bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP89 Stimulating And Assuring Evidence Quality From A Dutch Funders’ Perspective – Introducing The ZonMw Reporting Checklist
Authors: Reijmerink Wendy , Vianen Gerjanne , Bloemers Margreet , Durrani Abida , Stax Annelein 
Published: December 2022

IntroductionAll studies should report methods and findings in full, following credible and justifiable reporting guidelines. According to the guiding principles of the Ensuring Value in Research (EViR) Funders’ Forum (www.evir.org), this applies irrespective of the nature of the findings or whether the study was completed as planned.One way for a public funding agency to address evidence quality and transparency is to adaptively implement EQUATOR reporting guidelines (www.equator-network.org) in its funding procedure to ensure research quality ‘from proposal to publication’. The Netherlands Organisation for Health Research and Development, ZonMw, has created the ZonMw Reporting Checklist (ZRC), which was derived from EQUATOR reporting guidelines in order to systematically plan, monitor, and evaluate projects. The next step is experimenting with implementing the ZRC in ZonMw’s grant management system and procedures. Customization is possible based on the ‘comply or explain’ approach (80/20 rule).MethodsWe selected 15 EQUATOR reporting guidelines that covered basic research and health technology assessment through to implementation projects, supplemented with the reporting guideline for implementation studies (StaRI checklist). We conducted comparative content analyses (including rearrangement) to provide a greatest common denominator consisting of both standard and modular reporting elements. We completed the ZRC by adding other current requirements for responsible research practices with respect to diversity and gender, data management, open access, systematic reviews, recruitment and inclusion, registration, and impact.ResultsThe ZRC results in structured and validated in-house data on the objectives, design, conduct, and results of ZonMw projects. This is an important source for good research governance, impact assessment, and research on research.ConclusionsImplementation of the ZRC by a funding agency optimizes the quality, transparency, relevance, and impact of evidence, which legitimately and effectively improves health care for all.
PP96 Joint Clinical Assessments – Implementation And Lessons For The Next Stage Of EU HTA
Authors: Samaha Dima , Newton Max , Tavares Eliana , Kruger Robert , Szawara Paula , van Engen Anke 
Published: December 2022

IntroductionThe European Network for Health Technology Assessment (EUnetHTA) was a voluntary cross-border initiative on HTA harmonization established by European Commission in 2005. Between 2016-2021, EUnetHTA completed 56 Joint Assessments (28 drugs; 28 other technologies) including 14 reviews of COVID treatments.MethodsWe conducted a review of the 14 EUnetHTA joint assessment reports of drugs in non-COVID 19 indications. We cross-referenced recommendations with national guidance in 30 member countries (including UK) and conducted an analysis of time to national assessment, choice of comparator, direct reference to EUnetHTA assessment, and time to reimbursement decision.ResultsSix products in oncology, 2 in endocrine and metabolic diseases, 2 in infectious and parasitic diseases, and cardiovascular, digestive system, eye disorders and central nervous system (one each) were identified. On average, EUnetHTA published its recommendation 52 days after market authorization for oncology products and 33 days for non- oncology products. EUnetHTA recommendations considered on average 4 comparators (range 1-8) as part of the assessment. All of the 6 oncology products have been assessed by national HTA bodies, however uptake was low with an average of 5 reports referencing the EUnetHTA report. Similarly for the non-oncology products assessed only 3 of 30 HTA bodies cite the EUnetHTA report. Citing HTA bodies were: AETSA (Spain), HAS (France), INFARMED (Portugal), NoMA (Norway), and TLV (Sweden). There was no clear reduction in the time to reimbursement for these products in these markets.ConclusionsAccording to EUnetHTA, there has been an increased use and dissemination of joint assessment reports since 2016. Our analysis shows that the level of implementation across countries is heterogeneous despite publication of the EUnetHTA reports shortly after market authorization. The future the EU HTA will depend on the timeliness, rigor and transparency of joint clinical assessment reports and improved uptake of these reports at a national level.
PP100 Improving The Assessment Of Effectiveness For Digital Applications Using The B Statistic: Using WtsWrng As A Case Study
Authors: Abbas Ismail , Barcelo Carla Fernandez , Valdasquina Blanca , Rabasseda Nicolau , Sampietro-Colom Laura 
Published: December 2022

