bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP340 Kidney Patients’ Preferences For A Wearable Digital Health Technology To Support Self-Management Of Chronic Kidney Disease - A Discrete Choice Experiment
Authors: Gc Vijay S. , Iglesias Cynthia , Erdem Seda , Hassan Lamiece , Manca Andrea 
Published: December 2021

IntroductionWearable Digital Health Technologies (WDHTs) can support and enhance self-management by giving individuals with chronic conditions more control over their health, safety and wellbeing. Involving patients early on in the design of these technologies facilitates the development of person-centered products. It may increase the potential uptake of (and adherence to) any intervention they are designed to deliver. This research aims to elicit chronic kidney disease (CKD) patients’ preferences for WDHTs that may help patients manage their conditions.MethodsWe used discrete choice experiments (DCE) to elicit preferences for WDHTs characterized by their generalizable characteristics. The study design was informed by a multi-stage mixed-method approach (MSMMA). This included a review of the published literature, focus group interviews and one-to-one interactions with CKD patients to identify relevant characteristics (that is, attributes and levels) associated with wearable DHTs. We collected the data from 113 patients (age ≥18 years) with stage 3 or above CKD. The analysis started with a conventional multinomial logit model and was extended by investigating heterogeneity in preferences via latent class models.ResultsOur MSMMA yielded ten potential attributes for consideration in a choice task. The final list included five attributes, cross-checked and validated by the research team, and patient representatives. The most preferred attributes of WDHTs were device appearance, format and type of information provided, and mode of engagement with patients. Respondents preferred a discreet device, which offered options that individuals could choose from and provided medical information.ConclusionsWe show how to use MSMMA to elicit user preferences in (and to inform the) early stages of the development of WDHTs. Individuals with CKD preferred specific characteristics that would make them more likely to engage with the self-management support WDHT. Our results provide valuable insights that can be used to inform the development of different WDHTs for different segments of the CKD patients population, moving away from a one-size-fits-all provision and resulting in population health gains.
OP345 Evaluation Of An Artificial Intelligence-assisted Service For Cardiac Monitoring As Part Of A National Institute For Health And Care Excellence (NICE) Digital Health Technology Pilot
Authors: Erskine Jamie , Goddard Kate , Chalkidou Anastasia 
Published: December 2021

IntroductionZio XT Service was one of five Digital Health Technologies (DHTs) to be assessed by the National Institute for Health and Care Excellence (NICE) as part of their evaluation pilot. The King's Technology Evaluation Centre (KiTEC) act as an External Assessment Centre for NICE and worked on this pilot evaluation. The service comprises a I-Lead ECG patch, an inbuilt software that makes use of artificial intelligence (AI) algorithms to record, store and analyze ECG traces, and a team of cardiac physiologists.MethodsAlthough the methods were based on NICE's existing Medical Technologies Guidance Process, they were modified to suit the assessment of DHTs. The process was split into two sections, with the option to discontinue the assessment if it was considered that insufficient evidence was available for the technology. Clinical experts and patients were consulted through the process and clinical, economic and technical evidence was considered. Costs for three care pathways were modelled.ResultsA total of thirty relevant clinical studies were identified, with a further study being reviewed as part of a separate technical assessment, focusing on the AI component of the technology. Four of the studies were considered to be pivotal to the decision problem, one of which was a Randomized Controlled Trial. The technology was found to have a greater diagnostic yield than a standard ambulatory monitor, however diagnostic accuracy measures were absent in the literature. Three economic models were developed to represent three care pathways: patients with syncope, patients who have had a stroke or transient ischaemic attack and a third model assessing downstream costs associated with stroke treatment.ConclusionsDigital Health Technologies and Artificial Intelligence Technologies pose novel and unique challenges to health technology assessment (HTA) bodies. Zio XT Service is a diagnostic tool, with both human and AI input, making it a particularly complex technology to assess. This work serves as a case-study in the evaluation of DHTs and AI and the lessons learned may contribute to the development of guidelines for such technologies.
OP437 Use Of Real-World Evidence In Survival Analysis Adjusting For Treatment Crossover In Cutaneous T-Cell Lymphoma
Authors: Hawkins Neil , Evans Rachel , Muszbek Noemi , Jones Trefor , McNamara Linda 
Published: December 2021

