bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP01 Convergent Validity Between Discrete Choice Experiment And Other Stated Preference Methods: A Multistudy Comparison
Authors: Veldwijk Jorien , Tervonen Tommi , de Bekker-Grob Esther , Hauber Brett , Groothuis-Oudshoorn Catharina G.M. 
Published: December 2022

IntroductionTo assess convergent validity of stated preference methods in studies where they were used to elicit patient preferences for informing medical product decisions.MethodsIn four studies, two stated preference methods were used to elicit preferences of patients with neuromuscular diseases (NMD; n = 140, Discrete Choice Experiment [DCE] and Best-Worst Scaling [BWS] case 2), diabetes (n = 495, DCE and swing weighting [SW]), myocardial infarction (MI; n = 335, DCE and BWS case 1), and rheumatoid arthritis (RA; n = 982, DCE and probabilistic threshold technique [PTT]). In each study, results of the two methods were compared using a normalized preference measure for which confidence intervals (CIs) were estimated using nonparametric bootstrapping of 500 samples. Normalized preference measures comprised of mean relative attribute importance weights (NMD and diabetes studies), attribute uptake probability (MI study), or maximum acceptable risk (RA study).ResultsIn all four studies, attribute ranking showed similar patterns between DCE and other methods for the most important attributes. The same attribute had highest importance in three out of four studies. Significant differences were found in ranges of normalized preference measures of each study between DCE and the other methods: 4.1–43.4 versus 8.9–24.7 for DCE and BWS case 2 in NMD; 3.8–49.7 versus 11.9–16.8 for DCE and SW in diabetes; 2.0–85.5 versus 0.2–69.0 for DCE and BWS case 1 in MI; -3.5–49.2 versus 1.1–18.1 for DCE and PTT in RA.ConclusionsPreferences differed significantly between DCE and other preference methods implying limited convergent validity. The substantially larger ranges in normalized outcome measures in DCE compared to other methods, are likely due to differences in mechanics and bias related to the methods. Since none of the methods is considered the golden standard for measuring stated preferences as true preferences are unknown, further studies are necessary to compare stated preference methods, determine internal validity and data quality, and potentially measure external validity.
OP05 The Role Of Conditional Reimbursement In The Lifecycle Approach
Authors: Schelleman Hedi , van Montfort Simone , Sicking Lenneke , Haasnoot-Volker Daniëlle 
Published: December 2022

IntroductionIn 2012, the Netherlands introduced a conditional reimbursement (CR) program to give patients earlier access to promising treatments (i.e., drugs and medical devices). After completion of the CR-study the results are used to (re)assess whether the treatment fulfills the criteria for reimbursement.MethodsPromising treatments were identified via a bottom-up (physicians/researchers initiated) or top-down process (after the Dutch National Health Care Institute (Zorginstituut Netherland; ZIN) concluded that the intervention did not fulfill the criteria for reimbursement but the initial results seemed promising). A CR-grant was only approved if a committee determined the treatment had an added (health, social, ethical or organizational) benefit compared to Standard of Care (SoC) and the grant proposal was of good quality and fit for purpose. After approval from the Dutch Ministry of Health, all Dutch insurance companies were obliged to reimburse the treatment for patients participating in the CR-study. Researchers could also apply for a research grant (maximum EUR 400,000) from the Netherlands Organization for Health Research and Development (ZonMw), if there was no ‘wealthy’ manufacturer.ResultsCurrently, there are 23 (ongoing and completed) CR-studies. All of them are closely monitored by ZIN, ZonMw, and stakeholders. The results of all completed CR-studies (n = 11) have been used in a (re)assessment. ZIN concluded that five treatments were not effective compared to SoC. Six interventions were effective and cost-effective compared to SoC and are now reimbursed. In most cases (>80%) the physician and patient groups agreed with the conclusion about reimbursement. In some cases there were additional requirements to maintain the clinical effectiveness and cost-effectiveness in clinical practice (such as training of new physicians).ConclusionsData from CR-studies are important for reassessments. Factors with a positive influence are: a maximum duration for a CR-study, close monitoring, possibility to adapt the study design (only with approval from ZIN and ZonMw), and active involvement of stakeholders (physician and patient groups). A negative influence was: the legal requirements to ensure only reimbursement for patients participating in a CR-study.
OP06 Percutaneous Transforaminal Endoscopic Discectomy: From Insufficient Evidence To Reimbursement
Authors: Schelleman Hedi , de Groot Ingrid , Haasnoot-Volker Daniëlle , Jellema Petra 
Published: December 2022