IntroductionThe performance of diagnostic health technologies is usually assessed by comparing them with standard care using the kappa statistic. These comparisons are made based on comprehensive clinical information (e.g., anamnesis and complementary tests). However, not all digital applications (DAs) execute over complete information, which leads to work under non-uniform distribution of values. Using kappa statistic in this situation has serious methodological limitations. Kappa assumes that the marginal values are uniformly distributed and highly weights the discordant values when calculating concordance, which underestimates the real effectiveness of DAs (i.e., observed concordance). We aimed to present the application of the B statistic to WtsWrng, a symptom triage DA for individuals.MethodsWtsWrng was used by 382 patients at the emergency department of a hospital. Diagnoses provided by WtsWrng, given 19 symptoms, were compared with those logged in the hospital’s electronic clinical records at discharge. Observed concordance was calculated using contingency tables. The concordance using the kappa and B statistics were compared for the 12 most frequent diagnoses at hospital discharge. Sensitivity and specificity were also calculated.ResultsReal observed concordance fluctuated from 0.4 to 0.98 for the 12 most frequent diagnoses, eight of which had a concordance greater than 0.8. The results ranged from -0.005 to 0.37 when using the kappa statistic and from 0.36 to 0.99 when using the B statistic. The sensitivity and specificity of WtsWrng were greater than 0.8 for three and eight of the 12 diagnoses, respectively.ConclusionsThe results show that the B statistic is closer to the real observed concordance when kappa statistic assumptions are not fulfilled by a DA. Therefore, the B statistic is better suited for assessing the effectiveness of this type of technology. Analysis of WtsWrng using the B statistic showed that its diagnoses were close to those provided by clinicians, which were arrived at using complete clinical information. Moreover, the high specificity of the WtsWrng DA suggests that it is a good tool for determining the appropriate use of healthcare resources.
PP108 Multidimensional Analysis Of Peristeen Plus Medical Device
Authors: Cicchetti Americo , Fiore Alessandra , Rumi Filippo , Di Paolo Maria Giovanna , Fortunato Agostino 
Published: December 2022

IntroductionNeurogenic bowel dysfunction occurs in people with central nervous system disease or injury and causes loss of bowel control and severe constipation. These problems involve a lot of anxiety and discomfort and can reduce the quality of life of those who suffer from them, therefore the management of symptoms is very important. Peristeen Plus is a transanal irrigation (TAI) system for bowel dysfunction management, used to empty the rectum and distal sigmoid colon, to prevent uncontrolled bowel movements or to relieve and prevent constipation.MethodsA literature review was conducted. A total of 14 records were included to evaluate the benefits in terms of efficacy and safety associated with the adoption of the medical device. To assess the economic impact, two different budget impact models have been implemented. The first aimed at evaluating an incremental diffusion in a short-term time horizon (3 years) of the home distribution of the device compared to the direct and indirect distribution methods in the Italian context. The second model aimed at assessing the impact of the diffusion of the device in the clinical practice.ResultsOverall, most studies demonstrate improved endpoints related to the severity of fecal incontinence, constipation and intestinal disorders in patients using the device. The economic assessments conducted estimate that the increase in the Italian care setting is associated with a saving of resources in each year under analysis. The diffusion of home distribution of the device would potentially be able to offer a lower absorption of resources compared to other distribution methods. In addition to this, there is an incremental saving correlated to the degree of diffusion of Peristeen Plus.ConclusionsTAI is considered a safe and more effective method than conventional treatments for reducing fecal incontinence, constipation and improving quality of life. The results of our study confirm the benefits of TAI as a second-line treatment in case of failure of conventional medical therapy in the management of the neurogenic gut.
PP110 An External Combined Occipital And Trigeminal Neurostimulation Device For Migraine Treatment
Authors: Rodríguez-Ortiz-de-Salazar Begoña , Hijas-Gómez Ana Isabel , del Mar Polo de Santos María , Arias-Pacheco Amparo , Sánchez-Gómez Luis , Luengo-Matos Seteffilla 
Published: December 2022