Real-World Evidence is useful for validating crossover adjustment approaches, particularly when the adjustment is required because a trial does not accurately reflect a health technology assessment (HTA)-relevant population. We use the MAVORIC trial advanced stage mycosis fungoides and Sézary syndrome cutaneous T-cell lymphoma population and data from the Hospital Episodes Statistics to explore and validate crossover adjustment methods.IntroductionThe MAVORIC trial compared mogamulizumab to vorinostat in patients with mycosis fungoides (MF) or Sézary syndrome (SS), subtypes of cutaneous T-cell lymphoma. However, the treatment comparison within MAVORIC may not represent an HTA relevant population from a UK perspective: (i) 72.6 percent of patients randomized to vorinostat switched to mogamulizumab and (ii) vorinostat is not used in current clinical practice in the UK. This study explores methods to adjust treatment effect estimates using different crossover adjustment methods and Real-World Evidence.This medicine is subject to additional monitoring. This will allow quick identification of new safety information. See www.mhra.gov.uk/yellowcard for how to report side effects.MethodsAn advanced stage (stage ≥IIB MF and all SS) population was included. Three methods were considered for treatment crossover adjustment. A synthetic control arm was created using the Hospital Episodes Statistics (HES) dataset. Predicted survival for the MAVORIC control arm, post-crossover adjustment, was compared to the HES to inform the selection of the appropriate methods for adjustment. A direct comparison between mogamulizumab (reweighted to represent the distribution of MF/SS patients in the HES) and the synthetic control was also conducted.ResultsFollowing crossover adjustment of the vorinostat arm, using the inverse probability of censoring weighting method, the overall survival (OS) hazard ratio (HR) estimate for mogamulizumab vs. vorinostat was 0.45 (95% confidence interval (CI): 0.19, 1.07). This adjustment method was considered the most appropriate based on an assessment of assumptions and a comparison of OS between the adjusted vorinostat data and the HES data. The OS HR estimate for reweighted mogamulizumab vs. synthetic control from HES was 0.33 (CI: 0.21, 0.50).ConclusionsReal World Evidence from the HES database can be used to validate crossover adjustment methods and to better reflect current clinical practice in the UK. Results using both methods support each other.
PP89 The Investigation And Development Of A National Formulary Monitoring System Across Wales
Authors: Boldero Richard , Curran Thomas , Haines Kath , Morton Miranda , Routledge Phil A. 
Published: December 2021

IntroductionThe New Treatment Fund (NTF), launched in January 2017, aims to support the faster introduction of new medicines recommended by the National Institute for Health and Care Excellence (NICE) and the All Wales Medicines Strategy Group (AWMSG). The NTF requires seven health boards and one trust to make recommended medicines available within 60 days of any positive recommendation decision. The project goal was to develop a system for demonstrating how monitoring the NTF improves medicines access for the people of Wales.MethodsThe process was derived via a series of task and finish group meetings with relevant stakeholders. The monitoring criteria were agreed through a collaborative expert approach using a nominal group technique. This determined a minimal dataset of formulary status, which included time to formulary addition. Pre-NTF medicines data (n = 59) were available for a six-month period.ResultsBy the three-year milestone of the NTF, the average time taken for newly recommended medicines (n = 219) to become available to patients across Wales had decreased by eighty-five percent from 90 to 13 days (p < 0.01).ConclusionsAn innovative and robust system has been created for accurately monitoring the formulary addition of medicines within the NTF, supporting the rapid and comprehensive uptake of medicines deemed clinically and cost effective by NICE and the AWMSG.
PP94 Pandemic Preparedness: EUnetHTA COVID-19 Rapid Response With “Rolling Collaborative Reviews (RCR)”
Authors: Wild Claudia , Mayer-Ferbas Julia , Willemsen Anne 
Published: December 2021

IntroductionPotential therapies and interventions for COVID-19 are emerging and developing rapidly. In a response to this public health emergency, the European Network for Health Technology Assessment (EUnetHTA) aims to support health policy in preparation for evidence-based purchasing. To monitor the emerging evidence, a new EUnetHTA product was created: Rolling Collaborative Reviews (RCRs).MethodsRCRs are living documents that are descriptive in nature, updated monthly, and centrally coordinated. They are based on the following three sources of information: (i) published randomized controlled trials (RCTs) presented as a summary of efficacy and safety data (synthesized for a network meta-analysis conducted by the Department of Epidemiology Lazio Regional Health Service, Italy); (ii) published prospective observational studies for safety results, provided by the Map of COVID-19 Evidence conducted by the Norwegian Institute of Public Health, Norway; and (iii) RCTs registered in clinical trial registries (ClinicalTrials.gov, EudraCT Register, and the ISRCTN registry). Additionally, detailed stopping and starting rules were defined.ResultsAs of November 2020, 14 RCRs were ongoing. From the initial list of RCRs, one was suspended due to lacking effectiveness and two moved on to rapid collaborative reviews due to European Medicines Agency approvals. Four RCRs are updated on a bimonthly basis due to a lack of high-quality evidence, and five new RCRs will be started because of promising clinical studies.ConclusionsRCRs can be a means of providing timely and continuous policy support, but they require a high level of coordinated effort.
PP106 Twenty Years Of Orphan Medicines Regulation: Have Treatments Reached Patients In Need Across Europe And Canada?
Authors: Henderson Nadine , O'Neill Phill , Garau Martina 
Published: December 2021