IntroductionThe standard surgical technique for lumbosacral radicular syndrome in the Netherlands is open microdiscectomy (OM). An alternative technique, preferred by some Dutch physicians, is percutaneous transforaminal endoscopic discectomy (PTED). However, in 2006 the Dutch National Health Care Institute (Zorginstituut Netherland [ZIN]) concluded that the available evidence was insufficient, and a high quality randomized controlled trial (RCT) was needed to assess the cost-effectiveness of PTED compared to OM. The relevant physician group agreed with this conclusion, but they were unable to perform this RCT due to lack of funding and high treatment costs.MethodsIn 2012, the Netherlands introduced a conditional reimbursement (CR) program to give patients earlier access to promising treatments. Researchers, in collaboration with physicians and patients, submitted a grant proposal and in 2016 the Dutch Ministry of Health approved the CR of PTED. Due to this decision, insurance companies were obliged to reimburse PTED for patients participating in the PTED-study (NCT02602093). The Netherlands Organization for Health Research and Development (ZonMw) also provided a research grant to fund the PTED-study. In total, 682 adult patients with greater than 10 weeks of radiating pain, or greater than 6 weeks of excessive radiating pain and an indication for surgery were included. After 4 years and 5 months the PTED-study was completed.ResultsOutcomes of published studies and the unpublished PTED-study were used in the HTA reassessment. Results showed that PTED was noninferior to OM with regards to leg pain (Visual Analogue Scale: mean difference (MD) -0.73; 95% confidence interval [CI] -5.04, 3.59), functional status (Oswestry Disability Index: MD -2.07; 95% CI -3.61, -0.53), and rate of complications (relative risk 0.45; 95% CI 0.18, 1.12) after 6 months (GRADE level ‘moderate’). Furthermore PTED was, after the surgeons’ learning-curve, cost-effective.ConclusionsThis CR project was successful and PTED is now reimbursed as part of the Dutch healthcare package. However, in order to maintain high quality care in clinical practice, safeguards should be developed (including the appropriate training of surgeons). This example shows that CR programs are essential for promising treatments without ‘wealthy’ manufacturers. Additionally, all stakeholders are needed to make a CR-study successful.
OP12 Post-Launch Evidence Generation Among Health Technology Assessment Bodies In Europe
Authors: Lema Leonor Varela , Puñal-Riobóo Janet , Guilhaume Chantal , Galbraith Maggie , Bélorgey Chantal , Faraldo Vallés Maria José , Amélie Amélie Meillassoux 
Published: December 2022

IntroductionThe need for timely access to innovative technologies has placed a special focus on the development of policies and practices that can guarantee the availability whilst ensuring the safety of these technologies after launch or licensure. The aim of this paper is to present and discuss Post-Launch Evidence Generation (PLEG) practices among health technology assessment (HTA) bodies at the European level to explore cross-border collaboration opportunities.MethodsIn December 2019, a survey composed of nine closed-ended questions with multiple choice answers about the PLEG practices in each country was sent to 25 partners of the European Network of Health Technology Assessment (EUnetHTA) Joint Action 3. In addition to the survey, the national practices were discussed during a face-to-face meeting with all partners of the dedicated work package. A quantitative analysis and a qualitative synthesis of the results was carried out.ResultsTwelve HTA bodies completed the survey. Of these, 11 reported procedures in place for official PLEG requests. In nine of the agencies, the requests are made at the time of the assessment/appraisal. Data collection and analysis mainly lies with companies for pharmaceuticals (60%) while it is more the responsibility of the HTA body for medical devices (75%). Only one agency reported owning the data and being able to exchange the data without asking permission. During the face-to face discussions, it was acknowledged that PLEG practices differ between countries depending on the topic concerned, but most rely on the usage of registries (mainly disease registries) for data collection. Most agencies estimated that a European collaboration could take place.ConclusionsPLEG practices are in the remit of many European HTA bodies. Data sharing should be anticipated as only some own the data and can exchange them without asking permission. European collaboration on PLEG could commence once the evidence gaps have been defined or during the production of the HTA reports in the case of joint assessments.
OP15 Improving The Osteoporosis Care Trajectory By Collaboration Between Clinicians, Patients And Health Insurers Within the Appropriate Care Program
Authors: Groeneveld Iris , Witteveen Janneke , Boonzajer-Flaes Saskia , Mullenders Pé , Leenen Celine 
Published: December 2022

