bluuebunny/crossref_metadata_2025_split huggingface dataset:
OD18 Surrogate Measures And The Health Technology Assessment Of Cancer Drugs In Ireland: A Retrospective Analysis, 2017 to 2022
Authors: Assi Alexandre , Karbanova Tereza , Algawas Lama , Darren Lim Yue Xiang , Trela-larsen Lea , Usher Cara , Barry Michael , Walsh Caroline 
Published: January 2025

IntroductionSurrogate endpoints are increasingly being used in the pivotal trials of cancer drugs to underpin (conditional) regulatory approval. We examined the relationship between the use of surrogate measures in pivotal trials underpinning cancer drug approvals by the European Medicines Agency (EMA) between 2017 and 2022 and health technology assessment (HTA) recommendations made by the National Centre for Pharmacoeconomics in Ireland (NCPE).MethodsA previously published methodology was used to identify cancer drug indications that received (conditional) marketing authorization between 2017 and 2022, inclusive. EMA-approved cancer drugs were categorized using the following benefit categories, based on pivotal trial endpoints: overall survival (OS), progression-free survival (PFS), disease response (DR), and single-arm trials (SATs). The NCPE website was searched to identify indications that had undergone, at least, a rapid review (RR) assessment. The NCPE recommendation for each assessment was recorded. Additional data including the incremental quality-adjusted life years (QALY) gain reported in cost-effectiveness analyses were extracted for indications that had undergone a full HTA.ResultsOne hundred and eight cancer drug indications were identified, comprising 68 cancer drugs. In 2017, OS, PFS, and SAT benefit underpinned equal proportions of approvals (28.6% each). In 2022, SAT underpinned the largest proportion of approvals (53.6%). As of June 2023, 77 indications (71.3%) had undergone at least a RR assessment; 31 indications had completed a full HTA appraisal. All of the indications underpinned by SAT evidence (n=7) received a conditional negative recommendation. Indications with SAT evidence had a mean incremental QALY gain of 1.88 (standard deviation [SD] 1.20), whereas indications with an OS benefit had a mean incremental QALY gain of 0.81 (SD 0.36).ConclusionsThe proportion of cancer drug indications receiving regulatory approval on the basis of SAT evidence, where no direct comparative evidence is available, is increasing. This results in additional uncertainty in the comparative benefit of cancer drugs supported by SAT evidence. The study is limited by the sample size of HTA appraisals included. Further in-depth analysis of factors influencing NCPE recommendations is needed.
OD32 Single Nucleotide Polymorphism Marker Analysis In Donor-Derived Cell-Free DNA For Solid Organ Transplantation (Next-Generation Sequencing): Systematic Review
Authors: Kim Yoojin , Im Jiae , Lee Wurlsook , Choi Jieun 
Published: January 2025

IntroductionSingle nucleotide polymorphism marker analysis in donor-derived cell-free (dd-cf
) DNA for solid organ transplantation is a technique for post-transplantation monitoring, including early detection of injury to the transplanted organ, signs of infection, and treatment decision-making, by measuring dd-cf-DNAs as a percentage of the total cf-DNAs in the patient’s body in donors and recipients of kidney, heart, lung, or liver transplants.MethodsThe assessments were performed via a systematic review. Searching five databases (KoreaMed, Ovid MEDLINE, Ovid Embase, and Cochrane) yielded 1,619 related studies. Two reviewers independently assessed the quality of these studies using a Scottish Intercollegiate Guidelines Network checklist, and the assessment results were described based on the results of the quality appraisal and level of evidence.ResultsThe population of the included studies was patients who underwent kidney, heart, lung, or liver transplantation. Medical outcomes in kidney transplantation patients were reported in 15 studies. The index test was reported to have an area under curve (AUC) of 0.68 to 0.99 and a sensitivity of 0.24 to 1.00. Four studies reported effectiveness data for the index test for lung transplantation patients. The diagnostic accuracy of the index test for acute cell-mediated rejection was reported have an AUC of 0.72. Sensitivity was reported as 0.44 and specificity as 0.80 for heart transplant patients, and sensitivity as 0.73 to 1.00 and specificity as 0.60 to 0.95 for liver transplant patients.ConclusionsThis is a safe and effective technique for post-transplantation monitoring, including the early detection of injury to the transplanted organ and signs of infection, and for treatment decision-making, by measuring dd-cf-DNA as a percentage of total cf-DNA in the patient’s body in recipients and donors of kidney, heart, lung, or liver transplantation (level of evidence: C).
OD39 Evaluation Of Sustainability And Environmental Impact Measures In Health Technology Assessment Of Medical Technologies In Europe: Starting An Important Journey
Authors: Orozco María Álvarez , Monje Julen , Hallas Natalie , Vogt-Humberg Ilona , Borghetti Francesca , Mauratille Constance , Strachan Liesl 
Published: January 2025