IntroductionMigraine is a multifactorial pathology that affects 12 percent of the world’s population. Relivion® MG (Neurolief Inc., USA) is a non-invasive self-administered device for external combined occipital and trigeminal nerve stimulation that has been recently approved for marketing in Europe and the USA. The objective of this study was to conduct an early assessment of the efficacy and safety of Relivion for the treatment of migraine episodes.MethodsRelivion was identified by the early awareness and alert system, SINTESIS- nuevas tecnologías, of the Agencia de Evaluación de Tecnologías Sanitarias at the Instituto de Salud Carlos III. An early assessment of the technology was conducted by reviewing relevant clinical studies published to 29 September 2021. The literature was identified by searching PubMed, Embase, the International Clinical Trials Registry Platform, ClinicalTrials.gov, and the Cochrane Library.ResultsTwo randomized, sham-controlled double-blind trials were found. They assessed side effects and pain relief, response rate, and pain freedom two hours after treatment. One study included 55 patients and the other (the RIME study) included 131 patients. The rate of pain relief two hours after treatment ranged from 60 to 76 percent in the treatment group and from 31 to 37 percent in the control group (p<0.01). The response rate, defined as at least 50 percent pain reduction two hours after treatment, was significantly higher in the treatment group (67 to 70% versus 32 to 42%). The percentage of patients free of pain two hours after treatment ranged from 42 to 46 percent in the treatment group, compared with 11 to 12 percent in the control group (p<0.0001). No notable adverse events were recorded.ConclusionsPreliminary results indicated that Relivion effectively and safely relieves or eliminates acute migraine pain. However, more comparative studies are needed. The use of Relivion could improve the control of symptoms and improve quality of life in patients with migraine.
PP112 Reimbursement Decisions for Medical Services in Austria: An Analysis of Influencing Factors for the Hospital Individual Services Catalogue
Authors: Goetz Gregor , Panteli Dimitra , Busse Reinhard , Wild Claudia 
Published: December 2022

IntroductionThis study aims to (i) describe the (evidence-based) reimbursement process of hospital individual services, (ii) evaluate the accordance between evidence-based recommendations and reimbursement decision of individual services and (iii) elaborate potential aspects that play a role in the decision-making process in Austria.MethodsThe reimbursement process is described based on selected relevant sources such as official documents. Evidence-based recommendations and subsequent reimbursement decisions for the annual maintenance of the hospital individual service catalogue in Austria between 2008 and 2020 were analyzed using a mixed methods approach, encompassing descriptive statistics and a focus group with Austrian decisionmakers.ResultsOne hundred and eighteen evidence-based recommendations were analyzed. There were 93 (78.8%) negative and 25 (21.2%) positive evidence-based recommendations. In total, 107 out of 118 evidence-based recommendations (90.1%) did not lead to a deviating reimbursement decision. We identified six aspects that may have played a role in the decision-making process for the annual maintenance of the hospital individual service catalogue, with clinical evidence being the most notable. Further aspects included quality assurance/organizational aspects (i.e., structural quality assurance), costs (if comparable to already existing medical services, not: cost-effectiveness), procedural aspects (e.g., if certain criteria for adoption have not been met formally through the proposals), “other countries” (i.e., taking into account how other countries decided) and situational aspects (such as the COVID-19 pandemic).ConclusionsThere is good accordance between evidence-based recommendations and reimbursement decisions regarding hospital individual services in Austria. Beyond clinical evidence, organizational aspects seem to be considered often with regard to quality assurance but costs do not appear to play a major role. The Austrian system has mechanisms in place that can restrict widespread adoption of novel hospital individual services with uncertain clinical benefits. Future studies could investigate how well these mechanisms work and how they compare to other health systems in Europe.
PP124 The Importance Of Flexible PPI Approaches: Case Study On Flash Glucose Monitoring
Authors: Evans Alice , Washington Jenni , Jarron David 
Published: December 2022