IntroductionThe European Union regulation for orphan medicinal products (OMPs) was introduced to improve the quality of treatments for patients with rare conditions. To mark 20 years of European Union OMP regulation, this study compared access to OMPs and the length of their reimbursement process in a set of European countries and Canadian provinces. Access refers to their full or partial reimbursement by the public health service.MethodsData were collated on European Medicines Agency orphan designation and marketing authorizations, health technology assessment (HTA) decisions and reimbursement decisions, and the respective dates of these events for all the OMPs centrally authorized in 14 European countries (Belgium, England, France, Germany, Hungary, Italy, the Netherlands, Norway, Poland, Scotland, Slovakia, Spain, Sweden, and Switzerland) and four Canadian provinces (Alberta, British Columbia, Ontario, and Quebec).ResultsSince the implementation of the OMPs Regulation in 2000, 215 OMPs obtained marketing authorization. We found that Germany had the highest level of coverage, with 91 percent of OMPs being reimbursed. The three countries with the lowest reimbursement rates were Poland, Hungary, and Norway (below 30%). We observed that Germany had the quickest time to reimbursement following marketing authorization, followed by Switzerland and Scotland. We observed that Poland, Hungary, and Slovakia consistently had the longest time to reimbursement.ConclusionsWe observed substantial variation in the levels and speed of national reimbursement of OMPs, particularly when comparing countries in Eastern and Western Europe, which suggests that an equity gap between the regions may be present. The data also indicated a trend toward faster times to reimbursement over the past 10 years.
PP145 VALIDATE Methodology For A Medication-Related Clinical Decision Support System: Innovating Or Going Back To Basics?
Authors: Fernandez-Barceló Carla , Calvo-Cidoncha Elena , Sampietro-Colom Laura 
Published: December 2021

IntroductionIn the past decade, health technology assessment (HTA) has narrowed its scope to the analysis of mainly clinical and economic benefits. However, twenty-first century technology challenges require the need for more holistic assessments to obtain accurate recommendations for decision-making, as it was in HTA's foundations. VALues In Doing Assessments of health TEchnologies (VALIDATE) methodology approaches complex technologies holistically to provide a deeper understanding of the problem through analysis of the heterogeneity of stakeholders’ views, allowing for more comprehensive HTAs. This study aimed to assess a pharmaceutical clinical decision support system (CDSS) using VALIDATE.MethodsA systematic review of the empirical evidence on CDSS was conducted according to PRISMA guidelines. PubMed, the Cochrane Library, and Web of Science databases were searched for literature published between 2000 and 2020. Additionally, a review of grey literature and semi-structured interviews with different hospital stakeholders (pharmacists, physicians, computer engineers, etc.) were conducted. Content analysis was used for data integration.ResultsPreliminary literature results indicated consensus regarding the effectiveness of CDSS. Nevertheless, when including multistakeholder views, CDSS appeared to not be fully accepted in clinical practice. The main reasons for this appeared to be alert fatigue and disruption of workflow. Preliminary results based on information from the literature were contrasted with stakeholder interview responses.ConclusionsIncorporation of facts and stakeholder values into the problem definition and scoping for a health technology is essential to properly conduct HTAs. The lack of an inclusive multistakeholder scoping can lead to inaccurate information, and in this particular case to suboptimal CDSS implementation concerning decision-making for the technology being evaluated.
PP148 Liquid Biopsy For The Detection Of Ovarian Or Endometrial Cancer In Samples Taken From The Pap Smear: PapSEEK
Authors: García Vanesa Ramos , Perestelo-Pérez Lilisbeth , Rivero-Santana Amado , Duare-Díaz Andrea , Álvarez-Pérez Yolanda , Torres-Castaño Alezandra , Toledo-Chávarri Ana , Serrano-Aguilar Pedro 
Published: December 2021