IntroductionReducing environmental impact and ensuring sustainable practices are important to the medical technology (MedTech) industry. However, it is unclear how these factors will be formally addressed by health technology assessment (HTA). The objective of this research was to understand what sustainability measures are required and included in HTA reports on medical technologies in five European countries (France, England, Germany, Italy, and Spain).MethodsHTA guidelines, framework papers, and published HTA reports for devices were searched from January 2022 to November 2023 for inclusion of environmental aspects. Search terms included environmental sustainability, carbon footprint, greenhouse gas, and waste generation. Data were extracted into standardized templates and analyzed both quantitively and qualitatively for the inclusion of sustainability and environmental impact measures.ResultsHTA guidance on inclusion of sustainability and environmental measures is lacking. While some countries request the inclusion of these aspects in the HTA process, there is little detail on evidence requirements and how it will be evaluated. Of the over 450 HTA reports examined, less than 10 percent included environmental aspects, with most sustainability benefit claims made related to the reduction in waste or less resources used. Very few claims were supported by published evidence. This resulted in the exclusion of potential environmental benefits in final HTA recommendations. Costs associated with environmental impact of medical technologies were rarely assessed.ConclusionsCurrent inclusion and evaluation of sustainability measures in MedTech HTA in Europe is nascent. While countries like England [National Institute for Health and Care Excellence (NICE)] and France [Haute Autorité de santé (HAS)] have very recently made public statements regarding the importance of environmental impact, there is a growing need for detailed guidance on environmental evidence requirements and how these will be incorporated into the evaluation and decision-making processes.
OD45 Evaluating Quality In Health Economics: Quality Appraisal Checklist For Systematic Reviews Of Studies Eliciting Health State Utility Values
Authors: Muchadeyi Muchandifunga T. , Hernandez-Villafuerte Karla , Di Tanna Gian Luca , Eckford Rachel , Feng Yan , Meregaglia Michela , Peasgood Tessa , Petrou Stavros , Ubels Jasper , Schlander Michael 
Published: January 2025

IntroductionThe reliability of cost–utility analyses depends on the quality of health state utility values (HSUVs). Given the increasing number of studies eliciting HSUVs, systematic reviews (SRs) are vital to economic evaluations. Nevertheless, a universally acceptable quality appraisal (QA) tool specific to the SRs of HSUV studies is lacking—this study aimed to develop one and fill this gap.MethodsWe employed a mixed-method approach, starting with a rapid review to identify QA dimensions, QA items, and terminology in the SRs of HSUV-eliciting studies. This informed a modified Delphi process with a seven-member international expert panel, aiming to define key terms, refine the QA tool dimensions, and establish relevant signaling questions. The experts participated in two anonymous online survey rounds interspersed with structured feedback, enabling iterative refinement of their views. Following these surveys, a virtual face-to-face meeting was held to resolve outstanding issues. Consensus was defined a priori at all stages of the modified Delphi process.Results
The rapid review identified three QA dimensions and 16 initial items, noting the diverse terminologies in defining QA. Response rates to the first- and second-round questionnaires and the virtual consensus meeting were 100, 86, and 71 percent, leading to a consensus on the definitions of scientific quality, QA, the three QA dimensions (reporting, methodological limitation, and risk of bias and relevance), and scope of the QA tool. The number of QA items was refined to 14: all relevant to reporting, six to relevance, and 11 to methodological limitations and bias risk dimensions. The QA tool underscores distinct evaluations for each dimension.Conclusions
We present the first version of a QA checklist designed to provide SR authors with a tool to appraise the quality of HSUV-eliciting studies comprehensively. The QA tool aims to (i) facilitate QA in SRs of HSUV elicitation studies, (ii) promote consistency in the appraisal process, and (iii) emphasize the importance of differentiating between reporting quality, methodology, and relevance.
PP03 Investigating Technological Strategies In The Hospital Setting: Insights From The Dutch Context
Authors: Pinelli Maria , Lettieri Emanuele , Tummers Marcia , Grutters Janneke 
Published: January 2025

