bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP123 Fair Drug Pricing: Review Of Literature And Models
Authors: Wagner Olena , Hurst Samia 
Published: December 2022

IntroductionConstantly rising costs of pharmaceuticals and biologics spurred a debate in recent years leading to increasingly persistent public calls to overhaul the existing system of pricing and distributing health technologies. The COVID-19 pandemic exposed the controversies of the current model of access to health commodities in all evidence when the existing discrepancy between the global supply and the global demand in health technology can be viewed as an illustration of a conflict between the neoliberal free market ideal of health innovation genesis and ownership and the original democratic principles of what would now be called sustainable human development.MethodsPresented here is an integrative literature review of over 265 publications in peer-reviewed journals on Pubmed and Web of Science, academic and “grey” literature, mainly publicism, published in English. We reviewed and analyzed included literature for the purposes of identifying leading ideas with regards to ethical frameworks for evaluating or referencing value for pricing health commodities.ResultsSeven drug pricing models were analyzed in terms of them satisfying the three most common criteria of price adequacy – its fairness (viewed from the point of view of main schools of ethical thought), accountability-for-reasonableness (including transparency of decisions, relevance, existing mechanisms of revisions and governance to ensure compliance), and price functionality. One of the central ideas under controversy is value, its relative character for different contexts due to the high weight of the willingness-to-pay in the value-based health technology assessment (HTA) decisions and the relative value of money, and the attempts to quantify value in a universal way for institutions, patients, and originators.ConclusionsWhile the review scored the pricing models on their “public fairness” with volume-based rather than value-based pricing models leading the rating, the main conclusion of the review is that the main meaningful divide is between value creation and value extraction when pricing health innovation.
PP132 Disinvestment Initiatives In Health Care: A Scoping Review Of Systematic Reviews
Authors: Kamaruzaman Hanin , Grieve Eleanor , Wu Olivia 
Published: December 2022

IntroductionDisinvestment of ineffective, low value technologies is growing as a priority for international health policy in order to improve quality and maximize value in health care. Different strategies have been implemented at the international and national level using various methods of evidence gathering and technical assessment. However, the success of these initiatives is mixed, with fewer than half of the empirical studies reporting reductions in the use of low value services.This review explored the role of stakeholders in the disinvestment process by describing the initiatives and analyzing the methods used for reassessment. We also identified the facilitators and barriers related to disinvestment implementation.MethodsThis scoping review was guided by the JBI Manual for Evidence Synthesis and the PRISMA statement for scoping reviews. Strategic literature searches were performed to identify published reviews on disinvestment in health care using the MEDLINE, Web of Science, and Scopus databases. Data were extracted using a predesigned form and then synthesized narratively to identify similarities and differences across the approaches according to prespecified domains.ResultsSixteen reviews were included. We identified various disinvestment initiatives across 16 countries, with a minimum of 34 initiatives at different levels of implementation and with various agencies responsible for the activities. Two of the most used methods for facilitating disinvestment decisions were program budgeting and marginal analysis (PBMA) and health technology assessment (HTA). Stakeholder involvement was the most important aspect to be addressed since it acts as both a facilitator and a barrier in implementing disinvestment initiatives. Meaningful engagement may be strengthened with continuous stakeholder participation, transparency in methods and processes, and ongoing knowledge transfer.ConclusionsThis scoping review highlights the role of stakeholder involvement in disinvestment, which is a double-edged sword because it both facilitates and hinders disinvestment implementation. The most common methods for assessing candidates for disinvestment are PBMA and HTA, but there is a lack of clarity on which HTA dimension is suitable for a disinvestment process.
PP134 Health Technology Assessment Life Cycle Approach In Asthma Care
Authors: Have Pieter ten , Van Kemenade Geert-Jan 
Published: December 2022