IntroductionHealth Technology Wales (HTW) review guidance 3 years after publication to establish if reassessment is warranted because, for example, new evidence has become available. Since the publication of guidance on flash glucose monitoring (FGM) in 2018, HTW introduced a patient and public involvement (PPI) process with novel approaches to flexible engagement. This enabled HTW to include three streams of patient evidence into the review of FGM devices.MethodsHTW’s Patient and Public Involvement Standing Group (PPISG) considered appropriate methods of engagement using the HTW Patient and Public Involvement Mechanism Tool. This tool considers the nature of the health technology, the presence of appropriate patient organizations and questions that can be put to patients, as well as other approaches for obtaining patient evidence.ResultsHTW contacted Diabetes Cymru and met with them to discuss contributing to the appraisal of FGM devices. Diabetes Cymru produced a patient submission summarizing the experiences of their patient network, with particular focus on the expansion of the technology to closed-loop insulin systems. Diabetes Cymru later attended HTW’s Appraisal Panel committee and gave a presentation. Additionally, HTW conducted a patient evidence literature review. This review summarized published qualitative studies on a range of perspectives, including carer perspectives, family perspectives, children and adolescences perspectives as well as considerations from specific environments, such as schools, workplaces, homes, care homes and communities. In addition to new clinical and cost effectiveness evidence, this PPI input was used to formulate new guidance recommending more widespread adoption of FGM.ConclusionsThe introduction of flexible approaches to PPI enabled HTW to gain patient evidence from multiple sources. This ensured greater patient representation and a more detailed understanding of the role of FGM devices across different patient communities. This added considerable richness to the patient evidence, which is vital to understand the everyday impacts of FGM and its use amongst patients. Combining flexible PPI with the new clinical and cost effectiveness evidence resulted in a change in the original guidance recommendation.
PP129 Usefulness, Acceptability And Satisfaction Of A Decision Making Tool For Clinical Meso-Management In Type 2 Diabetes Mellitus
Authors: Ramallo-Fariña Yolanda , González-Pacheco Himar , Sacks Analía Abt , García-Hernández Laura , de Segura Ignacio Llorente-Gomez , Wägner Ana , Toledo-Chávarri Ana , Serrano-Aguilar Pedro 
Published: December 2022

IntroductionGesPeDia is a web-based application that provides aggregate clinical information, using outcome and process indicators, and disaggregated patient information. Information is obtained from the electronic medical records. GesPeDia aims to promote people-centered care, improve monitoring of patients’ health outcomes and quality of professional performance. This study aims to evaluate usability, acceptability and satisfaction of GesPeDia.MethodsNineteen evaluators were included (2 management technicians, 9 health center directors and 8 endocrine consultants). They had access to GesPeDia for two months. Perception of their usefulness for decision-making, acceptability and design satisfaction were measured with an online questionnaire. In addition, suggestions for improvements in the app’s functionalities were collected. Finally, a sample of the evaluators were included in a semi-structured interview to deepen the analysis of dimensions. A descriptive analysis of the data was performed.ResultsThe questionnaire was completed by 10 professionals, with mean age of 51.1 years and professional experience 16.5 years. Among the evaluators, 60 percent considered the app quite useful and only 10 percent found it inappropriate for their daily activities. Each of the indicator blocks was rated quite useful. Eight percent considered GesPeDia moderately fast, although for 20 percent navigation within the app was not very intuitive. Appearance was positively valued by 80 percent, despite the fact that 30 percent considered that design does not favor the understanding of contents. Seventy percent considered degree of reliability, relevance and clarity of the contents to be high. Most indicated that information provided by GesPedia is complete for decision-making.ConclusionsGesPeDia is valued positively by evaluators as a decision-making tool.
PD01 Using ELISA Tests For Monitoring Response To Anti-TNFs In Rheumatoid Arthritis: Findings From A NICE Health Technology Assessment
Authors: Yang Huiqin 
Published: December 2022