IntroductionRapid advancements in technology are significantly impacting the healthcare system, and decision-making regarding technology adoption occurs at multiple decentralized levels within hospitals. National bodies seek to standardize this process, yet differing visions and strategies hinder centralization. This study explores the relationship between technological innovation and hospital strategies, focusing on scanning and assessment, aiming to assess the feasibility of centralized decision-making.Methods
To do this, we performed a qualitative analysis through 23 semistructured interviews in seven hospitals in the Netherlands, a country characterized by strong healthcare innovation and decentralization. We interviewed different actors involved in technological innovation, on different levels in the organization: CEOs, medical doctors, medical physicists or similar roles, and innovation managers. Ethics approval was obtained, and interviews were conducted, recorded, transcribed, and shared with participants for accuracy confirmation. Thematic analysis via grounded theory methodology and ATLAS.ti software generated insights on technological innovation’s relationship to hospitals’ strategies. Initial codes were refined into themes relevant to the research question.ResultsHospitals primarily aim to provide optimal patient care, with academic hospitals emphasizing research and education. Some hospitals aspire to be pioneers in adopting new technologies, while patient-centric healthcare is a shared goal. Technological strategies are not precisely designed in hospitals, being shaped by factors like people, financial constraints, or external environments. Hospitals’ scanning of technologies lacks systematization, and evaluations before and after technology adoption are not univocally performed. The need for systematic scanning and assessment practices is recognized by some interviewees, while others emphasize the importance of experimenting without the constraint of evaluation, perceiving it as a hurdle delaying innovation.ConclusionsCentralization could represent a benefit for hospitals, allowing them more streamlined decision-making, but it could also be perceived as a barrier. Involving hospitals’ stakeholders in centralization would be crucial to achieve it through a joint effort. Suggestions for future research could include focusing on a specific hospital, involving more stakeholders, and exploring other decentralized healthcare systems.
PP04 Assessing The Utility Of Natural Language Processing In Generating A Granular Estimated Indication For A Horizon Scanning Database
Authors: Choitir Caitríona Ní , Harte Marie , Duell Daisy , Odnoletkova Irina , Christensen Anna Bergkvist , Persson Marie , Bräuner Helle , Guardian Marcus , Tuckova Alzbeta Alzbeta , Wilkinson Brian , Kuzmak Beth , Adams Roisin , Shaw John , Boyce Craig , Palakollu Prashanth , Erinoff Eileen 
Published: January 2025