IntroductionWithin Dutch health care, Zorginstituut Nederland regularly selects topics for reassessment of diagnostic and therapeutic interventions. Until now, about 15 topics have been investigated, such as hip/knee arthrosis, stable angina, low back pain, urinary incontinence, palliative care, retinopathy and asthma.MethodsFor each topic, stakeholders were asked to report what could be improved. Also a working group was created to analyze: what do guidelines recommend, what does actual care look like seen from administrative data, is there a gap between guidelines and delivered care, what can be improved? A report with recommendations was written with a calculation of the impact. Agreements were made on implementation. This report focused on findings relating to asthma.ResultsWe identified the multiple issues in the management of asthma: (i) Only 11 percent of patients had their diagnosis confirmed (with spirometry and reversibility or visit to pulmonologist). (ii) 60,000 patients had intensive short acting broncho-adrenegic agents (SABA) without inhaled corticosteroids (ICS) representing overuse of SABA. (iii) 200,000 patients use inhalers that can be empty, without the patient knowing this, as there is no indicator showing the number of actuation/puffs left, which leads to under treatment. (iv) 60.000 patients have aerosol and powder inhalers together, each requiring a different technique, leading to mistakes. (v) 67 percent of asthma adults get a chest x-ray on referral to hospital, which is a high proportion. (vi) 49 percent of patients use inhalers with propellant, which is needlessly high, given their undesirable impact on climate change. This causes 36 million kg co2 equivalent, the same as 36000 extra gasoline cars. (vii) Only 37 percent of patients receive yearly monitoring. We calculated that EUR 14 million annually can be saved as a result of better diagnosis leading to less overdiagnosis and overtreatment and less spacers.ConclusionsDespite that ‘HTA (Health Technology Assessment) at the doorstep’ has been applied for asthma interventions, we noticed considerable room for improvement. We consider this method important for real HTA life cycle approach.
PP147 Conditional Reimbursement Of Medicinal Products, A Procedure For Orphan Drugs, Conditionals and Exceptionals
Authors: van Heesch Floor , De Vries Marijke 
Published: December 2022

IntroductionIn 2018 the Dutch Ministry of Health (MoH) introduced a new policy regarding the conditional reimbursement (CR) of drugs in the basic health insurance package. This new policy offers patients with a serious, often rare disease for which no effective treatment is yet available, the possibility of obtaining controlled access to new promising drugs. In the meantime, additional data on (cost-) effectiveness is being collected. The aim was to assess whether this new policy allowed improved inclusion of drugs in the basic health insurance package.MethodsMarketing authorization holders (MAH) were able to apply for the CR. The drug had to be registered by the European Medicines Agency (EMA) as an orphan drug, conditional or exceptional and address an unmet medical need. The MAH had to submit a dossier which includes a study protocol together with the professionals’ associations, patients’ associations and a research institute. It was possible to engage an ongoing (international) study in the CR application. Based on the proposed study, the National Health Care Institute (ZIN) assessed whether it is possible to determine if the drug should be reimbursed at the end of the CR period. A reduced price was a condition for CR.ResultsFour drugs are currently reimbursed as part of the CR, being: parathyroid hormone, ataluren, larotrectinib and entrectinib. The proposed studies are ongoing and will generate data to support the final reimbursement decision. Progress will be monitored by the researchers and discussed with ZIN.ConclusionsFour drugs were successfully conditionally reimbursed, concluding the new CR procedure is feasible. Additional data is being collected to aid in the decision on the definitive reimbursement of these drugs. The upcoming period, the focus will be on the quality of the collected data and whether the inclusion of patients is proceeding as planned. The MoH will be informed by ZIN on the study progress annually. The final reimbursement decision is taken at the end of the CR period.
PP148 The Impact Of Health Technology Wales Guidance For Autologous Hematopoietic Stem Cell Transplantation: Two Years Post-Publication
Authors: Elston Lauren , Hughes Sophie , Glarou Eleni , Myles Susan 
Published: December 2022