IntroductionThe PapSEEK test is an emerging minimally invasive technique in which samples are collected from the endocervical or intrauterine cavity with the Papanicolaou (Pap) brush or the Tao brush to detect somatic mutations or aneuploidies indicating the presence of endometrial or ovarian cancer.MethodsWe systematically searched for articles published up to October 2020 in the following electronic databases: Medline, Embase, the Cochrane Library, and the Centre for Reviews and Dissemination. We included experimental studies, observational primary studies, and cost-effectiveness studies evaluating the safety, effectiveness, and cost effectiveness of the PapSEEK test for the early detection of ovarian or endometrial cancer. Relevant outcomes included sensitivity, specificity, the coefficient of variation, re-test rates, the incremental cost-effectiveness ratio, the incremental cost-utility ratio, and the cost of each alternative.ResultsA single relevant retrospective study was identified. In this study, samples from women with endometrial cancer (n = 656) and ovarian cancer (n = 254) were collected with the Pap brush and Tao brush and compared with samples from healthy women (n = 1,002). The diagnostic validity for somatic mutation or aneuploidies obtained with a Pap brush had a sensitivity of 81% for endometrial cancer and 33% for ovarian cancer, and a specificity of 99% for both conditions. When samples were collected from the intrauterine cavity with a Tao brush, the sensitivity increased to 93% for endometrial cancer and to 45% for ovarian cancer. The sensitivity of the PapSEEK test increased only for ovarian cancer when plasma samples to detect circulating tumor DNA were collected in addition to Pap smear samples. This strategy provided a diagnostic validity of 43%, which was higher in late-stage ovarian cancer (56% versus 35%), and a specificity of 100%.ConclusionsProspectively designed studies are required to assess the safety and effectiveness of the PapSEEK test in screening settings, as well as studies comparing the technology with conventional screening methods. No cost-effectiveness studies have been conducted for the PapSEEK test.
PP162 Use Of The RenalGuard® System To Prevent Contrast-Induced Nephropathy
Authors: Bektassova Perizat , Benberin Valeriy , Avdeyev Andrey , Shanazarov Nasrulla , Akhmedullin Ruslan , Okesh Makhabbat , Aimanova Tansholpan , Bariyeva Gulzada 
Published: December 2021

IntroductionContrast-induced nephropathy (CIN) is a common cause of hospital-acquired acute kidney injury (AKI) following the administration of contrast media for coronary interventions or procedures such as diagnostic coronary angiography. The optimal way of preventing CIN remains uncertain. However, preliminary intravenous hydration, minimizing the volume of contrast media, and avoiding the use of nephrotoxic drugs are recommended in current management guidelines. The aim of this analysis was to compare the RenalGuard® system with standard care.MethodsA comprehensive literature search was conducted in PubMed and Google Scholar to identify evidence on the clinical and economic effectiveness of forced diuresis with matched hydration using the RenalGuard system for preventing CIN. Multiple criteria decision analysis (MCDA) was used to assess the performance of the method in hospital settings, compared with alternative options.ResultsSeveral systematic reviews with meta-analyses demonstrated that forced diuresis with matched hydration using the RenalGuard system was associated with a significantly lower relative risk of CIN among high-risk patients with chronic kidney disease. However, the evidence supporting the advantage of the proposed method over current forced diuresis techniques with manual calculation of the volumes for matched hydration in the hospital setting was limited.ConclusionsAlthough the effectiveness of the RenalGuard system has been demonstrated in meta-analyses, its clinical advantage over forced diuresis with manual hydration calculation is uncertain. It is also worth noting the lack of evidence to date on this technology, the fact that it is still at the research stage in some countries, and that it is not included in CIN management guidelines.
PP216 Indirect Treatment Comparison Assessment: An Improvement Intervention In The Scottish Medicines Consortium
Authors: Scott John , McMurray Moira , O'Connell Rickie , McGuire Pauline , Downes Noreen 
Published: December 2021

IntroductionThe Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines on behalf of the National Health Service Scotland based on pharmaceutical company submissions. As the appraisals are conducted close to the point of marketing authorization, there is often a lack of direct head-to-head data. In 2019, assessment of relevant comparative efficacy was informed via indirect treatment comparisons (ITC) in 55 percent (36/66) of submissions. While the ITCs are essential to the decision-making process, they are frequently incomplete.MethodsA focus group was conducted with the clinical assessment team (n = 11) to explore problems in the submission process and to identify areas for improvement. It was agreed that providing improved guidance to companies prior to submission may prevent future inconsistencies. A working group (n = 5) was tasked with identifying and implementing potential solutions. The group reviewed the focus group findings, relevant literature, and guidance from other organizations. Draft guidance was developed that was reviewed by two pharmaceutical industry representatives (SMC subcommittee members).ResultsFindings from the focus group highlighted issues broadly related to the incomplete presentation and reporting of ITCs. The improved guidance document outlined specific requirements in a checklist format for reporting and presenting the results of different ITC data. This guidance was published in February 2020. To evaluate the impact of the updated guidance and to identify any further changes required, a follow-up focus group and survey of industry representatives is planned for March 2021.ConclusionsThe aim of the ITC guidance is to provide pharmaceutical companies with direction to improve the quality and transparency of reporting, which will in turn improve the quality of HTAs and thus strengthen the recommendations provided by the SMC. The follow-up focus groups and survey will assess the impact of the guidance. It is acknowledged that the results of this process may be limited by the small sample size and short duration of the assessment.