IntroductionDetailed, precise information on a pharmaceutical’s projected therapeutic use is required for horizon scanning. Inferring an estimated indication from trial protocols is a key skill of horizon scanners. The International Horizon Scanning Initiative (IHSI) database utilizes semi-automated data collection. This pilot aimed to verify that the extraction of relevant word sets to generate an estimated indication could be semi-automated.Methods
Ten drugs approved in Europe in 2021 were selected as the pilot test set. The test set included drugs approved for the treatment of rare diseases (n=4), haemato-oncology (n=3), and non-oncology conditions (n=3). Eight of the drugs were approved based on phase III trials. The assessment comprised a review of the pivotal trial that supported product registration for these drugs. We undertook a comparison between a human curator and a natural language processing (NLP) algorithm in generating granular tags relating to key aspects of the drugs’ estimated indication (stage of disease, patient-specific subgroup, and place in treatment).ResultsIn 50 percent of cases, the NLP accurately tagged a word or word set related to stage of disease, patient-specific subgroup, or place in treatment, which was also tagged by human curators. In 50 percent of cases, the NLP did not identify words or word sets tagged by human curators. Where relevant, the NLP successfully tagged the same word sets relating to stage of disease for all drugs in the test set. The same word sets relating to patient-specific subgroup were successfully tagged for three drugs in the set. NLP successfully tagged word sets relating to place in treatment for two drugs.ConclusionsThe NLP algorithm is successful in extracting relevant word sets, which can be used to generate an estimated indication in an automated or semi-automated process. The pilot highlighted that further testing is required to advance the sensitivity of the algorithm. Further piloting exploring both unsupervised and supervised modeling approaches (named entity recognition and deep neural networks, respectively) is planned.
PP12 Projecting The Potential Impact Of Disease-Modifying Therapies For Alzheimer’s Disease On The Carbon Emissions From Hospital Bed-Days in the UK
Authors: Trepel Dominic , Edwards Sophie , Ritchie Craig , Hahn-Pedersen Julie Hviid , Kettle Jamie , Chan Mei Sum , Bray Benjamin D , Clark Alice , Ivkovic Milana , Wichmann Christian Ahmad , Evans Marc 
Published: January 2025

IntroductionDisease-modifying therapies (DMTs) for Alzheimer’s disease (AD) are emerging treatment options. This study aimed to estimate the potential health system and associated environmental impacts of DMTs by modeling future bed-days and carbon dioxide equivalent (CO2e) emissions for the UK population under various scenarios for access to and efficacy of DMTs.MethodsA cohort Markov model was developed to predict the UK population distribution from 2020 to 2040 across five health states—cognitively unimpaired and four stages of AD (mild cognitive impairment, and mild, moderate, severe dementia). These distributions were estimated using national population projections, AD prevalence data, and stage-specific transition rates. Annual bed-days per person for each state and associated CO2e emissions from published literature were applied to estimate total bed-days and emissions. Modeled scenarios combined ranges of DMT efficacy estimates (20 to 30%) and access levels (25 to 58% eligible patients receiving treatment) elicited from expert opinion to explore the extent of potential DMT impacts.ResultsWithout DMT access, annual bed-days across the four AD stages were projected to increase from 5.5 million to 8.6 million from 2020 to 2040, with cumulative bed-days totaling 140 million. Associated annual emissions increased from 0.7 Mt to 1.1 Mt CO2e, reaching 17 Mt CO2e cumulatively from 2020 to 2040. Under the various high-access (58% eligible patients treated) DMT efficacy scenarios, relative to no DMT access, annual reductions of 430 thousand to 650 thousand bed-days and 54 kt to 81 kt CO2e were estimated by 2040, and cumulative emissions decreased by 419 kt to 633 kt CO2e. Decreasing DMT access to 25 percent, assuming 25 percent DMT efficacy, reduced annual bed-days by 230 thousand by 2040, and annual emission savings decreased to 29 kt CO2e.ConclusionsDMTs for AD may contribute to efforts by healthcare systems to reduce the carbon emissions from hospital inpatient care. Environmental sustainability should be considered as part of a holistic value proposition when assessing the benefits of new medicines.
PP13 Cost-Effectiveness Analysis Of Antiandrogen Therapies For Metastatic Castration-Sensitive Prostate Cancer: The Brazilian Healthcare System Perspective
Authors: Gargano Ludmila , Oliveira Layssa , De Oliveira Haliton Alves , Lucchetta Rosa 
Published: January 2025