IntroductionEvaluating the impact of health technology assessment (HTA) is vital to measure its contribution to health and social care decision-making and improving citizen outcomes. Health Technology Wales (HTW) is a HTA body committed to evaluating the impact of our work. Here we present HTW’s impact evaluation approach with a case study for autologous hematopoietic stem cell transplantation (AHSCT) for highly active relapsing remitting multiple sclerosis (RRMS).MethodsUsing an outcomes-focused approach based on contribution analysis, HTW has worked with an external evaluation organization to develop a framework to measure the impact of our work. Data on impact was collected from both qualitative and quantitative sources, including social media metrics, surveys, and informal feedback from stakeholders. We engaged with various stakeholders, including clinicians, academics, patient organizations and other HTA bodies.ResultsThe technology appraisal and guidance were published in July 2020, recommending AHSCT for routine adoption to treat highly active RRMS. Patient groups welcomed the appraisal findings as an important step forward in recognising the needs of people with RRMS and felt that “people living with MS were listened to throughout the process”. Following publication online, the guidance has had approximately 500 views, and featured on the MS Trust website and in several news articles. The Welsh Health Specialist Services Committee, a commissioning body in Wales, recommended AHSCT for RRMS as a ‘high priority’ for funding in the WHSSC Integrated Commissioning Plan 2021-22.ConclusionsSince its publication, we have been able to prospectively capture the impact of this guidance through various stakeholders groups and sources. Overall, responses have been positive and the guidance has supported decision makers in Wales. Ongoing evidence capture, including through HTW’s adoption audit processes, will add further understanding to the potential impact of our work.
PP149 A Multidimensional And Multistakeholder Approach: Assessing Ethical, Legal, Organizational, Social or Patient-centered (ELSI+) for Telemedicine In Neurological Diseases
Authors: Toledo-Chávarri Ana , Castaño Alezandra Torres , Rodríguez Janet Delgado , Sacks Analía Abt , Serrano-Aguilar Pedro 
Published: December 2022

IntroductionTelemedicine strategies have been broadly introducing in health services during the COVID-19 pandemic, including in care of neurological diseases.MethodsA rapid realist review was conducted using EUnetHTAs Core Model 3.0 and GRADE evidence to decision frameworks were used as frameworks to describe the ethical, legal, organizational, social and patient aspects (ELSI+) related to the use of teleneurology (TN) A scoping multistakeholder meeting helped defined the scope and research questions of the assessment. Patient representatives, clinicians, scientific society representatives with relevant experience in TN were invited and participated. Industry representatives were also present. Systematic searches for ethical, legal, organizational, social and patients related aspects were conducted. Additional manual searches contributed to contextualize these dimensions in the Spanish context. A narrative synthesis was undertaken.ResultsMain results of the assessment of the ELSI+ aspects of TN were described. TN applications are diverse depending on the condition, objective of care and technology used. The implementation of TN lacks specific legal frameworks which implies legal uncertainty. TN may increase geographical accessibility to neurological care in remote areas and by reducing difficult commuting to specialized care centers. Nevertheless, accessibility is challenged by reduced access to technology, the digital divide, lack of health literacy or technologies not adapted to functional diversity. Therefore, equity is not guaranteed if it is offered as a non-voluntary basis or with no support. TN tends to be accepted by patients and carers if it has enough quality, saves travelling time and costs and does not dehumanize care as it is perceived as more flexible and convenient. Quality of TN needs an interdisciplinary team with skills to coordinate organizational aspects of the implementation which include among others, the planification of the support to patients and carers before, during and after the consultation. Health professionals may also need to learn adapted communicational and technological skills.ConclusionsThe implementation of TN poses many ethical, legal, organizational, social or patient-centered challenges.
PD10 Quality Of Economic Evaluation Of Coronary Stents Based On CHEERS: A Scoping Review
Authors: Ren Yan Feng , Li Fu Ming , Chen Yingyao 
Published: December 2022

