML algorithms for LRs, with no additional information. IQWiG, in its general methods, recommends the use of ML-validated classifiers for identifying randomized controlled trials (RCTs) within bibliographic searches.ConclusionsOur research indicates that there is scarce guidance available for the use of AI in LRs for HTA submissions. However, considering the rapidly evolving nature of this field, it is anticipated that guidance documents and manuals will be updated in the near future.
OD19 Future Health And Economic Burden Of Cardiovascular Disease In Type 2 Diabetes In Qatar, From 2023 To 2032
Authors: Abushanab Dina , Al-Badriyeh Daoud , Marquina Clara , Liew Danny , Al-Zaidan Manal , Al-Kuwari Mohammed Ghaith , Abdulmajeed Jazeel , Ademi Zanfina 
Published: January 2025

IntroductionThe Middle East and North Africa region accounts for the largest prevalence of type 2 diabetes (T2D). Qatar ranks top 10 for global T2D prevalence, with 17 percent in 2022. Also, 50 to 70 percent of the cardiovascular events occurred in T2D. We sought to estimate the future health and economic burden of cardiovascular disease (CVD) in T2D from 2023 to 2032.MethodsA dynamic multistate model in people with T2D was constructed. The demographic profile of the population was based on Qatari citizens and residents with T2D aged 40 to 90 years in 2022. First CVD events (i.e., myocardial infarction [MI] and stroke) were calculated via the 2013 Pooled Cohort Equation using data from Primary Health Care Corporation. Recurrent CVD events were sourced from the global Reduction of Atherothrombosis for Continued Health (REACH) registry. Outcomes were MI and stroke, years of life lived, quality-adjusted life years (QALYs), total direct and productivity loss costs. Utility and cost model inputs were drawn from published sources. The model adopted a Qatari societal perspective.ResultsThe model estimates 123,524 non-fatal MIs (95% uncertainty interval [UI]: 116,923, 130,065), 70,466 non-fatal strokes (95% UI: 67,945, 73,476) and 15,410 CVD deaths (95% UI: 15,217, 15,794), respectively. T2D population accrued 4,834,146 (95% UI: 4,781,235, 4,881,695) total years of life lived and 3,817,246 (95% UI: 3,756,963, 3,870,616) total QALYs. Direct costs accounted for 59.52 percent of the total costs, with a projection of QAR43.59 billion (USD11.98 billion) (95% UI: QAR9.14 billion [USD2.5 billion], QAR134.20 billion [USD36.87 billion]), while the total indirect costs were expected to exceed QAR29.65 billion (USD8.14 billion) (95% UI: QAR2.40 billion [USD659.34 million], QAR113 billion [USD31.04 billion]).ConclusionsThis study highlights that the considerable rising burden of CVD in T2D in Qatar will impact not only the healthcare system but also the society overall. The findings may be used to prioritize strategies targeting T2D to prevent CVD burden.
OD23 Evolving Prostate Cancer Screening Strategies In Germany: A Cost–Utility Analysis Comparing Traditional And Emerging Modalities
Authors: Muchadeyi Muchandifunga T. , Hao Shuang , Hernandez-Villafuerte Karla , Khan Shah , Becker Nikolaus , Krilaviciute Agne , Seibold Petra , Gulati Roman , Albers Peter , Schlander Michael , Clements Mark 
Published: January 2025

IntroductionIn 2021, approximately 15,000 men in Germany died from prostate cancer (PCa). The national health policy is considering shifting from annual digital rectal examination (DRE)-based screening to an age-related prostate-specific antigen (PSA)-based risk-adapted and organized screening strategy. Our research investigated the cost–utility of the current DRE-based strategy versus organized age-related PSA-based risk-adaptive PCa screening strategies in Germany.MethodsWe adapted the Swedish Prostata model to the German context, recalibrating it with PCa clinical and epidemiological data from the national and state registries. The model includes preclinical and clinical disease health states defined by tumor, nodal, and metastatic stages and Gleason scores, and assumes that the benefits of screening arise from stage shift. We assessed the cost–utility of 14 strategies, ranging from no screening to DRE, and age-related, PSA-based, risk-adapted screening. Health state utility values and test characteristics were sourced from the literature. Inpatient and outpatient care costs were derived from the German diagnostic-related groups and uniform-based valuation systems.ResultsAmong all strategies evaluated and compared with no screening, the “DRE only” strategy led to substantial overdiagnosis, the highest incremental cost, and minimal quality-adjusted life years (QALY) gains. PSA testing starting at 50 to 60 years with reflex MRI for PSA greater than 3 ng/mL cases followed by combined systemic and targeted biopsy reduced the number of biopsies and overdiagnosis by 75 percent and 26 percent, albeit for fewer QALYs and higher costs (dominated) than the same strategy without reflex MRI. The PSA-based risk-adaptive strategy, starting at 50 to 60 years without reflex MRI, demonstrated an 85 percent probability of being cost effective within the EUR30,000 (USD32,211) to EUR100,000 (USD107,369)/QALY willingness-to-pay range.ConclusionsWhile Germany’s HTA emphasizes clinically added benefits and health-related quality of life, cost-effectiveness analysis substantiates this evidence. As a standalone early detection tool, DRE leads to substantial overdiagnosis, unnecessary biopsies, and increased healthcare costs. Overall, this study demonstrated the importance of age-related PSA risk-adaptive PCa screening. The value of MRI deserves further investigation, considering MRI’s positive effect on screening acceptability.