IntroductionThe cyclic appropriate care program of the National Health Care Institute aims at enhancing quality of care in the Netherlands. A full cycle consists of four phases: (i) screening each ICD-10 chapter and selecting a care trajectory, (ii) in-depth analysis of the care trajectory and formulating actions for improvement, (iii) implementation, and (iv) evaluation. Collaboration with and between relevant parties from the field is key to its success. We describe the 2nd and 3th phase of this cycle, aimed at osteoporosis.MethodsFirst, nine clinicians, one nurse practitioner, two physiotherapists and one pharmacist representing scientific organizations, two patient representatives and one health insurer, defined research questions. Second, by in-depth analysis on declaration data of health care activities, and comparing the results to the guidelines, research questions were addressed. Last, results were discussed among all parties and actions for making wiser choices and improving the appropriateness of care were formulated. For each action, the parties defined who would take the lead and who would be involved.ResultsThe analyses showed that only 26 percent of Dutch fracture patients 50 years of age and above underwent dexa-scanning, as opposed to more than 80 percent in the guideline. All parties underlined this percentage should be increased. Possible actions were defined: to describe the care trajectory in the update of the multidisciplinary guideline; to better inform fracture patients about the importance of dexa-scanning; and to make adjustments to the hospital electronic system in order to facilitate the ordering of dexa-scans. For these actions, medical specialists were in the lead. Other data-analyses showed that half of osteoporosis patients stopped using medication within two years. For the jointly defined actions aimed at raising medication adherence, such as better patient education, the general practitioner and pharmacist were in the lead. Currently, the National Health Care Institute facilitates implementation and monitors improvement.ConclusionsClose collaboration with relevant parties led to well-considered actions on improvement of osteoporosis care.
OP20 Is The Quality Of Evidence In Health Technology Assessment Deteriorating Over Time?: A Case Study On Cancer Drugs In Australia
Authors: Gao Yuan , Laka Mah , Merlin Tracy 
Published: December 2022

IntroductionRecently, there have been concerns regarding a trend toward poorer quality evidence being accepted by regulatory institutions and the consequent impact on health technology assessment (HTA) decision-making. This study aimed to determine whether there has been a change in the quality of evidence provided on cancer drugs proposed for listing on the Pharmaceutical Benefits Scheme, using data solely extracted from public summary documents (PSD) published by the Australian government.MethodsPSDs published from July 2005–2020 were reviewed. Metrics associated with quality of evidence were extracted, including the directness of comparison, study design, sample size, and risk of bias (RoB). Additional data were extracted to provide greater context to any observed trends in quality of evidence. Analyses were performed across different time periods. Associations between the quality of evidence and time periods were explored using logistic regression analysis.ResultsIn total, 214 PSDs were included in the analysis. Only 13 percent of submissions provided a single arm study or observational study as the key evidence; however, 37 percent of submissions did not contain a direct (‘head-to-head’) comparison relevant to Pharmaceutical Benefits Advisory Committee (PBAC) decision-making. Among all submissions containing direct evidence, about half had findings of a moderate/high/unclear RoB. Among all submissions containing indirect comparisons, over half had transitivity issues. In submissions containing direct comparisons, there was an increase in the RoB over time even after adjusting for trial data maturity and the rareness of the drug indication (odds ratio [OR] 1.30; 95% confidence interval [CI] 0.99, 1.70). There were no clear time trends observed in sample size, directness, study design, or transitivity issues during any of the observed time periods.ConclusionsIn the last 7 years, a high proportion of cancer drug submissions presented findings with a high RoB and transitivity issues. As the evidence dossiers provided to the PBAC are often congruent with submissions made elsewhere, this poor evidence quality is of concern and can only lead to higher levels of decision-maker uncertainty.
OP37 Lifecycle Evaluation Models And Frameworks Used To Assess Medical Devices: A Qualitative Evidence Synthesis
Authors: Harkin Kathleen , Sorensen Jan , Thomas Steve 
Published: December 2022

IntroductionDue to the iterative nature of medical device innovation and development, a single once-off assessment does not provide all the answers that decision-makers need over the device’s lifetime. Consequently, a lifecycle approach is frequently recommended. However, there is no lifecycle model recognized internationally for conducting such evaluations, nor is there explicit agreement regarding what is meant by, or evaluated over, the lifecycle. The purpose of this review was to identify and explore the range of models/frameworks used for evaluating medical devices across their lifetime – to determine what people mean by ‘the lifecycle’, what is evaluated, how, and why.MethodsA qualitative evidence synthesis of lifecycle models described in the literature from a wide variety of disciplines was performed. Literature searching and selection of models iterated with analysis. Similarities, differences, and patterns were identified, from which themes became apparent, and explanatory theories were developed.ResultsFifty-three models are included in the synthesis. The dimensions of difference include, amongst others, the lifecycle scope, level of application, evaluation timepoints and methods, factors included in the models, and the focus of interest. These are each influenced by the purpose of the lifecycle evaluation, which depends on the perspective and the decision or activity the evaluation is intended to inform. Few models provide a lifecycle approach to evaluating safety or efficacy. Theories explaining the differences are postulated.ConclusionsLifecycle evaluation means different things to different actors, with varied reasons for evaluation and different variables included in the models. Thus, discussions between different actors on lifecycle evaluation may be inadvertently at cross-purposes. Without first defining what is meant by the lifecycle (including the stages or phases of activity it covers) and the variables included in an evaluation, care must be exercised when discussing a lifecycle evaluation approach – to ensure that the meaning (and intended objective) is not lost in translation. Indeed, promoting lifecycle evaluation may result in necessary evidence not being generated early enough, being deferred instead until later.
OP42 Increasing Access To Real-World Data To Move From Health Technology Assessment To Health Technology Management
Authors: Rowark Shaun , Jonsson Pall , Kent Seamus 
Published: December 2022