IntroductionThe study aims to systematically review all articles on the economic evaluation (EE) of coronary stenting, to critically assess the reporting quality, and to summarize the results.MethodsA systematic search was undertaken through seven databases (PubMed, Web of Science, Embase, CNKI, Wanfang data, Vip data and SinoMed.) from inception until March 2021, to identify economic evaluation articles comparing coronary stenting with other therapies, or among different stenting procedures. After screening articles and extracting data independently, we summarized methods, contents, and outcomes of the included articles and appraised their methodological quality using the CHEERS (Consolidated Health Economic Evaluation Reporting Standards) checklists. Then, the literature scores were standardized as a proportion of the total score, and stepwise multiple regression was constructed to verify the factors that might influence the quality of literature.ResultsOf the 3,622 publications identified, 59 articles were included in this review. There were 33 cost-effectiveness studies and 26 were cost-utility studies. The quality of the reports varied between studies, with a standardized mean score of 0.76 (0.40-0.98). According to the Cheers checklist, “Introduction” had the lowest overall score (0.53), with many articles deficient in the description of the study’s perspective; “Discussion” had the highest overall score (0.86), with nearly three-quarters of the articles reporting the full content; “Title and abstract”, “Methods”, “Results”, and “Other” scored 0.71, 0.78, 0.74 and 0.66, respectively. According to the results of the stepwise multiple regression model, “Published year”, “National type”, and “Type of economic analysis” research were significantly associated with the quality of literature.ConclusionThe quality of current research reports on the economics of coronary stenting is generally satisfactory, but there is potential for improvement and high quality reports can provide evidence to support decision making for policy makers.
PD23 Assessing The Suitability Of Real-World Data For Answering Decision Problems – NICE’s Data Suitability Assessment Tool
Authors: Kincaid Lynne , Ayyar-Gupta Vandana , Rowark Shaun , Kent Seamus , Duffield Stephen , Jonsson Pall 
Published: December 2022

IntroductionThe National Institute for Health and Care Excellence (NICE) intends to increasingly use real-world evidence in developing guidance. To increase trust in such evidence, NICE has developed a framework for developing and assessing real-world evidence studies, including understanding the value of the selected data source for the decision problem.MethodsStarting with published high-quality studies about data quality, we developed a conceptual model of the elements needed to understand the quality of a data source. Results from a literature search were then mapped to the model. We used this to design a structured reporting tool, the data Suitability Assessment Tool (dataSAT), and tested it in several cases studies. Additionally, we engaged with internal and external stakeholders to obtain feedback on the tool and revised it accordingly.ResultsDataSAT covers provenance of the data, assessment of data quality, and the data’s relevance to the research question. For data provenance, information is requested about the data source independent of the study’s interests, including the purpose, setting, dates of operation, funding, data specification, and management and quality assurance plans for the data sources. Data quality is covered by quantitively assessing the completeness and accuracy of the following key study elements to inform critical appraisal of the study: population inclusion and exclusion criteria; intervention; comparator; and outcomes and key covariates. The findings on data sources and data quality are then interpreted in terms of relevance to the decision problem. This includes relevance to the population in the United Kingdom, the treatment pathway and care setting, the availability of key study elements, time-related factors such as length of follow up, and the effects of sample size and missing data on the validity of findings.ConclusionsDataSAT allows summary information on source data, including quality and relevance, to be reported in a structured manner, enabling decision makers to better understand how the data influence the robustness of analyses used in health technology assessment. This helps increase trust in the use of real-world evidence.
PD24 Robust Real-World Evidence Generation In Comparative Effects Studies – NICE’s Methods Guidance
Authors: Duffield Stephen , Kent Seamus , Sharma Manuj , Kincaid Lynne , Ayyar-Gupta Vandana , Rowark Shaun , Jonsson Pall 
Published: December 2022