IntroductionPatients with severe rheumatoid arthritis (RA) disease may be treated with tumor necrosis factor (TNF)-α inhibitors. However, their efficacy is reduced by the presence of anti-drug antibodies. The objective of this HTA was to conduct a systematic review to investigate the effectiveness of using enzyme-linked immunosorbent assay (ELISA) tests (Promonitor, IDKmonitor, LISA-TRACKER, RIDASCREEN, MabTrack, and Sanquin Diagnostic Services) to measure levels of the drug and anti-drug antibodies for monitoring response to TNF-α inhibitors in patients with RA.MethodsA range of bibliographic databases including MEDLINE, EMBASE and CENTRAL were searched from inception to November 2018. Studies were eligible for inclusion if they investigated ELISA tests in RA patients receiving treatment with a TNF-α inhibitor who had achieved the treatment target (remission or low disease activity [LDA]) or experienced a primary or a secondary non-response. The tests must compare with standard care where treatment decisions are based on clinical judgements and monitoring using a composite score such as the disease activity score 28 joints (DAS28). Risk of bias was assessed using the Cochrane (ROBINS-1) tool for non-randomized studies.ResultsTwo studies were included. One non-randomized controlled trial (non-RCT) compared standard care with therapeutic drug monitoring using Promonitor test kits in RA patients in remission/LDA receiving adalimumab, and a historically controlled study investigated Sanquin ELISA kits. The non-RCT study showed that there was a non-significant reduction in the risk of flare in the intervention group compared with the control group. Patients’ health-related quality of life outcomes were higher in the intervention group at all visits compared with the control group. However, this study had serious limitations because analyses were not performed using an intention-to-treat approach. The historically controlled study did not provide valid evidence on whether ELISA-based monitoring is clinically effective.ConclusionsThere is only limited, poor evidence available. There are considerable uncertainties on the effectiveness of using ELISA tests for monitoring response to TNF-α inhibitors in RA. Further controlled trials are required.
PD02 A Literature Review Of Treatment Schemes And Effects For Beta Thalassemia In China
Authors: Yuan Jiaqi , Qiao Jingyi , Weng Junling , Luo Bingxing , Yan Juntao , Ming Jian , Chen Yingyao 
Published: December 2022

IntroductionThe incidence of beta thalassemia varies greatly in different regions of China. Blood transfusion combined with iron chelation and hematopoietic stem cell transplantation (HSCT) is the main treatment for beta thalassemia in China. This study aimed to reveal the specific treatment schemes used for patients with beta thalassemia and to evaluate their effects.MethodsA search strategy was developed to identify articles published between 1 January 2010 and 30 August 2021 in the following literature databases: PubMed, Embase, the China National Knowledge Infrastructure, Wanfang Data, and the Chinese BioMedical Literature Database.ResultsThe most used iron chelation schemes in China for patients with beta thalassemia included deferoxamine (DFO) monotherapy, deferiprone (DFP) monotherapy, deferasirox (DFX) monotherapy, and combinations of DFP and DFO. Most studies reported that combinations of DFP, DFO, and DFX monotherapy performed better than DFO or DFP monotherapy alone in reducing the blood, heart, and liver iron load. However, the adverse effects of iron chelation affected patient compliance with treatment to a certain extent. Stem cells for patients receiving HSCT in China were mainly donated by human leukocyte antigen (HLA)-matched siblings or unrelated individuals. The five-year overall survival rate after HSCT ranged from 83 to 90 percent, while the five-year beta thalassemia-free survival rate ranged from 65 to 87 percent. Graft-versus-host disease and infection were the most common serious complications experienced by transplant recipients.ConclusionsFor patients in China with beta thalassemia, the most effective iron chelation treatment schemes were combinations of DFP, DFO, and DFX monotherapy. HSCT from HLA-matched siblings or unrelated donors resulted in a significant improvement in the cure rate for beta thalassemia. However, patients still need safer and more effective innovative treatments, and further evidence on existing treatments needs to be generated from larger scale studies in the Chinese population.