IntroductionWhen assessing existing or emerging technologies using a one-off health technology assessment (HTA) we do not take into consideration the effects on people who will receive the technology once approved. Developments in real-world data (RWD) can help to address this by moving to ongoing health technology management (HTM).MethodsTo move to HTM, we first need to develop HTM data requirements. We undertook user interviews with National Institute for Health and Care Excellence (NICE) HTA developers to develop a list of requirements. We surveyed the types of data that NICE currently has access to and performed a gap analysis to understand where further data is needed. We then worked with external systems partners to identify and review available data sources that could support HTM.ResultsFrom our user interviews we established eight HTM data requirements. Data needed to be linked, cover full care pathways, contain data from new collections, be shareable, have direct access, be of high quality, have comprehensive coverage, and be responsive to technological developments (such as artificial intelligence). The review of data sources revealed a fragmented landscape of health data in the United Kingdom (UK). We identified National Health Service Digital’s (NHSD) Trusted Research Environment as the main data source that could address HTM requirements. This addresses challenges with fragmented data by providing approved researchers with timely and secure access to a range of linked health and care data. We also identified that a large national data collection would not capture all technologies, such as orphan technologies for rare conditions. We therefore established a process for accessing data from smaller data collections such as disease specific registries. To address how we can use this data, we developed the NICE Real-World Evidence (RWE) Framework that provides clear guidance on the expectations for the planning, conduct, reporting, and appraisal of RWE studies.ConclusionsWe have established requirements for the type of data that will help to deliver HTM as well as developed a process for accessing several suitable data sources that meet these requirements.
OP46 Assessing The Quality Of Pharmacoeconomic Evaluations About Type 2 Diabetes Mellitus Drugs In National Reimbursement Drug List
Authors: Bao Shi-Yi , Liu Liu , Li Fuming , Yang Yi , Wei Yan , Shao Hui , Ming Jian , Yan Juntao , Chen Yingyao 
Published: December 2022

IntroductionWith the disease spectrum changing in China, type 2 diabetes mellitus (T2DM) has become the main chronic disease which affects people’s health severely, bring patients serious economic burden of disease. For T2DM patients, reliable quality of evidence in decision-making are significant, improving the efficiency of the adjustment of the National Reimbursement Drug List (NRDL). Based on the Consolidated Health Economic Evaluation Reporting Standards (CHEERS), we aimed to evaluate the quality of all published pharmacoeconomic evaluations on T2DM drugs in 2020 NRDL.MethodsBecause the 2020 NRDL came into effect on 1 March 2021, we searched all published pharmacoeconomic evaluations about T2DM drugs in 2020 NRDL before March 2021 in China National Knowledge Infrastructure (CNKI), Wan fang Data, China Science and Technology Journal Database (VIP), PubMed, and Web of Science. According to the criterion of inclusion and exclusion, all documents were screened and then relevant basic information of targeted documents was extracted. The quality was evaluated by calculating the final scores based on CHEERS. Two reviewers assessed each publication’s quality using the CHEERS instrument and summarized study quality.ResultsA total of 910 papers were searched, and 24 papers were included. These involved six T2DM drugs, specifically IDegAsp, exenatide, liraglutide, lixisenatide, dapagliflozin and empagliflozin. The average score was 18.31, the standard deviation was 3.67, and the average scoring rate was 77.41 percent. Among all items, “characterizing heterogeneity” scored 0.04, least satisfied with requirements. “Setting and location”, “choice of health outcomes” and “assumptions” scored one, most satisfied with requirements. Among the average scores of all parts, “results” scored lowest at 0.55, and “methods” scored highest at 0.85. The Wilcoxon sum-rank tests showed that score rate which represented reporting quality of economic evaluation (EE) was significantly related to “journal type”, “EEs type”, “model choice” and “study perspective”.ConclusionsThe methodological quality of pharmacoeconomic evaluations about T2DM drugs in 2020 NRDL needs to be improved. Improving the quality of literature is the basic guarantee of scientific decision-making in national medical insurance negotiation.