IntroductionRecent reviews have shown that many real-world evidence (RWE) studies suffer from avoidable methodological flaws. Meanwhile, the National Institute for Health and Care Excellence (NICE) is seeing an increase in RWE submissions in Health Technology Appraisals and is keen to support the use of this evidence. However, limited guidance exists for the development and assessment of RWE, risking both missed opportunities for unbiased evidence generation and inconsistent decision making based on that evidence. As part of its RWE framework, NICE has developed methods guidance to provide clear expectations for the conduct and reporting of non-randomized comparative effects studies using real world data.MethodsA conceptual model and draft framework were developed based on established international best practices in RWE and observational research. This was refined with focused literature searches, for example, on the use of external control arm studies. We then engaged with external stakeholders to incorporate their feedback and develop case studies. A reporting template was developed and tested on multiple use cases.Results & ConclusionsThe guidance stresses the central importance of a target trial approach to study design, e.g., adopting an active comparator, new user design, where possible. Target trial emulation is a useful tool to improve the quality and transparency of RWE studies, helping to overcome selection and confounding biases. Various other study design and analytical approaches are outlined for addressing confounding bias and biases due to missing data, measurement error, or misclassification, which are common challenges in RWE. Alongside traditional approaches to sensitivity analysis, the framework promotes quantitative bias analyses which includes a range of methods to assess and communicate the potential impact of remaining bias to study findings by quantifying the direction, magnitude, and uncertainty of bias. A reporting template, based on common methodological pitfalls, is provided to help evidence developers consider key areas of bias in their work and to inform reviewers of any approaches used to investigate or resolve these.
PD29 Systematic Review With Meta-analysis Of Pharmacokinetic Parameters Of Tyrosine Kinase Inhibitors Used In Chronic Myeloid Leukemia
Authors: Fachi Mariana , de Campos Michel , Junkert Allan , Vilhena Raquel , Böger Beatriz , Cobre Alexandre , Domingos Eric , Leonart Leticia , Tonin Fernanda Stumpf , Pontarolo Roberto 
Published: December 2022

IntroductionTherapeutic drug monitoring (TDM) is a cost-effective tool to increase treatments’ efficacy and safety. Analyses of pharmacokinetics proprieties of tyrosine kinase inhibitors (TKI) used for chronic myeloid leukemia (CML) can contribute towards effective TDM, development of tailored treatments and new dosing regimens. We aimed to synthesize the available evidence on pharmacokinetic parameters of imatinib, nilotinib, bosutinib, ponatinib in healthy individuals vs. CML patients.MethodsSystematic searches were conducted in PubMed, Scopus and Web of Science. We included in vivo studies addressing TKIs’ pharmacokinetics, including maximum observed concentration (Cmax), time of maximum observed concentration (Tmax) and half-life (t1/2). Meta-analyses of event rates (mixed-effect models) were performed for the parameters of interest: area under the concentration-time curve from time zero to the last measurable concentration (AUC0-t) and from time zero to infinity (AUC 0-∞). Results were presented as event rates with 95 percent confidence intervals. Heterogeneity was assessed using chi-square and I2 statistical tests and considered significant when p<0.05 and high when I2>75 percent (Comprehensive Meta-Analysis v.2 Biostat-Englewood, NJ).ResultsOverall, 50 articles were included for analyses (n=26 imatinib, n=11 nilotinib, n=8 bosutinib, n=5 ponatinib). Most studies were performed in the United States (46.0%), designed as open-label trials (70.0%). Several significant interactions between TKI with enzyme inhibitors (ketoconazole, midazolam, aprepitant, metoprolol, grapefruit juice), proton pump inhibitors (esomeprazole, lanzoprazole, omeprazole), antacids, H2 antagonists (famotidine) and enzyme inducers (St. John’s, rifampicin) were found (p<0.001). Given the significant increase in AUC and Cmax in patients with hepatic/liver impairments currently using TKI, strict therapeutic monitoring is paramount to maintain safety. The between study heterogeneity was rated as moderate to high (I2=75-90%) due the limited number of trials for some drugs, different study design, and populations.ConclusionsThe co-administration of TKI with hepatic enzyme inducers or inhibitors, proton pump inhibitors, antacids, H2 antagonists, as well as in patients with hepatic/liver failures requires caution and additional monitoring. Further well-designed trials are needed to strengthen this evidence for some TKIs, namely bosutinib and ponatinib.