IntroductionBrazilian Unified Health System (SUS) Clinical Practice Guidelines (CPG) for prostate cancer recommend the use of androgen deprivation therapy (ADT) ± docetaxel for metastatic castration-sensitive patients (mCSPC). However, new medications have been developed since the last CPG update. We assessed the cost-effectiveness of abiraterone, apalutamide, darolutamide, and enzalutamide for mCSPC as part of the CPG update process.Methods
A cost–utility analysis was built, with partitioned survival model composed of three states: progression-free, evidence of radiological progression, and death. The overall survival (OS) and progression-free survival (PFS) curves from docetaxel STAMPEDE trial were extrapolated using different distributions for a 20-year horizon. Visual inspection, clinical plausibility, and Akaike information criterion (AIC)/Bayesian information criterion (BIC) tests were considered to choose the best fit, which were adjusted by hazard ratios (HR) from indirect comparisons of interventions with docetaxel. Utility and disutility values were identified from the literature. Direct costs of medications and pre- and post-progression disease monitoring were considered. We considered a BRL120,000/quality-adjusted life year (QALY) (i.e., USD24,511) cost-effectiveness threshold.ResultsWhen compared to docetaxel+ADT, interventions with higher effectiveness gains were abiraterone+docetaxel and darolutamide+docetaxel (0.94 and 0.99 QALY, respectively). Apalutamide and enzalutamide showed the highest incremental cost (BLR637,342 and BLR516,313, [i.e., USD130,187 and USD105,465], respectively). Abiraterone monotherapy or +docetaxel presented the lowest incremental cost–utility ratio (ICUR) (BLR84,960 and BLR79,428/QALY gained [i.e., USD17,354 and USD16,224], respectively) and may be cost effective for the SUS. Apalutamide, enzalutamide, and darolutamide may not be cost effective for the SUS as ICUR were higher than the cost-effectiveness threshold. Probabilistic sensitivity analyses or using different distributions for survival curves confirmed the direction of the findings.ConclusionsRecent studies have demonstrated that adding docetaxel or antiandrogen drugs to ADT in men with mCSPC can improve OS and PFS compared with ADT alone. However, in this analysis, only abiraterone was cost effective, and the incorporation of apalutamide, darolutamide, and enzalutamide would require substantial price reductions to be cost effective for the SUS.
PP15 Cost–Benefit Analysis Of An Antimicrobial Stewardship Program In A Cancer Setting In Qatar
Authors: Abushanab Dina , Moustafa Diala Alhaj , Sankar Bhagyasree Adampally , Nasr Ziad , Al Hail Moza , Hamad Anas , Abdul Rouf Palli Valappila , Elkassem Wessam , Thomas Binny , Al-Badriyeh Daoud 
Published: January 2025

IntroductionThe inappropriate use of antimicrobial agents has been associated with increased healthcare costs and the spread of multidrug-resistant organisms. We aimed to estimate the economic impact of the developed antimicrobial stewardship program (ASP), after five years of implementation, versus the preliminary ASP, upon implementation, in the cancer setting at the National Center for Cancer Care and Research (NCCCR) in Qatar.MethodsThe research investigated the economic benefits of employing a preliminary ASP versus a developed ASP from the perspective of a public healthcare hospital. Preliminary ASP was defined as the 12 months following the establishment of the ASP (i.e., 1 May 2015 to 30 April 2016), while developed ASP was defined as the most recent 12 months of ASP implementation (i.e., 1 February 2019 to 31 January 2020). Patient records were retrospectively reviewed. The total economic benefit of ASP maturity was calculated as the sum of the cost savings and the cost avoidance associated with the service, minus the operational cost.ResultsA total of 1,000 patients were included in the study. The developed ASP was associated with substantial reduction in antimicrobial consumption and resource utilization. Total cost of resources to avoid during the developed ASP period was USD1,634,658, in contrast to USD4,923,024 during the preliminary ASP period, yielding a positive cost avoidance of USD3,288,366. Developed ASP incurred lower operating costs than preliminary ASP, resulting in positive change in operational cost of USD3,428. The benefit-to-cost ratio was 640. The net benefit due to ASP maturity was USD3,624,875. The robustness of the results is demonstrated by the sensitivity analysis.ConclusionsThis study underscores the benefits of ASP development in a cancer setting. The observed reductions in antimicrobial costs from reduced antimicrobial use and the added value of cost avoidance signify the positive impact of a well-developed ASP. These findings lend support to the broader implementation of comprehensive ASPs, which contribute not only to patient-care optimization but also to cost containment.